Larimar kept in limbo as FDA maintains clinical hold on leading candidate

The FDA is keeping its clinical hold on Larimar Therapeutics' leading candidate to treat Friedreich’s ataxia and is requesting additional data, the company announced Tuesday.

In August, Larimar said it was confident that there was a path forward toward resolving the hold on CTI-1601 and moving on to Jive open-label extension and pediatric multiple ascending dose trials by the first half of this year. 

Now, the company is questioning whether additional studies are warranted to satisfy regulators’ questions regarding the primate trial as well as whether they’ll need to postpone the other two as a result. 

It’s the latest gut punch for Larimar, which has failed to get the trial back on track since the hold was first announced (PDF) last May. Wall Street expectedly soured on the news, with premarket shares down more than 60% to $3.16. 

In a statement, CEO Carole Ben-Maimon, M.D., said the company still believed there was a path forward toward resolving the hold and is committed to the drug’s further development. She added that the company will operate under a “cost reduction plan” to extend its cash runway if needed, which she said currently reaches in 2023.

RELATED: Larimar Therapeutics slapped with FDA clinical hold, forced to halt $95M cash raise

The trial was initially paused after the company notified regulators that there were mortalities in the highest dosing level of a toxicology study using primates. At the time, the FDA requested a full report on the trial, and the company was unable to initiate any additional trials. Larimar also halted a $95 million cash raise it had announced four days earlier. 

The drug aims to deliver a specific human protein, frataxin, into the mitochondria of patients with Friedreich's ataxia who are unable to produce enough on their own. The disease is a neurodegenerative disease caused by mutations in the frataxin gene. 

According to the National Institutes of Health, the disease typically begins in mid-childhood, and patients are unable to walk or stand within 15 years of onset. 

A contact for the company did respond to additional questions about the clinical hold nor which data the FDA was requesting by the time of publishing.