Project Mercury, a global initiative dedicated to accelerating readiness for FSHD therapies, marked a significant milestone at its 5th Semi-Annual Global Task Force (GTF) meeting. Held on June 15–16, the roundtable brought together international Key Opinion Leaders (KOLs), patient advocates, and industry stakeholders to align on strategic priorities across three critical workstreams: Trial to Therapy (WG1), Access (WG2), and Sustainability (WG3).
Global Synergies in Action
The meeting underscored the power of cross-border collaboration. Representatives from Europe, North America, Latin America and Australia shared progress on national registries, clinical trial readiness, and advocacy efforts, reinforcing the value of shared governance and transparent communication.
Highlights from the Three Workstreams
- WG1 – Trial to Therapy: The group reviewed progress on clinical trial infrastructure, including the development of the Focus Database to securely manage trial data in compliance with GDPR. Discussions included regulatory alignment with the FDA and EMA, patient enrollment strategies, and the importance of unified messaging to manage expectations around upcoming Phase 3 trials.
- WG2 – Access: A global disease progression model is in development, integrating diverse data sources to support Health Technology Assessments (HTAs). The group also addressed the burden of illness, quality of life metrics, and cost-effectiveness studies, with a focus on ensuring data consistency and accessibility across countries.
- WG3 – Sustainability: A unified global fundraising and development strategy was proposed, with platforms now in place to ensure 100% pass-through donations. Country Working Groups (CWGs) will receive tailored guidance to meet fundraising goals, and quarterly meetings will be reinstated to foster alignment and long-term viability.
Looking Ahead
The GTF reaffirmed its commitment to transparency, inclusivity, and patient-centered governance. Plans are underway for a global summit to further align stakeholders on treatment readiness and advocacy. Project Mercury continues to serve as a catalyst for uniting scientific innovation with real-world impact, ensuring that no region is left behind in the journey toward effective FSHD therapies.
