Karyopharm Therapeutics (KPTI) Announces SINE Compounds Being Evaluated as ALS Treatment

Karyopharm Therapeutics (NASDAQ: KPTI) reported that its SINE nuclear transport compounds are being evaluated for the treatment of Amyotrophic Lateral Sclerosis (ALS) and that preclinical data abstracts will be presented at two upcoming neuroscience meetings. A recent publication appearing in the journal Nature confirmed that nuclear transport is disrupted in a common mutation found in ALS. Given the tremendous unmet need for new treatments for ALS, Karyopharm is advancing its oral SINE™ compound KPT-350 for this indication. While Karyopharm’s primary clinical focus is in advancing its lead SINE™ compound oral selinexor for human cancer, other SINE™ compounds have already shown beneficial pharmacological effects in several neurodegenerative and anti-inflammatory preclinical models, including multiple sclerosis, systemic lupus and traumatic brain injury. The Nature publication entitled “The C9orf72 repeat expansion disrupts nucleocytoplasmic transport”…


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