Medicxi Ventures’ asset-focused investment model has scored another hit, in a $450 million deal with Servier SA, for KER-0193, an oral small molecule that is ready for phase II development in the treatment of the inherited autism spectrum disorder, fragile X.

Braveheart Bio Inc. is paying $65 million up front to license Jiangsu Hengrui Pharmaceuticals Co. Ltd.’s oral hypertrophic cardiomyopathy drug candidate called HRS-1893.

Argenica Therapeutics Ltd. saw its stock drop 56% following mixed results for its ARG-007 phase II Seancon trial in acute ischemic stroke patients undergoing endovascular revascularization, or thrombectomy.

As Wave Life Sciences Ltd. released more results from its ongoing phase Ib/IIa study of small interfering RNA editing oligonucleotide WVE-006 for treating alpha-1 antitrypsin deficiency, the company’s stock (NASDAQ:WVE) dropped by 16.8% to close at $8 on Sept. 3.

Charm Therapeutics Ltd. is heading to the clinic after closing an oversubscribed series B, which will fund initial development of a menin inhibitor that is AI-designed to circumvent shortcomings of first-generation molecules.

Following positive data from competitors earlier this year, United Therapeutics Corp. rolled out impressive and unexpected phase III results of nebulized Tyvaso (treprostinil) for idiopathic pulmonary fibrosis (IPF), setting the stage for an sNDA filing with the U.S. FDA next year.  If approved for the indication, United, of Silver Spring, Md., and Research Triangle Park, N.C., could tap into what analysts say is a $4 billion-plus market.

Eli Lilly and Co.’s regulatory ducks are lined up nicely with the latest positive top-line results from the phase III Attain-2 trial testing orforglipron, an oral glucagon-like peptide 1 (GLP-1) receptor agonist, in adults with obesity or overweight and type 2 diabetes.