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  • Corporate Presentation December 2015Dual listed company

    Lead asset on track for EU approval

    Partnered with tier-one, big pharma company

    June 2017

  • 2

    This document does not constitute or form part of any offer or invitation to sell or issue, or any solicitation

    of any offer to purchase or subscribe for, any shares in the Company, nor shall any part of it nor the fact of

    its distribution form part of or be relied on in connection with any contract or investment decision relating

    thereto, nor does it constitute a recommendation regarding the securities of the Company.

    This document may contain forward-looking statements and estimates made by the Company, including

    with respect to the anticipated future performance of TiGenix and the market in which it operates. They

    include all statements that are not historical facts. Such statements, forecasts and estimates are based on

    various assumptions and assessments of known and unknown risks, uncertainties and other factors, which

    were deemed reasonable when made but may or may not prove to be correct. Actual events are difficult to

    predict and may depend upon factors that are beyond the Company's control. Therefore, actual results, the

    financial condition, performance or achievements of TiGenix, or industry results, may turn out to be

    materially different from any future results, performance or achievements expressed or implied by such

    statements, forecasts and estimates. Forward-looking statements, forecasts and estimates only speak as

    of the date of this document and no representations are made as to the accuracy or fairness of such

    forward-looking statements, forecasts and estimates. TiGenix disclaims any obligation to update any such

    forward-looking statement, forecast or estimates to reflect any change in the Company’s expectations with

    regard thereto, or any change in events, conditions or circumstances on which any such statement,

    forecast or estimate is based.

    Forward-Looking Statements

  • 3

    Compelling Investment Case Lead product near approval in Europe, strong partner, significant upside potential

     Seasoned management team with deep experience in drug development

     Well capitalized

     Traded on the Nasdaq and Euronext (TIG)

     Ex-US rights licensed to Takeda; key strategic partner for commercial launch

     Up to EUR 380M in potential milestones plus double-digit royalties

     Lead product Cx601 in development for complex perianal fistulas, an orphan indication with high unmet medical need

     Filed for approval in EU; decision expected in 2H2017

     Clear development plan for global pivotal phase III trial to start in 1H2017

     Multiple assets in clinical-stage development

  • 4

    Pipeline Clear commercial potential with future growth opportunities

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    Novel, locally administered treatment for a severe

    complication of Crohn’s disease

    EU approval decision expected 2H2017

  • 6

    Adipose-Derived Stem Cells are Potent Anti-inflammatory Agents Mechanism of Action (MoA) is IDO1-mediated

    • eACSs broadly interact with many players

    in the immune system

    • One of their key in vivo biological roles is

    the control of inflammation to prepare the

    return to homeostasis through:

    • Inhibition of T cell proliferation and pro-

    inflammatory cytokine secretion

    • Induction of anti-inflammatory cytokines

    • Induction of increased number of Tregs

    • Control of NK cell mediated killing

    • Control of monocyte and B cell maturation

    • IDO enzyme is a key player in the MoA

    1 IDO: Indoleamine 2,3-dioxygenase

    Image representation of the proprietary research that supports the key characteristics of eASC mechanism of action.

  • 7

    GMP Facility Approved for Commercial Manufacturing Consistent and robust process

    1 liposuction from healthy donor

    Allogeneic model translates into production scalability

    2,400 patients (Cx601)

  • 8

    Complex Perianal Fistulas A common and severe complication of Crohn’s disease



     Perianal fistulas are chronic, abnormal

    communication between the epithelialized

    surface of the anal canal and the perianal skin

     Perianal fistulas are a complication of Crohn’s

    disease for 30−50% of patients1

     Perianal fistulas in Crohn’s disease are

    difficult to treat with currently available

    therapies and often leads to pain, swelling,

    infection and incontinence

     70−80% of perianal fistulas are classified as


    • Most challenging to treat

    • Often refractory to conventional treatment

    and anti-TNF agents4-6

    1. Schwartz DA, et al. Gastroenterology. 2002;122:875-80; 2. Eglinton TW, et al. Dis Colon Rectum. 2012;55:773-7; 3. Bell SJ, et al

    Aliment Pharmacol Ther. 2003;17:1145-51; 4. Present DH, et al. N Engl J Med. 1999;340:1398-405; 5. Sands BE, et al. N Engl J Med.

    2004;350:876-85; 6. Pearson DC, et al. Ann Intern Med. 1995;123:132-42;

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    Cx601: A Completely Different and Simple Approach A single administration with long-term efficacy

    Method of administration of Cx601: Injection

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    1 Schwartz et al 2002. Gastroenterology 122:875-880 2 Kappelman et al 2013. Dig Dis Sci, 58:519-525 3 Khalili et al 2012. Gut :1686-1692 4 Study commissioned to Vencore Health Analytics Inc based on Truven MarketScan database 5 Göttgens et al 2017. Eur J Gastroenterol Hepatol [Epub ahead of print] 6 Burisch et al 2013. J Crohn’s Colitis 7:322-337

    Estimation of prevalence of Crohn’s Disease patients

    with perianal fistulas

    Adult Crohn’s patients with perianal fistulas (93% CD patients are adults 2)

    Crohn’s patients with perianal fistulas

    50,153 57,359


    From European published data 5,6From US published data 1,2,3 From US real world data (claims

    database) 4

    53,756 Crohn’s patients with perianal fistulas (average)

    Adult Crohn’s patients with perianal fistulas (93% CD patients are adults 2)


    Over 125,000 Adult Crohn’s Patients Suffer Perianal Fistulas 40% of these patients are in the US

  • 11

    With complex perianal fistulas:

    Adult Crohn’s Disease patients with perianal fistulas:




    With complex perianal fistulas and controlled luminal disease:

    With refractory complex perianal fistulas and controlled luminal disease:




    1 Bell et al 2003. Aliment Pharmacol Ther 17:1145-1151 2 Eglinton et al 2012. Dis Colon Rectum 55:773-777 3 Riss et al 2013. Tech Coloproctol 17:89-94 4 Molendijk et al 2014. Inflamm Bowel Dis 20:2022-2028

    In ~75% CD patients perianal fistulas are complex 1-5

    In ~66% CD patients with perianal fistulas luminal disease is controlled

    in ~90% CD patients perianal fistulas are treatment refractory 6-8

    5 Molendijk et al 2015. Gastroenterology 149:918-927 6 Sands et al 2004. N Engl J Med 350:876-885 7 Domenech et al 2005. Aliment Pharmacol Ther 22:1107-1113 8 Rayen et al 2017. Tech Coloproctol 21:119-124

    More than 55,000 Patients Fit the Expected Label Non-controlled luminal disease patients represent a very meaningful opportunity

    49,993 75,190

    Expected label

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    A Real Need for New Treatment Options Available treatments lack long term efficacy and present safety issues

    1 Sands et al 2004. N Engl J Med 350:876-885 2 Grimaud et al 2010. Gastroenterology 138:2275-2281 3 Domenech et al 2005. Aliment Pharmacol Ther

    22:1107-1113 4 Rayen et al 2017. Tech Coloproctol 21:119-124 5 Present et al 1999. N Engl J Med 349:1398-1405 6 Poritz et al 2002. Dis Colon Rectum 45:771-775

    ● Only ~1/5 patients see a response after 1 year of

    continued treatment (IV infusion every 8 weeks) 1,2

    ● Upon treatment discontinuation healed fistulas relapse

    in 2/3 patients within the next year 3

    ● Rarely achieves complete healing in the long-term (13%

    of patients) 4

    ● Efficacy does not increase in combination with

    immunosuppressants 5

    ● Treatment does not abolish the need for surgery 6

    ● Associated with serious infections in 9% of patients and

    to infusion reactions in 11% of patients 7

    ● An average of 4 surgeries over a period of years 9,10 with

    only half of patients healed 9

    ● Fistula surgery is linked to risk of sphincter damage

    leading to faecal incontinence 11,12

    ● 30% and 54% of patients report post-operative

    incontinence to solid and liquid stools 13

    ● High rate of recurrences and failures 8

    ● 33% and 13% of patients with complex anal fistulas

    require ostomy and proctectomy 14 , with no guarantee of

    healing 15,16

    Infliximab (anti-TNFα)

    7 D’Haens et al 2016. J Crohn’s Colitis [Epub] 8 Schwartz et al 2015. Inflamm Bowel Dis 21:723-730 9 Graf et al 2015. Colorectal Dis 18:80-851 10 Galandiuk et al 2005. Ann Surg 241:796-802 11 Norton et al 2013. J Crohn’s Colitis 7:e302-311 12 Geltzeiler et al 2014. Ann Gast