Larimar Therapeutics Advances Nomlabofusp for Friedreich's Ataxia; Reports Q3 2024 Results
核心洞察
Larimar Therapeutics reported a Q3 2024 net loss of $15.5 million, with a strong cash position of $203.7 million, expected to last into 2026.
The company's lead program, nomlabofusp for Friedreich's ataxia (搜索), is progressing with a program update expected in mid-December 2024.
Larimar plans to initiate a PK run-in study in adolescents by the end of 2024 and a global confirmatory study by mid-2025, targeting BLA submission in 2H 2025.
Larimar Therapeutics, Inc. (Nasdaq: LRMR) has announced its third-quarter 2024 financial results and provided an update on the development of nomlabofusp, its lead compound for the treatment of Friedreich's ataxia (搜索) (FA). The company reported a net loss of $15.5 million, or $0.24 per share, for the third quarter of 2024, compared to a net loss of $9.1 million, or $0.21 per share, for the third quarter of 2023. Despite the increased net loss, Larimar maintains a strong financial position with $203.7 million in cash, cash equivalents, and marketable securities as of September 30, 2024, which is projected to provide a cash runway into 2026.
The increased net loss reflects significant R&D investment scaling, with R&D expenses for the third quarter of 2024 totaling $13.9 million, compared to $6.6 million for the third quarter of 2023. This increase is primarily attributed to nomlabofusp manufacturing costs, personnel expenses, assay development costs, and clinical costs associated with the ongoing open-label extension (OLE) study. General and administrative expenses also increased to $4.3 million in the third quarter of 2024, compared to $3.8 million in the third quarter of 2023, driven by personnel expenses and professional fees related to commercial activity.
Nomlabofusp Development Program
Larimar's lead program, nomlabofusp, is being developed as a potential frataxin (搜索) protein replacement therapy for patients with FA. All sites are activated with ongoing enrollment in the OLE study, evaluating the long-term safety, pharmacokinetics (PK), and frataxin levels in patients with FA following daily subcutaneous administration.
According to Dr. Carole Ben-Maimon, President and CEO of Larimar, the company plans to provide a development program update in mid-December 2024, including available safety, PK, and frataxin (搜索) data from patients receiving 25 mg of nomlabofusp daily for up to 180 days in the OLE study. An update on enrollment in the OLE study will also be provided.
Larimar is on track to initiate a PK run-in study in adolescents (12 to 17 years of age) with FA by the end of 2024, followed by a second cohort of children (2 to 11 years of age) next year. The company is also planning to initiate a global confirmatory/registration study by mid-2025, with potential sites in the U.S., Europe, U.K., Canada, and Australia. Larimar continues to target Biologics License Application (BLA) submission for nomlabofusp in the second half of 2025 to support potential accelerated approval.
Regulatory Milestones
Larimar recently received Innovative Licensing and Access Pathway (ILAP) designation from the Medicines and Healthcare Products Regulatory Agency (MHRA) for nomlabofusp for the treatment of adults and children with FA. ILAP aims to facilitate patient access to novel treatments by accelerating time to market through enhanced engagements with U.K. regulatory authorities and other stakeholders. Nomlabofusp has also been granted orphan drug designations in the U.S. and the European Union (EU), Fast Track and Rare Pediatric Disease designations in the U.S., PRIME designation in the EU, and was selected for the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program by the FDA.
Upcoming Presentations
Data from Larimar’s nomlabofusp Phase 1 studies and Phase 2 dose exploration study will be presented at the International Congress for Ataxia Research (ICAR) meeting being held November 12-15, 2024, in London, U.K. These presentations will include new data on results from the completed dose exploration study, including exploratory gene and lipid expression results following nomlabofusp treatment, as well as data from patients participating in the Phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) studies and Phase 2 dose exploration study.