Magazine Article | April 7, 2017

Tackling The Challenges Of A Rare-Disease Clinical Trial

By Ed Miseta, Chief Editor, Clinical Leader
Follow Me On Twitter @EdClinical

Regulatory agencies today are looking for Phase 3 trials to demonstrate a reduction in mortality as well as greater patient mobility. Unfortunately, Phase 3 trials also cost a lot of money to run, making them a challenge for small biotech firms. For a rare disease, the challenge is even greater, since patient recruitment is more difficult.

Jean-Louis Dasseux knows just how difficult it can be to run these trials. Dasseux is president and CEO of Cerenis Therapeutics, which is headquartered in France and focused on cures for cardiovascular disease (CVD). “Every year CVD results in the death of 17.3 million people, or one in every three deaths,” he explains. Current treatments, which attempt to lower low-density lipoprotein (LDL, also known as bad cholesterol), only reduce cardiovascular events by 25 to 35 percent. The drug in development by Cerenis attempts to mimic the role of natural high-density lipoprotein (HDL, also known as good cholesterol). Low levels of HDL caused by genetics have no current treatment and qualify as a rare disease.

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