Co-Founder and former Chief Technical Officer of Arcutis Biotherapeutics brings extensive topical product development experience, including track record of translating science into commercially available therapies Dr. Osborne to guide expansion of QTORIN™ platform, including second QTORIN™ product candidate on track to be announced by year-end 2025 WAYNE, Pa., Sept. 03, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella or “the Company”), a clinical-stage biopharmaceutical company ...

Co-Founder and former Chief Technical Officer of Arcutis Biotherapeutics brings extensive topical product development experience, including track record of translating science into commercially available therapies Dr. Osborne to guide expansion of QTORIN™ platform, including second QTORIN™ product candidate on track to be announced by year-end 2025 WAYNE, Pa., Sept. 03, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella or “the Company”), a clinical-stage biopharmaceutical company ...

Core Insights - The Phase 3 SELVA trial for QTORIN™ rapamycin has completed enrollment, exceeding its target by over 25%, with top-line results expected in Q1 2026 [1][5] - The Phase 2 TOIVA trial for QTORIN™ rapamycin is on track for results in Q4 2025 [1][9] - QTORIN™ rapamycin aims to be the first approved therapy for microcystic lymphatic malformations and cutaneous venous malformations in the U.S. [1][3] - The company plans to announce a third clinical indication for QTORIN™ rapamycin and a second candidate from the QTORIN™ platform by the end of 2025 [1][9] Research and Development Highlights - Enrollment in the Phase 3 SELVA trial included 51 subjects, surpassing the original target of 40 [5] - The company received initial proceeds from an FDA Orphan Products Grant, potentially totaling $2.6 million, to support the SELVA trial [5] - Key presentations at scientific meetings included an oral presentation on the SELVA trial design and Phase 2 results [5] - The USPTO issued patent No. 12,268,673 for QTORIN™ rapamycin, with a patent life extending into 2038 [5] Financial Overview - As of June 30, 2025, the company reported cash and cash equivalents of $70.4 million, expected to fund operations into the second half of 2027 [1][9] - Research and development expenses for Q2 2025 were $5.1 million, up from $1.4 million in Q2 2024, primarily due to increased clinical development spending [9][17] - General and administrative expenses rose to $4.1 million in Q2 2025 from $1.5 million in Q2 2024, driven by increased employee compensation and public company operating costs [9][17] - The net loss attributable to common stockholders for Q2 2025 was $9.5 million, or $0.86 per share, compared to a net loss of $4.4 million, or $2.47 per share, in Q2 2024 [9][17] Corporate Developments - The company is expanding its rare disease pipeline with plans for two new development programs in 2025 [3][9] - The appointment of Ashley Kline as Chief Commercial Officer strengthens the executive leadership team [9] - The company was added to the Russell 3000® and Russell 2000® indexes on June 27, 2025 [9]

Core Viewpoint - Palvella Therapeutics, Inc. is set to report its second quarter 2025 financial results on August 14, 2025, and will host a conference call for investors to discuss these results and provide a corporate update [1][2]. Company Overview - Palvella Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for serious, rare genetic skin diseases that lack FDA-approved treatments [4]. - The company is developing a pipeline of product candidates utilizing its patented QTORIN™ platform, with a primary focus on lifelong rare genetic skin diseases [4]. - Palvella's lead product candidate, QTORIN 3.9% rapamycin anhydrous gel, is currently undergoing evaluation in the Phase 3 SELVA clinical trial for microcystic lymphatic malformations and the Phase 2 TOIVA clinical trial for cutaneous venous malformations [4]. Financial Communication - A conference call for investors will take place at 8:30 a.m. ET on August 14, 2025, to discuss the financial results and provide updates on the company [2]. - The call will be accessible via a live webcast and by phone, with a replay available approximately two hours after the call [3].

Company Overview - Palvella Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies for serious, rare genetic skin diseases that lack FDA-approved treatments [4] - The company is developing a pipeline of product candidates based on its patented QTORIN™ platform, with its lead product candidate being QTORIN™ 3.9% rapamycin anhydrous gel, currently in Phase 3 and Phase 2 clinical trials [4] Market Position - Palvella has been added to the Russell 3000 Index and the Russell 2000 Index, effective June 30, 2025, as part of the annual Russell indexes reconstitution [1][2] - Membership in the Russell 3000 Index indicates automatic inclusion in the large-cap Russell 1000 Index or small-cap Russell 2000 Index, which is significant for market visibility and investment opportunities [2] Industry Context - The Russell indexes are widely utilized by investment managers and institutional investors for index funds and as benchmarks for active investment strategies, with approximately $10.6 trillion in assets benchmarked against these indexes as of June 2024 [3]

Core Insights - Palvella Therapeutics has successfully completed the SELVA Phase 3 trial for QTORIN™ rapamycin, which targets microcystic lymphatic malformations, a serious genetic disease with no FDA-approved therapies [2][3] - The trial enrolled 51 subjects, exceeding the initial target of 40 by over 25%, indicating strong demand and interest in this treatment [2][3] - Top-line data from the SELVA trial is expected in the first quarter of 2026, with a New Drug Application submission planned for the second half of 2026 [5] Company Overview - Palvella Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for rare genetic skin diseases without FDA-approved treatments [7] - The company is developing a pipeline of product candidates based on its QTORIN™ platform, with QTORIN™ rapamycin as the lead candidate currently in clinical trials [7] - The company has received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA for QTORIN™ rapamycin, which may qualify for seven years of orphan drug market exclusivity if approved [4] Clinical Trial Details - The SELVA trial is a 24-week, single-arm, baseline-controlled study evaluating QTORIN™ rapamycin in individuals aged three years and older with microcystic lymphatic malformations [3] - Following an 8-week baseline period, eligible participants may continue treatment in an open-label extension study, which is also open to new subjects aged three to five years [3] Disease Background - Microcystic lymphatic malformations are characterized by malformed lymphatic vessels that lead to persistent lymph fluid leakage and bleeding, often resulting in serious infections [6] - The disease affects an estimated 30,000 diagnosed patients in the United States, with symptoms worsening over time and no current FDA-approved treatments available [6]

Core Viewpoint - Palvella Therapeutics has received a new patent for its lead product candidate QTORIN™ rapamycin, which solidifies its exclusivity in the market for treating rare genetic skin diseases [2][3]. Company Overview - Palvella Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing therapies for serious, rare genetic skin diseases without FDA-approved treatments [5]. - The company is led by veterans in rare disease drug development and is working on a pipeline based on its patented QTORIN™ platform [5]. Product Details - QTORIN™ rapamycin is a 3.9% anhydrous gel that has been granted Breakthrough Therapy, Orphan Drug, and Fast Track Designations by the FDA for treating microcystic lymphatic malformations [4]. - The newly issued patent (No. 12,329,748) covers a wide range of claims related to QTORIN™ rapamycin and other mTOR inhibitors, emphasizing its application in treating rare dermatologic conditions [3]. Financial Support - Palvella has been awarded an FDA Orphan Products Grant, which could provide up to $2.6 million to support the ongoing SELVA Phase 3 trial of QTORIN™ rapamycin [4]. Clinical Trials - QTORIN™ rapamycin is currently being evaluated in the Phase 3 SELVA clinical trial for microcystic lymphatic malformations and the Phase 2 TOIVA clinical trial for cutaneous venous malformations [5].

Core Insights - Palvella Therapeutics has received a grant from the FDA Office of Orphan Products Development to support its Phase 3 SELVA trial for QTORIN™ rapamycin, aimed at treating microcystic lymphatic malformations [2][4] - The SELVA trial is one of only seven new clinical trials selected for funding out of 51 applications in fiscal year 2024, highlighting its significance [3] - Microcystic lymphatic malformations are a rare genetic disease with no FDA-approved treatments, affecting over 30,000 patients in the U.S. [5] Company Overview - Palvella Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for serious, rare genetic skin diseases [6] - The company is led by veterans in rare disease drug development and is advancing a pipeline of product candidates based on its QTORIN™ platform [6] - QTORIN™ rapamycin is currently being evaluated in the Phase 3 SELVA trial and has the potential to become the first approved therapy for microcystic lymphatic malformations [4][6] Trial Details - The SELVA trial is a 24-week, single-arm, baseline-controlled study enrolling approximately 40 subjects aged three or older [3] - Top-line results from the SELVA trial are expected in the first quarter of 2026 [1][3] Grant Information - The FDA Orphan Products Grants Program provides non-dilutive funding to advance promising therapies for rare diseases, with Palvella's grant amounting to up to $2.6 million [1][4] - The program has supported clinical research leading to the approval of over 85 products since its inception [4]

Core Insights - The Phase 3 SELVA trial for QTORIN™ rapamycin has exceeded its enrollment target of 40 patients, with enrollment expected to close in June 2025 and top-line results anticipated in Q1 2026 [1][4] - The Phase 2 TOIVA trial for QTORIN™ rapamycin is on track to report top-line results in Q4 2025 [1] - QTORIN™ rapamycin has the potential to become the first approved therapy for microcystic lymphatic malformations and cutaneous venous malformations in the U.S. [1][4] Clinical Trials - The SELVA trial has expanded to include patients aged 3 to 5 years old, with a total of over 40 patients aged six and older enrolled [7] - A recent epidemiology study supports a significant market opportunity for QTORIN™ rapamycin if approved, as it targets a serious, rare genetic disease with no current FDA-approved therapies [4][7] Financial Overview - As of March 31, 2025, the company reported cash and cash equivalents of $75.6 million, expected to fund operations into the second half of 2027 [1][12] - Research and development expenses for Q1 2025 were $4.1 million, up from $1.0 million in Q1 2024, primarily due to increased spending on clinical trials [12] - General and administrative expenses rose to $3.8 million in Q1 2025 from $0.8 million in Q1 2024, driven by employee compensation and public company operational costs [12] Market Potential - An estimated 44,553 high probability lymphatic malformation patients with cutaneous involvement have been identified in the U.S., indicating a substantial market for QTORIN™ rapamycin [7] - The company has received a patent for QTORIN™ rapamycin for the treatment of microcystic LMs, extending its patent life into 2038 [7]

Core Viewpoint - Palvella Therapeutics, Inc. is set to report its first quarter 2025 financial results on May 15, 2025, and will host a conference call to discuss these results and provide a corporate update [1][2]. Company Overview - Palvella Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for serious, rare genetic skin diseases that lack FDA-approved treatments [4]. - The company is developing a pipeline of product candidates utilizing its patented QTORIN™ platform, with a primary focus on lifelong serious genetic skin diseases [4]. - The lead product candidate, QTORIN 3.9% rapamycin anhydrous gel, is currently undergoing evaluation in the Phase 3 SELVA clinical trial for microcystic lymphatic malformations and the Phase 2 TOIVA clinical trial for cutaneous venous malformations [4]. Financial Communication - A conference call for investors will take place at 8:30 a.m. ET on May 15, 2025, to discuss the financial results and provide updates on corporate activities [2]. - The call will be accessible via a live webcast and by phone, with a replay available approximately two hours after the call [3].