Company also announces appointment of Stanford mitochondrial disease expert to its Scientific and Clinical Advisory Board and launch of two new pipeline programs
SAN FRANCISCO–(BUSINESS WIRE)–Adjuvia Therapeutics, a biotechnology company developing novel therapeutics to treat mitochondrial dysfunction and disease, today announced that the first participant has been dosed in its Phase 1 clinical trial of ATI-105, its lead investigational therapeutic candidate for Friedreich’s ataxia (FA). The milestone follows the U.S. Food and Drug Administration’s (FDA) acceptance of the company’s Investigational New Drug (IND) application in September.
Adjuvia also announced the appointment of Dr. Gregory M. Enns, M.D., Professor of Pediatrics at Stanford University, to its Scientific and Clinical Advisory Board, as well as expansion of its pipeline programs.
ATI-105 Phase 1 Clinical Trial Underway
Friedreich’s ataxia is a rare, inherited neurodegenerative disease associated with mitochondrial dysfunction and progressive neurological and cardiac complications. The first-in-human Phase 1 trial (NCT07869654) is evaluating the safety and tolerability of ATI-105 in healthy volunteers through single ascending dose and multiple ascending dose cohorts.
“FDA clearance of our IND application and first patient dosing is a significant milestone for our company and, more importantly, for individuals living with Friedreich’s ataxia and rare mitochondrial diseases,” said Laura Hix Glickman, Ph.D., Co-founder and Chief Executive Officer of Adjuvia Therapeutics. “Our goal is to develop life-extending therapies for patients with severe mitochondrial diseases, starting with Friedreich’s ataxia.”
Adjuvia plans to advance ATI-105 into Phase 1/2 studies in FA in 2027, with a focus on addressing lethal cardiomyopathy, followed by expansions into Leigh syndrome and primary mitochondrial diseases (PMD) in 2028.
Adjuvia Appoints Dr. Gregory Enns to Scientific and Clinical Advisory Board
Adjuvia is pleased to announce the appointment of Dr. Gregory M. Enns, Professor of Pediatrics in the Division of Medical Genetics at Stanford University and Director of the Biochemical Genetics Program, to its scientific and clinical advisory board. His world-renowned clinical and scientific research experience spans mitochondrial genetic diseases, clinical biochemical genetics, and emerging gene therapies.
“Adjuvia’s technology has tremendous potential to treat multiple disorders that affect energy metabolism. I’m particularly excited about the ATI-105 mitochondrial disease program that focuses on severe neurodegenerative conditions with unmet clinical needs, as well as the ATI-107 approach for safe and systemic delivery of LNP mRNA for gene therapy,” said Dr. Enns.
Adjuvia Announces Expansion of Clinical Pipeline
Adjuvia has launched two new pipeline programs based on its proprietary BBB-penetrant astaxanthin lipid nanoparticle platform. ATI-107 is a novel nanoparticle co-formulation approach designed to enhance systemic delivery of existing lipid nanoparticle (LNP) mRNA and nucleic acid therapeutics, while inhibiting production of LNP-induced inflammation. ATI-107 has the potential to safely administer existing LNP-mRNA vaccines, as well as enable broad systemic delivery of LNP-based nucleic acid gene therapies, without inducing the harmful inflammation that can lead to serious adverse events and has hindered the development of non-viral LNP gene therapy.
Adjuvia also announces ATI-108, a novel lipid nanoparticle formulation of their proprietary astaxanthin that enables aqueous dispersion and is being developed as a commercial reagent for IVF cell culture. With approximately 600,000 mitochondria in a single human oocyte, ATI-108 is designed to rapidly repair mitochondrial damage in transferred oocytes, inhibit oxidative stress during embryo growth to improve IVF outcomes, and give a healthier start to the fetal organs the mitochondria will be transferred into.
These two programs join the company’s broader pipeline that includes ATI-105 for chronic metabolic diseases and longevity, as well as ATI-106, a green tea EGCC molecule incorporated into its novel oral BBB-penetrant lipid nanoparticle platform for neurodegenerative diseases, including Alzheimer’s and Parkinson’s.
Adjuvia is currently exploring pharmaceutical partnerships and licensing opportunities for these programs.
About Adjuvia Therapeutics
Founded in 2023, Adjuvia Therapeutics is a clinical-stage biotechnology company located in San Francisco, CA, developing innovative therapies for mitochondrial dysfunction and disease. Adjuvia’s lead candidate, ATI-105, is a proprietary BBB-penetrant lipid nanoparticle formulation of a novel astaxanthin molecule with superior bioactivity and bioavailability, designed to repair mitochondrial damage by simultaneously addressing oxidative stress, chronic inflammation, and organ degeneration. In preclinical studies across multiple models of mitochondrial disease, ATI-105 demonstrated unprecedented reductions in reactive oxygen species and chronic inflammation, along with evidence of cellular repair, improved organ function, and extended lifespan. ATI-105 is currently in Phase 1 clinical trials for Friedreich’s ataxia, a rare inherited mitochondrial disease with no approved life-extending treatments. Additional trials are planned for Leigh Syndrome and primary mitochondrial diseases (PMD). The company’s broader pipeline includes ATI-106, a novel oral BBB-penetrant lipid nanoparticle formulation of EGCG for neurodegenerative diseases; ATI-107, a novel astaxanthin-LNP nucleic acid platform for vaccines, immunotherapies and non-viral LNP-based gene therapies; and ATI-108, a novel lipid nanoparticle astaxanthin formulation optimized for IVF cell culture.
For more information on Adjuvia Therapeutics, please visit www.adjuvia.com.
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