FSHD Global Research Foundation in Sydney is excited to announce it has invested in miRecule’s second generation drug development program that will simultaneously target two disease mechanisms in facioscapulohumeral muscular dystrophy (FSHD). The 2nd generation program is designed to not only suppress DUX4 activity but also address downstream biological pathways involved in muscle damage and regeneration.
“While several therapies are now targeting DUX4, no single therapeutic approach is likely to address every aspect of FSHD. Supporting a diverse pipeline of complementary approaches increases the likelihood of delivering meaningful benefit across the spectrum of disease,” says Emma Weatherley, FSHD Global’s CEO and Managing Director.
miReculeTM is a biotechnology company with a strong track record: its first-generation compound (an siRNA inhibitor of Dux4) was licensed to Sanofi and first-in-human clinical trials are expected soon.
“Dux4 inhibition is a critical first step to treating the disease, but it won’t solve all the problems patients face. We are hoping this second-generation therapy may provide broader therapeutic benefit that will help with muscle regeneration and inflammation,” says Dr Anthony Saleh, miRecule CEO.
The second-generation program is in the discovery phase, and the funding from FSHD Global will enable proof of concept studies to be completed in 2026, with the expectation that lead compound development can be completed in 2027, if further funding is obtained.
“We are hugely excited about working with FSHD Global and potentially coming to Australia to do early phase trials,” continues Dr Saleh.
“What’s unique about FSHD Global is its focus on ensuring therapeutic development benefits the entire FSHD community. Through investments in clinical trial readiness, disease progression modelling and patient data, the Foundation is helping tackle one of the greatest challenges in FSHD, designing studies that can demonstrate benefit across a highly heterogeneous disease. I have FSHD also, and we share an impetus to bring forward products to address unique challenges.
“Many current clinical trials enrol ambulatory adults with moderate disease because these populations provide the clearest regulatory pathway. However, our long-term goal is to ensure future therapies can also be evaluated in children and people living with advanced stage FSHD. My hope is the 2nd generation drug will address their unique challenges better, but we had to go through the 1st generation research to get here.
“Drug development is inherently risky, with a 90% failure rate, and rushing makes that worse. At the end of the day, we want this to be successful for patients, so it’s important to take enough time to cross t’s and dot i’s. I believe our 1st generation is the best of Dux4 inhibitors, with great potential, and this 2nd generation offers even more benefits.
“Our hope is to work long-term with FSHD Global, not just as a funder but to bring these therapies to patients around the world and Australia,” says Dr Saleh.
“Targeting DUX4 is a critical first step, but it may not be the whole answer for people living with FSHD. What excites us about miRecule’s second-generation program is its ambition to tackle multiple aspects of the disease, including muscle regeneration and inflammation. Investing in innovative science like this is central to our mission of improving both function and quality of life for everyone living with FSHD,” says Weatherley.