The biotech sector was in focus with regular pipeline updates. While Sarepta Therapeutics, Inc. SRPT and Solid Biosciences SLDB gained on positive data, Anika Therapeutics, Inc. ANIK and Ziopharm Oncology, Inc. ZIOP declined on dismal results.
Recap of Important Stories
Sarepta, Solid Biosciences Surge : The going has been good for the Duchenne muscular dystrophy (DMD) market. Shares of Sarepta surged significantly after the company reported impressive preliminary results from its phase I/IIa gene therapy trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with DMD. The biopsies performed at the end of day 90 showed robust micro-dystrophin expression in muscle measured by all methods and observed in all three patients. The patients enrolled also showed a significant decrease in serum creatine kinase (CK) levels, with a mean reduction of CK of over 87% at Day 60. (Read more: Sarepta's Stock Soars on Encouraging Gene Therapy Results ).
Earlier, Solid Biosciences' shares surged too after the company announced that the FDA has lifted the clinical hold on its phase I/II trial, IGNITE DMD, for its experimental candidate, SGT-001. Notably, SGT-001, Solid Biosciences' lead candidate, is a microdystrophin gene transfer, which is being evaluated for the treatment of DMD. The clinical hold was lifted as the FDA acknowledged that Solid Biosciences has satisfactorily addressed all the issues raised. (Read more: Solid Surges as FDA Lifts Clinical Hold on DMD Drug ).
Alexion Submits BLA for ALXN1210 : Alexion Pharmaceuticals, Inc. ALXN announced that the company has submitted the Biologics License Application (BLA) to the FDA for approval of its experimental long-acting C5 complement inhibitor, ALXN1210, for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). Since the submission is for a rare disease using a priority review voucher, the BLA is entitled to an eight-month review by the FDA instead of the standard 12-month review. Alexion's lead drug, Soliris, is already approved for PNH and a tentative approval of ALXN1210 will give a significant boost to the company. ALXN1210 had earlier achieved non-inferiority to Soliris in complement inhibitor treatment-naive patients with PNH based on the co-primary endpoints of transfusion avoidance and normalization of LDH levels in a phase III study wherein ALXN1210 was administered intravenously every eight weeks.
Alexion carries a Zacks Rank #3 (Hold). You can see the complete list of today's Zacks #1 Rank stocks here.
Anika Plunges on Disappointing Data : Shares of Anika Therapeutics have dropped in after-market trading after the company reported disappointing results from a phase III trial on Cingal, which is being conducted to support registration in the United States. The 16-02 trial compared Cingal, a combination of cross-linked HA and triamcinolone hexacetonide (TH), with TH alone and cross-linked HA in treating patients with osteoarthritis in the knee. The primary endpoint was a comparison of the pain reduction when treated with Cingal compared with only TH at 26 weeks. However, the results did not show any statistical significance even though Cingal achieved greater pain reduction numerically at every time point in the study. Cingal is Anika's third-generation visco supplement, along with Orthovisc and Monovisc products, to treat pain associated with osteoarthritis of the knee.
Ziopharm Oncology Plunges As FDA Places Clinical Hold on CAR T Therapy : Shares of Ziopharm Oncology declined after the FDA placed a clinical hold on its phase I trial evaluating CD19-specific CAR-T therapies manufactured under point-of-care. The FDA has requested additional information relative to Chemistry, Manufacturing and Controls in support of the investigational new drug (IND) application for the trial. The therapy is being evaluated as an investigational treatment for patients with relapsed or refractory, CD19+ leukemias and lymphomas.
Amgen Got European Nod For Addition of Data to Blincyto : Amgen AMGN announced that the European Commission (EC) has granted a full marketing authorization for Blincyto (blinatumomab) based on encouraging overall survival (OS) data from the phase III study, TOWER, in adult patients with Philadelphia chromosome-negative (Ph-) relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). Results showed that Blincyto demonstrated a superior improvement in median OS over standard-of-care (SOC) chemotherapy. Median OS was 7.7 months for Blincyto versus four months for SOC.
Medical - Biomedical and Genetics Industry 5YR % Return
The NASDAQ Biotechnology Index gained 1.02% over the last five trading sessions. Among the major biotech stocks, Regeneron gained 5.67%. Over the past six months, Celgene has lost 25.80%, while Amgen has gained 6.88%. (See the last biotech stock roundup here: Biotech Stock Roundup: AXON Soars, Amgen Drugs Get FDA, EC Nod )
What's Next in Biotech?
Stay tuned for regulatory updates and pipeline development news.
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