Pralsetinib

这样的标的上哪找去？作者：西北哽阿斯利康的王牌药物奥希替尼，最近有点头大。先是3月9日，中国企业同源康医药发布了II期头对头试验结果：自己研发的艾多替尼对比奥希替尼，在治疗EGFR脑转移时显示出更大的临床收益；3月21日，2025欧洲肺癌大会上，强生又拿出了埃万妥单抗联合拉泽替尼头对头奥希替尼的研究成果。强生直击的是奥希替尼的命门：一线治疗EGFR 19外显子缺失和21外显子L858R突变。奥希替尼正是在2018年时拿下这个适应症的一线治疗，才踏上的封神之路。如今，强生要抄阿斯利康的后路。可是螳螂捕蝉，黄雀在后。强生埃万妥单抗联合疗法猛攻奥希替尼的同时，自家大本营也在遭遇攻击。今年1月，迪哲医药的舒沃替尼向FDA申请美国上市获受理，瞄准的是埃万妥单抗的EGFR 20外显子插入突变（exon20ins）适应症。迪哲虽然晚来一步，但小碎步一直没停，去年4月，在埃万妥单抗一线已经获批的前提下，舒沃替尼一线治疗FDA认定为突破性疗法，今年进入NDA，▌摆明了就是要抄埃万妥单抗的后路要是能在今年登陆美国市场，舒沃替尼将作为全球小分子肺癌靶向药的代表，创造新的销售纪录。1覆盖肺癌治疗的空白点全球范围内，EGFR exon20ins这个肺癌难治靶点目前全球就只有两种对症药物获批：强生的埃万妥单抗和迪哲的舒沃替尼。强生在全球药企营收冠军的位子上已经默默坐了好几年，但两个“百亿美元俱乐部”品种：达雷妥尤单抗和乌司奴单抗都面临专利期将至的问题。强生为此设定了一个“5年25款重磅药物”的计划，埃万妥单抗就是25款药之一，强生内部给出的预测是峰值销售额▌50亿美元强生是这么算的：奥希替尼去年销售额66亿美元，埃万妥单抗如果头对头获胜，市场空间理应超过奥希替尼。阿斯利康运气好，独霸三代EGFR市场10年，强生就没这么好运。迪哲在身后紧紧咬住，而且已经组织起了包围圈。在20外显子插入突变的适应症上，当年埃万妥单抗获批二线疗法时，依据的是I期CHRYSALIS研究：单药治疗随访9.7个月客观缓解率40%，中位缓解时间11.1个月；舒沃替尼获批的二线疗法依据的是WU-KONG6研究，入组患者里脑转移的就有32%，不少还是用过埃万妥单抗后进展的患者。即便如此，舒沃替尼随访7.6个月整体ORR达到61%，。一线治疗情况也是类似。埃万妥单抗的一线联用方案，中位PFS为11.4个月，客观缓解率为73%；而舒沃替尼正在申报的一线单药方案，客观缓解率78.6%，中位PFS是12.4个月。（摘自2023年ESMO会议）可以看出，舒沃替尼在20外显子插入突变这个适应症上，一直压着埃万妥单抗一头，▌就跟田忌赛马一样但是，迪哲的布局远不止这些。今年ELCC大会上，舒沃替尼披露了一项联合VEGFR的研究，针对EGFR-TKI耐药的晚期非小细胞肺癌患者。这是奥希替尼都未曾触碰到的领域；另外，戈利昔替尼也提交了一份联用PD-1治疗PD-1耐药的研究。今年6月ASCO上要公布的新一代EGFR抑制剂DZD6008，同样针对三代EGFR TKI等多线治疗失败伴脑转移的EGFR突变晚期肺癌。迪哲的管线布局，基本覆盖了现有主流肺癌药物的治疗空白，比几个行业巨头谋划的都要深远。肺癌治疗在医药巨头们的努力之下，战线已拉得很长。以上海为例，最新公布的肺癌五年生存率达到61.6%，越来越多的患者会进入后线治疗，迪哲的布局就准备要托住这些需求。如今我们看到的迪哲，只是宏大叙事的很小一部分。2锋芒刚刚展现4月29日晚，迪哲医药公布2024年年报，去年迪哲营业收入3.6亿元。因为戈利昔替尼是去年6月底上市，迪哲去年的营收大部分应当来自舒沃替尼。近几年上市的肺癌罕见靶点药物不少。2021年6月，和黄医药的MET抑制剂赛沃替尼获批上市，商业化由阿斯利康负责。赛沃替尼针对的MET14外显子跳跃突变，在非小细胞肺癌中的占比与exon20ins相当。从已公布的数据看，舒沃替尼2024年上半年营收2.04亿元，已经超过赛沃替尼上市第一个完整半年销售额的约1.7亿元。另一罕见靶点RET抑制剂普拉替尼也是类似，RET的突变概率只有2%。2024年第一个完整销售年度，基石药业披露：普拉替尼和另一款药物加一起，销售额1.75亿元。同样是罕见靶点肺癌药物，舒沃替尼的起步速度比几家同行要快得多。（数据来源：迪哲医药年报）财报的表现中，还有相当部分来自FIC药物戈利昔替尼。戈利昔替尼基本是目前治疗外周T细胞淋巴瘤的最佳药物，今年4月，戈利昔替尼获得CSCO最高的I级推荐，用于治疗复发难治性外周T细胞淋巴瘤；而且CSCO还推荐戈利昔替尼单药治疗复发、难治NK/T细胞淋巴瘤患者。市面上治疗外周T细胞淋巴瘤的药物，目前以微芯生物的西达苯胺为主。2024年西达苯胺销售额5亿元，已多年近乎原地踏步。相比之下，戈利昔替尼以“强效抑瘤、抗炎、免疫调节”三重机制突破了PTCL治疗瓶颈，未来在维持治疗上有很大探索余地，市场前景也会大得多。目前，基本无法用西达苯胺来估算戈利昔替尼的上限，更不要提未来戈利昔替尼肺癌上的拓展了。去年，《Science》重磅新发现，JAK抑制剂联合免疫检查点抑制剂，能够逆转T细胞耗竭，增加抗肿瘤T细胞和NK细胞数量，显著增强PD-1抑制剂治疗肺癌和淋巴瘤的效果。戈利昔替尼可能还会有更大的空间。3商业化造血能力首屈一指迪哲和年报同一天发布了2025年一季报：今年一季度迪哲营业收入同比接近翻倍。戈利昔替尼和舒沃替尼是今年1月开始走医保报销的，一季报应该已经反映出了医保的放量效应。最近两年医保谈判规则更加柔性化，而且各地对创新药在医院的“小准入”上都开出了具体要求，这使得医保的含金量越来越高。从迪哲销售费用率的变化就能看出医保的价值：2024年还是123%，到今年一季度直降到77%。（迪哲2024年主要财务指标，摘自公司微信公众号）产品销售就如同巨轮引擎一样，一旦启动就能快速带动财务数据的整体改善。今年一季度，迪哲存货周转天数、经营性现金流等考察营运能力的关键指标都有正向改善。从去年到今年，迪哲逐季度的亏损幅度一直在收窄。到目前为止，对2025年迪哲的所有预估都还没有考虑BD的可能性。这会是迪哲▌今年的最大彩蛋按照迪哲的说法：公司会从管线协同性、全球商业化能力、发展潜力、现金流等各维度进行评估，寻求最优方案。迪哲系出名门，研发团队主体立项、经历过奥希替尼的研发过程，对肺癌治疗药物是非常熟悉的。如今，迪哲猛攻肺癌领域，挑战“肺癌治疗王者”奥希替尼，从某个意义上说，也是超越自己的过去。舒沃替尼站稳一线治疗后，必然会向更多适应症开拓；戈利昔替尼已经在开展联用治疗肺癌的研究。血液肿瘤上，迪哲全球首创的非共价双靶点BTK 药物DZD8586今年6月将在ASCO上以口头报告形式公布针对既往接受过共价或非共价BTK抑制剂和BTK降解剂治疗的CLL/SLL最新数据。过去两年，迪哲研发费用整体维持在7-8亿元的水平，已经度过了投入最大的阶段。今年4月17日，迪哲成功定增近18亿元，泰康资产、朱雀基金、鹏华基金等看好加码，定增完成后，迪哲账面现金将充实到20亿元以上。机构愿意给迪哲投钱，是有原因的：迪哲2017年成立，2023年自有产品就实现了商业化，相比其他Biotech来说纯投入期很短，回血能力超强。迪哲未来还会开辟更多国际化研发项目，未来商业回报空间更大。这样好的标的，如今市面上到哪里去找？ 喜欢我们文章的朋友点个“在看”和“赞”吧，不然微信推送规则改变，有可能每天都会错过我们哦~免责声明“汇聚南药”公众号所转载文章来源于其他公众号平台，主要目的在于分享行业相关知识，传递当前最新资讯。图片、文章版权均属于原作者所有，如有侵权，请在留言栏及时告知，我们会在24小时内删除相关信息。信息来源：拇指药略往期推荐本平台不对转载文章的观点负责，文章所包含内容的准确性、可靠性或完整性提供任何明示暗示的保证。

First quarter 2025 total revenue of approximately $53.3 million, which includes net product sales of $43.6 million and contract revenues from collaborations of $9.8 million Generated $11.4 million of net income in the first quarter of 2025 R289 granted Orphan Drug designation for the treatment of MDS and Fast Track designation for the treatment of previously-treated transfusion dependent lower-risk MDS by the FDA 2025 Outlook: Total revenue of approximately $200 to $210 million Conference call and webcast scheduled today at 4:30 p.m. Eastern Time SOUTH SAN FRANCISCO, Calif., May 6, 2025 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, today reported financial results for the first quarter ended March 31, 2025, including sales of TAVALISSE® (fostamatinib disodium hexahydrate), GAVRETO® (pralsetinib) and REZLIDHIA® (olutasidenib), and recent business progress. "Our first quarter results reflect the continued strength of our growing commercial business. This robust year-over-year revenue growth coupled with our continued financial discipline enabled us to generate more than $11 million in net income this quarter. These results uniquely position us to invest in our pipeline, including our ongoing Phase 1b clinical study evaluating R289 in patients with relapsed or refractory lower-risk MDS," said Raul Rodriguez, Rigel's president and CEO. "With a strong start to the year, we are focused on continuing our commercial growth, and building and advancing our development pipeline, including sharing data from the dose escalation portion of our R289 study later this year." First Quarter 2025 Business Update Commercial Net product sales of $43.6 million, an increase of 68% from the same period of 2024. Year-over-year commercial strength was driven by the expansion of the commercial portfolio, including the successful integration of GAVRETO. Rigel's partner Kissei Pharmaceutical Co., Ltd. (Kissei) announced in January that the Korean Ministry of Food and Drug Safety approved TAVALISSE for the treatment of thrombocytopenia in adult patients with chronic idiopathic thrombocytopenic purpura who have had an insufficient response to a previous treatment. In the first quarter, Rigel recognized $3.0 million in regulatory milestone revenue in connection with this approval. Clinical Development R2891, a novel and selective dual interleukin receptor-associated kinases 1 and 4 (IRAK1/4) inhibitor, was granted Orphan Drug designation for the treatment of myelodysplastic syndromes by the U.S. Food and Drug Administration (FDA) in January. R289 was previously granted Fast Track designation for the treatment of previously-treated transfusion dependent lower-risk myelodysplastic syndrome (MDS) by the FDA. Rigel continues to advance its Phase 1b clinical study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of R289 in patients with relapsed or refractory (R/R) lower-risk MDS. Enrollment in the sixth dose level (500 mg twice daily) is ongoing. Corporate Dr. Mark Frohlich joined Rigel's Board of Directors as an independent director and member of the Board of Director's Corporate Governance, Health Care Compliance Oversight and Nominating Committee, and the Scientific and Clinical Trial Advisory Committee, effective March 6, 2025. In March, Rigel announced it entered into a settlement agreement with Annora Pharma Private Ltd., Hetero Labs Ltd., and Hetero USA, Inc. (collectively "Annora") resolving patent litigation related to TAVALISSE. The litigation resulted from submission by Annora of an Abbreviated New Drug Application to the FDA seeking approval to market a generic version of TAVALISSE in the United States. Under the terms of the settlement agreement, Annora will have a license to sell its generic product in Q2 2032 or earlier under certain circumstances. In accordance with the agreement, the parties terminated all ongoing litigation between Rigel and Annora regarding TAVALISSE patents pending in New Jersey. In late April, Rigel notified Eli Lilly and Company (Lilly) that it will not exercise its opt-in right related to the development and commercialization of ocadusertib (previously R552) for the treatment of non-central nervous system (CNS) diseases. As a result of this notification, in the second quarter of 2025, Rigel expects to recognize approximately $40.0 million in non-cash revenue resulting from the release of the remaining cost share liability currently on the balance sheet. Per the agreement with Lilly, Rigel will continue to be entitled to receive milestone and tiered royalty payments on future net sales of ocadusertib and its CNS penetrant program. First Quarter 2025 Financial Update For the first quarter ended March 31, 2025, total revenues were $53.3 million, consisting of $43.6 million in net product sales and $9.8 million in contract revenues from collaborations. Net product sales grew 68% compared to $26.0 million in the same period of 2024. TAVALISSE net product sales were $28.5 million, growth of 35% compared to $21.1 million in the same period of 2024. GAVRETO net product sales were $9.0 million. GAVRETO became commercially available from Rigel in June 2024. REZLIDHIA net product sales were $6.1 million, growth of 25% compared to $4.9 million in the same period of 2024. Contract revenues from collaborations primarily consisted of $4.7 million of revenue from Grifols S.A. related to delivery of drug supplies and earned royalties, $4.6 million of revenue from Kissei related to the milestone payment and delivery of drug supplies and $0.4 million of revenue from Medison Pharma related to delivery of drug supplies and earned royalties. Total costs and expenses were $40.6 million compared to $36.5 million for the same period of 2024. The increase in costs and expenses was mainly due to increased personnel-related costs, and higher research and development costs driven by timing of clinical activities related to R289 and olutasidenib. In addition, cost of product sales increased, driven by increased product sales, higher royalties and amortization of intangible assets. These increases were partially offset by decreased stock-based compensation expenses. Rigel reported net income of $11.4 million, or $0.64 basic and $0.63 diluted per share, compared to a net loss of $8.2 million, or $0.47 basic and diluted per share, for the same period of 2024. The basic and diluted share and per share amounts for the prior period have been restated to reflect the 1-for-10 reverse stock split effected on June 27, 2024 on a retroactive basis. Cash, cash equivalents and short-term investments as of March 31, 2025 was $77.1 million, compared to $77.3 million as of December 31, 2024. 2025 Outlook Rigel continues to anticipate 2025 total revenue of approximately $200 to $210 million, which includes: Net product sales of approximately $185 to $192 million. Contract revenues from collaborations of approximately $15 to $18 million. The revenue ranges above exclude approximately $40.0 million in non-cash revenue that Rigel expects to recognize in the second quarter of 2025 related to the release of the remaining cost share liability from Rigel's collaboration with Lilly for the development and commercialization of ocadusertib. The company anticipates it will report positive net income for the full year 2025, while funding existing and new clinical development programs. Conference Call and Webcast with Slides Today at 4:30pm Eastern Time Rigel will hold a live conference call and webcast today at 4:30pm Eastern Time (1:30pm Pacific Time). Participants can access the live conference call by dialing (877) 407-3088 (domestic) or (201) 389-0927 (international). The conference call will also be webcast live and can be accessed from the Investor Relations section of the company's website at . The webcast will be archived and available for replay after the call via the Rigel website. About ITP In patients with immune thrombocytopenia (ITP), the immune system attacks and destroys the body's own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. Patients suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs), and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP. About NSCLC It is estimated that over 226,000 adults in the U.S. will be diagnosed with lung cancer in 2025. Lung cancer is the leading cause of cancer death in the U.S, with non-small cell lung cancer (NSCLC) being the most common type accounting for 85-90% of all lung cancer diagnoses.2 RET fusions are implicated in approximately 1-2% of patients with NSCLC.3 About AML Acute myeloid leukemia (AML) is a rapidly progressing cancer of the blood and bone marrow that affects myeloid cells, which normally develop into various types of mature blood cells. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that there will be about 22,010 new cases in the United States, most in adults, in 2025.4 Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.5,6 Refractory AML, which affects between 10 and 40 percent of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.7 Quality of life declines for patients with each successive line of treatment for AML, and well-tolerated treatments in relapsed or refractory disease remain an unmet need. About TAVALISSE® TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. Please click here for Important Safety Information and Full Prescribing Information for TAVALISSE. About GAVRETO® GAVRETO is indicated for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test and adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).* *Thyroid indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). Please click here for Important Safety Information and Full Prescribing Information for GAVRETO. About REZLIDHIA® REZLIDHIA is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test. Please click here for Important Safety Information and Full Prescribing Information, including Boxed WARNING, for REZLIDHIA. To report side effects of prescription drugs to the FDA, or call 1-800-FDA-1088 (800-332-1088). TAVALISSE, GAVRETO and REZLIDHIA are registered trademarks of Rigel Pharmaceuticals, Inc. About Rigel Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) is a biotechnology company dedicated to discovering, developing and providing novel therapies that significantly improve the lives of patients with hematologic disorders and cancer. Founded in 1996, Rigel is based in South San Francisco, California. For more information on Rigel, the Company's marketed products and pipeline of potential products, visit . R289 is an investigational compound not approved by the FDA. The American Cancer Society. Key Statistics for Lung Cancer. Revised January 16, 2025. Accessed March 31, 2025: Kato, S. et al. RET Aberrations in Diverse Cancers: Next-Generation Sequencing of 4,871 Patients. Clin Cancer Res. 2017;23(8):1988-1997 doi: 10.1158/1078-0432.CCR-16-1679 The American Cancer Society. Key Statistics for Acute Myeloid Leukemia (AML). Revised March 4, 2025. Accessed March 31, 2025: Patel, A, et al. Outcomes of Patients With Acute Myeloid Leukemia Who Relapse After 5 Years of Complete Remission. 2021 Sep 7;28(7):811-814. doi: Thol F, Ganser, A. Treatment of Relapsed Acute Myeloid Leukemia. Curr. Treat. Options on Oncol. (2020) 21: 66. doi: Thol F, Schlenk RF, Heuser M, Ganser A. How I treat refractory and early relapsed acute myeloid leukemia. Blood (2015) 126 (3): 319-27. doi: Forward Looking Statements This press release contains forward-looking statements relating to, among other things, expected commercial and financial results, projected financial performance and outlook for 2025, expectations for growing our commercial business, potential investment in our pipeline, results of the dose escalation portion of our R289 study, non-cash revenue recognition relating to our agreement with Lilly, continued ability for developing and commercializing TAVALISSE, GAVRETO, and REZLIDHIA domestically and in certain international markets, and expectations for Rigel's partnering and collaboration efforts. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Forward-looking statements can be identified by words such as "anticipates", "plan", "outlook", "potential", "may", "look to", "expects", "will", "initial", "promising", and similar expressions in reference to future periods. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Rigel's current beliefs, expectations, and assumptions and hence they inherently involve significant risks, uncertainties and changes in circumstances that are difficult to predict and many of which are outside of our control. Therefore, you should not rely on any of these forward-looking statements. Actual results and the timing of events could differ materially from those anticipated in such forward looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with the commercialization and marketing of fostamatinib, olutasidenib and pralsetinib; risks that the FDA, European Medicines Agency, PMDA or other regulatory authorities may make adverse decisions regarding fostamatinib, pralsetinib or olutasidenib; risks that clinical trials may not be predictive of real-world results or of results in subsequent clinical trials; risks that fostamatinib, pralsetinib or olutasidenib may have unintended side effects, adverse reactions or incidents of misuses; the availability of resources to develop Rigel's product candidates; market competition; as well as other risks detailed from time to time in Rigel's reports filed with the Securities and Exchange Commission, including its Annual Report on Form 10-K for the year ended December 31, 2024 and subsequent filings. Any forward-looking statement made by us in this press release is based only on information currently available to us and speaks only as of the date on which it is made. Rigel does not undertake any obligation to update forward-looking statements, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise, and expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein, except as required by law. Contact for Investors & Media: Investors: Rigel Pharmaceuticals, Inc. 650.624.1232 [email protected] Media: David Rosen Argot Partners 646.461.6387 [email protected] SOURCE Rigel Pharmaceuticals, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED