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Vertex completed enrollment for the two Phase 3 studies of the next-generation corrector VX-659 in triple combination with tezacaftor and ivacaftor in people with cystic fibrosis with one F508del mutation and one minimal function mutation and in people with two F508del mutations. Vertex expects to report data from both Phase 3 studies of the VX-659 triple combination regimen in late 2018. Vertex expects to complete enrollment of the two Phase 3 studies of the next-generation corrector VX-445 in triple combination with tezacaftor and ivacaftor in Q4 and to report data from these studies in 1Q19. Vertex plans to evaluate data from both the VX-659 and VX-445 Phase 3 triple combination programs to choose the best regimen to submit for potential regulatory approval. The data expected in late 2018 for VX-659 and in 1Q19 for VX-445 are expected to provide the basis for submission of a New Drug Application to the FDA for people with one F508del mutation and one minimal function mutation no later than mid-2019. Vertex expects to disclose in late 2018 and 1Q19 only the topline results for the primary 4-week efficacy endpoints of the VX-659 and VX-445 Phase 3 studies, respectively, and whether the safety and efficacy profiles observed support a potential NDA submission. In 2H19, Vertex intends to disclose additional safety and efficacy data, including secondary endpoints, for each study following the completion of both the VX-659 and VX-445 Phase 3 triple combination programs.

Vertex Pharmaceuticals announces finalization of an agreement with the Australian Government that allows for reimbursement of Orkambi for people ages six and over with cystic fibrosis who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene. This agreement means lumacaftor/ivacaftor will be listed on the Pharmaceutical Benefits Scheme from 1 October and follows a positive recommendation from the Pharmaceutical Benefits Advisory Committee. A pathway to access for future Vertex CF medicine, tezacaftor/ivacaftor, has been established as part of this process.

Vertex Pharmaceuticals and Genomics plc announced a three-year collaboration, extendable to five years, to use human genetics and machine learning to improve discovery of targets for precision medicines, and to advance understanding of the clinical impact of human genetic variation and patient stratification in diseases with significant unmet need. The goal of the collaboration is to further advance Vertex's efforts to develop transformative precision medicines for people with serious diseases. Scientists from Vertex and Genomics will work closely together to incorporate their deep understanding of human genetics into research and development related to target discovery and target validation in certain diseases. Both companies will also have the opportunity to suggest additional diseases for the collaboration, where human genetic evidence may be particularly powerful. In addition to funding the resources committed by Genomics to the collaboration, Vertex will make milestone and royalty payments to the company for novel targets resulting from the collaboration that are taken through clinical development. Dr. Altshuler will also join the Genomics plc board. Additionally, Vertex has made a GBP 10.5M equity investment in Genomics plc as part of its GBP 25M Series B funding round.