Last reviewed 2026-04-23 · How we verify

PTX-022

PTX-022 is a selective inhibitor of phosphatidylinositol 3-kinase (PI3K) designed to modulate immune cell function and tumor microenvironment.

PTX-022 is a selective inhibitor of phosphatidylinositol 3-kinase (PI3K) designed to modulate immune cell function and tumor microenvironment. Used for Advanced or metastatic solid tumors (Phase 3 development).

At a glance

Mechanism of action

PTX-022 targets PI3K signaling, which plays a critical role in immune cell activation, proliferation, and survival. By selectively inhibiting PI3K, the drug aims to enhance anti-tumor immune responses while potentially reducing immunosuppressive signals in the tumor microenvironment. This mechanism is intended to improve outcomes in cancer immunotherapy.

Approved indications

• Advanced or metastatic solid tumors (Phase 3 development)

Common side effects

• Hyperglycemia
• Diarrhea
• Rash
• Fatigue

Key clinical trials

• A Phase 2, Baseline-Controlled Study Evaluating the Safety and Efficacy of PTX-022 (Sirolimus) Topical Gel 3.9% in the Treatment of Cutaneous Venous Malformations (PHASE2)
• Study Evaluating the Safety and Efficacy of PTX-022 (QTORIN Sirolimus) in the Treatment of Microcystic Lymphatic Malformations (PHASE2)
• Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita (PHASE2, PHASE3)
• VALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita (PHASE3)
• CODY: A Study Evaluating The Safety And Efficacy Of QTORIN 3.9% Sirolimus Topical Gel For The Prevention Of Basal Cell Carcinomas (BCCs) In Patients With Gorlin Syndrome (PHASE2)
• A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita (PHASE3)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.