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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 elucidated 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 QTORINTM Technology

PTX-022 leverages Palvella’s proprietary QTORINTM 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.

The VALO Study

We have completed enrollment for the VALO Study, a Phase 2/3 multicenter clinical study evaluating the safety and efficacy of PTX-022 (QTORINTM rapamycin) in adults with moderate to severe pachyonychia congenita (PC). To stay updated on our progress, please complete the form below.

Trial Requirements Include:
  • Moderate to Severe PC
  • Adults, Age 18 or Older
  • Able and willing to comply with all protocol-required activities

Resources for PC

Pachyonychia Congenita Project

PC Project is the leading patient advocacy group for PC, connecting patients, researchers, and physicians throughout the world in a united effort to help those with Pachyonychia Congenita.

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2018 FDA Externally-led Patient-Focused Drug Development Meeting for PC

PC project co-organized an Externally-led Patient Focused Drug Development Meeting (EL-PFDD) for FDA officials on April 6, 2018. Approximately 75 Pachyonychia Congenita (PC) patients and caregivers shared their voices through panel presentations, electronic polling, and moderated audience discussions.

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National Organization for Rare Disorders

NORD is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 280 patient organizations members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.

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Our Advocacy Work

Collaboration with patients and patient advocacy groups is at the heart of our work, bringing both drive and focus to everything we do.

2019 Pachyonychia Congenita Patient Support Meeting - Boston, MA
2018 London PC Patient Support Meeting
Wes Kaupinen (CEO, Palvella Therapeutics) and Jan Schwartz (Executive Director, PC Project) at the 2018 London PC Patient Support Meeting
FDA Patient-Focused Drug Development Meeting, 2018