BALA CYNWYD, Pa. - Larimar Therapeutics, Inc. (NASDAQ: LRMR), a clinical-stage biotechnology company valued at $393.69 million, announced today initial positive data from a study on nomlabofusp, a potential treatment for Friedreich's ataxia (FA). The ongoing open-label extension (OLE) study showed that daily subcutaneous injections of 25 mg of nomlabofusp were generally well tolerated for up to 260 days.
Participants in the study demonstrated a mean change from baseline in tissue frataxin (FXN) levels, with increases observed in both buccal and skin cells at Day 90. FXN levels are crucial, as FA is characterized by frataxin deficiency. The study noted early trends towards improvement in clinical outcomes, hinting at a broad potential benefit for patients with FA.
The pharmacokinetic data suggest that nomlabofusp plasma levels reached a steady state by Day 30, with no further accumulation following long-term daily administration. 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 OLE study after safety and pharmacokinetic data assessment.
Larimar also announced a strong financial position with $203.7 million in cash and investments as of September 30, 2024, extending its projected financial runway into the second quarter of 2026. According to InvestingPro data, the company maintains a robust current ratio of 13.1, with more cash than debt on its balance sheet. Based on InvestingPro's Fair Value analysis, LRMR stock currently appears fairly valued. Investors seeking deeper insights can access comprehensive financial analysis and 8 additional ProTips through InvestingPro's detailed research reports.
The global confirmatory/registration study for nomlabofusp is slated to commence in mid-2025, with a Biologics License Application (BLA) submission targeted for the second half of 2025 to support potential accelerated approval.
Dr. Carole Ben-Maimon, President and CEO of Larimar, expressed optimism about the therapeutic potential of nomlabofusp based on the totality of data collected. Dr. Rusty Clayton, Chief Medical (TASE:PMCN) Officer, highlighted the general tolerability of the treatment and the plan to increase the dose to 50 mg for all current and newly enrolled participants.
Despite two serious adverse events reported during the OLE study, which resolved within 24 hours, the trial is proceeding as planned. The company also highlighted its ongoing preparation for a global confirmatory study and continued productive discussions with the FDA. With the stock trading at $6.17 and analyst targets ranging from $12.36 to $36, InvestingPro subscribers can access detailed valuation metrics and expert analysis to better evaluate the company's potential. The next earnings announcement is scheduled for March 12, 2025.
This report is based on a press release statement from Larimar Therapeutics.
In other recent news, Larimar Therapeutics has been making significant strides with its lead product candidate, nomlabofusp, aimed at treating Friedreich’s ataxia (FA). The therapy, which targets frataxin deficiency, the root cause of FA, has shown positive results in Phase 1 and Phase 2 studies. H.C. Wainwright reaffirmed its Buy rating for Larimar, forecasting $356 million in sales by 2030. Citi and Leerink Partners also maintained their Buy and Outperform ratings respectively, while Wedbush assigned an Outperform rating, predicting nomlabofusp could generate $1.5 billion in revenue by 2031. Jones Trading and Baird initiated coverage on Larimar with Buy and Outperform ratings, projecting peak unadjusted sales of $1.3 billion by 2031 and a 60% probability of nomlabofusp gaining approval by 2026, respectively. These developments have placed Larimar in a positive light among investors and analysts alike.
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