CSTONE PHARMACEUTICALS (2616.HK) COMPANY PRESENTATION November, 2019
CSTONE PHARMACEUTICALS (2616.HK)
COMPANY PRESENTATION
November, 2019
2
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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
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3
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
4
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
5
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
6
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
7
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
8
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…
9
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
10
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
11
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+)
12
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
13
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
14
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
15
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
16
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)
17
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
18
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
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%
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
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.
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
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
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
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
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
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
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
Thank you!
Appendix
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
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
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
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
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