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Corporate PresentationDecember 2015Advanced biopharmaceutical company developing novel
therapies for serious medical conditions
Lead asset on track for EU approval
Partnered with tier-one, big pharma company
Technopolis, Mechelen
31st May 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 CaseLead product near approval in Europe, strong partner, significant upside potential
Strong financial position
Publicly quoted on Nasdaq and Euronext (TIG), supported by
specialized, institutional shareholders
Ex-US rights licensed to Takeda, a global leader with strategic
focus in gastroenterology
Best possible partner for commercial launch
Up to EUR 380M potential milestones plus double-digit royalties
Lead product in an indication of high unmet medical need
EU approval decision expected in 2H2017
Clear development plan for Global Pivotal phase III trial to start in1H2017
Pipeline with clinical-stage assets which provides further upside
4
PipelineClear commercial potential with future growth opportunities
5
Cx601
Novel, locally administered treatment for a severe
complication of Crohn’s disease
EU approval decision expected 2H2017
6
GMP Facility Approved for Commercial ManufacturingConsistent and robust process
1 liposuction from healthy donor
Allogeneic model translates into production scalability
2,400 finished products (Cx601)
7
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;
8
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
9
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
10
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
11
Cx601: A Real Improvement Versus Best Possible CareStatistically significant and clinically meaningful results
● Significantly quicker healing: median of 7 weeks vs. 14 weeks
● Significantly higher remission rate at 6 months, sustained at one year
● > 50% patients in clinical and radiological remission after one year
● Lower relapse rate from 6 months to one year: 25% vs. 45%
● No added adverse reaction burden
● Highly stringent endpoint, never used before (clinical + radiological -MRI-)
● 100% patients had complex fistulas
● 100% patients were treatment-refractory (> 60% to anti-TNFs)
● More severe patients in Cx601 arm (more multi-branch fistulas)
Despite…
Significant results with a single local administration
12
Cx601: Strong Recognition of ADMIRE-CD Trial ResultsECCO1 (EU), DDW2 (US), The Lancet3
Dr. Panés: Oral presentation, ECCO
Dr. Van Assche: Satellite Symposium, ECCO
1 European Crohn’s and Colitis Organization (ECCO), Amsterdam, March 16 – 19, 2016
2 Digestive Disease Week (DDW) in San Diego, California (USA), May 24, 2016
3 The Lancet [online]. Published online July 28, 2016, available at http://dx.doi.org/10.1016/S0140-6736(16)31203-X
13
“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
14
• Team with previous experience in obtaining MA1 of cell therapy product
• 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
15
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
16
Takeda is the Best Potential Partner for Cx601A global leader with strategic focus in GI
Source: Takeda presentation (JPM event 2017)
1717
Cx601: A Key Pillar in Takeda’s GI StrategyPerfect fit with existing IBD portfolio
Source: Takeda presentation (JPM event 2017)
18
Cx601: Takeda’s CEO CommentaryStrong synergy, preparing for launch
“… we are really working on the launch of the product [Cx601] in Europe... the synergy is very
strong because it's an IBD product, so we are present in this field, so we'll be able to leverage our
presence.
…it's a very innovative way of intervening... the medical need is very, very significant because
there is very little alternative except surgery. So I think for the moment, we are very much working
on preparing the launch, working on the supply chain because it's a new field with stem cell therapy.
… also what is very attractive in our mind is that this is potentially helping us to learn how to manage
this type of product which will -- there will be more and more of this product in the future. So I think
that's very exciting. And we'll continue to see whether we want to expand the partnership ...”
Christophe Weber, President and Chief Executive Officer of Takeda Pharmaceutical Company Ltd
Full Year 2016, Annual Results, May 10 2017
http://www.net-ir.ne.jp/presen/45021705e/pc/index.html
19
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
20
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
21
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
22
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
23
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)
24
Pipeline
Cx611 – novel treatment for severe sepsis
AlloCSC-01 – allogeneic cardiac stem cells to treat heart
disease
25
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 has received the support
of the Horizon 2020 European Commission
Program and the endorsement of Key Opinion
Leaders in Europe
• Recruitment in Phase SEPCELL ongoing
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
26
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
27
Outlook
28
Strong Financial PositionSignificant fundraising completed in 2016; marquee investors on board
EUR 78 million at Dec 31, 2016
18 – 24 months runway
EUR Million, except for share data (EUR) 31 Dec 2016 31 Dec 2015
Revenues 26.8 2.2
Royalties 0.4 0.5
License revenues 25.0 -
Grants and other operating income 1.4 1.7
Operating charges (29.8) (26.3)
Research and development expenses (21.4) (19.6)
General and administrative expenses (8.4) (6.7)
Operating Loss (3.0) (24.1)
Financial income 0.2 0.2
Interest on borrowing and other finance costs (7.3) (6.6)
Fair value gains/(losses) 11.6 (6.7)
Impairment and losses on disposal of financial instruments - (0.2)
Foreign exchange differences,net 0.2 1.0
Income tax benefits 2.1 1.3
Profit (Loss) for the year 3.8 (35.1)
Basic income (loss) per share (EUR) 0.02 (0.21)
Cash and cash equivalents at the end of the year 78.0 18.0
Net cash (used in)/provided by operating activities 3.5 (19.6)
29
Delivering on Transformation of TiGenixTransformation started in 2014; exceptional progress in 2016; momentum continues
Product 2014 2015 2016 2017
Cx601
Europe
US
AlloCSC-01
acute
myocardial
infarction
Cx611 severe
sepsis
ChondroCelect
3Q15 Phase 3 primary endpoint met (24 weeks)
1Q16 study results(1 year follow-up)
1Q16 EMA filing
3Q14 CMO selection
4Q14 SPA submission
4Q14 Phase 3 enrollment completed
Takeda Deal
3Q15 positive SPA
1Q14 manufacturing facility sold
2Q14 licensed to SOBI
2Q16 Phase 2 interim analysis 1H17 Phase 2
study results
1Q15 Phase 2 enrollment initiated
4Q15 Phase 2 enrollment completed
4Q14 Phase 1initiated
2Q15 Phase 1 study results
4Q16 Phase 2 Approved for
Enrolment
Termination of agreement with SOBI
Withdrawal of MAA requested
First Patient-in /
Dosed 1Q17
1Q17 study results(2 year follow-up)
D180 LoOI received
FDA’s agreementon SPA for global study
30
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
31
Thank you!
Any questions?
32
Cx611
A novel treatment approach to severe sepsis
Recruitment ongoing. Data expected 2019
33
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
Severe Sepsis Leading 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
• TiGenix’ Phase II trial has received the support
of the Horizon 2020 European Commission
Program and the endorsement of Key Opinion
Leaders in Europe
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
34
Cx611: Strong Efficacy and Favorable Safety“In Vivo” models show therapeutic effect and phase I study confirms safety
• CELLULA phase I trial results:
favorable safety and tolerability
profile of Cx611, consistent with
phase I/IIa in refractory RA patients
1 LPS: lipopolysaccharide. LPS model is based on endotoxemia induced by high-dose of endotoxin 2 CLP: cecal ligation and puncture. This model mimics the clinical situation of patients with colonic leakage
following surgical procedures or diffused peritonitis
• Demonstrated increased survival
in both LPS (dose dependent)
and CLP in vivo models
LPS1 Model CLP2 Model
* p < 0.001
* p<0.001
• This effect is due to a combination
of reducing pro-inflammatory and
increasing anti-inflammatory
mediators, production of anti-
microbial effectors
Source: Gonzalez-Rey et al. Gut. 2009 Jul;58(7):929-39
3535
Cx611: Phase I/II SEPCELL TrialRecruitment ongoing: data available in 2019
ICU patients with sCABP1 requiring mechanical
ventilation and/or vasopressors
Cx601 N=90
(160 Million)
Placebo N=90
1:1
RA
ND
OM
IZA
TIO
N
Primary safety endpoints: Safety measured throughout 90 days
by the incidence of treatment emergent adverse events (TEAEs)
judged related or not to study treatment
14 days90 days
365 days
Cx611 or placebo
(day 1 and day 3)
Secondary safety endpoints: potential host immune
response to administered cells. Side effects: frequency,
duration, severity, relatedness to study treatment
Exploratory Safety Endpoint:
Long-term safety follow-up
(through phone calls)
720 days
29 days
Secondary efficacy clinical endpoints: Survival; mechanical
ventilator and/or vasopressor treatment/free days; sCABP clinical
response; other efficacy endpoints, such as time to discharge
from ICU
1 Severe Community-Acquired Bacterial Pneumonia
36
AlloCSC-01
Intracoronary administration of allogeneic cardiac stem
cells for the treatment of acute ischaemic heart disease
Top-line results announced on March 13, 2017
37
AlloCSC-01: Preventing Chronic Heart FailureMyocardial repair may be the only feasible alternative
• 1.9M Acute Myocardial Infarctions (US+EU and Japan)1 occur annually, mostly treated
by PCI2 and stent implantation
• Successful treatment of Acute Myocardial Infarctions (“AMI”) has increased short term
survival but contributed to a Chronic Heart Failure (CHF) epidemic (26M patients
worldwide3)
• CHF post-AMI is a terminal disease with an annual mortality rate of ~5% after the
first episode, for which no curative treatment exists with the exception of heart
transplantation
1 Datamonitor: Stakeholder Insight: Acute coronary syndromes, DMHC2347, 2007 2 PCI: Percutaneous Coronary Intervention3 Ambrosy PA et al., J Am Coll Cardiol. 2014;63:1123-1133.
Myocardial repair is the only
feasible treatment to address the
post-acute phase of the disease
and prevent the onset of CHF
38
CAREMI Trial – Phase I/II in Acute Myocardial InfarctionTop-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 study included 49 patients in its randomized phase with a 12-month follow-up.
AlloCSC-01 were infused in the affected coronary between the 5th and 7th day post-
infarction. 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
39
Strong Financial
Position
40
Strong Financial PositionSignificant fundraising completed in 2016; marquee investors on board
EUR 78 million at Dec 31, 2016
18 – 24 months runway
EUR Million, except for share data (EUR) 31 Dec 2016 31 Dec 2015
Revenues 26.8 2.2
Royalties 0.4 0.5
License revenues 25.0 -
Grants and other operating income 1.4 1.7
Operating charges (29.8) (26.3)
Research and development expenses (21.4) (19.6)
General and administrative expenses (8.4) (6.7)
Operating Loss (3.0) (24.1)
Financial income 0.2 0.2
Interest on borrowing and other finance costs (7.3) (6.6)
Fair value gains/(losses) 11.6 (6.7)
Impairment and losses on disposal of financial instruments - (0.2)
Foreign exchange differences,net 0.2 1.0
Income tax benefits 2.1 1.3
Profit (Loss) for the year 3.8 (35.1)
Basic income (loss) per share (EUR) 0.02 (0.21)
Cash and cash equivalents at the end of the year 78.0 18.0
Net cash (used in)/provided by operating activities 3.5 (19.6)
41
Significant US Banking CoverageConsensus on rating TiGenix as “Buy”, “Outperform”