Rare Daily Staff

Palvella Therapeutics said that it had entered into a development funding and royalties agreement with Ligand Pharmaceuticals to advance Palvella’s lead product candidate PTX-022 for the treatment of pachyonychia congenita, a rare, genetic disease of the skin and nails of the hands and feet.

In pachyonychia congenita (PC), 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. People with the condition have difficulty walking, which frequently requires the use of ambulatory aids or can force them to crawl their hands and knees to get around.

PTX-022 is a novel formulation of the drug rapamycin, an immunosuppressant and mTOR inhibitor. Researchers discovered the mTOR inhibitors exhibit a direct mechanism of action on the mutant keratin genes underlying pachyonychia congenita. PTX-022 was developed in partnership with MedPharm and leverages Palvella’s proprietary and patent-pending QTORIN formulation and delivery technology the cells that harbor the mutant genes.

Under the terms of the agreement, Palvella will receive $10 million from Ligand in exchange for milestones and a tiered single-digit royalty on future sales of PTX-022. As part of the agreement, Palvella will continue to have responsibility for all clinical development, regulatory, manufacturing, marketing, and other commercialization activities on a worldwide basis.

“Ligand has a long and successful track record in partnering with leading biopharmaceutical companies to develop some of the world’s most important medicines,” said Wes Kaupinen, president and CEO of Palvella. “This agreement with Ligand represents another milestone for Palvella in our ambition to improve the lives of PC patients who currently have no FDA-approved treatments.”

In partnership with Pachyonychia Congenita Project, Palvella is entering a phase 2/3 clinical study of PTX-022 for the treatment of pachyonychia congenita.

December 19, 2018
Photo:
Wes Kaupinen, president and CEO of Palvella

 

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