NRG Therapeutics is advancing first-in-class, orally bioavailable, and CNS-penetrant mitochondrial permeability transition pore (mPTP) inhibitors to slow or halt neurodegeneration.
Our proprietary small molecules bind to NLRX1—a key mPTP component or modulator of mitochondrial permeability transition—demonstrating profound neuroprotective and anti-inflammatory activity in in vivo preclinical models of ALS and Parkinson’s.
Our pipeline features structurally distinct chemical series capable of delivering multiple differentiated clinical assets for ALS, Parkinson’s and potentially other disease indications.
| Discovery | Lead Optimization | IND Enabling | Phase 1 | Phase 2 |
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NRG5051-101 clinical trial
NRG5051, our first-in-class oral mPTP inhibitor, is in a Phase 1 trial assessing safety in healthy adults and people with Parkinson’s disease or ALS.
EUCT number: 2025-523872-22-00
We plan to progress NRG5051 into Phase 2 clinical proof-of-concept (PoC) trials in ALS and in parallel generate data demonstrating its potential use in Parkinson’s.
NRG5051 has the potential to become the first disease-modifying therapeutic for both sporadic ALS and Parkinson’s.
NRG is unique in targeting NLRX1 with small-molecule inhibitors.
Clinical and biomarker strategy
We are developing a biomarker strategy to support clinical development of NRG5051 and potentially patient stratification across ALS and Parkinson’s.
To demonstrate NRG5051’s neuroprotective benefit in ALS patients, we will measure a reduction in neurofilament light (NfL) chain, a fluid biomarker which is released following neuronal damage and axonal injury in the central nervous system. Additionally, we are developing other fluid biomarkers to monitor mitochondrial function and activation of the innate immune system, alongside evaluating potential imaging modalities such as positron emission tomography (PET) to demonstrate target engagement.
NRG5051 is an investigational drug product
NRG5051 is undergoing clinical trials to evaluate the safety and effectiveness in humans.
It has not received marketing authorization or approval by any regulatory agency, including the US Food and Drug Administration and the European Medicines Agency.
© NRG Therapeutics
