EMA approval of the Alzheimer’s disease therapy Leqembi (lecanemab) has stalled once again, after the European Commission did not as usual nod through the agency’s recommendation, but told it to examine safety data that have recently become available.

California Institute for Regenerative Medicine (CIRM) has awarded a $5.8 million translational research grant to a scientist at the University of California, Los Angeles (UCLA) to further support the development of a hematopoietic stem cell (HSC) gene therapy to treat Angelman syndrome, including preparation of a pre-IND package submission to the FDA.

An effort two decades in the making, Vertex Pharmaceuticals Inc.’s suzetrigine gained U.S. FDA approval as the first drug targeting the NaV1.8 pain signal. Branded Journavx, the oral small molecule is cleared for use as a non-opioid option for treating moderate to severe acute pain.

Alterity Therapeutics Ltd. reported positive top-line phase II results for lead candidate ATH-434 for treating multiple system atrophy, a rare neurological disorder similar to Parkinson's disease.

Neu Health Ltd. received $2 million in funding from Cedars-Sinai Intellectual Property Company and Oxford Science Enterprises (OSE) to bring its platform, designed for Parkinson’s disease and dementia care, into the U.S.

Researchers from Columbia University Irving Medical Center published data from a study that assessed GiGA1, a selective G protein-gated inwardly rectifying potassium channel (GIRK) activator, in physiologically and clinically relevant ex vivo mouse models of tumor-associated and provoked seizures.

Harness Therapeutics Ltd. has raised fresh financing to further develop its technology for upregulating the translation of mRNA into proteins, and in particular to take on a previously undruggable target in Huntington’s disease.

Bioage Labs Inc. has nominated BGE-102 as a development candidate. The orally available, small-molecule NLRP3 inhibitor has high potency and high brain penetration.