Quite a few biotech companies came out with pipeline and regulatory updates this week. While Amarin AMRN gained on a label expansion for its key drug, Amgen AMGN won approval for its osteoporosis drug in Europe. Both Gilead Sciences, Inc. GILD and Biogen BIIB came out with pipeline updates.
Recap of the Week’s Most Important Stories:
Gilead Announces Top-Line Results From NASH Study: Gilead announced mixed top-line results from the mid-stage study on combination and monotherapy of investigational treatments for advanced fibrosis (F3-F4) due to nonalcoholic steatohepatitis (NASH).
The 48-week, phase II ATLAS randomized, double-blind, placebo-controlled study evaluated the safety and efficacy of monotherapy and dual combination regimens of the nonsteroidal farnesoid X receptor (FXR) agonist cilofexor 30 mg, the acetyl-CoA carboxylase (ACC) inhibitor firsocostat 20 mg and selonsertib 18 mg in patients with advanced fibrosis (F3-F4) due to NASH.
The results showed that the investigational regimens did not lead to a statistically significant increase in the proportion of patients who achieved the primary efficacy endpoint of a ≥1-stage improvement in fibrosis without worsening of NASH.
Earlier, Gilead’s Kite and Kiniksa Pharmaceuticals, Ltd. KNSA entered a clinical collaboration to conduct a phase II, multicenter study on pipeline candidate mavrilimumab in combination with CAR T-cell therapy Yescarta. The objective of the study is to determine the effect of mavrilimumab on the safety of Yescarta.
Concurrently, Kite submitted a Biologics License Application (BLA) to the FDA for the investigational CAR T-cell therapy, KTE-X19, for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL).
Biogen's Gosuranemab Fails in Phase II Brain Disorder Study: Biogen announced that its phase II study — PASSPORT — evaluating pipeline candidate, gosuranemab (BIIB092), in patients with progressive supranuclear palsy (PSP) failed to meet its primary endpoint. Data from the study showed that improvement in PSP patients treated with gosuranemab as measured by PSP rating scale at week 52 was not statistically significant.
Moreover, the candidate failed to demonstrate efficacy in patients on key clinical secondary endpoints. Based on these data, the company has decided to discontinue further development of the candidate for PSP and other primary tauopathies.
However, Biogen stated that it will continue the development of gosuranemab for treating mild cognitive impairment due to Alzheimer’s disease. Currently, the candidate is being evaluated in the phase II TANGO study for this indication.
Amarin Up on FDA Approval of Vascepa Label Expansion: Shares of Amarin Corporation gained after the company announced that the FDA approved a label expansion of Vascepa. Vascepa is now approved as an adjunct to maximally tolerated statin therapy to reduce the risk of myocardial infarction, stroke, coronary revascularization and unstable angina requiring hospitalization in adult patients with elevated triglyceride (TG) levels (≥150 mg/dL) and established cardiovascular disease or diabetes mellitus and two or more additional risk factors for cardiovascular disease.
The label expansion should significantly boost sales as, per estimates, millions of high-risk patients in the United States could benefit from this one-of-a-kind prescription therapy. Vascepa will be a new FDA-approved treatment option to reduce the persistent cardiovascular risk faced by many patients despite the use of statins with other contemporary standard-of-care therapies.
Amgen's Evenity Gets Approval in Europe: Amgen and partner UCB announced that the European Commission (EC) has granted marketing authorization to Evenity (romosozumab) for the treatment of severe osteoporosis in postmenopausal women at high risk of fracture. The approval was supported by the positive opinion given by the Committee for Medicinal Products for Human Use (CHMP) in October.
The drug is expected to be launched in Europe in the first half of 2020. Evenity was approved in April by the FDA. It is the first and only approved drug for osteoporosis to increase bone mineral density (“BMD”) and reduce the risk of fracture.
Alexion Exercises Option for Two Additional RNAi Programs: Alexion ALXN announced that it has exercised its option for exclusive rights to two additional targets within the complement pathway for the discovery and development of GalXC RNAi molecules under its collaboration with Dicerna Pharmaceuticals. As a result, Alexion will pay Dicerna a total of $20 million, or $10 million in option exercise fees per additional target.
Both companies collaborated in October 2018 for the discovery and development of subcutaneously delivered GalXC RNAi molecules directed to a total of four complement pathway targets for the treatment of complement-mediated diseases. Dicerna is leading the joint discovery and research efforts through the preclinical stage, while Alexion will lead development efforts beginning the phase I studies.
Alexion currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Medical - Biomedical and Genetics Industry 5YR % Return
Medical - Biomedical and Genetics Industry 5YR % Return
The Nasdaq Biotechnology index inched up 0.64% in the last five trading sessions. Among the biotech giants, Amgen gained 3.85% in the period. Over the past six months, shares of Amgen have gained 38%. (See the last biotech stock roundup here (See the last biotech stock roundup here: Biotech Stock Roundup: ASH Data in Focus, Amgen Gets FDA Nod to Remicade Biosimilar)
What's Next in Biotech?
Stay tuned for more pipeline updates.
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