Pavella Therapeutics, a rare disease biopharmaceutical company focused on developing and commercializing pathogenetically targeted therapies for serious genetic diseases with no approved treatments, announced the closing of an oversubscribed $45 million Series C financing. Leading biotech investors participating in this round include CAM Capital, Samsara BioCapital, BVF Partners L.P., Adams Street Partners, Opaleye Management, Ligand Pharmaceuticals, Agent Capital, BioAdvance and Nolan Capital (the investment fund of former AveXis CEO Sean Nolan). Proceeds will be used to accelerate the company’s pipeline of rare disease therapies, including late-stage programs in Pachyonychia Congenita (PC) and Gorlin Syndrome.
“Palvella was founded on fundamental beliefs that every individual with a rare disease deserves a treatment and that significant value creation occurs upon the introduction of the first approved therapy for a serious rare genetic disease,” stated Wes Kaupinen, President and Chief Executive Officer of Palvella. “The capital invested from this highly regarded syndicate of public market and venture investors strengthens our team’s unwavering commitment to develop and commercialize targeted therapies to individuals suffering from serious, life-altering rare genetic diseases such as pachyonychia congenita (PC) and Gorlin syndrome.”
Pavella’s lead program is PTX-022 (QTORIN 3.9% rapamycin anhydrous gel) for the treatment of adults with PC, a rare, chronically debilitating and lifelong genetic disease. PTX-022 is a novel formulation of rapamycin and leverages Palvella’s QTORIN™ technology to enable localized distribution of rapamycin into the suprabasal keratinocytes which express the mutant keratin genes that are the primary defect in PC. There are currently no FDA-approved therapies for the over 9,000 individuals estimated to be living with PC in the U.S. PTX-022 is being developed in partnership with the Pachyonychia Congenita Project.
A Phase 2/3 pivotal study, VALO, completed enrollment in March of 2020. Top-line results from VALO are expected to be available in the fourth quarter of 2020. PTX-022 has received FDA Fast Track Designation, FDA Orphan Drug Designation and EMA Orphan Drug Designation.
Palvella’s second candidate, PTX-367 (QTORIN™ rapamycin), is being developed in partnership with the Gorlin Syndrome Alliance. PTX-367 is poised to enter a late-stage clinical study for patients with Gorlin syndrome, a genetic disease caused by a mutation in PTCH1, a tumor suppressor gene. Gorlin syndrome affects an estimated 10,000 people in the U.S. and there are no FDA-approved therapies.
PTX-022 is protected by multiple issued method-of-use patents in the U.S. broadly covering the use of rapamycin and derivatives thereof in treating PC that expire as late as 2032 and an allowed patent application in the U.S. covering the use of anhydrous gel formulations of rapamycin for treating PC and Gorlin syndrome that will expire in 2038.