JPM 2023 Vertex.pdf

1、J.P.MORGAN HEALTHCARE CONFERENCEJANUARY 2023RESHMA KEWALRAMANI,M.D.CEO AND PRESIDENT2023 Vertex Pharmaceuticals ISAFE HARBOR STATEMENT&NON-GAAP FINANCIAL MEASURESThis presentation contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995,including,without

2、 limitation,the information provided regarding future financial and operating performance andstatements regarding(i)expectations,development plans and timelines for the companys medicines,drug candidates andpipeline programs,including expectations for five potential launches in five years,multiple c

3、linical-stage programs withlaunch potential by 2030,anticipated benefits of new products and relevant patient populations,(ii)expectations foruptake of and expanded access to the companys medicines(iii)expectations for continued growth in the number of CFpatients treated with our therapies,including

4、 targeted 2023 global launch of TRIKAFTA/KAFTRIO in patients aged 2-5,anticipated serial innovation to reach all CF patients eligible for CFTRm and plans to reach the last 5,000 CF patients withmRNA therapy,(iv)expectations for the exa-cel program,including the potential of exa-cel to be a one-time,

5、functionalcure for patients with SCD and TDT,expectations for completion of US regulatory submissions in the first quarter of 2023,expectations for near-term launch and commercial potential,(v)expectations for our pain program,including enrollmentplans in the acute pain and neuropathic pain studies

6、and plans for near-term launch and commercial potential in acutepain,and expectation for treatment of acute pain without limitations of opioids,(vi)expectations for our T1D program,including availability of VX-880 clinical data in 2023 and Phase 1/2 clinical trial for VX-264 anticipated to begin in