MRx Pipeline - April 2022 by Prime/Magellan Rx

MR x PIPELINE MRx AAVIEW & TRADITIONAL TRADITIONALDRUGS DRUGS VIEWINTO INTO UPCOMING UPCOMING SPECIALTY SPECIALTY &

APRIL 2022 JANUARY 2022

Table of CONTENTS

EDITORIAL STAFF Maryam Tabatabai, PharmD Editor-in-Chief Vice President, Clinical Information Carole Kerzic, RPh Executive Editor Drug Information Pharmacist

EDITOR-IN-CHIEF'S MESSAGE

Consultant Panel

PIPELINE DEEP DIVE

Lara Frick, PharmD, BCPS, BCPP Drug Information Pharmacist

KEEP ON YOUR RADAR

PIPELINE DRUG LIST

GLOSSARY

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Michelle Booth, PharmD Director, Specialty Clinical Solutions

Robert Greer, RPh, BCOP Vice President, Clinical Strategy and Programs Katie Lockhart Manager, Forecasting and Pharmacoeconomics Brian MacDonald, PharmD Director, Specialty Clinical Strategy 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 2026. These figures are not specific to a particular commercial or government line of business; rather, they look at forecasted total US sales. Depending on a variety of factors, including the therapeutic categories, eventual FDA-approved indications, populations within the plan, and other indices, the financial impact could vary by different lines of business.

REFLECTION

Despite the pandemic’s ongoing challenges, in 2021, the US FDA approved 50 novel drugs. Thus far in 2022, the agency has approved 11 novel drugs. While it is still early in the year, this is about 40% fewer approvals compared to the same time last year. Notably, most of the novel drug approvals so far in 2022 use at least 1 of the FDA’s expedited approval methods and the majority are designated as Orphan Drugs. While numbers do not tell the entire story, they do represent significant innovation in patient care and advance public health for the American public.

ON THE HORIZON

As we look ahead, there is a continued trend towards the approval of specialty medications and drugs for rare and ultra rare conditions, with 68% and 36% of approvals expected, respectively, for agents with applications submitted to the FDA. There are 2 agents seeking FDA’s Accelerated Approval, which allows for earlier drug approval for serious conditions that fill an unlet need based on a surrogate endpoint reasonably likely to predict a clinical benefit. New treatment modalities using gene therapy and the growth of biosimilars including interchangeable biosimilars are expected. A new COVID-19 vaccine using a more traditional platform is seeking authorization. Other noteworthy pipeline trends to watch include the development of complex therapies, oncology, immunology, immunotherapy, RSV, new options for Alzheimer’s disease, and therapeutic options for rare hereditary diseases. Moreover, sprouting products for behavioral health including women’s mental health and therapeutic psychedelics await on the horizon. The drug pipeline ecosphere will continue to evolve as it faces challenges and successes. Innovative agents that show positive results without compromising patient safety and access offer true therapeutic advances and hold the promise to alter the treatment paradigm. Maryam Tabatabai, PharmD Editor-in-Chief, MRx Pipeline

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

SPECIALTY

PRIORITY REVIEW

BREAKTHROUGH THERAPY

92%

15%

BIOSIMILAR

ORPHAN DRUG

69%

23%

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

INFECTIOUS DISEASE

bulevirtide SC Gilead PROPOSED INDICATIONS

Treatment of hepatitis delta virus (HDV) infection in adults with compensated liver disease

CLINICAL OVERVIEW

Bulevirtide is an entry inhibitor that blocks the access of HDV into hepatocytes. The ongoing, open-label, phase 3 trial (MYR301) is evaluating the safety and efficacy of bulevirtide in 150 patients co-infected with HBV and HDV. Patients were randomized 1:1:1 to monotherapy with bulevirtide 2 mg (BLV2mg), bulevirtide 10 mg (BLV10mg), or no treatment (delayed treatment). The primary endpoint was a combined response, defined as an undetectable HDV RNA or an HDV RNA decrease by ≥ 2 log10 IU/mL plus ALT normalization. At the 24-week planned interim analysis, the primary endpoint was achieved by 36.7% and 28% of patients on BLV2mg and BLV10mg, respectively, compared to 0 patients who received no treatment (p<0.0001 for both active groups versus no treatment). In addition, 55.1% and 68% of patients who received BLV2mg and BLV10mg, respectively, achieved an HDV RNA decrease by ≥ 2 log10 IU/mL compared to 3.8% assigned to no treatment (p<0.0001 for both). Furthermore, ALT normalization was observed in 53.1% and 38% of patients in the BLV2mg and BLV10mg groups, respectively, compared to 5.9% in the no treatment group (p<0.0001 for both). Bulevirtide monotherapy was generally well tolerated. No serious TEAEs were reported. An ongoing, randomized, open-label, phase 2b study (MYR204) is also evaluating BLV2mg and BLV10mg combined with pegylated interferon alfa (pINF) in 175 patients with chronic HBV/HDV co-infection. Based on an interim analysis at 24 weeks, 24% and 34% of patients in the BLV2mg/pINF and BLV10mg/pINF groups achieved the primary endpoint of undetectable HDV RNA. This was compared to 12.5% who received only pINF and 4% who received BLV10mg. In this study, the BLV monotherapy group (BLV10mg) achieved the highest rate of ALT normalization. Overall, bulevirtide plus pINF was well tolerated. Final results of another phase 2b trial (MYR203; n=60) demonstrated higher off-treatment HDV RNA suppression rates measured 24 weeks after discontinuation of therapy with BLV/pINF compared to pINF alone. Bulevirtide was administered once daily via SC injection.

PLACE IN THERAPY

HDV can be an acute or chronic infection. It requires the presence of HBV for its replication; therefore, patients with HDV will always have HBV co-infection. Populations at highest risk for HBV/HDV co-infection include indigenous people, hemodialysis patients, and injection drug users. Worldwide, HDV infects approximately 5% of those with chronic HBV infection. HDV infection in the US is uncommon, with most cases due to migration or travel from countries with high rates of HDV. HBV/HDV co-infection is the most severe type of chronic viral hepatitis, since it progresses to hepatocellular carcinoma and death faster than HBV infection alone. While global HBV immunization has led to a decrease in HDV infection, there is no approved treatment for HDV infection. Off-label use of interferon alpha or pegylated interferon alpha for 48 weeks showed a low sustained virologic response (25%). Bulevirtide is a first-in-class entry inhibitor. If approved, it will be the first medication indicated for the treatment of hepatitis D. Clinical trials demonstrated that bulevirtide with and without pegylated interferon alfa is safe and effective for treating HDV infection. Eiger is also in late-stage development of lonafarnib/ ritonavir (± pINF alfa) and pegylated interferon lambda to treat HDV infection.

FDA APPROVAL TIMELINE July to November 2022

 Breakthrough Therapy

 Orphan Drug

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$49

$100

$166

$215

$249

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IMMUNOLOGY

deucravacitinib oral Bristol-Myers Squibb PROPOSED INDICATIONS

Moderate to severe plaque psoriasis (PSO)

CLINICAL OVERVIEW

Deucravacitinib is an oral, selective tyrosine kinase 2 (TYK2) inhibitor. It blocks the signaling of cytokines IL-23, IL-12, and type 1 interferon, which are involved in autoimmune diseases. Two randomized, double-blind, placebo-controlled, active-comparator-controlled, phase 3 trials, POETYK PSO-1 and POETYK PSO-2, support FDA submission of deucravacitinib. The trials enrolled a total of 1,686 adults with moderate to severe PSO. The active comparator in each trial was oral apremilast (30 mg twice daily). In the PSO-1 and PSO-2 trials, significantly more patients assigned to deucravacitinib (58.7% and 53.6%, respectively) achieved PASI 75 response at week 16 compared to those assigned to apremilast (35.1% and 40.2%, respectively; p<0.0001 in both trials for deucravacitinib versus apremilast). In addition, significantly more patients in both trials treated with deucravacitinib (53.6% and 50.3%, respectively) achieved a static PGA of 0 (clear) or 1 (almost clear) compared to those treated with apremilast (32.1% and 34.3%, respectively; p>0.0001 in both trials for deucravacitinib versus apremilast). Significant differences in PASI 75 and PGA between the medications continued through week 24. Through 52 weeks, deucravacitinib showed a durable PASI 75 response and was generally well tolerated. The most common TEAEs reported with the product were nasopharyngitis and upper respiratory tract infection. Headache, diarrhea, and nausea were reported less often with deucravacitinib than with apremilast. The incidence of malignancy, MACE, venous thromboembolism, and serious infections with deucravacitinib were low and similar to apremilast. Deucravacitinib was administered orally as 6 mg once daily.

PLACE IN THERAPY

Psoriasis is a chronic, multisystem, immune-mediated, inflammatory disease involving the skin and joints. It affects an estimated 8 million people in the US. PSO can present at any age, but onset is highest between 20 to 30 years and 50 to 60 years. Nearly 25% of people with PSO have moderate to severe cases, and approximately 60% report that the condition negatively impacts their daily life. Treatment is based on the severity of the condition. Targeted immunomodulators are indicated to treat moderate to severe PSO after the failure of topical therapy alone when phototherapy is not available. These include injectable monoclonal antibodies that reduce pathogenic cytokines levels (e.g., TNF-α, IL-17, and IL23), and the oral phosphodiesterase 4 (PDE4) inhibitor apremilast (Otezla®) that reduces proinflammatory mediator responses. Deucravacitinib is a first-in-class, oral, selective TYK2 inhibitor. If approved, it may compete with oral apremilast (Otezla) in the PSO space. In clinical trials, deucravacitinib demonstrated significant improvement in skin clearance and symptom burden and a more favorable safety profile and tolerability than apremilast. This, along with its convenient once-daily oral dosing schedule, could make deucravacitinib the desirable oral option for treating moderate to severe PSO. Deucravacitinib is also in late-stage development for PsA, CD, UC, and lupus.

FDA APPROVAL TIMELINE September 10, 2022

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$51

$295

$596

$905

$1,218

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WOMEN’S HEALTH

linzagolix oral Obseva PROPOSED INDICATIONS

Uterine fibroid (UF)-related heavy menstrual bleeding

CLINICAL OVERVIEW

Linzagolix is an oral gonadotropin-releasing hormone (GnRH) receptor antagonist. The safety and efficacy of linzagolix was evaluated in 2 randomized, double-blind, parallel-group, placebocontrolled, phase 3 trials, PRIMROSE-1 (n=574) and PRIMROSE-2 (n=535). Patients with UFs were randomized to once-daily oral linzagolix 100 mg or 200 mg alone and in combination with hormonal add-back therapy (ABT; estradiol 1 mg plus norethisterone acetate 0.5 mg). A responder was defined as a patient with menstrual blood loss (MBL) volume of ≤ 80 mL and a ≥ 50% reduction from baseline in MBL. Pooled data demonstrated that the response rate at 24 weeks was 56.6% with linzagolix 100 mg monotherapy and 84.7% with linzagolix 200 mg + ABT compared to 32.2% with placebo. Response rates were maintained at 52 weeks, reporting 56.4% with linzagolix 100 mg monotherapy and 89.3% with linzagolix 200 mg + ABT. Both trials also reported improvements in pain, amenorrhea, anemia, and QOL. The most common TEAEs were hot flushes, headache, and anemia. In PRIMROSE 2, at 52 weeks, similar mean decreases in lumbar spine bone mineral desnity (BMD)were seen with linzagolix 100 mg monotherapy (-2.4%) and linzagolix 200 mg + ABT (-2%). Notably, patients did not receive vitamin D or calcium during the trials.

PLACE IN THERAPY

Uterine fibroids (UFs) are the most common benign gynecologic tumors. They affect 50% to 60% of premenopausal women and are more common in African American women. UFs are an overgrowth of connective tissue and smooth muscle in and around the uterus. While most cases are asymptomatic, UFs can lead to heavy menstrual bleeding, pelvic pressure and pain, back pain, and infertility. Surgical treatment approaches include myomectomy, hysterectomy, and uterine artery embolization. Medical therapies for symptomatic management of UFs include oral contraceptives, progestin-releasing intrauterine devices (IUDs), NSAIDs, mifepristone, GnRH agonists, and GnRH receptor antagonists. Notably, low doses of estrogen and progestin are added (“add-back therapy”) to mitigate bone loss and vasomotor symptoms associated with GnRH-targeted therapies. In addition, tranexamic acid (Lysteda®) is approved to treat cyclic heavy menstrual bleeding. It is taken for up to 5 days during monthly menstruation. If approved, linzagolix will be the third oral combination of a GnRH receptor antagonist with estradiol and norethindrone. It will compete with once-daily relugolix/estradiol/norethindrone (Myfembree®) and twicedaily elagolix/estradiol/norethindrone (Oriahnn®) for the treatment of heavy menstrual bleeding associated with UFs. Despite the inclusion of estradiol and norethindrone, reductions in BMD have occurred with Myfembree and Oriahnn; therefore, treatment with these products is limited to 24 months. BMD losses with linzagolix 200 mg + ABT appear similar to or greater than those reported with Myfembree and Oriahnn. It remains to be seen whether the FDA will recommend a limited duration of therapy for linzagolix. Linzagolix is in phase 3 trials for the treatment of endometriosis-associated pain. Myfembree has been submitted to the FDA for this indication, with an FDA decision expected in May 2022. Elagolix monotherapy (Orilissa®) is currently approved to treat endometriosis.

FDA APPROVAL TIMELINE September 13, 2022

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$20

$41

$102

$176

$271

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METABOLIC

olipudase alfa IV Sanofi PROPOSED INDICATIONS

Treatment of non-central nervous system (CNS) manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease (NPD) types A, B, and A/B

CLINICAL OVERVIEW

Olipudase alfa is a recombinant human acid sphingomyelinase enzyme. The safety and efficacy of olipudase alfa were evaluated in the multinational, randomized, double-blind, placebo-controlled, phase 2/3 ASCEND trial in 36 adults with ASMD. At week 52, olipudase alfa demonstrated significant improvement in lung function based on the co-primary endpoint measure of percent predicted diffusing capacity of carbon monoxide (DLco) compared to placebo (22% versus 3%, respectively; p=0.0004). Olipudase alfa also significantly reduced spleen volume compared to placebo (change, -39.5% versus +0.5%, respectively; assessed as percent change from baseline in multiples of normal [MN]; p<0.0001). However, in the US, a combined endpoint, called the splenomegaly related score (SRS), was used that included the spleen volume and a patient-reported symptom measure. Consequently, this combination endpoint did not demonstrate a significant improvement in the SRS score with olipudase alfa compared to placebo (SRS reduction, 8 versus 9.3 points, respectively; p=0.7). Headache, nasopharyngitis, upper respiratory tract infection, cough, and arthralgia were reported more often in adults treated with olipudase alfa than with placebo. In addition, the open-label, phase 2 ASCEND-Peds trial evaluated olipudase alfa in pediatric patients with ASMD. The study enrolled 20 patients ages 1 to 17 years, all of whom received olipudase alfa. At week 52, DLco was 33% (among 9 evaluable patients; p=0.0053) and mean spleen volume decreased by 49% (assessed by mean MN). The most common TEAEs in pediatric patients were considered to be infusionassociated reactions (e.g., urticaria, pyrexia, vomiting). Open-label extension trials reported sustained benefit of olipudase alfa for up to 6.5 years in adult patients and for 2 years in pediatric patients. Olipudase alfa was administered via IV infusion every 2 weeks and titrated to a maintenance dose of 3 mg/kg.

PLACE IN THERAPY

ASMD, or NPD types A, B, and A/B, is an autosomal recessive inherited condition that affects 1 in 250,000 individuals. It is caused by the mutations in the SMPD1 gene, resulting in a lack of acid sphingomyelinase, an enzyme involved the breakdown of the fat sphingomyelin. Consequently, sphingomyelin accumulates in the spleen, liver, lungs, bone marrow, and brain, causing damage to these organs. Manifestations of NPD vary widely. NPD type A appears during infancy and is characterized by hepatosplenomegaly, recurrent respiratory infections, failure to thrive, and nervous system deterioration. Children with NPD type A often do not live past early childhood. NPD type B is a milder form of the disease with onset ranging from infancy to adulthood. Patients may have little or no neurological symptoms; however, hepatosplenomegaly, lung impairment, osteopenia, dyslipidemia, nystagmus, unsteady gait, and intellectual disability may be present. Intermediate forms of NPD are also reported. Current therapy for ASMD consists of symptom management and may require coordination between pediatricians, neurologists, hepatologists, ophthalmologists, and other healthcare professionals. If approved, olipudase alfa will be the first treatment indicated for individuals with ASMD (NPD types A, B, and A/B). In clinical trials, long-term safety, improved lung function, and decreased spleen volume were demonstrated.

FDA APPROVAL TIMELINE July 3, 2022

 Breakthrough Therapy

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$18

$40

$64

$85

$103

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ONCOLOGY

poziotinib oral Spectrum PROPOSED INDICATIONS

Locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring HER2 exon 20 insertion mutations in previously treated patients

CLINICAL OVERVIEW

Poziotinib is an oral, irreversible tyrosine kinase inhibitor (TKI) that targets EGFR and HER2 with exon 20 insertion mutations. Submission to the FDA was based on the ongoing, open-label, phase 2, multi-cohort ZENITH20 trial that assessed poziotinib in treatment-experienced and -naïve patients with NSCLC. All patients in the study received poziotinib. Cohort 2 enrolled 90 treatment-experienced patients with HER2 exon 20 insertion mutations exclusively, including heavily-pretreated patients (67% failed ≥ 2 prior therapies). Based on a median follow-up of 8.3 months, poziotinib led to an ORR of 27.8% (primary endpoint), disease control rate (DCR) of 70%, median DOR of 5.1 months, and median PFS of 5.5 months. A total of 14 patients in this cohort had stable CNS metastases at baseline. Of these 14 patients, at a median follow-up of 8.3 months, 1 patient achieved a complete CNS response, and the remaining patients (92.9%) experienced stable disease or better. The safety profile of poziotinib was generally consistent with the TKI class. Grade ≥ 3 adverse events included rash (30%) and diarrhea (26%). In Cohort 2, patients received poziotinib 16 mg orally once daily.

PLACE IN THERAPY

Lung cancer is the leading cause of cancer death in the US. It is estimated that 236,740 new cases of deaths and 130,180 deaths due to the condition will occur in the US in 2022. Among patients with NSCLC, an estimated 2% to 4% harbor HER2 mutations, of which 90% exhibit exon 20 insertions. HER2 mutation with exon 20 insertion is associated with resistance to available treatments. The National Comprehensive Cancer Network (NCCN) recommends off-label use of the HER2-targeting antibody-drug conjugates (ADCs) ado-trastuzumab emtansine (Kadcyla®) and fam-trastuzumab deruxtecannxki (Enhertu®) in patients with HER2-positive NSCLC. The NCCN recommendation is based on phase 2 trials that showed ado-trastuzumab emtansine led to a 44% partial response rate and fam-trastuzumab deruxtecan-nxki led to a 55% ORR in patients with HER2-positive NSCLC. Trastuzumab (Herceptin®) is not recommended due to a lower efficacy in HER2-positive tumors compared to the ADCs. Dacomitinib (Vizimpro®) and mobocertinib (Exkivity®) are oral TKIs with activity against HER2; however, neither is indicated or recommended for HER2-positive NSCLC. If approved, poziotinib will be the first medication indicated to treat NSCLC with HER2 exon 20 mutations in treatment-experienced patients. Notably, it failed to show significant efficacy in treating NSCLC with EGFR exon 20 mutation (ZENITH20 Cohorts 1 and 3). Poziotinib is also being studied for first-line treatment of NSCLC harboring HER2 mutations (ZENITH20 Cohort 4) and for breast cancer treament.

FDA APPROVAL TIMELINE November 24, 2022  Fast Track

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$27

$90

$144

$188

$224

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NEUROLOGY

sodium phenylbutyrate/taurursodiol oral Amylyx PROPOSED INDICATIONS

Amyotrophic lateral sclerosis (ALS)

CLINICAL OVERVIEW

Sodium phenylbutyrate is a chaperone agent designed to reduce the unfolded protein response (UPR). Taurursodiol is a BCL2-associated X protein (Bax) inhibitor. The agents are being developed as an oral fixeddose combination to reduce neuronal cell death and dysfunction associated with ALS. Sodium phenylbutyrate/taurursodiol (PB/TURSO) was evaluated in the randomized, double-blind, placebocontrolled, phase 2 CENTAUR trial in 137 patients with ALS. The primary outcome was the rate of decline in the total score on the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R; range, 0 to 48, with higher scores indicating better function). At 24 weeks in the mITT group, the least squares ALSFRS-R score was 29 points with the active drug compared to 26.7 points with placebo (difference, 2.32; p=0.03). In the study, 77% of patients were also on edaravone and/or riluzole. When corrected for the use of edaravone and riluzole, the between-group differences in ALSFRS-R scores were 2.15 and 2.34, respectively. Secondary outcomes did not differ significantly between the groups. These included rates of decline in isometric muscle strength, plasma phosphorylated axonal neurofilament H subunits, and slow vital capacity as well as time to death, tracheostomy, permanent assisted ventilation, or any hospitalization. The most common TEAEs were mild to moderate GI events. A survival analysis that followed patients for up to 3 years revealed a 44% lower risk of death with PB/TURSO than with placebo. In an open-label, long-term follow-up, the median survival was 25 months with PB/TURSO and 18.5 months with placebo. Amylyx has also initiated the double-blind, placebo-controlled, phase 3, PHOENIX trial. Primary data is expected in late 2023. Sodium phenylbutyrate 3 g and taurursodiol 1 g were administered orally as a fixed-dose combination once daily for 3 weeks, followed by twice daily thereafter.

PLACE IN THERAPY

ALS is a rare, progressive motoneuron disease (MND) characterized by voluntary and involuntary muscle weakness, atrophy, paralysis, respiratory failure, and premature death. It is estimated that 16,000 people in the US have ALS, with approximately 5,000 new cases each year. The age at diagnosis is typically between 40 to 75 years. Once symptoms develop, life expectancy is 2 to 5 years. ALS occurs at similar rates in men and women, and 5% to 10% of cases appear to be inherited. Two DMTs are approved to treat ALS. These include the oral glutamate pathway antagonist riluzole (generic, Exservan™, Rilutek®, Tiglutik®) and the IV-administered free radical scavenger edaravone (Radicava®). Riluzole may increase survival by several months and extend the time to mechanical ventilation. Investigational PB/TURSO targets neuronal degeneration pathways in ALS, such as those that involve Bax. Non-comparative studies reveal a similar improvement in the ALSFRS-R score over the control with PB/TURSO and edaravone (2.32 versus 2.49 point difference from control, respectively) after 24 weeks of therapy. If approved, PB/TURSO will be another DMT option for patients with ALS. Sodium phenylbutyrate is approved as adjunctive treatment for certain urea cycle disorders. An oral formulation of edaravone was submitted to the FDA for ALS, with an FDA decision expected in May 2022.

FDA APPROVAL TIMELINE June 29, 2022

On March 30, 2022, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 6 to 10 against the approval of PB/TURSO due to lack of effectiveness.  Orphan Review

Priority Review

FINANCIAL FORECAST (reported in millions) The financial forecast for PB/TURSO is not currently available. 9 | MAGELLANRX.COM

IMMUNOLOGY

spesolimab IV, SC Boehringer Ingelheim PROPOSED INDICATIONS

Generalized pustular psoriasis (GPP) flares

CLINICAL OVERVIEW

Spesolimab is an antibody that selectively inhibits the interleukin-36 receptor (IL-36R) signaling pathway involved in autoimmune diseases. Spesolimab was evaluated in a randomized, placebo-controlled, double-blind, placebo-controlled, phase 2 trial in 53 patients with a moderate to severe acute GPP flare. Patients were randomized 2:1 to received spesolimab or placebo on day 1. Patients in either group were eligible to receive rescue treatment with openlabel spesolimab on day 8 and/or after day 8. Patients were followed until week 12. One week after receiving the study drug, significantly more patients treated with spesolimab experienced the primary endpoint of clearance of GPP lesions (Generalized Pustular Psoriasis Physician Global Assessment [GPPGA] pustulation subscore of 0) compared to those who received placebo (54% versus 6%, respectively; p>0.001). In addition, 84.4% of patients who received spesolimab reported no visible pustules (GPPGA, 0) after 12 weeks. One week after the dose, infection was reported in 17% of those treated with a single dose of spesolimab compared to 6% who received placebo. Among patients who received spesolimab at any time during the trial, including open-label rescue with spesolimab, infection had occurred in 24 of 51 (47%) at week 12. Serious adverse events, including drug-induced hepatic injury, UTI, and arthritis, were reported with spesolimab (each reported in 1 patient). Spesolimab was administered as a 900 mg dose via IV infusion. Among patients assigned to spesolimab, rescue doses of spesolimab were administered to 12 patients at day 8 and 4 patients after day 8. In the placebo group, 15 and 2 patients received rescue spesolimab at day 8 and after day 8, respectively. An ongoing open-label, 5-year extension GPP trial is evaluating every 4-, 6-, and 12-week SC dosing regimens.

PLACE IN THERAPY

GPP is a rare, potentially life-threatening autoinflammatory neutrophilic skin disorder. In some cases, IL36RN gene mutations leading to unregulated inflammatory cytokine production are present. Patients experience widespread sterile pustules. Symptoms at GPP flare onset include fever, chills, headache, intense itching, malaise, fatigue, and arthritis. GPP lesions typically resolve within weeks, but relapse is common. GPP flares may be triggered by infection, stress, corticosteroid discontinuation, and pregnancy or may have no apparent trigger. Hospitalization for flares is common. Without effective treatment, death due to complications (e.g., heart failure, renal failure, sepsis) has been reported during the acute stage. GPP primarily affects adults but has been reported in children and occurs in approximately half of those who suffer from PSO. Topical treatment of areas affected by GPP includes emollients and corticosteroids. Systemic retinoids, cyclosporine, and methotrexate have been used off-label in the short term. Off-label use of biologicals, including infliximab, adalimumab, and etanercept, is also reported. If approved, spesolimab will be the first treatment indicated in the US to treat GPP flares. In clinical trials, spesolimab was associated with non-serious infections. AnaptysBio’s IL-36R inhibitor imsidolimab is also in late-stage development for GPP flares. In a phase 2 trial, imsidolimab was administered IV on day 1, followed by 3 monthly SC doses. Spesolimab is also in phase 3 trials for UC and phase 2 trials for atopic dermatitis and hidradenitis suppurativa.

FDA APPROVAL TIMELINE June to July 2022

 Breakthrough Therapy

Orphan Drug

Priority Review

FINANCIAL FORECAST (reported in millions)

The financial forecast for spesolimab is not currently available.

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ONCOLOGY

teclistamab SC Janssen PROPOSED INDICATIONS

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

CLINICAL OVERVIEW

Teclistamab is a bispecific antibody that targets B-cell maturation antigen (BCMA) and CD3 receptors. It redirects CD3-positive T cells to BCMA-expressing myeloma cells to induce tumor cell kill. The FDA submission of teclistamab was based on the phase 1/2, open-label MajesTEC-1 trial in 159 adults with R/R MM who received ≥ 3 prior lines of therapy, including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 antibody. At a median follow-up of nearly 8 months, teclistamab demonstrated an ORR of 62%, with a complete response rate of 29%. The median time to first confirmed response was 1.2 months (range, 0.2 to 5.5) among patients who responded to therapy. At 9 months, the PFS was 59% and the median OS was not reached. Cytokine release syndrome (CRS) occurred in 72% of patients (1 grade 3; all others grade ≤ 2) and immune effector cell-associated neurotoxicity syndrome (ICANS) was reported in 3% of patients. All CRS and ICANS events resolved without discontinuation of therapy. Neutropenia (66%), anemia (50%), and thrombocytopenia (38%) occurred, including serious cases (grade 3/4 in 57%, 35%, and 21%, respectively). Phase 2 dosing of teclistamab was 1,500 µg/kg SC once weekly following step-up doses of 60 µg/kg and 300 µg/kg.

PLACE IN THERAPY

MM is a malignancy of plasma cells that accumulate in bone marrow, resulting in marrow failure. Nearly 34,500 new cases of MM and over 12,600 deaths due to the condition are predicted in the US in 2022. 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, eventual 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 proteasome inhibitor, an immunomodulatory agent, or a monoclonal antibody. Newer alternatives for patients who have received ≥ 4 prior lines of therapies are monotherapy with the antibody-drug conjugate (ADC) belantamab mafodotin-blmf (Blenrep®) and the CAR-T therapies idecabtagene vicleucel (Abecma®) and ciltacabtagene autoleucel (Carvykti™). All 3 treatments target BCMA, which is present in 60% to 70% of patients with MM. If approved, teclistamab will be the first off-the-shelf T cell-redirecting bispecific antibody therapy, which differentiates it from CAR-T therapies. Initial approval is expected to be in the heavily-pretreated space. While teclistamab does not demonstrate the same efficacy as CAR-T therapy (ORR, 95% with Carvykti and 72% with Abecma), it has a more favorable tolerability profile overall compared to CAR-T therapies. Teclistamab also offers an off-the-shelf option that is administered SC and has the potential for combination use in earlier lines of treatment. Teclistamab is currently being studied in combination with daratumumab SC in patients with R/R MM who have received 1 to 3 prior lines of therapy.

FDA APPROVAL TIMELINE August to December 2022  Breakthrough Therapy

Orphan Drug

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$13

$52

$93

11 | MAGELLANRX.COM

DIABETES

tirzepatide SC Eli Lilly PROPOSED INDICATIONS

Type 2 diabetes mellitus (T2DM)

CLINICAL OVERVIEW

Tirzepatide is a dual glucose-dependent insulinotropic polypeptide (GIP)/glucagon-like peptide-1 (GLP-1) receptor agonist (GLP-1RA). The phase 3 SURPASS clinical study program demonstrated dose-dependent improvements in glycemic control and body weight with tirzepatide in adults with uncontrolled T2DM. Across the studies, once weekly SC tirzepatide doses of 5 mg, 10 mg, and 15 mg were assessed for 40 to 52 weeks. In SURPASS-1 (n=478; double-blind), tirzepatide monotherapy reduced mean HbA1c from baseline by 1.91% to 2.11% (p<0.001 for all doses) compared to placebo in antidiabetic medication-naïve patients. A dose-dependent decrease in body weight by 7 kg to 9.5 kg was also seen with tirzepatide. SURPASS-2 (n=1,879; open-label) enrolled patients whose T2DM was inadequately controlled with metformin. In this trial, all doses of tirzepatide were superior to once weekly SC semaglutide 1 mg based on a reduction in mean HbA1c from baseline (difference range, -0.15% to -0.45%; p≤0.02 for all). Significantly greater reductions from baseline in mean body weight were also seen across all tirzepatide doses compared to SC semaglutide 1 mg (difference range, -1.9 kg to -5.5 kg; p<0.001 for all doses). The incidence of hypoglycemia (blood glucose < 54 mg/dL) was higher with tirzepatide 15 mg (1.7%) than with SC semaglutide 1 mg (0.4%). The most commonly reported TEAEs were GI-related, generally mild to moderate in severity and occurred more often with tirzepatide than semaglutide. Tirzepatide also demonstrated statistically significant improvement in HbA1c and lower incidence of hypoglycemia compared to insulin degludec (SURPASS-3; n=1,444; open-label) and insulin glargine (SURPASS-4; n=2,002; open-label). Tirzepatide was administered once weekly via SC injection.

PLACE IN THERAPY

An estimated 37.3 million people in the US have diabetes, with most cases being T2DM. T2DM pathology involves insufficient GI secretion of the hormones GLP-1 and GIP that regulate glucose metabolism and increase energy expenditure. GLP-1RAs are recommended for glycemic control in T2DM, particularly in patients with or at risk of developing ASCVD, CKD, or HF, and when weight loss is desirable. If approved, tirzepatide will be the first agent to target both GLP-1 and GIP. It will likely compete with the injectable GLP-1RA semaglutide 1 mg (Ozempic®) in adults with T2DM. In clinical trials, tirzepatide led to dosedependent improvements in glycemic control and weight loss that exceeded SC semaglutide 1 mg, as well as insulin degludec (Tresiba®) and insulin glargine (Lantus®). Hypoglycemia occurred more often with tirzepatide than with SC semaglutide but less often when compared to the insulin products. In clinical trials, tirzepatide also improved serum lipid parameters to a greater extent than SC semaglutide 1 mg. Notably, tirzepatide has not been compared to oral semaglutide (Rybelsus®) or to the recently FDA-approved semaglutide (Ozempic) 2 mg SC dose. Since, to date, CV outcomes studies with tirzepatide have not been completed, the Institute for Clinical and Economic Review (ICER) concluded that there is uncertainty surrounding tirzepatide's clinical effectiveness relative to other available T2DM treatment options. Tirzepatide is also in phase 3 trials for obesity, an indication already held by SC semaglutide 2.4 mg (Wegovy®), and for chronic HF.

FDA APPROVAL TIMELINE May 30, 2022

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$79

$662

$1,675

$2,802

$3,793

12 | MAGELLANRX.COM

ONCOLOGY

tislelizumab IV, SC Novartis/Beigene PROPOSED INDICATIONS

Unresectable, recurrent, locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) after prior systemic therapy

CLINICAL OVERVIEW

Tislelizumab is a humanized IgG4 anti-PD-1 monoclonal antibody. It is designed to minimize binding to macrophage Fc gamma receptors (FcγRs) and potentially reduce anti-PD-1 resistance. The randomized, open-label, phase 3 RATIONALE 302 trial evaluated tislelizumab in 512 patients with advanced or metastatic ESCC that had progressed during or after first-line treatment. Among the patients, 64.9% had previously received radiotherapy, 37.7% had prior surgery, and 97.9% had previous platinumbased chemotherapy. Patients with an ECOG performance status of 0 or 1 were randomized 1:1 to tislelizumab or investigators’ choice of chemotherapy (e.g., paclitaxel, docetaxel, irinotecan). At the data cut-off, the median follow-up was 8.5 months in the tislelizumab group and 5.8 months in the chemotherapy group. Data revealed that the OS was significantly greater with tislelizumab compared to chemotherapy in the overall ITT study population (median OS, 8.6 versus 6.3 months, respectively; p=0.0001; primary endpoint) and among PD-L1-positive patients (median OS, 10.3 versus 6.8 months, respectively; p=0.0006; secondary endpoint). In addition, ORR and median DOR were higher with tislelizumab than with chemotherapy (ORR, 20.3% versus 9.8%, respectively; DOR, 7.1 versus 4 months, respectively). Fewer patients treated with tislelizumab experienced a grade ≥ 3 TEAE than those treated with chemotherapy (46% versus 68%, respectively). Tislelizumab was administered as 200 mg IV every 3 weeks until disease progression or unacceptable toxicity.

PLACE IN THERAPY

The incidence of esophageal cancer has been rising in the US. It is estimated that 20,640 esophageal cancer cases will be diagnosed and 16,410 deaths associated with the disease will occur in 2022. While esophageal adenocarcinoma is more prominent in the US compared to ESCC, ESCC is associated with earlier lymphatic spread and poorer prognosis. The 5-year survival rate for ESCC remains low. Major risk factors for ESCC are tobacco and alcohol use. First-line systemic treatment of unresectable locally advanced, recurrent, or metastatic ESCC includes a fluoropyrimidine agent (fluorouracil or capecitabine) plus a platinum agent, with or without pembrolizumab (Keytruda®; for PD-L1 combined positive score [CPS] ≥ 10). Second-line treatment of ESCC includes the PD-1 inhibitors nivolumab (Opdivo®) and pembrolizumab (for PD-L1 combined CPS ≥ 10) in patients with no prior tumor progression while on therapy with a checkpoint inhibitor. If approved, tislelizumab will provide a third PD-1 inhibitor option as second-line therapy in patients with unresectable, recurrent, locally advanced or metastatic ESCC. Tislelizumab differs from other PD-1 inhibitors by its lack of FcγR binding; however, it remains to be seen whether this confers clinical benefit over the available PD-1 inhibitors. Based on non-comparative data, tislelizumab appears to demonstrate similar efficacy (based on OS) to nivolumab and pembrolizumab for the proposed indication. Pembrolizumab is also indicated as a first-line treatment combined with chemotherapy for locally advanced or metastatic ESCC; nivolumab and tislelizumab are in phase 3 trials in the first-line setting.

FDA APPROVAL TIMELINE July 12, 2022

Orphan Drug

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales*

$58

$194

$307

$427

$580

*For all indications being investigated 13 | 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. Moreover, an interchangeable biological product is a biosimilar that is expected to produce the same clinical result as the reference product in any given patient. Switching or alternating between the reference and interchangeable products should have been evaluated and should not negatively impact the safety and efficacy of therapy. Many controversies surround biosimilars, and regulatory and litigation hurdles remain. The FDA has issued final and draft guidances. Select FDA biosimilar guidances are noted here. In January 2017, the Agency issued final guidance on the nonproprietary naming of biologic products, which also applies to biosimilars. The biological products must bear a core name followed by a distinguishing 4-letter, lowercase, hyphenated suffix that is devoid of meaning. The international nonproprietary name (INN) impacts interchangeability as it affects pharmacists’ ability to substitute an interchangeable biosimilar for the reference product. The FDA withdrew the September 2017 draft industry guidance on determining similarity of a proposed biosimilar product to its reference product to allow for further consideration of the most current and relevant scientific methods in evaluating analytical data. The agency focuses on providing flexibility for the efficient development of biosimilars while maintaining high scientific standards. In July 2018, the FDA finalized its guidance on labeling biosimilars. The guidance pertains to prescribing information (PI) but does not contain specific recommendations on interchangeability in the labeling. The labeling guidance provides recommendations on how to include, identify, and differentiate the biosimilar and the reference product in various sections of the PI. The basic premise remains that the originator product’s safety and effectiveness can be relied upon for HCPs to make prescribing decisions; therefore, a biosimilar should include relevant data from the originator in its PI. In May 2019, the agency released its final guidance on interchangeability. Most states have enacted biosimilar substitution laws. An interchangeable product may be substituted for the originator at the pharmacy without the involvement of the prescriber. The Purple Book is an FDA database of licensed biological products that lists biosimilar and interchangeable products. The FDA has approved 2 biosimilars for interchangeability to their reference product: insulin glargine-yfgn (Semglee®) and adalimumab-adbm (Cyltezo®). Biosimilars can receive extrapolation to gain an indication without direct trials of the biosimilar for the eligible indication(s) of the reference products without requiring additional trials. Nevertheless, as each biosimilar comes to market, it will need to be considered individually. The FDA historically regulated insulins as small molecules. However, effective March 23, 2020, drugs such as insulin and growth hormone were deemed biologics and transitioned from the drug pathway to the biologic pathway. Their licensure as biologics allows these agents to be considered in the biosimilar space and promotes competition and access.

PLACE IN THERAPY

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

14 | MAGELLANRX.COM

To date, a total of 35 biosimilars have received FDA approval. Of these, only 21 have entered the market. APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Zarxio® (filgrastim-sndz)

Novartis/Sandoz

March 2015

Inflectra® (infliximab-dyyb)

Pfizer/Celltrion

April 2016

Erelzi® (etanercept-szzs)

Novartis/Sandoz

August 2016

Amjevita™ (adalimumab-atto)

Amgen

September 2016

Renflexis® (infliximab-abda)

Samsung Bioepis/ Merck

May 2017

Cyltezo (adalimumab-adbm)

Boehringer Ingelheim

August 2017

Mvasi (bevacizumab-awwb)

Amgen

September 2017

Ixifi™ (infliximab-qbtx)†

Pfizer

December 2017

Ogivri® (trastuzumab-dkst)

Viatris

December 2017

Retacrit® (epoetin alfa-epbx)

Pfizer/Hospira

May 2018

Fulphila® (pegfilgrastim-jmdb)

Viatris

June 2018

Nivestym® (filgrastim-aafi)

Pfizer

July 2018

Hyrimoz™ (adalimumab-adaz)

Novartis/Sandoz

October 2018

Udenyca® (pegfilgrastim-cbqv)

Coherus

November 2018

Truxima® (rituximab-abbs)

Celltrion/Teva

November 2018

Herzuma® (trastuzumab-pkrb)

Celltrion/Teva

December 2018

Ontruzant® (trastuzumab-dttb)

Samsung Bioepis/ Merck

January 2019

Trazimera™ (trastuzumab-qyyp)

Pfizer

March 2019

Eticovo™ (etanercept-ykro)

Samsung Bioepis/ Merck

April 2019

Kanjinti™ (trastuzumab-anns)

Amgen

June 2019

Zirabev (bevacizumab-bvzr)

Pfizer

June 2019

Hadlima™ (adalimumab-bwwd)

Samsung Bioepis/ Merck

July 2019

Ruxience® (rituximab-pvvr)

Pfizer

July 2019

Abrilada™ (adalimumab-afzb)

Pfizer

November 2019

Ziextenzo® (pegfilgrastim-bmez)

Novartis/Sandoz

November 2019

15 | MAGELLANRX.COM

Interchangeable -

Commercially Available

 



    -

     -

 



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

APPROVED BIOSIMILARS continued APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Avsola® (infliximab-axxq)

Amgen

December 2019

Nyvepria™ (pegfiltrastim-apgf)

Pfizer

June 2020

Semglee (insulin glargine-yfgn)

Viatris

July 2021

Hulio® (adalimumab-fkjp)

Viatris

July 2020

Riabni™ (rituximab-arrx)

Amgen

December 2020

Byooviz (ranibizumab-nuna)

Biogen/Samsung Bioepis

September 2021

Rezvoglar (insulin glargine-aglr)

Eli Lilly

December 2021

Yusimry™ (adalimumab-aqvh)

Coherus

December 2021

Releuko (filgrastim-ayow)

Amneal

March 2022

Alymsys® (bevacizumab-maly)

Amneal

April 2022

Interchangeable

Commercially Available

Originator (Manufacturer) Remicade (Janssen)



  



Rituxan (Genentech)

Lucentis® (Genentech)

Neupogen (Amgen)

Avastin (Genentech)

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

Lantus (Sanofi) Humira (Abbvie)

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

Also available are Eli Lilly’s Basaglar® insulin glargine, a follow-on to Sanofi’s Lantus, and Sanofi’s Admelog® insulin lispro approved as a follow-on to Eli Lilly’s Humalog®. Specialty medications, which include biologics, continue to grow and constitute a large part of drug spend. In the US, it is estimated that biosimilars will cost approximately 15% to 35% less than the originator product, although price dynamics vary. Further, the potential cost savings can vary based on the market segment where brand contracts can play a role. A host of factors will contribute to market acceptability and the potential success of biosimilars. Payers, pharmacies, prescribers, and patients each play an important role in market adoption of biosimilars. 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.

16 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

IMMUNOLOGY

adalimumab SC Alvotech, Fresenius, and Celltrion are seeking approval for their investigational biosimilars to Abbvie’s Humira, a tumor necrosis factor alpha (TNF-α) blocker indicated for the treatment of autoimmune disorders, including rheumatoid arthritis (RA), juvenile idiopathic arthritis (JIA), ankylosing spondylitis (AS), plaque psoriasis (PSO), psoriatic arthritis (PsA), Crohn’s disease (CD) in adults and children, ulcerative colitis (UC), hidradenitis suppurativa (HS), and non-infectious uveitis. Pfizer is seeking interchangeability of FDA-approved adalimumab-afzb (Abrilada). Organon/Samsung Bioepis has submitted a 100 mg/mL concentration as an additional FDA-approved formulation for adalimumab-bwwd (Hadlima).

FDA APPROVAL TIMELINE

Alvotech (AVT-02) December 2022 for initial approval and interchangeability Celltrion (Yuflyma) August 2022 Fresenius (MSB11022) October to December 2022 Pfizer (Abrilada) October to December 2022 for interchangeability Organon/Samsung Bioepis (Hadlima 100 mg/mL) August 2022

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$18,552

$10,899

$7,231

$5,527

$4,423

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

OPHTHALMOLOGY

aflibercept intravitreal Viatris/Janssen Viatris/Janssen is seeking approval of their investigational biosimilar (M710) to Regeneron’s Eylea®, a vascular endothelial growth factor (VEGF) inhibitor indicated for the treatment of neovascular (wet) age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME), and diabetic retinopathy.

FDA APPROVAL TIMELINE October 31, 2022

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$6,152

$6,434

$6,226

$5,919

$5,424

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

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BIOSIMILAR OVERVIEW continued

ONCOLOGY

bevacizumab IV Bio-Thera Solutions/Novartis, Centus/AstraZeneca, Samsung Bioepis/Merck, and Viatris/Biocon are seeking approval for their investigational biosimilars to Genentech’s Avastin, a vascular endothelial growth factor (VEGF)-specific angiogenesis inhibitor indicated for the treatment of metastatic colorectal cancer, nonsquamous non-small cell lung cancer, glioblastoma, metastatic renal cell carcinoma (RCC), and recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer.

FDA APPROVAL TIMELINE

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

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$699

$562

$477

$428

$385

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

BLOOD MODIFIER

filgrastim IV, SC Apotex Apotex is seeking approval of their investigational biosimilar (Grastofil) 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

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$97

$92

$85

$77

$73

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

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BIOSIMILAR OVERVIEW continued

BLOOD MODIFIER

pegfilgrastim SC Amneal, Apotex, Lupin, and Merck/Fresenius are seeking approval for their 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 Amneal (TPI-120) Pending Apotex (Lapelga) Pending Lupin (Lupifil-P) April 2022 Merck/Fresenius (MSB-11455) Pending

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$1,329

$1,105

$985

$877

$794

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

IMMUNOLOGY

ranibizumab intravitreal Coherus Coherus is seeking approval for their investigational biosimilar (FYB201) to Genentech’s Lucentis, a vascular endothelial growth factor (VEGF) inhibitor indicated to treat wet age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME), diabetic retinopathy, and myopic choroidal neovascularization (mCNV).

FDA APPROVAL TIMELINE August 2, 2022

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$1,313

$1,056

$807

$636

$530

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

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BIOSIMILAR OVERVIEW continued

ONCOLOGY

trastuzumab IV Novartis and Tanvex are seeking approval for their investigational biosimilars to Genentech’s Herceptin, a HER2/neu receptor antagonist indicated for the treatment of HER2-overexpressing breast cancer and HER2-overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma.

FDA APPROVAL TIMELINE Novartis December 20, 2022 Tanvex (TX05) August 2022

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$486

$416

$368

$333

$303

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

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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 2026, are displayed. The financials are projected total annual US sales, reported in millions. zuranolone

adagrasib

$1,206

$796

Behavioral health

tabelecleucel Oncology

Oncology

autologous CRISPR-Cas9 modiﬁed CD34+ human hematopoietic stem and progenitor cells (CTX001) Hematology/Gene therapy

$533

$949

resmetirom

dextromethorphan/ bupropion

Endocrine

$602

Behavioral health

$812 pegcetacoplan Ophthalmics

donanemab

$843

Neurology

$3,516 Novavax COVID-19 vaccine (NVX-CoV2373)

etrasimod

Immunomodulators

COVID-19

$634

$5,532 nirsevimab

gantenerumab

$541

$672

Neurology

Infectious disease

mirikizumab Immunology

$649

lenadogene nolparvovec (GS-010)

Ophthalmology/Gene therapy

$178

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

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

IN PHASE PHASE 3 3 TRIALS TRIALS

68%

67%

32%

33%

36%

35%

26%

Specialty

28%

14%

15%

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

DEVELOPMENT STATUS

FDA DECISION

Submitted (New Drugs) pegfilgrastim (biosimilar to Amgen’s Neulasta)

Lupin

Neutropenia/leukopenia

Submitted – BLA

April 2022

penpulimab

Akeso

Nasopharyngeal cancer (metastatic, 3rd-line)

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

Apr–Jun 2022

mavacamten

Bristol-Myers Squibb

Obstructive hypertrophic cardiomyopathy

Oral

Submitted – NDA; Breakthrough Therapy; Orphan Drug

04/28/2022

meloxicam/rizatriptan

Axsome

Migraine treatment

Oral

Submitted – 505(b)(2) NDA

04/29/2022

surufatinib

Hutchmed

Neuroendocrine tumors

Oral

Submitted – NDA; Fast Track; Orphan Drug

04/29/2022

toripalimab

Coherus

Nasopharyngeal carcinoma (recurrent or metastatic, in combination with chemotherapy for 1stline, monotherapy for 2nd-line)

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

04/29/2022

treprostinil DPI (Tyvaso DPI)

United Therapeutics

PAH; Idiopathic pulmonary fibrosisassociated pulmonary hypertension

Inhaled

Submitted – NDA

May 2022

vonoprazan

Phathom

Helicobacter pylori infection (in combination with amoxicillin ± clarithromycin)

Oral

Submitted – NDA; Fast Track; Priority Review; QIDP

05/03/2022

edaravone

Mitsubishi Tanabe

ALS

Oral

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

05/12/2022

cantharidin

Verrica

Molluscum contagiosum

Topical

Submitted – NDA

05/24/2022

tapinarof

Roivant

PSO (mild-severe)

Topical

Submitted – NDA

05/26/2022

tirzepatide

Eli Lilly

T2DM

Submitted – NDA; Priority Review

05/30/2022

spesolimab

Boehringer Ingelheim

Generalized pustular psoriasis flares

IV, SC

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

Jun–Jul 2022

trientine tetrahydrochloride

GMP-Orphan

Wilson’s disease

Oral

Submitted – NDA; Orphan Jun–Jul 2022 Drug

sodium phenylbutyrate

Acer

Urea cycle disorders

Oral

Submitted – 505(b)(2) NDA

06/03/2022

tebipenem pivoxil HBr

Spero

UTI (complicated)

Oral

Submitted – NDA; Fast Track; Priority Review; QIDP

06/27/2022

sodium phenylbutyrate/ taurursodiol

Amylyx

ALS

Oral

Submitted – NDA; Orphan 06/29/2022 Drug; Priority Review

narsoplimab

Omeros

HSCT-associated thrombotic microangiopathy

IV, SC

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

23 | MAGELLANRX.COM

July 2022

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

bulevirtide

Gilead

Hepatitis D infection (with compensated liver disease) treatment

Submitted – BLA; Breakthrough Therapy; Orphan Drug

Jul–Nov 2022

olipudase alfa

Sanofi

Acid sphingomyelinase deficiency (non–CNS manifestations, also known as Niemann-Pick disease types A, B, and A/B)

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

07/03/2022

tislelizumab

Beigene

Esophageal squamous cell carcinoma (unresectable, recurrent, locally advanced/ metastatic, after prior systemic therapy)

Submitted – BLA; Orphan Drug

07/12/2022

casirivimab/imdevimab (Regen-Cov)

Regeneron

COVID-19 treatment (non-hospitalized patients); COVID-19 postexposure prophylaxis

IM, IV, SC

Submitted – BLA; Priority Review

07/13/2022

vutrisiran

Alnylam

Transthyretin amyloid polyneuropathy

Submitted – NDA; Fast Track; Orphan Drug

07/14/2022

cipaglucosidase alfa

Amicus

Pompe disease (in combination with oral miglustat)

Submitted – BLA; Breakthrough Therapy; Orphan Drug

07/29/2022

roflumilast cream

Arcutis/AstraZeneca

PSO (mild-severe)

Topical

Submitted – NDA

07/29/2022

adalimumab (biosimilar to Abbvie’s Humira)

Celltrion

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

Submitted – BLA

August 2022

trastuzumab (biosimilar to Genentech’s Herceptin)

Tanvex

Breast cancer; Gastric/ gastroesophageal cancer

Submitted – BLA

August 2022

teclistamab

Janssen

Multiple myeloma (R/R)

Submitted – BLA; Breakthrough Therapy; Orphan Drug

Aug–Dec 2022

ranibizumab (biosimilar to Genentech’s Lucentis)

Coherus

Wet AMD; Macular edema following RVO; Myopic choroidal neovascularization (mCNV)

Intravitreal

Submitted – BLA

08/02/2022

trivalent measles-mumpsrubella (MMR) vaccine

GlaxoSmithKline

Measles, mumps, and rubella immunization

Submitted – BLA

08/02/2022

teplizumab

Provention Bio

T1DM (delay/prevention)

Submitted – BLA; Breakthrough Therapy; Orphan Drug

08/17/2022

betibeglogene autotemcel (Zynteglo)

Bluebird Bio

β-thalassemia (transfusion-dependent)

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

08/19/2022

sodium thiosulfate

Fennec

Chemotherapy-induced ototoxicity prevention

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

September 2022

microbiota suspension

Ferring

Clostridioides difficile infection (recurrent)

Rectal

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

Sep–Nov 2022

eflapegrastim

Spectrum

Chemotherapy-induced neutropenia

Submitted – BLA

09/09/2022

deucravacitinib

Bristol-Myers Squibb

PSO (moderate-severe)

Oral

Submitted – NDA

09/10/2022

24 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

linzagolix

Obseva

Uterine fibroid-related Oral heavy menstrual bleeding

Submitted – NDA

09/13/2022

dasatinib

Xspray

CML

Oral

Submitted – 505(b)(2) NDA

09/16/2022

elivaldogene autotemcel (Lenti-D)

Bluebird Bio

Cerebral adrenoleukodystrophy (pediatrics)

Submitted – BLA; Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatrics Disease

09/16/2022

taurolidine/citrate/heparin

CorMedix

Reduction of catheterrelated bloodstream infections (CRBSIs) related to chronic hemodialysis

Submitted – NDA; Fast Track; QIDP

09/28/2022

ublituximab

TG Therapeutics

MS (relapsing)

Submitted – BLA

09/28/2022

futibatinib

Otsuka

Cholangiocarcinoma (advanced or metastatic, FGFR2 gene rearrangements)

Oral

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

09/30/2022

aflibercept (biosimilar to Regeneron’s Eylea)

Viatris/Janssen

DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD

Intravitreal

Submitted – BLA

10/31/2022

furosemide

scPharmaceuticals

Decompensated heart failure

Submitted – 505(b)(2) NDA

October 2022

adalimumab (biosimilar to Abbvie’s Humira)

Fresenius

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

Submitted – BLA

Oct–Dec 2022

bevacizumab-vikg

Outlook

Wet AMD

Intravitreal

Submitted – BLA

Oct 2022–Mar 2023

apomorphine infusion pump

Supernus

Parkinson’s disease

Submitted – NDA

10/07/2022

treosulfan

Medac

Allogenic-HSCT conditioning

IV, Oral

Submitted – NDA; Orphan 10/22/2022 Drug

poziotinib

Spectrum

NSCLC (locally advanced/ metastatic; HER2 exon 20 insertion mutations)

Oral

Submitted – NDA; Fast Track

11/24/2022

omecamtiv mecarbil

Cytokinetics

Heart failure with reduced ejection fraction (HFrEF)

Oral

Submitted – NDA; Fast Track

11/30/2022

adalimumab (biosimilar to Abbvie’s Humira)

Alvotech

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

Submitted – BLA; seeking biosimilar interchangeability

December 2022

adagrasib

Mirati

NSCLC (KRASG12C mutation, ≥2nd-line)

Oral

Submitted – NDA; seeking Accelerated Approval; Breakthrough Therapy; RTOR

12/14/2022

trastuzumab (biosimilar to Genentech’s Herceptin)

Novartis

Breast cancer; Gastric/ gastroesophageal cancer

Submitted – BLA

12/20/2022

mirvetuximab soravtansine

Immunogen

Ovarian cancer (folate receptor alpha [FRα]-high platinum-resistant)

Submitted – BLA; seeking Jan–Mar 2023 Accelerated Approval; Fast Track; Orphan Drug

sparsentan

Travere/BristolMyers Squibb

Immunoglobulin A nephropathy (Berger’s disease)

Oral

Submitted – NDA; Orphan Jan–Mar 2023 Drug

25 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

daprodustat

GlaxoSmithKline

CKD-related anemia (dialysis-dependent and -independent)

Oral

Submitted – NDA

02/01/2023

fexapotide triflutate

Nymox

Benign prostatic hyperplasia

Intratumoral

Submitted – NDA

03/03/2023

vonoprazan

Phathom

Esophagitis (in combination with amoxicillin ± clarithromycin)

Oral

Submitted – NDA

03/14/2023

omaveloxolone

Reata

Friedreich’s ataxia

Oral

Submitted – NDA; Fast Track; Orphan Drug

03/31/2023

omburtamab

Y-mAbs

Brain cancer (CNS/ leptomeningeal metastasis from neuroblastoma)

Intracerebroventricular

Submitted – BLA; Breakthrough Therapy; Orphan Drug

03/31/2023

pegzilarginase

Aeglea

Arginase 1 deficiency

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

04/12/2023

aviptadil

NRx

COVID-19 treatment (respiratory failure, no other options are available)

Inhaled

Submitted – EUA; Breakthrough Therapy; Fast Track

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Bio-Thera Solutions/ Novartis

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

Submitted – BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Centus/AstraZeneca

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

Submitted – BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Samsung Bioepis/ Merck

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

Submitted – BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Viatris/Biocon

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

Submitted – BLA

Pending

bimekizumab

UCB

PSO

Submitted – BLA

Pending

COVID-19 vaccine (NVXCoV2373)

Novavax

COVID-19 prevention (adults)

Submitted – EUA

Pending

dextromethorphan/ bupropion

Axsome

MDD

Oral

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

Pending

diazepam buccal film

Aquestive

Seizure clusters

Oral transmucosal

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

Pending

filgrastim (biosimilar to Amgen’s Neupogen)

Apotex

Neutropenia/leukopenia

IV, SC

Submitted – BLA

Pending

infliximab-dyyb (Inflectra)

Celltrion

IBS

Submitted – BLA

Pending

Moderna COVID-19 vaccine (mRNA-1273)

Moderna

COVID-19 prevention (ages 12–17 years, ages 2–6 years, ages 6 months to < 2 years)

Submitted – EUA; Fast Track

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Amneal

Neutropenia/leukopenia

Submitted – BLA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Apotex

Neutropenia/leukopenia

Submitted – BLA

Pending

26 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Merck/Fresenius

Neutropenia/leukopenia

Submitted – BLA

Pending

Pfizer-Biontech COVID-19 vaccine

Pfizer/Biontech

COVID-19 prevention (ages 5–11 years; booster dose)

Submitted – EUA

Pending

sodium oxybate (oncenightly)

Avadel

Narcolepsy-related excessive daytime sleepiness and cataplexy

Oral

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

Pending

Submitted (Supplementals) baricitinib (Olumiant®)

Eli Lilly

COVID-19 treatment

Oral

Submitted – sNDA; Priority Review

Apr–Jun 2022

fam-trastuzumab deruxtecan-nxki (Enhertu)

Daiichi Sankyo

Breast cancer (HER2+, unresectable or metastatic, ≥ 1 prior antiHER2-based regimens)

Submitted – sBLA; Breakthrough; Fast Track; Priority Review; RTOR

Apr–Jun 2022

brolucizumab-dbll (Beovu®)

Novartis

DME

Intravitreal

Submitted – sBLA

Apr–Sep 2022

tisagenlecleucel-t (Kymriah®)

Novartis

Follicular lymphoma (R/R, 3rd-line)

Submitted – sBLA; Orphan Drug; Priority Review; RMAT

04/27/2022

Supernus

ADHD (adults)

Oral

Submitted – sNDA

04/29/2022

faricimab-svoa (Vabysmo )

Genentech

Diabetic retinopathy

Intravitreal

Submitted – sBLA

May 2022

relugolix/estradiol/ norethindrone (Myfembree)

Myovant

Endometriosis

Oral

Submitted – sNDA

05/06/2022

ipilimumab (Yervoy®)

Bristol-Myers Squibb

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

Submitted – sBLA

05/27/2022

nivolumab (Opdivo)

Bristol-Myers Squibb

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

Submitted – sBLA

05/27/2022

ravulizumab-cwvz (Ultomiris®)

AstraZeneca

Myasthenia gravis

Submitted – sBLA; Priority Review

Jun–Jul 2022

dupilumab (Dupixent®)

Sanofi/Regeneron

Atopic dermatitis (moderate-severe; ages 6 months–5 years)

Submitted – sBLA; Breakthrough Therapy; Priority Review

06/09/2022

setmelanotide (Imcivree™)

Rhythm

Bardet-Biedle syndrome or Alström (related obesity and hunger)

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

06/16/2022

lisocabtagene maraleucel (Breyanzi®)

Bristol-Myers Squibb

DLBCL (R/R, 2nd-line)

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

06/24/2022

luspatercept-aamt (Reblozyl®)

Merck/Bristol-Myers Squibb

β-thalassemia (nontransfusion dependent)

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

06/27/2022

ravulizumab-cwvz (Ultomiris)

AstraZeneca

PNH; Hemolytic uremic syndrome (atypical)

Submitted – sBLA; Orphan Drug

July 2022

viloxazine (Qelbree®) ®

27 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

fam-trastuzumab deruxtecan-nxki (Enhertu)

Daiichi Sankyo

NSCLC (unresectable or metastatic, HER2+, ≥ 2nd-line)

Submitted – sBLA; Breakthrough; Priority Review

Jul–Sep 2022

tocilizumab (Actemra®)

Genentech

COVID-19 treatment (hospitalized adults on systemic corticosteroids and require assisted ventilation)

Submitted – sBLA; Priority Review

Jul–Dec 2022

pneumococcal 15-valent conjugate vaccine (Vaxneuvance™)

Merck

Invasive pneumococcal disease prevention (ages 6 weeks–17 years)

Submitted – sBLA; Breakthrough Therapy; Priority Review

07/01/2022

pegloticase (Krystexxa®)

Horizon

Gout (in combination with IV methotrexate)

Submitted – sBLA; Orphan Drug; Priority Review

07/07/2022

ruxolitinib cream (Opzelura®)

Incyte

Vitiligo

Topical

Submitted – sNDA; Priority Review

07/18/2022

risdiplam (Evrysdi®)

Genentech

SMA (presynaptic, ages < 2 months)

Oral

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

07/25/2022

adalimumab-bwwd (Hadlima) 100 mg/mL (biosimilar to Abbvie’s Humira)

Organon/Samsung Bioepis

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

Submitted – sBLA

August 2022

dupilumab (Dupixent)

Regeneron/Sanofi

Eosinophilic esophagitis (ages ≥ 12 years)

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

08/03/2022

pimavanserin (Nuplazid®)

Acadia

Alzheimer’s diseaseOral related hallucinations and delusions

Submitted – sNDA; Breakthrough Therapy

08/04/2022

ustekinumab (Stelara®)

Janssen

PsA (ages ≥ 5 years)

Submitted – sBLA

08/08/2022

baricitinib (Olumiant)

Eli Lilly

Alopecia areata

Oral

Submitted – sNDA; Breakthrough Therapy; Priority Review

08/26/2022

ivosidenib (Tibsovo®)

Les Laboratoires Servier

AML (previously untreated IDH1-mutated)

Oral

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

September 2022

aprepitant (Cinvanti®)

Heron

Postoperative nausea and vomiting prophylaxis

Submitted – 505(b)(2) sNDA

09/16/2022

cemiplimab-rwlc (Libtayo®)

Regeneron

NSCLC (advanced, firstline, in combination with chemotherapy)

Submitted – sBLA

09/19/2022

adalimumab-afzb (Abrilada)(biosimilar to Abbvie’s Humira)

Pfizer

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

Submitted - PAS BLA; seeking biosimilar interchangeability

Oct–Dec 2022

ibalizumab (Trogarzo®) IV push formulation

Theratechnologies

HIV-1 infection treatment

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

10/06/2022

lumasiran (Oxlumo®)

Alnylam

Primary hyperoxaluria type 1 (advanced)

Submitted – sNDA; Breakthrough Therapy; Orphan Drug

10/06/2022

Pfizer-Biontech COVID-19 vaccine (Comirnaty®)

Pfizer/Biontech

COVID-19 prevention (ages 12–15 years)

Submitted – sBLA; Fast Track

10/14/2022

28 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

risankizumab-rzaa (Skyrizi®)

Abbvie

Submitted – sBLA; Orphan Drug

10/20/2022

zanubrutinib (Brukinsa®)

Beigene

CLL/SLL

Oral

Submitted – sNDA; Orphan Drug

10/22/2022

upadacitinib (Rinvoq™)

Abbvie

Axial spondyloarthritis (non-radiographic)

Oral

Submitted – sNDA

11/07/2022

cariprazine (Vraylar®)

Abbvie

MDD (adjunct)

Oral

Submitted – sNDA

12/22/2022

abaloparatide (Tymlos®)

Radius

Osteoporosis (men, highrisk for fracture)

Submitted – sNDA

12/23/2022

ibrutinib (Imbruvica®)

Abbvie

GVHD treatment (chronic, ages ≥ 1 years, ≥ 2ndline)

Oral

Submitted – sNDA; Breakthrough Therapy; Orphan Drug

12/28/2022

darolutamide (Nubeqa®)

Bayer

Prostate cancer (metastatic, hormonesensitive, in combination with docetaxel)

Oral

Submitted – sNDA

01/09/2023

baricitinib (Olumiant)

Eli Lilly

Atopic dermatitis (moderate-severe)

Oral

Submitted – sNDA

Pending

upadacitinib (Rinvoq)

Abbvie

Oral

Submitted – sNDA

Pending

Phase 3 (New Drugs) AAV-RPGR gene therapy

Janssen

Retinitis pigmentosa (X-linked)

Intraocular

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

abaloparatide-TD

Radius

Osteoporosis/osteopenia

Transdermal

Phase 3 – NDA

TBD

acoramidis

Bridgebio/ AstraZeneca

Transthyretin amyloid cardiomyopathy (ATTRCM)

Oral

Phase 3 – NDA

TBD

adintrevimab

Adagio

COVID-19

Phase 3 – BLA

TBD

adjuvanted, stabilized prefusion F protein subunit vaccine

GlaxoSmithKline

RSV prevention

Phase 3 – BLA; Fast Track

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Amgen

DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Sam Chun Dang

DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Samsung Bioepis/ Biogen

DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Santo/Formycon

DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

aloradine

Vistagen

Social anxiety disorder

Intranasal

Phase 3 – NDA; Fast Track

TBD

amcenestrant

Sanofi

Breast cancer

Oral

Phase 3 – NDA

TBD

amubarvimab + romlusevimab

Brii

COVID-19

Phase 3 – BLA

TBD

anthrax vaccine, adsorbed

Emergent

Anthrax infection

Phase 3 – BLA; Fast Track

TBD

anti-betv1 monoclonal antibodies (REGN-57135714-5715) 29 | MAGELLANRX.COM

Regeneron

Birch allergy

Phase 3 – BLA

TBD

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

apolipoprotein A-I (human)

CSL

Atherosclerosis

Phase 3 – BLA

TBD

arfolitixorin hemisulfate

Isofol

CRC

Phase 3 – NDA; Fast Track

TBD

arimoclomol

Orphazyme

Niemann-Pick disease

Oral

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

TBD

ataluren

PTC

DMD

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells

CRISPR Therapeutics

SCD; Thalassemia

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

TBD

balstilimab

Agenus

Cervical cancer (R/R, in combination with zalifrelimab)

Phase 3 – BLA; Fast Track

TBD

bamlanivimab

Eli Lilly

COVID-19

Phase 3 – BLA

TBD

bempegaldesleukin

Nektar/Bristol-Myers Squibb

Bladder cancer; RCC

Phase 3 – BLA

TBD

bentracimab

Phasebio/ AstraZeneca

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

Green Cross

Hemophilia A

Phase 3 – BLA

TBD

betibeglogene autotemcel (Zynteglo)

Bluebird Bio

SCD

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

TBD

bevacizumab-vikg

Outlook

DME; Retinal vein occlusion-associated macular edema

Intravitreal

Phase 3 – BLA

TBD

BIIB059

Biogen

SLE

Phase 3 – BLA

TBD

bimekizumab

UCB

Axial spondyloarthritis; Hidradenitis suppurativa; PsA

Phase 3 – BLA

TBD

bis-choline tetrathiomolybdate

AstraZeneca

Wilson’s disease

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

BPR277

Lifemax/Novartis

Congenital ichthyosis

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

brensocatib

Insmed/AstraZeneca

Bronchiectasis

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

cannabidiol transdermal gel

Zynerba

Fragile X syndrome

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

capsaicin

Centrexion

Osteoarthritis pain (knee)

Intraarticular

Phase 3 – NDA; Fast Track

TBD

ceftobiprole medocaril

Basilea

ABSSSI; CAP; HAP; Septicemia

Phase 3 – NDA; Fast Track; QIDP

TBD

ceftriaxone wearable micropump

scPharmaceuticals

Gram+/Gram- infection

Phase 3 – NDA

TBD

CM-AT (pancreatic enzyme)

Curemark

Autism spectrum disorder

Oral

Phase 3 – BLA; Fast Track

TBD

cobitolimod

Index/Merck

Rectal

Phase 3 – NDA; Orphan Drug

TBD

concizumab

Novo Nordisk

Hemophilia A and B

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

30 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

copper histidine

Zydus Cadila

Menkes disease

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

TBD

cosibelimab

Fortress

Cutaneous squamous cell carcinoma (metastatic)

Phase 3 – BLA

TBD

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

AstraZeneca

COVID-19

Phase 3 – BLA

TBD

COVID-19 vaccine (INO4800)

Inovio

COVID-19

Phase 3 – BLA

TBD

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

Janssen

COVID-19

Phase 3 – BLA

TBD

COVID-19 vaccine (MT2766)

Mitsubishi Tanabe/ GlaxoSmithKline

COVID-19

IM, SC

Phase 3 – BLA; Fast Track

TBD

COVID-19 vaccine (SP0253)

Sanofi/ GlaxoSmithKline

COVID-19

Phase 3 – BLA

TBD

crovalimab

Genentech

Hemolytic uremic syndrome; PNH

IV, SC

Phase 3 – BLA; Orphan Drug

TBD

dabocemagene autoficel

Castle Creek

Epidermolysis bullosa

Intradermal

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

TBD

daprodustat

GlaxoSmithKline

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

Oral

Phase 3 – NDA

TBD

darvadstrocel

Takeda

Phase 3 – BLA; Orphan Drug

TBD

delandistrogene moxeparvovec

Sarepta/Genentech

DMD

Phase 3 – BLA; Fast Track

TBD

dengue tetravalent vaccine

Takeda

Dengue fever

Phase 3 – BLA; Fast Track

TBD

dersimelagon

Mitsubishi Tanabe

Porphyria

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

despropyl macitentan

Janssen

Hypertension

Oral

Phase 3 – NDA

TBD

difluprednate XR

Sun

Ocular pain/inflammation

Ophthalmic

Phase 3 – NDA

TBD

donanemab

Eli Lilly

Alzheimer’s disease (early)

IV, SC

Phase 3 – BLA; Breakthrough Therapy

TBD

donaperminogene seltoplasmid

Helixmith

Diabetic foot ulcers (chronic non-healing)

Phase 3 – BLA

TBD

doravirine/islatravir

Merck

HIV-1 infection treatment

Oral

Phase 3 – NDA

TBD

dovitinib lactate

Allarity

Breast cancer; RCC (3rdline)

Oral

Phase 3 – NDA

TBD

durlobactam/sulbactam

Entasis

Acinetobacter baumannii infection

Phase 3 – NDA; Fast Track; QIDP

TBD

dust mite immunotherapy

Stallergenes Greer

Allergic rhinitis

Phase 3 – BLA

TBD

EB-101 (gene therapy)

Abeona

Epidermolysis bullosa

Surgical application

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

TBD

eculizumab (biosimilar to Alexion’s Soliris®)

Amgen

PNH

Phase 3 – BLA; Orphan Drug

TBD

efanesoctocog alfa

Sanofi

Hemophilia A

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

efgartigimod

Argenx

Myasthenia gravis; Pemphigus vulgaris

Phase 3 – BLA; Orphan Drug

TBD

31 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

elacestrant

Menarini

Breast cancer

Oral

Phase 3 – NDA; Fast Track

TBD

elamipretide

Stealth

Barth syndrome

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

enmetazobactam

Allecra

UTI (complicated)

Phase 3 – NDA; Fast Track; QIDP

TBD

ensifentrine

Verona

COPD

Inhaled

Phase 3 – NDA

TBD

ensovibep

Novartis/Molecular Partners

COVID-19

Phase 3 – BLA; Fast Track

TBD

epcoritamab

Genmab/Abbvie

DLBCL

Phase 3 – BLA

TBD

epinephrine

Bryn

Anaphylaxis

Intranasal

Phase 3 – NDA; Fast Track

TBD

eplontersen

Ionis/AstraZeneca

Transthyretin amyloid polyneuropathy

Phase 3 – NDA; Orphan Drug

TBD

eprenetapopt

Aprea

Myelodysplastic syndrome

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

TBD

esreboxetine

Axsome/Pfizer

Fibromyalgia

Oral

Phase 3 – NDA

TBD

etavopivat

Forma

SCD

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

etesevimab

Lonza/Eli Lilly

COVID-19

Phase 3 – BLA

TBD

etranacogene dezaparvovec

CSL

Hemophilia B

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

etrasimod

Arena

Oral

Phase 3 – NDA; Orphan Drug

TBD

fasinumab

Regeneron

Osteoarthritis pain (knee)

Phase 3 – BLA

TBD

fezolinetant

Astellas

Menopause vasomotor symptoms

Oral

Phase 3 – NDA

TBD

fidanacogene elaparvovec

Pfizer/Genentech

Hemophilia B

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

filgotinib

Gilead

CD; UC

Oral

Phase 3 – NDA

TBD

firibastat

Quantum Genomics

Hypertension (systemic)

Oral

Phase 3 – NDA

TBD

firmacute eubacterial spores

Seres

C. difficile-associated 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

FVIII mimetic bi-specific antibody

Novo Nordisk

Hemophilia A

Phase 3 – BLA

TBD

gantenerumab

Genentech

Alzheimer’s disease (early)

Phase 3 – BLA; Breakthrough Therapy

TBD

garadacimab

CSL

HAE

Phase 3 – BLA; Orphan Drug

TBD

gavorestat

Applied Therapeutics Galactosemia

Oral

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

TBD

gepotidacin

GlaxoSmithKline

Oral

Phase 3 – NDA; QIDP

TBD

32 | MAGELLANRX.COM

UTI (uncomplicated)

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

giredestrant

Genentech

Breast Cancer

Oral

Phase 3 – NDA; Fast Track

TBD

giroctocogene fitelparvovec

Pfizer

Hemophilia A

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

TBD

glatiramer acetate depot

Viatris

Phase 3 – 505(b)(2) NDA

TBD

glofitamab

Genentech

DLBCL

Phase 3 – BLA

TBD

gold nanocrystal

Clene

ALS

Oral

Phase 3 – NDA; Orphan Drug

TBD

hypericin

Soligenix

Cutaneous T cell lymphoma (CTCL)

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

idursulfase

Takeda/Sanofi

Mucopolysaccharidosis II Intrathecal (MPS II; Hunter syndrome)

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

inavolisib

Genentech

Breast cancer

Oral

Phase 3 – NDA

TBD

inclacumab

Global Blood Therapeutics

SCD

Phase 3 – BLA

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

Prostate cancer

Oral

Phase 3 – NDA

TBD

iptacopan

Novartis

Complement 3 (C3) glomerulopathy; Hemolytic uremic syndrome; Immunoglobulin A nephropathy (Berger’s disease); PNH

Oral

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

TBD

itepekimab

Regeneron

COPD

Phase 3 – BLA

TBD

Lactobacillus reuteri

Infant Bacterial Therapeutics

Necrotizing enterocolitis

Oral

Phase 3 – BLA; Orphan Drug

TBD

lanifibranor

Inventiva

NASH

Oral

Phase 3 – NDA; Breakthrough Therapy; Fast Track

TBD

lazertinib

Genosco

NSCLC

Oral

Phase 3 – NDA

TBD

lebrikizumab

Eli Lilly

Atopic dermatitis (moderate-severe)

Phase 3 – BLA; Fast Track

TBD

lecanemab

Eisai

Alzheimer’s disease (early)

Phase 3 – BLA; Breakthrough Therapy; Fast Track

TBD

lenadogene nolparvovec (GS010)

Gensight

Leber’s hereditary optic neuropathy

Intravitreal

Phase 3 – BLA; Orphan Drug

TBD

leniolisib

Pharming/Novartis

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

levodopa/carbidopa patch pump

Mitsubishi Tanabe

Parkinson’s disease

Phase 3 – 505(b)(2) NDA

TBD

33 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

ligelizumab

Novartis

Urticaria

Phase 3 – BLA; Breakthrough Therapy

TBD

linrodostat

Bristol-Myers Squibb

Bladder cancer

Oral

Phase 3 – NDA

TBD

lorecivivint

Biosplice

Osteoarthritis pain (knee)

Intraarticular

Phase 3 – NDA

TBD

lotilaner

Tarsus

Demodex blepharitis

Ophthalmic

Phase 3 – NDA

TBD

magrolimab

Gilead

Myelodysplastic syndrome

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

TBD

marstacimab

Pfizer

Hemophilia A and B

IV, SC

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

masitinib

AB Science

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

Oral

Phase 3 – NDA; Orphan Drug

TBD

mavorixafor

Warts, Oral hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome

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

TBD

melphalan

Delcath

Uveal melanoma (hepatic-dominant)

Percutaneous hepatic perfusion

Phase 3 – NDA

TBD

meningococcal vaccine

GlaxoSmithKline

Meningococcal immunization

Phase 3 – BLA

TBD

meningococcal vaccine

Pfizer

Meningococcal immunization

Phase 3 – BLA

TBD

metachromatic leukodystrophy gene therapy

Orchard

Metachromatic leukodystrophy

Phase 3 – BLA; Orphan Drug; RMAT

TBD

midomafetamine

Multidisciplinary Association for Psychedelic Studies

PTSD

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

minocycline/edetate/ethyl alcohol

Citius

Catheter-related bloodstream infection (CRBSI)

Phase 3 – NDA; Fast Track; QIDP

TBD

mirikizumab

Eli Lilly

CD; UC

IV, SC

Phase 3 – BLA

TBD

mitapivat

Agios

SCD; Thalassemia

Oral

Phase 3 – NDA; Orphan Drug

TBD

molnupiravir

Merck

COVID-19

Oral

Phase 3 – NDA

TBD

momelotinib

Sierra Oncology/ Gilead

Myelofibrosis

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

mosunetuzumab

Genentech

Follicular lymphoma (3rdline)

IV, SC

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

motixafortide

Biolinerx

Stem cell mobilization

Phase 3 – NDA; Orphan Drug

TBD

nabiximols

Jazz

MS-related spasticity

Oral transmucosal

Phase 3 – NDA

TBD

nalbuphine ER

Trevi

Pruritus

Oral

Phase 3 – NDA; Fast Track

TBD

naloxone

Orexo

Opioid overdose

Intranasal

Phase 3 – 505(b)(2) NDA

TBD

naloxone hydrochloride dihydrate

Elorac

Pruritus

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

34 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

narsoplimab

Omeros

Hemolytic uremic syndrome

IV, SC

Phase 3 – BLA; Fast Track

TBD

natalizumab (biosimilar to Biogen’s Tysabri®)

Novartis

Phase 3 – BLA

TBD

navitoclax

Abbvie/Genentech

Myelofibrosis

Oral

Phase 3 – NDA; Orphan Drug

TBD

nedosiran

Novo Nordisk

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

nirsevimab

AstraZeneca

RSV prevention

Phase 3 – BLA; Breakthrough Therapy; Fast Track

TBD

nomacopan

Akari

Hemolytic uremic syndrome; HSCTassociated thrombotic microangiopathy; PNH

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

olezarsen

Akcea

Familial chylomicronemia syndrome

Phase 3 – NDA

TBD

OPT-302

Opthea

Wet AMD

Intravitreal

Phase 3 – BLA; Fast Track

TBD

OTL-103

Orchar

Wiskott-Aldrich syndrome IV

Phase 3 – BLA; Orphan Drug; RMAT

TBD

oxalobacter formigenes

Oxthera

Hyperoxaluria

Oral

Phase 3 – BLA; Orphan Drug; Rare Pediatric Disease

TBD

padeliporfin

Steba

Bladder cancer

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

palopegteriparatide

Ascendis

Hypoparathyroidism

Phase 3 – BLA; Orphan Drug

TBD

palovarotene

Ipsen

Fibrodysplasia ossificans progressiva

Oral

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

TBD

pamrevlumab

Fibrogen/BristolMyers Squibb

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

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

TBD

pegadricase

Swedish Orphan Biovitrum

Gout

Phase 3 – BLA

TBD

pegcetacoplan

Apellis

Dry AMD

Intravitreal

Phase 3 – NDA; Fast Track

TBD

pemafibrate

Kowa

Dry eye disease (associated with meibomian gland dysfunction)

Oral

Phase 3 – NDA

TBD

perfluorohexyloctane

Bausch

Dry eye disease

Ophthalmic

Phase 3 – NDA

TBD

pirtobrutinib

Eli Lilly

Mantle cell lymphoma

Oral

Phase 3 – NDA

TBD

plinabulin

Beyondspring

NSCLC

Phase 3 – NDA

TBD

plonmarlimab

I-Mab

COVID-19

Phase 3 – BLA

TBD

pollinex quattro grass

Allergy Therapeutics

Allergic rhinitis

Phase 3 – BLA

TBD

pollinex quattro ragweed

Allergy Therapeutics

Allergic rhinitis

Phase 3 – BLA

TBD

35 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

potassium citrate/ potassium bicarbonate

Advicenne

Renal tubular acidosis

Oral

Phase 3 – 505(b)(2) NDA

TBD

pozelimab

Regeneron

Paroxysmal nocturnal hemoglobinuria; Chaple disease

IV, SC

Phase 3 – BLA; Orphan Drug

TBD

pritelivir

Aicuris Anti-infective Cures

Herpes simplex virus treatment

Oral

Phase 3 – NDA; Breakthrough Therapy; Fast Track

TBD

proxalutamide

Kintor

COVID-19

Oral

Phase 3 – NDA

TBD

rabies monoclonal antibody cocktail

Sanofi/Janssen

Rabies treatment

Phase 3 – BLA; Fast Track

TBD

ralinepag

United Therapeutics

PAH

Oral

Phase 3 – NDA; Orphan Drug

TBD

ranibizumab (biosimilar to Genentech’s Lucentis)

Stada Arzneimittel/ Bausch

Wet AMD; Macular edema following RVO; Myopic choroidal neovascularization (mCNV)

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

relacorilant

Corcept

Cushing’s syndrome

Oral

Phase 3 – NDA; Orphan Drug

TBD

resmetirom

Madrigal

NASH

Oral

Phase 3 – NDA; Fast Track

TBD

reproxalap

Aldeyra

Allergic conjunctivitis; Dry eye disease

Ophthalmic

Phase 3 – NDA

TBD

rezafungin

Cidara

Candidemia/invasive candidiasis

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

ridinilazole

Summit

C. difficile-associated diarrhea

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

ritlecitinib

Pfizer

Alopecia areata

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

rituximab (biosimilar to Genentech’s Rituxan)

Archigen

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

Phase 3 – BLA

TBD

roflumilast

Arcutis/AstraZeneca

Atopic dermatitis

Topical

Phase 3 – NDA

TBD

rogaratinib

Bayer

Bladder cancer

Oral

Phase 3 – NDA

TBD

roluperidone

Minerva Neurosciences

Schizophrenia

Oral

Phase 3 – NDA

TBD

ropeginterferon alfa-2b

Pharmaessentia

Essential thrombocythemia

Phase 3 – BLA; Orphan Drug

TBD

roxadustat

AstraZeneca

Anemia due to cytotoxic chemotherapy

Oral

Phase 3 – NDA

TBD

rozanolixizumab

UCB

Myasthenia gravis

Phase 3 – BLA; Orphan Drug

TBD

RSV vaccine (JNJ64400141)

Janssen

RSV prevention

Phase 3 – BLA; Breakthrough Therapy

TBD

RSV vaccine (PF06928316)

Pfizer

RSV prevention

Injectable

Phase 3 – BLA; Breakthrough Therapy; Fast Track

TBD

36 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

ruxolitinib (deuterated)

Concert

Alopecia areata

Oral

Phase 3 – NDA; Breakthrough Therapy; Fast Track

TBD

sabatolimab

Novartis

Myelodysplastic syndrome

Phase 3 – BLA; Fast Track

TBD

sabizabulin

Veru

COVID-19 treatment

Oral

Phase 3 – NDA; Fast Track

TBD

seasonal influenza nanoparticle vaccine

Novavax

Seasonal influenza prevention

Phase 3 – BLA; Fast Track

TBD

seladelpar

Cymabay/Janssen

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

Chronic HFpEF; Heart failure in patients with T2DM

Oral

Phase 3 – NDA

TBD

sotatercept

Merck/Bristol-Myers Squibb

PAH

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

sparsentan

Travere/BristolMyers Squibb

Focal segmental glomerulosclerosis

Oral

Phase 3 – NDA; Orphan Drug

TBD

SPK-8011

Genentech

Hemophilia A

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

tabelecleucel

Atara

Epstein-Barr virus-associated post-transplant lymphoproliferative disease

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

TAK-755

Takeda

Thrombotic thrombocytopenic purpura (TTP)

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

tanfanercept

Hanall

Dry eye disease

Ophthalmic

Phase 3 – BLA

TBD

tapinarof

Roivant

Atopic dermatitis

Topical

Phase 3 – NDA

TBD

tarcocimab tedromer

Kodiak

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

Intravitreal

Phase 3 – BLA

TBD

tecarfarin

Espero

Anticoagulation

Oral

Phase 3 – NDA

TBD

tiragolumab

Genentech

Esophageal cancer; NSCLC

Phase 3 – BLA; Orphan Drug

TBD

tiratricol

Rare Thyroid Therapeutics

Resistance to thyroid hormone type beta (RTH-b)

Oral

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

TBD

tirzepatide

Eli Lilly

Obesity

Phase 3 – NDA

TBD

tislelizumab

Beigene/Novartis

Gastric cancer; HCC; NSCLC

Phase 3 – BLA; Orphan Drug

TBD

tixagevimab + cilgavimab (Evusheld™)

AstraZeneca

COVID-19

Phase 3 – BLA

TBD

tocilizumab (biosimilar to Genentech’s Actemra)

Biogen

Phase 3 – BLA

TBD

37 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

tocilizumab (biosimilar to Genentech’s Actemra)

Fresenius/Merck

Phase 3 – BLA

TBD

tofersen

Biogen

ALS

Intrathecal

Phase 3 – NDA; Orphan Drug

TBD

tominersen

Genentech

Huntington’s disease

Intrathecal

Phase 3 – NDA; Orphan Drug

TBD

tradipitant

Vanda/Eli Lilly

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

Oral

Phase 3 – NDA

TBD

travoprost implant

Glaukos

Glaucoma/ocular hypertension

Intraocular

Phase 3 – 505(b)(2) NDA

TBD

trofinetide

Acadia

Rett syndrome

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

tusamitamab ravtansine

Sanofi

NSCLC

Phase 3 – BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Amgen

PSO

IV, SC

Phase 3 – BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Formycon

PSO

IV, SC

Phase 3 – BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Hikma

PSO

IV, SC

Phase 3 – BLA

TBD

valoctocogene roxaparvovec

Biomarin

Hemophilia A

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

TBD

venglustat

Sanofi

Gaucher’s disease; GM2 gangliosidoses

Oral

Phase 3 – NDA; Orphan Drug

TBD

verbrinacogene setparvovec

Freeline

Hemophilia B

Phase 3 – BLA; Orphan Drug; RMAT

TBD

VGX-3100 therapeutic vaccine

Inovio

Cervical dysplasia (human IM papillovirus-positive)

Phase 3 – BLA

TBD

visomitin

Mitotech

Dry eye disease

Ophthalmic

Phase 3 – NDA

TBD

wilfactin

LFB

Von Willebrand disease

Phase 3 – BLA; Orphan Drug

TBD

zavegepant

Biohaven/BristolMyers Squibb

COVID-19; Migraine treatment & prevention

Intranasal, Oral

Phase 3 – NDA

TBD

zilucoplan

UCB

Myasthenia gravis

Phase 3 – NDA; Orphan Drug

TBD

zolbetuximab

Astellas

Gastric cancer

Phase 3 – BLA; Orphan Drug

TBD

zoliflodacin

Entasis

Gonorrhea

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

zuranolone

Sage

MDD; Postpartum depression

Oral

Phase 3 – NDA; Breakthrough Therapy; Fast Track

TBD

Phase 3 (Supplementals) adalimumab (Hulio; biosimilar to Abbvie’s Humira) baricitinib (Olumiant) benralizumab (Fasenra ) ®

38 | MAGELLANRX.COM

Viatris/Biocon

Hidradenitis suppurativa; Uveitis

Phase 3 – sBLA

TBD

Eli Lilly

JIA; Uveitis

Oral

Phase 3 – sNDA

TBD

AstraZeneca

ANCA-associated vasculitis; Bullous pemphigoid; Esophagitis

Phase 3 – sBLA; Orphan Drug

TBD

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

brexpiprazole (Rexulti®)

Otsuka

PTSD

Oral

Phase 3 – sNDA

TBD

dupilumab (Dupixent)

Sanofi

Allergic fungal rhinosinusitis; Bullous pemphigoid; COPD; Pruritus; Urticaria

Phase 3 – sBLA; Orphan Drug

TBD

efgartigimod (Vyvgart®)

Argenx

ITP

Phase 3 – sBLA; Orphan Drug

TBD

empagliflozin (Jardiance®)

Boehringer Ingelheim

Diabetic nephropathy

Oral

Phase 3 – sNDA

TBD

ferric carboxymaltose (Injectafer®)

Daiichi Sankyo

Anemia in heart failure

Phase 3 – sNDA

TBD

ferric derisomaltose (Monoferric®)

Pharmacosmos

Anemia in heart failure

Phase 3 – sNDA

TBD

fostamatinib (Tavalisse®)

Rigel

Autoimmune hemolytic anemia

Oral

Phase 3 – sNDA; Fast Track; Orphan Drug

TBD

guselkumab (Tremfya®)

Janssen

Phase 3 – sBLA

TBD

hydrogen peroxide (Eskata®)

Aclaris

Warts

Topical

Phase 3 – sNDA

TBD

immune globulin intravenous, human 10% (Octagam®)

Octapharma

COVID-19

Phase 3 – sBLA

TBD

inebilizumab-cdon (Uplizna®)

Horizon

Myasthenia gravis

Phase 3 – sBLA

TBD

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

Evofem

Chlamydia trachomatis infection; Neisseria gonorrhoeae infection

Intravaginal

Phase 3 – sNDA; Fast Track; QIDP

TBD

mepolizumab (Nucala®)

GlaxoSmithKline

COPD

Phase 3 – sBLA

TBD

meropenem/vaborbactam (Vabomere®)

Melinta

Bacteremia; HAP

Phase 3 – sNDA; QIDP

TBD

nirmatrelvir/ritonavir (Paxlovid™)

Pfizer

COVID-19 post-exposure prophylaxis; COVID-19 treatment

Oral

Phase 3 – sNDA

TBD

Lupin

COVID-19; Influenza

Oral

Phase 3 – sNDA

TBD

obeticholic acid (Ocaliva )

Intercept

NASH

Oral

Phase 3 – sNDA; Breakthrough Therapy

TBD

odevixibat (Bylvay™)

Albireo

Alagille syndrome-related Oral cholestatic pruritus

Phase 3 – sNDA; Orphan Drug

TBD

omalizumab (Xolair®)

Genentech

Food allergies

Phase 3 – sBLA; Breakthrough Therapy

TBD

patisiran (Onpattro®)

Alnylam

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

Phase 3 – sNDA; Orphan Drug

TBD

pegylated liposomal irinotecan (Onivyde®)

Ipsen

SCLC

Phase 3 – sNDA; Fast Track; Orphan Drug

TBD

ravulizumab-cwvz (Ultomiris)

Alexion

Pachyonychia congenita

IV, SC

Phase 3 – sBLA

TBD

risankizumab-rzaa (Skyrizi)

Abbvie

IV, SC

Phase 3 – sBLA

TBD

rituximab-arrx (biosimilar to Genentech’s Rituxan) (Riabni)

Amgen

Phase 3 – sBLA

TBD

rivaroxaban (Xarelto®)

Janssen

COVID-19

Oral

Phase 3 – sNDA

TBD

nitazoxanide (Alinia®) ®

39 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

romiplostim (Nplate®)

Amgen

Chemotherapy-induced thrombocytopenia

Phase 3 – sBLA; Orphan Drug

TBD

secukinumab (Cosentyx®)

Novartis

Hidradenitis suppurativa

Phase 3 – sBLA

TBD

tezepelumab-ekko (Tezspire™)

AstraZeneca

Nasal polyposis

Phase 3 – sBLA

TBD

ticagrelor (Brilinta)

AstraZeneca

SCD

Oral

Phase 3 – sNDA

TBD

upadacitinib (Rinvoq)

Abbvie

CD; Giant cell arteritis

Oral

Phase 3 – sNDA

TBD

Complete Response Letter (CRL) NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

bardoxolone methyl

Reata

Alport syndrome-related CKD

Oral

CRL

TBD

benralizumab (Fasenra)

AstraZeneca

Chronic rhinosinusitis with nasal polyps (CRSwNP)

CRL

TBD

episalvan

Amryt

Epidermolysis bullosa

Topical

CRL

TBD

immune globulin (human) 10%

Green Cross

PHI

CRL

TBD

lenacapavir

Gilead

HIV-1 infection treatment (MDR)

Oral, SC

CRL

TBD

risperidone ER

Teva

Schizophrenia

CRL

TBD

sintilimab

Eli Lilly

NSCLC (metastatic, nonsquamous, without EGFR or ALK mutations)

CRL

TBD

terlipressin

Mallinckrodt

Hepatorenal syndrome

CRL

TBD

vadadustat

Akebia/Otsuka

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

Oral

CRL

TBD

zolmitriptan microneedle system

Zosano

Migraine treatment; Cluster headache treatment

Transdermal

CRL

TBD

40 | MAGELLANRX.COM

GLOSSARY 6MWT 6 Minute Walking Test

CF Cystic Fibrosis

ABSSSI Acute Bacterial Skin and Skin Structure Infection

CHF Congestive Heart Failure

ACEI Angiotensin-Converting Enzyme Inhibitor

CKD Chronic Kidney Disease

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

CI Confidence Interval CLL Chronic Lymphocytic Leukemia CML Chronic Myeloid Leukemia CNS Central Nervous System

ACR70 American College of Rheumatology 70% Improvement

COPD Chronic Obstructive Pulmonary Disease

ADC Antibody-Drug Conjugate

CRC Colorectal Cancer

ADHD Attention Deficit Hyperactivity Disorder

CRL Complete Response Letter

ADL Activities of Daily Living

CRR Complete Response Rate

AED Anti-Epileptic Drug

CSF Colony Stimulating Factor

ALK Anaplastic Lymphoma Kinase

CV Cardiovascular

ALL Acute Lymphoblastic Leukemia

CVD Cardiovascular Disease

ALS Amyotrophic Lateral Sclerosis ALT Alanine Transaminase

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

AMD Age-Related Macular Degeneration

DEA Drug Enforcement Administration

AML Acute Myeloid Leukemia

DLBCL Diffuse Large B Cell Lymphoma

ANCA Antineutrophil Cytoplasmic Antibodies

DMARD Disease Modifying Antirheumatic Drug

ANDA Abbreviated New Drug Application

DMD Duchenne Muscular Dystrophy

ARB Angiotensin II Receptor Blocker

DME Diabetic Macula Edema

ARNI Angiotensin Receptor II Blocker – Neprilysin Inhibitor

DMT Disease Modifying Therapy

ART Antiretroviral Therapy

DOR Duration of Response

ARV Antiretroviral

DPI Dry Powder for Inhalation

AS Ankylosing Spondylitis

DPP-4 Dipeptidyl Peptidase 4

ASCVD Atherosclerotic Cardiovascular Disease

DR Delayed-Release

AST Aspartate Aminotransferase BCVA Best Corrected Visual Acuity

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

BLA Biologics License Application

ECOG Eastern Cooperative Oncology Group

BMI Body Mass Index

EDSS Expanded Disability Status Scale

BSA Body Surface Area

eGFR estimated Glomerular Filtration Rate

BsUFA Biosimilar User Fee Act

EGFR Epidermal Growth Factor Receptor

CABP Community Acquired Bacterial Pneumonia

ER Extended-Release

CAP Community Acquired Pneumonia

ESRD End-Stage Renal Disease

CAR T Chimeric Antigen Receptor T Cell

EUA Emergency Use Authorization

CD Crohn's Disease

FDA Food and Drug Administration

CDC Centers for Disease Control and Prevention

FH Familial Hypercholesterolemia

41 | MAGELLANRX.COM

COVID-19 Coronavirus Disease 2019

DNA Deoxyribonucleic Acid

GLOSSARY continued FLT3 FMS-Like Tyrosine Kinase-3

ITT Intent-To-Treat

G-CSF Granulocyte Colony Stimulating Factor

IV Intravenous

GI Gastrointestinal

JIA Juvenile Idiopathic Arthritis

GIST Gastrointestinal Stromal Tumor

LDL Low-Density Lipoprotein

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

LDL-C Low-Density Lipoprotein Cholesterol

GM-CSF Granulocyte-Macrophage Colony Stimulating Factor

LVEF Left Ventricular Ejection Fraction

GVHD Graft Versus Host Disease

MACE Major Adverse Cardiovascular Events

H Half HAART Highly Active Antiretroviral Therapy HAM-D Hamilton Depression Rating Scale HAP Healthcare-Associated Pneumonia Hb Hemoglobin HbA1c Hemoglobin A1c HBV Hepatitis B Virus HCC Hepatocellular Carcinoma HCP Healthcare Professional HCV Hepatitis C Virus HER Human Epidermal Growth Factor Receptor HER2 Human Epidermal Growth Factor Receptor 2 HF Heart Failure HFA Hydrofluoroalkane HFpEF Heart Failure with preserved Ejection Fraction HIT Heparin Induced Thrombocytopenia HIV Human Immunodeficiency Virus HIV-1 Human Immunodeficiency Virus-1 HPV Human Papilloma Virus HR Hazard Ratio HSCT Hematopoietic Stem Cell Transplant HTN Hypertension IBS Irritable Bowel Syndrome IBS-C Irritable Bowel Syndrome, Constipation Predominant IGA Investigator's Global Assessment IL-12 Interleukin-12 IL-17 Interleukin-17 IL-23 Interleukin-23 IM Intramuscular IRB Internal Review Board ITP Immune Thrombocytopenic Purpura

42 | MAGELLANRX.COM

mAb Monoclonal Antibody MADRS Montgomery – Åsberg Depression Rating Scale MAOI Monoamine Oxidase Inhibitor MDD Major Depressive Disorder MDI Metered Dose Inhaler MDR Multi-Drug Resistant mITT modified Intent-To-Treat 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 NIAID National Institute of Allergy and Infectious Diseases NSAID Non-Steroidal Anti-Inflammatory Drug NSCLC Non-Small Cell Lung Cancer NYHA New York Heart Association ODT Orally Disintegrating Tablet OR Odds Ratio ORR Overall/Objective Response Rate OS Overall Survival OTC Over-the-Counter PAH Pulmonary Arterial Hypertension PARP Poly(ADP-ribose) Polymerase PAS Prior Approval Supplement PASI Psoriasis Area and Severity Index PASI 50 Psoriasis Area and Severity Index 50% Reduction PASI 75 Psoriasis Area and Severity Index 75% Reduction PASI 90 Psoriasis Area and Severity Index 90% Reduction PASI 100 Psoriasis Area and Severity Index 100% Reduction PCI Percutaneous Coronary Intervention PCSK9 Proprotein Convertase Subtilisin Kexin 9

GLOSSARY continued PD-1 Programmed Death Protein 1

sNDA supplemental New Drug Application

PD-L1 Programmed Death-Ligand 1

SNRI Serotonin and Norepinephrine Reuptake Inhibitor

PDUFA Prescription Drug User Fee Application PFS Progression-Free Survival PGA Physician Global Assessment PHI Primary Humoral Immunodeficiency PNH Paroxysmal Nocturnal Hemoglobinuria PsA Psoriatic Arthritis

SOC Standard of Care sPGA static Physician Global Assessment SR Sustained-Release SSRI Selective Serotonin Reuptake Inhibitor SSSI Skin and Skin Structure Infection

PSO Plaque Psoriasis

T1DM Type 1 Diabetes Mellitus

PTCA Percutaneous Transluminal Coronary Angioplasty

T2DM Type 2 Diabetes Mellitus TBD To Be Determined

PTSD Post-Traumatic Stress Disorder

TEAE Treatment-Emergent Adverse Event

Q Quarter

TNBC Triple Negative Breast Cancer

QIDP Qualified Infectious Diseases Product

TNF Tumor Necrosis Factor

QOL Quality of Life

TNFα Tumor Necrosis Factor-alpha

R/R Relapsed or Refractory

UA Unstable Angina

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

UC Ulcerative Colitis

RA Rheumatoid Arthritis

UTI Urinary Tract Infection

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 RVO Retinal Vein Occlusion SARS-CoV-2 Severe Acute Respiratory SyndromeAssociated Coronavirus-2 sBLA supplemental Biologics License Application SC Subcutaneous SCCHN Squamous Cell Cancer of the Head and Neck SCD Sickle Cell Disease SCLC Small Cell Lung Cancer SCT Stem Cell Transplant SGLT2 Sodium-Glucose Co-Transporter 2 SL Sublingual SLE Systemic Lupus Erythematosus SLL Small Lymphocytic Lymphoma

43 | MAGELLANRX.COM

US United States VAS Visual Analog Scale VEGF Vascular Endothelial Growth Factor VTE Venous Thromboembolism WBC White Blood Cell WHO World Health Organization XR Extended-Release

MRx PIPELINE A VIEW INTO UPCOMING SPECIALTY & TRADITIONAL DRUGS

JANUARY 2022