NRG Therapeutics Raises $67 Million to Advance Novel Mitochondrial-Targeting Therapy for Parkinson's and ALS
核心洞察
NRG Therapeutics (搜索) secured £50 million ($67 million) in Series B funding to advance its lead compound NRG5051 into clinical trials for Parkinson's disease (搜索) and ALS (搜索).
The therapy targets mitochondrial permeability transition pore (搜索) (mPTP (搜索)) dysfunction, a pathology common to nearly all neurodegenerative diseases (搜索) that leads to inflammation and cell death.
Phase 1 clinical trials could begin as early as December 2025, supported by additional funding from the Michael J. Fox Foundation (搜索) for manufacturing and biomarker development.
UK-based NRG Therapeutics (搜索) has raised £50 million ($67 million) in Series B financing to advance its potentially first-in-class therapy for neurodegenerative diseases (搜索) into human testing. The funding round, led by SV Health Investors' Dementia Discovery Fund (搜索), will enable the company to take lead compound NRG5051 through initial proof-of-concept testing in amyotrophic lateral sclerosis (搜索) (ALS (搜索)) and Parkinson's disease (搜索).
The financing comes during a challenging period for the biotechnology industry and includes participation from the venture arms of major pharmaceutical companies Novartis and Merck KGaA, alongside new investors British Business Bank, M Ventures, and Criteria Bio Ventures. CEO Neil Miller noted that the fundraising "points to the interest that pharma has" in the firm's approach to tackling neurodegenerative diseases (搜索).
Novel Approach to Mitochondrial Dysfunction
NRG's therapeutic strategy centers on addressing mitochondrial dysfunction, which Miller describes as "common to nearly all neurodegenerative diseases (搜索)." The company has developed brain-penetrant, small-molecule inhibitors targeting the mitochondrial permeability transition pore (搜索) (mPTP (搜索)), which forms in the inner membrane of mitochondria and disrupts cellular energy production.
When the mPTP (搜索) opens, it leads to inflammation and neuronal death in neurodegenerative diseases (搜索). The company believes that pathological proteins in these conditions—alpha-synuclein (搜索) in Parkinson's disease (搜索) and TDP-43 in ALS (搜索)—are toxic to mitochondria and contribute to the mitochondrial dysfunction observed in both diseases.
The biological pathway involving mPTP (搜索) is well-established but has proven difficult to target therapeutically. To overcome this challenge, Miller explains that the team "went back to old-fashioned drug discovery" using phenotypic screening to identify a series of small molecules. This approach led to the identification of NRG5051, which potently and selectively binds to a specific protein target (referred to as protein A) while maintaining the ability to cross the blood-brain barrier.
Clinical Development Timeline
NRG5051 was selected as the lead candidate in October 2024, and Phase 1 clinical trials could commence as early as December 2025. The clinical program is receiving additional support through a $5 million grant from the Michael J. Fox Foundation (搜索), which will fund initial manufacturing and toxicology work.
The foundation grant will also support the development of a labeled form of the drug candidate for positron-emission tomography (PET) imaging, allowing researchers to track where the therapy is active in the central nervous system. The company plans to use some of the clinical trial data to validate biomarkers for use as surrogate endpoints in later studies.
Company Background and Pipeline
NRG Therapeutics (搜索) was co-founded in 2018 by seasoned drug hunter Neil Miller alongside biotech entrepreneurs Grant Hawthorne and Richard Rutter. The Stevenage-based company initially received seed funding from Parkinson's UK, a nonprofit organization, and has successfully secured additional grant funding from organizations including the Michael J. Fox Foundation (搜索).
Beyond NRG5051, the company is developing a broader pipeline of mPTP (搜索)-targeting compounds. Miller indicated that NRG has backup molecules from the same series as its lead candidate, as well as another series of compounds with different pharmacological properties.
The Series B funding represents a significant increase from the company's £16 million Series A round completed three years ago. Miller emphasized that developing new drugs for neurological diseases is "very challenging, but is receiving increased interest given the high unmet medical need and growing prevalence in ageing populations."
The new funding will provide NRG with the resources needed to advance its lead program through proof-of-concept testing while continuing to develop its portfolio of small molecule candidates for additional indications beyond ALS (搜索) and Parkinson's disease (搜索).