Phase 3 SELVA trial evaluating QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of microcystic lymphatic malformations (microcystic LMs) has exceeded enrollment target of 40 patients; enrollment expected to close in June 2025

Phase 3 SELVA trial top-line results anticipated in the first quarter of 2026

Phase 2 TOIVA trial evaluating QTORIN™ rapamycin for the treatment of cutaneous venous malformations top-line results on track for the fourth quarter of 2025

QTORIN™ rapamycin has the potential to be the first approved therapy and standard of care in the U.S. for microcystic LMs and cutaneous venous malformations

Cash and cash equivalents of $75.6 million as of March 31, 2025, expected to fund operations into the second half of 2027

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

WAYNE, Pa., May 15, 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 first quarter ending March 31, 2025 and provided a corporate update.

"We are pleased with the strong interest from clinical investigators, clinical trial sites, and study participants in our Phase 3 SELVA study which together has led to exceeding our Phase 3 enrollment target of 40 patients," said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. "Based on a recently presented epidemiology study estimating diagnosed U.S. prevalence and incidence of lymphatic malformations with cutaneous involvement, the data continue to support a large potential market opportunity for QTORIN™ rapamycin, if approved, as potentially the first targeted therapy for this serious, rare genetic disease which currently has no FDA-approved therapies."

Recent Research and Development Highlights

QTORIN™ rapamycin for the treatment of microcystic lymphatic malformations (microcystic LMs)

Announced results from the Phase 2 study of QTORIN™ rapamycin for the treatment of microcystic LMs were published in the Journal of Vascular Anomalies (JoVA).
Expanded SELVA, the Company's Phase 3 clinical trial of QTORIN™ rapamycin for the treatment of microcystic LMs, to include patients ages 3 to 5 years old.
The SELVA trial has exceeded its enrollment target of 40 patients ages six and older. Enrollment is expected to close in June 2025 and top-line results are anticipated in the first quarter of 2026.
Presented a poster highlighting the estimated diagnosed U.S. prevalence and U.S. annual incidence of lymphatic malformations with cutaneous involvement at the 82nd Annual Meeting of the Society for Investigative Dermatology.
- Quantitative analysis of medical claims indicates an estimated 44,553 high probability LM patients with cutaneous involvement are projected based on the methodology developed by the authors that includes having greater than or equal to two diagnosis claims related to LM.
Reported the United States Patent and Trademark Office granted a patent for QTORIN™ rapamycin for the treatment of microcystic LMs, the Company's fifth patent granted in the U.S. for QTORIN™ rapamycin, with anticipated patent life extending into 2038.

QTORIN™ rapamycin for the treatment of cutaneous venous malformations (cutaneous VMs)

Announced the dosing of the first patients in TOIVA, a Phase 2 single-arm, open-label, baseline-controlled clinical trial of QTORIN™ rapamycin for the treatment of cutaneous VMs. The trial is currently enrolling patients at six sites across the U.S.
Top-line results from TOIVA are anticipated in the fourth quarter of 2025.

First Quarter 2025 Financial Results

Cash and cash equivalents as of March 31, 2025 were $75.6 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 $4.1 million for the three months ended March 31, 2025, compared to $1.0 million for the three months ended March 31, 2024. The increase in research and development expenses was primarily due to increased spending on the clinical development of QTORIN™ rapamycin for the treatment of microcystic LMs and cutaneous venous malformations, including conducting our Phase 3 SELVA and Phase 2 TOIVA trials, which were initiated in 2024.
General and administrative expenses were $3.8 million for the three months ended March 31, 2025, compared to $0.8 million for the three months ended March 31, 2024. The increase in general and administrative expenses was primarily driven by employee compensation expense due to headcount additions, as well as increases in expenses related to operating as a publicly-traded company.
Net loss was $8.2 million or $0.74 per basic and diluted share for the three months ended March 31, 2025, compared to net loss of $2.7 million or $1.54 per basic and diluted share for the three months ended March 31, 2024.
Shares outstanding were 13,697,114 as of March 31, 2025, including 11,021,389 shares of common stock and 2,675,725 common share equivalents assuming conversion of our outstanding preferred shares and prefunded warrants.

Conference Call Details

Palvella will host a conference call and live audiovisual webcast to discuss the Company's first quarter 2025 financial results and provide a corporate update at 8:30 a.m. ET today. To access the live webcast of the call with slides, please click here or visit the "Events & Presentations" section of Palvella's website. To access the call by phone, please use this registration link, and you will be provided with dial in details. A replay of the webcast will be available approximately 2 hours after the conclusion of the call and archived for 90 days under the "Events & Presentations" section of the Company's website at www.palvellatx.com.

About Palvella Therapeutics

Founded and led by rare disease drug development veterans, Palvella Therapeutics, Inc. (NASDAQ:PVLA) is 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 FDA-approved therapies. Palvella is developing a broad pipeline of product candidates based on its patented QTORIN™ platform, with an initial focus on serious, rare genetic skin diseases, many of which are lifelong in nature. Palvella's lead product candidate, QTORIN 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), is currently being evaluated in the Phase 3 SELVA clinical trial in microcystic lymphatic malformations and the Phase 2 TOIVA clinical trial in cutaneous venous malformations. For more information, please visit www.palvellatx.com or follow Palvella on LinkedIn or X (formerly known as Twitter).

QTORIN™ rapamycin is for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency for any indication.

Forward-Looking Statements

This press release contains forward-looking statements (including within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended (Securities Act)). These statements may discuss goals, intentions, and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the management of Palvella, as well as assumptions made by, and information currently available to, the management of Palvella. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as "may," "will," "should," "would," "expect," "anticipate," "plan," "likely," "believe," "estimate," "project," "intend," and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Statements that are not historical facts are forward-looking statements. Forward-looking statements include, but are not limited to, statements regarding the expected timing of the presentation of data from ongoing clinical trials, Palvella's clinical development plans and related anticipated development milestones, Palvella's cash and financial resources and expected cash runway, and the potential of, and expectations regarding, Palvella's programs, including QTORIN™ rapamycin, and its research-stage opportunities, including its expected therapeutic potential and market opportunity. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the ability to raise additional capital to finance operations; the ability to advance product candidates through preclinical and clinical development; the ability to obtain regulatory approval for, and ultimately commercialize, Palvella's product candidates, including QTORIN™ rapamycin; the outcome of early clinical trials for Palvella's product candidates, including the ability of those trials to satisfy relevant governmental or regulatory requirements; the fact that data and results from clinical studies may not necessarily be indicative of future results; Palvella's limited experience in designing clinical trials and lack of experience in conducting clinical trials; the ability to identify and pivot to other programs, product candidates, or indications that may be more profitable or successful than Palvella's current product candidates; the substantial competition Palvella faces in discovering, developing, or commercializing products; the negative impacts of global events on operations, including ongoing and planned clinical trials and ongoing and planned preclinical studies; the ability to attract, hire, and retain skilled executive officers and employees; the ability of Palvella to protect its intellectual property and proprietary technologies; reliance on third parties, contract manufacturers, and contract research organizations; and the risks and uncertainties described in the filings made by Palvella with the Securities and Exchange Commission (SEC), including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available at www.sec.gov. The events and circumstances reflected in our forward-looking statements may not be achieved or occur, and actual results could differ materially from those projected in the forward-looking statements. New risk factors and uncertainties may emerge from time to time, and it is not possible for management to predict all risk factors and uncertainties that Palvella may face. Except as required by applicable law, Palvella does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

This press release contains hyperlinks to information that is not deemed to be incorporated by reference into this press release.

Contact Information

Investors

Wesley H. Kaupinen

Founder and CEO, Palvella Therapeutics

wes.kaupinen@palvellatx.com

Media

Marcy Nanus

Managing Partner, Trilon Advisors LLC

mnanus@trilonadvisors.com

PALVELLA THERAPEUTICS, INC.

CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

(in thousands, except share and per share amounts)

	Three months ended March 31,
	2025			2024
Operating expenses:
Research and development	$	4,074		$	984
General and administrative		3,797			775
Total operating expenses		7,871			1,759
Loss from operations		(7,871	)		(1,759	)
Total other income (expense), net		(314	)		(777	)
Net loss	$	(8,185	)	$	(2,536	)
Less: Cumulative Series D preferred dividends		—			(194	)
Net loss attributable to common stockholders	$	(8,185	)	$	(2,730	)

Net loss per share — basic and diluted	$	(0.74	)	$	(1.54	)
Weighted-average number of common shares used in computing net loss per share — basic and diluted		11,013,697			1,770,167

PALVELLA THERAPEUTICS, INC.

CONDENSED CONSOLIDATED BALANCE SHEET INFORMATION

(in thousands)

	March 31,		December 31,
	2025		2024
Assets
Cash and cash equivalents	$	75,626	$	83,602
Other current assets		3,813		4,632
Total current assets		79,439		88,234
Total assets	$	79,439	$	88,234
Liabilities and Stockholders' Equity
Current liabilities	$	8,983	$	12,038
Non-current liabilities		14,879		13,589
Total liabilities		23,862		25,627
Total stockholders' equity		55,577		62,607
Total liabilities and stockholders' equity	$	79,439	$	88,234

Palvella Therapeutics Reports First Quarter 2025 Financial Results and Provides Corporate Update