Palvella Received
QTORIN™ rapamycin has Potential to be First FDA Approved Therapy and Standard of Care in the
“We are pleased to expand our strategic partnership with
According to the terms of the amended agreement, Palvella received an upfront payment of
“Based on the positive Phase 2 results announced earlier this year and FDA’s recent granting of Breakthrough Therapy Designation, we are encouraged by the significant commercial potential of QTORIN rapamycin to be the first FDA approved therapy for Microcystic Lymphatic Malformations in the U.S.,” said
QTORIN rapamycin, the lead product candidate from Palvella’s QTORIN platform, is a novel, 3.9% rapamycin anhydrous gel currently under development for the treatment of Microcystic LMs and other serious, functionally debilitating skin diseases driven by the overactivation of the mammalian target of rapamycin (mTOR) pathway. There are currently no FDA-approved treatments for the estimated more than 30,000 diagnosed patients with Microcystic LMs in the
Palvella previously announced positive Phase 2 results from a multi-center, open-label trial of once-daily QTORIN rapamycin in Microcystic LMs. In the Phase 2 study, 100% of participants (n=12) were either “Much Improved” or “Very Much Improved” as rated by the Clinician Global Impression of Change after 12 weeks of once-daily QTORIN rapamycin treatment. The
QTORIN rapamycin is protected by multiple issued composition patents in the
About Palvella Therapeutics
Founded and led by rare disease veterans, Palvella Therapeutics is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no FDA-approved therapies. We are developing a broad pipeline of product candidates based on our patented QTORIN™ platform, with an initial focus on serious, rare genetic skin diseases, many of which are lifelong in nature. Our lead product candidate, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) is currently in late-stage clinical development for Microcystic Lymphatic Malformations (Microcystic LMs) and Basal Cell Carcinomas (BCCs) in Gorlin Syndrome (GS). QTORIN rapamycin has received FDA Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation for Microcystic LMs, and Fast Track Designation for the prevention of BCCs in GS.
Contact Information
Palvella Therapeutics
Investors:
Founder and CEO, Palvella Therapeutics
wes.kaupinen@palvellatx.com
Media:
Marcy Nanus
Managing Partner,
mnanus@trilonadvisors.com
Source: Palvella Therapeutics
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