Project Mercury unites patients, industry, research and clinical communities in groundbreaking initiative
from Fulcrum Therapeutics
Today on World FSHD Day, the FSHD Society and Fulcrum Therapeutics announce the launch of Project Mercury, a first-of-its-kind collaboration of global stakeholders committed to identifying and addressing the challenges that could impede the delivery of new medicines for this devastating condition.
Developing and delivering treatments for any rare disease involves unique challenges, including establishing trial outcome measures; enrolling small, and often undiagnosed, patient populations; managing financial risk and incentives; navigating extended diagnostic journeys and fractured care pathways; and generating clear and compelling evidence to support regulatory and reimbursement activities.
“We understand the gaps in improving the lives of rare disease patients, but no patient advocacy organization is capable of solving the myriad of challenges on its own,” said Mark Stone, President and CEO, FSHD Society and inaugural chair of the Project Mercury Global Task Force. “By synergizing the expertise, insights and influence of industry, researchers and others, we have greater chance at catalyzing change and meeting the needs of the FSHD community.”
Project Mercury consists of collaboration at the global level, through a Global Task Force, and at the local level, through Country Working Groups. Founding Project Mercury Global Task force members include: The FSHD Society, FSHD Canada Foundation and multiple other members of the World FSHD Alliance, Fulcrum Therapeutics, Avidity Biosciences, TREAT-NMD, FSHD patient advocates, leading FSHD clinicians and researchers, and experts in the fields of health economics and patient access.
FSHD is a rare genetic disorder that is estimated to affect 1 in 8,000 people worldwide. People with FHSD experience relentless and accumulating muscle and functional loss, which results in the inability to perform daily life activities due to significant impairment of upper extremity function, loss of mobility, and chronic pain. Typically beginning in the teenage years, around 20 percent of people diagnosed with FSHD will need a wheelchair by age 50. FSHD currently has no effective treatment or cure, however in recent years significant progress has been made, underscored by Fulcrum Therapeutics’ initiation of REACH, the first ever Phase 3 clinical trial for FSHD in 2022.
The unrelenting progression of FSHD illustrates the importance of providing rapid and equitable delivery of future treatments. Driven by this sense of urgency, Project Mercury seeks to mobilize all sectors of the FSHD community to unite for the singular purpose of ensuring that the barriers to access are removed, as quickly as possible.
Project Mercury is guided by the principle of shared burden to create shared benefit. Collectively, the members of the Global Task Force aim to accelerate progress in understanding and treating FSHD by:
- building a global cohort of 10,000 clinical-trial-ready, well-characterized patients for participation in clinical trials
- expanding and optimizing the world-wide clinical trial infrastructure
- removing the barriers that delay delivery of therapies once approved
“We are thrilled to be a global sustaining partner in this unique collaboration to ensure that patients benefit from innovation as quickly as possible,” said Robert J. Gould, Ph.D., Fulcrum’s interim president and chief executive officer. “The Project Mercury model can help align all stakeholders to overcome the barriers and inefficiencies that stand in the way of progress.”
Project Mercury’s Global Task Force and Country Working Groups are all led by patient advocacy organizations of the World FSHD Alliance. As the co-founding and convening partner, the FSHD Society provides management, resources, and implementation support for the coalition. As a founding and sustaining partner, Fulcrum Therapeutics is supporting Project Mercury by donating resources and support for communications. All members of the Global Task Force commit to sharing resources and dedicating staff to help Project Mercury achieve the greatest impact.
More information about Project Mercury can be found at https://www.projectmercuryfshd.org.
What does quickly as possible mean? 10 years? 20 years? 30 years?
Many things can go off the rails in drug development, but the first FSHD drug approval in the US might come as soon as in 2-3 years. The work needs to begin ASAP to prepare patient advocacy groups to make sure their voices are heard by regulators and payors about the outcomes that matter to them.
After 2 or 3 more years it will have have taken something like 7+ years to get access to a simple small molecule that already had been safety tested on thousands of people before FSHD trials. Clearly Losmapimod should already be accessible to those seriously affected through expanded access. It has has taken far too long with losmapimod and as things are done now, it will be 10+ years minimum before any of these other treatments like Avidity’s candidate could potentially be available to anyone seriously affected. These time frames are simply not acceptable. There will be no benefit to a very high percentage of people with FSHD who are alive today.
Project Mercury is an innovative initiative that brings together patients, industry, research, and clinical communities, fostering collaboration and breakthroughs in healthcare. This groundbreaking project has the potential to drive significant advancements in patient care, benefiting individuals and the healthcare industry as a whole.
das Project Mercury gibt doch Hoffnung, dass Losmapimod doch rasch zugelassen wird !
Nach den langjährigen Tests, die sehr positive Erfolge gezeigt haben, sollte für schwere FSHD Betroffene Losmapimod so rasch als möglich freigegeben werden !! Die rasche Zulassung der Chovid Impfstoffe hat gezeigt, dass es möglich ist wenn die Wissenschaft und die Politiker dies fordern !! Die Kooperation von Fulcrum und Sanofi erhöht die Hoffnung der vielen FSHD Betroffenen beachtlich ! Zuständige Gesundheitsminister weltweit sollten in den Zulassungsprozess von FSHD Medikamenten eingebunden werden um kompetente Informationen zu erhalten !!!