New infusion of NIH funds for Tregitope,“Paradigm-Shifting” Treatment

Providence, RI – April 10, 2012

EpiVax_Tregitope_10April2012

Providence-based biotech EpiVax, Inc. was awarded a new NIH SBIR Phase II grant totaling $1.5M in funding from the National Institutes of Health’s (NIH) NIDDK to explore formulation, dose, route and delivery vehicle for Tregitope through the NIH Small Business Innovation Research (SBIR) program. EpiVax anticipates receiving an additional SBIR award of approximately $775,000 within the next few months, bringing the total to over $2.25 million dollars for the first half of 2012.

The new funding from NIH will allow EpiVax to move this important treatment for autoimmune diseases forward in preclinical studies. Tregitopes were discovered in 2008 by the team of De Groot and Martin at EpiVax and the program is currently managed by Scientific Director and Brown U. Ph.D, Leslie Cousens. The original discovery was published in the journal Blood in 2008.

Tregitopes are linear sequences contained within the framework of monoclonal antibodies and immunoglobulin G (also known as gamma globulin). The Tregitopes act as a natural ‘off switch” and have been shown in standard preclinical models, and by collaborating laboratories, to suppress and treat autoimmune disease, allergy, and to effectively suppress the immunogenicity of co-administered proteins. According to Dr. Srini Kaveri of the Centre de Recherche des Cordeliers (INSERM) in Paris, “It is most likely that some of the well established and successfully practiced therapeutic strategies such as intravenous immunoglobulins to treat several serious autoimmune and inflammatory diseases in order to induce tolerance, may actually be harnessing the potential of Tregitopes”.

Anticipated uses of Tregitope include induction of tolerance to co-administered protein drugs, a market worth more than $100B globally. Also known as biotherapeutics, drugs such as Campath, Rituximab, enzyme replacement therapies such as Myozyme, and blood factors such as FVIII often induce antibodies, rendering the drugs less effective or ineffective. In addition, Tregitopes may have broad applications in Transplant according Nader Najafian, M.D., who is using Tregitopes in research being performed at Harvard Brigham and Women’s Hospital, and therapy for autoimmune diseases such as Multiple Sclerosis. Researcher Samia Khoury M.D., an internationally recognized MS researcher also based at Harvard Brigham and Women’s hospital, says that pre-clinical studies of Tregitope being carried out in her laboratory by researcher Wassim Elyaman, Ph.D., are “promising”. The Tregitope technology won awards from the American Transplant Association (ATA) and from the American Association of Pharmacologists (AAPS) in 2010 and 2011.

The receipt of the original Phase I SBIR grant in 2008 allowed EpiVax to generate substantial evidence that Tregitopes may explain the effectiveness of IVIg, a current leader in auto-immune treatment. In animal models, Tregitope appears to “reset” the immune response away from autoimmunity and towards tolerance, normalizing blood sugar levels.

“This is another great example of the important role the SBIR program has played in helping Rhode Island small businesses,” said Senator Sheldon Whitehouse, who worked to re-authorize the SBIR program in the Senate last year. “I commend EpiVax for its hard work and dedication over the years, and congratulate it on its SBIR grant. This funding will support Rhode Island’s knowledge economy by helping EpiVax continue its important research on the prevention and treatment of harmful diseases.”

The initial target for Tregitope therapy will be Type 1 diabetes (T1D). Each year more than 13,000 young people are diagnosed with T1D. Islet replacement therapy is still in the experimental stage for individuals with advanced disease, but EpiVax believes that T1D-Tregitope therapy will facilitate this novel treatment for individuals in the later stages of T1D. Furthermore, preliminary studies carried out by EpiVax and collaborators indicate that Tregitope may be useful for inducing tolerance to transplants, protein drugs, and blood replacement therapies, suggesting that Tregitope may be useful for patients with other auto-immune diseases. EpiVax will use this new round of funding to develop a dosing and delivery procedure for Tregitope therapy in preparation for an IND application. Preliminary “Safety and Toxicity” studies are also supported by the NIH Phase II.

“Developing more specific therapies to promote tolerance to the beta cell antigens that trigger the autoimmune response is a critical component of a comprehensive therapeutic approach to type 1 diabetes,” said Julia Greenstein, Ph.D., assistant vice president of cure therapies for JDRF, whose previous funding for EpiVax helped the company derive preliminary data to support the new NIH funding. “It’s great to see the NIH support EpiVax’s approach as this therapy may have the potential to reduce the harmful immune responses to the insulin-producing beta cells, thereby preserving the body’s ability to make its own insulin.”

“The endorsement, and more so the continued funding, by the National Institutes of Health of EpiVax’s Tregitope program, is further validation of the promising research pioneered by Dr. De Groot and her colleagues and collaborators,” stated Richard G. Horan, managing director at the Slater Technology Fund. “Rhode Islanders have been well-served by the support provided EpiVax from the Slater Fund in the company’s early years. In addition to generating a return on the fund’s investment, the company has generated over a decade of high value, high wage jobs funded by steadily-increasing grants and contracts with pharmaceutical and biotech companies. It’s a great example of academic innovation translating into robust economic development in biotechnology.”

Note- More information about current research on Tregitope will be available to the public at EpiVax’s 4th Annual Tregitope Symposium will be in Baltimore following IIR’s 13th Annual Immunogenicity for Biotherapeutics. Seating is limited. Please contact us for the hotel discount code and to reserve a seat.

For further information and to register for our seminar, please contact CMillerIV[at]EpiVax.com or AnnieD[at]EpiVax.com.

For more information on Tregitopes, the original paper can be found at this link:
http://tinyurl.com/ASDeGroot-Blood-2008.

An animation describing the proposed mechanism of action can be found at:http://www.epivax.com/epivax-animation4.html

For more information about Professor Anne S. De Groot, M.D.
see http://www.immunome.org.

For the relevance of Tregitope to biologics
http://tinyurl.com/ASDeGroot-Clin-Immunol-2009.