Dicerna Pharmaceuticals announced today that its investigational GalXC RNAi therapy belcesiran has achieved positive interim results in a phase 1 clinical trial
Belcesiran is an RNAi therapy under development for the treatment of alpha-1 antitrypsin deficiency-related liver disease (AATLD)
AATLD is a rare genetic disease.
Due to mutations in the SERPINA1 gene, α-1 antitrypsin (AAT) mutants misfold and accumulate in liver cells, which can lead to liver fibrosis, cirrhosis and hepatocellular carcinoma
Belcesiran aims to target genes that produce abnormal AAT proteins to reduce AAT production in the liver
The data of the interim analysis showed that after a single administration of belcesiran, serum α-1 antitrypsin (AAT) decreased in a dose-dependent manner
At the same time, belcesiran has acceptable safety profiles and is generally well tolerated
In this randomized, double-blind, placebo-controlled phase 1 clinical trial, healthy volunteers received a single injection of placebo or different doses of belcesiran
This interim analysis included injection doses of 0.
0, or 6.
The results of the interim analysis showed that belcesiran reduced AAT protein levels by an average of 50% (1.
0 mg/kg), 69% (3.
0 mg/kg) and 80% (6.
0 mg/kg) compared with baseline
In terms of safety, no serious adverse events were found, and 3 moderate treatment-related adverse events (TEAE) occurred, but they were not related to belcesiran, and other TEAEs were mild
None of the participants treated with belcesiran in this analysis reported clinically significant changes in lung function or laboratory tests
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 Dicerna Announces Interim Results From Phase 1 Trial of Belcesiran for Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease.
Retrieved July 21, 2021, from https://investors.