Arrowhead Research Corporation is looking to add another entrant into the clinical trial-based race to commercialize the first RNAi-based compound, a market category that could become a new class of drugs akin to monoclonal antibodies and produce revenues exceeding $1 billion by 2020 according to one projection. The company recently announced that it has submitted its Investigational New Drug application for its RNAi candidate, ARC-520, for the treatment of the chronic hepatitis B virus.
Alnylam today announced the addition of RNA interference therapies for the hepatitis delta virus and chronic liver infections to its clinical pipeline at the American Association for the Study of Liver Diseases in Boston. The company also said it remains on track to select a candidate for its hepatitis B program by the end of the year and hopes to file an investigational new drug application with the FDA in late 2015.
Arrowhead Research watched its shares nose-dive after a peek at Phase II data on its in-development treatment for hepatitis B revealed that the RNAi therapy isn't measuring up to its preclinical promise.
Arrowhead Research reported on Aug. 12 that patients in its Phase II clinical trial of the ARC-520 RNA interference candidate for hepatitis B had a "similar" knockdown to those reported in primate studies. The stock price fell at the very end of the trading day to close at $12.00 from an opening-day price of $13.00.
The drug is approved for treating--not preventing--hepatitis B, prompting FDA to call the advert misleading.
To help design next-generation drugs for hepatitis B and C, researchers have developed a humanized mouse model that contains human liver cells and mimics the immune system of people.
RNAi specialist Alnylam is adding a hepatitis B virus therapy to its pipeline of candidates that employ its Enhanced Stabilization Chemistry-GalNAc-conjugate technology enabling subcutaneous dosing and plans to file an IND by the end of 2015.
FDA and EMA requests for more data on hepatitis B candidate Heplisav have set Dynavax Technologies back time and again. Now, Dynavax is embarking on a new trial it hopes will address some of those safety concerns.
Arrowhead, fresh off a $120 million public offering in February, is paving the way on what it has called a "functional cure" for hepatitis B, beginning dosing in a Phase IIa trial for its RNAi drug designed to deliver the genetic material to the nucleus using a polymer-based conjugate.
Last week research firm GlobalData tipped the hepatitis B vaccine market to top $1 billion by 2022, with sales of Sanofi's Hexyon driving the sector's modest growth. Dynavax hopes to claim a slice of the market too, but its hepatitis B vaccine received another blow this week.