Acta Pharmaceuticals has raised $24 million in total seed financing to develop new therapies aimed at early intervention and the potential prevention of Alzheimer’s disease.
The company plans to use its scientific research to develop treatments that could help reduce the risk of Alzheimer’s disease before significant damage occurs in the brain. Its lead drug candidate, GSM-779690T, is being developed as a potential long-term treatment for early-stage Alzheimer’s disease and, eventually, disease prevention in at-risk populations.
Acta was founded to advance decades of research led by neuroscientist Dr. Rudolph E. Tanzi, who has spent more than 40 years studying Alzheimer’s disease. The company’s lead program builds on approximately 25 years of research into gamma-secretase modulators conducted by Dr. Tanzi and the late Dr. Steven L. Wagner at Massachusetts General Hospital and the University of California, San Diego.
“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,” said George Alex, Acta Pharmaceuticals’ co-founder and CEO. “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.”
Alzheimer’s disease affects more than seven million Americans. Many existing treatments focus on managing symptoms or slowing disease progression after harmful amyloid-beta proteins have accumulated in the brain.
Acta’s GSM-779690T is designed to take a different approach. The experimental drug aims to reduce the production of amyloid-beta 42, a protein associated with plaque formation, while increasing other forms, including amyloid-beta 37 and 38, that may help limit plaque development.
Unlike earlier drugs that broadly block gamma-secretase activity, GSMs are designed to adjust the enzyme’s activity while preserving its normal biological functions. According to the company, preclinical studies showed that GSM-779690T did not inhibit the processing of Notch, an important gamma-secretase substrate, at concentrations up to 20 micromolar.
The company is also developing combination therapies using research from the Alzheimer’s-in-a-Dish platform. Developed by Dr. Tanzi and his colleagues, the platform uses laboratory models of human brain cells to recreate important features of Alzheimer’s disease. It allows researchers to test hundreds of existing drugs and natural compounds simultaneously.
“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,” said Kevin Rynearson, Ph.D., Acta Pharmaceuticals’ chief science officer. “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. It’s a true honor to build upon the pioneering work of our Scientific Founder, Dr. Rudolph Tanzi, and my mentor, Dr. Steven Wagner, as we advance what we believe is the next generation of Alzheimer’s therapeutics.”
According to Acta, researchers have identified more than 30 validated drug combinations that reduce Alzheimer’s-related pathology using this approach. The company has licensed related intellectual property from Massachusetts General Hospital and is advancing selected candidates toward clinical evaluation.
Acta plans to evaluate GSM-779690T in a Phase 1 clinical study involving healthy adult volunteers. The randomized, placebo-controlled trial is designed to assess the drug’s safety, tolerability and pharmacokinetics. The candidate remains experimental and its safety and effectiveness in treating or preventing Alzheimer’s disease have not yet been established.
Acta’s shareholders include Alzheimer’s Disease Research Foundation, Dolby Family Ventures, Gamma Bio Investors, family offices and other mission-driven investors.
With the new funding, the company aims to advance its lead drug candidate and expand its pipeline of potential treatments designed to address Alzheimer’s disease earlier in its development.
