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Company Developing Novel Heart Therapy Based On UH Research

University of Hawaiʻi
Kelli Abe Trifonovitch, (808) 228-8108
Hawai‘i Innovation Initiative
Alexander Stokes, (808) 566-0909
Makai Biotech LLC
Posted: May 12, 2014

*video and sound available, details below

HONOLULU – 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.”



The University of Hawai‘i (UH) was established in 1907 and its campuses are all fully accredited by the Western Association of Schools and Colleges. The UH System now comprises all public higher education in the State and provides a rich array of associate, baccalaureate, graduate, and professional degrees and certificates to about 60,000 students through seven community colleges, two baccalaureate campuses and a major research university that holds land-, space- and sea-grant designations. For more information, visit


The University of Hawai‘i is working in partnership with the private sector and government to build a thriving $1-billion research enterprise in Hawai‘i that will develop a third major economic sector for the State, create thousands of high-quality living-wage jobs, and address the challenges and opportunities that face our communities and the world to improve our quality of life.  More information about the Hawai‘i Innovation Initiative is available online at


Makai Biotechnology LLC is developing novel therapies for the treatment of heart failure. A new ion channel target in this disease has been identified, along with multiple effective compounds that regulate this target. Makai Biotechnology plans to establish alliances with major pharmaceutical companies to develop and test compounds from pre-clinical through phase II human clinical trials.  For more information contact Alexander Stokes at (808) 566-0909 or  Company information is available online at

Safe Harbor:

This release may contain certain forward-looking statements regarding prospective performance and strategies by the University of Hawai‘i (including JABSOM and OTTED), the Hawai‘i Innovation Initiative, and Makai Biotechnology LLC (including owners and officers) (collectively the “Group”). Forward-looking statements, which are based on certain assumptions and describe future plans, strategies, and expectations of the Group, are generally identified by use of words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “plan,” “project,” “seek,” “strive,” “try,” or future or conditional verbs such as “could,” “may,” “should,” “will,” “would,” or similar expressions. Our ability to predict results or the actual effects of the Group’s plans or strategies is inherently uncertain. Accordingly, actual results may differ materially from anticipated results.

Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release. Except as required by applicable law or regulation, the Group undertakes no obligation to update these forward-looking statements to reflect events or circumstances that occur after the date on which such statements were made.



Link to download video and sound:

B-ROLL 1:15 Location: Stokes Laboratory, John A. Burns School of Medicine

SOUNDBITES: Dr. Alexander Stokes, John A. Burns School of Medicine, University of Hawai‘i

(1:13) We’ve shown in pre-clinical trials that treatment with a specific ion-channel-blocker can alleviate the progression to heart failure. (1:19) This has been published and is the basis of the intellectual property and the science that we’ve developed down at the John A. Burns School of Medicine.

(1:29) Well moving forward we hope to move into clinical trials as the company develops (1:35) and this should be reasonably rapid as we’re already using drugs that have been designed for alternative use that have been through some clinical trials already.