Accelerating glioblastoma therapeutics via venture philanthropy. Issue 7 (July 2021)

• Record Type: Journal Article
• Authors: Siah, Kien Wei; Xu, Qingyang; Tanner, Kirk; Futer, Olga; Frishkopf, John J.; Lo, Andrew W.
• Is Part Of: Drug discovery today. Volume 26:Issue 7(2021)
• Page Start: 1744
• Page End: 1749
• Publication Date: 2021-07
• Subjects: Glioblastoma -- Biomedical megafund -- Adaptive clinical trial platform -- Parallel drug discovery; Drugs -- Design -- Periodicals; Drugs -- Research -- Periodicals; 615.1
• DOI: 10.1016/j.drudis.2021.03.020
• Languages: English
• ISSNs: 1359-6446

Abstract

Highlights: Drug discovery for glioblastoma (GBM) is stagnant largely due to financial risks. Portfolio-based drug discovery strategy significantly reduces the financial risks. Risk–return profile of a GBM megafund is attractive to a wide group of investors. Success of a GBM megafund depends on biomedical expertise and diversification. GBM AGILE, a global clinical trial platform, expedites drug development process. Abstract: Development of curative treatments for glioblastoma (GBM) has been stagnant in recent decades largely because of significant financial risks. A portfolio-based strategy for the parallel discovery of breakthrough therapies can effectively reduce the financial risks of potentially transformative clinical trials for GBM. Using estimates from domain experts at the National Brain Tumor Society (NBTS), we analyze the performance of a portfolio of 20 assets being developed for GBM, diversified across different development phases and therapeutic mechanisms. We find that the portfolio generates a 14.9% expected annualized rate of return. By incorporating the adaptive trial platform GBM AGILE in our simulations, we show that at least one drug candidate in the portfolio will receive US Food and Drug Administration (FDA) approval with a probability of 79.0% in the next decade.