First-in-human trial now testing oral FA drug in healthy volunteers, recruiting
Novel med harvested from bacteria and engineered to enter brain
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A first-in-human trial testing Adjuvia Therapeutics‘ ATI-105 for Friedreich’s ataxia (FA) — an oral therapy derived from bacteria and engineered to cross a barrier protecting the brain — is now underway, having dosed its first participant.
The Phase 1 study (NCT07869654), sponsored by the California-based biotech, is testing the safety and pharmacological properties of ATI-105 in healthy adult volunteers. Recruitment is ongoing at a single site in Miami, where the drug, taken as an oral capsule, will be tested against a placebo. The trial is expected to enroll about 60 people, ages 18-60, who also meet certain body fat measures.
According to Adjuvia, the trial began dosing participants just weeks after the U.S. Food and Drug Administration (FDA) approved an investigational new drug application, or IND, which is a request for regulatory go-ahead to start clinical testing.
“FDA clearance of our IND application and first [participant] dosing is a significant milestone for our company and, more importantly, for individuals living with Friedreich’s ataxia and rare mitochondrial diseases,” Laura Hix Glickman, PhD, cofounder and CEO of Adjuvia, said in a company press release announcing the trial’s launch. The experimental therapy is ultimately designed to ease disease symptoms in people with FA.
Per the trial page, “the main questions this study is designed to answer include the following: How do single doses or doses for 14 days work in the body? Are these doses safe and tolerated? Should ATI-105 be taken with or without food?”
A genetic disease, FA is caused by mutations in the gene that provides instructions to make a protein called frataxin. This protein is vital for the functionality of mitochondria, the so-called powerhouses of a cell, responsible for helping to produce energy. In people with FA, mitochondria do not function properly, leading to cellular dysfunction that ultimately drives the hallmark symptom, ataxia, characterized by impaired coordination and muscle control during voluntary movements.
ATI-105 targets the powerhouses of cells
ATI-105 aims to repair mitochondrial damage, thereby reducing cellular dysfunction and ultimately easing symptoms. The therapy contains a formulation of astaxanthin, an antioxidant molecule that is naturally produced by Haematococcus pluvialis in response to stress. With ATI-105, astaxanthin is harvested from this bacterium and engineered into a nanoparticle formulation that allows the molecule to cross the blood-brain barrier — a cellular wall that regulates whether substances in the blood can pass into the brain.
Our goal is to develop life-extending therapies for patients with severe mitochondrial diseases, starting with Friedreich’s ataxia.
According to Adjuvia’s website, experiments in preclinical models have indicated that ATI-105 can help cells produce energy more effectively, thus improving cellular survival. Preclinical tests also suggest that the therapy can switch on the body’s own antioxidant defenses to quench reactive oxygen species (ROS), which are toxic oxygen-containing molecules that contribute to cellular damage in FA.
The therapy also has been shown to reduce inflammation and to activate the Nrf2 pathway. This molecular signaling pathway is notably targeted by Skyclarys (omaveloxolone), to date the only approved treatment for FA.
“Our goal is to develop life-extending therapies for patients with severe mitochondrial diseases, starting with Friedreich’s ataxia,” Glickman said.
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