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Breaking Pharma News

Informing the Ataxia community about drug development

RECENT NEWS

February 29, 2024

Biohaven Ltd. announced recent business developments in their latest press release. Biohaven has continued to have constructive dialogue with the FDA regarding its SCA development program and potential future data analyses to address regulatory concerns in the previously issued refuse-to-file decision on its NDA application for SCA3. Biohaven will provide further updates on the SCA development program as warranted by any continued positive progress from the outcome of future regulatory interactions on this topic.

Read the press release.

February 26, 2024

Voyager Therapeutics, Inc. announced that the joint steering committee with its collaborator Neurocrine Biosciences has selected a lead development candidate in the Friedreich’s ataxia (FA) program. The candidate combines a frataxin (FXN) gene replacement payload with an intravenously administered, blood-brain barrier penetrant, novel capsid derived from Voyager’s TRACER™ capsid discovery platform. The companies expect the program to advance into first-in-human clinical trials in 2025.

Read the press release.

February 12, 2024

Biogen Inc. announced the European Commission (EC) has authorized SKYCLARYS® (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older. SKYCLARYS is the first treatment approved within the European Union for this rare, genetic, progressive neurodegenerative disease.

Read the press release.

Related News: FDA Approves First Treatment for Friedreich’s Ataxia

February 12, 2024

Larimar Therapeutics, Inc. today announced positive top-line data and successful completion of its four-week, placebo-controlled Phase 2 dose exploration study of nomlabofusp (CTI-1601) in participants with Friedreich’s ataxia (FA). Nomlabofusp was generally well tolerated and demonstrated dose dependent increases in frataxin (FXN) levels in all evaluated tissues (skin and buccal cells) after daily dosing of 14 days followed by every other day dosing until day 28 in the 25 mg and 50 mg cohorts. Participants in the 25 mg (n=13) and 50 mg (n=15) cohorts were randomized 2:1 to receive subcutaneous injections of nomlabofusp or placebo.

Read the press release.

January 5, 2023

  • Funding supports ongoing Phase 1/2a clinical trial evaluating VO659 in patients with spinocerebellar ataxia type 3 and type 1 and Huntington’s disease

Vico Therapeutics B.V., a clinical-stage genetic medicines company developing therapies for severe neurological diseases, today announced a $60 million Series B financing. “This financing positions us to accelerate our efforts to advance our lead program VO659, currently in a Phase 1/2a clinical study of patients with spinocerebellar ataxia types 3 and 1 and Huntington’s disease,” said Micah Mackison, chief executive officer at Vico. 

The financing will support overall company operations including advancement of the ongoing Phase 1/2a multi-center, open-label basket trial of VO659 in patients living with spinocerebellar ataxia type 3 (SCA3) and type 1 (SCA1) and Huntington’s disease (HD). The trial is designed to assess the safety and tolerability of multiple ascending doses of VO659 administered intrathecally in participants with mild to moderate SCA3, SCA1 and early manifest HD, with the first patient dosed in April 2023. The financing will also help support discovery and research efforts to further expand the company’s pipeline in other genetic neurological diseases.

Read the press release.

November 30, 2023

Variantyx, a leader in genomic precision medicine, today announced that the set of short tandem repeat (STR) expansions detected by its Genomic Unity® line of whole genome-based tests has been expanded. With these additions, Variantyx becomes one of the first laboratories to offer testing for two recently characterized ataxia variants: the biallelic AAGGG expansion in RFC1 that causes cerebellar ataxia, neuropathy, vestibular areflexia syndrome (CANVAS) and the monoallelic, deep intronic GAA expansion in FGF14 that causes spinocerebellar ataxia 27B (SCA27B).

Read the press release.

August 1, 2023

PureTech Health announced that the company has been awarded up to $11.4 million from the U.S. Department of Defense (DoD) to advance its therapeutic candidate, LYT-300 (oral allopregnanolone), for the treatment of Fragile X-associated Tremor Ataxia Syndrome (FXTAS). The funds will support a Phase 2 trial of LYT-300 in collaboration with the University of California, Davis (UC Davis).

Read the press release.

Here is an SCAsource article that might be helpful in understanding the information in the press release above:

July 28, 2023

Biogen Inc. and Reata Pharmaceuticals, Inc. announced the companies have entered into a definitive agreement under which Biogen has agreed to acquire Reata for $172.50 per share in cash, reflecting an enterprise value of approximately $7.3 billion.

Reata has made significant advancements developing therapeutics that regulate cellular metabolism and inflammation in serious neurologic diseases. Reata’s FDA-approved SKYCLARYS® (omaveloxolone) is the first and only approved treatment for Friedreich’s ataxia (FA) in the United States, with a commercial launch underway, and European regulatory review ongoing. In addition, Reata is developing a portfolio of innovative products for a range of neurological diseases.

Read the press release.

July 27, 2023

Biohaven Ltd. today announced multiple updates on key programs including a regulatory update on troriluzole. The FDA informed Biohaven that it would not review the recently submitted NDA application for troriluzole given that the study’s primary endpoint was not met and thus, would not permit a substantive review. The communication from the FDA indicated that the Company may request a Type A meeting within 30 days. Biohaven is committed to working closely with the FDA to bring troriluzole to people with SCA3 as quickly as possible given no therapy is currently approved for this ultra-rare genetic disorder and is requesting a Type A meeting to comprehensively address FDA’s concerns cited in the refusal to file letter. Any updates regarding the Type A meeting will be provided subsequent to the upcoming regulatory interaction.

Read the press release.

Comment from NAF:

While NAF is disappointed to learn of the FDA’s decision to not review the new drug application for troriluzole, we are confident that FDA will follow established regulatory processes and precedent in determining next steps for the NDA. We look forward to learning more after the Type A meeting and will keep the Ataxia community informed of updates.

NAF’s Chief Scientific Officer Lauren Moore said, “It can be difficult to remain hopeful following setbacks in the drug development process. Having multiple family members affected by Ataxia, I understand the immense need for effective treatments NOW. While we wait to hear about what the future holds for troriluzole following this decision, I take strength in the momentum created by the unprecedented number of companies investing in Ataxia drug development.”

NAF will continue to represent the voice of the patient throughout the regulatory process and advocate for the needs of the Ataxia community.

July 25, 2023

Larimar Therapeutics announced that the FDA has cleared the Company’s four-week, placebo-controlled, Phase 2 dose exploration trial of CTI-1601 in patients with Friedreich’s ataxia (FA) to proceed to a 50 mg cohort in which participants will be dosed daily for the first 14 days, and then every other day until day 28. In addition, Larimar’s open label extension (OLE) trial was also cleared for initiation by the FDA. Participants in the OLE will receive 25 mg of CTI-1601 daily. CTI-1601 is a novel protein replacement therapy designed to deliver frataxin to the mitochondria of patients with FA who have low levels of frataxin.

Read the press release.

Here is an SCAsource article that might be helpful in understanding the information in the press release above:

June 27, 2023

Reata Pharmaceuticals (RETA) announced that the United States Food and Drug Administration (FDA) has approved the Prior Approval Supplement (PAS) to update the drug substance specification for SKYCLARYS® (omaveloxolone), the first and only FDA approved drug for the treatment of Friedreich’s Ataxia in adults and adolescents aged 16 years and older. With the approval of the PAS, SKYCLARYS is now available to patients with Friedreich’s Ataxia in the United States.

Read the press release.

Here is an SCAsource article that might be helpful in understanding the information in the press release above:

June 13, 2023

LEXEO Therapeutics (LEXEO), a clinical-stage gene therapy company advancing adeno-associated virus (AAV)-based gene therapy candidates for genetically defined cardiovascular diseases and a genetically defined sub-group of Alzheimer’s disease, announced today the completion of the first dose cohort and the dosing of the first patient in the second dose cohort in SUNRISE-FA, a Phase 1/2 clinical trial of LX2006 in patients with Friedreich’s ataxia (FA) cardiomyopathy.

In the first dose cohort, LX2006 has been well tolerated with no unexpected events or toxicities observed. Following the Data Safety Monitoring Board recommendation to proceed, investigators have initiated dosing in the second cohort.

 

Read the press release.

Here is an SCAsource article that might be helpful in understanding the information in the press release above:

May 31, 2023

Biohaven Ltd. (NYSE: BHVN) (“Biohaven”), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies for people with debilitating diseases, including ultra-rare disorders, will provide an overview of clinical progress, regulatory updates, and pipeline developments at an in-person R&D Day today, concurrently with the Yale Ventures’ Innovation Summit 2023 taking place on May 31-June 1. 

The clinical progress, regulatory updates, and pipeline developments included:

Troriluzole in SCA: Submitted New Drug Application (NDA) to the U.S. FDA for troriluzole for the treatment of spinocerebellar ataxia type 3 (SCA3), an ultra-rare, genetically-defined, neurodegenerative disease associated with progressive disability, frequent falls, loss of ambulation, speech and swallowing impairment, and premature death that is the most common SCA genotype worldwide.

  • NDA supported by consistent treatment benefits observed in patients with genotype SCA3 in Study BHV4157-206 across multiple outcome measures including the change from baseline f-SARA at Week 48, CGI-I total score at Week 48, and a robust reduction in fall risk over the study period. A rigorous analysis by genotype was possible as patients were randomized by genotype strata at baseline prior to randomization in the pivotal Phase 3 48-week, double-blind, placebo-controlled phase of Study BHV4157-206.
  • SCA3 represented 41% of study participants, consistent with being the most common subtype. SCA3 affects approximately 10,600 people in North America and in the EU and Japan.
  • NDA further supported by a composite scale development (SCACOMS) and analysis of Study BHV4157-206, and confirmatory evidence of efficacy provided by data from the 3-year, long-term open-label (OLE) extension phase of two studies (BHV4157-206 and BHV4157-201) using a Matching Adjusted Indirect Comparison (MAIC) to an external control group.
  • Biohaven has previously received Fast-Track and Orphan drug designation (ODD) from the FDA, and ODD from the European Medicines Agency, for troriluzole in SCA.

Read the press release.

Here is an SCAsource article that might be helpful in understanding the information in the press release above:

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 www.SKYCLARYS.com.

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 www.ataxiacongress.org.

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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