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Help patients with immunotherapy designed to reduce risk of GVHD in stem cell transplantations

Unlock full potential of haploidentical hematopoietic stem cell transplantations (HSCT), with allo-depleted T-cell product ATIR

Company presentation, February 25, 2018

Amsterdam, The NetherlandsEuronext (KDS)

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 or 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.

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Novel cell therapies establishing themselves

3

Selected companies Product Status Company valuation

Kymriah(list price $475,000)

FDA approved

Yescarta(list price $373,000)

FDA approved $11.9bn 8/2017 acquisition by Gilead

liso-celPotential FDA filing 2H 2018

$9.0bn 1/2018 offer by Celgene

darvadstrocelEMA positive CHMP opinion

$596M12/2017 acquisition by Takeda

tabelecleucel Phase 3 $1.7bn* market capitalization 1/2018 $128m capital raise

* As of market close February 13, 2018

The very first cell therapy: 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%)

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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

AML, 39%(early AML 21%,advanced AML 12%,transformed AML 6%)

CML, 2%

MDS/MPN, 12%MPN, 35%

ALL, 16%

Others, 1%AID, 0.1%

IDM, 0.8%PID, 3%

Thal/sickle, 3%

CLL, 2%

HD, 3%

NHL, 4%

PCD, 7.1%

Solid tumors, 0.2%BMF, 5%

Source: Solh 2017; McCurdy 2017; Broder 2017; Passweg 2017; CIBMTR 2016 summary slides; Besse 2015

MPN, 3%

Disease indications for allogeneic HSCT in Europe in 2015

Kiadis: aim to enable HSCT with reduced risk of GVHD

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FUNDAMENTALLY

SAFER HSCT

CLOSE TO

MARKET

STRONG PATIENT

BENEFIT

PLATFORM FOR

GROWTH

• ATIR allo-depleted T-Cell product, given after HSCT, designed to

reduce risk of GVHD, can unlock full HSCT potential

• Improvement in GVHD-Free and Relapse-Free Survival (GRFS) vs.

literature for PTCy/Baltimore protocol

• EMA potential approval 4Q18 (CHMP); Potential initial EU launch 2H19; FDA RMAT ‘breakthrough’ designation; Phase 3 enrolling

• Potential >27,900 target patients per year; Sustainable;

Attractive economics vs. CAR-T

• Ability to leverage cell therapy infrastructure: manufacturing,

supply chain, medical, commercial

ATTRACTIVE BUSINESS

OPPORTUNITY

HSCT challenge: give potent donor T-cells, yet avoid GVHD

Protect against viruses and tumor cells

Attack patient tissue

Donor stem cells (CD34+)

Source: Walker 2004

The ‘holy grail’ of HSCT: Give donor T-cells that protect

Remove donor T-cells that attackAvoid immunosuppressants

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Donor T-cells (CD3+)

Immunosuppressants(reduce GVHD, but

hamper engraftment and protection)

Stem cells engraft in patient bone marrow: create blood and immune system

But, stem cells alone not enough: T-cells take 6-12 months to re-emerge from stem cells; survival without T-cells 10-20%

Graft Versus Host Disease (GVHD): a terrible disease

Types Acute: can be life threatening (grade III/IV) Chronic: can be severely debilitating

Organs Skin, mouth, eyes, liver, GI tract, lungs

Manifestations Scleroderma, rash, ulceration, erythema, cirrhosis, immunodeficiency

EffectsSkin disease, infections, muscle constriction, bone loss, pulmonary disease, thyroid disfunction, ophthalmology, solid tumors, sleep deprivation, depression

Treatment Immunosuppression (steroids, MMF, MTX)

Subpopulation of mature T-cells from donor attack antigenically foreign antigens on patient tissues, due to

genetic differences between patient and donor (MHC Class II proteins)

Source: Jones 2016; Koreth 2013 7MMF: mycophenolate mofetilMTX: methotrexate

HSCT: Strong growth, still large unmet need

8Source: 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*

Haploidentical donors

Matched Unrelated Donor (MUD; registries)

Matched Related Donors (MRD)

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

Haplo PTCy/Baltimore vs MUD: improved GVHD, worse relapse

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* Not a ‘true’ average: Mix of indications (AML, NHL, HL), follow up (1.5, 2 and 3 yr) and patient populations; analysis based on ratio within each of the publications in the review paper shows similar resultsSource: Fuchs E 2017

38%

53%

38%

51%

26% 27%

45%49%

'Average'MatchedUnrelated Donor

'Average'Haploidentical(PTCy)*

Acute GVHD II-IV

Chronic GVHD

Relapse Survival

Post Transplant Cyclophosphamide (PTCy or Baltimore protocol): Trigger immediate ‘GVHD attack’ by activation of half-matched donor T-cells in the patient, treated with chemotherapy on days 3&5 and immunosuppressants

• Lower GVHD for PTCy than MUD (ie effective depletion of alloreactive T-cells)

• Higher relapse for PTCy than MUD (ie also depleting protective T-Cells)

MUD: Matched Unrelated Donor

Haplo HSCT 30 days after Haplo HSCT

GVHD Treatment/Prophylaxis Approach

KiadisT-cell depleted (‘safe’ stem cells)

ATIR: subset of T-cells, depleted of alloreactive T-cells (‘safe’ T cells)

No prophylactic immunosuppressant

PreventGVHD

PTCy or Baltimore protocol

T-cell replete (stem cells plus all T-cells)

Chemo (cyclophosphamide) & immunosuppressants to treat immediate attack from alloreactive donor T-cells

TreatGVHD

Kiadis: potential improvement versus PTCy/Baltimore

Kiadis: reduce risk of GVHD: give T-cells that protect; not T-cells that attack

10Table provided for illustrative purposes, not for direct comparison

ATIR: outpatient infused drug; add on to HSCT

Apheresis of patient and

donor

Infusion of ATIR

Central ATIR production, 5 day process

14 days before HSCT

~30 days after HSCT

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Conditioning of patient

Apheresis of donor, graft

infusion

Engraftment of donor

stem cells

ATIR (add on to HSCT)

HSCT

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

Step 1 (Day 1–4)

Patient cells inactivated by radiation

Healthy donor

Patient

Immune cells are collected and mixed

Step 3 (Day 5)Step 2 (Day 5)donor

patient

```

ATIR: remaining potent non-

alloreactive donor T-cells, infused on

day 28-32 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* induces

apoptosis: activated and 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

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ATIR production: fast & simple, own facility secured

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ü 5 day process

ü No genetic engineering (No viral vector production/transfection)

ü Clean rooms with LAF cabinets (No BL2; No bioreactors)

ü 42 hour hold time for ingoing material, final product frozen down

ü Own commercial manufacturing facility secured (lease)

Lower COGS per batch and capex compared to e.g. CAR-T

ATIR: Past and current clinical trials in blood cancers

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Phase 1 Phase 2 Phase 3

CR-GVH-001• Dose finding • 19 patients; Complete

CR-AIR-006• Historic observational cohort

(control)• 35 patients; Complete

CR-AIR-009• Randomized/controlled• 195 patients; Enrolling

CR-AIR-007• Open label single arm efficacy • 23 patients; Complete

CR-AIR-008• 9 patients only 1st dose; 6

patients also 2nd dose • 15 patients; Enrolment complete

ATIR as add on to haploidential CD34+ HSCTAML/ALL/MDS

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

15* 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%

0% 0%

4%5%

8%**

24%

Acute

GVHD III/IV

Chronic

GVHD

Severe

Chronic

GVHD

Phase 2 (007): relapse & GVHD vs literature for PTCy (1 yr)

Comparison provided for illustrative purposes, based on literature comparison, NOT based on randomized controlled trials* PTCy: Relapse: of which 11% relapsed and 18% relapsed and died

** Solomon 2012; Ciurea 2012; Esquirol 2016

*** Ciurea 2015; Piemontese 2017, Solomon 2012, Ciurea 2012; Devillier 2016; Di Stasi 2014; Esquirol 2016; Sugita 2015

**** Ciurea 2015; McCurdy 2017, Devillier 2016, Sugita 2015 (normalization based on Armand 2014); DRI CR-AIR-AIR-007: 53% high/47% intermediate

Overall SurvivalATIR Phase 2, 007

(MITT, n=23)

PTCy/Baltimore

Literature, at least 50%

AML/ALL*** (n=571)

Relapse and NRM*

9%

30%29%

22%

Relapse Non Relapse

Mortality

PTCy: results from

EBMT/CIBMTR databases,

Johns Hopkins (Baltimore)

and Northside (Atlanta)

61% 60%57%

Overall Survival

PTCy/Baltimore

Literature, adjusted for

DRI**** (n=561)

GVHD

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Phase 2 (007): GRFS versus literature for PTCy/Baltimore (1 yr)

GVHD-Free and Relapse-Free Survival (GRFS) Composite endpoint, capturing

survival and future outlook

Defined as survival without:• Chronic GVHD requiring

immunosuppression• Acute grade III/IV GVHD• Relapse

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

* Solh 2016 (Atlanta; DRI normalized GRFS 30%; n=128); McCurdy 2017 (Johns Hopkins; DRI normalized GRFS 38%; n=372)

57%

36%

GRFSATIR Phase 2

007 (MITT; n=23)

PTCy/ Baltimore

DRI adjusted* (n=500)

PTCy: single site results from Johns Hopkins (Baltimore) and

Northside (Atlanta)

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Future options for patients: ATIR after CD34+ or after PTCy

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Stem cells (CD34+; Phase 2)

PTCy/ Baltimore

(literature*)

+ ATIR20%

+ATIR ?57%

61%

future study

Overall Survival (OS) GVHD

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

* Ciurea 2015; McCurdy 2017, Devillier 2016, Sugita 2015 (normalization based on Armand 2014); DRI CR-AIR-AIR-007: 53% high/47% intermediate

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

* Various hemato-oncology products EMA (conditionally) approved 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) approval 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 received, adequate response end Q1 2018

FDA (US)

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)

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

Phase 3 (009): ATIR vs. PTCy/Baltimore protocol

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**)

Secondary endpoints: OS, Progression Free Survival, Relapse Related Mortality, Transplant Related Mortality

Event driven:Primary analysis at 93 GRFS events, Interim analysis at half GRFS events

195 patients* with acute leukemia or MDS40-50 sites in US, Canada and EU

Aligned with FDA and regulators in EU; Enrolling patients

* 80% powered to detect 20% GRSF 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 20

ATIR potential: target >27,900 annual patients US/EU

* EU Not Known; ** EU 2000, US 900Provided for illustrative purposesSource: CIBMTR summary slides 2017, Passweg 2017, Besse 2015;

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TBD

12,000

13,000 (US*)

2,900

Haploidentical transplants 2016**

Unmet need(lack of matched

donors)

Matched Unrelated Donor

Blood disorders, auto-immune

disease

Potential haploidentical

transplants

Growth haploidentical HSCT: Historic 32% compound annual growth with PTCy; potential for acceleration upon increased benefits of ATIR

>27,900

1 in 3

1 in 12

1 in 4Overallchronic GVHD

SeverechronicGVHD

Relapse

Reduce risk of (severe) GVHD and relapse

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Death• 50% five year mortality for

severe chronic GVHD

Quality of life• QALY loss: 27% for severe

GVHD (up to 68%) and 20% for

moderate GVHD *

• Quality of life with severe

GVHD worse than vision

impairment, MS, loss of

arm/leg, diabetes

• Impact on patients, donors,

families, physicians

Loss of income• 75% of patients lose 3 years of

earnings

• 25% of patients permanently

lose earnings

Risk with PTCy Risk with ATIR

1 in 28

1 in 9

0

ATIR market survey 100 US/EU KOLs (2013)

Note: Needs to be updatedSource: ATIR Assessment, September 2013 by Defined Health, based on Phase 1 data: 100 transplant physicians/KOLs (50/50 US/EU; eg Harvard, Johns Hopkins, MD Anderson, Stanford, Dresden, Saint Antoine)

In what percent of your haplo’swould you use ATIR given strong

Phase 3 data and formal approval?

It’s got to show an advantage over PTCysomewhere. I’m not saying necessarily

superiority of survival but even if there’s a

superiority in terms of infection rate, it’s got to show that it’s better in

some respect

EU

US

23

““55%

58%

ATIR potential reimbursement: drug, separate from HSCT

24

ATIR (Medicare: Part B, J-code)

• Outpatient infused drug (~30 days after HSCT, 1-2 weeks after end of hospitalization); no hospitalization for infusion*

• Cost of drug billed to payer (‘buy & bill’, mark up for physician); Apheresis reimbursed via procedure code or bundled into ATIR

• Examples: Checkpoint inhibitors

HSCT(Medicare: MS-DRG-14)

• Covers HSCT procedure, including hospitalizations and follow ups• Additional CD34 selection already routinely reimbursed within DRG • Cost savings to hospital due to reduced use of cyclophosphamide and

AEs (e.g., Hemorrhagic cystitis)

• CAR-T SAEs require hospitalization and thus inpatient/DRG• Source: interviews

Likely differentiated from CAR-T*

ATIR protection: patents, orphan drug, know how

Patents (owned/ licensed)

• Methods for reducing GVHD including composition (P015; October 2021)• More rhodamine derivatives (P019; January 2024)• Improved photodynamic process (P040; February 2036 – if granted)

Orphan drug designation

• US (7 years from launch): for prevention of GVHD or TRM• EU (10 years from launch): for treatment in HSCT regardless of disease,

for treatment of AML, for prevention of GVHD

Proprietary know how

• Manufacturing critical process parameters• Release assays based on critical quality attributes• Cell handling, storage, formulation and shipment• Patient specific supply chain and communication

Biosimilar unfeasible: head to head comparative trial not possible for patient specific cell therapy product

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Kiadis management: track record commercial/medical/ops

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Arthur Lahr (April 2017) Chief Executive OfficerChief Strategy Officer Crucell (8 years; head BD, M&A, M&S US/EU); Supervisory Board Sanquin (Dutch blood bank); McKinsey; Unilever

Robbert van HeekerenChief Financial OfficerHead Global Finance & Control Organon

Jan Feijen (April 2017)Chief Operations OfficerEVP Operations J&J Vaccines and VP Manufacturing and Ops J&J Vaccines & Advanced Therapies; VP Crucell Asia; Managing Director Operations Avebe; Gist-Brocades

Andrew Sandler (Oct 2017)Chief Medical OfficerSVP Medical Affairs Medivation; CMO Dendreon and Spectrum Pharma; Bayer; Berlex; Seattle Genetics; Board certified medical oncologist

Karl Hård (Sept 2017)Head IR & communicationsHead investor relations AstraZeneca; 10 years investor relations; 10 years pharma R&D; Assistant Professor chemistry

Margot HoppeGeneral Counsel20+ years in corporate legal,including Gist-Brocades, DSM

Kiadis: company at a glance

• Management track record operations, development and commercial (Crucell, Medivation, J&J, AstraZeneca, Organon, DSM, McKinsey)

• Strong supervisory board (Cipla CEO/Novartis ExCom; Actelion COO, Prosensa/Jerini CFO; Prof Dana Farber)

• Euronext Amsterdam/Brussels, listed in 2015

• Major shareholders (>5%): LSP, Draper Esprit, Alta

• Analysts: Jefferies, Canaccord, Oppenheimer, Chardan, KBC, Kempen, LifeSci

• Market cap: ~$248M / €202M

• 17.5 million shares outstanding

• Raised €40M in equity & debt since June 2017; €77M in/since IPO

27

ORGANIZATION

SHAREHOLDERS

FINANCIALS (February 21, 2018)

Kiadis key (potential) milestones

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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 ✔ (extra)

Secured own commercial manufacturing facility (lease) ✔ (extra)

New management and supervisory board members ✔ (extra)

2018

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

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

Potential (conditional) EMA approval in EU (CHMP opinion, Q4)

Updates Phase 2 data and Phase 3 enrollment

2019Potential initial commercial launch ATIR in first of EU5 countries (H2)

Initiate trial with ATIR as adjunctive to PTCy

Potential interim read out Phase 3

Kiadis: aim to enable HSCT with reduced risk of GVHD

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FUNDAMENTALLY

SAFER HSCT

CLOSE TO

MARKET

STRONG PATIENT

BENEFIT

PLATFORM FOR

GROWTH

• ATIR allo-depleted T-Cell product, given after HSCT, designed to

reduce risk of GVHD, can unlock full HSCT potential

• Improvement in GVHD-Free and Relapse-Free Survival (GRFS) vs.

literature for PTCy/Baltimore protocol

• EMA potential approval 4Q18 (CHMP); Potential initial EU launch 2H19; FDA RMAT ‘breakthrough’ designation; Phase 3 enrolling

• Potential >27,900 target patients per year; Sustainable;

Attractive economics vs. CAR-T

• Ability to leverage cell therapy infrastructure: manufacturing,

supply chain, medical, commercial

ATTRACTIVE BUSINESS

OPPORTUNITY

30

‘Practice two things in your dealings with disease: either help or do not harm the patient’

Epidemics, Book I, of the Hippocratic school

‘I will prevent disease whenever I can but I will always look for a path to a cure for all diseases’

Hippocratic oath, Louis Lasagna, Academic Dean of the School of Medicine at Tufts University (1964)

Attachments

31

Additional information

• Competition

• Pricing

• ATIR product characteristics

• Clinical trial information

• Disease Risk Index adjustment for literature comparisons

32

Haplo HSCTHaplo donor T-cell Product (after HSCT)

GVHD Treatment/Prophylaxis (in patient)

Approach to GVHD

ATIR (Kiadis) T-cell depleted

‘Safe’ subset of T-cells, depleted of alloreactive T-cells

No prophylactic immunosuppressant Prevent

Zalmoxis (MolMed)BPX-501 (Bellicum)

T-cell depleted

All T-cells, but engineered with ‘suicide gene’

Eliminate activated T-cells by infusing suicide agent, if GVHD occurs

Treat

PTCy or Baltimore protocol

T-cell replete (All T-cells)

Post Transplant Cyclo-phosphamide & immunosuppressants

Treat

Comparison Kiadis with MolMed/Bellicum

33Table provided for illustrative purposes, not for direct comparison

Patients

Effect of

product* Survival** Relapse NRM

Status

EMA

ATIR

(Kiadis)

Adult

Potential CHMP

Q4 2018

Zalmoxis

(MolMed)Adult

Approval Q4 2016

BPX-501

(Bellicum)Pediatric

Submission 2019

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

* ATIR 007 MITT data (N=23). Matched historical control for Zalmoxis includes T-cell replete and T-cell deplete, thus effect of product cannot be determined** Leukemia Free Survival for BPX-501 (BPX-501 Overall Survival is 89%, Overall Survival not reported for controls); *** Adults PTCy, pediatric MUD; except for ATIR not DRI adjusted/matchedLow GVHD for all three; 5% Grade III/IV for BPX-501 (resolved after rimiducid) Source: CHMP Assessment report (Zalmoxis); Merli EHA 2017 (BPX-501); Locatelli 2017 (BPX-501)

3.0x

1.1x

29

29

19

21

20

15

42

9

22

5

60

3

20

30

46

22

Competitive overview – available data January 2018 (1 year)

Product (Matched) Historical Control MUD or PTCy***

34

77

57

57

77

34

20

83

51

61

Zalmoxis (MolMed) – EMA filing data and EU pricing

Survival Relapse and NRM

9%

30%

42%

20%

29%

22%

Relapse Non RelapseMortality

61%

51%

60%

Overall Survival

4%7% 6%

5%

24%

AcuteGVHD III/IV

ChronicGVHD

GVHD

Comparison provided for illustrative purposes, based on literature comparison, NOT based on randomized controlled trials * CHMP Assessment report (aGVHD III/IV: Kempen 2017 report); CD34+ HSCT; 74% AML; 10% ALL; 16%MDS/NHL/HD; patients receiving Zalmoxis** Ciurea 2015 (CIBMTR); Piemontese 2017 (EBMT), Solomon 2012 (Atlanta), Ciurea 2012; Devillier 2016; Di Stasi 2014; Esquirol 2016; Sugita 2015*** Prices as at 16 January 2018 and 13 December 2017, respectively

ATIR Phase II (007, MITT, n=23)

Zalmoxis (MolMed)Phase II data in EMA filing, n=36*

Zalmoxis pricing/dose*** (1-4 doses per patient):

Germany: €163,900Italy: €149,000

0%

35

PTCy/BaltimoreLiterature, at least 50% AML/ALL** (n=571)

BPX-501 (Bellicum): AML/ALL pediatric (Zhou 2014)

36

Population• Pediatric, aged 3-17, 10 pts• CD34+ HSCT

1 Year data• Survival 80%• Relapse 30%• Non Relapse Mortality 0%

Observations• Low GVHD, at high doses of T

cells (incl. alloreactive T-cells)• 3 out of 4 patients treated with

rimiducid to treat GVHD subsequently died from relapse (day 158, 164, 591)

Infused T cells/kg

aGVHD cGVHD Administration of AP1903

Current status (day after SCT*

1x106 Grade I/II

None Yes Alive, CR (D+1440)

1x106 Grade I None Yes Relapse of ALL (D+552), death D+591

3X106 None None None Alive, CR (D+1388)

3X106 Grade I None Yes Relapse of ALL (D+57), death D+158

1X107 Grade I None Yes Relapse of ALL (D+158), death D+164

1X106 None None None Alive, CR (D+1016)

1X107 None None None Alive, CR (D+954)

1X107 None None None Alive, CR (D+835)

1X107

1X106None None None Relapse (D+312), alive, CR (D+475)

second allo-HSCT)

1X107

5X106None None None Death from respiratory failure secondary

to refractory AIHA (D+615)

CAR-T therapies

• First products approved by FDA (B-cell lymphomas), EU approval expected in 2018̶ Kymriah (Novartis): list price $475,000 (outcome based)̶ Yescarta (Kite/Gilead): list price $373,000

• In most cases used as a bridge to a HSCT• Most successes from targeting CD19

(i.e., ALL, CLL, B-cell lymphomas; esp. refractory ALL)• Also pursued by Juno, Cellectis, Amgen, Pfizer, Celgene, Merck KgaA• Reported issues

‒ Persistence: Even in ALL with remissions of 90%, many patients relapse (most in ALL)‒ Antigen-negative relapse: CAR-T no longer able to target because cells no longer display the desired antigen, so far

only with CD19‒ Safety: severe cytokine release syndrome and neurotoxicity correlated with efficacy‒ Manufacturing (viral vectors): complexity, time, capacity, yields, batch failures, costs

Source: Evaluate Pharma report 2016; Novartis and Gilead/Kite press releases 37

Healthcare costs of Allogeneic HSCTs*

38

* Includes Inpatient/Outpatient/pharmacy costs** Includes different physician charges, graft procurement costs

Total HSCT costs Period / Source

$401,000 (MA)$301,000 (RIC)

100 days; Broder 2017

$549,000 (MA)$432,000 (RIC)

1 year; Broder 2017

$893,000 180 days; Milliman 2017**

Excludes lifelong costs of chronic GVHD

MA: myeloablative conditioningRIC: reduced intensity conditioning

Costs of HSCT complications

HSCT complications Costs to healthcare system (US)

Cancer death $165,000

Relapse $69,000*

Hemorrhagic cystitis $242,000*/**

Acute GVHD $527,000*

Chronic GVHD moderate/mild $124,000 ($14,400 per year***)

Chronic GVHD severe $322,000 ($37,400 per year***)

39

* Cost based on Broder total cost and cost multiplier Khera

** Side effect of cyclophosphamide

*** 10 years, discounted

Sources: Mariotto 2011; Yu 2017; Broder 2017; Khera 2014; literature PTCy analysis

Examples EU conditionally approved ATMPs/leukemia drugs

40

Approved indication Trial Design Trial size List Price

Treatment of patients with ADA-SCID who cannot be treated by a bone-marrow transplant because they do not have a suitable, matched, related donor

Open-label, prospective, sequential study in children with SCID due to ADA deficiency who lacked a healthy HLA-identical sibling

12 patients GBP 594,000 (UK)

Treatment of moderate to severe limbalstem-cell deficiency caused by burns, including chemical burns, to the eyes

Two retrospective, multicentre, case series based, non-randomised, and uncontrolled observational studies

135 patients over 9 years

GBP 80,000 (UK)

Adjunctive treatment to CD34+ haploidentical stem cell transplantation

Open label, single arm phase II study 36 patients €163,900 per dose (DE); €149,000 per dose (Italy)

Treatment of adults with Philadelphia chromosome negative relapsed or refractory B-precursor acute lymphoblastic leukaemia (ALL)

Open-label, multicentre, single-arm Phase 2 study

€87,000 (UK, DE; per course)

Second or third-line treatment in chronic lymphocytic leukaemia (CLL) in the presence of 17p deletion or TP53 mutation in adult patients

Open-label, single-arm Phase 1 and 2 studies

€99,000 (DE)

ATIR101: alloreactive T-cells depleted, potency retained

Functional release assay based on Quality Target Product Profile

& Critical Quality Attributes

41Source: Bonig ISCT 2017

*

Control: no donorreactivity

Safety: depleted

allo-reactivity

Potency:other reactivity

retained

12

6

1.6

1.4

1.2

1.0Autologous Recipient 3rd Party CD3/28

Prol

ifera

tion

Inde

x (PI

) DonorATIR101

ATIR101: T-cells reactive against infections & tumor retained

Donor

ATIR Control

Myb628multimer

CD8 T-cells

Collaboration with Prof. Angela Krackhardt, Medizinische Klinik III, Klinikum Rechts der Isar, TU Munich, Munich, Germany 42Source: Bonig ISCT 2017

ATIR101: T-cells reactive against EBV retained – examples

Examples of two patients in clinical study with ATIR:

EBV reactivation triggered response of (viral specific) T-cells in several patients• Increase in CD3+ T-cells detected in

peripheral blood• EBV copy numbers reduced after

increase in CD3+ T-cells, indicating effective immunological T-cell response

43

Phase 1 (CR-GVH-001): Overall Survival (5 year)

Patients: 19 with advanced hematological malignancies (14 not in remission at transplant)

ATIR101 doses: 10k cells/kg to 5 mln cells/kg; 31 days after HSCT

Results:

• 67% Overall Survival at middle dose level after 5 years

• No acute grade III/IV GVHD related to ATIR101 at any dose

Overall Survival (OS)

Dose L1-L3

Dose L4-L6

Dose L7

0%

20%

40%

60%

80%

100%

0 6 12 18 24 30 36 42 48 54 60

Time after HSCT (months)

3 patients; 2,6-5M cells/kg)

7 patients, 10-130k cells/kg

9 patients, 320k-2M cells/kg

Note: un-manipulated haplo-identical Donor Lymphocyte Infusion escalated above 10k T cells/kg induce GVHDSource: Lewalle 2003 44

Phase 2 (CR-AIR-007): trial characteristics & endpoints

45

Design: Open-label, single arm, multi-center study

Patient population:• AML or ALL in first remission with high-risk features or in second or higher remission• No suitable matched donor• Haploidentical family

Locations: CA, BE , DE, UK (8 sites in total, patients from 4 sites)

Primary endpoint: Transplant Related Mortality (TRM) at 6 months

Secondary endpoints:

• Acute and chronic GVHD• Immune reconstitution• Infections• TRM, relapse, Overall Survival (OS)

Patient follow-up (per 27 September 2016):

Median 485 days (range 110 – 744)

Phase 2 (CR-AIR-007): patient & donor characteristics

1 Mrozek 20122 Armand 2014 46

Patient and donors

• N=23 patients (HSCT + ATIR101)• Median patient age (range):

41 years (21-64)

• Gender: 13 female, 10 male• Median donor age (range): 33 years (21 – 61)

DiagnosisAML: n=16 (70%):• 11 in CR1

• 5 in CR2

ALL: n=7 (30%):• 4 in CR1

• 3 in CR2

Risk classification

Cytogenetic risk profile1:

• Favorable 0

• Intermediate 9 (39 %)

• Adverse 14 (61 %)

Disease-risk index2:

• Low risk index 0

• Intermediate risk index 10 (43 %)

• High risk index 13 (57 %)

Phase 2 (CR-AIR-007): causes of deathPeriod post HSCT Classification No. of pts Classification of cause of death

< 6 months

Relapse 1

TRM – Infections 2 Adenovirus and JC virus infections

TRM – Other 1 Pulmonary embolism

6-12 months

Relapse 1

TRM – Infections 3 Respiratory/pulmonary infections/distress

TRM – Other 1 Multi-organ failure

12-24 months

Relapse 2

TRM-Infections 3* Pneumonia/sepsis/septic shock

Total 14 (61%)

* All 3 patients immunosuppressed, subsequently contracted infections, leading to death: 2 patients who received un-manipulated DLI’s and subsequently developed severe acute GVHD; 1 patient with chronic GVHD 47

Objective Extend the length of protection (further improve TRM); investigate flexibility for physicians

Design HSCT followed with ATIR101 at day 30, and additional dose of ATIR101 at day 72

Patients AML, ALL and MDS

StatusEnrolment completed: 15 patients treated with ATIR101: 9 with one dose (of which 5 with > 1 year follow up) and 6 with two doses (all with > 1 year follow up)Study ongoing, not all data yet monitored and thus subject to change

Interim results

Two doses (for 6 patients): grade III/IV acute GVHD in two patients within 30 days of 2nd doseSingle dose (for 5 patients that have > 1 year follow up): • No grade III/IV GVHD• 80% Overall Survival; 20% 1 year Relapse, 0% 1 year Non Relapse MortalitySingle dose (for 4 patients that have < 1 year follow up): 1 suspected grade III acute GVHD

Interim conclusion

Confirming safety/efficacy findings of 007 in the 5 patients with a single dose that have >1 year follow up; Not confirmed with two doses or with remainder of single dose patients.Phase 3 009 based on 007, not based on 008

Phase 2 (CR-AIR-008): second dose (January 2018)

48

Phase 2 (007 & 007/008) vs. PTCy literature (1 yr)ATIRPhase 2 007/008 (MITT; pts receiving ATIR; n=28*)

PTCyLiterature, adjusted for 007 DRI for OS (n=561)** & for GRFS (n=500)**

Comparison provided for illustrative purposes, based on literature comparison, NOT based on randomized controlled trials: * 23 AML/ALL patients from 007 (study completed) plus 5 AML/ALL patients from 008 that have completed 1 year follow up with single dose (008 data not all monitored and thus subject to change); for 28 AML/ALL 007/008 patients: Relapse rate 11% and NRM 25%, chronic GVHD 4%** Ciurea 2015 (CIBMTR); McCurdy 2017 (Baltimore), Devillier 2016, Sugita 2015 (57% high DRI; normalization based on Armand 2014) *** Solh 2016 (Atlanta; DRI normalized GRFS 30%; n=128); McCurdy 2017 (Johns Hopkins; DRI normalized GRFS 38%; n=372) 49

Survival GRFS

61%57%

54%

36% 37%

ATIRPhase 2 007 (ITT; pts receiving HSCT; n=26)

ATIRPhase 2 007 (MITT; pts receiving ATIR; n=23)

64%61%

58% 57% 59%

OS

PTCyLiterature, adjusted for 007/008 DRI for OS (n=561)* & for GRFS (n=500)**

Disease• Anemia due to defects in haemoglobin and red blood cells• Transfusion dependency and iron overload, leading to high mortality

HSCT• Potential cure of blood forming system, yet high risk• ATIR201 to increase safety

Trial design• ATIR201 as adjunctive to αβ T-cell depleted HSCT• 10 pediatric patients with β-thalassemia major

Status• No patient dosed yet• Postponed

ATIR201 Phase 1/2: β-thalassemia

50

DRI adjustment (Armand 2014)

DRI adjusted 1-year survival (OS)

comparison ATIR vs all HSCT

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

Armand 2014: 9849 HSCT patients

(CIBMTR 2008-2010):

• MRD, non-MRD, MUD, mismatched

• PB, BM, cord

• Leukemia, lymphoma, MM, etc

• Myeloablative, RIC

61%55%

GRFSATIR101

Phase 2

007

(n=25)

Average HSCT

in Armand 2014;

DRI adjusted

0 6 12 18 24

Months from transplantation

0

20

40

60

80

100

Ove

rall S

urv

iva

l (%

)

Very High Risk

High Risk

Intermediate Risk

Low Risk

P<0.0001

51

Conclusion: Disease Risk Index (DRI) is strongest prognostic factor for Overall Survival

52

…so that many more patients with otherwise incurable diseases will have a reasonable chance of long survival and cure

Dr. E. Donnall Thomasestablished bone marrow transplantation as a treatment for leukemiaNobel Lecture | 1990