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.
Market research firm Allied Market Research forecasts that RNA-based therapies will generate $1.2 billion in revenue by 2020, a compounded annual growth rate of 28.4%, according to a recent report.
A while ago, there was talk of a dreaded biotech bubble, and Novartis' decision to slam the breaks on its RNA interference research was making waves. But lately, the talk has turned positive on the promising yet unproven treatment paradigm, which utilizes RNAi to deliver gene-based therapy.
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.
Alnylam announced July 22 that its intellectual property estate has been strengthened due to the U.S. Patent Office's issuance of a Notice of Allowance for claims in one of its patent applications that covers "chemically modified RNA therapeutics conjugated with an N-acetylgalactosamine (GalNAc) ligand independent of length, sequence, or disease target," according to a statement.
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.
One of the chief drawbacks of RNA interference therapies so far has been the difficulty of delivering small interfering RNA to cells outside the liver. That's why a newly reported breakthrough in delivering siRNA to endothelial cells in the lung and other organs is a big deal.
The COO of RNAi specialist Alnylam was critical of Big Pharma in the wake of Novartis' decision to slam the breaks on its RNAi research. The news made investors nervous about the future of the promising, but nascent technology and Alnylam stock price is down more than 20% in the last week.