Adjuvia Therapeutics Doses First Participant in Phase 1 Trial of ATI-105 for Friedreich’s Ataxia
Adjuvia Therapeutics, a biotechnology company focused on developing novel treatments for mitochondrial dysfunction and associated diseases, has announced that the first participant has been dosed in its Phase 1 clinical trial of ATI-105, the company’s lead investigational therapeutic candidate for Friedreich’s ataxia (FA). The milestone marks an important step in the clinical development of ATI-105 following the U.S. Food and Drug Administration’s (FDA) acceptance of the company’s Investigational New Drug (IND) application in September 2026.
Alongside the clinical trial update, Adjuvia announced the appointment of Gregory M. Enns, M.D., Professor of Pediatrics at Stanford University, to its Scientific and Clinical Advisory Board. The company also disclosed the expansion of its development pipeline with two additional programs, ATI-107 and ATI-108, designed to address challenges in nucleic acid delivery, gene therapy and mitochondrial health.
The announcements reflect Adjuvia’s broader strategy to develop therapies targeting mitochondrial dysfunction while expanding the potential applications of its proprietary astaxanthin lipid nanoparticle technology. The company’s research programs span rare genetic diseases, neurodegenerative disorders, metabolic health and reproductive medicine.
ATI-105 Phase 1 Clinical Trial Begins
The first-in-human Phase 1 clinical trial of ATI-105 is now underway, evaluating the safety and tolerability of the investigational candidate in healthy volunteers. The study is registered on ClinicalTrials.gov under identifier NCT07869654 and includes both single ascending dose and multiple ascending dose cohorts.
The trial is designed to generate early clinical data on how ATI-105 is tolerated at different dose levels. Single ascending dose cohorts generally evaluate the safety of progressively higher doses administered on one occasion, while multiple ascending dose cohorts assess tolerability following repeated administration over a specified period.
These approaches are commonly used in early-stage clinical development to help researchers characterize a candidate’s safety profile and determine appropriate doses for subsequent studies. Depending on the study design and available data, Phase 1 trials may also assess pharmacokinetic characteristics, including how a candidate is absorbed, distributed, metabolized and eliminated from the body.
The initial study in healthy volunteers is an important step before ATI-105 advances into trials involving people with Friedreich’s ataxia. The results will help inform the company’s next development decisions, although safety and tolerability findings in healthy participants will not by themselves establish whether the treatment is effective in patients.
Adjuvia intends to advance ATI-105 into Phase 1/2 studies in Friedreich’s ataxia in 2027. The planned program will focus on addressing cardiomyopathy associated with the disease, a major contributor to serious complications and premature mortality among affected individuals.
The company also plans to explore additional applications for ATI-105 in Leigh syndrome and primary mitochondrial diseases in 2028. These future programs are intended to extend the candidate’s potential reach across rare disorders characterized by impaired mitochondrial function, subject to further development and clinical evaluation.
Friedreich’s Ataxia and the Need for New Treatments
Friedreich’s ataxia is a rare inherited neurodegenerative disease that affects the nervous system and can also cause significant cardiac complications. The condition is associated with abnormalities in mitochondrial function, which can interfere with the body’s ability to produce and manage the energy required by cells.
Mitochondria are specialized structures within cells that play a central role in energy production. They also contribute to several other essential biological processes. Because tissues such as the nervous system and heart have substantial energy requirements, mitochondrial dysfunction can have particularly serious consequences for these organs.
Friedreich’s ataxia is commonly associated with progressive problems involving coordination, balance and movement. Many affected individuals experience difficulties with walking and other motor functions as the disease advances. Additional complications can include scoliosis, diabetes and cardiomyopathy, although the severity and combination of symptoms vary among patients.
Cardiac disease is a particularly important concern. Friedreich’s ataxia can cause cardiomyopathy, in which the heart muscle becomes structurally or functionally abnormal. Cardiac complications can progress over time and may contribute substantially to the disease’s overall burden.
The condition is associated with abnormalities involving the FXN gene, which provides instructions for producing frataxin, a protein essential to mitochondrial function. Reduced frataxin levels disrupt normal cellular processes and contribute to mitochondrial stress and damage.
Although treatment and supportive care can help manage certain symptoms and complications, significant unmet medical needs remain. Researchers continue to investigate approaches that could address underlying disease mechanisms, preserve organ function and improve long-term outcomes.
ATI-105 is being developed within this broader effort to target mitochondrial dysfunction. However, the candidate remains investigational, and its clinical benefits, safety profile and potential impact on disease progression have yet to be established through clinical trials.
Company Leadership Highlights Development Milestone
Laura Hix Glickman, Ph.D., Co-founder and Chief Executive Officer of Adjuvia Therapeutics, emphasized the importance of beginning clinical evaluation of ATI-105 following the FDA’s acceptance of the IND application.
“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 Glickman.
“Our goal is to develop life-extending therapies for patients with severe mitochondrial diseases, starting with Friedreich’s ataxia,” she added.
The first participant dosing represents a transition from preclinical development to human clinical investigation. For biotechnology companies developing treatments for rare diseases, this stage is particularly important because it begins the process of evaluating whether laboratory findings and preclinical observations translate into an acceptable safety profile in humans.
The company plans to use the results from its early clinical program to guide further development of ATI-105. The proposed 2027 Phase 1/2 program will represent a subsequent step toward evaluating the candidate in individuals with Friedreich’s ataxia, including its potential relevance to disease-associated cardiac complications.
The planned expansion into Leigh syndrome and other primary mitochondrial diseases would broaden the program’s scope if supported by the necessary scientific, regulatory and clinical evidence.
Dr. Gregory Enns Joins Scientific and Clinical Advisory Board
Adjuvia has appointed Gregory M. Enns, M.D., to its Scientific and Clinical Advisory Board to strengthen the company’s scientific and clinical expertise as it advances its development programs.
Dr. Enns is a Professor of Pediatrics in the Division of Medical Genetics at Stanford University and Director of the Biochemical Genetics Program. His clinical and research experience includes mitochondrial genetic diseases, clinical biochemical genetics and emerging gene therapies.
His background is relevant to Adjuvia’s focus on disorders involving mitochondrial dysfunction and impaired energy metabolism. Expertise in medical genetics and biochemical genetics can help inform the evaluation of disease mechanisms, clinical manifestations and potential therapeutic approaches for inherited metabolic conditions.
As a member of the advisory board, Dr. Enns is expected to contribute scientific and clinical perspectives to the company’s research and development strategy. His experience in mitochondrial disease may be particularly relevant as Adjuvia prepares to move ATI-105 beyond its initial healthy-volunteer study and toward potential patient trials.
Dr. Enns also highlighted the potential applications of the company’s broader technology platform, including its efforts to improve the delivery of lipid nanoparticle-based messenger RNA therapeutics.
“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.
His appointment comes as Adjuvia expands its pipeline beyond its lead mitochondrial disease candidate, with new programs targeting nucleic acid delivery and mitochondrial support in reproductive medicine.
ATI-107 Targets Lipid Nanoparticle Delivery and Inflammation
One of the company’s newly announced programs, ATI-107, is based on a nanoparticle co-formulation approach intended to improve the systemic delivery of existing lipid nanoparticle (LNP) messenger RNA and other nucleic acid therapeutics.
Lipid nanoparticles are widely used as delivery vehicles for nucleic acids, including messenger RNA and other genetic materials. These systems can protect fragile molecules and help transport them into cells, where they may produce a therapeutic effect or influence cellular processes.
LNP technology has become an important component of modern drug development, particularly in mRNA vaccines and emerging genetic medicines. Nevertheless, challenges involving delivery efficiency, tissue distribution, immune activation and tolerability continue to influence the development of broader applications.
Adjuvia is developing ATI-107 to enhance the systemic delivery of existing LNP-based mRNA and nucleic acid therapeutics while inhibiting the production of LNP-induced inflammation.
According to the company, the program is intended to address inflammatory responses that can limit the use of certain nanoparticle delivery systems. Unwanted immune activation is an important consideration in the development of nucleic acid medicines because it can affect tolerability, restrict repeat administration or complicate the delivery of therapeutic cargo to intended tissues.
ATI-107 is designed to support the potential administration of existing LNP-mRNA vaccines and enable broader systemic delivery of LNP-based nucleic acid gene therapies. The company believes the approach could help address obstacles that have hindered the development of non-viral LNP gene therapy.
The platform is also associated with Adjuvia’s proprietary astaxanthin lipid nanoparticle technology, which the company describes as capable of crossing the blood-brain barrier. The blood-brain barrier is a selective biological interface that limits the movement of many substances from the bloodstream into the central nervous system, making it a major challenge for neurological drug development.
The company’s broader interest in this technology is to support delivery approaches that could be useful across different therapeutic areas.
ATI-107 remains a development-stage program, and its potential to improve delivery, reduce inflammation or enable new gene therapy applications will require further experimental and clinical validation. The company’s stated objectives should therefore be viewed as development goals rather than established clinical outcomes.
ATI-108 Designed for Mitochondrial Support in IVF
Adjuvia’s second new program, ATI-108, is being developed as a lipid nanoparticle formulation of the company’s proprietary astaxanthin. The formulation is designed to enable aqueous dispersion and is intended for development as a commercial reagent for in vitro fertilization (IVF) cell culture.
IVF involves fertilizing an egg with sperm outside the body and subsequently transferring an embryo into the uterus. The quality and developmental competence of oocytes, or egg cells, are important factors in reproductive outcomes. Cellular energy production and the regulation of oxidative stress are among the biological processes relevant to oocyte function and early embryonic development.
Mitochondria play an important role in these processes because they supply energy required for cellular activity. Adjuvia notes that a single human oocyte contains approximately 600,000 mitochondria, illustrating the substantial mitochondrial content of these cells.
Oxidative stress occurs when the production of reactive molecules exceeds the body’s ability to manage them through its antioxidant and protective systems. Excessive oxidative stress can damage cellular components, including lipids, proteins and genetic material. In reproductive biology, researchers continue to investigate how oxidative balance and mitochondrial function influence oocyte quality and embryo development.
ATI-108 is designed to address these areas by supporting mitochondrial health and reducing oxidative stress during embryo growth. The company aims to develop the formulation for use in IVF cell culture, with the goal of improving conditions that support early developmental processes.
Adjuvia’s stated development objectives include rapidly repairing mitochondrial damage in transferred oocytes, limiting oxidative stress during embryo growth and potentially supporting healthier fetal development.
These objectives represent the intended applications of ATI-108 rather than demonstrated clinical benefits. Evidence from appropriate laboratory studies and subsequent development will be necessary to establish whether the formulation can improve mitochondrial function, affect embryo quality or lead to better IVF outcomes.
The program adds a reproductive medicine component to Adjuvia’s pipeline, complementing its work in rare mitochondrial diseases, neurodegeneration and nucleic acid delivery.
Broader Pipeline Includes Neurodegenerative and Metabolic Disease Programs
ATI-107 and ATI-108 join Adjuvia’s existing pipeline, which includes ATI-105 and ATI-106.
ATI-105 is the company’s lead investigational candidate and is being developed initially for Friedreich’s ataxia, with additional potential applications in Leigh syndrome and primary mitochondrial diseases. The ongoing Phase 1 study will provide the initial human safety and tolerability data needed to inform further development.
ATI-106 is based on a green tea-derived epigallocatechin gallate (EGCG) molecule incorporated into Adjuvia’s oral lipid nanoparticle platform, which the company describes as capable of crossing the blood-brain barrier. The program is intended for neurodegenerative diseases, including Alzheimer’s disease and Parkinson’s disease.
Alzheimer’s disease is a progressive neurological disorder associated with cognitive decline, while Parkinson’s disease is characterized by the progressive loss or dysfunction of neurons involved in movement regulation. Both conditions represent substantial areas of unmet medical need, and researchers continue to investigate multiple biological pathways that could support new therapeutic approaches.
Adjuvia’s interest in these conditions reflects its broader focus on mitochondrial function, oxidative stress and the delivery of therapeutic compounds to tissues that may be difficult to reach using conventional formulations.
The company has also described ATI-105 as part of a broader strategy involving chronic metabolic diseases and longevity-related research. The specific clinical status and development plans for these broader applications will depend on further research and supporting evidence.
Together, the programs reflect an effort to apply the company’s technology to several different biological challenges. However, each candidate will require its own development pathway, with evidence of safety, efficacy and an appropriate benefit-risk profile established for the intended use.
Partnership and Licensing Opportunities
Adjuvia Therapeutics is exploring pharmaceutical partnerships and licensing opportunities for its development programs. Such arrangements can provide biotechnology companies with access to additional research capabilities, development expertise, manufacturing infrastructure and commercialization resources.
Partnerships may be particularly important for companies working across multiple therapeutic areas, where clinical development and regulatory requirements can differ considerably between programs.
For Adjuvia, potential collaborations could support the advancement of ATI-105 in rare mitochondrial diseases, the development of ATI-107 for nucleic acid delivery and the evaluation of ATI-108 for IVF cell culture applications. Licensing arrangements could also enable other companies to assess or develop particular technologies for specific indications or markets.
The company’s expanded pipeline and the appointment of Dr. Enns represent additional developments as it pursues these opportunities.
Next Steps in Adjuvia’s Development Strategy
The initiation of the ATI-105 Phase 1 trial marks a significant development milestone for Adjuvia Therapeutics as it begins evaluating its lead candidate in humans. The company’s next major objective is to generate initial safety and tolerability data and use the findings to inform its planned Phase 1/2 program in Friedreich’s ataxia in 2027.
Beyond ATI-105, Adjuvia is advancing a broader research strategy focused on mitochondrial health, neurodegenerative disease and delivery technologies for nucleic acid therapeutics. ATI-107 and ATI-108 expand this strategy into LNP-mediated delivery and reproductive medicine, while ATI-106 maintains a focus on neurological conditions.
The addition of Dr. Gregory Enns to the Scientific and Clinical Advisory Board provides the company with further expertise in mitochondrial genetics, biochemical genetics and emerging gene therapies as it develops these programs.
Although all four programs remain subject to their respective development requirements, Adjuvia’s latest announcements establish the direction of its research portfolio and its intention to pursue collaborations that could help advance the candidates.
For people affected by Friedreich’s ataxia and other rare mitochondrial disorders, the development of new therapeutic approaches remains an important area of medical research. The clinical evaluation of ATI-105 will help determine whether Adjuvia’s approach can progress toward a treatment option capable of addressing the serious complications associated with mitochondrial disease.
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.