Karyopharm Therapeutics Provides Endometrial Cancer Program Update
Karyopharm Therapeutics Provides Endometrial Cancer Program Update
NEWTON, Mass., Dec. 3, 2024 /PRNewswire/ -- Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, today announced that it is in discussions and has an upcoming meeting with the U.S. Food and Drug Administration (FDA) regarding the evolving treatment landscape in endometrial cancer and any implications this may have with respect to the Company's Phase 3 XPORT-EC-042 trial. In light of this, Karyopharm does not plan to discuss its endometrial cancer program during the Piper Sandler 36th Annual Healthcare Conference being held in New York, NY today. The Company intends to provide an update on its endometrial cancer program as soon as practical following the discussion with FDA.
馬薩諸塞州牛頓，2024年12月3日/美通社/ - karyopharm therapeutics公司（納斯達克股票代碼：KPTI）一家開拓新型癌症療法的處於商業階段的藥品公司，今日宣佈，就子宮內膜癌治療領域的發展格局以及其對公司第3期XPORt-EC-042試驗可能產生的任何影響，與美國食品和藥物管理局（FDA）進行討論，並將舉行即將舉行的會議。鑑於此，karyopharm沒有計劃在今天在紐約舉行的派傑投資第36屆年度醫療保健大會上討論其子宮內膜癌項目。公司打算在與FDA討論後儘快更新其子宮內膜癌項目。
A live webcast of the fireside chat at the Piper Sandler 36th Annual Healthcare Conference at 11:30 a.m. ET today can be accessed under "Events & Presentations" in the Investor section of the Company's website, , and will be available for replay following the event.
您可以在公司網站的投資者部分下的「活動和演示文稿」中查看今天美國東部時間上午11:30派傑投資第36屆年度醫療保健大會的現場網絡研討會，並在活動結束後進行重播。
About the EC-042 Study
EC-042 (XPORT-EC-042; NCT05611931) is a global, Phase 3, randomized, double-blind study evaluating selinexor as a maintenance therapy following systemic therapy in patients with TP53 wild-type advanced or recurrent endometrial cancer. The EC-042 study was initiated in November 2022 and is expected to enroll up to 220 patients who will be randomized 1:1 to receive either a 60 mg, once-weekly, administration of oral selinexor or placebo until disease progression. The primary endpoint of the study is progression free survival, as assessed by an investigator, with overall survival as a key secondary endpoint. Further, in connection with the EC-042 Study, Karyopharm entered into a global collaboration with Foundation Medicine, Inc. to develop FoundationOneCDx, a tissue-based comprehensive genomic profiling test to identify and enroll patients whose tumors are TP53 wild-type.
關於EC-042研究
EC-042（XPORt-EC-042; NCT05611931）是一項全球性、第3期、隨機、雙盲研究，評估塞利內索作爲一種維持治療，在已接受系統治療的TP53野生型晚期或複發性子宮內膜癌患者中的應用。EC-042研究於2022年11月啓動，預計將招募多達220名患者，這些患者將被隨機分配1:1，接受口服塞利內索60毫克每週一次或安慰劑，直至疾病進展。該研究的主要終點是由研究員評估的無進展生存，總體生存是關鍵的次要終點。此外，與EC-042研究相關，karyopharm與Foundation Medicine, Inc.達成全球合作，開發FoundationOneCDx，這是一種基於組織的全面基因組分析檢測，用於識別和招募腫瘤爲TP53野生型的患者。
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 patients with multiple myeloma after at least one prior therapy; (ii) in combination with dexamethasone in patients with heavily pre-treated multiple myeloma; and (iii) under accelerated approval in 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, China, 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.
關於XPOVIO（Selinexor）
XPOVIO是karyopharm首款核蛋白轉運體1（XPO1）抑制劑，也是治療癌症的karyopharm選擇性核蛋白輸出（SINE）化合物中的第一種。XPOVIO通過選擇性結合並抑制核蛋白轉運蛋白XPO1發揮作用。XPOVIO已在美國獲得批准，並由karyopharm在多種腫瘤適應症中銷售，包括：（i）與VELCADE（硼替佐米）和地塞米松（XVd）聯合應用，用於至少經歷一次前期治療的多發性骨髓瘤患者；（ii）與地塞米松聯合應用，用於經過多次治療的多發性骨髓瘤患者；以及（iii）在至少接受兩線系統治療的瀰漫大B細胞淋巴瘤患者中根據加速批准。XPOVIO（在某些國家也被稱爲NEXPOVIO）已在越來越多的歐美區域和國家得到各種適應症的監管批准，其中包括但不限於歐洲聯盟、英國、中國、澳大利亞、韓國、新加坡、以色列和加拿大。在這些相應的歐美區域，XPOVIO/NEXPOVIO由karyopharm的合作伙伴Antengene、Menarini、Neopharm和FORUS銷售。Selinexor也正被用於多個其他中晚期臨床試驗中，跨多個高需求癌症適應症，包括子宮內膜癌和骨髓纖維化的研究。
For more information about Karyopharm's products or clinical trials, please contact the Medical Information department at: Tel: +1 (888) 209-9326; Email: medicalinformation@karyopharm.com
如需了解更多關於Karyopharm產品或臨床試驗的信息，請聯繫醫療信息部門：電話：+1（888）209-9326；郵箱：medicalinformation@karyopharm.com
XPOVIO (selinexor) is a prescription medicine approved:
XPOVIO（selinexor）是一種經過處方批准的藥物：
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).
與硼替佐米和地塞米松聯合用於接受過至少一次治療的成年多發性骨髓瘤患者的治療（XVd）。
與地塞米松聯合用於至少接受過四種治療方案且疾病對至少兩種蛋白酶抑制劑、至少兩種免疫調節劑和一種抗CD38單克隆抗體具有耐藥性的成年患者的治療（Xd）。
針對成年患者治療復發或難治性瀰漫大B細胞淋巴瘤（DLBCL），否則未指明，包括來源於濾泡淋巴瘤的DLBCL，在至少兩種系統治療方案後。該適應症獲得加速批准，基於響應率。該適應症的持續批准可能取決於驗證和描述確認試驗中的臨床益處。
SELECT IMPORTANT SAFETY INFORMATION
重要的安全信息選擇性
Warnings and Precautions
警告和預防措施
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.
血小板減少症：在整個治療過程中監測血小板計數。通過劑量中斷和/或減少以及支持性護理進行管理。
中性粒細胞減少：在治療過程中監測中性粒細胞計數。可通過暫停劑量和/或減少劑量以及粒細胞集落刺激因子進行管理。
胃腸毒性：可能出現噁心、嘔吐、腹瀉、食慾減退和體重減輕。提供抗噁心預防。可通過暫停劑量和/或減少劑量、抗噁心藥物和支持護理進行管理。
低鈉血癥：在治療過程中監測血清鈉水平。糾正合並的高血糖和高血清漿蛋白水平。可通過暫停劑量、減少劑量或停藥以及支持護理進行管理。
嚴重感染：監測感染情況並及時治療。
神經毒性：建議患者避免駕駛和從事危險職業或活動，直到神經毒性消退。優化水合狀態和同時使用的藥物以避免頭暈或精神狀態改變。
胚胎-胚胎毒性：可能對胎兒造成傷害。告知生育潛能的女性及具有生育潛能的男性（有生育潛能的女性的伴侶），胎兒可能面臨的潛在風險以及使用有效避孕措施。
白內障：可能發展或進展白內障。白內障的治療通常需要手術切除白內障。
Adverse Reactions
副作用
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.
接受XVd治療的多發性骨髓瘤患者中，最常見的不良反應（≥20%）包括疲勞、噁心、食慾減退、腹瀉、周圍神經病變、上呼吸道感染、體重減輕、白內障和嘔吐。實驗室等級3-4異常反應（≥10%）包括血小板減少、淋巴細胞減少、低磷血癥、貧血、低鈉血癥和中性粒細胞減少。在波士頓試驗中，有6%的患者在最後一次治療後30天內發生了致命不良反應。52%的患者出現了嚴重不良反應。因不良反應中斷治療的比例爲19%。
接受Xd治療的多發性骨髓瘤患者中，最常見的不良反應（≥20%）包括血小板減少、疲勞、噁心、貧血、食慾減退、體重減輕、腹瀉、嘔吐、低鈉血癥、中性粒細胞減少、白細胞減少、便秘、呼吸困難和上呼吸道感染。在STORm試驗中，有9%的患者發生了致命的不良反應。有58%的患者出現了嚴重不良反應。因不良反應導致停止治療的比率爲27%。
患有瀰漫大B細胞淋巴瘤的患者中，最常見的不良反應（發病率≥20%）不包括實驗室異常，包括疲勞、噁心、腹瀉、食慾減退、體重減輕、便秘、嘔吐和發熱。實驗室等級3-4異常反應（≥15%）包括血小板減少、淋巴細胞減少、中性粒細胞減少、貧血和低鈉血癥。在SADAL試驗中，有3.7%的患者在30天內發生致命不良反應，5%的患者在最後一次治療後60天內發生; 最常見的致命不良反應是感染（患者的4.5%）。46%的患者出現嚴重不良反應; 最常見的嚴重不良反應是感染（患者的21%）。因不良反應中斷治療的比例爲17%。
Use In Specific Populations
Lactation: Advise not to breastfeed.
在特定人群中的使用
母乳餵養：建議停止母乳餵養。
For additional product information, including full prescribing information, please visit .
如需額外產品信息，包括完整的處方信息，請訪問。
To report SUSPECTED ADVERSE REACTIONS, contact Karyopharm Therapeutics Inc. at 1‐888‐209‐9326 or FDA at 1‐800‐FDA‐1088 or .
如有疑似不良反應，請聯繫Karyopharm Therapeutics Inc.，電話1-888-209-9326或FDA電話1-800-FDA-1088或。
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 a growing number of 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.
關於Karyopharm Therapeutics
karyopharm therapeutics公司（納斯達克代碼：KPTI）是一家商業化藥品公司，其開發新型癌症療法的奉獻源自對癌症患者非凡的力量和勇氣的信念。自成立以來，karyopharm一直是口服藥物領域的行業領先者，致力於解決核出口調控這一癌症生成的基本機制。karyopharm的首席化合物和口服第一類物質，選擇性核出口蛋白1（XPO1）抑制劑XPOVIO（Selinexor）已在美國獲得批准，並由公司在三種腫瘤學適應症中進行市場推廣。它還在越來越多的美國領土和國家（包括歐洲和英國（作爲NEXPOVIO）以及中國）的各種適應症中獲得了監管批准。karyopharm的專注管線針對多種存在高需求的癌症適應症，包括多發性骨髓瘤，子宮內膜癌，骨髓纖維化和彌散性大B細胞淋巴瘤（DLBCL）。有關我們的團隊，科學和項目管線的更多信息，請訪問，並在領英上關注我們，在X上關注@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 Karyopharm's clinical development plans and potential regulatory submissions of selinexor and the ability of selinexor to treat patients with multiple myeloma, endometrial cancer, myelofibrosis, diffuse large B-cell lymphoma, and other diseases. 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 September 30, 2024, which was filed with the Securities and Exchange Commission (SEC) on November 5, 2024, 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.
前瞻性聲明
本新聞稿包含根據1995年《私人證券訴訟改革法》中的前瞻性陳述。這些前瞻性陳述包括Karyopharm的臨床開發計劃以及selinexor的潛在監管提交，以及selinexor治療多發性骨髓瘤、子宮內膜癌、骨髓纖維化、瀰漫大B細胞淋巴瘤和其他疾病的能力。這些聲明受許多重要因素、風險和不確定性的影響，其中許多超出Karyopharm的控制，可能導致實際事件或結果與Karyopharm當前期望大相徑庭。例如，Karyopharm無法保證成功將XPOVIO商業化，也無法保證Karyopharm的任何藥物候選成功完成必要的臨床開發階段，或Karyopharm的任何藥物候選的開發將繼續。此外，無法保證Karyopharm的任何藥物候選組合的開發或商業化中出現的任何積極進展將導致股價上漲。管理層的期望，因此，本新聞稿中的任何前瞻性陳述也可能受到與許多其他因素相關的風險和不確定性的影響。包括以下因素：XPOVIO在商業市場上的接受程度、商業化XPOVIO或獲得監管批准的Karyopharm的任何藥物候選所涉及的時間和成本；獲得和保留XPOVIO或獲得監管批准的Karyopharm的任何藥物候選的監管批准的能力；Karyopharm在臨床試驗和臨床前試驗中的結果，包括對正在進行和將來試驗中收到的現有數據和新數據的後續分析；由美國食品和藥物管理局和其他監管機構、臨床試驗基地的調查審查委員會以及出版審查機構作出的決定的內容和時間，包括關於需要進行額外臨床試驗的事項；Karyopharm或其第三方合作方或利益受讓人根據適用協議履行各自義務的能力，以及此類協議可能帶來的潛在未來財務影響；Karyopharm招募臨床試驗患者的能力；未經計劃的現金需求和支出；存在Karyopharm能否繼續作爲持續經營實體的重大疑慮；Karyopharm競爭對手開發或監管批准的產品或產品候選品對Karyopharm目前正在商業化或開發的產品產生的直接或間接影響；COVID-19大流行或任何未來大流行對Karyopharm業務、運營結果和財務狀況的直接或間接影響；以及Karyopharm取得、保留和實施對其任何產品或產品候選的專利和其他知識產權保護的能力。這些和其他風險在Karyopharm於2024年9月30日結束的季度報告第10-Q的題注「風險因素」下有描述，該報告於2024年11月5日向美國證券交易委員會（SEC）提交，以及Karyopharm將來可能向SEC提交的其他文件中有描述。本新聞稿中包含的任何前瞻性陳述僅截至本日期，Karyopharm明確免除根據法律要求更新任何前瞻性陳述的任何義務，無論是基於新信息、未來事件還是其他原因。
XPOVIO and NEXPOVIO are registered trademarks of Karyopharm Therapeutics Inc.
XPOVIO和NEXPOVIO是Karyopharm Therapeutics Inc.的註冊商標。
SOURCE Karyopharm Therapeutics Inc.
消息來源：Karyopharm Therapeutics Inc.
譯文內容由第三人軟體翻譯。
