Allos Pharma has announced the exclusive license rights on arbaclofen in Fragile X Syndrome (FXS). The company is preparing the regulatory path for market authorization. Allos Pharma was founded to translate breakthrough discoveries in neurobiology into innovative drug treatments that improve the lives of patients and families with Fragile X Syndrome, autism, and other disorders of brain development.
"Arbaclofen is the active enantiomer of baclofen, a GABA-B receptor agonist. Animal research confirmed that arbaclofen can correct core pathophysiology in Fragile X that arises from dysregulation of neuronal protein synthesis and hyperexcitability" said Professor Mark Bear and co-founder of Allos Pharma.
"Statistically significant and clinically meaningful efficacy was observed in a prior Phase 3 trial conducted by Seaside Therapeutics in humans with Fragile X Syndrome." said Randy Carpenter, MD, and co-founder of Allos Pharma.
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"The Fragile X professional field is excited about the opportunity to further explore arbaclofen in fragile X syndrome. The Fragile X community deserves this follow up work and the NFXF fully supports the professionals who have continually advocated for the access of the past trial data to inform future work to make arbaclofen available to individuals with Fragile X Syndrome. NFXF supports the future work of Allos Pharma and looks forward to working in partnership to better the lives of individuals with Fragile X Syndrome and their families." said Linda Sorensen, Executive Director of the National Fragile X Foundation (NFXF).