Company Developing Novel Heart Therapy Based On UH Research

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UH logoHONOLULU – Biotech startup Makai Biotechnology LLC is licensing technology from the University of Hawai‘i to develop new cardiovascular drugs aimed at treating and preventing heart failure. Alexander Stokes, an assistant professor of cell and molecular biology at the John A. Burns School of Medicine (JABSOM), developed the science for the drugs.

At JABSOM, Stokes worked on identifying a new target and set of effective therapeutic compounds for the treatment and prevention of cardiac hypertrophy, heart failure, and associated pathologies. Patent rights are pending.  Makai Biotechnology LLC was recently formed by Stokes and David G. Watumull, who serves as senior advisor.   Watumull is also the CEO of Cardax, Inc., a publicly held life sciences company.


Many types of diseases ultimately affect the heart by making it work harder. The heart muscle compensates by getting bigger (cardiac hypertrophy).  The heart becomes stiffer and less functional, and eventually starts to fail.

Stokes said, “We have a way of protecting the heart with a completely new therapeutic approach.  This new therapy will allow the heart to compensate for the extra work it needs to perform, without losing function and failing.”

The American Heart Association reports that heart failure is the fastest growing clinical cardiac disease in the United States, with 670,000 new cases of heart failure diagnosed each year, and accounts for 34 percent of all cardiovascular related deaths. Moreover, heart failure represents one to two percent of all health care expenditures in the U.S. or approximately $40 billion a year.

“Drugs used to treat cardiovascular diseases, especially those needed to treat heart failure, are urgently needed, and represent a very large market worldwide,“ said Watumull.

Makai Biotechnology LLC is licensing intellectual property from the University of Hawai‘i’s Office of Technology Transfer & Economic Development (OTTED). UH holds an interest in Makai Biotechnology LLC, and may receive future revenue derived from this intellectual property.  Makai Biotechnology LLC plans to establish alliances with major pharmaceutical companies to develop and test compounds from pre-clinical through phase II human clinical trials.

The new treatment method focuses on the regulation of the ion channel TRPV1. This ion channel is best known for being activated by capsaicin, the hot component of chili peppers. Stokes’ lab at JABSOM recently published data that reveals that in pre-clinical trials, inhibition of TRPV1 with a small molecule compound can protect the heart from the pathological and functional changes associated with cardiac hypertrophy, heart failure and associated pathologies.

Stokes said, “The development of a clinical formulation, and phase II clinical trials, should be quite rapid, as many TRPV1 inhibitors have been already tested in multi phase clinical trials for alternative indications.”

Funding for the pre-clinical studies was provided through grants awarded by the National Institutes of Health (NIH) and the Hawaii Community Foundation, totaling approximately $1 million over 5 years, ending 2015. One of the NIH grants was part of the Research Multidisciplinary and Translational Research Infrastructure Expansion (RMATRIX) program designed to aid translational research at JABSOM.

“Our basic science researchers dream of translating their work from the laboratory bench to the patient’s bedside.” Dean of John A. Burns School of Medicine Dr. Jerris Hedges said. “The research underway by Dr.Stokes exemplifies this opportunity and advances our most important goal – which is to provide results that will make life better for patients in Hawai‘i.”

“It is a pleasure to partner with Dr. Stokes and Makai Biotechnology in developing and commercializing research that started at the University of Hawai‘i and could ultimately end up saving many lives,” Vice President for Research and Innovation Vassilis Syrmos said.  “It is also an example of the university’s commitment to building a research industry in our state in partnership with the community through the Hawai‘i Innovation Initiative.”