Cambrian Bio records a positive human trial with its AMPK Activator drug, ATX-304

Scientists may have finally found a way to safely restore AMPK function in humans, a milestone many researchers in the aging biology field have pursued for almost 30 years.

In a release on Monday, biotech company Cambrian Bio reported positive Phase 1b results for ATX-304, a clinical-stage drug designed to activate the AMPK metabolic pathway.

AMPK is a cellular enzyme that acts as a metabolic sensor that coordinates how cells take in and burn fuel. The problem is that AMPK activity declines with age, which, in turn, reduces metabolic efficiency.

Scientists have been on AMPK Activators for almost 30 years

For nearly three decades, scientists have reportedly sought to develop a drug that can safely restore AMPK function in humans, with ATX-304 now the first potential drug to enter clinical trials, according to Cambrian Bio.

In an 8-week randomized, double-blind, placebo-controlled study of 23 adults with obesity and prediabetes, followed by an 8-week OLE, the drug recorded significant metabolic improvements, including an 8% increase in resting metabolic rate, meaning participants burned more energy at rest.

Participants took either 400 mg of ATX-304 once daily or a placebo for eight weeks. Weight loss at this dose was minimal, which Cambrian said matched preclinical predictions.

The drug is said to work differently from GLP-1 weight-loss therapies that reduce appetite. ATX-304 instead targets cellular energy use, increasing both glucose uptake and mitochondrial respiration, with no signs of “mitochondrial uncoupling.”

Cambrian Bio says the effect resembles what happens during aerobic exercise training.

“The full AMPK Network Activation of ATX-304 elevates overall metabolic activity in a cell, driving up both substrate availability and use, increasing energy expenditure in a manner that closely resembles aerobic exercise training,” said James Peyer, PhD, CEO and Founder of Cambrian Bio.

Aging is tied to metabolic decline. Scientists being able to restore AMPK activity to youthful levels could prevent or delay the onset of multiple age-related cardiometabolic diseases, like Type 2 diabetes, cardiovascular disease, and fatty liver disease, ultimately prolonging human lifespan.

Anti-aging field continues to make more headway

Cambrian Bio says the next step is a two Phase 2 trials that would test higher doses of ATX-304 on patients. The company indicates that higher doses in future trials would target greater weight reduction.

The anti-aging field has made decent progress over the last few years, with several drugs moving into clinical trials, Cryptopolitan reported. Many of the companies are also successfully raising funds from investors to lead research into longevity, with the recent one being Coinbase CEO’s startup, NewLimit. 

Earlier this month, NewLimit raised $435M in a Series C round led by Founders Fund to bring its longevity medicine to human trials.

Aging is arguably the root cause of most major diseases (loss of function in our cells). Four years ago, we made a bet that aging was treatable, and NewLimit was born.

NewLimit now has a prototype drug that reverses the age of some human cells (restores function they had when… https://t.co/rUWpGFwBDX

— Brian Armstrong (@brian_armstrong) June 2, 2026

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