Principia: Consistent Positive Data in Thrombocytopenia Phase 1/2 Trial

Principia Biopharma Inc. (NASDAQ: PRNB) announced consistent positive data from an ongoing Phase 1/2 trial of its investigational treatment, PRN1008, in 31 highly treatment-resistant and refractory patients (median of six prior therapies) with immune thrombocytopenia (ITP). The data from the trial is being presented today by David Kuter, M.D., Director of Clinical Hematology at Massachusetts General Hospital and Professor of Medicine at Harvard Medical School, at an oral scientific session of the 61st American Society of Hematology Annual Meeting (ASH).

“We are very encouraged by the data so far and pleased to see meaningful clinical responses and quick onset in this highly pre-treated patient population. We are also pleased to observe that this investigational drug so far has not seen the typical BTK class side effects,” said Dr. Kuter, the trial’s principal investigator.

Of the 31 patients, 39 percent (80 percent confidence interval (CI) 28, 50), irrespective of dose and duration of treatment, 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, 45 percent (80 percent CI 34, 57) of enrolled patients achieved any two platelet counts ≥50,000/µL. Most patients who achieved the primary endpoint had a platelet count >30,000/µL by the first week of treatment. Preliminary data on 13 patients treated at higher doses (300mg and 400mg twice daily) and who had completed at least 12 weeks of therapy, demonstrated a response rate of 54 percent (80 percent CI 37, 70) and 62 percent (80 percent CI 44, 77) for both endpoints respectively. To date PRN1008 has been well-tolerated at all doses studied, whether given as a monotherapy or with allowed concomitant ITP therapy (thrombopoietin and steroids), with no reported treatment related bleeding or thrombotic events. Related treatment emergent adverse events (TEAEs) were reported in 35 percent of patients and were all grade 1 or 2.

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