According to an a press release published on Business Wire, Arrowhead Pharmaceuticals Inc. recently presented updated phase 2 data at the 2023 European Association for the Study of the Liver Congress. This trial was evaluating fazirsiran, an investigational drug, as a treatment for alpha-1 antitrypsin deficiency-associated liver disease. The findings are comparable to those in an open-label phase 2 trial that was recently published. The therapy will continue to be investigated in an ongoing phase 3 trial.

About Alpha-1 Antitrypsin Deficiency (A1AD)

Alpha-1 antitrypsin deficiency (A1AD) is a genetic mutation that can result in disease of the liver or lungs. Typically, lung problems begin between the ages of 20 to 50 years old. Symptoms include shortness of breath, wheezing, increased risk of lung infections, and COPD. Not all patients experience liver problems, but in some cases cirrhosis and liver failure may occur. Smoking can greatly worsen symptoms and outcomes, and even shorten life expectancy in people with A1AD. Most treatments are meant to improve symptoms and lung function. Transplantation may be an option in those with severe disease. In some patients, augmentation of the deficient A1AT protein may be viable and can stop the progression of symptoms and lung damage. People with liver complications are unfortunately not suitable candidates for this treatment. However, long term effects of this treatment have not been studied. Learn more about alpha-1 antitrypsin deficiency over at Patient Worthy’s disease cornerstone here.

Trial Findings

The press release indicates that Fazirsiran was able to demonstrate major impacts in several areas that indicate the presence and activity of alpha-1 antitrypsin deficiency liver disease. The drug reduced serum levels of Z-AAT, with increased effect seen with higher doses. The treatment also reduced levels of Z-AAT in the liver and hepatic globule burden. Treatment with the drug also brought down signs of liver inflammation and fibrosis (scarring).

In terms of safety, fazirsiran appears to be well tolerated, with no adverse side effects leading to cessation of treatment, study withdrawals, or interruptions in dosing. While the therapy will require continued evaluation in the ongoing phase 3 trial, these findings are largely positive and suggest that the drug will be well on its way to approval.

Full text of the press release is available over at Business Wire.