BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) announced this week that the U.S. Food and Drug Administration (FDA) has removed the partial clinical hold on its nomlabofusp (CTI-1601) clinical program. Nomlabofusp is being developed as a treatment for Friedreich’s Ataxia (FA), a rare genetic disorder affecting the nervous system and movement.
The FDA’s decision follows a review of data from Larimar’s recently completed Phase 2 study. This study explored doses of 25 mg and 50 mg, where patients received daily doses for 14 days, followed by every-other-day dosing until day 28. The FDA’s removal of the partial hold clears the way for Larimar to escalate dosing in its ongoing open label extension (OLE) study, which initially evaluates the 25 mg dose.
“We are very excited the FDA has removed the partial clinical hold on our nomlabofusp program following review of our Phase 2 data,” said Carole Ben-Maimon, MD, President and CEO of Larimar. “Helping patients with FA is our top priority and we appreciate the attention and thorough review by the FDA of all submitted data.”
Nomlabofusp is a novel protein replacement therapy designed to deliver frataxin, a crucial protein, to mitochondria. Mitochondria are the energy powerhouses of cells, and a deficiency in frataxin leads to the debilitating symptoms seen in FA patients. The Phase 2 study showed that nomlabofusp was generally well-tolerated and demonstrated predictable pharmacokinetics. Importantly, it increased frataxin levels in skin and buccal cells, with some patients reaching over 50% of the frataxin levels observed in healthy individuals.
This development holds significant implications for patients with Friedreich’s Ataxia. Increasing frataxin levels could potentially slow or reverse disease progression, offering hope for improved quality of life. The ability to escalate doses in the OLE study will allow Larimar to further test the efficacy and safety of higher doses, which could lead to more robust treatment outcomes.
The ongoing OLE study will continue to evaluate the long-term safety and effectiveness of nomlabofusp at the 25 mg dose. Larimar plans to increase the dose to 50 mg after further evaluation. This step is critical to understanding how the body processes and responds to frataxin delivered via nomlabofusp. The interim data from this study, expected in the fourth quarter of 2024, will be closely watched by both the medical community and investors.
Larimar aims to submit a Biologics License Application (BLA) for nomlabofusp in the second half of 2025. This timeline indicates the company’s confidence in their therapeutic approach and the FDA’s supportive stance following the positive Phase 2 results.
The removal of the clinical hold not only accelerates Larimar’s development timeline but also boosts investor confidence. It signals regulatory alignment and enhances the credibility of Larimar’s research and development efforts. As a result, Larimar is poised to make significant strides in addressing unmet medical needs in FA.
For patients and families affected by Friedreich’s Ataxia, this news brings renewed hope. If successful, nomlabofusp could transform the treatment landscape for this challenging condition, providing a much-needed option for managing and potentially improving patient outcomes.
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