New Qtorin Rapamycin Data Featured at World Congress of Pediatric Dermatology

Image Credit: © dermnetnz.org

Today, Palvella Therapeutics announced that Qtorin 3.9% rapamycin anhydrous gel (Qtorin rapamycin) was featured in Amy Paller, MS, MD’s oral presentation at the 15th World Congress of Pediatric Dermatology in Buenos Aires, Argentina.¹ The presentation was 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."

"There is an urgent need for a safe and effective targeted topical therapy for mosaic genetic skin disorders, including microcystic lymphatic malformation," said Paller, who is the Chair of Dermatology at 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."¹ Paller has also served as the 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).

In the presentation, Paller highlighted a multicenter, open-label, 12-week, phase 2 trial in which 100% of patients were either "very much improved” (41.7%) or “much improved” (58.3%), according to the Clinician Global Impression of Change (CGI-C). Additionally, 83% of participants were either “very much improved” (25%) or “much improved” (58.3%) as rated by the Patient Global Impression of Change. The therapy was well tolerated, with only mild or moderate adverse events and no discontinuations due to these.

Paller also discussed the upcoming SELVA study, a phase 3 single-arm, baseline-controlled clinical trial that mimics the aforementioned phase 2 study. The primary endpoint will be the 7-point fit-for-purpose Microcystic Lymphatic Malformations Investigator’s Global Assessment (mLM-IGA). Approximately 40 patients 3 years and older with moderate to severe disease will be included. The treatment duration will be extended to 24 weeks.

"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 CEO of Palvella.¹

Cutaneous venous malformations occur due to genetic mutations that overactivate the PI3K/mTOR signaling pathway, leading to dysfunctional vein formation within the skin. Affecting an estimated 75,000 diagnosed individuals in the US, this condition can cause severe complications, including bleeding, ulceration, thrombosis, and chronic pain.

Qtorin rapamycin is a topically administered 3.9% rapamycin anhydrous gel designed to target the mTOR pathway. By delivering rapamycin directly to the affected skin, Qtorin aims to achieve localized therapeutic effects while minimizing systemic exposure and its associated risks. This novel approach has the potential to provide clinical benefits previously observed only through off-label systemic mTOR inhibitors.

Patients are currently being enrolled for SELVA at 13 centers in the United States with top-line data anticipated for the first quarter of 2026. The trial is supported by an Orphan Products Grant of up to $2.6 million from the US Food and Drug Administration’s (FDA) Office of Orphan Products Development. The FDA has also granted Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation for Qtorin rapamycin.² There are currently no FDA-approved therapies for the rare, genetic disease.

References

1. 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. GlobeNewswire. April 11, 2025. Accessed April 11, 2025. https://www.globenewswire.com/news-release/2025/04/11/3060025/0/en/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-Chai.html

2. Palvella Therapeutics announces U.S. FDA Breakthrough therapy designation granted to QTORINTM 3.9% rapamycin anhydrous gel (QTORINTM rapamycin) for the treatment of microcystic lymphatic malformations. GlobeNewswire. November 16, 2023. Accessed April 11, 2025. https://www.globenewswire.com/news-release/2023/11/16/2781752/0/en/Palvella-Therapeutics-Announces-U-S-FDA-Breakthrough-Therapy-Designation-Granted-to-QTORIN-3-9-Rapamycin-Anhydrous-Gel-QTORIN-rapamycin-for-the-Treatment-of-Microcystic-Lymphatic-M.html