Dicerna Pharmaceuticals has secured a significant partnership with Roche, involving a $200 million upfront payment for the rights to a gene-silencing drug designed to treat hepatitis B virus (HBV) infections. The agreement could extend to $1.47 billion, contingent upon the drug, DCR-HBVS, successfully reaching the market and achieving sales milestones.
DCR-HBVS is in the early stages of clinical testing, specifically phase 1, and aims to deactivate genes that the HBV virus exploits to infiltrate liver cells. This innovative approach could potentially offer a functional cure by maintaining the virus at undetectable levels, thereby preventing its transmission.
The collaboration underscores the urgent need for more effective HBV treatments, despite existing preventive vaccines and oral therapies like tenofovir and entecavir. These current treatments can manage HBV infections but do not eliminate the virus, often necessitating lifelong therapy. Chronic HBV can lead to severe liver conditions, including cirrhosis and liver cancer, and affects approximately 257 million people globally, according to the World Health Organization.
Roche's investment in DCR-HBVS adds to its portfolio of HBV treatment candidates, which includes several early-stage clinical trials. Dicerna retains the option to co-fund the global pivotal development of DCR-HBVS, which would grant the company higher royalties and co-promotion rights in the United States.
This partnership further validates Dicerna's RNA interference (RNAi) platform, following previous collaborations with Eli Lilly, Boehringer Ingelheim, and Alexion. RNAi technology, which involves using RNA molecules to silence disease-causing genes, represents a cutting-edge approach in medical treatment, with recent FDA approvals for drugs targeting inherited amyloidosis.
Dicerna's RNAi strategy distinguishes itself by utilizing longer, double-stranded RNAs linked to sugar side chains, enhancing the potency and durability of the RNA's activity against messenger RNA (mRNA). This innovative method positions Dicerna alongside other companies like Arbutus and Arrowhead Pharma, which are also exploring RNAi-based solutions for HBV treatment.
