Palvella Therapeutics focuses on developing and commercializing novel therapies for serious, rare genetic skin diseases that currently lack FDA-approved treatments. The company advances a broad pipeline built on its patented QTORIN platform, initially targeting lifelong rare skin disorders. Palvella works with patient advocacy organizations and patient registries to design accelerated development programs to expedite patient access to targeted therapies. Its lead product candidate is QTORIN 3.9% rapamycin anhydrous gel, being developed for pachyonychia congenita, microcystic lymphatic malformations, and prevention of basal cell carcinomas in Gorlin syndrome. QTORIN rapamycin has received FDA Fast Track Designation for all three indications. The company recently completed a Series D financing to support advancement of QTORIN rapamycin across these indications. Palvella Therapeutics is a Wayne, PA–based rare disease biopharmaceutical company focused on developing and commercializing pathogenetically targeted therapies for serious genetic diseases with no approved treatments. Its lead program, PTX-022 (QTORIN™ 3.9% rapamycin anhydrous gel), is in a Phase 2/3 pivotal study for pachyonychia congenita (PC), a rare, chronically debilitating and lifelong genetic disease estimated to affect more than 9,000 individuals in the U.S. The company partners with patient advocacy organizations and patient registries to design fit-for-purpose, accelerated clinical development programs aimed at expediting targeted therapies to patients lacking approved options. Palvella intends to use the proceeds from the financing to advance PTX-022 in adults with PC. Wes Kaupinen serves as President and CEO. In conjunction with the funding, Scott Morenstein of CAM Capital and Cory Freedland of Samsara BioCapital joined Palvella’s Board of Directors. Palvella Therapeutics is a Wayne, PA-based rare disease biopharmaceutical company focused on developing and commercializing pathogenetically targeted therapies for debilitating, rare genetic diseases with no approved treatments. The company is led by Wes Kaupinen, president and chief executive officer. Its lead program, PTX-022 (QTORIN™ rapamycin formulation), is entering Phase 2/3 development for pachyonychia congenita (PC). PC is a rare, chronically debilitating lifelong monogenic disease in which mutations in keratin genes lead to dysregulated keratinocyte proliferation, increased skin fragility and impaired plantar skin barrier function. Palvella will continue to retain responsibility for all clinical development, regulatory, manufacturing, marketing and other commercialization activities worldwide. The company has secured external development funding to advance PTX-022.

Total raised

$93M

Funding rounds

3

Latest round

Series D

Latest activity

Jan 2023