Vertex’s rare lung disease treatment has achieved its Phase 2 goal, but is still stuck in the scrap pile

To overturn another perspective that biotechnology was developing for alpha-1 antitrypsin deficiency (AATD), a rare hereditary disease that can lead to liver and lung disease, Vertex blueprints came back.

Patients with AATD have mutations that produce abnormally shaped α-1 antitrypsin (AAT) proteins. These misfolded proteins are trapped in the liver rather than being released into the blood like regular AAT proteins. Abnormal accumulation of AAT in the liver, combined with reduced levels of AAT in the blood, can cause liver and lung disease.

Vertex’s VX-864 is a “correction molecule” designed to prevent misfolding of AAT and increase plasma AAT levels. Twenty-eight days later, in a phase II trial, three dose levels of VX-864 defeated placebo by boosting the patient’s plasma AAT levels.However, although the trial was technically successful, the drug was so ineffective that the company Unlikely “Convert to substantial clinical benefit”.

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As a result, Vertex will not move the VX-864 into late-stage development, but rather a new small molecule corrector that has the potential to enhance its clinical efficacy, “the company said in a statement Thursday afternoon.

According to the statement, the company plans to begin clinical trials of other drug prospects in 2022.

Vertex’s share price fell 14% in after-hours trading.

Vertex announced Phase 2 data and program discontinuation eight months later. Abandoned Another molecule, VX-814, caused abnormally high levels of liver enzymes in Phase 2 studies.

Investigators found elevated liver enzymes in “several patients,” four of whom showed spikes that were more than eight times the normal upper limit, the company said in a statement at the time. In addition, data analysis showed that only a small amount of the drug had moved where it should have been.

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The news was a blow to Vertex’s “widely thought to be the driving force of key value” programs other than the treatment of cystic fibrosis, JP Morgan analyst Collie Kasimov said at the time. I wrote it in a memo for.

After excluding VX-814, Vertex turned to VX-864. VX-864 is a “structurally different” compound from VX-814 and was already in Phase 2 research. Now that the VX-864 is being scrapped, the company looks to the preclinical pipeline.

“I was encouraged by the clear separation of AAT levels between the VX-864 treatment and placebo groups and the good safety profile,” said Vertex Chief Medical Officer and Executive Vice President of Global Drug Development and Medical Issues. Dr. Carmen Bozic said: statement. “Based on these findings, we continue to develop innovative therapies for AATD and quickly translate the lessons learned from this study to optimize the next set of small molecule correctors. And we are making it possible to fully realize the potential of this class of molecules. For those who live with this disease. “ Vertex’s rare lung disease treatment has achieved its Phase 2 goal, but is still stuck in the scrap pile

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