Lexeo Therapeutics Inc. feels like it’s in a faster lane to a BLA for its Friedreich ataxia cardiomyopathy therapy after a talk with the U.S. FDA. The agency told Lexeo that LX-2006 could be on the accelerated approval path if data are pooled from ongoing phase I/II studies with pivotal data. New interim results from the studies showed improvement in patients’ cardiac and neurologic health. LX-2006 is a one-time intravenous infusion treatment for the progressive, autosomal recessive and neurodegenerative disease that characterized by the body slowly losing its ability to control itself. The company’s stock (NASDAQ:LXEO) reacted well to the FDA feedback, with shares popping 26% upward to $8.20 each at midday.

Biomea’s menin inhibitor shows durability in hard-to-treat diabetes

Among severe insulin-deficient type 2 diabetes patients, 12 weeks of 100-mg, once-daily dosing of Biomea Fusion Inc.’s icovamenib lowered HbA1c by 1.8% from placebo at the 52-week timepoint, a significant benefit over and above what was seen at 26 weeks. The selective covalent menin inhibitor also demonstrated a clinically meaningful impact on patients unable to reach their target HbA1c while taking a GLP-1-based therapy. They, too, showed a 1.8% placebo-adjusted reduction by week 52 after receiving multiple doses in the first 12 weeks. Leading up to the data release just after market close Oct. 6, Biomea’s stock (NASDAQ:BMEA) shot up 24%, or 52 cents, to close at $2.67. Shares tumbled by 37% to $1.68 in early trading Oct. 7, following the pricing of a $25 million public offering.

IRA forecloses Novo Nordisk challenge to Medicare negotiations

Like the federal district court before it, the U.S. Court of Appeals for the Third Circuit said it lacks jurisdiction to rule on the merits of Novo Nordisk A/S’ claim that CMS violated the Inflation Reduction Act (IRA) when it treated six of the company’s insulin aspart products as one negotiation-eligible single-source drug. The appellate court cited the IRA’s provision barring judicial review of CMS’ “determination of negotiation-eligible drugs” or “the determination of qualifying single source drugs.” Thus, the court said, “We cannot review CMS’ determinations or the internal processes CMS used to make them.” Besides being a big loss for Novo Nordisk, which faces a similar issue with its semaglutide franchise, the court’s decision could impact future development of drugs and drug-device combination products.

New AI model can assign certainty, or uncertainty, to predictions

“Have you ever asked ChatGPT anything, and the response was, ‘I don’t know?’” Cheong Jae-ho, a specialist in tumor biology and professor at Yonsei University College of Medicine, asked during an interview with BioWorld. “Probably not, and that is the problem with AI now.” Asking AI about the weather is one thing, but using AI programs to diagnose tumors could have life-altering consequences for patients. A team of U.S. and South Korean researchers have developed an AI model called MSI-SEER that can not only predict microsatellite instability-high (MSI-H) tumors based on tissue slides, but also flag “what it does not know.”