Dear friends,
The treatment pipeline for Friedreich’s ataxia is in a critical phase of development. There are clinical trials in process and more to become available by the end of 2021 and into 2022. All these studies are important as we need multiple treatments to address the symptoms and unmet medical need for individuals with FA.
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Enrollment, participation, and completion of a clinical trial is an important commitment and a personal decision. Individuals who participate in a trial or research study are not only making a decision that can impact their own personal health, but also impact the whole FA community. We need individuals to voluntarily enroll and participate, to adhere to the study protocol, and to complete the study and final visits. Studies that fail or have inconclusive results due to lack of participation, lack of compliance, or high dropout rates, slows our progress towards treatments and a cure for FA.
When participants withdraw from a clinical trial due to travel fatigue to adhere to the clinical trial protocol, concern of being on placebo, or desiring to enroll in a different study, there is significant risk that the clinical trial will not reach conclusive results or meet its endpoint to determine safety and efficacy of the drug. Our persistence and dedication to research and advancing treatments for FA are bringing our community several choices of investigational studies with several more on the horizon. Please consider your personal situation and upcoming life events, along with the risks and benefits and goals of the trial or research study when enrolling in a clinical trial. Properly conducted and completed research studies deliver results we can have confidence in and will change the future for those diagnosed and living with FA.
Sincerely,
Jen
Jen Farmer,
Chief Executive Officer
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Design Therapeutics Reports GeneTAC™ Portfolio Progress
Design Therapeutics is a biotechnology company developing a new class of therapies based on a platform of gene targeted chimera (GeneTAC™) small molecules. The company’s GeneTAC molecules are designed to either turn on or turn off a specific disease-causing gene to address the underlying cause of disease. Design’s lead program is focused on the treatment of Friedreich's ataxia, and they announced their plans to begin clinical development in the first half of 2022. To read the update about the FA program, click here.
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Reata Pharmaceuticals, Inc. Announces Second Quarter 2021 Financial Results and Provides an Update on Clinical Development Programs
Omaveloxolone in Patients with Friedreich’s Ataxia (“FA”).
Based on a communication received from the U.S. Food and Drug Administration (“FDA”) regarding omaveloxolone for the treatment of FA, Reata withdrew their request for a Type C meeting and requested a pre-NDA meeting with the FDA. The pre-NDA meeting request has been granted, a pre-NDA meeting has been scheduled during the third quarter of this year, and they have submitted briefing materials for the meeting. Reata recently received a communication from the FDA requesting the estimated date of our New Drug Application (“NDA”) for its planning purposes. Reata plans to submit the NDA during the first quarter of 2022. To read the full update, click here.
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Larimar Therapeutics Reports Second Quarter 2021 Operating and Financial Results
“We finished the second quarter in a strong financial position and with a compelling clinical data set that demonstrates proof-of-concept for CTI-1601, which to our knowledge is the only clinical-stage candidate designed to address the root cause of Friedreich’s ataxia,” said Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar. “These positive Phase 1 data along with non-clinical pharmacology data demonstrate proof-of-concept, and CTI-1601’s differentiated mechanism of action helped us to earn a PRIME designation from the European Medicines Agency, providing us with valuable regulatory benefits and important external validation. Looking forward, we continue to collect and analyze data from our 180-day non-human primate toxicology study and remain confident that there is a path forward through the resolution of the CTI-1601 clinical hold and towards the initiation of our Jive open-label extension and pediatric multiple ascending dose trials.” To read the full press release, click here.
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FARA Newly Funded Research |
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Testing the efficacy of dietary butyrate in ameliorating ataxic symptoms in Friedreich’s ataxia mouse models
Daniele Lettieri-Barbato, PhD – University of Rome Tor Vergata, Italy
A direct link has been discovered in recent years between the gut and the brain and alteration of the composition of the microbial gut population is associated with several neurodegenerative diseases. In this project, by using mouse models of FRDA, Dr. Lettieri-Barbato and his team will investigate whether the gut microbiome is altered in FRDA with a particular focus on bacteria producing butyrate, a molecule produced by fermentation of fiber in the colon that has been shown to possess both a neuroprotective and anti-diabetic functions. FRDA mice will be treated with a diet rich in butyrate and the effect of butyrate on motor functions and other molecular hallmarks of the disease will be assessed.
Co-sponsor: AFAF
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Upcoming Grassroots Events |
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No matter where you are, we invite you to be a part of this year’s FARA Energy Ball, which will take place on International Ataxia Awareness Day, September 25!
If you are unable to travel to Tampa for the in-person event, you can take part with your very own watch party. Whether you wish to tune into the livestream with a few family members, or throw a glamorous party for your entire community, we welcome you to be a part of the energy!
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rideATAXIA Philly is back! Join us for the in-person ride on Saturday, October 9th in Harleysville, PA at the LBCB Church: BranchCreek Campus. We will have 1, 3, 6, 21, and 46-mile biking routes along with a lunch provided by Outback Steakhouse. You can register today by clicking the button below.
Along with Philly, the rideATAXIA Global Challenge returns for its second year! This virtual event will take place from September 28 - October 9, with ride day planned for the same day as rideATAXIA Philly.
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rideATAXIA Dallas returns on November 6th! Join us in person at UNT Discovery Park in Denton, Texas.
We will have 6, 20, and 50-mile bike rides, a delicious post-ride meal provided by Outback Steakhouse, along with great times with our good friends in Texas.
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Download the FA App today! Nearly 1,400 users have already downloaded the app, representing over 60 countries worldwide! Right now, there are 16 research studies that are actively enrolling through the app. The app also has options for 9 different languages, connects people in the FA Community from all over the world, and provides the latest in FA research and community news. You can join as an FAer, a parent, and/or a friend someone with FA.
You can download the app by searching for "The FA App" in the Apple Store or Google Play, or by visiting thefaapp.org. If you have any suggestions on how to improve the app, let us know at support@thefaapp.org
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Researchers at the Children’s Hospital of Philadelphia and the Hospital of the University of Pennsylvania are recruiting participants for a study to test the effects of a dietary supplement combined with exercise on aerobic capacity. Aerobic capacity refers to the body’s ability to take in, transport, and use oxygen during exercise.
Eligibility includes:
- Documented genetic diagnosis of FA
- Age 10 to 40
- Ability to complete exercise testing
- Not pregnant for women of child-bearing years
If you are selected to participate in this study, you would complete 2 in-person study visits, each lasting 2 days, over a 12-week study period at CHOP and Penn. During the study period, you may complete blood testing, exercise testing, administration of a dietary supplement, MRI scans, oral glucose tolerance testing (with a non-FDA approved stable isotope product) and optional muscle biopsies.
You will be compensated for your time.
To learn more about the study and determine your eligibility, contact the
(267) 425-1998.
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Enrollment now open at University of Iowa, University of South Florida, UCLA, Children’s Hospital of Philadelphia (CHOP), Murdoch Children’s Research Institute (Australia), CHUM (Canada) and University of Campinas (Brazil).
Sites in France and New Zealand are open for eligibility screening. Sites in Spain and Italy expected to open later in August.
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Sites are open for enrollment at University of Minnesota (Minneapolis), University of Florida (Gainesville), Children’s Hospital of Philadelphia, Monash University (Victoria, Australia) and RWTH Aachen University (Aachen, Germany). To contact a study coordinator, please click HERE.
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39 FA participants are needed to fill this study by the end of 2021. The outcomes of this study are critical for allowing remote data collection during future clinical trials.
For additional eligibility criteria, please contact study coordinator Hannah Casey (hannahcasey@uchicago.edu) or (773) 702-4610. Please specify your city of interest for enrollment - Baltimore, Boston, Chicago, Los Angeles or Portland (OR).
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Anyone considering participating in a clinical trial should discuss the matter with his or her physician. FARA does not endorse or recommend any particular study. |
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The goals of the FA Global Patient Registry (FAGPR) are to collect information on all FA patients in one registry, to develop the registry into a powerful resource for research, and to engage the FA community in studies aimed at advancing our knowledge of FA and the treatments being developed.
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The Pathway
You can help fund research progress all year by joining our monthly giving program, The Pathway. When you join The Pathway with a monthly donation of $10 or more, you are joining a community invested in setting a path to treatments and a cure for FA through research. Click the image to give today.
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