Larimar Therapeutics Inc. (NASDAQ: LRMR) has announced that it has received feedback from the US FDA about the clinical hold in its CTI-1601 program.
The FDA will retain clinical hold on CTI-1601
The FDA indicated that it is retaining its clinical hold for now and that more data is required to lift the hold. Larimar is delving deeper into previously completed studies to see if more research is necessary. The company also plans to work with the FDA to figure out the best way to deliver this data. As part of its efforts to satisfy the agency’s request, Larimar is reevaluating recommendations on scheduling the scheduled Jive open-label expansion and pediatric multiple-ascending dosage clinical trials.
CEO and President Carole Ben-Maimon stated, “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).”
CTI-1601 meant to treat Friedreich’s ataxia cause
Because CTI-1601 is designed to treat the fundamental cause of Friedreich’s ataxia (FA), it is ideally positioned to meet the critical demand for disease-modifying medications for this devastating condition. The company remains committed to the advancement of CTI-1601, and it is working toward this objective with a good cash position that gives it at least until 2023. Larimar expects to follow a cost-cutting strategy while addressing the clinical hold to limit burn and, if necessary, extend its cash runway.
Following the company’s report to the FDA of mortalities at the maximum dose intervals in a 26-week non-human primate (NHP) toxicity trial that was supposed to support continued dosing of subjects with CTI-1601, the FDA placed the study on clinical hold. However, following Larimar’s submission of a complete response, which included a thorough study result from the 26-week NHP toxicity trial, the FDA recently provided feedback.
