We believe steps should be taken, not endured.

PTX-022 for Pachyonychia Congenita

PTX-022 (QTORIN 3.9% rapamycin anhydrous gel) is an investigational therapy currently in a multi-center Phase 2/3 pivotal study. PTX-022 aims to be the first approved therapy for patients with Pachyonychia Congenita.

PC: A Disease of Limited Mobility and Pain

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 difficulty with ambulation which frequently necessitates the use of either ambulatory aids or alternative forms of mobility such as crawling on hands and knees. PC affects an estimated 9,330 patients in the US¹. There are currently no FDA or EMA approved treatments for PC.

^{1. Gallagher et al., Prevalence of Diagnosed/Highly Symptomatic Pachyonychia Congenita (PC) Patients Managed Annually by US Dermatologists-National Real World Occurrence (RWO) Physician Study. J Dermatol Dis 2019, 6:1}

Addressing the Root Cause of the Disease

The potential for rapamycin in PC was discovered by leading scientists and geneticists in the field of PC research who proposed a direct mechanism of action of mTOR inhibitors on translationally repressing the mutant keratin genes which are the root cause of PC.

PTX-022 is supported by multiple layers of protection, including multiple issued method of use patents in the U.S. broadly covering the use of mTOR inhibitors in PC. PTX-022 has been awarded Fast Track Designation, FDA Orphan Drug Designation, and EMA orphan drug designation.

Our QTORIN^TM Technology

PTX-022 leverages Palvella’s proprietary QTORIN^TM technology, which enables distribution of rapamycin into the basal keratinocytes which harbor the mutant keratin genes that are the primary defect in pachyonychia congenita. It also provides significant stability for high-concentration formulations at room temperature.