2016 half-year results and business update · 2018. 9. 13. · webcast presentation – 25 august...

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Nanobodies ® creating better medicines Webcast presentation 25 August 2016 2016 half-year results and business update

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Page 1: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

Nanobodies®

creating better medicines

Webcast presentation – 25 August 2016

2016 half-year results and business update

Page 2: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

2

Ablynx 2016 half-year results presentation

Participants on the call

Dr Robert K. Zeldin

CMO

Dr Edwin Moses

CEO

Wim Ottevaere

CFO

Page 3: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

3

Forward looking statements

Certain statements, beliefs and opinions in this presentation are forward-looking, which reflect the

Company or, as appropriate, the Company directors’current expectations and projections about

future events. By their nature, forward-looking statements involve a number of risks, uncertainties

and assumptions that could cause actual results or events to differ materially from those expressed

or implied by the forward-looking statements. These risks, uncertainties and assumptions could

adversely affect the outcome and financial effects of the plans and events described herein. A

multitude of factors including, but not limited to, changes in demand, competition and technology,

can cause actual events, performance or results to differ significantly from any anticipated

development. Forward looking statements contained in this presentation regarding past trends or

activities should not be taken as a representation that such trends or activities will continue in the

future. As a result, the Company expressly disclaims any obligation or undertaking to release any

update or revisions to any forward-looking statements in this presentation as a result of any

change in expectations or any change in events, conditions, assumptions or circumstances on

which these forward-looking statements are based. Neither the Company nor its advisers or

representatives nor any of its parent or subsidiary undertakings or any such person’s officers or

employees guarantees that the assumptions underlying such forward-looking statements are free

from errors nor does either accept any responsibility for the future accuracy of the forward-looking

statements contained in this presentation or the actual occurrence of the forecasted developments.

You should not place undue reliance on forward-looking statements, which speak only as of the

date of this presentation.

3

Page 4: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

4

Ablynx 2016 half-year results presentation

Agenda

• Welcome and introduction

• Significant achievements year-to-date

• Financial highlights

• Operational performance

• Outlook for the remainder of the year

• Q&A

• Conclusion

Page 5: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

Financial highlights

Financial results and shareholder information

Page 6: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

6

Financial summary

€ million H1 2016 H1 2015 % change

Total revenue and grant income 53.5 38.4 39%

R&D income 53.1 38.0 40%

Grants 0.4 0.4 -

Operating expenses (55.5) (45.9) 21%

R&D (49.0) (40.3) 22%

G&A (6.5) (5.6) 16%

Operating result (2.0) (7.4) 73%

Net financial result 24.9 (7.7) >100%

Net result 22.8 (15.2) >100%

Net operational cash flow (19.0) (1) (35.1) (2) 46%

Cash position at 30 June (5) 288.7 (3) 268.4 (4) 8%

(1) Excluding €71.4 million net proceeds from the private placement of new shares (announced on 1 June 2016)

(2) Excluding €97.2 million net proceeds from the convertible bond (announced on 20 May 2015)

(3) Including €1.3 million in restricted cash

(4) Including €1.6 million in restricted cash

(5) Cash, cash equivalents, restricted cash and short-term investments at the end of the period

Page 7: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

7

Shareholders

Diversified shareholder base – August 2016

• Ordinary shares listed on Euronext Brussels (ABLX)

• Sponsored Level I ADRs on the US OTC market (ABYLY)

• 60.9M shares outstanding

• 2.6M outstanding warrants (in number of shares)

Breakdown of share capital

US 41%

UK 21%

Belgium 24%

The Netherlands

9%

Scandinavia 1%

France 2% Other

1%

% of Institutional Shareholders by Geography (representing 78% of total S/O)

5% 5%

5%

5%

4%

4%

3%

Van Herk Investments B.V. (NL)

Bank of America (US)

Perceptive Advisors (US)

Fidelity Management Research (US)

Taube Hodson Stonex Partners (UK)

Boehringer Ingelheim (DE)

Oppenheimer Funds (US)

Other shareholders

Page 8: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

Product pipeline

Significant clinical pipeline achievements

>15 new pre-clinical programmes started

Page 9: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

9

Hybrid business model fuels the pipeline

>45 programmes with 8 Nanobodies in clinical development

Indication Product Target

aTTP

RSV

vobarilizumab

Pre-clinical Phase I Phase II Phase III

IL-6R

caplacizumab vWF

ALX-0171

ALX-0141 RANKL

ALX-0761

RSV

Bone disorders Greater China

IL-17A/IL-17F

ozoralizumab TNFα

Greater China

Filing

Japan

RA

RA

SLE

RA

Psoriasis

Immuno-Oncology

~ 15 wholly-

owned and

partnered

programmes

Up to 17

programmes

IL-6R

IL-6R

TNFα RA

Various

Various

+

CXCR2 Inflammation

Oncology VEGF/Ang2 BI 836880

Chronic kidney disease CX3CR1 BI 655088

Page 10: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

10

Vobarilizumab – anti-IL-6R Nanobody

Potential to be the best-in-class in RA

• Global exclusive option licensing deal with

AbbVie

• Compelling results from 2 Phase IIb RA

studies in a total of ~600 patients

• RA open-label extension study ongoing (94%

rollover rate)

• Phase II SLE study ongoing

• Opportunity in multi-billion dollar markets

RA: rheumatoid arthritis

SLE: systemic lupus erythematosus

Page 11: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

11

Vobarilizumab

2016 achievements

• Compelling results from the 12 week Phase IIb monotherapy study in

251 patients with moderate to severe RA

August July

• Compelling results from the 24 week Phase IIb combination therapy study

in 345 patients with moderate to severe RA

Page 12: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

12

Vobarilizumab Phase IIb study results in RA High ACR responses consistent across the two studies

• Strong effect on clinically meaningful efficacy endpoints resulting in high ACR50 and

ACR70 responses at week 12 (both as monotherapy and in combination with MTX)

• Continued improvement in ACR50 and ACR70 scores from week 12 to week 24 (in

combination with MTX)

MTX: methotrexate

Week 12

Phase IIb monotherapy

(vobarilizumab N=187; tocilizumab: N=64)

% r

esp

on

ders

* nominal p<0.05 vs. placebo; ** nominal p<0.01 vs placebo

Phase IIb combination therapy (+ MTX)

Week 12 Week 24

(vobarilizumab N=276; placebo: N=69)

% r

esp

on

ders

Page 13: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

13

Vobarilizumab Phase IIb study results in RA Strong impact on disease activity confirms best-in-class potential

Remission: DAS28CRP < 2.6; low disease activitiy: 2.6 ≤ DAS28CRP ≤ 3.2

• Vobarilizumab as a monotherapy induces clinical remission in up to 41% of patients as

compared to 27% for tocilizumab-treated patients

• Vobarilizumab in combination with MTX induces clinical remission in up to 49% of

patients at week 24

Week 12

Phase IIb monotherapy

* nominal p<0.05 vs. placebo; ** nominal p<0.01 vs. placebo; *** nominal p<0.001 vs. placebo

*** *** *

*** **

***

* ** ***

** *** ***

Phase IIb combination therapy (+ MTX)

Week 12 Week 24

% s

ub

jects

% s

ub

jects

42

57 60

44

Page 14: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

14

Vobarilizumab Phase IIb study results in RA Favourable safety profile confirmed in a larger patient population

• Side effect profile did not change with increased dosing

• Treatment-related serious adverse events occurred in:

- 0.5% of vobarilizumab-treated patients in the monotherapy study

(vs. 3.1% for tocilizumab-treated patients)

- 1.8% of vobarilizumab-treated patients in the combination therapy study

(vs. 2.9% for placebo-treated patients)

• In both studies, only infrequent abnormalities in laboratory parameters were

observed

• No negative effect on mean LDL/HDL cholesterol ratio across all doses

tested in both studies

Page 15: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

15

Vobarilizumab

Potential to be the best-in-class in RA

15

µ$

µµ

µ

µ

µµ

• Very strong impact on disease activity as measured by DAS28CRP

• Favourable safety and tolerability profile across all doses tested

• Opportunity for convenient monthly dosing

Potential to become a positively differentiated treatment option for patients suffering from RA

Page 17: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

17

ALX-0171 – inhaled anti-RSV Nanobody

First-in-class potential for treatment of RSV infections

• Biological drug delivered by inhalation – major

platform advantage

• Most advanced product in clinical development

to treat RSV infections in infants

• Critical pre-clinical and clinical milestones

achieved

• Phase II dose-ranging efficacy study in infants

planned to start in Q4 2016

• Opportunity in multi-billion dollar market

Page 18: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

18

Inhaled ALX-0171

• Recruitment from Q4 2014 to Q1 2016

• Study centres in Europe and Asia-Pacific region

• Adapted infant inhalation device (vibrating mesh)

• Inhaled ALX-0171 administered once/day, for 3 consecutive days

Phase I/IIa study in 53 hospitalised RSV-infected children

Primary endpoint:

Safety and tolerability of ALX-0171

Secondary endpoints:

Assessment of clinical effect (feeding, respiratory rate, O2

saturation, wheezing, coughing, general appearance), PD,

PK and immunogenicity

RA

ND

OM

ISA

TIO

N

2:1

Placebo N=10

ALX-0171 N=20 Open-label lead-in

N=5 Infants aged 3-24

months

Placebo N=6

ALX-0171 N=12

Infants aged 1-5

months Infants aged 5-24

months

DMC*

review

DMC*

review

* Data monitoring committee

Page 19: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

19

First-in-infant Phase I/IIa study

Safety and tolerability confirmed in the target population

Open-label group

ALX-0171 (N=5)

Randomised group

ALX-0171 (N=30)

Randomised group

Placebo (N=16)

Adverse events (AEs)

- number (%) of subjects with an AE 4 (80.0) 9 (30.0) 4 (25.0)

- number (%) of subjects with a treatment-related AE 1 (20.0) 2 (6.7) 0 (0.0)

Serious adverse events (SAEs)

- number (%) of subjects with an SAE 3* (60.0) 1** (3.3) 0 (0.0)

- number (%) of subjects with treatment-related SAEs 0 (0.0) 0 (0.0) 0 (0.0)

* 1 of whom discontinued ** subject discontinued

• Most common AEs were infections and respiratory disorders

• 3 AEs related to ALX-0171: mild cough, mild rhinorrhoea, mild fever 11 days after last dose

• 5 SAEs reported: hypo-responsiveness, hypotonia, pneumonia (2) and atelectasis

Page 20: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

20

First-in-infant Phase I/IIa study

Anti-viral effect – time to undetectable virus in culture

Pro

po

rtio

n o

f p

atie

nts

with

de

tecta

ble

RS

V

All Placebo (N=13)

All ALX-0171 (N=22)

Time (hours)

Treatment with ALX-0171 had an immediate and significant impact on viral replication in RSV-infected infants

Cox model to compare ALX-0171 and placebo with respect to time to first undetectable virus in culture (undetectable at 2

consecutive time points) from time of start of treatment

Time to undetectable virus ALX-0171 Placebo

Median hours 25.3 51.9

Nominal p-value 0.014

Page 21: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

21

First-in-infant Phase I/IIa study

Overall disease severity assessment – Global Severity Score*

Encouraging initial indication of therapeutic effect

* Overall disease severity assessment including feeding intolerance, medical intervention, respiratory difficulty, respiratory frequency, apnoea, general

condition and fever

Nominal p-value=0.0092

based on longitudinal analysis comparing ALX-0171

and placebo with respect to the Global Severity

Score, adjusting for baseline score and time

All ALX-0171 (N=26)

All Placebo (N=15)

Glo

ba

l S

eve

rity

Sco

re (

me

an

+ S

E)

Page 22: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

22

Inhaled ALX-0171

Potential breakthrough for the treatment of RSV infections

22

µ$

µµ

µ

µ

µµ

• Direct delivery to the site of infection through inhalation

• Good safety profile and no treatment-related SAEs in hospitalised

RSV infected infants

• Demonstrated anti-viral-effect in pre-clinical and clinical studies

• Encouraging indication of therapeutic effect in the clinic

Phase II dose-ranging efficacy study in ~180 hospitalised RSV-infected infants planned to start in Q4 2016

Page 23: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

23

Caplacizumab – anti-vWF Nanobody

First-in-class potential for treatment of aTTP

• Orphan Drug Status and patent protection to

2035

• Phase III ongoing with results expected by

end of 2017

• Planned filing for conditional approval in

Europe (Q1 2017) and BLA submission in

USA (2018)

• Ablynx to lead commercialisation in Europe

and the US

• Anticipated first launch in 2018 with peak

sales potential of ~€300M1

1 US, EU, Japan and other major markets

aTTP: acquired thrombotic thrombocytopenic purpura

Page 24: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

24

Caplacizumab

2016 achievements

• Publication of the Phase II TITAN study in patients with acquired TTP in The

New England Journal of Medicine

• Recruitment in HERCULES Phase III proceeding well with target enrolment

increased to approximately 130 patients (from 92 originally planned) with

topline results still expected before the end of 2017

• Preparations on-track to file for conditional approval of caplacizumab in

Europe in Q1 2017

Page 25: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

Outlook confirmed

Potential value enhancing events

Page 26: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

26

2016-2017 outlook confirmed

Focus on sustainable value creation

2016 2017

• Q1 2016

- Phase I start with VEGF/Ang2 (BI) - €8M

- publication of data from TITAN study in the NEJM

• Q2 2016

- Phase I start with CX3CR1 (BI) - €8M

- Phase I start with CXCR2 (Novartis)

- Phase I/IIa results with ALX-0171 (53 infants)

- successfully raised €74M in a Private Placement

• Q3 2016

- vobariluzumab PhIIb RA monotherapy study results

- vobariluzumab PhIIb RA combination study results

- start follow-up study with HERCULES patients

• Q4 2016

- opt-in decision by AbbVie for ALX-0061 in RA

- start Phase II dose-ranging efficacy study of ALX-0171

- pre-clinical milestones

• Caplacizumab (aTTP) – wholly-owned

- filing for conditional approval in Europe (H1)

- HERCULES Phase III study results (H2)

- life cycle management activities

• Vobarilizumab (RA) – preparations for

Phase III study initiation

• ALX-0171 (RSV) – wholly-owned

- life cycle management activities

• Immuno-oncology – with Merck & Co., Inc.

- start of multiple IND enabling studies

- pre-clinical milestones

• Various partnered programmes

advancing in the clinic

Page 27: 2016 half-year results and business update · 2018. 9. 13. · Webcast presentation – 25 August 2016 2016 half-year results and business update . 2 Ablynx 2016 half-year results

CONTACT DETAILS

Questions

+32 9 262 00 00 Investor

Relations

investors@

ablynx.com www.ablynx.com