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.
Kangbaida (Sichuan) Biotechnology Co. Ltd. has disclosed pyroptosis and NLRP3 inflammasome inhibitors reported to be useful for the treatment of cancer, type 1 diabetes, gout, inflammatory disorders, multiple sclerosis, psoriasis, autoimmune diseases and Alzheimer’s disease.
Pramana Pharmaceuticals Inc. has been awarded $1 million from the Alzheimer’s Association to support IND-enabling development of PRM-914, the company’s once-daily oral small-molecule therapeutic for Alzheimer’s disease. The funding will support final toxicology and translational studies required to advance PRM-914 into first-in-human evaluation.
Sarepta Therapeutics Inc. has filed a clinical trial application (CTA) in New Zealand seeking clearance to initiate a first-in-human trial of SRP-1005 (formerly ARO-HTT). Pending approval, the INSIGHTT trial is anticipated to begin in the second quarter of this year.
Lysoway Therapeutics Inc. has completed IND-enabling studies for its lead TRPML1 agonist, LW-1017. A first-in-human study is planned in Australia, with dosing set to commence in May.
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