Wayne-based Palvella Therapeutics stock jumped 35 percent following the announcement of positive late-stage testing for Qtorin rapamycin, its lead drug candidate for a rare skin disease, writes John George for the Philadelphia Business Journal.
The biopharmaceutical company now intends to file its first new drug application for the topical gel with the Food and Drug Administration in the second half of the year.
Qtorin rapamycin is an experimental therapy for microcystic lymphatic malformations, a rare and chronically debilitating genetic condition marked by malformed lymphatic vessels that can leak lymph fluid and bleed when breaking through the skin. More than 30,000 people in the United States currently live with a microcystic lymphatic malformations diagnosis.
Currently, treatment for the disease relies on laser therapy, but this approach addresses only surface-level issues and does not target the deeper abnormal vessels. Qtorin rapamycin is intended to shrink skin lesions while also slowing the growth of abnormal blood vessels within the skin.
In the latest study involving 50 patients, the drug achieved its primary objective as well as all secondary endpoints.
Palvella founder and CEO Wesley H. Kaupinen stated earlier that the diseases targeted by the company represent “multibillion-dollar market opportunities.”
Read more about Palvella Therapeutics’ stock and its lead drug candidate in the Philadelphia Business Journal.
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