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SCA1 Treatment Pipeline

Tracking progress of the development of SCA1 therapies.

Pipeline for SCA1

This is a visual tool created to show the progress of all SCA1 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 SCA1

Please note that a drug’s status is subject to change. Go to 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: Biohaven Pharmaceuticals, Inc


Troriluzole is a third-generation prodrug of riluzole, a small molecule hypothesized to decrease over-excitability of neurons in the brain by reducing levels of free glutamate. Glutamate levels have been shown to be abnormal in the brains of patients with different types of ataxias. In addition, ataxia model systems have shown over-excitability of Purkinje cells, a type of cerebellar neuron necessary for coordinated movement. Therefore, troriluzole is hypothesized to improve coordinated movement in ataxia patients through regulating glutamate levels in the brain and thereby improving neuronal function.

Ataxia Types Included in Clinical Trials to Date:


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


Current Stage of Development:

A Phase 3 clinical trial sponsored by Biohaven is ongoing and no longer recruiting. The trial has enrolled over 200 adults affected with SCA1, SCA2, SCA3, SCA6, SCA7, SCA8, and SCA10. Topline results of this trial were reported in the first half of 2022. Read more in their press release. Reference

An investigator-initiated Phase 3 open-label study of trigriluzole (BHV-4157) is also ongoing in SCA1, SCA2, SCA3, and SCA6 patients who were already taking Riluzole for more than 8 weeks, and in non-genetic pure cerebellar ataxia and MSA-C patients. Reference –

News/Press Releases:

February 25, 2022 – Biohaven announced that the company expects to report topline of troriluzole in Spinocerebellar Ataxia (“SCA”) in the first half of 2022.

March 15, 2019 – Biohaven announced that the company has initiated patient enrollment in a Phase 3 clinical trial of its third generation pro-drug troriluzole for the treatment of SCA.

October 2, 2017 – Biohaven reported negative topline results from SCA phase 2/3 trial. Patients that received Trigriluzole did not exhibit greater improvement in SARA scores than patients that received placebo (non-active drug). However, patients that received placebo exhibited a much higher “placebo response rate” than expected compared to prior randomized controlled trials in SCA meaning many patients who received a non-active drug showed an unanticipated improvement in symptoms likely due to psychological rather than physical affects.

May 30, 2017 – Biohaven announced that the company has completed enrollment in Spinocerebellar Ataxia Clinical Trial with trigriluzole.

May 15, 2017 – The Food and Drug Administration has granted Fast Track designation to trigriluzole (BHV-4157) for the potential treatment of SCA.


March 2022 – Safety and efficacy of riluzole in spinocerebellar ataxia type 2 in France (ATRIL): a multicentre, randomised, double-blind, placebo-controlled trial. In a Phase 3 trial of SCA2 only patients, riluzole did not lead to any clinical or radiological improvements in patients. Riluzole is the metabolite of troriluzole, but these are distinct candidate drugs. Therefore, lack of efficacy of riluzole doesn’t necessarily predict that troriluzole will also not be effective for treatment of SCA2 or other ataxia types.

October 2015 – Riluzole in patients with hereditary cerebellar ataxia: a randomised, double-blind, placebo-controlled trial. Results of a phase 2, randomized, double-blind clinical trial (NCT01104649) of 55 patients with either spinocerebellar ataxia showed that about half of the patients that were treated with riluzole for 1 year exhibited a decrease in SARA score, while only 11% of patients treated with placebo (non-active drug) exhibited a decrease in their SARA score. SARA SARA is a clinical test that assesses the severity of a person’s ataxia symptoms, where higher scores indicate worsening of symptoms. No severe adverse events were recorded.

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):


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


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, “as part of a pipeline assessment, the Synchrony-1 trial will not proceed as initially scheduled. Reference

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. Reference

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.


Sponsor: Sclnow Biotechnology Co., Ltd.

Overview: The purpose of this study is verify the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) therapy for patients with Spinocerebellar Ataxia, and in addition, explore the possible mechanisms of UC-MSC therapy in Spinocerebellar Ataxia.

Ataxia Types Included in Clinical Trials (planned):


Development Stage:
Phase 2 Reference


This information was obtained from

Sponsor: Vico Therapeutics

Overview: The U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for VO659, VICO’s investigational antisense oligonucleotide (AON) therapy for the treatment of spinocerebellar ataxia (SCA).

Ataxia Types Included in Clinical Trials (planned): SCA1, SCA3

Development Stage:
IND Granted Reference – 
N/A – Not yet available

This information was obtained from

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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