The Broad Institute, Inc.

The US Food and Drug Administration has approved the first cancer drug based on discoveries from Broad Institute scientists. The drug, sevabertinib, was developed by Bayer Healthcare Pharmaceuticals in close collaboration with the Broad, and is the first FDA-approved medicine from the long-standing Broad-Bayer oncology research alliance. Sevabertinib is a new oral treatment option for a type of non-small-cell lung cancer, the most common type of lung cancer. The medicine is a pill that patients can take at home and is approved for adult patients with certain HER2 mutations in their tumors and who have already received chemotherapy or immunotherapy. This translates to roughly 4,000 to 8,000 lung cancer patients in the US every year who could potentially benefit from this new drug. Many of these patients are women, including younger women who have never smoked. The drug's approval is based on data from Bayer’s Phase I/II clinical trial, which found that over 70 percent of the patients studied in one cohort saw their tumors shrink or disappear. Many patients experienced profound and durable responses. This achievement is the result of discoveries made by Broad scientists two decades ago and of the collaboration between Broad and Bayer Pharmaceuticals, which began in 2013 . The team's clinical success demonstrates that long-term academic-industry partnerships can close the gap between biological discoveries and new medicines that improve health. "The whole idea of the Broad Institute when it was founded more than 20 years ago was to bring the power of human genome science and high-throughput research to solving human disease," said Matthew Meyerson , a lead cancer biologist on the project, institute member at the Broad, the Charles A. Dana Chair in Human Cancer Genetics at Dana-Farber Cancer Institute, and professor of genetics and medicine at Dana-Farber and Harvard Medical School. "Today, with our industry partners at Bayer, we're now able to see the fruits of these efforts in a new drug that we hope can help many patients by extending their lifespan and improving their quality of life." New option for lung cancer patients Lung cancer is the leading cause of cancer-related deaths in the US, and non-small-cell lung cancer (NSCLC) accounts for more than 85 percent of lung cancer cases. Up to 4 percent of NSCLC cases carry a HER2 gene mutation, which causes the HER2 protein to become overactive and spur abnormal cell growth. Sevabertinib halts the activity of HER2 proteins with certain mutations, inhibiting the growth of tumors. Approved as a second-line treatment, it may serve a patient population that until recently had no remaining options after chemotherapy or immunotherapy for treating their cancer. The FDA granted sevabertinib a Breakthrough Therapy designation in 2024, signifying that the compound may provide substantial improvement over existing therapies in an area of high unmet medical need. In 2025, sevabertinib was additionally granted Priority Review status , meaning the FDA would aim to take action on an application within six months (compared to ten months under standard review). A research success story This milestone began with work first published in 2005 led by Meyerson and Heidi Greulich , an institute scientist and senior group leader in the Cancer Program at the Broad. In 2005, the Broad team reported that some lung cancer patients who didn't respond to existing drugs had a mutation in their tumors known as an “exon 20 insertion” — an extra bit of DNA tucked in at a specific location in the gene EGFR . Developing a drug to target this mutation was one of the first projects for the Broad-Bayer alliance when it launched in 2013, and the work soon evolved into creating a drug to target a similar mutation in HER2 , a gene closely related to EGFR that’s involved in lung and other cancers. "We've been collaborating closely with the Bayer team on sevabertinib and its precursor compounds for 12 years now,” said Greulich. “It really shows how the power of persistence can be brought to bear on difficult problems to help patients." A Phase III clinical trial is continuing to evaluate sevabertinib as a first-line treatment option for patients with NSCLC carrying HER2 mutations. The drug is also being studied in patients with other metastatic or unresectable solid tumors, beyond lung cancer, that have HER2 mutations. "We're in an extraordinary time for the development of new medicines to treat cancer patients," said Meyerson. "And I think that, with our industry partners, we have a huge opportunity to repeat this success not once, not twice, but many, many times over." 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.

Contributions to this project were also made by colleagues in the Broad’s technology platforms, including the Genetic Perturbation Platform , the Center for the Development of Therapeutics , and the Proteomics Platform , and by colleagues in the Broad’s Cancer Program . What were some of the challenges you faced in developing this new medicine? HG: We encountered many challenges during the course of this project, so it wasn't smooth sailing all the way through. In fact, when we started the project, there were no cellular models available so we had to come up with a cellular assay that we could use to test early compounds. Our initial cell assay was unstable, but heroic efforts from senior research associate Bethany Kaplan helped to stabilize the assay so that it was robust enough for compound testing. In addition, our chemistry colleagues at Bayer worked for years to improve the potency, selectivity, and stability of the compounds to eventually develop sevabertinib. How did two decades of basic cancer research contribute to the development of the new drug? MM: The development of sevabertinib is a real testament to the power of long-term collaborative partnerships. Heidi and I have been working together on EGFR and HER2 for 22 years now, and we've developed a lot of knowledge of these molecules and how they behave. Before that, Bill Sellers and I worked to explore alterations of tyrosine kinase enzymes in human cancer that led to the EGFR discovery. And going back even further, Bill and Broad Institute director Todd Golub and I worked to understand what happens in the genomes of human cancers more broadly. These long-term scientific collaborations and all the biological insight that came from them, along with our deep and continued commitment to solving the same problems, have really helped us to get to where we are today with the development of sevabertinib. What’s next for sevabertinib and similar efforts? MM: It is an extraordinary time to make many new treatments for patients with cancer and other diseases as well. The success of sevabertinib is a real proof-of-concept that we can do this again. If we can keep targeting the genomic causes of cancer in a disciplined and persistent way, we will continue making further advances in cancer treatment, so it’s important that we keep this momentum going forward. Here at the Broad, with our industry partners, we could do this again, not just once, not just twice, but many, many more times, and I’m looking forward to our opportunities to do that. Seeing this effort come to fruition after so many years of hard work, what are your reflections on this milestone? MM: As a cancer researcher, it's just an incredible honor and privilege to be part of a team that has brought forward a new medicine that can help people with the terrible disease of lung cancer. HG: Not all academic scientists have the opportunity to initiate and execute a drug discovery project that directly benefits patients, so we’ve been incredibly fortunate in this respect. The development of sevabertinib is also really meaningful for me personally. Both my parents passed away from cancer, and my father from lung cancer in particular. So the fact that I've made a contribution to help other lung cancer patients resonates with me and makes me really glad I come to work every day at the Broad. 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.

2025年11月19日，FDA（美国食品药品监督管理局）加速批准sevabertinib（Hyrnuo，拜耳健康保健制药有限公司），适用于局部晚期或转移性非鳞状非小细胞肺癌（NSCLC）成人患者，其肿瘤经FDA批准检测方法证实存在HER2（ERBB2）酪氨酸激酶域（TKD）激活突变，且既往接受过系统治疗。 sevabertinib推荐剂量为每天两次，口服，每次20mg，随食物服用，直到病情进展或出现不可接受的毒性。 （图片来源：FDA官网） 关注新药社，每日为你介绍全球新药信息！ Sevabertinib （代号：BAY 2927088）是由Bayer AG与Broad Institute of MIT and Harvard合作开发的一款口服、可逆的小分子酪氨酸激酶抑制剂（TKI）。它主要靶向突变型HER2（也称 ERBB2）——包括HER2 exon 20 插入和点突变，同时也具备对突变型EGFR的作用，并对野生型EGFR具备较高选择性。 2024年被FDA认定为“突破性疗法”，2025年5月，拜耳提交sevabertinib 的 NDA（新药申请），并被授予“优先审评”（Priority Review）程序。 FDA的加速批准是基于一项开放标签、单臂、多中心、多队列临床试验SOHO-01（NCT05099172），试验中评估了sevabertinib对既往接受过系统治疗、存在HER2（ERBB2）酪氨酸激酶域激活突变的不可切除或转移性非鳞状非小细胞肺癌患者的疗效。入组前由当地实验室通过肿瘤组织或血浆检测确认HER2（ERBB2）激活突变。 主要疗效评估指标为经BICR采用RECIST v1.1标准评估的确认客观缓解率（ORR）和缓解持续时间（DOR）。在70例局部晚期或转移性NSCLC且携带HER2（ERBB2）酪氨酸激酶域激活突变、既往接受过系统治疗但未接受过HER2突变靶向治疗的患者中，ORR为71%（95% CI： 59, 82），中位DOR为9.2个月（95% CI：6.3, 15.0），其中54%的应答患者DOR≥6个月。 在52例局部晚期或转移性NSCLC患者中，这些患者携带HER2（ERBB2）酪氨酸激酶域激活突变，且既往接受过包括HER2靶向抗体药物偶联物在内的系统治疗，ORR为38%（95% CI：25， 53），中位DOR为7.0个月（95% CI：5.6，不可评估），60%的应答患者DOR为≥ 6个月。 试验中最常见不良反应为腹泻（多数为 1/2 级），在某些队列中出现率达 80% 以上。 值得一提的是FDA还批准了Oncomine Dx靶向检测试剂盒（Life Technologies Corporation）作为伴随诊断设备，用于辅助检测非鳞状非小细胞肺癌患者中HER2（ERBB2）酪氨酸激酶域（TKD）激活突变。 今年7月，拜耳已在中国提交sevabertinib （代号：BAY 2927088片）的上市申请，也被CDE纳入突破性治疗和优先审评审评程序，如无意外，预计2026年4月获批。另外，拜耳还开发了sevabertinib的口服溶液剂型，目前尚未在中国申请上市。 根据CDE临床登记平台的公开信息，sevabertinib登记的两项临床试验值得关注：一是BAY 2927088对不同类型的HER2激活突变实体瘤患者疗效的篮式研究；二是评估BAY 2927088口服给药与标准治疗相比作为一线治疗的有效性和安全性的III期研究。 （图片来源：CDE官网） 在中国，今年8月批准了勃林格殷格翰的圣赫途®（中文通用名：宗艾替尼片，英文通用名：zongertinib），是全球首个获批的口服HER2-TKI。 声明：本公众号发布的关于新药研发进展、临床研究动态及药品获批信息等内容，仅为分享医药领域前沿资讯、提供行业信息参考，不构成任何形式的医疗建议、疾病诊断依据或用药指导！ 加入新药社交流群，每日获取全球新药动态