Allos Pharma Advances Arbaclofen Program for Autism and Fragile X

Allos Pharma Advances Arbaclofen Program for Autism and Fragile X

By, Admin 22 August 2025

A Major Step Forward in Neurodevelopmental Disorders

Allos Pharma Inc. has announced several key milestones that strengthen its mission to deliver effective treatments for Autism Spectrum Disorder (ASD), Fragile X Syndrome (FXS), and related conditions.

The centerpiece of this progress is global rights to arbaclofen, a GABA-B receptor agonist with one of the most promising data sets in ASD pharmacotherapy to date.

Securing Global Rights to Arbaclofen

Allos now holds worldwide rights to arbaclofen. This includes:

  • Clinical data from prior trials.
  • Regulatory assets enabling efficient advancement.
  • Manufacturing capabilities to support future development.

Dr. Randall Carpenter, Co-founder and CMO of Allos, explained the significance:

“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.”

Clinical Results Show Promise in ASD

Two recent placebo-controlled trials in Canada and the EU, sponsored by:

  • The POND Network
  • The EU AIMS-2-Trials Consortium

…replicated earlier positive findings from the Seaside Therapeutics Phase II trial.

Key outcomes:

  • Clinically meaningful improvements in social function.
  • Significant progress in communication.
  • Reduction in restricted and repetitive behaviors.

These results mark the strongest efficacy signals seen so far in ASD pharmacotherapy.

Insights from 16p11.2 Deletion Syndrome Study

The L16hthouse study, led by Clinical Research Associated LLC, was designed following strong preclinical evidence in 16p11.2 deletion syndrome— the most common genetic copy number variant linked to ASD.

Takeaways:

  • Data informs selection of primary endpoints.
  • Provides guidance for patient stratification in future precision medicine trials.

Grant from the Simons Foundation

Allos has also secured non-dilutive funding from the Simons Foundation. This grant will:

  • Support clinical development of arbaclofen.
  • Enable biomarker-driven analyses in ASD populations.

About Arbaclofen

Arbaclofen is a selective GABA-B receptor agonist that restores excitatory/inhibitory balance and corrects metabolic dysregulation in the brain.

Highlights:

  • Evaluated in over 700 children, adolescents, and adults with ASD and FXS.
  • Demonstrated exceptional safety and tolerability, including in long-term open-label studies.
  • Targets core domains of ASD, FXS, and 16p11.2 deletion syndrome.

About Allos Pharma

Allos Pharma is a biopharmaceutical company dedicated to developing therapies for autism and genetically defined neurodevelopmental disorders. Its lead candidate, arbaclofen, is positioned as a potential first-in-class treatment targeting both core symptoms and associated features of these conditions.

Backed by leading academic and philanthropic partners, Allos is committed to rigorous science, patient-centered clinical development, and long-term impact in neurodevelopmental health.

Final Word

With global rights secured, new clinical validation, and foundation funding, Allos Pharma is well-positioned to advance arbaclofen through the next stages of development. For families affected by ASD and Fragile X, these milestones bring renewed hope for effective therapies.

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