Larimar Therapeutics, Inc. (NASDAQ: LRMR) recently announced initial data from its ongoing long-term open-label extension (OLE) study evaluating daily subcutaneous injections of 25 mg nomlabofusp for Friedreich’s Ataxia. This news coincided with a drop in LRMR stock price, prompting investors to analyze the study results and their implications.
Friedreich’s Ataxia is a rare, inherited disease that causes progressive nervous system damage and movement problems. It arises from a mutation in the FXN gene, responsible for producing frataxin, a protein crucial for mitochondrial function. Mitochondria are the powerhouses of cells, and frataxin deficiency leads to cellular energy depletion. Nomlabofusp aims to address this deficiency by increasing frataxin levels.
The OLE study included 14 patients with Friedreich’s Ataxia, with over half being non-ambulatory, receiving daily 25 mg doses of nomlabofusp for up to 260 days (average 99 days). A key finding was the observed increase in tissue FXN levels. By day 90, mean frataxin levels in buccal (mouth lining) cells increased from a baseline of 15% of healthy volunteer levels to 30%. Even more significantly, skin cell frataxin levels rose from 16% to 72% of healthy volunteer levels. This suggests that nomlabofusp can effectively elevate and maintain frataxin levels in affected tissues.
Further analysis indicated that buccal cell frataxin levels reached a steady state around day 30. While the drug was generally well-tolerated, two participants experienced serious adverse events, resolving within 24 hours, leading to their withdrawal from the study. The most common side effects were mild and temporary injection site reactions.
Building on these findings, Larimar has initiated dose escalation to 50 mg daily in six participants within the OLE study. Additionally, the company is screening adolescents with Friedreich’s Ataxia for a pediatric pharmacokinetic (PK) run-in study, with dosing anticipated in early 2025. Adolescents completing this study will transition into the OLE study after safety and PK data assessment.
Despite these seemingly positive developments, LRMR stock experienced a significant decline, trading down 21.5% at $4.85 on Monday. This market reaction suggests that investors may be cautiously interpreting the data, potentially focusing on the small sample size, the occurrence of serious adverse events, or broader market trends.
In conclusion, the initial data from Larimar Therapeutics’ OLE study demonstrates nomlabofusp’s potential to increase and sustain frataxin levels in individuals with Friedreich’s Ataxia. However, the market response underscores the need for further research, including larger-scale trials and longer-term follow-up, to fully evaluate the efficacy and safety profile of nomlabofusp. The upcoming pediatric PK study and continued dose escalation within the OLE study will provide valuable insights into the drug’s potential in a broader patient population. Continued monitoring of LRMR stock performance and future data releases will be crucial for investors interested in this developing therapeutic area.
