“I’m a pediatrician in metabolic diseases, and every day in my clinical work I’m confronted with our lack in effective therapies for our patients.” That was the sobering introduction by Sabine Fuchs in her talk at the 2025 Congress of the European Association for the Study of the Liver in Amsterdam this week. The nature of metabolic diseases makes it difficult to develop treatments for them. “There are over 1,500 diseases known by now, and it is just very difficult to develop therapies for each and every individual rare disease.”

Less than a week after announcing it had a new CEO, privately held Creyon Bio Inc. began a licensing and research partnership with Eli Lilly and Co. Creyon is getting $13 million up front and could bring in more than $1 billion in milestone payments. The two plan to find, develop and commercialize RNA-targeted oligonucleotide treatments for a range of diseases.

The National Institutes of Health (NIH) is adopting a new initiative to expand innovative, human-based science while reducing animal use in research. Developing and using alternative nonanimal research models aligns with the FDA’s recent initiative to reduce testing in animals.

Metabolic dysfunction-associated steatotic liver disease (MASLD) is a growing global health concern, with an estimated prevalence of around 25% worldwide. This chronic liver condition is characterized by lipid deposition in the liver, which can lead to inflammation, scarring and even liver cancer if left untreated.

Ensem Therapeutics Inc. has gained IND clearance from the FDA for ETX-636, a novel allosteric pan-mutant-selective PI3Kα inhibitor and degrader. A first-in-human trial will begin this quarter. The phase I/II study will evaluate ETX-636 administered alone and in combination with fulvestrant in participants with advanced solid tumors, including breast cancer, harboring a PI3Kα mutation.

Elix Inc. has entered into a drug discovery collaboration with Prism Biolab Co. Ltd. to accelerate AI-driven drug discovery for challenging protein-protein interaction targets. The partnership will combine Elix’s AI drug discovery platform with Prism’s proprietary small-molecule peptide mimetic Pepmetics technology for controlling protein-protein interactions.

In a roadmap to change animal testing requirements for INDs, the U.S. FDA said its new approach will improve drug safety, hasten the evaluation process, and lower costs for companies and patients. It’s another step in a process of changing rules put in place decades ago.

Orexo AB has announced promising in vivo proof-of-concept data for a powder-based intranasal vaccine candidate based on Abera Bioscience AB’s BERA vaccine platform and formulated with Orexo’s Amorphox technology.