Apomorphine Hydrochloride

4月22日，百时美施贵宝（BMS）公布了3期ARISE临床试验的顶线结果。该研究评估了Cobenfy（占诺美林/曲司氯铵）作为辅助疗法在症状控制不佳的成人精神分裂症患者中的安全性和有效性。研究结果显示，与安慰剂联合非典型抗精神病药相比，Cobenfy辅助治疗在第6周时使阳性和阴性症状量表（PANSS）总分降低2.0分，但未达到主要终点的统计学显著性阈值（P=0.11）。初步分析表明，在特定患者亚组中，Cobenfy辅助治疗显示出优于安慰剂的症状改善趋势。值得注意的是，事后亚组分析揭示了显著的应答差异：以利培酮为基础治疗的患者与使用其他非利培酮类抗精神病药（包括帕利哌酮、阿立哌唑、齐拉西酮、鲁拉西酮和卡利拉嗪）的患者相比，表现出不同的治疗效果。ARISE试验（KAR-012）是一项为期6周、随机、双盲、安慰剂对照的多中心门诊研究，旨在评估Cobenfy对当前抗精神病药疗效不佳的成人精神分裂症患者（18-65岁）的辅助治疗效果。研究纳入标准包括：保持稳定的背景治疗，且筛选期PANSS总分≥70分。研究设置的主要终点为第6周时PANSS总分较基线的变化，次要终点包括：个人与社会功能量表（PSP）评分临床总体印象-严重度量表（CGI-S）评分PANSS Marder因子分应答率（定义为PANSS总分改善≥30%）用药偏好评估完成ARISE试验的合格受试者可进入为期52周的开放标签扩展研究（OLE），以进一步评估该疗法的长期安全性和耐受性特征。占诺美林/曲司氯铵是BMS去年收购的产品，是失败产品二次定位、成功盘活的经典案例。关于占诺美林的故事[1]Karuna成立于2009年，于2024年5月被百时美施贵宝耗资140亿美元收购。Karuna之所以如此值钱，是因为它成功盘活了一个“废弃”的高潜力药物占诺美林。占诺美林是一个选择性乙酰胆碱M1和M4受体激动剂，是礼来与诺和诺德开发的一个抗阿尔茨海默病（AD）药物。上世纪八九十年代，科学家认为AD与乙酰胆碱通路有关，并成功开发了多奈哌齐、卡巴拉汀、加兰他敏等产品。虽然临床试验证实占诺美林对AD有效，但因严重的胃肠道和心血管不良反应，临床试验在1998年被终止。在临床试验中，科学家发现占诺美林对AD“异常行为”有影响，如幻觉、妄想等，这让他们联想到该药物有望用于治疗精神分裂症。本世纪初，丹麦科学家就通过阿扑吗啡诱导建模，在动物试验中评估了占诺美林在对抗精神分裂方面的活性，而且礼来公司还开展了早期的临床试验初步证明了疗效，但因严重的不良反应，项目开发也相继终止。2012年，Karuna以“10万美元首付款+最高5400万美元里程碑付款+销售提成”的方式向礼来购买了占诺美林的全球开发权益，包括礼来持有的专利、监管文件和试验数据。随后，Karuna将占诺美林与M受体阻断剂曲司氯铵联用，巧妙地解决了占诺美林的不良反应问题。他们认为占诺美林的不良反应是因激动外周M受体引起，只要找到一种不能进入中枢的M受体阻断剂与之搭配使用，外周M受体激动引起的不良反应就能抵消。经过大量的筛选，科学家最终选择了曲司氯铵。一开始，Karuna几乎无法融资，硬着头皮在70名健康者中进行了一期临床试验，结果发现KarXT（占诺美林+曲司氯铵的固定剂量组合物）组的不良反应发生率相比占诺美林单药组下降了53%。这样的数据非常让人兴奋，2018年，Karuna在A轮融资中成功筹集了4200万美元，六个月后又在B轮融资中成功筹集到8000万美元。同年，Karuna的股票挂牌上市。2019年11月，KarXT的二期临床试验达到了预期的终点，Karuna的股价飞速上涨，市值从3亿多美元飞升至20多亿美元。2020~2024年间，Karuna在国际顶级期刊上发表了多篇文章，披露的临床数据非常惊艳。作为精神分裂症全新机制的疗法，市场潜力巨大，分析师认为该产品在2028年的销售额有望达到28亿美元。2024年5月，百时美施贵宝以140亿美元的天价收购了Karuna，而KarXT在2024年9月获得了FDA批准。[1] 内容摘自《探寻跨国药企的成功基因》，该书将在下半年出版发行药事纵横投稿须知：稿费已上调，欢迎投稿

AGOURA HILLS, Calif., April 22, 2025 (GLOBE NEWSWIRE) -- Oncotelic Therapeutics, Inc. (OTCQB:OTLC), a biotechnology company focused on nanomedicine and RNA-based therapeutics, announced today that its Chairman and CEO, Dr. Vuong Trieu, will deliver a keynote presentation at the 21st Annual Congress of International Drug Discovery Science & Technology (IDDST-Europe), scheduled for June 18-20, 2025, in Stockholm, Sweden. Dr. Trieu's presentation, titled " Deciparticles: Novel Sub-20 nm Nanoparticles for Advanced Drug Delivery" will highlight the groundbreaking Deciparticles™ platform developed by Oncotelic Therapeutics and Sapu Nano. Deciparticles are a novel class of sub-20 nm nanoparticles specifically engineered to overcome challenges associated with delivering insoluble drugs to solid tumors. Deciparticles platform is able to formulate more than 90% of all water insoluble drugs tested – including the taxanes, The presentation will outline key findings demonstrating that Deciparticles significantly improve tumor penetration and achieve superior antitumor efficacy compared to conventional formulations. A central feature of this innovation is the integration of biomarker-driven strategies, leveraging DNA methylation signatures and gene-expression profiles predictive of patient survival, potentially enabling personalized therapeutic interventions. Oncotelic's lead candidate, Sapu-001, has shown promising preclinical results, demonstrating a higher maximum-tolerated dose and reduced toxicity relative to existing therapies. Dr. Trieu emphasized, "With Deciparticles, we've entered a new era of targeted drug delivery—leveraging sub-20 nm nanoparticles combined with epigenetic biomarkers to significantly enhance treatment outcomes for patients. We anticipate Phase 1 clinical trials for Sapu-001 later this year." Presentation Details: Event: IDDST-Europe 2025 ConferenceDate: June 19, 2025, at 10:30 CETSpeaker: Vuong Trieu, Ph.D., Chairman & CEO, Oncotelic TherapeuticsTitle: " Deciparticles: Novel Sub-20 nm Nanoparticles for Advanced Drug Delivery" About Oncotelic Oncotelic (f/k/a Mateon Therapeutics, Inc.), was formed in the State of New York in 1988 as OXiGENE, Inc., was reincorporated in the State of Delaware in 1992, and changed its name to Mateon Therapeutics, Inc. in 2016, and Oncotelic Therapeutics, Inc. in November 2020. Oncotelic is seeking to leverage its deep expertise in oncology drug development to improve treatment outcomes and survival of cancer patients with a special emphasis on rare pediatric cancers. Oncotelic has rare pediatric designation for Diffuse Intrinsic Pontine Glioma (“DIPG” through OT-101) through its 45% joint venture, GMP Biotechnology Limited, melanoma (through CA4P), and Acute Myeloid Leukemia (“AML” through OXi 4503). Oncotelic also acquired PointR Data Inc. in November 2019 to build an AI driven biotechnology company. Further, Oncotelic acquired AL-101, during the 4th quarter of 2021, for the intranasal delivery of apomorphine. We intend to develop AL-101 for the treatment of Parkinson Disease, erectile dysfunction, female sexual disorder and hypoactive sexual desire disorder. All these ailments have a very large population suffering from them and there is a need for treatments for each. For more information on AL-101, refer to our 2023 Annual Report on form 10-K filed with the SEC on April 12, 2024. Oncotelic's Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this communication regarding strategy, future operations, future financial position, prospects, plans and objectives of management are forward-looking statements. Words such as "may", "expect", "anticipate" "hope", "vision", "optimism", "design", "exciting", "promising", "will", "conviction", "estimate," "intend," "believe", "quest for a cure of cancer", "innovation-driven", "paradigm-shift", "high scientific merit", "impact potential" and similar expressions are intended to identify forward-looking statements. Forward looking statements contained in this press release include, but are not limited to, statements about future plans related to the operations of the JV, taking the JV into an initial public offering or the success thereof, the progress, timing of clinical development, scope and success of future clinical trials, the reporting of clinical data for the company's product candidates and the potential use of the company's product candidates to treat various cancer indications as well as obtaining required regulatory approval to conduct clinical trials and upon granting of approval by the regulatory agencies, the successful marketing of the products; building and the success of our nanoparticle platform and the related success of launching the platform, the success of the launch of a company with a DAO infrastructure, the success of the entity and the plans surrounding the pet and animal health, the ability for the Company to register the tokens of Pet2DAO, the actual filing of a registration statement and approval of the PDAO, or any other tokens that we may launch, as registrable securities with the SEC through a registration statement, the ability of the tokens to be tradable or any value such tokens may have if they become tradable.. Each of these forward-looking statements involves risks and uncertainties, and actual results may differ materially from these forward-looking statements or may not occur at all. Many factors may cause differences between current expectations and actual results, including unexpected safety or efficacy data observed during preclinical or clinical studies, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, failure of collaborators to support or advance collaborations or product candidates and unexpected litigation or other disputes, taking the Company or its affiliates through initial public offerings. These risks are not exhaustive, the company faces known and unknown risks, including the risk factors described in the Company's annual report on Form 10-K filed with the SEC on April 12, 2024 and in the company's other periodic filings. Forward-looking statements are based on expectations and assumptions as of the date of this press release. Except as required by law, the company does not assume any obligation to update forward-looking statements contained herein to reflect any change in expectations, whether because of new information, future events, or otherwise. Contact Information: For Oncotelic Therapeutics, Inc.:Investor Relationsir@oncotelic.com