Retrophin罕见病药物RE-024临床研究发现可能引起肝部损伤

Retrophin公司于今年一月份成功在纳斯达克上市以进行一系列罕见病药物的研发。然而最近公司进行的治疗一种罕见神经退行性疾病的药物RE-024在首次临床研究中暴露出严重副作用为其未来蒙上一层阴影。Retrophin公司是在上个月进入RE-024的临床研究的。当研究人员对招募的这名患者给药后发现患者血液中肝部酶的含量显着升高，最后迫使研究人员暂停给药并随后降低给药量。

RE-024主要是用于治疗泛酸激酶相关神经退行性疾病，这种疾病的发病率约为百万分之一到百万分之三之间。此次Retrophin公司的研究受挫可能会影响投资者对生物医药公司的热情。

详细英文报道：

Retrophin ($RTRX), the biotech venture of former hedge fund manager Martin Shkreli, startled investors Friday morning when it revealed that elevated levels of liver enzymes briefly derailed a study of its rare disease candidate, sending the company's shares down more than 8%.

Last month, the company dosed its first patient with RE-024, a treatment for a neurodegenerative disorder that affects between 1 and 3 per million people. After two weeks, that patient developed "a modest elevation of liver enzymes," Retrophin said, leading the company to discontinue treatment. Six days later, however, the patient had trended back toward liver health, and Retrophin re-started treatment at a reduced dose, keeping up treatment through this week with no meaningful elevation in liver enzymes, the company said.

The drug, designed to treat pantothenate kinase-associated neurodegeneration (PKAN), is among the biotech's fleet of rare disease treatments it believes can bring in between $60 million and $70 million in 2015. Retrophin has watched its share price soar since jumping onto the Nasdaq in January, buoyed by a constant stream of acquisitions for rare ailments like cerebrotendinous xanthomatosis, cystinuria and infantile spasms.

The brief pause in RE-024's clinical development is Retrophin's first blemish of 2014, and the swift Wall Street reaction could signal a fair amount of skepticism among investors. The biotech, naturally, was quick to downplay any fears.

"The company does not believe data on a single patient can be interpreted in a meaningful fashion and is releasing this data to accommodate investor requests," Retrophin said in its disclosure. "Further, the results are inconclusive at this time with regard to RE-024's ability to treat patients that suffer from PKAN. Potential investors should not place undue reliance on this single-patient data."