Specialty Drug Pipeline Report

Jennifer Schonhorst, Pharm.D.

The PerformRx Pipeline Report provides a summary of potentially high-impact new medications and new indications that may be approved by the Food and Drug Administration in the next six months. It includes medications relevant to pharmacy and medical benefits. It is not inclusive of all new medications and new indications. For example, medications used for surgical purposes, such as general anesthetics, would not appear in this report. Medications are grouped by disease category and sorted alphabetically within each category.

The information contained in this report is compiled from independent third-party sources (see Bibliography) and is provided for informational purposes only. This information should not be solely relied upon for decisionmaking purposes. Prospective drug launch dates are estimates based on market information. PerformRx does not guarantee any dates and the actual release dates are subject to change without notice. PerformRx makes no endorsement, express or implied, of any product, service, or information, by its inclusion or exclusion from this document. For questions pertaining to this report, please contact Lauren Megargell, Director of Clinical Services, at lmegargell@performrx.com

DERMATOLOGY

ENDOCRINE

Lantidra (donislecel)

Ocaliva (Obeticholic Acid)

HEMATOLOGY

Jesduvroq (daprodustat)

Inc.

Pharmaceuticals, Inc.

Mellitus, Type I

Kline plc

Due to Chronic Renal Failure, DialysisIndependent

HEMATOLOGY continued

Ex-vivo gene therapy; potential first gene therapy approved for treatment of SCD.

Novel therapy. Targets mannan-binding lectinassociated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system.

Roctavian (valoctocogene roxaparvovec)

Infectious Disease

Evusheld (tixagevimab/ cilgavimab)

Being developed for people with limited treatment options. Novel mechanism antifungal therapy from the newly discovered orotomide class.

Single dose option and seeking indication for broader infant population vs solely preterm infants or those with high-risk conditions.

Would be the first fully approved therapy for COVID-19 treatment in non-hospitalized patients. Ronapreve (casirivimab/ imdevimab)

Would be the first approved therapy for COVID-19 post-exposure prophylaxis. No other products, including those Emergency Use Authorized, are indicated for post-exposure prophylaxis. METABOLIC

Two component therapy consisting of the rhGAA cipaglucosidase and particularly bis-phosphorylated mannose-6 phosphate (bis-M6P) glycans, to enhance cellular uptake; it is administered in combination with oral Zavesca (miglustat).

Delandistrogene moxeparvovec (SRP-9001)

Eplontersen Ionis Pharmaceuticals, Inc. Hereditary Transthyretin (hATTR) Amyloidosis with Polyneuropathy

Livmarli (maralixibat)

Pharmaceuticals, Inc.

Familial Intrahepatic Cholestasis

Gene therapy which delivers micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein.

METABOLIC continued

Palovarotene Ipsen SA Fibrodysplasia Ossificans Progressiva (FOP)

NEUROLOGY

Selective RARγ agonist for the prevention of heterotopic ossification (new bone formation) as a treatment option for individuals living with FOP.

PO

NDA Fast Track Orphan

Duchenne Muscular Dystrophy (DMD) 10/26/2023

Ischemic

Stroke IV

Disease

NurOwn BrainStorm Cell Therapeutics Inc. NDA

Avasopasem Manganese Galera Therapeutics, Inc.

Radiotherapyinduced severe oral mucositis in patients being treated for head and neck cancer (HNC)

Dasynoc (dasatinib) Xspray Pharma AB Chronic Myelogenous Leukemia (CML)

Eflornithine/ Sulindac Mallinckrodt plc Familial Adenomatous Polyposis (FAP)

IV IT

Fast Track Orphan Rare Disease 07/07/2023

IV NDA Breakthrough Fast Track

08/09/2023 First in class agent for treatment of severe oral mucositis. Reduces elevated levels of superoxide caused by radiation therapy by rapidly converting superoxide to hydrogen peroxide and oxygen.

PO NDA 505b2 On or before 06/30/2023

PO NDA Fast Track Orphan On or before 06/30/2023 patients with relapsed or refractory disease. Fruquintinib Takeda Pharmaceutical Co. Ltd. Colorectal Cancer PO NDA Fast Track 11/30/2023 A highly selective and potent oral inhibitor of VEGFR-1, -2 and -3. Seeking indication for refractory metastatic colorectal cancer. Glofitamab Roche Holding AG Diffuse Large B-Cell Lymphoma (DLBCL) - NHL IV BLA Rare Disease 07/01/2023 Seeking indication for relapsed/refractory dis- after antibody. engaging lymphocytes harvested from a patient's metastatic melanoma tumors, expanded in vitro, and infused back into the patient to attack the tumors. two or more lines of systemic therapy. 06/16/2023 Alternative therapeutic option. SUMMER 2023 PERFORMRX REVIEW 19

Bioequivalent to Sprycel with 30% lower dose.

Combination of eflornithine and sulindac. Eflornithine inhibits ornithine decarboxylase, reducing polyamine synthesis and destabilizing DNA to reduce cell growth. Sulindac is an NSAID that inhibits COX and prostaglandin synthesis and reduces inflammation.

ONCOLOGY continued

Aphexda (motixafortide)

Hepzato (melphalan hydrochloride for Injection/ Hepatic Delivery System)

Ltd.

Nirogacestat SpringWorks Therapeutics Inc.

Quizartinib Daiichi Sankyo Co., Ltd.

Repotrectinib Bristol Myers Squibb

Marrow Transplant and Stem Cell

Acute Myeloid Leukemia (AML)

Cell Lung Cancer (NSCLC)

Talquetamab Johnson & Johnson Multiple Myeloma (relapsed/ refractory)

A highly selective inverse agonist of chemokine (C-X-C motif) receptor 4, used to mobilize stem cells for bone marrow transplant in multiple myeloma patients.

Regionalized approach; administered via hepatic artery and the venous effluent of the liver is collected and filtered using a percutaneously placed catheter and filtration system. Submitted for approval of unresectable hepatic-dominant metastatic ocular melanoma.

A potential first-in-class and first-in-disease agent for desmoid tumors.

Targets the kinase FLT3, which is mutated and constitutively activated in 25-40% of AML patients and associated with poor prognosis.

Targeting treatment of patients with ROS1positive locally advanced or metastatic NSCLC.

Bispecific T-cell engager. Targets novel GPRC5D which is expressed on some normal cells but overexpressed on myeloma cells, and separately targets CD3.

Alternative therapeutic option. Designed to minimize binding to FcγR on macrophages which is theorized to improve efficacy relative to products lacking this feature.

BLA — biologics license application; ID — intradermal; IM — intramuscular; IN — intranasal; INS — instillation; INV—intravesical; IO — intraocular; IT—intrathecal; IV — intravenous; NDA — new drug application;

QIDP — Qualified Infectious Disease Product; sBLA — Supplemental Biologics License Application; SC — subcutaneous; sNDA — supplemental new drug application; SPA — special protocol assessment; TD— transdermal; TOP — topical

Glossary

Breakthrough — a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).1

Fast Track — a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.1

Orphan — drugs for the prevention, diagnosis, or treatment of diseases or conditions affecting fewer than 200,000 persons in the United States, or drugs that will not be profitable within seven years following approval by the U.S. Food and Drug Administration (FDA).2

Qualified Infectious Disease Product (QIDP) — a designation granted by the FDA to an antibacterial or antifungal drug for human use intended to treat serious or life-threatening infections. Products granted QIDP designation may also be eligible for Fast Track and Priority Review designations, and approved QIDP products gain an additional five years of regulatory exclusivity for the use for which the QIDP designation was granted.3

Regenerative Medicine Advanced Therapy (RMAT) — A drug designation applicable to cell therapies, therapeutic tissue engineering products, human cell and tissue products, or any combination product using such therapies or products; intended to treat, modify, reverse or cure a serious, lifethreatening disease or condition; which has the potential to address unmet medical needs.3

Special Protocol Assessment (SPA) — a process in which sponsors may request to meet with FDA to reach agreement on the design and size of certain clinical trials, clinical studies, or animal trials to determine if they adequately address scientific and regulatory requirements.4

Our Authors

Jennifer Schonhorst Pharm.D. PerformRx

References

1. “Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review,” U.S. Food and Drug Administration, February 23, 2018, https:// www.fda.gov/patients/learn-about-drug-and-device-approvals/ fast-track-breakthrough-therapy-accelerated-approval-priority-review

2. “Recommended Tips for Creating an Orphan Drug Designation Application,” U.S. Food and Drug Administration, 2018, https://www.fda.gov/media/111762/download

3. “Qualified Infectious Disease Product Designation Questions and Answers,” U.S. Food and Drug Administration, January 2018, https:// www.fda.gov/regulatory-information/search-fda-guidance-documents/ qualified-infectious-disease-product-designation-questions-and-answers

4. “Regenerative Medicine Advanced Therapy Designation,” U.S. Food and Drug Administration, May 17, 2019, https://www.fda. gov/vaccines-blood-biologics/cellular-gene-therapy-products/ regenerative-medicine-advanced-therapy-designation

5. “Special Protocol Assessment Guidance for Industry,” U.S. Food and Drug Administration, April 2018, https://www.fda.gov/regulatory-information/ search-fda-guidance-documents/special-protocol-assessment-guidance-industry

BIBLIOGRAPHY

Center for Drug Evaluation and Research, U.S. Food and Drug Administration, “Qualified Infectious Disease Product Designation Questions and Answers — Guidance for Industry,” January 2018, https://www.fda.gov/downloads/Drugs/ GuidanceComplianceRegulatoryInformation/Guidances/UCM594213.pdf

Informa Pharma Intelligence Inc., BioMedTracker, Informa PLC, https://www.biomedtracker.com

IPD Analytics, http://www.ipdanalytics.com

Manufacturer press releases and websites.

U.S. Food and Drug Administration, http://www.fda.gov