Friedreich's Ataxia Research Alliance (FARA)

Today, the Pediatric Inclusion Roundtable: Effective Inclusion of Children Early in Clinical Trials meeting brought together stakeholders to discuss pediatric drug development for rare diseases like Friedreich’s ataxia. FARA extends sincere gratitude to all of the speakers, including representatives from FDA, The National Institutes of Health, industry sponsors, and patient advocacy groups. Special thanks to Ron Bartek for spearheading this effort along with Leavitt Partners and all of the speakers, moderators, and participants today.

In a webcast today, Monday, July 15, 2024, Lexeo Therapeutics reported the interim clinical results of their SUNRISE-FA Phase 1/2 clinical trial for the treatment of Friedreich ataxia cardiomyopathy (NCT05445323) and the Weill Cornell Medicine investigator-initiated Phase 1A trial (NCT05302271) (from 8 participants with > 6-months of follow-up): -Among the participants with elevated left ventricular mass index (LVMI) at baseline, mean reduction in LVMI was 11.4% at 12 months (n=4) and 18.3% at 18 months (n=2) -Sustained and consistent improvements in other key measures of cardiac status, including left ventricular wall thickness (n=6) and troponin I (n=5), in majority of participants at 12 months -Increased post-treatment frataxin expression above baseline in those participants from the Lexeo study evaluated via myocardial biopsy to date -LX2006 was well tolerated with no treatment-related serious adverse events to date -SUNRISE-FA Phase 1/2 clinical trial is proceeding to Cohort 3; one participant dosed in this cohort to date Click here to read the full press release: https://buff.ly/467aWSZ Click here to access the webcast recording: https://buff.ly/467aX9v

Last week, FARA had the honor of participating in UMDF’s Mitochondrial Medicine Conference in Cleveland, OH (https://lnkd.in/gkVp4Jru). This annual gathering served as a global platform for leaders in the field, fostering discussions on the latest advancements, potential treatments, and cutting-edge research. The conference not only highlighted industry breakthroughs but also emphasized the crucial role of patient engagement in accelerating research.    FARA Scientific Advisory Board Member, Dr. David Lynch, MD, PhD, addressed topics such as clinical trial endpoints and biomarkers in Friedreich’s ataxia (FA), sparking insightful discussions. Additionally, Lindsay Marjoram, PhD, Director of Research for the Barth Syndrome Foundation and FARA Board Member and Finance Director, Ruth Acton, discussed the important role that patient advocacy groups play in providing educational resources to the Barth and Friedreich’s ataxia communities. Ruth highlighted FARA's Clinical Trials Participant Guide, gene therapy educational modules and stressed the importance of community engagement in advocating for access to treatment. Learn more about FARA's educational resources at curefa.org

Join Leavitt Partners and Friedreich's Ataxia Research Alliance (FARA) for a Pediatric Inclusion Roundtable.    The day will begin with discussions with FDA and The National Institutes of Health leadership on their vision for effective pediatric inclusion, followed by multi-stakeholder panel discussions centered around FDA’s three medical product centers: CDER, CBER, and CDRH.   We invite all stakeholders to join our discussions regarding the need for including children earlier in clinical development, the requirements for doing so, and stakeholder barriers.   For registration and agenda, see https://lnkd.in/eW4Cnh_5   Contact Elizabeth Hassett, MPH (elizabeth.hassett@leavittpartners.com) with questions or for more information.

Join Leavitt Partners and Friedreich's Ataxia Research Alliance (FARA) for a Pediatric Inclusion Roundtable. The day will begin with discussions with FDA and The National Institutes of Health leadership on their vision for effective pediatric inclusion, followed by multi-stakeholder panel discussions centered around FDA’s three medical product centers: CDER, CBER, and CDRH. We invite all stakeholders to join our discussions regarding the need for including children earlier in clinical development, the requirements for doing so, and stakeholder barriers. For registration and agenda, see https://lnkd.in/eW4Cnh_5 Contact Elizabeth Hassett, MPH (elizabeth.hassett@leavittpartners.com) with questions or for more information.

Join Leavitt Partners and Friedreich's Ataxia Research Alliance (FARA) for a Pediatric Inclusion Roundtable.    The day will begin with discussions with FDA and The National Institutes of Health leadership on their vision for effective pediatric inclusion, followed by multi-stakeholder panel discussions centered around FDA’s three medical product centers: CDER, CBER, and CDRH.   We invite all stakeholders to join our discussions regarding the need for including children earlier in clinical development, the requirements for doing so, and stakeholder barriers.   For registration and agenda, see https://lnkd.in/eW4Cnh_5   Contact Elizabeth Hassett, MPH (elizabeth.hassett@leavittpartners.com) with questions or for more information.

This week, FARA CEO Jen Farmer visited the CSIR-Institute of Genomics and Integrative Biology (IGIB), where she met with Dr. Mohammed Faruq and his team of ataxia researchers. Jen was warmly welcomed by the Director, Dr. Souvik Maiti, who invited her to attend their Research Council Meeting. It was an excellent opportunity to gain insights into the institute's diverse research initiatives, including a focus on FA research. During her visit, Jen also had the chance to tour their new GMP cell therapy facility as well as their genomics and zebrafish cores.

During their visit to New Delhi, India this week, Jen Farmer and Kyle Bryant represented FARA at the AIIMS (All India Institute of Medical Sciences, New Delhi) FA Research Community Meeting where they met with clinicians and researchers from AIIMS and other esteemed medical and research institutions across India. The meeting provided a valuable opportunity for face-to-face engagement with dedicated clinicians and researchers including Dr. Istaq Ahmad (Coordinator, AIIMS FA GCC site), Dr. Akhilesh Kumar Sonakar, Dr. Mohammed Faruq (Investigator, Senior Principal Scientist, CSIR-IGIB), Shweta Sahni (Coordinator, AIIMS FA GCC and FARA Grad Research Fellow), Jyoti Katoch, Dr. Achal Kumar Srivastava (Site PI, Professor, Department of Neurology, AIIMS), and Dr. Muvin Khan (Clinical Coordinator, FA GCC). AIIMS serves as a site for the Friedreich’s Ataxia Global Clinical Consortium (FA GCC) in India, having successfully enrolled nearly 150 individuals with FA in research initiatives like the UNIFAI natural history study. This consortium fosters collaboration among FA researchers and clinicians worldwide, facilitating the exchange of vital information and resources essential for advancing research.

On June 25, 18 families living with Friedreich’s ataxia (FA) attended the All India Institute of Medical Sciences (AIIMS) FA Community Symposium in New Delhi, India. We were honored to participate in this community education day and look forward to our continued collaboration with AIIMS in their efforts to advance FA research and raise awareness about this rare disease in India. The event garnered attention from local news stations, further spreading awareness about FA. FARA believes in the strength of a well-informed and connected global community, and we are grateful to the organizers of the event as well as the attendees for actively engaging and staying informed about FA.

Yesterday, FARA CEO Jen Farmer and rideATAXIA Director Kyle Bryant visited the Friedreich's Ataxia Global Clinical Consortium (FA GCC) site at AIIMS (All India Institute of Medical Sciences, New Delhi). The Director of AIIMS, Dr. M Srinivas, presented FARA with these beautiful silver plates in recognition of our partnership and visit. The FA GCC is an international group of clinical research centers that work together to advance treatments and clinical care for individuals with Friedreich’s ataxia. Learn more about FARA's global research network and the FA GCC here: https://lnkd.in/gzAYKzNs During this visit, Jen and Kyle met with Prof Dr. Achal Kumar Srivastava, the Principal Investigator of the AIIMS FA GCC site. They also had the opportunity to meet with many other researchers and clinicians including FARA-funded researcher, Dr. Shweta Sahni, to discuss how the FA GCC site at AIIMs functions. Learn more about Dr. Sahni's research here: https://lnkd.in/g3fkfJrj