Stoke Therapeutics Inc.’s speeded-up timeline for zorevunersen, the antisense oligonucleotide in development with Biogen Inc. as a first-in-class potential disease-modifying treatment for Dravet syndrome, put the rare, severe form of lifelong epilepsy in the spotlight. The news involved completion of enrollment and a phase III data readout from the Emperor study, as officials said signups of 150 patients are expected in the second quarter of the year, which puts the study on track for data in mid-2027.
With rumors regarding a couple of potential mega-mergers making the rounds, the week of the annual J.P. Morgan Healthcare Conference kicked off with the official disclosure of some billion-dollar collaborations, leading with Abbvie Inc.’s exclusive licensing deal with Remegen Co. Ltd. for PD-1/VEGF-targeted bispecific antibody RC-148.
For individuals with major depressive disorder (MDD) that does not respond to pharmacotherapy, recent decisions by the U.S. FDA enabling at-home use of neuromodulation devices may provide a bright spot in dark days. Neurolief Ltd. received FDA premarket approval for Proliv Rx, a physician-directed, brain neuromodulation therapy indicated for use as an at-home adjunctive treatment for adults with MDD who failed to respond adequately to at least one previous antidepressant on Jan. 7. That decision follows the December 510(k) clearance for at-home use of the Flow brain-stimulation device by Flow Neuroscience AB as either a monotherapy or as an adjunctive treatment.
Keiferx LLC has entered into a research collaboration and option agreement with Amneal Pharmaceuticals Inc. to support the pre-IND development of KFRX-06, Keiferx’s brain-penetrant preclinical candidate designed to inhibit leucine-rich repeat kinase 2 (LRRK2), a gene strongly implicated in Parkinson’s disease biology.
While Alzheimer’s disease is known for involving amyloid-b plaques and neurofibrillary tangles, it also involves metabolic dysfunction, further research into which could help scientists understand how the disease occurs and how it can be treated.
Entrada Therapeutics Inc. has updated progress across its preclinical portfolio of RNA-based programs for the potential treatment of neuromuscular and ocular diseases.
Dewpoint Therapeutics Inc. has announced the selection of a development candidate for its TDP-43 program. The first-in-class small molecule is designed to correct disease-associated TDP-43 condensates, restoring normal TDP-43 function and addressing the core molecular pathology that drives neurodegeneration in amyotrophic lateral sclerosis (ALS) and related diseases.
Debate over Vanda Pharmaceuticals Inc.’s Hetlioz (tasimelteon) for use in jet lag disorder looks to continue after the U.S. FDA rejected the latest supplemental NDA submission, with the agency’s Center for Drug Evaluation and Research concluding the application cannot be approved in its current form.
Shanghai Huilun Pharmaceutical Co. Ltd. has described compounds acting as 5-HT2A receptor agonists reported to be useful for the treatment of depression.
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