Allos Pharma, Inc.

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. Global Rights to Arbaclofen 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. "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." Encouraging ASD Clinical Results 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. 16p11.2 Data Analysis 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. Grant Awarded Allos has also received a non-dilutive grant from the Simons Foundation. The grant will support further clinical development of arbaclofen and enable biomarker-driven analyses in ASD. About Arbaclofen Arbaclofen is a GABA-B receptor agonist that modulates excitatory/inhibitory balance and metabolic pathways in the brain to correct core molecular pathophysiology in preclinical models of ASD, FXS, and 16p11.2 deletion syndrome. It has been administered to over 700 children, adolescents, and adults with ASD and FXS in previous trials and demonstrated exceptional safety and tolerability, including in long-term, open-label studies. About Allos Pharma Allos Pharma is a biopharmaceutical company developing novel therapies for autism and genetically defined neurodevelopmental disorders. The company's lead candidate, arbaclofen, targets core domains and associated symptoms of ASD, Fragile X syndrome, and 16p11.2 deletion syndrome. Allos is committed to rigorous science and clinical excellence, backed by leading philanthropic and academic partners. CONTACT: [email protected]. SOURCE Allos Pharma Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

CAMBRIDGE, Mass., Sept. 18, 2023 /PRNewswire/ -- Allos Pharma Inc. ("Allos"), a biopharmaceutical company specializing in the development of treatments for rare neurological disorders, announced today that it has expanded its intellectual property rights with a new patent family. A new patent family was filed to the United States Patent Trademark & Office earlier in 2023. This new patent family comes in addition to an existing granted patent family. Continue Reading Allos also announces today the worldwide exclusive rights to granted patents and clinical programs for arbaclofen in further indications, including Autism Spectrum Disorder. This is in addition to the current Fragile X Syndrome development program, for which there are no approved medications, and where arbaclofen has Orphan Drug Designation. Expanded arbaclofen rights with a newly filed patent family and with exclusive rights in all indications, incl. Autism. Tweet this "This license builds upon the progress in our Fragile X Syndrome program, where we have achieved alignment with the fragile X community and the FDA on the design of the confirmatory Phase 3 clinical trial. We are excited to have the rights to develop arbaclofen in additional indications as clear efficacy signals were observed in recent trials in individuals with idiopathic Autism Spectrum Disorder." said Dr. Randall Carpenter, MD, co-founder of Allos Pharma Inc. In May 2023, the results of two autism studies, in Canada (n=90) and EU (n=122), were presented at the annual meeting of the International Society for Autism Research. The two studies had nearly identical protocols and endpoints. Improvement was seen in both studies on the primary endpoint (Socialization Domain, Vineland-3) but did not reach statistical significance. Nominally significant improvements were seen in one or both studies on other measures of social skill (Social Responsiveness Scale, Aberrant Behavior Checklist-Social Withdrawal, Autism Impact Measure). A subject-level meta-analysis is planned. "We remain committed to providing new treatment options for patients and families affected by Fragile X Syndrome and Autism Spectrum Disorder, and we believe that arbaclofen has the potential to make a significant, positive impact on their lives." Dr. Randall Carpenter, MD, co-founder of Allos Pharma Inc About Allos Pharma Inc. Allos Pharma Inc. is a biopharmaceutical company dedicated to the development of treatments for rare neurological disorders. Allos' investigational drug, arbaclofen, is currently in development for the treatment of Fragile X Syndrome and Autism Spectrum Disorder. For more information, please visit Allos Pharma Inc.'s website at SOURCE Allos Pharma Inc.

CAMBRIDGE, Mass., May 23, 2023 /PRNewswire/ -- Allos Pharma Inc. ("Allos"), a biopharmaceutical company specializing in the development of treatments for rare neurological disorders, announced today that it has held a meeting with the U.S. Food and Drug Administration (FDA) to optimize the design of their Phase 3 trial. This trial is designed to support a New Drug Application (NDA) to address Fragile X Syndrome, for which there is no approved medicine today. Allos' investigational drug, arbaclofen, targets the molecular pathophysiology of Fragile X Syndrome and has demonstrated considerable efficacy in an FDA-compliant Phase 3 trial. The robust efficacy observed in children provides clarity on effective dosage, inclusion criteria, trial duration, safety, and endpoints, increasing confidence that the pivotal Phase 3 trial will yield positive results. During the FDA meeting, Allos received invaluable and constructive feedback that is being leveraged with input from the Clinical Trial Committee of the National Fragile X Foundation (NFXF) and will be incorporated into the design of the Phase 3 trial. Hilary Rosselot, Executive Director of the National Fragile X Foundation (NFXF) comments that "the Allos team has made significant progress in developing arbaclofen. The expert clinician members of our Clinical Trial Committee have provided input and guidance to optimize the design of the clinical trial and enthusiastically support performing this study. NFXF looks forward to working in partnership with Allos to better the lives of individuals with Fragile X Syndrome and their families." "We support the dedicated work of Drs. Bear and Carpenter and the rest of the Allos team to move ahead with arbaclofen to treat core symptoms in children with Fragile X. Many, many families are waiting eagerly to have access to arbaclofen again!" says Katie Clapp, President and co-founder of FRAXA Research Foundation. "We are extremely pleased with the outcome of the meeting with the FDA and grateful for their engagement, guidance and support as we advance our Phase 3 trial for Fragile X Syndrome," said Dr. Randall Carpenter, MD, co-founder of Allos Pharma Inc. "We remain committed to providing a new treatment option for patients and families affected by this debilitating disorder, and believe that arbaclofen has the potential to make a significant, positive impact on their lives." Additional data analyses suggested by the FDA bolster confidence that the statistically significant improvements observed in the Phase 3 trial are clinically meaningful. Specifically, anchor-based, within-patient responder analyses were performed on data collected in the Phase 3 studies previously conducted by Seaside Therapeutics. Significantly more children treated with arbaclofen had a clinically meaningful magnitude of improvement (P=0.015) than the placebo control group. These results were presented at 18th NFXF International Fragile X Conference July 14-17th 2022 (provide hotlink to the poster on our website). The robust dose-response observed on the clinical outcome assessments in this trial further support the likelihood that efficacy will be replicated in the confirmatory trial. About Allos Pharma Inc. Allos Pharma Inc. is a biopharmaceutical company dedicated to the development of treatments for rare neurological disorders. Allos' investigational drug, arbaclofen, is currently in Phase 3 clinical development for the treatment of Fragile X Syndrome. For more information, please visit Allos Pharma Inc.'s website at Contact: [email protected] SOURCE Allos Pharma Inc.