It was a busy week for the biotech sector with updates from quite a few companies. Solid Biosciences (SLDB - Free Report) plummeted after the FDA placed a clinical hold on its gene therapy candidate, while Amarin (AMRN - Free Report) gained ahead of an FDA committee meeting on the label expansion of its approved drug Vascepa. Meanwhile, Celgene Corp. and partner Acceleron (XLRN - Free Report) obtain FDA approval of Reblozyl for the treatment of anemia in adult patients with beta thalassemia.
Recap of the Week’s Most Important Stories:
Solid Biosciences Plunges As FDA Places Clinical Hold: Shares of Solid Biosciences plunged after the FDA notified it that a clinical hold has been placed on IGNITE DMD, its phase I/II study of lead candidate SGT-001, after a patient dosed in one of the cohorts experienced a serious clinical event (SAE).
Six patients have been dosed with SGT-001, the company’s gene transfer candidate under investigation for Duchenne muscular dystrophy (DMD). This includes three patients in the first cohort at a 5E13 vg/kg dose, who continue to do well and are being followed per the study protocol. Three patients were subsequently dosed in the second cohort at a 2E14 vg/kg dose.
However, the third patient in the 2E14 vg/kg cohort, dosed in late October, experienced an SAE deemed related to the study drug that was characterized by complement activation, thrombocytopenia, a decrease in red blood cell count, acute kidney injury, and cardio-pulmonary insufficiency. Thereafter, Solid Biosciences reported the event to the FDA and the study Data Safety Monitoring Board (DSMB). The hold comes as a setback for the company, which plans to report additional biomarker data from the study before the year-end.
Celgene & Acceleron Get FDA Nod for Rare Blood Disorder Drug: Celgene Corporation and partner Acceleron Pharma Inc. announced that the FDA has approved pipeline candidate Reblozyl (luspatercept-aamt) for the treatment of anemia in adult patients with beta thalassemia, who require regular red blood cell (RBC) transfusions. Per the company, Reblozyl is the first and only FDA-approved erythroid maturation agent, representing a new class of therapy for these patients. The approval comes ahead of the target action date of Dec 4.
The approval of the drug for beta thalassemia was based on encouraging results from the pivotal, phase III, randomized, double-blind, placebo-controlled, multicenter BELIEVE study. Meanwhile, the FDA is also evaluating luspatercept-aamt for the treatment of anemia in adults with very low- to intermediate-risk myelodysplastic syndromes (MDS), who have ring sideroblasts and require RBC transfusions. The agency has set a target action date of Apr 4, 2020, for the same.
Celgene currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Gilead Presents Data on HIV Therapy: Gilead Sciences (GILD - Free Report) announced encouraging data on pipeline candidate GS-6207 at the 17th European AIDS Conference (EACS) in Basel, Switzerland. GS-6207 is an investigational, novel, selective, first-in-class inhibitor of HIV-1 capsid function. New data from two phase I studies demonstrated that GS-6207 has potent antiviral activity and a potential dosing interval of up to every six months.
GS-6207 was generally well tolerated and no serious adverse events were reported in both clinical studies. Additionally, the vitro virology study results suggested that GS-6207 can potentially be used in a broad range of HIV patients regardless of their treatment history.
Amarin Up Ahead of FDA Committee Meet: Shares of Amarin gained 23.8% ahead of the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) meeting scheduled for Nov 14. The committee will decide on the label expansion of Amarin’s lead drug, Vascepa, which is indicated as an adjunct to diet to reduce triglyceride (TG) levels in adult patients with severe (≥500 mg/dL) hypertriglyceridemia. Amarin is seeking a label expansion of the drug to include cardiovascular risk reduction in statin-treated patients with only moderately increased triglyceride.
Biogen to Expand Biosimilars Portfolio: Biogen (BIIB - Free Report) announced a new collaboration with Samsung Bioepis Co., Ltd. to secure the exclusive rights to commercialize two new ophthalmology biosimilars, SB11 (biosimilar of Lucentis) and SB15 (biosimilar of Eylea), in major markets worldwide, including the United States, Canada, Europe, Japan and Australia. Additionally, the company will acquire exclusive commercialization rights for its anti-TNF portfolio, including Benepali (etanercept), Flixabi (infliximab) and Imraldi (adalimumab), in China.
Moreover, Biogen will acquire an option to extend its existing commercial agreement with Samsung Bioepis for this anti-TNF portfolio in Europe. Per the terms, Biogen will make a $100-million upfront payment to Samsung Bioepis. Additionally, the company may pay Samsung Bioepis up to $210 million in milestone payments.
The Nasdaq Biotechnology index gained 0.93% in the last five trading sessions. Among the biotech giants, Amgen gained 20.83% in the period. Over the past six months, shares of Biogen have gained 29.13%, whereas the Alexion stock has declined 13.09%. (See the last biotech stock roundup here: Biotech Stock Roundup: Biotech Stock Roundup: Q3 Earnings Beat at ALXN & AMGN, Pipeline Updates & More)
What's Next in Biotech?
Stay tuned for more pipeline updates.
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