Rare Daily Staff

Palvella Therapeutics said it has begun the phase 3 pivotal portion of its phase 2/3 study of PTX-022 for the treatment patients with pachyonychia congenita.

Pachyonychia congenital (PC) is a rare, chronically debilitating and lifelong monogenic disease in which mutations of genes responsible for keratin production lead to dysregulated keratinocyte proliferation, increased skin fragility and impaired skin barrier function on the plantar aspects of the feet. As a result, affected individuals experience pain and difficulty with walking, which frequently necessitates the use of either ambulatory aids or alternative forms of mobility such as crawling on hands and knees. There are currently no approved therapies for PC.

The phase 2/3 VALO study is the largest clinical study ever conducted of a therapy for PC and is currently enrolling patients at nine sites across the United States. In the phase 3 double-blind, placebo-controlled, randomized withdrawal portion of the study, enrolled patients who met the pre-specified clinical response criteria during the phase 2 portion will be assigned to one of three arms: placebo, twice-daily PTX-022, or once-daily PTX‑022. Following completion of phase 3, Palvella intends to initiate an open-label extension program where patients will have the option to continue to receive study drug.

“Commencing the Phase 3 portion of this study represents a significant milestone for all who have been involved in accelerating the development of PTX-022 for pachyonychia congenita,” said Palvella President and CEO Wes Kaupinen. “We’re grateful for the efforts of the patients, their families, the VALO clinical investigators and study coordinators, and our partners at PC Project. We look forward to reporting the study results in the second half of 2020.”

PTX-022 is a novel formulation of rapamycin which was discovered by leading scientists in the field of PC research to have a potential direct mechanism of action on the mutant keratin genes which are the root cause of PC. PTX-022 leverages Palvella’s formulation and delivery technology that employs a specific composition of excipients to enable distribution of rapamycin into the basal keratinocytes which harbor the mutant keratin genes that are the primary defect in pachyonychia congenita. In addition to PC, Palvella is investigating the use of its technology in other serious, rare genodermatoses.  

PTX-022 has received FDA Fast Track designation, FDA Orphan Drug designation, and EMA Orphan Drug designation. Photo: Wes Kaupinen, president and CEO of Palvella Therapeutics  

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