Palvella Therapeutics (PVLA) Study result summary

Study Overview and Objectives

• Phase 3 SELVA trial evaluated QTORIN 3.9% rapamycin gel for microcystic lymphatic malformations, a rare, lifelong, and debilitating disease with no FDA-approved therapies, affecting over 30,000 individuals in the U.S.

• The study enrolled 51 participants aged three and older, with 49 in the intent-to-treat population and nearly 90% retention at 24 weeks; 98% of completers entered the extension period.

• The trial was single-arm, baseline-controlled, supported by an FDA Orphan Products Grant, and received Breakthrough Therapy, Fast Track, and Orphan Drug designations.

• Most participants had failed prior therapies, highlighting high unmet need; current interventions are limited to surgery and laser, which are painful and often require repeated treatments.

• QTORIN rapamycin is designed for targeted, chronic topical delivery with minimal systemic exposure and deep dermal penetration.

Efficacy Results

• QTORIN rapamycin achieved a +2.13 mean improvement on the mLM-IGA primary endpoint at week 24 (p<0.001), exceeding predefined efficacy goals.

• 95% of participants improved on the primary endpoint; 86% were rated as much or very much improved at week 24.

• All primary, key secondary, and four additional secondary endpoints, including blinded clinician and patient-reported outcomes, showed highly statistically significant improvements (all p<0.001).

• Key secondary endpoint (blinded mLM-MCSS) showed a mean improvement of -3.36 (p<0.001).

• Efficacy was consistent across age groups and time points, with high concordance between endpoints.

Safety and Tolerability

• QTORIN rapamycin was well tolerated with a favorable safety profile across adults and children; no drug-related serious adverse events reported.

• 70% experienced treatment-emergent adverse events, mostly mild or moderate and local (application site acne, discoloration, pruritus); 34% had treatment-related events.

• No severe or serious treatment-related adverse events reported.

• Systemic rapamycin levels remained below 2 ng/mL for all participants at all timepoints.

• 98% of week 24 completers rolled over to the extension period, supporting chronic administration.