THE CHRONICLE HERALD: 2017 Game Changers awards recognize youth retention
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]]>
AGADA Biosciences Corp. was among 26 businesses to receive provincial government money Tuesday to help expand outside the region’s confines. The company, which received a $26,252 grant under the province’s Global Business Accelerator Program, has 10 people on staff, not including its two founders. According to Muscular Dystrophy Canada’s website, the disease can appear at any time in life. It is widely understood as a childhood disorder, but there are many types that afflict people. “It’s a horrible disease where children are diagnosed between two and four years of age; one hundred per cent of them die in their twenties,” AGADA co-founder Dr. Kanneboyina Nagaraju said in an interview Tuesday. “It’s a horrible thing for parents knowing that their children are going to die.” Nagaraju said the provincial money will help expand the young company — co-founded by Dr. Eric Hoffman, who in the late 1980s discovered the gene that causes muscular dystrophy — and enable AGADA to break into new markets in the United States, the United Kingdom, Japan, South Korea, France, Italy and Australia. The firm has contracts with groups in those countries. “In fact, this is one of the first human genes ever discovered; it remained as his main focus,” Nagaraju said of Hoffman. “He needs to find a cure … for the disease. “We wanted to expand our business in multiple different companies. We truly don’t have the expertise to go off to each market, so that means we have to hire people to help us.” AGADA will continue to work on treatments of its own and screen possible drugs for muscular dystrophy submitted by other pharmaceutical firms, he said. Nagaraju and Hoffman worked on a drug to combat the disease in 2008 and plan human trials next year. That drug was developed by the partners, who also own the firm ReveraGen, before they formed AGADA, said Nagaraju. AGADA did pre-clinical work on the drug. Hoffman, who is based in Washington, D.C., spends part of every year in Nova Scotia. Nagaraju, a professor of integrated systems, biology and pediatrics at the Children’s National Medical Centre in that city, frequently travels to the province. Nagaraju said big pharmaceutical companies often focus on diseases that affect a large number of people but not rare diseases, which AGADA focuses on. Agencies such as the U.S. Food and Drug Administration and European medical regulators have recently become more helpful, and the focus on rare disease has heightened, said Nagaraju. That shift has helped AGADA because big companies are bringing trial drugs to the Halifax firm for screening, he said. Blind trials are done at AGADA’s headquarters in the Dalhousie Life Sciences Building on Summer Street, where it has laboratories and offices. The company rents from Innovacorp and Dalhousie University. AGADA is just a year old, and Nagaraju said the funding will help accelerate it beyond Nova Scotia borders. “We are in the very early stages.” Source]]>
familial exudative vitreoretinopathy, or FEVR. One in 15 babies born in Canada has what’s called an orphan disease. And even though there are more than 7,000 such diseases that affect over 300 million people globally, they’re considered rare. “If you look at use of beds in pediatric hospitals, the estimate is one in three kids is there because of a genetic condition,” says Dr. Chris McMaster, professor and head of Dalhousie’s Department of Pharmacology. “This high incidence of hospitalization is due to the fact that 95 per cent of orphan diseases have no treatment.” Major pharmaceutical companies generally concentrate on drugs that treat common conditions, so orphan diseases – most of which are genetic – haven’t been a priority, as their name suggests.Working with partners such as Halifax’s Agada Biosciences and the Vancouver-based Centre for Drug Research and Development, Dalhousie Medical School researchers are taking new orphan disease treatments from the lab and moving them to market. “I’ve seen first-hand how Dr. McMaster and his team have been diligently working to advance their research and build partnerships,” said Stephen Hartlen, Dalhousie’s assistant vice-president of industry relations and executive director of the university’s Industry Liaison and Innovation Office. “The dedication and commitment to developing new medicines for patients who are impacted by these orphan diseases is both honourable and commendable.” Through a project announced Tuesday (September 13), $3-million in funding from the Atlantic Canada Opportunities Agency and additional project support from Agada Biosciences and Dalhousie Medical Research Foundation, making it worth a total of $4.5 million will enable orphan disease drug discovery work and will also result in the training of several highly qualified scientists with expertise in getting treatments from the laboratory to the medicine cabinet. “Ninety per cent of orphan diseases are life-limiting, and 35 per cent of children born with them don’t make it to their fifth birthday,” says Dr. McMaster. “However, advances in knowledge and technology by clinicians and scientists in Nova Scotia have made it possible to move toward treatments for orphan disease patients, so we can now help these Canadian children in need.” The zebrafish facility, located in Dalhousie’s Life Sciences Research Institute, will be key to advancing the drug discovery. Zebrafish share many genes with humans, so they’re excellent models for testing the safety and efficacy of treatments. “This project will help establish the Atlantic region as a centre of excellence in orphan disease drug development,” says Dr. McMaster. “We’ll be leveraging the scientific expertise within our Zebrafish Core Facility to conduct drug screenings and to identify future drug development efforts.” “It’s a brave new world for genetic conditions, and it’s a perfect storm for us right now in Halifax,” says Dr. McMaster. “We’re identifying genetic links behind orphan diseases, finding targets for therapeutic intervention, and developing compounds that can be turned into medications. So not only will we have faster, more precise diagnoses, we’ll have more precise, targeted treatments for patients.” Source]]>