Fed Governor Adriana Kugler to resign
BALA CYNWYD, Pa. - Larimar Therapeutics, Inc. (NASDAQ: LRMR), a biotechnology company focused on treatments for rare diseases, reported progress in discussions with the FDA regarding its leading drug candidate, nomlabofusp, for Friedreich’s ataxia (FA). According to InvestingPro data, the company’s stock has seen significant volatility, trading 77% below its analyst consensus target of $24.25, suggesting potential upside if regulatory milestones are met. The FDA has indicated a willingness to consider skin frataxin (FXN) concentrations as a surrogate endpoint for accelerated approval, a key development for the company’s planned Biologics License Application (BLA) submission by the end of 2025.
The FDA’s receptiveness to using FXN concentration as a reasonably likely surrogate endpoint (RSLE) emerged from the agency’s written correspondence in the context of the START pilot program. The regulator also acknowledged the relationship between increased FXN concentrations in skin cells and benefits in heart, dorsal root ganglia, and skeletal muscle tissues, based on data submitted by Larimar.
Larimar’s ongoing open label extension (OLE) study, which involves participants receiving a 50 mg dose of nomlabofusp, is expected to yield additional data in September 2025. The company has also amended the OLE protocol to include premedication aimed at reducing allergic reactions, following the identification of anaphylaxis as a likely adverse drug reaction to nomlabofusp.
In parallel, Larimar has initiated dosing in a pediatric pharmacokinetic (PK) study and plans to enroll children aged 2-11 in the clinical program. The adolescent cohort of this study is anticipated to be completed by the end of March 2025, with results expected in September.
The FDA has accepted a transition to a lyophilized form of nomlabofusp, which Larimar aims to commercialize. Feedback from both the FDA and the European Medicines Agency (EMA) has been obtained on the global Phase 3 study protocol, with the trial expected to commence in mid-2025.
Financially, Larimar reported a strong position with $183.5 million in cash, cash equivalents, and marketable securities as of December 31, 2024. The company’s net loss for the fourth quarter of 2024 was $28.8 million, with research and development expenses reflecting increased investment in nomlabofusp manufacturing and development. InvestingPro analysis reveals a robust current ratio of 13.1, indicating strong short-term liquidity, though the company is burning through cash rapidly. For deeper insights into Larimar’s financial health and detailed metrics, investors can access the comprehensive Pro Research Report, available exclusively on InvestingPro.
Larimar’s focus remains on advancing nomlabofusp as a potential first disease-modifying therapy for FA, with a projected cash runway into the second quarter of 2026. With an overall Financial Health score of "FAIR" from InvestingPro and strong analyst support (consensus "Strong Buy"), the company appears positioned to execute its development plans. The information in this article is based on a press release statement from Larimar Therapeutics. Subscribers to InvestingPro can access 8 additional ProTips and over 30 financial metrics for comprehensive investment analysis.
In other recent news, Larimar Therapeutics has taken steps to align executive compensation with company performance by issuing performance-based restricted stock units (PSUs) to its top executives. President and CEO Carole S. Ben-Maimon received 100,000 PSUs, while CFO Michael Celano was awarded 50,000 PSUs. Chief Development Officer Gopi Shankar and Chief Medical Officer Russell G. Clayton each received 25,000 PSUs. These awards are contingent on the company achieving specific regulatory milestones, reflecting a strategy to incentivize executives in line with shareholder interests. Meanwhile, JMP Securities has maintained a Market Outperform rating on Larimar Therapeutics, with a price target of $21.00. The firm’s positive outlook is driven by Larimar’s progress toward a Biologics License Application submission for its drug candidate, nomlabofusp, expected later this year. Larimar has begun dosing pediatric patients with nomlabofusp, which is seen as an indicator of the FDA’s comfort with the drug’s safety profile. The analyst from JMP Securities noted that upcoming long-term data from adult and adolescent trials could significantly impact the company’s stock.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.