Bitcoin price today: dips below $112k, near 6-wk low despite Fed cut bets
BALA CYNWYD, Pa. - Larimar Therapeutics, Inc. (NASDAQ:LRMR), a clinical-stage biopharmaceutical company with a market capitalization of $166 million, announced Monday it has received safety database recommendations from the U.S. Food and Drug Administration (FDA) for its Friedreich’s ataxia drug candidate nomlabofusp, refining its timeline for Biologics License Application (BLA) submission. According to InvestingPro data, the company maintains a strong liquidity position with a current ratio of 7.48, though it faces significant cash burn as it advances its clinical programs.
The company now plans to submit its BLA seeking accelerated approval in the second quarter of 2026, allowing time to include recommended safety data from both adults and children. The FDA advised Larimar to evaluate safety in at least 30 participants with continuous exposure for 6 months, including a subset of at least 10 with 1-year exposure, with the majority of data coming from participants receiving the 50 mg dose. Despite recent market volatility that has seen the stock decline 10% in the past week, analysts maintain a strong bullish consensus with price targets ranging from $10 to $40.
The FDA has indicated openness to using skin frataxin (FXN) concentrations as a reasonably likely surrogate endpoint for approval, according to the company’s press release statement.
Larimar expects to report data in September 2025 from its open label extension (OLE) study, which will include 30-40 participants who received at least one dose of nomlabofusp, including those on the 50 mg dose. Adolescent pharmacokinetic run-in data from 14 participants is also expected at that time.
The company reported that some participants in the OLE study have now been on treatment for up to 15 months at both 25 mg and 50 mg doses. The study has been expanded to include adolescents and patients who have not previously participated in Larimar’s clinical trials.
Preparations for a global Phase 3 study are underway with site identification and qualification ongoing in the U.S., Europe, U.K., Canada, and Australia. This study is expected to serve as the confirmatory trial required under the FDA’s accelerated approval pathway.
Nomlabofusp is being developed as a potential first disease-modifying therapy for Friedreich’s ataxia, a rare genetic disease.
In other recent news, Larimar Therapeutics has been actively advancing its drug candidate, nomlabofusp, with significant developments in its regulatory and clinical journey. The company reported a net loss of $28.8 million for the fourth quarter of 2024, while maintaining a strong cash position of $183.5 million as of December 31, 2024. Larimar is preparing for a Biologics License Application (BLA) submission by the end of 2025, with the FDA showing openness to using skin frataxin concentrations as a surrogate endpoint for accelerated approval. Analysts from Citizens JMP, Jones Trading, H.C. Wainwright, and Citi have all reiterated their positive ratings for Larimar, with price targets ranging from $14 to $21, reflecting confidence in the company’s prospects.
The FDA’s feedback has been crucial, as it recognizes the correlation between skin frataxin levels and critical tissues, which could pave the way for nomlabofusp’s approval. Larimar plans to release data from its ongoing open-label extension study in September 2025, which will provide further insights into the drug’s efficacy. The company has also initiated a pediatric study, with results expected in September 2025. Despite a recent challenge involving a drug-related anaphylaxis signal, Citi and other firms remain optimistic due to the positive regulatory developments.
Larimar’s strategic focus includes a confirmatory Phase 3 study set to begin mid-2025, aiming to enroll 100-150 patients. The company has received various regulatory designations for nomlabofusp, underscoring its potential as a treatment for Friedreich’s ataxia. As Larimar progresses through these critical milestones, investors and stakeholders are closely monitoring the developments, which could significantly impact the company’s future trajectory.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.