NRG raises £50m to start trials of Parkinson's, ALS drug

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Two cross-sections of mitochondria
Louisa Howard, Dartmouth College, via Wikipedia

Transmission electron microscope image of a thin section cut through an area of mammalian lung tissue. The high magnification image shows two mitochondria.

NRG Therapeutics has secured the funding it needs to take a potentially first-in-class therapy for neurodegenerative diseases from preclinical development into human testing.

The Stevenage, UK-based biotech has closed a £50 million ($67 million) Series B – three years after its £16 million first round – which it said would give it the resources needed to advance lead candidate NRG5051 through initial proof-of-concept (PoC) testing in amyotrophic lateral sclerosis (ALS, also known as motor neurone disease or MND), as well as Parkinson's disease.

NRG's R&D pipeline is focused on brain-penetrant, small-molecule inhibitors of the mitochondrial permeability transition pore (mPTP), which forms in the inner membrane of mitochondria – the energy-producing structure in the cell – leading to disruption of their activity and cell death.

According to NRG, the opening of the mPTP leads to inflammation and neuronal death in neurodegenerative diseases, and it has a pipeline of small molecules that have been shown to inhibit pore opening through a novel mechanism of action.

Clinical testing of the hypothesis that preventing mPTP opening can have a neuroprotective effect in ALS and Parkinson's, and significantly reduce neuroinflammation, will start early next year, according to the company.

The company believes that the two pathological proteins in the two diseases – alpha-synuclein in Parkinson's and TDP-43 in ALS – are toxic to mitochondria and contribute to the mitochondrial dysfunction that occurs in the two diseases.

NRG5051 was selected as its lead candidate last October, and the clinical programme is also being supported by a $5 million grant from the Michael J Fox Foundation that is going towards initial manufacturing and toxicology work, the development of a PET tracer to track where it is working in the central nervous system, and identifying and validating biomarkers that can be used as surrogate endpoints in clinical trials.

"Developing new drugs to treat neurological diseases is very challenging, but is receiving increased interest given the high unmet medical need and growing prevalence in ageing populations," said NRG's co-founder and chief executive, Neil Miller.

"These new funds provide the runway to advance our lead programme through PoC in ALS/MND, and to develop our portfolio of small molecule candidate drugs for other indications, including Parkinson's."

The Series B was led by SV Health Investors' Dementia Discovery Fund, and included new investors British Business Bank, M Ventures, Novartis Venture Fund, and Criteria Bio Ventures, along with existing investors Omega Funds and Brandon Capital.

In the first half of this year, the UK life sciences sector raised £1.23 billion in new venture capital backing, putting it on course to at least match the 2024 total for the full year, although, there have been signs of a slowdown in the second and third quarters.