Developer to test ALS therapy with help of Mass General’s MyMatch
Keros using biomarker-driven program to design clinical trial for rinvatercept
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- Keros Therapeutics will work with Massachusetts General Hospital's MyMatch program to design a clinical trial to test its ALS therapy rinvatercept.
- This therapy aims to preserve muscle strength and function, allowing people with ALS to maintain independence longer.
- MyMatch helps identify the specific patient group most likely to benefit from a treatment.
Keros Therapeutics is collaborating with Massachusetts General Hospital to design a clinical trial evaluating its therapy candidate rinvatercept in people with amyotrophic lateral sclerosis (ALS).
According to the developer, the therapy may help preserve muscle strength and function, and improve quality of life, for those with the progressive disease.
The Phase 2 trial will be designed within the ALS MyMatch program, a Mass General initiative that aims to accelerate the development of new ALS treatments. It works by matching patients to specific experimental therapies based on their own genetic and fluid biomarkers.
The program was launched last year and is led by the Sean M. Healey & AMG Center for ALS at Mass General Brigham. The biomarker-driven clinical trial initiative, which is “focused on expediting start up and recruitment,” seeks to identify the patient group most likely to benefit from a given therapy, according to a company press release from Keros. The program findings are then used to inform the design of larger Phase 2/3 clinical trials.
The collaboration with Keros follows the center’s recent call for applications for MyMatch involvement. Keros said it plans to engage with regulators on the trial design later this year.
“We are proud to be selected to the ALS MyMatch program to collaborate with the Healey & AMG Center at Mass General,” said Jasbir S. Seehra, PhD, president and CEO of Keros, who added that “rinvatercept represents a potentially novel approach to treat patients with ALS.”
“Based on the data we have generated to date, we believe there is a strong scientific rationale for the potential of rinvatercept to preserve muscle strength and function and improve quality of life in patients with ALS,” Seehra said.
Rinvatercept aims to support muscle strength, regeneration
ALS is caused by the progressive dysfunction and death of motor neurons, the nerve cells that control voluntary movement. As these nerve cells lose the ability to stimulate muscle fibers, muscles gradually weaken and shrink, leading to increasing muscle weakness and disability.
Rinvatercept is an investigational therapy designed to block the biological effects of myostatin and activin A. By doing so, the therapy aims to support muscle strength and regeneration, which may help people with ALS maintain their physical abilities and independence for longer.
In preclinical studies using a mouse model of ALS, rinvatercept preserved muscle strength and the communication between motor neurons and muscle cells. It also helped protect the neuromuscular junction — the site where nerves communicate with muscles, which is often dysfunctional in ALS.
Early data from a Phase 1 clinical trial in healthy volunteers showed the therapy was generally well tolerated, with no serious side effects or dose-related toxicities reported. It also showed signs of being able to increase muscle mass.
Merit Cudkowicz, MD, director of the Sean Healey Center and professor at Harvard Medical School, welcomed the drug developer to the Mass General program.
“ALS is a serious, progressive disorder with need of additional treatment options,” Cudkowicz said. “We look forward to partnering with Keros to evaluate rinvatercept within the ALS MyMatch program, bringing us closer to our goal of finding additional treatments for those battling ALS.”
Rinvatercept is also being developed for Duchenne muscular dystrophy, a neuromuscular disorder that’s also marked by the progressive loss of muscle mass and strength.
