Vertex Pharmaceuticals reported results from a phase 2 study evaluating the efficacy and safety of VX-864 in patients with alpha-1 antitrypsin deficiency (AATD) with the PiZZ genotype. 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 across three dose groups of VX-864 compared to placebo, meeting the primary endpoint. VX-864 was generally well tolerated. While the results provide proof-of-mechanism, Vertex stated the magnitude of treatment effect is unlikely to translate into substantial clinical benefit. As a result, VX-864 will not advance into late-stage development. The company will instead advance additional novel small molecule correctors with the potential for increased clinical efficacy into the clinic.
VX-864 is an oral small molecule Alpha 1 antitrypsin modulator.
About Alpha-1 Antitrypsin Deficiency
AATD is a rare, genetic disease characterized by a protein folding defect which can lead to liver and lung disease. AATD is caused by changes in the SERPINA1 gene that encodes the AAT protein. In the most common form of ...
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