Dicerna Posts Updated Data From Belcesiran Early-Stage Study For Genetic Liver Disease

Dicerna Pharmaceuticals Inc DRNA has announced interim results from the four completed active-treatment dose cohorts (0.1, 1.0, 3.0, and 6.0 mg/kg) of its Phase 1 trial of belcesiran.

• The GalXC RNAi therapeutic is in development for alpha-1 antitrypsin deficiency-associated liver disease (AATLD), a rare genetic condition that can lead to liver fibrosis, cirrhosis, and hepatocellular carcinoma. 
• The primary treatment evaluation period for the final dose cohort (12.0 mg/kg) is ongoing.
• Mean maximum serum AAT reductions from baseline achieved for doses greater than 0.1 mg/kg were: 50% (1.0 mg/kg), 69% (3.0 mg/kg) and 80% (6.0 mg/kg).
• In the four subjects receiving 6.0 mg/kg, maximum AAT reductions of 91%, 87%, 79%, and 62% were observed. The latter participant experienced a concomitant skin infection (unrelated to belcesiran) and markedly elevated C-reactive protein levels (a measure of inflammation).
• There were no serious adverse events reported. 
• Three moderate treatment-emergent adverse events were reported, though unrelated to belcesiran, the Company said.
• No clinically significant changes in lung function or laboratory tests were reported.
• Price Action: DRNA shares are down 5.51% at $37.78 during the premarket session on the last check Wednesday.