Benzinga | Jun 22, 2021 07:32AM EDT

Dicerna Initiates Patient Dosing In ESTRELLA Phase 2 Clinical Trial Of Belcesiran For Treatment Of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease

Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) (the "Company" or "Dicerna"), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, announced it has initiated patient dosing in the Company's Phase 2 ESTRELLA trial of belcesiran, an investigational GalXC(tm) RNAi therapeutic candidate for the treatment of alpha-1 antitrypsin (AAT) deficiency-associated liver disease (AATLD). AATLD is a rare genetic condition that can lead to liver fibrosis, cirrhosis and hepatocellular carcinoma.

"There are an estimated 183,000 people in Europe and the U.S. who carry a genetic mutation that can result in AAT deficiency and the associated liver and lung disease.1 With no therapies available to specifically address AAT liver disease, liver transplantation remains the only option for these patients," said Shreeram Aradhye, M.D., Executive Vice President and Chief Medical Officer at Dicerna. "Dicerna's investigational RNAi therapeutic, belcesiran, is designed to silence the gene that produces the abnormal AAT protein. Reducing production of this protein in patients with AATLD has the potential to allow their livers to clear the accumulated abnormal protein and restore liver health. We are very pleased to have now begun patient dosing in our Phase 2 ESTRELLA trial to better understand belcesiran's potential to treat the underlying cause of AATLD."

ESTRELLA (NCT04764448) is a randomized, multidose, double-blind, placebo-controlled Phase 2 trial evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of belcesiran in participants with AATLD. The study includes a 24-week cohort and a 48-week cohort to be conducted in parallel, each with up to 27 participants who have a diagnosis of PiZZ-type AAT deficiency and AATLD.

The ESTRELLA clinical trial is part of Dicerna's SHINE clinical development program to evaluate the safety and efficacy of belcesiran, formerly known as DCR-A1AT, for the treatment of AATLD. A Phase 1 trial of multiple doses of belcesiran in healthy volunteers is ongoing, with initial data from the Phase 1 trial expected in mid-2021.