Palvella Therapeutics — Third Quarter 2026 Earnings Summary

Financial Performance:

• Cash, cash equivalents, and short-term investments totaled $236.7 million as of September 30, 2026.
• Research and development expenses were $10.8 million for the three months ended September 30, 2026, compared to $6.5 million in the same period in 2025.
• General and administrative expenses were $11.9 million for the three months ended September 30, 2026, compared to $3.6 million in the same period in 2025.
• Net loss was $24.6 million, or $1.70 per basic and diluted share, for the three months ended September 30, 2026, compared to a net loss of $11.3 million, or $1.03 per share, in the same period in 2025.
• Weighted average shares outstanding were 14,531,595 for the three months ended September 30, 2026, and 13,996,326 for the nine months ended September 30, 2026.
• Shares outstanding were 15,844,715 as of November 2, 2026, including 14,618,457 shares of common stock and 1,226,258 common share equivalents from outstanding pre-funded warrants.

Guidance and Future Outlook:

• The FDA has set a Priority Review PDUFA target action date of February 28, 2027, for the NDA for QTORIN® rapamycin in microcystic lymphatic malformations.
• Palvella is preparing for a standalone U.S. commercial launch of QTORIN® rapamycin in the first half of 2027, contingent on FDA approval.
• Phase 3 trial initiation for QTORIN® rapamycin in cutaneous venous malformations is on track for the fourth quarter of 2026.
• Topline results from the Phase 2 LOTU trial for QTORIN® rapamycin in clinically significant angiokeratomas are expected in the second half of 2027.
• Phase 2 trial initiation for QTORIN® pitavastatin in disseminated superficial actinic porokeratosis is expected in the fourth quarter of 2026.
• Palvella plans to announce a fourth target clinical indication for QTORIN® rapamycin in the fourth quarter of 2026.
• Palvella plans to announce a third product candidate from the QTORIN® platform in the fourth quarter of 2026.

Business Segments and Product Lines:

• QTORIN® rapamycin (Microcystic Lymphatic Malformations): The NDA was accepted by the FDA; the company received a third year of FDA Office of Orphan Products Development grant funding for the Phase 3 SELVA trial and open-label extension study.
• QTORIN® rapamycin (Cutaneous Venous Malformations): A Phase 2 TOIVA poster presented at the 2026 PeDRA Annual Conference highlighted improvements in disease severity and reductions in lesion bleeding among pediatric participants.
• QTORIN® rapamycin (Clinically Significant Angiokeratomas): The LOTU Phase 2 trial is currently enrolling patients for this Fast Track-designated indication.
• QTORIN® pitavastatin (Disseminated Superficial Actinic Porokeratosis): U.S. claims data analysis identified an estimated 154,986 diagnosed patients in the U.S.
• Pipeline Expansion: The company is advancing preparations for a standalone U.S. commercial launch with established key commercial, medical, and patient services capabilities.

Market and Competitive Landscape:

• Microcystic lymphatic malformations is a serious, rare, lifelong disease affecting an estimated more than 30,000 diagnosed children and adults in the U.S., for which there are no FDA-approved therapies.
• Clinically significant angiokeratomas is a rare, chronic, and debilitating isolated lymphatic malformation affecting an estimated more than 50,000 diagnosed patients in the U.S., with no FDA-approved therapies.
• The company positions QTORIN® rapamycin as a potential first-line treatment and standard of care for microcystic lymphatic malformations.
• QTORIN® rapamycin demonstrated mean systemic exposure at least 40-fold lower than reported exposure with oral sirolimus.

Risks and Challenges:

• The company faces risks related to the ability to raise additional capital, advance product candidates through development, and obtain regulatory approval.
• Potential delays in the FDA's review of the accepted NDA or failure to obtain approval could impact commercialization plans.
• The company has limited experience in commercial manufacturing and designing clinical trials.
• Substantial competition exists in discovering, developing, or commercializing products for rare skin diseases and vascular anomalies.
• Risks include reliance on third parties, contract manufacturers, and contract research organizations, as well as the ability to protect intellectual property.

Management Commentary and Tone:

• CEO Wes Kaupinen stated the company is "closer than ever to bringing our first therapy to patients" following the FDA's Priority Review grant.
• Management emphasized a clear focus on successfully obtaining FDA approval, bringing the first therapy to patients, advancing additional QTORIN® programs, and building a leading company in the rare skin disease space.
• The company is actively recruiting additional top talent to shape an exceptional launch for the microcystic LM community.
• Chief Scientific Officer Jeff Martini was selected to provide public comment at an FDA public workshop on drug repurposing.

Other Key Points:

• James R. Treat, M.D., a nationally recognized pediatric dermatologist with nearly 20 years of experience, was appointed Chief Medical Officer effective December 21, 2026.
• The company presented data at Fall Clinical Dermatology 2026 and the 2026 Pediatric Dermatology Research Alliance (PeDRA) Annual Conference regarding clinical benefits and treatment satisfaction.
• Palvella is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies for serious, rare skin diseases and vascular anomalies for which there are no FDA-approved therapies.
• The company hosts a conference call and live webcast to discuss results and provide a corporate update.