The merger of Protagenic Therapeutics Inc. and Phytanix Bio Inc. combines two different approaches to treating obesity and metabolic issues. The all-stock deal will merge Protagenic’s peptide candidate in IND-enabling development for treating depression, anxiety, posttraumatic stress disorder and additional indications, along with Phytanix’s cannabinoid and cannabinoid-like molecules for bladder pain syndrome and treatment-resistant focal seizures.

It’s been a big week for Inozyme Pharma Inc. On the heels of a promising interim readout for phase III-stage enzyme replacement therapy (ERT) candidate INZ-701 in ENPP1 deficiency, the firm agreed to be acquired by Biomarin Pharmaceuticals Inc. in a deal valued at about $270 million, putting the rare disease ERT in the hands of an experienced commercial team.

Using a customized gene editing therapy, researchers at the Children’s Hospital of Philadelphia have reported success in treating an infant with a severe metabolic disorder. Kiran Musunuru, Barry J. Gertz Professor for Translational Research in the University of Pennsylvania’s Perelman School of Medicine, presented the case at the American Society of Gene and Cell Therapy’s 2025 annual meeting. The case study was simultaneously published in The New England Journal of Medicine.

In a deal that could top out at about $2.2 billion, Septerna Inc. is getting $200 million up front from Novo Nordisk A/S in a collaboration to develop oral treatments for obesity, type 2 diabetes and other cardiometabolic indications. There will be four programs for discovering, developing and commercializing small molecules targeting G protein-coupled receptors, which includes GLP-1, GIP and glucagon receptors, with both companies putting their shoulders to conducting research from discovery to choice of candidate.

Lysosomal storage disorder specialist Azafaros NV has raised €132 million (US$146.7 million) in a series B to fund two phase III studies of its lead product, a small-molecule glycosidase inhibitor that the company says has shown disease-modifying effects.

Immunity is not a function most people particularly associate with the liver. But because of its connection to the gut, the liver is exposed to bacterial metabolites as few other organs are. And when either the liver or the gut is not functioning well, it can adversely affect immunity as well. The liver is connected to the gut via both the biliary system and the portal vein. Those two conduits allow metabolites from the gut microbiome to influence what’s going on in the liver. Both liver and gut damage can affect this communication for the worse. And surprisingly, one of the consequences is immune dysfunction.

Ongoing policy issues in the U.S., including the Inflation Reduction Act and recent proposals under President Donald Trump’s administration, have wide ranging implications for the global biopharmaceutical industry, speakers at Bio Korea 2025 said May 8, including a heightened need for all biotechs to draft regulatory strategies.

“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.”