Larimar Therapeutics Chosen for FDA START Pilot with Nomlabofusp in Friedreich’s Ataxia

Larimar Therapeutics, Inc. has announced that the FDA has chosen its novel therapy, nomlabofusp, to participate in the START pilot program. This initiative, known as the Support for Clinical Trials Advancing Rare Disease Therapeutics (START), aims to expedite the development of innovative treatments for rare diseases by enhancing communication between developers and the FDA.

Nomlabofusp is a protein replacement therapy designed to tackle Friedreich’s ataxia (FA), a rare neurodegenerative disease. The therapy works by delivering frataxin, a protein that FA patients lack, directly to the mitochondria. The FDA selected nomlabofusp for this program due to its potential to meet significant unmet medical needs and its readiness for development. The selection criteria also included the alignment of chemistry, manufacturing, and controls (CMC) development timelines with clinical plans, as well as a proposed plan to accelerate communication and pivotal study initiation, ultimately aiming for a Biologics License Application (BLA) submission.

Launched in September 2023, the START pilot program seeks to speed up the development of up to six novel drugs that address rare or serious conditions with unmet medical needs. The program was divided equally between the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER), each responsible for three selections. Being part of START allows sponsors to have more frequent and rapid interactions with the FDA, facilitating quicker progress to pivotal clinical studies or pre-BLA stages and generating reliable data for BLA or New Drug Application (NDA) submissions.

Dr. Carole Ben-Maimon, President and CEO of Larimar, expressed enthusiasm about nomlabofusp's inclusion in the START program. She emphasized that this selection underscores the FDA's commitment to accelerating the pace of drug development through enhanced communication mechanisms. Dr. Russell Clayton, Larimar's Chief Medical Officer, highlighted the importance of increased FDA communication in expediting the nomlabofusp development program to the pre-BLA stage, offering hope to patients with FA.

Nomlabofusp is currently undergoing an open-label extension (OLE) study to evaluate its long-term safety, tolerability, pharmacokinetics, and frataxin levels in peripheral tissues of FA patients. Interim results from this study are anticipated in the fourth quarter of 2024.

Nomlabofusp has received several designations from regulatory bodies, including Rare Pediatric Disease and Orphan Drug designations from the FDA and Orphan Drug Designation from the European Commission. Additionally, it has been granted a PRIME designation by the European Medicines Agency, recognizing its potential to provide significant benefits for patients with FA.

Larimar Therapeutics is a clinical-stage biotechnology company focusing on developing treatments for rare and complex diseases. Their lead compound, nomlabofusp, also known as CTI-1601, aims to treat Friedreich's ataxia. The company plans to leverage its intracellular delivery platform to develop other fusion proteins targeting rare diseases caused by intracellular bioactive compound deficiencies.

In conclusion, the inclusion of nomlabofusp in the START pilot program represents a significant milestone for Larimar Therapeutics. This FDA initiative is expected to enhance the development efficiency of nomlabofusp, potentially bringing a much-needed treatment closer to patients suffering from Friedreich’s ataxia.