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Principia Presents Consistent Positive Data of PRN1008 for Immune Thrombocytopenia in Ongoing Phase 1/2 TrialORLANDO, Fla., Dec. 07, 2019 (GLOBE NEWSWIRE) -- Principia Biopharma Inc. (Nasdaq: PRNB), a late-stage biopharmaceutical company focused on developing novel therapies for immune mediated diseases, today 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. This analysis includes 31 adult patients who had a median baseline platelet count of 13,000/µL (entry criteria were two platelet counts <30,000/µL within 15 days prior to treatment). The patient population for this analysis had a median duration of disease of 7.8 years and a median of six prior ITP treatments. 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, and with the trial having a current median treatment duration of 12 weeks (range, 0.1-41.9). 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 throbotic events. Related treatment emergent adverse events (TEAEs) were reported in 35 percent of patients and were all grade 1 or 2. “As we continue to execute on our PRN1008 and BTK franchise strategy, these data represent yet another validation of our scientific platform as well as demonstration of our ability to develop oral therapies without compromising the desired efficacy or a favorable tolerability profile. We now have proof of concept in two immune mediated diseases,” said Martin Babler, Principia’s chief executive officer. These results are preliminary in nature and may change as patients progress in the trial and as additional patients may be enrolled. A complete analysis of this trial will be presented at an upcoming medical conference. Principia will hold an investor event today at 7:00 pm Eastern time to review the ITP data presented at ASH and the ITP landscape. The event will be available via live webcast on the “Events and Presentations” page of the “Investors” section of Principia’s website at www.principiabio.com. A replay of the webcast will be available after the conclusion of the live presentation. About ITP and PRN1008 About Principia Biopharma Forward-Looking Statements Investor Contact Christopher Chai, CFO [email protected] Media Contact Paul Laland, VP of Corporate Communications [email protected] 415.519.6610 |