Public Summaries of Past Applications for Advice from the Ataxia Advisory Committee for Therapeutics (Ataxia ACT)
Ataxia ACT plays a vital role in supporting the development of therapies by providing expert guidance on trial readiness for ataxia. While full reports are shared only with the applicants, sharing non-confidential summaries helps foster transparency and progress in ataxia research. Interested parties and potential funders are encouraged to contact the researchers directly to request a full report.
Autumn 2024: Solaxa
SLX-100 & SLX-200 for Symptomatic & Chronic Treatment of Ataxia in Children and Adults
Ataxia ACT was approached to provide a multi-disciplinary expert advice on SLX-100 a phase 3 clinical program, repurposing 4-Aminopyridine for spinocerebellar ataxia (SCA). Ataxia ACT acknowledged the robust pre-clinical and clinical data to support the rational. Repurposing of an already approved drug enables rapid advancement of the clinical program due to the streamlined regulatory path, and the development can be carried out relatively quickly. With potential capacity to alleviate ataxia symptoms across other types of hereditary ataxia, SLX-100 may have broader impact. Major advice by the Ataxia ACT committee was given on the proposed phase 3 clinical trial design. Additional recommendations were given on dosing, safety data, and regulatory path. The committee also suggested obtaining additional input from SCA patient communities.
Applicant: Solaxa