UK-based NRG Therapeutics has raised US$67m (£50m) in series B financing to take its lead drug for Parkinson’s disease and ALS into first-in-human clinical trials.
The fundraising round was led by the Dementia Discovery Fund, alongside the venture arms of Novartis and Merck KGaA.
The series B funding will support Phase 1 trials of NRG5051, which could begin as early as December.
The funding will also support development of a labelled form of NRG5051 for positron-emission tomography (PET) imaging, which would allow researchers to track the compound in the body.
The company’s approach targets mitochondrial dysfunction – damage to the cell’s energy-producing structures – which occurs in almost all neurodegenerative diseases.
NRG has focused on the mitochondrial permeability transition pore (mPTP), a channel that, when opened, triggers inflammation and cell death.
While this pathway is well known, designing drugs to influence it has proved difficult.
Chief executive Neil Miller said the team “went back to old-fashioned drug discovery” by using phenotypic screening – testing compounds on cells to observe their effects – to find a series of small molecules.
Further work identified the specific protein target, which Miller would only describe as “protein A”, and the lead compound NRG5051, which binds selectively to the protein and is able to cross the blood-brain barrier – the protective membrane that blocks most substances from reaching the brain.
Miller said the investment “points to the interest that pharma has” in the company’s approach to tackling neurodegenerative conditions.
The company aims to use part of the trial to validate a biomarker – a measurable indicator of disease – to help guide future studies.
Miller and fellow biotech entrepreneurs Grant Hawthorne and Richard Rutter founded NRG in 2018 with seed funding from Parkinson’s UK, a nonprofit.
Since then, the firm has secured grants from Parkinson’s UK and the Michael J. Fox Foundation, along with private investment.
Miller said NRG also plans to expand its mPTP-targeting pipeline, with backup molecules from the same series as NRG5051 and a second series of compounds with different pharmacology.
