Local Biotechs Face Clinical Trial Setbacks

Watertown clinical-stage biopharma Neumora Therapeutics’ Phase 1 trial of NMRA-266 has been placed on clinical hold by the FDA based on pre-clinical data showing convulsions in rabbits. Approximately 30 participants have been dosed in the Phase 1 study, with no evidence of convulsions observed in any participant.

“We are disappointed with the unanticipated safety findings in rabbits and are discussing next steps with the FDA,” said Neumora president and CEO Henry Gosebruch. “In parallel, we’re continuing to make significant progress across the rest of our portfolio as we seek to fulfill our mission to develop medicines for serious brain diseases. We anticipate several important milestones including Phase 3 data in major depressive disorder and the initiation of a Phase 2 study in bipolar depression…and the initiation of a Phase 1b study in agitation in Alzheimer’s disease…”

Two days later in Cambridge, Sage Therapeutics announced disappointing results from its Phase 2 study of the oral medicine dalzanemdor in people with mild cognitive impairment in Parkinson’s Disease (PD). Based on the data, Sage does not plan any further development of dalzanemdor in PD.

“Although cognitive impairment is common in neurodegenerative disorders, the underlying pathophysiology and symptomatology in Parkinson’s disease is distinctive, and these results do not necessarily predict results with dalzanemdor in other neurodegenerative conditions. We look forward to the topline data readouts from the Phase 2 studies in Huntington’s disease and Alzheimer’s disease expected later this year,” said Sage CEO Barry Greene. Founded in 2010, Sage developed the only two FDA-approved treatments indicated for postpartum depression and is advancing a robust pipeline to target unmet needs in brain health. (Source: Neumora Therapeutics Website, 15 April, 2024 and Sage Therapeutics Website, 17 April, 2024)