SpringWorks Therapeutics LLC

Europe's CHMP has recommended 10 drugs for marketing authorization, including GSK's cancer treatment Blenrep, which has also picked up recent nods from regulators in Japan and the U.K. Who doesn’t love a comeback story? Certainly not GSK, which has found a way to resuscitate its cancer treatment Blenrep as a combo agent after it was pulled from the U.S. market as a monotherapy in late 2022.This week, Europe’s Committee for Medicinal Products for Human Use (CHMP) recommended Blenrep for approval for patients with relapsed or refractory multiple myeloma.The CHMP has given Blenrep a thumbs-up in two combinations for patients who have received at least one prior therapy including Bristol Myers Squibb’s Revlimid (lenalidomide). One combo pairs Blenrep with the protease inhibitor Velcade (bortezomib) plus the corticosteroid dexamethasone. The other regimen includes Blenrep with BMS's Pomalyst (pomalidomide) and dexamethasone.In 2020, the FDA signed off on the antibody-drug conjugate as a monotherapy for patients who had tried at least four other treatments, but two years later, Blenrep came up short in a confirmatory trial.Since then, Blenrep has emerged victorious in two phase 3 trials, topping standard of care as a combo agent. In both trials, the Blenrep regimen showed a statistically significant and clinically meaningful edge in improving patients' progression-free survival.Last month, Blenrep earned an approval in the U.K. Then earlier this week, officials in Japan endorsed the GSK regimen. The FDA has a target date of July 23 to decide on the Blenrep combo.Novartis pulls Lutathera request Meanwhile, Novartis’ bid to expand the label of its radiotherapy Lutathera in Europe is on hold after the company withdrew its filing, according to the CHMP. The committee explained that Novartis made the decision after “there were still some unresolved issues,” after the company answered a round of questions about the application, which was backed by a trial of 226 patients.The CHMP said that while the study found that Lutathera increased the amount of time patients lived without disease progression, “its impact on extending patients’ lives had not been established.” The committee concluded that the benefits of the treatment did not outweigh the risks. Lutathera has been on the market for eight years as a treatment for rare cancers of the digestive tract. The company had been seeking approval for Lutathera to treat adults with newly diagnosed unresectable or metastatic, well-differentiated high-grade somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors.Other CHMP decisions The CHMP also signed off on Autolus Therapeutics’ Aucatzyl as a treatment for relapsed or refractory B-cell precursor acute lymphoblastic leukemia. The FDA endorsed Aucatzyl in the indication in November of last year.Also making the grade at this week's CHMP meeting was SpringWorks Therapeutics’ MEK inhibitor Ezmekly for patients with symptomatic, inoperable neurofibromatosis type 1–associated plexiform neurofibromas (NF1-PN), which lead to tumor growth inside and along the nerves. The recommendation follows a February approval for the treatment in the U.S., where it goes by the commercial name of Gomekli. The CHMP also blessed Roche’s Itovebi as a combo treatment with palbociclib and fulvestrant as a first-line regimen for PIK3CA-mutated, estrogen-receptor (ER)-positive, HER2-negative locally advanced and metastatic breast cancer. The combo scored a nod from the FDA in October of last year.Receiving a thumbs-down from the CHMP were 4SC’s Kinselby, for the treatment of advanced stage mycosis fungoides and Sezary syndrome, and Fresenius Kabi’s Atropine sulfate for children with myopia. 

GSK's bid to bring back multiple myeloma therapy Blenrep (belantamab mafodotin) got a major boost on Friday when the antibody-drug conjugate (ADC) received a positive opinion from the EU's drug advisory panel. Also receiving a positive vote from the European Medicine Agency's Committee for Medicinal Products for Human Use (CHMP) was Roche's breast cancer drug Itovebi (inavolisib). Survival data for the PI3K inhibitor was recently spotlighted in an abstract released ahead of the American Society of Clinical Oncology (ASCO) meeting (see – ASCO25 abstract roundup: Roche's Itovebi cuts risk of death).Autolus' CAR-T cell therapy Aucatzyl (obecabtagene autoleucel) also received a thumbs up from CHMP, as well as mirdametinib, a MEK inhibitor recently acquired by Merck KGaA in its $3.9-billion takeout of SpringWorks Therapeutics. Blenrep is backBlenrep was withdrawn from global markets in 2022 following disappointing results from a confirmatory trial, but after positive readouts from two pivotal Phase III trials  — DREAMM-7 and DREAMM-8 — GSK has been working to bring the BCMA-targeted ADC back. Specifically, CHMP recommended Blenrep to treat relapsed or refractory multiple myeloma in combination with bortezomib and dexamethasone (BVd) for adults who have received at least one prior therapy; and in combination with Bristol Myers Squibb's Pomalyst (pomalidomide) plus dexamethasone for patients who have received at least one prior treatment, including BMS's Revlimid (lenalidomide).   In DREAMM-7, Blenrep plus BVd achieved a progression-free survival (PFS) of 36.6 months, versus 13.4 months for the comparator regimen of Johnson & Johnson's Darzalex (daratumumab) plus BVd.DREAMM-8 compared Blenrep to bortezomib, both in combination with Pomalyst and dexamethasone. At nearly 22 months follow-up, median PFS was not yet reached with Blenrep compared to 12.7 months in the control arm.In April, the UK became the first country to reauthorise Blenrep. Regulatory applications for the ADC are under review in a dozen other countries, including the US, Switzerland, Japan, China and Canada. The FDA is expected to make a decision by July 23, although there is concern that recent mass layoffs at the FDA could slow down the review process (see – Spotlight On: Could key drug approvals be put at risk of delay by FDA layoffs?).GSK is positioning the drug as a key growth driver, projecting peak annual sales exceeding £3 billion (see – Spotlight On: Can GSK deliver much needed pipeline momentum?).Itovebi impressesItovebi scored a positive opinion for its use in combination with palbociclib and fulvestrant to treat PIK3CA‑mutated, ER‑positive/HER2‑negative, locally advanced or metastatic breast cancer, following recurrence on or within 12 months of completing adjuvant endocrine treatment.The PI3K inhibitor won a similar approval in the US last October based on data from the Phase III INAVO120 study showing a 57% benefit for the Itovebi triplet on the primary endpoint of PFS compared to palbociclib and fulvestrant alone. Patients achieved PFS of 15 months in the experimental arm versus 7.3 months for placebo.At ASCO, Roche plans to share updated survival data for Itovebi. The abstract revealed that patients who received the triplet experienced a median overall survival of 34 months compared to 27 months for controls, representing a 33% reduction in the risk of death. Additionally, the combination therapy delayed the time to chemotherapy from 12.6 months in the control arm to 35.6 months with Itovebi.Go-ahead for Aucatzyl, mirdametinibA CHMP recommendation also went to Aucatzyl to treat patients aged 26 and older with relapsed or refractory B cell precursor acute lymphoblastic leukaemia (ALL).Autolus' CD19-directed cell therapy was cleared by the FDA in November for a similar indication, based on data from the Phase Ib/II FELIX trial. Of 65 patients with ALL who received the CAR-T, 27 (42%) achieved a complete response (CR) within three months, with a median CR duration of 14.1 months.Meanwhile, Merck KGaA's newly gained mirdametinib received a positive opinion to treat symptomatic, inoperable plexiform neurofibromas (PN) in paediatric and adult patients with neurofibromatosis type 1 (NF1) aged 2 years and above. If fully approved by the European Commission, it will have the brand name of Ezmekly.In February, the MEK inhibitor was approved in the US for the same patient population, where it's marketed as Gomekli.

在生物医药行业经历前所未有的低迷之际，“如何生存” 成为所有从业者必须面对的核心命题。近期一场主题为 “我们到那儿了吗？在持续的生物科技衰退中生存与发展” 的行业研讨会揭示，尽管市场被形容为 “奄奄一息”（moribund），但企业仍在通过战略调整、资本运作和聚焦核心管线寻找破局之道。一、市场寒冬：IPO 冰封与并购放缓的双重压力当前生物科技市场的严峻程度超乎想象。Latigo Biotherapeutics 首席执行官尼玛・法尔赞（Nima Farzan）用 “奄奄一息” 形容现状，COUR Pharmaceuticals CEO 丹妮尔・阿佩尔汉斯（Dannielle Appelhans）则认为市场处于 “避险模式”，nChroma Bio CEO 杰夫・沃尔什（Jeff Walsh）更是直言 “我们被困住了”。数据显示，除了强生年初以 146 亿美元收购 Intra-Cellular Therapies 的重磅交易外，全年仅剩下默克收购 SpringWorks Therapeutics（39 亿美元）和诺华收购 Regulus Therapeutics（17 亿美元）等少数案例，并购活动显著降温。IPO 市场更是陷入深度冻结。企业不愿在政策多变的环境中浪费资源，正如 Pliancy 增长副总裁诺亚・塔利亚费里（Noah Tagliaferri）指出：“当你试图进行公开募股时，每天都有新消息变化，这实在缺乏吸引力。”Latigo 曾在疫情期间的泡沫期尝试过上市，法尔赞坦言那段经历 “非常痛苦”，因此这次选择通过 1.5 亿美元的 B 轮融资 “私下成熟”，避免过早暴露在波动的公开市场中。二、药企策略调整：聚焦数据、合并求生与押注核心管线在融资和并购双重受限的情况下，企业的生存策略呈现三大方向：1.数据为王，用临床进展撬动资本COUR Pharmaceuticals 用十年时间验证了 “数据至上” 的逻辑。其早期将乳糜泻治疗资产授权给武田，获得近 5 亿美元非稀释性资金，如今凭借三条自主管线重新出发。阿佩尔汉斯强调：“对我们而言，口号是‘数据、数据、数据’，就像房地产依赖‘地段、地段、地段’一样。”Latigo 同样将 1.5 亿美元融资用于生成关键临床数据，尽管 Vertex Pharma 的同类药物获批带来竞争压力，但其聚焦疼痛治疗的管线仍吸引了 10 家新投资者加入 B 轮。2.合并重组：抱团取暖应对资本寒冬nChroma Bio 由 Chroma Medicine 和 Nvelop Therapeutics 合并而成，并伴随 7500 万美元融资。沃尔什坦言，合并过程充满挑战，但在资本和并购机会有限的情况下，这是 “必要的生存手段”。他认为行业应出现更多类似整合，“问题是很多公司没意识到必须这样做，而我们很庆幸挺了过来”。这种 “强强联合” 模式正在成为无法独立上市企业的重要选项。3.押注高潜力领域，规避政策风险尽管市场整体低迷，疼痛治疗、基因编辑等领域仍获资本青睐。Latigo 受益于 Vertex 新药获批引发的疼痛治疗领域复兴，而 nChroma 聚焦的表观遗传编辑平台则瞄准乙肝等难治愈疾病。法尔赞指出，疫情期间 “只要是精准肿瘤学就能融资” 的粗放时代已结束，现在投资者要求深度尽职调查，企业必须在细分领域展现不可替代性。三、外部环境挑战：政策不确定性与全球化竞争特朗普政府的 “最惠国”（MFN）药品定价政策成为新的变量。法尔赞推测，药企高管层正被政策变动牵制，导致并购决策放缓：“业务开发团队仍在接触生物科技公司，但高层可能因优先处理政策事务而搁置交易。” 与此同时，美国生物科技的全球领导地位正面临挑战，塔利亚费里警示：“美国的创新优势并非天生，必须保护人才库并鼓励投身生物科技。”值得注意的是，中国生物科技公司正成为跨国药企的合作目标，辉瑞、诺华等巨头通过授权交易获取中国本土创新，这既带来竞争压力，也凸显美国企业需在技术差异化上加大投入。四、行业展望：寒冬中的韧性与希望尽管当下充满挑战，参会高管仍对行业韧性抱有信心。沃尔什认为：“所有困境都会消退，我们将以更强姿态应对未来挑战。” 他指出，美国仍拥有充足资本和人才储备，关键在于企业需保持纪律性 —— 避免盲目扩张，聚焦真正有临床价值的管线。对于初创企业，阿佩尔汉斯建议：“不要等待市场好转，而是主动创造转机。” 无论是通过数据驱动融资、战略性合并，还是深耕未满足需求的领域，生存之战的核心始终是 “创新质量” 与 “资本效率” 的平衡。正如法尔赞所言，生物科技的周期性低谷终将过去，但只有那些能在寒冬中打磨出核心竞争力的企业，才能迎接下一个春天。在这场没有硝烟的生存竞赛中，生物科技行业正经历凤凰涅槃般的蜕变。数据、整合与战略聚焦，或许就是穿越寒冬的密钥。参考来源：https://www.biospace.com/business/in-biotechs-moribund-market-what-does-it-take-to-survive识别微信二维码，添加生物制品圈小编，符合条件者即可加入生物制品微信群！请注明：姓名+研究方向！版权声明本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观点，不代表本站立场。