BAR HARBOR, MAINE - Novo Biosciences Inc., a spinoff of the MDI Biological Laboratory in Bar Harbor, Maine, has announced that it has received a two-year, $1.5 million Small Business Innovation Research (SBIR) grant from the National Institutes of Health (NIH) for the development of MSI-1436 as a potential regenerative medicine therapy for the treatment of patients who have suffered an acute heart attack.
The grant will allow Novo Biosciences to move ahead with studies of the effectiveness of MSI-1436 in pigs. The pig heart, which closely resembles that of humans, allows scientists to model a human heart attack. MSI-1436 has already been demonstrated to regenerate damaged heart muscle and improve heart function in adult zebrafish and mice. The study in pigs is the critical next step in moving the drug candidate into clinical trials.
"We believe MSI-1436 has enormous potential," said Kevin Strange, Ph.D., CEO of Novo Biosciences and president of the MDI Biological Laboratory. "No drug now exists to treat heart attack. Heart disease is the the world's leading killer. In order to advance MSI-1436 into clinical trials, we need to first test its effectiveness in a large animal model. We are deeply grateful to the National Heart, Lung, and Blood Institute for making this possible."
"MSI-1436 offers several significant advantages with regard to drug development," Strange said. "First, the fact that it works in zebrafish and mice, which are separated by more than 450 years of evolution, makes it more likely that it will work in humans too; second, it has been found in Phase I trials for an unrelated application to be well tolerated in humans at doses many times higher than those used for the treatment of heart injury in zebrafish and mice."
"The establishment of the safety of MSI-1436 in early-stage clinical trials significantly decreases the time and expense associated with developing this drug as a potential treatment for heart attack," he added.
The randomized, double-blind studies will be conducted in collaboration with scientists from the Louisiana State University Health Science Center Cardiovascular Center of Excellence in New Orleans. "If MSI-1436 is found to be effective in pigs, Novo Biosciences will seek an FDA IND (Investigational New Drug) authorization to conduct clinical trials in heart attack patients," Yin said.
The SBIR grant is supported by Senators Susan Collins (R-ME) and Angus S. King Jr. (I-ME), who have been advocates of federal initiatives to build a strong science and technology sector, including legislation to promote biotechnology research, development and manufacturing, and to protect intellectual property.
"Heart disease is the number one cause of death in America and affects thousands of Mainers every year. It is critical that we continue to support research that tests new and innovative therapies to prevent heart attacks and treat the damage they cause," Collins and King said in a joint statement. "By further advancing the science behind cardiovascular disease and healing mechanisms, we can help save lives and improve the health of our communities."
MSI-1436 is a natural compound that was originally discovered in the dogfish shark at the MDI Biological Laboratory in the 1990s by Michael A. Zasoff, M.D., Ph.D.. In response to Zasloff's hunch that MSI-1436 might also stimulate regeneration, MDI Biological Laboratory scientist Voot P. Yin, Ph.D., tested the compound for its ability to regenerate heart muscle tissue in zebrafish, and then in mice.
The results of those tests were extraordinary. In mice, which are mammals like humans, MSI-1436 improved heart function, increased survival, reduced scarring and stimulated the proliferation of heart muscle cells. Strange, Yin and Zasloff, who is scientific director of the MedStar-Georgetown Transplant Institute at Georgetown University Hospital, were subsequently awarded a patent for use of MSI-1436 to treat heart disease.
"The current therapies for heart damage are limited to efforts to prevent secondary heart attacks and to transplantation for heart failure," said Yin, who is the chief scientific officer of Novo Biosciences and the principal investigator for the SBIR grant. "If MSI-1436 shows results in humans that are anything like what we have demonstrated in mice, it will be a game-changer for patients who have suffered a heart attack."
"If the pig study is successful, Novo Biosciences will seek investors to move the potential drug through the multi-stage clinical trial process," Yin continued. MSI-1436 would likely be licensed to a pharmaceutical company with the expertise, contacts and financial resources to commercialize the research discovery, a process that could take 10 years or more and involve an investment of tens of millions of dollars.
The inventors of MSI-1436 are also exploring its use as a potential treatment for the regeneration of skeletal muscle tissue and cardiac muscle tissue in patients suffering from Duchenne muscular dystrophy, the most common form of the disease. Duchenne muscular dystrophy is characterized by progressive muscle degeneration and ultimately death due to heart and/or respiratory failure.
About the MDI Biological Laboratory
Our scientists are pioneering new approaches to regenerative medicine focused on drugs that activate our natural ability to heal, and that slow age-related degenerative changes. Our unique approach has identified new drugs with the potential to treat major diseases, demonstrating that regeneration could be as simple as taking a pill. As innovators and entrepreneurs, we also teach what we know. Our Maine Center for Biomedical Innovation prepares students for 21st century careers and equips entrepreneurs with the skills and resources to turn great ideas into successful products. For more information, please visit mdibl.org.
About Novo Biosciences
Novo Biosciences is a regenerative medicine company that is developing small molecule therapies that reactivate and stimulate innate healing abilities in humans and animals. For more information, please visit novobiosciences.com.
Research reported in this publication was supported by the National Heart, Lung, And Blood Institute of the National Institutes of Health under Award Number R44HL139161. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health."