November th FERCAP International Conference Nagasaki, Japan TransCelerate Overview

TransCelerate Background

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 3 R&D Leaders Identified Collaboration As A Key Opportunity For Generating Industry-wide Efficiencies ​ Conducted an industry survey on areas amenable to collaboration Doability Value Clinical Trial Execution Target Validation Translational Endpoints Data Sharing

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 4 TransCelerate Chartered in companies selected areas of focus that shared goals of increased Quality, Patient Safety, and Accelerated Development Timelines. 1.Model Approach for High- Quality Risk-Based Monitoring 2.Shared Site Qualification and Training 3.Shared Investigator Platform 4.Clinical Data Standards (as a collaborator in CFAST) 5.Comparator Drugs for Clinical Trials Founding Members Initiatives Started in 2012

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 5 Non-profit Entity Created to Drive Collaboration Our core values + Quality + Transparency & Openness + Trust & Integrity + Collaboration + Courage Our vision To improve the health of people around the world by accelerating and simplifying the research and development of innovative new therapies. Our mission To collaborate across the global research and development community to identify, prioritize, design and facilitate implementation of solutions designed to drive the efficient, effective and high quality delivery of new medicines.

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 6 Research and CRO Community Investigative sites Strategically focusing engagement efforts with selected key stakeholder groups – the intent is not to recreate, but partner whenever feasible Regulatory Bodies Industry Initiatives 6 An Entity That Engages With The Wider Clinical Ecosystem Globally

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved TransCelerate Members Today

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 8 TransCelerate Organization Structure External Counsel Accounting Firm Audit Firm Administrative Asst. Councils Regulatory Technology Change Management Retained Position Member Representatives Key: Head of Delivery Excellence & Corp Affairs Board of Directors CEO Finance Lead Dir. of Portfolio Dir. of Quality 2 Exploratory Concepts (New Ideas) 12 Active Workstreams (Design, Develop Deploy Phase) 2 Workstreams Closing Out (Business Continuity Phase) Workstreams Clinical Operations Committee Project Management Dir. of Communication Dir. of Operations Contracted Resources

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 9 Workstream Overview Clinical Data Standards Clinical Data Transparency Clinical Trial Diversification Site Qualification and Training Shared Investigator Platform Risk Based Monitoring Quality Management System Placebo / Standard of Care Data Sharing Pediatric Trial Efficiencies Investigator Registry eConsenteLabels Comparator Drugs Common Protocol Template Active Design, Develop, & Deploy Phase Business Continuity Execution Phase

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 10 WorkstreamObjective Shared Investigator Platform Facilitate interaction between investigators and multiple clinical trial sponsors, enabling study planning, study start-up and study conduct activities while reducing the administrative burden on site staff. Investigator Registry Enhance the Shared Investigator Platform and accelerate identification and recruitment of qualified investigators, which will avoid duplication of standard site qualification and the creation of investigator documentation, and thereby reducing cost, study start up time, and trial length. Site Qualifications and Training Collaboration with TransCelerate Member Companies, investigator sites, CROs, and Health Authorities to achieve the goal of enhancing and simplifying clinical trial SQT processes and to reduce administrative burden on sites. Active Workstreams with the Shared Goal of Decreasing the Burden on Investigator Sites

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 11 WorkstreamObjective Comparator Drugs for Clinical Trials Establish a reliable, rapid sourcing of quality products for use in clinical trials for participating Member Companies to avoid counterfeiting and avoid delays in study start-up. Quality Management System Explore ways to improve quality across the industry, through partnership with Health Authorities and other stakeholders. Risk Based Monitoring Create a model approach for implementation based on Health Authority guidance that seeks to improve data integrity and patient safety. Active Workstreams with the Shared Goal of Improving Patient Safety, Data Reliability & Integrity

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 12 WorkstreamObjective Common Protocol Template Work with industry stakeholders and regulators to create a model clinical trial protocol template containing a common structure and common language to improve accuracy in data recordation and speed study start up. Placebo / Standard of Care Data Sharing Enable the sharing of data to allow the re-use of clinical data collected historically from the placebo and standard of care control arms of clinical trials to improve trial design, safety signal interpretation and the patient experience through reduced exposure to sub-optimal therapies. Clinical Data Standards Develop industry-wide Data Standards in priority Therapeutic Area (TAs) to support the exchange and submission of clinical research and meta-data, while improving patient safety and outcomes. Clinical Data Transparency Develop a model approach for redacting privacy information found in clinical study reports and a model approach for the anonymization of patient level data shared with the broader healthcare community. Active Workstreams with the Shared Goal of Digitizing Trial Design (Opportunities to Improve End to End Data Flow in Clinical Studies)

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 13 WorkstreamObjective e-LabelsSupport TransCelerate Member Companies in establishing an innovative information channel using technology to enhance label utility for patients. eConsent (newly approved) Transform one of the most critical processes into a patient- focused, regulatory compliant and digitally supported process with cross-industry alignment. Active Workstreams with the Shared Goal of Innovative Operational Approaches to Speed Progress on Patient-Centered Clinical Trials

SQT Workstream Overview and GCP Training

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 15 Why Shared Site Qualification and Training? 15 Unmet Need: Disparate and redundant GCP training, and collection of non-study specific information, are pain points for investigators and sites, as well as for biopharmaceutical companies Objective: Program established for mutual recognition of GCP training and site qualification credentials Benefits: Realization of improved quality of clinical sites and accelerated study start-up times

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 16 Mutual Recognition of GCP Training A process for mutual recognition of GCP training across member companies, including a set of minimum criteria and a TransCelerate certificate. Unmet need: Instead of adding value, company specific GCP training has become a burden to the sites. Training has to be repeated for each sponsor and sometimes for each study. Member company’s staff spend time chasing site personnel to re-take the course. Benefits: Site personnel appreciate the fact that they now only have to re-take training every 3rd year. Consistent message to the sites; GCP training is important, and member companies accept each other‘s training certificates as long as minimum criteria has been included in the GCP training. 16 Link to the Site Qualification and Training resources site (see minimum criteria and a list of TransCelerate member’s GCP training meeting minimum criteria):

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved TransCelerate Member Companies established Minimum Criteria for GCP training content. 2 Training Providers self-attest to meeting Minimum Criteria via our GCP Training Attestation process. Training Providers receive a certificate and are added our list. 3 TRAINING PROVIDERS GCP TRAINING THAT MEETS THE MINIMUM CRITERIA Sites take GCP training from provider on our list Sites r eceive certificate from training provider Sites presents certificate from training provider for future studies Participating TransCelerate Member Companies can mutually recognize GCP training taken as a part of this program TransCelerate facilitates a GCP training program that creates on-going value for sites 17

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 18 Shared Site Qualification and Training: Forms and Informational Programs Forms Financial Disclosure Form (FiDS) Protocol Informed Consent Tracking Log Site Specific Informed Consent Tracking Log Site Profile Form Curriculum Vitae Template Site Signature and Delegation of Responsibilities Log Form FDA 1572 – Guidance for Completion Informational Programs for Investigator Sites Principal Investigator Oversight Informational Program Note: All assets above are located on the TransCelerate website.

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 19 Site Qualification and Training Roadmap

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