ACTA Pharmaceuticals

Early intervention

The next frontier in Alzheimer’s disease

Scientific progress has created new opportunities to intervene earlier in Alzheimer’s disease (AD). Acta Pharmaceuticals, Inc. is building on four decades of discoveries led by renowned neuroscientist Dr. Rudolph (Rudy) Tanzi to develop new therapies for early intervention and Alzheimer’s prevention.

Acta at a glance

Scientific founder

Rudolph E. Tanzi, Ph.D.

Harvard Medical School

Massachusetts General Hospital

40+ years

Pioneering Alzheimer’s research

$70M in funding

Supporting preclinical development

Lead clinical program

GSM-779690T

Phase 1 clinical trial

Translating decades of pioneering Alzheimer’s research into clinical innovation.

“We must protect brain health like we protect heart health: early, before symptoms begin.”

Rudolph E. Tanzi, Ph.D.

Scientific Founder

Our programs

Translating pioneering Alzheimer’s research into therapeutic innovation

Acta is advancing two complementary programs targeting early intervention and prevention

A clinical study for GSM-779690T, a next-generation gamma secretase modulator

A discovery platform with a pipeline of 50+ validated therapies for investigation

Oral small molecule

Selective GSM

Aβ42↓

Aβ37/Aβ38 ↑

Amyloid-peptide balance

Lead clinical program

GSM-779690T

GSM-779690T is engineered as a best-in-class gamma-secretase modulator (GSM) for the potential treatment and ultimately, prevention of Alzheimer’s disease. It’s the culmination of 25 years of research led by GSM inventors Dr. Tanzi and the late Dr. Steven L. Wagner through a collaboration among Massachusetts General Hospital, Harvard Medical School and the University of California, San Diego.

Explore GSM-779690T

Drugs + natural products

Alzheimer’s-in-a-Dish™

Candidates + combinations

Discovery platform

Combination therapies program

Acta’s Combination Therapies program builds on decades of research led by Dr. Tanzi using Alzheimer’s-in-a-Dish™, laboratory models of human brain cells in mini-organoids and “assembloids” that recreate key pathological features of AD and the aging brain. Using the model, Dr. Tanzi and colleagues screened thousands of compounds, identified hundreds of treatment candidates, and validated 50 for investigation, which could expand Acta’s treatment pipeline.

Explore Combination Therapies

Mechanism

Selective modulation

Aβ42↓

GSM

Aβ37↑

Aβ38↑

Designed to shift the amyloid-beta peptide (Aβ) balance

Investigational oral candidate being developed to reduce longer forms of amyloid-beta, primarily Aβ42, in favor of shorter forms such as Aβ37 and Aβ38, which are less likely to aggregate and can be neuroprotective.

Oral

Small molecule

Phase 1

Clinical trial

25 yrs

Research

Lead clinical program

GSM-779690T

Engineered for best-in-class potency

GSM-779690T is the culmination of 25 years of structural innovation within one of the world’s most comprehensive GSM portfolios. Researchers optimized GSM-779690T for pharmacologic activity and brain penetration. In preclinical studies, it demonstrated Aβ42 potency of 5.3 nanomolar (nM), near 1:1 brain-to-plasma ratio, oral bioavailability across multiple species and no inhibition of Notch cleavage.

Acta is advancing GSM-779690T in a phase 1 clinical trial.

Learn about GSM-779690T

GSM-779690T: a strong scientific foundation

Best-in-class potency

5.3 nM Aβ42 potency

Near 1:1 brain-to-plasma ratio

Orally bioavailable across species

Highly selective γ-secretase modulation with no inhibition of cleavage of the important gamma secretase substrate, Notch, at concentrations up to 20 micromolar (μM)

25 years of research

The culmination of 25 years of research by GSM inventors Drs. Tanzi and Wagner

Scientific collaboration

Long-standing scientific collaboration among Massachusetts General Hospital, Harvard Medical School and the University of California, San Diego

$70M preclinical development funding

Rigorous preclinical development, including support from the National Institutes of Health’s National Institute on Aging and the Cure Alzheimer’s Fund

Dr. Rudolph E. Tanzi

Dr. Rudolph E. Tanzi

Scientific Founder · Harvard Medical School · Massachusetts General Hospital

Scientific foundation

Built upon decades of pioneering Alzheimer’s research.

Dr. Rudolph E. Tanzi’s decades of discoveries have helped define the genetic and biological understanding of Alzheimer’s disease. His scientific foundation anchors Acta’s effort to translate pioneering research into investigational medicines.

40+

years of Alzheimer’s research

800+

scientific publications

50+

patents

Meet Our Scientific Founder

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