Robust platelet responses with cevidoplenib in ITP

Cevidoplenib was associated with robust platelet responses in patients with persistent and chronic immune thrombocytopenia (ITP) who had received at least one prior therapy. The agent was well-tolerated, and no new safety signals emerged in the current phase 2 trial.

Thrombopoietin receptor agonists are common second-line therapies for patients with ITP. “However, 30% of the patients do not respond to these agents. Next to the emerging therapies rilzabrutinib and efgartigimod, cevidoplenib, an investigational SYK inhibitor is being evaluated for the ITP population.” introduced Dr Jun-Ho Jang (Samung Medical Center, South Korea). He presented the findings of the phase 2, multicenter, randomised, double-blind, placebo-controlled trial (NCT04056195), which assessed the SYK inhibitor cevidoplenib in 60 participants with persistent and chronic ITP who had failed at least 1 prior therapy [1]. The participants were randomised 1:2:2 to placebo, 200 mg cevidoplenib (twice daily), or 400 mg cevidoplenib (twice daily). The primary endpoint was the participant’s platelet response at any visit during the 12-week treatment period, defined as a platelet count ≥30,000/μL and doubling the baseline count.

The primary endpoint was achieved by 33% of the participants in the placebo arm and by 54% in the cevidoplenib arms (P=0.333, see Figure). The rate of participants who reached the primary endpoint was numerically higher in the 400 mg dose group than in the 200 mg dose group (64% vs 46%). Participants on cevidoplenib were more likely to achieve the secondary endpoint of reaching ≥2 consecutive platelet counts ≥30,000/μL, irrespective of the dosing, than participants receiving a placebo (38% vs 50% vs 8%).

Grade 3 or 4 adverse events occurred in 16.7% of the participants on placebo and 7.7% and 22.7% of the participants on 200 mg and 400 mg cevidoplenib, respectively. The most common treatment-related adverse events were ALT increase (alanine aminotransferase, 8.3%), AST increase (aspartate aminotransferase, 6.3%), and nausea (6.3%).

“Further evaluation of cevidoplenib in a larger number of patients for an extended period is needed to confirm the durability of the observed clinical benefits,” concluded Dr Jang.

 

Figure: Primary efficacy results (platelet response) [1]



BID, bis in die, twice daily.

 

1. Jang J-H, et al. Cevidoplenib, a selective inhibitor of SYK, in persistent and chronic ITP: phase 2 study. Late-breaking oral session, EHA 2023 Annual Congress, 8─11 June, Frankfurt, Germany.

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Posted on 2 August 20232 August 2023