Larimar Therapeutics (NASDAQ:LRMR) has announced positive initial results from its ongoing open-label extension (OLE) study evaluating daily subcutaneous injections of 25 mg of nomlabofusp, either self-administered or administered by a caregiver, in participants with Friedreich’s ataxia (FA).
FA is a rare, inherited disease that causes damage to the nervous system, leading to a gradual loss of coordination.
In a statement, Carole Ben-Maimon, MD, president, and CEO of Larimar, said, “We are pleased with the advancement of our OLE study that includes 14 patients dosed for up to 260 days. Importantly, 25 mg of nomlabofusp administered daily increased and maintained tissue FXN levels over time, with mean levels increasing from 15% of healthy volunteers at baseline to 30% in buccal cells and from 16% to 72% in skin cells at Day 90.”
“Importantly, we are highly encouraged by the early trends towards improvement observed in clinical outcomes that could support the potential for nomlabofusp administration to result in a clinical benefit across a broad spectrum of patients with FA. To date we have reported data showing increases in FXN in three independent clinical studies, trends towards normalization in gene expression and lipid profiles, and we are now showing early trends in clinical outcomes. Thus, the totality of data continues to support the therapeutic potential of nomlabofusp. We are excited to be increasing the dose to 50 mg nomlabofusp daily for currently enrolled study participants as well as starting newly enrolled participants on 50 mg daily with data for the 50 mg dose expected mid-2025,” Dr. Ben-Maimon added.
Dr. Ben-Maimon continued, “The long-term safety, PK, and FXN data we are collecting in the OLE will be used to support a potential accelerated approval using FXN as a novel surrogate endpoint. Additionally, we are expanding clinical evaluation into adolescents with our recent initiation of our pediatric PK run-in study and expect initial data with the next update in mid-2025. Our global confirmatory and registrational study remains on track to initiate in mid-2025. Our interactions with the FDA continue to be productive and we are focused on our goal of submitting a BLA in the second half of 2025.”
