Stevenage, UK, 22 February 2023 - NRG Therapeutics, Ltd., an innovative neuroscience company targeting mitochondrial dysfunction, is pleased to announce that it has been awarded a second $500,000 grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF), to support its lead drug discovery programme and the development of a novel treatment for Parkinson’s.
NRG Therapeutics is applying breakthrough science in the field of mitochondrial biology to develop first-in-class treatments for Parkinson’s. Its approach is based on inhibiting the mitochondrial permeability transition pore (mPTP) in brain cells, which has been shown to be neuroprotective in several preclinical models of Parkinson’s and other neurodegenerative diseases.
Having met the goals of the grant it received from MJFF in 2021, this second grant will support experiments to validate a novel mitochondrial drug target for Parkinson’s. The work will determine whether genetic deletion of this mitochondrial protein in vitro and in vivo protects brain cells, and therefore, whether drug modulation of this protein has the potential to improve Parkinson’s symptoms. The overall goal of the programme is to develop drugs that block the opening of the mPTP, an event which is triggered by high concentrations of calcium ions, leading to the death of dopamine-producing brain cells.
NRG Therapeutics’ co-founder and CEO Dr Neil Miller said, “We have made great progress with our research funded by MJFF and we are delighted to have received a second award to fund further research to validate the molecular target for our mPTP inhibitors. There is a significant unmet medical need as all current treatments for Parkinson’s control symptoms but do not halt or slow progression of disease. Truly disease-modifying treatments for Parkinson’s and other neurodegenerative diseases remain the holy grail. It is our hope that our work could lead to new treatments that could positively change the lives of individuals with neurodegenerative disorders.”
Classical ‘first-generation’ inhibitors of the mPTP, such as cyclosporin A, inhibit cyclophilin D (CypD), a protein that is believed to regulate the pore. However, all historical attempts to develop CypD inhibitors that can cross the blood-brain barrier have been unsuccessful.
NRG has discovered novel ‘second-generation’ mPTP inhibitors that are small drug-like molecules, orally bioavailable and brain penetrant, and therefore have the potential for use in the treatment of chronic neurodegenerative disorders. NRG’s lead assets were identified through phenotypic screening in isolated mitochondria and have been shown to act independently of CypD.
“Our mission is to support development of life-changing treatments to fulfill the unmet needs of people with Parkinson’s disease, and we are pleased to provide funding for a second project with NRG toward that goal,” said Alexandra Vaiana, PhD, MJFF’s Associate Director of Research Programs.