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Supported Research

The research listed below was partially supported by FARA thanks to your generosity.

For more information on FARA-funded research & scientists, please visit FARA Funded Research, Active Clinical Trials and the Featured Scientist.

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.”


Healx launches partnership with Ataxia UK and FARA to find treatments for rare neurodegenerative condition

Ataxia UK is the leading charity in the United Kingdom for people affected by ataxia - an umbrella term for a group of neurological disorders that target the nervous system and affect balance, coordination and speech. FARA (Friedreich’s Ataxia Research Alliance) is a non-profit organisation in the US, dedicated to supporting research into treatments and cures for Friedreich’s ataxia, the most common type of hereditary ataxia. In this partnership, Healx will combine its AI technology and deep pharmacological expertise with Ataxia UK and FARA’s unparalleled patient and scientific insight to drive novel treatments for Friedreich's ataxia from prediction to clinic.

Cambridge UK – 7 April 2021
– Healx, the AI-powered, patient-inspired technology company accelerating the discovery and development of rare disease treatments at scale, is excited to announce its latest patient group partnerships. Working in collaboration with Ataxia UK and FARA, Healx will leverage its state-of-the-art AI platform and drug discovery expertise to develop novel treatments for Friedreich’s ataxia – a rare neurodegenerative condition that causes issues with balance, speech and coordination.

Read the Full article here

Design Therapeutics Launches with $45 Million to Develop a New Class of Disease-Modifying Therapies for Serious Degenerative Disorders

Series A Financing Funds Novel Pipeline for Patients with Nucleotide Repeat Disorders

Company Advancing Lead Program for Friedreich’s Ataxia toward Clinical Development

San Diego, Calif., March 20, 2020 – Design Therapeutics announced today that it is launching to create and develop a new class of therapies for patients with serious degenerative disorders caused by nucleotide repeat expansions. The company has closed a $45 million Series A financing led by SR One, with participation from Cormorant Asset Management, Quan Capital and WestRiver Group, to advance its lead therapeutic candidate into clinical development for the treatment of Friedreich’s ataxia, and support advancement of its discovery programs for multiple other degenerative diseases, including fragile X syndrome and myotonic dystrophy. The company’s pipeline is led by a novel program for Friedreich’s ataxia. Design Therapeutics has developed a novel program that unblocks transcription, thereby restoring the natural production and function of frataxin. With the use of proceeds from the Series A fundraising, Design Therapeutic intends to conduct IND-enabling studies and initiate clinical development for its program for Friedreich’s ataxia.

Read the entire article HERE

Chondrial Therapeutics Announces Dosing of First Patients in Phase 1 Clinical Program of CTI-1601 for Treatment of Friedreich’s Ataxia

CTI-1601 granted Rare Pediatric Disease Designation and Fast Track Designation by U.S. FDA

BALA CYNWYD, Pa., Dec. 18, 2019 (GLOBE NEWSWIRE) -- Chondrial Therapeutics, a clinical-stage biotechnology company focused on developing treatments for rare diseases, with an initial focus on Friedreich’s ataxia (FA), today announced that the first patients have been dosed in a Phase 1 clinical trial to evaluate the safety and tolerability of single ascending doses of CTI-1601. CTI-1601 is a recombinant fusion protein intended to deliver human frataxin to patients with FA, who have decreased levels of frataxin.

"CTI-1601 is designed to address the root cause of Friedreich’s ataxia – low levels of frataxin. The initiation of our Phase 1 clinical program was supported by positive preclinical data and is an important step forward for patients affected by FA, a devastating and progressive disease for which there is no cure," said Carole Ben-Maimon, MD, President and Chief Executive Officer of Chondrial Therapeutics. "We separately announced today a merger that will provide us with significant resources to advance our development of CTI-1601 and expand our efforts in other rare diseases that may benefit from a cell-penetrating peptide approach."

View the entire Press Release Here

Exicure Announces First Neurological Development Program in Friedreich’s Ataxia and Expands Scientific Advisory Board

- Exicure to collaborate with Friedreich’s Ataxia Research Alliance (FARA)

- Hank Paulson, MD, PhD and Susan Perlman, MD, added to Scientific Advisory Board

CHICAGO & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dec. 16, 2019-- Exicure, Inc. (NASDAQ:XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) technology, today announced Friedreich’s ataxia (FA) as the therapeutic indication for the company’s first neurology development program. Exicure also announced the expansion of its Scientific Advisory Board.

"Over the past year Exicure has developed extensive preclinical data supporting the development of our SNAs for neurological disorders. We’re eager to evaluate our technology for this important unmet medical need," said David Giljohann, PhD, Exicure’s chief executive officer. "We are also pleased to add the experience and team from the Friedreich’s Ataxia Research Alliance (FARA)," added Dr. Giljohann.

View the Exicure Press Release PDF Here

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