MRx Pipeline - October 2021 by Prime/Magellan Rx

MRx PIPELINE A VIEW INTO UPCOMING SPECIALTY & TRADITIONAL DRUGS OCTOBER 2021

EDITORIAL STAFF Maryam Tabatabai, PharmD Editor-in-Chief Vice President, Clinical Information Carole Kerzic, RPh Executive Editor Drug Information Pharmacist Consultant Panel Michelle Booth, PharmD Director, Medical Pharmacy Strategy Rebecca Borgert, Pharm D, BCOP Senior Director, Clinical Strategy and Programs Lara Frick, PharmD, BCPS, BCPP Drug Information Pharmacist Robert Greer, RPh, BCOP Senior Director, Clinical Strategy and Programs

Table of CONTENTS

Katie Lockhart Manager, Forecasting and Pharmacoeconomics Brian MacDonald, PharmD Director, Specialty Clinical Programs

EDITOR-IN-CHIEF'S MESSAGE

PIPELINE DEEP DIVE

KEEP ON YOUR RADAR

PIPELINE DRUG LIST

GLOSSARY

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Nothing herein is or shall be construed as a promise or representation regarding past or future events and Magellan Rx Management expressly disclaims any and all liability relating to the use of or reliance on the information contained in this presentation. The information contained in this publication is intended for educational purposes only and should not be considered clinical, financial, or legal advice. By receipt of this publication, each recipient agrees that the information contained herein will be kept confidential and that the information will not be photocopied, reproduced, distributed to, or disclosed to others at any time without the prior written consent of Magellan Rx Management.

Editor-in-Chief's MESSAGE Welcome to the MRx Pipeline. This quarterly publication offers clinical insights and competitive intelligence on anticipated drugs in development, so you are well-sourced on the drug pipeline. MRx Pipeline, our universal forecast, addresses trends applicable across market segments. Traditional and specialty drugs as well as agents under the pharmacy and medical benefits are featured. Also profiled in the report are new molecular entities, pertinent new and expanded indications for existing medications, and biosimilars. Clinical analyses, financial outlook, and pre-regulatory status are considered. The products housed in the MRx Pipeline have been researched in detail. They have been developed in consultation with our internal team of clinical and analytics experts.

METHODOLOGY

Emerging therapeutics continue to grow and influence the clinical and financial landscape. Therefore, Magellan Rx Management has developed a systematic approach to determine the products with significant clinical impact. For the in-depth clinical evaluations, the products’ potential to meet an underserved need in the market by becoming the new standard of care, and the ability to replace existing therapies were investigated. The extent to which the pipeline drugs could shift market share on a formulary and their impact on disease prevalence were also important considerations. In order to assist payers with assessing the potential impact of these pipeline drugs, where available, a financial forecast has been included for select products. Primarily complemented by data from EvaluateTM, this pipeline report looks ahead at the 5-year projected annual US sales through the year 2025. These figures are not specific to a particular commercial or government line of business; rather, they look at forecasted total US sales. Depending on a variety of factors, including the therapeutic categories, eventual FDA-approved indications, populations within the plan, and other indices, the financial impact could vary by different lines of business.

REFLECTION

Thus far in 2021, the FDA has approved 41 novel drugs putting the number of approvals at par at approximately the same time in 2020. For the remainder of this year, 32 notable drugs filed with the agency are profiled, each with an anticipated FDA decision in 2021. Should these approvals come to fruition, 2021 could become the year with the highest number of FDA approvals on record. The progress of these agents is being actively monitored through MRx Pipeline.

ON THE HORIZON

As we look ahead, there is a continued trend toward the approval of specialty medications and drugs for rare diseases, with 60% and 32% of approvals expected, respectively, for agents with applications submitted to the FDA. There are 2 agents seeking FDA’s Accelerated Approval, which allows for earlier drug approval for serious conditions that fill an unmet need based on a surrogate endpoint. The growth of biosimilars and new treatment modalities using gene therapy are expected. This will be another critical year in combatting COVID-19 with a public health arsenal of COVID-19 therapeutics and authorizing COVID-19 vaccines for new populations such as pediatrics. Other noteworthy pipeline trends to watch include the development of complex therapies, oncology, immunology, immunodermatology, therapeutic options for rare hereditary diseases. Moreover, sprouting products for cardiology, ophthalmology, and women’s health await on the horizon. The drug pipeline ecosphere will continue to evolve as it faces challenges and successes. Innovative agents that show positive results without compromising patient safety and access offer true therapeutic advances and hold the promise to alter the treatment paradigm. Maryam Tabatabai, PharmD Editor-in-Chief, MRx Pipeline

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Pipeline DEEP DIVE Objective evidence-based methodology was used to identify the Deep Dive drugs in the upcoming quarters. This section features a clinical overview and explores the potential place in therapy for these agents. Moreover, it addresses their FDA approval timeline and 5-year financial forecast.

SPECIALTY

PRIORITY REVIEW

BREAKTHROUGH THERAPY

80%

16%

BIOSIMILAR

ORPHAN DRUG

64%

24%

pecialty drug names appear in  S magenta throughout the publication.

RENAL DISEASE

bardoxolone methyl oral Reata PROPOSED INDICATIONS

Alport syndrome-related chronic kidney disease (CKD)

CLINICAL OVERVIEW

Bardoxolone methyl activates nuclear factor erythroid 2-related factor 2 (Nrf2), which promotes normal mitochondrial function in the cell. This action reduces oxidative stress and inflammation, and may improve kidney function. The randomized, double-blind, phase 3 CARDINAL trial evaluated the safety and efficacy of bardoxolone methyl in 157 patients 12 to 70 years of age with CKD due to Alport syndrome. Enrolled patients had a baseline eGFR of 30 to 90 mL/min/1.73 m2 and urinary albumin to creatinine ratio (UACR) ≤ 3,500 mg/g. In the modified ITT population, bardoxolone methyl led to a significantly greater mean change from baseline in on-treatment eGFR at week 100 (primary endpoint) and off-treatment eGFR at week 104 (secondary endpoint) compared to placebo (on-treatment difference, 11.3 mL/min/1.73 m2 [p=0.0001]; off-treatment difference, 4.3 mL/min/1.73 m2 [p=0.023]). Similar changes in eGFR at weeks 100 and 104 were seen among pediatric patients in the study (n=23) and the overall study population. In addition, among pediatric patients, the UACR, blood pressure, and height and weight growth curves remained generally unchanged with bardoxolone methyl relative to placebo. Most TEAEs were mild to moderate in severity and included muscle spasms and increased aminotransferases (transient increases reported in pediatric patients). A small extension study (EAGLE) demonstrated durability of response for up to 3 years. The oral bardoxolone methyl dose was titrated to a maximum of 20 mg or 30 mg once daily based on the patient’s UACR. The study allowed for a 4-week drug withdrawal period after weeks 48 and 100.

PLACE IN THERAPY

Alport syndrome is a rare disorder characterized by progressive kidney disease. It affects an estimated 30,000 to 60,000 people in the US, and accounts for approximately 3% of CKD in children and 0.2% of ESRD in adults. The onset and progression of kidney disease varies depending on the gene mutations involved. X-linked Alport syndrome (XLAS) is caused by COL4A5 gene mutations and accounts for 80% of all cases. Males with XLAS have earlier onset and progression of kidney disease (kidney failure between 25 to 60 years of age) than XLAS females. Mutations in the COL4A3 and/or COL4A4 genes are seen in autosomal recessive Alport syndrome (ARAS) and autosomal dominant Alport syndrome (ADAS). Kidney failure typically develops in teen or early adult years in males and females with ARAS, and later in adulthood in those with ADAS. There is no cure for Alport syndrome. Early off-label treatment with ACE inhibitors or ARBs may delay progression of kidney disease; however, even with ACE inhibitor or ARB therapy, the annual rate of eGFR decline is reported to be 4 to 5 mL/min/1.73 m2. Patients may require eventual dialysis or kidney transplant. Alport syndrome does not recur in kidney transplants, which may be preferred over dialysis, particularly in males with XLAS and ESRD. In clinical trials, bardoxolone methyl led to a significant increase in eGFR in patients with Alport syndrome. If approved, it may be the first therapy to improve kidney function and possibly slow the progression of kidney disease in patients with Alport syndrome. Ongoing trials report durable response for up to 3 years. Bardoxolone methyl is also in phase 3 clinical trials for polycystic kidney disease.

FDA APPROVAL TIMELINE February 25, 2022  Orphan Drug

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$85

$242

$319

$428

The forecast is a projection of total US sales per year.

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METABOLIC

cipaglucosidase alfa IV Amicus PROPOSED INDICATIONS

Late-onset Pompe disease (LOPD)

CLINICAL OVERVIEW

Cipaglucosidase alfa is a recombinant human acid alpha-glucosidase (rhGAA) enzyme. It is administered with oral miglustat, which stabilizes and enhances the exposure of cipaglucosidase alfa. The double-blind, parallel-group, phase 3 PROPEL study compared cipaglucosidase alfa plus miglustat to alglucosidase alfa (Lumizyme®) in enzyme-replacement therapy (ERT)-experienced and -naïve adults (n=123) with LOPD. In the overall population, at week 52, there was a numerically greater improvement in the 6-minute walk distance (6MWD) from baseline (primary endpoint) with cipaglucosidase alfa/miglustat compared to alglucosidase alfa (+20.8 versus +7.2 meters, respectively; p=0.072), but it did not reach statistical superiority. However, cipaglucosidase alfa/miglustat did lead to a nominally significant and clinically meaningful improvement in the mean forced vital capacity (FVC) over alglucosidase alfa (-0.9 versus -4 percent predicted, respectively; p=0.023; key secondary endpoint). Clinically significant improvements in other secondary endpoints also favored cipaglucosidase alfa/miglustat, including lower extremity manual muscle test (MMT), physical function and maneuvers, fatigue, and pertinent biomarkers (creatine kinase, urine hexose tetrasaccharide). The safety profile was similar between treatment arms. In the PROPEL study, patients were randomized to IV cipaglucosidase 20 mg/kg plus oral miglustat 260 mg or IV alglucosidase alfa 20 mg/kg plus oral placebo every 2 weeks.

PLACE IN THERAPY

Pompe disease is estimated to occur in 1 out of every 40,000 births. The genetic disorder results in a lack of acid alpha-glucosidase (GAA), an essential enzyme for glycogen metabolism. Without adequate GAA, excess glycogen accumulates in the lysosomes of the muscles and leads to tissue damage. Patients experience muscle weakness, including in pulmonary muscles, that progresses to respiratory failure and death. A complete lack of GAA results in early onset Pompe disease (EOPD; also known as infantile Pompe disease) with death commonly occurring before the first birthday. LOPD is the consequence of partial GAA deficiency. Its onset can occur during childhood or adulthood. The prognosis of LOPD depends on the age at onset and extent of respiratory muscle damage. ERT is the SOC to improve clinical outcomes in patients with Pompe disease. Available ERTs in the US include alglucosidase alfa (Lumizyme; indicated in all ages) and the recently approved avalglucosidase alfa-ngpt (Nexviazme®; indicated in patients ≥ 1 year of age). If approved, IV cipaglucosidase alfa combined with oral miglustat will provide a third ERT option in adults with LOPD. In the pivotal phase 3 trial, cipaglucosidase alfa/ miglustat narrowly missed demonstrating superiority over the established ERT Lumizyme, based on the primary endpoint of 6MWD. However, the combination did show statistically significant improvement over Lumizyme in FVC, a parameter that was the sole basis for Lumizyme approval. Notably, in its key clinical trial for FDA approval, avalglucosidase alfa also narrowly failed to demonstrate superiority to Lumizyme, based on FVC. A phase 3 trial is underway for cipaglucosidase alfa/miglustat in patients 12 to < 18 years of age with LOPD.

FDA APPROVAL TIMELINE

» May 29, 2022 – oral miglustat (in combination with cipaglucosidase) » July 29, 2022 – cipaglucosidase alfa  Breakthrough Therapy

 Orphan Drug

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$11

$31

$54

$71

The forecast is a projection of total US sales per year.

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OPHTHALMOLOGY

faricimab intravitreal Genentech PROPOSED INDICATIONS

» Neovascular (wet) age-related macular degeneration (AMD) » Diabetic macular edema (DME) » Diabetic retinopathy (DR)

CLINICAL OVERVIEW

Faricimab is a bispecific antibody that blocks vascular endothelial growth factor-A (VEGF-A) and angiopoietin-2 (Ang-2), two distinct pathways implicated in diseases of the retina. Wet AMD The identical, double-blind, phase 3 LUCERNE (n=658) and TENAYA (n=671) trials evaluated the efficacy and safety of intravitreal faricimab 6 mg administered every 8, 12, and 16 weeks compared to aflibercept 2 mg every 8 weeks in treatment-naïve patients with wet AMD. For both agents, the initial 4 doses were administered every 4 weeks. In both studies, faricimab was non-inferior to aflibercept based on the primary endpoint of average change in best-corrected visual acuity (BCVA) score from baseline through week 48. In LUCERNE and TENAYA, BCVA increases were 5.8 and 6.6 letters, respectively, with faricimab compared to 5.1 and 6.6 letters, respectively, with aflibercept. In addition, across both studies, faricimab given at intervals of up to 16 weeks provided similar reductions in central subfield thickness (CST) as every-8-week aflibercept. In the phase 2, double-blind STAIRWAY trial, 76 treatment-naïve patients with wet AMD were randomized to intravitreal ranibizumab 0.5 mg every 4 weeks, or to faricimab 6 mg every 12 or 16 weeks (following an initial 4 doses given every 4 weeks). At week 40, the adjusted mean BCVA gains from baseline (primary endpoint) with faricimab every 12 and 16 weeks were 9.3 and 12.5 letters, respectively, compared to 11.4 letters with ranibizumab. In addition, at week 24, 71% and 61% of patients in the faricimab 12- and 16-week groups, respectively, showed no signs of disease activity compared to 94% of those in the ranibizumab group. Both of the faricimab regimens led to CST reductions similar to ranibizumab. Durability of efficacy was seen through the study end at 52 weeks. DME and DR The identical, randomized, double-blind, phase 3 RHINE (n=951) and YOSEMITE (n=940) trials evaluated the efficacy and safety of intravitreal faricimab 6 mg given every 8 weeks or at individualized frequencies up to every 16 weeks compared to aflibercept 2 mg every 8 weeks in patients with DME. For both agents, maintenance dosing followed 4 initial doses given every 4 weeks. In both studies, faricimab demonstrated non-inferiority to aflibercept based on the primary endpoint of average change in BCVA score from baseline through week 52. BCVA increases in RHINE and YOSEMITE, respectively, were 11.8 and 10.7 letters with faricimab every 8 weeks, 10.8 and 11.6 letters with faricimab up to every 16 weeks, and 10.3 and 10.9 letters with aflibercept. In both studies, faricimab given every 16 weeks led to greater reductions in CST compared to aflibercept every 8 weeks. The absence of DME and intraretinal fluid (IRF) were reported more often with faricimab than aflibercept through week 56. However, similar rates of subretinal fluid (SRF) absence and DR improvements were seen with both agents. The double-blind, phase 2 BOULEVARD study compared faricimab 6 mg (n=82) and ranibizumab 0.3 mg (n=90) in patients with DME. Each agent was administered intravitreally every 4 weeks for a total of 6 doses (until week 20). Following a 16-week off-treatment period, in treatment-naïve patients (n=112), once-monthly faricimab 6 mg demonstrated a statistically significant BVCA gain of 3.6 letters over once-monthly ranibizumab 0.3 mg (BVCA gain, 13.9 versus 10.3 letters, respectively; p=0.03). Greater reductions in CST and a higher proportion of patients achieving ≥ 2-step improvement in the Diabetic Retinopathy Severity Scale (DRSS) were seen with faricimab 6 mg compared to ranibizumab. Among patients who received previous anti-VEGF therapy (n=60), similar mean changes from baseline in BCVA were found with faricimab 6 mg and ranibizumab 0.3 mg (9.6 versus 8.3 letters, respectively), as well as similar CST reductions and a comparable proportion of patients achieving a ≥ 2 step improvement in DRSS. Across all studies, faricimab was well-tolerated. No safety signals were identified. 6 | MAGELLANRX.COM

faricimab cont. PLACE IN THERAPY

AMD is a leading cause of irreversible vision loss in people ≥ 50 years of age. AMD is characterized by deterioration of light-detecting macular cells of the retina. Approximately 10% of cases progress from dry (non-neovascular) AMD to wet (neovascular) AMD caused by the formation of new, fragile, leaky blood vessels behind the retina that lead to scarring and vision loss. Intravitreal administration of VEGF inhibitors is considered first-line treatment for wet AMD, for which several agents are available. These include aflibercept (Eylea®), ranibizumab (Lucentis® and its biosimilar Byooviz™ [availability expected in June 2022]), and brolucizumabdbll (Beovu®), as well as off-label use of the lower cost bevacizumab (Avastin® and its biosimilars Mvasi® and Zirabev®). Approved VEGF inhibitors are administered by an HCP with maintenance dosing every 4 weeks (ranibizumab), every 4 to 8 weeks (aflibercept), or every 8 to 12 weeks (brolucizumab-dbll). DR and DME are major causes of vision loss and blindness in diabetic patients. DR is caused by damage to the retinal vasculature. DME is a progression of DR and is characterized by retinal thickening. It is estimated that 700,000 people in the US have proliferative DR and 500,000 have clinically significant macular edema. Pharmacologic treatment for DR and DME include intravitreal corticosteroids and VEGF inhibitors (aflibercept, ranibizumab [Lucentis only], and off-label bevacizumab). The frequency of HCP-administered VEGF inhibitor maintenance dosing is similar to that used for wet AMD treatment. Unlike other VEGF inhibitors, faricimab targets 2 distinct pathways, VEGF-A and Ang-2, that are implicated in retinal vascular disorders. In clinical trials, faricimab was well-tolerated and demonstrated non-inferiority to aflibercept (Eylea) in vision gains in patients with wet AMD or DME. Faricimab also led to higher absence rates of DME and IRF and similar rates of SRF absence and DR improvements compared to aflibercept. Furthermore, in phase 2 trials, faricimab demonstrated significant visual acuity gains over ranibizumab in patients with wet AMD or DME (when faricimab was dosed every 12 or 16 weeks for wet AMD and every 4 weeks for DME). Across all phase 3 trials, approximately 50% of patients treated with faricimab were able to extend dosing frequency to every 16 weeks. If approved, faricimab will provide an option that targets 2 significant pathways to treat diseases of the retina. On October 22, 2021, Genentech's ranibizumab injection for intravitreal use via ocular implant (Susvimo™) was FDA-approved to treat wet AMD. It employs a port delivery system that is refilled twice per year. The product is in phase 3 trials for DME and DR. Several other VEGF-targeting agents are also in phase 3 trials for retinal conditions.

FDA APPROVAL TIMELINE

» January 31, 2022 – Wet AMD and DME – Priority Review » May 2022 – DR

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$204

$340

$497

$627

The forecast is a projection of total US sales per year.

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INFECTIOUS DISEASE

maribavir oral Takeda PROPOSED INDICATIONS

Refractory cytomegalovirus (CMV) infection treatment in solid organ transplant (SOT) or hematopoietic cell transplant (HCT) recipients

CLINICAL OVERVIEW

Maribavir is an orally bioavailable benzimidazole riboside that is active against CMV. The randomized, open-label, phase 3 SOLSTICE trial compared maribavir (n=235) with investigator assigned conventional antiviral treatment (IAT; n=117) in HCT or SOT recipients. Enrolled patients had confirmed refractory CMV infection, with or without resistance to ≥ 1 conventional antiviral therapy, including ganciclovir, valganciclovir, foscarnet, and cidofovir. At treatment week 8, significantly more patients treated with maribavir (55.7%) compared to those treated with IAT (23.9%) achieved confirmed CMV viremia clearance (primary endpoint), defined as plasma CMV DNA < 137 IU/mL on 2 consecutive tests ≥ 5 days apart (p<0.001). CMV clearance was maintained through week 16 in each group (18.7% versus 10.3%, respectively; p=0.013; secondary endpoint). Lower rates of TEAEs were reported with maribavir compared to IAT. These included neutropenia versus valganciclovir/ganciclovir (1.7% versus 25%, respectively) and acute kidney injury versus foscarnet (1.7% versus 19.1%, respectively). Moreover, therapy discontinuation due to TEAEs was reported at a lower rate with maribavir than with IAT (13.2% versus 31.9%, respectively). One TEAE-related death occurred in each treatment group. Maribavir and IAT were given for 8 weeks. Maribavir was administered orally as 400 mg twice daily.

PLACE IN THERAPY

CMV is a beta-herpes virus and is a common infectious complication of HCT or SOT. CMV infection occurs primarily between 30 and 90 days after transplantation. Risk factors among transplant recipients include reactivation of latent infection (estimated to occur in 40% to 100% of adults), seropositive organ donors, and use of lymphocyte-depleting antibodies. In HCT or SOT recipients, IV ganciclovir and oral valganciclovir are approved to prevent CMV infection in select transplant recipients. Treatment of symptomatic CMV infection relies on off-label use of oral valganciclovir or IV ganciclovir. If resistance to these agents occurs (incidence, 5% to 14%), off-label IV foscarnet or cidofovir may be used. Despite the proven efficacy of the available CMV antivirals, their use may be limited by toxicities, such as myelosuppression (ganciclovir and valganciclovir) and nephrotoxicity (foscarnet and cidofovir). Notably, there are limited data on the benefit of the add-on use of CMV immune globulin for severe CMV infection. Based on its clinical benefit and limited TEAE profile, the FDA's Antimicrobial Drugs Advisory Committee (AMDAC) voted unanimously in favor of approval of maribavir to treat refractory CMV infection with or without genotypic resistance in transplant recipients. The panel stated that maribavir may improve outcomes and QOL in this patient population. If approved, maribavir will be the first agent indicated for the treatment of confirmed CMV infection. Takeda is currently recruiting patients in a phase 3 trial that will compare maribavir 200 mg twice daily to valganciclovir for CMV prevention in transplant recipients; although, a lower dosage of maribavir (100 mg twice daily) did not appear to provide benefit for prevention.

FDA APPROVAL TIMELINE November 19, 2021

 Breakthrough Therapy

 Fast Track

 Orphan Drug

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$19

$52

$94

$135

The forecast is a projection of total US sales per year.

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 Priority Review

CARDIOLOGY

mavacamten oral Bristol-Myers Squibb PROPOSED INDICATIONS

Symptomatic obstructive hypertrophic cardiomyopathy (oHCM)

CLINICAL OVERVIEW

Mavacamten is a reversible, cardiac myosin inhibitor. It reduces adenosine triphosphate (ATP) activity resulting in reduced cardiac muscle contractility. The randomized, double-blind, placebo-controlled, phase 3 EXPLORER-HCM trial evaluated the safety and efficacy of mavacamten in 251 adults with symptomatic (NYHA Class II or III) oHCM and a left ventricular outflow tract (LVOT) gradient ≥ 50 mm Hg. Most patients (97%) were on a background beta-blocker (BB) or calcium channel blocker (CCB). The primary composite efficacy endpoint was an achievement of either a ≥ 1.5 mL/kg/min increase in peak oxygen consumption (pVO2) plus a ≥ 1 NYHA class reduction OR a ≥ 3 mL/kg/min increase in pVO2 with no worsening in NYHA functional class. At week 30, significantly more patients treated with mavacamten met the primary endpoint compared to those given placebo (37% versus 17%, respectively; p=0.0005). Mavacamten also led to significantly greater improvements in secondary measures including post-exercise LVOT gradient and patient-reported symptom scores compared to placebo. Notably, at week 30, mavacamten produced higher reductions in the myocardial wall stress biomarker N-terminal-pro-brain natriuretic peptide (NT-proBNP) and high-sensitivity cardiac troponin I, by 80% and 41%, respectively, relative to placebo. Moreover, an EXPLORER-HCM substudy demonstrated that mavacamten has the potential to reverse cardiac remodeling and reduce left ventricular wall thickness. Mavacamten was generally well tolerated. An interim analysis of the MAVA-LTE extension study demonstrated durable improvements to week 36 in LVOT gradients, NYHA class, left ventricular filling pressures, and NT-proBNP. The initial dose of mavacamten was 5 mg orally once daily. The daily dose was adjusted, ranging from 2.5 mg to 15 mg daily, to maintain an LVOT < 30 mm Hg and mavacamten plasma concentration of 350 to 700 ng/mL.

PLACE IN THERAPY

Hypertrophic cardiomyopathy (HCM) is the most common inherited CV disorder and is characterized by abnormal thickening of the heart muscle. Sarcomere dysfunction distinguishes HCM from other cardiac conditions. LVOT obstruction affects roughly two-thirds of all cases. Approximately 1 in 3,200 people in the US experience symptoms (e.g., shortness of breath and chest pain upon exertion, dizziness, syncope, arrythmia). HCM onset usually occurs during the second or third decades of life. Regarding pharmacotherapy, off-label use of a BB and/or CCB (first-line) and disopyramide (second-line) offer limited relief of symptoms, and anticoagulants are recommended to prevent stroke if atrial fibrillation is present. Procedures, such as septal myectomy or ablation, can provide substantial benefit but are associated with risk of death and barriers of access to specialized centers of care. Notably, the use of implantable cardioverter-defibrillators (ICD) has significantly reduced the risk of sudden cardiac death in high-risk patients. If approved, mavacamten will be the first cardiac myosin inhibitor to treat the underlying cause of HCM. The Institute for Clinical and Economic Review (ICER) reported that the benefit derived from the addition of mavacamten to usual care (BB and/or CCB) over either usual care alone or disopyramide is promising but inconclusive. In addition, ICER states that there is concern among some clinical experts that the LVEF reduction observed in some patients treated with mavacamten may result in long-term harm rather than benefit. Market uptake of mavacamten could be positively impacted if the FDA grants clearance for MyoKardia’s wrist-worn photoplethysmography (PPG) digital device that was used to screen for oHCM in the EXPLORERHCM study. In addition, following positive phase 2 results, Bristol-Myers Squibb has announced a phase 3 trial of mavacamten for treating non-obstructive HCM. Other agents that target myosin for HCM are in phase 2 (aficamten) or earlier development.

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mavacamten cont. FDA APPROVAL TIMELINE January 28, 2022

 Breakthrough Therapy

 Orphan Drug

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$154

$444

$709

$962

The forecast is a projection of total US sales per year.

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METABOLIC

mitapivat oral Agios PROPOSED INDICATIONS

Pyruvate kinase deficiency (PKD)

CLINICAL OVERVIEW

Mitapivat is a first-in-class pyruvate kinase-R (PKR) enzyme activator. FDA submission of mitapivat is supported by the phase 3 ACTIVATE (n=80) and ACTIVATE-T (n=27) trials in adults with PKD. The randomized (1:1), double-blind, placebo-controlled, ACTIVATE study enrolled patients who were not regularly transfused and had a baseline Hb ≤ 10 g/dL. In this trial, 40% of patients treated with mitapivat achieved the primary endpoint of Hb response (defined as ≥ 1.5 g/dL increase in Hb from baseline that was sustained at ≥ 2 scheduled assessments at weeks 16, 20, or 24) compared to none in the placebo group (p=0.0001). Significant improvements in Hb level (difference in mean change from baseline, 1.8 g/dL; p<0.0001), hemolysis and hematopoietic activity markers, and patient-reported outcomes regarding symptom burden and QOL were also seen with mitapivat relative to placebo. The most common grade ≥ 3 TEAEs reported with mitapivat were hypertriglyceridemia (5%) and hypertension (5%). The open-label ACTIVATE-T trial enrolled regularly transfused patients (≥ 6 RBC transfusions in the prior 52 weeks). A transfusion reduction response (defined as a ≥ 33% reduction in RBC units transfused compared to individual historical transfusion burden) was achieved in 37% of patients treated with mitapivat (p=0.0001), and 22% of patients were transfusion-free. Normal Hb concentrations occurring ≥ 8 weeks after a transfusion were reported at least once in 11% of patients treated with mitapivat. No new safety signals were reported in ACTIVATE-T. In both trials, the initial dose of mitapivat was 5 mg orally twice daily, with 2 potential dose escalations to 20 mg twice daily and 50 mg twice daily over a 12-week period in ACTIVATE or a 16-week period in ACTIVATE-T. After the dose-escalation period, patients received a fixed dose for an additional 12 weeks in ACTIVATE or 24 weeks in ACTIVATE-T.

PLACE IN THERAPY

PKD is a rare genetic disorder characterized by chronic hemolytic anemia. The condition is caused by mutations in the PKLR gene leading to a deficiency of the pyruvate kinase enzyme, which is critical for glycolysis. RBCs require energy produced during glycolysis, and without an adequate supply, RBCs hemolyze prematurely (from 120-day lifespan to a few days or weeks). The severity of PKD can vary greatly from life-threatening at birth to mild or no symptoms into adulthood. Complications include gallstones, pulmonary hypertension, osteoporosis, and iron overload. Prevalence is estimated as 1 in 1,000,000 of the general population; however, PKD is most common in people of European descent. In November 2020, Agios and PerkinElmer Genomics launched the Anemia ID program to offer no-cost genetic testing to eligible patients with suspected hereditary anemias, including PKD, which are typically diagnosed using genetic testing and RBC analysis. RBC transfusion is used to treat pediatrics and adults with anemia associated with PKD; the frequency of transfusions depends on how an individual tolerates the hemolytic anemia. Regular use of RBC transfusion leads to iron overload, which necessitates the use of chelation agents or phlebotomy to decrease iron levels. Splenectomy has led to partial improvement in anemia. Supplementation with folic acid can also improve RBC production. If approved, mitapivat will be the first disease modifying therapy to treat PKD.

FDA APPROVAL TIMELINE February 17, 2022  Fast Track

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$29

$106

$181

$207

The forecast is a projection of total US sales per year. 11 | MAGELLANRX.COM

INFECTIOUS DISEASE (COVID-19)

molnupiravir oral Merck/Ridgeback

PROPOSED INDICATIONS

Emergency Use Authorization (EUA) for the treatment of mild to moderate severe acute respiratory syndromeassociated coronavirus-2 (SARS-CoV-2) infection (COVID-19) in adults who are at risk for progressing to severe COVID-19 and/or hospitalization

CLINICAL OVERVIEW

Molnupiravir is an oral, potent ribonucleoside analog that blocks the replication of the SARS-CoV-2 virus. Submission of the EUA is supported by the global, double-blind, placebo-controlled, phase 2/3 MOVe-OUT trial in unvaccinated, non-hospitalized adults with laboratory-confirmed mild to moderate COVID-19. The MOVe-OUT trial was conducted throughout North, South, and Central Americas, as well as Europe, the United Kingdom, Africa, Japan, Taiwan, and the Philippines. Enrolled patients (n=1,550) experienced symptom onset ≥ 5 days before randomization and had ≥ 1 risk factor associated with poor clinical outcomes. The primary efficacy outcome was the percentage of participants who were hospitalized and/or died between randomization through day 29. An interim analysis that included data from 775 patients reported fewer hospitalizations or deaths with molnupiravir compared to placebo (7.3% versus 14.1%, respectively; p=0.0012). The Delta, Gamma, and Mu variants accounted for nearly 80% of the SARS-CoV-2 variants captured in the interim data. No deaths occurred in the molnupiravir group compared to 8 in the placebo group. TEAEs were reported at similar rates in both groups. In the phase 3 period, molnupiravir was administered as 800 mg orally every 12 hours for 5 days (10 doses).

PLACE IN THERAPY

Both preventive and acute treatment options are crucial in defeating the global COVID-19 pandemic. Currently, there is 1 FDA-approved vaccine (Comirnaty®; Pfizer-Biontech) and 2 additional authorized vaccines (Moderna, Janssen) to prevent COVID-19 in the US. However, the availability of anti-SARS-CoV-2 antivirals for the acute treatment of COVID-19 is lacking. To date, Gilead’s IV-administered SARS-CoV-2 antiviral remdesivir (Veklury®) is the only medication FDA-approved to treat COVID-19 and is indicated for use in a hospital or comparable setting. The FDA has also authorized the use of the following IV anti-SARS-CoV-2 monoclonal antibody regimens to treat non-hospitalized patients with mild to moderate COVID-19 who are at high risk of severe illness: bamlanivimab + etesevimab, casirivimab + imdevimab, and sotrovimab. Bamlanivimab + etesevimab and casirivimab + imdevimab are also authorized for post-exposure prophylaxis in select individuals. Based on interim data, molnupiravir reduced the risk of hospitalization or death due to COVID-19 by approximately 50% compared to placebo. The authorization of oral molnupiravir as an at-home treatment of mild to moderate COVID-19 in at-risk adults could be a game changer in the battle against COVID-19. Merck has also initiated a phase 3 evaluation (MOVe-AHEAD) for post-exposure prophylaxis of COVD-19. Notably, other data (MOVe-IN trial) indicate that molnupiravir is unlikely to provide clinical benefit in hospitalized patients who typically have had a longer duration of symptoms. If molnupiravir is granted an EUA, Merck anticipates having 10 million treatment courses available by the end of 2021 for mild to moderate COVID-19 treatment. Other promising oral antiviral candidates in late-stage development for outpatient treatment of mild or moderate COVID-19 include AT-527 (Roche/Atea), favipiravir (Dr. Reddy’s/Appili), and PF-07321332 (Pfizer; in combination with ritonavir). While phase 3 studies for favipiravir and PF-07321332 include adults only, AT-527 is also being studied in adolescents. Various phase 3 trials are assessing PF-07321332 (plus ritonavir) in symptomatic patients with or without increased risk of severe illness and as post-exposure prophylaxis.

FDA APPROVAL TIMELINE

On November 30, 2021, the FDA’s Antimicrobial Drugs Advisory Committee (AMDAC) will review the EUA application for molnupiravir to treat mild to moderate COVID-19 in at-risk patients. FDA authorization is anticipated in December 2021.

FINANCIAL FORECAST (reported in millions)

The financial forecast for molnupiravir is not currently available.

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RESPIRATORY

tezepelumab SC Amgen/AstraZeneca PROPOSED INDICATIONS Severe asthma

CLINICAL OVERVIEW

Tezepelumab is a monoclonal antibody that inhibits the action of thymic stromal lymphopoietin (TSLP). TSLP is a cytokine derived from epithelial cells that is involved in asthma pathophysiology. It mediates interactions between structural cells and immune cells of the airway. TSLP levels are associated with airway obstruction, disease severity, and glucocorticoid resistance. The randomized, double-blind, placebo-controlled, phase 3 NAVIGATOR trial evaluated tezepelumab in patients ≥ 12 years of age with severe, uncontrolled asthma (n=1,061). Background asthma therapy was continued with no changes during the trial. At week 52, the study revealed a 56% reduction in the primary endpoint of annual asthma exacerbation rate (AAER) with tezepelumab compared to placebo (AAER, 0.93 versus 2.1, respectively; p<0.001). The AAER was reduced by 41% (AAER, 1.02 versus 1.73, respectively) in patients with a blood eosinophil count < 300 cells/μL and by 70% (AAER, 0.79 versus 2.66, respectively) in those with a blood eosinophil count ≥ 300 cells/μL. Greater decreases in blood eosinophil count and serum total immunoglobulin E (IgE) were seen with tezepelumab compared to placebo (least square mean differences, -130 cells/μL and -208 IU/mL, respectively). Improvements in forced expiratory volume in 1 second (FEV1) and QOL were also seen with tezepelumab. The safety profile for tezepelumab was similar to placebo. The tezepelumab dosage studied was 210 mg SC every 4 weeks.

PLACE IN THERAPY

It is estimated that over 25 million people in the US are affected by asthma. Up to 15% of cases are severe and difficult to control; of the severe cases, approximately 50% to 60% are eosinophilic phenotype. In patients with severe asthma, monoclonal antibody therapy with an anti-interleukin (IL)-5 agent (benralizumab [Fasenra®; ages ≥ 12 years], mepolizumab [Nucala®; ages ≥ 6 years], and reslizumab [Cinqair®; adults]), an anti-IL-4 agent (dupilumab [Dupixent®; ages ≥ 6 years]), or an anti-IgE agent (omalizumab [Xolair®; ages ≥ 6 years]) is recommended as add-on to inhaled corticosteroid (ICS)/long-acting beta2 agonist (LABA) treatment. Reslizumab and mepolizumab are administered every 4 weeks by an HCP (IV and SC, respectively). Dupilumab, omalizumab, and benralizumab may be self-administered as SC injections every 2, 4, and 8 weeks, respectively. If approved, tezepelumab will be the first-in-class TSLP inhibitor to treat severe eosinophilic phenotype asthma. In clinical trials, it reduced AAER regardless of blood eosinophil levels. As with other monoclonal antibodies indicated for asthma, tezepelumab’s use will likely be limited to those with severe asthma who are inadequately controlled with current SOC; however, oral corticosteroid (OCS) use was not evaluated in tezepelumab trials, unlike benralizumab, dupilumab, and mepolizumab trials that reported reductions in OCS use. No differences in safety and efficacy of patient-/caregiver-administered tezepelumab autoinjector were found in the phase 3 PATH-HOME study compared to the NAVIGATOR study.

FDA APPROVAL TIMELINE January 10, 2022

 Breakthrough Therapy

 Priority Review

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$129

$401

$633

$930

The forecast is a projection of total US sales per year.

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ONCOLOGY/NEUROLOGY

ublituximab IV TG Therapeutics PROPOSED INDICATIONS

» Chronic lymphocytic leukemia (CLL)/Small cell lymphocytic lymphoma (SLL) » Relapsing multiple sclerosis (MS)

CLINICAL OVERVIEW

Ublituximab is a glycoengineered anti-cluster of differentiate 20 (CD20) monoclonal antibody (mAb) that induces potent antibody-dependent cellular cytotoxicity and complement-dependent cytotoxicity. CLL/SLL The randomized, phase 3 UNITY-CLL trial compared ublituximab plus umbralisib (U+U) to obinutuzumab (an anti-CD20 mAb) plus the chemotherapeutic agent chlorambucil (O+C) in 421 patients with CLL who were treatment-naïve or had R/R disease. The study included high-risk patients with 17p deletion, 11q deletion, or unmutated immunoglobulin heavy chain gene (IGHV). At a median follow-up of 36.7 months, U+U demonstrated superiority to O+C (median PFS, 31.9 versus 17.9 months, respectively; p<0.0001). A significantly greater PFS was seen with U+U as first-line and subsequent therapy (HR, 0.482 and 0.601, respectively). The ORR was also significantly higher with U+U compared to O+C (83.3% versus 68.7%, respectively; p<0.001). Grade 3/4 TEAEs for U+U (versus O+C, respectively) included elevated ALT (8.3% versus 1%), elevated AST (5.3% versus 2%), and opportunistic infections (5.8% versus 1.5%). The randomized, phase 3 GENUINE study evaluated the addition of ublituximab to the Bruton’s tyrosine kinase inhibitor ibrutinib in 126 adults with R/R CLL who are at high risk (17p deletion, 11q deletion, and/ or TP53 mutation), have had ≥ 1 prior CLL therapy, and had an ECOG status ≤ 2. After a median follow-up of 41.6 months, a significantly higher ORR was reported with ublituximab/ibrutinib compared to ibrutinib alone (ORR, 83% versus 65%, respectively; p=0.02). Grade 3/4 TEAEs with ublituximab/ibrutinib versus ibrutinib included neutropenia (19% versus 12%, respectively), anemia (8% versus 9%, respectively), and diarrhea (10% versus 5%, respectively). Five deaths were reported due to TEAEs in the ibrutinib group; causality included cardiac arrest, stroke, Pneumocystis jirovecii pneumonia, and cause unknown. In both trials, ublituximab was administered as increasing IV doses during cycle 1 (≤ 150 mg on day 1, 750 mg on day 2, and 900 mg on days 8 and 15) and continued as 900 mg on day 1 of cycles 2 through 6 (28 days per cycle). After cycle 6, ublituximab was given as 900 mg every 3 cycles. In UNITY-CLL, the umbralisib dose was 800 mg orally once daily until disease progression or treatment discontinuation. In GENUINE, ibrutinib 420 mg was administered orally once daily during all cycles. Multiple Sclerosis The 96-week, randomized, double-blind, phase 3 ULTIMATE I and ULTIMATE II clinical trials compared ublituximab monotherapy to teriflunomide (Aubagio®) in 1,094 patients with relapsing MS. In the studies, ublituximab significantly reduced annualized relapse rate (ARR) by 59.4% (p<0.0001) and 49.1% (p=0.0022) relative to teriflunomide, respectively. In each trial, ublituximab reduced the number of contrast enhancing T1 lesions by 96.7% and 96.5%, respectively, and new/enlarging T2 lesions by 92.4% and 90%, respectively, relative to teriflunomide (p<0.0001 for all). Pooled data showed a significant increase in confirmed disability improvement (CDI) with ublituximab over teriflunomide at 12 and 24 weeks (116% [p=0.0003] and 103% [p=0.0026], respectively). However, there was no significant difference between the 2 medications in confirmed disability progression (CDP). The most frequently reported TEAEs with ublituximab were infusion-related reactions, headache, nasopharyngitis, and lymphopenia, which occurred at similar rates as teriflunomide. Serious TEAEs were reported in 9.5% of patients given ublituximab; most notable were infection and nervous system disorders. However, no cases of progressive multifocal leukoencephalopathy (PML) were reported. Three deaths occurred in the ublituximab group, 1 each due to encephalitis, salpingitis, and pneumonia. No deaths were reported with teriflunomide. In both ULTIMATE trials, patients received ublituximab 450 mg infused IV over 1 hour every 24 weeks (following day 1 infusion of 150 mg) or teriflunomide 14 mg orally once daily.

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ublituximab cont. PLACE IN THERAPY

CLL/SLL In CLL, leukemic cells accumulate over time in the blood, bone marrow, and lymphoid tissues, and in SLL these cells accumulate primarily in the bone marrow and lymphoid tissue. CLL is typically diagnosed at about 70 years of age and is considered the predominant type of adult leukemia in Western countries. In the US, it is estimated that 21,250 cases of CLL will be diagnosed and 4,320 related deaths will occur in 2021. Cytogenic abnormalities, such as 17p deletion, 11q deletion, TP53 mutation, and unmutated IGHV, are associated with poor prognosis. The treatment of CLL/SLL is individualized and ranges from observation to cytotoxic and biologic therapies. Preferred first-line and subsequent therapy options, including in high-risk patients, are ibrutinib, acalabrutinib ± obinutuzumab, and venetoclax ± obinutuzumab or rituximab. Chlorambucil plus obinutuzumab is also a first-line option in patients with significant comorbidities or ≥ 65 years of age. Other recommended regimens to treat R/R CLL/SLL include ibrutinib, idelalisib (± rituximab), acalabrutinib, duvelisib, venetoclax (± rituximab), and zanubrutinib. While there are several options to treat CLL/SLL, an unmet need still remains since patients eventually relapse. Combination therapy that includes ublituximab demonstrated significantly higher ORRs than select SOC therapies, as shown in the UNITY-CLL and GENUINE clinical trials. If approved, ublituximab may be an important addition to the CLL/SLL treatment armamentarium. Multiple Sclerosis MS is a complex human autoimmune-type inflammatory disease that causes demyelination and damage to the neurons in the brain and spinal cord. Relapsing forms of MS are characterized by isolated attacks of neurologic symptoms followed by periods of remission. It is estimated that nearly 1 million people in the US are living with MS. Significant physical disability occurs in over 30% of patients within 20 to 25 years of onset and cognitive dysfunction affects 40% to 70% of patients. There is no cure for MS. Treatment is aimed at preventing new MS attacks and improving physical function after an attack. There are currently several disease modifying therapies established in treating relapsing MS, including injectable and oral formulations. PML is a rare and serious brain infection that has been associated with some MS therapies, including other CD20-targeted mAbs when used to treat MS; however, to date, PML has not been reported with ublituximab for MS. If approved, ublituximab will be the third anti-CD20 mAb to treat relapsing MS, following ocrelizumab (Ocrevus®) and ofatumumab (Kesimpta®). In non-comparison trials, efficacy of ublituximab was similar to ofatumumab (51% to 59% relative reduction in ARR reported with ofatumumab compared to teriflunomide). Ocrelizumab (Ocrevus) was the first anti-CD20 mAb approved, and its utility in care is now established, which may hinder ublituximab’s uptake in the MS space. Furthermore, ocrelizumab and ofatumumab are also approved for progressive MS, an indication that currently does not appear to be studied with ublituximab.

FDA APPROVAL TIMELINE » March 25, 2022 – CLL/SLL  Fast Track

 Orphan Drug

» September 30, 2022 – MS

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$212

$661

$946

$1,456

The forecast is a projection of total US sales per year.

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Biosimilar Overview CLINICAL OVERVIEW

Biosimilars are very different from generic drugs in that they are not exact duplicates of their reference biologic product. The FDA approval process for biosimilars is designed to ensure that the biosimilar product is highly similar to the reference product without having any meaningful clinical differences. Moreover, an interchangeable biological product is a biosimilar that is expected to produce the same clinical result as the reference product in any given patient. Switching or alternating between the reference and interchangeable products should not negatively impact the safety and efficacy of therapy. Many controversies surround biosimilars, and regulatory and litigation hurdles remain. The FDA has issued final and draft guidances. Select FDA biosimilar guidances are noted here. In January 2017, the agency issued final guidance on the nonproprietary naming of biologic products, which also applies to biosimilars. The biological products must bear a core name followed by a distinguishing 4-letter, lowercase, hyphenated suffix that is devoid of meaning. The international nonproprietary name (INN) impacts interchangeability as it affects pharmacists’ ability to substitute an interchangeable biosimilar for the reference product. The FDA withdrew the September 2017 draft industry guidance on determining similarity of a proposed biosimilar product to its reference product to allow for further consideration of the most current and relevant scientific methods in evaluating analytical data. The agency will focus on providing flexibility for the efficient development of biosimilars while maintaining high scientific standards. In July 2018, the FDA finalized its guidance on labeling biosimilars. The guidance pertains to prescribing information (PI) but does not contain specific recommendations on interchangeability in the labeling. The labeling guidance provides recommendations on how to include, identify, and differentiate the biosimilar and the reference product in various sections of the PI. The basic premise remains that the originator product’s safety and effectiveness can be relied upon for HCPs to make prescribing decisions; therefore, a biosimilar should include relevant data from the originator in its PI. In May 2019, the agency released its final guidance on interchangeability. Most states have enacted biosimilar substitution laws. An interchangeable product may be substituted for the originator at the pharmacy without the involvement of the prescriber. The Purple Book is an FDA database of licensed biological products which lists biosimilar and interchangeable products. The FDA has approved 2 biosimilars for interchangeability to their reference product: insulin glargine-yfgn (Semglee®) and adalimumab-adbm (Cyltezo®). Biosimilars can use extrapolation of efficacy and safety data to receive an indication without direct trials of the biosimilar for the eligible indication(s) of the reference product without requiring additional trials. Nevertheless, as each biosimilar comes to market, it will need to be considered individually. The FDA historically regulated insulins as small molecules. However, effective March 23, 2020, drugs such as insulin and growth hormone were deemed biologics and transitioned from the drug pathway to the biologic pathway. Their licensure as biologics allows these agents to be considered in the biosimilar space and promotes competition and access.

PLACE IN THERAPY

The patents of several biologic drugs are set to expire in the next few years, opening the US market for biosimilar entry; however, patent litigation has resulted in significant launch delays of FDA-approved biosimilars. In June 2017, the US Supreme Court issued 2 landmark rulings: (1) allowing a biosimilar manufacturer to provide launch notice of commercial marketing to the originator manufacturer before or after FDA approval of the biosimilar product and (2) eliminating any federal requirement for disclosure, also known as the “patent dance.” Some states, however, mandate disclosure. These decisions may bring biosimilars to the market sooner and potentially create price competition in the marketplace. In July 2018, the FDA unveiled its Biosimilar Action Plan (BAP), a series of 11 steps to encourage biosimilar market competition, some of which were previously announced or underway. The BAP contains 4 key strategies: (1) improving the biosimilar development and approval process; (2) maximizing scientific and regulatory clarity for sponsors; (3) providing effective communications for patients, clinicians, and payers; and (4) reducing unfair tactics that may delay market approval and entry. The BAP strives to promote access to biosimilar products and reduce healthcare costs.

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To date, a total of 30 biosimilars have received FDA approval. Of these, only 20 have entered the market. APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Zarxio® (filgrastim-sndz)

Novartis/Sandoz

March 2015

Inflectra® (infliximab-dyyb)

Pfizer/Celltrion

April 2016

Erelzi® (etanercept-szzs)

Novartis/Sandoz

August 2016

Amjevita™ (adalimumab-atto)

Amgen

September 2016

Renflexis® (infliximab-abda)

Samsung Bioepis/ Merck

May 2017

Cyltezo (adalimumab-adbm)

Boehringer Ingelheim

August 2017

Mvasi (bevacizumab-awwb)

Amgen

September 2017

Ixifi™ (infliximab-qbtx)†

Pfizer

December 2017

Ogivri® (trastuzumab-dkst)

Viatris

December 2017

Retacrit® (epoetin alfa-epbx)

Pfizer/Hospira

May 2018

Fulphila® (pegfilgrastim-jmdb)

Viatris

June 2018

Nivestym® (filgrastim-aafi)

Pfizer

July 2018

Hyrimoz™ (adalimumab-adaz)

Novartis/Sandoz

October 2018

Udenyca® (pegfilgrastim-cbqv)

Coherus

November 2018

Truxima® (rituximab-abbs)

Celltrion/Teva

November 2018

Herzuma® (trastuzumab-pkrb)

Celltrion/Teva

December 2018

Ontruzant® (trastuzumab-dttb)

Samsung Bioepis/ Merck

January 2019

Trazimera™ (trastuzumab-qyyp)

Pfizer

March 2019

Eticovo™ (etanercept-ykro)

Samsung Bioepis/ Merck

April 2019

Kanjinti™ (trastuzumab-anns)

Amgen

June 2019

Zirabev (bevacizumab-bvzr)

Pfizer

June 2019

Hadlima™ (adalimumab-bwwd)

Samsung Bioepis/ Merck

July 2019

Ruxience® (rituximab-pvvr)

Pfizer

July 2019

Abrilada™ (adalimumab-afzb)

Pfizer

November 2019

Ziextenzo® (pegfilgrastim-bmez)

Novartis/Sandoz

November 2019

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Interchangeable -

Commercially Available

 



    -

     -

 



Originator (Manufacturer) Neupogen® (Amgen) Remicade® (Janssen) Enbrel® (Amgen) Humira® (Abbvie) Remicade (Janssen) Humira (Abbvie) Avastin (Genentech) Remicade (Janssen) Herceptin® (Genentech) Epogen® (Amgen) Procrit® (Janssen) Neulasta® (Amgen) Neupogen (Amgen) Humira (Abbvie) Neulasta (Amgen) Rituxan® (Genentech) Herceptin (Genentech) Herceptin (Genentech) Herceptin (Genentech) Enbrel (Amgen) Herceptin (Genentech) Avastin (Genentech) Humira (Abbvie) Rituxan (Genentech) Humira (Abbvie) Neulasta (Amgen)

APPROVED BIOSIMILARS continued APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Avsola® (infliximab-axxq)

Amgen

December 2019

Nyvepria™ (pegfiltrastim-apgf)

Pfizer

June 2020

Semglee (insulin glargine-yfgn)

Viatris

July 2021

Hulio® (adalimumab-fkjp)

Viatris

July 2020

Riabni™ (rituximab-arrx)

Amgen

December 2020

Byooviz (ranibizumab-nuna)

Biogen/Samsung Bioepis

September 2021

Interchangeable

Commercially Available

Originator (Manufacturer) Remicade (Janssen)



  



Rituxan (Genentech)

Lucentis (Genentech)

Neulasta (Amgen) Lantus® (SanofiAventis) Humira (Abbvie)

† Pfizer already has Inflectra on the market and has not announced plans to launch Ixifi.

Also available are Eli Lilly’s Basaglar® insulin glargine, a follow-on to Sanofi’s Lantus, and Sanofi’s Admelog® insulin lispro, approved as a follow-on to Eli Lilly’s Humalog®. Originally, in June 2020 the FDA approved insulin glargine (Semglee) as a branded biologic that was not biosimilar to Lantus. Subsequently, in July 2021 insulin glargine-yfgn (Semglee) received FDA approval under the 351(k) regulatory pathway as an interchangeable biosimilar. These two products have identical moieties but have different regulatory designations and NDCs. The original Semglee NDCs are expected to sunset. Further, Viatris is introducing insulin glargine-yfgn, its unbranded authorized biologic version of the interchangeable biosimilar. A host of factors will contribute to market acceptability and the potential success of biosimilars. Payers, pharmacies, prescribers, and patients each play a role in market adoption of biosimilars. Specialty medications, which include biologics, make up approximately 36% of global medication spend. Global specialty spending is projected to reach 40% by 2024, and reach 52% in developed markets. While < 2% of Americans use biologics, they account for 26% of all national prescription drug spending. Not surprisingly, there is a growing body of evidence on predicted biologic spend and potential biosimilar savings. A 2020 report by IQVIA forecasts that biosimilars are on track to reduce overall US drug spend by $100 billion over the next 5 years. In fact, the next 5 years are expected to have an estimated 5-fold increase in savings relative to the past 5 years as more biosimilars launch and existing biosimilars see more utilization and reductions in price. Three biosimilar launches in 2019 saw substantial uptake within the first year of commercialization, these are bevacizumab (42%), trastuzumab (38%), and rituximab (20%). In the US, it is estimated that biosimilars will cost approximately 15% to 35% less than the originator product, although price dynamics vary. The potential cost savings can vary based on the market segment where brand contracts can play a role. A 2017 report by the RAND Corporation estimates a $54 billion cost savings from biosimilars between 2017 and 2026. In July 2018, an FDA analysis reported that if Americans had access to FDA-approved biosimilars in 2017, it would have resulted in a $4.5 billion savings. A 2017 analysis by the Moran Company projects biosimilars could save the government an estimated $11.4 billion by 2027, but it would require the Centers for Medicare and Medicaid Services (CMS) to revise its reimbursement policy for biosimilars. Subsequently, in November 2017, the CMS revised its reimbursement policy. The CMS now issues a unique Healthcare Common Procedure Coding System (HCPCS) code to each individual biosimilar. Under this rule, Medicare Part B separately codes and pays for biosimilars and no longer groups them into a common payment code with originator agents. Biosimilars are paving the way for increased access to important biologic therapies that may increase survival and/ or QOL for many patients with difficult-to-treat diseases while also reducing costs.

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BIOSIMILAR OVERVIEW continued

IMMUNOLOGY

adalimumab SC AVT-02, CHS-1420, and Yuflyma are investigational biosimilars to Abbvie’s Humira, a tumor necrosis factor alpha (TNF-α) blocker indicated for the treatment of autoimmune disorders, including rheumatoid arthritis (RA), juvenile idiopathic arthritis (JIA), ankylosing spondylitis (AS), plaque psoriasis (PSO), psoriatic arthritis (PsA), Crohn’s disease (CD) in adults and children, ulcerative colitis (UC), hidradenitis suppurativa (HS), and non-infectious uveitis.

FDA APPROVAL TIMELINE Alvotech (AVT-02) Pending

Celltrion (Yuflyma) August 2022 Coherus (CHS-1420) December 2021

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$17,358

$16,324

$9,416

$6,655

$4,998

The forecast is a projection of total US sales per year for the branded originator product.

ONCOLOGY

bevacizumab IV Aybintio, BAT1706, BEVZ92, Bmab-100, and FKB238 are investigational biosimilars to Genentech’s Avastin, a vascular endothelial growth factor (VEGF)-specific angiogenesis inhibitor indicated for the treatment of metastatic colorectal cancer, non-squamous non-small cell lung cancer (nsNSCLC), glioblastoma, metastatic renal cell carcinoma (RCC), and recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer.

FDA APPROVAL TIMELINE Amneal (BEVZ92) April to May 2022

Bio-Thera Solutions (BAT1706) November 27, 2021 Centus/AstraZeneca (FKB238) Pending Samsung Bioepis/Merck (Aybintio) Pending Viatris/Biocon (Bmab-100) Pending

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$1,052

$784

$680

$602

$545

The forecast is a projection of total US sales per year for the branded originator product. 19 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

BLOOD MODIFIER

filgrastim IV, SC Apotex and Amneal are seeking approval of their investigational biosimilars to Amgen’s Neupogen, a leukocyte growth factor indicated for use in patients with nonmyeloid malignancies who are receiving myelosuppressive anti-cancer drugs; following induction or consolidation chemotherapy for acute myeloid leukemia (AML); with nonmyeloid malignancies in patients who are undergoing myeloablative chemotherapy followed by bone marrow transplantation; to mobilize autologous hematopoietic progenitor cells for collection by leukapheresis; with symptomatic congenital neutropenia‚ cyclic neutropenia‚ or idiopathic neutropenia; and in patients who are acutely exposed to myelosuppressive doses of radiation (hematopoietic syndrome of acute radiation syndrome [HSARS]).

FDA APPROVAL TIMELINE Amneal Pending

Apotex (Grastofil) Pending

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$69

$55

$49

$45

$39

The forecast is a projection of total US sales per year for the branded originator product.

ENDOCRINE

insulin aspart SC Viatris/Biocon Viatris/Biocon is seeking approval of their investigational biosimilar to Novo Nordisk’s Novolog®, a rapid-acting insulin to improve glycemic control in patients with T1DM or T2DM.

FDA APPROVAL TIMELINE Pending

INTERCHANGEABILITY

Interchangeability status is expected.

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$823

$701

$614

$564

$525

The forecast is a projection of total US sales per year for the branded originator product.

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BIOSIMILAR OVERVIEW continued

BLOOD MODIFIER

pegfilgrastim SC Lapelga, Lupifil-P, MSB-11455, and TPI-120 are investigational biosimilars to Amgen’s Neulasta, a leukocyte growth factor indicated for use in patients with non-myeloid malignancies who are receiving myelosuppressive anti-cancer drugs and in patients acutely exposed to myelosuppressive doses of radiation (hematopoietic syndrome of acute radiation syndrome [HSARS]).

FDA APPROVAL TIMELINE Amneal (TPI-120) January 13, 2022 Apotex (Lapelga) Pending Lupin (Lupifil-P) April 2022 Merck/Fresenius (MSB-11455) Pending

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$1,549

$1,289

$1,100

$969

$860

The forecast is a projection of total US sales per year for the branded originator product.

IMMUNOLOGY

ranibizumab intravitreal Coherus Coherus is seeking approval for their investigational biosimilar to Genentech’s Lucentis, a vascular endothelial growth factor (VEGF) inhibitor indicated to treat wet age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME), diabetic retinopathy, and myopic choroidal neovascularization (mCNV).

FDA APPROVAL TIMELINE August 2, 2022

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$1,272

$1,213

$1,122

$1,031

$945

The forecast is a projection of total US sales per year for the branded originator product.

21 | MAGELLANRX.COM

Keep on Your RADAR Notable agents that are further from approval have been identified in this unique watch list. These are products with the potential for significant clinical and financial impact. Their development status is being tracked on the MRx Pipeline radar. These pipeline products, their respective class or proposed indication, as well as an estimated financial forecast for the year 2025, are displayed. The financials are projected total annual US sales, reported in millions. zuranolone

abrocitinib

$1,248

$506

Behavioral health

tirzepatide

Diabetes/Metabolic

Dermatology

adagrasib Oncology

$2,481

$959

tiragolumab

deucravacitinib

$626

$1,289

Immunology

Oncology

sotrovimab

dextromethorphan/ bupropion

COVID-19

$612

Behavioral health

$1,289 Moderna COVID-19 vaccine

donanemab

COVID-19

Neurology

$2,699

$3,701 elivaldogene tavalentivec (Lenti-D)

mirikizumab Immunology

Neurology/Gene therapy

$583

$35 lenadogene nolparvovec (GS-010)

Ophthalmology/Gene therapy

$146

gantenerumab Neurology

$1,716

pecialty drug names appear in  S magenta throughout the publication.

Pipeline DRUG LIST The pipeline drug list is an aerial outline of drugs with anticipated FDA approval through 2023. It is not intended to be a comprehensive inventory of all drugs in the pipeline; emphasis is placed on drugs in high-impact categories. Investigational drugs with a Complete Response Letter (CRL) and those that have been withdrawn from development are also noted. APPLICATION APPLICATION SUBMITTED SUBMITTED TO THE FDA

IN PHASE PHASE 33 TRIALS TRIALS

60%

68%

40%

32%

33%

18%

Specialty

17%

14%

12%

Traditional

Orphan Drug

Priority Review

Breakthrough Therapy

Biosimilar

pecialty drug names appear in  S magenta throughout the publication.

PIPELINE DRUG LIST  Specialty drug names appear in magenta throughout the publication. NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

Submitted (New Drugs) donislecel

Celltrans

T1DM

Submitted – BLA; Orphan Drug

Oct-Dec 2021

lamotrigine

Azurity

Partial seizures

Oral

Submitted – 505(b)(2) NDA

Oct-Dec 2021

dapivirine ring

International Partnership for Microbicides

HIV-1 infection prevention

Intravaginal

Submitted – NDA

Oct-Dec 2021

phenylephrine/ tropicamide

Eyenovia

Pharmacologic mydriasis

Ophthalmic

Submitted – 505(b)(2) NDA

10/29/2021

testosterone undecanoate

Marius

Hypogonadism

Oral

Submitted – 505(b)(2) NDA

10/29/2021

triamcinolone

Bausch

Uveitis

Intravitreal

Submitted – 505(b)(2) NDA

10/29/2021

topiramate oral solution

Azurity

Partial seizures

Oral

Submitted – 505(b)(2) NDA

11/05/2021

treprostinil

Liquidia

PAH

Inhaled

Submitted – 505(b)(2) NDA

11/05/2021

ropeginterferon alfa-2b

Pharmaessentia

Polycythemia vera

Submitted – BLA; Orphan Drug

11/12/2021

maribavir

Takeda

Cytomegalovirus infection treatment (refractory, post solid organ or hematopoietic cell transplant)

Oral

Submitted – NDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review

11/19/2021

omidenepag isopropyl

Santen

Glaucoma/ocular hypertension

Ophthalmic

Submitted – NDA

11/19/2021

vosoritide

Biomarin

Achondroplasia

Submitted – NDA; Orphan 11/20/2021 Drug; Priority Review

sirolimus albumin-bound nanoparticle

Aadi

Perivascular epithelioid cell tumor (advanced, malignant)

Submitted – 505(b)(2) NDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review

11/26/2021

bevacizumab (biosimilar to Genentech’s Avastin)

Bio-Thera

Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC

Submitted – BLA

11/27/2021

sodium thiosulfate

Fennec

Chemotherapy-induced ototoxicity prevention

Submitted – NDA; Breakthrough Therapy; Fast Track; Orphan Drug

11/27/2021

ciltacabtagene autoleucel

Janssen/Legend

Multiple myeloma (R/R)

Submitted – BLA; Breakthrough Therapy; Orphan Drug; Priority Review

11/29/2021

episalvan

Amryt

Epidermolysis bullosa

Topical

Submitted – NDA; Fast Track; Orphan Drug; Priority Review; Rare Pediatric Disease

11/30/2021

24 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

pacritinib

CTI

Myelofibrosis

Oral

Submitted – NDA; seeking Accelerated Approval; Fast Track; Orphan Drug; Priority Review

11/30/2021

plinabulin

Beyondspring

Chemotherapy-induced neutropenia prevention

Submitted – NDA; Breakthrough Therapy; Priority Review

11/30/2021

trivalent hepatitis B vaccine

VBI Vaccines

Hepatitis B infection prevention

Submitted – BLA

11/30/2021

adalimumab (biosimilar to Abbvie’s Humira)

Coherus

RA; AS; PSO; PsA; JIA; CD; UC

Submitted – BLA

December 2021

molnupiravir

Merck/Ridgeback

COVID-19 treatment (outpatient setting)

Oral

Submitted – EUA

December 2021

clindamycin phosphate gel

Daré

Bacterial vaginosis

Intravaginal

Submitted – NDA; Fast Track; Priority Review; QIDP

12/07/2021

budesonide (long-acting)

Calliditas

Immunoglobulin A (IgA) nephropathy (Berger’s disease)

Oral

Submitted – 505(b)(2) NDA; seeking Accelerated Approval; Orphan Drug; Priority Review

12/15/2021

buprenorphine ER

Braeburn

Opioid use disorder (moderate to severe)

Submitted – 505(b)(2) NDA; Fast Track

12/15/2021

efgartigimod

Argenx

Myasthenia gravis

Submitted – BLA; Fast Track; Orphan Drug

12/17/2021

diazepam buccal film

Aquestive

Seizure clusters

Oral transmucosal

Submitted – 505(b)(2) NDA; Fast Track; Orphan Drug

12/23/2021

tadalafil + finasteride

Veru

Benign prostatic hyperplasia

Oral

Submitted – 505(b)(2) NDA

12/23/2021

pilocarpine 1.25%

Allergan

Presbyopia

Ophthalmic

Submitted – 505(b)(2) NDA

12/24/2021

inclisiran

The Medicines Company

Hypercholesterolemia (2nd-line)

Submitted – NDA; Orphan 12/31/2021 Drug

levoketoconazole

Strongbridge

Cushing’s syndrome

Oral

Submitted – 505(b)(2) NDA; Orphan Drug

12/31/2021

somatrogon

Opko/Pfizer

Growth hormone deficiency (pediatrics)

Submitted – BLA; Orphan Drug

January 2022

dexmedetomidine

Bioxcel

Bipolar disorder-related acute aggitation; Schizophrenia-related acute aggitation

Submitted – 505(b)(2) NDA; Fast Track

01/05/2022

carbetocin

Levo

Prader-Willi syndromerelated hyperphagia and behavioral distress

Intranasal

Submitted – NDA; Fast Track; Orphan Drug

01/06/2022

daridorexant

Idorsia

Insomnia

Oral

Submitted – NDA

01/07/2022

tezepelumab

Amgen/AstraZeneca

Asthma (severe)

Submitted – BLA; Breakthrough Therapy; Priority Review

01/10/2022

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Amneal

Neutropenia/leukopenia

Submitted – BLA

01/13/2022

25 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

oteseconazole

Mycovia

Vulvovaginal candidiasis (recurrent)

Oral

Submitted – NDA; Fast Track; Priority Review; QIDP

01/27/2022

mavacamten

Bristol-Myers Squibb

Obstructive hypertrophic cardiomyopathy (symptomatic)

Oral

Submitted – NDA; Breakthrough Therapy; Orphan Drug

01/28/2022

faricimab

Genentech

DME; Wet AMD

Intravitreal

Submitted – BLA; Priority Review

01/31/2022

mitapivat

Agios

Pyruvate kinase deficiency

Oral

Submitted – NDA; Fast Track; Orphan Drug; Priority Review

02/17/2022

dextroamphetamine

Hisamitsu

ADHD

Transdermal

Submitted – NDA

02/22/2022

tebentafusp

Immunocore

Uveal melanoma (metastatic)

Submitted – BLA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review

02/23/2022

bardoxolone methyl

Reata

Alport syndrome-related CKD

Oral

Submitted – NDA; Orphan 02/25/2022 Drug

immune globulin (human) 10%

Green Cross

Primary humoral immunodeficiency

Submitted – BLA

02/25/2022

lenacapavir

Gilead

HIV-1 infection (heavily treatment-experienced)

Submitted – NDA; Breakthrough Therapy; Priority Review

02/28/2022

sintilimab

Eli Lilly

NSCLC (metastatic, EGFR exon 20 insertion mutations)

Submitted – BLA

March 2022

tirzepatide

Eli Lilly

T2DM

Submitted – NDA; Priority Review

Mar-May 2022

donepezil

Corium

Alzheimer’s disease (mild to severe)

Transdermal

Submitted – 505(b)(2) NDA

03/11/2022

ganaxolone

Marinus

Cyclin-dependent kinaselike 5 (CDKL5) deficiency disorder-related seizures

IV, Oral

Submitted – NDA; Orphan 03/18/2022 Drug; Priority Review; Rare Pediatric Disease

relatlimab/nivolumab

Bristol-Myers Squibb

Melanoma (unresectable or metastatic, ages ≥ 12 years)

Submitted – BLA; Priority Review

03/18/2022

gefapixant

Merck

Chronic cough

Oral

Submitted – NDA

03/21/2022

ublituximab

CLL/SLL (in combination with umbralisib)

Submitted – BLA; Fast Track; Orphan Drug

03/25/2022

udenafil

Dong-A Socio

Single ventricle heart disease after Fontan palliation

Oral

Submitted – NDA; Orphan 03/25/2022 Drug

vadadustat

Akebia

Anemia due to CKD (dialysis-dependent, dialysis-independent)

Oral

Submitted – NDA

03/29/2022

benegrastim

Evive

Neutropenia/leukopenia

Submitted – BLA

03/30/2022

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Lupin

Neutropenia/leukopenia

Submitted – BLA

April 2022

bevacizumab (biosimilar to Genentech’s Avastin)

Amneal

Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC

Submitted – BLA

Apr-May 2022

risperidone ER

Teva

Schizophrenia

Submitted – 505(b)(2) NDA

Apr-May 2022

26 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

vonoprazan

Phathom

H. pylori infection (in combination with amoxicillin & clarithromycin)

Oral

Submitted – NDA; QIDP

Apr-Jun 2022

vutrisiran

Alnylam

Transthyretin amyloid polyneuropathy

Submitted – NDA; Fast Track; Orphan Drug

04/14/2022

surufatinib

Hutchmed

Neuroendocrine tumors

Oral

Submitted – NDA; Fast Track; Orphan Drug

04/29/2022

meloxicam/rizatriptan

Axsome

Migraine treatment

Oral

Submitted – 505(b)(2) NDA

04/30/2022

faricimab

Genentech

Diabetic retinopathy

Intravitreal

Submitted – BLA

May 2022

tapinarof

Roivant

PSO (mild-severe)

Topical

Submitted – NDA

05/26/2022

miglustat

Amicus

Pompe disease (in combination with cipaglucosidase alfa)

Oral

Submitted – NDA

05/29/2022

asciminib

Novartis

CML

Oral

Submitted – NDA; Breakthrough Therapy; Fast Track; Orphan Drug

June 2022

trientine tetrahydrochloride

GMP-Orphan

Wilson’s disease

Oral

Submitted – NDA; Orphan Jun-Jul 2022 Drug

sodium phenylbutyrate

Acer

Urea cycle disorders

Oral

Submitted – 505(b)(2) NDA

06/03/2022

betibeglogene autotemcel (Zynteglo)

Bluebird Bio

β-thalassemia (transfusion-dependent)

Submitted – BLA; Breakthrough Therapy; Fast Track; Orphan Drug

Jul-Sep 2022

Moderna COVID-19 vaccine (mRNA-1273)

Moderna

COVID-19 (ages ≥ 18 years)

Submitted – BLA; Fast Track

Jul-Sep 2022

tislelizumab

Beigene

Esophageal cancer

Submitted – BLA; Orphan Drug

07/12/2022

cipaglucosidase alfa

Amicus

Pompe disease (in combination with miglustat)

Submitted – BLA; Breakthrough Therapy; Orphan Drug

07/29/2022

adalimumab (biosimilar to Abbvie’s Humira)

Celltrion

RA; AS; PSO; PsA; JIA; CD; UC

Submitted – BLA

August 2022

toripalimab

Coherus

Nasopharyngeal carcinoma (recurrent or metastatic)

Submitted – BLA; Breakthrough Therapy; Orphan Drug

August 2022

trivalent measles-mumpsrubella (MMR) vaccine

GlaxoSmithKline

Measles, mumps, and rubella prevention

Submitted – BLA

08/02/2022

linzagolix

Obseva

Uterine fibroids

Oral

Submitted – NDA

09/15/2022

ublituximab

MS (relapsing)

Submitted – BLA

09/30/2022

roflumilast cream

Arcutis

PSO (mild to severe)

Topical

Submitted – NDA

10/04/2022

abrocitinib

Pfizer

Atopic dermatitis (moderate-severe)

Oral

Submitted – NDA; Breakthrough Therapy; Priority Review

Pending

adalimumab (biosimilar to Abbvie’s Humira)

Alvotech

RA; AS; PSO; PsA; JIA; CD; UC

Submitted – BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Centus/AstraZeneca

Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC

Submitted – BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Samsung Bioepis/ Merck

Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC

Submitted – BLA

Pending

27 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

bevacizumab (biosimilar to Genentech’s Avastin)

Viatris/Biocon

Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC

Submitted – BLA

Pending

bimekizumab

UCB

PSO

Submitted – BLA

Pending

dextromethorphan/ bupropion

Axsome

MDD

Oral

Submitted – 505(b)(2) NDA; Breakthrough Therapy; Fast Track; Priority Review

Pending

filgrastim (biosimilar to Amgen’s Neupogen)

Amneal

Neutropenia/leukopenia

IV, SC

Submitted – BLA

Pending

filgrastim (biosimilar to Amgen’s Neupogen)

Apotex

Neutropenia/leukopenia

IV, SC

Submitted – BLA

Pending

insulin aspart (biosimilar to Novo Nordisk’s Novolog)

Viatris/Biocon

T1DM; T2DM

Submitted – BLA; seeking Pending Interchangeability

Moderna COVID-19 vaccine (mRNA-1273)

Moderna

COVID-19 (ages 12 to 17 years)

Submitted – EUA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Apotex

Neutropenia/leukopenia

Submitted – BLA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Merck/Fresenius

Neutropenia/leukopenia

Submitted – BLA

Pending

sodium oxybate (oncenightly)

Avadel

Narcolepsy-related excessive daytime sleepiness and cataplexy

Oral

Submitted – 505(b)(2) NDA; Orphan Drug

Pending

tixagevimab + cilgavimab

AstraZeneca

COVID-19 post-exposure prophylaxis

IM, IV

Submitted – EUA

Pending

Submitted (Supplementals) andexanet alfa (Andexxa )

Portola

Acute intracranial IV hemorrhage while taking an oral Factor Xa inhibitor, including edoxaban and enoxaparin

Submitted – sBLA; Breakthrough Therapy; Orphan Drug

October 2021

dexamethasone intracanalicular insert (Dextenza®)

Ocular Therapeutix

Allergic conjunctivitis

Intraocular

Submitted – sNDA

October 2021

tacrolimus (Prograf®)

Astellas

Lung transplant rejection prevention

IV, Oral

Submitted – sNDA

October 2021

abemaciclib (Verzenio®)

Eli Lilly

Breast cancer (high risk HR+, HER2-, early disease)

Oral

Submitted – sNDA

Oct-Dec 2021

brexpiprazole (Rexulti®)

Otsuka

Schizophrenia (adolescents)

Oral

Submitted – sNDA; Priority Review

Oct-Dec 2021

zanubrutinib (Brukinsa®)

Beigene

Waldenstrom macroglobulinemia

Oral

Submitted – sNDA; Fast Track; Orphan Drug; Priority Review

10/18/2021

dupilumab (Dupixent)

Sanofi

Asthma (moderate-severe, SC adjunct, ages 6-11 years)

Submitted – sBLA

10/21/2021

pembrolizumab (Keytruda®)

Merck

RCC (adjuvant, intermediate-high or high risk of recurrence, post nephrectomy)

Submitted – sBLA; Breakthrough Therapy; Priority Review

12/10/2021

Amgen

PSO (mild-moderate)

Oral

Submitted – sNDA

12/17/2021

Intra-Cellular Therapies

Bipolar disorder

Oral

Submitted – sNDA

12/17/2021

apremilast (Otezla®) lumateperone (Caplyta ) ®

28 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

abatacept (Orencia®)

Bristol-Myers Squibb

Acute GVHD prevention (age ≥ 6 years, post HSCT from unrelated donor)

cabotegravir/rilpivirine (Cabenuva®)

Viiv

benralizumab (Fasenra)

DOSAGE FORM

FDA APPROVAL

Submitted – sBLA; Breakthrough Therapy; Priority Review

12/23/2021

HIV-1 infection (treatment, IM every 2-month dosing)

Submitted – sNDA

12/24/2021

AstraZeneca

Nasal polyposis

Submitted – sBLA

Jan-Apr 2022

cabotegravir (Vocabria)

Viiv

HIV-1 infection prevention (pre-exposure prophylaxis [PrEP])

Oral

Submitted – sNDA; Breakthrough Therapy; Priority Review

01/24/2022

vonicog alfa (Vonvendi®)

Takeda

Von Willebrand disease (prevention of bleeding episodes)

Submitted – sBLA; Orphan Drug

01/28/2022

cemiplimab-rwlc (Libtayo®)

Regeneron

Cervical cancer (recurrent or metastatic, after chemotherapy)

Submitted – sBLA; Priority Review; Project Orbis

01/30/2022

risankizumab-rzaa (Skyrizi®)

Abbvie

PsA

Submitted – sBLA

February 2022

tecovirimat (Tpoxx®)

Siga

Smallpox treatment (temporary alternative or oral Tpoxx)

Submitted – sNDA; Fast Track; Orphan Drug

03/04/2022

estradiol/progesterone (Bijuva®) 0.5 mg/100 mg (low-dose)

TherapeuticsMD

Menopause-related moderate to severe vasomotor symptoms

Oral

Submitted – 505(b)(2) sNDA

03/21/2022

umbralisib (Ukoniq®)

CLL/SLL (in combination with ublituximab)

Oral

Submitted – sNDA; Orphan Drug

03/25/2022

pembrolizumab (Keytruda)

Merck

Endometrial carcinoma (advanced, MSI-H or dMMR, monotherapy, ≥ 1 prior systemic therapy, curative surgery or radiation ineligible)

Submitted – sBLA

03/28/2022

semaglutide 2 mg (Ozempic®)

Novo Nordisk

T2DM

Submitted – sNDA

03/28/2022

copanlisib (Aliqopa®)

Bayer

NHL (indolent, R/R, in combination with rituximab)

Submitted – sNDA; Fast Track; Orphan Drug

April 2022

rivaroxaban (Xarelto®)

Janssen

Venous Oral thromboembolism (treatment and risk reduction of recurrence, ages birth to < 18 years); Thromboprophylaxis after Fontan procedure (ages ≥ 2 years)

Submitted – sNDA

Apr-Jun 2022

brolucizumab-dbll (Beovu)

Novartis

DME

Intravitreal

Submitted – sBLA

Apr-Sep 2022

eptacog beta (Sevenfact )

Hema

Hemophilia A and B (prevention of bleeding related to surgery or invasive procedure)

Submitted – sBLA

Apr-Sep 2022

viloxazine (Qelbree®)

Supernus

ADHD (adults)

Oral

Submitted – sNDA

04/29/2022

relugolix/estradiol/ norethindrone (Myfembree®)

Myovant

Endometriosis

Oral

Submitted – sNDA

05/06/2022

29 | MAGELLANRX.COM

IV, SC

APPROVAL STATUS

PIPELINE DRUG LIST continued NAME

DOSAGE FORM

CLINICAL USE

ipilimumab (Yervoy®)

Bristol-Myers Squibb

Esophageal squamous cell carcinoma (advanced or metastatic, 1st-line, in combination with nivolumab)

Submitted – sBLA

05/27/2022

nivolumab (Opdivo®)

Bristol-Myers Squibb

Esophageal squamous cell carcinoma (1st-line, in combination with ipilimumab or fluoropyriidine/platinum chemotherapy)

Submitted – sBLA

05/28/2022

axicabtagene ciloleucel (Yescarta®)

Gilead

Large B-cell lymphoma (R/R, adults)

Submitted – sBLA; Breakthrough Therapy; Orphan Drug

July 2022

upadacitinib (Rinvoq™)

Abbvie

Oral

Submitted – sNDA; Orphan Drug

July 2022

risankizumab-rzaa (Skyrizi)

Abbvie

Submitted – sBLA; Orphan Drug

07/20/2022

setmelanotide (Imcivree™)

Rhythm

Alström-related obesity and hunger; BardetBiedle syndrome-related obesity and hunger

Submitted – sNDA; Breakthrough Therapy; Orphan Drug

07/20/2022

fenfluramine HCl (Fintepla®)

Zogenix

Lennox-Gastaut syndrome

Oral

Submitted – sNDA; Orphan Drug

07/28/2022

abacavir/dolutegravir/ lamivudine (Triumeq) dispersible tablet

GlaxoSmithKline

HIV-1 treatment (pediatrics weighing 14 kg to < 40 kg)

Oral

Submitted – sNDA

August 2022

bupivacaine/meloxicam (Zynrelef™)

Heron

Postsurgical pain (foot and ankle, small–medium open abdominal, and lower extremity total joint arthroplasty surgical procedures)

Instillation

Submitted – sNDA; Breakthrough Therapy; Fast Track

08/04/2022

ustekinumab (Stelara®)

Janssen

PsA

Submitted – sBLA

08/08/2022

axicabtagene ciloleucel (Yescarta)

Gilead

Marginal zone lymphoma (after ≥ 2 prior lines of systemic therapy)

Submitted – sBLA; Breakthrough Therapy; Orphan Drug

Pending

baricitinib (Olumiant®)

Eli Lilly

Atopic dermatitis (moderate-severe)

Oral

Submitted – sNDA

Pending

infliximab-dyyb (Inflectra)

Celltrion

IBS

IV, SC

Submitted – sBLA

Pending

tofacitinib (Xeljanz / Xeljanz XR®)

Pfizer

Oral

Submitted – sNDA

Pending

upadacitinib (Rinvoq)

Abbvie

AS; Atopic dermatitis (moderate-severe; ages ≥ 12 years); PsA

Oral

Submitted – sNDA

Pending

APPROVAL STATUS

FDA APPROVAL

MANUFACTURER

Phase 3 (New Drugs) abaloparatide-TD

Radius

Osteoporosis/osteopenia

Transdermal

Phase 3 – NDA

TBD

acoramidis

Bridgebio

Transthyretin amyloid cardiomyopathy (ATTRCM); Transthyretin amyloid polyneuropathy

Oral

Phase 3 – NDA; Orphan Drug

TBD

adagrasib

Mirati

NSCLC

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

adalimumab (biosimilar to Abbvie’s Humira)

Fresenius

RA; AS; PSO; PsA; JIA; CD; UC

Phase 3 – BLA

TBD

30 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

aflibercept (biosimilar to Regeneron’s Eylea)

Samsung Bioepis/ Biogen

DME; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Santo/Formycon

DME; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Viatris/Janssen

DME; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

amcenestrant

Sanofi

Breast cancer

Oral

Phase 3 – NDA

TBD

anthrax vaccine, adsorbed

Emergent

Anthrax infection

Phase 3 – BLA; Fast Track

TBD

anti-Betv1 monoclonal antibodies (REGN-57135714-5715)

Regeneron

Birch allergy

Phase 3 – BLA

TBD

apolipoprotein A-I (human)

CSL

Atherosclerosis

Phase 3 – BLA

TBD

arfolitixorin hemisulfate

Isofol

CRC

Phase 3 – NDA

TBD

AT-527

Roche/Atea

COVID-19

Oral

Phase 3 – NDA

TBD

ataluren

PTC

DMD

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

autologous genetically modified human dermal fibroblasts

Castle Creek

Epidermolysis bullosa

Intradermal

Phase 3 – BLA; Fast Track; Orphan Drug; RMAT

TBD

baclofen/naltrexone/ sorbitol

Pharnext

Charcot-Marie-Tooth disease

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

balixafortide

Polyphor

Breast cancer

Phase 3 – NDA; Fast Track

TBD

bamlanivimab

Eli Lilly

COVID-19

Phase 3 – BLA

TBD

bardoxolone methyl

Reata

Polycystic kidney disease

Oral

Phase 3 – NDA; Orphan Drug

TBD

bentracimab

Phasebio

Ticagrelor (Brilinta®) reversal

Phase 3 – BLA; Breakthrough Therapy

TBD

beremagene geperpavec

Krystal

Epidermolysis bullosa

Topical

Phase 3 – BLA; Fast Track; Orphan Drug; RMAT

TBD

beroctocog alfa

Hemophilia A

Phase 3 – BLA

TBD

betibeglogene autotemcel (Zynteglo)

Bluebird Bio

Sickle cell disease

Phase 3 – BLA; Fast Track; Orphan Drug; RMAT

TBD

bevacizumab-vikg

Outlook

DME; Retinal vein occlusion-associated macular edema; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

bexagliflozin

Theracos

T2DM

Oral

Phase 3 – NDA

TBD

bimekizumab

UCB

AS; Hidradenitis suppurativa; PsA

Phase 3 – BLA

TBD

bintrafusp alfa

Merck

NSCLC

Phase 3 – BLA

TBD

bis-choline tetrathiomolybdate

AstraZeneca

Wilson’s disease

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

brensocatib

Insmed

Bronchiectasis

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

BRII-196/BRII-198

Brii

COVID-19

Phase 3 – BLA

TBD

bulevirtide

Gilead

Hepatitis D infection

Phase 3 – NDA; Breakthrough Therapy

TBD

cannabidiol

Zynerba

Fragile X syndrome

Transdermal

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

capsaicin

Centrexion

Osteoarthritis pain (knee)

Intraarticular

Phase 3 – 505(b)(2) NDA; Fast Track

TBD

31 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

ceftobiprole medocaril

Basilea

ABSSSI; CAP; HAP

Phase 3 – NDA; Fast Track; QIDP

TBD

ceftriaxone wearable micropump

scPharmaceuticals

Gram+/Gram- infection

Phase 3 – NDA

TBD

CM-AT (pancreatic enzyme)

Curemark

Autism spectrum disorders

Oral

Phase 3 – BLA; Fast Track

TBD

concizumab

Novo Nordisk

Hemophilia A and B

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

COVID-19 vaccine (AZD7442)

AstraZeneca

COVID-19

IM, IV

Phase 3 – BLA

TBD

COVID-19 vaccine (C19VAZ; formerly AZD1222; ChAdOx1)

AstraZeneca

COVID-19

Phase 3 – BLA

TBD

COVID-19 vaccine (INO4800)

Inovio

COVID-19

Phase 3 – BLA

TBD

COVID-19 vaccine (JNJ78436735; formerly Ad26. COV2-S)

Janssen

COVID-19

Phase 3 – BLA

TBD

COVID-19 vaccine (MT2766)

Medicago/ GlaxoSmithKline

COVID-19

Phase 3 – BLA; Fast Track

TBD

COVID-19 vaccine (NVXCoV2373)

Novavax

COVID-19

Phase 3 – BLA; Fast Track

TBD

COVID-19 vaccine (SP0253)

Sanofi/ GlaxoSmithKline

COVID-19

Phase 3 – BLA

TBD

crovalimab

Genentech

Paroxysmal nocturnal hemoglobinuria

IV, SC

Phase 3 – BLA; Orphan Drug

TBD

dalcetrapib

Dalcor

Acute coronary syndrome (ADCY9 AA genotype)

Oral

Phase 3 – NDA

TBD

daprodustat

GlaxoSmithKline

Anemia due to CKD (dialysis-dependent, dialysis-independent)

Oral

Phase 3 – NDA

TBD

darvadstrocel

Takeda

Phase 3 – BLA; Orphan Drug

TBD

dehydrated human Mimedx amnion-chorion membrane

Achilles tendonitis; Plantar fasciitis

Phase 3 – BLA

TBD

dengue tetravalent vaccine

Takeda

Dengue fever

Phase 3 – BLA; Fast Track

TBD

denosumab (biosimilar to Amgen’s Prolia®)

Novartis

Osteoporosis/osteopenia

Phase 3 – BLA

TBD

dersimelagon

Mitsubishi Tanabe

Porphyria

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

despropyl macitentan

Janssen

Hypertension

Oral

Phase 3 – NDA

TBD

deucravacitinib

Bristol-Myers Squibb

PSO

Oral

Phase 3 – NDA

TBD

diazoxide choline

Soleno

Prader-Willi syndrome

Oral

Phase 3 – 505(b)(2) NDA; Fast Track; Orphan Drug

TBD

difluprednate XR

Sun

Ocular pain/inflammation

Ophthalmic

Phase 3 – NDA

TBD

dihydroergotamine

Satsuma

Migraine treatment

Intranasal

Phase 3 – NDA

TBD

dociparstat

Chimerix

AML; COVID-19

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

donanemab

Eli Lilly

Alzheimer’s disease

IV, SC

Phase 3 – BLA; Breakthrough Therapy

TBD

32 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

donaperminogene seltoplasmid

Helixmith

Diabetic foot ulcers (chronic non-healing)

Phase 3 – BLA

TBD

doravirine/islatravir

Merck

HIV-1 infection treatment

Oral

Phase 3 – NDA

TBD

dovitinib

Allarity

Breast cancer; RCC

Oral

Phase 3 – NDA

TBD

durlobactam/sulbactam

Entasis

Acinetobacter baumannii infection

Phase 3 – NDA; Fast Track; QIDP

TBD

dust mite immunotherapy

Stallergenes Greer

Allergic rhinitis

Phase 3 – BLA

TBD

EB-101 (gene therapy)

Abeona

Epidermolysis bullosa

Surgical application

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug; RMAT

TBD

eculizumab (biosimilar to Alexion’s Soliris®)

Amgen

Paroxysmal nocturnal hemoglobinuria

Phase 3 – BLA

TBD

efanesoctocog alfa

Sanofi

Hemophilia A

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

efgartigimod

Argenx

ITP; Myasthenia gravis; Pemphigus vulgaris

IV, SC

Phase 3 – BLA; Orphan Drug

TBD

efruxifermin

Akero

NASH

Phase 3 – BLA; Fast Track

TBD

elivaldogene autotemcel (Lenti-D)

Bluebird Bio

Adrenoleukodystrophy

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

enmetazobactam

Allecra

UTI (complicated)

Phase 3 – NDA; Fast Track; QIDP

TBD

ensifentrine

Verona

COPD

Inhaled

Phase 3 – NDA

TBD

EP-2101 therapeutic vaccine

OSE Immunotherapeutics

NSCLC

Phase 3 – NDA; Orphan Drug

TBD

epinephrine

Bryn

Anaphylaxis

Intranasal

Phase 3 – NDA; Fast Track

TBD

eplontersen

Akcea

Transthyretin amyloid polyneuropathy

Phase 3 – NDA

TBD

eprenetapopt

Aprea

Myelodysplastic syndrome

Phase 3 – NDA; Breakthrough Therapy; Fast Track; Orphan Drug

TBD

esreboxetine

Axsome

Fibromyalgia

Oral

Phase 3 – NDA

TBD

etanercept (biosimilar to Amgen’s Enbrel)

Coherus

RA; Polyarticular JIA; AS; PSO; PsA

Phase 3 – BLA

TBD

etavopivat

Forma

Sickle cell disease

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

etesevimab

Eli Lilly

COVID-19

Phase 3 – BLA

TBD

etranacogene dezaparvovec

Uniqure

Hemophilia B

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

etrasimod

Arena

CD; UC

Oral

Phase 3 – NDA

TBD

etrolizumab

Genentech

Phase 3 – BLA

TBD

fasinumab

Regeneron

Osteoarthritis pain (knee)

Phase 3 – BLA

TBD

favipiravir

Dr. Reddy's/Appili

COVID-19; Influenza

Oral

Phase 3 – NDA

TBD

fexapotide triflutate

Nymox

Benign prostatic hyperplasia

Intratumoral

Phase 3 – NDA

TBD

fezolinetant

Astellas

Menopause vasomotor symptoms

Oral

Phase 3 – NDA

TBD

fidanacogene elaparvovec

Pfizer

Hemophilia B

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

33 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

filgotinib

Gilead

CD; UC

Oral

Phase 3 – NDA

TBD

firmacute eubacterial spores

Seres

Clostridium difficileassociated diarrhea

Oral

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

fitusiran

Sanofi

Hemophilia A and B

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

follitropin alfa (biosimilar to EMD Serono’s Gonal-F®)

Allergan

Female reproductive disorder

Phase 3 – BLA

TBD

follitropin alfa (biosimilar to EMD Serono’s Gonal-F)

Finox

Female reproductive disorder

Phase 3 – BLA

TBD

gantenerumab

Genentech

Alzheimer’s disease

Phase 3 – BLA; Breakthrough Therapy

TBD

gepotidacin

GlaxoSmithKline

UTI (uncomplicated)

Oral

Phase 3 – NDA; QIDP

TBD

giredestrant

Genentech

Breast cancer

Oral

Phase 3 – NDA; Fast Track

TBD

giroctocogene fitelparvovec

Pfizer

Hemophilia A

Phase 3 – BLA; Fast Track; Orphan Drug; RMAT

TBD

givinostat

Italfarmaco

DMD

Oral

Phase 3 – NDA; Orphan Drug; Rare Pediatric Disease

TBD

glatiramer acetate depot

Viatris

Phase 3 – NDA

TBD

glofitamab

Genentech

DLBCL

Phase 3 – BLA

TBD

hypericin

Soligenix

Cutaneous T-cell lymphoma (CTCL)

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

idursulfase

Takeda

Mucopolysaccharidosis II (Hunter syndrome)

Intrathecal

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

inclacumab

Global Blood Therapeutics

Sickle cell disease

Phase 3 – BLA

TBD

infliximab (biosimilar to Janssen’s Remicade)

Nichi-Iko

RA; AS; PSO; PsA; CD; UC

Phase 3 – BLA

TBD

ingenol disoxate

Leo

Actinic keratoses

Topical

Phase 3 – NDA

TBD

insulin aspart (biosimilar to Novo Nordisk’s Novolog)

Sanofi

T1DM; T2DM

Phase 3 – BLA

TBD

insulin glargine (biosimilar to Sanofi’s Lantus)

Gan & Lee

T1DM; T2DM

Phase 3 – BLA

TBD

insulin icodec (onceweekly)

Novo Nordisk

T2DM

Phase 3 – BLA

TBD

iodine-131 apamistamab

Actinium

AML

Phase 3 – BLA; Orphan Drug

TBD

ipatasertib

Genentech

Breast cancer (TNBC/HR+, 1st-line, in combination with chemotherapy); Prostate cancer

Oral

Phase 3 – NDA

TBD

iptacopan

Novartis

Immunoglobulin A Oral nephropathy (Berger’s disease); Paroxysmal nocturnal hemoglobinuria

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

KSI-301

Kodiak

DME; Retinal vein occlusion-associated macular edema; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

Lactobacillus reuteri

Infant Bacterial Therapeutics

Necrotizing enterocolitis

Oral

Phase 3 – BLA; Orphan Drug

TBD

34 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

lanifibranor

Inventiva

NASH

Oral

Phase 3 – NDA; Breakthrough Therapy; Fast Track

TBD

L-asparaginase

Erytech

Pancreatic cancer

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

lazertinib

Genosco

NSCLC

Oral

Phase 3 – NDA

TBD

lebrikizumab

Eli Lilly

Atopic dermatitis (moderate-severe)

Phase 3 – BLA; Fast Track

TBD

lecanemab

Eisai

Alzheimer’s disease (early)

Phase 3 – BLA; Breakthrough Therapy

TBD

lenadogene nolparvovec (GS010)

Gensight

Leber’s hereditary optic neuropathy

Intravitreal

Phase 3 – BLA; Orphan Drug

TBD

leniolisib

Pharming

Activated phosphoinositide 3-kinase (PI3K)-delta syndrome

Oral

Phase 3 – NDA; Orphan Drug

TBD

lenzilumab

Humanigen

COVID-19

Phase 3 – BLA

TBD

leriglitazone

Minoryx

Adrenoleukodystrophy

Oral

Phase 3 – NDA; Fast Track; Orphan Drug; Rare Pediatric Disease

TBD

leronlimab

Cytodyn

COVID-19; HIV-1 infection treatment (in combination therapy with HAART, highly treatmentexperienced)

Phase 3 – BLA; Fast Track

TBD

levodopa/carbidopa patch pump

Mitsubishi Tanabe

Parkinson’s disease

Phase 3 – 505(b)(2) NDA

TBD

lidocaine/prilocaine

Plethora Solutions

Premature ejaculation

Topical

Phase 3 – NDA

TBD

ligelizumab

Novartis

Urticaria

Phase 3 – BLA; Breakthrough Therapy

TBD

linrodostat

Bristol-Myers Squibb

Bladder cancer

Oral

Phase 3 – NDA

TBD

lorecivivint

Samumed

Osteoarthritis pain (knee)

Intraarticular

Phase 3 – NDA

TBD

lotilaner

Tarsus

Demodex blepharitis

Ophthalmic

Phase 3 – NDA

TBD

lutetium 177Lu-PSMA-617

Novartis

Prostate cancer

Phase 3 – NDA; Breakthrough Therapy

TBD

magrolimab

Gilead

Myelodysplastic syndrome

Phase 3 – BLA; Breakthrough Therapy; Fast Track; Orphan Drug

TBD

marstacimab

Pfizer

Hemophilia A and B

IV, SC

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

marzeptacog alfa

Catalyst

Hemophilia A and B

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

masitinib

AB Science

ALS; Alzheimer’s disease; Asthma (eosinophilic); MS

Oral

Phase 3 – NDA; Orphan Drug

TBD

mavorixafor

Primary immunodeficiencies

Oral

Phase 3 – NDA; Breakthrough Therapy; Fast Track; Orphan Drug; Rare Pediatric Disease

TBD

mazindol

NLS

Narcolepsy

Oral

Phase 3 – NDA; Orphan Drug

TBD

melphalan

Delcath

Uveal melanoma (hepatic-dominant)

Percutaneous hepatic perfusion

Phase 3 – NDA

TBD

35 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

meningococcal vaccine (PF-06886992)

Pfizer

Meningococcal infection prevention

Phase 3 – BLA

TBD

metachromatic leukodystrophy gene therapy

Orchard

Metachromatic leukodystrophy

Phase 3 – BLA; Orphan Drug; RMAT

TBD

microbiota suspension

Ferring

C. difficile infection (recurrent)

Rectal

Phase 3 – BLA; Breakthrough Therapy; Fast Track; Orphan Drug

TBD

minocycline/edetate/ethyl alcohol

Citius

Catheter-related bloodstream infection (CRBSI)

Phase 3 – NDA; Fast Track; QIDP

TBD

mirikizumab

Eli Lilly

CD; PSO; UC

IV, SC

Phase 3 – BLA

TBD

mirvetuximab soravtansine

Immunogen

Ovarian cancer

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

momelotinib

Sierra Oncology

Myelofibrosis

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

motixafortide

Biolinerx

Stem cell mobilization

Phase 3 – NDA; Orphan Drug

TBD

nabiximols

MS-related spasticity

Oral transmucosal

Phase 3 – NDA

TBD

nalbuphine ER

Trevi

Pruritus

Oral

Phase 3 – NDA

TBD

naloxone

Orexo

Opioid overdose

Intranasal

Phase 3 – 505(b)(2) NDA

TBD

naloxone hydrochloride dihydrate

Elorac

Pruritus

Topical

Phase 3 – 505(b)(2) NDA; Fast Track; Orphan Drug

TBD

narsoplimab

Omeros

Hemolytic uremic syndrome

IV, SC

Phase 3 – BLA; Fast Track

TBD

natalizumab (biosimilar to Biogen’s Tysabri®)

Novartis

Phase 3 – BLA

TBD

navitoclax

Abbvie

Myelofibrosis

Oral

Phase 3 – NDA; Orphan Drug

TBD

nedosiran

Dicerna

Hyperoxaluria

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug; Rare Pediatric Disease

TBD

nemolizumab

Galderma

Atopic dermatitis (moderate-severe); Pruritus

Phase 3 – BLA; Breakthrough Therapy

TBD

nipocalimab

Janssen

Autoimmune hemolytic anemia; Myasthenia gravis

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

nirsevimab

AstraZeneca

RSV infection prevention

N/A

Phase 3 – BLA; Breakthrough Therapy; Fast Track

TBD

nomacopan

Akari

Hemolytic uremic syndrome; HSCTassociated thrombotic microangiopathy; Paroxysmal nocturnal hemoglobinuria

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

olipudase alfa

Sanofi

Niemann-Pick disease

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

36 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

omecamtiv mecarbil

Cytokinetics

Chronic heart failure (with Oral reduced ejection fraction)

Phase 3 – NDA; Fast Track

TBD

OTL-103

Orchard

Wiskott-Aldrich syndrome IV

Phase 3 – BLA; Orphan Drug; RMAT

TBD

oxalobacter formigenes

Oxthera

Hyperoxaluria

Oral

Phase 3 – BLA; Orphan Drug; Rare Pediatric Disease

TBD

padeliporfin

Steba Biotech

Bladder cancer

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

palopegteriparatide

Ascendis

Hypoparathyroidism

Phase 3 – BLA; Orphan Drug

TBD

pamrevlumab

Fibrogen

COVID-19; DMD; IV Idiopathic pulmonary fibrosis; Pancreatic cancer

Phase 3 – BLA; Fast Track; Orphan Drug; Rare Pediatric Disease

TBD

pegadricase

Swedish Orphan Biovitrum

Gout

Phase 3 – BLA

TBD

pegcetacoplan

Apellis

Dry AMD

Intravitreal

Phase 3 – NDA; Fast Track

TBD

pemafibrate

Kowa

Dyslipidemia/ hypercholesterolemia

Oral

Phase 3 – NDA

TBD

perfluorohexyloctane

Bausch

Dry eye disease

Ophthalmic

Phase 3 – NDA

TBD

PF-07321332

Pfizer

COVID-19

Oral

Phase 3 – NDA

TBD

plinabulin

Beyondspring

NSCLC

Phase 3 – NDA

TBD

plonmarlimab

I-Mab

COVID-19

Phase 3 – BLA

TBD

pollinex quattro grass

Allergy Therapeutics

Allergic rhinitis

Phase 3 – BLA

TBD

pollinex quattro ragweed

Allergy Therapeutics

Allergic rhinitis

Phase 3 – BLA

TBD

potassium citrate/ potassium bicarbonate

Advicenne

Renal tubular acidosis

Oral

Phase 3 – NDA

TBD

pozelimab

Regeneron

Paroxysmal nocturnal hemoglobinuria; Chaple disease

IV, SC

Phase 3 – BLA; Orphan Drug

TBD

ranibizumab (biosimilar to Genentech’s Lucentis)

Stada Arzneimittel/ Bausch

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

rapamycin

Timber

Tuberous sclerosis complex-associated facial angiofibromas

Topical

Phase 3 – NDA

TBD

rapamycin (high-strength)

Palvella

Pachyonychia congenita

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

refanalin

Angion

Delayed graft function

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

regdanvimab

Celltrion

COVID-19

Phase 3 – BLA

TBD

relacorilant

Corcept

Cushing’s syndrome

Oral

Phase 3 – NDA; Orphan Drug

TBD

reproxalap

Aldeyra

Allergic conjunctivitis; Dry eye disease

Ophthalmic

Phase 3 – NDA

TBD

rezafungin

Cidara

Candidemia/invasive candidiasis

Phase 3 – NDA; Fast Track; TBD Orphan Drug; QIDP

ridinilazole

Summit

C. difficile-associated diarrhea

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

rilzabrutinib

Principia

ITP; Pemphigus vulgaris

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

ritlecitinib

Pfizer

Alopecia areata

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

37 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

rituximab (biosimilar to Genentech’s Rituxan)

Amgen

RA; CLL/SLL; NHL (indolent); ANCAassociated vasculitis

Phase 3 – BLA

TBD

rituximab (biosimilar to Genentech’s Rituxan)

Archigen

RA; CLL/SLL; NHL (indolent); ANCAassociated vasculitis

Phase 3 – BLA

TBD

rivipansel

Glycomimetics

Sickle cell disease

Phase 3 – NDA; Fast Track; Orphan Drug; Rare Pediatric Disease

TBD

roflumilast cream

Arcutis

Atopic dermatitis

Topical

Phase 3 – NDA

TBD

rogaratinib

Bayer

Bladder cancer

Oral

Phase 3 – NDA

TBD

ropeginterferon alfa-2b

Pharmaessentia

Essential thrombocythemia

Phase 3 – BLA; Orphan Drug

TBD

roxadustat

AstraZeneca

Anemia due to cytotoxic chemotherapy

Oral

Phase 3 – NDA

TBD

rozanolixizumab

UCB

ITP; Myasthenia gravis

Phase 3 – BLA; Orphan Drug

TBD

RSV vaccine (JNJ64400141)

Janssen

RSV infection prevention

Phase 3 – BLA; Breakthrough Therapy

TBD

ruxolitinib (deuterated)

Concert

Alopecia areata

Oral

Phase 3 – NDA; Breakthrough Therapy; Fast Track

TBD

sabatolimab

Novartis

Myelodysplastic syndrome

Phase 3 – BLA

TBD

seasonal influenza nanoparticle vaccine

Novavax

Seasonal influenza prevention

Phase 3 – BLA; Fast Track

TBD

seladelpar

Cymabay

Primary biliary cholangitis Oral

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

seltorexant

Janssen

MDD

Oral

Phase 3 – NDA

TBD

sepofarsen

Proqr

Leber’s congenital amaurosis

Intravitreal

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

sofpironium

Brickell

Axillary hyperhidrosis

Topical

Phase 3 – NDA

TBD

sotagliflozin

Lexicon

Heart failure in patients with T2DM

Oral

Phase 3 – NDA

TBD

sotatercept

Acceleron

PAH

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

sotrovimab

Vir

COVID-19

Phase 3 – BLA

TBD

sparsentan

Travere

Focal segmental glomerulosclerosis; Immunoglobulin A nephropathy (Berger’s disease)

Oral

Phase 3 – NDA; Orphan Drug

TBD

tapinarof

Roivant

Atopic dermatitis

Topical

Phase 3 – NDA

TBD

tebipenem pivoxil

Spero

UTI (complicated); Acute pyelonephritis

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

tecarfarin

Espero

Anticoagulation

Oral

Phase 3 – NDA

TBD

tezepelumab

AstraZeneca

Nasal polyposis

Phase 3 – BLA

TBD

timbetasin

Regentree

Dry eye disease

Ophthalmic

Phase 3 – BLA

TBD

tiragolumab

Genentech

NSCLC; SCLC

Phase 3 – BLA; Breakthrough Therapy

TBD

38 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

tirzepatide

Eli Lilly

Obesity

Phase 3 – NDA

TBD

tislelizumab

Beigene

HCC; NSCLC

Phase 3 – BLA; Orphan Drug

TBD

tocilizumab (biosimilar to Genentech’s Actemra®)

Bio-Thera

Phase 3 – BLA

TBD

tofersen

Biogen

ALS

Intrathecal

Phase 3 – NDA; Orphan Drug

TBD

tominersen

Genentech

Huntington’s disease

Intrathecal

Phase 3 – NDA; Orphan Drug

TBD

tradipitant

Vanda

Atopic dermatitis; COVID-19; Emesis; Gastroparesis; Pruritus

Oral

Phase 3 – NDA

TBD

trastuzumab (biosimilar to Genentech’s Herceptin)

Novartis

Breast cancer; Gastric/ gastroesophageal cancer

Phase 3 – BLA

TBD

trastuzumab (biosimilar to Genentech’s Herceptin)

Tanvex

Breast cancer; Gastric/ gastroesophageal cancer

Phase 3 – BLA

TBD

travoprost implant

Glaukos

Glaucoma/ocular hypertension

Intraocular

Phase 3 – NDA

TBD

trofinetide

Acadia

Rett syndrome

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

tusamitamab ravtansine

Sanofi

NSCLC

Phase 3 – BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Amgen

PSO

IV, SC

Phase 3 – BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Formycon

PSO

IV, SC

Phase 3 – BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Hikma

PSO

IV, SC

Phase 3 – BLA

TBD

valoctocogene roxaparvovec

Biomarin

Hemophilia A

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug; RMAT

TBD

venglustat

Sanofi

Gaucher’s disease; GM2 gangliosidosis (TaySachs disease, Sandhoff disease, AB variant); Polycystic kidney disease

Oral

Phase 3 – NDA; Orphan Drug

TBD

VGX-3100 therapeutic vaccine

Inovio

Cervical dysplasia (human IM papillovirus-positive)

Phase 3 – BLA

TBD

visomitin

Mitotech

Dry eye disease

Ophthalmic

Phase 3 – NDA

TBD

vonoprazan

Phathom

Esophagitis (in combination with amoxicillin ± clarithromycin)

Oral

Phase 3 – NDA

TBD

wilfactin

LFB

Von Willebrand disease

Phase 3 – BLA; Orphan Drug

TBD

zavegepant

Biohaven

COVID-19; Migraine treatment & prevention

Intranasal, Oral

Phase 3 – NDA

TBD

zilucoplan

UCB

Myasthenia gravis

Phase 3 – NDA; Orphan Drug

TBD

zolbetuximab

Astellas

Gastric cancer

Phase 3 – BLA; Orphan Drug

TBD

zoliflodacin

Entasis

Gonorrhea

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

39 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

zolmitriptan microneedle system

Zosano

Migraine treatment; Cluster headache treament

Transdermal

Phase 3 – 505(b)(2) NDA

TBD

zuranolone

Sage

MDD; Post-partum depression

Oral

Phase 3 – NDA; Breakthrough Therapy; Fast Track

TBD

Phase 3 (Supplementals) adalimumab (biosimilar to Abbvie’s Humira; Hulio)

Viatris/Momenta

Hidradenitis suppurativa; Uveitis

Phase 3 – sBLA

TBD

anakinra (Kineret®)

Swedish Orphan Biovitrum

COVID-19

Phase 3 – sBLA

TBD

baricitinib (Olumiant)

Eli Lilly

Alopecia areata; COVID-19; JIA; SLE; Uveitis

Oral

Phase 3 – sNDA; Breakthrough Therapy; Fast Track; Orphan Drug

TBD

benralizumab (Fasenra)

AstraZeneca

ANCA-associated vasculitis; Bullous pemphigoid; Esophagitis

Phase 3 – sBLA; Orphan Drug

TBD

cariprazine (Vraylar®)

Allergan

MDD

Oral

Phase 3 – sNDA

TBD

dupilumab (Dupixent)

Sanofi

COPD; Eosinophilic esophagitis; Pruritus; Urticaria

Phase 3 – sBLA; Breakthrough Therapy; Orphan Drug

TBD

empagliflozin (Jardiance®)

Boehringer Ingelheim

Chronic heart failure (with preserved ejection fraction); Diabetic nephropathy

Oral

Phase 3 – sNDA; Breakthrough Therapy; Fast Track

TBD

eptacog alfa (Novoseven®)

Novo Nordisk

Factor VIII intolerance

Phase 3 – sBLA

TBD

ferric carboxymaltose (Injectafer®)

Daiichi Sankyo

Anemia in heart failure

Phase 3 – sNDA

TBD

fostamatinib (Tavalisse®)

Rigel

Autoimmune hemolytic anemia; COVID-19

Oral

Phase 3 – sNDA; Fast Track; Orphan Drug

TBD

hydrogen peroxide (Eskata®)

Aclaris

Warts

Topical

Phase 3 – sNDA

TBD

immune globulin (human) 10% (Octagam®)

Octapharma

COVID-19; Dermatomyositis

Phase 3 – sBLA; Orphan Drug

TBD

inebilizumab-cdon (Uplizna®)

Horizon

Myasthenia gravis

Phase 3 – sBLA

TBD

L-lactic acid/citric acid/ potassium bitartrate (Phexxi®)

Evofem

Chlamydia trachomatis infection; Neisseria gonorrhoeae infection

Intravaginal

Phase 3 – sNDA; Fast Track; QIDP

TBD

mepolizumab (Nucala)

GlaxoSmithKline

COPD

IV, SC

Phase 3 – sBLA

TBD

meropenem/vaborbactam (Vabomere®)

Melinta

Bacteremia; HAP

Phase 3 – sNDA; QIDP

TBD

nitazoxanide (Alinia®)

Lupin

COVID-19; Influenza

Oral

Phase 3 – sNDA

TBD

obeticholic acid (Ocaliva )

Intercept

NASH

Oral

Phase 3 – sNDA; Breakthrough Therapy

TBD

odevixibat (Bylvay™)

Albireo

Alagille syndrome-related Oral cholestatic pruritus

Phase 3 – sNDA; Orphan Drug

TBD

omalizumab (Xolair)

Genentech

Food allergies

Phase 3 – sBLA; Breakthrough Therapy

TBD

40 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

patisiran (Onpattro®)

Alnylam

Transthyretin amyloid cardiomyopathy (ATTR-CM, wild type or hereditary)

Phase 3 – sNDA; Orphan Drug

TBD

pegylated liposomal irinotecan (Onivyde®)

Ipsen

SCLC

Phase 3 – sNDA; Fast Track; Orphan Drug

TBD

ranibizumab via ocular implant (Susvimo)

Genentech

Diabetic retinopathy; DME

Intravitreal

Phase 3 – sBLA

TBD

ravulizumab-cwvz (Ultomiris®)

Alexion

COVID-19; HSCTassociated thrombotic microangiopathy; Myasthenia gravis; Neuromyelitis optica (Devic’s syndrome)

IV, SC

Phase 3 – sBLA

TBD

risankizumab-rzaa (Skyrizi)

Abbvie

Phase 3 – sBLA

TBD

rivaroxaban (Xarelto)

Janssen

COVID-19

Oral

Phase 3 – sNDA

TBD

romiplostim (Nplate )

Amgen

Chemotherapy-induced thrombocytopenia

Phase 3 – sBLA; Orphan Drug

TBD

ruxolitinib cream (Opzelura™)

Incyte

Vitiligo

Topical

Phase 3 – sNDA

TBD

secukinumab (Cosentyx®)

Novartis

Hidradenitis suppurativa

IV, SC

Phase 3 – sBLA

TBD

ticagrelor (Brilinta)

AstraZeneca

Sickle cell disease

Oral

Phase 3 – sNDA

TBD

tisagenlecleucel-t (Kymriah®)

Novartis

DLBCL (1st relapse)

Phase 3 – sBLA

TBD

tocilizumab (Actemra)

Genentech

COVID-19

Phase 3 – sBLA

TBD

upadacitinib (Rinvoq)

Abbvie

CD; Giant cell arteritis

Oral

Phase 3 – sNDA

TBD

Complete Response Letter (CRL)/Withdrawn Drugs NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

balstilimab

Agenus

Cervical cancer (R/R)

Withdrawn

TBD

cantharidin

Verrica

Molluscum contagiosum

Topical

CRL

TBD

casirivimab/imdevimab

Regeneron

COVID-19 treatment (outpatient setting); COVID-19 post-exposure prophylaxis

IM, IV, SC

CRL

TBD

eflapegrastim

Spectrum

Neutropenia/leukopenia

CRL

TBD

narsoplimab

Omeros

HSCT-associated thrombotic microangiopathy

IV, SC

CRL

TBD

oportuzumab monatox

Sesen

Bladder cancer (highrisk, BCG-unresponsive, nonmuscle invasive)

Intravesical

CRL

TBD

palovarotene

Ipsen

Fibrodysplasia ossificans progressiva

Oral

CRL

TBD

pirfenidone (Esbriet®)

Genentech

Idiopathic pulmonary fibrosis (unclassified)

Oral

CRL

TBD

retifanlimab

Incyte

Anal cancer (squamous cell, locally advanced/ metastatic, failed on/ intolerant to platinumbased chemotherapy)

CRL

TBD

41 | MAGELLANRX.COM

NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

risperidone

Rovi

Schizophrenia

CRL

TBD

roxadustat

AstraZeneca

Anemia due to CKD (dialysis-dependent, dialysis-independent)

Oral

CRL

TBD

sulopenem etzadroxil/ probenecid

Iterum

Uncomplicated UTI (quinolone-resistant)

Oral

CRL

TBD

tenapanor (Ibsrela®)

Ardelyx

Hyperphosphatemia (dialysis-dependent patients)

Oral

CRL

TBD

treosulfan

Medac

Allogenic-HSCT conditioning

IV, Oral

CRL

TBD

treprostinil DPI

United Therapeutics

PAH; Idiopathic pulmonary fibrosisassociated pulmonary hypertenson

Inhaled

CRL

TBD

42 | MAGELLANRX.COM

GLOSSARY 6MWT 6 Minute Walking Test

CHF Congestive Heart Failure

ABSSSI Acute Bacterial Skin and Skin Structure Infection

CI Confidence Interval

ACEI Angiotensin-Converting Enzyme Inhibitor

CLL Chronic Lymphocytic Leukemia

ACR20 American College of Rheumatology 20% Improvement ACR50 American College of Rheumatology 50% Improvement

CKD Chronic Kidney Disease CML Chronic Myeloid Leukemia CNS Central Nervous System COPD Chronic Obstructive Pulmonary Disease

ACR70 American College of Rheumatology 70% Improvement

COVID-19 Coronavirus Disease 2019

ADC Antibody-Drug Conjugate

CRL Complete Response Letter

ADHD Attention Deficit Hyperactivity Disorder

CRR Complete Response Rate

ADL Activities of Daily Living

CSF Colony Stimulating Factor

AED Anti-Epileptic Drug

CV Cardiovascular

ALK Anaplastic Lymphoma Kinase

CVD Cardiovascular Disease

ALL Acute Lymphoblastic Leukemia ALS Amyotrophic Lateral Sclerosis

DAS28-CRP Disease Activity Score-28 with C Reactive Protein

ALT Alanine Transaminase

DEA Drug Enforcement Administration

AMD Age-Related Macular Degeneration

DLBCL Diffuse Large B Cell Lymphoma

AML Acute Myeloid Leukemia

DMARD Disease Modifying Antirheumatic Drug

ANCA Antineutrophil Cytoplasmic Antibodies

DMD Duchenne Muscular Dystrophy

ANDA Abbreviated New Drug Application

DME Diabetic Macula Edema

ARB Angiotensin II Receptor Blocker

DMT Disease Modifying Therapy

ARNI Angiotensin Receptor II Blocker – Neprilysin Inhibitor

DNA Deoxyribonucleic Acid

ART Antiretroviral Therapy

DPI Dry Powder for Inhalation

ARV Antiretroviral

DPP-4 Dipeptidyl Peptidase 4

AS Ankylosing Spondylitis

DR Delayed-Release

ASCVD Atherosclerotic Cardiovascular Disease AST Aspartate Aminotransferase

EASI-75 Eczema Area and Severity Index ≥ 75% reduction

BCVA Best Corrected Visual Acuity

ECOG Eastern Cooperative Oncology Group

BLA Biologics License Application

EDSS Expanded Disability Status Scale

BMI Body Mass Index

eGFR estimated Glomerular Filtration Rate

BsUFA Biosimilar User Fee Act

EGFR Epidermal Growth Factor Receptor

CABP Community Acquired Bacterial Pneumonia

ER Extended-Release

CAP Community Acquired Pneumonia

ESRD End-Stage Renal Disease

CAR T Chimeric Antigen Receptor T Cell

EUA Emergency Use Authorization

CD Crohn's Disease

FDA Food and Drug Administration

CDC Centers for Disease Control and Prevention

FH Familial Hypercholesterolemia

CF Cystic Fibrosis

FLT3 FMS-Like Tyrosine Kinase-3

43 | MAGELLANRX.COM

CRC Colorectal Cancer

DOR Duration of Response

GLOSSARY continued G-CSF Granulocyte Colony Stimulating Factor

MADRS Montgomery – Åsberg Depression Rating Scale

GI Gastrointestinal

MAOI Monoamine Oxidase Inhibitor

GIST Gastrointestinal Stromal Tumor

MDD Major Depressive Disorder

GLP-1RA Glucagon-Like Peptide-1 Receptor Agonist

MDI Metered Dose Inhaler

GM-CSF Granulocyte-Macrophage Colony Stimulating Factor

MRI Magnetic Resonance Imaging

GVHD Graft Versus Host Disease

MS Multiple Sclerosis

H Half HAART Highly Active Antiretroviral Therapy HAM-D Hamilton Depression Rating Scale HAP Healthcare-Associated Pneumonia Hb Hemoglobin

MRSA Methicillin-Resistant Staphylococcus Aureus N/A Not Applicable NASH Non-Alcoholic Steatohepatitis NDA New Drug Application NHL Non-Hodgkin Lymphoma

HbA1c Hemoglobin A1c

NIAID National Institute of Allergy and Infectious Diseases

HCC Hepatocellular Carcinoma

NSAID Non-Steroidal Anti-Inflammatory Drug

HCP Healthcare Professional

NSCLC Non-Small Cell Lung Cancer

HCV Hepatitis C Virus

NYHA New York Heart Association

HER Human Epidermal Growth Factor Receptor

ODT Orally Disintegrating Tablet

HER2 Human Epidermal Growth Factor Receptor 2

OR Odds Ratio

HFA Hydrofluoroalkane

ORR Overall/Objective Response Rate

HIT Heparin Induced Thrombocytopenia

OS Overall Survival

HIV Human Immunodeficiency Virus

OTC Over-the-Counter

HIV-1 Human Immunodeficiency Virus-1

PAH Pulmonary Arterial Hypertension

HPV Human Papilloma Virus

PARP Poly(ADP-ribose) polymerase

HR Hazard Ratio

PASI Psoriasis Area and Severity Index

HSCT Hematopoietic Stem Cell Transplant

PASI 50 Psoriasis Area and Severity Index 50%

HTN Hypertension

PASI 70 Psoriasis Area and Severity Index 70%

IBS Irritable Bowel Syndrome

PASI 90 Psoriasis Area and Severity Index 90%

IBS-C Irritable Bowel Syndrome, Constipation Predominant

PASI 100 Psoriasis Area and Severity Index 100%

IGA Investigator's Global Assessment

PCSK9 Proprotein Convertase Subtilisin Kexin 9

IM Intramuscular IRB Internal Review Board ITP Immune Thrombocytopenic Purpura ITT Intent-To-Treat IV Intravenous JIA Juvenile Idiopathic Arthritis LDL Low-Density Lipoprotein LDL-C Low-Density Lipoprotein Cholesterol mAb Monoclonal Antibody MACE Major Adverse Cardiovascular Events

44 | MAGELLANRX.COM

PCI Percutaneous Coronary Intervention PD-1 Programmed Death Protein 1 PD-L1 Programmed Death-Ligand 1 PDUFA Prescription Drug User Fee Application PFS Progression-Free Survival PGA Physician Global Assessment PsA Psoriatic Arthritis PSO Plaque Psoriasis PTCA Percutaneous Transluminal Coronary Angioplasty PTSD Post-Traumatic Stress Disorder Q Quarter

GLOSSARY continued QIDP Qualified Infectious Diseases Product

UC Ulcerative Colitis

QOL Quality of Life

US United States

R/R Relapsed or Refractory

UTI Urinary Tract Infection

R-CHOP Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, Prednisone

VAS Visual Analog Scale

RA Rheumatoid Arthritis

VTE Venous Thromboembolism

RBC Red Blood Cell RCC Renal Cell Carcinoma REMS Risk Evaluation and Mitigation Strategy RMAT Regenerative Medicine Advanced Therapy RNA Ribonucleic Acid RRR Relative Risk Reduction RSV Respiratory Syncytial Virus RTOR Real-Time Oncology Review sBLA supplemental Biologics License Application SARS-CoV-2 Severe acute respiratory syndromeassociated coronavirus-2 SC Subcutaneous SCCHN Squamous Cell Cancer of the Head and Neck SCLC Small Cell Lung Cancer SCT Stem Cell Transplant SGLT2 Sodium-Glucose Co-Transporter 2 SL Sublingual SLE Systemic Lupus Erythematosus SLL Small Lymphocytic Lymphoma sNDA supplemental New Drug Application SNRI Serotonin and Norepinephrine Reuptake Inhibitor SOC Standard of Care sPGA static Physician Global Assessment SR Sustained-Release SSRI Selective Serotonin Reuptake Inhibitor SSSI Skin and Skin Structure Infection T1DM Type 1 Diabetes Mellitus T2DM Type 2 Diabetes Mellitus TBD To Be Determined TEAE Treatment-Emergent Adverse Event TNBC Triple Negative Breast Cancer TNF Tumor Necrosis Factor TNFα Tumor Necrosis Factor-alpha UA Unstable Angina 45 | MAGELLANRX.COM

VEGF Vascular Endothelial Growth Factor WBC White Blood Cell WHO World Health Organization XR Extended-Release