Dicerna readies alpha-1 antitrypsin deficiency-related liver disease drug for trial

Dicerna Pharmaceuticals submitted a clinical trial authorization application to the Swedish Medical Products Agency to conduct a phase 1/2 study of DCR-A1AT for the treatment of alpha-1 antitrypsin deficiency-related liver disease, according to a press release.

DCR-A1AT is an RNA interference therapeutic that incorporates Dicerna’s proprietary GalXC technology, designed to create selective and safe RNAi therapies that are specific and potent in disease targeting.

“The launch of the DCR-A1AT clinical program is welcome news to the A1AT deficiency community, as there are currently no approved therapies that treat the liver manifestations of this condition,” Miriam O’Day, president and CEO of the Alpha-1 Foundation, said in the release. “We look forward to working with Dicerna as they advance DCR-A1AT through clinical trials.”

The proposed study would comprise a single ascending-dose cohort of 36 participants and a multiple ascending-dose cohort of 24 patients with A1AT deficiency-related liver disease.

“We are pleased to begin the clinical development phase of our [alpha-1 antitrypsin (A1AT)] deficiency-associated liver disease program, which serves two roles in Dicerna’s portfolio,” Douglas Fambrough, president and CEO of Dicerna, said in the release. “First, A1AT deficiency-associated liver disease fits with our rare disease strategy as an indication with a significant unmet medical need and a clear biomarker, presenting a rapid development path to approval. Second, the program will inform and aid our efforts in the much broader chronic liver disease field, where we believe our GalXC platform can have a major impact, and which provides ample opportunities to collaborate with larger pharmaceutical company partners.”

Reference: www.dicerna.com