Slide 1: Rigel Conference Call: FIT Phase 3 Study in ITP
Rigel Conference Call FIT Phase 3 Study in ITP October 20, 2016 5:00am PT / 8:00am ET
Slide 2: Agenda
Agenda Safe Harbor Statement D. Vance Introduction and Overview R. Rodriguez FIT Phase 3 Program Results Duliege FIT Phase 3 Commentary J. Bussel Q&A
Slide 3: Safe Harbor Statement
Safe Harbor Statement In the conference call accompanying these slides, Rigel management will be making some forward-looking statements, including statements relating to Rigel's plans for the future clinical development of fostamatinib and the timing of results thereof. Any statements contained in t
Slide 4: Introduction and Overview
Introduction and Overview Raul Rodriguez President and Chief Executive Officer
Slide 5: Participants
Participants Rigel Senior Management: Raul Rodriguez – President and Chief Executive Officer Anne-Marie Duliege, MD – Executive Vice President and Chief Medical Officer Dolly Vance – Executive Vice President, Corporate Affairs and General Counsel Ryan Maynard – Executive Vice President and Chief Fin
Slide 6: ITP Background
ITP Background Significant unmet need: Characterized by the destruction of platelets by the body’s own immune system Increased risk of severe bleeding events Can result in serious medical complications or even death Heterogeneous patient population, difficult to predict which of available therapies
Slide 7: Why Fostamatinib?
Why Fostamatinib? Novel mechanism of action Oral inhibitor of SYK, a key player in platelet destruction in ITP May uniquely address the underlying autoimmune basis of ITP by impeding platelet destruction Based on Phase 2 study: timely, substantial, and enduring benefit Primary endpoint responders do
Slide 8: Program Results
FIT Phase 3 Program Results Anne-Marie Duliege, MD Executive Vice President and Chief Medical Officer
Slide 9: FIT Phase 3 Program Design
Study 049 Open-Label Long-Term Extension *If <50,000 platelets/L at Week 3, then increase to 150 mg bid (bid = twice a day) ** n=118 with monitored data as of June 2016 Study 048 Phase 3 (FIT 2 Study) N=74 Fostamatinib 100 mg bid* N=50 Placebo n=24 RANDOMIZE 2:1 Study 047 Phase 3 (FIT 1 Study) N=76
Slide 10: Study 047: Primary Endpoint
From August announcement: FIT 1 Phase 3 – Study 047: Primary Endpoint p=0.0261 n=9 *Stable platelet response (primary endpoint): platelet count of >50,000/L on > 4 of the last 6 visits between Week 14 and Week 24 9/51=18% n=0 18% 0% 0% 20% 40% 60% 80% 100% Fostamatinib (n=51) Placebo (n=25) Percent
Slide 11: Study 047: Adverse Events
From August announcement: FIT 1 Phase 3 - Study 047: Adverse Events Number (n) and % of Patients with > one Adverse Event (AE) Fostamatinib N=51 Placebo N=25 n (%) N (%) Any AE 49 (96%)* 19 (76%) Serious AEs 8 (16%) 5 (20%) Treatment-related AEs 39 (77%) 7 (28%) Gastrointestinal complaints** 31 (61%
Slide 12: FIT Phase 3 Program Design
Study 049 Open-Label Long-Term Extension *If <50,000 platelets/L at Week 3, then increase to 150 mg bid (bid = twice a day) ** n=118 with monitored data as of June 2016 Study 048 Phase 3 (FIT 2 Study) N=74 Fostamatinib 100 mg bid* N=50 Placebo n=24 RANDOMIZE 2:1 Study 047 Phase 3 (FIT 1 Study) N=76
Slide 13: Study 048: Relevant Patient Baseline Characteristics
FIT 2 Phase 3 - Study 048: Relevant Patient Baseline Characteristics Patient Population: Adults with chronic/persistent ITP, defined as having consistently low platelet levels of <30,000 platelets/µL of blood All subjects have received prior treatment for ITP Fostamatinib N=50 Placebo N=24 Total N=7
Slide 14: Study 048: Primary Endpoint
FIT 2 Phase 3 – Study 048: Primary Endpoint p=.15 n=9 *Stable platelet response (primary endpoint): platelet count of >50,000/L on at least 4 of the last 6 visits between Week 14 and 24 9/50=18% n=1 18% 4% 0% 20% 40% 60% 80% 100% Fostamatinib (n=50) Placebo (n=24) Percent of Patients Stable Platelet
Slide 15: Study 048: Adverse Events
FIT 2 Phase 3 - Study 048: Adverse Events Number and % of Patients with > one Adverse Event (AE) Fostamatinib N=51 Placebo N=23 Any AE 36 (71%)* 18 (78%) Serious AEs 5 (10%) 6 (26%) Treatment-related AEs 20 (39%) 6 (26%) Gastrointestinal complaints** 11 (22%) 5 (22%) Nausea 4 (8%) 3 (13%) Diarrhea 9
Slide 16: Combined Study 047+048: Relevant Patient Baseline Characteristics
FIT Phase 3 – Combined Study 047+048: Relevant Patient Baseline Characteristics Patient Population: Adults with chronic/persistent ITP, defined as having consistently low platelet levels of <30,000 platelets/µL of blood All subjects have received prior treatment for ITP Fostamatinib N=101 Placebo N=
Slide 17: Combined Study 047+048: Median Platelet Counts Over Time
FIT Phase 3 – Combined Study 047+048: Median Platelet Counts Over Time Week (/L) *Data not shown for time points with n<3 patients (except for the placebo non responder, n=1) 50,000 0 20,000 40,000 60,000 80,000 100,000 120,000 140,000 0 2 4 6 8 10 12 14 16 18 20 22 24 Fosta Responder (n=18) Fosta N
Slide 18: Combined Study 047+048: Adverse Events
FIT Phase 3 – Combined Study 047+048: Adverse Events * AEs in the fostamatinib group were generally mild (74%) or moderate (22%) ** Nausea, vomiting, diarrhea, or abdominal pain Number (n) and % of Patients with > one Adverse Event (AE) Fostamatinib N=102 Placebo N=48 n (%) n (%) Any AE 85 (83%)* 37
Slide 19: Combined Study 047+048: Sub-Group Analysis
The response rate was statistically superior in the fostamatinib group vs. placebo across all sub-groups: Prior splenectomy / No prior splenectomy Prior TPO agent exposure / TPO naïve Baseline platelet count <15,000 / > 15,000 Fostamatinib benefit is consistent across patients with various clinical
Slide 20: FIT Phase 3 Program Design
Study 049 Open-Label Long-Term Extension *If <50,000 platelets/L at Week 3, then increase to 150 mg bid (bid = twice a day) **n=118 with monitored data as of June 2016 Study 048 Phase 3 (FIT 2 Study) N=74 Fostamatinib 100 mg bid* N=50 Placebo n=24 RANDOMIZE 2:1 Study 047 Phase 3 (FIT 1 Study) N=76 F
Slide 21: Long Term Extension Study
FIT Phase 3 Long Term Extension Study 049 Enduring benefit in fostamatinib patients who had a stable platelet response in Study 047 and 048 Have generally maintained platelet counts >50,000/L in Study 049 Median count = 96,000/L; total median exposure of 13 months to fostamatinib Response rate in pa
Slide 22: Fostamatinib Program Commentary
Fostamatinib FIT Phase 3 Program Commentary James Bussel, MD Principal Investigator
Slide 23: Regulatory Path Forward
Regulatory Path Forward In Next 3 Months Input from regulatory consultants Feed-back from FDA Pending FDA’s Feed-back NDA submission still planned for Q1 2017 Pending FDA’s Acceptance of the NDA submission: Potential FDA Advisory Committee Review (Q4 2017) FDA decision on approval anticipated in Q1
Slide 24: Conclusions
Conclusions Key Findings: All FIT studies demonstrate consistent clinical benefit in treating ITP Safety profile consistent with prior experience. AEs related to GI were most frequent. AEs were generally mild or moderate. Positive Risk/Benefit For Patients who met Primary Endpoint: Timely platelet r
Slide 25: Conclusions
Conclusions Key Findings: + All FIT studies demonstrate consistent clinical benefit in treating ITP Safety profile consistent with prior experience. AEs Auto-antibodies related to GI were most frequent. AEs were generally < AQ mild or moderate. + Positive Risk/Benefit For Patients who met Primary En
Slide 26: Q&A
Q&A R IGE L 2