Annabel Samimy

Catherine Novack

Danielle Brill

Dev Prasad

Ritu Baral

Ryan Deschner

Sam Slutsky

Whitney Ijem

- **Palvella Therapeutics** announced the **expansion of its QTORIN™ Rapamycin pipeline** to include **clinically significant angiokeratomas** as a third planned indication, targeting an estimated **>50,000 U.S. patients** with no FDA-approved therapies.  **[1583648_2155827:0]** **[1583648_2155827:3]** **[1583648_2155827:4]** 
- The company plans to initiate a **Phase 2 trial for angiokeratomas in 2H 2026**, following an FDA meeting in 1H 2026 to discuss study design and expedited programs.  **[1583648_2155827:3]** **[1583648_2155827:7]** 
- Key upcoming milestones include **Phase 2 topline data for Cutaneous VMs in December 2025** and **Phase 3 topline data for Microcystic LMs in Q1 2026**.  **[1583648_2155827:3]** 
- A **new QTORIN™ program** for another rare disease is slated for Q4 2025, aiming for Phase 2 proof-of-concept data within 2.5 years and under $10 million.  **[1583648_2155827:3]**

Palvella Therapeutics Expands QTORIN™ Rapamycin Pipeline and Provides Clinical Trial Updates

- Palvella Therapeutics announced the **expansion of its QTORIN™ rapamycin 3.9% anhydrous gel pipeline** to include clinically significant angiokeratomas, a rare, chronically debilitating lymphatic disease that currently has **no FDA-approved therapies**.  **[1583648_2155749_0]** **[1583648_2155749_1]** 
- This new indication targets an estimated **over 50,000 diagnosed patients** in the United States and is considered a **commercially compelling opportunity** with strong clinician demand for a novel topical therapy.  **[1583648_2155749_1]** **[1583648_2155749_5]** **[1583648_2155749_6]** 
- The company plans to initiate a **Phase 2 study** for clinically significant angiokeratomas in the **second half of 2026**, following an FDA meeting in the first half of 2026.  **[1583648_2155749_2]** **[1583648_2155749_6]** 
- Palvella also anticipates **top-line readouts** for its Phase 2 TOIVA study in **mid-December** (this year) and its Phase 3 SELVA study in **Q1 2026**.  **[1583648_2155749_1]**

Palvella Therapeutics Expands QTORIN™ Rapamycin Pipeline for Angiokeratomas

- Palvella Therapeutics, Inc. announced on **September 24, 2025**, the **expansion of its QTORIN™ 3.9% rapamycin anhydrous gel development program into Clinically Significant Angiokeratomas**.  **[1583648_0001104659-25-092681_tm2526823d1_ex99-1.htm:0]** 
- Clinically significant angiokeratomas are a **rare, chronically debilitating lymphatic disease** for which there are **no FDA-approved therapies**, affecting an estimated **more than 50,000 diagnosed U.S. patients**.  **[1583648_0001104659-25-092681_tm2526823d1_ex99-1.htm:0]** 
- The company plans to **initiate a Phase 2 trial** for this indication in the **second half of 2026**, following discussions with the FDA in the first half of 2026.  **[1583648_0001104659-25-092681_tm2526823d1_ex99-1.htm:0]** 
- QTORIN™ rapamycin is also currently being evaluated in the Phase 3 SELVA study, with top-line results anticipated in **Q1 2026**, and the Phase 2 TOIVA study, with top-line results expected in **mid-December 2025**.  **[1583648_0001104659-25-092681_tm2526823d1_ex99-1.htm:0]** **[1583648_0001104659-25-092681_tm2526823d1_ex99-1.htm:1]**

Palvella Therapeutics Expands QTORIN™ Rapamycin Development into Clinically Significant Angiokeratomas

- Palvella Therapeutics, Inc. (PVLA) announced anticipated top-line data for its QTORIN 3.9% rapamycin anhydrous gel, including **Phase 3 data in microcystic lymphatic malformations (mLMs) in Q1 2026** and **Phase 2 data in cutaneous venous malformations (cVMs) in December 2025**.  **[1583648_0001104659-25-086685_tm2525080d1_ex99-1.htm:5]** 
- The company expects to announce a **third clinical indication for QTORIN rapamycin in September 2025** and a **new QTORIN program in Q4 2025**.  **[1583648_0001104659-25-086685_tm2525080d1_ex99-1.htm:4]** **[1583648_0001104659-25-086685_tm2525080d1_ex99-1.htm:5]** 
- PVLA reported a cash position of **$70.4 million as of June 30, 2025**, with a projected cash balance of **~$55 million at year-end**, providing a runway into **2H 2027**.   
- The company has made key leadership hires, including **Ashley Kline as Chief Commercial Officer in May 2025** and **David Osborne, Ph.D. as Chief Innovation Officer in September 2025**.  **[1583648_0001104659-25-086685_tm2525080d1_ex99-1.htm:3]**

PALVELLA THERAPEUTICS (PVLA)