Vertex abandons rare disease drug despite positive trial

June 11, 2021

Vertex Pharmaceuticals has abandoned its prospect for alpha-1 antitrypsin deficiency (AATD) — a rare genetic disorder — despite the phase 2 trial meeting its primary endpoint.

AATD is a rare, genetic disease characterized by a protein folding defect which can lead to liver and lung disease. Vertex’s VX-864 is an oral small molecule corrector designed to promote the proper folding of the mutant Z-AAT protein.

According to Vertex, although results of the study provide proof-of-mechanism, "the magnitude of treatment effect observed in this study is unlikely to translate into substantial clinical benefit."  As such, the drugmaker has chosen not to advance VX-864 into late-stage development. Instead, Vertex will focus on advancing additional novel small molecule correctors with the potential for increased clinical efficacy.

“Based on these findings, we remain committed to developing transformative treatments for AATD and are working with urgency to translate the learnings from this study to optimize the next set of small molecule correctors so that we can fully realize the potential that this class of molecules may hold for people living with this disease,” said Carmen Bozic, chief medical officer at Vertex.

Read the press release