Recently, Ataxia UK had a meeting with representatives from Larimar Therapeutics, a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, to discuss possible collaborations to help progress research on a potential treatment for Friedreich’s ataxia (FA). In December, Larimar announced the completion of dosing from its Phase 1 single ascending dose (SAD) clinical trial evaluating CTI-1601 as a potential frataxin replacement therapy for patients with FA. Dosing has been completed and based on preliminary data, single subcutaneous injections of CTI-1601 at doses up to 100 mg are thought to have been well tolerated. Injection site adverse events were mild and transient, and no serious adverse events were reported. Analysis of clinical trial results remains ongoing Phase 1 multiple ascending dose (MAD) clinical trial. To date, dosing of the first two MAD cohorts has been completed, with dosing of the third cohort expected to begin in the first quarter of 2021. Larimar expects to report unblinded topline data from both the SAD and MAD trials in the second quarter of 2021. Patients completing the SAD and/or MAD clinical trials are eligible to screen for an open-label extension clinical trial, which is expected to initiate in the second half of 2021. Larimar also expects to initiate a MAD clinical trial in patients under 18 years of age in the second half of 2021.
The full press release can be viewed here.