On Tuesday, H.C. Wainwright maintained its Buy rating and $15.00 price target for Larimar Therapeutics (NASDAQ:LRMR), following the company's announcement of positive preliminary data from its ongoing study.
According to InvestingPro data, analysts are notably bullish on LRMR, with price targets ranging from $12.49 to $36, despite the stock's recent 26% decline over the past week. The research focuses on nomlabofusp, a potential treatment for Friedreich’s ataxia (FA), a debilitating genetic disease. The study is designed to assess the long-term safety, pharmacokinetics, and impact on frataxin levels in patients with FA.
The open label extension (OLE) study by Larimar Therapeutics reported encouraging initial results. Nomlabofusp, a subcutaneously administered recombinant fusion protein, is being investigated for its ability to deliver frataxin directly to the mitochondria of FA patients.
Addressing frataxin deficiency is crucial, as it is the fundamental cause of the disease. With a market capitalization of $308 million and a strong liquidity position evidenced by a current ratio of 13.1, InvestingPro analysis indicates the company maintains a solid financial foundation for its research initiatives.
The U.S. Food and Drug Administration (FDA) has granted nomlabofusp several designations that could expedite its development, including Rare Pediatric Disease Designation, Fast Track Designation, and Orphan Drug Designation. Additionally, the European Commission has awarded it Orphan Drug Designation, and the European Medicines Agency (EMA) has given it PRIME designation.
Larimar's ongoing study includes 14 adults with FA, with treatment durations ranging from 99 to 260 days. Notably, over half of the participants were non-ambulatory. The treatment with nomlabofusp has shown to increase and sustain tissue frataxin levels.
At Day 90, buccal cells exhibited a mean change from baseline frataxin levels of 1.32pg/µg, and skin cells showed an increase of 9.28pg/µg. These levels were maintained over time, rising from 15% to 30% of healthy volunteer levels in buccal cells and from 16% to 72% in skin cells.
The analyst projects a market introduction of nomlabofusp in 2026, with initial sales estimated at $6 million, potentially reaching $356 million by 2030. The reiterated Buy rating and $15 price target reflect the analyst's confidence in the drug's commercial prospects.
InvestingPro analysis suggests LRMR is currently undervalued, with additional insights available through the comprehensive Pro Research Report, which provides deep-dive analysis of the company's financial health, rated as FAIR with a score of 2.37.
In other recent news, Larimar Therapeutics has experienced a mix of promising and challenging developments.
Despite the occurrence of serious adverse events leading to two patients withdrawing from the study, the company's Friedreich’s ataxia treatment, nomlabofusp, was generally well tolerated in its ongoing open label extension study.
This data update led to a significant decline in Larimar's stock, although analysts from Leerink Partners, Citi, and Guggenheim maintain a positive outlook, emphasizing the encouraging biomarker and functional data, and the company's strong financial standing.
Larimar Therapeutics also disclosed positive initial data indicating that nomlabofusp increased and maintained tissue frataxin levels, showing potential clinical benefits. The company has begun dose escalation to 50 mg daily in six participants and plans to transition adolescents from a pediatric pharmacokinetic study into the open label extension study after safety and pharmacokinetic data assessment.
Larimar ended the third quarter of 2024 with $203.7 million in cash and investments, extending its financial runway into the second quarter of 2026. As the company moves toward the second half of 2025, it aims to submit a Biologics License Application under an accelerated approval pathway, with a focus on four key decisions that will affect their ability to file.
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