A Message from Harmony Biosciences about the RECONNECT Trial
To the Fragile X Community,
On behalf of Harmony Biosciences, thank you for your time and steadfast engagement with the RECONNECT clinical trial. This document is intended to provide a clear and comprehensive update around the RECONNECT clinical trial and its Open Label Extension (OLE) program, including responses to questions gathered by NFXF and FRAXA. We are writing to share this information directly. We know how much hope and energy so many of you invested in this study, and at Harmony, we approach our work with patient and advocacy communities as a partnership, grounded in respect, clarity, and thoughtful communication.
What We Can Share Today
Following a thorough analysis of the RECONNECT clinical data, we have decided to suspend further development of ZYN002. As a result, the Open Label Extension (OLE) program will be concluded. Across the overall dataset, there was a positive effect, however, the difference between the treatment and placebo groups was not statistically significant based on the larger than expected placebo response. Additional analyses to understand whether specific subgroups may have benefited more have not yielded a different outcome. The study was appropriately powered and increasing the number of study participants would not have changed the statistical outcome relative to the study endpoints.
Open Label Extension (OLE)
The final week of dosing with study drug for participants currently enrolled in the OLE program will be the week of March 30, 2026, with some limited medical exceptions for patients requiring a taper period. Participants currently enrolled in the OLE program will be contacted directly by their respective clinical trial site with information on the discontinuation of the program. Questions should be directed to the clinical sites and your treating physician. The OLE is not currently accepting new participants.
Trial Design and Safety
Regarding safety and tolerability, there were no unexpected results related to safety or tolerability, nor were there unexpected adverse events during the trial. The RECONNECT study was designed as an all‑comers study. The methylation assay was not used to qualify participants; rather, it was used to place participants into groups so we could evaluate ZYN002’s efficacy in specific populations.
Learnings and Context
The RECONNECT results are similar to previously completed Phase 3 studies in Fragile X syndrome in that there was no statistically significant difference between the active treatment and placebo. Future trials may benefit from more objective endpoints that can be quantified rather than described. We know there is interest in broader data sharing for research progress. While we are not able to provide specifics on sharing full trial data at this time, we remain committed to analyzing and publishing our research findings with the data we have collected at a future time to be determined.
How and Why We Communicate Results
Harmony Biosciences believes in the disclosure and communication of study results. As a publicly traded company, we are required to share material study results publicly and simultaneously. We are not permitted to provide updates directly to individual patients or trial participants prior to a public announcement. Participant communications occur through study doctors and the Clinical Research Organization to preserve the study’s integrity. Providing clinical updates directly to NFXF and FRAXA is the fastest and most compliant way for Harmony to communicate with Fragile X families. We encourage the community to stay engaged with these organizations.
All of us at Harmony share the disappointment felt by families related to this news. We are deeply grateful for the community’s participation, advocacy, and partnership throughout this journey. Your commitment has meaningfully contributed to the advancement of research in Fragile X syndrome, and we thank you for the trust you placed in us.
Sincerely,
Harmony Biosciences
Dr. Jeffrey Dayno
President and Chief Executive Officer
Dr. Kumar Budur
Chief Medical and Scientific Officer
