Latest news with #QTORIN
Yahoo
16-05-2025
- Business
- Yahoo
Palvella Therapeutics Inc (PVLA) Q1 2025 Earnings Call Highlights: Strong Clinical Progress ...
Cash and Cash Equivalents: $75.6 million as of March 31, 2025. Research and Development Expenses: $4.1 million for Q1 2025, up from just under $1 million in Q1 2024. General and Administrative Expenses: $3.8 million for Q1 2025, compared to $800,000 in Q1 2024. Net Loss: $8.2 million or $0.74 per diluted share for Q1 2025, compared to $2.7 million or $1.54 per diluted share in Q1 2024. Cash Spend Forecast: Approximately $30 million total cash spend for 2025, with an expected year-end cash balance of at least $55 million. Clinical Trials: Exceeded enrollment target of 40 patients in Phase 3 study for QTORIN and rapamycin; Phase 2 study for cutaneous venous malformations has six sites open and enrolling. Funding and Cash Runway: Financing provides cash runway into the second half of 2027. Warning! GuruFocus has detected 6 Warning Signs with TSX: Release Date: May 15, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Palvella Therapeutics Inc (NASDAQ:PVLA) has exceeded its enrollment target of 40 patients in the Phase 3 Selva study for QTORIN rapamycin, indicating strong interest and progress in their clinical trials. The company has received FDA's breakthrough therapy, fast track, and orphan drug designations for QTORIN rapamycin, which could expedite the regulatory approval process. Palvella Therapeutics Inc (NASDAQ:PVLA) is financially strong with $75.6 million in cash and cash equivalents, providing a clear cash runway into the second half of 2027. The company is planning to unveil two new QTORIN programs in the second half of the year, indicating a robust pipeline and potential for future growth. Palvella Therapeutics Inc (NASDAQ:PVLA) has expanded its intellectual property portfolio with the issuance of a fifth US patent, extending anticipated claims into 2038. Palvella Therapeutics Inc (NASDAQ:PVLA) reported a net loss of $8.2 million for Q1 2025, which is a significant increase from the $2.7 million loss in the same period in 2024. The company faces the challenge of commercializing its products in a market with no existing FDA-approved therapies, which requires careful planning and execution. There is uncertainty regarding the pricing strategy for QTORIN rapamycin, as it will depend on the results of the Phase 3 trial and further market analysis. Palvella Therapeutics Inc (NASDAQ:PVLA) is still in the process of hiring a Chief Commercial Officer, which is crucial for guiding the company's commercial strategy. The company needs to manage the complexities of a 505B2 submission pathway for NDA approval, which requires careful coordination with regulatory advisors. Q: What is the target enrollment for the Phase 3 MLM study, and are there any plans to explore additional subpopulations? A: The target enrollment has always been 40 patients, and we are pleased with the demand from all clinical study sites. The study is designed based on Phase 2 efficacy results, and with 40 patients, we are more than 99% powered for the Phase 3 study. (Wesley Kaupinen, CEO; Jeffrey Martini, CSO) Q: How does the recent epidemiology study impact the company's view on the total addressable market for MLM? A: The study aligns with previous estimates of over 30,000 diagnosed US patients. It also introduces new insights, suggesting over 1,500 new cases annually. This information will help refine our market model and commercial strategies, particularly targeting the 150 vascular anomaly centers. (Wesley Kaupinen, CEO) Q: What are the plans for commercializing the pipeline, and would you consider a global partner? A: We plan to prioritize a stand-alone US launch for our products. For markets outside the US, such as Europe and Japan, we will consider both stand-alone launches and partnerships, with a bias towards leveraging partners' footprints. (Wesley Kaupinen, CEO) Q: Can you provide details on the NDA submission timeline and any additional work required for the 505B2 submission? A: We aim to submit the NDA in the second half of 2026, following top-line data in Q1 2026. We expect a six-month priority review due to breakthrough and fast track designations. The 505B2 pathway will streamline the review process by referencing existing data on oral rapamycin. (Wesley Kaupinen, CEO) Q: How is the company addressing the heterogeneity in MLM and CBM patient recruitment for the Phase 3 study? A: The study is enriched for moderate to severe patients to ensure a clear treatment effect. We have strict inclusion criteria and controls to ensure the right patients are enrolled, which is crucial given the small sample size. (Wesley Kaupinen, CEO; Jeffrey Martini, CSO) For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Sign in to access your portfolio
Yahoo
04-04-2025
- Business
- Yahoo
Palvella Therapeutics to Present at the Jones Healthcare and Technology Innovation Conference
WAYNE, Pa., April 04, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella), 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 Food and Drug Administration (FDA)-approved therapies, today announced that Wes Kaupinen, Founder and Chief Executive Officer of Palvella will participate in a fireside chat at the Jones Healthcare and Technology Innovation Conference on Wednesday, April 9, 2025, at 2:00 p.m. PT. A live webcast of the fireside chat will be available on the Events and Presentations section of Palvella's website at An archived replay of the webcast will be available for approximately 90 days following the presentation. 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 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. Contact Information InvestorsWesley H. KaupinenFounder and CEO, Palvella MediaMarcy NanusManaging Partner, Trilon Advisors, LLCmnanus@
Associated Press
04-04-2025
- Business
- Associated Press
Palvella Therapeutics to Present at the Jones Healthcare and Technology Innovation Conference
WAYNE, Pa., April 04, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc. (Palvella), 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 Food and Drug Administration (FDA)-approved therapies, today announced that Wes Kaupinen, Founder and Chief Executive Officer of Palvella will participate in a fireside chat at the Jones Healthcare and Technology Innovation Conference on Wednesday, April 9, 2025, at 2:00 p.m. PT. A live webcast of the fireside chat will be available on the Events and Presentations section of Palvella's website at An archived replay of the webcast will be available for approximately 90 days following the presentation. 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 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. Wesley H. Kaupinen Media Marcy Nanus Managing Partner, Trilon Advisors, LLC
Yahoo
01-04-2025
- Business
- Yahoo
Is Palvella Therapeutics Inc. (PVLA) the Best Performing NASDAQ Stock So Far in 2025?
We recently published a list of . In this article, we are going to take a look at where Palvella Therapeutics Inc. (NASDAQ:PVLA) stands against other best performing NASDAQ stocks so far in 2025. On March 29, T. Rowe Price portfolio manager Tony Wang appeared on CNBC's 'Closing Bell Overtime' to discuss the current volatility in the tech sector, especially the NASDAQ. The NASDAQ underperformed throughout March and registered the largest decline since June 2022. But the question that now arises is whether this decline and volatility present a buying opportunity or not. Wang leans towards less caution and more confidence in this potential bull market. To support his sentiment, he cited that growth and momentum have both been high in recent years. Tech stocks in particular have seen substantial growth in the past 2 year. He thinks that valuations could further improve, given that the sector recent strong years came from AI infrastructure build out. Wang also acknowledged that companies are naturally questioning whether current spending levels in the sector are sustainable or not. But he reiterated that compute will retain its value, despite concerns about whether it has a cyclical position or not. He also noted that deep tech could potentially lower inferencing costs and expand market access for companies that enter with limited capital. Like any other sector, cost reduction is an ideal long-term outcome for the tech industry as well. We used the Finviz stock screener to compile a list of all the best-performing NASDAQ stocks. We then picked the 11 NASDAQ stocks with the highest year-to-date gains, as of March 31. The stocks are ranked in ascending order of their year-to-date performance. We have also added the hedge fund sentiment for each stock which was sourced from Insider Monkey's database. Why are we interested in the stocks that hedge funds pile into? The reason is simple: our research has shown that we can outperform the market by imitating the top stock picks of the best hedge funds. Our quarterly newsletter's strategy selects 14 small-cap and large-cap stocks every quarter and has returned 373.4% since May 2014, beating its benchmark by 218 percentage points (see more details here). A biopharmaceutical research lab with a team of scientists working on immunomodulatory therapies. Number of Hedge Fund Holders: 9 Average Upside Potential as of March 31: 136.33% Palvella Therapeutics Inc. (NASDAQ:PVLA) is a clinical-stage biopharmaceutical company that develops and commercializes novel therapies to treat patients with serious and rare genetic skin diseases. Its lead product candidate QTORIN rapamycin is in a Phase 3 clinical trial for the treatment of microcystic lymphatic malformations. The Phase 3 clinical trial is called SELVA and has around 40 participants, which also include children between the ages of 3 and 5. Positive results from the earlier held Phase 2 trial have shown 100% of participants showing improvements. Results from the SELVA trial are expected in Q1 2026. It has received a grant of up to $2.6 million from the FDA. QTORIN rapamycin is essentially a topical gel that targets the underlying pathway of Microcystic LMs. This disease affects around 30,000 people in the US alone, with no primary approved treatment in the market. Palvella Therapeutics Inc. (NASDAQ:PVLA) aims to be the first company to provide an approved therapy for patients suffering from Microcystic LMs. Overall, PVLA ranks 2nd on our list of best performing NASDAQ stocks so far in 2025. While we acknowledge the growth potential of PVLA, our conviction lies in the belief that AI stocks hold great promise for delivering high returns and doing so within a shorter time frame. If you are looking for an AI stock that is more promising than PVLA but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock. READ NEXT: 20 Best AI Stocks To Buy Now and 30 Best Stocks to Buy Now According to Billionaires. Disclosure: None. This article is originally published at Insider Monkey.
