Roche Bets $1.47 Billion on Functional Cure for Hepatitis B with Dicerna's RNAi Therapy DCR-HBVS

Today, Dicerna Pharmaceuticals, Inc. announced that it has entered into a research collaboration and license agreement with Roche to jointly develop innovative therapies for the treatment of chronic hepatitis B virus (HBV) infection. These therapies will be based on Dicerna’s proprietary GalXC RNAi technology platform. The collaboration will focus on the global development and commercialization of Dicerna’s investigational RNAi therapy, DCR-HBVS, and will also include the use of both companies’ technology platforms to develop therapies targeting multiple human and viral gene targets associated with HBV infection.

Hepatitis B virus (HBV) is the most common cause of severe liver infection worldwide. According to estimates from the World Health Organization (WHO), there are over 292 million people living with chronic hepatitis B globally. It causes more than 800,000 deaths annually. HBV is also the leading cause of liver cancer, which is the second leading cause of cancer-related deaths worldwide. Although current treatments for hepatitis B can suppress HBV replication, they rarely achieve a sustained “functional cure.”

Dicerna is a biotechnology company focused on developing innovative RNAi therapeutics. Because the HBV genome reuses the same sequence segments to generate multiple distinct mRNAs, the company’s RNAi therapeutic, DCR-HBVS, employs a single RNAi sequence to specifically knock down multiple genes associated with HBV mRNA production and HBV entry into hepatocytes.

Image source: Reference [2]

In preclinical animal studies, DCR-HBVS reduced hepatitis B surface antigen (HBsAg) levels in animals by more than 3.9 log units. This therapy is currently being evaluated in Phase 1 clinical trials, with the first patient receiving dosing in May this year.

Image source: Reference [2]

Under the agreement, Roche will obtain the global exclusive license for the research, development, and commercialization of DCR-HBVS. Dicerna will receive a $200 million upfront payment, up to $1.47 billion in milestone payments, and tiered royalties based on sales of products containing DCR-HBVS. Dicerna retains an option to co-develop DCR-HBVS.

“Dicerna is delighted to collaborate with Roche to realize the full potential of DCR-HBVS and leverage the GalXC platform to target and silence genes associated with chronic hepatitis B virus infection,” said Dr. Douglas M. Fambrough, President and Chief Executive Officer of Dicerna. “Roche is the ideal partner to accelerate the development and commercialization of DCR-HBVS. Together, we will develop curative therapies for chronic HBV infection, addressing a serious threat to global public health.”

Targeting the gene expression of the hepatitis B virus (HBV) has emerged as a promising therapeutic approach for achieving a “functional cure.” These therapies offer high specificity for HBV, significantly reducing serum hepatitis B surface antigen (HBsAg) levels and potentially leading to a substantial decrease in viral covalently closed circular DNA (cccDNA) levels. Achieving this reduction is a key objective in the functional cure of chronic HBV infection.

Currently, multiple biotechnology companies are utilizing RNA interference (RNAi) or antisense oligonucleotide therapies to target the gene expression process of hepatitis B virus (HBV). Meanwhile, large pharmaceutical companies are also establishing a presence in this field through research and development collaborations: Roche’s recent partnership, Johnson & Johnson’s collaboration with Arrowhead Pharmaceuticals last year, and GlaxoSmithKline’s (GSK) partnership with Ionis Pharmaceuticals this year all demonstrate the recognition by major pharmaceutical enterprises of this innovative therapeutic modality.

We look forward to this innovative treatment model bringing about a “functional cure” for chronic hepatitis B infection as soon as possible, thereby benefiting a vast number of patients.

References:

[1] Dicerna™ Enters Agreement with Roche to Develop and Commercialize DCR-HBVS for the Treatment of Chronic Hepatitis B Virus (HBV) Infection. Retrieved October 31, 2019, from https://www.businesswire.com/news/home/20191031005215/en/Dicerna%E2%84%A2-Enters-Agreement-Roche-Develop-Commercialize-DCR-HBVS

[2] Dicerna Corporate Overview. Retrieved October 31, 2019, from http://investors.dicerna.com/static-files/96600266-a42e-4065-8afd-90e3a98b4e0b

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