To date, the Phase 1/2 trial of PRN1008 has enrolled 26 adult patients who have had two platelet counts < 30,000/µL within 15 days prior to treatment. Oral PRN1008 starting doses were 200mg once daily, 400mg once daily, 300mg twice daily, and 400mg twice daily, with intra-patient dose escalation allowed every four weeks, with the trial having a current median treatment duration of 12.7 weeks (range 0.14 to 39.71).

Of the 26 patients enrolled to date, 39 percent (80% confidence interval (CI) 27.3, 51.0) achieved the trial’s primary endpoint of ≥ 2 consecutive platelet counts of ≥ 50,000/µL, separated by at least five days, and increased by ≥ 20,000/µL from baseline without requiring rescue medication. In addition, 46 percent (80% CI 34.2, 58.5) of enrolled patients achieved any 2 platelet counts ≥ 50,000/µL.

- From Principia press release.

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