Cell Therapy Limited Ajan Reginald, CEO EMA Roundtable SME Office - PowerPoint PPT Presentation

Cell Therapy Limited Ajan Reginald, CEO EMA Roundtable SME Office 27 th November 2015 This document outlines certain information regarding CTL. It is presented solely for purposes of discussion. Under no circumstances is this document to be used or considered as an offer to sell, or a solicitation of any offer to buy an interest or shares. This may only be made as a private offering to qualified investors. This material is confidential and shall not be reproduced in whole or in part for any purpose. It shall not be disclosed directly or indirectly to any party other than the recipient. By accepting this material the recipient agrees to be bound by all limitations stated herein. It is current as of the date indicated on the front page unless otherwise specified.

Introduction to Cell Therapy Limited  CTL is an SME based in Cardiff, Wales  Founded by Ajan Reginald (CEO), and Sir Martin Evans (CSO)  Lead product details:  Heartcel TM – Allogeneic immuno-modulatory progenitor cell  Tissue-engineered ATMP  Designed to regenerate heart tissue scarred by myocardial infarction  Adult indication in Congenital Coronary Artery Malformations (CCAM) targeted for Conditional MAA submission in 2016 – Letter of Intent to be submitted in January  Paediatric deferral to be requested – potential utility in rare paediatric diseases (Kawasaki Disease, ALCAPA) 1 Confidential March 2015

Directors & Scientific Advisory Board  Ajan Reginald CEO Founder and Director  Prof. Sir President & Founder and Director   Former Roche Global Head of Martin Evans Chief Nobel Laureate, 2007  Emerging Technologies Scientific Copley Medal, Royal Society   Former BCG Consultant Officer Knighted in 2004  Fulbright Scholar  Albert Lasker Award, 2001  Digby, Chairman Former UK Minister for Trade & Lord Jones Investment  Prof. Steve Cardiac Distinguished Ralph Cicerone  of Former Director-General of the Westaby Surgery Professor, Oxford University Birmingham Confederation of British Industry  Consultant Cardiac surgeon.  Chairman, Triumph Motorcycles John Radcliffe Hospital,  Advisor to BP, JCB, Jaguar Oxford  Mr Rhodri Non- Ex-leader of Welsh Government  Morgan Executive First Minister of Wales (2000- Director, 2009)  Head of Chancellor, Swansea University  Prof. Armand Chief Medical Director, Cell Therapy, Audit & Keating Advisor Princess Margaret Hospital Governance  Past President, American Society of Hematology   Dr. Non- Former cardiologist Professor of Medicine  Director, Div’n of Haematology  Francesco Executive Executive-in-Residence at  Epstein Chair in Cell Therapy Granata Director Warburg Pincus  Chairman at Circassia  Former senior executive at Pfizer, Schering-Plough, Biogen  Prof. Sir Cosmetics & Medical Director Mølholm Peter Dermatology Hospital, Denmark  Bjerring Professor, Faculty of Medicine  Dr. Darrin Non- PhD in Biotechnology,  President of European Society Disley Executive Cambridge  Director CEO, Horizon Discovery  Business Leader of the Year, European Life Science Awards  Mr Neil Cardio- MB ChB MRCS MD FRCS   Dr. Mubasher Non- Co-Founder Roberts vascular Consultant Heart Surgeon   Sheikh Executive Ex-Transplant Physician St. Bartholemews Heart  Director Head of Healthcare, Permira Hospital, London   Ex-Partner and Head of MD in progenitor cell therapy Pharmaceuticals, McKinsey

EMA Timelines Nov-15 Dec-15 Jan-16 Feb-16 Mar-16 Apr-16 May-16 Jun-16 Jul-16 Aug-16 Adult� CCAM Orphan� Validation Orphan� Decision CHMP� Scientific� Advice Submit� names� to� NRG Eligibility� Granted Rapporteur� Meetings ATMP� Classification Submit� Letter� of� Intent Submit� MAA Paediatric� Indications Submit� PIP PDCO� Opinion Protocol� Assistance

Overview of support provided to medicines developers by the European Medicines Agency Pre Authorisation MAA 1 Evaluation Post Authorisation Innovation task force i Scientific advice i • Informal meetings Qualification MAA Post-authorisation i i i covering regulatory, technical and Parallel • scientific issues ATMP 2 i scientific advice arising from the • Qualification of CONDITIONAL FEE FEE EXEMPTIONS FOR: • with health development of new EXEMPTION FOR technology • EXTENSION OF MAA innovative medicines, methodologies SMEs PROVIDED assessment MedDRA license new technologies and • Classification of for TYPE-IA, TYPE-IB AND • THAT AGENCY ’ S bodies ATMPs borderline products development TYPE-II VARIATIONS; SCIENTIFIC ADVICE 100% FEE REDUCTION FOR Parallel of medicines • • • Recommendation on • CERTIFICATION HAS BEEN RENEWAL OF MARKETING • SMES 3 scientific advice OF QUALITY FOLLOWED – eligibility for scientific AUTHORISATION; with US FDA services for combined AND NON- DEFERRAL OF FEE • TRANSFER OF MARKETING CLINICAL DATA and borderline PAYMENT • 90% FEE AUTHORISATION TO A FOR SMEs medicines REDUCTIONS PROVISION OF • SECOND SME; DEVELOPING FOR SMEs Pre-MAA inspections • Complimentary TRANSLATIONS OF ATMPs REFERRAL PROCEDURE • PRODUCT service for all INITIATED BY THE 90% FEE REDUCTION FOR • companies INFORMATION MARKETING SMEs – DEFERRAL OF FEE REQUIRED FOR AUTHORISATION HOLDER PAYMENT GRANT OF Including • MARKETING • ANNUAL FEE compassionate AUTHORISATION use, Art. 58 Post-MAA inspections Scientific services applications, i herbal medicines 90% FEE REDUCTION FOR • 90% FEE • SMEs FOR SMEs, ASSISTANCE • REDUCTIONS PROVIDED BY SME OFFICE FOR SMEs Regulatory and administrative assistance i Orphan incentives i Notes : • Protocol assistance, fee reductions Key: and market exclusivity 1. Marketing authorisation application. 2. Advanced therapy medicinal product. Available to everyone 3. Access to MedDRA through registration to EudraVigilance for AVAILABLE TO SMEs ONLY micro-sized and small companies only. 4 Confidential October 2012

✓ SUPPORT RECEIVED BY CTL TO DATE Overview of support provided to medicines developers by the European Medicines Agency Pre Authorisation MAA 1 Evaluation Post Authorisation ✓ Innovation task force i ✓ Scientific advice i • Informal meetings Qualification MAA Post-authorisation i i i covering regulatory, technical and Parallel • scientific issues ATMP 2 i scientific advice arising from the • Qualification of CONDITIONAL FEE FEE EXEMPTIONS FOR: • with health development of new EXEMPTION FOR technology • EXTENSION OF MAA innovative medicines, methodologies SMEs PROVIDED assessment MedDRA license new technologies and • Classification of for TYPE-IA, TYPE-IB AND • THAT AGENCY ’ S bodies ATMPs borderline products development TYPE-II VARIATIONS; SCIENTIFIC ADVICE 100% FEE REDUCTION FOR Parallel of medicines • • • Recommendation on • CERTIFICATION HAS BEEN RENEWAL OF MARKETING • SMES 3 scientific advice OF QUALITY FOLLOWED – eligibility for scientific AUTHORISATION; with US FDA services for combined AND NON- DEFERRAL OF FEE • TRANSFER OF MARKETING CLINICAL DATA and borderline PAYMENT • 90% FEE AUTHORISATION TO A FOR SMEs medicines REDUCTIONS PROVISION OF • SECOND SME; DEVELOPING FOR SMEs Pre-MAA inspections • Complimentary TRANSLATIONS OF ATMPs REFERRAL PROCEDURE • PRODUCT service for all INITIATED BY THE 90% FEE REDUCTION FOR • companies INFORMATION MARKETING SMEs – DEFERRAL OF FEE REQUIRED FOR AUTHORISATION HOLDER PAYMENT GRANT OF Including • MARKETING • ANNUAL FEE compassionate AUTHORISATION use, Art. 58 Post-MAA inspections Scientific services applications, i herbal medicines 90% FEE REDUCTION FOR • 90% FEE • SMEs FOR SMEs, ASSISTANCE • REDUCTIONS PROVIDED BY SME OFFICE FOR SMEs ✓ Regulatory and administrative assistance i ✓ Orphan incentives i Notes : • Protocol assistance, fee reductions Key: and market exclusivity 1. Marketing authorisation application. 2. Advanced therapy medicinal product. Available to everyone 3. Access to MedDRA through registration to EudraVigilance for AVAILABLE TO SMEs ONLY micro-sized and small companies only. 5 Confidential October 2012

CTL has received significant support  SME incentives and support received to date include:  Advice from SME office on administrative and procedural issues around:  ATMP classification  Orphan designation  Paediatric investigation plans  Protocol assistance from CHMP, including potential paediatric indications to consider, opportunity to submit for Conditional Approval  Fee reductions for scientific advice 6 Confidential March 2015