Palvella Therapeutics Reports Full Year 2024 Financial Results and Provides Corporate Update

Upon close of merger and $78.9mm concurrent private placement from a syndicate of leading healthcare-dedicated investors, completed transformation to a publicly traded rare disease biopharmaceutical company advancing a late clinical-stage pipeline and a platform for treating serious, rare genetic skin diseases

Top-line results from SELVA, a Phase 3 single-arm, baseline-controlled trial evaluating QTORIN$(TM)$ 3.9% rapamycin anhydrous gel (QTORIN(TM) rapamycin) for the treatment of microcystic lymphatic malformations (microcystic LMs), on track for the first quarter of 2026

Top-line results from TOIVA, a Phase 2 single-arm, baseline-controlled trial evaluating QTORIN(TM) rapamycin for the treatment of cutaneous venous malformations (cutaneous VMs), on track for the fourth quarter of 2025

Planned QTORIN(TM) pipeline expansion in second half of 2025

Cash and cash equivalents of over $83 million as of December 31, 2024 expected to fund operations into the second half of 2027

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

WAYNE, Pa., March 31, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella or "the Company"), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, reported financial results for the full year ending December 31, 2024 and provided a corporate update.

"2024 was marked by significant progress towards achieving our vision of becoming the leading rare disease biopharmaceutical company focused on serious, rare genetic skin diseases," said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. "Upon the close of our merger and concurrent private placement in December 2024, we were able to rapidly advance QTORIN(TM) rapamycin, our lead product candidate from the QTORIN(TM) platform, into the Phase 3 SELVA study for the treatment of microcystic lymphatic malformations and the Phase 2 TOIVA study for the treatment of cutaneous venous malformations. Microcystic LMs and cutaneous VMs are both serious, rare, and chronically debilitating genetic diseases for which QTORIN(TM) rapamycin has the potential to be the first FDA-approved therapy and standard of care in the U.S."

Mr. Kaupinen continued, "In addition to these two initial indications for QTORIN(TM) rapamycin, which we believe together could exceed $1 billion in U.S. peak annual sales, we plan to broaden our pipeline in 2025 by advancing new and existing QTORIN(TM) programs for additional serious, rare genetic skin diseases with no FDA-approved therapies."

Recent Research and Development Highlights

QTORIN(TM) rapamycin for the treatment of microcystic LMs

   -- Microcystic LMs are a rare, chronically debilitating genetic disease 
      caused by dysregulation of the phosphatidylinositol 3-kinase 
      (PI3K)/mammalian target of rapamycin (mTOR) pathway. The disease is 
      characterized by malformed lymphatic vessels that protrude through the 
      skin and persistently leak lymph fluid (lymphorrhea) and bleed, often 
      leading to recurrent serious infections and cellulitis that can cause 
      hospitalization. 
 
   -- There are no FDA-approved treatments for the estimated more than 30,000 
      individuals in the U.S. with microcystic LMs. 
 
   -- In October 2024, the Company was awarded an Orphan Products Clinical 
      Trials Program grant of up to $2.6 million from the FDA Office of Orphan 
      Products Development to support the Phase 3 SELVA study, a 24-week, Phase 
      3, single-arm, baseline-controlled clinical trial of QTORIN(TM) rapamycin 
      for the treatment of microcystic LMs. Out of 51 grant applications 
      received by the FDA Orphan Products Grants Program in fiscal year 2024, 
      Palvella's Phase 3 clinical trial was one of seven new clinical trials 
      that was awarded a grant. 
 
   -- Two posters, including data supporting QTORIN(TM) rapamycin as a 
      potential targeted therapy for the treatment of microcystic LMs and a 
      review of the Phase 3 SELVA study, were presented at the 12th Pediatric 
      Dermatology Research Alliance (PeDRA) Annual Conference in October 2024. 
 
   -- In November 2024, Palvella dosed the first patient in the Phase 3 SELVA 
      study. 
 
   -- Breakthrough Therapy Designation, Orphan Drug Designation, and Fast Track 
      Designation from the U.S. FDA have been granted to QTORIN(TM) rapamycin 
      for the treatment of microcystic LMs. Orphan Drug Designation has also 
      been granted by the European Medicines Agency. 
 
   -- Top-line results from SELVA are anticipated in the first quarter of 2026. 

QTORIN(TM) rapamycin for the treatment of cutaneous VMs

   -- Cutaneous VMs are a rare genetic disease caused by mutations in genes 
      that cause overactivation of the PI3K/mTOR signaling pathway, leading to 
      dysfunctional veins within the skin. These malformations can cause 
      substantial morbidity and functional impairment, significantly impact 
      quality of life, and are associated with severe bleeding, ulceration, and 
      other potential complications. 
 
   -- There are no FDA-approved treatments for the estimated more than 75,000 
      individuals in the U.S. with cutaneous VMs. 
 
   -- Published case studies and real-world evidence from over 20 publications 
      have provided preliminary evidence of clinical benefit from the off-label 
      use of systemic rapamycin in the treatment of patients with venous 
      malformations who have TIE2 and PIK3CA mutations while highlighting the 
      need for topical agents which could potentially reduce the toxicities 
      associated with systemic therapy. 
 
   -- In January 2025, Palvella announced the dosing of the first patients in 
      TOIVA, a Phase 2 single-arm, open-label, baseline-controlled clinical 
      trial of QTORIN(TM) rapamycin for the treatment of cutaneous VMs. 
 
   -- Fast Track Designation from the FDA has been granted to QTORIN(TM) 
      rapamycin for the treatment of venous malformations. 
 
   -- Top-line results from TOIVA are anticipated in the fourth quarter of 
      2025. 

QTORIN(TM) rapamycin and QTORIN(TM) platform expansion

   -- The next target clinical indication for QTORIN(TM) rapamycin is 
      anticipated in the second half of 2025. The expansion of QTORIN(TM) 
      rapamycin into additional indications is supported by comprehensive 
      publications by leading researchers, including Andrew Swarbrick et al 
      (2021) and Dr. Joyce Teng and colleagues (Fogel et al, 2015) which 
      highlight the broad potential of rapamycin in several difficult to treat, 
      mTOR-driven skin diseases while advocating for targeted, topical 
      approaches suited to improve tolerability and safety. 
 
   -- The second product candidate from the QTORIN(TM) platform is anticipated 
      in the second half of 2025. Similar to QTORIN(TM) rapamycin, we believe 
      this product candidate will have the potential to be developed for 
      several serious, rare genetic skin diseases. 

Recent Corporate Highlights

   -- In July 2024, Palvella announced a definitive merger agreement with 
      Pieris Pharmaceuticals. In December 2024, the Company closed the merger 
      and a concurrent private placement of $78.9 million, co-led by BVF 
      Partners L.P. and Frazier Life Sciences. Additional new investors 
      included Blue Owl Healthcare Opportunities, Nantahala Capital, DAFNA 
      Capital Management, ADAR1 Capital Management, and a healthcare dedicated 
      fund. Existing investors Samsara BioCapital, Petrichor, CAM Capital, 
      Ligand Pharmaceuticals (Nasdaq: LGND), Integrated Finance Group (an 
      AscellaHealth partner company), BioAdvance, and Gore Range Capital also 
      committed to participate in the financing. 
 
   -- Matthew Korenberg was appointed as Chief Financial Officer in October 
      2024. Mr. Korenberg is a seasoned executive with significant operational 
      and financial leadership experience, including senior roles at Ligand 
      Pharmaceuticals $(LGND)$ and in healthcare investment banking at 
      Goldman Sachs. 

Full Year 2024 Financial Results

   -- Cash and cash equivalents as of December 31, 2024, were $83.6 million. 
      Palvella expects such resources will be sufficient to fund its operations 
      into the second half of 2027, and sufficient to accomplish its current 
      strategic agenda. 
 
   -- Research and development expenses were $8.2 million for the twelve months 
      ended December 31, 2024, compared to $8.8 million for the twelve months 
      ended December 31, 2023. 
 
   -- General and administrative expenses were $5.9 million for the twelve 
      months ended December 31, 2024, compared to $3.1 million for the twelve 
      months ended December 31, 2023. The increase in G&A expenses was 
      primarily driven by increases in expenses related to becoming a public 
      company. 
 
   -- Net loss was $17.4 million or $7.83 per basic and diluted share for the 
      twelve months ended December 31, 2024, compared to net income of $17.9 
      million or $2.19 and $2.17 per basic and diluted share, respectively, for 
      the twelve months ended December 31, 2023. 
 
   -- Shares outstanding were 13,687,830 as of December 31, 2024, including 
      11,012,105 shares of common stock and 2,675,725 common share equivalents 
      assuming conversion of our outstanding preferred shares and prefunded 
      warrants. 

Conference Call Details

(MORE TO FOLLOW) Dow Jones Newswires

March 31, 2025 07:30 ET (11:30 GMT)