Nomlabofusp

Friedreich’s Ataxia News highlighted 2024's top FA research and treatments, including vatiquinone's FDA approval pursuit, gene therapy advancements, and novel treatments like DT-216P2 and PPL-001. Studies showed improvements in FA symptoms, mitochondrial function, and heart defects in mice, aiming for better FA management.

Larimar Therapeutics reported positive initial data from its OLE study on nomlabofusp for Friedreich’s ataxia, showing increased FXN levels and early clinical improvement trends. The study is expanding to a 50 mg dose, with long-term data expected by mid-2025. Plans include a global confirmatory study and FDA discussions for accelerated approval.

Larimar Therapeutics reported initial data from an OLE study on nomlabofusp for FA, showing it's well-tolerated with mild adverse events. FXN levels increased in buccal and skin cells, indicating potential clinical benefits. The study also noted early improvement trends in clinical outcomes. Plans include dose increases and global site evaluations for further studies.

Larimar Therapeutics announced positive initial data from a long-term study of nomlabofusp for Friedreich's Ataxia, showing increased frataxin levels, potentially slowing disease progression. A registrational study is planned for mid-2025, with a Biologics License Application submission in late 2025.

Nomlabofusp treatment modifies gene expression, lipid profiles, and increases frataxin levels in Friedreich’s ataxia patients. Modeling predicts 50 mg daily achieves FXN levels ≥50% of healthy controls. Study participants represent the broad FA population, with consistent relationships between FXN levels and disease characteristics. Nomlabofusp program update expected mid-December 2024.

Nomlabofusp treatment in FA patients modified gene expression, lipid profiles, and increased FXN levels. Modeling predicts 50 mg daily achieves FXN levels ≥50% of healthy controls. Study participants' disease characteristics were representative of the broader FA population. Nomlabofusp program update expected mid-December 2024.

Trump's reelection may boost biotech innovation and M&A activity, potentially repealing the Inflation Reduction Act. Key roles in federal health agencies will influence drug pricing reforms. The Zacks Medical-Drugs industry, despite headwinds, shows promising trends with innovation in diabetes/obesity, inflammation, and neuroscience. Small drugmakers rely on collaboration partners and R&D funding, with notable stocks including Corcept Therapeutics, Madrigal Pharmaceuticals, Catalyst Pharmaceuticals, Theravance Biopharma, and Larimar Therapeutics.

Larimar Therapeutics reported Q3 2024 net loss of $15.5 million ($0.24 per share) with $203.7 million in cash, projecting runway into 2026. Their lead program, nomlabofusp for Friedreich's ataxia, advances with milestones: program update in mid-December 2024, PK run-in study in adolescents by year-end, global confirmatory study mid-2025, and BLA submission targeted for 2H 2025. The company received ILAP designation from MHRA for accelerated UK market access.