$4M Grant Will Help Advance Research into NVP015 Treatment for Genetic Mitochondrial Disease

The U.S. Department of Defense (DoD) has awarded a $4 million, three-year grant to the Children’s Hospital of Philadelphia (CHOP) to advance NeuroVive Pharmaceutical AB‘s NVP015 program for genetic mitochondrial diseases.

The donation was made by DoD’s Office of the Congressionally Directed Medical Research Programs.

“We are delighted by the news that our research partners at CHOP are receiving this grant. It further validates the extensive interest our novel succinate prodrug approach to treating mitochondrial diseases has generated. … The outcomes of the grant will expedite the development of our NVP015 project into first-in-man studies,” Eskil Elmér, MD, PhD, and NeuroVive Chief Scientific Officer, said in a news release.

Apart from symptom-based management, mitochondrial disease treatment focuses on using preventive measures to control symptoms during periods of body stress, such as infection, dehydration, or surgery.

The NVP015 program aims at creating a medicine that can bypass mitochondria dysfunction.

NVP015 enables mitochondrial substrate succinate delivery in the cell via a prodrug technology, which ultimately increases cell energy production. A prodrug is an inactive drug that is activated when ingested by transformation of its chemical structure.

Under the NVP015 program, experimental studies showed that one specific molecule was well- tolerated in a roundworm model (Caenorhabditis elegans) of mitochondrial disease. It was also able to reach the brain while retaining its stability in the blood, further validating its potential as a therapeutic candidate.

Based on those results, researchers ultimately want to develop a therapeutic candidate for Investigational New Drug (IND) submission to the U.S. Food and Drug Administration (FDA) and begin Phase 1 trials.

The work will be led by Todd Kilbaugh, MD, associate professor of anesthesiology, critical care and pediatrics, and medical director of the ECMO Center at Children’s Hospital of Philadelphia.

The grant marks the most extensive alliance between the NVP015 program developer and CHOP. It is the third time Neurovive and CHOP have worked together.

Both institutions started by working together on the ongoing NVP015 research, centered on the lead candidate’s therapeutic use in genetic mitochondrial disease. They are also investigating NVP015’s effectiveness in alleviating the harmful effects of toxic chemicals.