PVLA

EX-99.1

2 pvla-ex99_1.htm

EX-99.1

EX-99.1

Palvella Therapeutics Reports Second Quarter 2026 Financial Results and Provides Corporate Update

First module of the rolling NDA for QTORIN™ rapamycin for microcystic lymphatic malformations submitted to FDA, with completion of the NDA submission on track for the second half of 2026

Preparing for a planned standalone U.S. commercial launch of QTORIN™ rapamycin for microcystic lymphatic malformations in the first half of 2027, if approved

Initiation of Phase 3 trial of QTORIN™ rapamycin for the treatment of cutaneous venous malformations planned for the fourth quarter of 2026

Initiation of Phase 2 trial of QTORIN™ pitavastatin for the treatment of disseminated superficial actinic porokeratosis planned for the second half of 2026

Topline results from the Phase 2 LOTU trial of QTORIN™ rapamycin for clinically significant angiokeratomas expected in the second half of 2027

Cash, cash equivalents and short-term investments of $250.6 million as of June 30, 2026

Company to host conference call at 8:30 a.m. ET today

WAYNE, Pa., August 4, 2026 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc. (Palvella or “the Company”) (Nasdaq: PVLA), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies for serious, rare skin diseases and vascular malformations for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today reported financial results for the second quarter ending June 30, 2026 and provided a corporate update.

“We made significant progress during the second quarter, completing our pre-NDA meeting with FDA and initiating the rolling NDA submission for QTORIN™ rapamycin in microcystic lymphatic malformations,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. “We are working closely with FDA under the program’s Breakthrough Therapy and Fast Track designations to expedite development and review, with the objective of potentially introducing the first approved therapy to pediatric and adult patients living with this serious, lifelong disease. Completion of the NDA submission remains on track for the second half of 2026, and we are preparing for a planned standalone commercial launch in the first half of 2027, if approved. We have recruited commercial and medical affairs leaders with deep experience in rare disease and dermatology who are now in the field executing key pre-launch activities, while we continue to advance our other rare disease programs and pursue additional opportunities across the QTORIN™ platform.”

Recent Research and Development Highlights

QTORIN™ rapamycin for microcystic lymphatic malformations (microcystic LMs)

• James Treat, M.D., of Children’s Hospital of Philadelphia presented additional Phase 3 SELVA data during a late-breaking session at the International Society for the Study of Vascular Anomalies World Congress (ISSVA), including a statistically significant improvement in the 6–11-year-old cohort and other supportive findings showing improvements in clinical signs and patient-reported outcomes with QTORIN™ rapamycin.

• Completed an in-person pre-New Drug Application (NDA) meeting with FDA which addressed nonclinical, clinical pharmacology, clinical information, and the planned evidence package for the NDA.

• Following the pre-NDA meeting, FDA granted Palvella’s request for Rolling Review of the QTORIN™ rapamycin NDA for microcystic LMs, allowing the Agency to begin reviewing completed portions of the application before submission of the full NDA.

• Submitted the first module of its rolling NDA to the FDA seeking approval of QTORIN™ rapamycin for the treatment of microcystic LMs.

• The Company remains on track to complete the NDA submission in the second half of 2026.

QTORIN™ rapamycin for cutaneous venous malformations (cutaneous VMs)

• In May 2026, at the 83rd Annual Meeting of the Society for Investigative Dermatology (“SID”), Palvella presented new data from our Phase 2 TOIVA trial of QTORIN™ rapamycin for the treatment of cutaneous VMs highlighting that 100% of patients with bleeding at baseline demonstrated improvement on the Cutaneous Venous Malformations Investigator Global Assessment Bleeding scale at Week 12.

• Dr. Treat presented additional Phase 2 TOIVA data, including 24-week results, at ISSVA demonstrating statistically significant improvements in both cVM-MCSS Height/Engorgement and cVM-MCSS Appearance at all measured time points, with increasing clinical response observed with longer duration of QTORIN™ rapamycin therapy.

• Phase 3 trial initiation remains on track for the fourth quarter of 2026 following completion of the planned End-of-Phase 2 meeting.

QTORIN™ rapamycin for clinically significant angiokeratomas

• Dosed the first patients in LOTU, a multicenter Phase 2 trial evaluating Fast Track-designated QTORIN™ rapamycin for clinically significant angiokeratomas, a rare, chronic and debilitating isolated lymphatic malfor

EX-99.1

2 pvla-ex99_1.htm

EX-99.1

EX-99.1

Palvella Therapeutics Reports First Quarter 2026 Financial Results and Provides Corporate Update

FDA Pre-New Drug Application (NDA) meeting granted for QTORIN™ rapamycin for the treatment of microcystic lymphatic malformations, with meeting expected in second quarter of 2026; NDA submission on track for second half of 2026

Accelerating U.S. launch readiness for QTORIN™ rapamycin for microcystic lymphatic malformations; BEYONDmLM.com disease awareness campaign launched to educate, engage, and empower patients, caregivers, and healthcare professionals

Initiation of Phase 3 trial of QTORIN™ rapamycin for the treatment of cutaneous venous malformations planned for second half of 2026

Initiation of Phase 2 trial of QTORIN™ pitavastatin for the treatment of disseminated superficial actinic porokeratosis planned for second half of 2026

Phase 2 LOTU trial of QTORIN™ rapamycin for clinically significant angiokeratomas initiated with topline results expected in second half of 2027

Completed upsized, oversubscribed equity financing of $230.0 million in February 2026; cash, cash equivalents and short-term investments of $261.9 million as of March 31, 2026

Company to host conference call at 8:30 a.m. ET today

WAYNE, PA., May 7, 2026 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc. (Palvella or “the Company”) (Nasdaq: PVLA), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare skin diseases and vascular malformations for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today reported financial results for the first quarter ending March 31, 2026 and provided a corporate update.

“Following the positive Phase 3 SELVA topline results, we believe Palvella is on a clear path toward near-term NDA submission for QTORIN™ rapamycin in microcystic lymphatic malformations, with the potential for approval and U.S. commercial launch in the first half of 2027,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. “Our $230.0 million financing with participation from high-quality new and existing investors meaningfully strengthens our balance sheet, which supports our plan for U.S. commercialization, and enables us to attract key talent as we prepare for a potential first-in-disease launch. At the same time, we continue to rapidly advance our pipeline beyond microcystic LMs, with the goal of expanding our QTORIN™-derived programs across six serious, rare skin diseases and vascular malformations by year-end.”

Recent Research and Development Highlights

QTORIN™ rapamycin for microcystic lymphatic malformations (microcystic LMs)

• Reported positive topline data from the Phase 3 SELVA study demonstrating statistically significant results across the primary and all pre-specified secondary endpoints.

• In collaboration with leading nonprofit and advocacy organizations, launched the “BEYOND mLM” disease awareness campaign and BEYONDmLM.com to educate and engage patients, caregivers, and healthcare professionals on microcystic lymphatic malformations.

• Announced publication in the Journal of Vascular Anomalies highlighting the distinct biology and significant unmet need in microcystic lymphatic malformations, supporting early intervention and reinforcing the scientific rationale for QTORIN™ rapamycin as a targeted therapeutic approach.

• Strengthened global intellectual property for QTORIN™ rapamycin with issuance of a European patent covering anhydrous topical compositions and methods of use, providing protection through 2038.

• Phase 3 SELVA study results to be presented at the International Society for the Study of Vascular Anomalies World Congress 2026 on May 20, 2026, marking the first presentation of these data at a major medical meeting.

• Pre-New Drug Application (NDA) meeting with the FDA granted; meeting expected in the second quarter of 2026.

• NDA submission remains on track for the second half of 2026.

QTORIN™ rapamycin for cutaneous venous malformations (cutaneous VMs)

• Phase 2 TOIVA study results to be presented at the International Society for the Study of Vascular Anomalies World Congress 2026 on May 20, 2026; additional data presentation planned at the 83rd Annual Meeting of the Society for Investigative Dermatology on May 12, 2026.

• Submitted application for FDA Breakthrough Therapy Designation in the second quarter of 2026.

• Phase 3 study initiation anticipated in the second half of 2026.

QTORIN™ rapamycin for clinically significant angiokeratomas

• Dosed first patients in the Phase 2 LOTU trial, a single-arm, baseline-controlled clinical trial of QTORIN™ rapamycin administered topically once daily for the treatment of clinically significant angiokeratomas.

• Topline results from LOTU are expected in the second half of 2027.

QTORIN™ pitavastatin for disseminated superficial actinic porokeratosis (DSAP)

• Presented q

EX-99.1

2 pvla-ex99_1.htm

EX-99.1

EX-99.1

Palvella Therapeutics Reports Full Year 2025 Financial Results and Provides Corporate Update

New Drug Application (NDA) for QTORIN™ rapamycin for the treatment of microcystic lymphatic malformations (microcystic LMs) on track for planned submission in second half of 2026

Accelerating U.S. launch readiness for QTORIN™ rapamycin for microcystic LMs; potential to become the first FDA-approved therapy and first-line, standard-of-care treatment for serious, lifelong disease affecting an estimated more than 30,000 diagnosed patients in the U.S.

Initiation of Phase 3 trial of QTORIN™ rapamycin for the treatment of cutaneous venous malformations planned for second half of 2026

Initiation of Phase 2 trial of QTORIN™ rapamycin for the treatment of clinically significant angiokeratomas planned for second quarter of 2026

Initiation of Phase 2 trial of QTORIN™ pitavastatin for the treatment of disseminated superficial actinic porokeratosis planned for second half of 2026

Pro forma cash of approximately $274 million as of December 31, 2025 reflects net proceeds from a February 2026 equity financing; cash and cash equivalents of $58.0 million as of December 31, 2025

Company to host conference call at 8:30 a.m. ET today

WAYNE, PA., March 31, 2026 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc. (Palvella or “the Company”) (Nasdaq: PVLA), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare skin diseases and vascular malformations for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today reported financial results for the full year ending December 31, 2025 and provided a corporate update.

“2025 was a landmark year for Palvella, and we carried that momentum into 2026 with positive Phase 3 SELVA results in microcystic lymphatic malformations, marking a major milestone for the company and putting us on a path toward our first potential FDA approval in the first half of 2027,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. “Following SELVA results, we significantly strengthened our balance sheet through an oversubscribed $230.0 million financing, enabling us to accelerate U.S. launch readiness and continue advancing novel topical product candidates from our QTORIN™ platform. Supported by Breakthrough Therapy, Fast Track, and Orphan designations, we are now focused on advancing QTORIN™ rapamycin toward an NDA submission while preparing for a planned standalone U.S. commercial launch. We believe this momentum positions us to advance our vision of building the leading rare disease biopharmaceutical company addressing serious, rare skin diseases and vascular malformations with no FDA-approved therapies.”

Recent Research and Development Highlights

QTORIN™ rapamycin for microcystic LMs

• In February 2026, reported positive topline results from the Phase 3 SELVA study:

o Primary endpoint met with statistically significant improvement (mean change of +2.13; p<0.001) on the Microcystic Lymphatic Malformation Investigator Global Assessment (mLM-IGA)

o Achieved statistical significance on pre-specified key secondary endpoint (p<0.001) and all four secondary efficacy endpoints (all p<0.001)

o 95% of trial participants aged 6 years and older who completed the efficacy evaluation period improved on the mLM-IGA at Week 24

o 86% of trial participants aged 6 years and older who completed the efficacy evaluation period were rated as “Much Improved” (+2) or “Very Much Improved” (+3) on the mLM-IGA at Week 24

o QTORIN™ rapamycin was well-tolerated, with no drug-related serious adverse events reported and systemic rapamycin levels below 2ng/mL at all timepoints for all participants

o 98% of participants who completed the efficacy evaluation period elected to continue to receive QTORIN™ rapamycin in the ongoing treatment extension period

• In Q4 2025, FDA awarded year two non-dilutive proceeds from the FDA Orphan Products Grant program to support the SELVA trial.

• In March 2026, submitted Pre-NDA meeting request to FDA, with meeting anticipated in the second quarter of 2026.

• NDA planning is underway, with submission on track for the second half of 2026.

QTORIN™ rapamycin for cutaneous venous malformations (cutaneous VMs)

• In December 2025, announced positive topline efficacy results from the Phase 2 TOIVA study, achieving statistical significance on multiple pre-specified clinician-reported and patient-reported efficacy endpoints, including dynamic change endpoints and static severity endpoints.

o 73% of trial participants (11/15 participants) improved on the Overall Cutaneous Venous Malformations Investigator Global Assessment (Overall cVM-IGA) at Week 12; 67% of trial participants (10/15 participants) rated as “Much Improved” (+2) or “Very Much Improved” (+3) on the Overall cVM-IGA at Week 12.

• Recently completed a Preliminary Brea