Bexorg Awarded Grant from The Michael J. Fox Foundation to Identify Translational Biomarkers for TRPML1-Targeted Parkinson’s Disease Therapies

- TRPML1 is a promising target¹ for Parkinson’s disease treatments and identifying translational biomarkers will help confirm target engagement in clinical studies

- Bexorg will leverage its postmortem whole-human brain platform to identify biomarkers that track TRPML1 drug activity in human brain tissue

NEW HAVEN, Conn., Jan. 28, 2026 (GLOBE NEWSWIRE) -- Bexorg, Inc., the first integrated AI and whole-human brain platform company transforming central nervous system (CNS) drug discovery and development, today announced that it has been awarded a research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) through the Targets to Therapies Initiative to identify translational biomarkers for TRPML1-targeted therapies.

MJFF has convened leading experts to prioritize promising therapeutic targets for Parkinson’s disease. Among these, TRPML1 has emerged as a compelling target for drug candidates.¹ However, to date, the lack of translational biomarkers to confirm target engagement in clinical trials poses a significant obstacle to the advancement of TRPML1-targeted therapies. To address this gap, MJFF is providing funding to Bexorg to apply its proprietary BrainEx platform, the world’s first postmortem whole-human brain platform, to identify such biomarkers in brains donated by people who lived with Parkinson’s disease.

“Through this grant, MJFF is supporting Bexorg to leverage its postmortem whole-human brain platform in advancing TRPML1 research to bridge the translational gap between preclinical drug discovery and advancement of therapies in clinical trials,” said Paul Wes, Ph.D., Vice President of Drug Discovery at Bexorg. “With multiple biotechnology and pharmaceutical companies pursuing TRPML1-targeted programs, our collaboration with MJFF aims to accelerate the entire field toward developing disease-modifying therapies for people living with Parkinson’s disease.”

“Validating TRPML1 will close important translational gaps and inform multiple therapeutic programs,” said Shalini Padmanabhan, Ph.D., Senior Vice President of Translational Research at MJFF. “Bexorg’s capabilities will help advance this work and may also inform future efforts to evaluate additional targets for Parkinson’s therapies.”

Terms of the grant were not disclosed. The postmortem whole brains will be sourced from LifeNet Health, an organ procurement organization that provides high-quality, ethically sourced human biological materials for research.

Overcoming the Translational Gap in CNS Drug Discovery
Industry reports have found that the clinical trial failure rate in CNS drug development exceeds 95%, largely because cellular and preclinical models used in drug discovery fail to accurately represent the complexity and biology of the human brain. Bexorg is shifting this paradigm with its platform built entirely on human-derived data generated from wet-lab experimentation in physiologically maintained postmortem whole human brains from people who suffered from neurological disorders or passed away from unrelated causes. This unique data set directly powers AI models that deliver high-fidelity target identification, novel biomarker discovery, and go/no-go decisions with unprecedented accuracy and reliability.

Since its founding in 2021, Bexorg has rapidly advanced its drug discovery platform. Over the last 18 months, the company has expanded its proprietary data set to include hundreds of whole-brain experiments spanning Alzheimer’s disease, Parkinson’s disease, and other neurodegenerative conditions, creating one of the world’s largest repositories of human CNS data. Building on continuous growth of its data set, Bexorg is now training foundation models on petabyte-scale human brain molecular data, with the goal to establish the first AI system grounded in experimentally measured human neurobiology, and redefining how CNS therapies are discovered, validated, and advanced to the clinic.

About Bexorg
Bexorg is a privately held company transforming CNS drug discovery and development with the world’s first integrated AI and whole-human brain platform. Founded by Dr. Zvonimir Vrselja and Dr. Nenad Sestan at Yale University, Bexorg integrates its BrainEx platform with advanced AI models to create one of the most clinically predictive systems in neuroscience. The company operates under rigorous legal framework and bioethics standards, guided by an independent board of experts. For more information, visit www.bexorg.com or follow us on LinkedIn.

About The Michael J. Fox Foundation’s “Targets to Therapies Initiative”
The Michael J. Fox Foundation for Parkinson’s Research (MJFF) launched its Targets to Therapies Initiative in 2024 to expand the number of promising, druggable biological targets within the Parkinson’s disease (PD) translational pipeline. Leveraging MJFF’s many years of experience supporting target validation and advancement, the initiative takes a fresh approach combining community-sourced target nomination with expert panel-guided prioritization and validation roadmaps to deliver targets that are ripe for Parkinson’s therapeutic development. These targets stem from efforts across the field, including rich inputs from MJFF research program datasets and collaboration with the Aligning Science Across Parkinson’s (ASAP) initiative-supported programs including the Collaborative Research Network (CRN), the Parkinson’s Progression Markers Initiative (PPMI) and the Global Parkinson’s Genetics Program (GP2). Learn more here.

Reference
¹ Pollmanns MR, et al. Activated Endolysosomal Cation Channel TRPML1 Facilitates Maturation of α-Synuclein-Containing Autophagosomes. Front Cell Neurosci. 2022 Jul 6;16:861202. doi: 10.3389/fncel.2022.861202. PMID: 35875350; PMCID: PMC9296810.

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JPA Health 
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Sarah McCabe
JPA Health
smccabe@jpa.com

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