Larimar Therapeutics is making strides in the development of nomlabofusp, a potential first-in-class frataxin protein replacement therapy for Friedreich's ataxia (FA). The company plans to provide a comprehensive program update in mid-December, including crucial safety, pharmacokinetic (PK), and frataxin data from patients in the open-label extension (OLE) study receiving a daily 25 mg dose of nomlabofusp for 30 to 180 days.
Clinical Development Milestones
The OLE study is currently enrolling patients across multiple sites to assess the long-term safety, PK, and frataxin levels following subcutaneous administration of nomlabofusp. Carole Ben-Maimon, MD, President and CEO of Larimar, highlighted the ongoing progress and the anticipation of presenting new data at the International Congress for Ataxia Research (ICAR) meeting in November. This data will include results from the completed dose exploration study, featuring exploratory gene and lipid expression results after nomlabofusp treatment.
Further presentations at ICAR will cover baseline disease characteristics, tissue frataxin levels, and the relationship between dose, PK, and tissue frataxin levels, utilizing modeling and simulation techniques. The company remains on track to initiate a PK run-in study in adolescents with FA by the end of 2024, with a subsequent study planned for younger children in 2025. Participants in the PK run-in study will be randomized 2:1 to receive either nomlabofusp or placebo daily, with eligible participants then able to screen for the OLE study.
Regulatory and Commercial Strategy
Larimar has received Innovative Licensing and Access Pathway (ILAP) designation from the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K., aiming to expedite patient access to nomlabofusp. The company also held initial meetings with the FDA as part of the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. These interactions are designed to refine the development program and address unmet needs in the FA therapeutic landscape.
In parallel with clinical and regulatory advancements, Larimar is scaling up manufacturing efforts and collecting data to support a potential accelerated approval path, with a Biologics License Application (BLA) submission targeted for the second half of 2025. A global confirmatory/registration study is planned for mid-2025, including potential sites in the U.S., Europe, U.K., Canada, and Australia.
Financial Position
As of September 30, 2024, Larimar reported a strong financial position with $203.7 million in cash, cash equivalents, and marketable securities, providing a projected cash runway into 2026. This financial stability supports the ongoing and planned clinical trials, manufacturing scale-up, and regulatory activities necessary for the development and potential commercialization of nomlabofusp.
