BioBiz: Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy fails

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Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy failed to beat placebo in a phase 2 clinical trial. Functional motor ability scores in the SRP-9001 arm were statistically no better than in the placebo group, almost halving Sarepta’s stock price and raising doubts about a key program.  

Investigators randomized 41 DMD patients aged four to seven years old to take SRP-9001 or placebo. At 48 weeks, children treated with the gene therapy had a 1.7 mean improvement on the 17-point NSAA functional motor ability scale. The mean improvement in the placebo group was 0.9, causing the clinical trial to miss its primary functional endpoint. 

DBV Technologies’ journey to have Viaskin, its peanut allergy skin patch approved has been a tough nut to crack. The FDA turned the down the patch—saying its designs would need to be modified and retested. Today, DBV says it will lay off more than two-thirds of its staff by the end of the quarter. The French developer first announced plans to restructure its global workforce last June, a little more than a month before the FDA issued a complete response letter for the patch. The agency is now demanding a new human factor study, additional clinical data and more information on the patch’s chemistry and manufacturing process.

Q BioMed Inc. announced today that the Centers for Medicare and Medicaid Services updated the Outpatient Prospective Payment System (OPPS) reimbursement rate for its FDA approved non-opioid metastatic bone pain drug Strontium89 (Strontium Chloride Sr-89 Injection, USP) on January 3, 2021. The update ensures all Medicare patients have access to Strontium89 in the appropriate setting. With the CMS update, all Medicare providers are now able to be reimbursed appropriately for providing Strontium89 treatment.

DiCE Molecules announced the completion of an $80 million Series C financing. The financing was led by RA Capital Management with participation from new investors including Eventide Asset Management, New Leaf Venture Partners, Soleus Capital, Driehaus Capital Management, Osage University Partners and Asymmetry Capital Management. Existing investors Northpond Ventures, Sands Capital, Sanofi Ventures, Alexandria Venture Investments, Altitude Life Science Ventures and Agent Capital also participated.

OncoDNA, a theranostic and genomic company specializing in precision oncology, has signed a subcontract service agreement with HalioDx’s U.S.-based CLIA-certified laboratory located in Richmond, Virginia. This agreement will enable OncoDNA to expand its unique offering of services to biopharmaceutical companies and research institutions in North America.