Harmony's ZYN002 Shows Clinically Meaningful Improvements In Fragile X Syndrome Patients

(RTTNews) - Harmony Biosciences Holdings, Inc. (HRMY), Tuesday announced the updated data from its Open-Label Extension or OLE study ZYN2-CL-017, evaluating the safety and effectiveness of ZYN002 in children, adolescents, and adults with Fragile X syndrome, a rare genetic disorder.

Participants entered the OLE trial from the Phase 2/3 CONNECT-FX trial, and the Phase 1/2 FAB-C signal detection open-label trial.

The findings revealed clinically meaningful improvements in irritability-related symptoms prevalent in individuals with FXS.

Additionally, over 60 percent of participants in both groups achieved clinically meaningful improvement of at least 9 points on the ABC-CFXS Irritability scores at two consecutive visits.

Additionally, 40.6 percent of participants who received active treatment in the CONNECT-FX trial demonstrated clinically meaningful improvement in behavior based on the Caregiver Global Impression of Change scores.

At the end of three years, 73.3 percent of patients showed clinically meaningful improvements.

ZYN002 is currently being evaluated as an investigational treatment for FXS in the RECONNECT study, evaluating the safety and efficacy of ZYN002 in patients with FXS ages 3 to under 30 years.

Currently, Harmony's stock is trading at $29.18, up 2.80 percent on the Nasdaq.