Pharma Focus America

Larimar Therapeutics Receives FDA Approval to Progress to 50 mg Cohort in Phase 2 Friedreich's Ataxia Trial for CTI-1601 and to Commence Open Label Extension Trial

Wednesday, July 26, 2023

Larimar Therapeutics, a biotechnology company focused on developing treatments for rare diseases, has received clearance from the U.S. Food and Drug Administration (FDA) for the next phase of its clinical trial involving CTI-1601 for Friedreich's ataxia (FA). The trial will now progress to a 50 mg cohort, where participants will be administered daily doses for the first 14 days, followed by every other day dosing until day 28. Additionally, Larimar's open label extension (OLE) trial has also been approved to commence, with participants receiving a daily dose of 25 mg of CTI-1601. CTI-1601 is a novel protein replacement therapy designed to increase frataxin levels in patients with FA who have low frataxin levels.

The FDA clearance came after reviewing Larimar's response to its partial clinical hold, which included safety, pharmacokinetic (PK), and pharmacodynamic (PD) data from the completed 25 mg cohort of the Phase 2 trial. The data showed that CTI-1601 was well-tolerated and resulted in increased frataxin (FXN) levels compared to placebo in the evaluated tissues at day 14.

Carole Ben-Maimon, MD, President, and CEO of Larimar, expressed the significance of these milestones, as CTI-1601 could potentially become the first therapy to address frataxin deficiency in FA patients. The company is looking forward to data from the Phase 2 trial's 50 mg cohort, which is expected in the first half of 2024, to further characterize the safety and PK profiles of CTI-1601 and its dose-dependent effect on frataxin levels.

Participants who complete treatment in the Phase 2 trial or have participated in previous CTI-1601 trials are eligible for the OLE trial, which is scheduled to begin in Q1 2024, with interim data expected in Q4 2024.

Larimar aims to expand its clinical program globally to address the needs of the FA community worldwide, as around 75% of individuals with FA live outside the U.S. CTI-1601 has already received several designations, including Orphan Drug (U.S. and Europe), Rare Pediatric Disease (U.S.), Fast Track (U.S.), and PRIME (Europe) designations for FA.

The Phase 2 trial for CTI-1601 is a four-week, placebo-controlled dose exploration study, intending to assess the safety, pharmacokinetic, and pharmacodynamic profiles of the drug. Eligible participants, aged 18 and above, include both ambulatory and non-ambulatory individuals with Friedreich's ataxia (FA). During the trial, participants in each cohort are randomly assigned with a 2:1 ratio to receive either CTI-1601 or placebo. The drug is administered daily through subcutaneous injections for the first 14 days and then every other day until day 28. The trial is currently composed of two cohorts. Cohort 1, consisting of 13 participants (9 on CTI-1601 and 4 on placebo), evaluated a 25 mg dose of CTI-1601. Cohort 2, which is about to commence, will include 12-15 participants, randomized with a 2:1 ratio, and will evaluate a 50 mg dose of CTI-1601. Key endpoints include safety assessments, pharmacokinetic assessments, and measurements of frataxin levels and other pharmacodynamic markers in peripheral tissues. 

The open label extension trial is a multi-center study designed for individuals with FA who have previously participated in a clinical trial involving CTI-1601. Participants will receive daily subcutaneous injections of 25 mg of CTI-1601, with the option for self-administration or administration by a caregiver. The primary objectives of the trial are to evaluate the safety, tolerability, and pharmacokinetics of long-term subcutaneous administration of CTI-1601, along with measurements of frataxin levels and other pharmacodynamic markers in peripheral tissues. Additional objectives include assessing the effects of long-term subcutaneous administration of CTI-1601 on clinical function measures. Data collected during the trial will be compared to a matched set of untreated patients from the Friedreich’s Ataxia Clinical Outcome Measures Study (FACOMS) database.

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