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Cancer Vaccine Therapies: Failures and Future Opportunities Michael D Cancer Vaccine Therapies: Failures and Future Opportunities Michael D. Becker, Janet Dally, and Jeffrey Martini, Ph.D. Michael D. Becker President & CEO [email protected] Janet Dally Senior Vice President [email protected] Jeffrey Martini, Ph.D. Vice President [email protected] MD Becker Partners LLC 12 Penns Trail, #231 Newtown, PA 18940 Main phone: 267-756-7094 Email: [email protected] Web: www.mdbpartners.com www.mdbpartners.com Copyright © 2010 MD Becker Partners LLC All rights reserved MD Becker Partners LLC [This page intentionally left blank] Cancer Vaccine Therapies: Failures and Future Opportunities Page 2 MD Becker Partners LLC MD Becker Partners’ reports are limited publications that contain valuable information provided to a select group of customers. Our reports are for our customers’ internal use and not for general publication or disclosure to third parties. No part of this report may be given, lent, resold, or disclosed to non-customers without written permission. Furthermore, no part may be reproduced, stored in a retrieval system, or transmitted in any form or by any means, electronic, mechanical, photocopying, recording, or otherwise, without the permission of the publisher. For additional information, please refer to Appendix G: Legal Disclaimer. For information regarding permission, write: MD Becker Partners LLC 12 Penns Trail, #231 Newtown, PA 18940 Main phone: 267-756-7094 Email: [email protected] Web: www.mdbpartners.com MD Becker Partners is a boutique management and strategy consulting firm focusing on both public and private companies in the life sciences industry, including pharmaceuticals, biotechnology, and medical devices. We also work with venture capitalists, institutional investors, and others that provide capital to these companies. As a strategic supporter and partner, everything we do is focused on helping our clients increase visibility, unlock stakeholder value and access resources to grow their business. To accomplish this, we integrate strategy, relations, and operational capabilities and apply them to carefully conceived and expertly enacted tactics. In addition, MD Becker Partners limits its client base in order to provide the personalized and dedicated service that only a boutique firm can offer. Cancer Vaccine Therapies: Failures and Future Opportunities Page 3 MD Becker Partners LLC TABLE OF CONTENTS EXECUTIVE SUMMARY .................................................................................................................... 7 THE SCIENCE BEHIND CANCER VACCINES AND IMMUNOTHERAPY ............................................. 11 THE IMMUNE RESPONSE OVERVIEW ................................................................................................................................ 11 SCIENTIFIC RATIONALE FOR IMMUNE SYSTEM MODULATION ................................................................................................ 13 CANCER VACCINES AND IMMUNOTHERAPY ................................................................................ 14 PASSIVE IMMUNOTHERAPY AND ANTIBODIES..................................................................................................................... 16 PROPHYLACTIC ONCOLOGY VACCINES .............................................................................................................................. 17 AUTOLOGOUS AND ALLOGENEIC CANCER IMMUNOTHERAPIES .............................................................................................. 18 CELLULAR VACCINES ..................................................................................................................................................... 22 DENDRITIC CELLS ......................................................................................................................................................... 24 PEPTIDE VACCINES ....................................................................................................................................................... 26 GENE TRANSFER .......................................................................................................................................................... 32 CLINICAL TRIAL DIFFICULTIES ........................................................................................................ 34 TOO EARLY FOR THE CLINIC ........................................................................................................................................... 34 WEAK IMMUNE RESPONSE AND IMMUNOSUPPRESSION ...................................................................................................... 36 CLINICAL TRIAL DESIGN AND ENDPOINTS .......................................................................................................................... 37 THE MARKET ................................................................................................................................. 40 THE CANCER MARKET ................................................................................................................................................... 40 COMMERCIALIZATION CHALLENGES ................................................................................................................................. 41 COMPETITION ............................................................................................................................................................. 42 THE FUTURE OF CANCER VACCINES ............................................................................................. 49 POTENTIAL 2010 INDUSTRY CATALYSTS ....................................................................................... 50 APPENDIX A: PHYSICIAN ROUNDTABLE DISCUSSION ................................................................... 51 APPENDIX B: PHYSICIAN BIOGRAPHIES ........................................................................................ 69 JAMES L. GULLEY, M.D., PH.D., F.A.C.P. ....................................................................................................................... 69 SUSAN SLOVIN, M.D., PH.D. ......................................................................................................................................... 70 DAVID BERD, M.D. ...................................................................................................................................................... 71 Cancer Vaccine Therapies: Failures and Future Opportunities Page 4 MD Becker Partners LLC APPENDIX C: COMPANY PROFILES ................................................................................................ 77 ADVAXIS, INC. (ADXS.OB) ........................................................................................................................................... 77 ALPHAVAX, INC. (PRIVATE) ............................................................................................................................................ 79 ANTIGENICS, INC. (AGEN) ............................................................................................................................................ 80 APTHERA, INC. (PRIVATE) .............................................................................................................................................. 82 ARGOS THERAPEUTICS (PRIVATE) .................................................................................................................................... 83 AVAX TECHNOLOGIES, INC. (AVXT.PK) .......................................................................................................................... 84 BAVARIAN NORDIC A/S (BAVA.CO) .............................................................................................................................. 86 BIOSANTE PHARMACEUTICALS, INC. (BPAX) .................................................................................................................... 87 BIOVEST INTERNATIONAL, INC. (BVTI.PK) ........................................................................................................................ 89 BRISTOL-MYERS SQUIBB (BMY) ..................................................................................................................................... 91 CEL-SCI CORPORATION (CVM) ..................................................................................................................................... 93 CELLDEX THERAPEUTICS, INC. (CLDX) ............................................................................................................................. 95 CYTOS BIOTECHNOLOGY AG (CYTN.SW) ........................................................................................................................ 97 DENDREON CORPORATION (DNDN) ............................................................................................................................... 98 GENEREX BIOTECHNOLOGY (GNBT) .............................................................................................................................. 100 GENITOPE CORPORATION (GTOP.PK) ........................................................................................................................... 102 GERON CORPORATION (GERN).................................................................................................................................... 104 GLAXOSMITHKLINE PLC (GSK) ....................................................................................................................................
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