Investigating therapies for rare diseases and common diseases that have a genetic component
We are developing a pipeline of RNAi therapies that aim to restore health by addressing the underlying cause of disease. We currently have three clinical development programs:
- PHYOX™ Clinical Program: evaluating nedosiran (DCR-PHXC) for the treatment of all three known genetic types of primary hyperoxaluria (PH)
- Hepatitis B Virus (HBV) Infection Clinical Program: investigating DCR-HBVS for the treatment of patients with non-cirrhotic chronic HBV infection
- SHINE Clinical Program: developing DCR-A1AT for the treatment of patients with alpha-1 antitrypsin deficiency-associated liver disease
PHYOX1 (DCR-PHXC-101) is a study of nedosiran in healthy volunteers (HVs) and patients with PH1 and PH2. Results from the PHYOX1 clinical trial show normalization or near normalization of urinary oxalate levels in a majority of participants and favorable safety and tolerability profiles after a single dose of nedosiran for further clinical evaluation.
PHYOX2 (DCR-PHXC-201) is a double-blind, randomized, placebo-controlled trial in approximately 36 patients with PH1 and PH2. PHYOX2 is a pivotal trial, and data from this trial will be submitted to the U.S. Food and Drug Administration (FDA) for approval of nedosiran.
PHYOX3 (DCR-PHXC-301) is a long-term, multi-dose, open-label, rollover extension trial designed to further evaluate the safety and efficacy of nedosiran.
Participants in this trial will receive the same frequency and dose level as patients in PHYOX2. This will enable continuous readouts of multi-dose data, making PHYOX3 a proxy for the PHYOX2 pivotal trial data.
Patients enrolled in clinical trials of nedosiran may remain on treatment until drug approval by entering PHYOX3.
We are working with Roche to develop DCR-HBVS for the treatment of patients with non-cirrhotic chronic HBV. A randomized, placebo-controlled Phase 1 clinical trial designed to evaluate the safety and tolerability of DCR-HBVS in healthy volunteers and in patients with non-cirrhotic chronic HBV is ongoing.
We are investigating DCR-A1AT in the SHINE clinical development program. The Phase 1/2 clinical trial, named ESTRELLA, is a first-in-human study that we will conduct in two phases: a single ascending-dose (SAD) phase in healthy volunteers (HV; Group A) and a multiple ascending-dose (MAD) phase in participants with alpha-1 antitrypsin (A1AT) deficiency-associated liver disease.