Inflammasome Therapeutics to Collaborate with Healey & AMG Center on New ALS Treatment Study
Inflammasome Therapeutics, a clinical-stage biotechnology company advancing a new category of drug candidates known as Kamuvudines for ophthalmic and neurological diseases, announced that it has signed an agreement with The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital. Through this partnership, the organizations will design a new Healey ALS MyMatch clinical trial to evaluate oral Kamuvudine-9 (K9) in individuals diagnosed with amyotrophic lateral sclerosis (ALS).
This collaboration comes in response to the Healey & AMG Center’s Spring 2025 call for applications for inclusion in the next round of ALS MyMatch trials. Inflammasome Therapeutics is among the first companies chosen to participate, marking an important milestone for both its neuroinflammation drug platform and the forward-looking MyMatch program.
Paul Ashton, Ph.D., co-founder and CEO of Inflammasome Therapeutics, expressed enthusiasm for the opportunity. “We are honored to be selected and delighted to enter this collaboration with the Healey & AMG Center at Mass General,” he said. Ashton noted that the company’s focus on developing compounds that inhibit neuroinflammatory pathways is relevant to a broad spectrum of diseases—including ALS, multiple sclerosis, Parkinson’s disease, Alzheimer’s disease, and macular degeneration. Two Kamuvudine molecules are already in clinical testing and have demonstrated meaningful potential, reinforcing confidence in the therapeutic promise of the platform.
Co-founder Jayakrishna Ambati, M.D., highlighted the compelling preclinical foundation behind K9, the company’s lead ALS candidate. “Preclinical studies of K9 have been extremely encouraging, showing a profound effect in reducing a biomarker that is prognostic of disease progression,” he explained. “Specifically, K9 reduces levels of neurofilament light (NfL) chain in human ALS motoneurons and in animal models by 80 percent or more.” NfL is a biomarker widely studied across neurodegenerative conditions, and reductions in NfL have been correlated with slower clinical progression.
Dr. Ambati added that K9 is closely related to—and works through a similar mechanism as—Kamuvudine-8 (K8), another Inflammasome Therapeutics candidate currently being evaluated in a Phase 2 clinical trial (NCT06164587) for macular degeneration, a disease that also involves neuroinflammation. “Early data from this trial are very promising,” he said. “These results give us further confidence in our ALS program, and we are pleased that the ALS MyMatch committee found our data compelling.”
Advancing ALS Trials Through Biomarker-Driven Personalization
The Healey ALS MyMatch program represents a paradigm shift in the way early-stage ALS clinical trials are designed and conducted. Rather than using traditional one-size-fits-all approaches, the MyMatch platform integrates comprehensive biomarker data—including genetic markers and biofluid profiles—to match individuals with ALS to therapies most relevant to their underlying biology. This personalized approach is intended to enhance the likelihood of detecting meaningful biological responses and guide more efficient progression to larger Phase 2 or Phase 3 trials.
MyMatch consists of a series of Phase 1b/2a studies focused on gaining a deeper understanding of each investigational therapy’s biological effects. By aligning trial enrollment with defined biomarker patterns, the program seeks to determine which subgroups of individuals may benefit most from specific experimental treatments. This helps accelerate development, reduce unnecessary exposure to ineffective therapies, and build a robust evidence base more quickly than conventional trial structures allow.
One of the core biomarkers guiding current ALS development is neurofilament light chain. “There are very few treatments for ALS that are directed toward the disease process,” Dr. Ambati explained. “One of those drugs, Qalsody, was approved by the FDA based on its ability to reduce blood levels of NfL by around 55 percent. However, this treatment is available only for the small fraction of patients who carry a specific genetic mutation.” Approximately 95 percent of individuals with ALS are not eligible for this therapy, underscoring the need for more broadly applicable treatments such as K9.
Dr. Ashton added that while additional biomarkers of ALS progression are under investigation, NfL remains the best-validated marker currently available. “NfL is released by dying neurons. Although it is not specific to a single disease, its levels are elevated two- to threefold in people with Parkinson’s disease and approximately tenfold in individuals with ALS,” he noted. “Activation of inflammasomes Therapeutics drives neuroinflammation, which is closely associated with neuronal death. Because K9 acts as a multi-inflammasome Therapeutics inhibitor, we believe it has considerable promise for treating ALS as well as other chronic neuroinflammatory diseases such as multiple sclerosis, Alzheimer’s disease, and Parkinson’s disease.”
A Multi-Site Research Network to Accelerate ALS Innovation
Healey ALS MyMatch is a multisite collaborative program that currently connects four major ALS research centers known for rapid and high-quality enrollment. These include Massachusetts General Hospital in Boston; the University of Minnesota in Minneapolis, Minnesota; Northwestern University in Evanston, Illinois; and Nova Southeastern University in Fort Lauderdale, Florida. The program is affiliated with the Northeast ALS (NEALS) Consortium and is designed to expand over time, with additional high-performing research centers expected to join future MyMatch studies.
The initiative has also partnered with the Acceleration Centers of Enrollment (ACE) program. ACE is a community-driven philanthropic effort that supports research centers in expediting both study start-up and participant recruitment—two common bottlenecks in ALS clinical development. By streamlining administrative processes and enabling more predictable enrollment timelines, ACE improves the pace at which promising experimental therapies can enter and advance through clinical testing.
About the Healey & AMG Center for ALS
The Sean M. Healey & AMG Center for ALS at Mass General was launched in 2018 with a mission to accelerate the global search for effective ALS treatments. The Center has built a broad network of scientists, clinicians, foundations, government agencies, individuals living with ALS, and caregivers. This collaboration-driven structure allows the Center to deploy innovative trial models—including the platform-style Healey ALS Platform Trial and, now, the biomarker-guided MyMatch program. The Center’s mission is rooted in advancing new therapies more efficiently while ensuring that individuals with ALS have expanded access to cutting-edge research opportunities.
About Inflammasome Therapeutics
Inflammasome Therapeutics is a private clinical-stage biotechnology company working to develop novel, first-in-class therapies for neurodegenerative and ocular diseases. The company’s pipeline is centered around Kamuvudines, a family of drugs designed to inhibit inflammasomes Therapeutics—key drivers of inflammation implicated in several conditions marked by neuronal cell loss. Two of the company’s leading drug candidates, K8 and K9, are currently being evaluated in Phase 1 and Phase 2 clinical trials. K8 is delivered via a sustained-release implant for ocular diseases, while K9 is administered orally for systemic neuroinflammatory conditions.
With its inclusion in the Healey ALS MyMatch program, Inflammasome Therapeutics is taking an important step toward translating its scientific insights into potential new treatment options for people affected by ALS. Through this collaboration, both organizations hope to advance the understanding of inflammasome Therapeutics-mediated neurodegeneration and accelerate the development of therapies capable of addressing the unmet needs of the broader ALS patient community.
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