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PTX-367

Development stage: Unknown
Lead developer: Palvella Therapeutics
Modality: Small Molecules, Transdermal Patches → Device-based Delivery → Drug Delivery Systems, Hydrogel Systems → Drug Delivery Systems
Administration: Topical

Overview

PTX-367 is a proprietary topical gel formulation of the mTOR inhibitor sirolimus (rapamycin), developed using Palvella Therapeutics’ QTORIN technology for chronic, localized delivery into diseased skin. It is being advanced for rare genodermatoses including pachyonychia congenita and Gorlin syndrome, conditions driven by aberrant keratinocyte proliferation and signaling where local mTOR pathway inhibition is expected to reduce lesion burden and associated morbidity.[5][3][9][13]

Brand names: QTORIN rapamycin
Other names: QTORIN sirolimus topical gel

Targets

mTOR (Mammalian target of rapamycin kinase)

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