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Larimar Therapeutics Announces Dosing of Patients in Third Cohort of Phase 1 SAD Trial of CTI-1601 for Treatment of Friedreich’s Ataxia
BALA CYNWYD, Pa., July 20, 2020 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Nasdaq:LRMR), a clinical-stage biotechnology company focused on developing
About this update from Larimar Therapeutics, Inc.
[{"type":"text","content":"BALA CYNWYD, Pa., July 20, 2020 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Nasdaq:LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced that patients have been dosed in the third cohort of a Phase 1 clinical trial to evaluate the safety and tolerability of single ascending doses (SAD) of CTI-1601 for the treatment of Friedreich’s ataxia (FA). The trial was previously delayed due to the impact of the COVID-19 pandemic. CTI-1601 is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with FA who are unable to produce enough of this essential protein.\n The double-blind, placebo-controlled trial is evaluating the safety, tolerability and pharmacokinetics of single ascending doses of subcutaneously administered CTI-1601 in patients over age 18 with FA. To date, two cohorts of patients have completed the Phase 1 clinical trial. Topline results are planned for the first half of 2021. “We’re pleased that our Phase 1 clinical trial has resumed and we can continue to move forward with our lead product candidate, CTI-1601, which has the potential to become the first frataxin replacement therapy for patients with FA,” said Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar Therapeutics. “Our highest priority remains the health of our employees and patients especially given the COVID-19 pandemic. We have thoughtfully re-engaged with our clinical site to mitigate the safety risks.” Additional information on the trial can be found on www.clinicaltrials.gov using the identifier NCT04176991. About CTI-1601 CTI-1601 is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with Friedreich’s ataxia (FA) who are unable to produce enough of this essential protein. Currently in a Phase 1 clinical trial, CTI-1601 has been granted Rare Pediatric Disease designation, Fast Track designation and Orphan Drug designation by the U.S. Food and Drug Administration (FDA). To date, two cohorts of patients have completed the single ascending dose (SAD) Phase 1 clinical trial. Topline results from the Phase 1 clinical program are planned for the first half of 2021. About Friedreich’s ataxiaFriedreich’s ataxia (FA) is a rare, progressive, multi-symptom genetic disease that typically pr...