Science

Pipeline

Numerous opportunities to change patients’ lives

ProgramIndicationDiscoveryIND enablingPhase 1
Anti-Nitrated Alpha Synuclein Antibody Parkinson's Disease

NDC-0524

 
Synuclein Nitrase Inhibitor Parkinson's Disease

 
Platform Oncology

 
Anti-Nitrated Alpha Synuclein Antibody
Parkinson's Disease
DiscoveryIND enablingPhase 1

NDC-0524

Synuclein Nitrase Inhibitor
Parkinson's Disease
DiscoveryIND enablingPhase 1

Platform
Oncology
DiscoveryIND enablingPhase 1

Parkinson’s Disease

A key example of the power of our NITROME platform is our discovery of Synuclein Nitrase (the specific synuclein nitration enzyme). This represents a significant scientific breakthrough that provides an ideal therapeutic target to halt Parkinson’s disease (PD). Currently, there is no effective intervention to slow, halt or reverse the progression of Parkinson’s disease.

The buildup of aggregated alpha-synuclein is a hallmark of PD. Our data suggest a key role for the nitration of alpha synuclein in mediating disease pathology and dysfunction in PD.

These synuclein aggregates are heavily nitrated and this nitration strongly induces neurodegenerative disease in preclinical models, promoting neuronal death. Additionally, we have found that nitrated synuclein is elevated in the cerebral spinal fluid of PD patients suggesting it could be the pathogenic species driving spread between brain regions.

We have developed antibodies against nitrated synuclein, and data presented at the AD/PD conference in March 2024 demonstrated that our first-in-class antibody reduced PD progression in in vitro and in vivo PD models. The antibody significantly reduced the spread of alpha-synuclein pathology from one area of the brain to another. Based on these data, we are advancing our development candidate NDC-0524 into IND-enabling studies.

Download AD/PD 2024 Presentation

Cancer

The NITROME platform has also identified several nitrases and nitro-substrates that play an important role in cancer as well as in immuno-oncology.

These nitrases and their nitro-substrates have been shown to play a key role as tumor survival factors, mediators of apoptosis and promoters of resistance to PD-1 checkpoint inhibitors.

Several studies have shown that the overexpression of some nitrases was associated with poorer overall survival in multiple cancer types and that elimination of the nitrase in cellular models slowed tumor growth, reduced viability, and increased the rate of apoptosis.

We are identifying inhibitors of nitrases to target cancer-promoting nitrases and their nitro-substrates in underserved solid and hematological tumors.

Future Opportunities

Given the broad and diverse biology of nitrases and their nitro-substrates, Nitrase Therapeutics is also evaluating programs in other therapeutic areas where nitrases and nitro-substrates play a role in disease establishment or progression, including acute lung injury, lung fibrosis, and autoimmune diseases.

Based on the breadth of nitrases and nitro-substrates that Nitrase Therapeutics has discovered to-date, the pipeline and potential collaboration opportunities across therapeutic areas is vast.