- First development candidate selected, NRG5051, with potential to treat Parkinson’s and Amyotrophic Lateral Sclerosis (ALS) (also known as Motor Neuron Disease, MND) and other neurodegenerative diseases
- NRG5051 prevents mitochondrial dysfunction via inhibition of the mitochondrial permeability transition pore (mPTP) through a novel protein target
- Inhibition of the mPTP in the brain prevents neuronal cell death and reduces neuroinflammation in animals
- $5M grant from The Michael J. Fox Foundation (MJFF) to advance NRG5051 into the clinic
- Grant will support CMC, GLP Toxicology, development of a PET tracer and building a biomarker strategy for Parkinson’s
- NRG5051 anticipated to enter first-in-human clinical studies by end of 2025
STEVENAGE, United Kingdom, Oct. 31, 2024 (GLOBE NEWSWIRE) -- NRG Therapeutics, Ltd., an innovative neuroscience company targeting mitochondrial dysfunction, is pleased to announce that it has achieved a key milestone with the nomination of its first development candidate, NRG5051 and has been awarded a $5M grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to 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).
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 (a-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.”
Alexandra Vaiana, PhD, MJFF’s senior scientific portfolio manager, said, “MJFF continues to fund critical pre-clinical research and development. We look forward to seeing the results of NRG’s first-in-class mPTP inhibitor as a novel disease-modifying treatment for Parkinson’s.”
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.
Media enquiries (for NRG Therapeutics)
Sue Charles, Charles Consultants - +44 7968 726585 sue@charles-consultants.com
About NRG Therapeutics – http://www.nrgtherapeutics.com
NRG Therapeutics is a neuroscience drug discovery company building a pipeline of disease-modifying mitochondrial therapeutics to slow or halt the progression of neurodegenerative diseases such as Parkinson’s and Amyotrophic Lateral Sclerosis (ALS), also known as motor neuron disease (MND).
The company’s pre-clinical pipeline of small molecules is based on inhibiting the mitochondrial permeability transition pore (mPTP) through a novel mechanism of action. Inhibition of the mPTP has been shown to protect mitochondria, reduce neuroinflammation and prevent neuronal death in pre-clinical disease models.
Based at the Stevenage Bioscience Catalyst (SBC), UK, 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).
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