Palvella Therapeutics Secures $10 Million Funding from Ligand Pharmaceuticals to Advance PTX-022 for Pachyonychia Congenita

VCBeat (WeChat ID: vcbeat) has learned that on December 18, U.S. rare-disease biopharmaceutical company Palvella Therapeutics entered into a development funding and royalty agreement with Ligand Pharmaceuticals (NASDAQ: LGND) to advance Palvella’s lead candidate, PTX-022 (QTORIN™, a rapamycin formulation), for the treatment of pachyonychia congenita (PC).

Under the terms of the agreement, Palvella will receive $10 million from Ligand in exchange for milestone payments and single-digit royalty rates on future sales of PTX-022. As part of the agreement, Palvella will continue to be responsible for all global clinical development, regulatory, manufacturing, marketing, and other commercialization activities for PTX-022.

Palvella is a rare disease biopharmaceutical company founded in Pennsylvania, United States, in 2015 by veterans of the life sciences and rare disease industries. The company is dedicated to serving patients with debilitating rare genetic diseases through the development and commercialization of life-changing, pathogen-targeted therapies, which are not yet approved.

“Ligand has a proven track record of collaborating with the world’s leading biopharmaceutical companies to develop important medicines,” said Wes Kaupinen, President and CEO of Palvella. “The partnership with Ligand marks another milestone in Palvella’s efforts to improve the quality of life for patients with PC, and this funding will further enhance the commercial potential of Palvella’s development of PTX-022.”

Currently, Palvella is conducting a Phase 2/3 clinical study of PTX-022 for the treatment of PC. PC is a rare, chronic, debilitating, lifelong monogenic disorder in which mutations in genes responsible for keratin production lead to dysregulated keratinocyte proliferation, thereby increasing skin fragility and compromising the plantar skin barrier function of the feet. Consequently, individuals affected by this disease experience difficulty walking and often require assistive devices or alternative modes of mobility, such as crawling on their hands and knees.

Leading scientists in the field of pachyonychia congenita (PC) research have identified the potential of QTORIN™ in treating PC. They posit that mTOR inhibitors exert a direct mechanistic effect on mutant keratin genes, which are the root cause of hereditary pachyonychia congenita. QTORIN™ utilizes a highly specific combination of excipients to enable the delivery of mTOR inhibitors to basal keratinocytes. These cells harbor the mutant keratin genes and constitute the primary pathological basis of congenital pachyonychia congenita.

PTX-022 is supported by multiple method-of-use patents in the United States, which will broadly cover the application of mTOR inhibitors in congenital pachyonychia until 2032. PTX-022 has also received FDA Fast Track designation, FDA Orphan Drug designation, and EMA Orphan Drug designation.

Ligand, founded in 1987 and headquartered in California, USA, is a publicly traded biopharmaceutical company focused on developing or acquiring technologies that assist pharmaceutical companies in discovering and developing drugs. (NASDAQ: LGND)

Ligand excels in drug discovery, early-stage drug development, product restructuring, and collaborations, frequently partnering with other pharmaceutical companies to leverage their core competencies for late-stage drug development. The company’s business model creates value for shareholders by providing a diversified portfolio of revenue streams from biotechnology and pharmaceutical products.