CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY...

35
CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019

Transcript of CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY...

Page 1: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

CSTONE PHARMACEUTICALS (2616.HK)

COMPANY PRESENTATION

November, 2019

Page 2: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

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Presentation Disclaimer

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

The information in this presentation has been prepared by representatives of CStone Pharmaceuticals (the "Company" and, together with its subsidiaries, the "Group") for

use in presentations by the Group for information purpose. No part of this presentation will form the basis of, or be relied on in connection with, any contract or commitment

or investment decision.

Certain statements contained in this presentation and in the accompanying oral presentation, may constitute forward-looking statements. Examples of such forward-looking

statements include those regarding investigational drug candidates and clinical trials and the status and related results thereto, as well as those regarding continuing and

further development and commercialization efforts and transactions with third parties. Such statements, based as they are on the current analysis and expectations of

management, inherently involve numerous risks and uncertainties, known and unknown, many of which are beyond the Company’s control. Such risks include but are not

limited to: the impact of general economic conditions, general conditions in the pharmaceutical industry, changes in the global and regional regulatory environment in the

jurisdictions in which the Company’s does business, market volatility, fluctuations in costs and changes to the competitive environment. Consequently, actual future results

may differ materially from the anticipated results expressed in the forward-looking statements. In the case of forward-looking statements regarding investigational drug

candidates and continuing further development efforts, specific risks which could cause actual results to differ materially from the Company’s current analysis and

expectations include: failure to demonstrate the safety, tolerability and efficacy of the Company’s drug candidates, final and quality controlled verification of data and the

related analyses, the expense and uncertainty of obtaining regulatory approval, the possibility of having to conduct additional clinical trials and the Company’s reliance on

third parties to conduct drug development, manufacturing and other services. Further, even if regulatory approval is obtained, pharmaceutical products are generally

subject to stringent on-going governmental regulation, challenges in gaining market acceptance and competition. These statements are also subject to a number of material

risks and uncertainties that are described in the Company’s prospectus published onto the websites of the Company and The Stock Exchange of Hong Kong Limited and

the announcements and other disclosures we make from time to time. The reader should not place undue reliance on any forward-looking statements included in this

presentation or in the accompanying oral presentation. These statements speak only as of the date made, and the Company is under no obligation and disavows any

obligation to update or revise such statements as a result of any event, circumstances or otherwise, unless required by applicable legislation or regulation.

Forward-looking statements are sometimes identified by the use of forward-looking terminology such as "believe," "expects," "may," "will," "could," "should," "shall," "risk,"

"intends," "estimates," "plans," "predicts," "continues," "assumes," "positioned" or "anticipates" or the negative thereof, other variations thereon or comparable terminology or

by discussions of strategy, plans, objectives, goals, future events or intentions.

No representation or warranty, express or implied, is 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, revision, verification and amendment and such information may

change materially.

This presentation and the information contained herein is highly confidential and being furnished to you solely for your information and may not be reproduced or

redistributed in any manner to any other person, in whole or in part. In particular, neither the information contained in this presentation nor any copy hereof may be, directly

or indirectly, taken or transmitted into or distributed in any jurisdiction which prohibits the same except in compliance with applicable securities laws. This presentation and

the accompanying oral presentation contains data and information obtained from third-party studies and internal company analysis of such data and information. We have

not independently verified the data and information obtained from these sources.

By attending this presentation, you acknowledge that you will be solely responsible for your own assessment of the market and the market position of the Group and that

you will conduct your own analysis and be solely responsible for forming your own view of the potential future performance of the business of the Group.

Page 3: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

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Industry-leading

Management Team

Integrated

Biopharma with Clear

Focus on Clinical

Development

$150M

Series A

$262M

Series B

(July 2016) (May 2018)

$328M

HK IPO

(Feb 2019)

Well-balanced

Oncology Portfolio with a Focus

on Immuno-oncology and

Precision Medicine

Bring Innovative Oncology Therapies to Cancer Patients Worldwide

3+ Years Since Company

Inception

HKEx listed

2616.HK

Page 4: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

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Industry Leading Management Team with Proven Track

Record and Complementary Expertise

Frank Jiang, MD, PhDChairman, Chief Executive Officer

Jason Yang, MD, PhD

Chief Medical OfficerRichard Yeh, MBA

Chief Financial OfficerBing Yuan, PhD, MBA

Chief Business Officer

Archie Tse, MD, PhD

Chief Translational Medicine

Officer

Jon Wang, PhD

Chief Scientific Officer

Jingrong Li, PhD

SVP, Product Development

& Manufacturing

Sanhu Wang

SVP, Government Affairs

Yinghua Zhang

VP, Operations

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Distinguished World-Class Scientific Advisory Board

With Deep Oncology and IO Expertise

Note: ASCO = American Society of Clinical Oncology; AACR = American Association for Cancer Research.

Paul Bunn

MD

Weiping Zou

MD, PhD

Richard Finn

MD

Former AACR President

2018-2019

Professor of Oncology,

Johns Hopkins

University

Chair, AACR Cancer

Immunology

Charles B.de Nancrede

Professor,

University of Michigan

Former International

Liver Cancer

Association President

Clinical Professor,

UCLA

Elizabeth Jaffee

MD

Former ASCO President

2002-2003

Distinguished Professor,

University of Colorado

Page 6: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

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Well-balanced Oncology Portfolio with a Focus on

Immuno-oncology and Precision Medicine

Drug

Candidate

Molecular

Target/

Signaling

Pathway

Lead

Indication(s)

and Line(s) of

Therapies

Drug

Candidate Category

Commercial

RightsPartner Pre-clinical

IND

Filing

Dose

EscalationPOC Pivotal NDA

CS1001

(Core Product)PD-L1

R/R cHL, R/R NKTL,

NSCLC2, Solid

tumors3

Biologics, 1 Worldwide

CS10031 PD-1 HCC, Solid tumors3 Biologics, 1 Worldwide

Ivosidenib IDH1R/R AML, 1L AML,

Cholangiocarcinoma

Chemicals, 1

(MRCT for AGILE);

Chemicals, 5.1

(IND for R/R AML)

Greater China

AvapritinibKIT &

PDGFRα

PDGFRα/ 2L / 3L

GIST,

AdvSM, ISM

Chemicals, 1 Greater China

Pralsetinib RET1L / 2L NSCLC,

1L MTC4 Chemicals, 1 Greater China

Fisogatinib FGFR4 1L / 2L HCC Chemicals, 1 Greater China

CS10021 CTLA-4 Solid tumors3 Biologics, 2 Worldwide

CS30061 MEK Solid tumors3 Chemicals, 1 Worldwide

CS3003 HDAC6Solid tumors3,

R/R MM5 Chemicals, 1 Worldwide

CS3002 CDK4/6 Solid tumors3 Chemicals, 1 Worldwide

ND021PD-L1/4-

1BB/HSASolid tumors3 Biologics, 1

Greater China,

South Korea,

Singapore

CS3004 Worldwide

CS1009 Worldwide

CS3005 Worldwide

CS2004 Worldwide

Source: Company

1 Denotes we currently have clinical trials ongoing in Australia for the product candidate. 2 Line of therapies include 1L Stage IV NSCLC and consolidation therapy after chemoradiotherapy for Stage III NSCLC. 3 Because there are no clinical

efficacy data on the drug candidate, no specific types of solid tumors are established as lead indications at this stage. 4 The clinical data published so far by Blueprint demonstrated that pralsetinib is effective in the treatment of certain NSCLC

and MTC patients. 5 Available clinical data from other HDAC6 inhibitor studies provides the basis to suggest that CS3003 may be effective in treating MM; we are considering to evaluate the clinical efficacy of CS3003 in MM and various types

of solid tumors in the Phase Ib dose expansion.

Note: AML= Acute Myeloid Leukemia, AdvSM = Advanced Systemic Mastocytosis, cHL= Classical Hodgkin’s Lymphoma, GIST = Gastrointestinal Stromal Tumor, HCC = Hepatocellular Carcinoma, ISM = Indolent Systemic Mastocytosis,

NKTL = Natural KILLER/T Cell Lymphoma, NSCLC = Non-small Cell Lung Cancer, MTC = Medullary Thyroid Cancer, R/R = Relapsed or Refractory, SM = Systemic Mastocytosis, MM = Multiple Myeloma.

Pre

-cli

nic

al

Late

-sta

ge

Undisclosed

US FDA Approved (Agios)

China Status

China Status

Rest of the World Status

China Status

Rest of the World Status

Rest of the World Status

China Status

Rest of the World Status

China Status

Rest of the World Status

China Status

China Status

NDA submission in Taiwan

NDA submission in the US and EU accepted(Blueprint)

Cli

nic

al/

IND

China Status

Rest of the World Status

China Status

China Status

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Focus on China’s largest indications, covering 55%+ of

total cancer incidences

PD-L1 Mono

Stage III NSCLC

Lung Cancer

774,323

Gastric Cancer Liver CancerEsophagus

Cancer

456,124 392,868 307,359

PD-L1 + Chemo

Stage IV NSCLC

PD-L1 + Chemo

Advanced GC/GEJ

PD-L1+Regorafenib

Advanced GC/GEJ

PD-1 + VEGFRi

Advanced HCC

PD-L1 + Fisogatinib

FGF19+ HCC

PD-L1 + Chemo

Advanced ESCC

Source: Globocan 2018

Note: NSCLC = non-small cell lung cancer; GC = gastric adenocarcinoma; GEJ = gastro-esophageal junction adenocarcinoma; HCC = hepatocellular

carcinoma; ESCC = esophageal squamous cell carcinoma; CRC = colorectal cancer; VEGFRi = inhibitor of vascular endothelial growth factor receptor

Cancer type

New cases

(2018)

5-year prevalence

(2018) 716,411 603,851 296,780 284,163

Ongoing and

planned

clinical trials

Colorectal

Cancer

516,859

1,237,145

PD-L1+Regorafenib

Advanced CRC

Pralsetinib

RETm NSCLC

registrational

Fisogatinib

FGF19+ HCC

PD-1+Regorafenib

Advanced CRC

PD-1+Regorafenib

Advanced GC/GEJ

exploratory

Page 8: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

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Large Scale and Right Mix of Pipelines to Drive Success

in IO Combo Therapy

Only company in China which owns

clinical stage PD-L1, PD-1 and CTLA-4

Pralsetinib

(RET)

CS3002

(CDK4/6)

CS3003

(HDAC6)

CS1009

CS3005

Avapritinib(KIT&PDGFRα)

Fisogatinib (FGFR4)

CS3004

CS2004

PD-L1

CTLA-4PD-1

ND-021

(PD-L1x4-1BB)

Ou

tlo

ok

Str

ate

gy

Combination therapy trials

10 by year-end

PD-(L)1 + Regorafenib

PD-(L)1 + chemo/radiation

De-risked Combo

PD-(L)1 + CDK4/6

PD-L1 + Fisogatinib

PD-(L)1 + IvosidenibPD-(L)1 + CTLA4

Novel Combo

Unique to CStone

Ivosidenib(IDH1)

CS3006

(MEK)

Combination

potentialCost control Flexibility

More… More…

Page 9: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

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Significant Clinical Progress – OverviewLeveraging unparalleled clinical development engine

FY2019

2 years from first trial (2017 Oct)

28 clinical trials by year end, of which 10 are PD-(L)1 combo trials

Including 13 registrational studies

PD-L1: 6 registrational studies including several in large

indications

PD-1: 1 registrational study

Licensed-in assets: 6 registrational studies

Page 10: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

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Significant Clinical Progress (1/11) CS1001 (PD-L1): 6 Registrational Trials Ongoing with More Combos

in Large Indications Planned

Upcoming Development Plan

New trials by year-end 2019:

Ph I trial of CS1001+fisogatinib (FGFR4)

for HCC in China

CS1001 + Regorafenib in CRC

Ph Ib trial of CS1001+PARP inhibitor for

solid tumors globally

New trials planned in 2020:

Regorafenib + CS1001 for GC,HCC,

GBM and BTC in AUS, CN, HK & TW

Donafenib + CS1001

More…

Indication

Mono-

/Combo-

Therapy

Phase Location

Expected

trial

completion

date (1)

REGISTRATIONAL

Stage III

NSCLC Mono III China 2020

Stage IV

NSCLC

Combo (with

SoC)III China 2020

Gastric

cancer

Combo (with

SoC)III China 2021

Esophageal

Cancer

Combo (with

SoC)III China 2022

cHL Mono II China 2019

NKTL Mono II China 2020

EARLY PHASE AND EXPLORATORY

Solid tumors Mono I U.S.

Solid tumors

and

lymphoma

Mono Ib China

Ongoing Clinical Trials (900+ patients dosed

by October 17, 2019)

1. Denotes the date on which the last patient is enrolled

Page 11: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

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A total of 29 patients

Well tolerated and demonstrated good safety

profile

— Low infusion-related reaction

— No DLT observed

— No Treatment-related SAE

— MTD not reached

— Comparable AE profile

Efficacy comparable to similar drugs

— 24% ORR, 7 confirmed PRs

PK profile showed concentration proportional

to dosage, with T½ of around 2 weeks

— ADA positive rate was 24%

Designed with distinct characteristics:

fully human with potentially less ADA and

toxicity

Potentially CStone’s first self-developed drug

to be approved in China

Potentially the first domestic anti-PD-L1 to be

launched in China

Overview

Ph Ia

Highlight

Source: Company, Ph1a data presented at 2019 ASCO

Note: PR = partial response; PK = Pharmacokinetic; ADA = anti-drug antibody; DLT = dose-limiting toxicity; SAE = serious adverse event; MTD = maximum tolerated dose; BOR = best overall response;

SLD = sum of longest diameters

Significant Clinical Progress (2/11)China’s First Fully Human, Full-length IgG4 PD-L1 mAb: Safe and

Efficacious in Early Clinical Trial

v

7/29 (24.1%) patients achieved a partial response (PR) as

assessed by investigators per RECIST 1.1

9/29 (31.0%) patients remained on study treatment, with a

median duration of treatment being 126 days (range, 21 to 408+)

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Significant Clinical Progress (3/11)Safe and Efficacious in ESCC as Combo with Chemotherapy (Ph Ib)

Source: Company, Ph1b data presented at 2019 CSCO

Note: ORR = Overall Response Rate; DOR = Duration Of Response;

Esophageal Squamous Cell Carcinoma:

ORR=77.8% (14/18), DCR=88.9% (16/18)

77.8

46.7

33.3

80

0

20

40

60

80

100

OR

R (

%)

CS1001 Tislelizumab

Durvalumab+

Tremelimum

ab

Camrelizumab

+

Apatinib

Class PD-L1 PD-1 PD-L1 PD-1

n 23 15 6 30

ECOG0: 21.3%

1: 78.3%

0: 26.7%

1: 73.3%

0: 0%

1: 100%

0: 83.3%

1: 16.7%

Chemo

RegimenCF CF CF

liposomal

paclitaxel+

nedaplatin

DOR (m)NR

(0.03+~8.4+)12.8 Not reported Not reported

Source CSCO 2019 CSCO 2019ASCO GI

2019ASCO 2019

Page 13: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

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Significant Clinical Progress (4/11)Safe and Efficacious in GC/GEJ as Combo with Chemotherapy (Ph Ib)

Source: Company, Ph1b data presented at 2019 CSCO

Note: ORR = Overall Response Rate; DOR = Duration Of Response;

Gastric Cancer/Gastro-Esophageal Junction Cancer:

ORR=62.1% (18/29), DCR=82.8% (24/29)

62.1

48.6

6068.4 66.7

85

0

20

40

60

80

100

OR

R (

%)

CS1001Keytruda

(062)

Keytruda

(059)Opdivo

Camrelizu

mabSintilimab

Class PD-L1 PD-1 PD-1 PD-1 PD-1 PD-1

n 21 257 vs 250* 25 38 48 20

ECOG0: 41.4%

1: 58.6%

0: 46.0%

1: 54.0%

0: 60.0%

1: 40.0%

0: 50.0%

1: 50.0%

0: 41.7%

1: 58.3%

0: 45.0%

1: 55.0%

Chemo

RegimenXELOX

Cisplatin + 5-

FU or

Capecitabine

Cisplatin + 5-

FU or

Capecitabine

SOX or

XELOXXELOX XELOX

DOR (m)

6.2

(0.03+ ~

6.21+)

6.8

4.6

(2.6 ~

20.3+)

9.9

(5.8, NR)NR

5.3

(4.8~7.2)

SourceCSCO

2019ASCO 2019 ESMO 2017

Ann Oncol.

2019 Feb

1;30(2):250-

258

ASCO

2019ASCO 2019

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Significant Clinical Progress (5/11)Safe and Efficacious in CC/GBC as Monotherapy (Ph Ib)

Source: Company, Ph1b data presented at 2019 CSCO

Note: ORR = Overall Response Rate; DOR = Duration Of Response;

Cholangiocarcinoma/Gallbladder Cancer:

ORR=10.3% (3/29), DCR=37.9% (11/29)

10.3

4.7

13

5.8

11.1

3.3

22

0

5

10

15

20

25

OR

R (

%)

CS1001Imfinzi

Asia

Keytruda

(PD-

L1>=1%)

Keytruda

(global)

Keytruda

(PD-

L1>=1%)

Korean

Opdivo

Japan

Opdivo

US

Class PD-L1 PD-L1 PD-1 PD-1 PD-1 PD-1 PD-1

n 29 42 24 104 39 30 54

ECOG

0: 14(48.3)

1: 13(44.8)

NA: 2 (6.9)

0: 27(64)

1: 15(36)

0: 9(37.5)

1: 15(62.5)

0: 42(40.4)

1: 62(59.6)

Not

reported

Not

reported

Not

reported

Regime

n

1200mg IV

Q3W

10 mg/kg

Q2W

10mg/kg

IV Q2W

200mg IV

Q3W

200mg IV

Q3W

Mono:

nivolumab

240mg

Q2W

240mg

Q2W for

16 weeks

then

480mg

Q4W

DOR (m)

5.39

(1.91+,8.0

2)

9.7

NR

(21.5 -

29.4+)

NR

(6.2-23.2+)

Not

reported

Not

reported

Not

reported

SourceCSCO

2019

JCO

2019

ASCO

2019

ASCO

2019JCO 2019

Lancet

2019 JCO 2019

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Significant Clinical Progress (6/11)Safe and Efficacious in MSI-H/dMMR Cancer as Monotherapy (Ph Ib)

Source: Company, Ph1b data presented at 2019 CSCO

Note: ORR = Overall Response Rate; DOR = Duration Of Response;

MSI-H/dMMR Cancer:

ORR=38.1% (8/21), DCR=57.1% (12/21)

38.1

22.6

39.6

31.1

19

0

10

20

30

40

50

OR

R (

%)

CS1001 Imfinzi Keytruda OpdivoTislelizuma

b

Class PD-L1 PD-L1 PD-1 PD-1 PD-1

n 21 62 149 74 16

ECOG0: 0

1: 100%Not reported

0:36%

1:64%

0:43%

1:57%Not reported

Regime

n

1200mg iv

q3w

10mg/kg

q2w

200mg q3w

/10mg/kg

q2w

3mg/kg q2w 200mg q3w

DOR (m)NR

(0.03+, 8.6+)Not reported

NR

(1.6+, 22.7+)NR NR

Source CSCO 2019ASCO GI

2019PI, 05/2017

Lancet

Oncol 2017

CSCO

2019

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Source: Company, CS1001-201 Abstract #2833 in 2019 American Society of Hematology (ASH) Annual Meeting

Data cut-off 17th June 2019.

Updated data with more details will be presented at the upcoming ASH in Dec 2019

Note: rr-ENKTL=relapsed or refractory extranodal natural killer (NK)/T-cell lymphoma; rr-PTCL=relapsed or refractory peripheral T cell lymphoma, ENKTL is one

of PTCL subtype; ORR = Overall Response Rate; CR: Complete Remission; PR: Partial Remission; DOR = Duration Of Response; HSCT: Hematopoietic Stem

Cell Transplantation[1] Li CC, et al. Cancer. 2004 Jan 15;100(2):366-75; [2] Zhang L, et al. Cancer Med. 2016 Jan;5(1):33-40; [3] HJ Kim, et al. Bone Marrow Transplantation; [4] Shi, Y., et al. Ann Oncol, 2015; [5] Shi, Y., et al. J Hematol Oncol, 2017;

rr-ENKTL:

CR=31.8% (7/22), ORR=40.9% (9/22)

31.8

7.1 6.3 6.1

0

10

20

30

40

CR

(%

)

CS1001 SintilimabChidamide

(Approved in rr-PTCL, China, 2014)

Class PD-L1 PD-1 HDAC

Study CS1001-201 ORIENT-4Registration

Study

Real World

Study

n 22 28 16 33

CR 31.8% (7/22) 7.1% (2/28) 6.3% (1/16) 6.1% (2/33)

DOR

(m)

NR

(0.03+~8.61+)

4.1

(0+,4.2+)UNK UNK

Source ASH 2019 ASCO 2019Ann Oncol,

2015[4]

J Hematol

Oncol, 2017[5]

Preliminary efficacy dataCS1001 demonstrated promising antitumor activity with

a high CR rate and durable response in rr-ENKTL

patients

Among the 22 efficacy-evaluable patients, the

investigator-assessed ORR was 40.9%

7 (31.8%) patients achieved complete response

The duration of response (DOR) ranged from 0.03+

to 8.61+ months, and the median DOR was not

reached

8 additional pts reached response assessment time

point, the updated data will be reported as poster

presentation at the 2019 ASH conference

CR is more clinically meaningful for r/r ENKTL

• Patients with CR have long duration of response

while patients with PR generally progress quickly[1,2].

• High CR rate translates to potential allogeneic

HSCT and curation of the disease [3].

Significant Clinical Progress (7/11) Efficacious in rr-ENKTL as Monotherapy (PII Registrational)

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Significant Clinical Progress (8/11)Four Innovative Precision Medicines: Global Development and

Regulatory Status

Note: AML= Acute Myeloid Leukemia, Non-IC= Non-eligible for intensive chemotherapy; SM = Systemic Mastocytosis, GIST = Gastrointestinal Stromal Tumor,

HCC = Hepatocellular Carcinoma, NSCLC = Non-small Cell Lung Cancer, MTC = Medullary Thyroid Cancer

* Non-pivotal trials

Drug

CandidateTarget Indications

Pivotal / Registrational Trial

Patient Enrollment

NDA

Submission

NDA

Approval

Ivosidenib

(CS3010)IDH1

R/R AML

1L AML

monotherapy

1L Non-IC

combo with AZA

2/3L

Cholangiocarcinoma

monotherapy

Avapritinib

(CS3007)KIT & PDGFRα

PDGFRα D842

GIST, 4L GIST

3L GIST

2L GIST

AdvSM, ISM

Pralsetinib

(CS3009)RET

2L NSCLC

1L NSCLC

2L MTC

Fisogatinib*

(CS3008)FGFR4

1L / 2L

FGF19 (+) HCC

Achieved To be achieved in 2020

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Significant Clinical Progress (9/11)Four Innovative Precision Medicines: Greater China Development

and Regulatory Status

Drug

CandidateIndications Region

Mono

/ComboStudy Initiation Patient Enrollment NDA Submission NDA Approval

Ivosidenib

(CS3010)

IDH1m R/R AML Taiwan Mono

IDH1m 1L AML

(AGILE)China Mono

IDH1m R/R AML China Mono

Avapritinib

(CS3007)

PDGFRα D842V

GISTTaiwan Mono

GIST 3L China Mono

PDGFRα D842V

GISTChina Mono

GIST 2L China Mono

Adv SM China Mono Exploring trial waiver

Pralsetinib

(CS3009)

NSCLC 2L China Mono

MTC 1L China Mono

NSCLC 1L China Mono

Multiple tumors China Mono

Fisogatinib

(CS3008)

Tki Naïve HCC China Mono

HCC China Combo

Joining global study

Registration with US NDA data

Bridging study

Joining global study

Joining global study

Joining global study

China study

Joining global study

Joining global study

Joining global study

Registrational Study

Registration with US NDA data

Bridging study

Joining global study

Non-Registrational Study NDA submission in 2020

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19

Best

Change i

n T

arg

et

Lesio

n

from

Baselin

e (

%)

PRSDPD

CS1003 200 mg CS1003 60 mg

1 2 3 4 5 6 7 8 9 10 11 12 13

Str

ate

gic

valu

e

■ A full-length, fully human IgG1 mAb against CTLA4

■ Amino acid sequence identical to ipilimumab (Yervoy)

Ph

1 D

ata

Hig

hli

gh

ts

■ CS1002 monotherapy was well tolerated up to 10 mg/kg

Q3W, with no DLT and no MTD reached (N=13)

■ CS1002 demonstrated dose-proportional PK profile with

T1/2 of 12~15 days; increase in absolute lymphocyte count

(ALC) observed indicating target engagement

■ Overall clinical profile is consistent with that of ipilimumab

Asset

ov

erv

iew

CS1002

Significant Clinical Progress (10/11)Two Additional I/O Backbones: CS1002 (CTLA-4) and CS1003 (PD-1)

CS1003

■ Potentially become another outstanding CTLA-4 inhibitor

after Ipilimumab, which has not been launched in China

■ One of 3 I/O backbones to enable flexible combo strategy,

including chemo-free I/O-I/O combo with CS1003

■ Humanized IgG4 anti-PD-1 mAb

■ Recognize both human & murine PD-1 with unique

advantage to evaluate efficacy in syngeneic mouse

models, esp. for testing combinations with small molecules

■ Bridging Ph1 conducted in China showed that CS1003

monotherapy was safe and tolerable at 60mg and 200mg

Q3W; no DLT or MTD was observed (N=19)

■ Dose proportional increase in systemic exposure to

CS1003; low immunogenicity of CS1003 without treatment-

induced/-enhanced ADA positivity

■ Preliminary anti-tumor activity of CS1003 observed in

multiple tumor types (PR 3/16 (18.8%))

■ One of 3 I/O backbones with current safety and efficacy

data support further clinical development of CS1003

■ Initiating combo studies with CS1002, and VEGF TKI,

including registration trial in 1L HCC

1 mg/kg

(N=6)3 mg/kg

(N=3)

10 mg/kg

(N=4)10 mg/kg

CS1002 Ipilimumab

CS1002 induced early ALC increase, similarly to Ipilimumab Preliminary efficacy in multiple tumor types

DLT: dose-limiting toxicity; MTD: maximum tolerated dose; Q3W: once every 3 weeks; PK: pharmacokinetics; ADA: anti-drug antibody; PR: partial response; VEGF: vascular endothelial growth factor; TKI:

tyrosine kinase inhibitor; HCC: hepatocellular carcinoma

1=Squamous cell carcinoma of the cervix; 2=Synovial sarcoma; 3=Hepatocellular carcinoma;4=Gastric adenocarcinoma; 5=Leiomyosarcoma; 6=Malignant melanoma; 7= Leiomyosarcoma; 8= Fibromatosis;

9= Follicular dendritic cell sarcoma; 10=Uterine Leiomyosarcoma; 11= Laryngeal squamous cell carcinoma; 12= Oesophageal squamous cell carcinoma; 13= Oesophageal squamous cell carcinoma.

18.8%25.0%37.5%

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20

Significant Clinical Progress (11/11)Progress on Other Clinical-Stage Candidates

CS3002

(CDK4/6)

Plan to conduct a Ph I trial for solid tumors as a mono by year-end 2019

and subsequently combo with CS1003 (PD-1) in Australia and/or China

CS3003

(HDAC6)

IND/CTA approvals received in China and Australia in March 2019 and

April 2019 respectively

CS3006

(MEK)

Conducting a Ph I trial in Australia and expect to complete the dose

escalation portion in 1H20

Conducting a Ph I trial as a mono for advanced solid tumors in China

and expect to complete the dose escalation portion by year-end 2019

and initiate a dose expansion portion to confirm RP2D in 2020

Note: CSCO = Chinese Society of Clinical Oncology; RP2D = recommended phase 2 dose

Page 21: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

21

Pipeline 2.0 for Sustained Growth of CStone

Advanced

Portfolio

Precision Medicine

De-risked

I/O Backbone & Assets

GAP Analysis Pipeline 2.0

New

Targets

Pipeline 1.0

Balanced

Portfolio

Convincing combo activity

with PD-(L)1 or pipeline 1.0

candidates?

“Practice changing” potential? FIC/FW or BIC multispecific

mAbs/scaffolds

TME modulators to

maximize PD-(L)1 efficacy

Cancer vaccines

Novel pathway inhibitors“Disruptive” biology?Validated & Novel

I/O Combination

External

Partnerships

Internal

Development

Dual Sources of Innovation

R&D / Partnership

MeetingsDatabase

KOL

ConnectionsLiterature Academia Network

Note: FIC = first in class; FW = first wave; BIC = best in class; TME = tumor microenvironment.

Page 22: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

22

Potentially Best-in-class PD-L1/HSA/4-1BB tri-specific antibody-based molecule

Designed to significantly broaden safety window and higher efficacy vs current PD-(L)1

therapies

Validated dosing and longer half-life enable convenient dosing schedules vs competing

molecules in the same class

ND021 tethers 4-1BB and activates T cells only upon engagement

of PDL1-expressing tumor cells & overcomes 4-1BB mAb toxicity

Monovalent

without Fc

Highlights

Strategic Value

Access to Numab’s novel multi-specific technology platform

Complement to CStone’s IO strategy

One Step Towards Pipeline 2.0 – A Potential Next

Generation PD-(L)1 Therapy

Page 23: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

23

… More to come

Avapritinib (KIT & PDGFRα)

Globally first-in-class

Pralsetinib (RET)

Fisogatinib (FGFR4)

Enrich CStone oncology pipeline

Enable combo development

Access to fast-growing but

complex China market

Global quality and compliance

standard

Partner-driven Innovation

Engine Validated by

High-profile Partnerships

Ivosidenib (IDH1)

FDA approved

Globally first-in-class

Crown Jewel Assets from

World Class Partners

Clinical Collaborations Focused

on IO Combo Therapy

ND021

Next generation IO

Potential best-in-class

Partnering with Domestic and Global Leading

Biopharmaceutical Companies

… More to come

CS1001 (PD-L1) + Donafenib

Over 1,000 patients with advanced

malignancies having received

treatment with Donafenib

CS1001 (PD-L1) + Regorafenib

Global collaboration with China focus

Treatment for gastric cancer and

other serious malignancies

CS1001 (PD-L1) + IMP4297

Potential best-in-class

Target cancers with BRCA mutation

or DNA repair deficiency

Page 24: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

24

Global Collaboration Deal with China Focus to Evaluate

PD-L1 in Combination with Regorafenib in Key Indications

PD-L1

First collaboration with a MNC

pharma, one of the very few without

PD-(L)1 – a vote of confidence in

CStone and CS1001 (PD-L1)

Regorafenib reported promising data with PD-1 in

gastric cancer and colorectal cancer at ASCO 2019

Indication Mono/Combo ORR

Advanced GCPembro 13%1

Rego + Nivo 44%2

pMMR/MSS

CRC

(95% of

mCRC)

Pembro 0%3

Rego 2%4

Atezo 2%4

Atezo + MEK 3%4

Rego + Nivo 33%2

Note: 1. KEYNOTE059; 2. 2019 ASCO data, All respondents were

Microsatellite stable (MSS); 3. 2015 ASCO data; 4. IMBlaze370

pMMR = mismatch repair proficient

Further strengthens our core

strategy in IO combination therapy

A big step forward for CStone’s

global strategy in case of positive

data

Hig

hlig

hts

Str

ate

gic

Valu

e

Page 25: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

25

Ramp up Commercial Capabilities to Sync Up with

Multiple Product Launches across Indications in the Next

Few Years

▪ Establish commercial

organization with core

functions for Greater China

▪ Focus on self-build

commercial model while

exploring potential value-driving

strategic partnership

▪ Full launch of PD-L1

and in-licensed assets

across multiple major

indications

▪ Scale up Greater

China commercial

presence

Launch &

commercialization

Build and scale up commercial capabilities (Sales & Marketing; Access; Medical Affairs; Distribution;

Commercial Excellence etc.) to support China and global launches, ramped up 18-24 months in advance of

each launch

1 2 3

Commercial model

Stage 2

Stage 1 2018-2021

2021-2023

Stage 3 2023+

▪ First wave of product

launches outside China1,

preferably with partner

▪ Scale up global commercial

organization to support pre-

launch planning and launch

Core Commercial Organization

Already Established

MarketingSales

Management

Commercial

Excellence

Medical Affairs

GAMASales

Commercial

Excellence

Medical

Affairs

GAMA

Marketing

Full-fledged Commercial Organization

By 4Q 2023

Page 26: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

26

Developing Strong Manufacturing Capabilities for Both

Biologics and Small Molecules

Small Molecules Biologics

Compliance with GMP requirements

in China and globally

Planned capacity of biologics plant design:

26,000L for biologics and 1 billion tablets for small molecule

In August, we entered into an agreement with

Sungent (state-owned controlled by Suzhou

Industrial Park) to build manufacturing facility

Planned building area of approximately

100,000 sqm

Commissioned to a third party and is

scheduled to break ground in 2020

Capability: once completed, the complex will

be equipped with integrated capabilities for

R&D, Pilot Plant, and full commercial scale

manufacturing

Planned capacity: 26,000L for

macromolecule biologics and 1 billion tablets

and capsules for small molecule drugs

Strategic partnership with Wuxi Biologics

on clinical and commercial stage

manufacturing

Better Quality Control

DriveDownCost

Protection of Key Know-how

Compliance Monitoring

Efficient Planning

Long-term Stable Supply

Value Creation

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27

27

Series B

$150 Million

$262 Million

Series A

Record

Breaking1

Record

Breaking1

2016/07 2018/05 2019/02

HKEX

IPO

$328 Million2

Other global

leading institutional

investors

Cornerstone

investors

Successfully Listed on HKEx on February 26, 2019

Note:1At the time of the deal; 2Post-shoe

Page 28: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

28

Upcoming

data

publication

Note: NKTL = Natural Killer T Cell Lymphoma, ASH = American Society of Hematology, ASCO = American Society of Clinical Oncology, NSCLC =

Non-small cell lung cancer, GIST = Gastrointestinal Stromal Tumor, MTC = Medullary Thyroid Cancer

Upcoming Major Data Release Plan

NKTL registrational trial data at ASH, Orlando, Dec 7

to Dec 10, 2019

Stage IV NSCLC squamous and non-squamous Ib

trial data at ASCO 2020

Top-line

data by

2020

Stage IV NSCLC registrational trial data by Q3 2020

Stage III NSCLC registrational trial data by Q4 2020 /

Q1 2021

3L GIST registrational trial data by Q2 2020

GIST with PDGFRa D842V registrational trial data by

Q3 2020

2L NSCLC registrational trial data by Q3 2020

1L MTC registrational trial data by Q4 2020

PD-L1

PD-L1

Avapritinib

Pralsetinib

Page 29: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

Thank you!

Page 30: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

Appendix

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31

Stage III

NSCLC

Stage IV

NSCLC

GC

HCC

EC

cHLNKTL

Peak Sales

USD

700M

Total China PD-(L)1 Market Estimated at USD 5-10B:

CS1001 expected to reach peak annual revenue of

USD 700M in current indications

Source: CStone analysis

Note: NKTL = Natural killer/T-cell lymphoma; cHL = classical Hodgkin’s lymphoma; NSCLC = non-small cell lung cancer; HCC = hepatocellular carcinoma; GC = gastric cancer

Page 32: CSTONE PHARMACEUTICALS (2616.HK) COMPANY … · CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019. 2 ... that are described in the Company’sprospectus published

32

TIBSOVO®: Globally First-in-Class IDH1 Inhibitor US FDA approved for Acute Myeloid Leukemia with IDH1 mutation

Fast Track Review

Breakthrough Therapy Designation

Priority Review

0 2 4 6 8 1 0 1 2 1 4 1 6 1 8 2 0 2 2 2 4 2 6 2 8 3 0 3 2 3 4 3 6 3 8 4 0

0 .0

0 .1

0 .2

0 .3

0 .4

0 .5

0 .6

0 .7

0 .8

0 .9

1 .0

O v e ra ll s u rv iv a l (m o n th s )

Su

rv

iva

l p

ro

ba

bil

ity

N u m b e r o f p a t ie n ts a t r is k :

N o n -re s p o n d e rs

57 50 32 16 45757 43 25 11 456 215 47 3

N o n -C R /C R h re s p o n d e rs

C R + C R h

C R + C R h

N o n -C R /C R h re s p o n d e rs

N o n -re s p o n d e rs

O ve ra ll

12 1

18 10 3 11517 6 214 0

1 0 4 29 9 35577 15 6 038 2

C e n s o re d

Durable Remissions with Ivosidenib in IDH1-Mutated Relapsed or Refractory AML

In this high-risk, molecularly

defined R/R AML patient

population, ivosidenib induced

durable responses:

CR+CRh rate 32%,

duration 8.2 months,

median overall

survival 18.8 months

ORR 42%, duration 6.5

months

Total Newly Diagnosed Addressable Incidences (2018)

Source: Globocan 2018; CStone analysis

Note: AML = acute myeloid leukemia; MDS =

myelodysplastic syndromes; iCCA = intrahepatic

cholangiocarcinoma; CRC = colorectal cancer;

Prostate = prostate cancer; B-All = B-acute

lymphoblastic leukemia; GBM = glioblastoma;

LGG = low grade glioma

Immuno-Oncology Combination Potential

Active Clinical Programs

AML Chondro

-sarcoma

MDSiCCA CRC Prostate B-ALL Melanoma GBM LGG

Cancer Types Harbouring IDH1 Mutation

Peak Sales in AML only USD 50M

Total:

33,408

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33

Avapritinib: Globally First-in-Class PDGFRA/KIT Inhibitor First US NDA filed for PDGFRA exon 18 mutant and fourth-line GIST

Fast Track Review

2X Breakthrough Therapy Designation

Priority Review

Encouraging Clinical Activity Shown in NAVIGATOR Registrational Phase 1 Study

Phase 1 NAVIGATOR study

demonstrates favorable

tolerability and encouraging

clinical activities across lines of

therapy

86% ORR in PDGFRA

Exon 18 mutant GIST

22% ORR in fourth-list

GIST

Total Newly Diagnosed Addressable Incidences (2018)

Source: Globocan 2018; CStone analysis;

Avapritinib CTOS 2018 Presentation

Note: GIST = gastrointestinal stromal tumors;

ASM = advanced systemic mastocytosis; ISM =

indolent systemic mastocytosis; AML = acute

myeloid leukemia; LGG = low grade glioma; EC

= esophageal cancer; HNSCC = head and neck

squamous cell carcinoma; HCC = hepatocellular

carcinoma; sqNSCLC = squamous non-small

cell lung cancer; nsqNSCLC = non-squamous

non-small cell lung cancer; CRC = colorectal

cancer; CC = cervical cancer

GIST: Prevalence is over

10X of incidence

Active Clinical ProgramsCancer Types Harbouring PDGFRA exon 18

and/or KIT exons 9/11/13/14/17 mutations

Peak sales in GIST indications only USD 90M

GIST Incidences

GIST Prevalence Total:

37,454

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34

Pralsetinib: Potentially First-in-Class RET InhibitorFirst US NDA to be filed in RET-fusion NSCLC in 1Q 2020

2X Breakthrough Therapy Designation

Broad and Durable Antitumor Activity in Patients with RET Fusion+ NSCLC

Pralsetinib demonstrates broad

and durable antitumor activity in

patients with RET fusion+

advanced NSCLC

60% ORR and 100%

DCR in patients

previously treated with

platinum chemotherapy,

and 58% ORR in all RET

fusion+ patients

Total Newly Diagnosed Addressable Incidences (2018)

Source: Globocan 2018; CStone analysis;

Pralsetinib ASCO 2019 Presentation

Note: NSCLC = non-small cell lung cancer; MTC

= medullary thyroid cancer; PTC = papillary

thyroid cancer; CRC = colorectal cancer; GC =

gastric cancer; BC = breast cancer; STS = soft

tissue sarcoma; OC = ovarian cancer; Liver =

liver cancer; CC = cervical cancer; Pancreas =

pancreatic cancer; EC = esophageal cancer;

NEN = neuroendocrine neoplasm

Active Clinical Programs

Targeted Therapy Combination Potential (e.g. Tagrisso®)

Cancer Types Harbouring RET Mutation

Peak Sales in lung and MTC only USD 180M

Total:

73,576

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35

Fisogatinib: Potentially First-in-Class FGFR4 InhibitorPotent, highly selective FGFR4i designed for cancers with FGFR4

pathway activation

Anti-tumor activity in heavily pre-treated FGF19 IHC+ patients

Fisogatinib provides acceptable tolerability, pathway engagement and anti-tumor

activity in heavily pre-treated FGF19 IHC+ patients

Fisogatinib demonstrates clinical activity regardless of HCC etiology and

prognostic factors

CS1001 (PD-L1) and Fisogatinib combination study to be initiated by year end

Source: Globocan 2018; CStone analysis; Fisogatinib ESMO 2017 Presentation; Role of fibroblast growth factor receptor 4 in cancer by Tang et al.

Note: HCC = hepatocellular carcinoma

Broad cancer types

Alterations of FGFR4 has

been detected in multiple

types of human cancer,

such as breast cancer,

liver cancer, colon cancer,

prostate cancer,

rhabdomyosarcoma,

esophageal cancer, and

head and neck cancer