Dear friends,
International Ataxia Awareness Day is quickly approaching on September 25th, and there are many ways to get involved throughout the Fall. This newsletter will highlight some of the upcoming events such as United Against Ataxia Hill Day, the FARA and CHOP Symposium, the Energy Ball, rideATAXIA, as well as new clinical studies and more. We look forward to seeing you online or in-person for one or several of these gatherings.
Sincerely,
Jen
Jen Farmer,
FARA Chief Executive Officer
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Larimar Therapeutics Announces FDA has Partially Lifted the Clinical Hold and Plans for a Phase 2 Dose Exploration Trial of CTI-1601 in Friedreich’s Ataxia Patients
The FDA has cleared the initiation of 25 mg cohort of a Phase 2, four-week, placebo-controlled, dose exploration trial of CTI-1601 in Friedreich’s ataxia (FA) patients. In a written communication to Larimar, the FDA indicated it was lifting its full clinical hold on the CTI-1601 program and imposing a partial hold. The FDA will review data from the 25 mg cohort prior to escalating the dose in the second cohort. Larimar expects to begin the Phase 2 trial in Q4 2022.
Larimar’s upcoming Phase 2 trial is designed to further characterize CTI-1601’s safety, pharmacodynamic (PD), and pharmacokinetic (PK) profiles to provide information about the preferred long-term dose and dose regimen. Eligible patients will include ambulatory and non-ambulatory individuals with FA who are at least 18 years old.
Click here for the full press release which contains additional background and information on this program.
FARA is working with Larimar to host a webinar with Larimar Therapeutics for the patient community next week. We will share date and time and registration details in the next few days.
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The FARA Grant Program is excited to announce that the 2022 Award for Innovative Mindset has been granted to Anna Stepanova, PhD at the Weill Cornell Medical College for Calcium communication among intracellular compartments in FA patient-derived cells. Learn more about this award and the grant program here.
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United Against Ataxia Hill Day |
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Although registration has closed, you can still be part of the fourth United Against Ataxia Hill Day! Email or call your Senator or Congressman/woman next Wednesday, September 21!
There are just three easy steps to add your voice to the collective effort:
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Look up your Congressional Members at house.gov (upper right hand corner) or senate.gov (upper left hand corner)
- Call or email your Senator or Congressman/woman. Share your story of ataxia and ask them to support initiatives that will expedite treatments. Here is a template that you can use:
Dear Senator __________ or Congressman/woman _____________,
Today, many of my fellow community members are meeting with your colleagues for the fourth United Against Ataxia Hill Day. I was unable to attend the meetings but wanted to share my story and explain how you can help.
<insert a short story describing your ataxia journey>
Research funding is our top priority since there are currently no approved treatments for any of the 70+ types of ataxia. There are several ways you can help faciliate this initiative:
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Support the continued inclusion of Hereditary Ataxia in the Congressionally Directed Medical Research Programs (CDMRP). Friedreich's Ataxia was added to the CDMRP in FY22, and the FY23 Senate draft bill has broadened that the title to include all hereditary ataxias. Several similar appropriations requests have been submitted to the House Defense Appropriations sub-commitee.
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Increase funding for federal agencies that support research and drug development AND pass a timely budget. Continuing resolutions hamper research efforts and slow treatments to patients.
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Pass the Prescription Drug User Fee Act (PDUFA) by September 30 to guarantee that the FDA has the staff and resources to efficiently review new drug applications.
Let brigid.brennan@curefa.org know how it goes! You can even cc her on the email, so FARA can keep everyone informed of any future initiatives.
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NIH Eye on the Future Video Contest |
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Congratulations to 15-year-old Claire Juip for receiving an honorary award in the NIH Eye on the Future Video Contest. Her video describes a future when we’ve found a cure for Friedreich’s Ataxia, including its impact on eyesight. View Claire’s video HERE.
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The FARA store is now open on Bonfire! Outfit yourself, your kids, and even your four-legged friends in FARA apparel with a wide selection of styles, colors, and designs. A portion of each sale is donated back to FARA. Click the link below to shop now!
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Join us on Saturday, September 24, 2022 in Harleysville, PA for rideATAXIA Philly! Enjoy a 46, 21, 6, or 3 mile ride or a 1 mile walk/ride with lunch to follow from Outback Steakhouse. |
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The Friedreich's Ataxia Research Alliance (FARA) and the Friedreich’s Ataxia Center of Excellence at Children’s Hospital of Philadelphia (CHOP) will host a hybrid in-person and virtual symposium September 24th & 25th in King of Prussia, PA.
The symposium includes a panel discussion on Clinical Stage Programs in FA Research, a presentation on Gene Therapy and Gene Editing, and more.
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Join us for the FARA Energy Ball on Saturday, October 15th!
*NEW LOCATION*
JW Marriott
510 Water St.
Tampa, FL 33602
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Join us on November 5, 2022 for rideATAXIA Dallas in Denton, TX for 42, 17, and 6 mile route options, as well as a 1.5 mile walk and a General Admission registration option. Lunch will be catered by Outback Steakhouse, and we are looking forward to an awesome celebration of your fundraising efforts. |
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New Clinical Studies/Trials |
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Three new clinical studies/trials are open for enrollment.
This study is currently recruiting at University of California Los Angeles. Other sites, including University of South Florida, University of Iowa, and Mayo Clinic, will start recruiting at a later date.
The screening period will take up to 3 months, and then there will be 16 office visits during the first year following the investigational therapy administration. After that, there will be a long-term follow up period consisting of 10 additional office visits over the next 4 years.
Click HERE to access training modules on gene therapy.
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1. Measure patient-relevant changes in disease burden over time FA-HI (Friedreich Ataxia-Health Index)
2. Measure caregiver-reported changes in disease burden over time FACR-HI (Friedreich Ataxia Caregiver Reported-Health Index).
The study will require completion of surveys (lasting 15-30 minutes) either by an FA patient or an FA caregiver at four timepoints- baseline, 6 months, 12 months, and 18 months.
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Click the titles for additional information about each study.
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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 studies. |
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There are several clinical studies and trials open and enrolling participants. Click the titles below to learn more about the research studies, participating sites, and contact information for study coordinators.
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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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