Proteon Therapeutics, a company developing novel, first-in-class therapeutics to address the medical needs of patients with kidney and vascular diseases, has announced that its investigational treatment, vonapanitase, has received Breakthrough Therapy designation from the FDA for increasing arteriovenous fistula secondary patency (i.e., survival of the fistula without abandonment) and use for hemodialysis in patients on or expected to initiate hemodialysis.
Secondary patency and fistula use for hemodialysis are the co-primary endpoints of Proteon’s ongoing pivotal phase III clinical trial, PATENCY-2. As previously announced, the FDA has confirmed that the PATENCY-2 trial together with data from previously completed studies would provide the basis for a Biologics License Application (BLA) submission as a single pivotal study if PATENCY-2 is successful in showing statistical significance (p=0.05) on each of the co-primary endpoints.
The FDA awards Breakthrough Therapy designations to expedite the development and review of drugs that are intended to address a serious or life-threatening condition and preliminary clinical evidence indicates that the drug may offer a substantial improvement over available therapies on one or more clinically significant endpoints. Proteon’s Breakthrough Therapy designation is supported by data from PATENCY-1, the company’s first phase III clinical trial evaluating vonapanitase in patients with chronic kidney disease (CKD) undergoing surgical creation of a radiocephalic arteriovenous fistula. In that study, vonapanitase demonstrated clinically meaningful improvements in secondary patency and use for hemodialysis, although it did not meet the primary endpoint of improving primary patency.
“We believe the decision by the FDA to grant vonapanitase a Breakthrough Therapy designation speaks to the clinical importance of fistula survival and use for hemodialysis to patients with chronic kidney disease,” said Timothy Noyes, president and chief executive officer of Proteon Therapeutics. “Our productive ongoing dialogue with the FDA has helped to create a clear path forward for vonapanitase, and we look forward to continuing to work closely with the FDA to expedite our development efforts for this important program.”
Enrollment in the PATENCY-2 trial is expected to complete in the first quarter of 2018 and Proteon expects to report top-line data in the first quarter of 2019. Proteon also expects to submit a BLA to the FDA in 2019.
In the most severe stage of chronic kidney disease (CKD), also known as kidney failure, the kidneys can no longer function to sustain life. The majority of patients with kidney failure undergo chronic hemodialysis, which requires a high-flow vascular access to repeatedly connect the patient’s bloodstream to a hemodialysis machine for this life-saving treatment. The preferred form of vascular access for hemodialysis is a radiocephalic arteriovenous fistula, created when a surgeon connects a vein to an artery in the forearm, resulting in a substantial increase in blood flow and vein dilation.
Vonapanitase is an investigational drug intended to improve hemodialysis vascular access outcomes. Vonapanitase is applied in a single administration and is currently being studied in a phase III program in patients with CKD undergoing surgical creation of a radiocephalic arteriovenous fistula for hemodialysis. Vonapanitase has received Breakthrough Therapy, Fast Track and Orphan Drug designations from the FDA, and Orphan Medicinal Product designation from the European Commission, for hemodialysis vascular access indications. In addition, vonapanitase may have other surgical and endovascular applications in diseases or conditions in which vessel injury leads to blockages in blood vessels and reduced blood flow. Proteon is currently conducting a phase I clinical trial of vonapanitase in patients with peripheral artery disease (PAD).