Sio Gene Therapies Inc. CEO Pavan Cheruvu said the company sees no “significant rate limiters to moving forward” with FDA fast-tracked AXO-AAV-GM1 for the treatment of GM1 gangliosidosis. Shares of the New York-based firm (NASDAQ:SIOX) closed at $2.36, up 21 cents, as Wall Street welcomed positive interim data from the ongoing phase I/II study with the adeno-associated viral vector 9-based gene therapy for GM1 gangliosidosis.
Stealth Biotherapeutics Corp.’s refusal to file (RTF) letter from the FDA regarding the NDA for elamipretide, a candidate that targets mitochondria in the treatment of Barth syndrome, hardly knocked the stock, as investors likely saw the bad news coming.
Phase II results from Atea Pharmaceuticals Inc. with AT-527 in COVID-19 treatment had pundits scrambling to stack the odds of success for the direct-acting oral antiviral against those of Merck & Co. Inc.’s molnupiravir, though cross-trial comparisons are beset by the usual hurdles, with two especially dramatic ones.
Omeros Corp.’s complete response letter (CRL) from the FDA regarding its BLA for narsoplimab did not come as much of a surprise to Wall Street, but still was enough to drive shares (NASDAQ:OMER) down 26.6% to close Oct. 18 at $5.67, a loss of $2.06.
A few months after disclosing its merger with Chiasma Inc., Amryt plc rolled out second quarter financials pleasing to Wall Street, where considerable interest has begun to manifest in the company’s epidermolysis bullosa program with Filsuvez, also known as Oleogel-S10.
Recent news that The University of Texas MD Anderson Cancer Center is teaming up with Schrödinger Inc. to work on the latter’s WEE1 inhibitor – along with data disclosed at the 2021 meeting of the European Society for Medical Oncology (ESMO) – shone a light on the gatekeeper checkpoint kinase, which a number of players are busy exploring, though nothing in the class has been approved so far.
Poseida Therapeutics Inc.’s R&D Day in February – where much of its technology was made public for the first time – created “a flood of interest” in deals and officials were “pretty selective,” said CEO Eric Ostertag, whose remarks came as the company nailed down a whopping research collaboration and exclusive license agreement with Takeda Pharmaceutical Co. Ltd. The arrangement will deploy Poseida’s Piggybac and Cas-CLOVER, as well as biodegradable DNA and RNA nanoparticle delivery technology and other genetic engineering platforms to come up with as many as eight gene therapies.
Supernus Pharmaceuticals Inc. CEO Jack Khattar, in disclosing his firm’s plan to acquire Adamas Pharmaceuticals Inc., said the team there has made “significant progress in redirecting” the Parkinson’s disease (PD) drug Gocovri (amantadine), but plenty more potential remains on the table with the oral selective RET kinase inhibitor.
Allogene Therapeutics Inc. officials took many questions but had few answers during a conference call regarding the FDA’s clinical hold after the report of a chromosomal abnormality in ALLO-501A CAR T cells in a patient treated in the phase I/II Alpha2 study.
With its topical gel for primary axillary hyperhidrosis (excessive underarm sweating) in adults and children 9 years of age and older, Brickell Biotech Inc. is gearing up to challenge Journey Medical Corp.’s Qbrexza (glycopyrronium), an anticholinergic agent contained in a single-use, pre-moistened, medicated cloth.
