Corbus Pharmaceuticals Holdings, Inc. · NASDAQ:CRBP
Corbus Pharmaceuticals — Technical Presentation (November 2016)
33 slides from Corbus Pharmaceuticals's technical presentation, Nov 14, 2016
Slide 1: JBT101-SSc-001 Top-Line Data Phase 2 Study in Systemic Sclerosis
NASDAQ:CRBP | CORBUSPHARMA.COM JBT101-SSC-001 TOP-LINE DATA PHASE 2 STUDY IN SYSTEMIC SCLEROSIS NOVEMBER 14, 2016 Exhibit 99.2
Slide 2: Forward-Looking Statements
FORWARD-LOOKING STATEMENTS This presentation contains certain forward-looking statements, including those relating to the Company’s product development, clinical trials, clinical and regulatory timelines, market opportunity, competitive position, possible or assumed future results of operations, bus
Slide 3: Agenda
AGENDA Opening Remarks Yuval Cohen, Ph.D. – Chief Executive Officer Review of JBT-101 Phase 2 Efficacy and Safety Top-Line Results Barbara White, M.D. – Chief Medical Officer Comments Robert Spiera, M.D. – Director of the Vasculitis and Scleroderma Program at the Hospital for Special Surgery, Weill
Slide 4: Trial Design
TRIAL DESIGN a
Slide 5: JBT101-SSc-001 Phase 2 Trial Design
JBT101-SSc-001 PHASE 2 TRIAL DESIGN Double-blind, randomized, placebo-controlled, Phase 2 trial (Part A) 9 clinical sites in the U.S. Adults ages 18 to 70 with diffuse cutaneous systemic sclerosis (SSc) Inclusive eligibility criteria Disease duration ≤ 3 years with any modified Rodnan skin score (mR
Slide 6: Duration and Dosing
DURATION AND DOSING 16 week trial including 12 weeks of active dosing 2:1 overall ratio of JBT-101:placebo Weeks 1-4: JBT-101 5 mg once a day, JBT-101 20 mg once a day, JBT-101 20 mg twice daily or placebo in 2:2:2:3 randomization for dose response for biomarkers, plasma concentrations, safety, and
Slide 7: Objectives
OBJECTIVES Primary Objectives: Evaluate safety and tolerability Evaluate efficacy, using the American College of Rheumatology Combined Response Index in diffuse cutaneous Systemic Sclerosis (CRISS) score Secondary Objectives: Evaluate efficacy using levels of CRISS response and in each domain of the
Slide 8: American College of Rheumatology CRISS Score
AMERICAN COLLEGE OF RHEUMATOLOGY CRISS SCORE Composite score of improvement from baseline Exponential, weighted algorithm CRISS score > 20% can be considered medically significant Input 2 physician assessments – skin thickness (mRSS) and global health (MDGA) 2 patient assessments – Health Assessment
Slide 9: Safety and Tolerability Outcomes
SAFETY AND TOLERABILITY OUTCOMES Safety Measures Treatment-emergent adverse events Other assessments: Vital signs, laboratory safety testing, electrocardiograms, Addition Research Center Inventory – Marijuana questionnaire Tolerability Measures Discontinuation of study product because of treatment-e
Slide 10: Sample Size
SAMPLE SIZE Safety: 24 subjects in JBT-101 treatment group yield at least 95% probability of detecting adverse events that occur at a true rate of 12% or higher Not formally powered for efficacy First-in systemic sclerosis Phase 2 study First study to use CRISS as primary outcome
Slide 11: Subject Disposition and Baseline Characteristics
SUBJECT DISPOSITION AND BASELINE CHARACTERISTICS
Slide 12: Subject Disposition
SUBJECT DISPOSITION 1/27 (3.7%) dosed with JBT-101 withdrew for adverse event 61 Subjects Screened 43 Randomly Assigned 27 Dosed with JBT-101 15 Dosed with Placebo 24 Completed 16 Weeks 3 Withdrew 14 Completed 16 Weeks 1 Withdrew 1 Treatment Emergent Adverse Event
Slide 13: Demographics
DEMOGRAPHICS Characteristics JBT-101 n = 27 Placebo n = 15 Female, n (%) 23 (85.2%) 9 (60.0%) Age, mean ± SD 48.7 ± 10.4 46 ± 11 Disease duration1 Months, mean ± SD 38 ± 19 41 ± 19 > 3 ≤ 6 years 12 (42.1%) 7 (46.7%) Immunosuppressive or immuno-modulating drugs 26 (92.9%) 12 (80.0%) 1 Since first non
Slide 14: Baseline CRISS Domains
BASELINE CRISS DOMAINS Domain Mean ± SD (range) JBT-101 n = 27 Placebo n = 15 Modified Rodnan skin score (0 – 51) 23.5 ± 10.4 (9 - 46) 26.2 ± 10.7 (8 - 47) Physician global assessment (0-10) 4.6 ± 1.8 (2 - 10) 5.2 ± 2.1 (2 - 9) Patient global assessment (0-10) 4.9 ± 2.3 (0 - 8) 4.9 ± 2.8 (0 - 9) Hea
Slide 15: Efficacy Results
EFFICACY RESULTS a
Slide 16: CRISS Scores by Week and Subject
CRISS SCORES BY WEEK AND SUBJECT 1 mITT, LOCF population, 1- sided, mixed model repeated measures using rank transformed data 8 12 16 Week 8 12 16 Week JBT-101 (n = 26) Circle = subject Median = black bar Interquartile range = red bars P1 = 0.044 Placebo (n = 15)
Slide 17: CRISS Scores by Week
CRISS SCORES BY WEEK 1 Modified intent to treat population, last observation carried forward 2 (25th percentile, 75th percentile) Group Median CRISS Score1, % (Interquartile Range)2 Week 4 Week 8 Week 12 Week 16 JBT-101, n = 26 3.0% (0.6%, 11.4%) 19.0% (0.3%, 69.2%) 27.5% (1.9%, 67.8%) 33.0% (0.8%,
Slide 18: Change in HAQ-DI by Week
CHANGE IN HEALTH ASSESSMENT QUESTIONNAIRE - DISABILITY INDEX (HAQ-DI) BY WEEK P = 0.04 P = 0.006 P = 0.006 P = 0.03 1-sided p value, least squares mean difference, analysis of covariance model
Slide 19: Change in Patient Global Assessment (PtGA) by Week
CHANGE IN PATIENT GLOBAL ASSESSMENT (PTGA) BY WEEK P = 0.08 P = 0.10 1-sided p value, least squares mean difference, analysis of covariance model
Slide 20: Change in Modified Rodnan Skin Score (mRSS) by Week
CHANGE IN MODIFIED RODNAN SKIN SCORE (MRSS) BY WEEK P = 0.085 1-sided p value, least squares mean difference, analysis of covariance model
Slide 21: Change in Physician Global Assessment (MDGA) by Week
CHANGE IN PHYSICIAN GLOBAL ASSESSMENT (MDGA) BY WEEK P = 0.02 P = 0.13 1-sided p value, least squares mean difference, analysis of covariance model
Slide 22: Change in Forced Vital Capacity (FVC) by Week
CHANGE IN FORCED VITAL CAPACITY (FVC) BY WEEK 1-sided p value, least squares mean difference, analysis of covariance model
Slide 23: Safety Results
SAFETY RESULTS a
Slide 24: Safety: Treatment-Emergent Adverse Events
SAFETY: TREATMENT-EMERGENT ADVERSE EVENTS 2 serious TEAEs, both unrelated to study drug JBT-101: dehydration of moderate severity treated in hospital Placebo: abdominal pain and nausea of severe severity treated in hospital No unexpected TEAEs related to JBT-101 Only two severe TEAEs occurred, both
Slide 25: Tolerability
TOLERABILITY JBT-101: 1 withdrawal at 4 weeks for TEAE of moderate dizziness probably related to study product
Slide 26: TEAEs in Subjects With 5% Difference Between Groups
TEAES IN ≥ 2 SUBJECTS AND ≥ 5% MORE SUBJECTS IN ONE GROUP COMPARED TO THE OTHER GROUP Organ System Class Term JBT-101 (n = 27) Placebo (n = 15) Subjects n (%) Events n Subjects n (%) Events n Gastrointestinal disorders Nausea 1 (3.7%) 1 2 (13.3%) 3 Diarrhea 2 (7.4%) 3 General disorders Fatigue 5 (18
Slide 27: Conclusions
CONCLUSIONS In a 16 week study in diffuse cutaneous SSc, JBT-101 provided significant and medially meaningful efficacy, as assessed with the ACR CRISS score Results of secondary efficacy outcome measures support efficacy of JBT-101 in diffuse cutaneous SSc The safety profile of JBT-101 was acceptabl
Slide 28: Acknowledgments
ACKNOWLEDGMENTS We are grateful to the patients, physicians, and study staff at the sites who participated in this trial Investigator Staff Robert Spiera, MD Chris Hatzis, Emily Bakaj Lori Chung, MD Amanda Foster, Donna Adelman, Joel Nicholus Robyn Domsic, MD Dana Ivanco Tracy Frech, MD Fumiko Alger
Slide 29: Comments
COMMENTS a
Slide 30: Q&A
Q&A a
Slide 31: Appendix
APPENDIX a
Slide 32: Examples of Categorical CRISS Responses From the Trial
EXAMPLES OF CATEGORICAL CRISS RESPONSES FROM THE TRIAL Number following CRISS indicates CRISS score is ≥ that number A decrease in all numbers except FVC is an improvement The individuals measurements that comprise the CRISS do not necessarily improve or worsen in parallel Level Change from Baseline
Slide 33: CRISS Categories by Week
CRISS CATEGORIES BY WEEK *** CRISS20 CRISS40 * P ≤ 0.1, **P ≤ 0.05, ***P ≤ 0.01, mITT, observed values, one-tailed Fisher’s exact test JBT-101 = blue Placebo = orange CRISS30 CRISS50 ** * * ** *
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