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National Ataxia Foundation

2023 AAC

Breaking Pharma News

Informing the Ataxia community about drug development


May 23, 2023

PTC Therapeutics, Inc. (NASDAQ: PTCT) today reported topline results from the MOVE-FA trial of vatiquinone in patients with Friedreich ataxia. The study did not meet its primary endpoint of statistically significant change in mFARS score at 72 weeks in the primary analysis population. However, vatiquinone treatment did demonstrate significant benefit on key disease subscales and secondary endpoints. In addition, in the population of subjects that completed the study protocol, significance was reached in the mFARS endpoint and several secondary endpoints.

Read the press release.

May 15, 2023

Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced preliminary top-line data from the 25 mg cohort of its Phase 2, four-week, placebo-controlled, dose exploration trial of CTI-1601 in participants with Friedreich’s ataxia (FA). Participants in the trial’s 25 mg cohort (n=13) were randomized to receive subcutaneous injections of 25 mg CTI-1601 (n=9) or placebo (n=4) daily for 14 days and then every-other-day thereafter until day 28. Data from the cohort indicate CTI-1601 was generally well tolerated and showed increases in frataxin (FXN) levels from baseline compared to placebo in all evaluated tissues (skin and buccal cells) at day 14.

Read the press release.

May 10, 2023

In early March 2023, Reata deployed their fully trained and experienced field sales team as well as the infrastructure that enables physicians to initiate start forms for FA patients. They have received approximately 500 patient start forms to date.

Reata has completed the final stages of SKYCLARYS drug product manufacturing and packaging. An NDA supplement was submitted to the FDA to increase the drug substance specification for a process impurity that was observed above the current specification during process validation. The approval of this supplement is required to release SKYCLARYS drug product to Reata’s specialty pharmacy. The FDA is reviewing the supplement as a Prior Approval Supplement under Priority Review with an approval target action date in mid-August 2023. The FDA stated that the review of the supplement was prioritized and that the approval potentially should come earlier than the action date. Reata believes that the information provided in the NDA supplement, which was developed based upon FDA and international harmonized guidance, is sufficient to support the proposed change. Provided that there are no major review issues with the supplement, Reata believes that SKYCLARYS will be available through the specialty pharmacy no later than mid-August 2023.

Read the press release.

May 9, 2023

Design Therapeutics is evaluating its lead GeneTAC™ small molecule, DT-216, in an ongoing Phase 1 MAD clinical trial designed to evaluate the safety, tolerability, pharmacokinetic, biodistribution, and pharmacodynamic effects of three weekly doses of DT-216 in adults with FA. An unanticipated vendor issue related to the study drug vial stopper caused a short delay in product supply, which has been resolved. Design now anticipates presenting initial results from the MAD trial in the third quarter of 2023. The company plans to initiate a Phase 2 trial in the second half of 2023.

Read the press release.

April 25, 2023

Biogen announced their decision to stop the MERA study for Spinocerebellar Ataxia type 3. The decision was made after careful assessment of the nonclinical safety data, clinical pharmacodynamic data, and future development of BIIB132.

Dr. Rosalind Chuang said, “This is the not the outcome we had hoped for and we acknowledge that this news may be disappointing to SCA3 patients and the entire Ataxia community. We greatly appreciate the dedication and support by patients, their families, and patient advocacy. Biogen remains committed to Ataxia research.”

Read the notice.

April 3, 2023 – Leiden, Netherlands

Vico Therapeutics B.V., a clinical-stage genetic medicines company developing therapies for severe neurological diseases, today announced that the first patient has been dosed in a Phase 1/2a clinical study evaluating VO659 for the treatment of Huntington’s disease (HD), spinocerebellar ataxia type 1 (SCA1) and type 3 (SCA3). VO659 is an antisense oligonucleotide (ASO) investigational therapy designed to target the CAG repeat expansion that causes all nine known polyglutamine diseases including HD, SCA1 and SCA3. The Phase 1/2a clinical trial is a multi-center, open-label basket study designed to assess the safety and tolerability of multiple ascending doses of VO659 administered intrathecally in participants with early manifest HD or mild to moderate SCA1 or SCA3. The study is expected to enroll approximately 71 participants at locations in Europe.

For more information, read the press release.

Here are SCAsource articles that might be helpful in understanding the information in the press release above:

April 3, 2023

Seelos Therapeutics, Inc. (Nasdaq: SEEL) (“Seelos”), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, today announced they are temporarily pausing additional enrollment of patients in the SLS-005-302 study in spinocerebellar ataxia type 3 (SCA3). Patients already enrolled will continue in the study and data will continue to be collected in order to make decisions for resuming enrollment in the future. This temporary pause has been implemented as a business decision due to financial considerations, and is not based on any data related to safety or therapeutic effects.

For more information, read the press release.

February 28, 2023

Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” “our,” “us,” or “we”), a biopharmaceutical company focused on developing and commercializing novel therapies for patients with severe diseases, announced that the U.S. Food and Drug Administration (“FDA”) has approved SKYCLARYS™ (omaveloxolone) for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. With this approval, the FDA granted a rare pediatric disease priority review voucher.

For more information, read the press release.

For information about SKYCLARYS, visit their patient website at

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.

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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