Principia Biopharma: PRN1008 achieved primary endpoint in 50% of patients

Principia Biopharma says PRN1008 achieved primary endpoint in 50% of patients  Principia announced top-line data from the completed open-label Phase 2 trial of PRN1008 in patients with pemphigus, or PV, and pemphigus foliaceus, or PF and the initiation of a Phase 3 trial of PRN1008 in pemphigus. The primary efficacy endpoint of the Phase 2 trial, control of disease activity within four weeks, was achieved by more than 50% of patients and PRN1008 was generally well tolerated. Based on the results of the Phase 2 trial, Principia has initiated the PEGASUS study, a global, randomized, double-blind, placebo-controlled, pivotal, Phase 3 clinical trial of PRN1008 in patients with moderate to severe pemphigus. The Phase 2 trial included 27 patients with pemphigus in an open-label trial to evaluate PRN1008’s potential to induce rapid onset of clinical response, enable tapering and/or avoidance of CS use, and lower autoantibody levels while having minimal and reversible effects on the patient’s immune system. The primary efficacy endpoint was the initial control of disease activity during the first four weeks of therapy, where new lesions cease to form and existing lesions begin to heal, without the need for prednisone-equivalent CS doses greater than 0.5 mg/kg/day. The Phase 2 trial included patients with newly diagnosed or relapsing, mild or moderate pemphigus, for whom an initial period of PRN1008 monotherapy or combination with low doses of CS is judged to be clinically acceptable. PRN1008 was administered orally twice daily with a treatment period of 12 weeks and a post-treatment follow-up period of 12 weeks to patients with PV and a small number of patients whom, although originally diagnosed with PV, had a PF antibody profile. Ninety-five percent of patients had confirmatory anti-desmoglein antibodies. The drug was generally well tolerated. Principia has initiated an extension to this trial to increase the active treatment period from 12 to 24 weeks, with a post-treatment follow-up period of four weeks, and anticipates announcing top-line results of this extension in the second half of 2019.

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