Indiana University's Innovation and Commercialization Office (ICO) has published IU’s current pharmaceutical pipeline of potential drug candidates. (See chart below.) A total of 36 therapeutic technologies are currently under development for treating diseases ranging from cancer to metabolic disorders, autism and others – including two technologies directly linked to the fight against COVID-19.
Clinical trials are an essential part of the drug development process and are used to assess the safety and effectiveness of new drugs prior to approval by regulatory agencies for sale. IU's ICO works closely with researchers and other stakeholders, overseeing all aspects of the technology transfer and commercialization process for advancing therapeutic technologies to market. Most of the products currently under development are part of IU startups that have raised significant private investment funds to develop and commercialize these life-changing technologies.
Among these therapeutic technologies, the story of burosumab highlights how the drug development process works to successfully bring a new treatment to market after decades of research.
Michael Econs and Kenneth E. White, of the IU School of Medicine, led the research that resulted in the development of a novel treatment for the debilitating disease X-Linked Hypophosphatemia (XLH). XLH is a deforming bone disorder that causes rickets or softening of the bones and other complications.
The treatment is based on Econs' and White’s initial discovery of Fibroblast Growth Factor-23 (FGF23), which set the foundation for the development of burosumab.
“I first started seeing XLH patients in 1986 and started doing research on XLH shortly thereafter,” said Econs, who is Distinguished Professor of Medicine and Medical and Molecular Genetics at the IU School of Medicine. “When burosumab was finally approved, it was like a dream come true.”
IU Innovation and Commercialization Office licensed the FGF23 patents to Kyowa Kirin Co., Ltd., a Tokyo-based pharmaceutical and biotechnology company. Kyowa Kirin discovered and manufactured burosumab, based on this licensed technology, and entered into a collaboration and license agreement with Ultragenyx Pharmaceutical of Novato, Calif., to develop and commercialize the drug.
Burosumab obtained U.S. Food and Drug Administration (FDA) approval in 2018 and is now being marketed under the brand name Crysvita. This past June, the FDA also approved Crysvita for the treatment of tumor-induced osteomalacia, highlighting another success for the FGF23 technology.
Another product in late-stage development is acamprosate for treatment of core social and communication impairments caused by the Fragile X Syndrome (FXS) and autism spectrum disorders. These applications are being developed by Confluence Pharmaceuticals LLC, an Indiana-based company.
Craig A. Erickson, now at Cincinnati Children's Hospital, conducted open-label pilot studies and discovered acamprosate’s application for FXS in 2009, during his time at the IU School of Medicine.
Since then, Confluence Pharmaceuticals has developed a unique sprinkle formulation for acamprosate and successfully completed Phase I clinical trials. Currently, Confluence is preparing to advance its sprinkle formulation into Phase II clinical trials in pediatric and adolescent Fragile X Syndrome patients. After successful completion of Phase II and Phase III clinical trials, Confluence Pharmaceuticals will be able to assess the suitability of the acamprosate formula to treat patients with autism spectrum disorders.
In the drive to develop acamprosate, Confluence Pharmaceuticals licensed intellectual property developed at IU through the ICO. It also received a $200,000 investment from the IU Philanthropic Venture Fund in 2019.
Confluence Pharmaceuticals CEO Steve Johns said the money from the Philanthropic Venture Fund is being used to accelerate clinical development and regulatory activities. Since receiving the investment, the company has been preparing for an FDA-required toxicology study and Phase II clinical trial protocols.
“IU has been an excellent partner, providing significant value in terms of resources and support,” Johns said. “We are very excited about the prospect of our product getting closer to market.”
“The technology transfer and commercialization process can be very complicated, and it can take years,” said Simran Trana, associate vice president of innovation and commercialization at IU. “Our goal is to find the best and the most efficient path to support IU researchers to bring their technologies to market. This is particularly relevant, given the societal and economic toll of the current pandemic, as IU researchers develop potential treatments for COVID-19.”
See other key technologies from the latest IU pharmaceutical pipeline in the chart at the bottom of this page.
About the Indiana University Innovation and Commercialization Office
Indiana University ICO is tasked with the protection and commercialization of technology emanating from innovations by IU researchers. Since 1997, IU research has generated almost 3,200 inventions resulting in more than 4,800 global patent applications. These discoveries have generated more than $145 million in licensing and royalty income, including more than $115 million in funding for IU departments, labs and inventors.