Jenny Leigh Gonzalez-Armenta

Jenny Gonzalez-Armenta from Leerink Partners, on behalf of Joseph Schwartz, inquired about the AMT-130 surgical procedure from a patient's perspective, the associated time commitment, and the likely commercial patient population, including potential early adopters.

Answer

Chief Medical Officer Walid Abi-Saab described the MRI-guided neurosurgical procedure as 'low complexity' for experienced surgeons, with patients typically discharged within 24 hours and recovering quickly. CEO Matt Kapusta added that with no other disease-modifying therapies available, he expects the overwhelming majority of eligible Stage 2 and 3 patients to seriously consider the treatment.

Jenny Leigh Gonzalez-Armenta, on for Joseph Schwartz, asked about the new CBER head's views on patient advocacy groups, uniQure's relationship with the Huntington's Disease Society of America, and the general sentiment of the HD community.

Answer

CMO Dr. Walid Abi-Saab emphasized uniQure's long and productive history with patient advocacy groups like CHDI, stating their work and sophisticated natural history data have been essential for the field. He described these organizations as extremely balanced, well-respected by the FDA, and crucial partners, noting that without them, uniQure and others would not be where they are today.

Jenny Leigh Gonzalez-Armenta from Leerink Partners asked about the potential benefits of exceeding the 11 micromolar AAT protein threshold in the Alpha-1 program and the key differentiators between RNA and DNA editing from a patient's perspective.

Answer

President and CEO Dr. Paul Bolno stated that exceeding the 11 micromolar threshold could offer additional benefits, such as faster clearance of liver aggregates, and that upcoming multi-dose data will be crucial. He contrasted RNA editing with DNA editing by highlighting the risks of bystander edits and misfolded proteins with DNA-based approaches. He also noted a key patient-centric advantage: Wave's GalNAc-conjugated WVE-006 avoids the need for LNP delivery, which carries a risk of hepatic injury, a significant concern for patients with liver disease.