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Pillars of WARF Therapeutics: Invest - Develop - Partner

Pillars of WARF Therapeutics: Invest - Develop - Partner. WARF Therapeutics: A team of industry trained “Drug Hunters” with expertise in navigating the pre-clinical drug discovery space Aspirational Goals & Benefits:

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Pillars of WARF Therapeutics: Invest - Develop - Partner

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  1. Pillars of WARF Therapeutics: Invest - Develop - Partner • WARF Therapeutics:A team of industry trained “Drug Hunters” with expertise in navigating the pre-clinical drug discovery space • Aspirational Goals & Benefits: • Continue the UW legacy & deliver novel medicines to patients with unmet medical needs • Revenue stream for PIs, UW, and WARF • Mission: To partner with UW and MIR Principal Investigators (PIs) interested in translational research, invest to discover & develop novel drug like molecules that modulate validated targets and improve human disease, and partner with bio-centric companies to develop and commercialize

  2. WARF Therapeutics Virtual Laboratory: Bridging the Gap between Novel UW Discoveries & Bio-Pharma Target Discovery Preclinical Drug Discovery Drug Development UW & MIR PIs Pharma/Biotech Partners • Novel biological target • Therapeutic hypothesis • Rigorous target validation • Preclinical Drug Discovery Experts • Small Molecule Design & Optimization • Project Management • Novel Intellectual Property (IP) • IND Tox • Clinical Trials • NDA Sanford Burnham Prebys Research Institute Pharmaron/Wuxi CRO Institute for Clinical and Translational Research (ICTR) Institute for Clinical and Translational Research (ICTR) • Risk/reward sharing partner • Early Discovery • MSA signed • Flexible resource (FFS) • Late Discovery • MSA signed • Preclinical research support • Clinical research support High Potential Licensable Assets Novel Hits Early Leads Late Leads Clinical Candidate

  3. Rationale for the 5R Philosophy

  4. Why do Drugs Fail in the Clinic? AstraZeneca Determined Late-Stage Failure Linked to Target Selection • Efficacy- This could be due to: • Insufficient target engagement and/or poor pharmacokinetics • Preclinical models did not translate into human disease modulation • Insufficient target validation data linking target modulation (GOF and LOF) to disease • Safety- This could be due to: • Mechanism based toxicity • Off-target toxicity • Molecule (or metabolite) toxicity Nature Reviews Drug Discovery 2016, 15, 817

  5. AstraZeneca’s 5R Philosophy Focused Resources on Most Validated Targets Which Led to Improved Productivity Metrics • The Right Target • Strong link (human genetics) between target & disease • Strong Target Validation • Available & predictive biomarkers • The Right Tissue • Adequate drug exposure in tissue of interest • Defined PD biomarkers • Clear understanding of PK/PD/functional relationships • The Right Safety • Understanding ALL on-target modulation effects (therapeutic index) • The Right Patients • Know how to identify and stratify patients • The Right Commercial Potential • Differentiated versus standard of care (SOC) Nature Reviews Drug Discovery 2014, 13, 419

  6. Definition of Target • Target:is a molecule in the body, usually a protein, that is intrinsically associated with a particular disease process and that could be addressed by a drug to produce a desired therapeutic effect.

  7. Definition of Target Validation Gain of Function (GOF), Loss of Function (LOF) experiments Disease association, genetics and expression data In vitro and in vivo disease models Target ID & Validation • Target Validation: the process of physiologically, pathologically, and pharmacologically evaluating a biomolecule. Might be performed at the molecular, cellular, or whole animal level. Transgenic models (knock in or knock out) Understanding of molecular signaling pathways Pharmaco-logical modulation with Tool cmpd

  8. TEMPLATES&INSTRUCTIONS

  9. Message to PI’s: Interact with WARF Therapeutics Early & Often • Pre-submission: PI and WARF Therapeutics can have regular meetings to discuss idea(s) • Review program data, identify gaps and plan next steps • Submission Process to WARF Therapeutics • PI submits IDR & “Program Nomination Form” • Formal presentation made to Scientific Advisory Board • Program Decision & Prioritization • If Program accepted, next steps in collaboration with WARF Therapeutics include • Develop work plan, Go / No Go decisions, and timelines • Discuss and agree on public disclosure/publication plan • Determine model and allocate resources • If Program not accepted • Provide feedback and rationale for the decision • Suggest experiments that if successful could lead to future acceptance

  10. Name/Department of Principal Investigator: insert name here Target and Therapeutic Indication: insert target & indication here Date of Submission (Month/day/year): insert date here Program Nomination Form: Executive Summary This page is for instructional purposes only. View as a template. Use the blank page that follows as your Executive Summary and complete the requested information for each row

  11. The Right Target (primary data to the questions below: 3-10 slides as needed) • This page is for instructional purposes only for the “Right Target” • View as a template • There is no slide count limitation for each topic: for slide 2 of the“Right Target”, create duplicate slide and make Title “The Right Target (continued) • Insert Tables, figures, images that support the 3 questions for the “Right Target” and target validation • Specify the target • Specify the intended or potential disease(s) and the therapeutic hypothesis • Provide the existing (and/or planned) target validation data: for example target modulation-functional relationships with either CRISPR, siRNA, or small molecule pharmacological modulation (in vitro and/or in vivo), human genetic-disease data • Disease association, genetics and expression data • Gain of Function (GOF), Loss of Function (LOF) experiments • In vitro and in vivo disease models • Transgenic models (knock in or knock out) • Understanding of molecular signaling pathways • Pharmacological modulation with Tool compound • Annotate with text to articulate key messages

  12. The Right Target (continued)

  13. The Right Tissue (primary data to the questions below: 3-10 slides as needed) • This page is for instructional purposes only View as a template • There is no slide count limitation for each topic • Insert Tables, figures, images that support the 3 questions for the “Right Tissue” • Specify which tissues where the target is expressed • Specify which tissue is the intended target • What potential target engagement and/or functional biomarkers and if these assays currently exist, would need to be developed or validated? • Annotate with text to articulate key messages

  14. The Right Safety (primary data to the questions below: 3-10 slides as needed) • This page is for instructional purposes only View as a template • There is no slide count limitation for each topic • Insert Tables, figures, images that support the questions for the “Right Safety” • Specify known target related pharmacology that could impact the therapeutic index in patients • Specify proteins with high sequence homology • Annotate with text to articulate key messages

  15. The Right Patients (primary data to the questions below: 3-10 slides as needed) • This page is for instructional purposes only View as a template • There is no slide count limitation for each topic • Insert Tables, figures, images that support the questions for the “Right Patients” • Specify what patients would be targeted for this drug • How would patients be identified or stratified? • Annotate with text to articulate key messages

  16. The Right Commercial Potential (primary data to the questions below: 3-10 slides as needed) • This page is for instructional purposes only View as a template • There is no slide count limitation for each topic • Insert Tables, figures, images that support the questions for the “Right Commercial Potential” • Specify what is the Standard of Care (SOC) for this disease • How will a drug for this target differentiate versus SOC • Specify the competitive landscape for this target or indication? • Annotate with text to articulate key messages

  17. The Right Modality / Assay (primary data to the questions below: 3-10 slides as needed) • This page is for instructional purposes only View as a template • There is no slide count limitation for each topic • Insert Tables, figures, images that support the questions for the “Right Modality / Assay” • Specify the preferred modality (small molecule, PROTAC, peptide, protein, antibody, anti-sense, siRNA) • Has this target been screened before? If so, was it successful or unsuccessful? • What assays and reagents are available for the proposed screen? • Why is the proposed approach thought to be innovative and potentially grant competitive? • Annotate with text to articulate key messages

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