Vertex Pharmaceuticals Investor Presentation — June 2021

Document content

VX-864 PHASE 2 RESULTS AND ALPHA-1 ANTITRYPSIN DEFICIENCY (AATD) PROGRAM UPDATE

©2021 Vertex Pharmaceuticals IncorporatedJUNE 10,2021global.vrtx.comFor Q&A

©2021 Vertex Pharmaceuticals Incorporatedglobal.vrtx.com2VX-864 PHASE 2 RESULTS AND ALPHA-1 ANTITRYPSIN DEFICIENCY (AATD)PROGRAM UPDATEAGENDAReshma Kewalramani, M.D., Vertex’s CEO and President

David Altshuler, M.D., Ph.D., Vertex’s EVP, Global Research, and Chief Scientific Officer

global.vrtx.com3SAFE HARBOR STATEMENTThispresentation contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, (i) the company’s intention to apply insights from this study and its plan to advance novel small molecule correctors with the potential for increased clinical efficacy in 2022 and (ii) the company’s expectations regarding the clinical path for future molecules. While Vertex believes the forward-looking statements contained in this presentation are accurate, theseforward-looking statements represent the company's beliefs only as of the date of this presentation and there area number ofrisks and uncertainties that could cause actual events or results todiffer materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, risks related to the company’s AATD research programs, that data from the company's research and development programs may not support registration or further development of its compounds due to safety, efficacy, or other reasons, and other risks listed under the heading Risk Factors in Vertex's annual report filed with the Securities and ExchangeCommission and available through the company's website at www.vrtx.comand on the SEC’s website at www.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this presentation as new informationbecomes available.©2021 Vertex Pharmaceuticals Incorporatedglobal.vrtx.comglobal.vrtx.com

VX-864 PHASE 2 RESULTS

©2021 Vertex Pharmaceuticals Incorporated4global.vrtx.comglobal.vrtx.com5Placebo (N=7)STARTVX-864DAY 28POST-TREATMENTFOLLOW-UP(28 days)•Change from baseline in plasma antigenic AAT levels at Day 28•Pharmacokinetic parameters of VX-864 •Change from baseline in plasma functional AAT levels at Day 28•Safety and tolerabilityPrimary EndpointsSecondary EndpointsN = 44 subjects500 mg q12h (N=18)300 mg q12h (N=9) 100 mg q12h (N=10)PHASE 2 STUDY AIMED TO ASSESS THE ABILITY OF VX-864 TO INCREASE LEVELS OF FUNCTIONAL AATIN PLASMA AND SAFETY/TOLERABILITY

Preview truncated — download the full document for the complete content.