Insmed公司上周宣布公司開發(fā)的一種利用脂質(zhì)體載藥系統(tǒng)輸送的治療肺部感染的吸入式藥物Arikayce出現(xiàn)重大轉(zhuǎn)折。此前這一藥物的二期臨床研究結(jié)果顯示該藥物未能達到其預(yù)期目標(biāo)，然而現(xiàn)在68名完成這一研究的志愿者中，有21名患者已經(jīng)無法檢測到肺部感染的跡象。這一結(jié)果無疑為Insmed公司帶來一線希望。此前公司在今年三月份宣布這一二期臨床研究宣告失敗。

 Arikayce是一種以脂質(zhì)體形式藥物，它具有一個含水的內(nèi)核和一個磷脂雙分子層，保證其能夠同時裝載疏水性藥物和親水性藥物。同時磷脂雙分子層表面的負電荷能夠阻止細菌與肺部細胞的結(jié)合以進一步抑制感染。（生物谷Bioon.com）

 詳細英文報道：

 Insmed's ($INSM) Phase II trial of Arikayce, or inhaled liposomal amikacin for the treatment of nontuberculous mycobacterial lung infections (NTM), did not meet its primary endpoint, but new data released May 20 from the 84-day open-label extension show that patients receiving the compound experienced significant improvement, the company said.

 Out of 68 patients who completed both phases of the study, 21 showed no sign of the bacteria.

 Investors punished the company in March for failing to meet the primary endpoint during the 84-day double-blind phase, but judging by their positive reaction to the latest news (Insmed stock rose nearly 7%), the candidate and its proprietary liposomal technology for delivery of drugs to the lung is still promising.

 Arikayce is delivered in liposomes consisting of a water core containing water-soluble drugs and a hydrophobic chain bilayer interior containing water-insoluble drugs. Arikayce says the liposomes' neutral charge helps them treat infections by resisting attraction to negatively charged compounds on the surface of mucus and protective biofilm.

 Other advantages include localized delivery and the liposomes' small size, which the company says enables the drug to access bacteria within infected microphages. The technology is also being used in Insmed's Phase III candidate for the treatment of cystic fibrosis.

 Arikayce is inhaled using an investigational nebulizer developed by PARI Pharma.

 The study of Arikayce for the treatment of NTM consisted of an 84-day double-blind portion comparing the standard of care for NTM plus placebo to the standard of care plus Arikayce. It was followed by an 84-day open-label phase in which all subjects received Arikayce as a supplement to the standard of care. Patients in the trial were on standard care unsuccessfully for at least 6 months, according to the statement.

 Arikayce did not demonstrate statistical significance for the measurement of the change in mycobacterial density on a 7-point scale by the end of the double-blind phase of the trial. The trial resulted in a p-value of 0.148 for that measure, short of the prespecified target, according to Insmed's statement. However, the trial did meet its secondary endpoint for the proportion of subjects with culture conversion to negative.

 It will be interesting to watch how FDA interprets the mixed results from the trial. It has given the candidate Orphan Drug, Qualified Infectious Disease Product and Fast Track designations for the treatment of NTM.