This week, quite a few biotech companies came out with pipeline and regulatory updates. Epizyme (EPZM - Free Report) got a significant boost with the FDA approval of its lead drug. Both Acceleron Pharma Inc. (XLRN - Free Report) and Hepion Pharmaceuticals, Inc. (HEPA - Free Report) surged on positive pipeline updates. Incyte (INCY - Free Report) too came out with positive results from a late-stage study on ruxolitinib cream, while Gilead Sciences (GILD - Free Report) and Regeneron (REGN - Free Report) provided regulatory updates on their candidates.
Recap of the Week’s Most Important Stories:
Epziyme Wins FDA Nod for Tazverik: The FDA granted accelerated approval to Epizyme’s lead drug Tazverik (tazemetostat) for the treatment of adults and pediatric patients aged 16 years or older with metastatic or locally advanced epithelioid sarcoma (ES) not eligible for complete resection. Tazverik is a methyltransferase inhibitor. The approval was based on the overall response rate and the duration of response in a phase II study. An approval was in the cards as the Oncologic Drugs Advisory Committee (ODAC) of the FDA had voted (11-0) in favor of the benefit-risk profile of the drug for the above-mentioned indication in December.
The continued approval of Tazverik for this indication is contingent upon verification and description of clinical benefit in a confirmatory trial. Epziyme’s ongoing, global, randomized, controlled confirmatory trial assessing the combination of Tazverik and doxorubicin compared with doxorubicin plus placebo as a front-line treatment for ES is underway. The approval will significantly boost Epizyme’s portfolio, which did not have any approved drug in its kitty till now.
Acceleron Soars on Success of PAH Drug: Acceleron Pharma Inc. surged after it announced that the mid-stage study on pipeline candidate, sotatercept, in patients with pulmonary arterial hypertension (PAH) was successful.
The phase II study, PULSAR, is a randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy and safety of sotatercept in PAH patients. In the study, 106 patients were randomized to receive placebo, 0.3 mg/kg of sotatercept, or 0.7 mg/kg of sotatercept subcutaneously, every 21 days, in combination with stable background PAH-specific therapies over a 24-week treatment period.
The study results showed that the candidate demonstrated a statistically significant reduction in pulmonary vascular resistance PVR, the trial’s primary endpoint, at week 24 versus placebo. PVR, as measured by right heart catheterization, is the resistance that the heart must overcome to pump blood through the pulmonary circulatory system. The study also achieved statistically significant improvements in the key secondary endpoint of six-minute walk distance (6MWD) and other secondary endpoints, including amino-terminal brain natriuretic propeptide (NT-proBNP) and World Health Organization (WHO) functional class.
Incyte Announces Positive Data From Atopic Dermatitis Study: Incyte announced positive top-line results from a late-stage study evaluating the safety and efficacy of ruxolitinib cream in adolescent and adult patients with atopic dermatitis (AD). The randomized, vehicle-controlled, pivotal phase III TRuE-AD2 study met its primary endpoint as a larger number patients treated with ruxolitinib cream 0.75% and 1.5% achieved Investigator’s Global Assessment Treatment Success (IGA-TS) — defined as an IGA score of 0 (clear) or 1 (almost clear) with at least a two-point improvement from baseline at Week 8 — than those treated with vehicle control (non-medicated cream). The long-term safety portion of the study will continue as planned. Additionally, the overall efficacy and safety profile of ruxolitinib cream is consistent with previous data.
Gilead’s Marketing Application for CAR T Cell Therapy Validated: Gilead Sciences announced that its Marketing Authorization Application (MAA) for the experimental chimeric antigen receptor (CAR) T cell therapy, KTE-X19, for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) has been fully validated. It is now under evaluation with the European Medicines Agency (EMA). The MAA is supported by positive data from the single-arm, open-label, phase II ZUMA-2 study. KTE-X19 has been granted Breakthrough Therapy Designation (BTD) by the FDA and Priority Medicines (PRIME) by the EMA. A Biologics License Application (BLA) for the same was also submitted to the FDA in December 2019.
Gilead currently carries a Zacks Rank#3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Regeneron, Sanofi’s sBLA for Dupixent Accepted: Regeneron Pharmaceuticals, Inc. and partner Sanofi (SNY - Free Report) announced that the FDA has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Dupixent (dupilumab). Both companies are seeking the approval of the drug as an add-on maintenance treatment for children aged 6-11 years with moderate-to-severe atopic dermatitis, whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. The FDA has set a target action date of May 26, 2020. The drug is already approved in the United States to treat patients aged 12 years or older with moderate-to-severe atopic dermatitis.
Hepion Surges on Positive Study Data: Shares of Hepion Pharmaceuticals, Inc. surged after it announced positive data from an expanded study with human precision cut liver slices (PCLS). Hepion's drug candidate, CRV431, prevented experimentally induced liver fibrosis to a larger extent than four other leading non-alcoholic steatohepatitis (NASH) drug candidates — obeticholic acid, elafibranor, resmetirom and Aramchol — in this specialized experimental model using human liver tissue. Hepion is evaluating CRV431 for NASH, fibrosis and other liver diseases. A phase I, single ascending dose study previously showed CRV431 to be safe and well tolerated in humans. The candidate is currently being administered to humans in a 28-day multiple ascending dose study.
The Nasdaq Biotechnology index lost 2.65% in the last five trading sessions. Among the biotech giants, Regeneron lost 5.40% in the period. Over the past six months, shares of Vertex have gained 39.55%. (See the last biotech stock roundup here: Biotech Stock Roundup: Updates From HZNP & CLVS, BioNTech to Acquire Neon)
What's Next in Biotech?
Stay tuned for earnings updates from leading biotech giants.
Looking for Stocks with Skyrocketing Upside?
Zacks has just released a Special Report on the booming investment opportunities of legal marijuana.
Ignited by new referendums and legislation, this industry is expected to blast from an already robust $6.7 billion to $20.2 billion in 2021. Early investors stand to make a killing, but you have to be ready to act and know just where to look.
See the pot trades we're targeting>>