A U.S. biotech company will work with a Swiss pharmaceutical giant to develop and commercialize RNA interference-based therapies for chronic hepatitis B.
Cambridge, Massachusetts-based Dicerna Pharmaceuticals said Thursday that it had entered a partnership with Roche to develop RNAi-based drugs for HBV, with a particular focus on the drug DCR-HBVS, which is currently in Phase I development. Under the deal, Roche will pay Dicerna $200 million upfront, as well as up to $1.47 billion in milestone payments. Dicerna will have the option to fund registrational development of the drug worldwide and promote in the U.S. together with Roche with enhanced royalties.
Shares of Dicerna rose nearly 15 percent on the Nasdaq Thursday morning following the news.
“With its deep expertise in HBV and established global infrastructure, Roche is ideally suited to help us accelerate the development and commercialization of DCR-HBVS, pursue a cure for chronic HBV infection and address this serious global threat to public health,” Dicerna CEO Douglas Fambrough said in a statement.
In a note to investors, Cowen analyst Yaron Werber wrote that the deal means Dicerna will likely provide full Phase I data for its drug in the middle of next year, as there will now be less need for data on the first cohort of patients. The deal with Roche, he wrote, provides an important validation, lucrative terms and an experienced partner.
Roche markets the injected biologic Pegasys (peginterferon alfa-2a) for hepatitis B and C. Meanwhile, Gilead Sciences markets Hepsera (adefovir dipivoxil) for chronic hepatitis B. Gilead’s annual report for 2018 grouped sales figures for Hepsera together with the hepatitis C drug Sovaldi (sofosbuvir) and the antibiotic Cayston (aztreonam), with the three having combined global sales of $320 million.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
RNAi is a type of drug mechanism that involves RNA molecules inhibiting the expression of genes. Other companies developing RNAi-based medicines include Alnylam, which is developing the drug inclisiran under a partnership with The Medicines Co. That drug is designed to lower cholesterol by inhibiting the production of the protein PCSK9 in the liver.
Another RNAi drug in development for hepatitis B is ARC-520, under development by Pasadena, California-based Arrowhead Pharmaceuticals. A study published last week in the journal Hepatology found that the drug was active in patients with hepatitis B e antigen-positive and -negative disease.
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