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Innovative Therapies for Retinal Disorders: Developing Disease Modifying Treatments for Diabetic Eye Disease Dr. Patrik De Haes, CEO Dominique Vanfleteren CFO 2017 Full Year Results Presentation - March 15, 2018

Innovative Therapies for Retinal Disorders: Developing ... BU FY17 Concall... · freed up in January 2018 following completion of NVS ... o THR-149 is targeting the treatment of diabetic

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Page 1: Innovative Therapies for Retinal Disorders: Developing ... BU FY17 Concall... · freed up in January 2018 following completion of NVS ... o THR-149 is targeting the treatment of diabetic

Innovative Therapies for Retinal Disorders:Developing Disease Modifying Treatments for Diabetic Eye Disease

Dr. Patrik De Haes, CEO

Dominique Vanfleteren CFO

2017 Full Year Results Presentation - March 15, 2018

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Disclaimer

By attending the meeting where this presentation is made, or by reading the presentation slides,you agree to be bound by the following limitations:

o This document has been prepared by ThromboGenics NV (the "Company") and is being supplied to you solely for your information and use by you at the Company presentation. This document and its contents are confidential and may not be further distributed or passed on to any other person or published or reproduced, in whole or in part, by any medium or in any form for any purpose. All the numerical data provided in this document are derived from ThromboGenics’ consolidated financial statements.

o No representation or warranty expressed or implied is or will be made as to, and no reliance should be placed on, the fairness, accuracy, completeness, or correctness of the information or opinions contained herein. The information set out herein may be subject to updating, completion, revision, verification, and amendment, and such information may change materially. The Company is under no obligation to update or keep current the information contained in this document or the presentation to which it relates, and any opinions expressed in it are subject to change without notice. None of the Company or any of its affiliates, its advisors, or representatives shall have any liability whatsoever (in negligence or otherwise) for any loss whatsoever arising from any use of this document or its contents or otherwise arising in connection with this document.

o The following information does not constitute investment advice, and shall not constitute an offer or invitation for the sale or purchase of securities or assets of ThromboGenics in any jurisdiction. No securities of ThromboGenics may be offered or sold within the United States without registration under the U.S. Securities Act of 1933, as amended, or in compliance with an exemption therefrom, and in accordance with any applicable U.S. state securities laws.

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PhI/II TB-403

5 I/O projects

(preclinical)

• +82% (>85%) THR owned

• Fully funded 3 yr

• VIB leads I/O developments

• Phase I/II clinical trial TB-403

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Global BU

Commercial

• 100% THR owned

• US cash neutral

• Ex-US in transition from NVS

3 Disease Modifying Treatments

Targeting Unmet Medical Needs

in Diabetic Retinopathy

• THR-317 – anti-PlGF – Phase I/IIa

• THR-149 – plasma kallikrein inh.

• THR-687 – integrin antagonist

• Cash position = >€100 million

• Funded –2-3 yrs

3

ThromboGenics value proposition: focused on development of promising diabetic eye disease pipeline

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2017/18 Highlights

o Continued to advance promising clinical and preclinical pipeline of diabetic eye disease candidateso THR-317 – last patient enrolled in Phase I/IIa study in DME – initial results expected by end of Q1o THR-149 – expected to enter the clinic in H1 2018o THR-687 – expected to enter the clinic mid-2018o At least one new drug candidate to enter development in 2018

o ThromboGenics received €53.7 million in cash and a €10 million equity investment from Novartis as part of regaining non-US rights to JETREA®

o Oncurious acquired 5 next generation immuno-oncology assets as part of agreement with VIB

o ThromboGenics strengthened its leadership in November with two new executive team members: o Susan Schneider, MD - Chief Medical Officer (CMO)o Vinciane Vangeersdaele - Chief Commercial Officer (CCO)

o Thomas Clay was elected as the new Chairman of the Board of Directors, effective 15 June 2017

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€ Million FY 2017 FY 2016

Income 9.1 7.1

US 2.9 4.4

Ex-US 2.9 2.7

Other 3.3 * NA

R&D expenses (23.2) (24.6)

Selling expenses (4.2) (4.3)

Other income 50.4 ** NA

Net result 22.6 (60.4) ***

EPS (€) 0.62 (1.67)

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Financial Review FY 2017

* Settlement dispute vials price

** Including €45m contract ending compensation and €4.5m intervention obsolent drug materials

*** Including €26.6 millon impairment charge taken on JETREA®sales

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Financial Review FY 2017

• Cash and investments of €115.7m end December 2017, including restricted cash

freed up in January 2018 following completion of NVS investment (vs. €80.4 m end

December 2016)

• Net profit of €22.6m end of December 2017 : extra ordinary income of €53.7 (from

Novartis)

• Continued tight operational cash management

• 2017 US operations break-even continued

• Available cash to fund ongoing and planned development activities for foreseeable

future

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THR-317 update

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THR-317: Targeting diabetic macular edema

o Phase I/ IIa study enrolment and treatment complete

o Only drug in development that targets solely PlGF -anti-inflammatory & anti-fibrotic

o Topline results anticipated end of Q1 2018

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Anti-PlGF (Placental Growth Factor)

Placental growth factor (PlGF) is an optimal target to treat more

advanced stages in diabetic eye disease

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THR-317: Phase I/IIa Study Overview

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Treatment population:

o Anti-VEGF naïve patients : 40

o Anti-VEGF poor responders : 10

n=50

THR-317 low dose

THR-317 high dose

SAFETY FOLLOW-UP

randomized 1:1

DAY 0 DAY 90 DAY 150DAY 30 DAY 60

EFFICACY EVALUATION

o BCVA

o Retinal Thickness

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THR-149 update

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Plasma kallikrein and DME patients

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0

500

1000

1500

2000

2500

3000

3500

0

5

15

10

20

PK

al (

fold

incr

ease

)

VEG

F (p

g/m

L)

DME patients ID #

PKal levels

VEGF levels

1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20

Adapted: Kita et al. 2015 Diabetes 64:3588–99

• Plasma kallikrein is a key driver in diabetic macular edema

• Potential as a stand-alone treatment and/or treatment in refractory DME patients to current therapy

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THR- 149 : Phase I study to be initiated H1 2018

o THR-149 is a potent and selective plasma kallikrein inhibitor

o THR-149 is targeting the treatment of diabetic macular edema (DME)

o impact on disease on-set and progression

o THR-149 offers the potential for two treatment modalities

o stand-alone therapy in diabetic retinopathy with DME

o refractory DME to current treatment

o Sizeable target population

o Phase I study to be initiated H1 2018

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THR-687 update

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THR-687: Phase I study to be initiated around Mid 2018

o THR-687 is a novel and potent integrin antagonist

o THR-687 has a broad therapeutic potential

o diabetic retinopathy with and without DME

o NPDR / PDR indication

o treatment of diabetic retinopathy

o induction of total posterior vitreous detachment

o Sizeable target population

o Phase I study to be initiated around Mid 2018

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Oncurious update - focused on immuno-oncology

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o Oncurious reached agreement to acquire VIB portfolio of 5 next-generation immuno-oncology projects targeting a broad spectrum of cancers in September

o VIB to receive royalty on future sales of these assets

o VIB increased shareholding in Oncurious

o ThromboGenics invests €2.1 million in Oncurious as part of this agreement

o This investment will return ThromboGenics’ ownership of Oncurious to 85%

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Recent Oncurious development – Entering I/O

Acquired exciting pipeline of next-generation immuno-oncology drugs targeting a broad spectrum of cancers

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Wrap-up

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PRECLINICAL

THR-317(anti-PIGF)

THR-149(plasma kallikrein

inhibitor)

THR-687(integrin antagonist)

Discovery (2)

PREP

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Pipeline newsflow for 2018/19:

2018 2019

PHASE I/ II PHASE II

Topline

PREP PHASE I!

FPI

PHASE I

FPI

!

FPI

!!

‘Multiple Shots on Goal´ – different mechanisms of action – data read outs targeting mid 2019

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ThromboGenics - focus on developing promising diabetic eye disease pipeline

o 3 clinical projects by mid 2018

o Over €100 million in cash to invest in pipeline development

o Pioneer of new drug class of pharmacological vitreolysis and 100% owner of

JETREA® (ocriplasmin)

o +82% (>85%) ownership of Oncurious NV: 5 next gen I/O projects + ongoing TB-

403 clinical trial

o Experienced management team focused on delivering value generating milestones

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Thank you for your interest

For questions and information :Please send your message at [email protected]