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Intercept治疗胆汁性肝硬化疾病药物OCA三期临床结果良好

2014年04月24日

最近美国生物医药公司Intercept Pharmaceuticals在欧洲肝病研究学会(European Association for the Study of the Liver, EASL)上公布了其治疗原发胆汁性肝硬化(primary biliary cirrhosis, PBC)药物OCA的临床三期研究成果。公司希望借此在今年晚些时候向欧洲和美国药物管理部门提出上市申请。

根据Intercept公司公布的数据,OCA能够降低患者体内的碱性磷酸酶水平约50%,而对照组仅为10%。同时数据还显示患者服用这种药物的两周后病情就有所改善,持续服用6个月后药效最为显著。另一方面,在这次临床三期研究中,研究人员并未发现OCA有非常明显的副作用。而目前市面上治疗 PBC的药物主要是熊去氧胆酸,这种药物对一些患者疗效并不明显。因此,Imtercept公司将OCA看作是占领PBC药物市场的重点产品。

详细英文报道:

Investigators for Intercept Pharmaceuticals spelled out the impact of its lead therapy on rare cases of a liver disease called primary biliary cirrhosis (PBC), fleshing out the results of a late-stage study that it will use to seek U.S. and European approvals later in the year.

Intercept ($ICPT) had already unveiled the key takeaway from Phase III: Its drug significantly reduced levels of alkaline phosphatase in close to half of all patients in two drug arms, compared to 10% of the patients taking a placebo. In a late breaker at a meeting of the European Association for the Study of the Liver (EASL), investigators added that the positive impact of the drug could be seen after two weeks in some patients, with peak efficacy at 6 months. Cases of pruritis--severe itching--were the chief concern in the study.

Intercept, based in New York with development activities in San Diego, has been positioning OCA as an important new therapy for a disease that has traditionally been treated with ursodiol. The older drug often works for the disease, a swelling of the biliary ducts that causes a backup of bile that threatens the liver, but executives for the biotech say that there's a big market waiting if they can get an approval for treating up to half of all patients who don't respond adequately to the mainstay drug.

"PBC has been a rather neglected disease, an orphan disease but not ultra rare, affecting 1 out of 1,000 women over the age of 40," David Shapiro, Intercept's chief medical officer, tells FierceBiotech. With up to half of those women achieving only a suboptimal response, "there's a need for a better therapy."

"This is a drug which is working in second line, which is the most difficult one," adds Frederik Nevens, the chief investigator in the study. Intercept has now tested this drug on more than 500 patients, he adds, in mid- and late-stage studies. And all the data point to its potential as a "highly effective" new treatment.

Shapiro also notes that there were no signs of cardiovascular side effects in the Phase III. Intercept had startled investors a few weeks ago with a 10-K filing noting some cardiovascular incidents among patients, but quickly managed to tamp down the fretting with the late-stage data.

The biotech believes OCA is a first-in-class agonist of the farnesoid X receptor with a broad ability to guard the liver. The NIH had been studying OCA for nonalcoholic steatohepatitis, or NASH, which bears all the signs of liver disease seen in alcoholics, but in people who don't drink. The disease is triggered by a diet high in fat and sugar, and it's been spreading around the world after growing into a serious health problem in the U.S. and Europe.