NVP015 is a program aimed at developing a systemic treatment for patients with genetic mitochondrial neurological diseases such as Leigh syndrome, and MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes). Developed by the mitochondrial medicine company NeuroVive Pharmaceutical, the NVP015 program’s lead compound has produced positive results in experimental C. elegans models of genetic mitochondrial diseases.

Patients with mitochondrial disorders often experience symptoms such as muscle weakness, epileptic seizures, and other neurological manifestations. Currently, patients with genetic mitochondrial disorders are offered treatments that provide only symptomatic relief. However, the main aim of the NVP015 program is to create a medicine that can deliver systemic treatment.

How the lead compound of the NVP015 program works

Mitochondria are small cellular structures responsible for producing energy. Genetic diseases such as Leigh syndrome have been linked to dysfunctions in mitochondria. These dysfunctions occur as a result of genetic mutations and lead to the dysregulation of energy-producing mechanisms in the mitochondria. The NVP015 program has identified a molecule that is expected to bypass this mitochondrial deficiency, restore the energy-production mechanisms, and help mitochondria function properly.

Studies involving the NVP015 program

The tolerability of the lead molecule identified in NVP015 was investigated in experimental models. Top-line data from these experimental studies, which were announced in a press release by NeuroVive, showed that the lead compound in NVP015 was well-tolerated, reached the brain, and retained stability in the blood.

Based on the promising results in the experimental studies, the lead molecule identified by the program has been selected for further investigation of its therapeutic potential in preclinical studies. The main objective of these studies is to develop an orphan drug based on this compound.

Other information

In addition to the NVP015 program, NeuroVive also is working on a small molecule medicine known as KL1333 for mitochondrial disorders. The safety and tolerability of KL1333 are being investigated in healthy volunteers in a Phase 1 clinical trial (NCT03056209), which is still recruiting participants.

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