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 (NASDAQ:RGLS), 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 (NASDAQ:GILD) 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.
Sean Williams has no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong.
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