Palvella Therapeutics Announces Positive Topline Results from Phase 2 Study of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the Treatment of Microcystic Lymphatic Malformations, a Serious, Rare Genetic Skin Disease with No FDA-approved Therapies

QTORIN™ rapamycin generally well-tolerated; no drug related severe adverse events and no observed rapamycin in systemic circulation

100% of participants were either “Much Improved” or “Very Much Improved” as rated by the Clinician Global Impression of Change following 12-weeks of QTORIN™ rapamycin

End of Phase 2 meeting completed with U.S. Food and Drug Administration in February 2023; pending additional interactions with FDA, anticipate potential initiation of pivotal Phase 3 study in second half of 2023

FDA previously granted Fast Track Designation and Orphan Drug Designation to QTORIN™ rapamycin for Microcystic Lymphatic Malformations

QTORIN™ rapamycin has potential to become first therapy and standard of care for the estimated more than 30,000 individuals with Microcystic Lymphatic Malformations in U.S., if approved

Excerpt from the Press Release:

WAYNE, Pa., March 09, 2023 (GLOBE NEWSWIRE) — Palvella Therapeutics, Inc., a late clinical-stage biopharmaceutical company whose vision is to become the leading rare disease company focused on developing and commercializing novel therapies to treat individuals suffering from serious, rare genetic skin diseases for which there are no FDA-approved therapies, today announced positive topline results from the Company’s Phase 2 study of QTORIN™ rapamycin in Microcystic Lymphatic Malformations (Microcystic LM). Microcystic LM is a rare, chronically debilitating genetic disease caused by dysregulation of the PI3K/mTOR pathway. The disease is characterized by localized masses of malformed lymphatic vessels that protrude through the skin barrier and persistently leak lymph fluid (lymphorrhea) and bleed, often leading to recurrent serious infections and cellulitis. There are no FDA-approved treatments for the estimated more than 30,000 individuals living with Microcystic LM in the U.S.

“People living with Microcystic LMs face daily challenges and frequent, sometimes life-threatening hospitalizations due to cellulitis. Current treatments are inadequate, invasive, and do not address the underlying mechanisms of this debilitating disease,” said James Treat, MD, a pediatric dermatologist at Children’s Hospital of Philadelphia and study investigator. “The encouraging results from the Phase 2 study of QTORIN™ rapamycin build upon the large, growing evidence base supporting targeted therapeutic intervention of Microcystic LMs via the mTOR pathway.

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