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National Ataxia Foundation

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Ataxia Treatment Pipeline

Tracking progress of the development of Ataxia therapies.

Treatment development is a long process that involves multiple phases of a clinical trial and oversight by the Food and Drug Administration (FDA). NAF is committed to empowering you with up-to-date information about Ataxia treatment development. The pipelines are visual tools that were created to show the progress of all Ataxia therapies that are currently being developed. There is a pipeline for each type of Ataxia that currently has therapies in development. 

Select a type of Ataxia to learn more: 

Ataxia Treatments in the Pipeline

This is a listing of drugs to treat Ataxia that are currently in the development pipeline. 

About the Treatments

If you are interested in participating in a study, contact the sites listed on their clinicaltrials.gov page or reach out to the sponsor company directly. Select the drug or study name for more information:

Sponsor: Biohaven Pharmaceuticals, Inc

Overview:

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:

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

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

Yes

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.

Clinicaltrials.gov Reference https://clinicaltrials.gov/ct2/show/NCT03701399

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.

Clinicaltrials.gov Reference – https://clinicaltrials.gov/ct2/show/NCT03408080

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.

Results/Publications:

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: 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

Clinicaltrials.gov Reference – 
N/A – Not yet available

This information was obtained from vicotx.com

Stemchymal (Allogenic Stem Cells)

Sponsor: Steminent Biotherapeutics

Overview: Stemchymal® is a formulation allogenic of stem cells isolated and cultured with from human adipose tissue.

Ataxia Types Included in Clinical Trials (planned):

SCA2, SCA3

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

Yes

aCurrent Stage of Development: A Phase 2 clinical trial in SCA2 and SCA3 patients has been planned since 2018. The current status of this trial is unknown.

News/Press Releases:

July 18, 2018 – The FDA has accepted Steminent’s Investigation New Drug (IND) application of Stemchymal for the treatment of polyglutamine spinocerebellar ataxia.

Publications

Mesenchymal stem cell transplantation ameliorates motor function deterioration of spinocerebellar ataxia by rescuing cerebellar Purkinje cells.

Steminent has completed a phase I/II clinical trial with Stemchymal® for the treatment of SCA. Evidence of Stemchymal® safety was generated in this trial with no biological-related adverse events observed in the 12-month follow up. Moreover, evidence of potential efficacy was observed in the preliminary Phase I/II data from two efficacy  assessments:  Scale for the Assessment and Rating of Ataxia (SARA) and the Sensory Orientation Test (SOT).  Sixty-six percent of the subjects showed improvement in both the SARA score and the SOT score in the first month following a single infusion of Stemchymal®.  These measured functional improvements were maintained in study subjects for up to 6 months.

In December 2015, Steminent received US FDA Orphan Drug Designation for Stemchymal® in the treatment of PolyQ SCAs. The US FDA’s Orphan Drug Designation program provides orphan status to drugs and biologics intended for both safe and effective treatment of rare indications that affect fewer than 200,000 people in the U.S. The granted designation also allows Steminent to enjoy a 7-year market exclusivity upon approval of Stemchymal® and other development incentives including tax credits for clinical research costs and Prescription Drug User Fee Act (PDUFA) fee exemption.

To further investigate the safety and efficacy of Stemchymal® for the treatment of SCAs, Steminent is currently conducting a randomized, double-blind/placebo-controlled phase II clinical trial (NCT02540655).

Sponsor: Changhua Christian Hospital

Overview: Spinocerebellar ataxia type 3 (SCA3) is one of autosomal dominant hereditary ataxias. Standing imbalance, unsteady gait, dysmetria, fatigue, and depression would occur gradually. There are no effective treatment or palliative methods for patients in the present days. However, low-dose growth hormone, or its downstream product, insulin-like growth factor I (IGF-1), may deter the progress of SCA3 in transgenic mice. The main bioactive constituent among the Chinese medicine WT possesses neuroprotective function against glutamate-induced toxicity, which is one major pathology of SCA3. It promotes neurogenesis, and increases the protein expression of IGF-1 in ischemic brains of rats. Thus, we designed a randomized, double-blind trial for patients with SCA3, if WT is a possible neuroprotective medicine.

Ataxia Types Included in Clinical Trials (planned): SCA3

Development Stage:
Phase 2

Clinicaltrials.gov Reference – 
https://clinicaltrials.gov/ct2/show/NCT05038306

 

This information was obtained from clinicaltrials.gov

Sponsor: Biogen

Overview: The primary objective of this study is to evaluate the safety and tolerability of multiple ascending doses of BIIB132 administered via intrathecal (IT) injection to participants with spinocerebellar ataxia type 3 (SCA3). The secondary objective of this study is to characterize the multiple-dose pharmacokinetics (PK) of BIIB132 administered via IT injection to participants with SCA3.

Ataxia Types Included in Clinical Trials (planned): SCA3

Development Stage:
Phase 1

Clinicaltrials.gov Reference – 
https://clinicaltrials.gov/ct2/show/NCT05160558

This information was obtained from clinicaltrials.gov

Sponsor: Seelos Therapeutics

Overview: Trehalose is a type of sugar molecule that has been shown to activate autophagy, a cellular process that help clear material from cells. Dysregulation of autophagy and accumulation of toxic protein aggregates is characteristic of many different types of ataxias.  Trehalose is hypothesized to help clear aggregates from the brain and thereby improve neuronal health and survival.

Ataxia Types Included in Clinical Trials (planned):

SCA3

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

Yes

Current Stage of Development: A Phase 2b/3 clinical trial in SCA3 patients began recruiting in 2022.  

Clinicaltrials.gov Reference – 
https://clinicaltrials.gov/ct2/show/NCT05490563

News/Press Releases:

November 8, 2021 – The FDA has accepted Seelos’ Investigation New Drug (IND) application to study SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) for the treatment of spinocerebellar ataxia (SCA). The FDA has also granted the program Fast Track designation in the U.S. for SCA, and SLS-005 has previously received Orphan Drug designation for spinocerebellar ataxia type 3 (SCA3) from the FDA and from the European Medicines Agency in the EU. Global Phase IIb/III Study for SLS-005 to be Initiated in Early 2022

February 19, 2019 – Bioblast Pharma Ltd. Announced the sale of its trehalose clinical development programs to Seelos Therapeutics, Inc.

January 19, 2017 – Bioblast Pharma Ltd.ORPN announced positive results from a phase IIa study of trehalose for the treatment of patients with spinocerebellar ataxia type 3 (SCA3). Results from the open label Phase 2a study showed that the 14 patients treated with trehalose exhibited a stabilization in their SARA scores for up to 12 months. In comparison, natural history data suggests that individuals with SCA3 would be expected to show a measurable increase on SARA within a 12-month period, which is indicative of disease progression and worsening of symptoms. 

Publications

Trehalose in Machado-Joseph Disease: Safety, Tolerability, and Efficacy. Results from the open label Phase 2a study showed that the 14 patients treated with trehalose exhibited a stabilization in their SARA scores for up to 12 months. In comparison, natural history data suggests that individuals with SCA3 would be expected to show a measurable increase on SARA within a 12-month period, which is indicative of disease progression and worsening of symptoms. 

Trehalose alleviates the phenotype of Machado–Joseph disease mouse models

Sponsor: National University of Malaysia

Overview: This study evaluates the effectiveness of oral trehalose in alleviating the neuropathological and motor behaviour deficits among patients with SCA3. A total of 40 participants with SCA3 will be recruited, with 20 participants to be administered with trehalose while another 20 participants to be administered with a maltose placebo.

Ataxia Types Included in Clinical Trials (planned): SCA3

Development Stage:
Phase 2

Clinicaltrials.gov Reference – 
https://clinicaltrials.gov/ct2/show/NCT04399265

This information was obtained from clinicaltrials.gov

Please note: Some of the studies listed did not have more information available at this time.

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

Please note that a drug’s status is subject to change. Go to clinicaltrials.gov for the most current information on a therapy. 

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