Ruxolitinib Phosphate

– Top-Line Results Anticipated in March 2026 – NEWTON, Mass., Sept. 10, 2025 /PRNewswire/ -- Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, today announced that it has completed enrollment in the Phase 3 SENTRY trial, which is evaluating selinexor in combination with ruxolitinib in JAKi-naïve myelofibrosis patients. "We are excited to announce that we have completed enrollment of our Phase 3 SENTRY trial and look forward to sharing top-line data from this pivotal trial in March 2026," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "Selinexor plus ruxolitinib has the potential to be the first combination therapy approved for the treatment of myelofibrosis, depending on the outcome of the data. By combining selinexor with the current standard of care, we believe we have the potential to redefine the way people living with myelofibrosis are treated." "I am grateful for the patients, their families and caregivers, the investigators and their clinical trial staff, as well as the extraordinary efforts of the Karyopharm team and our external partners for their help in successfully achieving this important milestone," said Reshma Rangwala, MD, PhD, Chief Medical Officer and Head of Research of Karyopharm. "This trial is advancing our understanding of the treatment of myelofibrosis and the potential role that XPO1 inhibition may play in this disease. People living with myelofibrosis deserve new treatment options and everyone involved in SENTRY is making an important contribution towards our common goal of providing additional options to patients with this disease." "We are encouraged by the work that Karyopharm is doing in myelofibrosis and eagerly await data from the Phase 3 SENTRY trial," said Kapila Viges, Chief Executive Officer of MPN Research Foundation. "The myelofibrosis community is in need of new, more effective therapies that can help a greater number of patients beyond what is available with currently approved options. Efforts to develop new therapies bring hope to the myelofibrosis community and open the potential for patients to have more treatment options. For patients, options matter." SENTRY (XPORT-MF-034; NCT04562389) is a Phase 3 clinical trial evaluating a once-weekly dose of 60 mg of selinexor in combination with ruxolitinib compared to placebo plus ruxolitinib in JAKi-naïve myelofibrosis patients with platelet counts >100 x 10 9/L. Patients are randomized 2-to-1 to the selinexor arm. The co-primary endpoints for this trial are spleen volume response rate ≥ 35% (SVR35) at week 24 and the average change in absolute total symptom score (Abs-TSS) over 24 weeks relative to baseline. The Phase 3 trial enrolled 353 patients. About Myelofibrosis Myelofibrosis is a rare blood cancer that affects approximately 20,000 patients in the United States and 17,000 patients in the European Union1. The disease causes bone marrow fibrosis (scarring in the bone marrow), which makes it difficult for the bone marrow to make healthy blood cells, splenomegaly (enlarged spleen), progressive anemia which often leads to symptoms like fatigue and weakness, and other disease associated symptoms including abdominal discomfort, pain under the left ribs, early satiety, night sweats and bone pain. The only approved class of therapies to treat myelofibrosis are JAK inhibitors, including ruxolitinib. Patients treated with the most commonly prescribed JAK inhibitor often require blood transfusions, and more than 30% will discontinue treatment due to anemia.2 Anemia and transfusion dependence are strongly correlated with poor prognosis and shortened survival.3 1. Clarivate/DRG (2023) 2. Palandri, F., Palumbo, G.A., Elli, E.M. et al. Ruxolitinib discontinuation syndrome: incidence, risk factors, and management in 251 patients with myelofibrosis. Blood Cancer J. 11, 4 (2021). 3. Pardanani, A., & Tefferi, A. (2011). Prognostic relevance of anemia and transfusion dependency in myelodysplastic syndromes and primary myelofibrosis. Haematologica, 96(1), 8–10. About XPOVIO® (selinexor) XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor and the first of Karyopharm's Selective Inhibitor of Nuclear Export (SINE) compounds for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved in the U.S. and marketed by Karyopharm in multiple oncology indications, including: (i) in combination with VELCADE® (bortezomib) and dexamethasone (XVd) in adult patients with multiple myeloma after at least one prior therapy; (ii) in combination with dexamethasone in adult patients with heavily pre-treated multiple myeloma; and (iii) under accelerated approval in adult patients with diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. XPOVIO® (also known as NEXPOVIO® in certain countries) has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including but not limited to the European Union, the United Kingdom, Mainland China, Taiwan, Hong Kong, Australia, South Korea, Singapore, Israel, and Canada. XPOVIO®/NEXPOVIO® is marketed in these respective ex-U.S. territories by Karyopharm's partners: Antengene, Menarini, Neopharm, and FORUS. Selinexor is also being investigated in several other mid- and late-stage clinical trials across multiple high unmet need cancer indications, including in endometrial cancer and myelofibrosis. For more information about Karyopharm's products or clinical trials, please contact the Medical Information department at: Tel: +1 (888) 209-9326; Email: [email protected] XPOVIO® (selinexor) is a prescription medicine approved: In combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy (XVd). In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti‐CD38 monoclonal antibody (Xd). For the treatment of adult patients with relapsed or refractory diffuse large B‐cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). SELECT IMPORTANT SAFETY INFORMATION Warnings and Precautions Thrombocytopenia: Monitor platelet counts throughout treatment. Manage with dose interruption and/or reduction and supportive care. Neutropenia: Monitor neutrophil counts throughout treatment. Manage with dose interruption and/or reduction and granulocyte colony‐stimulating factors. Gastrointestinal Toxicity: Nausea, vomiting, diarrhea, anorexia, and weight loss may occur. Provide antiemetic prophylaxis. Manage with dose interruption and/or reduction, antiemetics, and supportive care. Hyponatremia: Monitor serum sodium levels throughout treatment. Correct for concurrent hyperglycemia and high serum paraprotein levels. Manage with dose interruption, reduction, or discontinuation, and supportive care. Serious Infection: Monitor for infection and treat promptly. Neurological Toxicity: Advise patients to refrain from driving and engaging in hazardous occupations or activities until neurological toxicity resolves. Optimize hydration status and concomitant medications to avoid dizziness or mental status changes. Embryo‐Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential and males with a female partner of reproductive potential, of the potential risk to a fetus and use of effective contraception. Cataract: Cataracts may develop or progress. Treatment of cataracts usually requires surgical removal of the cataract. Adverse Reactions The most common adverse reactions (≥20%) in patients with multiple myeloma who receive XVd are fatigue, nausea, decreased appetite, diarrhea, peripheral neuropathy, upper respiratory tract infection, decreased weight, cataract and vomiting. Grade 3‐4 laboratory abnormalities (≥10%) are thrombocytopenia, lymphopenia, hypophosphatemia, anemia, hyponatremia and neutropenia. In the BOSTON trial, fatal adverse reactions occurred in 6% of patients within 30 days of last treatment. Serious adverse reactions occurred in 52% of patients. Treatment discontinuation rate due to adverse reactions was 19%. The most common adverse reactions (≥20%) in patients with multiple myeloma who receive Xd are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%. The most common adverse reactions (incidence ≥20%) in patients with DLBCL, excluding laboratory abnormalities, are fatigue, nausea, diarrhea, appetite decrease, weight decrease, constipation, vomiting, and pyrexia. Grade 3‐4 laboratory abnormalities (≥15%) are thrombocytopenia, lymphopenia, neutropenia, anemia, and hyponatremia. In the SADAL trial, fatal adverse reactions occurred in 3.7% of patients within 30 days, and 5% of patients within 60 days of last treatment; the most frequent fatal adverse reactions was infection (4.5% of patients). Serious adverse reactions occurred in 46% of patients; the most frequent serious adverse reaction was infection (21% of patients). Discontinuation due to adverse reactions occurred in 17% of patients. Use In Specific Populations Lactation: Advise not to breastfeed. For additional product information, including full prescribing information, please visit . To report SUSPECTED ADVERSE REACTIONS, contact Karyopharm Therapeutics Inc. at 1‐888‐209‐9326 or FDA at 1‐800‐FDA‐1088 or . About Karyopharm Therapeutics Karyopharm Therapeutics Inc. (Nasdaq: KPTI) is a commercial-stage pharmaceutical company whose dedication to pioneering novel cancer therapies is fueled by a belief in the extraordinary strength and courage of patients with cancer. Since its founding, Karyopharm has been an industry leader in oral compounds that address nuclear export dysregulation, a fundamental mechanism of oncogenesis. Karyopharm's lead compound and first-in-class, oral exportin 1 (XPO1) inhibitor, XPOVIO® (selinexor), is approved in the U.S. and marketed by the Company in three oncology indications. It has also received regulatory approvals in various indications in 50 ex-U.S. territories and countries, including Europe and the United Kingdom (as NEXPOVIO®) and China. Karyopharm has a focused pipeline targeting indications in multiple high unmet need cancers, including in multiple myeloma, endometrial cancer, myelofibrosis, and diffuse large B-cell lymphoma (DLBCL). For more information about our people, science and pipeline, please visit , and follow us on LinkedIn and on X at @Karyopharm. Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the timing of reporting of top-line data from the SENTRY trial; the ability of selinexor to treat patients with multiple myeloma, endometrial cancer, myelofibrosis, diffuse large B-cell lymphoma and other diseases; and expectations with respect to the clinical development plans and potential regulatory submissions of selinexor. Such statements are subject to numerous important factors, risks and uncertainties, many of which are beyond Karyopharm's control, that may cause actual events or results to differ materially from Karyopharm's current expectations. For example, there can be no guarantee that Karyopharm will successfully commercialize XPOVIO or that any of Karyopharm's drug candidates, including selinexor, will successfully complete necessary clinical development phases or that development of any of Karyopharm's drug candidates will continue. Further, there can be no guarantee that any positive developments in the development or commercialization of Karyopharm's drug candidate portfolio will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: the adoption of XPOVIO in the commercial marketplace, the timing and costs involved in commercializing XPOVIO or any of Karyopharm's drug candidates that receive regulatory approval; the ability to obtain and retain regulatory approval of XPOVIO or any of Karyopharm's drug candidates that receive regulatory approval; Karyopharm's results of clinical trials and preclinical trials, including subsequent analysis of existing data and new data received from ongoing and future trials; the content and timing of decisions made by the U.S. Food and Drug Administration and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies, including with respect to the need for additional clinical trials; the ability of Karyopharm or its third party collaborators or successors in interest to fully perform their respective obligations under the applicable agreement and the potential future financial implications of such agreement; Karyopharm's ability to enroll patients in its clinical trials; unplanned cash requirements and expenditures; substantial doubt exists regarding Karyopharm's ability to continue as a going concern; development or regulatory approval of drug candidates by Karyopharm's competitors for products or product candidates in which Karyopharm is currently commercializing or developing; the direct or indirect impact of the COVID-19 pandemic or any future pandemic on Karyopharm's business, results of operations and financial condition; and Karyopharm's ability to obtain, maintain and enforce patent and other intellectual property protection for any of its products or product candidates. These and other risks are described under the caption "Risk Factors" in Karyopharm's Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, which was filed with the Securities and Exchange Commission (SEC) on August 11, 2025, and in other filings that Karyopharm may make with the SEC in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and, except as required by law, Karyopharm expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. XPOVIO® and NEXPOVIO® are registered trademarks of Karyopharm Therapeutics Inc. SOURCE Karyopharm Therapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

摘要：2025 年以来，瑞士制药巨头诺华的业务发展团队动作频频，在并购与授权交易上已投入 172.3 亿美元。其中，心血管领域成为重点布局方向，仅 9 月就达成三笔交易，包括以 14 亿美元收购 Tourmaline Bio。这一系列动作旨在应对多款核心药物即将失去专利保护的挑战，通过 “补强型交易” 充实管线，尤其是在心律失常等公司具有优势的治疗领域。 九月三笔交易凸显并购力度 进入 9 月，诺华的并购步伐明显加快。9 日，该公司宣布以 14 亿美元收购心血管生物科技公司 Tourmaline Bio，借此获得其针对动脉粥样硬化性心血管疾病（ASCVD）的 IL-6 靶向单克隆抗体 pacibekitug。据 BMO Capital Markets 分析，诺华为此支付了 37% 的溢价，凸显出该药物的潜力，它将与诺和诺德通过收购 Corvidia Therapeutics 获得的 III 期疗法 ziltivekimab 展开竞争。 而在 9 月初，诺华还与 Argo Biopharmaceutical、Arrowhead Pharma 达成两项授权交易，总潜在价值达 75.6 亿美元。其中，与 Argo 的合作涉及多款针对心血管靶点的 siRNA 资产，交易总价值 53.6 亿美元，包括 1.6 亿美元预付款和 52 亿美元里程碑付款；与 Arrowhead Pharma 的交易则聚焦于针对突触核蛋白病的 siRNA 疗法 ARO-SNCA，总价值 22 亿美元，包含 2 亿美元预付款和 20 亿美元里程碑付款。巨额投入背后的管线焦虑 诺华如此密集且大额的并购投入，与公司面临的专利悬崖压力密切相关。今年，其慢性心力衰竭药物 Entresto 将失去专利保护，此外，Xolair 今年也将遭遇仿制药竞争，Jakafi、Cosentyx 和 Tafinlar 等也将在 2030 年底前陆续失去专利 exclusivity。 为应对这一局面，充实管线成为诺华的重要战略。在 7 月的二季度财报电话会议上，首席财务官 Harry Kirsch 就表示，公司正在寻求 “创造价值的补强型交易”。他还提到，由首席战略增长官 Ronny Gal 领导的业务发展团队在过去两年已签署 30 笔交易，今年 4 月与 Regulus Therapeutics 达成的 17 亿美元交易便是典型案例，该交易为诺华的寡核苷酸产品线增添了常染色体显性多囊肾病（ADPKD）疗法 farabursen。聚焦优势领域，剑指大市场 在布局方向上，诺华将目光投向了自身具有独特优势的领域。公司高管曾在 2024 年底表示，希望到 2025 年底在心律失常领域拥有多个临床阶段资产，首席执行官 Vas Narasimhan 在财报电话会议上重申了这一目标的重要性。 为此，诺华已签署多项临床前资产的授权交易，包括 6 月与 Flagship Pioneering 旗下 ProFound Therapeutics 的合作。“心律失常领域是我们拥有独特专长的领域，可能也是少数仍在致力于解决心脏节律问题的公司之一。”Narasimhan 向分析师表示，“显然，若能成功，这里的市场规模相当大，对于患者而言，药物治疗相较于基于设备的心脏复律具有显著吸引力。” 从目前的交易来看，心血管领域已成为诺华 2025 年的布局重点，年内已达成四笔相关交易。业内人士认为，随着并购的持续推进，诺华有望在应对专利悬崖的同时，为未来的增长奠定基础。 参考来源：https://www.biospace.com/business/novartis-busy-bd-team-keeps-2025-bolt-on-deal-promises 识别微信二维码，添加生物制品圈小编，符合条件者即可加入 生物制品微信群！ 请注明：姓名+研究方向！ 版 权 声 明 本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观不本站。