Vertex-JPM-20240108.pdf

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1、J.P.MORGAN HEALTHCARE CONFERENCEJANUARY 2024RESHMA KEWALRAMANI,M.D.CEO AND PRESIDENT2024 Vertex Pharmaceuticals IncorporatedSAFE HARBOR STATEMENT&NON-GAAP FINANCIAL MEASURESThis presentation contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995,includ

2、ing,without limitation,the information provided regarding and expectations for future financial and operating performance andstatements regarding(i)expectations,development plans and timelines for the companys medicines and pipeline programs,including anticipated 10 disease areas in clinical develop

3、ment in 2024,expectations for five potential launches in five years,anticipated near-term commercial opportunities in TDT,CF and acute pain,anticipated benefits of new products and relevant patientpopulations,and plans to broaden and deepen R&D pipeline across modalities,(ii)plans to launch CF medic

4、ines in younger agegroups globally and to bring additional molecules to market to get CF patients to carrier levels of SwCl,(iii)plans to advance VX-522 toreach the 5,000 CF patients who cannot benefit from a CFTR modulator,(iv)expectations for CASGEVY,including the potentialbenefits for patients wi

5、th SCD and TDT,expectations for broad access in the US,expectations for ex-US access initially through EarlyAccess Programs and efforts for long-term reimbursement agreements,plans to obtain approvals in additional geographies,and plansto complete dosing in younger age groups,(v)expectations for our

6、 pain program,including plans for near-term launch andcommercial potential in acute pain,expectation for treatment of acute pain without side effects of addictive properties of opioids,plans to share data from the acute pain pivotal program and submit regulatory filings,plans to advance VX-993 into