Neurocrine Biosciences生物公司在去年由于其研發的用于治療罕見的運動機能紊亂疾病的50mg劑量藥物二期研究未能達到預定目標而宣告失敗使公司股價一度遭受重挫。然而現在公司研發的治療遲發性運動障礙新藥NBI-98854二期研究傳出了積極的結果,根據公司數據透露,此次二期研究的藥物劑量約為75mg,其效果相比去年的50mg劑量明顯許多。這一結果也促使公司股價飆升60%之多。NBI-98854是一種小分子VMAT2抑制劑,此次二期研究反映出其藥效和安全性都值得肯定。公司計劃在進行三期研究前將藥物提交到FDA進行預審核。直到現在,市面上并沒有一種特效藥治療遲發性運動障礙,這也使得市場對NBI-98854的期望相當之大。
詳細英文報道:
Back in September, shares of Neurocrine Biosciences ($NBIX) were subjected to a beating at the hands of investors unhappy with the news that the 50-mg dose of its experimental therapy for a rare movement disorder failed the primary endpoint in a Phase IIb study--even though the 100-mg dose performed as hoped. Last night, the biotech experienced a dramatic improvement in its fortunes as a 75-mg dose delivered the goods in a follow-up Phase IIb trial.
Shares of Neurocrine ($NBIX) shot up 60% on the successful study of NBI-98854, a small-molecule VMAT2 inhibitor designed to treat tardive dyskinesia. And with the mid-stage program wrapped on a positive note, the company says it's ready to ask the FDA for a meeting in advance of a pivotal, late-stage trial.
"The profound response in this Kinect 2 study demonstrates the potential of NBI-98854 as both a safe and highly effective treatment for patients suffering from tardive dyskinesia," said Christopher O'Brien, the chief medical officer of Neurocrine Biosciences, in a statement. "It is clear from these results that the use of blinded central AIMS raters coupled with the ability to titrate up to 75 mg of NBI-98854 were both critical to the success of this trial."
There are no therapies for this disease, which had helped spur some enthusiasm for the drug among some analysts.
Neurocrine's lead program is for elagolix, an endometriosis therapy that won a $575 million deal back in 2010 with Abbott ($ABT), which later spun off its drug business as AbbVie ($ABBV). That's still the primary focus of the company and analysts. A Phase III study for that drug was launched in 2012, and the partners say they can file for an approval in 2016, provided the data are right.
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