Palvella Therapeutics Announces QTORIN™ Rapamycin 3.9% Anhydrous Gel for the Treatment of Microcystic Lymphatic Malformations Featured in Oral Presentation by Amy Paller, M.S., M.D., Chair of Dermatology at Northwestern University’s Feinberg School of Medicine, at the 15th World Congress of Pediatric Dermatology
Presentation highlighted the recent expansion of the Phase 3 SELVA trial to include children 3 to 5 years old
Presentation reviewed clinically and statistically significant Phase 2 results and the design of the ongoing Phase 3 SELVA trial
Top-line results from SELVA remain on track for the first quarter of 2026
QTORIN™ 3.9% rapamycin anhydrous gel has the potential to be the first approved therapy and standard of care for microcystic lymphatic malformations in the U.S.
WAYNE, Pa., April 11, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella or “the Company”), 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 U.S. Food and Drug Administration (FDA)-approved therapies, today announced QTORIN™ rapamycin 3.9% anhydrous gel (QTORIN™ rapamycin) for the treatment of microcystic lymphatic malformations (microcystic LMs) was featured by Dr. Amy Paller in an oral presentation at the 15th World Congress of Pediatric Dermatology in Buenos Aires, Argentina. Dr. Amy Paller is the Walter J. Hamlin Professor and Chair of Dermatology, Professor of Pediatrics, and Principal Investigator of the NIH-funded Skin Biology and Diseases Resource-based Center at Northwestern University’s Feinberg School of Medicine and has served as President of the Society for Investigative Dermatology (SID), the Society for Pediatric Dermatology (SPD), the International Eczema Council (IEC), the Pediatric Dermatology Research Alliance (PeDRA), and the Women’s Dermatological Society (WDS).
"There is an urgent need for a safe and effective targeted topical therapy for mosaic genetic skin disorders including microcystic lymphatic malformation," said Amy Paller, M.S., M.D., Chair of Dermatology, Northwestern University's Feinberg School of Medicine. "Many patients have considerable complications associated with this disease, and I am looking forward to the Phase 3 results early next year."
The oral presentation titled, "SELVA: A Phase 3 study with a fit-for-purpose primary endpoint evaluating QTORIN™ 3.9% rapamycin anhydrous gel in the treatment of microcystic lymphatic malformations in patients 3 years of age and older," highlighted:
Microcystic LMs are congenital mosaic lesions that gradually increase in size with risk of complications and are best managed aggressively during childhood
Microcystic LMs are proliferative with no spontaneous regression
QTORIN™ rapamycin, an investigational therapy designed to selectively inhibit the mammalian target of rapamycin (mTOR) in the skin, potentially reduces endothelial cell hyper-proliferation and vascular endothelial growth factor signaling, both of which are the result of overactive mTOR signaling in microcystic LMs
A multicenter, open-label, 12-week, Phase 2 study evaluating the safety and efficacy of QTORIN™ rapamycin for microcystic LMs demonstrated:
100% of participants were either "Very Much Improved” (41.7%) or “Much Improved” (58.3%) as rated by the Clinician Global Impression of Change (CGI-C), a 7-point change scale conducted by live clinician assessment
83% of participants were either “Very Much Improved” (25%) or “Much Improved” (58.3%) as rated by the Patient Global Impression of Change, a 7-point change scale reported by patients
QTORIN™ rapamycin was generally well-tolerated; all treatment related adverse events were moderate or mild and there were no discontinuations due to adverse events.
SELVA, a 24-week, Phase 3, single-arm, baseline-controlled clinical trial of QTORIN™ rapamycin for the treatment of microcystic LMs, mimics the Phase 2 study, with key study elements including the following:
Based on data from Phase 2 and clinician interviews, the primary endpoint is the fit-for-purpose Microcystic Lymphatic Malformations Investigator’s Global Assessment (mLM-IGA), a 7-point change scale conducted by live clinician assessment with similarities to the CGI-C
Patient population enriched to include patients with moderate to severe disease
Target sample size of 40 subjects
Treatment duration extended to 24-weeks
Enrollment criteria expanded to include patients 3 years and older
"Microcystic LMs are a serious, rare, and chronically debilitating genetic disease with a pediatric onset and lifelong course. Early intervention is essential to minimizing disease burden for this patient population who currently have no FDA-approved therapies. Palvella is pleased to include patients younger than 6 years old in the ongoing Phase 3 SELVA study," said Wes Kaupinen, Founder and Chief Executive Officer of Palvella.
SELVA is currently enrolling patients at 13 centers in the United States. Top-line data is anticipated in the first quarter of 2026. The U.S. FDA has granted Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation to QTORIN™ rapamycin for the treatment of microcystic LMs. Additionally, the SELVA study is supported by an Orphan Products Grant of up to $2.6 million from FDA’s Office of Orphan Products Development.
About Microcystic Lymphatic Malformations Microcystic LMs are a rare, chronically debilitating genetic disease caused by dysregulation of the phosphatidylinositol 3-kinase (PI3K)/mammalian target of rapamycin (mTOR) pathway. The disease is characterized by malformed lymphatic vessels that protrude through the skin and persistently leak lymph fluid (lymphorrhea) and bleed, often leading to recurrent serious infections and cellulitis that can cause hospitalization. The natural history of microcystic LMs is persistent and progressive without spontaneous resolution, with symptoms generally worsening during life, including increases in the number and size of malformed vessels that lead to complications and lifetime morbidity. There are currently no FDA-approved treatments for the estimated more than 30,000 diagnosed patients with microcystic LMs in the United States.
About Palvella Therapeutics Founded and led by rare disease drug development veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) 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. Palvella is developing a broad pipeline of product candidates based on its patented QTORIN™ platform, with an initial focus on serious, rare genetic skin diseases, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), is currently being evaluated in the Phase 3 SELVA clinical trial in microcystic lymphatic malformations and the Phase 2 TOIVA clinical trial in cutaneous venous malformations. For more information, please visit www.palvellatx.com or follow Palvella on LinkedIn or X (formerly known as Twitter).
QTORIN™ rapamycin is for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency for any indication.
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