Sangamo Therapeutics Inc. rolled out pleasing preliminary data from the first four patients treated in the phase I/II study known as Staar, evaluating isaralgagene civaparvovec, or ST-920, a gene therapy for Fabry disease. Results as of the Sept. 17, 2021, cutoff date from the four patients in the first two dose cohorts showed that the drug was generally well-tolerated, and all four patients exhibited above normal alpha-galactosidase A activity.
Antios Therapeutics Inc. followed up its $96 million series B financing in April with another $75 million series B-1 round that reflects faith in the potential of lead candidate ATI-2173, bound for phase IIb investigation as the backbone of a once-daily, curative regimen in chronic hepatitis B virus (HBV) infection.
Though Wall Street may not have caught on to the value of Clene Inc.’s phase II results with gold nanocrystal suspension CNM-Au8 in amyotrophic lateral sclerosis (ALS), CEO Rob Etherington said his firm is “truly excited” about the data, which bode well for the next stage of development, already underway.
Abbvie Inc.’s FDA clearance of Vuity (pilocarpine HCl ophthalmic solution) 1.25% for the treatment of presbyopia in adults, could bring relief to about 128 million Americans: almost half of the U.S. population, the company said. It’s the first and only eye drop given U.S. regulators’ go-ahead for what’s known as age-related blurry near vision – but plenty more have generated clinical data.
Travere Therapeutics Inc.’s mid-September deal with Vifor Pharma Group – a collaboration and licensing agreement for the commercialization of sparsentan in Europe, Australia and New Zealand – brought further, well-deserved attention to the dual endothelin angiotensin receptor antagonist, in the works for rare kidney disorders, specifically focal segmental glomerulosclerosis and IgA nephropathy.
As the company unveiled 18-month results from the phase III study called Helios-A, Alnylam Pharmaceuticals Inc.’s president of R&D, Akshay Vaishnaw, said “new exploratory data with the cardiac endpoints are extremely encouraging.” The study is testing RNAi therapy vutrisiran in polyneuropathy associated with hereditary transthyretin-mediated (hATTR) amyloidosis, where Helios-A met all secondary endpoints measured at 18 months.
What Cortexyme Inc.’s chief operating officer Christopher Lowe called “a giant step forward” in Alzheimer’s disease (AD) research with atuzaginstat (COR-388) was viewed differently by Wall Street, which walloped shares (NASDAQ:CRTX) by 76%, or $44.17, causing the stock to close at $13.51.
Mozart Therapeutics Inc. CEO Katie Fanning said the firm’s $55 million series A financing will allow the filing of an IND, probably in early 2024, for a prospect in celiac disease. Founded in July 2020, Seattle-based Mozart is based on research into the CD8 T-cell regulatory network, which has been found to play an important role in surveillance, recognition and elimination of inappropriately activated autoreactive and pathogenic immune cells.
CEO Gil Beyen of Erytech Pharma AS said the firm is mulling next steps in the aftermath of phase III data with eryaspase in second-line pancreatic cancer, where the compound – which consists of L-asparaginase encapsulated in a donor-derived red blood cell – missed its primary endpoint of overall survival. “We will have to regroup and see how we can further continue on the different programs that are ongoing,” he said during a conference call with investors.
Metacrine Inc.’s departure, in the wake of mixed phase IIa results, from the fiercely competitive nonalcoholic steatohepatitis (NASH) space with farnesoid X receptor (FXR) agonist MET-642, put a serious dent in shares and brought implications for other players. The San Diego-based firm’s stock (NASDAQ:MTCR) closed at $1.63, down $2.20, or 57%, as its plans to shift emphasis from NASH to inflammatory bowel disease (IBD) were made known.
