SAN MATEO, Calif.SAN MATEO, Calif.SAN MATEO, CA - AcelRx Pharmaceuticals, Inc. (Nasdaq: ACRX), (AcelRx), a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for use in medically supervised settings, today announced that it is advancing Niyad™ (a lyophilized formulation of nafamostat) into a registrational study following the recent approval of an Investigational Device Exemption (IDE) submission to the United States Food and Drug Administration (FDA). This clinical trial will evaluate the safety and efficacy of Niyad to support a premarket approval application (PMA) expected to be submitted in the second half of 2024. The single study will consist of 166 adult patients undergoing renal replacement therapy (RRT) who cannot tolerate heparin or are at risk for bleeding. If approved, Niyad would be the first-ever FDA-approved regional anticoagulant for the extracorporeal circuit in the U.S.
"We are thankful that the FDA has reviewed and approved our IDE application so we can now proceed with our registrational study to evaluate our lead product candidate, Niyad, which we believe to be a paradigm-shifting product candidate for the medically supervised setting," said Vince Angotti, Chief Executive Officer of AcelRx. "We are on track to start this study later this year, with top-line data expected by mid-2024 and submission of a PMA planned in the second half of 2024."
The registration study is designed as a prospective, double-blinded trial to be conducted at approximately 10 U.S. hospital intensive care units that will enroll and evaluate 166 patients that will be randomized into either active or placebo arm. The primary endpoint of the study is mean post-filter activated clotting time (ACT) over the first 24 hours versus placebo. Key secondary endpoints include filter lifespan, number of filter changes over 72 hours, number of transfusions over 72 hours and dialysis efficacy (based on urea concentration) over the first 24 hours.
Pamela P. PalmerPamela P. Palmer, M.D., Ph.D., Chief Medical Officer and Co-Founder of AcelRx, commented, "Importantly, we are encouraged by our interactions with the FDA - not only by being granted Breakthrough Device Designation status for Niyad, but also in terms of the Agency's acceptance of a straightforward registrational trial design to evaluate Niyad. Based on the feedback from the Emergency Use Authorization request, the FDA stated that AcelRx should proceed with the clinical study. We're looking forward to our upcoming Investigator's Meeting just prior to starting enrollment. We remain steadfast in our commitment to advance Niyad for use as a regional anticoagulant during renal replacement therapy, as current options are limited and patients often receive care below the recommended international standards. We also look forward to publishing our independent qualitative market research related to these findings later this year."
