Press release
Vertex Announces Primary Endpoint Achieved in Phase 2 Study of VX-864 in Alpha-1 Antitrypsin Deficiency
- Treatment with VX-864 led to a statistically significant increase from baseline in plasma functional AAT levels as compared to placebo and was generally
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n- Treatment with VX-864 led to a statistically significant increase from baseline in plasma functional AAT levels as compared to placebo and was generally well tolerated -\n\n- Results provide proof-of-mechanism, although magnitude of treatment effect observed unlikely to translate into substantial clinical benefit; VX-864 will not advance into late-stage development - \n\n- Vertex to advance additional novel small molecule(s) with potential for greater clinical efficacy into the clinic in 2022 -\n\n- Vertex will host an investor conference call and webcast today, Thursday, June 10 at 4:30 p.m. EDT - \n\n BOSTON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that in a Phase 2 proof-of-concept study, VX-864 achieved rapid, consistent and statistically significant increases in mean functional alpha-1 antitrypsin (fAAT) levels of 2.2 to 2.3 micromolar from baseline in people with alpha-1 antitrypsin deficiency (AATD) with the PiZZ genotype, across three dose groups of VX-864 compared to placebo. VX-864 was generally well tolerated in the Phase 2 study. These data provide clear evidence that an oral small molecule corrector designed to promote the proper folding of the mutant Z-AAT protein can increase plasma levels of fAAT in patients with AATD. Although results provide proof-of-mechanism, the magnitude of treatment effect observed in this study is unlikely to translate into substantial clinical benefit. As such, Vertex will not advance VX-864 into late-stage development and instead will advance additional novel small molecule correctors with the potential for increased clinical efficacy into the clinic.\nThis press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210610005939/en/Figure 1: Statistically significant increase in mean functional and antigenic AAT observed at day 28 compared to placebo (Graphic: Business Wire)\n“This is the first time that dosing of a small molecule corrector of the Z-AAT protein resulted in significant elevations in both functional and antigenic levels of AAT in people with AATD. We are encouraged by the clear separation of AAT levels in the VX-864 treated groups versus placebo and the favorable safety profile,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, ...