NRG Therapeutics launches Series B after $5m boost from Michael J. Fox Foundation

The new grant will support the development of NRG5051 as a novel disease-modifying treatment for Parkinson’s disease. NRG5051 also has potential in other neurological diseases such as ALS (also known as MND).

NRG Therapeutics has embarked on a series B financing to fund progression of NRG5051 into the clinic and through a Proof-of-Mechanism study in patients.

NRG5051 is a first-in-class inhibitor of the mitochondrial permeability transition pore (mPTP) and is designed to penetrate the brain when taken orally.

It has been shown in vitro to protect mitochondrial function via inhibition of a novel protein that is essential for pore opening and thus prevent neuronal cell death. In pre-clinical models of Parkinson’s and ALS, NRG5051 is neuroprotective and significantly reduces the neuroinflammation observed in these diseases.

NRG’s grant from MJFF’s Therapeutics Pipeline Program will fund the completion of key IND-enabling studies with the aim of advancing NRG5051 into first-in-human clinical studies in 2025.

This new grant will support the manufacture of drug substance for both 28-day GLP-toxicology studies and Phase 1 first-in-human human clinical trials. It will also enable the development of a safe low-dose PET tracer that can be used to demonstrate CNS target engagement of NRG5051 in Phase 1 clinical trials.

In addition, it will support the identification and validation of new biomarker(s) that will be used in the clinic to determine if NRG5051 is inhibiting the mPTP in the brain and producing the desired biological effects.

Mitochondria, the batteries of cells, are crucial for energy production, especially in brain cells. Substantia nigra neurons (Parkinson’s) and motor neurons (ALS) have very high energy demands and consequently are particulary sensitive to mitochondrial health.

Furthermore, it is now known that the pathological proteins in Parkinson’s (α-synuclein) and ALS (TDP-43) are toxic to mitochondria and contribute to the mitochondrial dysfunction which is a common underlying pathology in neurodegenerative diseases. Inhibition of mPTP opening has been shown to protect mitochondria from this gain-of-function protein toxicity and to preserve neurons in pre-clinical models.

NRG Therapeutics co-founder and CEO Dr Neil Miller said:“The selection of our first development candidate is a major milestone for the company. NRG5051 has been shown in vivo in pre-clinical models of Parkinson’s and ALS to prevent the death of brain cells and to reduce neuroinflammation, and we are excited by its potential to halt or significantly slow the progression of disease in individuals with Parkinson’s and ALS.

“We are grateful for MJFF grant support to help advance NRG5051 into the clinic as quickly as possible.”

Based at the Stevenage Bioscience Catalyst (SBC), NRG Therapeutics is a private company with equity investment from Parkinson’s UK, Omega Funds and Brandon Capital.

The company has also received grant funding from Innovate UK (Biomedical Catalyst Award), The Michael J. Fox Foundation and Target ALS, and is the industrial partner for a FightMND Drug Discovery grant awarded to WEHI (Walter and Eliza Hall Institute of Medical Research).