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National Ataxia Foundation


Breaking Pharma News

Informing the Ataxia community about drug development


October 27, 2022

LEXEO Therapeutics (LEXEO), a clinical-stage biotech company advancing a pipeline of adeno-associated virus (AAV)-based gene therapy candidates for cardiovascular and central nervous system (CNS) diseases, today announced it will be presenting clinical and preclinical data at three upcoming scientific meetings. LEXEO will present at the inaugural International Congress for Ataxia Research (ICAR) held live in Dallas, Texas from November 1-4, 2022, the American Heart Association Scientific Sessions held live in Chicago, Illinois and virtually from November 5-7, 2022, and the Clinical Trials for Alzheimer’s Disease (CTAD) conference held live in San Francisco, California from November 29-December 2, 2022. 

For more information, read the press release.

For information about ICAR, visit

Seelos Therapeutics Doses First Patient in a Registrational Phase II/III study of SLS-005 in Spinocerebellar Ataxia

October 25, 2022

Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, today announced dosing of the first patient in a registrational phase II/III study of SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) for the treatment of spinocerebellar ataxia focusing on participants with type3 (SCA3, also known as Machado-Joseph disease) and also announced that it will participate in the International Congress for Ataxia Research (ICAR) in Dallas, Texas November 1st – 4th, 2022.

For more information, read the press release.

If you have SCA3 and are interested in participating in this trial, read the recruitment information.

Reata Announces FDA Filing Acceptance and Priority Review Designation for the NDA for Omaveloxolone

May 26, 2022

Reata Pharmaceuticals  announced today the FDA have granted their Omaveloxolone application ‘Priority Review’ designation. This means the FDA will review their application for regulatory approval within 8 months rather than the usual 12 months. Omaveloxolone is a drug being tested for the treatment of FA.

For more information, read the press release.

Update on Biohaven Clinical Trial

May 23, 2022

Biohaven Pharmaceuticals reported top-line results of its phase 3 clinical trial of troriluzole, an investigational drug for patients with SCA.  While we share in your disappointment that the drug did not meet its primary endpoint in the overall SCA population, there is much more to this story than just that headline.  Further analysis by SCA type suggests a positive treatment effect in patients with SCA3, the most common form of SCA.  There was also a reduction in the relative risk of falls in troriluzole-treated patients with all types of SCA included in the trial versus the placebo group.

We are pleased that Biohaven will be presenting this data to the FDA as soon as possible.  “The f-SARA change in the SCA3 troriluzole treated group is compelling,” stated Dr. Susan Perlman, Director of Ataxia Clinic and Neurogenetics Clinical Trials at the David Geffen School of Medicine at UCLA.  “Given the excellent safety profile of troriluzole, and the fact that there are no approved treatment options for patients with this devastating neurodegenerative disorder, I am thrilled for my patients that Biohaven is planning to engage with the FDA to potentially move this program forward.”

For more information, read the press release.

Pfizer to Buy Biohaven Pharmaceuticals

May 10, 2022

Biohaven Pharmaceuticals, the company running a phase three clinical trial of troriluzole for the treatment of Spinocerebellar Ataxia, has been purchased by Pfizer. Pfizer is buying Biohaven’s big migraine drug, NURTEC. Then a new publicly traded company will be created that will retain Biohaven’s pipeline for neurological and other disorders. NAF has confirmed with the company that the troriluzole trial remains on track and will report topline results in mid-2022.

For more information, read the press release.

Reata Pharmaceuticals Provides an Update on Omaveloxolone for the Treatment of Friedreich's Ataxia

May 10, 2022

Reata Pharmaceuticals recently announced that the FDA has granted Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation to omaveloxolone for the treatment of Friedreich’s Ataxia. 

For more information, read the press release.

Seelos Therapeutics Announces FDA Acceptance of Investigational New Drug Application and Grant of Fast Track Designation for SLS-005 (IV Trehalose) for the Treatment of Spinocerebellar Ataxia

November 8, 2021

Seelos Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, announced today that 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.

For more information, read the press release.

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