From target to translational funding: a journey from academic discovery to an investable therapeutic

Presenters: Jon Collins, PhD, and Bryan Baines This presentation will provide an overview of key elements of early-stage drug development to consider when seeking translational funding. It will delve into the significance of protein target selection, pharmacological target validation, and clinical tractability, emphasizing the importance of these factors to funding groups. The presentation will extend to descriptions of unmet medical need, the Target Product Profile (TPP), and the Target Candidate Profile (TCP), as well as commercial factors. Lastly, the presentation sheds light on the translational funding landscape, offering insights into various sources of support that can accelerate the translation of scientific discoveries from the academic laboratory to the clinic. Jon Collins is the CSO of Pinnacle Hill and Director of Therapeutic Research Translation at UNC Chapel Hill. Prior to joining UNC, he served as Director of the Discovery Partnerships with Academia at GSK where he led joint academia-GSK drug discovery collaborations across the US. He has 29 years’ experience leading drug development teams across all phases of early drug discovery. He received a PhD in Organic Chemistry from Indiana University at Bloomington. Bryan Baines is Director of Scientific Collaborations and Liaison to Four Points Innovation, a strategic partnership focused on supporting and accelerating the translation of Duke R&D projects throughout preclinical stages of drug discovery and development. Bryan brings more than 30 years of broad knowledge and business experience in the biopharmaceutical industry and in-depth understanding of preclinical and clinical drug development, IP protection, and licensing. This presentation was given on July 30, 2024, to an invited audience. This video is for educational purposes only. Presentation Bookmarks 00:00 Welcome 02:10 Presentation overview 03:48 The Target Product Profile (TPP): A living document 05:15 Drug discovery & development is risky and expensive (mAb example) 09:13 Why do drugs fail? How to mitigate this risk? 13:08 Support of genetic evidence for the most prescribed and top grossing drugs 14:10 Human genetics data & assessment of target safety 16:44 Target validation is critical 21:00 Tool compounds & target validation: Quality matters! 24:28 Pillars of pharmacological tool validation 27:05 Building the preclinical assays 31:37 Preclinical tractability 32:58 Clinical tractability 35:45 Unmet need & the medicine proposition 36:15 Commercial Considerations 40:14 Target Product Profile for Addiction (Example) 41:49 The Target Development Candidate Profile (TCP) 43:22 Translational funding, how do I get there? 44:42 Examples of Translational Funding vehicles for Therapeutics (Retained IP rights) 46:11 Hallmark criteria for Translational Granting mechanisms 47:27 Q&A The Catalyze program utilizes a new approach to translational research by supporting both the technical needs of an innovation and the management, commercialization, regulatory, and training requirements of the innovators developing them. Projects supported by Catalyze receive funding and advisory support through the National Heart, Lung, and Blood Institute (NHLBI), as well as project management assistance, access to technical services and expertise, and training opportunities through the Catalyze Coordinating Center. This research was funded, in part, by the National Institutes of Health (NIH) Agreement OT2 HL157844-01. The views and conclusions of the presenters are their own and should not be interpreted as representing the official policies, either expressed or implied, of the NIH. NHLBI Catalyze Public Site: https://nhlbicatalyze.org This research was, in part, funded by the National Institutes of Health (NIH) Agreement OT2 HL157844-01. The views and conclusions contained in this document are those of the authors and should not be interpreted as representing the official policies, either expressed or implied, of the NIH.