Medicines shares surge on early-stage study suggesting cholesterol drug's effectiveness

Shares of The Medicines Co. surged in premarket trading Monday after the company and its partner Alnylam Pharmaceuticals Inc. reported study results suggesting effectiveness for a potential cholesterol drug.

The companies said that results from an ongoing Phase 1 clinical trial showed that their experimental treatment ALN-PCSsc lowered bad cholesterol by up to 83 percent compared to published results from studies with anti-PCSK9 MAbs, another class of drugs aimed at treating cholesterol. Both classes of drugs work by blocking a gene related to high cholesterol.

Shares of Medicines jumped $5.98, or 17.8 percent, to $39.62 in premarket trading.

The study only involved 69 patients and focused on safety. Phase 1 clinical trials are too small to test for a drug's effectiveness, though companies will suggest if a drug candidate seems effective based on the small pool of data. Also, Alnylam and Medicines are comparing figures in their ongoing study to published figures in prior studies and not on a head-to-head basis with competing treatments.

In this case, Medicines and Alnylam are testing the drug candidate against a class of biotechnology-based drugs called anti-PCSK9MAbs that lower cholesterol levels. Regeneron Pharmaceuticals Inc. and Sanofi sell Praluent and Amgen Inc. sells Repatha in that category.

Alnylam and Medicines said their early-stage study suggests that reductions in cholesterol were maintained for over 140 days, which supports once-quarterly or biannual injections of the developing drug.

The companies plan to move the drug candidate into Phase 2 clinical development by the end of the year and Phase 3 clinical development by end of 2017. Those studies will be larger and focus on the drug's potential effectiveness.