CSTONE PHARMACEUTICALS (2616.HK) COMPANY …

CSTONE PHARMACEUTICALS (2616.HK)

COMPANY PRESENTATION

January 2021

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

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3

Table of Contents

1 Introduction 4

2 Clinical Progress Overview 14

3 CStone Pipeline 2.0 Advancement 28

4 Full-fledged Biopharma Capabilities 32

4

Introduction

5Note: NDA = New Drug Application

Industry-leading

management team

Integrated

biopharma with clear focus

on clinical development, fast

ramp-up of manufacturing

capability, and transitioning

into commercial stage

$150M

Series A(July 2016)

$262M

Series B(May 2018)

$328M

HK IPO(Feb 2019)

Well-balanced

oncology portfolio with a focus

on immuno-oncology and

precision medicine with 5+ NDA

submissions in 2020

$200M

Pfizer strategic investment(Sep 2020)

To become a world-renowned biopharmaceutical company that is

leading the way to conquering cancer

5 Years Since Company

Inception

HKEx listed

2616.HK

6

Industry leading management team with proven track

record and complementary expertise

Shirley Zhao, MD, MBA

Greater China GM,

Head of Commercial

Frank Jiang, MD, PhDChairman, Chief Executive Officer

Jason Yang, MD, PhD

Chief Medical Officer

Archie Tse, MD, PhD

Chief Scientific Officer

Jingrong Li, PhD

Chief Technology Officer

Sanhu Wang, MPH

SVP, Government and

Regulatory Affairs

7

Clinical development engine: strong in-house team led by

experienced leaders and supported by global CROs

7

▪ Former SVP and Head of Clin Dev for Beigene, led development of PD-1, BTK, PARP and RAF dimer inhibitors

▪ Led 40+ global and China trials

Jason Yang, MD, PhD

CMO

▪ Former Executive Director of Early Clin Dev at

MSD (US)

▪ Led 30+ FIH oncology trials

▪ Led 20+ I/O combination trials

Archie Tse, MD, PhD

CSO

▪ Former Head of APAC R&D for Sanofi

▪ Led a 21,000 patient mega-trial

▪ Led 79 clinical trials and 30 NDAs

within five years

Frank Jiang, MD, PhD

Chairman and CEO

Keep clinical strategy planning & development oversight in-house, while outsourcing day-to-day

execution to global CROs to ensure optimal balance between efficiency and scalability

Clin

ica

l

De

ve

lop

me

nt &

PV

1

Clin

ica

l

Op

era

tio

ns

Bio

me

tric

s &

Me

dic

al W

ritin

g

Qu

alit

y

Assu

ran

ce

Responsible for late-stage clinical

development and regulatory affairs

Ea

rly

De

ve

lop

me

nt

Clin

ica

l

Ph

arm

aco

logy

Tra

nsla

tio

nal

Me

dic

ine

&

Bio

ma

rke

r

Mo

lecu

lar

Dia

gn

ostics

Responsible for early-stage clinical

development and diagnostics/biomarkers

Strong in-house team with ~160 clinical staff, representing ~30% of total employees, of which ~70%

hold advanced degrees2 and ~70% have clinical development experience at MNCs

Note: 1. Includes GI cancer, lung cancer, hematology & other solid tumors, and pharmacovigilance. 2.Master and above

8

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

9

Well-balanced oncology portfolio of 14 innovative assets

focused on immuno-oncology and precision medicine

9

Source: Company

Note: Assets status denote progress in the region noted in the column titled “Rights”.

* CStone obtains the exclusive global right to lead development and commercialization of LCB71 outside the Republic of Korea

POC = Proof of Concept, AML= Acute Myeloid Leukemia, AdvSM = Advanced Systemic Mastocytosis, ISM = Indolent Systemic Mastocytosis, 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.

Drug Candidate

Lead Indication(s)

and Line(s) of

Therapies

Rights Pre-clinicalDose

EscalationPOC Pivotal NDA Partner

Pralsetinib

(RET)

1L / 2L NSCLC,

1L MTC

Avapritinib

(KIT / PDGFRA)

PDGFRA exon 18 GIST,

AdvSM, ISM

Ivosidenib

(IDH1)

R/R AML, 1L AML,

Cholangiocarcinoma

Sugemalimab

(CS1001, PD-L1)

NSCLC, GC, EC

R/R NKTLOut-licensed

CS1003

(PD-1)HCC

Fisogatinib

(FGFR4)1L / 2L HCC

CS1002

(CTLA-4)Solid tumors

CS3002

(CDK4/6)Solid tumors

CS3005

(A2aR)Solid tumors

NM21-1480

(PD-L1/4-1BB/HSA)Solid tumors

CS5001/LCB71

(ROR1)

Solid tumors,

hematologic cancers

Multi-Specific #2 Undisclosed

Multi-Specific #3 Undisclosed

ADC #2 Undisclosed

Pre

-cli

nic

al

La

te-s

tag

eC

lin

ical/

IND

Taiwan and Singapore NDA submission

Mainland China and Taiwan NDA submission

Mainland China Ex-Greater China

Ex-Greater China

ChinaGlobal Korea Singapore

Mainland China NDA submission

Mainland China NDA submission

*

10

Partnering with and recognized by Pfizer through

strategic and multi-dimensional collaboration

Co-development of

Pfizer’s assets

Joint in-licensing of global

innovative drugs

China commercialization of

sugemalimab

Equity investment: $200mm (at approximately HK$13.37 per share)

Long-term collaboration

solidified through equity

investment

Competitive platform for in-

licensing deals to allow

rapid expansion of pipeline

Faster and broader

commercialization of

sugemalimab in China

Synergistic Collaboration

Pfizer

◼ A global pharmaceutical leader with

prestigious brand value

◼ Extensive commercialization

network in China

◼ Leading oncology franchise with

robust pipeline of drug candidates

CStone

◼ An emerging leader in the

biopharmaceutical industry in China

◼ Sugemalimab, a commercial-ready,

potential best-in-class PD-L1 asset

for large indications

◼ Superior clinical capabilities with

strong execution

In addition to the equity investment

premium, CStone is entitled to receive

▪ Up to $280mm in milestone

payments, and

▪ Tiered, mid-to-high teens royalties

▪ Post proof-of-concept oncology

assets

▪ CStone to receive low double-

digit royalties

▪ Jointly in-license for Greater China

market

▪ CStone retains an option to

participate in co-promotion

1 2 3

11

Partnering with and recognized by EQRx, a global top-

notch biotech, through significant license-out transaction

Meaningful

immediate financial

benefits

Maximize global

commercial potential of

two lead I/O assets

▪ EQRx’s innovative model to position CStone’s assets competitively in large indications such as

NSCLC

▪ EQRx’s exceptionally experienced “all-star” team with stellar track record of success

▪ Broad potential to pursue CS1003 drug combos for the China market

▪ Immediate and significant capital proceeds to invest in strategic initiatives, as we transition into a

fully integrated biopharma and pursue CStone Pipeline 2.0 strategy

Capability of

forming global

partnership

▪ Continue to build on the successful track record of forming strategic partnership

▪ Elevate CStone as a partner of choice for global biotech and biopharma

Importance for

patient community

▪ Make CStone’s assets available to global patient community

▪ EQRx’s innovative business model to enlarge access and make drugs more affordable for patients

CStone to out-license ex-China rights to EQRx for two key late-stage

assets, sugemalimab (PD-L1) and CS1003 (PD-1)

▪ EQRx will lead clinical development and commercialization of these

two assets outside of Greater China

▪ CStone to receive US$150m upfront payment, up to US$1.15bn

milestone payments and tiered royalties on net sales

12

We are advancing Pipeline 2.0 development with

multiple first-in-class / best-in-class molecules

▪ We have secured the global rights for multiple innovative molecules with first-in-class / best-in-class potential

▪ Starting from multi-specific molecules and ADCs, our portfolio is well positioned to advance towards Pipeline 2.0

1 NM21-1480

(PD-L1 x 4-1BB x HSA)

• Rights in Greater China, Korea and

Singapore

• Tri-specific molecule with the

potential to be BIC molecule as the

next generation PD-(L)1 inhibitor

• In-licensed from Numab

3 Multi-Specific #3

1 LCB71 (ROR1 ADC)

2 Multi-Specific #2

2 ADC #2

• Global rights

• Global FIC

• Nominated and designed by

CStone

• Global rights (outside Korea)

• Potentially a BIC ROR1 ADC with

differentiated technology

• In-licensed from LegoChem

• Global rights

• Global FIC

• Nominated and designed by

CStone

• Global rights

• Potentially a BIC molecule

• Nominated and designed by CStone

Multi-specific

molecules

ADC

And more…

And more…

Others And more…

13

Clear strategy towards building a full-fledged commercial

organization with near-term ambitions to reach “critical mass”

Note: I/O = immuno-oncology; EAP = early access program

Reach “Critical Mass” with

strong commercial platform

▪ Oncology focused portfolio with 3+

precision medicine and multiple I/O

combos

▪ Well-established sales team with

broad hospital coverage in China

▪ EQRx to maximize commercial value of

sugemalimab and CS1003 in ex-China

▪ Continue to explore potential

partnership with global partners for

value creation, with ex-China rights in

hand

Achieve “Global Vision”:

▪ To become globally recognized as

the leading Chinese biopharma

Stage 1 (2020)

Stage 2 (in 3-5 years)

Stage 3 (beyond)

Develop “Full-Fledged”

commercial organization

• Commercial organization with

core competencies and team

ready by year-end of 2020

• Focus on launching precision

medicines. Initiated Bo’ao EAP

precision medicine pilot program

in Hainan

14

Clinical Progress Overview

15

Note:

AML= Acute Myeloid Leukemia,, cHL= Classical Hodgkin’s Lymphoma, GIST = Gastrointestinal Stromal Tumor, HCC = Hepatocellular Carcinoma,

ENKTL = Extranodal Natural KILLER/T Cell Lymphoma, NSCLC = Non-small Cell Lung Cancer, EC = Esophagus Cancer, MTC = Medullary Thyroid

Cancer, R/R = Relapsed or Refractory

Significant clinical progress up to date

30 clinical trials with 17 registrational trials by 2021

Sugemalimab

(PD-L1)

CS1003

(PD-1)

Ivosidenib

(IDH1)

Avapritinib

(KIT/PDGFRA)

Pralsetinib

(RET)

• 2L NSCLC• Mainland China NDA accepted in 2020 with priority review designation

• Taiwan NDA filing expected in 2021

• 1L NSCLC • Last patient in by 2020 and mainland China NDA filing expected in 2021

• 1L MTC• Last patient in and breakthrough therapy designation granted by 2020,

mainland China NDA filing expected in 2021

• PDGFRA exon 18 GIST• Mainland China NDA accepted in 2020 with priority review designation

• Taiwan NDA filed in 2020

• R/R AML• Singapore NDA filed in 2020

• Last patient in by 2020 and mainland China NDA filing expected in 2021

• 1L AML • Patient enrollment on track

• 1L Stage IV NSCLC • Mainland China NDA accepted in 2020

• Stage III NSCLC • Last patient in by 2020 and mainland China NDA filing expected in 2021

• 1L GC • Patient enrollment on track

• 1L ESCC • Patient enrollment on track

• R/R ENKTL • Patient enrollment on track

• 1L HCC • Patient enrollment on track

Indication Status

16

Significant clinical progress – pralsetinib (RET) Global and China development and regulatory status

U.S.

Greater China

Registrational Potential Study

PartnerDrug

CandidateIndications Region

Mono

/ComboStudy Initiation

Patient

EnrollmentNDA Submission NDA Approval

Pralsetinib

(RET)

NSCLC 2L China Mono

MTC 1L China Mono

NSCLC 1L China Mono

Basket cohort China Mono

Joining global study




PartnerDrug

CandidateTarget Indications

Pivotal / Registrational Trial

Patient Target Enrollment

U.S. NDA

Submission

U.S. NDA

Approval

Pralsetinib RET

NSCLC * **

TC/MTC **

Achieved

Registrational StudyNote:

* Phase 3 AcceleRET Lung trial is ongoing

** In collaboration with Roche. Blueprint Medicines and Roche have co-exclusive rights to develop and commercialize pralsetinib in the U.S.

GAVRETOTM (pralsetinib) was granted accelerated approval by the FDA for the treatment of three indications: adult patients with metastatic RET fusion-positive NSCLC as detected by an FDA approved

test, adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant MTC who require systemic therapy, and adults and pediatric patients 12 years of age and older with

advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). Blueprint Medicines, GAVRETO

and associated logos are trademarks of Blueprint Medicines Corporation.

NDA = New Drug Application, NSCLC = Non-small Cell Lung Cancer, TC = Thyroid Cancer, MTC = Medullary Thyroid Cancer

17

Pralsetinib: potentially first-in-class RET inhibitorTargeted therapy with significant indication expansion potential

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

Total Newly Diagnosed Incidences in China (2020)

Source: Globocan 2020; CStone analysis;

Pralsetinib ASCO 2020 Presentation

Active Clinical Programs

In Partnership With

Not for promotional use.

ARROW

Note: ORRs are for response-evaluable patients.

Top-line data from the Phase 1/2 ARROW trial in patients with RET fusion NSCLC. Data cutoff date: November 18, 2019.

1. Two responses pending confirmation. 2. All responses confirmed.

Safety Summary

◼ Safety results (N=354; 400 mg

QD, all tumor types) were

consistent with prior data; most

reported AEs were grade 1 or 2;

no treatment-related grade 5 AEs

were reported.

◼ Overall, 4% of patients

discontinued treatment due to a

treatment-related AE.

ORR1: 61% (RET-fusion NSCLC

with prior platinum chemotherapy)

ORR2: 73% (RET-fusion NSCLC

with no prior systemic therapy)

ALL NSCLC PATIENTS (400 MG QD)

PER CENTRAL RADIOLOGY

Total Incidences:

80,832

Cancer Types Harboring RET Mutation

2X Breakthrough Therapy Designation

Orphan Drug Designation

Priority Review

Accelerated Approval

18

Significant clinical progress – avapritinib (KIT/PDGFRA) Global and China development and regulatory status

PartnerDrug

CandidateTarget Indications

Pivotal / Registrational Trial

Patient Target Enrollment

U.S. NDA

Submission

U.S. NDA

Approval

Avapritinib KIT/PDGFRA

PDGFRA exon 18

GIST

Advanced SM

Indolent SM Ongoing

Achieved

U.S.

Greater China

PartnerDrug


Mono


Patient


Avapritinib

(KIT/PDGFRA)

PDGFRA

exon 18 GISTTaiwan Mono

PDGFRA

exon 18 GISTChina Mono

Advanced SM China Mono Exploring trial waiver

Indolent SM China Mono Exploring trial waiver

Registration with US NDA data

Note: AYVAKIT™ (avapritinib) is approved by the U.S. FDA for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations

Blueprint Medicines, AYVAKIT and associated logos are trademarks of Blueprint Medicines Corporation.

(1) Sufficient enrollment has been achieved to enable interim analysis and supplemental NDA filed in Q4 2020 in the U.S.

GIST = Gastrointestinal Stromal Tumors, SM = Systemic Mastocytosis

Registrational Study

(1)

Bridging study

19

Avapritinib: potentially first-in-class KIT/PDGFRA inhibitor Targeted therapy with significant indication expansion potential

Robust Clinical Activity Shown in NAVIGATOR Registrational Phase 1 Study

Fast Track Review


Priority Review


Source: Globocan 2020; CStone analysis; Avapritinib

ESMO 2020 Presentation Active Clinical Programs

Cancer Types Harboring PDGFRA exon 18

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

In Partnership With

Total Incidences:

38,589


Efficacy Summary

◼ ORR: 91% for all doses

◼ mDOR with avapritinib 300/400 mg: 22

months (95% CI, 14–NR); mPFS: 24

months (95% CI, 18–NR); mOS: not

reached

Safety Summary

◼ Most AEs were grade 1 or 2, with a lower

incidence of commonly reported AEs in

the 300 mg QD dose group compared

with the 400 mg QD dose group.

◼ No treatment-related grade 5 AEs were

reported.

Data cut-off: March 9, 2020. Long-term efficacy, tolerability and overall survival in patients with unresectable or metastatic PDGFRA D842V-

mutant gastrointestinal stromal tumor treated with avapritinib: NAVIGATOR phase 1 trial update. 2020 ESMO Virtual Congress

Orphan Drug Designation

Approval for PDGFRA exon 18 mutant GIST

PFS of Avapritinib in PDGFRA exon 18 mutant GIST

20

Significant clinical progress – ivosidenib (IDH1) Global and China development and regulatory status

Partner Drug Candidate Target IndicationsPivotal / Registrational Trial

Patient Enrollment

NDA

SubmissionNDA Approval

Ivosidenib IDH1

R/R AML

IC-Ineligible 1L AML

monotherapy

IC-Eligible 1L AML

combo with 7+3

IC-Ineligible 1L AML

combo with AZA

2/3L Cholangiocarcinoma

monotherapy

Achieved

PartnerDrug


Mono


Patient


Ivosidenib

(IDH1)

IDH1m R/R AML Taiwan Mono

IC-Ineligible 1L

AML

combo with AZA

China Combo

IDH1m R/R AML China MonoIncluded in the list of drugs of urgent

clinical needs in mainland China

IDH1m R/R AML Singapore Mono



Bridging study

Global

Greater China and Singapore

Note: AML= Acute Myeloid Leukemia, R/R = Relapsed or Refractory, IC = intensive chemotherapy, AZA = azacytidine; 7+3 is a chemotherapy regimen for AML, where “7” refers to Cytarabine given daily

for 7 days and “3” refers to Daunorubicin given daily for 3 days


Registrational Study

21

TIBSOVO®: global first-in-class IDH1 inhibitor No immediate competitor in China; significant indication expansion potential

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

Fast Track Review


Priority Review


Source: Globocan 2020; CStone analysis Active Clinical Programs

AML Chondro

-sarcomaMDSiCCA CRC Prostate B-ALL Melanoma GBM LGG

Cancer Types Harboring IDH1 Mutation

Total Incidences:

34,151

In Partnership With

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

rviv

al

pro

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


Orphan Drug Designation (R/R AML, CCA, GBM and MDS)

Approval for R/R AML and IC-Ineligible 1L AML

22

Asset overview

Note: as of Dec 31, 2020, including patients dosed with placebo

DLT = dose limiting toxicity, MTD = maximum tolerated dose, ADA = anti-drug antibodies, IgG4 = Immunoglobulin G4, ADCP = Antibody-Dependent

Cellular Phagocytosis, ADCC = Antibody-Dependent Cell-Mediated Cytotoxicity, CDC = Complement Dependent Cytotoxicity, EC = Esophageal Cancer

■ Fully-human, full length IgG4 derived from Ligand’s OmniRat® platform – minimal

possibility of generating ADA;

■ The only PD-L1 antibody that naturally lacks ADCC/CDC activity – better safety and

avoid unwanted attack of T cells;

■ Retains ADCP activity that potentially induces direct tumor killing by macrophages

and enhances tumor antigen presentation for long-term anti-tumor immunity – more

efficacious in certain indications like NKTL

Significant clinical progress – sugemalimab (PD-L1) (1/4)

Unique design

Prominent

safety profile

Best-in-class

potential

Significant

development

progress

■ Early phase studies demonstrated that Sugemalimab was safe & well tolerated

‒ No DLT1 from 3 mg/kg to 40 mg/kg, MTD2 not reached

‒ No infusion reactions; low ADA3 rate

‒ Low frequency of severe irAEs

■ Data showed potential of best-in-class PD-L1

‒ Encouraging anti-tumor activities observed in phase Ia dose-escalation study,

multiple phase Ib cohorts, phase II and phase III studies

‒ In particular, promising data in NSCLC, ESCC and NKTL

■ >1,600 patients dosed, with 5 registration trials ongoing, including 1 pivotal Ph II

study and 4 Ph III studies*

■ Strategically targeting China prevalent cancers including S3 and S4 NSCLC, GC and

ESCC

23

Significant clinical progress – sugemalimab (PD-L1) (2/4)

Note: cross trial comparison subject to the usual caveat; NDA status as of Jan 22nd, 2021; mNSCLC = metastatic non-small cell lung cancer, sq = squamous, nsq = non-squamous

* PFS HR data of POSEIDON trial not publicly available

Source: public information and CStone analysis

48

46

44

29

67

0 10 20 30 40 50 60 70

Tislelizumab (sq, RATIONALE 307)

Sintilimab (sq, ORIENT-12)

Pembrolizumab (sq, Keynote-407)

Atezolizumab (sq, IMpower 131)

Durvalumab (sq, POSEIDON)*

Sugemalimab (sq, GEMSTONE-302)

PD-(L)1 for 1L

treatment of

mNSCLC

(sq)

PD-L1 PD-1

▪ Potentially the most efficacious PD-(L)1 for first line treatment of mNSCLC (sq)

▪ Potentially the best-in-class PD-L1 for first line treatment of mNSCLC

PD-(L)1 for 1L

treatment of

mNSCLC

(sq & nsq)

NDA StatusReduction of the Risk of Disease Progression or Death (%)

Under review

Not approved

Not approved

Approved

Under review

Approved

36

48

52

46

39

48

44

40

36

29

50

0 10 20 30 40 50 60

Tislelizumab (nsq, RATIONALE 304)

Tislelizumab (sq, RATIONALE 307)

Sintilimab (nsq, ORIENT-11)

Sintilimab (sq, ORIENT-12)

Camrelizumab (nsq, SHR-1210-303)

Pembrolizumab (nsq, Keynote-189)

Pembrolizumab (sq, Keynote-407)

Atezolizumab (nsq, IMpower 132)

Atezolizumab (nsq, IMpower 130)

Atezolizumab (sq, IMpower 131)

Durvalumab (sq&nsq, POSEIDON)*

Sugemalimab (sq&nsq, GEMSTONE-302) Under review

Not approved

Not approved

Approved

Not approved

Approved

Approved

Approved

Under review

Under review

Approved

Under review

24

Significant clinical progress – sugemalimab (PD-L1) (3/4)Phase Ib study data in 1L ESCC and Phase II study data in rr-ENKTL

Source: Company, ESCC Ph Ib data presented at 2020 ESMO, rr-ENKTL Ph II data presented at 2020 CSCO

Note: PTCL = peripheral T-cell lymphoma; HDAC = histone deacetylase; ORR = Overall Response Rate; DOR = Duration Of Response;

Sugemalimab + CF (Cisplatin + 5 FU) in 1L ESCC

ORR = 67.6% (25/37), DCR = 89.2% (33/37)

Sugemalimab as monotherapy in rr-ENKTL

ORR = 44.7% (17/38), CR = 31.6% (12/38)

mDOR = 16.8 months, 1 Yr OS rate: 55.5%

Sugemalimab SintilimabChidamide

（Approved in rr-PTCL, China, 2014)

Class PD-L1 PD-1 HDAC

Study CS1001-201 ORIENT-4 Registration Study Real World Study

n 38 28 16 33

CR 31.6% (12/38) 7.1% (2/28) 6.3% (1/16) 6.1% (2/33)

DOR (m)16.8

(1.0,16.8+)

4.1

(0+,4.2+)UNK UNK

Source CSCO 2020 ASCO 2019 Ann Oncol, 2015[4] J Hematol Oncol,

2017[5]

31.6

7.1 6.3 6.1

0

10

20

30

40

CR

(%

)

ime on reatment ays

atients

CR R

R

CR RS reatment ngoing anti cancer therapy eath

ORR*: 67.6% (25/37)

DCR: 89.2% (33/37)

DOR: not reached

Sugemalimab TislelizumabDurvalumab+

Tremelimumab

Class PD-L1 PD-1 PD-L1

n 37 15 6

ECOG0: 20.5%

1: 79.5%

0: 26.7%

1: 73.3%

0: 0%

1: 100%

Chemo

RegimenCF CF CF

DOR (m)NR

(0.03+~13.3+)12.8 Not reported

Source ESMO 2020 CSCO 2019 ASCO GI 2019

67.6

46.7

33.3

0

20

40

60

80

OR

R (

%)

* 20 confirmed PR; 5 PR to be confirmed as of data cut-off on 19 February 2020

25

Sugemalimab + XELOX (Capecitabine

+ Oxaliplatin) in 1L GC/GEJ (Gastric or

Gastroesophageal Junction

Adenocarcinoma)

* 17 confirmed PR; 1 PR to be confirmed as of data

cut off on 19 February 2020

Best Response

Partial Response (PR)

Stable Disease (SD)

Progressive Disease (PD)

30% Shrinkage

ORR*: 62.1% (18/29)

DCR: 82.8%

DOR: 11.3m

Target Lesion Shrinkage from Baseline

Change (

%)

to B

aselin

e

Sugemalimab (PD-L1) as monotherapy

in CC/GBC (Cholangiocarcinoma or

Gallbladder Carcinoma)

* 2 confirmed PR; 1 PR to be unconfirmed as of data

cut-off on 1 July 2019


Change (

%)

to B

aselin

e30% Shrinkage

ORR*: 10.3% (3/29)

DCR: 37.9%

DOR: 5.39m

Sugemalimab (PD-L1) as monotherapy

in MSI-H/dMMR Cancer

Pancreatic*

30% Shrinkage

Intestinal*

** Unless specified, all patients were Colorectal Cancer

Best Response


Stable Disease (SD)


Best Response


Stable Disease (SD)


ORR*: 38.1% (8/21)

DCR: 57.1%

DOR: not reached


Significant clinical progress – sugemalimab (PD-L1) (4/4)Phase Ib study data: other cohorts

Change (

%)

to B

aselin

e

62.1

48.6

60

68.4 66.7

85

0

10

20

30

40

50

60

70

80

90

OR

R (

%)

CS1001Keytruda

(062)

Keytruda

(059)Opdivo

Camrelizu

mabSintilimab

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

n 29 257 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%

Regimen XELOXCisplatin + 5-

FU or

Capecitabine

Cisplatin + 5-

FU or

Capecitabine

SOX or

XELOXXELOX XELOX

DOR (m)11.3

(1.0+ ~14.1+)6.8

4.6

(2.6 ~ 20.3+)

9.9

(5.8, NR)NR

5.3

(4.8~7.2)

Source ESMO 2020 ASCO 2019 ESMO 2017

Ann Oncol.

2019 Feb

1;30(2):250-

258

ASCO

2019ASCO 2019

26

Str

ate

gic

va

lue

Ph

I D

ata

Hig

hli

gh

tsA

ss

et

ove

rvie

wSignificant clinical progress – CS1003 (PD-1) & CS1002 (CTLA-4) Two Additional I/O Backbones: preliminary data and development strategy

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.

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

■ Amino acid sequence identical to ipilimumab (Yervoy)

■ 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; increase in

absolute lymphocyte count (ALC) observed indicating

target engagement

■ Overall clinical profile is consistent with that of ipilimumab

CS1002

■ 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

CS1002 induced early ALC increase, similarly to Ipilimumab

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 Ph I conducted in China showed that CS1003

monotherapy was safe and tolerable at 60mg and 200mg

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

■ Ph Ib data showed that ORR and mPFS reached 40%

and 8.4 months respectively among 20 patients that

received the treatment of CS1003 + lenvatinib

■ One of 3 I/O backbones with multiple combo studies

ongoing, including the combo study of CS1003 + CS1002

and the global randomized Ph III trial of CS1003 +

lenvatinib in first line treatment for advanced

unresectable HCC (CS1003-305)

Preliminary efficacy of CS1003 + lenvatinib in HCC

1 mg/kg (N=6) 3 mg/kg (N=3) 10 mg/kg (N=4) 10 mg/kg

CS1002 Ipilimumab

27

Progress on other clinical-stage assets

CS3002

(CDK4/6)

◼ Conducting a Ph I trial for solid tumors as monotherapy in Australia/China

CS3005

(A2aR)

◼ Conducting a Ph I trial for solid tumors as monotherapy in Australia/China and plan

to initiate a combo study with CS1003 (PD-1) thereafter

CS3008 /

Fisogatinib *

(FGFR4)

◼ Conducting a Ph I trial (join global) for HCC as monotherapy in China and

published results (CN part) in CSCO 2020, exploring the registration path for

monotherapy in 3L HCC in China

◼ Conducting a Ph Ib/II trial for HCC as combo with sugemalimab in China and

initiated dose expansion in 3Q2020

NM21-1480

(PD-L1 / 4-1BB /

HSA)

◼ Conducting Ph I trials for solid tumors in US and Taiwan by Numab

Note: in partnership with Blueprint Medicines

HCC = Hepatocellular Carcinoma

28

CStone Pipeline 2.0 Advancement

29

Pipeline 2.0: CStone-driven innovation model for global

FIC/BIC assets

I/O (Combo) & Precision Medicine

TME Multi-specifics ADCCancer

Vaccine(Opportunistic)

CStone-Driven

Innovation

Model

Idea Generation

Project Design1 FIC/BIC

2 Global rightsWork with CRO

on execution

Examples

• 3 multi-specifics,

incl. NM21-1480

• 2 ADCs, incl.

CS5001

Sources of Innovative Ideas

Protein Chemistry PharmacologyProject

Management

Clinical

Pharmacology

DMPK /

Toxicology

Bioinformatics Translation

Med/BiomarkerSABAcademic

Observa-tions

made in the clinic

BD partners

CRO platforms

Archie Tse, MD, PhD

Chief Scientific Officer

Line of sight from Discovery Research to PoC under a single leadership

Note: I/O = immuno-oncology, TME = tumor microenvironment, ADC = antibody-drug conjugate, FIC = first-in-class, BIC = best-in-class, PoC = Proof of Concept

Early Development

Molecular

Diagnostics

• Physician-scientist with 20+ years of

experience in translational oncology

research covering cytotoxics, targeted

agents, and immunotherapies

• Oncology TA Head at Daiichi-Sankyo

responsible for the development and

implementation of overall Oncology TA

strategy

• Oversaw early development of 14 IO

assets of different MOAs and

modalities at MSD

30

■ Combination therapy in one molecule

→expands combo options with SoC and

other ICIs

■ Monovalent 4-1BB binding remains

inactive until PD-L1 engagement

→maximizes safety

■ Ultra-high affinity (pM) to PD-L1

→broadens PD-L1+ tumor types

■ Standard affinity to 4-1BB (nM)

→ensures effective activation

■ HSA extends T1/2 & 100% effector null

→enables convenient dosing & eliminated

undesirable FcgR-mediated activation

■ MW ~70kDa, only half of conventional

mAb

→leads to better tumor penetration &

efficacy

4-1BB

T cell

PD-1

PD-L1

4-1BB

Cancer

T cell

scMATCH3

PD-L1 Negative PD-L1 Positive

Urelumab

NM21-1480

500

1000

Mean relative tumor volume

Time (days)

mean R

TV

(%

of baseline)

0 3 7 10 17 21 2414

Irrelevant IgG (Palivizumab)1

NM21-1186 (0.02 mg/occasion)2



Avelumab7

PDL1 IgG6

PD-L1 IgG + 4-1BB IgG5

Urelumab8

NM21-1480 (PD-L1x4-1BBxHSA) has the potential to be

the BIC molecule as the next generation PD-(L)1 inhibitor

■ Bridge new biology

■ Improve therapeutic index & reduce

unwanted toxic effects

■ Expand combo options & improve

administration convenience

Six key features of NM21-1480

31

LCB71 has the potential to be best-in-class ROR1 ADC

with differentiated technology

Collaboration highlights Differentiated payload design

1

2

▪ CStone to acquire rights for development and commercialization

outside Korea for LCB71, an ROR1 antibody drug conjugate

(“ADC”)

▪ A potential global first-wave / best-in-class drug with monotherapy

and combination applications for multiple solid and hematological

malignancies

▪ ROR1 protein expression is prevalent in a variety of cancers including

leukemia, non-Hodgkin lymphoma, breast, lung, and ovarian cancers

▪ LCB71 leverages proprietary tumor-selective linker (denoted as 1 in

the graph on the right) and tumor-activated pyrrolobenzodiazepine

(“ ”) prodrug toxin (denoted as 2 in the graph on the right) to

address the typical toxicity problem associated with traditional

PBD payloads

▪ It utilizes site-specific conjugation for a precise drug antibody ratio

(“DAR”), enabling homogeneous production and large-scale

manufacturing

Asset Development stage Indications Global value

LCB71

(CStone / LegoChem)

▪ Pre-clinical

▪ Potential best-in-class with differentiated

toxin design and conjugation technology

▪ Multiple solid and

hematological malignancies

VLS-101

(Merck / VelosBio)

▪ Ph I/II

▪ Global first-in-class

▪ TNBC, NSCLC, CLL and

MCL, etc.

• In Nov 2020, Merck acquired VelosBio

$2.75bn for all shares

NBE-002

(BI / NBE Therapeutics)

▪ Entered Ph I in Oct 2020 ▪ TNBC and advanced solid

tumor, etc.

• In Dec 2020, Boehringer Ingelheim

acquired NBE Therapeutics for

~$1.4bn (Euro 1.18bn) for all shares

Recent transactions for ROR1 ADC

Note: TNBC = Triple Negative Breast Cancer, NSCLC = Non-Small Cell Lung Cancer, CLL = Chronic Lymphocytic Leukemia, MCL = Mantle Cell Lymphoma

32

Full-fledged Biopharma Capabilities

33

State-of-the-art manufacturing facility in construction to

be in pilot operation by 2021

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 molecules

◼ In August 2019, CStone entered into an

agreement with Sungent (state-owned

controlled by Suzhou Industrial Park) to

build manufacturing facility

◼ Commencement of the construction in the

first half of 2020 with pilot operation in 2021

◼ Planned building area of approximately

100,000 sqm

◼ Capabilities: 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

34

Continuing to bring breakthrough therapies to

cancer patients in China and worldwide

Note:1. For in-licensed assets NDA approval time will depend on our partners’ NDA approval time by US FDA

GIST = Gastrointestinal Stromal Tumor, AML= Acute Myeloid Leukemia, R/R = Relapsed or Refractory, NSCLC = Non-small Cell Lung Cancer, sq = squamous, nsq = non-

squamous, MTC = Medullary Thyroid Cancer, SM = Systemic Mastocytosis, HCC = Hepatocellular Carcinoma, NKTL = Natural KILLER/T Cell Lymphoma, ESCC = Esophageal

Squamous Cell Carcinoma, GC = Gastric Cancer

Self-developed From partners

Expect 4 products

Across 4+ indications1

Expect 6+ products


Expect 10+ potentially

approved products


Near-term (2020~2021) Mid-term (2022~2025) Long-term (2026~)

Fisogatinib

HCC

Avapritinib


SM

Ivosidenib

IDH1 r/r AML, 1L AML

CS3002

CDK4/6

ND21-1480

(PD-L1/4-1BB/HSA)

CS1003 (PD-1)

HCC

Pralsetinib

RET 1L / 2L NSCLC, 1L MTC

Sugemalimab

Stage IV NSCLC (sq & nsq),

Stage III NSCLC, ESCC, GC,

NKTL, CRC (w/ regorafenib)

CS1002

(CTLA-4)

CS3005

A2aR Pralsetinib

RET 2L NSCLC

Sugemalimab

Stage IV NSCLC (sq &

nsq)

CS1003 (PD-1)

HCC

Pralsetinib

RET 1L NSCLC,

RET 2L NSCLC,

1L MTC

SugemalimabStage IV NSCLC (sq &

nsq), Stage III NSCLC,

ESCC, GC, NKTL

Fisogatinib

HCC

Avapritinib


SM

Ivosidenib

IDH1 r/r AML,

1L AMLAvapritinib

PDGFRA exon 18 GIST

Ivosidenib

IDH1 r/r AMLMolecules from co-development with Pfizer

Molecules from co-development and joint in-licensing with Pfizer

More molecules under co-development or in-licensing agreement with partners

35

Financial summary for 1H 2020

Cash Balance

◼ RMB2,124 million of cash, cash equivalents, and time deposits as of

June 30, 2020 vs. RMB2,726 million as of December 31, 2019

◼ Cash position decreased by RMB602 million mainly due to R&D expenses,

administrative and selling expenses

Sources of

Cash

◼ IPO proceeds: HK$2,394 million (post greenshoe)

◼ Bank borrowing: Available bank loan facility up to RMB200 million for

working capital improvement and the construction of the manufacturing

facility and other facilities

Uses of Cash

◼ R&D expenses (non-IFRS1): RMB470 million

◼ Administrative and selling expenses (non-IFRS1): RMB100 million

Note: 1. Adjusted for share-based compensation

36

Following a fruitful 2020, we expect various thrilling

milestones in 2021

4 products across 4+ indications

5+ NDA filings2 for 3 products

30 trials incl. 17 for registration

Multiple data readout

Revamp of research

organization for Pipeline

2.0 with global

FIC/BIC molecules

Multiple IND filings

Post-PoC

asset nomination for

co-development

with Pfizer

Pilot

operation of

the state-of-the-

art manufacturing

facility

Avapritinib

(KIT/PDGFRA

inhibitor)

Pralsetinib

(RET inhibitor)Sugemalimab1

(PD-L1

antibody)

Ivosidenib

（IDH1 inhibitor)

Commercialization Clinical Development

ResearchBusiness

DevelopmentManufacturing

Note:

1. Pfizer to lead commercialization of sugemalimab in mainland China

2. Expected NDA filings include but not limited to (1) sugemalimab: stage III NSCLC in mainland China; (2) pralsetinib: 1L NSCLC in mainland China; (3) pralsetinib: 1L MTC in

mainland China; (4) pralsetinib: 2L NSCLC in Taiwan; (5) ivosidenib: r/r AML in mainland China

Thank you!

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