By: Maureen Newman, UVC Biomedical Engineering Community Connector
With a strong history in investigational new drug (IND) enabling processes, Lee Henderson, PhD, President/CEO/Chairman/Founder of Vybion, is no stranger to the world of drug discovery and the formality of clinical trials. Currently, Vybion is 18 months from leaving preclinical studies to enter clinical trials for INT41, which is an Gene Therapy delivery of an intrabody to treat Huntington’s Disease, developed using Vybion’s proprietary technology ProCode™.
Dating back to 2007, Henderson visited the technology transfer office (Center for Technology Licensing) at Cornell University. He was drawn to the technology behind ProCode for its potential to characterize protein-protein interactions and discover protein drugs. Vybion bought the license and began to develop it further. “The technology was in fairly early stages,” recalled Henderson. “In fact, only last year we terminated both licenses because we developed a technology that accomplishes the same thing. Their technology had a high rate of false positives, and we found a way to eliminate those in a pretty robust and scalable fashion.” After several years of back-and-forth denial and rebuttal of patent applications for ProCode, Vybion eventually attained claims that have been approved in other countries.
Before buying the license, Vybion was involved in both diagnostics and drug development in collaboration with small and large pharmaceutical companies but spent nearly five times the effort on drug development. When Vybion developed INT41 using ProCode in collaboration with Caltech (who discovered the prototype), management decided to focus fully on its development and sold its diagnostic operations to Meridian Life Sciences. “We felt that strategically it was difficult to be a small company with multiple businesses,” said Henderson.
As a “mostly virtual” company currently, Vybion is outsourcing the manufacture of INT41 in California, conducting animal studies in Europe, and conducting clinical pathology research in the United States. “We successfully tested INT41 in an animal model, then focused on mechanism of action using cell-based studies. Our results were consistent with findings in the field about what drives Huntington’s disease, but one never really knows until it gets into humans.” They entered a strategic agreement with CHDI Foundation to enable upcoming animal studies that will lead to IND studies that will be completed in mid-summer 2015. “If those studies are successful, we will not have difficulty raising funds for partnering,” predicted Henderson.
When asked about the challenges Vybion faces in these upcoming studies, Henderson replied, “A lot of it is funding. Upstate is not known as a powerhouse for funding biotech, and even in Boston some are reluctant to invest in Ithaca.” This is one of the reasons why Vybion is focusing its efforts on proving the efficacy of INT41 in the clinic before spending capital on research and development of other new drug classes. “Getting the first one of a new drug class proven in the clinic is key,” explained Henderson. “We are completely focused on Huntington’s disease at this point.” With this focus, Vybion is around 18-24 months away from phase 1/2 clinical trials.
On the other end of the resource spectrum, Henderson indicated the need for finding a strong team that is versed in drug development. “We use a variety of consultants in San Diego, UCSF, and San Francisco. Our manufacturing expert is in Connecticut,” said Henderson. “If you were in Boston, you would find lots of people, but when you are in Ithaca, you need to bring people in.”
Since Vybion does not have a full pipeline of multiple drugs, the company chooses to use consultants with experience in drug development rather than hire. When selecting these individuals, Henderson looks at their expertise. “We try to collaborate with the best,” he said. “A group at UCSF developed the delivery technology for INT41 using Gene Therapy. They are the leaders in the world for the delivery technology we have, probably more knowledgeable than any other group. But with clinical management, we have a group in the United Kingdom who manages clinical trials for only nervous system drugs.”
Turning the tables, Henderson addressed the rigor Vybion goes through when finding investors. “CHDI Foundation spent three months going through copies of notebooks and data to do due diligence,” he said. “Smart investors are going to do that, and even investors without that knowledge base will bring in experts.” Henderson said the collaborative agreement with CHDI Foundation marked an important validation milestone for INT41. He looks forward to what the future holds for clinical trials.