Trial to Evaluate Inflammasome’s Kamuvudine-9 (K9) in Individuals with ALS

Inflammasome Therapeutics (https://www.inflam.com), a clinical-stage biotech company developing a new class of drugs, Kamuvudines, for ophthalmic and neurological conditions, announced the company has signed a contract with The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital to design a new Healey ALS MyMatch trial evaluating oral Kamuvudine-9 (K9) in individuals with amyotrophic lateral sclerosis (ALS).

The collaboration is in response to the Healey & AMG Center’s Spring 2025 call for applications for the ALS MyMatch program. Inflammasome Therapeutics is one of the first companies selected for inclusion in ALS MyMatch, according to the Center.

“We are honored to be selected and delighted to enter this collaboration with the Healey & AMG Center at Mass General,” said Paul Ashton, Ph.D. co-founder and CEO of Inflammasome Therapeutics. “We are developing a series of compounds targeting neuroinflammation, a disease process that characterizes such diverse diseases as ALS, multiple sclerosis, Parkinson’s disease, Alzheimer’s disease, and macular degeneration, and two of these drugs are already in clinical trials and showing great promise.”

“Preclinical studies of K9, our lead drug for ALS, have been extremely encouraging, showing a profound effect in reducing a biomarker that is prognostic of disease progression,” noted Jayakrishna Ambati, M.D., co-founder of Inflammasome Therapeutics. “Specifically, K9 reduces levels of neurofilament light (NfL) chain in human ALS motoneurons and in animal models by 80% or more,” he added. K9 is also related to and has a similar mode of action as K8, which is being tested in a Phase 2 clinical trial (NCT06164587) in macular degeneration, another neuroinflammatory disease. “Early data from this trial are very promising, giving us further confidence in our ALS program. We are pleased the ALS MyMatch committee found these data compelling,” Dr. Ambati continued.

The Healey ALS MyMatch program is helping to reshape the future of ALS clinical trials by harnessing the power of biomarker-driven personalized trial approaches. By integrating comprehensive genetic and biofluid markers, the program matches subgroups of ALS individuals to various experimental therapies based on their disease markers. ALS MyMatch is an ongoing series of early-phase clinical trials (Phase 1b/2a) aimed at deepening the understanding of biological effects of experimental products and identifying the optimal population for future Phase 2/3 clinical trials.

“There are very few treatments for ALS directed toward the disease process. One of those drugs, Qalsody, was approved by the FDA based on its ability to reduce blood levels of NfL by about 55%. However, this treatment is indicated for the small fraction of patients who have a mutation to a specific gene,” said Dr. Ambati. “Unfortunately, about 95% of individuals with ALS don’t qualify for this treatment.”

Dr. Ashton noted that while other biomarkers of ALS progression are being studied, none have yet been as well-validated as NfL. “NfL is released by dying neurons. Although it’s not specific for a particular disease, it’s two-to-three times higher in those with Parkinson’s disease and 10-times higher in individuals with ALS,” he noted. “Activation of inflammasomes causes neuroinflammation, which, in turn, is associated with diseases that involve neuronal cell death. Thus, we believe that K9, a multi-inflammasome inhibitor, has great potential for treating chronic diseases such as ALS, multiple sclerosis, Parkinson’s disease, and Alzheimer’s disease.”

Healey ALS MyMatch is a multi-site collaborative initiative that currently brings together four trial-ready, high enrolling ALS research centers and is a Network of Excellence for ALS (NEALS) Consortium affiliated program. Research centers include MGH in Boston; University of Minnesota in Minneapolis, Minn; Northwestern University in Evanston, Ill. And Nova Southeastern University in Fort Lauderdale, Fla. ALS MyMatch has partnered with the Acceleration Centers of Enrollment (ACE) program, a community driven philanthropic partnership program focused on expediting start up and recruitment of study centers. Additional trials and high performing sites will be added as the program grows.

The Sean M. Healey & AMG Center for ALS at Mass General, launched in 2018, brings together a global network of scientists, physicians, nurses, foundations, federal agencies and people living with ALS, their loved ones, and caregivers to accelerate the pace of ALS therapy discovery and development.

Inflammasome Therapeutics is a private, clinical-stage biotech company developing novel, first-in-class treatments for multiple ocular diseases, with potential as treatments in neurodegenerative conditions. The company is currently in Phase 1 and 2 clinical trials with two Kamuvudines, K8 and K9. The drugs are delivered via a sustained release implant and orally, respectively.

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