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

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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

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SLIDE 1

Cell Therapy Limited

Ajan Reginald, CEO EMA Roundtable SME Office

27th 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

  • ffer 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

  • therwise specified.
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SLIDE 2

CTL is an SME based in Cardiff, Wales Founded by Ajan Reginald (CEO), and Sir Martin Evans (CSO) Lead product details:

 HeartcelTM – 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)

Introduction to Cell Therapy Limited

1 Confidential March 2015

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SLIDE 3

Dr. Francesco Granata Non- Executive Director

  • Former cardiologist
  • Executive-in-Residence at

Warburg Pincus

  • Chairman at Circassia
  • Former senior executive at

Pfizer, Schering-Plough, Biogen Mr Rhodri Morgan Non- Executive Director, Head of Audit & Governance

  • Ex-leader of Welsh Government
  • First Minister of Wales (2000-

2009)

  • Chancellor, Swansea University

Digby, Lord Jones

  • f

Birmingham Chairman

  • Former UK Minister for Trade &

Investment

  • Former Director-General of the

Confederation of British Industry

  • Chairman, Triumph Motorcycles
  • Advisor to BP, JCB, Jaguar
  • Dr. Darrin

Disley Non- Executive Director

  • PhD in Biotechnology,

Cambridge

  • CEO, Horizon Discovery
  • Business Leader of the Year,

European Life Science Awards

  • Prof. Steve

Westaby Cardiac Surgery

  • Distinguished Ralph Cicerone

Professor, Oxford University

  • Consultant Cardiac surgeon.

John Radcliffe Hospital, Oxford

  • Prof. Sir

Peter Bjerring Cosmetics & Dermatology

  • Medical Director Mølholm

Hospital, Denmark

  • Professor, Faculty of Medicine
  • President of European Society

Mr Neil Roberts Cardio- vascular

  • MB ChB MRCS MD FRCS
  • Consultant Heart Surgeon
  • St. Bartholemews Heart

Hospital, London

  • MD in progenitor cell therapy
  • Prof. Armand

Keating Chief Medical Advisor

  • Director, Cell Therapy,

Princess Margaret Hospital

  • Past President, American

Society of Hematology

  • Professor of Medicine
  • Director, Div’n of Haematology
  • Epstein Chair in Cell Therapy

Directors & Scientific Advisory Board

  • Dr. Mubasher

Sheikh Non- Executive Director

  • Co-Founder
  • Ex-Transplant Physician
  • Head of Healthcare, Permira
  • Ex-Partner and Head of

Pharmaceuticals, McKinsey

  • Prof. Sir

Martin Evans President & Chief Scientific Officer

  • Founder and Director
  • Nobel Laureate, 2007
  • Copley Medal, Royal Society
  • Knighted in 2004
  • Albert Lasker Award, 2001

Ajan Reginald CEO

  • Founder and Director
  • Former Roche Global Head of

Emerging Technologies

  • Former BCG Consultant
  • Fulbright Scholar
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SLIDE 4

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

  • f

Intent Submit MAA Paediatric Indications Submit PIP PDCO Opinion Protocol Assistance

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SLIDE 5

4 Confidential October 2012

MedDRA license

  • 100% FEE REDUCTION FOR

SMES3

Pre-MAA inspections

  • 90% FEE REDUCTION FOR

SMEs – DEFERRAL OF FEE PAYMENT

MAA1 Evaluation Post Authorisation

Overview of support provided to medicines developers by the European Medicines Agency

Notes:

  • 1. Marketing authorisation application.

2. Advanced therapy medicinal product. 3. Access to MedDRA through registration to EudraVigilance for micro-sized and small companies only.

Qualification ATMP2 Scientific services Regulatory and administrative assistance Orphan incentives MAA

  • Qualification of

new methodologies for development

  • f medicines
  • Classification of

ATMPs

  • CERTIFICATION

OF QUALITY AND NON- CLINICAL DATA FOR SMEs DEVELOPING ATMPs

  • CONDITIONAL FEE

EXEMPTION FOR SMEs PROVIDED THAT AGENCY’S SCIENTIFIC ADVICE HAS BEEN FOLLOWED – DEFERRAL OF FEE PAYMENT

  • PROVISION OF

TRANSLATIONS OF PRODUCT INFORMATION REQUIRED FOR GRANT OF MARKETING AUTHORISATION

  • Including

compassionate use, Art. 58 applications, herbal medicines

  • 90% FEE

REDUCTIONS FOR SMEs

  • FOR SMEs, ASSISTANCE

PROVIDED BY SME OFFICE

  • Protocol assistance, fee reductions

and market exclusivity

Innovation task force

Pre Authorisation

Scientific advice

  • Parallel

scientific advice with health technology assessment bodies

  • Parallel

scientific advice with US FDA

  • 90% FEE

REDUCTIONS FOR SMEs

Post-MAA inspections

  • 90% FEE REDUCTION FOR

SMEs

i i i i i i i i

  • Informal meetings

covering regulatory, technical and scientific issues arising from the development of innovative medicines, new technologies and borderline products

  • Recommendation on

eligibility for scientific services for combined and borderline medicines

  • Complimentary

service for all companies

Key:

Available to everyone AVAILABLE TO SMEs ONLY

Post-authorisation i

FEE EXEMPTIONS FOR:

  • EXTENSION OF MAA
  • TYPE-IA, TYPE-IB AND

TYPE-II VARIATIONS;

  • RENEWAL OF MARKETING

AUTHORISATION;

  • TRANSFER OF MARKETING

AUTHORISATION TO A SECOND SME;

  • REFERRAL PROCEDURE

INITIATED BY THE MARKETING AUTHORISATION HOLDER

  • ANNUAL FEE
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SLIDE 6

5 Confidential October 2012

MedDRA license

  • 100% FEE REDUCTION FOR

SMES3

Pre-MAA inspections

  • 90% FEE REDUCTION FOR

SMEs – DEFERRAL OF FEE PAYMENT

MAA1 Evaluation Post Authorisation

Overview of support provided to medicines developers by the European Medicines Agency

Notes:

  • 1. Marketing authorisation application.

2. Advanced therapy medicinal product. 3. Access to MedDRA through registration to EudraVigilance for micro-sized and small companies only.

Qualification ATMP2 Scientific services Regulatory and administrative assistance Orphan incentives MAA

  • Qualification of

new methodologies for development

  • f medicines
  • Classification of

ATMPs

  • CERTIFICATION

OF QUALITY AND NON- CLINICAL DATA FOR SMEs DEVELOPING ATMPs

  • CONDITIONAL FEE

EXEMPTION FOR SMEs PROVIDED THAT AGENCY’S SCIENTIFIC ADVICE HAS BEEN FOLLOWED – DEFERRAL OF FEE PAYMENT

  • PROVISION OF

TRANSLATIONS OF PRODUCT INFORMATION REQUIRED FOR GRANT OF MARKETING AUTHORISATION

  • Including

compassionate use, Art. 58 applications, herbal medicines

  • 90% FEE

REDUCTIONS FOR SMEs

  • FOR SMEs, ASSISTANCE

PROVIDED BY SME OFFICE

  • Protocol assistance, fee reductions

and market exclusivity

Innovation task force

Pre Authorisation

Scientific advice

  • Parallel

scientific advice with health technology assessment bodies

  • Parallel

scientific advice with US FDA

  • 90% FEE

REDUCTIONS FOR SMEs

Post-MAA inspections

  • 90% FEE REDUCTION FOR

SMEs

i i i i i i i i

  • Informal meetings

covering regulatory, technical and scientific issues arising from the development of innovative medicines, new technologies and borderline products

  • Recommendation on

eligibility for scientific services for combined and borderline medicines

  • Complimentary

service for all companies

Key:

Available to everyone AVAILABLE TO SMEs ONLY

Post-authorisation i

FEE EXEMPTIONS FOR:

  • EXTENSION OF MAA
  • TYPE-IA, TYPE-IB AND

TYPE-II VARIATIONS;

  • RENEWAL OF MARKETING

AUTHORISATION;

  • TRANSFER OF MARKETING

AUTHORISATION TO A SECOND SME;

  • REFERRAL PROCEDURE

INITIATED BY THE MARKETING AUTHORISATION HOLDER

  • ANNUAL FEE

✓ ✓ ✓ ✓ ✓

SUPPORT RECEIVED BY CTL TO DATE

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SLIDE 7

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

CTL has received significant support

6 Confidential March 2015

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SLIDE 8

7 Confidential October 2012

MedDRA license

  • 100% FEE REDUCTION FOR

SMES3

Pre-MAA inspections

  • 90% FEE REDUCTION FOR

SMEs – DEFERRAL OF FEE PAYMENT

MAA1 Evaluation Post Authorisation

Overview of support provided to medicines developers by the European Medicines Agency

Notes:

  • 1. Marketing authorisation application.

2. Advanced therapy medicinal product. 3. Access to MedDRA through registration to EudraVigilance for micro-sized and small companies only.

Qualification ATMP2 Scientific services Regulatory and administrative assistance Orphan incentives MAA

  • Qualification of

new methodologies for development

  • f medicines
  • Classification of

ATMPs

  • CERTIFICATION

OF QUALITY AND NON- CLINICAL DATA FOR SMEs DEVELOPING ATMPs

  • CONDITIONAL FEE

EXEMPTION FOR SMEs PROVIDED THAT AGENCY’S SCIENTIFIC ADVICE HAS BEEN FOLLOWED – DEFERRAL OF FEE PAYMENT

  • PROVISION OF

TRANSLATIONS OF PRODUCT INFORMATION REQUIRED FOR GRANT OF MARKETING AUTHORISATION

  • Including

compassionate use, Art. 58 applications, herbal medicines

  • 90% FEE

REDUCTIONS FOR SMEs

  • FOR SMEs, ASSISTANCE

PROVIDED BY SME OFFICE

  • Protocol assistance, fee reductions

and market exclusivity

Innovation task force

Pre Authorisation

Scientific advice

  • Parallel

scientific advice with health technology assessment bodies

  • Parallel

scientific advice with US FDA

  • 90% FEE

REDUCTIONS FOR SMEs

Post-MAA inspections

  • 90% FEE REDUCTION FOR

SMEs

i i i i i i i i

  • Informal meetings

covering regulatory, technical and scientific issues arising from the development of innovative medicines, new technologies and borderline products

  • Recommendation on

eligibility for scientific services for combined and borderline medicines

  • Complimentary

service for all companies

Key:

Available to everyone AVAILABLE TO SMEs ONLY

Post-authorisation i

FEE EXEMPTIONS FOR:

  • EXTENSION OF MAA
  • TYPE-IA, TYPE-IB AND

TYPE-II VARIATIONS;

  • RENEWAL OF MARKETING

AUTHORISATION;

  • TRANSFER OF MARKETING

AUTHORISATION TO A SECOND SME;

  • REFERRAL PROCEDURE

INITIATED BY THE MARKETING AUTHORISATION HOLDER

  • ANNUAL FEE

✓

SUPPORT CTL INTENDS TO SEEK

✓ ✓ ✓ ✓ ✓

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SLIDE 9

SME incentives and support to be requested:

 Certification of Quality and Non-Clinical Data for ATMP  Advice from SME office on administrative and procedural issues around Conditional MAA  Scientific advice on Conditional MAA in adults  Scientific advice on Paediatric plans  Scientific Services on Compassionate Use Programmes  Fee reductions for procedures in the pre- and post-marketing- authorisation phases, including scientific advice, inspections, line extensions and variations  Assistance in translating product information submitted within the Conditional MAA  Deferral of the fee payable for an application for marketing authorisation or related inspection

CTL intends to ask for additional support

8 Confidential March 2015