FDA OKs trial testing treatment for muscle regeneration in FSHD adults
Canadian nonprofit commits up to $5M to back therapy's development
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The U.S. Food and Drug Administration (FDA) has given the go-ahead for a mid-stage clinical trial testing the oral therapy forazapadin — designed to protect muscles and potentially enhance their regeneration — in adults with facioscapulohumeral muscular dystrophy (FSHD), marked by muscle weakness that particularly affects the face, shoulders, and upper arms.
Satellos Bioscience, the developer behind forazapadin, formerly called SAT-3247, now plans to launch a Phase 2 study before the end of the year to test the drug’s safety and potential effectiveness in people with this form of muscular dystrophy (MD). The FSHD Canada Foundation has committed funding of as much as $5 million in exchange for a share of revenue if the therapy is ultimately approved.
The FDA cleared Satellos’ investigational new drug application (IND), giving the study the green light to begin. According to the developer, the trial will test two doses of forazapadin — 60 and 120 mg — against a placebo, with the goals of evaluating its safety profile, pharmacological properties, and early efficacy.
“Progress in medicine happens when researchers, clinicians, industry partners and advocacy organizations come together around a common goal, and we are grateful to the FSHD Canada Foundation for its partnership and confidence in our work,” Frank Gleeson, cofounder and CEO of Satellos, said in a company press release. “This support is expected to enable us to advance forazapadin into clinical development in FSHD and extend our muscle regeneration strategy to a second patient community.”
Satellos is also developing forazapadin for Duchenne muscular dystrophy (DMD), the most common type of MD, which is characterized by progressive muscle weakness throughout the body. In DMD, which primarily affects boys and men, weakness typically first affects the muscles around the hips and upper legs.
To help fund the upcoming trial in FSHD, Satellos struck an agreement with the Canadian nonprofit, which has the “singular goal” of finding a cure for this MD type. The foundation will provide financing toward the development of forazapadin for this indication and receive a revenue-sharing interest in any FSHD-related sales of the therapy.
Forazapadin is expected to boost muscle stem cell growth
FSHD is a genetic disorder characterized by muscle weakness in the upper body, including the facial muscles, that worsens over time. It is caused by abnormal activation of the DUX4 gene, and to date, there are no approved treatments that can alter disease progression.
When muscle tissue is damaged, specialized cells called muscle stem cells are normally able to grow to replace the damaged tissue. However, in muscular dystrophies, this process may be impaired.
Forazapadin is designed to block the activity of a protein called AAK1, which is expected to boost muscle stem cell growth and ultimately help facilitate muscle regeneration. The therapy has been shown to improve muscle function in a mouse model of FSHD.
“People living with FSHD, like me, are eager to find treatments that can stop our muscles from getting weaker. But we would also like to get some of those muscles back. That is what makes this announcement so meaningful,” said Neil Camarta, cofounder of the FSHD Canada Foundation. “Seeing forazapadin advance into clinical trials to evaluate the potential for muscle regeneration in FSHD is an important step for our community.”
Camarta added: “While we know there is still a long road ahead, it is encouraging to see innovative approaches like this moving into the clinic.”
People living with FSHD … are eager to find treatments that can stop our muscles from getting weaker. But we would also like to get some of those muscles back.
The FDA previously gave Satellos the go-ahead to start a clinical trial testing forazapadin in boys with DMD. That study, dubbed BASECAMP (NCT07287189), began dosing earlier this year. It involves an estimated 51 boys with DMD, ages 7 to 9, at sites in North America, Europe, and Australia. According to the developer, early trial data suggest that forazapadin has an acceptable safety profile, and imaging data indicate the therapy may help promote muscle regeneration.
Wildon Farwell, MD, chief medical officer of Satellos, said the company is “excited” to now have regulatory clearance to test forazapadin for FSHD.
“In particular, we are delighted that the clearance included 60 mg and 120 mg dose levels of forazapadin, enabling evaluation of two doses of our small molecule drug candidate,” Farwell said. “We look forward to working with the FSHD community to evaluate the potential of forazapadin to impact muscle regeneration and benefit people living with FSHD.”
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