Full Press Release Details
BALA CYNWYD, Pa., June 23, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced FDA safety database recommendations and refined timeline for Biologics License Application (BLA) submission to allow for the inclusion of the recommended safety data from adults and children with Friedreich’s Ataxia (FA). This comes following written responses from the U.S. Food and Drug Administration (FDA) based on discussions under the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program.
“We are thrilled to have clarity from FDA on the safety database recommendations following submission of safety information included in a briefing package from our nomlabofusp program. Importantly, we now have written recommendations from FDA on critical elements of the BLA submission including the safety database as well as the use of skin frataxin (FXN) concentrations as a reasonably likely surrogate endpoint (RLSE). Enrollment in our open label extension (OLE) study continues to progress and we have recently expanded enrollment to include adolescents and patients who have not participated in prior clinical studies and therefore have not been exposed to nomlabofusp previously. Based on the FDA’s safety database recommendations and our plan to request approval to treat a broad population of patients including adults and children, we now plan to submit our BLA seeking accelerated approval in the second quarter of 2026,” said Carole Ben-Maimon, MD, President, and Chief Executive Officer of Larimar. “Our participation in the START program has been incredibly valuable and continues to help us expedite clinical and regulatory development for the nomlabofusp program. We are on track to report data in September 2025 including data on the 50 mg dose from our OLE study, as well as adolescent pharmacokinetic (PK) run-in data. Nomlabofusp has the potential to be the first disease modifying therapy for FA and we look forward to expanding the clinical program to patients around the world with the initiation of our global Phase 3 study.”
Dr. Rusty Clayton, Chief Medical Officer of Larimar added, “Our long-term OLE study is further advancing, with some participants now on treatment for up to 15 months. This includes exposure at both the 25 mg and 50 mg doses. The high adherence rates we are seeing for daily subcutaneous injections in participants over the long term is very encouraging. We have begun transitioning adolescents from the PK run-in study and have amended the protocol to include patients who have never participated in any of our prior clinical trials. Overall, we are pleased with our progress and the recommendations we now have from FDA on the safety database to achieve our near-term registrational goals.”
Clear FDA Expectations for Path to Submission of Nomlabofusp BLA Seeking Accelerated Approval
Near-term Milestones
Conference Call and WebcastLarimar will host a conference call and webcast today, June 23, 2025, at 8:00 a.m. ET. To access the webcast, please visit thislinkto the event. To participate by phone, please dial 1-877-407-9716 (domestic) or 1-201-493-6779 (international) and refer to conference ID 13754491 or click on thislinkand request a return call. Following the live event, an archived webcast will be available on the“Events & Presentations”page of the Larimar website.
About Larimar TherapeuticsLarimar Therapeutics, Inc. (Nasdaq: LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. Larimar’s lead compound, nomlabofusp, is being developed as a potential treatment for Friedreich's ataxia. Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds. For more information, please visit:https://larimartx.com.
Forward-Looking StatementsThis press release contains forward-looking statements that are based on Larimar’s management’s beliefs and assumptions and on information currently available to management. All statements contained in this release other than statements of historical fact are forward-looking statements, including but not limited to statements regarding Larimar’s ability to develop and commercialize nomlabofusp and other planned product candidates, Larimar’s planned research and development efforts, including the timing of its nomlabofusp clinical trials, interactions and filings with the FDA, expectations regarding potential for accelerated approval or accelerated access and time to market and overall development plan and other matters regarding Larimar’s business strategies, ability to raise capital, use of capital, results of operations and financial position, and plans and objectives for future operations.
In some cases, you can identify forward-looking statements by the words “may,” “will,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “ongoing” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. These statements involve risks, uncertainties and other factors that may cause actual results, performance, or achievements to be materially different from the information expressed or implied by these forward-looking statements. These risks, uncertainties and other factors include, among others, the success, cost and timing of Larimar’s product development activities, nonclinical studies and clinical trials, including nomlabofusp clinical and regulatory milestones and continued interactions with the FDA; that preliminary clinical trial results may differ from final clinical trial results, that earlier non-clinical and clinical data and testing of nomlabofusp may not be predictive of the results or success of later clinical trials, and assessments; that the FDA may not ultimately agree with Larimar’s nomlabofusp development strategy; the potential impact of public health crises on Larimar’s future clinical trials, manufacturing, regulatory, nonclinical study timelines and operations, and general economic conditions; Larimar’s ability and the ability of third-party manufacturers Larimar engages, to optimize and scale nomlabofusp’s manufacturing process; Larimar’s ability to obtain regulatory approvals for nomlabofusp and future product candidates; Larimar’s ability to develop sales and marketing capabilities, whether alone or with potential future collaborators, and to successfully commercialize any approved product candidates; Larimar’s ability to raise the necessary capital to conduct its product development activities; and other risks described in the filings made by Larimar with the Securities and Exchange Commission (SEC), including but not limited to Larimar’s periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available atwww.sec.gov. These forward-looking statements are based on a combination of facts and factors currently known by Larimar and its projections of the future, about which it cannot be certain. As a result, the forward-looking statements may not prove to be accurate. The forward-looking statements in this press release represent Larimar’s management’s views only as of the date hereof. Larimar undertakes no obligation to update any forward-looking statements for any reason, except as required by law.