Palvella Therapeutics Inc Stock Price, News & Analysis

Palvella Therapeutics (PVLA) has completed enrollment for its Phase 3 SELVA trial of QTORIN rapamycin, a treatment for microcystic lymphatic malformations (LMs). The trial significantly exceeded its enrollment target by 25%, with 51 subjects enrolled versus the planned 40. QTORIN rapamycin, which has received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA, is being evaluated as the first targeted therapy for this rare genetic disease. The 24-week trial includes subjects aged three years and older, with an open-label extension study available for eligible participants. The company expects to release top-line data in Q1 2026 and plans to submit a New Drug Application in H2 2026. Palvella has also received an FDA Orphan Products Grant worth up to $2.6 million to support the trial.

Palvella Therapeutics (PVLA) has secured its sixth U.S. patent (No. 12,329,748) for QTORIN™ rapamycin, extending protection until at least 2038. The patent covers anhydrous compositions containing 0.1-20% rapamycin and other mTOR inhibitors, along with their methods of use. QTORIN™ 3.9% rapamycin anhydrous gel, the company's lead product candidate, has received multiple FDA designations including Breakthrough Therapy, Orphan Drug, and Fast Track for treating microcystic lymphatic malformations. The company has also been awarded an FDA Orphan Products Grant worth up to $2.6 million to support its SELVA Phase 3 trial. If approved, QTORIN™ rapamycin could receive seven years of orphan drug market exclusivity in the U.S.

Palvella Therapeutics (NASDAQ: PVLA) has received initial funding from an FDA Orphan Products Grant to support its Phase 3 SELVA trial of QTORIN rapamycin for microcystic lymphatic malformations (LMs). The grant, worth up to $2.6 million in non-dilutive funding, was one of only seven new clinical trials selected from 51 applications, and notably the only Phase 3 trial awarded. The SELVA trial is a 24-week, single-arm study enrolling approximately 40 subjects aged three or older at U.S. vascular anomaly centers. The trial aims to evaluate QTORIN 3.9% rapamycin anhydrous gel administered once daily, with top-line results expected in Q1 2026. This development is significant as there are currently no FDA-approved treatments for microcystic LMs, a rare genetic skin disease.

Palvella Therapeutics (NASDAQ: PVLA) has appointed Ashley Kline as Chief Commercial Officer to lead the commercial launch of QTORIN™ rapamycin for microcystic lymphatic malformations. The appointment is significant given Kline's impressive track record at Dompé Pharmaceuticals, where she led Oxervate® to achieve over $500 million in annual U.S. sales. QTORIN™ aims to be the first FDA-approved therapy for microcystic lymphatic malformations, a rare genetic disease affecting over 30,000 patients in the U.S. Kline's expertise in rare disease commercialization, particularly her success with Oxervate's capital-efficient launch strategy, positions her well to build Palvella's commercial organization.

Palvella Therapeutics (PVLA) reported Q1 2025 financial results and provided updates on its clinical trials. The company's Phase 3 SELVA trial for QTORIN™ rapamycin gel has exceeded its 40-patient enrollment target, with results expected in Q1 2026. The drug aims to treat microcystic lymphatic malformations (LMs). A parallel Phase 2 TOIVA trial for cutaneous venous malformations is progressing with results expected in Q4 2025. Financially, Palvella reported $75.6 million in cash, expected to fund operations into H2 2027. The company posted a net loss of $8.2 million ($0.74 per share) for Q1 2025, compared to $2.7 million loss in Q1 2024. Research shows an estimated 44,553 high-probability LM patients with cutaneous involvement in the U.S., representing a significant market opportunity as QTORIN™ rapamycin could become the first FDA-approved therapy for these conditions.

Palvella Therapeutics (NASDAQ: PVLA), a clinical-stage biopharmaceutical company specializing in treatments for rare genetic skin diseases, has scheduled its first quarter 2025 financial results and corporate update conference call for May 15, 2025, at 8:30 a.m. ET. The company, which focuses on developing therapies for conditions currently lacking FDA-approved treatments, will provide a webcast with slides accessible through their website's "Events & Presentations" section. Investors can register for dial-in details through a provided link, and a replay will be available approximately 2 hours after the call, remaining archived for 90 days on the company website.

Palvella Therapeutics (PVLA), a clinical-stage biopharmaceutical company focused on developing therapies for rare genetic skin diseases, announced it will ring the Nasdaq opening bell on May 12, 2025. The ceremony, led by CEO Wes Kaupinen along with management team, board members, investors, and advisors, will begin at 9:15 am ET at the Nasdaq MarketSite Tower in New York City.

The event marks Palvella's milestone as a publicly traded, Nasdaq-listed company. The company specializes in developing treatments for serious, rare genetic skin diseases that currently lack FDA-approved therapies. The ceremony will be live-streamed on Nasdaq's X and Facebook pages.

Palvella Therapeutics (NASDAQ: PVLA) has announced groundbreaking research on lymphatic malformations (LMs) with cutaneous involvement in the U.S., to be presented at the Society for Investigative Dermatology Annual Meeting in May 2025.

The medical claims-based analysis, conducted over 7 years using Komodo Health data, reveals an estimated 44,553 high probability LM patients (with ≥2 diagnosis claims) and 48,414 potential LM patients (with ≥1 diagnosis claim), totaling up to 92,967 patients with cutaneous involvement.

The research, led by Dr. Ionela Iacobas from Texas Children's Hospital and David Lapidus from LapidusData, represents the first quantitative analysis of the U.S. LM population using medical claims data. The findings indicate growing demand for multidisciplinary care in complex vascular anomalies treatment.

Palvella Therapeutics (PVLA) has been granted its fifth U.S. patent (No. 12,268,673) for QTORIN™ rapamycin, extending patent protection until 2038. The patent broadens protection for QTORIN™ 3.9% rapamycin anhydrous gel and its clinical applications in treating microcystic lymphatic malformations (LMs).

QTORIN™ rapamycin has received multiple FDA designations including Breakthrough Therapy, Orphan Drug, and Fast Track for treating microcystic LMs. If approved, it could become the first FDA-approved therapy for this rare genetic skin disease and receive seven years of orphan drug market exclusivity in the U.S.