Clinical hold of deucrictibant for on-demand treatment lifted by FDA

Agency's pause on testing of deucrictibant as prophylactic therapy remains

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has lifted its hold on clinical trials of deucrictibant for the on-demand treatment of hereditary angioedema (HAE).

The therapy’s developer Pharvaris now expects to resume RAPIDe-2 (NCT05396105), a long-term extension study evaluating PHVS416, an immediate-release capsule formulation of deucrictibant, for the on-demand treatment of HAE.

“The lift of the clinical hold on our on-demand clinical trials enables us to continue development of PHVS416 (deucrictibant immediate-release capsules) in the U.S., including resuming RAPIDe-2, our extension study for acute treatment of attacks,” Peng Lu, MD, PhD, chief medical officer of Pharvaris, said in a company press release.

Deucrictibant, previously known as PHA121, is an oral therapy designed to block the activity of receptors for bradykinin — the signaling molecule that drives swelling attacks in HAE. The FDA placed a clinical hold on the therapy last year, based on a review of nonclinical data.

The lift of the hold for the on-demand use of deucrictibant was based on interim data from a 26-week nonclinical study, which is still ongoing. The FDA’s hold on clinical testing of deucrictibant as a prophylactic (preventive) treatment for HAE remains in place pending the completion of that study.

“The 26-week nonclinical study to address the remaining hold on prophylactic deucrictibant in the U.S. is still progressing and we plan to submit the data from that study to the FDA by the end of the year,” Lu said.

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Deucrictibant as a prophylactic treatment

Pharvaris is currently running a Phase 2 clinical trial, called HAE CHAPTER-1 (NCT05047185), to assess the safety and efficacy of deucrictibant as a prophylactic treatment in adults with HAE types 1 and 2. While the study is still on hold in the U.S., recruitment is ongoing at sites in Austria, Ireland, Bulgaria, Canada, Spain, Germany, Israel, Italy, Poland, and the U.K. Results are expected by year’s end.

“Based on current enrollment, we confirm that top-line data from CHAPTER-1, our Phase 2 proof-of-concept study of PHVS416 for the prophylactic treatment of HAE, remains on track to be announced by the end of the year,” Lu said.

With these Phase 2 studies well underway, Pharvaris also is looking to start Phase 3 testing of deucrictibant for on-demand control of HAE swelling.

“Our team plans to request an end of Phase 2 meeting with the agency and is preparing for RAPIDe-3, our global Phase 3 study of PHVS416 for the on-demand treatment of HAE, to include U.S. sites,” Lu said.