The past week was busy for the biotech sector with pipeline updates for most biotech bigwigs. While Amgen (AMGN - Free Report) received an FDA nod for its osteoporosis drug, Gilead Sciences, Inc. (GILD - Free Report) has submitted an application for the label expansion of its HIV drug, Descovy. Celgene Corporation (CELG - Free Report) too has filed a Biologics License Application (BLA) for its pipeline candidate, luspatercept, to the FDA. Meanwhile, Regeneron Pharmaceuticals, Inc. (REGN - Free Report) has extended its collaboration agreement with Alnylam.
Recap of the Week’s Top Stories:
FDA Approves Amgen’s Osteoporosis Drug Evenity: Amgen and partner UCB announced that the FDA has approved osteoporosis drug, Evenity, for the treatment of osteoporosis in postmenopausal women at high risk for fracture. The FDA approval was based on the results of two phase III studies, namely FRAME and ARCH. Treatment with Evenity led to a significant reduction of new vertebral (spine) fracture at 12 months compared with placebo as results from the FRAME study demonstrate. Data from the ARCH study showed that treatment with EVENITY for a 12-month regime followed by 12 months of alendronate, significantly reduced the incidence of new vertebral fracture at 24 months. However, the label comes with a boxed warning of increase in the risk of myocardial infarction (heart attack), stroke and cardiovascular death.
Amgen currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Gilead Seeks Label Expansion of Descovy: Gilead Sciences announced the submission of a supplemental New Drug Application (sNDA) to the FDA for Descovy (emtricitabine 200 mg and tenofovir alafenamide 25 mg tablets). The company is seeking an FDA approval for Descovy as pre-exposure prophylaxis (PrEP) to lower the risk of sexually acquired HIV-1 infection among the individuals, who are HIV-negative and at risk for HIV. While Descovy is already approved in combination with other antiretroviral agents for the treatment of HIV infection in patients weighing greater or equal to 25 kg, it is not indicated for PrEP yet.
A priority review voucher was submitted along with the filing. Hence, the anticipated review time for the sNDA is expected to be six months. The filing is based on the findings from the phase III study, DISCOVER, which evaluated the safety and efficacy of Descovy compared with Truvada in men and transgender women, who sexually engage with men at a high risk of contracting HIV infection. The outcomes showed that Descovy achieved non-inferiority to Truvada in study participants, who were at substantial and sustained risk of HIV acquisition.
Celgene, Acceleron Submit BLA for Luspatercept: Celgene Corporation announced that it has submitted a Biologics License Application (BLA) for the pipeline candidate, luspatercept, a first-in-class erythroid maturation agent (EMA) that regulates late-stage red blood cell (RBC) maturation. The company is developing luspatercept in collaboration with partner Acceleron Pharma Inc.
The BLA is seeking an FDA approval for luspatercept to treat adult patients with very low-to-intermediate risk myelodysplastic syndromes (MDS)-associated anemia, those with ring sideroblasts and require RBC transfusions plus adult subjects with beta-thalassemia-associated anemia, who need RBC transfusions.
The BLA was filed on the basis of safety and efficacy results from the phase III studies, programs — MEDALIST and BELIEVE. Both companies plan to submit a marketing application to the European Medicines Agency in second-quarter 2019. Meanwhile, the candidate is also being evaluated in phase III study, COMMANDS, for the treatment of erythropoiesis-stimulating agent (ESA) naïve, lower-risk MDS patients. Luspatercept is also being evaluated in the BEYOND phase II study for non-transfusion-dependent beta-thalassemia and a phase II analysis for myelofibrosis.
Regeneron Collaborates With Alnylam for RNAi-based Therapeutics: Regeneron announced a collaboration agreement with Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) . Both companies will coordinate to discover, develop and commercialize new RNA interference (RNAi) therapeutics for a broad range of indications by addressing the disease targets expressed in the eye and the central nervous system (CNS) in addition to a select number of targets expressed in the liver.
Moreover, Alnylam is eligible to receive up to an additional $200 million as milestone fees. Regeneron will provide Alnylam with $2.5 million in funding at the initiation of any program and an additional $2.5 million for identifying the lead candidate. Alnylam will also get an extra $30 million in annual discovery funding.
United Therapeutics Plunges on Study Failure: Shares of United Therapeutics Corporation (UTHR - Free Report) declined after the company announced that a late-stage study on its pipeline candidate, esuberaprost, did not meet its primary endpoint. The BEAT study was evaluating the pipeline candidate, esuberaprost, in combination with the marketed pulmonary arterial hypertension (PAH) drug, Tyvaso as Tysuberprost, in patients with PAH, who showed signs of deterioration with Tyvaso or had a less than optimal response to the Tyvaso treatment. The primary endpoint of the delayed time to the first clinical worsening event was not successfully met in the study.
The NASDAQ Biotechnology index declined 3.16% in the past five trading sessions. Among the major biotech biggies, Biogen lost 28.1% of value. Over the past six months, shares of Regeneron have gained 3.60% while the Gilead stock has declined 15.1%. (See the last biotech stock roundup here: Biotech Stock Roundup: BIIB & CNAT Crash on Study Failures, LXRX Gets CRL)
What's Next in Biotech?
Stay tuned for regular pipeline developments.
Zacks' Top 10 Stocks for 2019
In addition to the stocks discussed above, would you like to know about our 10 finest buy-and-holds for the year?
Who wouldn't? Our annual Top 10s have beaten the market with amazing regularity. In 2018, while the market dropped -5.2%, the portfolio scored well into double-digits overall with individual stocks rising as high as +61.5%. And from 2012-2017, while the market boomed +126.3, Zacks' Top 10s reached an even more sensational +181.9%.
See Latest Stocks Today >>