MANAGEMENT PRESENTATION - Jefferies · Presentation Disclaimer. ... tolerability and efficacy of the Company’s drug candidates, final and quality controlled verification of data

MANAGEMENT PRESENTATION

JUNE, 2019

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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") foruse 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 commitmentor investment decision.

Certain statements contained in this presentation and in the accompanying oral presentation, may constitute forward-looking statements. Examples of such forward-lookingstatements include those regarding investigational drug candidates and clinical trials and the status and related results thereto, as well as those regarding continuing andfurther development and commercialization efforts and transactions with third parties. Such statements, based as they are on the current analysis and expectations ofmanagement, inherently involve numerous risks and uncertainties, known and unknown, many of which are beyond the Company’s control. Such risks include but are notlimited to: the impact of general economic conditions, general conditions in the pharmaceutical industry, changes in the global and regional regulatory environment in thejurisdictions in which the Company’s does business, market volatility, fluctuations in costs and changes to the competitive environment. Consequently, actual future resultsmay differ materially from the anticipated results expressed in the forward-looking statements. In the case of forward-looking statements regarding investigational drugcandidates and continuing further development efforts, specific risks which could cause actual results to differ materially from the Company’s current analysis andexpectations include: failure to demonstrate the safety, tolerability and efficacy of the Company’s drug candidates, final and quality controlled verification of data and therelated analyses, the expense and uncertainty of obtaining regulatory approval, the possibility of having to conduct additional clinical trials and the Company’s reliance onthird parties to conduct drug development, manufacturing and other services. Further, even if regulatory approval is obtained, pharmaceutical products are generallysubject to stringent on-going governmental regulation, challenges in gaining market acceptance and competition. These statements are also subject to a number of materialrisks 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 andthe announcements and other disclosures we make from time to time. The reader should not place undue reliance on any forward-looking statements included in thispresentation or in the accompanying oral presentation. These statements speak only as of the date made, and the Company is under no obligation and disavows anyobligation 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 orby 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 theinformation, or opinions contained herein. The information set out herein may be subject to updating, revision, verification and amendment and such information maychange 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 orredistributed 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, directlyor indirectly, taken or transmitted into or distributed in any jurisdiction which prohibits the same except in compliance with applicable securities laws. This presentation andthe accompanying oral presentation contains data and information obtained from third-party studies and internal company analysis of such data and information. We havenot 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 thatyou 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.

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World-classManagement Team

15 Assets, All Oncology4 Potentially First-in-Class Assets

3 Clinical-stage IO Backbones to Drive Combo Potential5 Late-stage Assets, 1 NDA submission

13 Clinical Trials, 20 IND / CTA submissions1

IntegratedBiopharma with Clear

Focus on Clinical Development

$150MSeries A

$262MSeries B

(July 2016) (May 2018)

$328MHK IPO

(Feb 2019)

To Become Globally Recognized as the Leading Chinese Biopharma

3+ Years Since Company Inception

Note: 118 approved, 1 withdrawn, 1 under review

4

Clinical DevelopmentSVP/Head

Asia PacificHead, R&D

GlobalPhase 2 Trial

U.S.21K Lovenox Mega Trial

Frank Jiang, MD, PhDChairman, Chief Executive Officer

Jason Yang, MD, PhDChief Medical Officer

Industry Leading Management Team with Proven Complementary Expertise

Richard Yeh, MBAChief Financial Officer

Bing Yuan, PhD, MBAChief Business Officer

Jon Wang, PhDChief Scientific Officer

Jingrong Li, PhDSVP, Product Development & Manufacturing

Managing DirectorAsia Pacific Healthcare

equity research lead analyst

Biotech sector research

Drug research

Executive Director & Global Oncology BD Lead

Keytruda Combo Partnership

Oncology Global Marketing

Director of Immuno-Oncology Research

Immuno-Oncology ResearchDeep Manufacturing and

Quality Expertise

Executive DirectorEarly Clinical Development of

Immuno-oncology

Senior DirectorClinical Development

Archie Tse, MD, PhDChief Translational Medicine Officer

5

Distinguished World-class Scientific Advisory Board With Deep Oncology and IO Expertise

Paul BunnMD

Weiping ZouMD, PhD

Richard FinnMD

AACR President2018-2019

Professor of oncology,Johns Hopkins

University

Chair, AACR CancerImmunology

Charles B.de NancredeProfessor,

University of Michigan

International Liver CancerAssociation Former President

Clinical Professor,UCLA

Elizabeth JaffeeMD

ASCO President2002-2003

Distinguished Professor,University of Colorado

6

DrugCandidate

Molecular Target/

Signaling Pathway

Lead Indication(s)

and Line(s) of Therapies

Drug Candidate Category

Commercial Rights Partner Pre-clinical IND

FilingDose

Escalation POC Pivotal NDA

ivosidenib(CS3010, AG-120)

IDH1 R/R AML, 1L AML, Cholangiocarcinoma

Chemicals, 1(MRCT for AGILE);

Chemicals, 5.1(IND for R/R AML)

Greater China

CS1001(Core Product) PD-L1

R/R cHL, R/R NKTL,NSCLC7, Solid

tumors3Biologics, 1 Worldwide

avapritinib(CS3007, BLU-285)

KIT & PDGFRα

PDGFRα/ 2L / 3L GIST,

AdvSM, ISMChemicals, 1 Greater China

CS3009(BLU-667) RET 1L / 2L NSCLC,

1L MTC5 Chemicals, 1 Greater China

CS3008(BLU-554) FGFR4 1L / 2L HCC Chemicals, 1 Greater China

CS10022 CTLA-4 Solid tumors3 Biologics, 2 Worldwide

CS10032 PD-1 Solid tumors3 Biologics, 1 Worldwide

CS30062 MEK Solid tumors3 Chemicals, 1 Worldwide

CS3003 HDAC6 Solid tumors3, R/R MM4 Chemicals, 1 Worldwide

CS3002 CDK4/6 Solid tumors3 Chemicals, 1 Worldwide

ND021 PD-L1/4-1BB/HSA Solid tumors3 Biologics, 1

Greater China, South Korea,

Singapore

CS3004 Worldwide

CS1009 Worldwide

CS3005 Worldwide

CS2004 Worldwide

Synergized Oncology Portfolio Capturing Value in the Era of Combo Therapy

Source: Company1 Some indication(s) may not require a non-pivotal Phase II clinical trial prior to beginning pivotal Phase II or III clinical trials. 2 Denotes we currently have clinical trials ongoing in Australia for the product candidate 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. 4Available clinical data from other HDAC6 inhibitor studies provides the basis to suggest that CS3003 may be effective in treating MM; we plan to assess the clinical efficacy of CS3003 in MM and various types of solid tumor patients in the Phase Ib dose expansion trial. 5The clinical data published so far by Blueprint demonstrated that BLU-667 (CS3009) is effective in the treatment of certain NSCLC and MTC patients.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-

clin

ical

Clin

ical

/IN

D

Undisclosed

US FDA Approved (Agios)

Pivotal Phase III trial in the US ongoing (Blueprint)

Phase Ib trial in the US ongoing (Blueprint)

Phase Ib trial in the US ongoing (Blueprint)

China Status

China Status

Rest of the World Status

China Status

China StatusRest of the World Status

China StatusRest of the World Status

China StatusRest of the World Status

China StatusRest of the World Status

China Status

China Status

China Status

China Status

China Status

NDA submission in Taiwan

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PD-1/PD-L1 INHIBITORS AND CTLA-4 MABS HAVE BECOME THE MOST

ADOPTED IO BACKBONES

Cancer Treatment Paradigm Shifts Towards Combo Therapy Given Significant Potential to Improve Survival and Response Rates

Source: NMPA, Goldman Sachs Research, clinicaltrials.gov, Frost & Sullivan Analysis, Cell, literature research1 For illustrative purpose only. Source: Cell, April 2015 (Sharma, Allison).2 As of year-end 2017.

1

1716

2009 2018

PD-(L)1 combination trials globally, including combination with CTLA-4

COMBO THERAPY BELIEVED TO BE THE MOST PROMISING CANCER TREATMENT

PARADIGM SHIFT TOWARDS COMBO THERAPY

28%74%

72%26%

China US

Breakdown of PD-(L)1 trials by combination therapy / single-agent therapy2

Single Agent Combo Therapy

Improved overall survival and response rates1

SURVIVAL DURATION

Chemotherapy

Immune Checkpoint Therapy

Targeted Therapy

IO Combo Therapy

%SURVIVAL

Response Rate DurabilityEfficacy

COMBINATION THERAPIES BASED ON IO IS A FUTURE TREND SUPPORTED BY THERETICAL BASIS AND CLINICAL DATA

8

15 Pipeline Assets

# OF POTENTIAL

COMBOS

# OF ASSETS

CStone

Large Scale and Right Mix of Pipelines to Drive Success in Combo Therapy

NumbersCOMBINATIONS INCREASE EXPONENTIALLY

WITH NUMBER OF ASSETSRight Mix and Large Scale

THE RIGHT BACKBONE ASSETS UNLOCK COMBO POTENTIAL

Only company in China whichowns clinical stage PD-L1, PD-1 and CTLA-4

CS3009 (FGFR4)

CS3002 (CDK4/6)

CS3003 (HDAC6)

CS1009

CS3005

TIBSOVO®(CS3010)

(IDH1)

Avapritinib(CS3007)

(KIT&PDGFRα)

CS3008 (RET)

CS3006 (MEK)

CS3004

CS2004

PD-L1

CTLA-4PD-1

ND-021(PD-L1x4-1BB)

FlexibilitySTAY AT THE FOREFRONT OF MEDICAL

ADVANCEMENT Rapidly adapt to the cutting edge therapeutic development and focus on the most promising combination possibilities

Cost ControlDEVELOPMENT & TREATMENT COST

CONTROL

In-house combo agents ensure effective control of the development and treatment cost

$

9

Significant Potential to Multiply the Clinical Value with Rich IO and Other Oncology MOAs in Combo Therapies

1015+

1

10+

2018 2019

Mono Combo

Planned Trial Number By Type

Pipe

line

2.0

CS1001 (PDL1) + VEGF

CS1002 (CTLA4) + CS1003 (PD1)

Novel Combo -Unique to CStoneDe-risked Combo

CS1001 (PDL1) + ivosidenib (IDH1) in cholangiocarcinoma

CS1001 (PDL1) + BLU554 (FGFR4) in HCC

PD(L)1 + CS3002 (CDK4/6)CS1001 (PDL1) + IMP4297 (PARP)

Advanced Portfolio

Super-charge combo strategy based on PoC data

Novel molecular scaffolds & multi-specific antibodies

Cancer vaccines TME modulators

Pipe

line

1.0

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1. By Agios Pharmaceuticals.Note: AML = Acute Myeloid Leukemia; GIST = Gastrointestinal Stromal Tumor; SM = Systemic Mastocytosis; NSCLC = Non-small Cell Lung Cancer; MTC = Medullary Thyroid Cancer; HCC

= Hepatocellular Carcinoma.

Four Highly Innovative Assets for Greater China:Two First-in-class and Two Potentially First-in-class

CS3010(AG-120, Ivosidenib)

CS3007(BLU-285, Avapritinib)

CS3009(BLU-667)

CS3008(BLU-554)

Target Indication

IDH1m AML

IDH1m Cholangiocarcinoma

GIST

SM

RET-altered NSCLC

RET-altered MTC

HCC

Molecular Target

IDH1m KIT and PDGFRα RET FGFR4

Market Opportunity

The number of patients addressable by IDH1 inhibitors in China in 2017 was ~1,600 for AML and ~6,900 for cholangiocarcinoma

The number of patients addressable by KIT and PDGFRα inhibitors in Chinain 2017 was ~20,900 for GIST and ~1,300 for SM

The number of patients addressable by RETinhibitors in China in 2017 was ~11,300 for NSCLC and ~3,000 for MTC

The number of patients addressable by FGFR4inhibitors in China in 2017 was ~53,100 for HCC

First-in-classPotential

The first and only FDA-approved1 therapy for patients with R/R AML and an IDH1 mutation

The only IDH1m inhibitor under clinical development in China currently

Globally, there is nomarketed PDGFRα-selective inhibitor

The only PDGFRα inhibitor under clinical development in China

Globally, there is no marketed RET-selective inhibitor

Globally only two drug candidates selectively targeting RET, BLU-667 and LOXO292 (by Loxo Oncology)

Globally, there are currently no marketed FGFR4 inhibitors, however some FGFR4 inhibitor drug candidates are undergoing clinical development

FIRST-IN-CLASS POTENTIALLY FIRST-IN-CLASS

11

Note: AML= Acute Myeloid Leukemia, SM = Systemic Mastocytosis, GIST = Gastrointestinal Stromal Tumor, HCC = Hepatocellular Carcinoma, NSCLC = Non-small Cell Lung Cancer, MTC = Medullary Thyroid Cancer, FIC = First-in-class, FPFD = First patient first dose

Avapritinib(KIT&PDGFRα)

BLU-667(RET)

BLU-554(FGFR4)

(IDH1)

Greater China

Status

• Accepted for a China bridging study for R/R IDH1m AML

• Approved to join global Ph3 trial for 1L IDH1m AML

• NDA submission for R/R AML in Taiwan in Jun 2019, priority reviewstatus granted

Asset

• Approved to join global Ph3 trial for GIST in China

• Approved to initiate a China bridging study in GIST with PDGFRαD842V mutation in China

• Approved to join global Ph1 trial for NSCLC and MTC in China

• Consider to initiate Basket cohort in other niche indications

• Approved to join global Ph1 trial for HCC

• Combo with PD-L1 IND approved in China

• Achieved FPFD in HCC mono in May 2019

Highlight• Global FIC• US FDA approved• US FDA designations:

Breakthrough therapy for IDH1m AML

• Global FIC• US FDA designations:

Breakthrough therapy, Fast track for GIST and SM

• Potentially Global FIC• US FDA designation:

Breakthrough therapy for NSCLC and MTC

• Potentially Global FIC

Achieved FPFD in BLU554 one month ahead of schedule; expect FPFD in the other 3 assets by 19Q3

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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: full human with potentially less ADA and tox

Potentially CStone’s first self-developed drug to be approved in China

One of the first domestic anti-PD-L1 drugs to be launched in China

500+ patients dosed across 7 trials as of May 31, 2019

Overview

Highlight

#1: stage IV cervical cancer with lymph node metastases; 1200 mg Q3W cohort; BOR is PR (SLD decrease of 69%, response was ongoing)

#2: stage IV MSI-H ampullary carcinoma with retroperitoneal lymph node metastases; 3 mg/kg Q3W cohort; BOR is PR (SLD decrease of 62%, response was ongoing)

#3: stage IV NSCLC and adenocarcinoma with multiple extrathoracicmetastases; 20 mg/kg Q3W cohort; BOR is PR (SLD decrease of 57%, response was ongoing)

#4: stage IV cholangiocarcinoma with liver and retroperitoneal lymph node metastases; 20 mg/kg Q3W cohort; BOR is PR (SLD decrease of 44%, response was ongoing)

#5: stage IV mixed histology of esophagus cancer and malignant melanoma with lymph node metastases; 1200 mg Q3W cohort; BOR is PR (SLD decrease of 32%, response was ongoing)

Source: Company, presented at 2019 ASCONote: 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

China’s First Full Human, Full-length IgG4 mAb: Proving Safe And Efficacious In Early Clinical Trials

13

Comprehensive Winning Strategy for Our PD-L1 from Three Dimensions

Source: GBI; Clinicaltrials.gov; Frost & Sullivan analysis; Chinadrugtrials.org; press search; company websites1 Advanced NSCLC, HCC, GC2 SOC = Standard of Care3 Company is evaluating both PD-L1 and PD1 as combo partner for this indicationNote: NSCLC = Non-small Cell Lung Cancer; HCC = Hepatocellular Carcinoma; GC = Gastric Cancer

Cancer Type Indication Treatment

Ranking in Domestic Competitors

NSCLC

1st Wave

HCC3 1st Wave

Gastric cancer 1st Wave

~1

Consolidation therapy after chemoradiotherapy, Unresectable Stage III NSCLC

All comers, Stage IV NSCLC

Advanced HCC3

1L

PD-L1(+), GC1L

PD-L1mono

PD-L1+ SOC2

PD-L1 + SOC2

PD-L1 + SOC2

FAST TO MARKET MARKET OCCUPANCY MAXIMIZATION

COMBO FLEXIBILITYAND POTENTIAL

1st wave to launch in large indications1 covering 1.2 million of the cancer incidence in China

Our own internal assets

External assets

Adaptive Trial Design

Sensitive / Niche Indications

Leverage Overseas Sites

# OF POTENTIAL

COMBOS

PD-L1 combina-

tions

# OF ASSETS

1 2

3

14

Robust Business Model Propelled by Our Clinical Development Engine, with Rapidly Developing Commercial and Manufacturing Capabilities

MANUFACTURING: “CMO + IN-HOUSE” APPROACH

BUSINESS DEVELOPMENT

Transitioning toCommercial Stage

Partnership

Internal development

DEVELOPMENT COMMERCIALIZATION

Overcoming Clinical Development Bottleneck

RESEARCH

Dual Sources of Innovation

Suzhou Translational Medicine Research Center

Unparalleled clinical development engine in China

Partnership

In-house

15

Unparalleled Clinical Development Engine with a Proven Track Record

CLINICAL DEVELOPMENT STRATEGY

Front-loaded Trial Design

FIH/POC

Pivotal ✓Accelerated

Approvalvs. Conventional: Ph I / Ph II

Streamlined, Parallel decision making

Predefined Go/No-Go Criteria

vs. Conventional Internal Review and Planning Steps

Strategic Choice of Trial Location

1/2 Time to Human Data

Data Credibility

Combo Potential

Accelerate China Development

CLINICAL OPERATIONAL EXCELLENCE

Seamless Operational Collaboration

OutsourceDay-to-day Execution Activities

Keep Strategy/ Oversight Monitoring In-house

Medical SciencePharmacovigilanceClinical OperationBiometrics

Industry Leading Team

IN-HOUSETRANSLATIONAL

RESEARCH CAPABILITY

Pre-clinical Clinical

Use biomarkers and preclinical discoveries to guide patient selection and predict treatment response in clinical trials

Increase success rate of clinical trials

Use clinical findings to guide preclinical discovery of drug resistance mechanisms

Frank Jiang, MD, PhDChairman and CEO

21,000 patient mega-trial79 clinical trials30 NDA obtained

US Transferability

Jason Yang, MD, PhDCMO

Led numerous global and China trials; designed and conducted several PD-(L)1 and combos trials

Archie Tse, MD, PhDCTMO

Over 20 years of global oncology experience

16

Translational Medicine Research Center in Suzhou (TMRC) – a part of our clinical development engine & a window to the world

1

Tissue banks & KOLs Expertise

Breakthrough Targets

Academic Labs

AcademicBioinformatics

Capabilities

DrugDiscovery

& Pipeline Support

BiomarkerDiscovery

&Translation Research

>1,000 Biotech & Pharma companies

Proven Drug Discovery Platforms

@CROs

Advanced Genomic data collection tools

17

Dual Sourcing of Innovation Through Internal Development and External Partnership

1

Competitiveness

Druggability

Novelty

Scientific rationale

Clinical evidence

R&D / Partnership MeetingsDatabaseKOL

Connections Literature Academia Network

External Partnership

Internal Development

18

Exclusive Regional Licensing Agreement For A Next Generation IO Therapy That Could Replace Current PD-(L)1 Therapies

Highlight

• 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

• Access to Numab’s novel multi-specific technology platform

• Complement to CStone’s IO strategy

ND021 tethers 4-1BB and activates T cells only upon engagement of PDL1-expressing tumor cells

Monovalent without Fc

19

Pipeline 2.0 for Sustained Growth of CStone

1

Advanced Portfolio

Precision Medicine

De-risked I/O Backbone & Assets

GAP Analysis Pipeline 2.0

New Targets (3-5 years)

Pipeline 1.0

Balanced Portfolio

Convincing combo activity with PD-(L)1 or pipeline 1.0

candidates?

Promising “practice changing” potential? FIC or BIC multispecific

mAbs/scaffolds TME modulators to

maximize PD-(L)1 efficacy Cancer vaccines Novel pathway inhibitors“Disruptive” biology?

To build a world class multi-billion dollar oncology company, we must have a world class pipeline, know the gaps and fill them with innovative drug candidates for sustained growth;

Aim to deliver 1-2 IND each year

Validated & Novel I/O Combination

Portfolio Prioritization

20

Partnership with Leading Biopharmaceutical Companies Globally

Avapritinib Globally first-in-class

BLU-554 (FGFR4) BLU-667 (RET)

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

… More to come

Ivosidenib FDA approved Globally first-in-class

Crown Jewel Assets from World Class Partners

“Best of Both Worlds“Value Proposition

Well Positioned asLeading Partner

ND021 Next generation IO Potential BIC

LARGE MULTINATIONAL

PHARMA

CHINA-DOMICILED

PHARMACStone

21

Stage 2

Stage 1 2018-2021

2021-2023

Stage 3 2023+

Mature asset or team (opportunistic)Buy mature asset / whole team (sales, logistics, etc.) and executive to accelerate commercial set up

Buy

Large indication in China and all indications ex-ChinaFirst large potential and highly competitive product CS1001 (PD-L1), leverage partnership to maximize value and learn from partner

Only ex-China Selectively ex-China

Part

ners

hip

Mature asset or team (opportunistic) Mature asset or team (opportunistic)

Small indication in ChinaHighly innovative FIC products (Ivo, Ava) as the foundation, more concentrated, scientific and biomarker driven, well suited for properly setting up a professional in-house team

Fully in-house in China

Fully in-house in China and mostly in-house global

In-h

ouse

Started Building Commercial Capabilities in China in Anticipation of Product Launches

22

Hybrid Manufacturing Model

CMO Outsourcing Planned In-house Manufacturing Facilities

• Outsourcing the production of drug candidates to a limited number of industry-leading, highly reputable CMOs

• To secure product supply for R&D needs and potential product launches

• For ivosidenib (CS3010) and avapritinib (CS3007), we will import drug products from our licensing partners at the initial commercialization stage

Small Molecules Biologics

Compliance with GMP requirements in China and globally

Planned capacity of biologics plant design: 10,000L (4x2,000L and 2x1,000L)

Stay Flexible Win on Speed

StayFocused on

Clinical Development

Better Quality Control

DriveDownCost

Protection of Key Know-how

Compliance Monitoring

Efficient Planning

Long-term Stable Supply

Value Creation

Hybrid Manufacturing Strategy for Both Small Molecules and Biologics

23

23

Series B

$150 Million

$262 Million

Series A

Record Breaking1

Record Breaking1

2016/07 2018/05 2019/02

HKEXIPO

$328 Million2

Other global institutional

investors (undisclosed)

Cornerstone investors

Record Breaking Financing by Prestigious Investors

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

24

Near-term Catalysts

2019

Initiate a China bridging study in the indication of GIST with PDGFRa D842V mutation for registration

Initiate the China portion of a phase III trial in the indication of 3L GIST for registration

Expect Blueprint to publish clinical data for GIST at academic conference (ASCO, CTOS)

Expect Blueprint to publish clinical data for SM at academic conference (EHA, ASH)

Potentially obtain IND approval for 2L GIST

Avapritinib Initiate a registrational trial in

China for the indication of 1L IDH1m AML by joining the global study

Initiate a China bridging study in the indication of R/R IDH1m AML for registration

File a China bridging study in the indication of IDH1m Cholangiocarcinoma for registration, pending partners’ data

Expect to submit NDA in Q2 for R/R IDH1m AML in Taiwan

IvosidenibPD-L1 Initiate 2 phase III trials (GC,

EC) in China and/or globally for registration

To publish clinical data at academic conference (ASCO, ESMO and CSCO)

Initiate combo trial with PARP

Initiate combo trial with FGFR4

Initiate combo trial(s) with VEGFRi

FGFR4

PD-1

CTLA-4

HDAC6

MEK

CDK4/6

1. Initiate the China portion of a phase I trial (PoC) in the indication of HCC 2. Initiate a phase I trial (PoC) in the indication of HCC for FGFR4 in combo with PD-L1

1. Expand clinical study into the US 2. To publish clinical data at ESMO annual meeting 3. Initiate a pivotal study in China based on data from phase I study (HCC or ES-SCLC)

1. Initiate the dose escalation portion of combination with PD-1 in the ongoing Australian phase I study 2. Initiate phase I study in China in patients with advanced tumors

1. Initiate the dose escalation portion of combination with PD-1 in the ongoing Australian and China phase I studies 2. To publish clinical data at ESMO and CSCO

1. Initiate a phase I trial in patients with advanced solid tumors and multiple myeloma as monotherapy and combination with PD-(L)1 in China and Australia

1. Initiate a phase I trial in patients with advanced solid tumors as monotherapy and combination with PD-(L)1 in Australia and China

Initiate the China portion of a global trial in the indications of RET fusion driven NSCLC, MTC for registration

Consider to initiate Basket cohort in other niche indications

RET

Legend• On track• Update• New

Thank you!

26

Press Releases since IPO Scientific Advisory Board Appointed three distinguished oncology leaders Drs. Paul A. Bunn, Jr., Elizabeth M. Jaffee and Richard S. Finn to Scientific

Advisory BoardBusiness development Exclusive regional licensing agreement with Numab for a next generation IO therapy that could replace current PD-(L)1

therapiesCS1001 (PD-L1) First patient dosed in Phase III GEMSTONE-303 study for CS1001 in combination with chemotherapy in first-line gastric

adenocarcinoma and gastro-esophageal junction adenocarcinomaCS1003 (PD-1) Presented pre-clinical data of CS1003 at the 2019 American Association for Cancer Research (AACR) Annual Meeting Received IND approval in China for combination trial with CS3008 (FGFR4)CS3003 (HDAC6) Received IND approval in China for HDAC6 inhibitor CS3007 (KIT & PDGFRα) Received approval in China to join global phase III clinical trial for GISTCS3008 (FGFR4) Dosed first patient in China as part of global phase I clinical trial in late stage HCCCS3009 (RET) Received approval in China to join global phase I clinical trial for RET Inhibitor BLU-667 CS3010 (IDH1) Agios received FDA Breakthrough Therapy designation for TIBSOVO® (ivosidenib) in combination with Azacitidine for the

treatment of newly diagnosed acute myeloid leukemia (AML) with an IDH1 mutation in adult patients ineligible for intensive chemotherapy

Agios reported updated data from phase I study of ivosidenib in combination with Azacitidine demonstrating deep and durable responses in newly diagnosed IDH1 mutant acute myeloid leukemia (AML) patients

Agios announced FDA acceptance of supplemental New Drug Application for TIBSOVO® (ivosidenib) for the treatment of patients with newly diagnosed acute myeloid leukemia with an IDH1mutation not eligible for standard therapy

Agios Announces the Randomized Phase 3 ClarIDHy Trial of TIBSOVO® (ivosidenib) Achieved its Primary Endpoint in Previously Treated IDH1 Mutant Cholangiocarcinoma Patients