Larimar Therapeutics(LRMR) - 2025 Q4 - Annual Results

Drug Development and Regulatory Plans - Larimar is developing nomlabofusp as the first potential disease-modifying therapy for Friedreich's ataxia (FA), targeting a patient population of approximately 20,000 globally, with around 5,000 in the U.S.[3] - The company plans to submit a Biologics License Application (BLA) for nomlabofusp in Q2 2026, with a U.S. launch targeted for early 2027, following FDA recommendations for accelerated approval based on frataxin levels as a surrogate endpoint[7] - The company has received multiple regulatory designations for nomlabofusp, including Orphan Drug and Fast Track designations in the U.S., and PRIME designation in the EU[7] - The START Pilot Program continues to expedite the clinical and regulatory development of Nomlabofusp, selected as one of 7 novel drug development programs by the FDA[54] - The FDA's new milestone-driven program launched in September 2023 aims to accelerate the development of therapies for rare diseases, providing more frequent and rapid interactions with the FDA[54] - The program is designed to facilitate the development of programs to the pre-BLA meeting stage, ensuring high-quality and reliable data for BLA support[54] Clinical Study Results - Data from a long-term open-label study indicates that 100% of participants at Day 180 had skin frataxin (FXN) levels greater than 50% of healthy volunteers, demonstrating sustained increases over time[11] - The median skin FXN levels increased from 2.70 pg/µg at baseline to 13.44 pg/µg at Day 180, indicating significant improvement in treatment efficacy[12] - Nomlabofusp demonstrated a median improvement of -2.25 in mFARS scores compared to a worsening of 1.00 in the FACOMS reference group after one year of treatment[18] - 10 out of 10 participants in the open-label study achieved skin FXN levels over 50% of median levels in healthy volunteers after 6 months of treatment[20] - The ongoing long-term open-label study has shown consistent directional improvement across four key clinical outcomes after one year of treatment[20] - In a cardiac mouse model, nomlabofusp increased median survival from 98 days (vehicle) to 166 days, demonstrating its potential efficacy[26] Safety and Tolerability - Safety observations from the open-label study showed that the most common adverse events were mild to moderate injection site reactions, with 7 participants experiencing anaphylaxis, all of whom recovered without complications[14] - The dosing regimen for nomlabofusp has been revised to a test dose of 5 mg followed by 25 mg daily for the first 30 days, then increasing to 50 mg daily, aimed at reducing the severity of potential anaphylaxis[15] - Nomlabofusp has shown a long-term safety profile with most adverse events being mild to moderate and not leading to withdrawals[19] - The Phase 2 study was generally well tolerated, with mild to moderate injection site reactions being the most common adverse events[40] Patient Population and Unmet Need - A national survey of clinicians treating FA patients indicated that 98% believe there is a need for treatments targeting frataxin levels directly, highlighting the high unmet medical need in this area[5] - The global Phase 3 study will include 100-150 ambulatory participants aged 2-40 years, focusing on safety and tolerability as well as upright stability and mFARS as primary outcomes[21] - The trial population included a broad representation of adults with Friedreich's Ataxia, with over 50% being non-ambulatory at baseline[48] Intellectual Property and Market Position - Larimar's intellectual property portfolio for nomlabofusp includes patents extending into 2040, providing a strong foundation for market exclusivity upon approval[8] Collaboration and Data Access - Larimar has established a strong relationship with FARA, joining the TRACK-FA Neuroimaging Consortium as an industry partner[57] - FARA provides access to a Global Patient Registry with demographic and clinical information on over 1,000 FA patients, aiding in patient recruitment and education[57] - The TRACK-FA initiative collects natural history data to establish disease-specific neuroimaging biomarkers for potential use in clinical trials[57] - Larimar will have access to all study data from TRACK-FA for regulatory filings as appropriate[57]