3. Larimar Therapeutics, Inc. (NASDAQ:LRMR)
1-Year Share Price Performance: ~233%
2-Year Share Price Performance: ~159%
Average Price Target Upside: 182.59%
Larimar Therapeutics, Inc. (NASDAQ:LRMR) is a biotechnology company at the clinical stage, focusing on therapies for rare and complex diseases. Its main candidate, nomlabofusp, is in development to potentially treat Friedreich’s ataxia.
Friedreich’s ataxia is a rare genetic disorder that affects the nervous system and muscles, leading to progressive movement and coordination difficulties. The disorder primarily impacts the spinal cord and peripheral nerves, which help control muscle movement. Symptoms usually appear in childhood or adolescence and may include loss of coordination, muscle weakness, difficulty walking, speech problems, and heart disease. The company also aims to expand its intracellular delivery platform to create fusion proteins for other rare diseases that involve intracellular bioactive compound deficiencies.
Larimar (NASDAQ:LRMR) shared its third-quarter 2024 financial and operational updates, emphasizing the progress of nomlabofusp. The company has activated all trial sites for its open-label extension (OLE) study, where patients receive a daily 25 mg dose of nomlabofusp to assess safety, pharmacokinetics (PK), and frataxin levels. The company expects to present data in December 2024 and provide enrollment updates. Additionally, in November, it will present new findings at the ongoing International Congress for Ataxia Research (ICAR) based on its completed dose exploration studies.
Regulatory developments include receiving the U.K.’s ILAP designation from the MHRA, which could expedite nomlabofusp’s market access in the U.K. Larimar has also begun engagement with the FDA’s START pilot program. The company aims to refine its market strategy by consulting with stakeholders and is on track to submit a Biologics License Application (BLA) in the latter half of 2025 to pursue accelerated approval.
Future plans include a PK run-in study with adolescents by year-end and younger children next year, followed by a larger confirmatory study across the U.S., Europe, and additional locations in mid-2025. As of September 30, 2024, Larimar (NASDAQ:LRMR) held $203.7 million in cash, cash equivalents, and marketable securities, supporting operations into 2026.
On October 31, Citi initiated a “90-day positive catalyst watch” on Larimar (NASDAQ:LRMR), as reported by The Fly. The firm maintained a Buy rating with a $14 price target on the stock. It expects initial data from the open-label extension study of nomlabofusp to be released mid-December.
The data is anticipated to provide valuable information on long-term frataxin concentration kinetics and safety, extending beyond the 28-day dosing from the Phase 2 study. Citi sees potential gains for the stock if the results confirm a strong safety profile, sustained frataxin levels, and early indications of positive treatment outcomes.
