The BioWorld Neurological Diseases Index (BNDI) ended May up 12.29% for the year, outpacing both the Nasdaq Biotechnology Index (up 4.95%) and the Dow Jones Industrial Average (up 6.18%), a reversal from 2025 when the BNDI trailed both broader benchmarks.
Achieving high marks in the phase II Amplify-BD trial for bipolar I and II depression, Autobahn Therapeutics Inc.’s elunetirom, a brain-penetrant central nervous system thyroid hormone receptor agonist, or thyromimetic, is moving toward a registrational path and a clinical readout in major depressive disorder.
Bial-Portela & Ca SA has patented new orexin OX1 receptor antagonists potentially useful for the treatment of anxiety disorders, depression, eating disorders, schizophrenia, obesity, sleep disorders, post-traumatic stress, substance abuse and dependence, among others.
Idefine, The Kleefstra Syndrome Foundation, has established a collaboration with UT Southwestern Medical Center to advance development of a potential gene therapy to treat Kleefstra syndrome, a rare neurodevelopmental disorder caused by changes or loss of the EHMT1 gene, which plays a critical role in brain development and function.
Changchun Genescience Pharmaceuticals Co. Ltd. has discovered new ubiquitin carboxyl-terminal hydrolase 30 (USP30) inhibitors potentially useful for the treatment of mitochondrial and Parkinson’s disease.
Montara Therapeutics Inc. has been awarded a research grant of approximately $1 million from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to advance a Brainonly therapy targeting mTOR for Parkinson’s disease.
Janssen Pharmaceutica NV has identified new endosomal/lysosomal proton channel TMEM175 activators that are potentially useful for the treatment of Parkinson’s disease.
Lysoway Therapeutics Inc. has been awarded an additional research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support development of its TMEM175 agonist program for Parkinson’s disease.
Voyager Therapeutics Inc. has obtained IND clearance from the FDA for VY-1706, the company’s investigational gene therapy for the treatment of Alzheimer’s disease.
Edgewise Therapeutics Inc. is pulling in $1.55 billion up front by selling its muscular dystrophy business, including its fast skeletal myosin inhibitor, sevasemten (EDG-5506), to Servier SA. The deal is potentially worth up to $2.65 billion when including a potential $1.1 billion in milestone payments.
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