BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) has announced encouraging initial results from its ongoing open-label extension (OLE) study evaluating long-term daily injections of nomlabofusp for Friedreich’s ataxia (FA). The findings demonstrate increased tissue frataxin (FXN) levels and early clinical improvement trends, providing a strong foundation for the drug’s continued development.
The OLE study showed that 25 mg of nomlabofusp, administered daily, significantly increased FXN levels in buccal and skin cells over time. By the 90th day of treatment, FXN levels rose from 15% to 30% in buccal cells and from 16% to 72% in skin cells, compared to levels in healthy individuals. The drug was generally well tolerated among the 14 study participants, with safety data supporting ongoing administration.
“Our data show that 25 mg of nomlabofusp administered daily increased and maintained tissue FXN levels over time,” said Dr. Carole Ben-Maimon, President and Chief Executive Officer of Larimar. “Importantly, we are highly encouraged by the early trends towards improvement observed in clinical outcomes that could support the potential for nomlabofusp to benefit a broad spectrum of patients with FA.”
Dose Escalation and Future Plans
Following the positive outcomes in the 25 mg dose group, Larimar has begun escalating to 50 mg daily in six participants. The study will expand this higher dose to all enrolled participants, with data collection continuing into mid-2025.
The company is also advancing its pediatric development program, with dosing for adolescents aged 12-17 set to begin as part of a pharmacokinetic (PK) run-in study in early 2025. Younger children aged 2-11 are expected to enroll later that year. The pediatric studies will transition participants into the OLE study for long-term evaluation.
Further plans include the initiation of a global confirmatory and registration study in mid-2025, with a Biologics License Application (BLA) submission for potential accelerated approval targeted for the second half of the year.
Broadening Understanding of Nomlabofusp
According to Dr. Ben-Maimon, the data collected from multiple studies highlight nomlabofusp’s therapeutic potential. “To date, we have reported data showing increases in FXN across three independent clinical studies, trends towards normalization in gene expression and lipid profiles, and now early trends in clinical outcomes. The totality of data continues to support the therapeutic potential of nomlabofusp,” she said.
Dr. Rusty Clayton, Larimar’s Chief Medical Officer, reaffirmed the drug’s safety profile. “While serious adverse events occurred in two study participants, these issues resolved within 24 hours, and the participants returned to their usual state of health,” he explained. “The long-term safety, PK, and FXN data we are collecting will support a potential accelerated approval using FXN as a novel surrogate endpoint.”
Clinical Outcomes and Future Implications
Early improvements in clinical measures such as the modified Friedreich Ataxia Rating Scale (mFARS) and other functional assessments were observed by Day 90. If sustained, these trends may indicate that nomlabofusp could play a critical role in slowing disease progression.
Dr. Susan Perlman, a principal investigator for the OLE study and Professor of Neurology at UCLA, emphasized the significance of these findings. “Friedreich’s ataxia is caused by frataxin deficiency, and disease progression is more rapid in patients with lower frataxin levels,” she said. “Increases in frataxin levels in patients with FA may lead to the slowing of progression.”
With a strong financial position of $203.7 million in cash and investments as of September 30, 2024, Larimar is well-positioned to advance nomlabofusp toward commercialization while fostering hope for the FA community.
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