Vertex Shares Drop After Company Stops Testing on Protein Deficiency Treatment

Shares of pharmaceutical company Vertex Pharmaceuticals were falling this week after the company announced that it was going to stop testing on a drug designed to treat protein deficiencies.
Based on early data of its Phase II trial of VX-814 in Alpha-1 antitrypsin deficiency (AATD), the company is halting the trial and ending development of the drug.

AATD is a genetic disease resulting in a missing liver protein and children with AATD either do not produce enough of the alpha-1 protein or the protein they do produce is abnormal. The accumulation of the abnormal protein is what leads to the damage to the liver.

The company’s trial had about 50 patients and was meant to evaluate the safety and pharmacokinetics of VX-814 and its ability to increase functional levels of alpha-1 antitrypsin over 28 days.

The patients demonstrated elevated liver enzymes, AST/ALT, and in four patients across different doses, the elevated liver enzymes were eight times greater than the upper limit of normal. To date, the elevated liver enzymes have either resolved or are resolving.

Vertex decided that based on the data, it would not be feasible to safely reach exposure levels.

“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,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.

“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.”
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Disclaimer: We have no position in any of the companies mentioned and have not been compensated for this article.