VRTX · Earnings call · 2026Q3T · Full report

Next-Generation CF Modulators

VERTEX PHARMACEUTICALS INC / MA · 2026-11-02 · Importance null · Surprise 42

Vertex reported that VX-828, tested in small cohorts of patients with F/M/F mutations, reduced sweat chloride in the triple-combination regimen versus a Trikafta baseline, while the dual combination did not change sweat chloride relative to baseline. The oral regimen is once daily and had a favorable safety, tolerability, and drug-interaction profile. Because ALYFTREK sets a high bar, Vertex plans to compare VX-828 with the other next-generation 3.0 correctors, VX-581 and VX-272, before deciding whether to advance a candidate into pivotal development; next-generation 4.0 molecules are in late preclinical development.

Key facts

• Vertex will wait for all data from the next-generation 3.0 family before deciding whether to advance a candidate into pivotal development.
• Vertex expected results from VX-581 and VX-272 in 2027 and planned to select the best of the three 3.0 medicines after reviewing all results.
• In a cohort of eight people with CF, VX-828 triple-combination treatment reduced sweat chloride by 6.6 mmol/L at day 28 versus the Trikafta baseline and by 9.4 mmol/L through day 28.
• VX-828 was studied as a triple combination in eight people with CF and as a dual therapy in another eight; all 16 patients had F/M/F mutations.
• VX-828 dual combination did not alter sweat chloride relative to the Trikafta baseline in the studied F/F population.
• VX-581 and VX-272 were in first-in-human studies, while next-generation 4.0 molecules were in late preclinical development.