Seeking Alpha: Vertex Pharma -7.9% as it drops VX-814 study, development (updated) | ARWR Message Board Posts

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Msg  10166 of 10169  at  10/14/2020 5:11:16 PM  by

Licensing Guy


Strong Buy

Seeking Alpha: Vertex Pharma -7.9% as it drops VX-814 study, development (updated)

Vertex Pharmaceuticals (NASDAQ:VRTX) has slipped 7.9% after new that it's discontinuing its Phase 2 study of VX-814 and dropping development.

That's based on safety and pharmacokinetic data, the company says.

The study of about 50 patients was designed to evaluate safety/PK in VX-814 and its ability to increase functional levels of alpha-1 antitrypsin over 28 days of dosing.

"Elevated liver enzymes (AST/ALT) were observed in several patients," Vertex says. "In four patients, across different doses studied, elevations greater than 8 times the upper limit of normal were noted; the elevated liver enzymes have either resolved or are resolving."

The company concluded that it's not feasible to safely reach targeted exposure levels (and thus meaningful increases in AAT levels).

A Phase 2 trial of VX-864 - structurally distinct from VX-814 - is still ongoing after its July start, the company says. It also continues to advance multiple small molecule correctors in late stage research with a goal of advancing at least one additional molecule into development in 2021.

Updated 4:55 p.m.: Arrowhead Pharmaceuticals (NASDAQ:ARWR) - also focused on liver diseases associated with alpha-1 antitrypsin deficiency - is up 14.9% after hours.



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