Acta Pharmaceuticals has raised $24 million in total seed financing to advance a pipeline of therapies designed for earlier intervention in Alzheimer’s disease, including its lead gamma-secretase modulator, GSM-779690T.
The Boston-based company was created to translate more than four decades of Alzheimer’s research from scientific founder Dr. Rudolph E. Tanzi into potential new therapies. Its lead program is the result of approximately 25 years of gamma-secretase modulator research conducted by Tanzi and the late Dr. Steven L. Wagner at Massachusetts General Hospital and the University of California, San Diego.
GSM-779690T is being developed as a once-daily oral small-molecule treatment intended for early-stage, long-duration disease modification and potentially prevention. The drug is designed to lower levels of Aβ42, an amyloid-beta protein associated with plaque formation, while increasing Aβ37 and Aβ38, which can interfere with aggregation. Unlike earlier gamma-secretase inhibitors, the approach selectively modulates the enzyme while seeking to preserve its normal biological functions.
Acta is evaluating GSM-779690T in a randomized, double-blind, placebo-controlled Phase 1 study in healthy adult volunteers. The trial is designed to assess safety, tolerability, and pharmacokinetics across single and multiple ascending doses. Preclinical studies cited by the company showed single-digit nanomolar potency against Aβ42, a near 1:1 brain-to-plasma ratio in mice, and oral bioavailability across multiple species.
Beyond its lead program, Acta is developing combination therapies based on research using Alzheimer’s-in-a-Dish, a laboratory platform developed by Tanzi and Massachusetts General Hospital colleagues. The models recreate important features of Alzheimer’s pathology and the aging brain and allow researchers to screen large numbers of approved drugs and natural compounds. The research has identified more than 30 validated combinations that the company said can curb Alzheimer’s pathology, with Acta licensing related intellectual property from MGH and advancing selected candidates toward clinical evaluation.
Tanzi’s research has played a significant role in the scientific understanding of Alzheimer’s disease. He co-discovered the familial Alzheimer’s genes APP, PSEN1, and PSEN2 and has authored more than 800 scientific papers and holds approximately 50 patents. He is Director of the Genetics and Aging Research Unit and the McCance Center for Brain Health at Massachusetts General Hospital and serves as Joseph P. and Rose F. Kennedy Professor of Neurology at Harvard Medical School.
Acta said approximately $70 million in preclinical development funding supported the research that ultimately led to GSM-779690T. The company’s shareholders include Alzheimer’s Disease Research Foundation, Dolby Family Ventures, Gamma Bio Investors, family offices, and other mission-driven investors.
The new financing will support Acta as it moves its lead program through clinical development while expanding a broader pipeline aimed at intervening earlier in the Alzheimer’s disease process. GSM-779690T remains investigational and has not been approved by any regulatory authority.
KEY QUOTES:
“We founded Acta Pharmaceuticals to translate more than four decades of Dr. Rudolph Tanzi’s discoveries into new Alzheimer’s disease treatments to enable early intervention and prevention. GSM-779690T is the culmination of 25 years of research at Massachusetts General Hospital and the University of California, San Diego and approximately $70 million in preclinical development funding. We’re privileged to build on Drs. Tanzi and Wagner’s pioneering GSM research and decades of discoveries to give the broadest group of people the opportunity for early prevention and treatment of one of the world’s most devastating and costly diseases.”
George Alex, Co-Founder And CEO Of Acta Pharmaceuticals
“We’re entering a golden age in Alzheimer’s research. Our understanding of genetic risk, new diagnostic tools and growing evidence showing that lifestyle habits can delay and even prevent disease give us an unprecedented opportunity to proactively protect brain health, just as we protect heart health today—before symptoms start. Our vision is that GSM-779690T could someday serve as the equivalent of a ‘statin for the brain’: a once-daily oral therapy designed to restore a healthier balance of amyloid-beta protein generation in the brain before irreversible damage occurs, giving people an opportunity to intervene with the AD pathological process as early as possible. All the Alzheimer’s research undertaken during the past four decades is coming to fruition at this moment, and I’ve never been more optimistic that Alzheimer’s prevention is within reach.”
Dr. Rudolph E. Tanzi, Scientific Founder Of Acta Pharmaceuticals
“As the culmination of more than 25 years of structural innovation, we believe GSM-779690T is a potential best-in-class gamma-secretase modulator for the treatment and potential prevention of Alzheimer’s disease. Acta evaluated hundreds of analogues to optimize potency, brain penetration and drug-like properties, ultimately identifying a single structurally distinct development candidate. Published preclinical studies demonstrated single-digit nanomolar potency against amyloid-beta 42 together with exceptional brain penetration, supporting efficacy at lower dose levels.”
Kevin Rynearson, Ph.D., Chief Scientific Officer Of Acta Pharmaceuticals
