Madrigal Pharmaceuticals Inc.’s long-awaited business development pact became reality by way of an exclusive global license agreement that could be worth more than $2 billion with CSPC Pharmaceutical Group Ltd., of Shijiazhuang, China, for SYH-2086. The candidate is a preclinical oral, small-molecule glucagon-like peptide-1 (GLP-1) receptor agonist and orforglipron derivative.
PTC Therapeutics Inc. will be launching its oral phenylketonuria therapy, Sephience (sepiapterin) in both the U.S. and Europe this summer, following the U.S. FDA approval just ahead of its July 29 PDUFA date. Sephience previously gained marketing authorization by the European Commission, roughly three months after a thumbs up from the EMA’s Committee for Medicinal Products for Human Use.
With more-than-satisfying phase III data in hand, Minneapolis-based Celcuity Inc. is eyeing an NDA submission in the fourth quarter of this year for gedatolisib (geda) in breast cancer. Shares of the company (NASDAQ:CELC) closed July 28 at $36.79, up $23.02, or 167%, after Wall Street learned of stellar top-line results from the PIK3CA wild-type cohort of the phase III Viktoria-1 trial.
After Recognify Life Sciences Inc.’s phase IIb failure in treating the cognitive impairment in those with schizophrenia, the indication is no closer to a U.S. FDA approved therapy. The placebo-controlled study of inidascamine missed its primary endpoint to improve cognition, joining a list of companies that have tried and failed to find a successful treatment.
Suzhou, China-based Kintor Pharmaceutical Ltd.’s KX-826 met the primary endpoint in a phase II trial for treating male adult androgenetic alopecia (AGA). Kintor is developing KX-826 (pyrilutamide), an androgen receptor inhibitor, for topical treatment of AGA as well as acne vulgaris.
Becoming the first approved treatment for chronic hand eczema in the U.S., Leo Pharma A/S’s Anzupgo (delgocitinib) received FDA clearance for an indication that affects 10% of adults worldwide. The cream, dosed at 20 mg/g and applied topically, will treat moderate to severe cases in those not responsive to, or unable to take, topical corticosteroids.
Amplia Therapeutics Ltd. is raising AU$27.5 million (US$18.12 million) to advance lead compound narmafotinib (AMP-945), a focal adhesion kinase (FAK) inhibitor, into new indications beyond pancreatic cancer.
Apnimed Inc.’s lead oral therapy for obstructive sleep apnea has delivered a second round of positive phase III data, bringing a potential U.S. FDA filing closer. Top-line results taken from the second pivotal late-stage study show AD-109 (aroxybutynin 2.5mg/atomoxetine 75 mg) hit its primary endpoint with clinically meaningful and statistically significant drops in airway obstruction at 26 weeks.
Following up on the $175 million it raised in September 2023, Avalyn Pharma Inc. secured an additional $100 million in an oversubscribed series D financing round to support development of its inhaled treatments for pulmonary fibrosis.
As Wall Street awaits key phase III data from Abivax SA with microRNA-124 (miR-124)-targeting obefazimod in ulcerative colitis, due this quarter, the inflammatory bowel disease (IBD) space – which also takes in Crohn’s disease – continues to grow and shift with new findings across varied fronts.
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