Although we don't believe in timing the market or panicking over market movements, we do like to keep an eye on big changes – just in case they're material to our investing thesis.
What: Shares of Regulus Therapeutics (RGLS -5.13%), a biopharmaceutical company focused on the development of therapies utilizing its proprietary microRNA product platform, saw its shares skyrocket as much as 127% at one point today after announcing the interim results for its human proof-of-concept trial for its early stage hepatitis C drug RG-101.
So what: According to Regulus' press release, RG-101 was safe and well-tolerated, and delivered sustained and significant viral load reductions with a single subcutaneous treatment in a varied group of hepatitis C genotypes, including tough to treat genotypes, and those who'd experienced a viral relapse. Regulus specifically noted that six of 14 patients had HCV RNA levels below quantification at day 29, signaling just how quickly the RG-101 appears to have worked in eliminating detectable levels of the disease. Additionally, given how successful RG-101 has been in this interim analysis, the study protocol was amended to follow patients for up to six months following their dosing.
Now what: Though we don't have a ton of empirical data on cure rates, aside from the six out of 14 patients that had no detectable levels of disease by day 29, the real allure of RG-101 is that it could cure varying types of HCV in a single dose!
The current standard of treatment is offered by Gilead Sciences (GILD -1.15%) through Sovaldi, or the newly approved Harvoni, which is a cocktail drug that combines Sovaldi and ledipasvir. Harvoni, which is widely expected to be the lead hepatitis C therapy moving forward, still requires HCV patients to take the drug for between eight and 24 weeks, depending on whether they're treatment-naïve or experienced, and whether or not they have liver cirrhosis. The thought here is that Regulus' single-dose cure could knock Harvoni out of the picture if its success rate is high and continues well beyond this interim proof-of-concept trial.
I personally wouldn't bet the boat on Regulus just yet as we're talking about an early stage study, but I would certainly advise adding Regulus to your watchlist.
