Specialty Pipeline Monthly Update

Critical updates in an ever changing environment

February 2019

New drug information

●● Cablivi® (caplacizumab-yhdp): The Food and Drug Administration (FDA) approved Sanofi’s Cablivi for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy. The first day of treatment includes both an 11 mg bolus intravenous injection prior to plasma exchange followed by an 11 mg subcutaneous injection after plasma exchange. Treatment continues with self-administered injections once daily until 30 days after the last plasma exchange; but may be extended for a maximum of 28 more days. Sanofi will launch Cablivi in the first quarter of 2019 with a list price of $270,000 for a typical episode of aTTP.1

●● Esperoct® (turoctocog alfa pegol, N8-GP or antihemophilic factor (recombinant) glycoPEGylated-exei): Novo Nordisk received FDA approval for the extended half-life factor VIII molecule, Esperoct, for adults and children with hemophilia A for:

→→ routine prophylaxis to reduce the frequency of bleeding episodes,

→→ on-demand treatment and control of bleeding episodes, and

→→ perioperative management of bleeding.

Novo Nordisk will not launch Esperoct until 2020 due to third-party intellectual property agreements.2 New indications

●● Imbruvica® (ibrutinib): The FDA granted approval to Janssen and Abbvie’s Imbruvica, in combination with Genentech’s Gazyva® (obinutuzumab) for the first-line treatment of adult patients with chronic lymphocytic leukemia/small lymphocytic lymphoma. Gazyva had previously been approved for use in combination with chlorambucil for patients with previously untreated CLL. A Phase 3 trial (iLLUMINATE) found patients who received the combination of Imbruvia-Gazyva achieved a significantly longer median progression-free survival as compared to the patients who received chlorambucil-Gazyva.3

continued

While the information in this newsletter is from sources we believe to be reliable, we do not warrant that the information in this document is free from error. Use it only as a guide. Statements regarding drugs or manufacturers are not intended as promotion; those statements should not be used to make assumptions about formulary status. Each trademarked drug name is the property of its respective owner. Specialty Pipeline Update: February 2019 Page 2

February news

●● “A readout from the first late-stage clinical trial intended to treat the fatty liver disease known as NASH has delivered disappointing results. Gilead Sciences said that its experimental drug, called selonsertib, failed to improve liver scarring compared to a placebo in a Phase 3 clinical trial. The study enrolled nearly 900 patients with compensated cirrhosis, an advanced form of NASH at higher risk for liver-related death.”4

●● “Mark Pruzanski, president and chief executive officer of Intercept, said the company is thrilled to report the positive late-stage results of the REGENERATE trial. With the primary endpoint hit for the 25 mg dose, Pruzanski said the company believes it could be in the position to receive the first regulatory approval for treatment of liver fibrosis due to NASH in the United States.”5

●● “Bluebird bio has announced an ‘aggressive’ plan to file three potential blockbuster drugs with regulators over the next three years, including its CAR-T therapy. Cambridge, Massachusetts-based bluebird bio has been working with Celgene on a chimeric antigen receptor T-cell (CAR-T) therapy, and is also developing therapies for a type of sickle-cell disease and a rare and deadly genetic brain disease.”6

●● “The FDA’s Psychopharmacologic Drug Advisory Committee and Drug Safety and Risk Management Advisory Committee voted in favor (14 yes, 2 no, 1 abstain) of the approval of esketamine nasal spray (Spravato™; Janssen) for the treatment of adult patients with treatment-resistant depression (TRD).”7

●● “AbbVie, a research-based global biopharmaceutical company, has announced that the FDA has accepted for priority review its New Drug Application (NDA) for upadacitinib for the treatment of adult patients with moderate to severe rheumatoid arthritis. Upadacitinib is an investigational once-daily oral JAK1-selective inhibitor being studied for multiple immune-mediated diseases. AbbVie anticipates a regulatory decision in Q3 2019.”8

References 1. https://www.fiercepharma.com/pharma/sanofi-scores-fda-nod-for-nanobody-drug-cablivi-its-first-from-ablynx-deal 2. https://seekingalpha.com/news/3434530-fda-oks-novos-esperoct-hemophilia 3. https://news.abbvie.com/news/abbvie-announces-us-fda-approval-imbruvica-ibrutinib-plus-obinutuzumab-gazyva--first-chemotherapy-free-anti-cd20-combination-regimen-approved-for-chronic-lymphocytic- leukemiasmall-lymphocytic-lymphoma-cllsll-in-previously-untreated-pati.htm 4. https://www.statnews.com/2019/02/11/a-fatty-liver-drug-from-gilead-sciences-posts-negative-results-in-late-stage-clinical-trial/ 5. https://www.biospace.com/article/intercept-pharma-is-one-step-closer-to-filing-for-approval-of-first-nash-treatment/ 6. https://pharmaphorum.com/news/bluebird-bio-aims/ 7. https://www.empr.com/home/news/ 8. https://www.prnewswire.com/news-releases/abbvie-announces-new-drug-application-accepted-for-priority-review-by-us-fda-for-upadacitinib-for-treatment-of-moderate-to-severe-rheumatoid-arthritis-300797706.html drugs-in-the-pipeline/fda-committees-vote-on-esketamine-nasal-spray-for-treatment-resistant-depression/