07/02/2023 - Vertex Pharmaceuticals Incorporated: Q4 2022 Presentation

SAFE HARBOR STATEMENT & NON-GAAP FINANCIAL MEASURES

This presentation contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the information provided regarding future financial and operating performance, full-year 2023 financial guidance, and statements regarding our (i) expectations, development plans, and timelines for the company's products, product candidates, and pipeline programs, including expectations for five potential launches in five years, multiple clinical-stage programs with launch potential by 2030, anticipated benefits of new products, patient populations, study designs, clinical site initiations, patient enrollment, data availability, anticipated regulatory filings, approvals, and timing thereof, (ii) expectations for continued growth in the number of CF patients treated with our existing

therapies, including targeted 2023 global launch of TRIKAFTA/KAFTRIO in patients aged 2-5, (iii) expectations to reach all CF patients eligible for CFTRm, including the last >5,000 CF patients (ineligible for a CFTRm) with VX-522, our plans to complete a single ascending dose study and initiate multiple ascending dose study for VX-522 in 2023, (iv) expectations on continued innovation in CF, including the benefits of vanzacaftor triple combination therapy, our plan to complete Phase 3 studies in 2023, and plans for near-term launch and commercial potential, (v) expectations for the exa-cel program, including the potential of exa-cel to be a one- time, functional cure for patients with SCD and TDT, expectations to complete U.S. regulatory submissions in the first quarter of 2023, expectations for near-term launch and commercial potential, (vi) expectations for our pain program, including its potential to broadly and effectively treat acute pain without the limitations of opioids, the market size and opportunity for VX-548, the anticipated timeline to complete the Phase 3 pivotal program for VX-548 in acute pain and progress enrollment in Phase 2 studies of VX-548 in neuropathic pain, and plans for near-term commercial launch in moderate-to-severe acute pain, (vii) our expectations and beliefs regarding our pivotal program for inaxaplin, including its potential to treat the underlying cause of AMKD, plans to complete Phase 2B studies in 2023, our beliefs regarding anticipated results of the study and the potential to seek accelerated approval in the U.S., (viii) expectations for the development of our T1D programs, including the patient population, potential curative benefits and safety of VX-880, plans to continue to progress the Phase 1/2 program for VX-880 and availability of updated clinical data in 2023, plans to initiate VX-264 in Canada, and expectations that our recent ViaCyte acquisition may accelerate the development of our T1D program, (ix) plans for continued advancement of VX-634 and VX-864, (x) plans for our DMD program, including expectations to file an IND, (xi) beliefs about rapid development of treatments and potential cures for more patients in multiple new disease areas, and plans to continue to invest in our pipeline and commercial readiness activities for our programs, and (xi) expectations regarding the company's tax rates, revenue growth, and the impact of foreign exchange rates on revenue growth. While Vertex believes the forward-looking statements contained in this presentation are accurate, these forward-looking statements represent the company's beliefs only as of the date of this presentation and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the company's expectations regarding its future financial and operating performance may be incorrect (including because one or more of the company's assumptions underlying its expectations may not be realized), that the company may not be able to submit anticipated regulatory filings on expected timelines, or at all, that external factors may have different or more significant impacts on the company's business or operations than the company currently expects, that data from preclinical testing or clinical trials, especially if based on a limited number of patients, may not be indicative of final results, that patient enrollment in our trials may be delayed, that actual patient populations able to participate in our trials or eligible for our products may be smaller than we anticipated, that data from the company's development programs may not be available on expected timelines, or at all, and may not support registration or further development of its potential medicines due to safety, efficacy or other reasons, and other risks listed under "Risk Factors" in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission (SEC) and available through the company's website at www.vrtx.com and on the SEC's website at www.sec.gov. You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this presentation as new information becomes available.

©2023 Vertex Pharmaceuticals Incorporated