Larimar: FDA Clinical Hold on CTI-1601; Termination of Financing

 Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for Friedreich’s ataxia (FA) and other complex rare diseases, today announced that the United States Food and Drug Administration (FDA) has placed a clinical hold on the CTI-1601 clinical program and that the company will not be closing a previously announced private placement financing. 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.

The clinical hold follows the previous notification by Larimar to the FDA of mortalities which occurred at the highest dose levels in an ongoing 180-day non-human primate (NHP) toxicology study, which is designed to support extended dosing of patients with CTI-1601. In the clinical hold letter, the FDA stated it needs a full study report from the ongoing NHP study and Larimar may not initiate additional clinical trials until the company has submitted the report and received notification from the agency that additional clinical trials may commence.

“While the notification of a formal clinical hold is disappointing, it does not change our previously stated clinical development strategy for CTI-1601,” said Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar. “Patient safety is our top priority, and we will continue with our plan to complete the NHP toxicology study, assess the data, and discuss that data with FDA to obtain their consent prior to initiating our Jive and pediatric MAD trials. Based on all of the information we have regarding CTI-1601’s safety profile to date, we continue to believe there is a path forward toward the initiation of our Jive and pediatric MAD trials. However, due to the additional regulatory requirements that come with responding to a formal clinical hold, we believe there is a possibility that the initiation of these trials may be delayed into 2022. Regarding the termination of the previously announced private placement financing, as of March 31, 2021, we have $81.4 million in cash and investments, which provides cash runway through the first half of 2022.”

https://finance.yahoo.com/news/larimar-therapeutics-reports-fda-clinical-214100959.html