Larimar Therapeutics Inc. (LRMR) said Tuesday that the United States Food and Drug Administration has placed a clinical hold on the CTI-1601 clinical program.
LRMR closed Tuesday regular trading at $13.51 down $1.60 or 10.59 percent. In the after-hours trading, the stock further dropped $6.11 or 45.23 percent.
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.
CTI-1601 is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with Friedreich's ataxia or FA who are unable to produce enough of the essential protein.
CTI-1601, which is currently in Phase 1 clinical trials in the U.S., has been granted Rare Pediatric Disease designation, Fast Track designation and Orphan Drug designation by the U.S. Food and Drug Administration, Orphan Drug Designation by the European Commission, and PRIME designation by the European Medicines Agency.
The safety of CTI-1601 was previously evaluated in Phase 1 single- and multiple-ascending dose clinical trials. Recently announced data from the trials indicated that repeated subcutaneous injections of CTI-1601 were generally well tolerated at doses up to 100 mg administered daily for 13 days.
Data from the MAD trial also showed that daily subcutaneous injections of CTI-1601 at doses of 50 mg or 100 mg resulted in frataxin levels in peripheral tissues (buccal cells) that were at or in excess of those that would be expected in phenotypically normal heterozygous carriers.
Larimar said it will continue with its plan to complete the NHP toxicology study, assess the data, and discuss that data with FDA to obtain their consent prior to initiating its Jive and pediatric MAD trials.
Larimar noted that it continues to believe there is a path forward toward the initiation of Jive and pediatric MAD trials. However, the company believes there is a possibility that the initiation of these trials may be delayed into 2022, due to the additional regulatory requirements that come with responding to a formal clinical hold.
Meanwhile, the company said it terminated recently announced private placement financing. As of March 31, 2021, the company has $81.4 million in cash and investments, which provides cash runway through the first half of 2022.
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