Only two days after Bridgebio Pharma Inc. impressed investors with data from BBP-418 in limb-girdle muscular dystrophy type 2I/R9, the company was back at it again, this time reporting positive top-line results from its global phase III study of encaleret in autosomal dominant hypocalcemia type 1, a genetic form of hypoparathyroidism.
MBX Biosciences Inc. posted positive top-line phase II study data in treating chronic hypoparathyroidism, positioning itself to take on a U.S. FDA-approved therapy for the rare endocrine disease. MBX’s Avail study of canvuparatide hit its primary endpoint in treating adults, demonstrating statistical significance at week 12 and positive six-month results from the open-label extension study. MBX said it is poised to begin a phase III study of once-weekly canvuparatide next year.
A recent paper in the Journal of Clinical Medicine quantified some of the potential bodily ravages ahead for patients with hypoparathyroidism (hypoPT), specifically the loss of kidney function, with the risk of chronic kidney disease going up every year by 11%. A handful of drug developers continue to advance prospects in hypoPT, where Ascendis Pharma A/S’ hormone replacement therapy Yorvipath (palopegteriparatide) – the first and only treatment for adults with the rare endocrine disease – has set the bar for efficacy.
Visen Pharmaceuticals Co. Ltd. announced its initial public offering (IPO) on the Hong Kong Stock exchange (HKEX) to raise roughly HKD$603.3 million (US$77.6 million) to advance its pipeline of endocrinology assets.
Visen Pharmaceuticals Co. Ltd. announced its initial public offering (IPO) on the Hong Kong Stock exchange (HKEX) to raise roughly HKD$603.3 million (US$77.6 million) to advance its pipeline of endocrinology assets.
Septerna Inc. has announced its decision to discontinue a healthy volunteer phase I trial of SEP-786, an oral small-molecule agonist of the parathyroid hormone 1 receptor being developed for the treatment of hypoparathyroidism.
Septerna Inc.’s stock plunged as much as 68% throughout the day Feb. 18 on news that the company was stopping a phase I trial of SEP-786 in healthy volunteers following two severe events of elevated unconjugated bilirubin in the highest dose cohort of the multiple ascending-dose portion of the study.
The approval of Ascendis Pharma A/S’ hormone replacement therapy Yorvipath (palopegteriparatide) hypoparathyroidism – the first and only treatment for adults with the rare endocrine disease – did little to sate the market’s appetite for new drugs in the indication, where a number of players are busy in various stages of development.
South San Francisco-based Septerna Inc. filed an S-1 with the U.S. SEC to conduct an IPO on Nasdaq about two years and eight months after launching operations with a $100 million series A led by Third Rock Ventures. The company is focused on G protein-coupled receptor oral small molecules derived from its Native Complex Platform, aimed at treating diseases within the endocrinology, immunology and inflammation, and metabolic diseases realms.
Three biopharmas debuted on Nasdaq Sept. 13, raising a combined $703 million for two Massachusetts-based companies focused on bifunctional antibodies for autoimmune diseases and cancer, and one Midwestern firm developing peptide therapies for endocrine and metabolic disorders.
