Palvella Therapeutics, Inc. Expands QTORIN™ 3.9% Rapamycin Program

• Palvella Therapeutics, Inc. (NASDAQ:PVLA) is advancing its QTORIN™ 3.9% rapamycin anhydrous gel program to include a Phase 2 trial for angiokeratomas, aiming to fill a significant unmet medical need.
• The company plans to meet with the FDA in early 2026 to discuss the Phase 2 study design, targeting the first FDA-approved therapy for angiokeratomas.
• Despite a negative P/E ratio of -26.45 and a negative enterprise value to operating cash flow ratio of -42.71, Palvella maintains a strong liquidity position with a current ratio of 7.67.

Palvella Therapeutics, Inc. (Nasdaq: PVLA) is a clinical-stage biopharmaceutical company focused on developing treatments for rare skin diseases. The company is expanding its QTORIN™ 3.9% rapamycin anhydrous gel program to include clinically significant angiokeratomas, a rare lymphatic disease affecting over 50,000 patients in the U.S. With no FDA-approved therapies available, Palvella aims to fill this gap by initiating a Phase 2 trial in the latter half of 2026.

Wes Kaupinen, the CEO of Palvella, is optimistic about the potential of QTORIN rapamycin to meet this unmet medical need. The company is committed to introducing the first FDA-approved therapy for angiokeratomas, aligning with its strategy to target serious, rare skin conditions. The current treatment options for angiokeratomas are limited and often involve painful procedures with risks of scarring and recurrence.

Palvella plans to meet with the FDA in early 2026 to discuss the Phase 2 study design, which will involve 10-20 patients. The company is also evaluating QTORIN rapamycin in other studies, with results expected in early 2026 and mid-December 2025. This expansion was discussed in a webcast conference call on September 24, 2025, as Palvella continues to leverage its patented QTORIN platform for novel therapies.

On the financial front, H.C. Wainwright upgraded PVLA to "Buy" on September 25, 2025, with a raised price target from $75 to $95. At the time, the stock was priced at $58.87. Annabel Samimy from Stifel Nicolaus also set a price target of $80, indicating a potential price increase of about 33.62% from the then-current price of $59.87.

Despite a negative price-to-earnings (P/E) ratio of -26.45 and challenges in generating cash flow, as indicated by a negative enterprise value to operating cash flow ratio of -42.71, Palvella maintains a strong liquidity position. The company's current ratio of 7.67 suggests it has sufficient current assets to cover its liabilities, providing a cushion as it advances its clinical programs.