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.
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.
Visen Pharmaceuticals Co. Ltd.’s palopegteriparatide met both primary and secondary endpoints in the phase III Pathway trial conducted in China in adults with chronic hypoparathyroidism, according to top-line data. In the 26-week randomized, double-blind, placebo-controlled Pathway trial, 77.6% of patients treated with palopegteriparatide achieved the primary multicomponent endpoint compared to 0% for placebo.
After many months of jockeying, the U.S. FDA has approved Ascendis Pharma A/S’ hormone replacement therapy Yorvipath (palopegteriparatide) for treating hypoparathyroidism. Ascendis said this is the first and only treatment for adults with the rare endocrine disease.
China’s Visen Pharmaceuticals (Shanghai) Co. Ltd. is targeting an IPO on the Hong Kong stock exchange with three rare endocrine disease therapies licensed-in from Denmark’s Ascendis Pharma A/S, including U.S. FDA-approved Skytrofa (lonapegsomatropin).
China’s Visen Pharmaceuticals (Shanghai) Co. Ltd. is targeting an IPO on the Hong Kong stock exchange with three rare endocrine disease therapies licensed-in from Denmark’s Ascendis Pharma A/S, including U.S. FDA-approved Skytrofa (lonapegsomatropin).
