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Page 1: Annual Results 2017 & Business Update 13 April 2018 · This presentation is not intended to be relied upon as advice to investors or potential investors and does not take into account

1

Annual Results 2017 & Business Update

13 April 2018

Page 2: Annual Results 2017 & Business Update 13 April 2018 · This presentation is not intended to be relied upon as advice to investors or potential investors and does not take into account

Disclaimer

These slides and the accompanying oral presentation contain forward-looking statements and information. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels of activity, performance or achievements to be materially different from those anticipated by such statements. The use of words such as “may”, “might”, “will”, “should”, “could”, “expect”, “plan”, “anticipate”, “believe”, “estimate”, “project”, “intend”, “future”, “potential” or “continue”, and other similar expressions are intended to identify forward looking statements. For example, all statements we make regarding (i) the initiation, timing, cost, progress and results of our preclinical and clinical studies and our research and developmentprograms, (ii) our ability to advance product candidates into, and successfully complete, clinical studies, (iii) the timing or likelihood of regulatory filings and approvals, (iv) our ability to develop, manufacture and commercialize our product candidates and to improve the manufacturing process, (v) the rate and degree of market acceptance of our product candidates, (vi) the size and growth potential of the markets for our product candidates and our ability to serve those markets, and (vii) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates, are forward looking. All forward-looking statements are based on current estimates, assumptions and expectations by our management that, although we believe to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that we expected. Any forward-looking statement speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. This presentation is not, and nothing in it should be construed as, an offer, invitation or recommendation in respect of our securities, or an offer, invitation or recommendation to sell, or a solicitation of an offer to buy, any of our securities in any jurisdiction. Neither this presentation nor anything in it shall form the basis of any contract or commitment. This presentation is not intended to be relied upon as advice to investors or potential investors and does not take into account the investment objectives, financial situation or needs of any investor.

2

Page 3: Annual Results 2017 & Business Update 13 April 2018 · This presentation is not intended to be relied upon as advice to investors or potential investors and does not take into account

HIGHLIGHTS - Operational

• Filed Marketing Authorization Application with the European Medicines Agency for ATIR101 in blood cancers

• Submitted responses to the EMA to enable a conditional marketing approval from the European Commission - potentially allowing an opinion from the EMA in Q4 2018

• Received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA for ATIR101

• First patient enrolled in Phase 3 trial for ATIR101 in adult patients with blood cancer

• Leased existing commercial manufacturing facility in The Netherlands

• Strengthened Organization and Supervisory Board

3

Page 4: Annual Results 2017 & Business Update 13 April 2018 · This presentation is not intended to be relied upon as advice to investors or potential investors and does not take into account

HIGHLIGHTS – Financial

• Raised more than EUR 60 million in equity and debt since June 2017

• End of March 2018: EUR 47.7 million cash

4

2017 2016 Change

Total revenue and other income - - -

Total operating expenses (16.1) (11.4) (4.7)

Research and development (11.2) (8.2) (3.0)

General and administrative (4.9) (3.2) (1.7)

Operating result (16.1) (11.4) (4.7)

Net financial result (0.9) (3.4) 2.5

Net result (17.0) (14.8) (2.2)

Net operating cash flow (15.9) (14.3) (1.6)

Cash position at end of year 29.9 14.6 15.3

Equity 15.9 9.4 6.5

Earnings per share before dilution (EUR) (1.14) (1.08) (0.06)

(Amounts in EUR million, except per share data)

Page 5: Annual Results 2017 & Business Update 13 April 2018 · This presentation is not intended to be relied upon as advice to investors or potential investors and does not take into account

ATIR Regulatory Status

5

Product Pre-ClinicalPhase

IPhase

IIPhase

III Filing CatalystsCommercial

Rights

ATIR101(Europe)

- CHMP Opinion 4Q18

- EU Launch 2H19

ATIR101(USA)

- Phase III (interim) read outOrphan Drug & RMAT Designations

Orphan Drug Designation

Page 6: Annual Results 2017 & Business Update 13 April 2018 · This presentation is not intended to be relied upon as advice to investors or potential investors and does not take into account

The very first cell transplant method: allogeneic HSCT

Allogeneic Hematopoietic Stem Cell Transplantation (HSCT):

• Curative intent: replace disease blood/immune system with healthy one from donor

• Risk of Graft versus Host disease (GVHD): Donor immune system attacks the patient

• Mostly blood cancers (85%) and adults (82%)

6

Adoption of HSCT limited by high risk

Blood cancers: Only 20-30% long term GVHD-Free and Relapse-Free Survival (GRFS)

Inherited blood disorders or autoimmune disease: Risk of replacing chronic disease with (chronic) GVHD

Page 7: Annual Results 2017 & Business Update 13 April 2018 · This presentation is not intended to be relied upon as advice to investors or potential investors and does not take into account

HSCT: Strong growth, still large unmet need (U.S.)

7Source: CIBMTR 2017 Summary slides; Fuchs 2017; Gragert 2014; Besse 2015

Historical: Matched Related or Unrelated Donors• Donor availability 20-80% (due to

family size & genetic diversity)• Declining, despite unmet need

Emerging: Haploidentical or half matched donors• Donor availability >95%

(parents/children)• 32% compound annual growth• Made possible due to Post

Transplant Cyclophosphamide (PTCy) or ‘Baltimore’ protocol*

Unmet need: 13,000 per year (lack of matched donors)

* Cyclophosphamide (chemotherapy, days 3 and 5) & immunosuppressants to treat immediate attack from alloreactive haploidentical donor T-cells

Haploidentical donors

Matched Unrelated Donor (MUD; registries)

Matched Related Donors (MRD)

Page 8: Annual Results 2017 & Business Update 13 April 2018 · This presentation is not intended to be relied upon as advice to investors or potential investors and does not take into account

Kiadis: potential improvement vs. PTCy/Baltimore

8

Kiadis’ ATIR (add on to HSCT)

PTCy

Apheresis of patient and

donor

Central ATIR production,

5 day processConditioning of

patient

Apheresis of donor,

graft infusion

Engraftment of donor

stem cellsInfusion of

ATIR

14 days before HSCT ~30 days after HSCT Aims to prevent GVHD

& relapse

stem cells only

Conditioning of patient

Apheresis of donor,

graft infusion

Engraftment of donor

stem cells

stem cells + all T-cells

Chemo & immuno-

suppressants

Day 2 & 5 after HSCT Treats GVHD

HAPLOIDENTICAL HSCT

Page 9: Annual Results 2017 & Business Update 13 April 2018 · This presentation is not intended to be relied upon as advice to investors or potential investors and does not take into account

ATIR production: subset of T-cells that protect, but not attack

ATIRTM MANUFACTURING PROCESS

MANUFACTURING

Kiadis Pharma has established a GMP-compliant, manufacturing process that has been successfully transferred to three GMP-

manufacturing sites in North America and Europe. The Company is one of seven companies to have ever been issued an Advanced

Therapy Medicinal Product (ATMP) certif cate for manufacturing quality and non-clinical data by the European Medicines Agency

(EMA).

MARKET POTENTIAL

ATIR101 has the potential of providing life-saving products to ~19,000 blood cancer patients a year, with a substantial expanding

primary market and complementary product trends. The Company estimates that approximately 35% of patients who are eligible

for, and who are in urgent need of, HSCT will not f nd a matching donor in time. A partially matched (haploidentical) family donor,

however, will be available to over 95%. Recent developments in cell-based immunotherapy have resulted in approached which

may allow more patients with blood cancers to go into remission, including various CAR-T approaches. These patients will then

be eligible for curative HSCT. The product may benef t from market exclusivity up to 10 years post launch in the EU and 7 years in

the US due to its orphan drug status.

ATIR101 AIMS TO ADDRESS THE RISKS ANS LIMITATIONS OF HSCT

Current sequence of options physicians will consider

Haploidentical donor + ATIR101

IdenticalSibling(SIB)

MatchedUnrelated

(MUD)

Haploidenticaldonor +

Cyclophosphamide*

Umbilical Cord Blood

(UCB)

Time to collect stem cells < 1 month < 1-2 months 3-6 months < 1 month 2-3 months

Donor availability >95% ≤ 25% 45-55% >95% Limited

Risk of life-threatening GVHD Low Low Moderate - High Moderate - High Moderate

Need for immune suppressants No Yes Yes Yes Yes

Risk of opportunistic infections Low Moderate Moderate Moderate - High High

Risk of relapse in leukemia Low High Moderate - High High High

* Often referred to as the Baltimore protocol. Treatment method was established at the John Hopkins Hospital, Baltimore, USA. (Luznik et al., Biol Blood Marrow

Transplant. 2008)

Source table: Company information, CIBMTR, 2014 and Defin

e

d He al th, 2013 commi ssi oned by the Co mp any .

Survey included 50 US and 50 EU physicians.

Step 1 (Day 1–4)

Patient cells

inactivated

by radiation

Healthy

donor

Patient

Immune cells are collected and mixed

Certain T-cells from the donor are activated by the

presence of the ‘foreign’ patient cells. If not eliminated,

these cells would cause GvHD in the patient

Step 3 (Day 5)

Mixture is exposed to light.

TH9402 is activated by light, causing the

‘stained’ GvHD-causing T-cells to self-destruct

The remaining product is infused back

and helps rebuild their immune system

to f ght infections and eliminate

remaining tumor cells

Final Step - Infusion of ATIR101Step 2 (Day 5)

TH9402 is introduced.

TH9402 is retained ONLY in the GvHD-causing T-cells

FEBRUARY 2017

donor

patient

ATIRTM MANUFACTURING PROCESS

MANUFACTURING

Kiadis Pharma has established a GMP-compliant, manufacturing process that has been successfully transferred to three GMP-

manufacturing sites in North America and Europe. The Company is one of seven companies to have ever been issued an Advanced

Therapy Medicinal Product (ATMP) certif cate for manufacturing quality and non-clinical data by the European Medicines Agency

(EMA).

MARKET POTENTIAL

ATIR101 has the potential of providing life-saving products to ~19,000 blood cancer patients a year, with a substantial expanding

primary market and complementary product trends. The Company estimates that approximately 35% of patients who are eligible

for, and who are in urgent need of, HSCT will not f nd a matching donor in time. A partially matched (haploidentical) family donor,

however, will be available to over 95%. Recent developments in cell-based immunotherapy have resulted in approached which

may allow more patients with blood cancers to go into remission, including various CAR-T approaches. These patients will then

be eligible for curative HSCT. The product may benef t from market exclusivity up to 10 years post launch in the EU and 7 years in

the US due to its orphan drug status.

ATIR101 AIMS TO ADDRESS THE RISKS ANS LIMITATIONS OF HSCT

Current sequence of options physicians will consider

Haploidentical donor + ATIR101

IdenticalSibling(SIB)

MatchedUnrelated

(MUD)

Haploidenticaldonor +

Cyclophosphamide*

Umbilical Cord Blood

(UCB)

Time to collect stem cells < 1 month < 1-2 months 3-6 months < 1 month 2-3 months

Donor availability >95% ≤ 25% 45-55% >95% Limited

Risk of life-threatening GVHD Low Low Moderate - High Moderate - High Moderate

Need for immune suppressants No Yes Yes Yes Yes

Risk of opportunistic infections Low Moderate Moderate Moderate - High High

Risk of relapse in leukemia Low High Moderate - High High High

* Often referred to as the Baltimore protocol. Treatment method was established at the John Hopkins Hospital, Baltimore, USA. (Luznik et al., Biol Blood Marrow

Transplant. 2008)

Source table: Company information, CIBMTR, 2014 and Defin

e

d He al th, 2013 commi ssi oned by the Co mp any .

Survey included 50 US and 50 EU physicians.

Step 1 (Day 1–4)

Patient cells

inactivated

by radiation

Healthy

donor

Patient

Immune cells are collected and mixed

Certain T-cells from the donor are activated by the

presence of the ‘foreign’ patient cells. If not eliminated,

these cells would cause GvHD in the patient

Step 3 (Day 5)

Mixture is exposed to light.

TH9402 is activated by light, causing the

‘stained’ GvHD-causing T-cells to self-destruct

The remaining product is infused back

and helps rebuild their immune system

to f ght infections and eliminate

remaining tumor cells

Final Step - Infusion of ATIR101Step 2 (Day 5)

TH9402 is introduced.

TH9402 is retained ONLY in the GvHD-causing T-cells

FEBRUARY 2017

ATIRTM MANUFACTURING PROCESS

MANUFACTURING

Kiadis Pharma has established a GMP-compliant, manufacturing process that has been successfully transferred to three GMP-

manufacturing sites in North America and Europe. The Company is one of seven companies to have ever been issued an Advanced

Therapy Medicinal Product (ATMP) certif cate for manufacturing quality and non-clinical data by the European Medicines Agency

(EMA).

MARKET POTENTIAL

ATIR101 has the potential of providing life-saving products to ~19,000 blood cancer patients a year, with a substantial expanding

primary market and complementary product trends. The Company estimates that approximately 35% of patients who are eligible

for, and who are in urgent need of, HSCT will not f nd a matching donor in time. A partially matched (haploidentical) family donor,

however, will be available to over 95%. Recent developments in cell-based immunotherapy have resulted in approached which

may allow more patients with blood cancers to go into remission, including various CAR-T approaches. These patients will then

be eligible for curative HSCT. The product may benef t from market exclusivity up to 10 years post launch in the EU and 7 years in

the US due to its orphan drug status.

ATIR101 AIMS TO ADDRESS THE RISKS ANS LIMITATIONS OF HSCT

Current sequence of options physicians will consider

Haploidentical donor + ATIR101

IdenticalSibling(SIB)

MatchedUnrelated

(MUD)

Haploidenticaldonor +

Cyclophosphamide*

Umbilical Cord Blood

(UCB)

Time to collect stem cells < 1 month < 1-2 months 3-6 months < 1 month 2-3 months

Donor availability >95% ≤ 25% 45-55% >95% Limited

Risk of life-threatening GVHD Low Low Moderate - High Moderate - High Moderate

Need for immune suppressants No Yes Yes Yes Yes

Risk of opportunistic infections Low Moderate Moderate Moderate - High High

Risk of relapse in leukemia Low High Moderate - High High High

* Often referred to as the Baltimore protocol. Treatment method was established at the John Hopkins Hospital, Baltimore, USA. (Luznik et al., Biol Blood Marrow

Transplant. 2008)

Source table: Company information, CIBMTR, 2014 and Defin

e

d He al th, 2013 commi ssi oned by the Co mp any .

Survey included 50 US and 50 EU physicians.

Step 1 (Day 1–4)

Patient cells

inactivated

by radiation

Healthy

donor

Patient

Immune cells are collected and mixed

Certain T-cells from the donor are activated by the

presence of the ‘foreign’ patient cells. If not eliminated,

these cells would cause GvHD in the patient

Step 3 (Day 5)

Mixture is exposed to light.

TH9402 is activated by light, causing the

‘stained’ GvHD-causing T-cells to self-destruct

The remaining product is infused back

and helps rebuild their immune system

to f ght infections and eliminate

remaining tumor cells

Final Step - Infusion of ATIR101Step 2 (Day 5)

TH9402 is introduced.

TH9402 is retained ONLY in the GvHD-causing T-cells

FEBRUARY 2017ATIRTM MANUFACTURING PROCESS

MANUFACTURING

Kiadis Pharma has established a GMP-compliant, manufacturing process that has been successfully transferred to three GMP-

manufacturing sites in North America and Europe. The Company is one of seven companies to have ever been issued an Advanced

Therapy Medicinal Product (ATMP) certif cate for manufacturing quality and non-clinical data by the European Medicines Agency

(EMA).

MARKET POTENTIAL

ATIR101 has the potential of providing life-saving products to ~19,000 blood cancer patients a year, with a substantial expanding

primary market and complementary product trends. The Company estimates that approximately 35% of patients who are eligible

for, and who are in urgent need of, HSCT will not f nd a matching donor in time. A partially matched (haploidentical) family donor,

however, will be available to over 95%. Recent developments in cell-based immunotherapy have resulted in approached which

may allow more patients with blood cancers to go into remission, including various CAR-T approaches. These patients will then

be eligible for curative HSCT. The product may benef t from market exclusivity up to 10 years post launch in the EU and 7 years in

the US due to its orphan drug status.

ATIR101 AIMS TO ADDRESS THE RISKS ANS LIMITATIONS OF HSCT

Current sequence of options physicians will consider

Haploidentical donor + ATIR101

IdenticalSibling(SIB)

MatchedUnrelated

(MUD)

Haploidenticaldonor +

Cyclophosphamide*

Umbilical Cord Blood

(UCB)

Time to collect stem cells < 1 month < 1-2 months 3-6 months < 1 month 2-3 months

Donor availability >95% ≤ 25% 45-55% >95% Limited

Risk of life-threatening GVHD Low Low Moderate - High Moderate - High Moderate

Need for immune suppressants No Yes Yes Yes Yes

Risk of opportunistic infections Low Moderate Moderate Moderate - High High

Risk of relapse in leukemia Low High Moderate - High High High

* Often referred to as the Baltimore protocol. Treatment method was established at the John Hopkins Hospital, Baltimore, USA. (Luznik et al., Biol Blood Marrow

Transplant. 2008)

Source table: Company information, CIBMTR, 2014 and Defin

e

d He al th, 2013 commi ssi oned by the Co mp any .

Survey included 50 US and 50 EU physicians.

Step 1 (Day 1–4)

Patient cells

inactivated

by radiation

Healthy

donor

Patient

Immune cells are collected and mixed

Certain T-cells from the donor are activated by the

presence of the ‘foreign’ patient cells. If not eliminated,

these cells would cause GvHD in the patient

Step 3 (Day 5)

Mixture is exposed to light.

TH9402 is activated by light, causing the

‘stained’ GvHD-causing T-cells to self-destruct

The remaining product is infused back

and helps rebuild their immune system

to f ght infections and eliminate

remaining tumor cells

Final Step - Infusion of ATIR101Step 2 (Day 5)

TH9402 is introduced.

TH9402 is retained ONLY in the GvHD-causing T-cells

FEBRUARY 2017

`ATIRTM MANUFACTURING PROCESS

MANUFACTURING

Kiadis Pharma has established a GMP-compliant, manufacturing process that has been successfully transferred to three GMP-

manufacturing sites in North America and Europe. The Company is one of seven companies to have ever been issued an Advanced

Therapy Medicinal Product (ATMP) certif cate for manufacturing quality and non-clinical data by the European Medicines Agency

(EMA).

MARKET POTENTIAL

ATIR101 has the potential of providing life-saving products to ~19,000 blood cancer patients a year, with a substantial expanding

primary market and complementary product trends. The Company estimates that approximately 35% of patients who are eligible

for, and who are in urgent need of, HSCT will not f nd a matching donor in time. A partially matched (haploidentical) family donor,

however, will be available to over 95%. Recent developments in cell-based immunotherapy have resulted in approached which

may allow more patients with blood cancers to go into remission, including various CAR-T approaches. These patients will then

be eligible for curative HSCT. The product may benef t from market exclusivity up to 10 years post launch in the EU and 7 years in

the US due to its orphan drug status.

ATIR101 AIMS TO ADDRESS THE RISKS ANS LIMITATIONS OF HSCT

Current sequence of options physicians will consider

Haploidentical donor + ATIR101

IdenticalSibling(SIB)

MatchedUnrelated

(MUD)

Haploidenticaldonor +

Cyclophosphamide*

Umbilical Cord Blood

(UCB)

Time to collect stem cells < 1 month < 1-2 months 3-6 months < 1 month 2-3 months

Donor availability >95% ≤ 25% 45-55% >95% Limited

Risk of life-threatening GVHD Low Low Moderate - High Moderate - High Moderate

Need for immune suppressants No Yes Yes Yes Yes

Risk of opportunistic infections Low Moderate Moderate Moderate - High High

Risk of relapse in leukemia Low High Moderate - High High High

* Often referred to as the Baltimore protocol. Treatment method was established at the John Hopkins Hospital, Baltimore, USA. (Luznik et al., Biol Blood Marrow

Transplant. 2008)

Source table: Company information, CIBMTR, 2014 and Defin

e

d He al th, 2013 commi ssi oned by the Co mp any .

Survey included 50 US and 50 EU physicians.

Step 1 (Day 1–4)

Patient cells

inactivated

by radiation

Healthy

donor

Patient

Immune cells are collected and mixed

Certain T-cells from the donor are activated by the

presence of the ‘foreign’ patient cells. If not eliminated,

these cells would cause GvHD in the patient

Step 3 (Day 5)

Mixture is exposed to light.

TH9402 is activated by light, causing the

‘stained’ GvHD-causing T-cells to self-destruct

The remaining product is infused back

and helps rebuild their immune system

to f ght infections and eliminate

remaining tumor cells

Final Step - Infusion of ATIR101Step 2 (Day 5)

TH9402 is introduced.

TH9402 is retained ONLY in the GvHD-causing T-cells

FEBRUARY 2017

``

ATIR: remaining potent non-

alloreactive donor T-cells, infused ~30 days after

HSCT

Add TH9402*, which accumulates only in

activated T-cells (MDR pump is switched off in

activated T-cells)

*TH9402 – proprietary selective rhodamine derivative, modified to become cytotoxic under green light

Mix patient cells & haplo donor T-cells: alloreactive donor

T-cells become activated (Mixed

Lymphocyte Reaction)

Expose to green light: TH9402*, which

induces apoptosis, is activated & thus

alloreactive T-cells are killed

Protect: Retain protective T-cells to fight relapse and infections& Not attack: Reduce risk of GVHD by depleting alloreactive T-cells ex vivo

9

Page 10: Annual Results 2017 & Business Update 13 April 2018 · This presentation is not intended to be relied upon as advice to investors or potential investors and does not take into account

Phase 2 (007): potent T-cell product, yet low GVHD (1 yr)

• no acute grade III/IV

• 3 acute grade II (13%)

• 1 chronic (4%)

007: Haplo CD34+ plus single dose ATIR

• Open label single arm 2013-18• 23 AML/ALL patients receiving ATIR (MITT)• 4 sites Canada/EU • Dose 2 million cells/kg*

006: Haplo CD34+

• Historical observational cohort 2006-13• 35 patients, similar indications/sites• Protocol based on EMA scientific advice

Low GVHD due to ATIR

2 million cells/kg of potent T-cells:

increasing survival 3x, yet low GVHD**

No need for prophylactic immunosuppression

10* Non allodepleted donor lymphocyte infusion can cause severe GVHD at 10,000 cells/kg

Improved Overall Survival due to ATIR

CD34+ stem cells with ATIR

CD34+ stem cells without ATIR

3x

61%

20%

Page 11: Annual Results 2017 & Business Update 13 April 2018 · This presentation is not intended to be relied upon as advice to investors or potential investors and does not take into account

11

Phase 2 (007): relapse, GVHD & GRFS* vs. literature for PTCy

Comparison provided for illustrative purposes, based on literature comparison, NOT based on randomized controlled trials

* Defined as survival without chronic GVHD requiring immunosuppression, acute grade III/IV GVHD or relapse ** Ciurea 2015; Piemontese 2017, Solomon 2012, Ciurea 2012; Devillier 2016; Di Stasi 2014; Esquirol 2016; Sugita 2015*** Solh 2016 (Atlanta; DRI normalized GRFS 30%; n=128); McCurdy 2017 (Johns Hopkins; DRI normalized GRFS 38%; n=372)

**

***

Page 12: Annual Results 2017 & Business Update 13 April 2018 · This presentation is not intended to be relied upon as advice to investors or potential investors and does not take into account

Filed in EU & received ‘breakthrough’ in US, based on Phase 2

* Various hemato-oncology products (conditionally) approved by EMA based on Phase 2, e.g., Zalmoxis (MolMed, 36 patients, versus matched historical control), Blincyto, Venclexta, Bosulif

EMA (EU)

Marketing Authorization Application filed, potential (conditional) opinion Q4 2018

• ATMP certificate for quality and non-clinical data in 2015

• Pediatric Investigation Plan agreed

• Phase 2 and historical control accepted for filing and review*

• Day 120 questions submitted end Q1 2018

FDA (U.S.)

Regenerative Medicine Advanced Therapy designation received (same benefits as Breakthrough)

• Increased access to FDA (not limited to customary timepoints)

• Possibility for priority/rolling review of BLA

• Development support (program/endpoints)

12

Page 13: Annual Results 2017 & Business Update 13 April 2018 · This presentation is not intended to be relied upon as advice to investors or potential investors and does not take into account

Objectives: demonstrate superior clinical benefit and collect pharmacoeconomical data (cost, days in hospital, incidence of severe infections and quality of life)

Commenced Phase 3 Clinical Study

R

RandomizedControlled(1:1)

HSCT plus ATIR: CD34+ HSCT + single dose ATIR101

PTCy/Baltimore protocol: post-HSCT cyclophosphamide & immunosuppressant

Primary endpoint: GVHD-Free and Relapse-Free Survival (GRFS**)

195 patients* in U.S., Canada and EU

Aligned with FDA and regulators in EU; Enrolling patients

* 80% powered to detect 20% GRFS difference; 15% difference will be statistically significant; allowed under protocol to increase sample size; 245 pts would give 80% power to detect 18% difference** Survival without chronic GVHD requiring immunosuppression, acute grade III/IV GVHD or relapse

13

Page 14: Annual Results 2017 & Business Update 13 April 2018 · This presentation is not intended to be relied upon as advice to investors or potential investors and does not take into account

Kiadis key milestones and upcoming catalysts

14

2017

EMA submission of ATIR for marketing authorization approval ✔

First patient enrolled for ATIR Phase 3 ✔

Updates enrollment, regulatory, new clinical sites ✔

FDA Regenerative Medicine Advanced Therapy designation ✔

Secured own commercial manufacturing facility (lease) ✔

New management and supervisory board members ✔

2018

Completion of enrollment of second Phase 2 trial (CR-AIR-008) ✔

Submission of answers to EMA Day 120 questions (End Q1) ✔

Potential EU CHMP opinion, Q4

Updates Phase 2 data and Phase 3 enrollment

2019

Potential initial commercial launch ATIR in first of EU5 countries (H2)

Initiate trial with ATIR as adjunctive to PTCy

Potential interim read out Phase 3