To date, MedPharm has employed its specialist formulation expertise to support Palvella’s development of a novel, high-strength rapamycin topical formulation for application to the skin (PTX 022) as a disease-modifying treatment for pachyonychia congenita (PC). MedPharm has also made arrangements to manufacture the clinical (IMP) batches for use in Palvella’s upcoming Phase 2/3 clinical study.

“MedPharm is very proud to be part of a collaboration with Palvella and the PC community which will potentially benefit PC patients directly. Currently this is a serious unmet medical need that has no approved treatments,” said Professor Marc Brown, MedPharm’s chief scientific officer and co-founder. “I have met many PC patients over the years and am excited to be continuing our support of Palvella and PC Project as they move closer to making a real difference to these patients’ lives.”

“We are very pleased with the expertise and the results that MedPharm have brought to Palvella’s lead development program for PC,” added Wes Kaupinen, Palvella’s president and chief executive officer. “In addition to their rigorous scientific approach, the actions of the entire MedPharm organization, from their dedicated scientists to their senior leadership, have shown a deep commitment to working towards the betterment of patients suffering from PC.”

PC is a rare, chronically debilitating, and lifelong genetic disease in which mutated genes responsible for keratin production lead to extreme cell fragility. This dramatically limits patients’ ability to walk and perform everyday tasks. Most patients suffer from deformed nails and almost all experience some level of pain. No treatments are currently approved for PC in the USA or Europe.