BeyondSpring, Inc. · NASDAQ:BYSI
BeyondSpring — Investor Presentation (June 2021)
46 slides from BeyondSpring's investor presentation, Jun 18, 2021
Slide 1: Corporate Presentation
Corporate Presentation June 2021 | NASDAQ: BYSI 1
Slide 2: Disclaimer
Disclaimer This presentation has been prepared for informational purposes only. No money or other consideration is being solicited, and if sent in response, will not be accepted. This presentation shall not constitute an offer to sell, or the solicitation of an offer to buy, any securities, nor shal
Slide 3: Investment Highlights
Investment Highlights Research subsidiary, SEED Therapeutics, with $800 million Partnerships Committed to raising the standard partnership with Eli Lilly (Proprietary TPD Platform) of care for cancer patients, in the largest global markets, with first-in- Cash position $90.6 million as of March 31,
Slide 4: Two Near-Term NDAs & Robust Drug Development Pipeline
Two Near-Term NDAs & Robust Drug Development Pipeline Indication / Trial Name / Commercial Program Preclinical Phase 1 Phase 2 Phase 3 Status/Next Milestone Target Collaborator Rights1 CIN • U.S. NDA accepted with Priority PROTECTIVE-1 (All cancer, all Plinabulin + pegfilgrastim Phase 3 primary endp
Slide 5: Plinabulin Value Generation Roadmap
Plinabulin Value Generation Roadmap Proven Expand Transform Promising early clinical efficacy data Phase 3 study fully enrolled (n=559) – June 2021 (ASCO) & Expected in 2022 & Topline OS data - mid-2021 Proven in 6 clinical studies(n>1200) & Filed for NDA approval – Mar. 2021 CIN (BTD) NSCLC Multipl
Slide 6: Plinabulin: Pipeline in a Drug - First-in-Class SIMBA
Plinabulin: “Pipeline in a Drug” - First-in-Class, Selective Immunomodulating Microtubule Binding Agent (SIMBA) 6
Slide 7: Plinabulin: A SIMBA with Potential for Multiple Cancer Indications
Plinabulin: A SIMBA with Potential for Multiple Cancer Indications First-in-class Agent Plinabulin’s immune mechanism designed to GEF-H1 activation2 enable its effects in multiple cancer Plinabulin1 indications: Rho/ROCK activation1 Chemotherapy Induced Microtubules Neutropenia (CIN): Designed to
Slide 8: Plinabulin Induces Dendritic Cell Maturation in IO Combo
Plinabulin Induces Dendritic Cell Maturation, a Key Step in Initiating Anti-cancer Durable Response in IO Combo ❷ Plinabulin 2 3 ❸ Checkpoint Inhibitors Hit the Gas Release the Brakes Stimulate maturation of dendritic Optimize T cell response cells to increase antigen presentation Dendritic cells ar
Slide 9: Triple I/O Combo: Best Tumor Response in PD-1 Non-Responsive Model
Triple I/O Combo: Plinabulin + PD-1 + Radiation (IR), Best Tumor Response in PD-1 Non-Responsive Tumor Model (MD Anderson) Tumor Volume (mm3) 2000 1000 0 DC activation is most dramatic in triple T cell doubles in triple I/O Combination I/O combination vs. PD1 + IR Biomarker data in tumor 30 days aft
Slide 10: Plinabulin Clinical Development Program
Plinabulin Clinical Development Program NB SS DI A Q Study 101 Phase 1/2 Phase 2 Efficacy Data Phase 2 Phase 3 Registration Protective-1: Plinabulin vs. Pegfilgrastim Study 105 (PROTECTIVE-1) Study 105 (PROTECTIVE-1) Plinabulin (various dose) vs. Plinabulin vs. Pegfilgrastim (n=105) Pegfilgrastim (n
Slide 11: Building the Plinabulin Franchise
Building the Plinabulin Franchise Anti-Cancer with Anti-Cancer with CIN Chemotherapy Immuno-Oncology Raise the Standard of Improve survival and Potential APC Care quality of life cornerstone of emerging regimens 11
Slide 12: Severe Unmet Medical Need for Breakthrough Designation and Priority Review
Severe Unmet Medical Need is Basis for Breakthrough Designation and Priority Review for Plinabulin + G-CSF Regimen in CIN Prevention Despite widespread G-CSF use, CIN #1 reason for FN, ER visits, hospitalization, sepsis, mortality, and chemotherapy disruption1 Short-term Outcome Benefit Long-term Ou
Slide 13: Monotherapy G-CSF Fails to Prevent Chemo Regimen Changes
Monotherapy G-CSF Fails to Prevent Chemo Regimen Changes Percent of patients with significant regimen changes mFOLFOX6 CRC Chemo regimen FOLFOX4 changes are NSCLC Cisplatin, vinorelbine unacceptably high Ovarian carboplatin, paclitaxel Patients’ experience: AC→doceltaxel • Dose reduction • Dose dela
Slide 14: Plinabulin + G-CSF Combination Addresses Unmet Medical Need
Plinabulin + G-CSF Combination Addresses Unmet Medical Need Plinabulin is the only product – in development – that has demonstrated the potential to elevate the standard of care (SOC) to prevent CIN 7.0 Neulasta 6 mg (n=22) • Breakthrough Therapy Designation: Unmet Plinabulin 20 mg/m2 + Neulasta 6 m
Slide 15: Protective-2 Ph3: Registration Study Design
Protective-2 (Study 106) Ph 3: Registration Study Design • Double blind, global study (19 centers); 4 cycles • Covance: CRO • Covance central lab: ANC evaluation Plinabulin 40mg + Pegfilgrastim 6 mg N=111 Breast Cancer, R 1:1 TAC Therapy Placebo + Pegfilgrastim 6 mg N=110 15
Slide 16: PROTECTIVE-2 Ph3: Primary Endpoint Met
PROTECTIVE-2 Ph3: Primary Endpoint Met Proportion of Patients with No Grade 4 Neutropenia (or DSN= 0 Days) in Cycle 1 35% 31.5% 30% p-value=0.0015 25% Patients 20% (%) 15% 13.6% 10% 5% N=110 N=111 0% Pegfilgrastim 6 mg Plinabulin 40 mg + Pegfilgrastim 6 mg • Grade 4 neutropenia (ANC < 0.5 x 109 cell
Slide 17: PROTECTIVE-2 Ph3: Clinical Consequences of CIN Also Reduced
PROTECTIVE-2 Ph 3: Clinical Consequences of CIN also Reduced by Combination Pegfilgrastim 6 mg Plinabulin 40 mg + Pegfilgrastim 6 mg ANC Nadir N=110 N=111 Mean nadir for FN patients 0.06 0.13 (x109 cells/L) Mean nadir for all patients 0.31 0.54 (x109 cells/L) ANC benefit of the combination improves
Slide 18: Adverse Reactions Occurring in ≥5% of Patients
Adverse Reactions Occurring in ≥ 5% of Patients in the Plinabulin + Pegfilgrastim vs. Pegfilgrastim Pegfilgrastim 6 mg Plinabulin 40 mg** + Pegfilgrastim 6 mg N=132 N=127 Body System/Preferred Term All Grade Grade 3/4 All Grade Grade 3/4 Gastrointestinal disorders 22 (16.7%) 1 (0.8%) 49 (38.6%) 9 (7
Slide 19: Superior Improvement in Clinically Meaningful Endpoints vs Pegfilgrastim
The combination with Superior Improvement in Clinically Meaningful Endpoints compared to Pegfilgrastim alone Reduction of Incidence and Reduction of Profound Improvement of Severity of FN and Neutropenia (PN) Related Hospitalization Benefits Quality of Life Clinical Endpoints Profound Neutropenia En
Slide 20: Favorable Benefit/Risk Ratio
Favorable Benefit/Risk Ratio (Plinabulin + G-CSF vs. G-CSF alone) Improved Efficacy (ANC based Improved Efficacy (FN) Favorable Safety in Cycle 1) – 106 Phase 3 – 106 Phase 3 – 106 Phase 2+3 No Grade 4 Neutropenia FN Grade 4 TEAE • 31.5% vs. 13.6% (incidence), p=0.0015 • 3.6% vs. 6.3% (incidence) •
Slide 21: Regulatory Strategy for CIN: FDA NDA Acceptance with Priority Review
Plinabulin’s Regulatory strategy for CIN, FDA NDA Acceptance with Priority Review: Superior Profile in a Broad Label Plinabulin shown to statistically reduce Grade 4 neutropenia in 6 clinical trials (1,200+ patients) Registration Study Supporting Studies Supporting Studies Plinabulin + G-CSF combo P
Slide 22: Plinabulin + G-CSF Combination - Commercial Plan in CIN Prevention
Plinabulin + G-CSF Combination - Commercial Plan in CIN Prevention 22
Slide 23: Chemotherapy Without Compromise: Turning the Ds into the Ss
Chemotherapy Without Compromise: Turning the 4 Ds into the 4 Ss DECREASED STABLE DOSE recommended dose maintaining >85% Plinabulin + G-CSF DELAYED cycles SUSTAINED CYCLES • Differentiated clinical cycles on time profile, potential to improve SOC DISCONTINUED chemotherapy STAY THE COURSE complete all
Slide 24: CIN: Large and Expanding Market Potential
CIN: Large and Expanding Market Potential Plinabulin can be used with each cycle of G-CSF in non-myeloid cancers to provide improved protection from neutropenia NCCN Guidelines Doubled the US Market for CIN Chemotherapy remains 650,000 US Historic NCCN CIN THE CORNERSTONE OF Chemotherapy prophylaxis
Slide 25: Prioritize on Large and Rapid Adopters at Launch
Prioritize on Large and Rapid Adopters at Launch Pegfilgrastim Patient Distribution1 – Top 1200 Centers US Regional Coverage Top 3 deciles = 81% 99% 100% 98% 100,000 of pts (~360 accts) 94% 96% 100% 91% 90,000 87% 90% 85,879 81% 80,000 80% 72% Cumulative Contribution (%) 70,000 70% G-CSF concentrati
Slide 26: Plinabulin: Potential for Use Across the Spectrum of Solid Tumors
Plinabulin: Potential for Use Across the Spectrum of Solid Tumors G-CSF Administrations: Solid Tumor Rectal Cancer Gynecological Cancer, Other 5% G-CSF use by cancer type: Ovarian 5% Cancer • Improved control of CIN with 5% Plinabulin can prove important in Prostate Cancer cancers with more aggressi
Slide 27: Targeted Commercialization Plan
Targeted Commercialization Plan Drive awareness of the "Neutropenia FOCUS: Elevating the SOC in chemotherapy 01 Vulnerability Gap” and unmet need Position Plinabulin with key decision 02 makers Key Account Neutropenia Plinabulin Activation Vulnerability Positioning /Access & Gap Availability Activat
Slide 28: Detailed Commercialization Plan
Detailed Commercialization Plan 1 2 3 Neutropenia Vulnerability Gap and Positioning Plinabulin with Activating Key accounts and Unmet Need Awareness Key Decision Makers Ensuring Broad Patient Access KOLs: • Advocacy and Expert network State of the Art Promotion Programs: • Major health care congress
Slide 29: Breakthrough Therapy with FDA NDA Priority Review
Breakthrough Therapy with FDA NDA Priority Review: Potential to Elevate SOC for CIN Prevention Opportunity Unmet need Product differentiation Market size Grade 4 neutropenia complications Plinabulin + G-CSF addresses 3 oncologist needs: Market growth CIN: #1 reason for therapy change NCCN guide
Slide 30: Building the Plinabulin Franchise
Building the Plinabulin Franchise Anti-Cancer with Anti-Cancer with CIN Chemotherapy Immuno-Oncology Raise the Standard of Improve survival and Potential APC Care quality of life cornerstone of emerging regimens 30
Slide 31: Unmet Medical Need – 2nd/3rd Line NSCLC, EGFR Wild Type
Unmet Medical Need – 2nd/3rd Line NSCLC, EGFR Wild Type • Large patient population with limited Efficacy treatment options o EGFR wild type: ~85% western NSCLC and Safety ~70% of Asian NSCLC patients o only four therapies approved o TKI is worse than docetaxel1 • Limited efficacy • Severe side effec
Slide 32: Four Therapies Approved in NSCLC: Limited Options
Limited Options • Four therapies approved in NSCLC (2nd/3rd line, EGFR wild type) • SOC Docetaxel: Limited overall survival with CIN severe neutropenia ~40% Pemetrexed and PD-1/PD-L1 Moved To First Line Treatment Ramuciramab + Docetaxel vs. Docetaxel1 Pemetrexed vs Docetaxel2 Nivolumab (PD-1 Ab) vs.
Slide 33: Rationale for Advancing Plinabulin in NSCLC Study
Rationale for Advancing Plinabulin in NSCLC Study Mode of Action Rationale Preliminary Clinical Evidence (Phase 2) Higher GEF-H1 immune signatures associated with longer OS in cancer 1 • Post-Hoc Ph2 data from Plinabulin in NSCLC in mechanism targeted patients (measurable lung lesion) shows overall
Slide 34: DUBLIN-3 (Study 103): Phase 3 in 2nd/3rd NSCLC, EGFR Wild Type
DUBLIN-3 (Study 103): Phase 3 in 2nd/3rd NSCLC, EGFR Wild Type Design EGFR wild-type NSCLC (Pre-specified MOA target patients: Measurable lung lesion) • Plinabulin + docetaxel vs docetaxel, 1:1 randomization, n=559 (fully enrolled) • Approval possible with a single, qualified study • Final analysis:
Slide 35: Target Product Profile
Target Product Profile Current Standard of Care Plinabulin - Docetaxel Combination • Potential survival benefit, with more • Modest survival benefit long survivals due to GEF-H1 IO MOA • Potential superior safety profile, • Severe safety concerns, e.g. CIN including CIN reduction • Poor Quality of L
Slide 36: Building the Plinabulin Franchise
Building the Plinabulin Franchise Anti-Cancer with Anti-Cancer with CIN Chemotherapy Immuno-Oncology Raise the Standard of Improve survival and Potential APC Care quality of life cornerstone of emerging regimens 36
Slide 37: Triple I/O Combo Development for Multiple Cancer Indications
Triple I/O Combo Development for Multiple Cancer Indications in PD- 1/PD-L1 Failed Patients – Severe Unmet Medical Needs Trial Name / Indication / Target Program Commercial Rights Status Collaborator SCLC Plinabulin + 7 US sites, including Phase 1 completed, Checkpoint naïve and Nivolumab (PD-1) + R
Slide 38: Plinabulin+Nivolumab+Ipilimumab in SCLC: Study Design
Plinabulin+ Nivolumab +Ipilimumab in SCLC: Study Design Dose-escalation phase I study 3+3 Design In patients with extensive-stage SCLC who had progressed on or after prior platinum-based chemotherapy (±PD-1/PD-L1) Primary objective To determine dose-limiting toxicities (DLT’s) and recommended Ph
Slide 39: Efficacy Analysis
Efficacy Analysis Data cutoff –December 30,2020 PD-1/PD-L1 PD-1/PD-L1 resistant Efficacy Analysis therapy naïve (n=7) (n= 6) Number of 3 (50%) 3 (43%) patients with PR *PR –Partial Response - RESIST 1.1 : At least a 30% decrease in the sum of diameters of target lesions, taking as reference the base
Slide 40: Efficacy Summary
Plinabulin+ Nivolumab +Ipilimumab in SCLC: Big Ten ITT Phase 1 Study Improvement of Overall Response Rate 46% 50% 43% 45% 40% 35% Efficacy Summary 30% 23% 25% 20% 11% 15% 10% 5% 0% Nivolumab Nivolmumab + Plinabulin + Plinabulin + Ipilimumab Nivolumab + Nivolumab + Ipilimumab Ipilimumab (CPI resistan
Slide 41: Plinabulin as a Potential Synergistic Cornerstone Agent in IO Therapy
Plinabulin as a Potential Synergistic “Cornerstone” Agent in IO Therapy Data Conclusion • High response rate to previous • Immune system re-sensitized CPI failures (43%) • Improved Anti-cancer Response • Increased antigen presentation (46% ORR vs. 12-23% CPI) simulates T cell activation • Durable re
Slide 42: Recent Goals Achieved, Near Term Milestones for Value Creation
Recent Goals Achieved, Near Term Milestones for Value Creation CIN (Target broad range of cancer and chemotherapy) Value creation in elevating SOC; Life cycle management Plinabulin Life-cycle Management Anti-cancer NSCLC (with chemo): 2022+ I/O Combo • Large and growing population Early Clin.
Slide 43: Corporate Highlights
BeyondSpring Corporate Highlights
Slide 44: SEED Therapeutics Subsidiary – Pipeline Potential
SEED Therapeutics Subsidiary – Pipeline Potential SEED: subsidiary pursuing "Molecular Glue" targeted protein degradation to degrade disease-causing proteins previously believed to be undruggable • $800M collaboration with Eli Lilly on three targets • Own targets (e.g., KRAS) • Structure conducive t
Slide 45: BeyondSpring: Key Highlights
BeyondSpring: Key Highlights Committed to raising the standard of care for cancer patients in the largest global markets with first-in-class Mission treatments that improve lives and clinical outcomes for millions of patients in need Near-term Global Capabilities Broad Pipeline Global Market Opportu
Slide 46: www.beyondspringpharma.com
www.beyondspringpharma.com 46