排名前五的靶点	数量

CDK2(细胞周期蛋白依赖性激酶2)	7
c-Kit(干细胞生长因子受体)	2
HPK1(造血祖细胞激酶1)	2
FGFR3(成纤维细胞生长因子受体-3)	1
PIK3C2B(phosphatidylinositol-4-phosphate 3-kinase catalytic subunit type 2 beta)	1

关联

项与 Blueprint Medicines Corp. 相关的药物

Pralsetinib

普拉替尼

靶点

RET

作用机制

RET抑制剂

在研机构

基石药业 [+5]

原研机构

Blueprint Medicines Corp.

在研适应症

RET突变阳性甲状腺髓样癌 [+5]

非在研适应症

晚期非小细胞肺癌 [+8]

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2020-09-04

Avapritinib

阿伐替尼

靶点

PDGFRα x c-Kit

作用机制

PDGFRα抑制剂 [+1]

在研机构

Blueprint Medicines (Netherlands) BV [+3]

原研机构

Blueprint Medicines Corp.

在研适应症

胃肠道间质瘤 [+10]

非在研适应症

晚期恶性实体瘤 [+5]

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2020-01-09

Elenestinib

靶点

c-Kit

作用机制

c-Kit抑制剂

在研机构

Blueprint Medicines Corp.

原研机构

Blueprint Medicines Corp.

在研适应症

髓性系统性肥大细胞增多症

非在研适应症

侵袭性系统性肥大细胞增多症 [+2]

最高研发阶段临床2/3期

首次获批国家/地区-

首次获批日期-

项与 Blueprint Medicines Corp. 相关的临床试验

NCT07629960

/ Recruiting临床1/2期

A Phase 1/2, Open-Label, Dose-Escalation, Dose-Enrichment, and Dose-Expansion Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Antitumor Activity of BLU-924 (SAR449336) as Monotherapy and Combination Therapy in Participants With Advanced Pancreatic Cancer, Non-Small Cell Lung Cancer, or Colorectal Cancer Harboring KRAS Mutations

A first in human study to evaluate the safety, tolerability, pharmacokinetics, and antitumor activity of BLU-924 / SAR449336, a pan-KRAS inhibitor, in participants with advanced Pancreatic Cancer, Non-Small Cell Lung Cancer, or Colorectal Cancer harboring KRAS mutations.

开始日期2026-06-30

申办/合作机构

Blueprint Medicines Corp.

[+1]

NCT07264959

/ RecruitingN/A

An Observational Study in Patients With Indolent Systemic Mastocytosis

This is a Phase 4, non-interventional, observational study to collect detailed data on the clinical characteristics, clinical outcomes and medical management of ISM in real-world settings. The study will describe the demographic and clinical characteristics of ISM participants, including anaphylaxis and bone manifestations in ISM. Quality of life and disease control will be assessed through participant questionnaires. The study will also evaluate real world ISM treatment management, including use of avapritinib.

开始日期2026-04-15

申办/合作机构

Blueprint Medicines Corp.

NCT07388511

/ Completed临床1期

A Phase 1, Open-Label Study to Evaluate the Effect of Elenestinib on the Pharmacokinetics of Midazolam and Combined Oral Contraceptives, Levonorgestrel/Ethinyl Estradiol, in Healthy Adult Female Participants

The main objectives of this study are to determine the effect of elenestinib on the pharmacokinetic parameters (how the drug is absorbed, distributed, and processed by the body) of midazolam, and to determine the effect of elenestinib on the pharmacokinetic parameters of levonorgestrel/ethinyl estradiol, when given as a combined oral contraceptive.
Healthy adult participants will receive midazolam and levonorgestrel/ethinyl estradiol with and without elenestinib and have blood samples taken.

开始日期2026-02-05

申办/合作机构

Blueprint Medicines Corp.

100 项与 Blueprint Medicines Corp. 相关的临床结果

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0 项与 Blueprint Medicines Corp. 相关的专利（医药）

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项与 Blueprint Medicines Corp. 相关的文献（医药）

2026-06-01·JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY

Avapritinib improves cutaneous involvement in patients with indolent systemic mastocytosis: Results from the randomized, phase 2, interventional PIONEER study

Article

作者: Florell, Scott ; Ogbogu, Princess U ; George, Tracy I ; Broesby-Olsen, Sigurd ; Panse, Jens ; Siebenhaar, Frank ; Barete, Stéphane ; Bidollari, Ilda ; Reiter, Andreas ; Wada, David A ; Gotlib, Jason ; Schafhausen, Philippe ; Álvarez-Twose, Iván ; Dybedal, Ingunn ; Castells, Mariana ; Rafferty, Mark ; Cerquozzi, Sonia ; Shaheen, Daniel ; Akin, Cem ; Sabato, Vito ; DeAngelo, Daniel J ; Hong, Janet ; Livideanu, Cristina Bulai ; Rets, Anton ; Lin, Hui-Min ; Radia, Deepti H ; Van Daele, Paul ; Lampson, Benjamin ; Hartmann, Karin ; Ustun, Celalettin ; Ungerstedt, Johanna

BACKGROUND:

Indolent systemic mastocytosis (ISM), a clonal mast cell disease driven by the KIT D816V mutation, can often cause debilitating dermatologic symptoms.

OBJECTIVE:

Assess improvement of ISM-related skin manifestations after treatment with avapritinib, a highly selective KIT D816V inhibitor, vs placebo in Part 2 of the PIONEER study (NCT03731260).

METHODS:

Patients with moderate to severe ISM received avapritinib 25 mg once daily (n = 141) or placebo (n = 71). Endpoints included skin lesion area and pigmentation at week 24, skin mast cell burden, and change in symptoms.

RESULTS:

Mean percent reduction in lesional surface area was -36.6% with avapritinib vs -1.8% with placebo in the most affected area; 86% vs 0% had improved skin lesion color. Mean percent change in skin mast cell burden decreased with avapritinib (-22.1%) vs placebo (10.1%). Avapritinib vs placebo significantly improved skin symptom domain score (mean change -7.2 vs -2.8; P < .0001), including the individual skin symptoms itching, flushing, and spots. Avapritinib was well tolerated.

LIMITATIONS:

Photography was optional, so analysis population for lesion area and color were smaller (n = 111) than the overall study population (n = 212).

CONCLUSION:

Avapritinib treatment improved dermatologic symptoms, decreased skin lesion size, normalized skin lesion color, and reduced skin mast cell burden in patients with ISM.

2026-05-01·The journal of allergy and clinical immunology. Global

Symptom patterns before and after indolent systemic mastocytosis: A matched cohort analysis

Article

作者: Yuen, Chris ; Lampson, Benjamin ; Atif, Hiba ; Saparudin, Mary ; Tse, Kevin Y ; Avila, Chantal ; Chen, Wansu ; Puttock, Eric J ; Zeiger, Robert S ; Zhou, Matt ; Sullivan, Erin ; Powell, Dakota ; Xie, Fagen ; Miller, Kerri

Background:

Systemic mastocytosis (SM) is a clonal mast cell disorder driven in most cases by KIT D816V mutation. The evolution of symptoms before and after diagnosis remains poorly understood.

Objective:

We sought to longitudinally describe various aspects of the real-world patient journey in the year leading up to and the year following diagnosis of SM.

Methods:

This retrospective, observational study included a previously confirmed cohort of patients with SM within a large integrated health system. We assessed physician-coded diagnoses, dispensing of medications, and patient-reported symptoms during the year before and after diagnosis, supplementing manual review with natural language processing outputs to enhance accuracy of symptom identification. Findings were compared with 2 matched comparison groups: patients with chronic spontaneous urticaria (CSU) and patients without SM or CSU.

Results:

Among 75 patients with SM (59 nonadvanced, 16 advanced), CSU patients with CSU, and 150 patients without SM or CSU, SM was associated with higher frequencies of cardiovascular disease, hepatosplenomegaly, osteoporosis, anemia, thrombocytopenia, eosinophilia, and elevated serum tryptase compared with both comparison groups. Gastrointestinal and neuropsychiatric symptoms were consistently more common in patients with SM, often persisting or worsening after diagnosis despite treatment. Advanced SM was distinguished by more pronounced hematologic and gastrointestinal abnormalities, whereas nonadvanced SM exhibited greater prevalence of cutaneous and neuropsychiatric symptoms.

Conclusions:

Patients with SM often present with heterogeneous, nonspecific symptoms that overlap with several other conditions, contributing to substantial diagnostic delays. Our findings underscore the need for more inclusive diagnostic approaches and improved management strategies that address the full spectrum of patient experience before and after diagnosis.

2026-04-23·JOURNAL OF MEDICINAL CHEMISTRY

Design and Synthesis of BLU-654, a Potent and Selective Mutant KIT V654A Inhibitor for the Treatment of Imatinib-Resistant GIST

Article

作者: Dineen, Thomas A. ; Choi, Yeon Sook ; Perola, Emanuele ; Moine, Ludivine ; Hu, Wei ; Barvian, Kevin ; Kim, Joseph L. ; Davis, Alison ; Guo, Jian ; Ahmad, Omar K. ; Grassian, Alexandra

Gastrointestinal stromal tumor (GIST) is the most common type of sarcoma of the gastrointestinal tract, with approximately 5000 new cases annually in the USA. Approximately 80% of GIST cases are driven by activating mutations in KIT in exon 9 or 11. Resistance to present therapies like imatinib often arises from secondary KIT mutations, especially V654A (exon 13), which is the most frequent resistance mutation. Tyrosine kinase inhibitors (TKIs) currently approved for GIST can cause dose-limiting side effects due to off-target inhibition of other kinases. Herein, we report the discovery and optimization of BLU-654 (compound 18), a highly potent and kinome-sparing KIT V654A inhibitor. Preclinical efficacy studies demonstrated its prolonged antitumor activity in a KIT V654A cell-derived xenograft mouse model. BLU-654 offers a potent and selective profile suitable for combination therapy for KIT-mutant GIST patients.

799

项与 Blueprint Medicines Corp. 相关的新闻（医药）

2026-07-03

·跨境药研所

一支基础胰岛素,是怎么从赛诺菲的"印钞机",变成人人买得起的"平价刚需"的?

导语有些药,救的是命,赚的是钱,写的却是一部行业史。来得时(Lantus)就是这样一支针。它让千万糖尿病人平稳地活着,也让赛诺菲稳稳地赚了四分之一个世纪;它曾是贵到让人掰着用的"奢侈品",如今又被一路砍成人人够得着的"平价刚需"。这支针的二十五年,几乎把现代医药商业里所有的门道——专利、定价、医保、集采、颠覆——都演了一遍。这篇长文,我们就顺着它的沉浮,把这门"救命生意"的底层逻辑,一层层拆开给你看。一 | 同一支针,两种人生:从三位数账单到 35 美元封顶 ▲ 曾经的月度账单动辄三位数,如今被一刀压到 35 美元封顶先讲个场景。在美国,一位普通的 2 型糖尿病人,过去每个月要为一支叫"来得时"(Lantus)的长效胰岛素,面对一张动辄两三百美元的账单。这药不是可有可无的保健品——不打,血糖压不住;打,钱包扛不住。于是在很长一段时间里,美国社交媒体上流传着一种黑色幽默:有人把胰岛素针剂掰着用,有人开车去加拿大"代购",还有人干脆在众筹平台上为自己的救命针发起募捐。这不是段子。糖尿病人对基础胰岛素的依赖,是一种近乎"呼吸"式的刚性——它不治愈任何东西,只是日复一日地替你把血糖的底盘稳住。正因为刚需到了这个份上,它的价格才格外扎眼:同样一支来得时,在美国的标价一度是欧洲、加拿大的好几倍。然后,转折来了。 2023 年 3 月,赛诺菲宣布把来得时在美国的官方列价一次性砍掉 78%;2024 年 1 月正式生效后,一支 10 毫升的西林瓶列价降到约 64 美元,预填充笔约 96 美元,同时对所有商业保险患者的自费部分设了每月 35 美元的封顶线。加上更早之前对无保险人群"从 99 美元降到 35 美元"的安排,这支药几乎在一夜之间,从"掏空钱包"变成了"一顿饭钱"。真正让来得时跌下神坛的,从来不是一纸专利到期通知,而是医保规则、政治压力与舆论合力扣下的那记扳机。这里藏着本文想讲的第一个反差:一款药的商业命运,往往不由它的疗效决定,而由它所处的支付体系决定。来得时的疗效二十多年没变,变的是它被谁定价、被谁买单、被谁盯上。一支针的沉浮,照见的是整个医药商业逻辑的底层规则。再往深一层看,这场降价背后还站着一整排推手。近几年,美国国会山上关于胰岛素价格的听证会一场接一场,参议员桑德斯反复把三大胰岛素巨头(礼来、诺和诺德、赛诺菲)拎出来"公开处刑",质问它们为什么把一款百年老药卖到普通人负担不起。舆论层面,"胰岛素比房租还贵""为了省钱少打针而丢命"的报道屡见报端。政策层面,《通胀削减法案》先给医保(Medicare)人群划下了每月 35 美元的自付上限,一项针对医保补助(Medicaid)的通胀返利新规又让药企继续大幅涨价变得"得不偿失"。三股力量拧成一股绳,巨头们几乎是被推着、赶着、你追我赶地在三周之内先后宣布降价——礼来先动,诺和诺德跟进,赛诺菲压轴。这里有个格外讽刺的注脚:胰岛素的发现者班廷,当年把专利以象征性的一美元卖给多伦多大学,只因为他认为"胰岛素属于全世界,而不该用来牟利"。一百年过去,他的这份初心,竟要靠一场夹杂着政治博弈、舆论声讨和医保新规的"合力围剿",才在美国被勉强找回一角。一支针能不能让人买得起,从来就不是纯粹的市场问题。好了,情绪归情绪,我们把镜头拉远。要真正读懂来得时这条抛物线,先得看清它脚下这门生意到底有多大。术语解读基础胰岛素也叫"长效胰岛素",负责在没有进食时提供一个平稳、持续的"底层"胰岛素水平,把空腹和夜间的血糖压住。它对应的是"餐时胰岛素"(短效/速效)——后者管吃饭那一下的血糖高峰。来得时正是基础胰岛素里的经典代表。二 | 全景:糖尿病到底是一门多大的生意？要理解一支针的价值,得先看它站在多大的池塘里。答案是:一个还在持续变大的、深不见底的池塘。 ▲ 数据来源:IDF《全球糖尿病地图》第11版(2025)、赛诺菲财报、中国医保局国际糖尿病联盟(IDF)2025 年发布的第 11 版《全球糖尿病地图》给出了一组让人心里一沉的数字:全球 20—79 岁成年人中,已有约 5.89 亿人患糖尿病,相当于每 9 个成年人里就有 1 个;按现有趋势,到 2050 年这个数字会攀到约 8.53 亿,再涨约 46%。其中九成以上是 2 型糖尿病,而 2 型,正是基础胰岛素的主战场。更刺眼的是钱:糖尿病相关的全球年度卫生支出已经突破 1 万亿美元,17 年间暴涨了 338%。换句话说,这不是一门会自然萎缩的生意,而是一条随着人口老龄化、城市化和生活方式变迁不断变宽的河。药企们争的,不是要不要下场,而是在这条河里能占多长一段河道。 “糖尿病是极少数"患者只会越来越多、且几乎终身用药"的疾病——这既是它的人道悲剧,也是它长盛不衰的商业底色。那么赛诺菲靠来得时挣的是什么钱? 说穿了,是一门"卖水"的生意。淘金热里最稳的不是挖矿的,是卖水、卖铲子的。糖尿病治疗的"金矿"是患者对稳定血糖的终身需求,而基础胰岛素就是那桶每天都要喝的水。它不需要治愈,只需要"每天都用、用一辈子"。这决定了它的商业模式:靠庞大的存量患者、靠复购、靠渗透率,而不是靠一次性的"神药时刻"。但"卖水"也分好坏。真正让赛诺菲把来得时做成现象级大单品的,是它在三件事上同时占了先手: 第一,产品本身有代际优势——它是第一个真正意义上的"无峰"长效基础胰岛素,一天一针就能覆盖近 24 小时; 第二,它踩中了"胰岛素类似物"替代"人胰岛素"的升级周期,把老一代产品的用户往上迁; 第三,也是最关键的,它在美国这个全球利润最肥的市场里,建立了一套围绕医保目录、药品福利管理方(PBM)和回扣的复杂分销体系。前两件事决定了它能卖,第三件事决定了它能卖出高价。这套分销体系值得单独拆一拆,因为它是理解"为什么美国胰岛素这么贵"的钥匙。在美国,礼来、诺和诺德、赛诺菲三家常年合计握着约九成的胰岛素市场——这是一个典型的寡头格局。寡头之间不打明面上的价格战,反而心照不宣地一起把"列价"往高抬。因为真正决定一款胰岛素能不能进保险目录、能不能被优先报销的,不是列价本身,而是药企私下给 PBM 的回扣有多丰厚。于是形成了一个荒诞的循环:列价越抬越高,回扣越给越多,保险公司和 PBM 拿走差价,而夹在中间、没有议价能力的无保险者和高自付患者,却要照着那个虚高的列价掏钱。来得时的"账面高价"和"实际净价"之间,长期隔着一条巨大的鸿沟。这也解释了赛诺菲那次降价的另一面。把列价砍掉 78%,看上去是"大出血",但因为列价本就虚高,砍完之后它反而能少付一大笔按列价计算的医保返利——有测算认为,这一手甚至可能让赛诺菲在来得时上少付数亿美元返利、并额外多赚一笔利润。所以你看,连"降价"这种看似让利的动作,背后也是一本精算到小数点后的商业账。这就是重磅药的生意本质:它从来不是"一手交钱一手交药"那么简单,而是一张由列价、净价、回扣、返利、目录位次层层缠绕的复杂网。再补一个常被忽略的细节:胰岛素卖的不只是"药",还是"器械"。来得时的主流卖法是预填充注射笔(SoloStar),一次性、好上手、适合居家自用。这套"笔"的设计、专利和使用习惯,本身就构成一道隐形壁垒——患者一旦用惯了某个品牌的笔,切换成本并不低。这就像剃须刀和刀片的生意:药是刀片,笔是刀架,黏住用户的往往是后者。生物类似药即便分子做得再像,也得在装置体验和使用惯性上重新和患者磨合。把这三件事放到同一条赛道上看,就更清楚了。长效基础胰岛素这条赛道,从来不是来得时一家独大,而是"五强对垒"。下面这张表,把同赛道几个关键玩家的定位摊开来对比——你会发现,它们卖的几乎是同一类东西,活法却各不相同。 ▲ 长效基础胰岛素五强定位对比(资料来源:各公司公开资料、行业公开报道) 看懂这张表,你就理解了来得时的处境:它是这条赛道的"开山鼻祖",却也是被后来者反复围猎的那个靶心。赛诺菲自己的 Toujeo(甘精 U300)想接它的班,诺和诺德的诺和达(德谷胰岛素)用"更长效更灵活"抢它的高端用户,国产甘李药业的长秀霖用价格啃它的下沉市场,而 Basaglar、Semglee 这些生物类似药干脆贴着它的分子做平价替身。一款好产品的宿命,就是把整条赛道的对手都培养成了自己的掘墓人。还有一个不能回避的背景:胰岛素市场长期是一个"三足鼎立"的全球寡头格局——礼来、诺和诺德、赛诺菲三家,各自握着一两款拳头产品,合力掌控着全球尤其是发达市场的绝大部分份额。这种结构带来了效率,也带来了争议: 一方面,它保证了产品质量和供应的稳定; 另一方面,长期缺乏真正的价格竞争,也是全球尤其是美国胰岛素价格畸高、许多中低收入国家患者"用不起救命药"的重要原因。世界卫生组织和多国政府这些年反复呼吁改善胰岛素的可及性,正是冲着这个格局去的。来得时的降价与集采,某种程度上,也是这场"让救命药回归可负担"的全球运动里的一个缩影。术语解读 PBM 与回扣(Rebate)在美国,药品福利管理方(PBM)是横在药企和患者之间的"中间商",负责替保险公司谈判药价。药企往往先把"列价"标得很高,再私下给 PBM 一笔"回扣"来换取进医保目录的好位置。结果就是:列价虚高、净价打折,而没保险或高自付的患者,常常要按虚高的列价掏钱。这套机制是美国胰岛素价格畸高的核心推手之一。三 | 拆解:一支甘精胰岛素,凭什么卖了二十多年？先把它的身份证亮出来：来得时,通用名甘精胰岛素(insulin glargine),由赛诺菲的前身(法国安万特、更早的赫斯特·马里恩·鲁塞尔)在德国法兰克福的生物技术中心研发,2000 年 4 月获美国 FDA 批准、6 月获欧盟批准上市,适用于 1 型和 2 型糖尿病的基础血糖控制。它一度是德国最重要的出口药品之一。它的技术内核,其实是一个精巧的"pH 小魔术"。研究者把人胰岛素的分子动了两处小手术：A 链第 21 位的天冬酰胺换成甘氨酸，B 链尾部再接上两个精氨酸。这两处改动把分子的"等电点"从大约 5.4 右移到 6.7。听起来很玄,落到实际就一句话——它在偏酸性(pH 约 4)的澄清药液里能稳稳溶解、乖乖待在笔芯里;一旦被注射到皮下(那里的 pH 接近 7.4),它立刻"水土不服",析出成细小的微沉淀,像一块慢慢融化的方糖,持续、平缓地把胰岛素单体释放进血液,做到近 24 小时几乎"无峰"的平稳降糖。 ▲ 来得时预填充注射笔(SoloStar)与 10 毫升西林瓶示意图 · 100 单位/毫升一处氨基酸的替换,换来了近 24 小时的平稳血糖——来得时真正的护城河,一开始就写在了分子结构里。正是这个"无峰、平稳、一天一针"的特性,让它相比更早的中效人胰岛素(比如 NPH)大幅降低了夜间低血糖的风险,患者依从性更好。对一款要用一辈子的药来说,"少半夜被低血糖惊醒"这件事,就是实打实的临床价值。这也是它能在竞争中站稳二十多年的根本。还有一层被外行低估的壁垒,是"制造"本身。很多人以为专利一过,仿制版就该像感冒药那样满地都是、价格立刻跳水。但胰岛素是生物大分子,靠活细胞发酵表达,纯化、灌装、质控的工艺极其复杂,建一条合规产线动辄要投入数亿乃至上十亿的资金和好几年时间。这就是为什么,即便甘精胰岛素的核心专利早在 2014、2015 年前后就到期,真正能造出合格生物类似药、并通过监管审评的厂家,依然屈指可数;头几年入场的,也就是礼来的 Basaglar/Abasaglar、Viatris 的 Semglee 这么几家。 "造得出、造得稳、造得便宜",本身就是一道把大多数追赶者挡在门外的高墙。原研药企正是靠着这道墙,为自己争取到了宝贵的缓冲期。再看它的商业地理。来得时的钱,并不是均匀地从全球赚来的。美国长期是它最肥的利润池——同样一支药,美国的净价远高于欧洲和新兴市场。欧洲市场规矩、价格受管制,利润薄但稳定; 新兴市场(包括中国、印度、拉美)则是它后期增长的主要来源,靠的是渗透率的提升和患者基数的膨胀。这种"美国供利润、新兴市场供增量"的结构,在它上升期是加速器,可到了下坡阶段,反而成了放大器: 美国的回扣战和目录调整先砍掉它的利润,中国的集采再削掉它的价格,两头一起使劲,曲线自然就陡了。再看它的商业曲线。来得时的销售额,画出来是一条教科书般的抛物线。上市后一路爬坡,到 2014 年触及巅峰——全球销售额约 74 亿欧元,折合约 88 亿美元。这是什么概念? 一款单品的年销售额,已经超过不少中小型国家的年度财政收入。它长期贡献着赛诺菲糖尿病业务的半壁江山,是名副其实的"印钞机"。 ▲ 来得时全球销售额:依据公开财报数据近似重构(单位:欧元) 然后就是下坡。 2014—2015 年前后,它在美国和欧洲的核心专利陆续到期;紧接着,生物类似药入场、PBM 把它从优选目录里踢出、竞品用回扣战术抢份额……销售额从巅峰的 70 多亿欧元,一路滑到 2023 年的约 14 亿欧元。九年时间,蒸发掉近八成。这条抛物线,几乎浓缩了所有"重磅原研药"的共同命运——学名叫"专利悬崖"。区别只在于:有的药一到期就断崖跳水,有的药靠品牌、靠临床惯性、靠剂型升级,把下坡的坡度磨得缓一些。来得时属于后者,它没有一夜清零,但也没能逃过引力。而真正决定它"摔得多惨"的,不是分子本身,是它接下来要面对的三重外力:生物类似药、支付方,以及一个谁都没料到的搅局者。术语解读专利悬崖(Patent Cliff)指原研药的专利保护到期后,仿制药或生物类似药蜂拥而入,原研药销售额在短时间内大幅下滑的现象,曲线上像一道悬崖。它是几乎所有重磅药物都躲不过的宿命,也是药企必须靠"下一代产品"和"新管线"来对冲的头号风险。四 | 护城河:一支老药,凭什么还没被抄死？专利到期,不等于游戏结束。恰恰相反,专利悬崖之后,才是真正考验一家药企"护城河"深浅的时候。赛诺菲围着来得时,前前后后修了四道防线。 ▲ 从分子设计到管线转型:赛诺菲围绕甘精胰岛素的四道防线第一道,是剂型迭代。早在专利到期前,赛诺菲就备好了"接班人"——Toujeo(甘精胰岛素 U300),把浓度提高到每毫升 300 单位,注射体积更小、释放更平缓,主打"夜间低血糖更少"。它的算盘很清楚:趁着来得时的用户还在,把他们平滑迁移到一款新专利保护的产品上,让品牌红利再续一程。这是原研药企对抗专利悬崖最经典的一招——"自己迭代自己"。第二道,更狠,是"自我颠覆"。生物类似药迟早要来,与其被别人用平价版抢走市场,不如自己先出一个平价版。赛诺菲在美国率先推出了一款无品牌的甘精生物制品,定价比来得时列价低约六成。用大白话说,就是"左右手互搏":用自己的低价产品去堵别人低价产品的路,哪怕牺牲一部分利润,也要把渠道和患者攥在自己手里。当模仿者必然到来时,最聪明的防守不是筑更高的墙,而是抢在对手之前,亲手把自己的城墙拆出一个平价的缺口。第三道,是把基础胰岛素塞进新赛道——组合疗法。赛诺菲把甘精胰岛素和一款 GLP-1 类药物(利司那肽)做成复方针剂 Soliqua,一针同时管两件事。这么做,一方面延长了甘精分子的生命周期,另一方面也是在向当时已经隐隐崛起的 GLP-1 大潮"投诚":打不过,就先把对方的机制拉进自己的产品里。第四道,也是格局最大的一道,是整个公司的"战略换血"。赛诺菲这些年推行一套叫"Play to Win"(志在必得)的战略,核心就一句话:把资源从回报率下降的老业务(比如成熟的糖尿病、消费者健康)里抽出来,重仓押注免疫学和疫苗。它把当家花旦从来得时换成了免疫学重磅药 Dupixent(度普利尤单抗)——后者的销售峰值目标被一路上调到 130 亿欧元以上,横跨十余个适应症。它还把消费者健康业务 Opella 分拆出售、接连收购 Blueprint、Vigil、Dynavax 等公司,给管线补血。看懂这四道防线,你就明白了一件事:对赛诺菲来说,来得时早已不是"未来",而是"底盘"。它贡献稳定的现金流,却不再是增长引擎。公司真正的赌注,已经押到了免疫学的牌桌上。这是所有老牌药企都要经历的"换发动机"——一边靠老药的现金流活着,一边拼命在新赛道上找下一个"来得时"。区别只在于,谁换得更早、更狠、更准。值得多说一句的是这场"换发动机"的力度。赛诺菲的当家新品 Dupixent,已经从一款单一适应症的皮炎药,滚雪球般拓展到特应性皮炎、哮喘、慢阻肺、鼻窦炎等十余个适应症,成了公司增长的绝对主引擎; 与之配套,它把免疫学领域一批处于中后期、单品峰值潜力动辄二三十亿欧元的管线资产同时往前推,还立下了到 2030 年免疫学业务做到二百多亿欧元、疫苗做到上百亿欧元的军令状。为了给这场豪赌腾钱腾人,它一边压缩成本、剥离出售消费者健康业务 Opella,一边靠一连串并购(Blueprint、Vigil、Dynavax 等)给管线补血。一句话:赛诺菲不是在"经营来得时",而是在"用来得时经营未来"。这背后其实是一个更冷峻的产业规律：在今天的制药业,慢病领域(尤其是已经高度成熟、且被集采和生物类似药双向挤压的糖尿病基础用药)越来越像"红海里的公用事业",利润薄、增长慢、政策风险高；而真正能撑起估值和想象空间的,是免疫、肿瘤、罕见病这些"高壁垒、高定价、高增长"的新蓝海。赛诺菲围绕来得时修的四道护城河,与其说是为了"守住这款药",不如说是为了"体面地、有节奏地从这款药身上撤退",把撤下来的资源,投到能打赢下一场仗的地方去。除了这四道明面上的防线,还有一道很少被写进财报、却真实存在的"隐形护城河"——医生和患者的使用惯性。一款用了二十多年的老药,积累了海量的真实世界使用经验、指南推荐和处方习惯。医生开惯了、患者用惯了、剂量调教熟了,这种惯性本身就是一种黏性。哪怕生物类似药便宜一截,要撬动一位血糖控制稳定、已经用顺手的老患者去换药,阻力也远比想象中大。正是这种"临床惯性",让来得时即便在专利到期、平价替身环伺的情况下,依然能守住一块相当可观的存量市场,把下坡的坡度磨得更缓——这也是所有经典老药共同的、最后的倔强。术语解读生物类似药 / 可互换生物类似药生物类似药,是与已上市生物原研药(如来得时)高度相似、疗效和安全性无临床意义差异的"仿制版",但因为是大分子、工艺复杂,做不到化学仿制药那种"完全一致"。而"可互换"(interchangeable)是美国 FDA 的一个更高认证——拿到它,药师可以在不经医生同意的情况下,直接把原研药替换成这款生物类似药。Semglee 就是首个获此认证的甘精胰岛素,平价替代的杀伤力因此更大。五 | 中国战场:集采一声令下,量价乾坤大挪移如果说美国市场教会来得时"怕政治与舆论",那么中国市场教会它的,是"怕集采"。 2021 年 11 月,中国启动了第六批国家组织药品集中带量采购——这一批很特殊,专门针对胰岛素,而且是国家层面第一次把"生物药"纳入集采。规则简单粗暴又高效:国家把全国公立医院的巨大采购量打包成订单,谁愿意降价换量,谁就能拿走这块蛋糕。胰岛素被分成六个组(餐时人胰岛素、基础人胰岛素、预混人胰岛素,以及三种对应的胰岛素类似物),甘精、地特、德谷这类长效基础类似物被归在同一组同台竞价。要理解这一步的分量,得先知道集采这套打法是怎么一路走来的。它最早在 2018 年从几个试点城市起步,针对的都是专利到期的化学仿制药——逻辑是"同一个通用名、质量过一致性评价的产品,凭什么价差好几倍",于是用国家采购量把价格往地板上摁。此后一路扩围到全国、扩展到几十个批次。但胰岛素这种生物药,长期被认为"太难集采":它不是简单的化学分子,不同厂家的产品在装置、纯度、临床习惯上都有差异,很难像仿制药那样直接"一刀切"比价。武汉等地先做了地方试点趟路,直到 2021 年,国家才第一次啃下这块硬骨头,专门为胰岛素设计了一套分组竞价、按报价高低把中选企业分成 A 到 D 不同梯队、并给出最高有效申报价的复杂规则,才把这块"最难集采的生物药"装进了框里。结果是一场量价的乾坤大挪移。 42 个产品中选,全场平均降幅约 48%,单品最大降幅高达约 74%(由礼来的一款预混产品报出)。来得时选择了"大降价保份额"的打法,甘精产品降价约 66%,靠着这记"割肉",守住了相当一部分公立医院的市场份额。据测算,这一轮集采首年采购量超过 2.1 亿支,预计为国家医保省下约 90 亿元。 ▲ 2021 胰岛素国家集采:一次砍价砍出了什么(数据来源:新华社、PharmCube、中国医保局) 集采的逻辑,从来不是"把外资赶走",而是用整个国家的采购量做筹码,逼所有玩家在同一条价格线上重新排队。但"保住份额"和"保住利润"是两码事。赛诺菲当时就预告,受集采影响,其甘精胰岛素在中国的销售额预计会下滑约三成——这是典型的"以价换量":卖得更多了,赚得却更少了。而在天平的另一端,是国产厂商的集体上位。甘李药业(长秀霖)、通化东宝这些本土玩家,凭借更低的成本和更狠的报价,在集采里大举拿量。到 2025 年前后,国产厂商合计已经拿下中国公立医院胰岛素市场过半的用量份额。这里就是本文的爆点所在:对普通患者而言,集采无疑是天大的好事——同样的甘精胰岛素,自付一下子少了一大截,救命药真正变成了"平价刚需"; 可对赛诺菲这样的外资原研厂商而言,中国市场的游戏规则被彻底改写了。它不再是那个"靠品牌溢价躺着数钱"的市场,而变成了一个"要么大幅降价换量、要么把高端产品(比如 Toujeo)另辟战场绕开集采"的残酷战场。于是你能看到赛诺菲在中国的两手策略: 一手,用来得时在集采里"以价换量",守住基本盘; 另一手,拼命把用户往未纳入集采、仍有溢价空间的 Toujeo(多睦达)上引导,同时押注糖尿病之外的新品(比如用于延缓 1 型糖尿病进展的 Tzield,已于 2025 年获中国药监部门批准)。说到底,在中国这块土地上,来得时的角色已经从"利润奶牛"降级成了"份额压舱石"。把视角切换到患者这一端,这笔账才算真正算明白。集采之前,一支甘精胰岛素在中国零售端要几十上百元,一位需要长期用药的糖尿病人,一年的药费是一笔实实在在压在家庭账本上的开支; 集采之后,同一支药的价格被腰斩甚至打到三折以下,叠加医保报销,很多患者每月的自付负担降到了以前的零头。对一个要用一辈子药的慢病群体来说,这种降幅不是"省点小钱",而是"能不能长期规范治疗、会不会因为舍不得钱而偷偷减量"的分水岭。从这个意义上说,集采真正的价值,不只是替医保省了钱,更是把一部分本来在"用不起"边缘挣扎的患者,重新拉回了规范治疗的轨道。当然,故事没有到此为止。集采不是一锤子买卖,而是有周期、要续标的。首轮协议到期后,续采的规则、企业的报价、以及"保供应还是保利润"的权衡,都会再博弈一轮。对赛诺菲这样的外资原研厂商而言,每一次续标都是一道选择题:是继续跟着国产厂商把价格往下压、死守医院渠道,还是干脆把重心转向院外市场、零售药房和高端分层产品,绕开这台"降价绞肉机"。这道题没有标准答案,但可以肯定的是——在中国,靠一款老胰岛素"躺着数钱"的时代,已经一去不复返了。术语解读带量采购(VBP / 集采)全称"药品集中带量采购"。核心是国家或地方医保部门把公立医院的采购量集中打包,以"确定的采购量"换取"更低的价格",通过竞标让企业大幅降价中选。它对患者是减负,对企业则是"以价换量"的生死考验——降得不够狠就丢标丢份额,降得太狠又伤利润。2021 年的胰岛素专项集采,是中国首次把生物药纳入这一机制。六 | 未来十年:守成,还是被"减肥针"改写？把时间线拉长,来得时这二十多年的沉浮,其实可以浓缩成五个节点:2015 年专利到期、2021 年中国集采、2024 年美国大降价……以及,一个正在改写整个代谢病版图的新变量。 ▲来得时命运的五个关键节点,以及正在逼近的 GLP-1 大潮这个新变量,就是眼下最火的 GLP-1 受体激动剂——诺和诺德的司美格鲁肽(Ozempic / Wegovy)和礼来的替尔泊肽(Mounjaro / Zepbound)。它们本是为 2 型糖尿病开发的,却因为能显著减重,一跃成了全球最炙手可热的药物类别。仅 2024 年,诺和诺德与礼来两家的 GLP-1 相关收入合计就高达约 457 亿美元;有机构预测,整个 GLP-1 市场规模会从 2025 年的约 644 亿美元,一路冲到 2033 年的约 1708 亿美元。 GLP-1 正在做一件对基础胰岛素而言最危险的事:它同时抢走了医生的处方笔、患者的注意力,和支付方的预算。这对基础胰岛素意味着什么? 短期看,胰岛素依然是刚需——GLP-1 替代不了所有需要注射胰岛素的患者,尤其是 1 型糖尿病和病程较晚的 2 型患者。但从中长期看,GLP-1 正在把大量原本可能"升级"到胰岛素的 2 型患者截流在更前面的治疗环节,同时把整个糖尿病领域的研发投入、医生心智和医保预算,大规模地吸走。当一个新物种以这样的速度扩张时,老一代产品的增长天花板,只会越压越低。更要命的是,GLP-1 的杀伤力还在"外溢"。它最初是降糖药,后来被发现能大幅减重,一脚踏进了规模更大的肥胖市场; 再往后,心血管获益、慢性肾病、睡眠呼吸暂停、脂肪肝等新适应症一个接一个被验证,支付方和保险开始愿意为它买单的场景越铺越广。与此同时,口服版 GLP-1、以及减重效果更猛的双靶点、多靶点新药也在路上。这意味着它抢的不只是"糖尿病用药"这一小块蛋糕,而是在重塑整个代谢病乃至慢病治疗的版图。基础胰岛素在这张越来越大的新版图里,位置只会越来越靠后、越来越"基础设施化"。有意思的是,连"平价化"这件事也要轮到 GLP-1 头上了。司美格鲁肽的专利将从 2026 年起在印度、中国、巴西等多个大市场陆续到期,仿制版和生物类似药已经在门口排队。这几乎是来得时故事的重演——只不过这次,站在"专利悬崖"边上的,换成了当年掀翻它的那批新贵。历史不会简单重复,但总在押着相似的韵脚:今天最风光的重磅药,大概率就是十年后被集采和生物类似药按在地上摩擦的下一个"来得时"。这也正是赛诺菲当年那个决定的高明之处——与其在糖尿病这条已经能望见结局的路上死磕,不如趁早换到免疫学的新牌桌。更微妙的是,连"平价化"这件事也要轮到 GLP-1 头上了。司美格鲁肽的专利将从 2026 年起在印度、中国、巴西等多个大市场陆续到期,仿制版和生物类似药已经在门口排队。这几乎是来得时故事的重演——只不过这次,站在"专利悬崖"边上的,换成了当年掀翻它的那批新贵。历史不会简单重复,但总在押着相似的韵脚。那么来得时的下一个十年会怎样? 我的判断是三句话。 ▸其一,它不会消失,而会彻底回归"平价刚需"的底盘角色——全球范围内价格继续被集采、被政策、被生物类似药往下压,量还在,利润薄了。 ▸其二,它在中国的故事,本质是"外资守成 vs 国产替代"的持久战,甘李、通化东宝们会继续用成本优势蚕食份额,而赛诺菲会尽量把战火引向高端和创新。 ▸其三,对赛诺菲这家公司而言,来得时早已不是叙事的主角——它更像一位功勋卓著的老将,负责在后方稳稳供着现金流,让公司腾出手去打免疫学和疫苗那场更大的仗。一支针,养活一家跨国药企四分之一个世纪;从巅峰 88 亿美元,到被砍成人人够得着的地板价。来得时的沉浮,不是一款药的成败,而是一部浓缩的现代医药商业史——它写着专利的红利与代价、支付体系的权力、政策之手的分量,也写着"再强的护城河,也挡不住时代换赛道"的残酷与公平。回过头看,来得时留给行业的启示,或许比它本身的商业成绩更值钱。它告诉后来者:一款重磅药的黄金期是有限的,真正的高手,是在巅峰时就开始为下坡铺路——迭代剂型、自我颠覆、储备管线、切换赛道,一步都不能等。它也提醒所有人:在慢病这样的"民生刚需"面前,纯粹的市场定价终究要让位于政策与公共利益,今天你能靠信息差和支付体系赚到的超额利润,明天大概率会被集采、被医保、被舆论一点点还回去。这不是某一家药企的委屈,而是整个行业必须接受的新常态。看清了这一点,来得时的故事就不再只是一段"由盛转衰"的挽歌,而是一面照见未来的镜子。对患者,它从奢侈品变回了必需品,这是进步;对企业,它从印钞机变回了压舱石,这是宿命。一支针的两面,恰好是这个行业最真实的样子。术语解读 GLP-1 受体激动剂一类模拟人体"胰高血糖素样肽-1"的药物,能促进胰岛素分泌、抑制食欲、延缓胃排空,既能降糖又能显著减重,近年更被证实对心血管、肾脏等有额外获益。代表药物是诺和诺德的司美格鲁肽(Ozempic/Wegovy)和礼来的替尔泊肽(Mounjaro/Zepbound,双靶点)。它正以史上最快的速度扩张,深刻改变着糖尿病与肥胖的治疗格局,也是悬在传统胰岛素头上最大的变量。 The End 数据来源与参考(截至 2026 年 6 月) 1.国际糖尿病联盟(IDF)《全球糖尿病地图》第 11 版(IDF Diabetes Atlas, 11th Edition, 2025):全球患病人数、2050 年预测、卫生支出等。 2.赛诺菲(Sanofi)历年财报与新闻稿(SEC 6-K / 官网 press releases, 2015—2024):来得时/Toujeo 销售、中国集采影响、美国降价与 35 美元封顶、Play to Win 战略等。 3.DrugPatentWatch、Statista:来得时全球销售额历史(2014 年峰值约 74 亿欧元 / 约 88 亿美元、2023 年约 14 亿欧元)。4.美国降价信息:Sanofi 官方新闻稿(2023-03-16)、STAT、NBC、CNN、Axios、Fierce Pharma 等公开报道(2023—2024):列价降 78%、Apidra 降 70%、10mL 瓶约 64 美元 / 预填充笔约 96 美元、35 美元自费封顶、IRA 与 Medicaid 返利背景。5.中国胰岛素集采:新华社、ChemLinked、PharmCube、BMJ Global Health、Frontiers in Public Health 等(2021—2025):第六批(胰岛素专项)平均降幅约 48%、单品最大降幅约 74%、首年采购量超 2.1 亿支、甘精降幅约 66%、国产份额过半等。6.GLP-1 相关:IQVIA、Grand View Research、GlobeNewswire/Yahoo Finance、C&EN、IMARC(China Diabetes Market)等(2024—2026):2024 年 GLP-1 合计收入约 457 亿美元、市场规模预测、司美格鲁肽 2026 年起专利到期等。7.甘精胰岛素机制与研发史:Wikipedia(insulin glargine)、PubMed、PMC、ScienceDirect、Google Patents 等:分子改造、等电点位移、微沉淀缓释机制、2000 年获批、生物类似药(Basaglar/Semglee/Rezvoglar)等。8.说明:文中销售额曲线为依据上述公开财报数据的近似重构,用于呈现趋势,非逐年精确值;涉及汇率换算(欧元/美元)为约数。免责声明本文为医药商业与产业趋势的科普性深度分析,仅代表作者基于公开信息的个人观察与判断,不构成任何投资建议、诊疗建议或用药指导。文中涉及的药品(包括来得时/甘精胰岛素及其他胰岛素、GLP-1 类药物等)均为处方药,是否使用、如何使用请务必遵从执业医师与药师的专业意见,切勿自行调整用药方案。文中所引数据与观点均来自公开渠道,已尽力核实并标注来源,但因信息更新与口径差异,不保证完全准确无误;若有出入,以各机构、公司官方发布为准。文中提及的公司、品牌、商标均归其各自权利人所有,本文使用仅为客观陈述与分析之目的,不含任何贬损或商业诋毁意图。原创声明本文为原创作品,配图均为作者原创制作的信息示意图(非药品官方实拍图,产品图为示意)。未经授权,禁止任何形式的转载、摘编或改编; 如需转载、授权或商务合作,请通过公众号后台留言联系。如果觉得内容还不错，别忘了点分享、收藏、在看、点赞哦！

专利到期

2026-07-01

·BioSpace

BridgeBio Raises $1 Billion in Preferred Equity to Accelerate Present and Upcoming Launches

- Preferred equity investment led by Sixth Street and with participation from HealthCare Royalty, a business of KKR, with an initial conversion price of approximately $138 per share (more than 100% premium to Company’s 30-day VWAP) - The financing significantly strengthens the Company’s balance sheet, enabling it to efficiently allocate capital across its highest return opportunities - The financing comes at a pivotal moment for the Company, as Attruby ® continues to grow into a multi-billion-dollar blockbuster drug, and as BridgeBio prepares for three additional potential blockbuster U.S. product launches over the next 12 months across BBP-418 for LGMD2I/R9, encaleret for ADH1, and infigratinib for achondroplasia PALO ALTO, Calif., July 01, 2026 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage, multi-product biopharmaceutical company focused on developing medicines for genetic conditions, today announced that it has entered into an agreement with funds managed by Sixth Street (“Sixth Street”) and funds managed by HealthCare Royalty, a business of KKR (“HCRx” and, together with Sixth Street, the “Purchasers”) under which the Purchasers have invested up to $1 billion in newly issued convertible preferred equity of the Company. The Series A Cumulative Convertible Participating Preferred Stock has the following principal terms: 7.00% initial dividend, payable in kind or in cash at the Company’s election Initial conversion price of $137.79 per share (more than 100% premium to BridgeBio’s 30-day volume-weighted average price), increasing to $153.10 per share (more than 125% premium) from the fifth anniversary Permanent equity with no scheduled maturity and no redemption at the holder’s option BridgeBio may redeem the preferred stock for cash or, in certain circumstances, convert it into common stock, in each case on the terms set forth in the definitive agreements Sixth Street funded $800M as the lead investor, and HealthCare Royalty funded $133.9M at today’s close of the preferred equity investment. “We are privileged to be partnering with Sixth Street and HealthCare Royalty at this pivotal time in BridgeBio’s trajectory. This financing represents the best of our dual mission – 1) to put patients first and ensure that we have the resources to do so, and 2) that we execute those responsibilities in a manner that maximizes the economic value of our Firm. Access to this type and quantum of capital ensures we can deliver on the promise of our launching medicines and beyond,” said Neil Kumar, Ph.D., Co-Founder and CEO of BridgeBio. “Sixth Street is proud to support BridgeBio’s mission of bringing meaningful medicines to patients during this exciting stage as the company is on the cusp of potential approval and launch of three important new therapies,” said Jeff Pootoolal, Partner at Sixth Street. “Providing flexible capital at scale to leading developers of transformative medicines is central to what we do, and we look forward to a long and productive partnership with the BridgeBio team." “The BridgeBio management team has a proven track record in launching and developing life-changing therapies, and we are pleased to partner with them on this transaction,” said Clarke Futch, Chairman and CEO of HealthCare Royalty. “This capital support reaffirms our belief in the company’s growth and ability to bring to market multiple products that serve high unmet medical needs.” Latham & Watkins LLP served as legal advisor to BridgeBio. Evercore served as financial advisor and Sullivan & Cromwell LLP and Mintz LLP served as legal advisors to Sixth Street. Gibson, Dunn & Crutcher LLP served as legal advisor to HealthCare Royalty. Additional details about the transaction and the related definitive agreements will be included in a Current Report on Form 8-K to be filed by the Company. About BridgeBio Pharma, Inc. BridgeBio Pharma, Inc. (BridgeBio; Nasdaq: BBIO) exists to develop transformative medicines for genetic conditions. Millions of people worldwide living with genetic conditions lack treatment options, often because drug development for small patient populations can be commercially challenging. We aim to bridge the gap between advancements in genetic science and meaningful medicines for underserved patient populations. Our decentralized, hub-and-spoke model is designed for speed, precision, and scalability. Autonomous and empowered teams focus on individual conditions, while a central hub provides the clinical, regulatory, and commercial capabilities needed to bring innovation to market. For more information visit bridgebio.com and follow us on LinkedIn , X , Facebook , Instagram , and YouTube . About Sixth Street Sixth Street is a global investment firm with over $130 billion in assets under management and committed capital. Sixth Street uses its long-term flexible capital, data-enabled capabilities, and One Team culture to develop themes and offer solutions to companies across all stages of growth. Sixth Street Healthcare and Life Sciences invests thematically throughout the healthcare ecosystem, providing flexible capital solutions to companies addressing our most pressing healthcare challenges and improving patient outcomes. Investments in the sector include Apellis Pharmaceuticals, Arrowhead Pharmaceuticals, Arsenal Biosciences, Beam Therapeutics, Biohaven, Blueprint Medicines, Caris Life Sciences, Chroma Medicine, ConcertAI, Datavant, Essential Pharma, Immunogen, Ironwood, Mammoth Biosciences, Paratek Pharmaceuticals, and Velocity Clinical Research, among many others. Founded in 2009, Sixth Street has more than 750 team members including approximately 300 investment professionals around the world. For more information, visit , or follow Sixth Street on LinkedIn . About HealthCare Royalty HealthCare Royalty (“HCRx”) is a leading royalty acquisition company founded in 2006 that is majority owned by KKR & Co. Inc. (NYSE: KKR). Over two decades, the HCRx team has developed a strong track record of investing in commercial-stage and near-commercial-stage biopharmaceutical assets, committing $7+ billion in over 110 biopharmaceutical products. With offices in New York, Stamford, San Francisco, Boston, London and Miami, HCRx continues to advance biopharmaceutical innovation by providing innovative capital solutions to counterparties. For more information, visit HEALTHCARE ROYALTY®, HEALTHCARE ROYALTY PARTNERS® and HCRx® are registered trademarks of HealthCare Royalty Management, LLC BridgeBio Pharma, Inc. Forward-Looking Statements This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “continues,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,” “should,” “will,” and variations of such words or similar expressions. BridgeBio intends these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements include express and implied statements relating to the Company’s expectations regarding its anticipated growth and expected product launches and intentions for investing in indication expansions. Such statements reflect the Company’s current views about the Company’s plans, intentions, expectations and strategies, which are based on the information currently available to it and on assumptions the Company has made. Although the Company believes that its plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, the Company can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, initial and ongoing data from the Company’s clinical trials not being indicative of final data, the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales, despite having ongoing and future interactions with the FDA or other regulatory agencies to discuss potential paths to registration for the Company’s product candidates, the FDA or such other regulatory agencies not agreeing with the Company’s regulatory approval strategies, components of the Company’s filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, increasing rates of inflation and changing interest rates, on business operations and expectations, as well as those risks set forth in the Risk Factors section of the Company’s most recent Annual Report on Form 10-K and the Company’s other filings with the U.S. Securities and Exchange Commission. Moreover, the Company operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of the Company’s management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, BridgeBio assumes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. CONTACT: BridgeBio Media Contact: Bubba Murarka, Executive Vice President contact@bridgebio.com (650)-789-8220 BridgeBio Investor Contact: Kristen Kelleher, Director of Investor Relations ir@bridgebio.com

并购

2026-06-25

·BioSpace

Genomic Profiling With MammaPrint + BluePrint Predicts Anthracycline Chemotherapy Benefit and Guides Personalized Treatment Selection in HR+HER2- Early-Stage Breast Cancer

FLEX Study data shows that patients with MammaPrint High Risk 2 tumors have improved 3-year survival with anthracycline-containing regimens, while patients with High Risk 1 tumors show no additional benefit IRVINE, Calif. & AMSTERDAM--(BUSINESS WIRE)--Agendia ® , Inc., a leader in precision oncology for breast cancer, today announced the publication of a new paper in JCO Precision Oncology, titled “Prediction of Anthracycline Benefit in Hormone Receptor–Positive, Human Epidermal Growth Factor Receptor 2–Negative Early-Stage Breast Cancer by the MammaPrint 70-Gene Signature for Patients Enrolled in the FLEX Study.” The study, based on data from the prospective, real-world FLEX Study ( NCT03053193 ), found that MammaPrint ® + BluePrint ® can help identify a subset of patients with hormone receptor positive, HER2-negative (HR+HER2-) early-stage breast cancer (EBC) most likely to benefit from anthracycline-based chemotherapy. This research led to an update in the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology (NCCN Guidelines ® ) recognizing MammaPrint as the only genomic test to guide personalized anthracycline use in this patient population. “Patients with HR+HER2-negative breast cancer who are candidates for adjuvant chemotherapy continue to face uncertainty around whether they will derive meaningful benefit from anthracycline-containing regimens,” said Joyce O’Shaughnessy, M.D., principal investigator for the FLEX Study. “This long-standing clinical dilemma underscores the need for predictive biomarkers that can identify which patients are most likely to benefit from anthracyclines and who may safely avoid unnecessary toxicity.” While anthracyclines are highly effective antitumor agents, they are associated with potentially serious toxicities, including cardiotoxicity and secondary leukemias. Multiple randomized controlled trials – including the ABC trials 1 , Plan B trial 2 , the MASTER trial 3 and the DBCG 07-READ trial 4 – have attempted to determine whether anthracycline-free chemotherapy regimens are non-inferior to anthracycline-containing regimens in clinically high-risk HER2-negative EBC but found no significant difference among HR+HER2- patients. This observational study from FLEX included 1,259 patients with stages I to III, HR+HER2- EBC, classified as MammaPrint High Risk 1 (H1) or High Risk 2 (H2) and BluePrint Luminal B, who received adjuvant chemotherapy with either anthracycline and taxane-based chemotherapy (AC-T) or adjuvant taxane with cyclophosphamide (TC), and had a median follow-up of 3.2 years. Inverse probability of treatment weighting (IPTW), a well-established statistical method for the analysis of real-world evidence, was performed to balance clinical characteristics between treatment groups and approximate the design of a randomized trial. Key findings from the study include: Patients with H2 tumors demonstrated significantly improved 3-year invasive disease-free survival (IDFS) with AC-T versus TC, with IDFS rates of 100% vs 94.8%, respectively – an absolute benefit of 5.2% (p=0.023). Patients with H1 tumors showed no significant difference in 3-year IDFS with identical IDFS rates of 95.9% for both regimens, despite overlapping high-risk clinical features. A statistically significant treatment-by-MammaPrint interaction demonstrated that AC-T benefit increased with higher genomic risk (p=0.036), showing that MammaPrint H2 is predictive of anthracycline benefit. “This study answers a question that randomized clinical trials have been unable to resolve, illustrating the value of genomic profiling to predict benefit from a specific cancer therapy,” said William Audeh, M.D., M.S., Chief Medical Officer of Agendia and co-author of the study. “The FLEX Study highlights the power of a multi-modal real-world evidence database to produce robust, clinically relevant data which not only inform treatment decisions, but also change clinical practice guidelines.” References 1. Flynn PJ, Yothers G, et al. Anthracyclines in early breast cancer: The ABC Trials—USOR 06-090, NSABP B-46-I/USOR 07132, and NSABP B-49 (NRG Oncology). J Clin Oncol. 2017;35(23):2647-55. doi:10.1200/JCO.2016.71.4147 2. Nitz U, Gluz O, Clemens M, et al. West German Study PlanB Trial: Adjuvant four cycles of epirubicin and cyclophosphamide plus docetaxel versus six cycles of docetaxel and cyclophosphamide in HER2-negative early breast cancer. J Clin Oncol. 2019;37(10):799-808. doi:10.1200/JCO.18.00028 3. Yu KD, Liu XY, Chen L, et al. Anthracycline-free or short-term regimen as adjuvant chemotherapy for operable breast cancer: A phase III randomized non-inferiority trial. Lancet Reg Health West Pac. 2021;11:100158. doi:10.1016/j.lanwpc.2021.100158 4. Ejlertsen B, Tuxen MK, Jakobsen EH, et al. Adjuvant cyclophosphamide and docetaxel with or without epirubicin for early TOP2A-normal breast cancer: DBCG 07-READ, an open-label, phase III, randomized trial. J Clin Oncol. 2017;35(23):2639-2646. doi:10.1200/JCO.2017.72.3494. About Agendia Agendia is a global leader in precision oncology focused on early-stage breast cancer . The company’s genomic assays, MammaPrint + BluePrint , deliver essential biological insights to inform personalized treatment decisions for patients and their care teams. With operations in Amsterdam and Irvine, Agendia partners with academic and community oncology centers worldwide to generate real-world evidence through the landmark FLEX Study (NCT03053193) , the largest whole-transcriptome registry of early-stage breast cancer. About MammaPrint MammaPrint is a gene expression profiling test that assesses a woman’s risk of distant metastasis in early-stage breast cancer. By analyzing 70 key genes in a tumor, it stratifies risk into four categories — UltraLow Risk, Low Risk, High Risk 1, and High Risk 2 — to help guide personalized treatment planning, including chemotherapy benefit and de-escalation decisions. About BluePrint BluePrint is an 80-gene molecular subtyping assay that reveals the functional biology driving tumor growth, classifying tumors as Luminal-type, HER2-type, or Basal-type. By defining intrinsic subtypes beyond traditional immunohistochemistry, BluePrint provides critical insights to optimize treatment selection and improve outcomes. Contacts Media contact marketing@agendia.com

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