Latest news with #rapamycin
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
