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Scientific News

FARA funds research progress

In this section, you will find the most recent FA research publications, many of which are funded by FARA, as well as information on upcoming conferences and symposiums. You can search for articles by date using the archive box in the right hand column. To locate FARA Funded or Supported Research, click the hyperlink in the right hand column. You may also search for specific content using key words or phrases in the search button at the top right of your screen. Please be sure to visit other key research sections of our website for information on FARA’s Grant Program and the Treatment Pipeline.

Study That Measures Efficacy of Experimental Friedreich’s Ataxia Treatment posted in medRXiv

Natural History study, funded by FARA, a Friedreich’s Ataxia patient advocacy organization, provides data to help evaluate treatment effect of Omaveloxolone

PRESS RELEASE - Downingtown, PA – August 16, 2022 – The Friedreich’s Ataxia Research Alliance (FARA), a leading patient advocacy organization dedicated to developing treatments and cures for Friedreich’s Ataxia (FA), announced today that data from a FARA-sponsored natural history study – the FA Clinical Outcomes Measures Study (FACOMS) -- has helped provide evidence and understanding of the treatment effect of Omaveloxolone, a potential treatment currently under review for approval by the Food and Drug Administration (FDA).

FACOMS is a multicenter natural history study that aims to understand the clinical symptoms, progression, and long-term outcomes for individuals with FA. Initiated by a core group of clinical researchers and supported by FARA, the FACOMS natural history study is the largest, most diverse source of longitudinal clinical data in FA. FACOMS has facilitated the development of clinical outcome assessments being used in clinical trials, such as the modified Friedreich’s Ataxia Ratings Scale (mFARS), which assesses neurological function in FA patients.

The newly published study using FACOMS data is entitled Direct utility of natural history data in analysis of clinical trials: Propensity match-based analysis of Omaveloxolone in Friedreich ataxia using the FACOMS dataset, and is available via the preprint repository for health sciences- medRXiv The Propensity match-based analysis of Omaveloxolone in FA using the FACOMS dataset compares 136 individuals who received Omaveloxolone (Omav) for FA in an open label extension study for an average of three years to well-matched individuals in FACOMS. The primary endpoint, change from baseline in mFARS score at year three, showed a 55% slowing in disease progression as measured by mFARS. The matched FACOMS cohort progressed by 6.6 mFARS points over three years and individuals in the MOXIe extension study (treated with Omav) progressed only 3.0 points (difference =-3.6 points; p=0.0001).

Jennifer Farmer, Chief Executive Officer of FARA, states, “This publication demonstrates the power of the natural history study in providing critical data to support clinical trials and shaving time off drug development. FARA is proud to have invested in and fostered this research. We are hopeful that this study will provide confirmatory evidence of the treatment effect of Omav observed in the MOXIe Part 2 study and can support an FDA decision to approve Omav for FA. In a progressive neuromuscular disease, we do not have the luxury of time and must operate with efficiency and urgency.”

Ron Bartek, President and Co- Founder of FARA adds, “Behind these data are more than 20 years of dedicated time from FA families, investigators throughout our Collaborative Clinical Research Network in FA, as well as generous contributions from donors to fund the work. The average distance a family travels to participate in a natural history visit is over 500 miles. We are grateful to all FA families for their long-standing commitment to research progress. We are beyond proud and excited to be a part of helping to demonstrate Omav can have a meaningful impact on the disease progression and hope that all FA community members who want access to Omav will have it soon.”


Replication dependent and independent mechanisms of GAA repeat instability

(GAA)n repeat instability arises during both replication-dependent processes, such as cell division and intergenerational transmission, as well as in terminally differentiated somatic tissues. Here, the authors provide a brief historical overview on the discovery of (GAA)n repeat expansions and their association to FRDA, followed by recent advances in the identification of triplex H-DNA formation and replication fork stalling. The main body of this review focuses on the last decade of progress in understanding the mechanism of (GAA)n repeat instability during DNA replication and/or DNA repair. The Authors propose that the discovery of additional mechanisms of (GAA)n repeat instability can be achieved via both comparative approaches to other repeat expansion diseases and genome-wide association studies. Finally, advances towards FRDA prevention or amelioration that specifically target (GAA)n repeat expansions are discussed.

Read the Full article here

An open-label pilot study of recombinant granulocyte-colony stimulating factor in Friedreich's ataxia

This paper describes an open-label, pilot study of recombinant human granulocyte-colony stimulating factor (G-CSF) administration in seven people with FA (EudraCT: 2017-003084-34); each participant receiving a single course of G-CSF (Lenograstim; 1.28 million units per kg per day for 5 days). The primary outcome is peripheral blood mononuclear cell frataxin levels over a 19-day period. The secondary outcomes include safety, haematopoietic stem cell (HSC) mobilisation, antioxidant levels and mitochondrial enzyme activity. The trial meets pre-specified endpoints. The authors show that administration of G-CSF to people with FA is safe. Mobilisation of HSCs in response to G-CSF is comparable to that of healthy individuals. Notably, sustained increases in cellular frataxin concentrations and raised PGC-1α and Nrf2 expression are detected. These findings show potential for G-CSF therapy to have a clinical impact in people with FA.

Read the Full article here

Larimar Therapeutics Provides Updates on CTI-1601 Clinical Program Following a Type C Meeting with the U.S. Food and Drug Administration and Reports Second Quarter 2022 Operating and Financial Results

Larimar Therapeutics Provides Updates on CTI-1601 Clinical Program Following a Type C Meeting with the U.S. Food and Drug Administration and Reports Second Quarter 2022 Operating and Financial Results

  • Larimar plans to submit a complete response to CTI-1601’s clinical hold in the third quarter of 2022
  • In conjunction with the complete response, Larimar is proposing a Phase 2, four-week dose exploration study in Friedreich’s ataxia (FA) patients as CTI-1601’s next clinical trial
  • Cash and marketable debt securities at June 30, 2022 of $54.9 million provides projected cash runway through the third quarter of 2023

Read the Full article here

Reata Pharmaceuticals Announces Three Month Extension of the Review Period for New Drug Application for Omaveloxolone for the Treatment of Friedreich’s Ataxia

Reata Submitted New Data and Analyses to Address FDA Questions During Mid-Cycle Meeting

FDA Extended PDUFA Date to Provide Time for Full Review of New Submissions

PDUFA Date Extended to February 28, 2023

PLANO, Texas--(BUSINESS WIRE)-- Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” “our,” “us,” or “we”), a clinical-stage biopharmaceutical company, announced that on August 8, 2022, after the U.S. financial markets closed, we received a communication from the U.S. Food and Drug Administration (“FDA”) informing us that they have extended the review timeline for the New Drug Application (“NDA”) for omaveloxolone for the treatment of Friedreich’s ataxia by three months.

Read the Full article here

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