Biopharmaceutical public benefit corporation Solaxa Inc. has announced plans to advance its investigational therapy SLX-100 into a Phase 3 registrational clinical trial for spinocerebellar ataxia type 27B (SCA27B), a rare hereditary neurological disorder currently lacking FDA-approved treatment options.
The pivotal study will be partially funded through a $7.3 million grant awarded to Dr. Susan Pearlman, MD, at the University of California, Los Angeles (UCLA) by the Congressionally Directed Medical Research Program. Solaxa will maintain its position as the regulatory sponsor for the trial while establishing partnerships with leading ataxia centers of excellence across the country.
"We are thrilled to move one step closer to impacting the lives of people living with SCA27B," said Christian Walker, CEO and Founder of Solaxa, highlighting the significance of this advancement for patients suffering from this rare condition.
Understanding Spinocerebellar Ataxia Type 27B
Spinocerebellar ataxia type 27B belongs to a group of rare hereditary ataxias characterized by progressive degeneration of the cerebellum and spinal cord. This neurodegenerative disorder typically manifests with symptoms including uncoordinated movement, impaired balance, slurred speech, and difficulty with fine motor skills.
SCA27B specifically results from genetic mutations affecting cerebellar function and represents one of the rarer subtypes within the spinocerebellar ataxia spectrum. The progressive nature of the disease significantly impacts patients' quality of life and functional independence over time.
Clinical Trial Design and Collaboration
While specific details regarding the trial design have not yet been disclosed, the company has indicated that the study will be "pivotal-powered," suggesting it is designed with sufficient statistical power to potentially support regulatory approval if successful.
The collaboration between Solaxa and academic medical centers represents a significant public-private partnership in rare disease research. Dr. Pearlman's involvement as the grant recipient highlights the academic expertise supporting this clinical development program.
Additional clinical trial sites beyond UCLA are expected to be announced later this year, expanding the geographical reach of the study and potentially increasing access for patients with this rare condition.
Addressing an Unmet Medical Need
The development of SLX-100 addresses a critical unmet need in the treatment landscape for SCA27B. Currently, management of spinocerebellar ataxias primarily focuses on symptomatic treatment and supportive care rather than disease modification.
If successful, SLX-100 could become the first FDA-approved therapy specifically targeting SCA27B, potentially changing the treatment paradigm for affected patients. The public benefit corporation status of Solaxa aligns with its mission to address rare neurological conditions that might otherwise receive limited commercial attention.
Looking Forward
The advancement of SLX-100 to a Phase 3 trial represents a significant milestone in the development pathway for this investigational therapy. For patients with SCA27B and their families, this progress offers hope for a potential treatment option for a condition that currently has limited therapeutic interventions.
Interested parties seeking more information about the upcoming clinical trial can contact Solaxa directly at their Bethesda, Maryland headquarters or via email at info@solaxa.com.
