Slide 1: Rheumatoid Arthritis Program: Top-line Phase 2 Results
For personal use only Rheumatoid Arthritis Program: Top-line Phase 2 Results
Slide 2: Cautionary Note Regarding Forward-Looking Statements
For personal use only CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS This presentation includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, le
Slide 3: Greatest Unmet Need in Rheumatoid Arthritis: Biologic Refractory Patients
Greatest Unmet Need in Rheumatoid Arthritis: Biologic Refractory Patients For personal use only In 2016, over 5m individuals are impacted by rheumatoid arthritis (RA) across the 7 major pharmaceutical markets (7MM) with ~70% suffering from moderate to severe disease1,2 Major advances in the trea
Slide 4: MPC-300-IV for Treatment of Chronic Inflammatory Diseases Through Immunomodulation
MPC-300-IV for Treatment of Chronic Inflammatory Diseases Through Immunomodulation For personal use only Inflammation induces production of immuno-modulatory factors by Mesenchymal Precursor Cells (MPCs), which regulate multiple immune pathways concurrently MPCs have receptors that respond to infl
Slide 5: MPC-300-IV for Modulation of Cytokine Production by Synoviocytes in RA
MPC-300-IV for Modulation Of Cytokine Production By Synoviocytes/Synovial Fibroblasts In Rheumatoid Arthritis TNFα For personal use only LPS Acidosis Synoviocytes IL-6 PGE2 MPC TGFβ PGE2/TGFβ Mediated Inhibition of NF-κB in synoviocytes PGE2 and TGFβ secreted by MPCs following surface signaling in
Slide 6: A Sheep Model of Collagen-Induced Arthritis (CIA)
A Sheep Model of Collagen-Induced Arthritis (CIA) Thorpe et al. Clinical & Exp. Rheumatology 10: 143 –150 (1992) For personal use only Knee joint Bone and Blood Aim: to determine the anti- inflammatory effects and dose Synovial Fluid ranging of ovine MPCs in an ovine model of collagen-induced acute
Slide 7: Features of CIA and Human RA: Invading Synovial Pannus and Cartilage Erosion
Features Of CIA And Human RA: Invading Synovial Pannus And Cartilage Erosion For personal use only Col II Injected Joint (Day 42) Normal joint Inflammatory cell pannus Erosion of cartilage 7
Slide 8: Allogeneic Sheep MPCs Reduce Synovial Pannus/Inflammatory Cytokines, Improve Symptoms in CIA
Allogeneic Sheep MPCs Reduce Synovial Pannus/ Inflammatory Cytokines, Improve Symptoms in CIA For personal use only 8
Slide 9: MSB-RA001: Study Design
MSB-RA001 : Study Design Phase 2 double-blind, randomized, placebo-controlled, dose-escalating study comparing a single For personal use only infusion of two MPC doses (1M/kg and 2M/kg) with placebo in patients with active RA – Inadequate response to at least 1 anti-TNF +/- other biologics – On a
Slide 10: MSB-RA001: Safety Summary
MSB-RA001 : Safety Summary For personal use only All infusions were well-tolerated without any acute infusion reactions or adverse events (AEs) reported either during infusion or the 6-h post-infusion monitoring period No serious AEs (SAEs) were reported during the 12- week primary study period
Slide 11: Profile of Marketed Non-TNFα/JAK Inhibitors Suggest Clinical Need for Reduced Safety Risk
Profile of Marketed Non-TNFα/JAK Inhibitors Suggest Clinical Need for Agents with Reduced Safety Risk For personal use only To date, no safety concerns have been identified with MPC-300-IV across multiple therapeutic indications, including in biologic refractory RA patients Currently marketed no
Slide 12: RA001: ACR70 Response (%) over Weeks
RA001: ACR70 Response (%) over 12 Weeks All Subjects and Subgroup of 1-2 Prior Biologics1,2 For personal use only All Subjects 1-2 Prior Biologics P la c e b o M P C 1 M /kg M P C 2 M /kg NOTE: 1) Values are observed n/N (%) at each time point 2) *P values vs. Placebo computed using Fisher exact tes
Slide 13: RA001: ACR50 Response (%) over Weeks
RA001: ACR50 Response (%) over 12 Weeks All Subjects and Subgroup of 1-2 Prior Biologics1,2 For personal use only All Subjects 1-2 Prior Biologics 60 P la c e b o (N = 1 6 ) M P C 1 M / k g (N = 1 6 ) 50 M P C 2 M / k g (N = 1 6 ) A C R 5 0 (% o f P a tie n ts) 40 * p = 0 .0 4 3 30 * p = 0 .0 4 3 20
Slide 14: One Dose of MPC-300-IV Provides Efficacy in the Biologic Refractory Population
One Dose of MPC-300-IV Provides Compelling Efficacy at 12 weeks in the Biologic Refractory Population* ACR70 @ 3 Months For personal use only 40% Range of Marketed Products 36% 35% 30% Range of Pipeline Products 27% 25% 20% 22% 15% 14% 10% 5% 11% 6% 0% Inhibitors of IL-6, Jak Inhibitors2 MPC 2m/kg M
Slide 15: RA001: ACR20 Response (%) over Weeks
RA001: ACR20 Response (%) over 12 Weeks All Subjects and Subgroup of 1-2 Prior Biologics1,2 For personal use only All Subjects 1-2 Prior Biologics P la c e b o M P C 1M /kg M P C 2M /kg NOTE: 1) Values are observed n/N (%) at each time point 2) *P values vs. Placebo computed using Fisher exact test
Slide 16: Health Assessment Questionnaire – Disability Index (HAQ-DI)
Health Assessment Questionnaire – Disability Index (HAQ-DI) Least Squares Mean (SE) Change from Baseline at 4 and 12 Weeks All Subjects and Subgroup of 1-2 Prior Biologics1,2,3 For personal use only All Subjects 1-2 Prior Biologics P la c e b o (n = 8 ) B a s e lin e : 1 .3 P la c e b o B a s e lin
Slide 17: HAQ-DI: Percent Reaching Minimal Clinically Important Reduction
Health Assessment Questionnaire – Disability Index (HAQ-DI) Percent that reached minimal clinically important reduction of -0.22 at 4 and 12 weeks All Subjects and Subgroup of 1-2 Prior Biologics1 For personal use only All Subjects 1-2 Prior Biologics P la c e b o (N = 9 ) P la c e b o M P C 1 M / k
Slide 18: Disease Activity Score-28 –CRP (DAS28-CRP)
Disease Activity Score-28 –CRP (DAS28-CRP) at Week 12 LS mean change from baseline and proportion of patients that achieved low disease activity state (defined as DAS28 <3.2) All Subjects and Subgroup of 1-2 Prior Biologics For personal use only All Subjects 1-2 Prior Biologics P la c e b o B a s e
Slide 19: MPC-300-IV: Conclusions
MPC-300-IV: Conclusions For personal use only The biologic refractory rheumatoid arthritis (RA) population accounts for approximately one-third of all RA patients who have received anti-TNF or other biologic agents, is the hardest to treat, and requires new therapies that are both effective and sa
Slide 20: References
References References For personal use only Abatacept (OrenciaR ) Prescribing Information Álvaro-Gracia JM et al. Ann Rheum Dis. 2016 Jun 7. doi:10.1136/annrheumdis-2015-208918 (in press) Burmester GR et al. Lancet 2013; 381:451-60 Cohen SB et al. Arthritis Rheum 2006; 54:2793–2806 Emory P et al. An