BALA CYNWYD, PA — The clinical-stage biotech firm Larimar Therapeutics has reported promising top-line data from its Phase 2 dose exploration study. The groundbreaking study tested the medication ‘nomlabofusp’ on patients living with Friedreich’s Ataxia (FA), a debilitating genetic disease.
Nomlabofusp is a new-gen recombinant fusion protein that aims at restoring the deficient levels of frataxin – a crucial protein – in FA patients’ mitochondria. Participants in the study received daily subcutaneous injections of either 25mg or 50mg of nomlabofusp or a placebo. The compelling results showed a dose-dependent surge in frataxin levels within 14 days of treatment – a breakthrough that could potentially decelerate disease progression in FA patients.
The study revealed that, remarkably, all patients in the 50mg cohort achieved frataxin levels in skin cells exceeding 41.25% of the average level found in healthy individuals. The findings also highlighted skin cells’ reliability and reproducibility over buccal cells, leading the company to plan an Open Label Extension (OLE) study for further efficacy evaluation.
Commenting on these results, Dr. Carole Ben-Maimon, President, and CEO of Larimar was bullish on nomlabofusp’s therapeutic potential. She expressed unabridged confidence in this ground-breaking drug, given the clear dose-response and increase in peripheral tissue frataxin level. Dr. Russell Clayton, Chief Medical Officer, commended the drug’s promising safety profile.
Larimar is not stopping with these results – it has already initiated preliminary discussions with the Food and Drug Administration (FDA) regarding the utilization of tissue frataxin levels as a potentially groundbreaking surrogate endpoint. If accepted, this has the potential to significantly expedite the approval process for nomlabofusp – an incredible development for individuals living with FA.
The FDA has acknowledged the critical role of frataxin deficiency in FA’s pathogenic mechanism and the urgent demand for treatments addressing the disease’s underlying physiology. With this endorsement, Larimar plans to submit a Biologics License Application (BLA) for accelerated approval in late 2025, demonstrating their commitment to advancing FA treatment options.
In further impressive updates from Larimar, the company unveiled its solid cash position of $86.8 million, providing them with enough runway to last until the first quarter of 2025. This financial stability allows Larimar to focus on conducting a confirmatory study and accumulating additional nonclinical pharmacology data, with the goal of leveraging their Rare Pediatric Disease designation, Fast Track designation, and Orphan Drug designation awarded to nomlabofusp by the FDA. These designations highlight the potential and significance of Larimar’s innovative pharmaceutical developments, positioning them for future success in the industry.
In summary, Larimar has achieved a groundbreaking leap forward in the treatment of Friedreich’s ataxia (FA), unveiling the remarkable potential of nomlabofusp to elevate frataxin levels and effectively tackle the underlying causes of this debilitating disease. With a clear path to an accelerated approval application and comprehensive plans for further clinical investigations and studies, the future appears exceedingly promising for this transformative and life-altering treatment approach.
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