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 Dicerna Pharmaceuticals Inc (NASDAQ: DRNA ) are trading over 20% higher today following the company's pre-market press release announcing positive preclinical trial results for DCR-PH1.
So what: Dicerna recently presented preclinical data on the aforementioned DCR-PH1 at the latest Primary Hyperoxaluria Workshop in Chicago. The drug is a treatment for primary hyperoxaluria type 1 (PH1), which is a rare inherited liver disorder that often results in severe kidney damage. Dicerna's study involved genetically modified mouse models, which showed a 97% reduction of the HAO1 gene thought to be responsible for PH1 in their livers after just one dose of DCR-PH1, and which also showed near-baseline levels of urinary oxalate.
Dicerna plans to begin phase 1 (human) trials for DCR-PH1 next year. According to Dicerna's press release, PH1 afflicts anywhere from 300 to 900 people in the United States (its estimated prevalence is one to three cases per million people), and they must often undergo kidney and liver transplants to avoid death.
Now what: The mouse data was certainly encouraging, but there's a big difference between curing a few mice and helping a specific human patient population. It's likely to be years before DCR-PH1 reaches commercialization, if it actually does, and Dicerna's track record as a public company -- shares are still over 50% below their IPO price -- indicates less optimism than caution regarding the company's prospects. Keep in mind that even if Dicerna charges patients $100,000 a year to take a commercialized DCR-PH1, and it successfully reaches all of a potential 900 patients, the drug would still be worth roughly $90 million a year in revenue -- hardly blockbuster status, even considering Dicerna's tiny market cap. I'd watch from the sidelines for now.
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