Positive results for Revolution Medicines Inc.’s phase I studies of the RAS-blocker daraxonrasib for pancreatic ductal adenocarcinoma prompted the company to say it’s time for a phase III study in the aggressive cancer. Revolution said it plans to begin a global, randomized and registrational trial in first-line metastatic disease sometime in the fourth quarter of 2025. The data had propelled the company’s stock (NASDAQ:RVMD) 12% higher at midday, with shares going for $45.40 each. The U.S. FDA granted the RAS(ON) multi-selective inhibitor breakthrough therapy designation in June.

Phase I outcome bolsters Maze thesis in PKU, CKD

Shares of Maze Therapeutics Inc. (NASDAQ:MAZE) were trading at $22.82, up $6.80, or 42%, after the firm offered positive results from the phase I healthy volunteer study of MZE-782, an oral small molecule targeting the solute transporter SLC6A19. Maze said the latest data further suggest that the drug could be a best-in-class therapy for patients with the metabolic disorder phenylketonuria plus a first-in-class therapy for patients with chronic kidney disease. Separately, the company raised $150 million in a private placement.

Neuro-focused LB breaks IPO drought with $285M raised

LB Pharmaceuticals Inc. became the first traditional biopharma company to price an IPO in the U.S. since February, raising $285 million through the upsized sale of 19 million shares at $15 apiece. Underwriters have a 30-day option for another 2.85 million shares, which are now listed on Nasdaq under the ticker symbol LBRX. By midday Sept. 11, shares of the company, which is developing LB-102 for neuropsychiatric disorders, were up 19% to $17.92. While two botanical drug companies, Jyong Biotech Ltd. and Curanex Pharmaceuticals Inc., conducted IPOs on Nasdaq in June and August, the most recent traditional biopharma IPO in the U.S. was done by Aardvark Therapeutics Inc. in February, when it raised $97.9 million.

Capsida's gene therapy trial paused upon first patient's death

Two months after starting the phase I/II Synrgy trial with its gene therapy CAP-002, enrolling 12 pediatric patients with rare disease STXBP1 encephalopathy, Capsida Biotherapeutics paused the study following the death of the trial’s first patient. “We understand this devastating news will raise questions and uncertainty, and we are working with urgency to gather information and find answers,” Capsida said in a letter on its website to the STXBP1 community. The company has alerted the U.S. FDA of the death and is working to “determine the root cause of the patient’s passing.” Synrgy is evaluating adeno-associated virus (AAV)-based CAP-002 in children ages 18 months to 7 years with syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE), a rare neurodevelopmental disorder affecting up to one in 26,000 births globally and about 5,000 pediatric patients in the U.S. and Europe. STXBP1-related disorders were discovered in 2008 in studies of some patients with Ohtahara syndrome, according to the STXBP1 Foundation, which was founded in 2017 by parents.

US Congress, courts weighing 340B program – again

The debate around the U.S. 340B prescription drug discount program is once again heating up in court and in Congress. Even as the American Hospital Association this week called on the FTC and Department of Justice to investigate alleged antitrust issues with the rebate models a few drug companies have proposed, some members of Congress raised concerns about how providers are abusing the program. Those concerns are based on a new Congressional Budget Office report that found the 340B program is a profit stream for covered entities and a factor in driving vertical consolidation among providers, increasing overall health care costs. Meanwhile, a U.S. appellate court heard arguments this week on whether states can fill in the silences of the federal law that created the program.

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Abcellera, Acesis, Actuate, Akeso, Alnylam, Amylyx, Avidity, Bioxcel, Camp4, Celcuity, Corcept, Coregen, Dianthus, Greenwich, Hoth, Instil, Intensity, Johnson & Johnson, Kriya, Maxymune, Mei, Novo Nordisk, Odyssey, Perspective, PMV Rapport, Resolution, Sanofi, Scipher, Serina, Sobi, Travere