RARE Daily

Palvella Raises $9.7 Million to Support Late-Stage Development and Commercialization of Rare Genetic Skin Disease Therapy

January 6, 2023

Palvella Therapeutics, a rare disease company focused on novel therapies for serious, rare genetic skin diseases, said it has raised $9.7 million in an initial closing of its series D financing of up to $37.7 million.

Petrichor led the financing with participation by new investor Gore Range Capital and existing investors Samsara BioCapital, BVF Partners, Agent Capital, Nolan Capital, and BioAdvance. Tadd Wessel, founder and managing partner of Petrichor, has joined the Palvella Board of Directors.

Proceeds from the financing will be used to advance the development of Palvella’s lead product candidate Qtorin 3.9 percent rapamycin anhydrous gel for the treatment of the rare conditions Pachyonychia Congenita (PC) and Microcystic Lymphatic Malformations (Microcystic LM), and for the prevention of Basal Cell Carcinomas (BCCs) in Gorlin Syndrome (GS). These three initial clinical indications for Qtorin rapamycin share similarities in the underlying disease pathology whereby the mammalian target of rapamycin, or mTOR, pathway is overactivated leading to chronically debilitating disease burdens for affected individuals. By inhibiting the mTOR pathway and limiting the systemic exposure of rapamycin, the company believes Qtorin rapamycin has the potential to become the first approved therapy and standard of care for these indications. Qtorin rapamycin has received U.S. Food and Drug Administration Fast Track designation for PC, Microcystic LM, and for the prevention of BCCs in GS.

“The funding will accelerate our efforts to develop and commercialize Qtorin rapamycin for individuals suffering from Pachyonychia Congenita, Microcystic Lymphatic Malformations, and Gorlin Syndrome,” said Wes Kaupinen, founder and CEO of Palvella.

Palvella anticipates several key catalysts within the next three fiscal quarters, including reporting phase 2 top-line results in Microcystic LM in the first quarter of 2023, phase 2b top-line results in GS in the first half of 2023, and phase 3 top-line results in PC in mid-2023.

Author: Rare Daily Staff

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