Partnership expands to develop a new treatment for pachyonychia congenita

19 Feb 2019

MedPharm and Palvella Therapeutics hope to develop the first approved treatment for PC in the US and Europe.

MedPharm has announced the expansion of their partnership with Palvella Therapeutics. 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). Most recently, MedPharm has made arrangements to manufacture the clinical (IMP) batches for use in Palvella’s upcoming Phase II/III clinical study.

Partnership expands to develop a new treatment for pachyonychia congenita

“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 CSO 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 CEO. “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 US or Europe.

As it has advanced the PTX-022 project, MedPharm has remained attentive to the needs of PC patients as part of the company’s partnership with Palvella, and has attended Pachyonychia Congenita Project (PC Project) patient meetings where research is discussed and patient feedback is collected.

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