MedPharm announce expansion of partnership with Palvella Therapeutics in developing a new treatment for the debilitating rare disease, pachyonychia congenita
MedPharm Ltd have announced the expansion of their partnership with Palvella Therapeutics, Inc., a Philadelphia-based biopharmaceutical company focused on developing and commercialising therapies for debilitating, rare genetic diseases. To date, MedPharm has employed its world-renowned 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 2/3 clinical study.
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.
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.
“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.”
Notes to editors
MedPharm is the world’s leading contract provider of topical and transdermal product design and formulation development services. MedPharm are experts at reducing risk and accelerating development times for generic and proprietary pharmaceutical customers through their unique, cost-effective and industry-leading performance testing models. Well established as the global leaders in dermatology, nail, mucosal membrane, and transdermal product development, MedPharm can also offer innovative solutions for ophthalmic and airway preparations recognised for their scientific rigour by regulators and investors. MedPharm has fully established R&D centres in the USA and UK and has its global HQ in Guildford, UK.
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Founded and led by life sciences and orphan drug veterans, Palvella Therapeutics is a rare disease biopharmaceutical company focused on developing and commercializing pathogenetically targeted therapies for debilitating, rare genetic diseases with no approved treatments. Palvella’s lead program PTX-022 is entering Phase 2/3 development for pachyonychia congenita, a rare, chronically debilitating, and lifelong genetic disease in which mutated genes responsible for keratin production lead to extreme cell fragility. This causes impaired ambulation which frequently necessitates the use of either ambulatory aids or alternative forms of mobility such as hands and knee crawling. More information can be found at www.palvellatx.com.