34 slides from Beone Medicines Ltd. Class A's investor presentation, Aug 10, 2017
Slide 1: Disclosures
2 Disclosures Certain statements contained in this presentation and in the accompanying oral presentation, other than statements of fact that are independently verifiable at the date hereof, may constitute forward-looking statements. Examples of such forward-looking statements include those regardin
Slide 2: Investment Highlights
Investment Highlights Global oncology company with four internally developed assets, each with demonstrated proof-of-concept and a potentially differentiated profile Lead asset BGB-3111, a potentially best-in-class BTK inhibitor, is in registrational trials both globally and in China; PD-1 mAb in re
Slide 3: Our Business Model: Multiple Sources of Value Creation
Our Business Model Multiple Sources of Value Creation Broad Portfolio First-in-China Globally Differentiated Proprietary Combinations First-in-China Proprietary Combinations Globally Differentiated 1 2 3 Each program is supported by clinical or preclinical data that suggest potential for meaningful
Slide 4: China Oncology Market Already Large and Growing Rapidly; Structural Advantages as a Local Player
China Oncology Market Already Large and Growing Rapidly; Structural Advantages as a Local Player Already the second largest pharmaceutical market Three EGFR inhibitors combined had a revenue of $441M in 2016 Expanding reimbursement coverage could significantly increase commercial opportunity The lat
Slide 5: Company Growth and Expanding Capabilities
Company Growth and Expanding Capabilities 2013-2015 Entering the Clinic ~150 Employees 2011-2012 Foundation and Research ~100 Employees 2016-2017 Late-stage Development 400+ Employees Founding of company Research buildout Clinical development buildout initiated in China Established global clinical d
Slide 6: Strategic Collaboration With Celgene
Strategic Collaboration With Celgene BGNE to Acquire China Commercial Ops; CELG to License A317 in Major Markets ex-China Collaboration transforms BeiGene into commercial-stage company BeiGene to acquire Celgene’s operations in China BeiGene to license and assume commercial responsibility for Celgen
Slide 7: Building Manufacturing Capacity
Building Manufacturing Capacity Partnership with Guangzhou Development District for Biologics Manufacturing Joint venture (JV), BeiGene Biologics, formed with Guangzhou Development District (GDD) Between BeiGene Hong Kong and Guangzhou GET Technology Development, an affiliate of GDD To build a biolo
Slide 8: The BeiGene Team and Scientific Advisory Board
Ron Levy, M.D. Stanford, former Chief of Oncology The BeiGene Team Scientific Advisory Board Charles Sawyers, M.D. Past President of AACR. NCI Board of Scientific Councilors David Schenkein, M.D CEO of Agios; adjunct attending physician in hematology at Tufts Medical Center Neal Rosen, M.D., Ph.D. M
Slide 9: Clinical Pipeline: Lead Asset in Global Phase 3
In total, over 1,400 patients and healthy adults3 enrolled across four programs, including combination trials Clinical Pipeline: Lead Asset in Global Phase 3 Additional Registrational Programs Expected to Start in 2017 Program (Molecular Target) Commercial Rights Pre-clinical Dose Escalation Dose Ex
Slide 10: Combinations in Development
Combination (Mechanism) Pre-clinical Dose Escalation Dose Expansion* Registrational** Phase 1a Phase 1b Phase 2 Phase 2 Phase 3 BGB-3111 + Gazyva® (BTK + CD20) BGB-A317 + BGB-290 (PD-1 + PARP) BGB-A317 + BGB-3111 (PD-1 + BTK) Global Global Global External Combo Internal Combo 11 Combinations in Deve
Slide 11: BGB-3111 Overview
Overview Selective and potent BTK inhibitor (BTKi) Preclinical studies indicate higher selectivity against BTK than ibrutinib* Phase 1 data suggest higher drug exposure than ibrutinib** We believe BGB-3111 is the only BTKi that has demonstrated sustained target inhibition in disease originating tiss
Slide 12: Target Inhibition by Ibrutinib Appears Sub-Optimal
BGB-3111 Target Inhibition by Ibrutinib Appears Sub-Optimal Potentially better bioavailability and higher exposure of BGB-3111 may allow deeper target suppression in disease-relevant tissues *Animal studies Note: PBMC = Peripheral Blood Mononuclear Cell; Source: BeiGene data and Byrd et al, NEJM, 20
Slide 13: Pharmacokinetic Profiles of Lead BTK Inhibitors
Adapted from Advani et al., JCO, 2013 BGB-3111 Pharmacokinetic Profiles of Lead BTK Inhibitors Cmax and AUC of BGB-3111 at 80mg QD appear to be similar to those of ibrutinib at 560mg Free drug exposure of BGB-3111 at 40mg QD appears to be comparable to that of ibrutinib at 560mg Distinct profile com
Slide 14: Deep and Sustained BTK Inhibition Observed in Phase 1
BGB-3111 Deep and Sustained BTK Inhibition Observed in Phase 1 15 PBMC* Lymph Node Complete BTK inhibition in PBMCs at the starting dose (40mg) Paired lymph node biopsies were collected during screening or pre-dose on day 3 Median trough occupancy: 100% (160mg BID) vs 94% (320mg QD), p=0.002 Proport
Slide 15: BGB-3111 Phase 1 Data Suggest Deep Response in WM
Cross-Trial Comparison BGB-31112 (IWWM 2016) BGB-31113 (ASH 2016) BGB-31111 (14-ICML) Ibrutinib4 (Treon) Ibrutinib5 (PCYC-1127) Trial Phase Phase 1 Phase 1 Phase 1 Phase 2 Phase 3 sub-study Patient Number 24 32 42 63 31 Follow-up (med) 8.0 mo 9.6 mo 12.3 mo 19.1 mo* 17.1 mo VGPR 33% 34% 43% 16% 13%
Slide 16: Phase 1 Results Suggest High Activity in CLL/SLL
17 Treatment Naive (n = 16) Relapsed/ Refractory (n = 50) Total (n = 66) Median follow-up (range) 7.6 (3.7-11.6) 14.0 (2.2-26.8) 10.5 (2.2-26.8) Best Response ORR CR PR PR-L SD D/C prior to assessment 16 (100%) 1 (6%) 13 (81%) 2 (13%) 0 0 46 (92%) 1 (2%) 41 (82%) 4 (8%) 3 (6%) 1 (2%) 62 (94%) 2 (3%)
Slide 17: BGB-3111 Well-Tolerated in CLL/SLL and WM: Adverse Events
18 BGB-3111 Well-Tolerated in Phase 1 Trials in CLL/SLL and WM: Adverse Events Independent of Causality Most Frequent Adverse Events (>10%) Independent of Causality CLL (n=69)1 WM (n=48)2 AE, n (%) All Grade Grade 3-4 AE, n (%) All Grade Grade 3-4 Petechiae/purpura/contusion 32 (46%) 1 (1%) Petechia
Slide 18: BGB-3111 + Obinutuzumab: Does Not Impair ADCC
19 BGB-3111 + Obinutuzumab BGB-3111 Does Not Appear to Impair Rituximab-Induced ADCC 1 Data are from preclinical studies Source: Li N, et al. Cancer Res. 2015;75:2597 Published preclinical data suggest that off-target effects of ibrutinib may be detrimental to CD20 mAb-induced ADCC and the activity
Slide 19: BGB-3111 + Obinutuzumab Initial Data in CLL/SLL and FL
20 BGB-3111 + Obinutuzumab Initial Data Show CRs and High ORR in CLL/SLL and FL After a Short Follow-Up 1Occurred on July 20, 2017 Source: Tam et al., 14-ICML (abstract 103), 2017, presentation based on data cutoff of March 31, 2017 Follow-up and Response TN CLL/SLL (n = 18) R/R CLL/SLL (n = 25) FL
Slide 20: BGB-3111 Near-Term Development Plan
BGB-3111 Near-Term Development Plan Global Monotherapy China Global Combination Phase 1 CLL, WM, MCL, DLBCL, FL, MZL, HCL, RT Phase 1 B-cell Malignancies Phase 1b 3111 + GA101 CLL, FL, DLBCL, NHL Phase 1b 3111 + A317 FL, DLBCL+, 1° CNS lymphoma Phase 2 Mono in R/R MCL Phase 2 Mono in R/R CLL/SLL Pha
Slide 21: BGB-3111 Phase 3 Trial Design in WM
Stratification factors at randomization: CXCR4 status (WHIM vs WT) No. of lines of prior therapy (0 vs. 1-3 vs. >3) Inclusion Criteria: Age >18y Indication for treatment per IWWM ECOG 0-2 No prior BTK inhibitor Primary Endpoint CR/VGPR rate Secondary Endpoints MRR (>PR) PFS Duration of response Symp
Slide 22: BGB-3111 Phase 3 Trial Design in Treatment-Naïve CLL
Inclusion Criteria: Indication for treatment per iwCLL Unsuitable for treatment with FCR ECOG 0-2 No prior BTK inhibitor Primary Endpoint PFS Secondary Endpoints ORR Duration of response OS Patient-reported outcomes Previously Untreated CLL/SLL BGB-3111 160mg BID to progression N=210 Bendamustine +
Slide 23: Phase 2 Registrational Trial Design in Relapsed or Refractory FL
Relapsed or Refractory FL N=210 BGB-3111 + Obinutuzumab N=140 Obinutuzumab* N=70 2:1 Randomization Stratification factors: number of prior lines of therapy (2-3 and > 3) Primary Endpoint ORR Secondary Endpoints Duration of Response PFS OS Time-to-response Expedited development in FL where there is a
Slide 24: BGB-A317 Overview
Overview Potential differentiation vs. currently approved PD-1 antibodies Potential cell biology differentiation in lack of Fc receptor binding, which may have a negative effect on the activity of other PD-1 antibodies based on our preclinical data and recent publications1 Superior PD-1 inhibition a
Slide 25: Tumor Types Responsive to PD-1 Antibodies Are Among the Most Common Cancers in China
China % of World (2012) Tumor Types That Have Shown Activity to PD-1 Antibodies Are Among the Most Common Cancers in China Source: Data from World Health Organization (2012); Chen et al., CA Cancer J Clin, 2016 Cervix Breast Colon Liver Esophagus 0.25 Gastric Lung All types 22% 36% 43% 49% 51% 19% 1
Slide 26: BGB-A317 Phase 1 Data Show Proof of Principle
27 BGB-A317 Phase 1 Data Show Proof of Principle and Encouraging Clinical Activity Note: 93 pts included in the chart, the remaining 6 pts were not evaluable for target lesion response based on imaging assessment at the cutoff time Source: 1 Phase 1 data as of September 30, 2016, presented at the So
Slide 27: BGB-290 Overview
Overview Highly selective PARP1 and PARP2 inhibitor with strong PARP trapping activity Significant brain penetration in preclinical studies, potential for treating brain tumors and metastases Well-tolerated in monotherapy dose escalation data1 with significant anti-tumor activity: Few AEs of myelosu
Slide 28: BGB-290 Monotherapy Dose Escalation Data
All Grade Grade 3-4 N = 29 Description n (pts) % n (pts) % Gastrointestinal Disorders Nausea 11 38% 0 Vomiting 4 14% 0 Diarrhea 3 10% 0 Dry Mouth 1 3% 0 General Disorders and Administration Site Conditions Fatigue 8 28% 0 Nervous System Disorders Lethargy 2 7% 0 Dysgeusia 1 3% 0 Hypoesthesia 1 3% 0
Slide 29: BGB-A317 + BGB-290 Dose Escalation Data
A. Change in Tumor Volume by Baseline Tumor Type BGB-A317 + BGB-290 Dose Escalation Data Generally Well-Tolerated with Preliminary Anti-Tumor Activity in Multiple Tumor Types :1 bile duct, 1 peripheral nerve sheath tumor Best overall responses included 1 CR, 3 confirmed PRs and 7 unconfirmed PRs Ova
Slide 30: BGB-283 Overview
31 Overview Potential to be a first-in-class RAF dimer inhibitor globally Vemurafenib and dabrafenib are only active against the BRAF monomer Potentially addresses several limitations of current BRAF inhibitors, including: Large unmet need in RAS-mutated tumors Rapid development of resistance Proof-
Slide 31: BGB-283 Phase 1a/1b: Durable Responses in K-RAS-Mutated Cancers
Best Response in Target Lesions * Duration of Treatment (day) BGB-283 Phase 1a/1b: Durable Responses in K-RAS-Mutated Cancers * On Treatment Source: Dose escalation / expansion data as of September 17, 2016, presented at AACR 2017 (Desai et al) 32
Slide 32: Financial Position and Investor Base
33 Financial Position and Investor Base Strong Balance Sheet Position Cash, Cash Equivalents, and Short-term Investments (6/30/2017) $407M (Unaudited) Stable and Established Investor Base Private Round Investors Investor Base Established Through Private and Subsequent Public Offerings Select New Pub
Slide 33: Near-term Milestones and Plans
34 Near-term Milestones and Plans Event Expected Timing BGB-3111 (BTK Inhibitor) Present data from the combination trial with BGB-A317 at a medical conference Present additional data from the dose-expansion phase of the Phase 1 monotherapy trial Present additional data from the Phase 1 combination t
Slide 34: Near-term Milestones and Plans
Near-term Milestones and Plans BGB-3111 (BTK Inhibitor) ey Present data from the combination trial with BGB-A317 at a medical conference 2017 Present additional data from the dose-expansion phase of the Phase 1 monotherapy 2017 trial Present additional data from the Phase 1 combination trial with ob