2021 Interim Results Presentation - Sep 2021
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2616.HK
2021 Interim Results Presentation
Sep 2021
1
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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
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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
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2
Introduction & Overview
3
Introduction & Overview
2021YTD Business Highlights
Successfully launched two FIC products, achieving significant sales in a short
period of time. AYVAKIT® (avapritinib) launched in mainland China and Taiwan, China in
Commercial May and June, respectively. GAVRETO® (pralsetinib) launched in mainland China in
June. Together, they generated revenue* of RMB79 million by 30 June
Primary endpoint met for sugemalimab in stage III NSCLC in “all-comers” setting.
Clinical Secured industry-leading 3 NDA approvals to facilitate the above launches, and
Development submitted 4 NDAs covering a third FIC product – ivosidenib – and indication /
geographic expansion for pralsetinib, avapritinib and sugemalimab
Selected Pfizer’s lorlatinib for co-development in China, deepening the partnership
Business established in 2020, and accelerated registration of sugemalimab in multiple
Development countries ex China with EQRx
IND accepted for CS2006 (PD-L1×4-1BB×HSA tri-specific) in China, and
Pipeline 2.0 completing IND-enabling studies of CS5001 (ROR1 ADC) for global submission
Capital Markets Included in Hang Seng Composite Index and Hong Kong Stock Connect
4
Note: * To-market sales. NDA = New Drug Application, IND = Investigational New Drug, NSCLC = Non-small Cell Lung Cancer, AML= Acute Myeloid Leukemia
Introduction & Overview
2616.HK
Full-fledged Biopharma
5 Years Since Company Inception
Industry leading management Solid financial position and
team with proven track record ability to support growth
and complementary expertise initiatives
Well-balanced oncology portfolio with 15 immuno- Full-fledged biopharma with a premier clinical
oncology and precision medicine assets, 7 of development engine, established commercial capabilities
which are at commercial/late stage and developing manufacturing capability
Today, CStone has achieved commercialization of multiple products and
expanded the global dimensions of its business, establishing its position among
the leading biopharmas in China
5
Introduction & Overview
Industry leading management team
Leadership with proven track record and complementary expertise
Frank Jiang, MD, PhD
Chairman, Chief Executive Officer
Shirley Zhao, MD, MBA Jason Yang, MD, PhD
Greater China GM Chief Medical Officer
Archie Tse, MD, PhD Jingrong Li, PhD
Chief Scientific Officer Chief Technology Officer
Sanhu Wang, MPH Michael Choi, MBA
SVP, GA & RA Chief Business Officer
Note: GA=Government Affairs; RA=Regulatory Affairs 6
Introduction & Overview
Well-balanced oncology portfolio of 15 innovative assets
Focused on immuno-oncology and precision medicine
Approval
Drug candidate Rights Indication Pre-clinical FIH POC Pivotal NDA Marketed Partner
CN5 TW HK US
2L NSCLC
Pralsetinib 1L NSCLC
(RET) 1L MTC / TC
Multiple tumors
PDGFRA exon 18
Avapritinib GIST
(KIT/PDGFRA)
AdvSM1
1L Stage IV NSCLC
Stage III NSCLC
Sugemalimab Out- 1L GC Mainland China
(PD-L1) licensed 1L ESCC
R/R ENKTL
Ex-Greater China
R/R ENKTL
R/R AML
Ivosidenib (IDH1)
1L AML
CS1003 (PD-1) Ex-Greater
1L HCC China
Lorlatinib 4
(ROS1/ALK) NSCLC
(ALK)
Fisogatinib (FGFR4) HCC
CS1002 (CTLA-4) Solid tumors
CS20062
Solid tumors
(PD-L1/4-1BB/HSA)
CS3002 (CDK4/6) Solid tumors
CS3005 (A2aR) Solid tumors
Solid tumors
CS50013 (ROR1) hematologic
malignancies
CS2007 (Undisclosed
Solid tumors
Multi-specific)
CS2008 (Undisclosed
Solid tumors
Multi-specific)
CS5002 (Undisclosed
Solid tumors
ADC)
Greater China Global Expedited
Note: Assets status denote progress in the region noted in the column titled “Rights”; CN = Mainland China, FIH = First in Human POC = Proof of Concept, NSCLC = Non-small Cell
Lung Cancer, MTC = Medullary Thyroid Cancer, TC = Thyroid Cancer, GIST = Gastrointestinal Stromal Tumor, AdvSM = Advanced Systemic Mastocytosis, GC = Gastric Cancer, ESCC registration
= Esophageal Squamous Cell Carcinoma, R/R = Relapsed or Refractory, NKTL = Natural KILLER/T Cell Lymphoma, AML= Acute Myeloid Leukemia, HCC = Hepatocellular Carcinoma Greater China Global
1.POC was conducted in the U.S. and no clinical trials have been conducted in China; 2.CS2006 is currently under PhI dose escalation study in Taiwan & IND preparation in mainland 7
China; 3.CStone obtains the exclusive global right to lead development and commercialization of LCB71/CS5001 outside the Republic of Korea; 4. Co-development in Greater China; 5.
Mainland China
Korea Singapore
Interim Business Review
8
Commercial 2 3 4 5 Interim Business Review
Established strong commercial foundation with seasoned
industry leaders
Supporting CStone's oncology leadership with successful launches
• 25+ years of experience in the industry (former Greater China GM of BMS, Allergan,
Genzyme)
• Built several organizations into industry leaders
• Deep experience spanning 30+ product launches and building leading brands in
oncology, rare disease & medical aesthetics
Shirley Zhao, MD, MBA
Greater China GM
Critical mass of CStone Commercial platform: > 300 FTEs by 2021
• Outstanding leadership team with diverse range
of experience at MNCs and innovative biotechs
• Strong track record with 30+ successful
launches in oncology & hematology
• Covering approximately 70-80% of potential
Sophia Lee Zezhou Wang Philip Chen markets for precision medicines
TW & HK Sales Broad Market Access
Note: MNC = multinational company; FTE = Full Time Equivalent
9
Commercial 2 3 4 5 Interim Business Review
Launches set industry benchmark for speed of delivery
Go-to market strategy enabled rapid and broad day one sales
MNC CStone
Quality Speed
GAVRETO® AYVAKIT®
Achieved rapid NMPA approval,
Time to reach distribution partners
6.5 months within a shorter timeframe than 4 days from time of arrival in China
typically required
Accessible in 64 cities within 30 Accessible in 52 cities within 30
64 cities provinces through DTPs on first day 52 cities provinces through DTPs on first day
Achieved product revenue* of RMB33.6 mn
Achieved product revenue* of RMB45.8 mn
since launch in Mainland China in May 2021
since launch in Mainland China in June 2021
and in Taiwan, China in June 2021
Note: * To-market sales. MNC = multinational company; Rx = prescription; NMPA = National Medical Products Administration; DTP = Direct-to-Patient 10
(pharmacy); TFDA = Taiwan Food and Drug AdministrationCommercial 2 3 4 5 Interim Business Review
Expanding accessibility of assets on the market
Commercial team taking additional steps to bolster sales growth
Reimbursement Coverage Scientific Influence
▪ 20 of the major government and commercial ▪ >7 national treatment & diagnosis guidelines
insurance plans have included avapritinib and Now include AYVAKIT®, GAVRETO®, TIBSOVO®
pralsetinib in their plans since launch and/ or testing
▪ ~40 million urban population covered with strong
reimbursement momentum
11Commercial 2 3 4 5 Interim Business Review
Bolstering commercial potential of pipeline developments
Preparing market for indication expansions and upcoming launches
Indication expansion of launched assets Launch preparation of more assets
GAVRETO® SUGEMALIMAB
• NSCLC – 1L RET • NSCLC – Stage III
fusion + (concurrent &
NDA filing to NMPA expected in sequential) Mainland China
the second half of 2021
NDA filed to NMPA in Aug 2021 Joint efforts with Pfizer
in
• MTC – RET-mutant • NSCLC – 1L Stage Ex-Greater China
NDA filing accepted by NMPA in in Mainland China
Joint efforts with EQRx
Apr 2021 IV (sq & nsq)
NDA filing accepted by NMPA in
• TC – RET fusion + Nov 2020
NDA filing accepted by NMPA in
Apr 2021
AYVAKIT® TIBSOVO®
• GIST – PDGFRA • r/r AML – IDH1+
in Mainland China
D842V mutant in Hong Kong, China
NDA filing accepted by NMPA in
Aug 2021
NDA filing accepted by HK DoH in
May 2021
12
Note: NMPA = National Medical Products Administration, HK DoH = Hong Kong Department of HealthCommercial 2 3 4 5 Interim Business Review
Preparing Sugemalimab for full-scale commercial launch
Harnessing Pfizer’s commercial leadership/infrastructure in China
US$200mn equity investment with three paths for collaboration
Commercialization of Co-development of
1 Sugemalimab 2 Pfizer assets 3
▪ Up to $280mm in milestone payments ▪ Two post-PoC oncology assets ▪ Jointly in-license for Greater China
▪ Tiered, mid-to-high teens royalties ▪ CStone to receive double-digit royalties ▪ CStone retains option for co-promotion
Positioning & Pricing & Go-To-Market Key
Adoption Market Access Model Differentiation
Next generation Competitive local Broadly cover The ONLY PD-(L)1
PD-(L)1 with global pricing; targeting >4,600 hospitals with superior efficacy
branding and new 2022 NRDL inclusion (~90% of market) & safety profile for
standard of care to maximize patient both stage III&IV
for 1L I/O suitable accessibility NSCLC patients
patients
Collaboration achieved Work in progress 131 Clinical 3 4 5 Interim Business Review
Unrivalled clinical development engine
Robust strategy, innovative trial designs and agile execution
Jason Yang, MD, PhD, Chief Medical Officer
➢ A physician scientist and senior executive with 25+ years biomedical research and biopharma R&D experience in oncology
➢ Led 60+ global and China trials, brought over 5 assets (tislelizumab, zanubrutinib, pamiparib, avapritinib and pralsetinib) to
market, including zanubrutinib to global market, and 2 additional market approvals pending (sugemalimab and ivosidenib)
➢ Built Beigene’s and CStone’s Clinical Development team & established efficient project centric work models
➢ Ph.D trained with Nobel laureates Dr. Mike Brown and Joseph Goldstein at UT Southwestern Med. Ctr.; Postdoctoral training
with Dr. Stuart Schreiber at Harvard University
Innovative Clinical Development Strategy to Set New Track Record in China
Indication strategy: focus on China’s largest Covering 50%+ of total cancer incidences: lung cancer,
indications with unmet need gastric cancer, liver cancer and esophagus cancer
Adaptive ph I/II design: seamless transition from Three years from ph I first patient dosed to first NDA filing for
dose escalation to multiple POC studies sugemalimab
2 pathologies in one trial: squamous + non-squamous
Innovative ph III trial design to accelerate NDA
2 treatment modality population in one trial: concurrent +
submission in large indications
sequential
Cost effective bridging strategy for accelerated Avapritinib and pralsetinib approved in China two years after
approval of in-licensed assets in Greater China IND approval
141 Clinical 3 4 5 Interim Business Review
Poised for rapid growth with numerous clinical successes
Industry leading number of NDA submissions, approvals, data readouts
Roadmap for new product
Indication & geographic expansion of launched products Ivosidenib
AML (R/R)
Pralsetinib Avapritinib ▪ NDA accepted in Mainland China
for R/R AML with priority review
NSCLC (1L/2L) GIST
▪ Positive data in Chinese R/R
▪ Positive data from registrational study ▪ NDA accepted in Hong Kong for
AML patients accepted for oral
in 1L RET fusion-positive NSCLC, with PDGFRA D842V mutant GIST
presentation in ESMO 2021
1L/2L NSCLC data accepted for ▪ Oral presentation for GIST in ESMO
presentation in WCLC 2021 GI 2021 AML (1L)
MTC (RET-mutant) AdvSM ▪ Servier announced positive
▪ Positive data from registrational study topline data from the global
▪ In discussion with CDE regarding
in RET-mutant MTC phase 3 study in combination
accelerated registration pathway
▪ NDA accepted in Mainland China for for AdvSM with Azacitidine in patients with
RET-mutant MTC with BTD and previously untreated IDH1-
▪ Approved in U.S. mutated AML
priority review
ISM
TC (RET fusion-positive)
▪ BTD granted by U.S. FDA for Cholangiocarcinoma
▪ NDA accepted in Mainland China for moderate to severe ISM ▪ Approved in U.S.
RET fusion-positive TC with priority
review ▪ Registrational trial data expected in ▪ Exploring China bridging
2022 strategy
Basket trial
Others
▪ Ongoing trial with registration potential
▪ Exploring additional indications, i.e.
AML, by partner
Note:
BTD = breakthrough designation, NSCLC = Non-small Cell Lung Cancer, MTC = Medullary Thyroid Cancer, TC = Thyroid Cancer, GIST = Gastrointestinal Stromal Tumor,
CDE = Center for Drug Evaluation, AdvSM = Advanced Systemic Mastocytosis, ISM = Indolent Systemic Mastocytosis, r/r = relapsed or refractory, AML = Acute Myeloid 15
Leukemia, WCLC = World Conference on Lung Cancer, ESMO = European Society for Medical Oncology1 Clinical 3 4 5 Interim Business Review
Expanding addressable patient population for key assets
Trials for additional indications to unlock full commercial potential
~90,000
1L NSCLC
Pralsetinib
Avapritinib 2L NSCLC
Ivosidenib x225% MTC
PTC
Basket trial
GIST (mPDGFRα）
AdvSM
~40,000 ISM
x400% 1L NSCLC
2L NSCLC
Others1
MTC r/r AML
PTC
Basket trial 1L AML (IC ineligible)
GIST (mPDGFRα） CCA
~10,000 AdvSM
r/r AML MDS
2L NSCLC 1L AML (IC ineligible)
GIST (mPDGFRα） CCA Others2
2021 2022 – 2023 2023 and beyond
Source: Clarivate DRG; Globocan 2020; CStone analysis; NEJM
1. Esophageal cancer, Hepatocellular carcinoma, NSCLC, Cervical cancer, etc.; 2. Colorectal cancer, Prostate cancer, Glioblastoma, Melanoma, etc.
NSCLC = Non-small Cell Lung Cancer, MTC = Medullary Thyroid Cancer, PTC = Papillary Thyroid Cancer, GIST = Gastrointestinal Stromal Tumor, AdvSM =
Advanced Systemic Mastocytosis, ISM = Indolent Systemic Mastocytosis, R/R = Relapsed or Refractory, AML= Acute Myeloid Leukemia, CCA = 16
cholangiocarcinoma; MDS = Myelodysplastic Syndromes1 Clinical 3 4 5 Interim Business Review
Sugemalimab poised to reshape lung cancer landscape
Only PD-(L)1 with efficacy in stage III & IV NSCLC in “all-comers” setting
The first PD-(L)1 to cover the entire advanced Positioned to become physicians’ preferred
NSCLC through innovative trial design PD-(L)1 for its broad applicability and safety
■ Differentiated design: Fully-human, full length IgG4 derived from
GEMSTONE-301 study Ligand’s OmniRat® platform – minimalizing ADA occurrence;
• First ph III trial to cover patients with either retaining ADCP activity for potentially enhanced efficacy
concurrent or sequential chemoradiotherapy in ■ Outstanding efficacy:
one trial, reflecting real-world clinical
practice and covering a broader population • The first PD-L1 in combo with chemo to demonstrate clinical
efficacy in both sq and nsq stage IV NSCLC patients, with
• Detailed data to be presented in 2021 ESMO PFS HR of 0.48, among the best of all competitor PD-(L)1
• The first PD-(L)1 to demonstrate PFS improvement in
patients with stage III NSCLC following concurrent or
sequential chemoradiotherapy
GEMSTONE-302 study • Superior clinical efficacy also observed in ESCC, GC, R/R
ENKTL, etc. “Breakthrough Designation” granted by both
• First ph III trial in China to cover 1L patients FDA and CDE
with both squamous and non-squamous NSCLC
in one trial vs two separate trials, with Hazard ■ Commercial opportunity:
Ratio further improved in the final analysis* • I/O drug of choice for advanced stage NSCLC
- PFS (HR: 0.48; mPFS: 9.0 vs. 4.9 months) • ~850K addressable stage III & IV NSCLC patients in
- OS (HR: 0.67; mOS: 22.8 vs. 17.7 months) China/US/EU5/Japan
• Detailed data to be presented in 2021 WCLC • Large addressable patients targeted ongoing registration trial
in ESCC, GC and R/R ENKTL
Stage III NSCLC: China NDA filed in August 2021
Stage IV NSCLC: China NDA approval expected in Q4 2021
Working closely with EQRx on NDA submission in multiple countries with U.S. BLA filing expected in 2022
17
Note: Data cutoff: Mar 15th, 20211 Clinical 3 4 5 Interim Business Review
Accelerated clinical advancement of sugemalimab
Entered the 1st tier of NSCLC from the 12th place with “CStone Speed”
From IND to Stage IV NDA approval Cover both Stage III and Stage IV NSCLC
1st PD-(L)1
for the treatment
of both stage III
4th PD-(L)1
and IV NSCLC in
all-comer setting
approved in both
sq & non-sq stage Est. 2022 Q4
Iv NSCLC with
BIC potential
Take the lead in
Est. 2021 Q4 advancing I/O therapy to
12th clinical stage III NSCLC patients
by ~2 years
PD-(L)1 in China
Serve Stage IV NSCLC
patients with efficacy
2017 Q4 and safety advantages
181 2 BD 4 5 Interim Business Review
Growing BD team expands global partnership network
Deeper capabilities to support pipeline and commercialization efforts
Michael Choi, Chief Business Officer
➢ 23+ years of experience in biopharmaceutical strategy and business development
➢ Executive experience with Pfizer and Sun Pharma Advanced Research Company
➢ 40+ transactions across 6 continents totaling multiple billion dollars in transaction value
Expanded Global Coverage
Seattle Beijing
New York
New Jersey
Shanghai
Expanded BD Priorities
Commercial BD Pipeline Building Strategic Partnerships
Drive near term cash flow Address clinical unmet need / Build up company capabilities /
and ROI on Field Force Generate clinical catalysts Evolve into a global player
• Commercial partnerships • China: Clinical post-POC assets • Multi-specific, ADC, AI
• Accretive near market assets • Global: Paradigm shifting preclinical assets discovery platforms
(e.g. FIC/BIC/FW, multi-specific, ADCs, etc.) • Bio-incubator programs
191 2 BD 4 5 Interim Business Review
Further deepening our strategic partnership with Pfizer
Agreement to co-develop Lorlatinib strengthens lung cancer offering
US$200mn equity investment with three paths for collaboration
Commercialization of Co-development of
1 Sugemalimab 2 Pfizer assets 3
▪ Up to $280mm in milestone payments ▪ Two post-PoC oncology assets ▪ Jointly in-license for Greater China
▪ Tiered, mid-to-high teens royalties ▪ CStone to receive double-digit royalties ▪ CStone retains option for co-promotion
Lorlatinib (ROS1/ALK)
Sizable patient Significant unmet Post-PoC asset Pioneering
population clinical need with high PoS clinical program
Over 670K diagnosed No approved targeted Demonstrated potent World’s 1st pivotal
incidence of NSCLC therapies in TKI and selective study of Lorlatinib on
in China, 2-3% of refractory setting, and inhibitory activity ROS1 positive
which are ROS1+ limited efficacy of against ROS1- patients in TKI
existing treatment for positive advanced refractory setting with
patients with brain NSCLC IND filing expected
metastases in Q4 2021
Note: TKI = Tyrosine Kinase Inhibitor, PoC = Proof-of-Concept, PoS = Probability of Success Collaboration achieved Work in progress 20
Source: Clarivate DRG; Zhang et al. Prevalence of ROS1 fusion in Chinese patients with non small cell lung cancer, Thorac Cancer 2019 10(1): 47–53.1 2 BD 4 5 Interim Business Review
Preparing sugemalimab for global launch
Leveraging EQRx’s business model to penetrate PD-L1 market
US$150mn upfront payment, up to US$1.15bn milestone
payments and tiered double-digit royalties on net sales
~US$30bn in market value1
(NSCLC, Gastric, Esophageal)
Rest of
US UK EU
the world2
First BLA filing Regulatory communications underway
in 2022
Working closely with EQRx to accelerate the registration of PD-L1 for NSCLC indications, and
collaborating for PD-L1 in other indications and PD-1 in HCC
Source: EQRx roadshow presentation, data based on EvaluatePharma July 2021 and Cowen PD(L)1 market model update Dec 2019
Note: 1. Global drug spend reflects 2026 estimated net prescription drug sales 211 2 3 Pipeline 2.0 5 Interim Business Review
Translating innovation into safe and effective therapies
Accelerating the development of assets from lab to post-POC stage
Archie Tse, MD, PhD, Chief Scientific Officer
➢ 20+ years of experience in translational oncology research covering cytotoxics, targeted agents, and immunotherapies
➢ Oncology TA Head at Daiichi-Sankyo, led the Phase 3 study of pexidartinib, the first systemic therapy approved for
tenosynovial giant cell tumor
➢ Oversaw early development of 10+ IO assets of different MOAs and modalities at MSD
➢ M.D-Ph.D, University of Southern California; faculty at Memorial Sloan-Kettering Cancer Center
Abundance of sources for In-house infrastructure in place Sustainably deliver
cutting-edge drug dev. ideas for implementation innovative drugs
Clinical
Observations
Kanglin Trans-
SAB Research
Protein DMPK & lational
Institute Chemistry Toxicology Science/ Pipeline 2.0
Biomarker
BD
partners
FIC/BIC/FW
Pharma
iSAT Project Molecular
cology/
Mgmt. Diagnostics
QSP Global rights
CRO
platforms
1~2 INDs per year
Clinical Bioinfor- Early
Conferences CI
Pharmaco matics Dev.
JITRI logy
Note: SAB = Science Advisory Board; JITRI = Jiangsu Industrial Technology Research Institute; FIC = First in class; BIC = Best in class; FW = First wave; 22
iSAT = innovation sourcing and alignment team; QSP = Quantitative System Pharmacology1 2 3 Pipeline 2.0 5 Interim Business Review
Harnessing full potential of next-gen candidates
Significant progress developing portfolio of FIC/BIC/FW assets
ADC PCC Declaration IND Submission
CS5001 (ROR1 ADC)
1 of top 3 ADCs globally and the first ADC in China Pre-IND
CS5002 (ADC)
Potential BIC with global rights Close to PCC
Multispecific
CS2006 (PD-L1 x 4-1BB x HSA) IND filling accepted
2021/7
Potential BIC with rights in Greater China, Korea and Singapore
CS2007 (Multi-Specific)
Global FIC with global rights Discovery / Pre-PCC
CS2008 (Multi-Specific)
Global FIC with global rights Discovery / Pre-PCC
231 2 3 Pipeline 2.0 5 Interim Business Review
ROR1 ADC: an asset with transformational potential
FIC/BIC asset in new modality with highly differentiated features
Differentiation In Design Clinical & Business Value
Potentially wider ➢ Potential applications for a wide range
therapeutic window of tumor types
Fully human mAb v.s. • NSCLC, TNBC, ovarian cancer, leukemia, NHL
Controllable quality humanized mAb in VLS-
and production 101 and NBE-002 • Over 3M annual incidence globally
Site-specific conjugation Proprietary tumor- ➢ Early promising data have led to
for a homogeneous selective cleavable extremely high transaction value in
drug antibody ratio linker, highly stable in
(“DAR”) (DAR=2) serum
ROR1 related deals
• Merck acquired VelosBio for $2.75 Bn
Tumor-activated PBD Core asset: VLS-101 (phase I/II)
dimer toxin prodrug • BI acquired NBE for $1.4 Bn
Core asset: NBE-002 (phase I)
Leading Position
1 of top 3 ADCs globally and the first ADC in China
IND filling in US IND filling in Potential accelerated Limited pricing
& AU expected by China expected registration path pressure maximizes
2021 in 1H 2022 Fast to market and cost- potential commercial
efficient development return of the asset
Note: NSCLC = non-small-cell lung cancer, TNBC = triple negative breast cancer, NHL = non-Hodgkin lymphoma, PBD = pyrrolobenzodiazepine; 24
FIC=First in class, BI = Boehringer Ingelheim1 2 3 4 Finance & Capital Markets Interim Business Review
Healthy financial profile supported by solid execution
Well capitalized for growth initiatives and commercial stage operations
• RMB2,447.2 million of cash, cash equivalents and time deposits
Cash Balance
as of June 30, 2021
• Product revenue*: RMB79.4 million
- GAVRETO (pralsetinib): RMB45.8 million (launched in Mainland
China in June 2021)
Key Income - AYVAKIT (avapritinib): RMB33.6 million (launched in Mainland
Statement China and Taiwan, China in May 2021 and June 2021 respectively)
Metrics
• R&D expenses (non-IFRS): RMB444.8 million
• Administrative, selling and marketing expenses (non-IFRS):
RMB214.3 million
25
Note: * To-market sales1 2 3 4 Finance & Capital Markets Interim Business Review
Expanding capital markets access
Inclusion in key index to support share trading and broaden ownership
• Included in Hang Seng Composite • Included in Hong Kong Stock Connect
Index on August 20, 2021, and effective on September 6, 2021
from September 6, 2021
Expecting improved liquidity, more efficient price discovery, and greater diversification of our
investor base, in particular more onshore institutional investors in mainland China
26Business Outlook
27Business Outlook
Vigorous pace of activity for remainder of 2021
Bringing significant clinical and commercial developments to fruition
Pipeline 2.0
Commercial Clinical
& Others
Preparation for new NDA approvals Data readout / presentation IND filings & approvals
product & indication launch • Sugemalimab stage IV NSCLC • Sugemalimab stage III NSCLC • ROR1 ADC IND / CTA filing in
in Mainland China oral presentation in ESMO U.S. / Australia
• TIBSOVO® r/r AML in Mainland
China • Ivosidenib Chinese R/R AML
• Ivosidenib R/R AML in • Lorlatinib IND filing in
oral presentation in ESMO
• GAVRETO® 1L NSCLC, MTC, Mainland China (by 2021/ Q1 Mainland China
TC in Mainland China 2022) • Sugemalimab stage IV NSCLC
oral presentation in WCLC • PD-L1/4-1BB/HSA IND
• GAVRETO® in other territories approval in Mainland China
• Pralsetinib 1/2L NSCLC oral
• AYVAKIT® in other territories NDA fillings presentation in WCLC
• Pralsetinib 1L NSCLC in • Pralsetinib MTC data
• Joint efforts with Pfizer for Mainland China Pilot operation of
launch of sugemalimab in presentation
• Pralsetinib 1L/2L NSCLC in manufacturing facility
Mainland China • Sugemalimab ENKTL data
Taiwan, China • Pilot operation of
presentation
• Pralsetinib 1L/2L NSCLC in manufacturing facility in
Broader patient access Hong Kong SAR, China Suzhou
• 10 commercial health insurance
plans and 15+ city insurance
• Tendering and hospital listing
for GAVRETO® and AYVAKIT®
28Introduction & Overview
Business outlook for 2022
Unlocking the global potential of our business and portfolio
Maximizing commercial potential with market expansion and penetration
Commercial
Target 5 NDA approvals of products
Expedite full slate of clinical development programs
Clinical
Expect 5+ NDA filings, 3 data readouts and 3+ registrational trial initiations, expanding our
Development presence in other high-prevalence cancers, along with the established lung portfolio
Drive innovative drug discovery and harness full potential of Pipeline 2.0
Pipeline 2.0 Submit IND for 1~2 highly-differentiated molecule(s) with FIC/BIC/FW potential and global rights,
and fully develop current suite of molecules, i.e., ROR1 ADC and PD-L1/4-1BB/HSA tri-specific
Business Support global ambitions with multi-dimensional partnerships
Pursue flexible deal structures for in-licensing and other partnerships to support pipeline
Development development and commercialization efforts in China and abroad
Prepare for pilot manufacturing operations
Manufacturing Submission for manufacturing site and material change and completion of technology transfer
for multiple products
29THANK YOU
2616.HK
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