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Allos Pharma Acquires Global Rights to Arbaclofen, Reports Positive ASD Trial Results and Grant Award

Allos Pharma Inc., announces key milestones advancing its mission to deliver effective treatments for Autism Spectrum Disorder (ASD), Fragile X Syndrome, and related neurodevelopmental disorders.

articleAllos S.a.August 21, 20252/company/allos-sa/news/allos-pharma-acquires-global-rights-to-arbaclofen-reports-positive-asd-trial-results-and-grant-award
Allos Pharma Acquires Global Rights to Arbaclofen, Reports Positive ASD Trial Results and Grant Award

About this update from Allos S.a.

[{"type":"text","content":"CAMBRIDGE, Mass., Aug. 21, 2025 /PRNewswire/ -- Allos Pharma Inc., announces key milestones advancing its mission to deliver effective treatments for Autism Spectrum Disorder (ASD), Fragile X Syndrome, and related neurodevelopmental disorders.","length":243,"tagName":"p"},{"type":"text","content":"Global Rights to Arbaclofen ","length":27,"tagName":"p"},{"type":"text","content":"Allos has secured worldwide rights to arbaclofen, a selective GABA-B receptor agonist previously evaluated in multiple Phase II and III trials in ASD, Fragile X syndrome (FXS) and 16p11.2 deletion syndrome. This transfer includes clinical data, regulatory assets, and manufacturing capabilities.","length":295,"tagName":"p"},{"type":"text","content":""This marks a foundational moment for Allos," said Dr. Randall Carpenter, Co-founder and CMO of Allos Pharma. "Owning full rights to arbaclofen enables us to move swiftly and with strategic clarity as we build a sustainable pipeline targeting core features of ASD and genetically defined neurodevelopmental syndromes."","length":338,"tagName":"p"},{"type":"text","content":"Encouraging ASD Clinical Results","length":32,"tagName":"p"},{"type":"text","content":"Recently completed placebo-controlled trials in Canada and the EU, sponsored by the POND Network and the EU AIMS-2-Trials Consortium respectively, independently replicate the clinically meaningful improvements in social function observed in the Phase II Seaside Therapeutics clinical trial. Significant improvements were also observed in the core ASD domains of communication and restricted/repetitive behaviors. These results provide the strongest efficacy seen to date in ASD pharmacotherapy.","length":494,"tagName":"p"},{"type":"text","content":"16p11.2 Data Analysis","length":21,"tagName":"p"},{"type":"text","content":"The recently completed L16hthouse study, sponsored by Clinical Research Associated LLC, was motivated by strong efficacy in preclinical models of 16p11.2 deletion syndrome, the most common genetic copy number variant cause of ASD. The results of this pilot study inform selection of primary endpoints and patient stratification strategies for future precision trials in this genetically defined orphan disease.","length":410,"tagName":"p"},{"type":"text","content":"Grant Awarded ","length":13,"tagName":"p"},{"type":"text","conte...

More updates from Allos S.a.

Fragile X Syndrome16p11.2 deletion syndromeAllosneurodevelopmental disordersAutism Spectrum Disorderworldwide rightsreceptor agonistPharmaRandall Carpenter