Larimar Therapeutics, Inc. (NASDAQ: LRMR) saw a 17.19% increase in after-hours trading on Monday, reaching $8.52, following a positive regulatory update. Investor excitement over recent advancements in the company’s clinical trials is reflected in this notable boost.
FDA Lifts Partial Hold On Nomlabofusp Program
Larimar Therapeutics (LRMR) announced that the U.S. Food and Drug Administration (FDA) has lifted the partial clinical hold on its nomlabofusp (CTI-1601) clinical program. Nomlabofusp, a pioneering protein replacement therapy, is being developed to treat Friedreich’s Ataxia (FA).
This therapeutic approach aims to address the underlying cause of FA by delivering frataxin directly to mitochondria. The FDA made its judgment after carefully examining the information from the recently finished four-week Phase 2 dosage exploration trial for Larimar, which was placebo-controlled.
Two cohorts participated in the trial; they were given dosages of 25 mg and 50 mg, respectively, and were dosed daily for the first 14 days, then every other day until day 28. Larimar was able to move forward with increasing the dosage in its current Open-Label Extension (OLE) trial to 50 mg as a result of this evaluation.
Promising Interim Data And Future Plans
The OLE study, which is currently evaluating the long-term safety and pharmacokinetics of nomlabofusp, will initially focus on 25 mg daily subcutaneous injections, either self-administered or administered by a caregiver. Larimar plans to escalate to a 50 mg dose after further characterizing frataxin pharmacodynamics (PD) at the 25 mg level. Should there be a need to escalate beyond 50 mg, additional data will be required for FDA review.
Nomlabofusp was generally well tolerated, with a predictable pharmacokinetic profile and dose-dependent increases in skin and buccal cell frataxin levels, according to the Phase 2 research. Notably, patients in the 50 mg cohort achieved significant increases in frataxin levels, with some reaching over 50% of the average levels observed in healthy individuals.
Interim data from the OLE study are anticipated in the fourth quarter of 2024, which will provide further insights into the long-term effects of nomlabofusp in patients with FA. This data is crucial for determining the future trajectory of the drug’s development and potential FDA approval for broader use.
