First Approved Cancer Treatment for TGCT Included DILIsym Simulations in FDA Review

Software: DILIsym®, RENAsym®
Division: DILIsym Services

DILIsym Services, Inc., a Simulations Plus company (Nasdaq: SLP) and a leading provider of modeling and simulation software for pharmaceutical safety and efficacy, today announced that simulations using their DILIsym® software were noted in a U.S. Food and Drug Administration (FDA) review of the New Drug Application for Pexidartinib (Turalio®, marketed by Daiichi Sankyo, Inc.), which was subsequently approved as the first systemic therapy for treatment of symptomatic tenosynovial giant cell tumor (TGCT). The FDA review also included the general comment by the FDA that DILIsym is helpful for hepatocellular (liver cell) injury. The Turalio® submission is the latest example of how results from the DILIsym software can be valuable in FDA interactions by contributing to safety predictions and safety management strategies.

DILIsym modeling supports key drug development decisions by predicting potential drug-induced liver injury (DILI) risk of new drug candidates. The modeling also identifies the biochemical events that lead to DILI caused by a drug and can thereby predict certain subgroups of patients at increased risk for DILI from that drug. The information from DILIsym modeling serves to help guide go/no-go decisions on major drug development projects, potentially avoiding the disastrous financial effects of failed clinical trials, or better, providing assurances that drug-induced liver injury will not be an insurmountable obstacle to FDA approval. Pexidartinib is a good example of the utility of DILIsym to help with safety evaluation and management, as well as benefit-risk analysis. DILIsym is the product of an ongoing public-private partnership (the DILI-sim Initiative) involving scientists from the pharmaceutical industry and academia.

Dr. Paul Watkins, the Director of the Institute for Drug Safety Sciences at University of North Carolina and chair of the DILI-sim Scientific Advisory Board, said: “With DILIsym, regulators and drug developers alike can better assess safety risks to patients and thereby speed delivery of important new treatments, like pexidartinib, to the patients who need them. I continue to be amazed at the power of DILIsym and the positive impact it is having on drug development programs.”

Dr. Scott Siler, Chief Scientific Officer of DILIsym Services, added: “It gives me great pride to have been involved in the Pexidartinib work, a therapy that can save the lives of patients. Our DILIsym software, now coupled with our new kidney injury software in development, RENAsym, comprises the best QST solutions available on the market. At the end of the day, our work is about the patients who need these important medicines in oncology but also many other therapeutic areas. We are glad to see our tools continue to show up in regulatory reviews.”

Views expressed in this press release do not necessarily reflect the official policies of the Department of Health and Human Services; nor does any mention of trade names, commercial practices, or organizations imply endorsement by the United States Government.