Larimar Therapeutics Publishes Cross-Species Findings Supporting Skin as a Surrogate for the Measurement of Frataxin in Tissues Clinically Relevant to Friedreich’s Ataxia
Rhea-AI Summary
Larimar Therapeutics (Nasdaq: LRMR) published a peer-reviewed article reporting that treatment with nomlabofusp raised frataxin (FXN) levels across mice, rats, non-human primates and in patients with Friedreich’s ataxia (FA).
Measured FXN increases in accessible peripheral tissues (skin, buccal cells) correlated with levels in clinically relevant organs; these data were reviewed by the FDA in support of the potential use of skin FXN as a reasonably likely surrogate endpoint. Larimar plans a BLA submission in June 2026 and to pursue accelerated approval.
Positive
- Cross-species FXN increases observed in mice, rats, and non-human primates
- Correlation between skin/buccal FXN and organ FXN in patients with FA
- FDA reviewed data supporting potential use of skin FXN as an RLSE
- BLA submission planned for June 2026
Negative
- Regulatory acceptance of skin FXN as a surrogate endpoint is not finalized
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BALA CYNWYD, Pa., April 30, 2026 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced the publication entitled “Nomlabofusp Treatment Produces Frataxin Levels That Correlate Across Peripheral Tissues: Preclinical and Clinical Support for Surrogate Tissue Sampling” in the journal Clinical and Translational Science, an official peer-reviewed publication of the American Society for Clinical Pharmacology and Therapeutics (ASCPT). The article is now available online (link).
Nonclinical data across mice, rats, and non-human primates consistently showed that treatment with nomlabofusp increases frataxin (FXN) levels in tissues clinically relevant to Friedreich’s ataxia (FA). These increases correlated with levels observed in accessible peripheral tissues such as skin and buccal cells. Additionally, correlations between the FXN increases in these two peripheral tissues were also observed in patients with FA after administration of nomlabofusp. These data were part of the package reviewed by the U.S. Food and Drug Administration (FDA) in support of the potential use of skin FXN concentrations as a reasonably likely surrogate endpoint (RLSE) for Larimar’s registrational program, which Larimar plans to pursue under the accelerated approval pathway.
“We are pleased to publish these findings that further reinforce nomlabofusp’s mechanism of action and ability to restore FXN levels in clinically-relevant tissues. Importantly, our studies show that after treatment, the increased levels in accessible tissues like skin and buccal cells reflect levels achieved in critical organs across mice, rats, and non-human primates. Correlations in FXN levels between peripheral tissues has also been shown in patients with FA,” said Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar. “The consistency seen across species further strengthens the translational relevance of our program, supports our planned accelerated approval pathway, and underpins our Biologics License Application (BLA) submission planned for June 2026. We are focused on continued execution across our near-term registrational milestones and advancing nomlabofusp as the first potential disease-modifying therapy for patients with FA.”
About Larimar Therapeutics
Larimar 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 Statements
This 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 any 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 the timing of the BLA submission, the expectations of the timing of, and potential for, accelerated approval or accelerated access, time to launch and market and overall development plans 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 milestones and continued interactions with the FDA and Larimar’s ability to timely implement the revised dosing regimen in its clinical program for nomlabofusp; 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; Larimar’s ability to realize the benefits of Breakthrough Therapy Designation; 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 at www.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.
| Investor Contact: Joyce Allaire LifeSci Advisors jallaire@lifesciadvisors.com (212) 915-2569 | Company Contact: Michael Celano Chief Financial Officer mcelano@larimartx.com (484) 414-2715 |
