A molecule with huge potential?

EpiVax Inc., a Providence-based biopharmaceutical company that has toiled in relative obscurity for almost a decade, may have hit the jackpot.
The company, which has offices and labs in the Jewelry District, says it has discovered a single molecule that could lead to the development of a new generation of drugs to treat and prevent autoimmune disorders, including juvenile diabetes, multiple sclerosis, rheumatoid arthritis, psoriasis, lupus, the rejection of donor organs and even common allergies.
If EpiVax is successful in clinical trials at harnessing the power of the molecule to prevent disease, it would represent a paradigm shift in the treatment all human health problems that involve an unwanted immune response, said Dr. Anne S. De Groot, the company’s president and CEO, who co-founded EpiVax with William Martin, the company’s chief operating officer.
The molecule, which EpiVax is calling “Epi-13,” is found in several naturally occurring proteins in the human body. The molecule activates regulatory T-cells that suppress immune responses, De Groot said.
By coupling the Epi-13 molecule with proteins associated with autoimmune diseases, EpiVax hopes to shut down the unwanted autoimmune responses that cause those illnesses, in a molecular process that could be compared to turning off a switch in the body, she said.
“You can imagine that with a protein like insulin, to which diabetics are basically allergic, it will suppress that immune response,” De Groot said. “It’ll suppress immune response to dust mites, it will suppress immune response to ragweed, it will suppress immune response to cat dander. You know, it’s really a very exciting discovery.”
EpiVax, which De Groot founded in 1998, has worked to develop treatments for a wide range of diseases using the company’s EpiMatrix System, a closely guarded drug and vaccine design technology that De Groot developed in the 1980s and 1990s while working at Brown and previously at the National Institutes of Health.
The EpiMatrix computer system uses algorithms to identify the links on a gene sequence that will activate T-cells, which direct all other cells in the immune system. The company had worked for years to identify cells that trigger immune responses when it unexpectedly discovered the molecule that dampens immune reactions about a year and a half ago, De Groot said.
“It is a molecule that we really literally stumbled on. This was a ‘eureka’ moment,” De Groot said.
EpiVax will most likely shift its entire business to focus on developing new drug treatments using the molecule, which has demonstrated its potential to dramatically improve existing medical treatments for a wide range of health problems, she said.
The Epi-13 molecule would be a coveted target for purchase by major pharmaceutical companies if clinical trials prove successful. Just in recent weeks, two small biopharmaceutical companies each sold molecules they had developed that interfere with immune response, in deals worth hundreds of millions of dollars.
On Oct. 23, for example, GlaxoSmithKline bought the exclusive rights to an antibody being developed by Cambridge, Mass.-based Tolerx Inc. to develop drugs for diabetes and other autoimmune diseases. Tolerx received an up front payment worth $70 million, and the total value of the deal could exceed $700 million if the antibody is successfully developed and approved as several drugs.
The Epi-13 could potentially fetch an even higher price, De Groot said, because unlike the treatments developed by Tolerux, which suppress the entire immune system, EpiVax’s approach would target individual proteins rejected by the immune systems of patients.
“What we’re working on is sort of like a fine-needle approach to surgery rather than a sledgehammer,” De Groot said. “The whole market for this type of molecule is huge. They’re big, big, billion-dollar markets.”
In pre-clinical trials conducted in the past year, EpiVax has used Epi-13 to successfully treat common allergies in lab mice and human cells. The company is currently testing the molecule in pre-clinical trials to treat multiple sclerosis and to prevent the rejection of transplanted organs, De Groot said.
On Oct. 25, EpiVax announced it has received a $351,000 grant from the Juvenile Diabetes Research Foundation to develop an Epi-13 treatment for Type I diabetes, a devastating and chronic autoimmune disease that currently affects 3 million Americans.
The research program will be carried out in collaboration with Dr. David Scott of the University of Maryland and with Robert Smith of the Hallett Center for Diabetes and Endocrinology at Rhode Island Hospital, an expert in the treatment of Type I diabetes.
“This research deals with a critically important clinical problem, and the approach EpiVax is taking in developing new diabetes therapies holds great promise,” Smith said. •