Esperion治療低密度膽固醇藥物即將進入臨床三期研究

　　總部設在密歇根州安娜堡的生物醫藥公司Esperion最近迎來了2015年的第一個好消息。公司透露FDA已經批準了公司開發的膽固醇藥物ETC-1002進入臨床三期研究。公司表示FDA已經取消了對于ETC-1002臨床研究不得超過6個月的限制。FDA此前對這種藥物的毒性表示了憂慮。不過，去年Esperion公司向FDA提交了大量的臨床前研究數據，使得FDA改變了這種看法。公司計劃在今年招募大約4000名患者進行臨床研究以檢驗其降低LDL膽固醇的效果。

　　近年來市場上不乏一些效果極佳的降LDL膽固醇藥物，一些制藥巨頭如安進公司、賽諾菲公司都在這一領域有自己的產品。不過，這些藥物大都屬于昂貴的抗體藥物。而ETC-1002則是一種小分子藥物。公司CEO認為ETC-1002將為許多患者提供除他汀類藥物以外的又一種選擇，而昂貴的抗體藥物如一些PCSK9抑制劑類抗體藥物也將降成為歷史。

　　作為一家小型生物醫藥公司，公司在去年進行了總額1億美元的IPO，但是公司表示目前還在尋找更具經驗的合作伙伴。（生物谷Bioon.com）

　　詳細英文報道：

　　Ann Arbor, MI, biotech Esperion Therapeutics ($ESPR) finally resolved the FDA snag standing in the way of its ambitious plans for a new cholesterol drug, clearing the company to begin late-stage trials with its potentially disruptive pill.

　　The agency has lifted a partial clinical hold that limited Esperion from running studies longer than 6 months, the company said. The FDA had previously expressed concern about the drug's toxicity, but, vetting preclinical data the biotech submitted late last year, regulators have given Esperion the go-ahead to keep moving.

　　Now the biotech plans to take its oral cholesterol fighter, ETC-1002, into Phase III later this year, CEO Tim Mayleben said. Esperion is planning to enroll about 4,000 patients in a late-stage study to confirm the treatment's effect on LDL cholesterol, marking the next step in Esperion's plan to shoulder in on the blockbuster race for next-generation cardio drugs.

　　ETC-1002 is a small-molecule therapy that has successfully slashed levels of LDL, or "bad," cholesterol in clinical trials. So, too, have the blockbuster-in-waiting injections from Amgen ($AMGN) and partners Sanofi ($SNY) and Regeneron ($REGN). But, unlike those likely expensive antibodies, Esperion's candidate is positioned as a cheaper, easier solution, Mayleben said at last month's JP Morgan Healthcare Conference.

　　As the CEO sees it, his company's pill will be an ideal second option for the millions of patients who either can't tolerate statins or can't get to healthy LDL levels on the generic drugs. The antibodies, which work by blocking the proteinPCSK9, will end up as third-line therapies, he said, subscribing to "the radical belief that patients will prefer an oral therapy over injection."

　　First Esperion will have to come through in Phase III, of course. Thanks to a nearly $100 million public offering late last year, the biotech has enough cash to go it alone, but Mayleben said Esperion may look to partner up with a larger drugmaker with experience running large cardiovascular trials.

　　Beyond ETC-1001, Esperion has only one other disclosed asset, a preclinical metabolic treatment. That's a reflection of what Mayleben said is an all-hands-on-deck devotion to the top prospect. Esperion is effectively a one-trick pony, he said, but that's "not so bad if you're riding Seabiscuit."