Larimar Therapeutics’ stock $LRMR shot up by as much as 20% after announcing Monday that the FDA had lifted its partial clinical hold on its treatment for Friedreich’s ataxia, a rare disorder that affects the nervous system.
The company wrote that the FDA lifted the hold after reviewing data from its Phase 2 dose exploration study for its drug nomlabofusp. Larimar’s treatment was put under clinical hold in 2021 following three primate deaths in a preclinical study and then partially lifted the following year.
Carole Ben-Maimon“Importantly, we are now cleared to dose escalate to the 50 mg dose in our ongoing [open label extension] study which we plan to do following further characterization of frataxin [pharmacodynamics] at the 25 mg dose,” Larimar president and CEO Carole Ben-Maimon said in a statement.
The open-label extension study is investigating both the long-term safety as well as frataxin levels following daily dosing of nomlabofusp, with interim data expected in the fourth quarter of 2024, Ben-Maimon said.
Larimar released Phase 2 data earlier this year, showing that nomlabofusp increased levels of the protein frataxin. When frataxin levels are low, it leads to symptoms including muscle weakness and a loss of balance.
William Blair analysts wrote in a Monday note that they “see the hold removal as regulatory buy-in that restoring [frataxin] expression could provide a functional and disease-modifying benefit to FA patients and that nomlabofusp’s risk/benefit is favorable beyond the [nonhuman primate] mortalities observed over 3 years ago.”