AGADA Biosciences: Facilitating Orphan Drug Development in Nova Scotia

LINK Drs. Eric Hoffman and Kanneboyina Nagaraju could have chosen a number of cities to relocate their growing business when they began to outgrow their lab space in Washington, D.C. With its established lab spaces, educated young talent, and business acceleration programs, Halifax was the clear winner. ReveraGen BioPharma was operating out of the Children’s National Medical Center in Washington, DC, where Dr. Hoffman and his team were developing a new drug, Vamorolone, for Muscular Dystrophy. However, with traditional approaches to drug development taking upwards of 15 years and a billion dollars to produce they faced many barriers. The industry was also experiencing changes at the time, that caused Dr. Hoffman to re-evaluate the research models. “Common diseases are becoming stratified into smaller and smaller groups and each group requires its own treatment regimen. There has been a lot of emphasis on rare diseases and orphan drug development over the last number of years,” says Dr. Hoffman. In recent years, there has been an increase in the number of incentive programs available for rare diseases research and with the Internet’s ability to consolidate those stakeholder communities internationally, it has created new opportunities for researchers. But, this also creates a fundamental problem; because these drugs are very targeted and if each drug costs a billion dollars and 15 years to make, the costs to patients will be astronomical. Realizing the shift in the industry and the need for better services, Drs. Hoffman and Nagaraju opened AGADA Biosciences in the Life Sciences Research Institute in 2013. The company’s primary goal is to meet the rapidly growing need for services that accelerate drug development for orphan diseases, with specific expertise in preclinical efficacy studies with biomarkers support. ReveraGen BioPharma recently developed a drug for Muscular Dystrophy that AGADA was able to bring to phase II clinical trials with Muscular Dystrophy patients. “At this point in time, it looks like we will receive approval in about eight years and for 30 million as opposed to 15 years and a billion dollars,” says Dr. Hoffman. AGADA’s success can be attributed to their first step of de-risking drugs and avoiding late stage failures. To do this, AGADA uses Dalhousie University’s state of the art mouse lab facility, also located in the Life Sciences Research Institute. Mice who develop muscular dystrophy have the same genetic mutation as human patients do, so if you develop a drug that makes the mice better, then you have de-risked that drug considerably. Dr. Hoffman states that proof of principle in an animal model is becoming increasingly requested by regulators, particularly in orphan diseases. “Traditionally, animal testing was used for toxicity, making sure you weren’t making an animal terribly sick before making a human terribly ill,” says Dr. Hoffman. “But, the whole paradigm is shifting – at AGADA we are working to make the mice better which anticipates making the human better.” AGADA has become the top tester of drugs in muscular dystrophy models and has grown quickly over the past three years. The company has seen a 60 percent growth rate each year and currently employs 13 people, with more than half coming from Nova Scotia universities. “One of the reasons we chose Halifax was because of the quality of a young, educated workforce,” says Dr. Hoffman. “We participate in the co-op programs at Dalhousie and Acadia because we see the value in keeping talent in the province.” Most recently, AGADA invested one million dollars along with three million from ACOA and half of a million from Dalhousie to develop “A Scientific and Clinical Hub for Orphan Drug Development” in Halifax that will help to accelerate orphan drug development beginning with developing treatments for three indications. The diseases in question are labeled “orphan diseases” but they occur in one in 5,000 to one in 10,000 babies. Preclinical research with the new therapies in zebrafish and mice have shown promising results that are expected to propel these programs through the clinical research stage. The project is expected to put Halifax and Nova Scotia on the map as the best place in Canada and one of the best sites in the world for such research and clinical work. Learn more about AGADA Biosciences at agadabio.com]]>