VAPAUS Study

About the VAPAUS Study

Leveraging key learnings from the largest clinical study in Pachyonychia Congenita to date, the phase 2/3 VALO study, Palvella is proud to continue our commitment to therapeutic development in Pachyonychia Congenita by sponsoring the VAPAUS study in collaboration with the Pachyonychia Congenita (PC) Project. VAPAUS is a pivotal, phase 3 study evaluating the safety and effectiveness of QTORIN™ rapamycin 3.9% in adults with PC. The VAPAUS study will determine if QTORIN™ rapamycin 3.9% is safe and effective in helping improve the overall condition of adults with moderate-to-severe Pachyonychia Congenita, measured by their difficulty in carrying out daily activities that involve being on their feet.

About Pachyonychia Congenita

Pachyonychia Congenita  is a rare, autosomal-dominant disease caused by a mutation in one or more of the genes responsible for the production of keratins. The production of mutated keratin filaments is the primary driver of Pachyonychia Congenita.  The stress from simple activities such as walking or standing causes trauma to these weakened cells, which triggers the production of mutated keratins, which in turn exacerbate these manifestations and contribute to increased functional debilitation and ability to do activities on one’s feet.   Furthermore, genetic defect leads to the exquisite plantar pain derived from blistering, the presence of neurovascular structures, dermal inflammation and atypical painful callous formations on the feet.

There are currently no FDA- approved therapies for Pachyonychia Congenita.

About QTORIN  rapamycin 3.9% anhydrous gel

QTORIN™ rapamycin 3.9% is a novel targeted topical therapy being developed to treat a number of rare genetic diseases including Pachyonychia Congenita. Rapamycin has been shown to inhibit the production of a kinase (a type of protein responsible for transcription) called mTOR, which is involved in the production of certain keratin proteins. In disorders such as Pachyonychia Congenita that are associated with overproduction of aberrant keratin proteins, the inhibition of mTOR may decrease the translation of mutant keratin-expressing genes and potentially block signaling pathways linked to skin fragility, keratoderma, pain and ambulatory impairment.