*NEW* Ataxia treatment pipeline launched! It is a visual look at drug development progress. LEARN MORE!

National Ataxia Foundation

Accelerate!

SCA17 Treatment Pipeline

Tracking progress of the development of SCA17 therapies.

Pipeline for SCA17

This is a visual tool created to show the progress of all SCA17 therapies that are currently being developed. Along the vertical axis, treatments are grouped by their mechanism, or how the drug works. The horizontal axis shows the stage of research where the drug is in development. Phases 1 through 3 involve clinical trials with human participants. Visit our Clinical Trials page to learn more about each phase. If a clinical trial provides enough evidence to prove that the drug is safe and effective, the sponsor can file a New Drug Application (NDA) with the FDA. After an NDA is approved, the drug can become available to individuals through their healthcare provider.

Graph depicting the phase of drug development for various drugs to treat SCA17

Please note that a drug’s status is subject to change. Go to clinicaltrials.gov for the most current information on a therapy. Detailed information is available for some of the drugs listed. Select the drug name from the list below for more information:

Sponsor: Novartis Therapeutics

Overview: CAD-1883 is a small molecule and modulator of calcium-activated potassium ion channels. It is hypothesized to improve symptoms in patients with spinocerebellar ataxia through regulating neuronal firing of cells in the cerebellum.

Ataxia Types Included in Clinical Trials (planned):

SCA1, SCA2, SCA3, SCA6, SCA7, SCA8, SCA10, SCA17, ARCA1

Potential Utility in Other Ataxias (i.e. sporadic, unknown cause, other SCAs, etc,)

Yes

Current Stage of Development: A Phase 2 clinical trial sponsored by Cadent Therapeutics was planned to begin in 2022 and would have included participants with SCA1, SCA2, SCA3, SCA6, SCA7, SCA8, SCA10, SCA17, and ARCA1. However, the clinical trial was withdrawn in January 2021 following the acquisition of Cadent Therapeutics by Novartis Therapeutics. According to clinicaltrials.gov, “as part of a pipeline assessment, the Synchrony-1 trial will not proceed as initially scheduled.  

Clinicaltrials.gov Reference https://clinicaltrials.gov/ct2/show/NCT04301284

A Phase 2 clinical trial sponsored by Cadent Therapeutics of CAD-1883 in individuals with essential tremor was completed in 2019. No serious adverse events were observed in treated patients in this study.

Clinicaltrials.gov Reference https://www.clinicaltrials.gov/ct2/show/results/NCT03688685

News/Press Releases:

December 17, 2020 – Novartis announced that the company has entered an agreement to acquire Cadent Therapeutics. The acquisition included the CAD-1883 clinical stage program for movement disorders.

June 4, 2019 – FDA granted Orphan Drug designation to CAD-1883 (Cadent Therapeutics), an investigational treatment for spinocerebellar ataxia.

 

Studies that are listed as “recruiting” are looking for participants. To learn more about participating in research visit our Clinical Trials page.

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