NRG Therapeutics selects first development candidate, NRG5051
Stevenage, UK, 31 October 2024 - 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.
