Rigel Conference Call - R788 Phase 2 Trial in ITP. December 4, :30pm Pacific/4:30pm Eastern

Transcription

1 Rigel Conference Call - R788 Phase 2 Trial in ITP December 4, :30pm Pacific/4:30pm Eastern

2 Agenda Safe Harbor Statement D. Vance Introduction and Overview J. Gower R788 Phase 2 Trial in ITP Results E. Grossbard Trial Commentary J. Bussel Q&A 1

3 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 R788 and the timing of results thereof. Any statements contained in this call that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "anticipates," "plans," "intends," "expects" and similar expressions are intended to identify these forward-looking statements. These forward-looking statements are based on Rigel's current expectations and involve risks and uncertainties. There are a number of important factors that could cause Rigel's results to differ materially from those indicated by these forward-looking statements, including risks associated with the timing and success of clinical trials, and other risks detailed in Rigel's SEC reports, including its Quarterly Report on Form 10-Q for the quarter ended September 30, Rigel expressly disclaims any obligation or undertaking to update the forward-looking statements discussed in this call. 2

4 Participants Rigel Senior Management James Gower - Chairman and Chief Executive Officer Elliott Grossbard - SVP of Medical Development Donald Payan - EVP and Chief Scientific Officer Raul Rodriguez - EVP and Chief Operating Officer Dolly Vance - SVP and General Counsel Ryan Maynard - VP and Chief Financial Officer Weill Cornell Medical Center James Bussel - Principal Investigator and Director of the Platelet Research and Treatment program at the Phyllis and David Komansky Center for Children's Health and NewYork-Presbyterian Hospital/Weill Cornell Medical Center Anna Podolanczuk Allison Schindler 3

5 Overview R788 in ITP - Phase 2 Results R788 Phase 2 Trials in ITP, RA and Lymphoma - Update on Status and Timeframes 4

6 Background Syk is a key kinase enzyme involved in Fc receptor (FcR) and B cell receptor signaling in various inflammatory cells FcRγ activation and signaling has been implicated in the immune destruction of platelets, a hallmark of ITP R788, an orally bioavailable small molecule, is a potent and relatively selective inhibitor of Syk In a murine model of ITP, treatment with R788 ameliorated platelet destruction 5

7 Study Design Adult patients with chronic refractory ITP Primary Efficacy: Platelet counts increased by 20K from baseline, to over 30K platelets Dosing: treated with mg PO BID of R788 Duration: 30 days dosing with extension, most responders treated for weeks 6

8 Patients Enrolled Age (years) Gender (n) Baseline characteristics median 66 range 31,81 Male 6 Female 10 Race (n) White or Caucasian 13 Black or African American 0 Hispanic or Latino 1 Asian 1 Other 1 Duration of ITP (years) median 10 range 1,29+ Prior treatment history (n,%) =3 prior treatments 16 (100) Prior treatments (n,%) Splenectomy 12 (75) IVIG 15 (94) Anti-D 8 (50) Steroids 16 (100) Rituximab 13 (81) Anti-CD40L 2 (13) H Pylori 1 (6) Thrombopoietic agents 5 (31) Vincristine 5 (31) Azathioprine 6 (38) Danazol 8 (50) GMA161 3 (19) Baseline platelet count (lowest count within 1 mo of starting drug) median 16 range 3,28 Older population Highly refractory ITP ITP for 10 years (median) All tried at least 3 prior treatments All not controlled w/steroids 13 failed Rituxan 5 failed TPO agents 12 had failed splenectomy Very difficult population to treat Frequent ITP related health issues 7

9 Efficacy Results Clinically-significant response was seen in 12/16 (75%) patients Increased platelet counts Reduced need for IVIg treatment Steroid tapering Durable response (platelet count increased by >20K to at least 30K on at least 75% of study visits) was seen in 8/16 people 4 patients were withdrawn from the study due to failure to respond 1 patient withdrew despite adequate platelet response, due to hospitalization for UTI and subsequent DVT 1 patient withdrew despite adequate platelet response, due to nausea and vomiting, but was re-enrolled with concomitant antinausea medications 8

10 Efficacy Results Median Max Platelet Count (in 000s) Responder (Maintained) n=8 128 Responder (Non- Maintained) n= Non-Responder n=4 = Baseline Plt Counts % Visits >30K Plts 100% 48% 0% % Visits >50K Plts 93% 19% 0% %Visits Treated w/ IVIg 0% 3% 53% 9

11 Safety Adverse Reaction N=16 N=14 Nausea 4 Diarrhea 4 SBP increased by >10 4 Drowsiness 3 Weight gain >5 kg 3 Constipation 2 ALT >2x ULN 2 Vomiting 1 Abdominal pain 1 Headache 1 The nausea and vomiting were amenable to treatment with anti-nausea agents. 10

12 R788 Broader Clinical Safety Experience This R788 trial in ITP is part of a broader clinical effort: Approx. 236 patients have been treated with R788 in Phase 2 trials in: RA: >140 patients treated at doses of 50/100/150mg BID Lymphoma: >80 patients treated at doses of 200/250mg BID ITP: 16 patients treated at doses of mg BID 11

13 Conclusions This preliminary ITP study found that: R788 is effective in maintaining adequate platelet counts in some patients with severely refractory ITP The majority (75%) of patients on drug achieved a clinical response and a subset maintained a durable platelet response Increased platelet counts reduced need for IVIg and allowed for steroid tapering The most frequent side-effects were GI related (nausea, diarrhea); nausea and vomiting were amenable to treatment with anti-nausea agents 12

14 Comments James Bussel, M.D., Principal Investigator 13

15 Q&A 14