MRx Pipeline - July 2021 by Prime/Magellan Rx

MRx PIPELINE A VIEW INTO UPCOMING SPECIALTY & TRADITIONAL DRUGS JULY 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 Senior Manager, 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.

LOOKING BACK

Thus far in 2021, the FDA has approved 31 novel drugs including a controversial Accelerated Approval for Alzheimer's disease based on a surrogate marker. There have been a number of Emergency Use Authorizations (EUAs) for COVID-19. Furthermore, FDA inspections for domestic plants transitioned back to normal in July 2021; overseas site inspections that are critical remain a priority. This new development allows the agency to address the pandemic backlog of facility visits.

ON THE HORIZON

As we look ahead, there is a continued trend toward the approval of specialty medications and drugs for rare and ultra rare diseases, with 62% and 33% of approvals expected, respectively, for agents with applications submitted to the FDA. There are 6 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 including the first interchangeable biosimilar insulin 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 the road to FDA-approved COVID-19 vaccines. Other noteworthy pipeline trends to watch include the development of complex therapies, agents for oncology, immunology, and immunodermatology, and therapeutic options for rare hereditary diseases. Moreover, sprouting products for hematology, 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

92%

23%

BREAKTHROUGH THERAPY

12%

BIOSIMILAR

ORPHAN DRUG

62%

27%

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

NEUROLOGY

atogepant oral Abbvie/Merck PROPOSED INDICATIONS

Prophylaxis of episodic migraine

CLINICAL OVERVIEW

Atogepant is a calcitonin gene-related peptide (CGRP) receptor antagonist. The 12-week, double-blind, phase 3 ADVANCE trial enrolled 910 patients who experienced 4 to 14 monthly migraine days (MMD). Patients were randomized to once-daily oral atogepant 10 mg, 30 mg, or 60 mg or placebo. All atogepant doses led to a significant decrease in MMD (primary endpoint) by 3.69, 3.86, and 4.2 days, respectively, compared to a decrease of 2.48 MMD with placebo (p≤0.0001 for all dose groups versus placebo). In addition, 55.6%, 58.7%, and 60.8% of patients in the respective atogepant treatment arms achieved at least a 50% reduction in MMD compared to 29% in the placebo arm (p≤0.0001 for all dose groups versus placebo). Statistically significant and clinically meaningful improvements in emotional impact of migraine and ability to carry out daily activities were revealed by secondary endpoints. The most common TEAEs reported were constipation (6.9% to 7.7%), nausea (4.4% to 6.1%), and upper respiratory tract infection (3.9% to 5.7%), the majority being of mild to moderate severity. Significant MMD reductions were also shown with atogepant once-daily doses of 10 mg, 30 mg, and 60 mg and twice-daily doses of 30 mg and 60 mg as evaluated in a 12-week, placebo-controlled, phase 2b/3 clinical trial. In addition, a durable response with atogepant 60 mg and no new safety signals were evident at 1 year in an open-label, long-term study (n=744). Among atogepant responders, at 1 year, 84.2% experienced ≥ 50% reduction in MMD, 69.6% experienced ≥ 75% reduction in MMD, and 48.4% experienced 100% reduction in MMD.

PLACE IN THERAPY

Migraine affects approximately 39 million men, women, and children in the US, the majority of whom have a family history of the condition. Migraine attacks can be debilitating with pain that can last hours to days. Triptan therapy is the SOC to treat acute moderate to severe migraine episodes. Since 2018, the FDA has approved 4 injectable CGRP receptor-directed monoclonal antibodies (eptinezumab-jjmr [Vyepti®], erenumab [Aimovig®], fremanezumab [Ajovy®], and galcanezumab [Emgality®]) for migraine prophylaxis in adults. In 2019 and 2020, the oral CGRP receptor antagonists ubrogepant (Ubrelvy®) and rimegepant (Nurtec® ODT) gained FDA approval for the acute treatment of migraine with or without aura in adults. In 2021, the indication for rimegepant was expanded to include preventive treatment of episodic migraine in adults. If approved, atogepant will be the second oral CGRP receptor antagonist to prevent episodic (< 15 MMD) migraine attacks. Rimegepant is dosed every other day while atogepant requires daily administration; however, atogepant demonstrated safety and efficacy across multiple dosage strengths and may provide more dose flexibility compared to rimegepant. Similar efficacy was observed between the 2 agents in non-comparative trials, but rimegepant was associated with a lower incidence of nausea (2.7%); additionally, constipation and upper respiratory tract infection are not reported in the rimegepant labeling. Liver toxicity has not been associated with atogepant, ubrogepant, or rimegepant, unlike other CGRP antagonists that are no longer in development. Atogepant is also in phase 3 trials for the prevention of chronic (≥ 15 MMD) migraine. In addition, the investigational oral CGRP receptor antagonist zavegepant is in phase 3 trials for acute treatment and prevention of migraine.

FDA APPROVAL TIMELINE August to September, 2021

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$102

$277

$429

$590

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

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ONCOLOGY

ciltacabtagene autoleucel (cilta-cel) IV Janssen/Legend PROPOSED INDICATIONS

Relapsed or refractory (R/R) multiple myeloma (MM)

CLINICAL OVERVIEW

Cilta-cel is CAR T therapy that binds to the B-cell maturation antigen (BCMA) on the MM cell surface. This results in CAR T cell proliferation, cytokine secretion, and subsequent MM tumor cell destruction. FDA submission was supported by the phase 1b/2 CARTITUDE-1 trial that enrolled patients with ≥ 3 prior lines of therapy (median, 6) including treatment with an immunomodulatory agent, a proteasome inhibitor, and a CD38-directed antibody. Bridging therapy was allowed after apheresis and cilta-cel was given 5 to 7 days after lymphodepleting chemotherapy (cyclophosphamide and fludarabine). A total of 97 patients with a median of 6 prior lines of therapy (range, 3 to 18) were treated with cilta-cel. At a median follow-up of 18 months, ORR was 97.9% (primary endpoint), with a 80.4% stringent complete response (sCR) rate. The median time to first response was 1 month. The DOR was 21.8 months. The overall 18-month PFS and OS rates were 66% and 80.9%, respectively; in patients who achieved a sCR, the PFS at 18-months was 75.9%. Based on safety data at 12-months, grade 3/4 cytopenia was reported in ≥ 95% of patients. Cytokine release syndrome (CRS) was reported in 95% of patients, of which 4% of cases were grade 3/4. Neurotoxic events (NEs) were reported in 21% of patients, including 10% with grade ≥ 3 NEs. A total of 21 deaths were reported (10 due to disease progression, 6 due to TEAEs, 5 due to non-treatment-related adverse events). Manufacturing failure did not occur with any of the cases, and 1 patient received retreatment with cilta-cel. In addition, the phase 2 CARTITUDE-2 trial evaluated cilta-cel in 20 patients with R/R MM and 1 to 3 prior lines of therapy. It demonstrated an ORR of 95% and sCR rate of 45% at a median 5.8-month follow-up. The overall safety profile was similar to the CARTITUDE-1 trial. Cilta-cel was administered as a single IV infusion with a target dose of 0.75 x 106 CAR+ viable T cells/kg.

PLACE IN THERAPY

MM is a malignancy of plasma cells that accumulate in bone marrow resulting in marrow failure. Nearly 35,000 new cases of MM and over 12,400 deaths due to the condition are predicted in the US in 2021. MM is usually diagnosed in adults ≥ 65 years old and is twice as likely to occur in African Americans than Caucasians. Risk factors may include obesity/overweight, other active plasma cell diseases, and family history of MM. There is no cure for MM. While patients typically respond to initial therapy, eventually treatment-resistant relapse usually occurs, including after HSCT and radiation therapy. Triplet therapy is the standard pharmacotherapy for previously treated MM. Preferred regimens consist of dexamethasone plus 2 of the following: a proteosome inhibitor, an immunomodulator, or a monoclonal antibody. Newer alternatives for patients who have received ≥ 4 prior lines of therapies are monotherapy with belantamab mafodotin-blmf (Blenrep®), an antibody-drug conjugate (ADC), and the CAR T therapy idecabtagene vicleucel (ide-cel; Abecma®). Both treatments target BCMA, which is present in 60% to 70% of patients with MM. If approved, cilta-cel will be the second CAR T therapy approved for MM. Non-comparative clinical trials revealed a higher ORR with cilta-cel versus ide-cel (ORR, 97% versus 72%, respectively) in heavily-treated patients. Furthermore, the Institute for Clinical and Economic Review (ICER) determined that available evidence is inadequate to demonstrate a net health benefit of ide-cel compared to cilta-cel.

FDA APPROVAL TIMELINE November 29, 2021

 Breakthrough Therapy

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$50

$150

$300

The forecast is a projection of total US sales per year. 5 | MAGELLANRX.COM

IMMUNOLOGY

efgartigimod IV Argenx PROPOSED INDICATIONS

Generalized myasthenia gravis (MG)

CLINICAL OVERVIEW

Efgartigimod is an antibody fragment that blocks the neonatal Fc receptor (FcRn) resulting in a reduction in and recycling of immunoglobulin G (IgG) antibodies, which are drivers of autoimmune diseases. The safety and efficacy of efgartigimod were evaluated in the 26-week, randomized, double-blind, placebocontrolled, phase 3 ADAPT trial in 167 adults with generalized MG. Eligible patients were on a stable regimen of ≥ 1 treatment for generalized MG, and 129 patients tested positive for acetylcholine receptor-antibodies (AChR-Ab). Response was defined as a ≥ 2-point improvement in the myasthenia gravis activities of daily living (MG-ADL) score for ≥ 4 consecutive weeks (primary endpoint) and a ≥ 3-point improvement in the quantitative myasthenia gravis (QMG) score (secondary endpoint). After the first treatment cycle, significantly more AChR-Ab-positive participants achieved a response with efgartigimod than with placebo, based on the MG-ADL score (67.7% versus 29.7%, respectively; p=0.0001) and QMG score (63.1% versus 14.1%, respectively). Similar results were reported after the second treatment cycle. Benefit was also seen in patients with no detectable AChR-Ab, among which 47.4% in the efgartigimod group were responders based on both the MG-ADL and QMG scores compared to 21.1% in the placebo group. Notably, efgartigimod was associated with decreases in total IgG and AChR-Ab levels by 61.3% and 57.6%, respectively, in AChR-Abpositive patients; similar IgG decreases were reported in AChR-Ab-negative patients. Onset of effect was evident within 2 weeks of starting efgartigimod and responses for ≥ 12 weeks duration were reported. The safety profile of efgartigimod was similar to placebo. Long-term safety and efficacy are being monitored in the ongoing 3-year, open-label ADAPT+ extension trial. Efgartigimod was administered as 10 mg/kg via IV infusion once weekly for 4 doses per cycle for a total of 26 weeks. After the first cycle, the need for repeated cycles was individualized based on clinical response.

PLACE IN THERAPY

MG is a chronic autoimmune neuromuscular disorder that affects an estimated 20 out of 100,000 people in the US. It typically presents earlier in females than males (mean age, 28 and 42 years, respectively). MG is characterized by weakness of voluntary muscles due to a dramatic reduction in acetylcholine receptors that transmit signals from neurons to muscle fibers. The reduction in receptors is most often caused by AChR-Abs that destroy or block the receptor site. MG episode triggers include infection, stress, surgery, and select medications. Up to 20% of patients with MG will experience an MG crisis with respiratory failure at least once in their lifetime. Treatment includes anticholinesterase inhibitors and immunosuppressive agents as well as thymectomy. Plasmapheresis and IV immunoglobulin have been helpful in severe cases. Eculizumab (Soliris®) is the only biologic indicated to treat MG, specifically in AChR-Ab-positive adults. If approved, efgartigimod will be a first-in-class FcRn inhibitor and will likely be distinguished by its demonstrated efficacy in AChR-Ab-negative patients. Notably, eculizumab carries a boxed warning for serious meningococcal infection, which has not been detected with efgartigimod. While clinical response dictates the need for repeat dosing of efgartigimod beyond the first cycle, eculizumab requires every-2-week IV maintenance dosing for MG. An efgartigimod SC formulation is also in phase 3 development. Other agents in phase 3 trials for generalized MG include rozanolixizumab (FcRn inhibitor), ravulizumab-cwvz (Ultomiris®), and inebilizumab-cdon (Uplizna®).

FDA APPROVAL TIMELINE December 17, 2021  Fast Track

 Orphan Drug

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$127

$297

$575

$811

The forecast is a projection of total US sales per year. 6 | MAGELLANRX.COM

METABOLIC

maralixibat oral Mirum PROPOSED INDICATIONS

Alagille syndrome (ALGS)-related cholestatic pruritus

CLINICAL OVERVIEW

Maralixibat is a non-systemic, apical sodium-dependent bile acid transport (ASBT) inhibitor that increases bile acid excretion in the intestine, resulting in lower systemic bile acid concentrations. Safety and efficacy of maralixibat to treat ALGS-related cholestatic pruritus were demonstrated in the USbased, single-arm, long-term IMAGINE II trial (extension of placebo-controlled, double-blind ITCH trial). The study included patients 12 months to < 18 years of age (mean age, 7 years) with ALGS. Twenty-eight of the total 34 patients in the trial were evaluated. At week 48, clinically meaningful reductions in pruritus were demonstrated by a 1.9-point mean reduction in both the Itch Reported Outcome Observer (ItchRO[Obs]) score and the Clinician Scratch Score (CSS); both measures evaluate pruritus on scales from 0 to 4. In addition, pediatric QOL (PedsQL) was significantly improved by 10.2 points (significant response is defined as ≥ +5 points), and serum bile acid (sBA) concentration was decreased. Response was stable through 96 weeks. An increase in height z-scores was also observed at 96 weeks. Three patients withdrew from the study due to elevated ALT, 2 patients withdrew due to liver transplant, and 1 patient each withdrew due to hematochezia, pancreatitis, nonadherence, progressive cholestasis, elevated total bilirubin, and autoimmune hepatitis. In addition, the pivotal, non-US-based, phase 2, ICONIC trial in 31 patients (mean age, 5.7 years) demonstrated significant reductions in pruritus (measured by ItchRO[Obs] and CSS), sBA, and xanthomas compared to placebo. Long-term accelerated growth was also observed. Maralixibat was generally welltolerated throughout the study. The most frequent TEAEs were diarrhea and abdominal pain. Maralixibat was administered orally once daily. Maximum dose was 280 mcg/kg.

PLACE IN THERAPY

ALGS is a rare, autosomal dominant disorder with an estimated prevalence of 1:30,000 to 1:100,000. Most cases are due to a mutation in the jagged-1 (JAG1) gene and a small percentage (2%) in the notch-2 gene. About half of all cases are not inherited (de novo). ALGS may affect multiple organ systems including liver, heart, kidneys, skeleton, CNS, blood vessels, and eyes. The majority of cases (approximately 90%) involve a lack of bile ducts in the liver. Cholestasis typically presents during the first months of life. An accumulation of sBA results in xanthomas and severe pruritus, which can significantly impact QOL. Dietary treatment is required to correct nutritional imbalances and complications (e.g., fat-soluble vitamin malabsorption, failure to thrive, poor linear growth, visual changes) associated with ALGS. Pruritus is often refractory to medical therapy (e.g., ursodeoxycholic acid, antihistamines, cholestyramine, rifampin, naltrexone). Biliary diversion and eventual liver transplant may be indicated in select patients with refractory disease. If approved, maralixibat will be the first medication with a potential to provide relief of ALGS-related cholestatic pruritus. Maralixibat is also in phase 3 development for progressive familial intrahepatic cholestasis (PFIC)associated pruritus. In addition, Albireo’s oral ASBT inhibitor odevixibat (BylvayTM) was recently approved for PFIC-related pruritus and is in phase 3 trials for ALGS-related pruritus.

FDA APPROVAL TIMELINE September 29, 2021  Breakthrough

 Orphan Drug

 Priority Review

 Rare Pediatric Disease

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$10

$77

$201

$312

$391

The forecast is a projection of total US sales per year including ALGS-related cholestatic pruritus and biliary atresia.

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ONCOLOGY

mobocertinib oral Takeda PROPOSED INDICATIONS

Metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations after platinum-based chemotherapy

CLINICAL OVERVIEW

Mobocertinib is an oral tyrosine kinase inhibitor (TKI) that selectively targets EGFR exon 20 insertion mutations. The phase 1/2 EXCLAIM trial enrolled 114 patients who were previously treated with platinum-based chemotherapy for metastatic NSCLC that harbors EGFR exon 20 insertion mutations. At a median follow-up of 14.2 months, the ORR as assessed by a blinded independent review committee (primary endpoint) was 28% (all partial responses) and disease control rate (DCR) was 78%. The median OS was 24 months, median DOR was 17.5 months, and median PFS was 7.3 months. In addition, among patients who also received prior immunotherapy and prior EGFR TKI therapy, ORR was 25% and 21%, respectively. The most common TEAEs were diarrhea (91%), rash (45%), paronychia (38%), decreased appetite (35%), nausea (34%), dry skin (31%), and vomiting (30%). The most notable grade ≥ 3 TEAE was diarrhea (21%). Approximately 17% of patients discontinued therapy due to adverse events, most notably diarrhea (4%) and nausea (4%). Mobocertinib was administered orally at a once-daily dose of 160 mg.

PLACE IN THERAPY

Lung cancer is the leading cause of death due to cancer in the US. NSCLC accounts for 80% to 85% of all lung cancers, and 2% to 3% of patients have EGFR exon 20 insertion mutations (10% to 14% of all EGFR mutations). EGFR 20 insertion mutations are associated with rapid disease progression and worse prognosis compared to other exon mutations. Most patients with NSCLC and EFGR 20 insertion mutations are nonsmokers, women, and of Asian ethnicity, and have adenocarcinoma histology. First-line treatment of advanced or metastatic NSCLC includes platinum-based chemotherapy with or without immunotherapy. The FDA recently approved the IV-administered EGFR-directed mAb amivantamab-vmjw (Rybrevant™) as treatment in patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations whose disease progressed on or after platinum-based chemotherapy. If approved, mobocertinib will be the second agent indicated to treat patients with NSCLC that harbors exon 20 insertion mutations, and the first-in-class oral TKI available to selectively target this mutation. In non-comparative trials, IV mobocertinib demonstrated a lower ORR and a higher DOR compared to oral amivantamab-vmjw (ORR, 28% versus 40%, respectively; DOR 17.5 versus 11.1 months, respectively). Notably, mobocertinib is associated with a considerable discontinuation rate (17%) due to adverse events. Takeda has initiated a phase 3 trial (EXCLAIM-2) comparing mobocertinib with platinum-based chemotherapy as first-line treatment of NSCLC with exon 20 insertion mutations. The anticipated primary completion date is June 2022. In addition, Takeda has collaborated with Foundation Medicine to create a companion diagnostic to identify eligible patients for mobocertinib treatment who have the EGFR exon 20 insertion mutation.

FDA APPROVAL TIMELINE October 26, 2021

 Breakthrough Therapy

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$27

$88

$147

$204

$230

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

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 seeking Accelerated Approval

ONCOLOGY

oportuzumab monatox intravesical Sesen PROPOSED INDICATIONS

High-risk, Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle-invasive bladder cancer (NMIBC)

CLINICAL OVERVIEW

The antibody-drug conjugate (ADC) oportuzumab monatox is a recombinant fusion protein. It targets the epithelial cell adhesion molecule (EpCAM) found on the surface of tumor cells to deliver the potent protein synthesis inhibitor Pseudomonas Exotoxin A. In the open-label, phase 3 VISTA trial, a total of 133 patients with BCG-unresponsive, high-risk NMIBC were treated with oportuzumab monatox. Patients were evaluated based on the type of disease and time to relapsed or refractory (R/R) disease following the last BCG dose (Cohort 1: carcinoma in situ [CIS] disease and R/R within 6 months; Cohort 2: CIS disease and R/R between 6 and 11 months; Cohort 3: T1 or high-grade Ta disease and R/R within 6 months). Among 82 evaluable patients in Cohort 1, the CRRs (primary endpoint) at 3, 6, 9, and 12 months were 39%, 26%, 20%, and 17%, respectively. The CRRs among the 7 evaluable patients in Cohort 2 at these time points were 57%, 57%, 43%, and 14%, respectively. The median DOR in Cohort 1 was 273 days. In Cohort 3 (n=40), the median time to recurrence was 402 days. The following were estimated across all 133 patients: 29% event-free rate at 12 months, 90% PFS for ≥ 2 years, over 75% cystectomy-free at 2.5 years, and 96% OS of ≥ 2 years. Oportuzumab monatox was well tolerated. The most serious TEAEs were reported in 3 patients and included pyrexia (grade 2), acute kidney injury (grade 3), cholestatic hepatitis (grade 4), and renal failure (grade 5). Oportuzumab monatox was studied as 30 mg via intravesical administration twice weekly for 6 weeks, then once weekly for 6 weeks during induction therapy. Maintenance therapy was 30 mg every other week for up to 104 weeks (24 months).

PLACE IN THERAPY

Bladder cancer is the 6th most common cancer in the US. It is predicted that there will be 83,730 new cases of bladder cancer and 17,200 deaths due to the condition in 2021. The median age at diagnosis is 73 years. Approximately 75% of new cases are NMIBC, and EpCAM is overexpressed in > 98% of high-grade NMIBCs. The standard initial treatment for high-risk NMIBC (Ta [papillary], T1 [submucosal invasive], and Tis [CIS]) is transurethral tumor resection followed by intravesical BCG therapy. Intravesical chemotherapy (e.g., gemcitabine, mitomycin) is an alternative to BCG induction. Up to 75% of cases will develop tumor recurrence after BCG therapy, and 20% will experience disease progression within 5 years. Notably, Merck is the sole supplier of the TICE® BCG strain in the US and a number of other countries, and supply constraints have impacted the availability of BCG live. Radical cystectomy is recommended in patients who do not respond to intravesical BCG therapy. In patients who wish to preserve their bladder or who are ineligible for cystectomy, nonsurgical options are limited to IV pembrolizumab (Keytruda®; administered every 3 or 6 weeks) and intravesical valrubicin (Valstar®; administered weekly for 6 weeks). Both options are indicated in patients with BCG-unresponsive CIS bladder cancer. If approved, oportuzumab monatox will be the first ADC that targets EpCAM for the treatment of BCG-unresponsive NMIBC. Fergene is also seeking approval for its adenoviral vector-based gene therapy, nadofaragene firadenovec (every 3 month intravesical administration), for highrisk BCG-unresponsive NMIBC.

FDA APPROVAL TIMELINE August 18, 2021  Fast Track

 Priority Review

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$65

$117

$171

$232

The forecast is a projection of total US sales per year. 9 | MAGELLANRX.COM

ONCOLOGY

pacritinib oral CTI Biopharma PROPOSED INDICATIONS

Myelofibrosis (MF) with severe thrombocytopenia (platelet count < 50,000/µL)

CLINICAL OVERVIEW

A key driver of MF is overactive Janus kinase (JAK)/signal transducer and activator of transcription (STAT) signaling, leading to bone marrow fibrosis, cytopenias, splenomegaly, and debilitating systemic symptoms. Pacritinib is an oral kinase inhibitor with specificity for Janus kinase 2 (JAK2) and minimal activity on Janus kinase 1 (JAK1). Pacritinib also inhibits interleukin 1 receptor-associated kinase 1 (IRAK1) which may reduce inflammation. Two randomized, open-label, phase 3 trials evaluated the safety and efficacy of pacritinib in patients with MF. The PERSIST-1 trial (n=327) compared pacritinib 400 mg once daily and best available therapy (BAT; excluding JAK inhibitors). PERSIST-2 (n=311) compared pacritinib 400 mg once daily or 200 mg twice daily to BAT (including JAK inhibitor ruxolitinib). Based on pooled data from both trials in the intent-to-treat (ITT) population, in patients with a baseline platelet count ≤ 50,000/µL, the co-primary endpoint of spleen volume reduction (SVR) of ≥ 35% from baseline to week 24 was achieved by 23% of patients treated with pacritinib (doses combined) versus 2% of those given BAT (p=0.0007). In addition, at week 24, the co-primary efficacy endpoint of total symptom score (TSS) reduction by ≥ 50% from baseline was reported in 25% of those in the pacritinib group and 11% in the BAT group (p=0.0894). TSS was based on a 6-symptom score of early satiety, abdominal discomfort, night sweats, pruritus, bone pain, and left-sided pain under the ribs. Further analysis of the data revealed that patients with a low (< 50%) or no JAK2 V617F allele burden had a lower baseline platelet count, more severe anemia, smaller spleen size, and higher probability of primary MF. Pacritinib resulted in a significant SVR response rate of 21% in those with allele burdens ranging from 0% to 25% and a SVR response rate of 15% in those with an allele burden of > 25% to 50%; there was no SVR response in patients with low allele burden treated with BAT. In addition, separate analyses of the 2 pacritinib dosages demonstrated that pacritinib 200 mg twice daily led to significant improvements in SVR and TSS response rates compared to BAT; however, pacritinib 400 mg once daily resulted in only a higher SVR response rate compared to BAT. In general, twice-daily dosing was also better tolerated. The most common TEAE reported with pacritinib in the safety population (n=152) was diarrhea (60.6%). Other TEAEs reported with pacritinib (≥ 20% and at higher rates than BAT) were anemia, nausea, thrombocytopenia, and vomiting. Also of note is that the FDA had placed a clinical hold on the 2 studies due to statistically insignificant safety concerns, including mortality, in patients on pacritinib; however, the hold was lifted after further analysis of the trials and the phase 2 PAC203 trial having showed that there were no drug- or dose-related safety concerns, including cardiac or bleeding events.

PLACE IN THERAPY

Myelofibrosis (MF) is a rare myeloproliferative neoplasm (MPN) characterized by the production of abnormal hematopoietic stem cells and scarring (fibrosis) within the bone marrow. Patients may exhibit anemia, leukopenia/leukocytosis, and thrombocytosis/thrombocytopenia. The estimated prevalence of MF in the US is 13,000 and the median age at diagnosis is 65 years, but it can occur at any age. Most often, MF is idiopathic (primary) in nature (> 50%), but MF can also result from a malignant or hematologic condition (secondary), such as polycythemia vera and essential thrombocythemia. Primary MF may progress slowly, and patients may be asymptomatic for many years; however, some cases may transform into acute myeloblastic leukemia (AML). Approximately 60% of patients with primary MF have a mutation of the JAK2 V617F gene. Mutations in the calreticulin (CALR) gene or the MPL thrombopoietin receptor gene have also been found in 20% to 35% and 5% to 8% of patients with primary MF, respectively. JAK2 V617F and MPL mutations confer a poorer overall survival (OS) (9 years) than the CALR mutation (18 years); however, triple-negative MF has a reported 3-year OS. Pharmacologic management is driven by the mutation detected. In clinical trials, pacritinib demonstrated similar SVR responses across all levels of JAK2 allele burden, whereas BAT (including ruxolitinib) showed no SVR response in patients with low allele burden.

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pacritinib cont. PLACE IN THERAPY cont.

In asymptomatic patients, clinicians may adopt a watch-and-wait approach. In early primary MF, pegylated interferon may reduce bone marrow fibrosis and spleen size in low-risk patients. The oral JAK2 inhibitors ruxolitinib (Jakafi®; JAK1/2 inhibitor; therapy of choice) and fedratinib (Inrebic®; selective JAK2 inhibitor) are approved for the treatment of intermediate- or high-risk primary or secondary MF in patients with platelet count > 50,000/µL. Allogeneic HSCT may be curative, but the associated risk of morbidity and mortality limits its use to patients with advanced disease. Palliative therapy for MF includes androgens, erythropoietin, hydroxyurea, thalidomide, lenalidomide, corticosteroids, radiation therapy, chemotherapy, splenectomy or splenic embolization, and RBC and/or platelet transfusions. If approved, pacritinib will likely compete with ruxolitinib and fedratinib in patients with intermediate- or high-risk MF. Unlike these JAK inhibitors, pacritinib does not inhibit JAK1, which is associated with immune dysfunction, lymphomas, and worsening of cytopenias; therefore, pacritinib has the potential for use in patients with baseline platelet count ≤ 50,000/µL, a population with limited treatment options. Furthermore, fedratinib carries a boxed warning for serious encephalopathy, which has not been reported with pacritinib, to date. The investigational JAK inhibitor momelotinib is also in phase 3 trials for MF.

FDA APPROVAL TIMELINE November 30, 2021  Fast Track

 Orphan Drug

 Priority Review

 seeking Accelerated Approval

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$41

$288

$412

$535

$617

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

11 | MAGELLANRX.COM

INFECTIOUS DISEASE

reltecimod IV Atox Bio PROPOSED INDICATIONS

Necrotizing soft tissue infection (NSTI)-related organ dysfunction/failure

CLINICAL OVERVIEW

An excessive immune response to NSTI can lead to organ failure. Reltecimod is a synthetic peptide that binds to CD28 expressed on T cells, resulting in mitigation of acute inflammation and subsequent systemic organ failure. The double-blind, placebo-controlled, phase 3 ACCUTE study evaluated the safety and efficacy of reltecimod as adjunct to SOC (e.g., surgical intervention, antimicrobial therapy, and critical care support) in 290 patients ≥ 12 years of age with NSTI and organ failure (identified as sequential organ failure assessment [SOFA] ≥ 3). The clinical composite endpoint was defined as: alive at day 28, ≤ 3 debridements, no amputation beyond the first operation, and day 14 modified SOFA ≤ 1 with at least a 3-point SOFA reduction (organ dysfunction resolution). In the per-protocol (PP) population, early administration of reltecimod compared to placebo led to a significant improvement in the primary clinical composite endpoint (54.3% versus 40.3%, respectively; p=0.021); although, the study failed to show a statistically significant improvement in the composite endpoint with reltecimod compared to placebo in the modified ITT (mITT) population (48.6% versus 39.9%, respectively; p=0.135). However, statistically significant resolution of organ dysfunction was achieved with reltecimod compared placebo in both groups (PP: 70.9% versus to 53.4%, respectively [p=0.005]; mITT: 65.1% versus 52.6%, respectively [p=0.041]). In the ACCUTE trial, reltecimod was evaluated as a single 0.5 mg/kg dose administered IV within 6 hours of NSTI diagnosis.

PLACE IN THERAPY

NSTI is a rare, rapidly progressing infection caused by “flesh-eating bacteria.” NSTI results in significant tissue damage and is associated with an inflammatory cytokine response that can lead to organ failure and death. It is estimated that 500 to 1,500 NSTIs occur in the US annually with an associated 35% mortality rate. NSTI requires early aggressive treatment to mitigate complications and improve survival. These serious infections are managed with broad-spectrum antibiotic therapy, surgical debridement, supportive care of organ dysfunction, and adjunctive IV immunoglobulin and hyperbaric oxygen therapies. The development of reltecimod for NSTI is a collaboration between Atox Bio and the US Biomedical Advanced Research and Development Authority (BARDA), which aids in the advancement of therapies for public health medical emergencies. If approved, reltecimod will be the first immune modulator targeting CD28 to treat organ dysfunction and failure that is a consequence of NSTIs. The pivotal ACCUTE trial reported mixed results, showing significant clinical response in the per protocol populations, but not in the mITT population; however, resolution of organ dysfunction was demonstrated in both groups.

FDA APPROVAL TIMELINE September 30, 2021  Fast Track

 Orphan Drug

 seeking Accelerated Approval

FINANCIAL FORECAST (reported in millions)

The financial forecast for reltecimod is not currently available.

12 | MAGELLANRX.COM

ENDOCRINE

somatrogon SC Opko/Pfizer PROPOSED INDICATIONS

Growth hormone deficiency (GHD) in pediatrics

CLINICAL OVERVIEW

Somatrogon is a long-acting human growth hormone (hGH). A randomized, open-label, global, phase 3 trial compared once-weekly somatrogon with once-daily somatropin (Genotropin®) in 224 pre-pubertal, treatment-naïve children (3 to 11 years old) with GHD. After 12 months, the least square mean height velocity (HV) with somatrogon was 10.12 cm/year compared to 9.78 cm/year with somatropin (difference, 0.33; 2-sided 95% CI, -0.39 to 1.05), and somatrogon was considered non-inferior to somatropin. Somatrogon was well tolerated; the TEAEs’ type and incidence were similar to somatropin. After completing the study, children had the option to enroll in a long-term extension trial. Those initially treated with somatropin could switch to somatrogon; approximately 95% of patients switched. The somatrogon study dose was 0.66 mg/kg administered SC once weekly. Somatropin was administered SC as 0.034 mg/kg/day (equal to 0.24 mg/kg/week) using a multi-dose, prefilled pen.

PLACE IN THERAPY

GHD is a rare disorder characterized by a lack of growth hormone (GH) production in the anterior pituitary gland. Causes of childhood-onset GHD may be considered congenital, acquired (e.g., brain trauma, CNS infection, or tumor), or idiopathic. GHD results in growth retardation, short stature, and delays in lengthening of the bones of the extremities. Current treatment for GHD includes daily SC injections of somatropin (various brands). Somatrogon uses glycosylation and C-terminal peptide technology to slow the excretion and extend the half-life of the molecule. Hence, it offers the convenience of once-weekly dosing that the caregiver or patient can administer. If approved, somatrogon may be the second once-weekly hGH for pediatric patients with GHD, following the potential approval of Ascendis’s long-acting hGH lonapegsomatropin that is scheduled to occur by September 25, 2021. Once-weekly lonapegsomatropin also demonstrated non-inferiority to daily somatropin (Genotropin).

FDA APPROVAL TIMELINE October 2021

 Orphan Drug

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$16

$35

$49

$56

The forecast is a projection of total US sales per year and could include pediatric and adult growth hormone deficiency.

13 | MAGELLANRX.COM

ONCOLOGY

tisotumab vedotin IV Seagen/Genmab PROPOSED INDICATIONS

Recurrent or metastatic cervical cancer with disease progression on/after chemotherapy

CLINICAL OVERVIEW

Tisotumab vedotin is an antibody-drug conjugate (ADC) directed to tissue factor (TF), a cell-surface protein expressed on various solid tumors including cervical cancer. TF is associated with tumor growth, angiogenesis, metastasis, and poor prognosis. An ongoing, pivotal, single-arm, phase 2 trial (InnovaTV 204) evaluated monotherapy with tisotumab vedotin in 101 patients with recurrent or metastatic cervical cancer. Enrolled patients were previously treated with SOC first-line doublet chemotherapy with or without bevacizumab and received up to 2 prior lines of therapy for recurrent or metastatic disease. The trial demonstrated an ORR of 24% (95% CI, 15.9% to 33.3%), including complete response in 7% of patients. The median time to response was 1.4 months. After a median follow-up of 10 months, the median DOR was 8.3 months (95% CI, 4.2 to not reached). The study also revealed a median PFS of 4.2 months (95% CI, 3 to 4.4) and OS of 12.1 months (95% CI, 9.6 to 13.9). In general, response to tisotumab vedotin was not impacted by tumor histology or by type of or response to prior therapy. Alopecia, epistaxis, nausea, conjunctivitis, fatigue, and dry eye were reported in ≥ 20% of patients treated. Peripheral neuropathy was also reported (grade 1, 17%; grade 2, 9%; grade 3, 7%) and was managed with dose adjustments. One death was reported as a result of septic shock. Tisotumab vedotin was administered as 2 mg/kg IV every 3 weeks until disease progression or toxicity.

PLACE IN THERAPY

While the incidence and mortality of cervical cancer are on the decline in the US, largely due to effective screening, they remain high in Black, Hispanic/Latino, and Asian populations. It is predicted that nearly 14,500 new cases and approximately 4,300 deaths due to the disease will occur in the US in 2021. The average age at diagnosis is 50 years. Estimated overall 5-year survival is 60% but is < 30% for stage III (spread to vagina, pelvic walls, and/or nearby lymph nodes) and stage IV disease (spread to bladder, rectum, or beyond pelvic area). Following treatment, approximately 35% of patients with invasive cervical cancer develop persistent or recurrent disease. Recurrence typically occurs within 2 years of treatment, particularly in those with stage III-IV disease. The leading risk factor for developing cervical cancer is persistent human papillomavirus (HPV) infection, a vaccine-preventable infection. The majority of cervical cancers are squamous cell carcinomas or adenocarcinomas. Treatment consists of surgery, radiation, and/or chemotherapy. Chemotherapy may be recommended in women with extrapelvic metastases or recurrent disease who are ineligible for radiation therapy or salvage surgery. First-line systemic chemotherapy for recurrent or metastatic disease consists of paclitaxel plus a platinum agent (preferred) or topotecan. Targeted therapy with bevacizumab is typically also included. Monotherapy with pembrolizumab is a second-line option in select patients. Unfortunately, the current therapies for recurrent or metastatic cervical cancer lead to limited response (ORR < 15%) and a median OS of 6 to 9.4 months. If approved, tisotumab vedotin will provide another option for patients with recurrent or metastatic cervical cancer, a hard to treat population. Notably, a phase 2 trial of tisotumab vedotin for cervical cancer reported ocular events and peripheral neuropathy, which will be further evaluated in an ongoing phase 3 trial.

FDA APPROVAL TIMELINE October 8, 2021

 Priority Review

 seeking Accelerated Approval

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$16

$97

$210

$357

$492

The forecast is a projection of total US sales per year for all indications being investigated. 14 | MAGELLANRX.COM

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. 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 FDA is still considering how to implement the nomenclature for previously approved biosimilar products. 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 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. There are currently no FDA-approved interchangeable biosimilars listed in the Purple Book – an FDA database of licensed biological products. Most states have enacted biosimilar substitution laws. Biosimilars are expected to receive full extrapolation for the eligible indications of the reference products without requiring additional trials. Nevertheless, as each biosimilar comes to market, it will likely 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.

15 | MAGELLANRX.COM

To date, a total of 29 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)

Mylan

December 2017

Retacrit (epoetin alfa-epbx)

Pfizer/Hospira

May 2018

Fulphila® (pegfilgrastim-jmdb)

Mylan

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

16 | MAGELLANRX.COM

Commercially Available

  -

 -

    -

     -

  -

 -



Originator Product (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

Hulio® (adalimumab-fkjp)

Mylan

July 2020

Riabni™ (rituximab-arrx)

Amgen

December 2020

Commercially Available

  -



Originator Product (Manufacturer) Remicade (Janssen) Neulasta (Amgen) Humira (Abbvie) Rituxan (Genentech)

* 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®. In June 2020, the FDA approved insulin glargine (Semglee®) by Viatris (Mylan)/Biocon under an abbreviated 505(b)(2) New Drug Application (NDA) pathway; the reference product was Lantus. Semglee is considered a biologic under section 351(a) rather than a 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. However, 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 FDAapproved 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.

17 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

IMMUNOLOGY

adalimumab SC AVT-02 and CHS-1420 are biosimilars to Abbvie’s Humira, tumor necrosis factor alpha (TNFα) 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) September 2021

Coherus (CHS-1420) December 2021

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$17,377

$17,789

$9,608

$6,632

$4,966

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

ONCOLOGY

bevacizumab IV Aybintio, BEVZ92, Bmab-100, BAT1706, 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 (Mylan)/Biocon (Bmab-100) Pending

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$1,097

$806

$688

$600

$538

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

18 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

BLOOD MODIFIER

filgrastim IV, SC Apotex, Amneal, and Tanvex are seeking 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 Apotex (Grastofil) Pending

Amneal January 13, 2022 Tanvex (Tx-01) Pending

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$71

$57

$49

$43

$37

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

ENDOCRINE

insulin aspart SC Viatris (Mylan)/Biocon Viatris (Mylan)/Biocon (MYL-1601D) is seeking biosimilar approval to Novo Nordisk’s Novolog®, a rapid-acting insulin to improve glycemic control in patients with T1DM or T2DM.

FDA APPROVAL TIMELINE July 2021

INTERCHANGEABILITY

Interchangeability status is expected.

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$834

$720

$630

$579

$539

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

19 | MAGELLANRX.COM

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 nonmyeloid malignancies who are receiving myelosuppressive anti-cancer drugs and in patients acutely exposed to myelosuppressive doses of radiation (HSARS).

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

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$1,564

$1,282

$1,077

$928

$798

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

IMMUNOLOGY

ranibizumab intravitreal Samsung Bioepis/Biogen Samsung Bioepis/Biogen is seeking biosimilar approval 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 September to October 2021

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$1,558

$1,534

$1,395

$1,272

$1,159

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

20 | 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

$910

$513

Behavioral health

tirzepatide

Dermatology

Diabetes

adagrasib Oncology

$2,389

$959

tezepelumab

bardoxolone methyl

$886

$1,079

Respiratory

Renal

sotrovimab (VIR-7831)

deucravacitinib Immunology

COVID-19

$1,336

$669

dextromethorphan/ bupropion

NVX-CoV2373 vaccine COVID-19

$2,711

Behavioral health

$924

mirikizumab

elivaldogene tavalentivec (Lenti-D)

Immunology

$583

Neurology/Gene therapy

$35

mavacamten

faricimab

Cardiovascular

$871

lenadogene nolparvovec (GS-010)

Ophthalmology

$616

Ophthalmology/Gene therapy

$150

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 2022. 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

62%

69%

38%

31%

33%

35%

26%

Specialty

18%

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) inolimomab

Elslys

GVHD (acute, steroidrefractory)

Submitted – BLA; Orphan Drug; RTOR

July 2021

insulin aspart (biosimilar to Novo Nordisk’s Novolog)

Viatris (Mylan)/Biocon

T1DM; T2DM interchangeability

Submitted – BLA

July 2021

abrocitinib

Pfizer

Atopic dermatitis (moderate-severe)

Oral

Submitted – NDA; Breakthrough Therapy; Priority Review

Jul-Aug 2021

celecoxib + tramadol

Mundipharma

Pain (moderate-severe)

Oral

Submitted – 505(b)(2) NDA

Jul-Dec 2021

donislecel

Celltrans

T1DM

Submitted – BLA; Orphan Drug

Jul-Dec 2021

lamotrigine

Azurity

Partial seizures

Oral

Submitted – 505(b)(2) NDA

Jul-Dec 2021

roxadustat

AstraZeneca

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

Oral

Submitted – NDA

Jul-Dec 2021

penpulimab

Akeso

Nasopharyngeal cancer

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

07/23/2021

retifanlimab

Incyte

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

Submitted – BLA; Orphan Drug; Priority Review

07/23/2021

sulopenem etzadroxil/ probenecid

Iterum

Uncomplicated UTI (quinolone-resistant)

Oral

Submitted – NDA; Fast Track; Priority Review; QIDP

07/23/2021

tick-borne encephalitis vaccine

Pfizer

Tick-borne encephalitis

Submitted – BLA; Priority Review; Rare Pediatric Disease

August 2021

treosulfan

Medac

Allogenic-HSCT conditioning

IV, Oral

Submitted – NDA; Orphan Drug

August 2021

atogepant

Abbvie/Merck

Migraine prevention

Oral

Submitted – NDA

Aug-Sep 2021

dapivirine ring

International Partnership for Microbicides/Janssen

HIV-1 prevention

Intravaginal

Submitted – NDA

Aug-Dec 2021

tretinoin/benzoyl peroxide

Sol-gel

Acne vulgaris

Topical

Submitted – 505(b)(2) NDA

08/01/2021

topiramate oral solution

Azurity

Partial seizures

Oral

Submitted – 505(b)(2) NDA

08/06/2021

avalglucosidase alfa

Sanofi

Pompe disease

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

08/18/2021

oportuzumab monatox

Sesen

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

Intravesical

Submitted – BLA; Fast 08/18/2021 Track; Priority Review

23 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

vosoritide

Biomarin

Achondroplasia

Submitted – NDA; Orphan Drug; Priority Review

08/20/2021

dextromethorphan/ bupropion

Axsome

MDD

Oral

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

08/22/2021

difelikefalin

Cara

Pruritus (hemodialysisrelated)

Submitted – NDA; Breakthrough Therapy; Priority Review

08/23/2021

adalimumab (biosimilar to Abbvie’s Humira)

Alvotech

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

Submitted – BLA

September 2021

ranibizumab (biosimilar to Genentech’s Lucentis)

Samsung Bioepis/Biogen

Wet AMD

Intravitreal

Submitted – BLA

Sep-Oct 2021

paliperidone palmitate 6-month injectable

Janssen

Schizophrenia

Submitted – NDA

09/02/2021

dihydroergotamine mesylate

Impel Neuropharma

Migraine treatment

Intranasal

Submitted – 505(b)(2) NDA

09/06/2021

belzutifan

Merck

RCC

Oral

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

09/15/2021

ruxolitinib cream

Incyte

Atopic dermatitis (mildmoderate)

Topical

Submitted – NDA; Priority Review

09/21/2021

cantharidin

Verrica

Molluscum contagiosum

Topical

Submitted – NDA

09/23/2021

risperidone long-acting in situ microparticle

Rovi

Schizophrenia

Submitted – 505(b)(2) NDA

09/24/2021

lonapegsomatropin

Ascendis

Growth hormone deficiency (pediatrics)

Submitted – BLA; Orphan Drug

09/25/2021

maralixibat

Mirum

Alagille syndrome-related Oral cholestatic pruritus

Submitted – NDA; 09/29/2021 Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatric Disease

reltecimod

Atox Bio

Necrotizing soft tissue infection (NSTI)-related organ dysfunction/failure (ages ≥ 12 years)

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

09/30/2021

somatrogon

Opko/Pfizer

Growth hormone deficiency (pediatrics)

Submitted – BLA; Orphan Drug

October 2021

RVT-802 (cultured human thymus tissue)

Enzyvant

DiGeorge syndrome (congenital athymia)

Surgical implant

Submitted – BLA; Breakthrough Therapy; Orphan Drug; Rare Pediatric Disease; RMAT

10/08/2021

tisotumab vedotin

Seagen/Genmab

Cervical cancer (recurrent or metastatic, prior chemotherapy)

Submitted – BLA; seeking Accelerated Approval; Priority Review

10/08/2021

bimekizumab

UCB

PSO

Submitted – BLA

10/15/2021

24 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

narsoplimab

Omeros

HSCT-associated thrombotic microangiopathy

IV, SC

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

10/15/2021

sodium oxybate

Avadel

Narcolepsy-related excessive daytime sleepiness and cataplexy

Oral

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

10/15/2021

varenicline

Oyster Point

Dry eye syndrome

Intranasal

Submitted – 505(b)(2) NDA

10/18/2021

ranibizumab intravitreal implant

Genentech

Wet AMD

Intravitreal

Submitted – BLA; Priority Review

10/23/2021

mobocertinib

Takeda

NSCLC (metastatic, Oral EGFR exon 20 insertion mutations, after platinumbase chemotherapy)

Submitted – NDA; seeking Accelerated Approval; Breakthrough Therapy; Orphan Drug; Priority Review

10/26/2021

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

10/30/2021

treprostinil

Liquidia

PAH

Inhaled

Submitted – 505(b)(2) NDA

11/07/2021

naloxone (high-dose)

US Worldmeds

Opioid overdose

Submitted – 505(b)(2) NDA

11/12/2021

ropeginterferon alfa-2b

Pharmaessentia

Polycythemia vera

Submitted – BLA; Orphan Drug

11/13/2021

omidenepag isopropyl

Santen

Glaucoma/ocular hypertension

Ophthalmic

Submitted – NDA

11/19/2021

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 (relapsed/refractory)

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

11/29/2021

episalvan

Amryt

Epidermolysis bullosa

Topical

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

11/30/2021

pacritinib

CTI

Myelofibrosis

Oral

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

11/30/2021

25 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

palovarotene

Ipsen

Fibrodysplasia ossificans progressiva

Oral

Submitted – NDA; Breakthrough Therapy; 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

diazepam

Aquestive

Seizure clusters

Oral transmucosal

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

December 2021

balstilimab

Agenus

Cervical cancer

Submitted – BLA; seeking Accelerated Approval; Fast Track; Priority Review

12/16/2021

efgartigimod

Argenx

Myasthenia gravis

Submitted – BLA; Fast 12/17/2021 Track; Orphan Drug

gefapixant

Merck

Chronic cough

Oral

Submitted – NDA

12/21/2021

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

levoketoconazole

Strongbridge

Cushing’s syndrome

Oral

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

12/31/2021

Pfizer-Biontech COVID-19 vaccine (BNT162b2)

Pfizer/Biontech

COVID-19 prevention (ages ≥ 16 years)

Submitted – BLA; Fast January 2022 Track; Priority Review

mitapivat

Agios

Pyruvate kinase deficiency

Oral

Submitted – NDA; Fast Track; Orphan Drug

Jan-Jun 2022

oteseconazole

Mycovia

Vulvovaginal candidiasis (recurrent)

Oral

Submitted – NDA; Fast Track; QIDP

Jan-Jun 2022

dexmedetomidine

Bioxcel

Bipolar disorderrelated acute agitation; Schizophrenia-related acute agitation

Submitted – 505(b)(2) NDA; Fast Track

01/05/2022

daridorexant

Idorsia

Insomnia

Oral

Submitted – NDA

01/07/2022

tezepelumab

AstraZeneca

Asthma (severe, uncontrolled)

IV, SC

Submitted – BLA; Breakthrough Therapy; Priority Review

01/10/2022

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Amneal

Neutropenia/leukopenia

Submitted – BLA

01/13/2022

budesonide (long-acting)

Calliditas

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

Oral

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

01/15/2022

26 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

maribavir

Takeda

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

Oral

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

01/21/2022

mavacamten

Bristol-Myers Squibb

Obstructive hypertrophic cardiomyopathy

Oral

Submitted – NDA; Breakthrough Therapy; Orphan Drug

01/28/2022

dextroamphetamine

Hisamitsu

ADHD

Transdermal

Submitted – NDA

02/22/2022

bardoxolone methyl

Reata

Alport syndrome-related CKD

Oral

Submitted – NDA; Orphan Drug

02/25/2022

immune globulin IV

Green Cross

Primary humoral immunodeficiency

Submitted – BLA

02/25/2022

sintilimab

Eli Lilly

NSCLC (metastatic, EGFR exon 20 insertion mutations)

Submitted – BLA

March 2022

ublituximab + umbralisib

TG Therapeutics

CLL/SLL

IV, Oral

Submitted – BLA; Fast 03/25/2022 Track; Orphan Drug

udenafil

Dong-A Socio

Single ventricle heart disease after Fontan palliation

Oral

Submitted – NDA; Orphan Drug

03/26/2022

vadadustat

Akebia

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

Oral

Submitted – NDA

03/29/2022

benegrastim

Evive

Neutropenia/leukopenia

Submitted – BLA

03/31/2022

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

dihydroergotamine autoinjector

Amneal

Migraine treatment; Cluster headache treatment

Submitted – 505(b)(2) NDA

Apr-Sep 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

tapinarof

Roivant

PSO (mild-severe)

Topical

Submitted – NDA

05/26/2022

lenacapavir

Gilead

HIV-1 infection (heavily treatment-experienced)

Submitted – NDA; Breakthrough Therapy

06/28/2022

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

bevacizumab (biosimilar to Genentech’s Avastin)

Viatris (Mylan)/Biocon

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

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

filgrastim (biosimilar to Amgen’s Neupogen)

Tanvex

Neutropenia/leukopenia

IV, SC

Submitted – BLA

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

Submitted (Supplementals) insulin glargine (Semglee)

Viatris (Mylan)/Biocon

T1DM; T2DM - for interchangeability

Submitted – sBLA

July 2021

levonorgestrel 52 mg intrauterine device (Mirena®)

Bayer

Contraception

Intrauterine

Submitted – sNDA

July 2021

zoster vaccine recombinant, adjuvanted (Shingrix)

GlaxoSmithKline

Herpes zoster prevention (ages ≥ 18 years at increased risk)

IM, SC

Submitted – sBLA

July 2021

baricitinib (Olumiant®)

Eli Lilly

Atopic dermatitis (moderate-severe)

Oral

Submitted – sNDA

Jul-Aug 2021

mepolizumab (Nucala®)

GlaxoSmithKline

Chronic rhinosinusitis with nasal polyps

Submitted – sBLA

Jul-Aug 2021

tofacitinib (Xeljanz®/ Xeljanz XR®)

Pfizer

Ankylosing Spondylitis

Oral

Submitted – sNDA

Jul-Aug 2021

upadacitinib (Rinvoq™)

Abbvie

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

Oral

Submitted – sNDA; Breakthrough Therapy

Jul-Aug 2021

eptacog beta (Sevenfact®)

Hema

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

Submitted – sBLA

Jul-Sep 2021

tenapanor (Ibsrela®)

Ardelyx

Hyperphosphatemia (dialysis-dependent patients)

Submitted – sNDA

07/29/2021

selexipag (Uptravi®)

Janssen

PAH (temporary switch from oral selexipag)

Submitted – sNDA; Orphan Drug

07/30/2021

sodium oxybate (Xywav™)

Jazz

Idiopathic hypersomnia

Oral

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

08/12/2021

lenvatinib (Lenvima®)

Eisai

RCC (1st-line, advanced disease, in combination with pembrolizumab)

Oral

Submitted – sNDA; Breakthrough Therapy; Priority Review

08/25/2021

pembrolizumab (Keytruda)

Merck

RCC (1st-line, advanced disease, in combination with lenvatinib)

Submitted – sBLA; Breakthrough Therapy; Priority Review

08/26/2021

rivaroxaban (Xarelto®)

Janssen

Peripheral arterial disease (post recent lower-extremity revascularization)

Oral

Submitted – sNDA

08/26/2021

28 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

ivosidenib (Tibsovo®)

Les Laboratoires Servier

Biliary tract cancer (previously treated, isocitrate dehydrogenase 1 [IDH1] mutated)

Oral

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

09/01/2021

lenvatinib (Lenvima)

Eisai

Endometrial carcinoma (advanced, after prior systemic therapy; surgery or radiation ineligible)

Oral

Submitted – sNDA; Priority Review

09/03/2021

nivolumab (Opdivo®)

Bristol-Myers Squibb

Bladder cancer (adjuvant, post surgical resection, high-risk, muscleinvasive)

Submitted – sBLA; Priority Review

09/03/2021

pembrolizumab (Keytruda)

Merck

Endometrial carcinoma (advanced, after prior systemic therapy; surgery or radiation ineligible)

Submitted – sBLA; Priority Review

09/03/2021

pembrolizumab (Keytruda)

Merck

Squamous cell carcinoma (locally advanced)

Submitted – sBLA

09/09/2021

zanubrutinib (Brukinsa®)

Beigene

Marginal zone lymphoma (prior anti-CD20 therapy)

Oral

Submitted – sNDA; Priority Review

09/19/2021

ruxolitinib (Jakafi)

Incyte

GVHD (chronic, steroidrefractory, ages ≥ 12 years)

Oral

Submitted – sNDA; Orphan Drug; Priority Review

09/22/2021

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 hormone receptorpositive, HER2-, early disease)

Oral

Submitted – sNDA

Oct-Dec 2021

brexucabtagene autoleucel Gilead (Tecartus®)

ALL

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

10/01/2021

zanubrutinib (Brukinsa)

Beigene

Waldenstrom macroglobulinemia

Oral

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

10/18/2021

treprostinil DPI (Tyvaso® DPI)

United Therapeutics

PAH; Idiopathic pulmonary fibrosisassociated pulmonary hypertension

Inhaled

Submitted – sNDA; Priority Review

10/19/2021

dupilumab (Dupixent®)

Sanofi

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

Submitted – sBLA

10/21/2021

apremilast (Otezla®)

Amgen

PSO (mild-moderate)

Oral

Submitted – sNDA

12/17/2021

lumateperone (Caplyta )

Intra-Cellular Therapies

Bipolar disorder

IV, Oral

Submitted – sNDA

12/17/2021

cabotegravir/rilpivirine (Cabenuva®)

Viiv

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

Submitted – sNDA

12/24/2021

29 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

vonicog alfa (Vonvendi®)

Takeda

Von Willebrand Disease (prevention of bleeding episodes)

Submitted – sBLA; Orphan Drug

01/28/2022

risankizumab-rzaa (Skyrizi®)

Abbvie

PsA

Submitted – sBLA

February 2022

tecovirimat (Tpoxx®)

Siga

Smallpox treatment (unable to swallow Tpoxx capsule)

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

semaglutide (Ozempic®) 2 mg

Novo Nordisk

T2DM

Submitted – sNDA

03/28/2022

cabozantinib (Cabometyx®)

Exelixis

Thyroid cancer (treatment-experienced, radioactive iodinerefractory, differentiated)

Oral

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

April 2022

copanlisib (Aliqopa®)

Bayer

NHL (indolent, relapsed/ refractory, 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

axicabtagene ciloleucel (Yescarta®)

Gilead

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

Submitted – sBLA; Breakthrough Therapy; Orphan Drug

Pending

infliximab-dyyb (Inflectra)

Celltrion

IBS

IV, SC

Submitted – sBLA

Pending

upadacitinib (Rinvoq)

Abbvie

AS; PsA

Oral

Submitted – sNDA

Pending

Phase 3 (New Drugs) abaloparatide-TD

Radius Health

Osteoporosis/osteopenia

Transdermal

Phase 3 – NDA

TBD

acoramidis

Bridgebio

Transthyretin amyloid cardiomyopathy; 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

aflibercept (biosimilar to Regeneron’s Eylea®)

Samsung Bioepis/Biogen

Diabetic macular edema; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Santo/Formycon

Diabetic macular edema; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Viatris (Mylan)/Janssen

Diabetic macular edema; 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

30 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

apolipoprotein A-I (human)

CSL

Atherosclerosis

Phase 3 – BLA

TBD

arfolitixorin hemisulfate

Isofol

CRC

Phase 3 – NDA

TBD

asciminib

Novartis

Chronic myelogenous leukemia

Oral

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

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

AZD7442

AstraZeneca

COVID-19

IM, IV

Phase 3 – BLA

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

Alport syndrome; 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; Thalassemia

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

TBD

bevacizumab-vikg

Outlook

Diabetic macular edema; Retinal vein occlusionassociated 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

Biliary tract cancer; NSCLC

Phase 3 – BLA; Orphan Drug

TBD

bis-choline tetrathiomolybdate

Alexion

Wilson’s disease

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

brensocatib

Insmed

Bronchiectasis

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

budesonide/albuterol

AstraZeneca

Asthma

Inhaled

Phase 3 – NDA

TBD

bulevirtide

Gilead

Hepatitis D infection

Phase 3 – NDA; Breakthrough Therapy

TBD

C19VAZ COVID vaccine (formerly AZD-1222; ChAdOx1)

AstraZeneca

COVID-19

Phase 3 – BLA

TBD

cannabidiol gel

Zynerba

Fragile X syndrome

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

31 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

capsaicin

Centrexion

Osteoarthritis pain (knee)

Intraarticular

Phase 3 – 505(b)(2) NDA; Fast Track

TBD

casirivimab/imdevimab

Regeneron

COVID-19

IM, IV, SC

Phase 3 – BLA

TBD

CD24Fc

OncoImmune

COVID-19

Phase 3 – BLA

TBD

ceftobiprole medocaril

Basilea

ABSSSI; CAP; HAP

Phase 3 – NDA; Fast Track; QIDP

TBD

ceftriaxone wearable micropump

scPharmaceuticals

Gram+/gram- infection

Phase 3 – NDA

TBD

cipaglucosidase alfa

Amicus

Pompe disease

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

clindamycin phosphate gel

Daré

Bacterial vaginosis

Intravaginal

Phase 3 – NDA; Fast Track; QIDP

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

coronavirus vaccine

Sanofi/GlaxoSmithKline

COVID-19

Phase 3 – BLA

TBD

crovalimab

Genentech

Paroxysmal nocturnal hemoglobinuria

IV, SC

Phase 3 – BLA; Orphan Drug

TBD

dactolisib

Restorbio

COVID-19

Oral

Phase 3 – NDA

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 Pharma Advanced Research

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

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

Oral

Phase 3 – NDA

TBD

dovitinib

Allarity

Breast cancer; RCC

Oral

Phase 3 – NDA

TBD

dust mite immunotherapy tablet

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

elamipretide

Stealth

Barth syndrome

Phase 3 – NDA; Fast Track; Orphan Drug

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

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

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

faricimab

Genentech

Diabetic macular edema; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

fasinumab

Regeneron

Osteoarthritis pain (knee)

Phase 3 – BLA

TBD

favipiravir

Dr. Reddy’s

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

ganaxolone

Marinus

Status epilepticus; CDKL5 deficiency disorderrelated seizures

IV, Oral

Phase 3 – NDA; Orphan Drug; Rare Pediatric Disease

TBD

gantenerumab

Genentech

Alzheimer’s disease

Phase 3 – BLA

TBD

gepotidacin

GlaxoSmithKline

UTI (uncomplicated)

Oral

Phase 3 – NDA; QIDP

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 (Mylan)

Phase 3 – NDA

TBD

human plasminogen

Kedrion

Ligneous conjunctivitis

Ophthalmic

Phase 3 – BLA; Orphan Drug

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

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

Johnson & Johnson COVID-19 (Ad26.COV2-S) vaccine

Janssen

COVID-19

Phase 3 – BLA

TBD

KSI-301

Kodiak

Diabetic macular edema; Retinal vein occlusionassociated macular edema; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

34 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

Lactobacillus reuteri

Infant Bacterial Therapeutics

Necrotizing enterocolitis

Oral

Phase 3 – BLA; Orphan Drug

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

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 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

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

linzagolix

Obseva

Endometriosis; Uterine fibroids

Oral

Phase 3 – NDA

TBD

lorecivivint

Samumed

Osteoarthritis (knee)

Intraarticular

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

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

melphalan

Delcath

Uveal melanoma (hepatic-dominant)

Percutaneous hepatic perfusion

Phase 3 – NDA

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

35 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

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

Moderna COVID-19 vaccine (mRNA-1273)

Moderna

COVID-19

Phase 3 – BLA; Fast Track

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 hydrochloride dihydrate

Elorac

Pruritus

Topical

Phase 3 – 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

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

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

nomacopan

Akari

Hemolytic uremic syndrome; HSCTassociated thrombotic microangiopathy; Paroxysmal nocturnal hemoglobinuria

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

NVX-CoV2373 vaccine

Novavax

COVID-19

Phase 3 – BLA; Fast Track

TBD

odevixibat

Albireo

Alagille syndrome-related Oral cholestatic pruritus

Phase 3 – NDA; Orphan Drug

TBD

olipudase alfa

Sanofi

Niemann-Pick disease

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

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

pacritinib

CTI

COVID-19

Oral

Phase 3 – NDA

TBD

palopegteriparatide

Ascendis

Hypoparathyroidism

Phase 3 – BLA; Orphan Drug

TBD

36 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

pamrevlumab

Fibrogen

DMD; COVID-19; Idiopathic pulmonary fibrosis

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

TBD

perfluorohexyloctane

Bausch Health

Dry eye syndrome

Ophthalmic

Phase 3 – NDA

TBD

pevonedistat

Takeda

Myelodysplastic syndrome

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

Pfizer-Biontech COVID-19 vaccine (BNT162b2)

Pfizer/Biontech

COVID-19 (ages < 16 years); COVID-19 (pregnant individuals)

Phase 3 – BLA; Fast Track

TBD

plinabulin

Beyondspring

NSCLC

Phase 3 – NDA

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)

Coherus

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

ranibizumab (biosimilar to Genentech’s Lucentis)

Stada Arzneimittel/ Bausch Health

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

ranibizumab intravitreal implant

Genentech

Diabetic macular edema; Diabetic retinopathy

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; Pancreatic cancer

Oral

Phase 3 – NDA; Orphan Drug

TBD

reproxalap

Aldeyra

Allergic conjunctivitis; Dry eye syndrome

Ophthalmic

Phase 3 – NDA

TBD

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

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

37 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

rivipansel

Glycomimetics

Sickle cell disease

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

TBD

roflumilast cream

Arcutis

Atopic dermatitis; PSO

Topical

Phase 3 – NDA

TBD

rogaratinib

Bayer

Bladder cancer

Oral

Phase 3 – NDA

TBD

ropeginterferon alfa-2b

Pharmaessentia

Essential thrombocythemia

Phase 3 – BLA

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 nanoparticle vaccine

Novavax

RSV prevention

Phase 3 – BLA; Fast Track

TBD

ruxolitinib cream

Incyte

Vitiligo

Topical

Phase 3 – NDA

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-7831)

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

tebentafusp

Immunocore

Uveal melanoma

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

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 syndrome

Ophthalmic

Phase 3 – BLA

TBD

tiragolumab

Genentech

NSCLC; SCLC

Phase 3 – BLA; Breakthrough Therapy

TBD

tirzepatide

Eli Lilly

T2DM

Phase 3 – NDA

TBD

38 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

tislelizumab

Beigene

Esophageal cancer; 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

toripalimab

Coherus

Nasopharyngeal carcinoma (recurrent or metastatic)

Phase 3 – BLA; Breakthrough Therapy; 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

ublituximab

CLL/SLL; MS

Phase 3 – BLA; Orphan Drug

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

valoctocogene roxaparvovec

Biomarin

Hemophilia A

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

TBD

venglustat

Sanofi

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

Oral

Phase 3 – NDA; Orphan Drug

TBD

VGX-3100 therapeutic vaccine

Inovio

Cervical dysplasia

Phase 3 – BLA; Orphan Drug

TBD

visomitin

Mitotech

Dry eye syndrome

Ophthalmic

Phase 3 – NDA

TBD

vonoprazan + amoxicillin ± clarithromycin

Phathom

Esophagitis; H. pylori infection

Oral

Phase 3 – NDA; QIDP

TBD

wilfactin

LFB

Von Willebrand disease

Phase 3 – BLA; Orphan Drug

TBD

zavegepant

Biohaven

Migraine treatment & prevention; COVID-19

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

39 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

zoliflodacin

Entasis

Gonorrhea

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

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 (Mylan)/Momenta

Hidradenitis suppurativa; Uveitis

Phase 3 – sBLA

TBD

anakinra (Kineret®)

Swedish Orphan Biovitrum

COVID-19

Phase 3 – sBLA

TBD

atezolizumab (Tecentriq®)

Genentech

NSCLC

Phase 3 – sBLA

TBD

baricitinib (Olumiant)

Eli Lilly

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

Oral

Phase 3 – sNDA; Breakthrough Therapy; Fast Track

TBD

benralizumab (Fasenra®)

AstraZeneca

ANCA-associated vasculitis; Bullous pemphigoid; Esophagitis; Nasal polyposis

Phase 3 – sBLA; Orphan Drug

TBD

cabotegravir (Vocabria)

Viiv

HIV-1 infection prevention (PrEP)

Oral

Phase 3 – sNDA; Breakthrough Therapy

TBD

cariprazine (Vraylar®)

Allergan

MDD

Oral

Phase 3 – sNDA

TBD

dupilumab (Dupixent)

Sanofi

Eosinophilic esophagitis; COPD; Pruritus; Urticaria

Phase 3 – sBLA; Breakthrough Therapy; Orphan Drug

TBD

empagliflozin (Jardiance®)

Boehringer Ingelheim

Diabetic nephropathy

Oral

Phase 3 – sNDA

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 intravenous (human) 10% (Octagam®)

Octapharma

COVID-19; Dermatomyositis

Phase 3 – sBLA; Orphan Drug

TBD

L-lactic acid/citric acid/ potassium bitartrate

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

40 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

omalizumab (Xolair®)

Genentech

Food allergies

Phase 3 – sBLA; Breakthrough Therapy

TBD

patisiran (Onpattro®)

Alnylam

Transthyretin amyloid cardiomyopathy (wild type or hereditary)

Phase 3 – sNDA; Orphan Drug

TBD

pegylated liposomal irinotecan (Onivyde®)

Ipsen

SCLC

Phase 3 – sNDA; Fast Track

TBD

ravulizumab-cwvz (Ultomiris)

Alexion

ALS; COVID-19; HSCTTMA; Myasthenia gravis; Neuromyelitis optica (Devic’s syndrome)

IV, SC

Phase 3 – sBLA

TBD

relugolix/estradiol/ norethindrone (Myfembree®)

Myovant

Endometriosis

Oral

Phase 3 – sNDA

TBD

risankizumab-rzaa (Skyrizi)

Abbvie

CD; UC

Phase 3 – sBLA; Orphan Drug

TBD

rivaroxaban (Xarelto)

Janssen

COVID-19

Oral

Phase 3 – sNDA

TBD

romiplostim (Nplate )

Amgen

Chemotherapy-induced thrombocytopenia

Phase 3 – sBLA; Orphan Drug

TBD

secukinumab (Cosentyx®)

Novartis

Hidradenitis suppurativa

Phase 3 – sBLA

TBD

sodium hyaluronate/ triamcinolone hexacetonide (Cingal®)

Anika

Osteoarthritis (knee)

Intraarticular

Phase 3 – sNDA

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; UC

Oral

Phase 3 – sNDA; Orphan Drug

TBD

Complete Response Letter (CRL)/Withdrawn Drugs NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

arimoclomol

Orphazyme

Niemann-Pick disease

Oral

CRL

TBD

dehydrated alcohol

Eton

Methanol poinsoning

CRL

TBD

natalizumab (Tysabri)

Biogen

MS (relapsing)

CRL

TBD

pegunigalsidase alfa

Chiesi

Fabry’s disease

CRL

TBD

pineapple proteolytic enzymes extract

Vericel

Burn Injury

Topical

CRL

TBD

Provention Bio

T1DM (delay/prevention)

CRL

TBD

teriflunomide (Aubagio )

Sanofi

MS (pediatrics, relapsing)

Oral

CRL

TBD

tralokinumab

AstraZeneca

Atopic dermatitis (moderate-severe)

CRL

TBD

tramadol

Fortress

Postsurgical pain

CRL

TBD

zonisamide oral suspension

Azurity

Partial seizures

Oral

CRL

TBD

teplizumab ®

41 | MAGELLANRX.COM

GLOSSARY 6MWT 6 Minute Walking Test

CAP Community Acquired Pneumonia

ABSSSI Acute Bacterial Skin and Skin Structure Infection

CAR T Chimeric Antigen Receptor T Cell

ACEI Angiotensin-Converting Enzyme Inhibitor ACR20 American College of Rheumatology 20% Improvement

CD Crohn's Disease CDC Centers for Disease Control and Prevention CF Cystic Fibrosis

ACR50 American College of Rheumatology 50% Improvement

CHF Congestive Heart Failure

ACR70 American College of Rheumatology 70% Improvement

CKD Chronic Kidney Disease

ADC Antibody-Drug Conjugate ADHD Attention Deficit Hyperactivity Disorder ADL Activities of Daily Living AED Anti-Epileptic Drug ALK Anaplastic Lymphoma Kinase ALL Acute Lymphoblastic Leukemia ALS Amyotrophic Lateral Sclerosis ALT Alanine Transaminase AMD Age-Related Macular Degeneration AML Acute Myeloid Leukemia

CI Confidence Interval

CLL Chronic Lymphocytic Leukemia CNS Central Nervous System COPD Chronic Obstructive Pulmonary Disease COVID-19 Coronavirus Disease 2019 CRC Colorectal Cancer CRL Complete Response Letter CRR Complete Response Rate CSF Colony Stimulating Factor CV Cardiovascular CVD Cardiovascular Disease

ANCA Antineutrophil Cytoplasmic Antibodies

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

ANDA Abbreviated New Drug Application

DEA Drug Enforcement Administration

ARB Angiotensin II Receptor Blocker

DLBCL Diffuse Large B Cell Lymphoma

ARNI Angiotensin Receptor II Blocker – Neprilysin Inhibitor

DMARD Disease Modifying Antirheumatic Drug

ART Antiretroviral Therapy ARV Antiretroviral AS Ankylosing Spondylitis ASCVD Atherosclerotic Cardiovascular Disease AST Aspartate Aminotransferase BCVA Best Corrected Visual Acuity BLA Biologics License Application

DMD Duchenne Muscular Dystrophy DMT Disease Modifying Therapy DNA Deoxyribonucleic Acid DOR Duration of Response DPI Dry Powder for Inhalation DPP-4 Dipeptidyl Peptidase 4 DR Delayed-Release

BMI Body Mass Index

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

BsUFA Biosimilar User Fee Act

ECOG Eastern Cooperative Oncology Group

CABP Community Acquired Bacterial Pneumonia

EDSS Expanded Disability Status Scale

42 | MAGELLANRX.COM

GLOSSARY continued eGFR estimated Glomerular Filtration Rate

IGA Investigator's Global Assessment

ER Extended-Release

IM Intramuscular

ESRD End-Stage Renal Disease

IRB Internal Review Board

FDA Food and Drug Administration

ITP Immune Thrombocytopenic Purpura

FH Familial Hypercholesterolemia

ITT Intent-To-Treat

FLT3 FMS-Like Tyrosine Kinase-3

IV Intravenous

G-CSF Granulocyte Colony Stimulating Factor

JIA Juvenile Idiopathic Arthritis

GI Gastrointestinal

LDL Low-Density Lipoprotein

GIST Gastrointestinal Stromal Tumor

LDL-C Low-Density Lipoprotein Cholesterol

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

mAb Monoclonal Antibody

GM-CSF Granulocyte-Macrophage Colony Stimulating Factor

MACE Major Adverse Cardiovascular Events

GVHD Graft Versus Host Disease H Half HAART Highly Active Antiretroviral Therapy HAM-D Hamilton Depression Rating Scale HAP Healthcare-Associated Pneumonia Hb Hemoglobin HbA1c Hemoglobin A1c HCC Hepatocellular Carcinoma HCP Healthcare Professional HCV Hepatitis C Virus HER Human Epidermal Growth Factor Receptor

MADRS Montgomery – Åsberg Depression Rating Scale MAOI Monoamine Oxidase Inhibitor MDD Major Depressive Disorder MDI Metered Dose Inhaler MRI Magnetic Resonance Imaging MRSA Methicillin-Resistant Staphylococcus Aureus MS Multiple Sclerosis N/A Not Applicable NASH Non-Alcoholic Steatohepatitis NDA New Drug Application NHL Non-Hodgkin Lymphoma

HER2 Human Epidermal Growth Factor Receptor 2

NIAID National Institute of Allergy and Infectious Diseases

HFA Hydrofluoroalkane

NSAID Non-Steroidal Anti-Inflammatory Drug

HIT Heparin Induced Thrombocytopenia

NSCLC Non-Small Cell Lung Cancer

HIV Human Immunodeficiency Virus

ODT Orally Disintegrating Tablet

HIV-1 Human Immunodeficiency Virus-1

OR Odds Ratio

HR Hazard Ratio

ORR Overall/Objective Response Rate

HSCT Hematopoietic Stem Cell Transplant

OS Overall Survival

HTN Hypertension

OTC Over-the-Counter

IBS Irritable Bowel Syndrome

PAH Pulmonary Arterial Hypertension

IBS-C Irritable Bowel Syndrome, Constipation Predominant

PARP Poly(ADP-ribose) polymerase

43 | MAGELLANRX.COM

PASI Psoriasis Area and Severity Index

GLOSSARY continued PASI 50 Psoriasis Area and Severity Index 50%

SCT Stem Cell Transplant

PASI 70 Psoriasis Area and Severity Index 70%

SGLT2 Sodium-Glucose Co-Transporter 2

PASI 90 Psoriasis Area and Severity Index 90%

SL Sublingual

PASI 100 Psoriasis Area and Severity Index 100%

SLE Systemic Lupus Erythematosus

PCI Percutaneous Coronary Intervention

SLL Small Lymphocytic Lymphoma

PCSK9 Proprotein Convertase Subtilisin Kexin 9

sNDA supplemental New Drug Application

PD-1 Programmed Death Protein 1

SNRI Serotonin and Norepinephrine Reuptake Inhibitor

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

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

PTSD Post-Traumatic Stress Disorder

TBD To Be Determined

Q Quarter

TEAE Treatment-Emergent Adverse Event

QIDP Qualified Infectious Diseases Product

TNBC Triple Negative Breast Cancer

QOL Quality of Life

TNF Tumor Necrosis Factor

R/R Relapsed or Refractory

TNFα Tumor Necrosis Factor-alpha

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

UA Unstable Angina

RA Rheumatoid Arthritis 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 SC Subcutaneous SCCHN Squamous Cell Cancer of the Head and Neck SCLC Small Cell Lung Cancer 44 | MAGELLANRX.COM

UC Ulcerative Colitis US United States UTI Urinary Tract Infection VAS Visual Analog Scale VEGF Vascular Endothelial Growth Factor VTE Venous Thromboembolism WBC White Blood Cell WHO World Health Organization XR Extended-Release