Cabaletta: Interim Phase II data

Interim data from 25 patients with OPMD in the open-label, international Phase II HOPEMD trial showed that once-weekly 30 g IV Cabaletta led to stabilization or improvement in 10 of 12 patients evaluable for VFS-PAS. Additionally, Cabaletta led to a 35.3% improvement in timed drinking test (p<0.0001) and a 12.1% improvement in Swallowing QOL (SWAL-QOL) questionnaire score (p=0.0448) from baseline to week 24. On lower extremity muscle strength tests, Cabaletta improved knee extension by 13.9% (p=0.0058), foot extension by 24.3% (p=0.0381) and hip flexion by 3.5% (p=0.4348) from baseline to week 24. On a composite of shoulder abduction and arm flexion upper extremity muscle strength tests, Cabaletta led to a 12.9% improvement on the endpoint from baseline to week 24 (p=0.0836). ...