At the 2025 Alzheimer’s Association International Conference, one of the bigger splashes was made by an experimental cardiovascular drug. Newamsterdam Pharma Co. NV presented data showing that its cholesterol drug obicetrapib lowered levels of the Alzheimer’s disease (AD) biomarker p-tau217. The data were generated as part of the Broadway trial, a phase III trial to evaluate its ability to lower LDL-C in adult patients with established atherosclerotic cardiovascular disease and/or heterozygous familial hypercholesterolemia. That means it was not primarily designed to show an effect on AD progression. And p-tau217 is a biomarker, not a cognitive or clinical measurement. Improvement occurred not just in p-tau127, but across a group of biomarkers that measure inflammation, neuronal damage, and other aspects of Alzheimer’s disease. And the treatment worked better in older patients, and in homozygotes for the ApoE4 allele, which is by far the strongest risk factor for late-onset Alzheimer’s disease.

Prasad’s CBER departure spurs Street celebrations

The news that Vanay Prasad has stepped down as CBER director had some biotech stocks literally jumping in joy this morning. Replimune Group Inc.’s stock (NASDAQ:REPL) shot up 93% today, while Sarepta Therapeutics Inc. (NASDAQ:SRPT) and Capricor Therapeutics Inc. (NASDAQ:CAPR) saw jumps of 22% and 23%, respectively. Both Replimune and Capricor had received complete response letters from CBER over the past few weeks, and Sarepta has been engaged in a heated back-and-forth with CBER over the continued marketing of its muscular dystrophy gene therapy Elevidys (delandistrogene moxeparvovec). Those actions were among several controversial regulatory decisions coming out of CBER since Prasad took the reins in May, said Leerink Partners LLC’s Joseph Schwartz. Prasad’s departure comes on the heels of the FDA giving its blessing this week for Sarepta to resume marketing of Elevidys.

Rezdiffra combo eyed via Madrigal’s potential $2B-plus CSPC deal

Madrigal Pharmaceuticals Inc. signed an exclusive global license agreement with CSPC Pharmaceutical Group Ltd., of Shijiazhuang, China, for SYH-2086, a preclinical oral, small-molecule glucagon-like peptide-1 (GLP-1) receptor agonist and orforglipron derivative. Clinical development will start in the first half of next year. Conshohocken, Pa.-based Madrigal wants to develop new combo treatments for metabolic dysfunction-associated steatohepatitis (MASH) using the firm’s Rezdiffra (resmetirom), the thyroid hormone receptor-beta agonist approved by the U.S. FDA in March of last year for the indication, previously known as nonalcoholic steatohepatitis. The plan with CSPC’s GLP-asset is to come up with a once-daily, well-tolerated pill for MASH. Under the terms, CSPC is banking an up-front payment of $120 million and is eligible to collect up to $2 billion if certain development, regulatory, and commercial milestones are achieved, plus royalties on net sales.

Cardiff has positive cancer data but a stock slide, too

Cardiff Oncology Inc.’s mid-stage and ongoing clinical trial of its PLK1 inhibitor in colorectal cancer produced promising data but the company’s stock had lost more than a quarter of its value at midday. The randomized phase II study of onvansertib combined with a standard-of-care treatment in first-line, RAS-mutated metastatic colorectal cancer produced a 49% confirmed objective response rate at the 30-mg dose level compared to 30% in the control arm. The new results are the latest from the study that in December showed an objective response rate of 50%, 64% and 57% at 20 mg, 30 mg and all onvansertib patients vs. 33% for the control group. Cardiff’s shares (NASDAQ:CRDF) were down 29% at midday to $2.35 each.

Galapagos does an about-face on cell therapy, small-molecule plans

As biotech zombies with failed programs and money in hand go, Galapagos NV is a notable example, with a number of misses in the clinic, $3.1 billion in the bank, and a market capitalization of $2.19 billion. The $3.1 billion cash balance was reported in the 2025 half-year results when the company announced an about-face on the previous rescue plan to ditch small molecules and focus attention on commercializing cell therapy assets acquired in 2022. But on May 13, Galapagos said it was having a rethink and announced July 23 the intention to offload the cell therapy business instead, conserving as much cash as possible to acquire new programs and rebuild the pipeline.

Prime editing could cure a rare childhood hemiplegia disorder

An experimental gene therapy based on the prime editing technique could become an effective treatment for alternating hemiplegia of childhood (AHC), a severe and currently incurable rare disease. David Liu’s lab at the Broad Institute, the inventor of this gene edition methodology, together with scientists from The Jackson Laboratory, successfully reversed the effects of five mutations associated with this disorder in a mouse model. “This study is an important milestone for prime editing and one of the most exciting examples of therapeutic gene editing to come from our team,” said co-senior author David Liu, professor and director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute.

Also in the news

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