Allos Pharma, Inc.

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.

CAMBRIDGE, Mass., July 14, 2022 /PRNewswire/ -- Allos Pharma Inc ("Allos"), a late-stage pharmaceutical company developing therapeutics for neurodevelopmental disorders, announces presentation of "Arbaclofen produces clinically meaningful improvements in individuals with Fragile X" at the 18th NFXF International Fragile X Conference, July 14-17th, 2022. 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. Meaningful change thresholds (MCTs) were applied for improvement on three subscales of the Fragile X-specific aberrant behavior checklist (ABCFX), Irritability, Social Avoidance and Socially Unresponsive/Lethargic to assess efficacy in the Phase 3 trial in children, aged 5-11. Clinically meaningful magnitudes of improvement were observed on all three ABCFX subscales for 45% of individuals receiving the high dose of arbaclofen vs 4% of those treated with placebo (P=0.00003), clearly demonstrating the efficacy of arbaclofen in individuals with Fragile X. "These findings comport remarkably well with experiences reported by clinicians involved in the clinical trials, who reported that ~50% of those receiving arbaclofen showed clear and substantial improvements." Says Dr. Elizabeth Berry-Kravis, MD, Rush University Medical Center. "We are presenting new analyses of the two phase 3 trials for arbaclofen in Fragile X Syndrome. In addition to the efficacy previously reported, statistically significant efficacy was demonstrated using the current FDA standards for within-subject responder analyses. This represents an important milestone in our work with the regulatory agencies to bring arbaclofen to market" says Randy Carpenter, MD, and co-founder of Allos. "This result supports our hypothesis that arbaclofen correction of core pathophysiology in Fragile X Syndrome arising from dysregulation of neuronal protein synthesis and hyperexcitability will confer broad therapeutic benefit. Combining the new results with the existing literature forms a positive picture of the efficacy of arbaclofen." says Professor Mark Bear and co-founder of Allos. About Allos Pharma, Inc. Headquartered in Cambridge, Massachusetts, Allos Pharma is a late-stage pharmaceutical company developing therapeutics for neurodevelopmental disorders. Our mission is 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. Co-founders Mark Bear and Randy Carpenter previously founded Seaside Therapeutics and advanced arbaclofen through the Phase 3 trials in Fragile X Syndrome. This domain knowledge and expertise will be directly leveraged to advance arbaclofen through NDA approval. For more information visit . Contact: [email protected] SOURCE Allos Pharma Inc.