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Soleno Therapeutics Announces Multiple Presentations at the 2025 United in Hope: International Prader-Willi Syndrome Conference
GlobeNewswire News Room· 2025-06-20 12:00
Core Viewpoint - Soleno Therapeutics, Inc. is actively participating in the 2025 United In Hope: International Prader-Willi Syndrome Conference, showcasing its commitment to advancing research and improving the lives of individuals affected by Prader-Willi syndrome (PWS) through multiple presentations [1][3]. Company Overview - Soleno Therapeutics focuses on developing novel therapeutics for rare diseases, with its first commercial product, VYKAT™ XR (diazoxide choline) extended-release tablets, recently approved by the FDA on March 26, 2025 [11]. Conference Participation - The conference, anticipated to be the largest focused on PWS, is a collaboration between the Prader-Willi Syndrome Association | USA, the Foundation for Prader-Willi Research, and the International Prader-Willi Syndrome Organisation [2]. - Soleno will present one oral and six poster presentations, highlighting the significance of data and advocacy in addressing the needs of the PWS community [1][3]. Presentation Details - Key presentations include: - "Swallowability and Dosing Compliance of Diazoxide Choline Extended-Release (DCCR) Tablets in Patients with Prader-Willi Syndrome" [4]. - "Diazoxide Choline Extended-Release (DCCR) Tablets Significantly Reduce Hyperphagia in Patients with PWS Who Are Managed with Strict Food Controls" [4]. - "Long-term Efficacy Results of Diazoxide Choline Extended-Release (DCCR) Tablets in Participants with Prader-Willi Syndrome" [7]. - Presenters include experts from Queen Mary University of London and the University of Florida, indicating a strong academic collaboration [4][7][8]. About Prader-Willi Syndrome - PWS is a rare genetic neurodevelopmental disorder affecting approximately one in every 15,000 live births, characterized by hyperphagia, which can lead to severe health complications [9][10].
Telix Precision Medicine Announces AlFluor Radiochemistry Platform
GlobeNewswire News Room· 2025-06-20 12:00
Core Insights - Telix Pharmaceuticals has launched a new PET radiochemistry solution called AlFluor™, based on 18F-aluminium fluoride, aimed at enhancing PSMA imaging capabilities [1][2]. Group 1: Product Development - The AlFluor™ platform allows flexible radiolabeling of PSMA with either AlF or gallium-68, and can also be used with ligands targeting neuroendocrine tumors and fibroblast activation protein [2]. - A strategic agreement has been signed with University Hospital Ghent and Ghent University for the development of a novel [18F]AlF-PSMA-11 targeting agent, which includes a comprehensive CMC package for a Drug Master File [3]. - The Phase 3 trial involving 96 prostate cancer patients showed that PSMA-11 labeled with 18F demonstrated diagnostic performance comparable to commercial 68Ga-labeled agents, achieving high specificity (~90%) for metastatic detection [4]. Group 2: Clinical and Regulatory Engagement - [18F]AlF-PSMA-11 has shown favorable biodistribution and high tumor-to-background ratios, with low off-target uptake in multiple studies, indicating its potential effectiveness [4]. - Telix is currently engaging with regulators to determine the approval pathway for [18F]AlF-PSMA-11, indicating progress towards commercialization [4]. Group 3: Company Strategy and Vision - The CEO of Telix emphasized the company's goal to expand PSMA-PET imaging through innovative and flexible product deployment, aligning with the evolving needs of physicians and patients [5]. - The introduction of AlFluor™ is part of Telix's strategy to offer a broad range of PSMA-PET imaging agents, enhancing service, flexibility, and reliability for customers [5]. Group 4: Company Overview - Telix Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing therapeutic and diagnostic radiopharmaceuticals, with operations in multiple countries including the U.S., Brazil, Canada, Europe, and Japan [6]. - The company is listed on both the Australian Securities Exchange and the Nasdaq Global Select Market, indicating its international presence and investment appeal [6].
SpringWorks Therapeutics Receives Positive CHMP Opinion for Nirogacestat for the Treatment of Adults with Desmoid Tumors
Globenewswire· 2025-06-20 12:00
Core Insights - Nirogacestat is poised to become the first and only approved therapy in the EU for desmoid tumors if the European Commission grants marketing authorization, with a decision expected in Q3 2025 [1][2] Company Overview - SpringWorks Therapeutics, Inc. is a biopharmaceutical company focused on severe rare diseases and cancer, currently advancing nirogacestat for desmoid tumors [2][10] - The company has previously received Orphan Drug designation for nirogacestat from the European Commission [3] Clinical Trial Results - The positive opinion from the EMA's CHMP is based on the Phase 3 DeFi trial, which demonstrated a 71% lower risk of disease progression for nirogacestat compared to placebo [3][6] - The DeFi trial involved 142 patients, with nirogacestat showing significant improvements in progression-free survival, objective response rate, and patient-reported outcomes [6] Safety Profile - Nirogacestat has a manageable safety and tolerability profile, with common adverse reactions including diarrhea, nausea, and fatigue [4] Disease Context - Desmoid tumors are rare, aggressive tumors with an estimated 1,300-2,300 new cases diagnosed annually in the EU, predominantly affecting individuals aged 20 to 44 [7][8] - Despite not metastasizing, desmoid tumors have high recurrence rates, leading to a shift in treatment guidelines recommending systemic therapies as first-line interventions [8]
Soleno Therapeutics Announces Multiple Presentations at the 2025 United in Hope: International Prader-Willi Syndrome Conference
Globenewswire· 2025-06-20 12:00
Group 1: Conference Overview - The 2025 United In Hope: International Prader-Willi Syndrome Conference is the first-ever international meeting jointly hosted by the Prader-Willi Syndrome Association | USA, the Foundation for Prader-Willi Research, and the International Prader-Willi Syndrome Organisation [1][2] - The conference is expected to be the largest focused on Prader-Willi syndrome (PWS) ever held, taking place from June 24-28, 2025, in Phoenix, AZ [2] Group 2: Company Involvement - Soleno Therapeutics, Inc. announced one oral and six poster presentations at the conference, highlighting its commitment to advancing research and improving the lives of those impacted by PWS [1][3] - The company's first commercial product, VYKAT™ XR (diazoxide choline) extended-release tablets, was approved by the U.S. FDA on March 26, 2025, for the treatment of hyperphagia in individuals with PWS [8] Group 3: Presentation Details - The oral presentation will cover long-term efficacy results of diazoxide choline extended-release tablets in participants with PWS from completed studies, presented by Evelien Gevers [4] - Multiple poster presentations will address various aspects of diazoxide choline extended-release tablets, including swallowability, dosing compliance, and the emotional impact of hyperphagia [4][5] Group 4: About Prader-Willi Syndrome - Prader-Willi syndrome (PWS) is a rare genetic neurodevelopmental disorder caused by an abnormality in gene expression on chromosome 15, occurring in approximately one in every 15,000 live births [6][7] - The defining symptom of PWS is hyperphagia, which can lead to severe health issues and significantly diminish the quality of life for affected individuals and their families [7]
Axsome Therapeutics Champions Brain Health During Alzheimer's & Brain Awareness Month
Globenewswire· 2025-06-20 11:00
Core Insights - Axsome Therapeutics is collaborating with the Alzheimer's disease advocacy community to raise awareness about the increasing impact of Alzheimer's disease in the U.S. and the significance of brain health during Alzheimer's & Brain Awareness Month [1][2] Industry Overview - There are currently over 7 million Americans living with Alzheimer's disease, a number projected to rise to approximately 14 million by 2060 [2] - Alzheimer's disease is the most common form of dementia, characterized by cognitive decline and various behavioral symptoms [2] - Nearly 12 million individuals serve as caregivers for those affected by Alzheimer's disease, highlighting the extensive burden on public health [2] - The Global Burden of Disease classification indicates that Alzheimer's disease has escalated from the 12th to the 6th most burdensome disease in the U.S. from 1990 to 2016, based on disability-adjusted life years (DALYs) [2][8] Company Initiatives - Axsome Therapeutics is actively participating in raising awareness and sharing resources related to Alzheimer's disease, alongside advocacy and patient communities [2] - The company is involved in scientific and medical conferences throughout the year to further its mission [2] Research and Development Focus - Axsome Therapeutics is dedicated to addressing central nervous system (CNS) disorders through innovative treatments, focusing on novel mechanisms of action to improve patient outcomes [3][5] - The company's neuroscience portfolio includes FDA-approved treatments for various conditions, including major depressive disorder and narcolepsy [5]
Ipsen receives positive CHMP opinion for Cabometyx® in previously treated advanced neuroendocrine tumors
Globenewswire· 2025-06-20 10:19
Core Viewpoint - Ipsen has received a positive opinion from the CHMP of the EMA for Cabometyx® (cabozantinib) for adult patients with unresectable or metastatic neuroendocrine tumors, based on the CABINET Phase III trial results, with a final decision expected soon [1][6]. Company Summary - Ipsen is a global biopharmaceutical company focused on oncology, rare diseases, and neuroscience, with nearly 100 years of development experience [11]. - The company has exclusive rights for the commercialization and clinical development of Cabometyx outside the U.S. and Japan, while Exelixis retains rights in the U.S. [8]. Industry Summary - The incidence of neuroendocrine tumors (NETs) is increasing, with a higher prevalence than pancreatic or bladder cancer, and treatment options are often limited upon disease progression [2]. - The CABINET Phase III trial demonstrated significant efficacy for Cabometyx, showing median progression-free survival (PFS) of 13.8 months for pNET patients compared to 4.4 months for placebo [7]. - For advanced gastrointestinal and lung NETs, the five-year survival rates are 68% and 55%, respectively, while advanced pNET has a poor prognosis with a five-year survival rate of only 23% [3].
Despite Fast-paced Momentum, Fortress Biotech (FBIO) Is Still a Bargain Stock
ZACKS· 2025-06-19 13:51
Group 1: Momentum Investing Overview - Momentum investing deviates from the traditional strategy of "buying low and selling high," focusing instead on "buying high and selling higher" for quicker profits [1] - Fast-moving trending stocks can be challenging to enter at the right time, as they may lose momentum if future growth does not justify their high valuations [2] Group 2: Investment Strategy - A safer investment approach involves targeting bargain stocks that exhibit recent price momentum, utilizing tools like the Zacks Momentum Style Score to identify promising candidates [3] - Fortress Biotech (FBIO) is highlighted as a strong candidate, showing a price increase of 6.5% over the past four weeks, indicating growing investor interest [4] Group 3: Fortress Biotech (FBIO) Performance - FBIO has demonstrated a 7.7% price gain over the past 12 weeks, with a beta of 1.76, suggesting it moves 76% more than the market [5] - The stock has a Momentum Score of A, indicating a favorable entry point for investors looking to capitalize on its momentum [6] Group 4: Earnings Estimates and Valuation - FBIO's upward trend in earnings estimate revisions has earned it a Zacks Rank 2 (Buy), which is associated with strong momentum effects [7] - The stock is currently trading at a Price-to-Sales ratio of 0.93, suggesting it is undervalued, as investors pay only 93 cents for each dollar of sales [7] Group 5: Additional Opportunities - Besides FBIO, there are other stocks that meet the criteria of the 'Fast-Paced Momentum at a Bargain' screen, presenting further investment opportunities [8] - Investors can explore over 45 Zacks Premium Screens tailored to different investing styles to identify potential winning stocks [9]
AlloVir (ALVR) Earnings Call Presentation
2025-06-19 13:33
Company Overview June 2025 1 Forward-Looking Statements & Disclaimer This presentation contains "forward-looking statements" within the meaning of the U.S. Private Securities Litigation Reform Act of 1995 and Section 21E of the Securities Exchange Act of 1934, as amended, that involve substantial risk and uncertainties. All statements, other than statements of historical fact, contained in this presentation, including statements regarding the strategy, future operations, future financial position, projected ...
BridgeBio Pharma (BBIO) Earnings Call Presentation
2025-06-19 13:29
Corporate Presentation April 29th, 2025 Forward Looking Statements and Disclaimer The presentation contains forward-looking statements. Statements made or presented may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Words such as "believe," "anticipate," "plan," "expect," "intend," "will," "may," "goal," "potential," "should," " ...
大阪世博会“广东活动周”6月下旬举办,聚焦产业投资推介
据悉,广东活动周开幕式、粤港澳大湾区投资环境推介暨中日大健康产业对接会两场大型活动将在"广 东活动周"举行。在对接会上,将有300多名中日企业代表与会共商合作,就大健康、智能制造等领域进 行对接交流。会上还将举办广州、韶关、中山、阳江等重点城市专题推介活动,面向日本工商界代表, 介绍当地营商环境、优惠政策和重点园区,促进产业对接和投资合作,推动共赢发展。 2025年日本大阪世博会中国馆广东活动周将于6月27至29日在日本大阪世博会中国馆举行。 南方财经记者谭海燕 广州报道 6月19日,在"2025年日本大阪世博会中国馆广东活动周"新闻发布会上,广东省贸促会副会长谢惠蓉介 绍,为期三天的"广东活动周"将突出岭南地方特色和文化特点,围绕"绿美广东,智创未来"主题,举办 一系列经贸、文化、科技交流活动。 另一方面,开展"南粤之窗"国际文化交流系列活动,英歌舞、粤剧、广东音乐、醒狮等广东的非遗民俗 精品将到世博舞台进行全方位展示。通过舞蹈、武术、音乐等形式,创新结合多种表演方式,全面呈现 广东民俗文化的内涵和价值,向世界展示展现传统与现代交融的岭南文化新活力。 南方财经记者了解到,广东共有11个地市组织经贸代表团参加 ...