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Our Pipeline

Leading the way for a new era in the treatment of rare genetic diseases.

Preclinical
Phase 1
Phase 2
Phase 3

PTX-022
(QTORINTM 3.9% rapamycin)

Pachyonychia Congenita

PTX-022
(QTORINTM 3.9% rapamycin)

Gorlin Syndrome

PTX-271
(QTORINTM platform)

Undisclosed Rare Disease

PTX-014

Undisclosed Rare Disease
PTX-022
(QTORINTM 3.9% rapamycin)
Phase 3
Pachyonychia Congenita
PTX-022
(QTORINTM 3.9% rapamycin)
Phase 2b
Gorlin Syndrome
PTX-271
(QTORINTM platform)
Preclinical
Undisclosed Rare Disease
PTX-014
Preclinical
Undisclosed Rare Disease

Our Programs

Pachyonychia Congenita (PC)

Pachyonychia Congenita (PC)

PTX-022 (QTORIN™ 3.9% rapamycin anhydrous gel), currently in a Phase 2/3 pivotal study, aims to be the first treatment for Pachyonychia Congenita by targeting the root genetic basis of the disease. PC is a chronically debilitating rare genetic disease that causes limited mobility and severe pain on the plantar aspects of the feet.

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Gorlin Syndrome

Gorlin Syndrome

Palvella is currently planning a Phase 2b clinical study for PTX-022 (QTORIN™ rapamycin) for patients with Gorlin Syndrome. Gorlin Syndrome is a rare genetic disease causing those affected to develop up to thousands of basal cell carcinomas, a form of cancerous skin tumors, often beginning in adolescence.

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QTORIN™ Platform

QTORIN™ Platform

Palvella’s proprietary QTORIN™ technology was developed over several years of research to overcome inherent challenges with topical delivery of mTOR inhibitors such as rapamycin – including chemical stability, skin penetration, and skin distribution.

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We’re uncovering more therapies every day. Stay updated.