Hartford Business Journal

8 Hartford Business Journal • August 29, 2016 www.HartfordBusiness.com Lipid Genomics hopes investors aid therapeutic's commercial viability By John Stearns jstearns@HartfordBusiness.com D r. Annabelle Rodriguez is driven to find a therapeutic cure for people with good levels of the healthy HDL cholesterol but who are still at risk for heart disease and heart attacks — even if it means waiting another 10 years to get medication in people's hands. It was more than a decade ago, when she worked at Johns Hopkins University School of Medicine in Baltimore, that she discov- ered genetic variations in the HDL choles- terol gene, SCARB1, among patients who had good levels of HDL but, paradoxically, had heart disease or were at risk for it. She set out not only to create a reliable test for the genetic variation, which she did, but also a way to treat it, eventually forming in 2010 a company, Lipid Genomics Inc., to develop that therapeutic remedy. Rodriguez, a physician-scientist at UConn Health, is at the point now where the Farming- ton startup needs investors to take a proposed therapeutic through the expensive and time consuming drug-trial process. "I know that when we're starting to look at therapeutics, it also takes about a decade," Rodriguez said. "So I've just learned to be patient again and think, 'Well, I'm 59, I think I can do it until 69,' " she said with a laugh, "that we'll find that therapeutic — that would be wonderful." Currently, patients with the gene varia- tion are treated with statins, but Rodriguez, an endocrinologist who's studied cholesterol disorders her entire career, isn't sure that's the best option. She calls it the still-unan- swered question. The market potential for a treatment is a multi-billion-dollar one, according to Gerry Smith, who became CEO of Lipid Genomics last fall and also splits his time as CEO of Hershey, Pa.-based Targepeutics Inc., which is develop- ing targeted therapeutics for cancer treatment. "The prevalence of the genetic variations that Annabelle's identified is very, very large, so it applies to a lot of patients," Smith said from his office near Harrisburg, Pa. Smith has been talking with potential investors to help fund the company through Phase 1 clinical trials of a drug, probucol, that was used in the U.S. from 1977 to 1995, then licensed in Japan, where it's used to treat the bad cholesterol, LDL, but not for the same issues Rodriguez identified. She'd like to see if it would help people in the U.S. with the HDL problem she identified. It works well in mice, she said. She proposed a repurposing of the drug for treating the issues she discovered related to good levels of HDL and received an Inves- tigational New Drug approval from the Food and Drug Administration in 2013, but Lipid Genomics needs funding to proceed with Phase 1 trials in humans in the U.S. The company has all its intellectual prop- erty and other groundwork in place and Smith hopes the quality of Rodriguez's science woos investors. Rodriguez's science is applicable to a broader market than people with healthy lev- els of HDL with genetic cholesterol disorders who are at risk of heart disease, Smith said. "If it's successful in that market, it can be meaningful in other markets as well, so the science that she's putting out, we're having a lot of dialog with big pharma and we're look- ing for a quality syndicate of venture capital- ists," Smith said. Rodriguez's research also has explored the link to inflammation and changes in one's immune system that can be linked to cholesterol and heart attacks. At UConn, she's developed a blood test to identify pro- tein markers for the inflammation, which can lead to heart disease and heart attacks, and is studying ways to treat it, hypothesiz- ing that probucol could be helpful. Discussions with potential investors are occurring on both the repurposed probucol and commercializing the protein-marker diagnostic test, she said. There seems to be momentum, she said, noting some investors are doing due diligence. Breaking down the science Smith said Rodriguez's research provides a radically new understanding of how the body works, backed by tests on about 6,000 blood samples from people on the genetic test she developed at Johns Hopkins and the protein marker test she developed at UConn, the latter Building Bioscience A N H B J S E R I E S O N C T ' S B I O S C I E N C E S E C T O R from page 1 Annabelle Rodriguez (third from right) is flanked by UConn Health researchers in a lab at UConn's Cell and Genome Sciences Building in Farmington, where Lipid Genomics is trying to develop a therapeutic for people with good levels of healthy cholesterol but who still suffer from heart disease. H B J P H O T O | J O H N S T E A R N S

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