ARPA-H launches program to develop predictive drug safety and efficacy modeling platforms for Investigational New Drug candidates 

Program aims to reduce drug safety failure rate and improve efficacy prediction before clinical trials start 

The Advanced Research Projects Agency for Health (ARPA-H), an agency within the U.S. Department of Health and Human Services (HHS), today announced a new funding opportunity through its Computational ADME-Tox and Physiology Analysis for Safer Therapeutics (CATALYST) program. CATALYST intends to create human physiology-based computer models to accurately predict safety and efficacy profiles for Investigational New Drug (IND) candidates.  

Current clinical drug development can be a long, expensive, and inefficient process. Over 90% of drug candidates never reach the commercial market. About half of these failures are due to efficacy with many resulting from incorrect dosing and non-availability of drugs for specific parts of the body. Further, a quarter of these failures result from safety issues occurring during clinical trials that were not predicted before first-in-human studies. More accurate preclinical safety and efficacy assessments could result in lower drug costs and increase orphan drug development.  

“Imagine a future where we could predict drug safety and efficacy accurately before clinical trials even begin,” said ARPA-H Director Renee Wegrzyn, Ph.D. “CATALYST’s digital models represent a first step toward that vision, working to modernize safety testing for drug development, have a positive impact on rare disease therapies, and help under-represented populations in clinical trials.” 

CATALYST aims to lessen the use of insufficiently predictive preclinical animal studies with more accurate, faster, and cost-effective in silico drug development tools grounded in human physiology. These technologies could reduce the failure rate of drug candidates, ensure that medicines reaching clinical trials have confident safety profiles, and better protect trial participants. By bringing in drug developers for real-world proof of concepts, CATALYST has the potential of moving far beyond discovery into practical application within just a few years. 

“The failure to accurately predict drug effects, and the current approach which uses animals for these predictions, is a huge bottleneck in drug development,“ said CATALYST Program Manager Andy Kilianski, Ph.D. “CATALYST seeks to transform how we predict clinical effects and will support the adoption of these novel drug development tools for use in regulatory science. By designing better in silico models and demonstrating their effectiveness in preclinical studies, CATALYST’s predictive tools will break through these bottlenecks. This leads to more representative safety assessments, shorter development timelines for orphan drugs, less expensive therapies, and better patient safety for all.” 

Through a forthcoming Innovative Solutions Opening (ISO) solicitation, CATALYST invites proposals across three technical areas: data discovery and deep learning methods for drug safety models, living systems tools for model development, and in silico models of human physiology. If successful, CATALYST will significantly improve drug safety testing in preclinical and clinical studies and create a drug development tools pipeline for use in regulatory science applications. 

ARPA-H is working across HHS to ensure computational models developed by CATALYST address drug development inefficiencies and are built to align to regulatory standards. The program intends to reach clinical trial readiness based on validated, in silico safety data and help meet the targets of the U.S. Food and Drug Administration’s Modernization Act. In addition, CATALYST will drive adoption of these predictive tools for use in regulatory filings by supporting their use in preclinical pharmaceutical drug development. 

Multiple awards under this ISO are anticipated. Resources available will depend on the quality of the proposals received and the availability of funds. Learn more about CATALYST on its program page, including information about the solicitation, Proposers’ Day registration, and how to state interest to form an applicant team.