TAVALISSE delivered rapid, robust, and durable improvements in platelet counts
Response rates achieved in the 24-week evaluation period (double-blind, placebo-controlled trials)
Median time to first response: 15 days4,‡
*Stable platelet response: achievement of a platelet count ≥50 x 109/L on ≥4 of the 6 visits during weeks 14-24 without need for rescue treatment.
†Overall response: achievement of a platelet count ≥50 x 109/L at least once during the first 3 months/12 weeks without need for rescue treatment.
‡Time to first response was a post hoc analysis.
TAVALISSE DEMONSTRATED DURABLE BENEFIT BEYOND 3 YEARS
Overall response observations from a 3-year post hoc interim analysis3,§,||
§1 month = 4 weeks. Data cutoff date: March 8, 2018.
||Overall response: achievement of a platelet count ≥50 x 109/L at least once during the first 3 months/12 weeks without need for rescue treatment.
- Patients were evaluated every 2 weeks in the placebo-controlled trials (FIT-1 + FIT-2) and no less often than monthly for 18 months, and then bimonthly for up to 5 years in the open-label extension study (FIT-3)2
- A total of 102 patients exited the placebo-controlled trials early (at or after week 12) and entered the open-label extension study (FIT-3)1
RESPONSE TO TAVALISSE WAS SEEN IN PATIENTS ACROSS ALL LINES OF THERAPY
A post hoc analysis of the phase 3 clinical studies was conducted to evaluate patient response by line of therapy
Time to response (platelet count ≥50 x 109/L) in responders to TAVALISSE5:
- 56% of 2nd-line patients responded within 4 weeks
- 76% of 2nd-line patients responded within 12 weeks
- 81% of patients across all lines of therapy responded within 12 weeks
¶Data cutoff date: December 2019.
#Platelet response was assessed by the proportion of patients achieving platelet counts of ≥50 x 109/L and of ≥30 x 109/L at any visit (without rescue therapy within 4 weeks).
**Percentage of patients achieving platelet counts of ≥30 x 109/L calculated using data on file.3
This analysis measured the percentage of treatment days that patients maintained their response
The majority of 2nd-line patients with platelet counts ≥50 x 109/L maintained their response for >36 months5
- Rates of adverse events in these subgroups were consistent with those in patients treated with TAVALISSE in the placebo-controlled trials4,5
††Durability of response: median percentage of treatment days that patients maintained a response of ≥50 x 109/L or ≥30 x 109/L, with loss of response at the first of two platelet counts ≤30 x 109/L or ≤20 x 109/L, respectively, at least 4 weeks apart, or use of rescue therapy.
TAVALISSE demonstrated efficacy across patient subgroups compared to placebo
Treatment benefit favored TAVALISSE in the phase 3 clinical trials (FIT-1 + FIT-2)3
‡‡Post hoc subgroup.
TAVALISSE PATIENTS HAD FEWER BLEEDING EPISODES AND REQUIRED LESS RESCUE MEDICATION COMPARED TO PLACEBO
TAVALISSE patients had a lower incidence of bleeding episodes than placebo patients1,3
- In the placebo-controlled trials, the incidence of bleeding in patients who received TAVALISSE (regardless of responder status) was 29%, compared with 37% in patients who received placebo
Of patients treated with TAVALISSE in the phase 3 clinical studies (FIT-1 + FIT-2 + FIT-3), 59% had no bleeding episodes.3,§§
TAVALISSE patients required less rescue medication than placebo patients4
- In the placebo-controlled trials, 30% of patients who received TAVALISSE (regardless of responder status) required rescue medication, compared with 45% of patients who received placebo
Of patients treated with TAVALISSE in the phase 3 clinical studies (FIT-1 + FIT-2 + FIT-3), 59% did not require rescue medication at any visit.3,§§
§§Compiled from interim analysis. Data cutoff: March 8, 2018.
References: 1. TAVALISSE® [package insert]. South San Francisco, CA: Rigel Pharmaceuticals, Inc.; April 2018. 2. Bussel JB, Arnold DM, Boxer MA, et al. Long-term fostamatinib treatment of adults with immune thrombocytopenia during the phase 3 clinical trial program. Am J Hematol. 2019;94(5):546-553. 3. Data on file, Rigel Pharmaceuticals, Inc. April 2018. 4. Bussel J, Arnold DM, Grossbard E, et al. Fostamatinib for the treatment of adult persistent and chronic immune thrombocytopenia: results of two phase 3, randomized, placebo-controlled trials. Am J Hematol. 2018;93(7):921-930. 5. Boccia R, Cooper N, Ghanima W, et al. Fostamatinib is an effective second-line therapy in patients with immune thrombocytopenia. Br J Haematol. 2020;190(6):933-938.