Palvella Raises $45 Million to Speed Development of Rare Disease Therapies

Rare Daily Staff

Palvella Therapeutics, a company focused on developing targeted therapies for serious genetic diseases, said it raised $45 million in an oversubscribed series C financing.

Investors in the round included CAM Capital, Samsara BioCapital, BVF Partners, Adams Street Partners, Opaleye Management, Ligand Pharmaceuticals, Agent Capital, BioAdvance, and Nolan Capital.

“Palvella was founded on fundamental beliefs that every individual with a rare disease deserves a treatment,” said Wes Kaupinen, president and CEO of Palvella, “and that significant value creation occurs upon the introduction of the first approved therapy for a serious rare genetic disease.”

Proceeds from the financing will be used to accelerate development of Palvella’s pipeline of rare disease therapies, including late stage programs in pachyonychia congenita and Gorlin syndrome.

Pachyonychia congenita (PC) is a rare, chronically debilitating and lifelong monogenic disease in which mutations of genes responsible for keratin production lead to dysregulated keratinocyte proliferation, increased skin fragility and impaired skin barrier function on the plantar aspects of the feet. As a result, affected individuals experience pain and difficulty with walking, which frequently necessitates the use of either ambulatory aids or alternative forms of mobility such as crawling on hands and knees. There are currently no approved therapies for PC.

Palvella’s experimental therapy PTX-022 is being studied in partnership with the Pachyonychia Congenita Project, with enrollment in a pivotal phase 2/3 study completed in March. PTX-022 is a novel formulation of rapamycin, which was shown by geneticist Roger Kaspar to have a direct mechanism of action on mutant keratin genes, which are the root cause in PC. PTX-022 leverages Palvella’s proprietary technology, Qtorin, to enable localized distribution of rapamycin into the suprabasal keratinocytes, which express the mutant keratin genes that are the primary defect in PC. The company expects top-line results from the study to be available in the fourth quarter of 2020.

In partnership with the Gorlin Syndrome Alliance, Palvella’s second candidate, PTX-367 will enter into a late-stage clinical study for individuals with Gorlin syndrome, a genetic disease caused by a mutation in PTCH1, a tumor suppressor gene. Individuals afflicted with Gorlin syndrome can develop hundreds of basal cell carcinomas (BCCs), oftentimes beginning in adolescence. BCCs are a malignant skin cancer requiring repeated and potentially disfiguring surgical removal for individuals with Gorlin syndrome. Palvella initiated internal research efforts on the potential for Qtorin rapamycin in Gorlin syndrome in 2017. Gorlin syndrome affects an estimated 10,000 people in the United States and there are no FDA-approved therapies.

PTX-022 has received Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration and Orphan Drug designation from the European Medicines Agency.

Photo: Wes Kaupinen, president and CEO of Palvella