Pipeline Report May 2020 Pipeline Report May 2020 © 2020 Envolve. All rights reserved. Page 1 This quarterly at-a-glance publication is developed by our Clinical Pharmacy Drug Information team to Table of Contents increase your understanding of the drug pipeline, ensuring you’re equipped with insights to prepare Recent Specialty Drug Approvals 1 for shifts in pharmacy benefit management. In this issue, you’ll learn more about these key themes and notable drugs: Recent Non-Specialty Drug Approvals 2 > Despite the FDA’s recent intense focus on COVID- 19-related activities, oncology drug pipeline is one Upcoming Specialty Products 3 therapeutic area which remains vibrant in the immediate term. This is evidenced by the consistent rate of FDA approvals, as well as acceptance of New Drug and Biologic License Applications for review. In fact, multiple oncology agents were Upcoming Non-Specialty Products 7 FDA-approved several months ahead of schedule, underscoring the urgency with which the FDA continues to act on oncologic agents. Biosimilars 9 > Several pipeline agents on this quarterly report are worth highlighting as possible new alternatives in high-cost disease states with few therapeutic options. These Generic Specialty Agents 10 agents include viltolarsen in the treatment of Duchenne muscular dystrophy (DMD), fostemsavir in the treatment of heavily treatment-experienced adult patients with HIV, and roxadustat which offers the first oral alternative to Generic Non-Specialty Agents 11 injectable agents such as Aranesp, Epogen and Procrit, for the treatment of anemia associated with chronic kidney disease. To prepare this report, our team accesses a wide range of clinical resources. This information is then analyzed, resulting in updates across multiple disease states including recent and anticipated drug approvals, key changes in the biosimilar agent landscape, and notes on recent and anticipated generic product launches. Our pipeline report is just one of many ways we’re committed to providing helpful tools and resources to our clients and partners. We look forward to sharing more updates with you in the months ahead. Ross Hoffman, MD To provide comments, feedback or requests for report enhancements, please email us at [email protected]. Pipeline Report Recent Specialty Drug Approvals May 2020 FDA Approval AcariaHealth (AH) Cost (AWP Drug Name Manufacturer Indication(s) Comments Date Access Status Package Price) ENDOCRINOLOGY Isturisa • For the treatment of adults who either cannot undergo pituitary osilodrostat • Novartis • Cushing’s disease 3/6/2020 Pending launch gland surgery or have undergone the surgery but still have the $410,400/ year oral tablets disease. NEUROLOGY Vyepti • Alder • Efficacy demonstrated for both episodic and chronic migraines. eptinezumab-jjmr • Migraine headaches 2/21/2020 Limited access $7,176/ year BioPharmaceuticals • 30-minute infusion once every 12 weeks. intravenous infusion Zeposia • Relapsing forms of multiple • Same mechanism of action as Gilenya, but potentially less cardiac ozanimod • Celgene 3/25/2020 Pending launch Pending launch sclerosis (MS) adverse effects. oral capsules ONCOLOGY Sarclisa • Proposed for use in combination with pomalidomide and isatuximab-irfc • Sanofi • Muliple myeloma 3/2/2020 Limited access dexamethasone for third-line treatment. $141,960/ year intravenous infusion • Same mechanism of action and indication as Darzalex. Koselugo • Neurofibromatosis type 1 (NF1) • AstraZeneca • Approved for the treatment of pediatric patients aged two years and selumetinib with plexiform neurofibromas 4/10/2020 Limited access $156,960/ year • Merck older with symptomatic, inoperable PNs. oral capsules (PN) Jelmyto • Is the only FDA-approved non-surgical treatment for this indication, mitomycin • UroGen for the treatment of low-grade upper tract disease that is $363,392/ 14-month • Urothelial carcinoma 4/15/2020 Pending launch pyelocalyceal instillation Pharmaceuticals unamenable to endoscopic resection or with contraindication to course of therapy gel nephroureterectomy. Pemazyre • For the treatment of locally advanced/metastatic or surgically pemigatinib • Incyte • Cholangiocarcinoma 4/17/2020 Limited access unresectable disease with FGFR-2 translocation in patients who $285,600/ year oral tablets failed previous therapy. • Approved for use in combination with trastuzumab and capecitabine Tukysa • Cascadian for patients who have received one or more prior anti-HER2-based tucatinib • Breast cancer 4/17/2020 AH has access $266,400/ year Therapeutics regimens in the metastatic setting. oral tablets • Showed efficacy in patients both with or without brain metastases. Trodelvy • Antibody-drug conjugate (ADC). sacituzumab govitecan- • Triple negative breast cancer • Impressive clinical activity seen in a refractory setting. • Immunomedics 4/22/2020 Limited access $251,160/ year hziy (TNBC) • Is the first and only ADC approved for the treatment of metastatic intravenous infusion TNBC. • Approved for both first-line and previously treated patients with Tabrecta • Non-small cell lung cancer metastatic MET exon 14 skipping (METex14) mutated disease. capmatinib • Novartis 5/6/2020 Pending launch Pending launch (NSCLC) • The MET mutation is seen in an estimated 3% - 4% of all patients oral tablets with NSCLC. © 2020 Envolve. All rights reserved. Page 1 Pipeline Report Recent Non-Specialty Drug Approvals May 2020 FDA Approval Cost (AWP Package Drug Name Manufacturer Indication(s) Comments Date Price) CARDIOVASCULAR Nexletol • For use in combination with maximally tolerated statin therapy. • Esperion bempedoic acid • Hypercholesterolemia 2/21/2020 • Not as potent as Praluent and Repatha, but considerably less $4,818/ year Therapeutics, Inc. oral tablets expensive. Nexlizet • For use in combination with maximally tolerated statin therapy. • Esperion bempedoic acid /ezetimibe • Hypercholesterolemia 2/26/2020 • Not as potent as Praluent and Repatha, but considerably less Pending launch Therapeutics, Inc. oral tablets expensive. GASTROINTESTINAL Pizensy • Chronic idiopathic • Once daily dosing. lactitol • Braintree labs 2/12/2020 Pending launch constipation • Mixed with 4-8 oz of fluid (water, juice, coffee, etc.). oral solution Barhemsys • Post-operative nausea and amisulpride • Acacia 2/26/2020 • Approved for use as both treatment and prophylaxis. Pending launch vomiting intravenous infusion NEUROLOGY Nurtec ODT • Will compete with generically available oral triptans for the same rimegepant • Biohaven • Migraine headaches 2/27/2020 $12,240/ year indication. oral tablets Ongentys • Once daily dosing. • Neurocrine opicapone • Parkinson’s disease (PD) 4/24/2020 • Intended as adjunct to levodopa therapy in patients experiencing “off” Pending launch Biosciences oral capsules episodes. © 2020 Envolve. All rights reserved. Page 2 Pipeline Report Upcoming Specialty Products May 2020 Anticipated Approval Drug Name Manufacturer Indication(s) Mechanism of Action Comments Date CARDIOVASCULAR • Potentially large impacted population. Revascor • Proposed for use in left ventricular systolic dysfunction. rexlemestrocel-L • Mesoblast • Chronic heart failure • Stem cell therapy 2020 • Would be reserved for use in patients who had failed multiple other intramyocardial injection therapies. • Administered in the doctor’s office as twice yearly dosing. • Proprotein convertase subtilisin/ inclisiran • Would compete with the PCSK9 binding inhibitors for high • Novartis • Hypercholesterolemia kexin type 9 (PCSK9) synthesis 12/1/2020 subcutaneous injection cardiovascular risk patients who are already on maximized statin inhibitor therapy. COAGULATION DISORDERS Roctavian • Proposed for the treatment of adults with severe disease (~60% of the valoctocogene • BioMarin • Hemophilia A • Gene therapy total hemophilia A population). 8/21/2020 roxaparvovec • Current standard of care is factor VIII replacement therapy. intravenous infusion AMT-061 • For the treatment of adults with severe disease (~40% of the total etranacogene • Uniqure • Hemophilia B • Gene therapy hemophilia B population). 2020-2021 dezaparvovec • Current standard of care is factor IX replacement therapy. intravenous infusion • For the treatment of adults with severe disease (~60% of the total SPK-8011 • Spark • Hemophilia A • Gene therapy hemophilia A population). 2021 intravenous infusion • Roche • Current standard of care is factor VIII replacement therapy or Hemlibra. ENDOCRINOLOGY UX 007 • Currently available therapies include avoidance of fasting, low-fat/high • Long-chain fatty acid triheptanoin • Ultragenyx • Fatty acid replacement therapy carbohydrate diets, carnitine and medium-chain triglyceride (MCT) oil, 7/31/2020 oxidative disorder oral liquid a medical food product. • Once daily oral therapy. BCX7353 • Hereditary angioedema • Selective inhibitor of plasma • All other HAE therapies are IV or SC injections. berotralstat • BioCryst 12/3/2020 (HAE) kallikrein • High demand for this more convenient, less invasive dosage form is oral capsules anticipated. HEPATOLOGY OCA • Non-alcoholic • Farnesoid X-activated receptor • New indication for Ocaliva. obeticholic acid • Intercept 6/26/2020 hepatosteatosis (NASH) agonist • Would be the first FDA-approved pharmacologic therapy for NASH. oral tablets © 2020 Envolve. All rights reserved. Continued Page 3 Pipeline Report Upcoming Specialty Products May 2020 Anticipated Approval Drug Name Manufacturer Indication(s) Mechanism of Action Comments Date IMMUOLOGY • IV infusion twice weekly for