A 7-year-old pharmaceutical company that’s developed a new way to create and deliver an old kind of drug is making the critical transition to the clinical stage as it prepares to take its drug for Crohn’s disease and ulcerative colitis into human trials.
The U.S. Food and Drug Administration has cleared the IND application filed by Avaxia Biologics, which develops orally administered antibody drugs that act on biological targets located in the digestive tract. Its lead product, AVX-470, was designed to neutralize the effects of an established target for irritable bowel disease called tumor necrosis factor (PDF).
Antibodies are proteins, so they’re digestible, which is why antibody drugs like Janssen’s Remicade and Abbott Laboratories’ Humira are injected, Avaxia vice president of corporate development Mike Rivard explained. Avaxia’s antibodies, he continued, are “engineered by nature to resist digestion.” They’re generated in dairy cows and isolated from the cows’ early milk.
So, rather than being injected into the bloodstream, the drug goes right to where inflammation occurs in the GI tract. Existing TNF inhibitors are distributed throughout the body and may cause side effects associated with immunosuppression like increased risk of infection.
A phase 1b trial is planned to begin shortly. It will be a double-blind, placebo-controlled study with three dosing cohorts made up of eight patients each, with six of them taking the drug and two taking a placebo. While the primary endpoint is safety, the company will also look for early signs that the antibody is reaching the right places in the colon and providing an anti-inflammatory effect, Rivard said.
Antibodies are an established therapeutic class with $45 billion in global sales. But that’s just one approach to treating inflammatory bowel disease, which lately has drawn interest from Big Pharma and startups alike. Earlier this year, an experimental Takeda antibody drug produced positive top-line results in a phase 3 trial. AstraZeneca and Amgen are also collaborating to co-develop two monoclonal antibodies for Crohn’s or ulcerative colitis.
Meanwhile, Cleveland-based Athersys is working with Pfizer to investigate its stem cell treatment for IBD in clinical trials, Santarus is seeking FDA approval of its steroid-based oral drug for bowel disease and Altheus Therapeutics is testing its combination drug for ulcerative colitis.
Two million people in the U.S., Europe and Japan have a form of IBD and that number is expected to grow, driving the global market value for therapeutics up to nearly $7 billion by 2015, according to visiongain.
Avaxia has other preclinical projects in diabetes, celiac disease, oral mucositis and acute radiation syndrome, but Rivard said it’s sharply focused for now on getting AVX-470 into the clinic.
Founded in 2005 by CEO Barbara Fox, the Lexington, Massachusetts company raised an undisclosed series A last year. It’s also been supported by friends-and-family money and some early government grants, Rivard said.