EX-99.1 2 s001719x1_ex99-1.htm EXHIBIT 99.1

Exhibit 99.1

 Transformational Science Advancing Oncology 
 

 Disclaimer  This presentation contains forward-looking statements about BeyondSpring Inc. (“BeyondSpring” or the “Company”). Forward looking statements are based on our management’s beliefs and assumptions and on information currently available to our This presentation contains forward-looking statements about BeyondSpring Inc. (“BeyondSpring” or the “Company”). Forward looking statements are based on our management’s beliefs and assumptions and on information currently available to our management, including those described in the forward-looking statements and risk factors sections of the Company’s 20-F and other filings with the SEC, which are available on BeyondSpring’s Investor Relations website.Such statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels of activity, performance, or achievements to be materially different from those anticipated by such statements. In some cases, you can identify forward-looking statements by terminology such as “may,” “will,” “should,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “intends,” or “continue,” or the negative of these terms or other comparable terminology. Forward-looking statements contained in this presentation include, but are not limited to, (i) statements regarding the timing of anticipated clinical trials for our product candidates and our research and development programs; (ii) the timing of receipt of clinical data for our product candidates; (iii) our expectations regarding the potential safety, efficacy, or clinical utility of our product candidates; (iv) the size of patient populations targeted by our product candidates and market adoption of our product candidates by physicians and patients; and (v) the timing or likelihood of regulatory filings and approvals. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. 
 

 Agenda 
 

 *  Corporate Overview  Lead drug candidate Plinabulin with two indications in pivotal studyPrevention of Neutropenia caused by chemotherapy regimensNon Small Cell Lung Cancer (NSCLC) in patients with measurable lesionsDual-track development in US and China, supported by one global study Follow-on indications for Plinabulin (PD-1 combination, RAS mutations, brain tumors, etc)  Wholly owned portfolio of novel immuno-oncology assets  Scalable Business Model  Well Positioned to Execute on Strategic Plan  Seasoned management team supported by KOLs and medical advisory boardCash of $54.6M* - funds pipeline development, including through submission of 2 NDA’s   Focus on approvals in U.S. and China, with unique access to large marketsPotential for accelerated clinical developmentEarly-stage pipeline includes next-generation immuno-oncology assets and new products from Fred Hutchinson/UW collaboration  * As of March 31, 2017 
 

 *  Plinabulin   Preclinical  Phase 1  Phase 2  Phase 3  Status  Plinabulin             Prevention of Neutropenia(in chemo induced Neutropenia) Study 105 (docetaxel)          IND Initiation 2Q 2017First Data Readout: 2H 2017   Study 106 (TAC)          First Data Readout: 1H 2018  NSCLC: Measurable lung tumor (+ Docetaxel)          Initiation 2Q 2016 Readout: 1Q 2018 (interim)  PD-L1+(+ Nivolumab)          Initiation 2H 2016Readout: 2017  KRAS Mutant(+ Docetaxel)           Initiation 2017  Metastatic Brain Tumor (+ Radiation)          Initiation 2017  IO Compound BPI-002 (T-cell activation)          Preclinical  IO Compound BPI-003 (IKK inhibitor)          Preclinical  IO Compound BPI-004 (Neo-Antigen)          Preclinical  Fred Hutchinson/UW Collaboration (Ubiquitination technology platform)          Preclinical  Pipeline Overview 
 

 *  Plinabulin  Dendritic Cell  Neutropenia Prevention Effects  Plinabulin: Novel Structure and Novel IO Target (GEF-H1)   JNK Activation  Rho Activation  Immune – Related Anticancer Effects  Plinabulin  Kashyap A et al. Keystone Meeting,Mar. 2017Wang Y et al. FEBS J. 2016; 283: 102-111  300 derivatives 
 

 * 
 

 *   Chemotherapy-Induced Neutropenia Causes:   Neutropenia Treatment With G-CSF Causes:   Bacterial related mortalitiesDelays in chemo dosingChemo Dose reductionsEarly termination of chemo  Enlarged spleenAcute respiratory distress syndromeAnaphylaxisSickle cell disorderBone pain   Addition to docetaxel led to significant reduction in grade 3 and 4 neutropenia (P<0.003)No added toxicityMore patients continue on full dose docetaxel for a longer period  Neutropenia: An Underserved Market   Plinabulin Product Profile  Competitive efficacy profileEnhanced safety, including lower incidence of bone pain Plinabulin can be dosed one hour after docetaxel use, unlike G-CSF’s 24 hour delay 
 

 *  Severe Neutropenia Seen After Docetaxel Monotherapy  Source: Quartino et al Invest New Drugs 2012; 30:833-845  Neutropenia Is Marked By: Severe InfectionsSepsisHospitalizationsHigh Mortality  Cycle 1 Docetaxel Treatment Model  Duration of Severe Neutropenia (DSN) refers to the number of days with neutrophil levels below 0.5 x109 cells/LA lower nadir correlates with a longer Duration of Severe Neutropenia (DSN)  Day 8  DSN 
 

 *  Phase 2 Data presented at ASH December 2016  Docetaxel Monotherapy  DSN Estimate Model Based on Nadir- Cycle 1 Day 8   Phase 2 Data Supports Favorable Product Profile  P < 0.0003  33.3%  4.6% *  ~5%   Both 20mg and 30mg Plinabulin cohort p-value <0.0003* Planned Phase 3 doseGrade 4/Severe Neutropenia: ANC < 0.5 x 109 cells/L  Docetaxel Monotherapy    0 1 2 3 4   Docetaxel + Plinabulin    Projected DSN  Mean DSN: 1.1 days   Mean DSN: 0.065 days  67%  95% 
 

 *  Efficacy Differentiation: Plinabulin vs. G-CSF  Percentage of Patients Experiencing Grade 3 and 4 Neutropenia†  Percentage of Patients Experiencing Grade 3 and 4 Neutropenia†  Percentage of Patients Experiencing Grade 3 and 4 Neutropenia†  Percentage of Patients Experiencing Grade 3 and 4 Neutropenia†  Percentage of Patients Experiencing Grade 3 and 4 Neutropenia†  Percentage of Patients Experiencing Grade 3 and 4 Neutropenia†  Neutropenia  Docetaxel100 (n=49)*  Docetaxel100+ G-CSF(n=60)**  Docetaxel75 (n=55)*  Docetaxel75 (n=51)***  Docetaxel75+ Plinabulin(n=81)***  Grade 3/4   85.7%  41%  67.3%  66.7%  11.5%  † We have not conducted head-to-head trials of G-CSF and Plinabulin in combination with docetaxel for the prevention of docetaxel-induced grade 3 and 4 neutropenia. As a result, the data derived from these separate clinical trials may not be comparable and would not form a basis for marketing Plinabulin, if submitted for regulatory approval.* Shepherd et al J Clin Oncol 2000;18:2095-2103.** Alexopoulos Cancer Chemother Pharmacol 1999;43:25.*** Phase 2 portion of our Phase 1/2 trial described above  The above data form the basis of our belief that Plinabulin may be as effective as G-CSF for the prevention of docetaxel-induced grade 3/4 Neutropenia 
 

 *  Plinabulin: Differentiated from G-CSF in Neutropenia Prevention 
 

 *  Plinabulin Neutropenia Clinical Program (Study 105)  Neulasta 0.6 mg/m2 (n=10)  Plinabulin 5 mg/m2 (n=10)  Plinabulin 10 mg/m2 (n=10)  Plinabulin 20 mg/m2 (n=10)  Arm 1Arm 2Arm 3Arm 4  Phase 2 Portion   Phase 3 Portion  Plinabulin 20 mg/m2* (n=75)  *Planned Phase 3 Dose  Neulasta 0.6 mg/m2 (n=75)  CurrentFirst patient enrolled in US, April 2017  H2 2017First Data Readout  H1 2018Interim AnalysisNon-Inferiority  H1 2019Final ReadoutNon-Inferiority  Study 105 (Docetaxel) Phase 2/3- 200 patients  Interim Analysis at n=50/Arm Primary Endpoint: DSN in Cycle 1Non-Inferiority Margin: 0.65 days 
 

 *  Neulasta 0.6 mg/m2 (n=20)  Plinabulin 10 mg/m2 (n=20)  Plinabulin 20 mg/m2 (n=20)  Arm 1Arm 2Arm 3  Phase 2 Portion   Phase 3 Portion  Neulasta 0.6 mg/m2 (n=60)  Plinabulin 20 mg/m2 (n=60)  *Planned Phase 3 Dose  Plinabulin Neutropenia Clinical Program (Study 106)  H1 2017Study to Initiate  H1 2018 First Data Readout  H1 2019Final ReadoutSuperiority  Study 106 (TAC) Phase 2/3- 200 patients  Final Analysis at n=60/ArmSequential AnalysisPrimary Endpoint: DSN in Cycle 1Non-InferioritySuperiority  First patient enrolled in the US in April 2017 
 

 *  Indication: Prevention of All Chemo-induced Neutropenia  For concurrent administration with a myelosuppressive chemotherapeutic regimen in patients with non-myeloid malignancies for the prevention of chemotherapy induced neutropenia (with Study 105 and 106) Plinabulin’s mechanism of action (MOA) is in protecting neutrophils from apoptosis via its immuno-oncology effect (independent of chemotherapy), and has shown to be effective in chemotherapy regimens with different mechanisms in pre-clinical models. As such, we expect Plinabulin’s neutropenia reduction effect to be ubiquitous to treatment.Primary Investigator, Dr. Blayney, Stanford Professor, Founding member of NCCN guidelines for neutropenia management   TARGET INDICATION 
 

 * 
 

 *  NSCLC: Large Market Opportunity for Plinabulin  Lung cancer is a leading cause of death worldwide~1.8 million diagnoses globally (87% have NSCLC)Nearly 1/3 of global patients are in ChinaDespite multiple approved therapies, the lung cancer treatment market remains fragmented, marked by modest survival benefits, limited by:Effectiveness only in specific tumor mutationsLimited patient response in many patientsUnique safety risks across therapeutic modalitiesPD-1 antibody Drugs are effective in a small percentage of NSCLC patients, typically between 15-30%  Plinabulin Target Patient Sub-Groups in 2nd/3rd Line NSCLC  30% Asian10% Western  70% Asian90% Western 
 

 *  Phase 2 Data: Oral Presentation at ASCO-SITC Meeting (Feb.2017)  Durable Response and Extended Survival Benefit of 4.6 Months  * We attribute the non-significant P-value to a small number of patients in each treatment arm 
 

 *  Plinabulin + Nivolumab (PD-1 Antibody) for NSCLC  Animal studies provide indications of an immune MOA, which primarily acts through activation and proliferation of tumor antigen-specific CD4 T-cells, a white blood cell active in immune responses. Combo of Plinabulin and PD-1 antibody in immune competent breast cancer model  Breast cancer model- Dr. Zippelius Lab at Univ. of Basel   U.S. Immuno-Oncology Trial Overview (Investigator Initiated IND)Phase 1/2 study started at UCSDGoal: Efficacy Synergy similar to PD-1+ CTLA-4 Antibodies, but SaferClinical sites: UCSD and Fred HutchinsonPatient: 2nd/3rd line NSCLC Timeline: 2H 2016 initiation, safety and biomarker read-out in 4Q 2017  UCSD Trial Design:28 days per cyclePlinabulin (IV): Day 1, 8, 15Nivolumab (IV): Day 1, 15  Pre-Clinical  Clinical 
 

 *  Plinabulin Clinical Program in Cancer   Exploring The Synergy of Plinabulin with Standard of Care Therapies in Multiple Patient Sub-Groups  Plinabulin + Docetaxel*EGFR wild typePD-1/PD-L1 failed patients includedPhase 3: 550 patients to be enrolled(80% from China)  Plinabulin+ PD-1 Antibody**PD-L1+Phase 1/2 study started at UCSD  * Combination with Docetaxel in all docetaxel approved indications including NSCLC, gastric cancer, breast cancer, head and neck cancer, and prostate cancer. Potential other chemo combo with Plinabulin.** Combination with PD-1/PD-L1 Antibody in PD-1 approved indications including NSCLC, melanoma, renal cancer, Hodgkin’s lymphoma, head and neck cancer, and urothelial carcinoma   Plinabulin + Docetaxel*RAS MutationsPhase 2 study expected to begin enrollment at UC Davis in 2017 
 

 * 
 

 *  Seasoned Leadership Team  Lan Huang, PhD: Co-Founder, Chairman & Chief Executive OfficerCo-founded clinical CRO Paramax and spearheaded its sale to global CRO RPS, Co-founded Wuxi MTLH Biotech, and sold self-designed peptide drug China rights to Shanghai Pharmaceutical companyBoard member of Oncology Drug Review Board of China Pharma Assoc.“Thousand Talent Innovator Award” Drug development recognition from the President of China  Ramon Mohanlal, MD, PhD: Executive Vice President; Chief Medical OfficerMore than 20 years experience in strategic drug development, including executive positions at GSK and VertexHead of Established Products Oncology for Novartis  G. Kenneth Lloyd, PhD: Chief Scientific OfficerGreater than 45 years experience in Pharma Industry drug discovery and developmentExecutive & Scientific positions at Roche; Synthelabo, Wyeth-Ayerst, SIBIA, and Nereus  Gordon Schooley, PhD: Chief Regulatory Officer Executive for more than 20 years leading clinical and regulatory affairsExecutive & Regulatory positions at SkyePharma, Alliance Pharmaceutical and Pacira Pharma  Richard Brand, MBA: Chief Financial Officer20 years of investment banking experience and 12 years of institutional investment manager experienceCFO experience in growth companies, including a $6 billion public company 
 

 *  Unique Access to Large China Market 
 

 *  China  U.S./ Europe  Seek broad indication from U.S. FDA in preventing Neutropenia, and in NSCLC in patients with measurable lung lesions Partner with one or more global pharmaceuticals companies for both Neutropenia and NSCLC indications  Pursue accelerated development in China for NSCLC indicationBuild an internal sales force for NSCLC indicationRetain a contract sales organization for secondary markets in China  Plinabulin Commercial Strategy 
 

 *  BeyondSpring Inc. (BYSI)–NASDAQ IPO 3/9/2017   IPO & Concurrent Private Placement: $54.3 M ($48 M net)Investors Included:HuaRong, $150 B fund, largest Asset Management Co. in ChinaSangel, top 10 Biotech Focused Fund in China Fosun, $70 B Investment Conglomerate 
 

 *  Expected Company Milestones  6 Months   12-18 Months   24-30 Months   P3 FinalNeutropenia NSCLCIND submission P3 PD-1 ComboBPI-004 [IO]Ubiquitination Technology Platform asset: Fred Hutchinson/UW collaboration   P3 InterimNeutropenia NSCLCDataPD-1 ComboIND submission BPI-002 [IO]  P2 DataNeutropenia  All funded by current cash runway 
 

 *  Thank You!Contact: Lan Huang, Ph.D.lhuang@beyondspringpharma.comBeyondSpring Inc.28 Liberty Street, 39th Floor, New York, NY 10005Tel: 646-305-6387 | Fax: 646-219-9660 
 

 *  Addendum 
 

 *  Robust Patent Estate  
 

 *  Additional Assets 
 

 *  Patients included:2nd/3rd line NSCLC, stage 3b/4n=163  Docetaxel 75mg/m2n=55   Docetaxel 75mg/m2 + Plinabulin 30mg/m2n=50  Docetaxel 75mg/m2n=18   Docetaxel 75mg/m2 + Plinabulin 20mg/m2n=40  Cohort 1: 30mg/m2  Cohort 2: 20mg/m2   Endpoints:Primary: Overall Survival (OS)Safety (includes Neutrophil count)  Completed by Nereus – CRO Quintiles; >70% US+ Western Patient Population; Randomized Phase 2 portion of Phase 1/2 trial   Plinabulin + Docetaxel in NSCLC: Phase 1/2 Trial 
 

 *  Plinabulin’s Neutropenia Benefit (Cycle 1-4)  Plinabulin’s Effect in Preventing Grade 4 and Grade 3 Neutropenia is Persistent  Grade 3 Neutropenia   Cycle 1 Cycle 2 Cycle 3 Cycle 4   Blue: Docetaxel alone (N=73)Red: Docetaxel + Plinabulin (N=90)   Grade 4 Neutropenia 
 

 *     30 DN(n=50)  20 DN (n=40)  20+30 D(n=73)  Nausea  48;4  40;0  39;0  Fatigue  52;4  30;3  40;10  Diarrhea   58;8  35;5  33;6  Constipation   36;0  28;0  29;1  Anorexia   34;0  25;3  33;0  Pyrexia   30;0  23;0  26;2  Vomiting   34;4  35;0  25;1  Cough   22;0  33;0  32;0  Alopecia   28;0  25;0  33;0  Dyspnea   22;4  28;5  25;14  Neutropenia   8;8  8;5  33;26  Myalgia   22;2  8;0  19;0  Anemia   24;8  20;5  16;2  Asthenia   8;2  20;13  26;4  Headache   22;0  26;3  11;0  Dizziness   22;0  5;0  9;0  Hypokalemia   20;0  5;5  4;1  Leukopenia   6;2  7;0  12;9  Tachycardia   14;0  7;0  8;0  Arthralgia   14;0  15;0  14;0  Transient Hypertension   32;20  23;5  4;0  Safety Data (Phase 2, NSCLC, Well Tolerated)  Common(>=20%) AEs(%Grade 1-4; %Grade 3-4) 
 

 *  Study 105 Design: Docetaxel  Neulasta 0.6 mg/m2 (n=10)  Plinabulin 5 mg/m2 (n=10)  Plinabulin 10 mg/m2 (n=10)  Plinabulin 20 mg/m2 (n=10)  Arm 1Arm 2Arm 3Arm 4  Phase 2 Portion   Phase 3 Portion  Interim Analysis at n=50/Arm  Primary Endpoint: DSN in Cycle 1 Non-Inferiority Margin: 0.65 days  Plinabulin 20 mg/m2* (n=75)  *Current Recommended Phase 3 Dose  Docetaxel in patients with NSCLC, breast cancer and prostate cancer  Neulasta 0.6 mg/m2 (n=75)  Primary EndpointDSN in cycle 1Secondary Endpoints:Incidence of: (1) Grade 4 neutropenia; (2) Febrile Neutropenia; (3) Infection; (4) antibiotic use; (5) docetaxel dose changeIncidence and duration of hospitalization due to Febrile Neutropenia Incidence, occurrence, and severity of bone pain 
 

 *  Neulasta 0.6 mg/m2 (n=20)  Plinabulin 10 mg/m2 (n=20)  Plinabulin 20 mg/m2 (n=20)  Arm 1Arm 2Arm 3  Phase 2 Portion   Phase 3 Portion  Final Analysis at n=60/ArmSequential Analysis (DSN cycle 1)Non-InferioritySuperiority  Neulasta 0.6 mg/m2 (n=60)  Plinabulin 20 mg/m2 (n=60)  *Current RP3D  Numeric Results for Testing the Difference Between Two Poisson RatesAlternative Hypothesis:  Two-Sided                         Group 1:  ControlTest Statistic:                Large-Sample                     Group 2:  Treatment                                                                              Grp 1           Grp 2                                                                                                                                          Event            Event                                                                                                                                            Rate            Rate              Diff           Ratio                         Power            N1                N2                 N                λ1                λ2            λ2-λ1          λ2/λ1            Alpha0.90248          53                53              106             1.20             0.60            -0.60          0.5000            0.0500.90051          80                80              160             1.20             0.70            -0.50          0.5833            0.0500.90096         246              246             492             1.20             0.90            -0.30          0.7500            0.050  Statistics for Superiority in Phase 3 Portion  Study 106 Design: TAC 
 

 *  Docetaxel +Plinabulin  Plinabulin + Docetaxel Phase 3 Trial for NSCLC  Patient Criteria:Patients with at least 1 measurable lung lesionPD-1/PD-L1 antibody failures (stratified)EGFR wild type, mutations not eligible; no restriction on histologyOne prior platinum-based chemotherapy; no restriction on biological therapySAP Plan: KRAS mutant subgroup; PD-L1 expression subgroup; tumor size subgroup; prior treatment include PD-1/PD-L1 or not  Patients, n=5502nd/3rd Line NSCLC1:1 Randomization  Docetaxel 75mg/m2   Docetaxel 75mg/m2 + Plinabulin 30mg/m2  Endpoints:Primary: Overall SurvivalSecondary: Neutropenia prevention, DOR, QoL Questionnaires, RR, PFS