WAYNE, PA — Palvella Therapeutics, Inc., a clinical-stage biopharmaceutical company, recently announced topline results from VAPAUS, a 24-week, randomized, double-blind, placebo-controlled pivotal Phase 3 study of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of Pachyonychia Congenita (PC).
In the Intent-to-Treat population (n=87), QTORIN rapamycin did not show a treatment effect on the Patient Global Assessment of Activities Difficulty primary endpoint, a daily patient-reported outcome measure which assessed the difficulty of patients carrying out activities on their feet, when compared to placebo. QTORIN rapamycin was well-tolerated in the study and no participants withdrew due to drug-related adverse events. No drug-related serious adverse events were reported, and all other adverse events were deemed mild or moderate in nature with the most common treatment emergent adverse events reported in the category of infections and infestations (nasopharyngitis, COVID-19, and upper respiratory tract infections).
“We would like to sincerely thank PC patients, the leadership of PC Project with whom we have an incredible partnership, the VAPAUS study investigators in the U.S. and U.K., the Palvella team, and our investors who together supported this study,” said Wes Kaupinen, Founder and Chief Executive Officer. “While we are disappointed that the VAPAUS study did not meet its primary endpoint, we hope that Palvella’s efforts to raise awareness of PC and encourage the advancement of novel therapies for this devastating disease will be enduring contributions that one day result in a better quality of life for this deserving patient community.”
The company plans to further analyze and share the Phase 3 VAPAUS study results with key stakeholders, including PC Project, physicians, and scientists. Based on these top-line results, Palvella does not plan to invest in additional clinical studies or commercial preparation activities for QTORIN rapamycin for the treatment of PC.
Mr. Kaupinen continued, “Our vision at Palvella remains focused on becoming the leading rare disease company developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases in indications for which there are no FDA-approved therapies.”