In two phase III studies, Merck & Co. Inc.’s oral, once monthly proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor produced statistically significant and clinically meaningful cuts in low-density lipoprotein cholesterol levels. The results come from the Coralreef studies of enlicitide decanoate in adults with hyperlipidemia. Both studies, with results on the way from a third clinical trial of the PCSK9 inhibitor in the same indication, hit their primary and key secondary endpoints. PCSK9 drugs approved in the U.S. include subcutaneously administered Leqvio (inclisiran, Novartis AG), Praluent (alirocumab, Sanofi SA) and Repatha (evolocumab, Amgen Inc.).

Metsera’s long-acting amylin analogue delivers in phase I

The big question going into the phase I readout for Metsera Inc.’s amylin analogue, MET-233i, was whether findings would support once-monthly dosing for the potential obesity candidate. And they did, with the company reporting what it called the “most durable pharmacokinetic profile of any known amylin analogue,” with an observed half-life of 19 days. Even better, Metsera executives pointed to results showing MET-233i yielded dose-dependent weight loss of up to 8.4% and offer an exposure profile that aligns with the company’s GLP-1 drug, MET-097i, supporting combination studies.

Open-source AI model can predict small-molecule binding affinity

Researchers at the Massachusetts Institute of Technology and Recursion Pharmaceuticals Inc. have released an open-source AI model that can predict the binding strength of small molecules as well as structures of proteins and biomolecular complexes. The model, which is called Boltz-2 and was released by the research team on the developer platform Github on Friday, addresses a major bottleneck in drug discovery with its improved ability to predict binding strengths.

Corestemchemon preps US BLA filing for stem cell ALS therapy

Corestemchemon Inc. is planning to file a BLA for Neuronata-R (lenzumestrocel) by the end of 2025 to gain accelerated approval from the U.S. FDA. Neuronata-R is an autologous bone marrow-derived mesenchymal stem cell therapy that first gained approval in South Korea in 2014 to delay the progression of amyotrophic lateral sclerosis. The FDA filing announcement came as a surprise, however, considering Neuronata-R failed to meet its primary endpoint in a top-line phase III Alsummit study readout in December 2024. Upon analyzing the raw data, however, the company’s bioinformatics and clinical team identified a statistically significant difference between the control group and a subset of patients labeled as the slow progressor group.

MHRA offers clarity for bacteriophage development, licensing

The U.K. Medicines and Healthcare products Agency (MHRA) has issued its first official guidance on how to develop bacteriophages as licensed medicinal products. This covers personalized phage therapies designed for specific patients – at present the only form in which they are available – but also is relevant to the development of off-the-shelf products for treating common infections. The guidance brings together relevant standards to provide clarity for companies and researchers, said Lawrence Tallon, the recently appointed new chief executive of MHRA. “We’re committed to working with industry to support innovation in this space,” Tallon said.

Canceling claims not an appropriate sanction, USPTO says

Absent extraordinary circumstances, the Patent Trial and Appeal Board (PTAB) “should never cancel claims it has not determined to be unpatentable as a sanction” for misconduct during a board hearing, according to the acting director of the U.S. Patent and Trademark Office (USPTO). That decision, handed down last week, modified the previous director’s review decision upholding a PTAB decision to cancel 183 challenged patent claims as a sanction against Longhorn Vaccines and Diagnostics LLC for misconduct.

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