Experts from the Applied Pharmaceutical Innovation (API) discussed approaches to streamline and reduce risks in drug development during a recent webinar, according to an April 6 statement. Sir Michael Houghton, Chief Scientific Officer at API and Director of the Li Ka Shing Applied Virology Institute at the University of Alberta, led the discussion on navigating the challenging transition from preclinical to clinical stages.
The process of moving a small molecule from discovery to first-in-human studies can cost between $3-5 million. This significant investment highlights the importance for developers to have clear strategies for funding and risk management early in their projects. "It’s never too early for developers to consider how to translate research to the clinic," Houghton said.
Daren Ure, Executive Scientist for Discovery & Early Development at API, emphasized that successful programs require robust proof-of-concept data supporting mechanism-of-action evidence, potency, safety, selectivity, pharmacokinetic profiles and drug interactions evaluated across multiple models. Ure also noted that artificial intelligence tools are helping filter large discovery libraries into smaller sets of candidates suitable for trials: "Although AI has not routinely delivered a single 'perfect' molecule without further experimentation, it is already improving efficiency."
Daniel Trepanier, Executive Scientist for Preclinical Development at API, said staged derisking programs—starting with computational filters followed by laboratory and animal testing—can lower failure rates: "Ultimately, derisking programs translate into a reduction of failure probability." The speakers also advised academic teams to conduct SWOT analyses and map out clinical development plans from project initiation.
Launa Aspeslet, Chief Translational Officer at API, stressed aligning preclinical models with target trial populations and planning manufacturing scalability early on. Patent life pressure was another concern raised by both Houghton and Trepanier; they explained that balancing timely patent filings with disclosure risks is crucial given typical drug development timelines.
Manufacturing considerations were highlighted as essential even in early stages. Trepanier recommended designing synthetic routes that are scalable using stable substances and accessible reagents before engaging contract manufacturers (CDMOs). Houghton noted that API’s facility aims to be one of North America’s largest biomanufacturing sites—a factor influencing his decision to join as CSO—and will support production efforts such as RNA hepatitis C vaccines.
