Concert Pharma公布新数据助推抗癫痫药物C-10068进入临床研究

Concert Pharmaceuticals公司是一家致力于开发中枢神经系统相关疾病的生物医药公司。不同于其他公司，Concert医药公司相信利用氘代化合物能够为治疗包括癫痫、痉挛等神经系统疾病开辟一条新道路。而2014年，凭借在这一领域的出色工作，Concert公司成功进行了一项总额达84亿美元的IPO。

最近，公司再次公布了其开发的氘代化合物疗法C-10068在动物模型上治疗癫痫的最新成果。C-10068是一种含氘元素的sigma-1拮抗剂，这种药物被认为能够通过作用于神经通路来治疗包括癫痫在内的一系列中枢神经系统相关疾病。

根据公司最近发表在Journal of Neurotrauma上的最新研究成果显示，C-10068在大鼠模型上表现出了良好的治疗效果。研究人员利用外伤性脑损伤大鼠模型检测C-10068的治疗效果，结果发现C-10068对癫痫大鼠有着出色的治疗效果。同时，C-10068的治疗效果还呈现出剂量依赖的关系，随着药物剂量的提升，大鼠的非痉挛性癫痫症状缓解率从20%增至70%，而癫痫持续时间也有明显缩短。

对于这一结果，Concert公司可谓是欢欣鼓舞。公司的首席营运官Nancy Stuart表示，下一步公司将着力开始推动C-10068进入临床研究阶段，他同时还坚信C-1006是证明氘代化合物能够辅助提高CNS相关疾病药物的疗效的又一力证。

不过，也有分析人士并不买账并直言在中枢神经系统研究领域，大鼠模型的相关研究并不可靠。Concert公司仍需要进行更多的研究才能得到更准确的结论。

详细英文报道：

A year after Concert Pharmaceuticals translated its work using deuterium to amp up the efficacy and stability of drugs into an upsized $84 million IPO, the biotech says it's completed a positive animal study on a new treatment designed to preventseizures. And now the biotech ($CNCE) wants to complete a preclinical foundation that can be used to support its advance into the clinic.

Their drug is called C-10068, a deuterium-containing sigma-1 agonist that is based on a compound initially developed at the Walter Reed Army Institute of Research. It has an impact on neurochemical pathways in the central nervous system.

Using a rat model for traumatic brain injury, the investigative team says that the drug spurred a dramatic, dose-dependent reduction of nonconvulsive seizures ranging from 20%, 42% and 70%. The animal model also demonstrated a reduction of the duration of seizures.

It should be noted here that rodent models for CNS have often proven to be unreliable in testing therapies preclinically. But Concert says that after more testing, it will seriously consider adding this treatment to its pipeline of clinical-stage therapies.

"C-10068 represents an opportunity in our R&D pipeline that may be considered for advancement into the clinic in the future based on additional preclinical studies," said Nancy Stuart, the chief operating officer of Concert Pharmaceuticals. "C-10068 is another example of how applying deuterium chemistry can enhance the pharmacologic properties of novel therapeutics for CNS diseases, and builds on our pipeline of drug candidates for a range of CNS diseases including spasticity, narcolepsy, Alzheimer's agitation and major depressive disorder."