Latest news with #rapamycin
Medical News Today
23-06-2025
- Health
- Medical News Today
Rapamycin may extend lifespan as effectively as dietary restrictions
Research is ongoing about potential strategies to prolong life. A meta-analysis found that the drug rapamycin prolongs life in several vertebrate appeared to prolong life at a level similar to dietary restrictions. How to prolong life is a key area of scientific research. Experts are interested in medications that have the potential to boost longevity.A recent meta-analysis published in Aging Cell explored how rapamycin and metformin influenced longevity among several results confirmed that dietary restriction appears to prolong life and that rapamycin offers similar benefits. Researchers also found that metformin did not seem to prolong life. More research is required to see how rapamycin might help boost longevity in people. Rapamycin: Does it increase lifespan?In this paper, researchers note that decreasing food intake without malnourishment appears to prolong life but that this strategy is difficult for people to stick to. Thus, looking into possible medications that produce similar effects is an area of research. The two medications that were the focus of this analysis were rapamycin and metformin. According to the National Cancer Institute, rapamycin has a few functions, such as being an immunosuppressant and antibiotic, and it can help people who get helps with type 2 diabetes management. This analysis involved a systematic literature search to find relevant data. The final analysis included data from 167 papers looking at eight total vertebrate species, seeking to see how both medications affected longevity and how they compared to dietary extracted information on average and median lifespan from the papers. For this analysis, the two types of dietary restriction were caloric reduction and fasting, and researchers also sought to see if the results differed based on the sex of the animals involved. The data came from animals like mice, rats, turquoise killifish, and rhesus macaques. Overall, there were more males studied than females. There was also the most data on dietary restriction, and the most common type of dietary restriction was decreasing the number of calories. Regarding dietary restriction, the findings suggested great variation regarding the effects. Overall, researchers found that dietary restriction and rapamycin had a similar impact and appeared to contribute to prolonged life. Metformin appeared to only have a minimal impact on life from one metformin model, there appeared to be no consistent differences between male and female animals regarding longevity. Study author Zahida Sultanova, PhD, a Leverhulme Early Career Research Fellow with the University of East Anglia, in the United Kingdom summarized the key findings of the study to Medical News Today: 'We checked whether the two best-known 'diet-mimic' drugs increase lifespan similar to eating less in animals. By pooling data from 167 studies, we found that rapamycin is almost as reliable as eating less for increasing lifespan, whereas metformin is not. In other words, a compound that was extracted from soil bacteria 50 years ago seems able to copy many of the biological effects of a permanent diet, at least in lab animals.'Do the same benefits apply to people?This research analyzed animal data but did not include data about people. Additionally, most of these studies involved these animals in a laboratory setting and only looked at a small number of meta-analysis was also the work of only three researchers, sometimes with only one researcher doing a component of the work, which could have impacted the had the least amount of data on metformin, so more research about this medication might be helpful. They also operated under the assumption that if a paper did not specify male or female subjects, it was a mixed group, which could have been incorrect. The authors further note that the 'results were sensitive to how lifespan was reported.' Researchers also acknowledge strong publication bias and a lot of heterogeneity. Additionally, the type of measure used in study reporting affected results. In one measurement, the impact on life extension disappeared for the most part, the authors did not find a consistent difference in results based on the sex of the animals. They explain this could be because of 'differences in taxonomic groups studied […] and the calculated effect size.' Sultanova noted the following cautions regarding the findings: 'This study includes a high number of scientific studies conducted on different organisms such as mice, fish and monkeys. However, survival results in humans are not included because these drugs were not tested in humans for lifespan extension. Even if they are, the studies will take a long time considering the length of human lifespan. So, we do not recommend people to take rapamycin before the results of human trials consistently show that there are no side effects.'Why is it hard to study rapamycin in humans?Researchers suggest the need for research involving other species in natural and laboratory settings. They also note the need to understand the difference in impact for 'different strains of the same species exposed to the same treatment.'Future research can further focus on the differences between rapamycin and metformin and why they impact lifespan differently. More research into the differences in rapamycin's results in males and females could be helpful as well. More research can be done to see if rapamycin can promote prolonged life in people, but there may be some challenges in this Ali, MD, a board-certified general surgeon, bariatric surgeon, and medical director of MemorialCare Surgical Weight Loss Center at Orange Coast Medical Center in Fountain Valley, CA, who was not involved in the study, told MNT that it 'shows the contribution of the immune system to lifespan, as rapamycin is an immunosuppressive medication.'According to him: 'The most logical next step is to explore the findings in humans; however, this would be a difficult study to design as rapamycin is a medication used in specific cancers and organ transplant and has significant side effects.'Despite this, the results show a potential benefit of rapamycin that warrants more explained that: 'Clinically, that puts rapamycin (and the mTOR pathway it targets) at the front of the queue for future anti-ageing therapies in humans. The compound had already been used for organ-transplant patients, so medical professionals understand its potential side effects.''The next step is waiting for the results of ongoing human trials that test lower and intermittent doses of rapamycin and refining the compound to 'rapalog' versions that keep the benefits while omitting side-effects such as immune suppression,' she told us.'Another important next step would be developing drugs that are similar in structure and function to rapamycin but without the side-effects. Scientists have already started refining rapamycin and producing the so-called rapalogs,' Sultanova noted.
Yahoo
23-06-2025
- Business
- Yahoo
Palvella Therapeutics Granted Sixth U.S. Patent Covering 0.1–20% Anhydrous Compositions of Rapamycin and Other mTOR Inhibitors
New intellectual property builds on Palvella's multi-layered exclusivity strategy Patent term expected through at least 2038 WAYNE, Pa., June 18, 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 U.S. Food and Drug Administration (FDA)-approved therapies, today announced that the United States Patent and Trademark Office (USPTO) issued patent No. 12,329,748 for claims related to the Company's lead product candidate QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin). The patent provides broad protection for the proprietary anhydrous topical composition and methods of use for QTORIN™ rapamycin. 'Our sixth issued U.S. patent marks another meaningful milestone in solidifying the exclusivity position of QTORIN™ rapamycin,' said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. 'We're executing a multi-layered strategy to protect and maximize the long-term value of QTORIN™ rapamycin—anchored by strong intellectual property, proprietary formulation and manufacturing trade secrets, and regulatory exclusivities.' The newly granted U.S. patent encompasses a wide range of composition and method-of-use claims for QTORIN™ rapamycin and other mTOR inhibitors—including temsirolimus and everolimus—formulated in anhydrous compositions. It also covers treatment of a broad spectrum of dermatologic conditions, with emphasis on rare and difficult-to-treat diseases such as microcystic lymphatic malformations and venous malformations. QTORIN™ rapamycin has been granted Breakthrough Therapy, Orphan Drug, and Fast Track Designations by the FDA for the treatment of microcystic lymphatic malformations. If approved, QTORIN™ rapamycin is expected to qualify for seven years of orphan drug market exclusivity in the U.S. Palvella has also been named an awardee of an FDA Orphan Products Grant from the FDA's Office of Orphan Products Development and is eligible to receive up to $2.6 million over the life of the grant to support the ongoing SELVA Phase 3 trial of QTORIN™ rapamycin. 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. 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 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 Media Marcy Nanus Managing Partner, Trilon Advisors LLC mnanus@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
The Independent
29-05-2025
- Health
- The Independent
Drug combination found to extend lifespan by 30 per cent
A combination of two cancer drugs significantly boosts lifespan in mice, according to a new study that may lead to better strategies for longevity. The drugs rapamycin and trametinib given together as a combination can increase mice lifespan by up to 30 per cent, say researchers, including from the Max Planck Institute for Biology of Ageing. Trametinib alone can extend lifespan by 5 to 10 per cent and rapamycin by 15–20 per cent, according to the study published in the journal Nature Ageing. Researchers say the drug combination has several other positive effects on mice health in old age, including less chronic inflammation in tissues and a delayed onset of cancer. Previous studies showed potent anti-ageing effects of rapamycin in several animals. Trametinib wasn't known to extend lifespan in mice but previous research in flies indicated it might. In humans, the two drugs have been used for cancer treatment. While both drugs act on the same chemical network in the body, their combination appears to achieve novel effects that are likely not attributable to just an increase in dosage. Gene activity analysis of mice tissues shows the combination influences genes differently than is achieved by administering the drugs individually. The analysis reveals specific gene activity changes that are only caused by the combination of the two drugs. In further studies, researchers hope to determine the optimal dose and route of administration of trametinib to maximise its life-prolonging effects while minimising unwanted side effects. 'Trametinib, especially in combination with rapamycin, is a good candidate to be tested in clinical trials as a geroprotector,' Sebastian Grönke, a co-author of the study, says. 'We hope that our results will be taken up by others and tested in humans. Our focus is on optimising the use of trametinib in animal models.' While the exact same kind of effect may not be possible in humans, researchers hope the drugs can help people stay healthy and disease-free for longer in life. "Further research in humans in years to come will help us to elucidate how these drugs may be useful to people and who might be able to benefit,' British geneticist Dame Linda Partridge said in a statement. Scientists hope the drug combination may be developed into a promising strategy for combating age-related diseases and promoting longevity.
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
