ITP Press Release
Principia Biopharma Inc. (the “Company”) issued a press release on December 7, 2019 announcing the presentation of consistent positive data from its ongoing Phase 1/2 clinical trial of its proprietary drug candidate, PRN1008, for the treatment of immune thrombocytopenia (ITP) at an oral scientific session at the 61st American Society of Hematology Annual Meeting.
The analysis presented included 31 adult patients. The median treatment duration of the reported data was 12 weeks (range 0.1 to 41.9). PRN1008 starting doses in the trial were 200mg once daily, 400 mg once daily, 300 mg twice daily, and 400 mg twice daily, with intra-patient dose escalation allowed every four weeks. Of the 31 enrolled patients, 39% (80% confidence interval (CI) 28, 50) achieved the trial’s primaryend-point of two or more consecutive platelet counts of³ 50,000/µL, separated by at least five days, and increased by³ 20,000/µL from baseline without requiring rescue medication. In addition, 45% (80% CI 34, 57) of enrolled patients achieved any two platelet counts³ 50,000/µL. Most patients who achieved the primary endpoint had a platelet count of³ 30,000/µL by the first week of treatment. These results were observed despite the limited duration of therapy and patients at multiple escalating dose levels. The data on asub-set of 13 patients treated at higher doses and who had completed at least 12 weeks of therapy, demonstrated a response rate of 54% (80% CI 37, 70) and 62% (80% CI 44, 77) for both endpoints, respectively. The data on all 31 patients showed PRN1008 had 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 were reported in 35% of patients and were all grade 1 or 2. There have been four serious adverse events (SAEs) reported in the ongoing Phase 1/2 trial in ITP and one SAE in the ongoing Phase 2 Part B trial in pemphigus, none of which were related to drug. These results are preliminary in nature and may change as additional patients are enrolled and progress in the trial. A copy of the press release is attached hereto as Exhibit 99.1 and is incorporated herein by reference.
ITP Presentation
As detailed in the press release, the Company also held an investor event on December 7, 2019 to review the ITP data and the ITP landscape. The investor event was available via live webcast. A copy of the ITP data slide presentation that was presented at the event is attached hereto as Exhibit 99.2 and is incorporated herein by reference.
Disclosure Channels to Disseminate Information
The Company disseminates information to the public about the Company, its drug candidates and pipeline, its science and technology and other matters through various channels, including the Company’s investor relations website (https://ir.principiabio.com), SEC filings, press releases, public conference calls and webcasts, in order to achieve broad,non-exclusionary distribution of information to the public. The Company encourages investors and others to review the information it makes public through these channels, as such information could be deemed to be material information.
| Item 9.01 | Financial Statements and Exhibits. |
(d) Exhibits
