Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) stock gained by 1.5% at last close while the VRTX stock price declines during after-hour by 12.95%. Vertex is a multinational biotechnology business that invests in scientific research to develop life-changing treatments for patients suffering from critical illnesses. Multiple drugs approved by VRTX treat the underlying cause of cystic fibrosis (CF).
What is happening?
In a Phase 2 proof-of-concept study, VX-864 experienced high, reliable, and statistically significant rises in mean functional alpha-1 antitrypsin (fAAT) levels of 2.2 to 2.3 micromolar from baseline in individuals with alpha-1 antitrypsin deficiency (AATD) with the PiZZ genotype along with all three dose groups of VX-864 relative to placebo. In the Phase 2 trial, VX-864 was usually well tolerated.
These findings show that an oral small molecule corrector that promotes normal folding of the mutant Z-AAT protein can raise plasma levels of fAAT in AATD patients. Although the findings show that the mechanism is working, the amount of treatment impact seen in this research is unlikely to translate into significant therapeutic benefit. As a result, Vertex will not move VX-864 into late-stage development, preferring to focus on developing new small molecule correctors with the potential to improve clinical effectiveness.
At day 28 of therapy, all VX-864 dosing groups showed highly statistically significant increases in plasma fAAT levels from baseline, if compared to placebo, indicating that the trial fulfilled its primary aim. In comparison to the placebo, treatment with VX-864 resulted in a mean rise of 2.2 to 2.3 micromolar in fAAT levels across the three dosage groups investigated.
Only one patient in this trial did not finish therapy. There were no withdrawals owing to adverse events (AEs) or serious adverse events (SAEs) that were thought to be connected to the study medication. The majority of adverse events were mild to moderate in intensity and did not interfere with therapy.
The information gathered is expected to aid in the optimization of Vertex’s small molecule corrector strategy in AATD, as well as the quick development of a portfolio of novel compounds with improved clinical effectiveness into the clinic by 2022. In addition, the VX-864 study’s findings are likely to improve clinical trial design, recruitment, and execution for future assets.