Advancement of AMT-130 for Huntington's Disease
AMT-130 has received breakthrough therapy designation from the FDA, indicating its potential as the first disease-modifying therapy for Huntington's disease. The company achieved regulatory alignment with the FDA on the statistical analysis plan and CMC requirements for a planned BLA submission in early 2026.
Encouraging Data for AMT-260 in Epilepsy
The first patient treated with AMT-260 for mesial temporal lobe epilepsy experienced a 92% reduction in seizure frequency over five months with no significant adverse events.
Strong Financial Position
Cash, cash equivalents, and investment securities totaled $377 million as of June 30, 2025, providing a solid foundation to fund operations into the second half of 2027.
Progress in Broader Clinical Pipeline
Advancements were made in clinical programs for AMT-191 for Fabry disease and AMT-162 for SOD1-ALS, with initial data expected in September 2025 and first half of 2026, respectively.