FDA keeps clinical hold in place on Larimar's lead drug with job cuts likely on the horizon
The FDA will extend its clinical hold on Larimar Therapeutics’ lead rare disease drug CTI-1601, asking for more data before it will reconsider the company’s case, Larimar said in a release Tuesday.
The agency initially placed a hold on the Phase I program in Friedreich’s ataxia back in May after the company reported multiple deaths of non-human primates in preclinical testing.
Shares in $LRMR were trading down roughly 62% Tuesday morning, from $8.56 to $3.40.
With the hold now in place for even longer, Larimar said it is evaluating data from completed studies to figure out if it wants to take on the headache of a new study. The company is also reconsidering timing on a planned open-label expansion study, as well as an ascending-dose pediatric trial.
“Our next step is to engage with the agency to determine how we can meet their request in the most efficient and expeditious manner,” CEO Carole Ben-Maimon said in a statement. “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.”
Once known as Chondrial Therapeutics, Larimar went public back in 2019 as part of a SPAC reverse merger with a platform based on protein replacement therapies. CTI-1601 was conceived as the flagship program in that platform, looking to treat the rare genetic disease Friedreich’s ataxia.
The non-human primates that died in a preclinical trial had been given the highest doses of CTI-1601, a recombinant fusion protein designed to deliver human frataxin into the mitochondria, as part of a study around extended dosing of the drug. Patients with FA are unable to produce enough of the protein, which is essential for metabolic functions.
Now, Larimar is looking to slash and burn as it paves its cash runway out past 2023, Ben-Maimon said in a release:
We remain committed to CTI-1601’s further development and are working towards this goal with a strong cash position which 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.