PALVELLA THERAPEUTICS (PVLA) Company Report

Palvella Therapeutics announced positive Phase 2 TOIVA topline data for QTORIN™ rapamycin in Cutaneous Venous Malformations (cVMs) on December 15, 2025.
The study achieved statistical significance on multiple clinician-reported and patient-reported efficacy endpoints.
73% of participants (11/15) demonstrated at least a 1-point improvement on the Overall cVM-IGA at Week 12, with a mean effect size of +1.5 (p<0.001).
QTORIN™ rapamycin was generally well-tolerated, with all treatment-related adverse events being moderate or mild.
Palvella plans discussions with the FDA in early 2026 for potential Breakthrough Therapy Designation and a Phase 3 pivotal study, positioning QTORIN™ rapamycin as a potential first FDA-approved therapy for cVMs.

Dec 15, 2025, 1:30 PM

Presentation

Palvella Announces Positive Phase 2 Results for Qtorin Rapamycin in Cutaneous Venous Malformations

PVLA

Product Launch

New Projects/Investments

Palvella (PVLA) announced positive top-line results from its Phase 2 TOIVA study evaluating Qtorin rapamycin for the treatment of cutaneous venous malformations (CVM), a serious rare genetic disease for which there are currently no FDA-approved therapies.
The study demonstrated highly statistically significant improvements on multiple clinician-reported and patient-reported efficacy endpoints.
At week 12, 73% of patients (11 out of 15) showed a one-point or greater improvement on the overall CVM IGA, and 67% (10 out of 15) were rated as either "much improved" or "very much improved".
Qtorin rapamycin was generally well tolerated, consistent with previous clinical trials, and systemic absorption was very low.
Palvella plans to engage the FDA regarding potential breakthrough therapy designation and to align on the design of a Phase 3 pivotal study, with the goal of Qtorin rapamycin becoming the first FDA-approved therapy for CVM.

Dec 15, 2025, 1:30 PM

Transcript

Palvella Therapeutics Announces Positive Phase 2 Study Results

PVLA

New Projects/Investments

Palvella Therapeutics announced positive top-line results from its phase 2 study evaluating Qtorin rapamycin for the treatment of cutaneous venous malformations (CVM).
The study demonstrated highly statistically significant results, with 73% of patients (11 out of 15) showing a one-point or greater improvement on the overall CVM Investigator's Global Assessment (IGA) at week 12, and 67% of patients (10 out of 15) rated as "much improved" or "very much improved". The mean effect size at week 12 was 1.5 with a p-value of less than 0.001.
Qtorin rapamycin was generally well tolerated, with the most common treatment-emergent adverse event being erythema in 25% of patients, and systemic absorption was very low.
Palvella plans near-term discussions with the FDA to explore breakthrough therapy designation, align on the design of a phase 3 pivotal study, and engage on the newly announced plausible mechanism pathway. These results support the potential for Qtorin rapamycin to become the first FDA-approved therapy for the estimated more than 75,000 individuals in the U.S. living with CVM.

Dec 15, 2025, 1:30 PM

Transcript

Palvella Therapeutics announces positive Phase 2 TOIVA clinical trial results for QTORIN™ rapamycin

PVLA

Palvella Therapeutics announced positive topline results from its Phase 2 TOIVA clinical trial of QTORIN™ 3.9% rapamycin anhydrous gel for the treatment of cutaneous venous malformations (cutaneous VMs) on December 15, 2025.
The study showed that 73% of trial participants (11/15) demonstrated at least a 1-point improvement on the Overall Cutaneous Venous Malformations Investigator Global Assessment (Overall cVM-IGA) at Week 12, with 67% (10/15) rated as "Much Improved" (+2) or "Very Much Improved" (+3).
QTORIN™ rapamycin achieved statistical significance on multiple pre-specified clinician-reported and patient-reported efficacy endpoints and was generally well-tolerated, with no drug-related serious adverse events reported.
Based on these results, Palvella plans to pursue near-term discussions with the FDA regarding the potential for Breakthrough Therapy Designation and a Phase 3 pivotal study.

Dec 15, 2025, 11:05 AM

8-K

Palvella Therapeutics Provides Clinical Pipeline Update and Financial Runway

PVLA

New Projects/Investments

Guidance Update

Palvella Therapeutics (PVLA) is focused on developing first FDA-approved therapies for serious rare skin diseases, leveraging its Qutorin platform.
The pivotal Phase 3 study for microcystic lymphatic malformations (MLM), using Qutorin Rapamycin, is fully enrolled with 51 patients and is expected to read out in Q1 2026, with potential regulatory approval in H1 2027.
Data from the Phase 2 study for cutaneous venous malformations (CVM), also with Qutorin Rapamycin, is anticipated in mid-December. The company is looking for approximately 30% clinical improvement to advance to Phase 3.
Palvella has expanded its pipeline to four diseases across two product candidates, including new programs for angiocheratomas and disseminated superficial actinic porokeratosis (DSAP).
The company has a cash runway into H2 2027, supported by a $80 million PIPE raised in December of last year, which funds upcoming clinical milestones.

Nov 12, 2025, 9:00 PM

Transcript

Palvella Therapeutics Reports Q3 2025 Financials and Pipeline Progress

PVLA

Earnings

New Projects/Investments

Guidance Update

Palvella Therapeutics ended Q3 2025 with $63.6 million in cash and equivalents as of September 30, 2025, providing a cash runway into the second half of 2027.
The company anticipates top-line data from its Phase 2 TOIVA study for cutaneous venous malformations next month (December 2025) and a top-line readout from its Phase 3 SELVA study for microcystic lymphatic malformations next quarter (Q4 2025/Q1 2026).
Palvella plans to submit an NDA for Qutoran rapamycin in microcystic lymphatic malformations in the second half of 2026, with a potential standalone U.S. launch anticipated in 2027.
New clinical programs, Qutoran rapamycin for clinically significant angiocheratomas and Qutoran Pentavastatin for disseminated superficial actinic porokeratosis (DSAP), are both on track to initiate Phase 2 studies in the second half of 2026.
The company holds six granted patents in the U.S. covering Qutoran rapamycin, with claims extending through at least 2038, and benefits from seven years of regulatory exclusivity for orphan designation upon approval.

Nov 11, 2025, 1:30 PM

Transcript

Palvella Therapeutics Reports Q3 2025 Financial Results and Pipeline Progress

PVLA

Earnings

New Projects/Investments

Guidance Update

Palvella Therapeutics ended Q3 2025 with $63.6 million in cash and equivalents as of September 30, 2025, providing approximately one and a half years of runway into the second half of 2027. Total operating expenses for the period were $10.2 million.
The company expects top-line data from its Phase 3 SELVA study of Qutoran rapamycin for microcystic lymphatic malformations in Q1 2026, with a planned NDA submission in H2 2026 and a potential U.S. launch in 2027. Additionally, top-line data from the Phase 2 TOIVA study of Qutoran rapamycin in cutaneous venous malformations is anticipated next month (December 2025).
Palvella is expanding its pipeline, with Qutoran Pentavastatin for disseminated superficial actinic porokeratosis expected to enter the clinic in H2 2026, and a Phase 2 study for Qutoran rapamycin in clinically significant angiocheratomas planned for H2 2026. The company is also accelerating commercial readiness for a standalone U.S. launch.

Nov 11, 2025, 1:30 PM

Transcript

PVLA Reports Q3 2025 Financials and Pipeline Advancements

PVLA

Earnings

New Projects/Investments

Guidance Update

Palvella ended Q3 2025 with $63.6 million in cash and equivalents, providing a runway into the second half of 2027.
The Phase 3 SELVA trial for Qutoran rapamycin in microcystic lymphatic malformations is on track, with top-line data expected in Q1 2026 and a planned NDA submission in the second half of 2026.
The company announced two new clinical programs: Qutoran Pentavastatin for disseminated superficial actinic porokeratosis (DSAP) and Qutoran rapamycin for clinically significant angiocheratomas, with Phase 2 studies planned to initiate in the second half of 2026 for both.
Palvella is accelerating its transition to a commercial-stage company, building out its U.S. commercial and medical affairs organizations for an anticipated standalone U.S. launch in 2027.

Nov 11, 2025, 1:30 PM

Transcript

Palvella Therapeutics Provides Q3 2025 Business and Pipeline Update

PVLA

Product Launch

New Projects/Investments

Guidance Update

Palvella Therapeutics has expanded its rare disease pipeline to four serious rare skin diseases, introducing Qtorin pitavastatin as a second product candidate for disseminated superficial actinic keratosis (DSAP).
The Phase 3 SELVA study for Qtorin Rapamycin in microcystic lymphatic malformations (MLM) over-enrolled and is on track for a top-line readout next quarter (Q4 2025), with NDA submission planned for 2026 and a U.S. launch anticipated in 2027.
The Phase 2 study of Qtorin Rapamycin in cutaneous venous malformations (CVM) achieved full enrollment and expects top-line data next month (Q4 2025).
New Phase 2 studies for Qtorin Rapamycin in clinically significant angiokeratomas and Qtorin pitavastatin in DSAP are both expected to initiate in 2026.
The company estimates a multi-billion dollar total addressable market for Qtorin Rapamycin in MLM, with over 30,000 diagnosed U.S. patients.

Nov 11, 2025, 1:30 PM

Transcript

Palvella Therapeutics Provides Q3 2025 Financial Update and Pipeline Milestones

PVLA

Earnings

Guidance Update

New Projects/Investments

Palvella Therapeutics reported $63.6 million in cash as of September 30, 2025, with a projected cash runway into 2H 2027. The company's R&D and G&A spend for Q3 2025 was $10.2 million, and projected cash at year-end is approximately $55 million.
The company anticipates Phase 2 top-line data for QTORIN™ Rapamycin in Cutaneous Venous Malformations (cVMs) in mid-December 2025.
Phase 3 top-line data for QTORIN™ Rapamycin in Microcystic Lymphatic Malformations (LMs) is expected in Q1 2026, with a potential NDA submission in 2H 2026 and FDA approval in 1H 2027.
Palvella announced two new QTORIN™ programs: QTORIN™ Rapamycin for Clinically Significant Angiokeratomas (announced September 2025) and QTORIN™ Pitavastatin for Disseminated Superficial Actinic Porokeratosis (DSAP) (announced November 2025), both targeting Phase 2 trial initiation in 2H 2026. These programs address conditions with >50k U.S. patients each.