Larimar Therapeutics Q3 2024 Financial and Operating Results

Larimar Therapeutics, Inc., a clinical-stage biotechnology company, revealed its performance and progress for the third quarter of 2024. The company, which concentrates on developing therapies for rare, complex diseases, is particularly focused on advancing its nomlabofusp program, a potential treatment for Friedreich’s ataxia (FA), a rare genetic disorder.

The nomlabofusp program is making significant strides and might become the first treatment to replace frataxin protein in FA patients. Enrollment is ongoing in the open-label extension (OLE) study, which is evaluating the long-term safety, pharmacokinetics (PK), and frataxin levels in FA patients administered with daily subcutaneous doses of nomlabofusp. Larimar plans to release an update on the program in mid-December, providing available safety, PK, and frataxin data from patients who have been receiving a daily 25 mg dose of the treatment for up to 180 days. Additionally, an update on the study's enrollment will be provided.

The company is preparing to present new data at the upcoming International Congress for Ataxia Research (ICAR) in November. This data includes results from their completed dose exploration study, which examines exploratory gene and lipid expression following nomlabofusp treatment. Two posters will also be presented, one detailing baseline disease characteristics and baseline tissue frataxin levels in patients, and the other illustrating the relationship between dose, PK, and tissue frataxin levels using modeling and simulation.

Larimar is on track to begin a PK run-in study in adolescents with FA by the end of this year, marking the first step towards evaluating nomlabofusp in pediatric patients. This study is crucial for assessing the treatment's safety and exposure in younger patients. The initiation of a global confirmatory/registration study is planned for mid-2025, with potential sites in the U.S., Europe, U.K., Canada, and Australia. The company aims to submit a Biologics License Application (BLA) for nomlabofusp in the second half of 2025 to support a potential accelerated approval.

The company achieved a significant milestone by receiving the Innovative Licensing and Access Pathway (ILAP) designation from the Medicines and Healthcare Products Regulatory Agency (MHRA). This designation is intended to expedite patient access to novel treatments in the U.K. Nomlabofusp has also received orphan drug designations in the U.S. and EU, Fast Track and Rare Pediatric Disease designations in the U.S., PRIME designation in the EU, and has been selected for the START pilot program by the FDA. These designations highlight the therapeutic potential of nomlabofusp and Larimar's commitment to meeting the unmet needs of FA patients.

Financially, Larimar reported having cash, cash equivalents, and marketable securities totaling $203.7 million as of September 30, 2024, providing a projected cash runway into 2026. For the third quarter of 2024, the company recorded a net loss of $15.5 million, or $0.24 per share, compared to a net loss of $9.1 million, or $0.21 per share, for the same period in 2023. Research and development expenses increased significantly, driven by higher manufacturing costs for nomlabofusp, increased personnel expenses, and costs associated with the OLE study and other clinical trials.

For the nine months ending September 30, 2024, Larimar reported a net loss of $51.8 million, or $0.86 per share, compared to a net loss of $24.0 million, or $0.55 per share, for the same period in 2023. The increase in research and development expenses was primarily due to higher manufacturing costs for nomlabofusp, increased personnel expenses, clinical costs associated with ongoing studies, and other related expenses.

General and administrative expenses also saw an increase, mainly due to higher personnel expenses and professional fees. Despite the financial losses, Larimar remains focused on advancing its nomlabofusp program and achieving its milestones to bring this potentially life-changing therapy to FA patients.