In a 10-1 vote, the U.S. FDA’s Psychopharmacologic Drugs Advisory Committee said Otsuka Pharmaceutical Co. Ltd.’s studies of its combination post-traumatic stress disorder (PTSD) treatment did not show enough efficacy. Had the adcom felt otherwise, the path may have been smoother for a new treatment in an indication that hasn’t seen regulatory advancement in decades. The committee’s conclusion, however, is not binding on the FDA, which will make its own decision whether to approve.
Carving out $400 million in annual cost savings is going over well with Sarepta Therapeutics Inc.’s investors, as the company’s stock got a 19.6% boost on July 17. Its big seller, the gene therapy Elevidys (delandistrogene moxeparvovec), continued its revenue numbers decline, so Sarepta chopped operating expenses by letting about 500 employees go in a 36% cutback and pared its development path.
The U.S. FDA is insisting that another study is needed to bolster the sNDA for Rexulti (brexpiprazole) from Otsuka Pharmaceutical Co. Ltd. in treating adults with post-traumatic stress disorder (PTSD). The agency’s Psychopharmacologic Drugs Advisory Committee and the company, one of Japan’s biggest pharmas, will discuss this during July 18’s adcom hearing regarding the efficacy of Rexulti in combination with sertraline.
Manufacturing issues are the latest problem for Ultragenyx Pharmaceutical Inc. to solve after last week’s disappointment in a phase III study to treat brittle bones. The U.S. FDA gave the company a complete response letter (CRL) regarding the BLA for its gene therapy to treat Sanfilippo syndrome type A, saying it needs more details and improvements made about CMC after having finished manufacturing facility inspections.
Capricor Therapeutics Inc. received a complete response letter (CRL) from the U.S. FDA on the BLA for deramiocel to treat cardiomyopathy in Duchenne muscular dystrophy patients. The company’s CEO said the letter was unexpected.
Ultragenyx Pharmaceutical Inc. and Mereo Biopharma Group plc, along with investors, had hoped for an early halt to the phase III study of setrusumab in treating brittle bones. They didn’t get it, as the clinical trial’s data monitoring committee wants the study to roll on through its final analysis.
In the second largest M&A deal of the year, Merck & Co. Inc. has strengthened its cardiopulmonary portfolio with the $10 billion acquisition of London-based Verona Pharma plc. The deal brings Merck Ohtuvayre (ensifentrine) for chronic obstructive pulmonary disease.
A mixed bag of top-line phase II data prompted stellar stock results for Prokidney Corp. and its chronic kidney disease (CKD) and diabetes therapy, rilparencel. The results came from two arms of the study, each with its own treatment regime. The market heartily took to the results as the company’s stock (NASDAQ:PROK) closed 515% upward at $3.73 per share after starting the day under $1 each.
After delaying a June PDUFA date, the U.S. FDA has approved Kalvista Pharmaceuticals Inc.’s Ekterly (sebetralstat) for hereditary angioedema (HAE) in those aged 12 and older. The plasma kallikrein inhibitor now joins a market with previously approved drugs for the rare, genetic, life-threatening condition, as well as other companies with HAE drugs in development. Ekterly is the first orally-delivered on-demand treatment, as all others in the U.S. are intravenously or subcutaneously administered.
Unnatural Products Inc. notched another collaboration by signing on with Argenx SE in a multitarget research collaboration. Unnatural Products, which is getting up-front, near-term payments and R&D funding, could end up with about $1.5 billion in milestones and options payments plus tiered royalties on net sales.
