Defactinib

LGSOC Awareness Day 2025: A breakthrough FDA approval proves rare cancers deserve focus, funding, and a future of hope. This FDA approval proves what we’ve always believed: when rare cancers get the focus they deserve, breakthroughs follow.” — Emily Campbell MIAMI, FL, UNITED STATES, September 9, 2025 / EINPresswire.com / -- September 9 marks the second annual Low-Grade Serous Ovarian Cancer (LGSOC) Awareness Day. But 2025 isn't just another awareness campaign. It’s the year a community of advocates came together to make change. Low-grade serous ovarian cancer affects 6,000 to 8,000 women in the U.S. and 80,000 worldwide every year. LGSOC strikes younger women with a median age of 45 versus 63 for high-grade ovarian cancer, yet it has historically received a fraction of research attention and funding. The research tells a stark story: chemotherapy for LGSOC has low success rates — only 4% of patients responded when treated before surgery and 25% when treated after surgery. For every 100 people diagnosed, more than 80 will have their cancer come back. Yet until this year, there were zero FDA-approved treatments specifically designed for LGSOC. At Not These Ovaries , we see these numbers as more than statistics. They represent thousands of women who deserved better, faster answers. The moment that changed everything Last May, medical history was made. The FDA granted accelerated approval to avutometinib and defactinib (Avmapki Fakzynja Co-Pack), the first-ever treatment approved for LGSOC patients, specifically for recurrence with KRAS mutations. But here's what's critical: this approval comes with specific requirements. The FDA approved this treatment specifically for adult patients who meet the following criteria: - Have low-grade serous ovarian cancer (LGSOC) - Have recurrent disease (cancer that has returned after prior treatment) - Have a confirmed KRAS mutation in their tumor (this is a critical requirement) - Have received prior systemic therapy Without a confirmed KRAS mutation, patients cannot access this breakthrough treatment. This makes genetic testing not just helpful, but crucial for treatment decisions. The Phase 2 RAMP 201 trial demonstrated a remarkable 44% overall response rate in KRAS-mutated patients, with responses lasting anywhere from 3.3 to 31.1 months. This breakthrough targets approximately 30% of LGSOC patients who carry KRAS mutations, transforming their journey from "managing with treatments designed for other cancers" to accessing purpose-built therapy. Why this matters beyond the medicine This isn't just about one drug. It's validation that LGSOC deserves recognition as a distinct disease requiring specialized research and targeted treatments. For too long, LGSOC patients received what we call "treatment leftovers" and off-label use of medication: therapies designed for other cancers, with limited effectiveness. This approval proves that when you fund research specifically for rare diseases, breakthroughs follow. And when patient communities partner with researchers who listen, we can accelerate progress that seemed impossible just years ago. What comes next The ongoing Phase 3 RAMP 301 trial is evaluating this combination in broader LGSOC populations, both with and without KRAS mutations. This could potentially expand treatment options for even more patients, which is exactly why continued research funding matters. For the 90% of LGSOC patients who report that their lives "revolve around cancer," this approval offers something revolutionary: the possibility that effective treatment might allow life to revolve around living instead. Action steps for LGSOC Awareness Day This LGSOC Awareness Day, here's how you can drive real change: - Share life-saving awareness: The symptoms of LGSOC — persistent bloating, pelvic pain, feeling full quickly, urinary urgency, and abdominal discomfort — shouldn't be dismissed, especially in younger women. Share these symptoms widely. - Fund research that happens now: Organizations like Not These Ovaries fund clinical trials that start immediately. Your donation today goes 100% toward research, not overhead. - Advocate for genetic testing: KRAS mutation testing is now critical for LGSOC treatment decisions. Ensure patients know to request comprehensive tumor profiling, not just basic genetic screening. - Connect patients with specialists: Link people to gynecologic oncologists experienced in rare ovarian cancer subtypes and LGSOC-specific support networks. Action and understanding will save lives Today, we celebrate a medical milestone that seemed impossible just years ago. Tomorrow, we continue working toward a future where no LGSOC patient faces their diagnosis feeling alone, uninformed, or without hope. The approval of avutometinib and defactinib proves our core belief: when rare diseases receive focused attention and urgent funding, breakthrough treatments follow. This LGSOC Awareness Day, we're not just raising awareness; we're demonstrating that advocacy, research, and swift action can literally save lives. The question isn't whether we can develop treatments for rare cancers. This approval proves we can. The question is: how fast can we fund the next breakthrough? Chris Campbell Not These Ovaries impact@nottheseovaries.org Visit us on social media: LinkedIn Instagram Facebook YouTube X Frequently Asked Questions about LGSOC Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

创新药板块持续火热，带动资本市场的创业热度再次回升。8月25日，应世生物提交港股上市申请，该公司3天前刚完成3400万美元的C轮融资。 应世生物的创始人王在琪曾是K药、HPV疫苗在中国上市的推手。2011年底，默沙东在中国设立研发中心，王在琪担任临床研究负责人，在他的带领下临床团队从1个人做到30多个人，注册了8个产品，其中就包括K药和四价HPV疫苗。2017年，王在琪创立应世生物。 应世生物尚未有产品获批上市，公司进度快的管线是2017年从勃林格殷格翰手里引进的FAK抑制剂IN10018。这个靶点当时全球尚未有产品获批上市，应世生物捡了个漏，仅以150万美元就接手了这款产品，后续付出的里程碑款也不过200万美元。公司预计，2025年底或2026年初，这款产品将提交上市申请。 目前，应世生物累计融资超1.3亿美元，投后估值达到3.06亿美元。 “药王”推手自立门户 应世生物的创始人王在琪从2001年开始就从事药物研发工作，先后在礼来、先灵葆雅、默沙东、罗氏等多家跨国药企工作过，领导过国内外20余个新药分子的开发，包括10项创新药上市关键性临床研究。 在一次采访中，王在琪谈到：4价HPV疫苗的审批上市经历了数年时间，而9价疫苗仅用了两个星期。作为HPV疫苗项目负责人和亲历者，他亲身感受到国内鼓励创新的春风吹来了。 应世生物成立之际，正值中国创新药企的一轮创办高峰，四面八方都是利好：有巨大的医药市场，充足的资金、创新技术及人才，还有政府政策法规的大力支持。不过很多企业都没能熬过前两年的融资寒冬。 应世生物的经营运作比较谨慎，选择肿瘤治疗耐药和转移后的解决方案，瞄准了FAK靶点。这不是一个新靶点。早在2008年，辉瑞就有两个FAK产品进入临床I期研究，2009年GSK也开始I期临床。遗憾的是，单药抗肿瘤效果都平平无奇。跨国药企纷纷放弃了对FAK抑制剂的研究，辉瑞将Defactinib转让，勃林格殷格翰的FAK抑制剂也被应世生物接手。 在王在琪看来，耐药可能和肿瘤主动进化出来的防御机制有关，FAK 在这个过程中扮演了关键角色，针对这个靶点开发创新药可能是解决肿瘤耐药问题的关键所在。尽管那么多跨国大公司都放弃了，但临床的需求依然在那儿，靶点整体也是安全的，FAK抑制剂有很大的机会，值得去尝试。 在2023年的一次采访中，王在琪预计次年提交IN10018的新药上市申请。不过直到现在，这款药物也没能正式提交上市申请。 今年5月，全球首个FAK抑制剂Defactinib在美国获批上市，证明了这个靶点的成药性，应世生物这才有了出头的机会，也有了IPO的勇气。目前，应世生物在尝试联合用药，根据不同的癌种设计不同的给药方案，IN10018联合KRAS G12C治疗一线NSCLC、治疗突变CRC进入注册性临床阶段，获得突破性疗法资格。 一款FAK抑制剂难当家 客观来说，应世生物大概是想借着全球首个FAK药物上市的机会冲刺港交所，所以整个融资过程略显仓促，而且IN10018这款药物的市场说服力也不足。 FAK抑制剂主打其他抗癌药物治疗的“后线”。在美国，首个上市的Defactinib适应症是KRAS突变低级别浆液性卵巢癌，应世生物首个申报的是铂耐药性卵巢癌（PROC）。即便在药价较高的美国市场，华尔街分析师预测Defactinib卵巢癌适应症的销售峰值只有7-9亿美元。中国市场上，IN10018的想象空间就更加有限了。应世生物在招股书中提到，2024年中国PROC患者数量大概只有1.86万人。 IN10018想要打开更大的想象空间，还是得拿下肺癌这样的大适应症。不过，其治疗KRAS G12C突变NSCLC的三期临床试验，预计到2027年才能完成，距离上市还有很远的距离。 除了IN10018之外，应世生物后续管线中产品不多，且绝大多数处于临床前期，目前进度最靠前的是引进的一款FAP ADC药物OMTX705，处于I期临床阶段。此外，应世生物自研的第二代FAK抑制剂IN10028，预计在今年四季度才会提交临床试验申请，另外两款ADC管线则还处于临床前阶段。 这也引发了一些投资者的担忧，IN10018临床数据还可以，但核心产品是收购来的，总担心公司自身研发能力跟不上。 摆在应世生物面前的更大挑战，还是资金问题。截至今年3月底，应世生物手里的现金及现金等价物仅有1.34亿元，同期公司流动负债净额高达7.9亿元。即便算上刚到手的约1.64亿元C轮融资，公司现金储备依紧张。 匮乏的管线意味着，应世生物不太能通过BD缓解资金压力。因此，眼下能否成功冲刺港股IPO，对于应世生物的未来发展相当重要。 撰稿 | 方涛之 编辑｜江芸 贾亭 运营 | 廿十三 声明：健识局原创内容，未经许可请勿转载 内容如有问题，请邮件联系： jianshiju@boyamedia.com 商务咨询：13288688512