Palvella Therapeutics has announced positive topline results from its Phase III SELVA study of QTORIN 3.9% rapamycin anhydrous gel (QTORIN rapamycin), an investigational therapy under development for the treatment of microcystic lymphatic malformations (microcystic LMs).
The company has been developing the therapy with partner MedPharm, which has worked closely alongside Palvella's research and development team and leadership to advance the formulation strategy from initial concept through clinical development and preparation for commercial manufacturing.
Palvella's QTORIN rapamycin has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for the treatment of microcystic LMs, as well as an FDA Orphan Products Development grant.
QTORIN rapamycin is also under development for cutaneous venous malformations and clinically significant angiokeratomas.
According to MedPharm, Rapamycin is an "inherently challenging molecule" due to its high molecular weight, poor solubility, chemical instability and complex physicochemical properties.
The company provided support in advancing the formulation strategy for QTORIN rapamycin, conducting a multi-year formulation development programme which involved complex physicochemical optimisation to achieve the performance characteristics required by Palvella.
MedPharm also stated that it supported Palvella in scale-up and manufacturing readiness, including enabling the production of clinical trial supply and preparation for anticipated near-term commercial-scale manufacturing.
"I am delighted that MedPharm has been able to contribute to Palvella reaching this important milestone," said Bill Humphries, CEO of MedPharm.
As global leaders in the development of semi-solid drug formulations, MedPharm looks forward to supporting Palvella with their vision to become the leading rare disease biopharma company serving patients with serious, rare skin diseases and vascular malformations.
MedPharm added that it has also been selected by Palvella to advance the development of QTORIN pitavastatin for the treatment of porokeratosis, a serious and rare genetic skin disease characterised by chronic, often itchy lesions and an increased risk of progression to skin cancer.
Currently, there are no FDA-approved therapies for porokeratosis. If approved, QTORIN pitavastatin could become the first therapy specifically targeted to treat this condition, addressing a significant unmet medical need for patients.