Arbaclofen Returns After 7 Years!

May 2013 left many in the Fragile X community devastated when financial pressures led Seaside Therapeutics to stop developing an experimental drug, arbaclofen, for Fragile X syndrome (FXS).

Today arbaclofen rises again! Allos Pharma Inc, a late-stage pharmaceutical company developing therapeutics for neurodevelopmental disorders, is launching a new development program for arbaclofen in Fragile X syndrome. Allos Pharma was recently cofounded by Mark Bear, PhD, MIT Picower Professor of Neuroscience, and Randy Carpenter, MD. Dr. Bear has been a FRAXA Investigator since 1999 and is a member of FRAXA’s Scientific Advisory Board.

Many people in the Fragile X community felt that arbaclofen helped their family members, and a small group created a website, STX209Stories, which featured nearly 50 stories from families who had experienced positive results from the experimental drug. Parents printed out the stories and delivered them to Members of Congress with the hope of continued access the medication. One of them was Holly Usrey-Roos, whose experience was featured in the New York Times’ An Experimental Drug’s Bitter End. We spoke with Holly today:

I never expected to get a phone call telling me that arbaclofen, the drug that changed my son’s life in the best ways, would be back. Losing arbaclofen seven years ago was devastating. With it, we were able to do things just like everyone else: we could go to the public pool, we could go to the movies, and best of all, it led to my son talking. We fought to keep it but we couldn’t. We were forced to move on.

I’m sure I am not alone in believing everyone had moved on and we would never hear about arbaclofen again. But we were wrong! Dr. Mark Bear never gave up on it. He never gave up on our children, on finding a way to make arbaclofen available again.

I’ve cried a lot during this pandemic, but these are the first tears of pure happiness. This news not only brings back hope for my son and the Fragile X community but reminds me that the amazing people who are dedicated to Fragile X will never give up on us.

Holly Usrey-Roos

Press Release by Allos Pharma

BOSTON, Nov 19, 2020 /PRNewswire/ — Allos Pharma Inc, a late-stage pharmaceutical company developing therapeutics for neurodevelopmental disorders, 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 hyperexcitabilitysays 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.says Randy Carpenter, MD, and co-founder of Allos Pharma.

“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.” says Linda Sorensen, Executive Director of the National Fragile X Foundation (NFXF).

“We are delighted that Drs. Bear and Carpenter and the rest of the Allos team are moving ahead with arbaclofen to treat core symptoms in children with Fragile X. Many, many families have been waiting eagerly for this news!” says Katie Clapp, President and co-founder of FRAXA Research Foundation.

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 www.allospharma.com or email contact@allospharma.com.

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Massachusetts, USA (Nov 18, 2020) — Allos Pharma Inc, a late-stage pharmaceutical company developing therapeutics for neurodevelopmental disorders, 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 hyperexcitabilitysays 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.says Randy Carpenter, MD, and co-founder of Allos Pharma.

“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.” says Linda Sorensen, Executive Director of the National Fragile X Foundation (NFXF).

“We are delighted that Drs. Bear and Carpenter and the rest of the Allos team are moving ahead with arbaclofen to treat core symptoms in children with Fragile X. Many, many families have been waiting eagerly for this news!” says Katie Clapp, President and co-founder of FRAXA Research Foundation.

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 www.allospharma.com or email contact@allospharma.com.

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