Regenerative Medicine Industry Outlook 2014

1.  About ARM a.  Review of Regenerative Medicine b.  RM Stakeholders c.  Major Industry Sub-Sectors

2.  RM Product and Clinical Trial Overview 3.  2013 RM Financial Performance Analysis 4.  Highlights from ARM Pharmaceutical Survey 5.  Panel Discussions

a.  Key Clinical Data Inflection Points for 2014 b.  Recent Financings in the Sector

•  Founded in 2009, ARM includes all of the major stakeholders in the field of RM working together to bring transformative healthcare solutions to patients

•  ARM’s activities focus on reducing uncertainty in the RM field through legislative, advocacy, regulatory, reimbursement, informational and scientific initiatives

5%

33%

24%

17%

9%

9% 3% Big Pharma (5%)

Therapeu8c Companies (33%)

Tools and Service Companies (24%) Pa8ent Advocates, Founda8ons and Associa8ons (17%) Academic Research Ins8tu8ons (9%) Tissue Engineering, Biomaterials and Devices (9%) Capital Providers and other Financial Ins8tu8ons (3%)

ARM Membership Demographic 152 Members

Regenerative Medicine is a revolutionary field focused on translating fundamental knowledge in biology, chemistry and physics into materials, devices, systems and a variety of therapeutic strategies which augment, repair, replace or regenerate organs and tissues.

60%

8%

5%

27%

Cell Therapy Companies

Gene Therapy Companies

Small Molecule and Biologic Companies -‐ Endogenous Ac8va8on Tissue Engineering Companies

Therapeu8c and Device Companies

40%

Service Companies

12%

Cell & Tissue Banks 33%

Tools & Reagent

Companies 15%

Regen Med Industry Sectors

476 Companies Analyzed

Company Breakdown: Therapeutics

2010 2011 2012 2013

Product Revenue

$460 Million

$730 Million

$900 Million

$1.3 Billion

Cell Therapy Product Revenue Sample of Leading Commercial Cell Therapy Products

There are Currently Over 40 Cell Therapy Products Commercially Available

35%

35%

11%

10%

7% 2%

Non-healing wounds

Musculoskeletal

Skin

Cancer

Ocular

Cardiovascular

Company Product AlloSource (distributed by NuVasive) Osteocel Alphatec Spine PureGen Anterogen Cupistem Avita Medical ReCell BioD (distributed by Amedica) BioDfactor BioD (distributed by Amedica) BioDfence Dendreon Provenge Fibrocell LAVIV Genzyme, a Sanofi company Carticel Genzyme, a Sanofi company Epicel Genzyme, a Sanofi company MACI Medipost Cartistem NuVasive Osteocel Organogenesis Apligraf Organogenesis GINTUIT Orthofix Trinity/Trinity Evolution Osiris Therapeutics Grafix Pharmacell Heartcelligram-AMI Shire Dermagraft TiGenix ChondroCelect Zimmer and ISTO Technologies DeNovo NT

By Jurisdiction

United States Dermagraft Osteocel PureGen BioDfactor BioDfence LAVIV Provenge Carticel Epicel Nucel Apligraf GINTUIT Trinity Evolution Grafix DeNovoET Prokera AmnioGraft

Europe MySkin CryoCell ReCell CartiCel Epicel MACI AlloStem BioSeed-C Co.don Chondrospheres Epidex EpiGraft ChrondroCelect

Other Regulated Jurisdictions Cupistem (S.Korea)

Heartcelligram (S. Korea)

Cartistem (S. Korea)

J-TEC Epidermis (Japan)

J-TEC Cartilage (Japan)

J-TEC Corneal Epithelium (Japan) CureXcell (Israel)

Prochymal (Canada & NZ)

Source: ARM 2013 Annual Report

“The number of cell and gene therapies in clinical pipelines has grown from 429 to 575 in the past five years according to McKinsey & Co. The pipeline is also maturing, with more treatments in Phase III trials.”

Diller, Wendy "Shedding Earlier Fears, Pharma Takes A Fresh Look At Cell, Gene Therapies."

Elsivier Business Intelligence: In Vivo; December 23, 2013.

Cancer, 28%

Cardiovascular, 12%

Non-‐healing wounds,

8%

Musculoskeletal, 8%

AutoImmune, 7%

Ocular, 5%

Stroke, 5%

Diabetes, 4%

Spinal Cord Injury , 3%

Skin, 2%

Other, 18%

Research Pre-Clinical Early-Stage (Ph. I) Mid-Stage (Ph. II) Late-Stage (Ph. III)

58 Trials 245 Trials 70 Trials 174 Trials 24 Trials

ARM Database

Ø  185 Therapeutic Companies

Ø  320 Regen Med Products

Ongoing Industry-Sponsored RM Trials by Stage

Late-‐Stage Industry-‐Sponsored RM Trials Early-‐to-‐Mid Stage Industry-‐Sponsored RM Trials

Cancer, 32%

Musculoskeletal, 28%

Non-healing wounds, 15%

Cardiovascular, 11%

AutoImmune, 4%

Ocular, 4%

Stroke, 2% Skin, 2% Other, 2%

Date Company Product Indication Milestone

1Q14 Pluristem PLX-PAD Trauma Phase I/II results

1H14 Cytori ADSRCs Myocardial Ischemia Phase II results

1H14 Athersys MultiStem Ischemic Stroke Preliminary Phase II results

2Q14 Athersys MultiStem Ulcerative Colitis Phase II results

3Q14 NeoStem AMR001 Myocardial Infarction Phase II results

4Q14 TiGenix Cx601 Perianal Fistula Phase III results

2H14 ReNeuron ReN009 Critical Limb Ischemia Phase I results

2H14 Fibrocell Laviv Burn Scar Phase II results

2014 Juventas JVS100 Cardiovascular Failure Phase II results

4Q15 Cardio3 C-Cure Congestive Heart Failure Phase III results

2015 Mesoblast Revascor Congestive Heart Failure Market launch

2015 bluebird LentiGlobin Sickle Cell/Beta Thalassemia Market launch

Mid-2015 ReNeuron ReN001 Cerebral Ischemia Phase II End

Source: Medtrack

2014 Cell-Based Immunotherapy Trials to Watch For

Late-Stage Mid-Stage Early-Stage

Number of Trials 13 54 10

Industry-Sponsored Cell-Based Immunotherapy Trials

Source: Medtrack

Date Company Product Indication Milestone

1Q2014 Northwest Biotherapeutics

DCVax Direct Solid Tumors Phase I/II results

2H2014 Opexa Tcelna MS Phase IIb End

3Q2014 Prima BioMed Cvac. Ovarian Cancer Interim Phase III results

4Q2014 Northwest Biotherapeutics

DCVax L Glioblastoma Phase III results

Source: ARM 2013 Annual Report

Deal Type Company(s) Deal Value/Upfront Date

Collaboration Sangamo / Biogen Idec 20M 1/9/14

Collaboration/Licensing Deal Capricor / J&J 12.5M 1/6/14

Acquisition Intrexon / Medistem 26M 12/20/13

Licensing Deal Pluristem / CHA Biotech 10.4M 12/17/13

Licensing Deal Cytori / Lorem Vascular 24M 11/4/13

Acquisition Mesoblast / Osiris 100M 10/11/13

Collaboration Stratatech / BARDA Contract 47.2M 7/31/13

Collaboration Tengion / Celgene 33.6M 7/1/13

Merger Capricor / Nile Therapeutics NA 7/8/13

Acquisition Smith & Nephew / Healthpoint 782M 11/28/12

Acquisition Royal DSM / Kensey Nash 360M 5/3/12

Acquisition Cytomedix / Aldagen 16M 2/8/12

Licensing Deal Sangamo / Shire 13M 2/1/12

Company Amount Raised Date Exchange Performance since IPO

Cardio 3 Biosciences $31.3M 6/4/13 EBR 53.45%

bluebird bio $116M 6/18/13 NASDAQ (22.04%)

Capricor Therapeutics N/A 7/8/13 OTCMKTS 853.95%

Cellular Dynamics International $46M 7/24/13 NASDAQ 82.84%

Organovo $46.6M 8/7/13 Up-listed to NYSE 125.6%

Intrexon $184M 8/13/13 NYSE (3.76%)

Regeneus Ltd $10.5M 9/12/13 ASX 88.89%

Fate Therapeutics $40.4M 10/4/13 NASDAQ (3.78%)

UniQure $75M (expected) 1/2/14 (file date) NASDAQ N/A

Initial Public Offerings by Regenerative Medicine Companies in 2013

2012 2013

PIPES, VC & Private Equity $765M $644.1M

Public Offerings $170M $736.6M

TOTAL $935M $1.381B

$0

$100

$200

$300

$400

$500

$600

$700

$800

$900

2012 2013

PIPES, VC & Private Equity

Public Offerings

0% 10% 20% 30% 40% 50% 60% 70% 80%

Survey Participants: Allergan, Amgen, Baxter, Biogen Idec, Boehringer Ingelheim, Celgene, Eli Lilly, GSK, Johnson & Johnson, Merck Serono, Novartis, Novo Nordisk, Pfizer, Roche, Sanofi-Genzyme, Shire

0% 10% 20% 30% 40% 50% 60% 70%

Where Pharma is Investing in Regenerative Medicine

Pharma’s Interest in the Pillar Technologies •  Areas showing moderate to significant interest

0%

10%

20%

30%

40%

50%

60%

70%

80%


0% 10% 20% 30% 40% 50% 60% 70%

Over Next

10 yrs Where Pharma Sees Therapeutic Opportunities


What Pharma Sees as the Key Challenges •  Areas posing significant and moderate concerns

0%

5%

10%

15%

20%

25%

30%

35%

40%

45%

50%

significant concern/challenge

moderate concern/challenge


•  We realize it’s a frontier technology beyond a five year time horizon and we don’t want to miss the boat. Our company is engaged in various levels and resources are internally devoted.

•  Focusing on rare diseases allows us to test transformative platforms on small patient groups with lower regulatory boundaries.

•  We would like to invest early, close to proof-of-concept. We will continue to invest in areas such as stem cells, gene therapy and other RM venture investments.

•  In 10 years we will crack Diabetes. This is a major area of interest. •  Most promising areas of RM include the ischemic space/cardiovascular, autoimmune/UC/IB/

GVHD, skin and musculoskeletal related injury and disease. •  We believe that monogenic disease is where you can focus and be successful because

there's no other therapeutic option. We can also be successful in this area because the MOA is 100% clear. Large indications are tough because we don't really understand the disease.

•  iPSCs for drug discovery, toxicology and modeling is our core focus in regenerative medicine. This technology not only enhances drug discovery, it is a paradigm shift in drug discovery.

•  Most of our efforts in RM would be through partnerships…we want to be working with the experts.

Moderator: •  Jason Kolbert, Managing Director, Maxim Group

Panelists: •  Robert Brenner, President & CEO, AlloCure •  Andrew Pecora, Chief Visionary Officer, NeoStem •  Marc Penn, Chief Medical Officer, Juventas Therapeutics;

Co-PI of MultiStem Cardio Trial, Athersys •  Eliseo Salinas, EVP & Head of R&D, StemCells, Inc.

Moderator: •  Dave Stadinski, Head, Equity Capital Markets, Piper Jaffray

Panelists: •  Christopher Calhoun, CEO, Cytori Therapeutics •  Keith Murphy, Chairman & CEO, Organovo •  Michael Schuster, Founding Executive & Head of

Global Therapeutic Products, Mesoblast •  Jeffrey Walsh, Chief Operating Officer, bluebird bio

Regenerative Medicine Industry Outlook 2014

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