Pipeline

Pipeline

Developing next-generation RNAi therapies.

Dicerna has a growing pipeline of product candidates to address unmet medical needs in diseases involving the liver, including rare diseases, viral infectious diseases, chronic liver diseases, and cardiovascular diseases. Our optimized, subcutaneously administered GalXCTM molecules are designed to potently and selectively silence genes that are implicated in these disorders.

Key Clinical Programs

Primary Hyperoxaluria (PH)

Dicerna is developing DCR-PHXC, a subcutaneously delivered GalXCTM candidate for the treatment of patients with all three genetic forms of primary hyperoxaluria (PH), a family of severe, rare, genetic liver disorders characterized by overproduction of oxalate, a natural chemical in the body that is normally eliminated as waste through the kidneys. DCR-PHXC is being tested in a variety of PHYOX trials:

  • PHYOX1 (DCR-PHXC-101): Single ascending dose study of DCR-PHXC in healthy volunteers (HVs) and patients with PH 1 and 2
  • PHYOX2 (DCR-PHXC-201): Double-blind, randomized, placebo-controlled, pivotal trial (2:1 randomization) in ~36 patients with PH1 and PH2
  • PHYOX3 (DCR-PHXC-301): Long-term, multi-dose, open-label, rolloverextension trial allowing readouts of multi-dose data and long-term access to DCR-PHXC for patients who previously participated in PHYOX study

For more information about our PHYOX trials, please visit PHYOXTrials.com.

 

Chronic Hepatitis B Virus (HBV) Infection 

Dicerna’s DCR-HBVS is a GalXC RNAi platform-based product candidate for the treatment of chronic hepatitis B virus (HBV) infection. Current therapies for HBV rarely lead to a long-term immunological cure as measured by the clearance of HBV surface antigen (HBsAg) and sustained HBV deoxyribonucleic acid (DNA) suppression in patient plasma or blood. DCR-HBVS is currently being tested in the DCR-HBVS-101 clinical trial, a randomized, placebo-controlled study designed to evaluate the safety and tolerability of DCR-HBVS in HVs and in patients with non-cirrhotic chronic HBV.

For more information about our DCR-HBVS clinical trial, please visit clinicaltrials.gov, identifier: NCT03772249.

A1AT Deficiency-associated Liver Disease

Dicerna is developing DCR-A1AT, an investigational therapy from our GalXC technology platform for the treatment of alpha-1 antitrypsin (A1AT) deficiency-associated liver disease. A1AT deficiency is a rare genetic disorder that can cause liver disease in children and adults, leading to complications such as weight loss, fatigue, jaundice and life-threatening conditions such a cirrhosis. There are currently no approved therapies that treat the liver manifestations of this condition.

Dicerna announced submission of a Clinical Trial Authorization (CTA) application to the Swedish Medical Products Agency (MPA) on June 26, 2019 to conduct the first-in-human Phase 1/2 study of DCR-A1AT. The proposed parallel-group, placebo-controlled, Phase 1/2 study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of DCR-A1AT in adult healthy volunteers and patients with A1AT deficiency-associated liver disease. The study will consist of two phases:

  • Group A: a single ascending-dose phase in HVs, enrolling up to 36 participants in as many as six cohorts
  • Group B: a multiple ascending-dose phase in patients with A1AT deficiency-associated liver disease, consisting of up to 24 participants in three or fewer cohorts