NeuroVive Pharmaceutical has made significant advances in its NVP015 program for mitochondrial diseases with the identification of a lead compound to develop in preclinical studies.
Preliminary analyses in experimental models of genetic mitochondrial disease revealed one specific compound that was well tolerated and able to reach the brain, while retaining its stability in the blood.
“We are very pleased with the progress in the NVP015 program which, together with our KL1333 program, shows our strong commitment to the development of novel treatments for genetic mitochondrial diseases,” Magnus Hansson, MD, PhD, chief medical officer and vice president of preclinical and clinical development at NeuroVive, said in a press release.
Mitochondria disorders, including Leigh syndrome and MELAS, present common features such as muscle weakness, epileptic episodes, and other severe neurological manifestations that can be fatal. These are caused by deregulation of the energy-producing mechanism, due to genetic mutations that alter the activity of one of five critical protein complexes involved in the process.
NeuroVive’s NVP015 program aims to develop a systemic treatment for these patients that will counteract acute energy crises and prevent organ damage. The company is working with Dr. Marni Falk and the research team at the Children’s Hospital of Philadelphia (CHOP) to evaluate the NVP015 compound and its therapeutic potential.
Dr. Todd Kilbaugh, an anesthesiologist and the medical director of ECMO Center at CHOP, received a grant from the Countermeasures Against Chemical Threats (CounterACT) NIH program to evaluate NVP015 as a novel therapeutic option for chemical threats. In addition NeuroVive earlier this year received a grant from the Swedish innovation agency to support the development of the NVP015 program.
“This is a breakthrough which takes us a step closer to our goal of developing a therapy for acute energy crisis situations in genetic mitochondrial disease patients,” said Eskil Elmér, MD, PhD, chief scientific officer and vice president of discovery at NeuroVive.
“Currently, patients with these types of genetic mitochondrial disorders can only be offered symptomatic treatment alternatives, whereas the aim of the NVP015 program is to create a drug that can bypass the dysfunction, helping the mitochondria to function properly,” Elmér said.
NeuroVive is developing a second treatment targeting mitochondria disorders. KL1333 was designed to improve energy production by modulating the activity of a co-enzyme involved in the process. This new drug is currently in a Phase 1 clinical trial (NCT03056209) to assess its safety in healthy volunteers.