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VRTX JPM 2026 Presentation_vFINAL.pdf

上传人: 明**** 编号:1069848 2026-01-17 21页 1.98MB

1、2026 Vertex Pharmaceuticals Incorporated2026 Vertex Pharmaceuticals IncorporatedJ.P.Morgan Healthcare ConferenceJanuary 2026Reshma Kewalramani,M.D.CEO and President2Safe Harbor Statement&Non-GAAP Financial MeasuresThis presentation contains forward-looking statements as defined in the Private Securi

2、ties Litigation Reform Act of 1995,including,without limitation,the information provided regarding future financial and operating performance and statements regarding(i)expectations,goals,development and commercialization plans and timelines for the companys products,product candidates and pipeline

3、programs,including beliefs regarding the emerging renal business,multiple launch opportunities,treatable patient populations,and additional opportunities in mid-and-late stage programs,including more than 10 disease areas with multi-billion dollar market potential,(ii)expectations for sustained grow

4、th with respect to the companys CF medicines,including with respect to the ongoing commercial launch of ALYFTREK,that the majority of eligible CF patients will switch from TRIKAFTA to ALYFTREK over time,treating younger patients,filing for approval for TRIKAFTA in patients 1 to 2 years of age in H1

5、2026 and securing approval in H2 2026,sharing data from Phase 3 study of ALYFTREK in patients 2 to 5 years of age and submitting for regulatory approval in 2026,initiating a Phase 3 study of ALYFTREK in patients 1 to 2 years of age in 2026,CF population growth and patients living longer,reaching add

6、itional geographies,the goal to advance additional small molecules to be able to bring all eligible patients to carrier levels of CFTR function,and extending the CF portfolios market leadership with IP through 2040,(iii)plans to advance VX-522 to reach the 5,000 CF patients who cannot benefit from a

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1. **核心业务与增长**:Vertex聚焦三大领域(囊性纤维化CF、血液病、肾脏病),通过差异化研发模式推动长期增长,2025年营收达119-120亿美元,研发投入占比高。 2. **关键产品进展**: - **CF**:5款药物覆盖95%患者,专利保护至2040年;2026年将提交TRIKAFTA(1-2岁)和ALYFTREK(2-5岁)申请,推进VX-522(mRNA疗法)等。 - **CASGEVY**:2025年营收超1亿美元,目标成为十亿美元级产品,2026年将提交5-11岁申请。 - **JOURNAVX**:2026年目标处方量增长超3倍,加拿大申请H1/2026提交。 3. **肾脏病管线**:Povetacicept(IgAN)2026年H1提交BLA寻求加速批准,Inaxaplin(AMKD)2026年底公布III期中期数据。 4. **财务策略**:持续投资研发,现金储备120亿美元,优先用于创新与股票回购。
**Vertex 2026亮点?** **CF新药进展如何?** **肾科布局有何突破?**
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