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.
Perceive Pharma Inc., a spin out from Perceive Biotherapeutics Inc., has closed a $15 million series A funding round to help it advance first-in-class therapies to prevent vision loss in glaucoma, and additional undisclosed disease areas.
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.
Becoming the second drug approved by the U.S. FDA for tenosynovial giant cell tumors, Deciphera Pharmaceuticals Inc.’s oral CSF1R inhibitor vimseltinib, newly branded Romvimza, could take significant market share from a competitor in the same class. A phase III trial showed a higher objective response rate with Romvimza than a separate phase III trial showed with Daiichi Sankyo Co. Ltd.’s Turalio (pexidartinib), and the latter’s label contains a boxed warning on rare but serious and sometimes life-threatening liver side effects.
Pliant Therapeutics Inc., which offered few details earlier this week when it voluntarily paused enrollment in the phase IIb Beacon-IPF trial testing bexotegrast in idiopathic pulmonary fibrosis (IPF), has taken an unusual step as it assembles a panel of outside experts to review unblinded data from the study with the goal of providing an independent recommendation.
Snagging its second U.S. FDA approval since being spun out of Pfizer Inc. in 2017, Springworks Therapeutics Inc. is aiming to position Gomekli (mirdametinib), cleared for neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) not amenable to complete resection, as a first-in-class drug for adults and potentially best-in-class option for children. The priority approval, which came after market close Feb. 11, more than two weeks ahead of the Feb. 28 PDUFA date, marks the second MEK1/2 inhibitor to hit the market for NF1-PN, a rare, genetic condition.
Bioversys AG has announced that its BV500 NTM program has reached its second milestone with the identification of up to five optimized lead compounds, under the CF AMR Syndicate collaborative discovery program agreement.
The closely watched chronic spontaneous urticaria (CSU, or hives) space chalked more early stage KIT inhibitor data as Third Harmonic Bio Inc. rolled out results from the phase I single and multiple ascending-dose trial with THB-335 in healthy volunteers, along with plans to move the oral candidate into phase II development by the middle of 2025.
Cognition Therapeutics Inc. evolved from the work of a neuroscientist and a chemist working in the San Francisco Bay area, seeking out targets to block the effects of Alzheimer’s disease. Since the company’s 2007 inception, it has received close to $200 million in U.S. NIH grant funding. Investors often tell CEO Lisa Ricciardi, who joined the company in 2020: “’That’s because you have a relationship with the FDA.’ Well, no. It’s because it’s competitive” and the company’s research has met the muster. “You have to apply two or three times. … It’s with rigor that these results are generated and that we’re able to get more funding.”
A mysterious turn in the phase IIb story with idiopathic pulmonary fibrosis (IPF) candidate bexotegrast sent shares of Pliant Therapeutics Inc. (NASDAQ:PLRX) down 60.6%, or $4.72, to close Feb. 10 at $3.07.
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