Nomlabofusp’s positive long-term open‑label results strengthen Larimar’s regulatory prospects but significant funding and commercialization risks remain.
Larimar Therapeutics reported positive long-term open‑label results for nomlabofusp in participants with Friedreich’s ataxia, showing benefit at both 25 mg and 50 mg doses delivered by daily subcutaneous self-injection.
The data represent a consequential signal for a rare, progressive neurodegenerative disorder that currently lacks effective disease-modifying treatments. Clinically meaningful durability across doses could reduce regulatory uncertainty around a future Biologics License Application and strengthen the case for accelerated approval pathways.
Despite the clinical upside, Larimar remains pre-revenue and unprofitable, and it forecasts continued losses for several years. Recent equity offerings have diluted shareholders and the share price stays volatile. Key near-term execution risks include securing additional capital, meeting regulatory milestones, scaling manufacturing, and executing commercialization if approved.
Market sentiment and valuation estimates vary widely — community fair-value ranges span roughly $2.60 to $26.00 per share — reflecting divergent views on clinical translation and financing risk.
In short, nomlabofusp’s durable open‑label performance materially improves Larimar’s therapeutic credibility, but substantial operational and funding hurdles must still be cleared before it can become a sustainable rare-disease franchise.