Larimar Therapeutics Issues Update on CTI-1601 Clinical Program
Feb 15, 2022 By MarketDepth
Larimar Therapeutics, Inc. (NASDAQ: LRMR) has reported receipt of feedback from the United States Food and Drug Administration in regards to the clinical hold on Larimar’s CTI-1601 program. The FDA has stated that it is maintaining its clinical hold at this time and require additional data to resolve the current clinical hold. Larimar is analyzing the previously completed studies and is evaluating if additional studies are required. The company will engage the FDA to determine the best steps to provide the required data. “Patient safety is our top priority,” said Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar. “Our next step is to engage with the agency to determine how we can meet their request in the most efficient and expeditious manner. Based on all available clinical and non-clinical data, we continue to believe there is a path forward through the resolution of the CTI-1601 clinical hold. We have a robust Phase 1 dataset, which demonstrates proof-of-concept for CTI-1601 as a frataxin replacement therapy and its differentiated mechanism of action (MOA). We believe this MOA leaves CTI-1601 uniquely positioned to address the urgent need for disease modifying therapies in Friedreich’s ataxia (FA), as it is designed to address the root cause of this devastating disease. We remain committed to CTI-1601’s further development and are working towards this goal with a strong cash position that provides runway at least into 2023. We intend to operate under a cost reduction plan while resolving the clinical hold to manage burn and extend our cash runway if needed.”
No Adverse Effects
The Phase 1 single- and multiple-ascending dose clinical trials displayed repeated subcutaneous injections of CTI-1601 were well tolerated at doses up to 100 mg administered daily for up to 13 days. There were no serious adverse events, important medical events or treatment related severe adverse events reported in the trial. The severity of adverse events did not increase with increased exposure to CTI-1601.