Panitumumab

BridgeBio Pharma has received European approval for a drug that treats cardiomyopathy stemming from a rare metabolic condition, strengthening the biotech’s position to take market share from the blockbuster Pfizer drug that is currently the standard treatment. The European Commission granted marketing authorization to acoramidis, which BridgeBio developed as treatment for transthyretin amyloidosis (ATTR), a disease in which a genetic mutation leads to abnormal versions of the liver protein transthyretin (TTR). The misfolded proteins can cause nerve and heart problems. BridgeBio’s acoramidis, which will be commercialized in Europe as Beyonttra, is a small molecule designed to stabilize TTR to treat the cardiomyopathy caused by the disease. The biotech contends its drug is a better stabilizer than the drugs marketed by Pfizer. For 2024, Pfizer reported $3.3 billion in revenue from its Vyndaqel family of TTR stabilizers, a 36% increase compared to 2023. The FDA approved the BridgeBio TTR drug this past November and it is marketed in the U.S. under the brand name Attruby. In a prescriptions update included in its report of fourth quarter and full year 2024 financial results, BridgeBio said that as of Feb. 17, 1,028 unique patient prescriptions for the drug have been written by 516 unique prescribers since the U.S. approval. That’s an increase from what the company reported last month during the annual J.P. Morgan Healthcare Conference last month, which Leerink Partners said indicates a broadening prescriber base. presented by Health IT Transforming Patient Care: The Role of AI-Powered Assistants The progress in artificial intelligence (AI) is reshaping patient care, across various dimensions, from facilitating faster discharge to curating treatment plans and suggesting lifestyle changes. By IT Medical In Europe, the BridgeBio drug will be commercialized by Bayer per terms of an agreement signed last year. BridgeBio is in line to receive milestone payments and royalties from sales. The first payment is $75 million for the European approval. Here’s a recap of other recent regulatory developments: More Rare Disease Drug Approvals —The FDA approved Mirum Pharmaceuticals drug chenodiol, brand name Ctexli, for treating rare lipid storage disease cerebrotendinous xathomatosis (CTX). This genetic metabolic disorder results in deficient levels of an enzyme important for breaking down fats. Chenodiol is a naturally occurring human bile acid. Under the brand name Chenodal, chenodiol has been marketed for treating gallstones, but it has been used off label as a treatment for CTX. The new FDA approval makes Ctexli the first FDA-approved treatment for CTX. Mirum acquired the drug from Travere Therapeutics in 2023. Sponsored Post Tips for Greater Healthcare Practice Success in 2025 Join us on February 26 at 3 pm ET, and we’ll dive into the key areas where practices are losing time and money and provide solutions to overcome them. By Elation Health and MedCity News —The European Commission approved Livdelzi, a Gilead Sciences drug developed for the rare liver disease primary biliary cholangitis (PBC). The conditional marketing authorization covers use of the daily pill in combination with ursodeoxycholic acid, an older drug that is the standard PBC treatment. The European decision for Livdelzi follows FDA approval of the drug last August. Livdelzi comes from Gilead’s $4.3 billion acquisition of Cymabay Therapeutics last year. —The FDA approved SpringWorks Therapeutics drug Gomekli as a treatment for tumors caused by the rare genetic disorder neurofibromatosis type 1 (NF1). The approval covers treatment of these tumors in both adults and children. That’s an advantage over AstraZeneca’s Koselugo, which is only approved for treating pediatric NF1 patients. —CSL’s garadacimab received approvals in Europe and Japan for treating adults and adolescents age 12 and older who have hereditary angioedema (HAE). The rare disease leads to swelling attacks in tissue beneath the skin. This swelling can close off the airway, putting a patient at risk of death. CSL’s drug, brand name Andembry, is an antibody engineered to inhibit activated Factor XII, a protein that starts the cascade of events leading to unpredictable HAE swelling attacks. Andembry is still under regulatory review in the U.S., Canada, and Switzerland. —Evrysdi, a drug marketed by Roche for the rare disease spinal muscular atrophy, is now approved in a tablet formulation. The product, which was initially developed by PTC Therapeutics and later partnered with Roche, was first approved in 2020 as a liquid administered via an oral syringe or through a feeding tube. The liquid formulation must be refrigerated and kept away from light. The new Evrysdi tablet offers patients a simpler dosing option that can be stored at room temperature. Cancer Drug Approvals —Merck’s Welireg is now approved in Europe as a treatment for adults with von Hillel-Lindau (VHL) disease, a rare genetic disorder that causes benign tumors in the body that can become cancerous. The European Commission’s conditional approval covers patients whose VHL is unsuitable for surgery and requires therapy for renal cell carcinoma, central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors. The commission’s decision also covers the treatment of adults with advanced clear cell renal cell carcinoma that has progressed after two or more lines of therapy. Welireg is a once-daily pill designed to block HIF-2 alpha, inhibiting this protein’s ability to contribute to cancer progression. The FDA approved Welireg in 2021. —The FDA approved Romvimza, an Ono Pharmaceutical drug for tenosynovial giant cell tumor (TGCT). The drug, which came to Ono via the 2024 acquisition of its developer, Deciphera Pharmaceuticals, offers more convenient dosing and a lower risk of liver toxicity compared to Turalio, a Daiichi Sankyo drug that was the first approved systemic therapy for TGCT. —Datroway, a TROP-2-targeting antibody drug conjugate (ADC) from partners AstraZeneca and Daiichi Sankyo, landed FDA approval for treating advanced and unresectable breast cancer that’s classified as HR positive and HER2 negative. It will compete with Trodelvy, Gilead Sciences’ TROP-2-targeting ADC is already approved in the same indication. —Axsome Therapeutics’ Symbravo won FDA approval for acute treatment of migraine with or without aura in adults. The tablet pairs two compounds that target multiple pathways behind a migraine attack. Symbravo is made with proprietary Axsome technology that enables the drug to more quickly achieve maximum concentration in the blood and to maintain a long half-life. Symbravo’s label carries a black box warning for cardiovascular and gastrointestinal risks. The approval marks a comeback for the drug, which received an FDA complete response letter in 2022 due to manufacturing issues. —Foundation Medicine received FDA approval for FoundationOne CDx as a companion diagnostic for the brain cancer drug Ojemda. The test detects the BRAF V600 mutation addressed by the drug, developed by Day One Biotherapeutics. The FDA approved Ojemda last April as a treatment for advanced cases of pediatric low-grade glioma. —Adcetris, an antibody drug conjugate (ADC) developed by Pfizer subsidiary Seagen, added to its label the treatment of advanced cases of diffuse large B-cell lymphoma (DLBCL). The new approval covers use of the drug in combination with the cancer drugs Revlimid and Rituxan. The new FDA approval is based on Phase 3 results showing the Adcetris regimen led to a 37% reduction in risk of death compared to Revlimid, Rituxan, and a placebo. Adcetris was first approved in 2011 for Hodgkin lymphoma and systemic anaplastic large cell lymphoma. The drug now has eight FDA approvals in cancer. Pfizer has rights to the drug in the U.S. and Canada; Takeda Pharmaceutical has rights to the product in the rest of the world. —Blockbuster AstraZeneca cancer drug Calquence, first approved in 2017 for treating advanced mantle cell lymphoma after at least one prior line of therapy, is now approved as a first-line treatment for this rare and aggressive blood cancer. The drug, a BTK inhibitor, is the first in its class approved as a first-line MCL treatment. —Amgen cancer drug Lumakras expanded its FDA approval to metastatic colorectal cancer that is positive for the KRAS G12c mutation and is also unresponsive or resistant to chemotherapy. The regulatory nod in this indication covers use of the daily pill in combination with Vectibix, an Amgen antibody drug approved for colorectal cancer. Lumakras was initially approved in 2022 as a treatment for non-small cell lung cancer driven by the KRAS G12C mutation. Approvals in Immunology —Galderma drug Nemluvio received European Commission approval as a treatment for the inflammatory skin disorders atopic dermatitis and prurigo nodularis. The once-monthly injection is an antibody designed to block IL-31 signaling. The FDA approved Nemluvio for prurigo nodularis and atopic dermatitis last year. —Eli Lilly’s Omvoh, a biologic drug first approved in 2023 as a treatment for ulcerative colitis, added moderately to severely active Crohn’s disease to its label. The drug is an antibody that targets IL-23p19, a protein that contributes to gastrointestinal inflammation. Regulatory submissions in Crohn’s are currently under review in Europe and Japan. In ulcerative colitis, the drug is approved in 44 countries. —Palforzia, a peanut allergy immunotherapy marketed by Stallergenes Greer, expanded the product’s approved uses in Europe to include the treatment of children ages 1 through 3. The initial approval for Palforzia covered children age 4 through 17. The new approval in Europe comes about six months after the therapy secured a similar regulatory decision from the FDA. Privately held Stallergenes Greer added Palforzia to its allergy products portfolio via a 2023 deal with Nestlé. Approvals for Neuro, Pain Meds —Supernus Pharmaceuticals received FDA approval for Onapgo, a drug/device combination product that continuously infuses the drug apomorphine to treat the “off” episodes experienced by Parkinson’s disease patients. Onapgo joins a Supernus Parkinson’s drug lineup that includes Apokyn, an injection pen product that administers apomorphine, and Gocovri, a capsule approved to treat dyskinesia and off time in Parkinson’s patients. —Vertex Pharmaceuticals’ non-opioid drug Journavx won FDA approval as a first-in-class treatment for moderate-to-severe acute pain in adults. The twice-daily pill targets NaV1.8, a pathway in the peripheral nervous system. By stopping pain signals in the periphery before they reach the brain, drugs in this class are intended to avoid the addiction risks poised by opioids, which hit targets in the brain. —The FDA approved Johnson & Johnson’s Spravato as a monotherapy for adults with treatment-resistant depression. The nasal spray drug’s initial approval in 2019 covered its use in combination with an oral antidepressant for treatment-resistant depression in adults. Vaccine Approvals —Bavarian Nordic received FDA approval for its virus-like particle chikungunya vaccine, which will be marketed under the brand name Vimkunya. The FDA nod covers use of the vaccine in those age 12 and older. That’s a broader range than Valneva’s Ixchiq, which became the first FDA-approved chikungunya vaccine in 2023. Ixchiq is indicated for use in those age 18 and older. Bavarian Nordic’s new approval was accompanied by a tropical disease priority review voucher, which the company said it plans to sell. —A GSK vaccine covering five groups of bacteria that cause meningococcal disease is now FDA approved for use in those ages 10 through 25. The vaccine, brand name Penmenvy, combines components of two already available GSK meningococcal vaccines, Bexsero and Menveo. Penmenvy will now compete with Pfizer’s Penbraya, which also covers five bacterial groups. Both vaccines are administered as two intramuscular injections given six months apart. —A Covid-19 vaccine developed by partners Arcturus Therapeutics and CSL is now approved in Europe. The vaccine, brand name Kostaive, employs self-amplifying RNA. Unlike traditional mRNA vaccines, self-amplifying RNA vaccines instruct the body to make more mRNA and protein to boost the immune response. Kostaive was originally developed by Arcturus. In 2022, CSL paid $200 million up front to collaborate on the vaccine. Per deal terms, Arcturus is responsible for regulatory filings in the U.S. and Europe as well as R&D of next-generation vaccine candidates. CSL subsidiary Seqirus takes the lead on all other R&D in Covid-19, influenza, and other fields. More Regulatory Approvals —Izervay, a geographic atrophy drug marketed by Astellas Pharma, is now approved for longer dosing. The product’s initial 2023 approval permitted the once-monthly eye injection to be administered for up to 12 months. The new approval removes the 12 month restriction. However, the Astellas product did not receive the FDA O.K. for every-other-month administration. That additional flexibility would have put it on par with Apellis Pharmaceuticals’ geographic atrophy drug Syfovre, which is approved for monthly and every-other-month dosing. —Novo Nordisk drug Ozempic is now approved for treating chronic kidney disease in patients with type 2 diabetes. The blockbuster drug was initially approved for type 2 diabetes, which is a risk factor for chronic kidney disease. In the pivotal study supporting the latest regulatory nod, Ozempic led to a 24% reduction in kidney disease complications compared to a placebo. —AbbVie’s pairing of the antibiotics aztreonam and avibactam received FDA approval for the treatment of complicated intra-abdominal infections, giving clinicians another tool to address antimicrobial resistance. The therapy, which will be marketed under the brand name Emblaveo, is indicated for use in combination with Flagyl, a Pfizer antibiotic. Emblaveo was developed in partnership with Pfizer. AbbVie has commercialization rights to the drug in the U.S and Canada; Pfizer is responsible for the antibiotic’s commercialization in the rest of the world. Clinical Holds and Regulatory Setbacks —The FDA sent Harmony Biosciences a refuse to file letter for pitolistant, a drug the biotech is seeking to commercialize as a treatment for excessive daytime sleepiness in adults who have idiopathic hypersomnia. In a Phase 3 withdrawal study, the once-daily tablet did not achieve statistical significance compared to placebo. The biotech said a Phase 3 registrational study in idiopathic hypersomnia is on track to begin in the fourth quarter of this year. Pitolistant is already available as a treatment for excessive daytime sleepiness or cataplexy in adults with narcolepsy, marketed as Wakix in this indication. —Moderna’s Phase 3 test of its two-season norovirus vaccine candidate, mRNA-1403, is under an FDA clinical hold. The company said the clinical trial halt is due to a single report of Guillain- Barré syndrome, nerve damage that can be a complication of vaccines. The adverse event is under investigation. The company does not expect the clinical hold will affect the trial readout timeline in the northern hemisphere, where the study is already fully enrolled. The timing of a data readout will depend on case accruals. Second season enrollment is being prepared in the southern hemisphere. —The FDA placed a clinical hold on Ebvallo, a treatment for advanced cases of Epstein-Barr virus positive post-transplant lymphoproliferative disease. The hold came days after the FDA rejected the immunotherapy due to findings from an inspection of a third-party manufacturing facility. The company said the Ebvallo clinical hold relates to the issues flagged in the complete response letter. Ebvallo, which is made by engineering T cells from healthy donors, was approved in Europe in 2022.

近年来，靶向和免疫药物势如破竹的研发和上市，让肺癌的治疗步入了黄金时代，在刚刚过去的2024年，22款肺癌明星药物纷纷获批，其中中国上市16款肺癌新药，美国上市7款，这意味着一些幸运的病友等来了新的选择和希望！ 好消息是，目前国外上市的以及中国自主研发的几款针对肺癌EGFR,ALK,ROS1,RET,MET,NTRK靶点的抗肿瘤新药，开始招募国内患者了！这意味着，国内的患者也终于有机会免费用上这些效果更好，堪称天价的抗癌新疗法！全球肿瘤医生网将2025年肺癌病友们可以申请的17款新药临床试验信息整理如下，想申请的患者可以提交病历至全球肿瘤医生网将医学部进行初步评估！ 2025非小细胞肺癌17款“续命”新药 01 EGFR EGFR(epidermal growth factor receptor，ErbB-1或HER1)表皮生长因子受体主要在肺腺癌、亚裔、非吸烟及女性患者中，大约有 15% 的白种人和 30-50% 的亚洲人中有 EGFR 基因突变。无吸烟史者，比例高达 50-60%，常见的突变位点是外显子19和21，占90%，称为经典型突变，其余10%为外显子18和20的突变。目前多款国内和国际的明星新药正在招募。 01 Ifebemtinib（IN10018） 药品名称： 甲磺酸哆希替尼片 作用靶点： EGFR 研发公司： 应世生物 药物介绍： 是一种高效、高选择性的FAK抑制剂。IN10018早期临床数据显示了其良好的安全性和多癌种有效性。最新研究成果表明IN10018有望通过克服肿瘤基质纤维化屏障和调节肿瘤免疫抑制性微环境，实现突破肿瘤防御机制，有效克服耐药和转移，成为多种治疗方案的基石分子。 适应症： EGFR突变肺癌 入组条件（部分）： 1.有EGFR-TKI敏感性相关的EGFR突变，包括Ex19del或L858R。除外EGFR-TKI敏感突变，可合并其他EGFR突变类型如T790M等。 2.既往经三代EGFR-TKI治疗进展，并接受过1-2线化疗（需至少包括 1线含铂化疗），且化疗进展或不耐受的NSCLC受试者. 02 SKB264 药品名称： SKB264 作用靶点： EGFR 研发公司： 科伦博泰生物 药物介绍： 注射用SKB264是靶向人滋养层细胞表面抗原2（TROP-2，在多种上皮来源肿瘤中高表达）的抗体偶联药物（ADC）药物，其偶联方式和毒素小分子具有自主知识产权，拟用于恶性实体瘤的治疗。早期临床研究结果显示了SKB264在转移性实体瘤患者中良好的耐受性、安全性和令人鼓舞的抗肿瘤活性。 适应症： EGFR突变肺癌 入组条件（部分）： 1.经肿瘤组织学或细胞学或血液学证实存在EGFR敏感突变；2.既往接受过EGFR-TKI治疗且治疗失败；3.既往接受过针对局部晚期或转移性NSCLC的至少一种含铂化疗失败。 03 JMT-101 药品名称： JMT-101 作用靶点： EGFR20 ins 研发公司： 津曼特 药物介绍： JMT101为人源化抗EGFR单克隆抗体，具有自主知识产权，该产品相较于国内外已上市同类产品西妥昔单抗、帕尼单抗等具有亲和力更高、免疫原性低、药效更优的特点。该产品已于2016年5月获得CFDA临床批件，现已进入I期临床研究，拟用于治疗非小细胞肺癌，结直肠癌等实体瘤。疾病控制率100%，我国自主研发广谱新药重磅登场！临床招募进行中 适应症： 一线或二线治疗EGFR突变20ins阳性肺癌 招募信息（部分）： ⅢB或Ⅳ期非小细胞肺癌，不适合进行根治性手术或放疗，且证实具有EGFR 20号外显子插入突变（包括重复突变），初治或一线治疗失败后的患者。 04 伏美替尼 药品名称： 伏美替尼 (AST2818，艾弗沙) 作用靶点： EGFR 20ins 研发公司： 艾力斯 药物介绍： 第三代EGFR抑制剂。2021年3月在国内获批上市，治疗EGFR T790M突变型非小细胞肺癌。国研第三代EGFR抑制剂，上市！新适应症临床试验开启！ 适应症： EGFR突变20ins阳性肺癌 招募信息（部分）： ⅢB或Ⅳ期非小细胞肺癌，不适合进行根治性手术或放疗，且证实具有EGFR 20号外显子插入突变（包括重复突变）。 05 PLB1004 药品名称： 安达替尼（PLB1004） 作用靶点： EGFR 20ins 研发公司： 鞍石生物 适应症： EGFR突变20ins阳性肺癌 药物介绍： 中国原研、具有全球知识产权的新型单苯胺基嘧啶小分子EGFR抑制剂，可有效且不可逆地靶向外显子20插入突变，同时还有效针对经典 EGFR 突变 ExDel19、L858R 和 T790M，与野生型 EGFR 相比具有高度选择性。这款药物是在保持第三代EGFR TKI药效优势的前提下，针对EGFR Ex20插入设计的全新一代EGFR TKI。 招募信息（部分）： ⅢB或Ⅳ期非小细胞肺癌，且证实具有EGFR 20号外显子插入突变（包括重复突变）。 02 ALK 对于确定为ALK阳性的患者是幸运的，因为针对ALK的靶向药有效率超高，副作用不大，一不注意肿瘤就给“吃没了”。因此，ALK突变被称为“钻石突变”，虽然仅有5%，但可用的靶向药却很多，显着增加了治疗机会，ALK最常见的融合伴侣是ALK-EML4（棘皮类微管相关样蛋白4）。它更容易出现在既往少量 / 无吸烟史和年轻的患者身上。 01 TL139 作用靶点： ALK/ROS1/NTRK融合 研发公司： 苏州韬略 药物介绍： TL139是一款新一代NTRK/ROS1/ALK多靶点抑制剂。 适应症： 二线治疗ALK/ROS1/NTRK阳性实体瘤 招募信息（部分）： ALK阳性，既往接受过≤2种ALK抑制剂的治疗（用过化疗没用过ALK抑制剂的也可以） ROS1阳性，既往接受过≤1种ROS1抑制剂的治疗 NTRK阳性，接受一代NTRK抑制剂后进展（拉罗，恩曲，TL118） 03 ROS1 ROS1 全称 c-ros 原癌基因，是一种跨膜的受体酪氨酸激酶基因。出现ROS1突变的患者，更多的是年轻的、非吸烟的肺癌患者，其中肺腺癌居多。突变者约占 NSCLC 总数的 3%。 01 瑞普替尼 药品名称： TPX-0005（Repotrectinib，瑞普替尼） 作用靶点： ALK/ROS1/NTRK突变 研发公司： TurningPoint/再鼎医药 药物介绍： 第二代ALK/ROS1/NTRK抑制剂。这款全新一代的ROS1，pan-TRK和ALK抑制剂，是一种经过特殊设计的低分子量大环TKI，具有高度选择性，对ROS1，TRKA-C和ALK具有很高的效力。临床证实其抗ROS1能力可以达到克唑替尼和恩曲替尼（Entrectinib，Etreso）的90倍以上！在ROS1融合的患者中，接受克唑替尼治疗后三分之一的患者出现现 ROS1 G2032R ，还有5%的患者会出现 ROS1 D2033N 。TPX-0005 能够克服临床中出现的所有已知ALK,ROS1和NTRK耐药问题。 适应症： ROS1突变阳性肺癌 招募信息（部分）： 1、年龄：18~75，男性或女性; 2、ECOG评分0~1分，预期寿命≥3个月; 3、未经治疗的ROS1阳性肺癌 02 他雷替尼 药品名称： Taletrectinib（DS-6051b / AB-106） 作用靶点： ROS1/NTRK突变 研发公司： TurningPoint/再鼎医药 药物介绍： 第二代ROS1/NTRK抑制剂。Talectrectinib代号为DS-6051b/AB-106，是一款新型、有效、选择性高的下一代ROS1和NTRK双靶点小分子抑制剂，可穿越血脑屏障。 适应症： 二线治疗ROS1突变阳性肺癌 招募信息（部分）： 1、年龄：18~75，男性或女性; 2、ECOG评分0~1分，预期寿命≥3个月; 3、标准治疗失败的ROS1+非小细胞肺癌患者 03 ICP-723 药品名称： ICP-723 作用靶点： ROS1突变 研发公司： 诺诚建华 药物介绍： ICP-723是一种国内研发的第二代泛酪氨酸受体激酶（泛TRK）小分子抑制剂，用于治疗不同肿瘤类型的神经营养性酪氨酸激酶（NTRK）阳性的肿瘤患者以及由于抗性TRK基因突变而对第一代酪氨酸激酶（TRK）抑制剂产生耐药性的患者。2021年8 月 31 日，获得美国食品药品监督管理局（FDA）的研究性新药批准在美国开始 I 期临床试验。 适应症： 1.ROS1阳性非小细胞肺癌； 2.克唑替尼或化疗后进展。 04 TL139 药品名称： TL139 作用靶点： ALK/ROS1/NTRK融合 研发公司： 苏州韬略 药物介绍： TL139是一款新一代NTRK/ROS1/ALK多靶点抑制剂。 适应症： 二线治疗ALK/ROS1/NTRK阳性实体瘤 招募信息（部分）： ALK阳性，既往接受过≤2种ALK抑制剂的治疗（用过化疗没用过ALK抑制剂的也可以） ROS1阳性，既往接受过≤1种ROS1抑制剂的治疗 NTRK阳性，接受一代NTRK抑制剂后进展（拉罗，恩曲，TL118） 05 HG030 药品名称： HG030 作用靶点： ALK/ROS1重排 研发公司： 成都先导 药物介绍： HG030是新一代NTRK /ROS1双靶点酪氨酸激酶抑制剂。 适应症： 治疗NTRK/ROS1重排非小细胞肺癌 招募信息（部分）： Ⅲ期或Ⅳ期非小细胞肺癌，存在ROS1重排； 04 RET RET激酶的基因组改变包括融合和点突变，从而导致RET信号过度活细胞生长不受控制。RET融合和突变发生在多种肿瘤类型中，且发生频率不同。据统计，在非小细胞肺癌里面，大约1%-2%的患者有RET基因融合，在甲状腺髓样癌中超过60%的患者有RET基因突变，在乳头状甲状腺癌中10%的患者有RET基因融合. 01 HS-10365 药品名称： HS-10365 作用靶点： RET 研发公司： 豪森药业 药物介绍： HS-10365是具有自主知识产权的1类创新药，它专门针对RET基因改变的实体瘤进行治疗。这款药物通过抑制RET酪氨酸激酶的活性，阻断肿瘤细胞的信号传导通路，从而有效抑制肿瘤细胞的生长和增殖，为患者带来新的治疗选择和希望。 适应症： 二线治疗RET融合阳性肺癌 招募信息（部分）： 经组织病理学或细胞学证实的复发、不可切除的局部晚期或转移的RET阳性非小细胞肺癌。 05 MET MET 是一种络氨酸激酶受体，它的过度激活与肿瘤发生、发展、预后与转归密切相关。它在肺腺癌中发生率为3%，其最常见的变异类型是外显子14的供体位点剪接突变。近年来，MET抑制剂的研究层出不穷，其中沃利替尼、Tepotinib和Capmatinib（卡马替尼，又名INC280）是目前研究数据相对较多的3个药物。 01 伯瑞替尼 药品名称： 安达替尼（PLB1004）+伯瑞替尼 作用靶点： c-Met过表达 研发公司： 鞍石生物 适应症： EGFR-TKI治疗失败后伴c-Met过表达或c-Met扩增 招募信息（部分）： 经组织学或细胞学确诊的，经临床诊断为不能手术治疗且不能接受根治性同步放化疗的局部晚期（ⅢB/ⅢC期）、转移性或复发性（IV期）NSCLC的患者； 既往未接受过针对晚期/转移性疾病的任何系统性抗肿瘤治疗； 携带与EGFR-TKI敏感性相关的EGFR突变（包括外显子19缺失和外显子21 L858R突变）； 经中心实验室确认c-Met过表达：通过IHC检测，组织样本≥50%肿瘤细胞的染色强度3+； 02 谷美替尼 药品名称： 谷美替尼 作用靶点： MET蛋白过表达 研发公司： 海和 药物介绍：谷美替尼（SCC244） 是一种国研高选择性 MET 抑制剂，够选择性抑制c-Met激酶活性，进而抑制肿瘤细胞的增殖、迁移和侵袭。在携带 MET 外显子 14 跳跃突变的非小细胞肺癌患者中表现出持久疗效。这款药物的上市为具有间质-上皮转化因子（MET）外显子14跳变的局部晚期或转移性非小细胞肺癌患者提供了新的治疗选择。 适应症： 一线治疗MET蛋白过表达的非小细胞肺癌。 招募信息（部分）： c-Met+ NSCLC或是存在已证实的MET 基因过表达。 06 KRAS RAS家族中的NRAS，HRAS和KRAS突变引起的癌症占所有人类癌症的近四分之一，使其成为与癌症相关的最常见基因突变之一。其中，KRAS是最常见的致癌基因（所有RAS突变的85％），存在于 90%的胰腺癌中，30-40%的结肠癌中，15-20%的肺癌中 （大多为非小细胞肺癌）。遗憾的是从发现这一靶点至今的30年里，没有任何一款直接针对KRAS突变的靶向药物获批。因此，人们一直认为KRAS是一种“坚不可摧”蛋白质。 01 GH21 药品名称： GH21 作用靶点： Kras G12c突变 研发公司： 勤浩医药 药物介绍： GH21具有低脱靶风险、口服生物利用率高，并展现出较大的安全性窗口，是一种具有广阔开发前景的小分子抗肿瘤药物。临床前的数据表明，GH21与D-1553的联合使用在携带KRASG12C突变的肿瘤动物模型中展现出强大的药物效果，并且动物的耐受性良好，没有观察到增加的毒性。 适应症： 二线治疗KRAS突变阳性非小细胞肺癌 招募信息（部分）： 1、年龄≥18岁，男性或女性; 2、ECOG评分0~1分，预期寿命≥3个月; 3、标准治疗失败的KRAS+实体瘤 02 HRS-4642 药品名称： HRS-4642 作用靶点： Kras G12d突变 研发公司： 恒瑞医药 药物介绍： HRS-4642是第一个具有良好临床疗效的KRASG12D抑制剂且耐受性良好，没有新的安全性信号。 适应症： 二线治疗KRAS阳性实体瘤 招募信息（部分）： 1、年龄：18~75，男性或女性; 2、ECOG评分0~1分，预期寿命≥3个月; 3、标准治疗失败的KRASG12D实体瘤 07 BRAF BRAF突变是人类致癌的关键驱动因素之一。据报道， BRAF突变存在于多种常见类型癌症中，包括 黑色素瘤（40-50%） 、 甲状腺癌（29-83%） 、 结直肠癌 (CRC；10-20%) 和 非小细胞肺癌 (NSCLC；2-4%) ， 最常见的BRAF突变亚型是BRAF V600。 01 HL-085 药品名称： HL-085胶囊联合维莫非尼 作用靶点： BRAF V600E 研发公司： 科州生物 适应症：BRAF V600E 阳性肺癌 药物介绍： 科露平（妥拉美替尼/HL-085）是我国自主研发的首款高选择性的MEK抑制剂，2024年3月获批上市，成为全球首个且唯一获批针对NRAS突变晚期黑色素瘤适应症的MEK抑制剂，可与RAS-RAF-MEK-ERK信号通路中的MEK1/2结合，阻断下游信号通路的传导，从而抑制肿瘤的生长。 招募信息（部分）： 1.经病理组织学/细胞学确认的手术无法切除，局部晚期或转移性的 IIIb、IIIc、IV 期 NSCLC； 2.未经系统治疗或经过系统治疗的人群均可纳入； 3.基线时能够提供 BRAF V600E 突变的基因检测报告 未完待续！期待更多新药开花结果 过去十年，肺癌的靶向免疫治疗已经取得了长足的进展，逆转了晚期患者的生存期，除了上面正在招募的药物，还有更多的新药正在研发中，下一期我们将为病友们系统整理肺癌各靶点的全球研发进展，想接受治疗的患者可以申请专家会诊评估用药方案或参加方舟计划获得免费用药的机会。让我们拭目以待，期待更多新药早日上市，造福大众！ 本文为全球肿瘤医生网原创，未经授权严禁转载