According to a news release from Vertex Pharmaceuticals, the company is stopping the dosing of VX-814 in its Phase 2 trial of the drug. The company came to this decision based on the pharmacokinetic profile and safety of the treatment.
Adding to this negative news, Vertex Pharmaceuticals says that it’s stopping the development of VX-814 completely. The company was testing the drug’s “increase functional levels of alpha-1 antitrypsin over 28 days of dosing.”
The news release from Vertex Pharmaceuticals notes that the clinical trial saw elevated liver enzymes in several patients. In the case of four patients, this increase was eight times beyond normal levels. The company notes that elevated liver enzymes are resolved or currently resolving.
Carmen Bozic, M.D., executive vice president of Global Medicines Development and Medical Affairs and CMO of Vertex Pharmaceuticals, said this about the news.
“Based on the liver enzyme elevations observed, along with the determination that we would not be able to safely achieve targeted exposure levels with VX-814, we are discontinuing further development of this molecule. We are grateful to the AATD patients and investigators who participated in the VX-814 studies and we remain committed to transforming the treatment of this disease. We look forward to continuing clinical study of VX-864 and other molecules targeting the underlying cause of AATD.”
VRTX stock was down 19.9% as of Thursday morning.
On the date of publication, William White did not have (either directly or indirectly) any positions in the securities mentioned in this article.