AMT-130: Robust 3‑Year Clinical Efficacy
Phase I/II 3‑year data demonstrated a statistically significant 75% slowing of disease progression on the composite UHDRS and a 60% slowing on Total Functional Capacity versus an external Enroll HD comparator; reduction in neurofilament light and supportive trends across other clinically meaningful endpoints. Company plans a 4‑year analysis in Q3 2026 and intends to amend the SAP and submit the analysis to regulators.
AMT-191 (Fabry): Durable Enzyme Activity and ERT Withdrawal
Preliminary Phase I/II data from 11 patients showed dose‑dependent elevation in alpha‑Gal A enzyme activity durable from 4 months up to >1 year; as of the call all 11 patients have been withdrawn from enzyme replacement therapy.
AMT-260 (Temporal Lobe Epilepsy): Early Promising Signals
Case study of first patient with up to 6 months follow‑up showed a promising reduction in seizure frequency and no serious adverse events; enrollment completed for five additional patients in cohort 1 (six total) with cohort 2 enrollment underway and a planned Q2 update on six patients with ≥6 months follow‑up.
Strong Cash Position and Capital Raise
Cash, cash equivalents and investment securities totaled $622.5 million at Dec 31, 2025 vs $367.5 million at Dec 31, 2024, an increase of ~$255.0 million (+69.4%), primarily from ~$404.2 million in proceeds from public offerings and prefunded loans; company expects cash to fund operations into the second half of 2026.
Operational Efficiency from Divestiture
Cost of contract manufacturing revenues was nil in 2025 versus $17.1 million in 2024 following the 2024 divestiture of the Lexington manufacturing facility; R&D 'other' costs decreased by $26.0 million driven by lower employee/contractor/severance and facility costs.
Regulatory Engagement and Global Pathway Strategy
Company is actively engaging the FDA (Type B planned for Q2 2026), pursuing dialogue with ex‑U.S. regulators (MHRA, EMA and others), and evaluating named‑patient/early access programs and ex‑U.S. regulatory pathways to accelerate patient access.
Strong Community and KOL Support
Broad advocacy from the Huntington's disease community and positive feedback from neurosurgeons, neurologists and HD centers of excellence reinforce clinical interest and urgency for regulatory flexibility and access.