RNAi specialist Tekmira Pharmaceuticals expanded its reach with the announcement of the purchase of OnCore Biopharma, developer of an all-oral candidate for hepatitis B, saying that a combination therapy approach is the optimal way to treat that disease.
Alnylam and Isis Pharmaceuticals announced an intellectual property cross-licensing agreement that they hope will leave them both better equipped to deal with the challenges of developing the first commercial RNAi therapy, including utilization of the promising but still-unproven RNA interference drug delivery pathway, and sometimes jittery investors.
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.
Saying it is on track to exceed its goal of having 5 key products in clinical development through 2015, Alnylam recently announced that it intends to focus on three strategic therapeutic areas, or what it dubs "STArs," as it races to commercialize its first candidate delivered using the RNAi pathway and reassure sometimes antsy investors.
Researchers at the University of California in San Diego have developed a way to disguise the genetic material itself so that it can enter cells and shut down genetic diseases at the core.
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.
RNA interference specialist Arrowhead presented new data from an ongoing Phase IIa study of its hepatitis B treatment ARC-520, demonstrating a reduction of the disease's surface antigens in what the company says is a first for RNAi.
RNAi specialist Alnylam is focusing on rare diseases at the moment (with the exception of hepatitis B), but Chief Business Officer Laurence Reid told investors at the Leerink Rare Disease Roundtable conference that the company is also looking to enter bigger markets via partnerships.
RNAi leader Alnylam, the American Porphyria Consortium and The European Porphyria Network are launching the Explore observational study of patients with the rare disease, with the hope of advancing Alnylam's candidate for porphyria, with a goal of filing for approval in late 2014 or early 2015.
Tekmira, developer of an RNAi treatment for Ebola, has come to terms with regulators on an access program for its in-development drug, allowing the company to provide doses to patients who have contracted the deadly virus.