PRINCIPIA BIOPHARMA INC. Item 8.01 Other Events ITP Press Release
On
The analysis presented included 47 heavily pre-treated (median of six prior therapies) adult patients enrolled with a median follow-up of 18 weeks. The primary endpoint was the proportion of patients able to achieve two or more consecutive platelet counts, separated by at least five days, of ³ 50,000/µL and an increase of platelet count of ³20,000/µL from baseline, without use of rescue medication.
Rilzabrutinib treatment at 400 mg twice daily led to both a rapid response detectable at the first platelet measurement (day eight), and a durable response. Fifty percent of patients who started at 400 mg twice daily and had at least 12 weeks of treatment (n=26) achieved the primary endpoint (80 percent confidence interval (CI) 38, 62). In the overall patient population, the primary endpoint was met by 43 percent of patients (80 (CI) 34, 52), irrespective of dose and duration of treatment. Among the patients who started on 400 mg twice daily, 53 percent achieved a clinically significant platelet count of ³30,000/µL on day eight. Among the patients that achieved the primary endpoint, 79 percent had a platelet count ³30,000/µL by day eight, and these patients had sustained responses ³50,000/µL for 71 percent of the time. In addition, responders achieved platelet counts ³20,000/µL above baseline 88 percent of the time. To date rilzabrutinib has been well tolerated, whether given as a monotherapy or with allowed concomitant ITP therapy (thrombopoietin receptor agonists and corticosteroids), with no reported treatment related bleeding or thrombotic events. Related treatment emergent adverse events were reported in 45% of patients and were all grade 1 or 2.
Based on this data, the Company's goal is to initiate a pivotal Phase 3 trial,
assuming no future COVID-19 related impact, by the end of 2020. In addition, the
Company believes that a single Phase 3 trial, if successful, will be acceptable
for approval in
These results are preliminary in nature and may change as patients progress in the trial and as additional patients may be enrolled. A copy of the press release is attached hereto as Exhibit 99.1 and is incorporated by reference.
Pemphigus Data Release
On
In the Phase 2 Part B trial of 15 patients with newly diagnosed or relapsed mild-to-severe pemphigus, rilzabrutinib demonstrated a 40% complete remission (CR) rate after 24 weeks of treatment, while the median corticosteroid (CS) dose was reduced significantly. The median CS dose was 18 mg/day (0.201 mg/kg/day) at baseline and in the 14 patients that completed 12 and 24 weeks of treatment the median CS dose decreased to 11 mg/day (0.125 mg/kg/day) at 12 weeks and decreased again to 6 mg/day (0.076 mg/kg/day) at 24 weeks. Additionally, 60 percent and 87 percent of patients achieved control of disease activity (CDA) by weeks 4 and 12, respectively. The results also demonstrated that while patients on 400 mg once-a-day dosing were able to reach CDA, 400 mg twice-a-day dosing is needed to achieve rapid CR rates. Related treatment emergent adverse events were mild-to-moderate and were all grade 1 or 2.
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),
--------------------------------------------------------------------------------
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits Exhibit No. Description 99.1 Press Release issued byPrincipia Biopharma Inc. datedJune 12, 2020 .
--------------------------------------------------------------------------------
© Edgar Online, source
