Two companies have enhanced their partnership to develop a treatment for an orphan disease
MedPharm has announced the expansion of its partnership with Palvella Therapeutics, a Philadelphia-based biopharmaceutical company focused on developing and commercialising therapies for debilitating, rare genetic diseases. This next step in the partnership with Palvella will be to develop a new treatment for the rare disease, pachyonychia congenita.
To date, MedPharm has employed its specialist formulation proficiency 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.
Commenting on the announcement, Prof Marc Brown, MedPharm’s CSO and Co-founder, said: “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. 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.”
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.