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Page 1: Presentación de PowerPoint - Jefferies NV(1).pdf · of the treated perianal fistula confirmed by centrally blinded MRI assessment in patients with clinical remission at any previous

Corporate PresentationDecember 2015Dual listed company

Lead asset on track for EU approval

Partnered with tier-one, big pharma company

June 2017

Page 2: Presentación de PowerPoint - Jefferies NV(1).pdf · of the treated perianal fistula confirmed by centrally blinded MRI assessment in patients with clinical remission at any previous

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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

Page 3: Presentación de PowerPoint - Jefferies NV(1).pdf · of the treated perianal fistula confirmed by centrally blinded MRI assessment in patients with clinical remission at any previous

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Compelling Investment CaseLead 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 orphanindication 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

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PipelineClear commercial potential with future growth opportunities

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Cx601

Novel, locally administered treatment for a severe

complication of Crohn’s disease

EU approval decision expected 2H2017

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Adipose-Derived Stem Cells are Potent Anti-inflammatory AgentsMechanism 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.

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GMP Facility Approved for Commercial ManufacturingConsistent and robust process

1 liposuction from healthy donor

Allogeneic model translates into production scalability

2,400 patients (Cx601)

Page 8: Presentación de PowerPoint - Jefferies NV(1).pdf · of the treated perianal fistula confirmed by centrally blinded MRI assessment in patients with clinical remission at any previous

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Complex Perianal FistulasA common and severe complication of Crohn’s disease

Fistula

Fistula

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

complex2,3

• 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 ApproachA single administration with long-term efficacy

Method of administration of Cx601: Injection

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1 Schwartz et al 2002. Gastroenterology 122:875-8802 Kappelman et al 2013. Dig Dis Sci, 58:519-5253 Khalili et al 2012. Gut :1686-16924 Study commissioned to Vencore Health Analytics Inc based on Truven MarketScan database5 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

80,849

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

database) 4

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

Adult Crohn’s patients with perianal fistulas (93%

CD patients are adults 2)75,19049,993

Over 125,000 Adult Crohn’s Patients Suffer Perianal Fistulas40% of these patients are in the US

Page 11: Presentación de PowerPoint - Jefferies NV(1).pdf · of the treated perianal fistula confirmed by centrally blinded MRI assessment in patients with clinical remission at any previous

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With complex perianal fistulas:

Adult Crohn’s Disease patients with perianal fistulas:

37,495

24,746

22,272

With complex perianal fistulas and controlled luminal disease:

With refractory complex perianal fistulas and controlled luminal disease:

56,392

37,219

33,497

1 Bell et al 2003. Aliment Pharmacol Ther 17:1145-11512 Eglinton et al 2012. Dis Colon Rectum 55:773-7773 Riss et al 2013. Tech Coloproctol 17:89-944 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 350:876-8857 Domenech et al 2005. Aliment Pharmacol Ther 22:1107-11138 Rayen et al 2017. Tech Coloproctol 21:119-124

More than 55,000 Patients Fit the Expected LabelNon-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 OptionsAvailable treatments lack long term efficacy and present safety issues

1 Sands et al 2004. N Engl J Med 350:876-8852 Grimaud et al 2010. Gastroenterology 138:2275-22813 Domenech et al 2005. Aliment Pharmacol Ther

22:1107-11134 Rayen et al 2017. Tech Coloproctol 21:119-1245 Present et al 1999. N Engl J Med 349:1398-14056 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 21:723-7309 Graf et al 2015. Colorectal Dis 18:80-85110 Galandiuk et al 2005. Ann Surg 241:796-80211 Norton et al 2013. J Crohn’s Colitis 7:e302-311 12 Geltzeiler et al 2014. Ann Gastro 27:320-33013 Riss et al 2013. Tech Coloproctol 17:89-94

Surgery

14 Molendijk et al 2014. Inflamm Bowel Dis 20:2022-2028

15 Figg et al 2009. Dis Colon Rectum 52:646-65016 Lee et al 2017. Colorectal Dis 19:418-429

Only medical treatment approved for fistulas in Crohn’s

disease

Complex fistulas require surgical intervention 8

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Pivotal Phase III Design Validated by EMA and FDAComplex fistulas refractory to best available standard of care

• Randomized, double-blind, placebo controlled

• 212 patients randomized 1:1

• Patients with non-active or mildly-active Crohn’s Disease

• 40% of patients with multiple tract fistulas

• Draining fistulas despite active treatment (majority anti-TNF)

• All patients stayed on their best standard of care treatment

• All draining tracts treated

• Efficacy defined as combined remission: clinical remission and lack of

abscesses with magnetic resonance imaging

• Efficacy measured at 24 and 52 weeks. Safety follow up to 104 weeks

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Cx601: Primary Endpoint Met at Week 24

Patients receiving Cx601 had a 44% greater probability of achieving Combined

Remission1 than placebo patients. Results published in The Lancet in July 2016

Shorter median time to clinical remission (6.7 weeks for Cx601 vs 14.6 weeks in

the control group)

51.5%

35.6%

0

20

40

60%

Combined Remission at W24 (mITT2 Population n= 204)

p = 0.021

1 Closure of all treated external openings draining at baseline despite gentle finger compression, and absence of collections > 2cm

by MRI (Magnetic Resonance Imaging)2 mITT: modified Intention To Treat i.e. patients randomized, treated and with ≥1 post-baseline assessment. Efficacy results are

consistent across all statistical populations

Cx601 Control

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Cx601: Benefit Sustained, Lower Relapse Rate at Week 52

1 Closure of all treated external openings draining at baseline despite gentle finger compression, and absence of collections > 2cm by MRI

(Magnetic Resonance Imaging)2 Relapse: reopening of any of the treated external openings with active drainage as clinically assessed, or development of perianal collection ≥2cm

of the treated perianal fistula confirmed by centrally blinded MRI assessment in patients with clinical remission at any previous visit3 mITT: modified Intention To Treat i.e. patients randomized, treated and with ≥1 post-baseline assessment. Efficacy results are consistent across all

statistical populations

75.0% of the patients treated with Cx601 who were in combined remission at W24 did not

relapse2, compared to 55.9% for patients in the placebo arm

75.0%

55.9%

0

20

40

60

80

56.3%

38.6%

0

20

40

60

More than 50% of the patients receiving Cx601 had all treated fistulas in Combined

Remission1 one year after a single administration of the product

Combined Remission at W52

(mITT3 Population n= 204)

p = 0.010

No Relapse Rate at W52

Cx601 Control Cx601 Control

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Cx601: KOLs Voice Excitement Over AMDIRE-CD ResultsECCO1 (EU), DDW2 (US), The Lancet3

Dr. Panés: Oral presentation, ECCO 2016

Dr. Van Assche: Satellite Symposium, ECCO 2016

1 European Crohn’s and Colitis Organization (ECCO), Amsterdam, March 16 – 19, 2016

2 Digestive Disease Week (DDW) in San Diego, May 24, 2016 and Chicago, May 4, 2017

3 The Lancet [online]. Published online July 28, 2016, available at http://dx.doi.org/10.1016/S0140-6736(16)31203-X

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“Key Opinion Leader” Event, 8th May 2017, ChicagoDoctors express disappointment with current treatments

1 Key Opinion Leader event webcast: http://www.wsw.com/webcast/cc/tig/

“Gastroenterology is one of Takeda’s core therapeutic

areas of focus. When we look at leadership in GI it is all

about delivering very innovative drugs in a population

which has high unmet need. And Cx601 truly

underscores that commitment.” 1

Dr. Uthra Sundaram, VP of the GI Therapeutic Area

and Global Commercial Leader at Takeda

Pharmaceuticals

“…This is a really debilitating, serious medical condition. The more complex fistulas historically have had poor

healing rates, which is one of the unmet need areas that stem cell therapy could potentially help. Fistulizing Crohn's

disease is fairly common and the incidence increases as the disease duration goes up. Gastroenterologists,

surgeons and radiologists collaborate to take care of these patients and what we really need is some new treatment,

which is what brings us here today.“ 1

Dr. William J. Sandborn, MD, Professor of Medicine and Adjunct Professor of Surgery Chief, Division of

Gastroenterology Vice Chair for Clinical Operations, Department of Medicine Director, UCSD IBD Center University

of California San Diego and UC San Diego Health System

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• Team with previous experience in obtaining MA1 of cell therapy product

• Orphan Designation received 2009

• 5 Scientific Advice Meetings held with EMA2 (2 pre-clinical, 2 CMC3, 1 clinical)

• Approved PIP4 with 20 patients to be started not before 2020

• GMP license for commercial manufacturing granted

• Final approval expected in 2H2017

Cx601: EU Approval Decision Expected 2H2017Clear and fast pathway to the market built on a solid regulatory strategy

1 MA: Marketing Authorization2 EMA: European Medicines Agency3 CMC: Chemistry Manufacturing and Controls4 PIP: Pediatric Investigational Plan5 MAA: Marketing Authorization Application

6 AR: Assessment Report7 LoQ: List of Questions8 LoOI: List of Outstanding Issues9 CHMP: Committee of Human Medicinal Products (within

EMA)

AR6 LoQ7 Responses Joint AR LoOI8 Responses

D121 D181

CHMP9

Opinion

D2101st Clock

Stop

2nd Clock

Stop

MAA5

Submitted

1Q2016

EU Decision

D277

D1 D80 D120

Start of the

procedure

D150

D180

Current status

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Ex-US Rights of Cx601 Licensed to TakedaTiGenix keeps significant upside potential

• Takeda acquired the exclusive ex-US development and commercialization

rights to Cx601 for the treatment of complex perianal fistulas in Crohn’s

disease patients

• TiGenix retains the right to develop Cx601 in new indications

• Takeda paid EUR 25M up front and a EUR 10M equity investment

• TiGenix eligible to receive potentially up to EUR 355M in regulatory and

sales milestones, including a EUR 15M EU marketing approval milestone

• Double-digit royalties on net sales, tiered to reimbursement price

• Takeda will assume manufacturing responsibilities for Cx601 after an initial

period of product supply by TiGenix at cost for the EU

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Takeda is the Best Potential Partner for Cx601A global leader with strategic focus in GI

Source: Takeda presentation (JPM event 2017)

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2121

Cx601: A Key Pillar in Takeda’s GI StrategyPerfect fit with existing IBD portfolio

Source: Takeda presentation (JPM event 2017)

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Cx601: European Launch Expected 1H2018Significant progress since signing licensing agreement

1 European Crohn’s and Colitis Organization2 Digestive Disease Week, May 6-9 Chicago, IL, USA

First wave of pre-launch activities completed or ongoing by Takeda

Value proposition, positioning and branding

Large-scale multi-stakeholder market research to understand unmet need, value perception and potential usage

Price and market access research

Preliminary structure of cost-effectiveness model

Centers mapping

Sizing of potential population eligible for Cx601 treatment

Stakeholder facing function training

Oral presentation on Cx601 and symposium at ECCO1 2017

Oral presentation at the 2017 DDW2 session dedicated to Controlled Clinical Trials in Inflammatory Bowel Diseases

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Cx601: Approach to US MarketLeveraging EU data with approved phase III protocol

• Solid regulatory and clinical development strategy

• Type B meeting with FDA1 confirmed:

• Adequacy of existing non-clinical package to support an IND2 filing

• Acceptability of using data from the ADMIRE-CD trial to support BLA

• SPA3 for US Phase III protocol agreed with FDA:

• Primary end-point identical to ADMIRE-CD trial

• p-value < 0.05 (vs. p-value <0.025 in ADMIRE-CD trial)

• Global phase III trial scheduled to start in 1H2017

• Lonza selected as contract manufacturing organization for Cx601 in the US,

technology transfer ongoing

• Exploring different expedited pathways with the FDA

1 FDA: Food and Drug Administration 2 IND: Investigational New Drug 3 SPA: Special Protocol Assessment

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The 21st Century Cures Act and the New RMAT Designation A potential avenue to accelerate Cx601 availability up to 24 months

The 21st Century Cures Act is an extremely significant healthcare legislation that

may have a direct and beneficial impact on TiGenix’s clinical development

programs and progress towards approvals with at least 4 RMAT designationsgranted since program launch in January 2017

Human acellular vessel in Vascular Access for Hemodialysis (Humacyte), 20 March 2017

Allogeneic thymic tissue in complete DiGeorge Syndrome (Enzyvant), 17 April 2017

Human retinal progenitor cells in Retinitis Pigmentosa treatment (jCyte), 2 May 2017

Autologous bone marrow derived multicellular therapy in Advanced Heart Failure Due to Ischemic Dilated Cardiomyopathy (Vericel), 10 May 2017

Expedited approval pathway specific for regenerative advanced therapies

Allows shorter timeline for marketing approval

Creates medical and economic benefits for the healthcare system and reduces costs of

development Facilitates early patient access to therapies with proven efficacy

Definition

Real cases

Benefits

Page 25: Presentación de PowerPoint - Jefferies NV(1).pdf · of the treated perianal fistula confirmed by centrally blinded MRI assessment in patients with clinical remission at any previous

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“Group A”Controlled

luminal disease

Antib. / Immuno.

Not responding

“Group B” Controlled

luminal disease

Infliximab

Not responding

“Group C”Controlled

luminal disease

Second biologic

Not responding

“Group D” Controlled

luminal disease

After failure of

repair surgery

Non-controlled

luminal disease

Infliximab

Not responding

21% 38% 17% 24% ∆ 54% patients upside

50% 45% 85% 90% 60%

11% 17% 14% 21% ∆ 51%patients upside

% of patients in US within each subgroup

25

Patient subgroups within expected label in US

% patients in US within each group treated with Cx601

% patients in US treated with Cx601 (from total within expected label)

Sources: Simon-Kucher & Partners; Expert discussions in June 2016 with 5 medical directors, 2 ex-Medicare carriers, 2 hospital pharmacy directors, 5 KOLs and 16 HVPs (gastroenterologists and colorectal surgeons) in the US

Cx601 Product Profile is Extremely CompetitiveUS doctors may also use Cx601 in other patient groups

Physicians in the US would prescribe Cx601 to around two thirds of all patients included in

the expected label

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46%

59%

90%100% 95%

99%

0%

20%

40%

60%

80%

100%

120%

At increasing prices, physicians would prescribe Cx601 to less patients given likely

restrictions of payers. However, profits would be optimized between $60 and $120k

Sources: Simon-Kucher & Partners; expert discussions June 2016 with 5 medical directors, 2 ex-Medicare carriers, 2

hospital pharmacy directors, 5 KOLs and 16 HVPs. Assumptions on costs provided by TiGenix

Pre

fere

nce

sh

are

fo

r C

x6

01

34%29%

27%

21%

16%14%

0%

10%

20%

30%

40%

50%

WAC price per treatment (thousand $)

Aggregated price-response curve

(physicians’ coverage and payers’ Rx)

Pro

fit

ind

ex (

incl

ud

ing

CO

GS

)

WAC price per treatment (thousand $)

Profit index function for Cx601

(physicians’ coverage and payers’ Rx)

30 40 60 80 100 12030 40 60 80 100 120

Payer adjusted scenario

Profit-optimal price range

Cx601: US Price Range Allows Significant Margin Research indicates profit-optimal prices between $60 and $120k

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Addressable patient population70,000 – 37,000

10% - 30%

USD 40,000 – 50,000

Penetration rate

Price per dose

70,000 – 40,000

15% - 25%

USD 20,000 – 30,000

Peak Year Sales

Cx601 Peak Year Sales Analysts’ ConsensusSeveral variables kept today on the “conservative side” provide future upside

USD 270M – 663M USD 205M – 300M

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Cx601: Pipeline Expansion Under EvaluationPotential for Cx601 growth beyond complex perianal fistulas

• Rectovaginal fistulas in Crohn’s

• Enterocutaneous fistulas in Crohn’s

• Complex anal fistulas in non-Crohn’s

• Intestinal ulcers in Crohn’s disease

• Intestinal ulcers in Ulcerative Colitis

Other gastrointestinal fistulasGastrointestinal indications other than fistulas

Other indications

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Cx601: Significant Potential in Other Gastrointestinal FistulasAddressable population could be four times larger

1 Study Commissioned to Vencore Health Analytics Inc, 2016

Patients under

expected label Cx601

Patients with other

fistulas

* Complex perianal fistulas out of the expected label include those in patients with non-controlled luminal

symptoms, those that are not refractory to currently available therapies, and those affecting children

Source: Truven MarketScan® database1

Estimated Patient Populations in US (2014)

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Pipeline

Cx611 – novel treatment for severe sepsis

AlloCSC-01 – allogeneic cardiac stem cells to treat heart

disease

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1 The Lancet Infectious Diseases; Volume 12; issue 2; page 89; February 20122 Martin GS Expert Rev Anti Infect Ther. 2012 June ; 10(6): 701–706. 3 Adapted from Lagu, T., et al. Critical Care Medicine, 40(3):754-761; 2012 4 Adapted from: Elixhauser et al. Septicemia in U.S. Hospitals 2009, AHRQ, Healthcare Cost Brief No. 122 October 2011

Cx611 – A Novel Treatment Approach to Severe SepsisLeading cause of mortality in the developed world

• Sepsis is a life-threatening complication of infection

leading to systemic inflammation and organ failure

• Between 15M to 19M sepsis cases occur worldwide

each year1. Mortality reaches 50% for severe

sepsis raising to 80% in septic shock2

• Cx611’s novel mechanism of action may offer an

innovative alternative to the treatment of severe

sepsis: efficacy in in vivo models and good Phase I

results

• TiGenix’ Phase II trial (SEPCELL) has received the

support of the Horizon 2020 European Commission

Program and the endorsement of Key Opinion

Leaders

• SEPCELL is a randomised, multicentre, double-

blind, placebo-controlled study. Recruitment

ongoing in patients with severe community-acquired

bacterial pneumonia (sCABP). Primary endpoint

safety; secondary endpoints include improved

survival, and/or clinical cure of the CABP, and other

infection-related endpoints (at 90 days)

600

800

1000

1200

1400

1600

1800

2000 2002 2004 2006 2008 2010

Dis

ch

arg

es th

ou

sa

nd

s

Trend in U.S. hospital stays with septicemia 2000−20094

8% CAGR

750,000

466,000

375,000

84.000

0

200,000

400,000

600,000

800,000

1,000,000

1,200,000

Sepsis Breast, Prostate Cancer &AIDS

Diagnosed cases and mortality of Sepsis vs. Breast Cancer, Prostate Cancer & AIDS3

Diagnosis

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AlloCSC-01: Allogeneic Cardiac Stem CellsTop-line results met all safety objectives. Study revealed valuable insight

“First-in-human” phase I/II trial focused on a safety primary objective and the

evaluation of the feasibility of an intracoronary infusion of 35 million of AlloCSC-01 in

patients following a high-risk AMI

The main findings were:

• Safety primary objective was met: no death or major cardiac adverse events at 30

days. Same results at 6 or 12 months; no immune-related adverse events

throughout the trial

• A larger reduction in infarct size was found in the AlloCSC-01 arm in a pre-

specified subgroup of patients with poor long-term prognosis associated with a

characteristic MRI signature, offering an exciting prospect for further targeted trials in

this population

• Top-line results announced on March 13, 2017. Full results to be presented at

upcoming medical congress

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Outlook

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Strong Financial PositionSignificant fundraising completed in 2016; marquee investors on board

• EUR 23,75 million raised in private placement with specialized investors

• USD 35,65 million raised with NASDAQ IPO. 4 US analysts coverage

• EUR 10 million equity investment from Takeda

• EUR 78 million (18 – 24 months runway) at Dec 31, 2016

• Shareholders over 3%1

• Grifols (16%)

• Cormorant (6%)

• Takeda (5%)

• Hillhouse (4%)

• Source: transparency notification, 13-F Reports, Nasdaq IR insight

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2017

2018

2019

• Cx601 IND and start of recruitment in US centers

• Takeda to launch Cx601 in EU markets

• Start of global phase III for Cx601 BLA at FDA

• Cx601 EU marketing approval decision

• €15M milestone on Cx601 EU MA decision

• Plan on new indications for Cx601

Several Key Future Milestones Short-term catalysts and long-term value-creation opportunities

• End of Cx601 recruitment

• Sepsis phase II data

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