Zafgen治疗下丘脑损伤引起的肥胖症药物beloranib中期研究再获成功

Zafgen治疗下丘脑损伤引起的肥胖症药物beloranib中期研究再获成功

                               
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发布日期：2015-01-09  来源：生物谷

最近，Zafgen医药公司宣布，公司开发的专门用于治疗下丘脑损伤引起的肥胖症（HIAO）药物beloranib在又一次临床二期研究中取得了显著进展。

                               
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不知不觉中，肥胖已经成为了摆在许多人面前的一个难题。随着对肥胖研究的深入，人们逐渐发现一些患者可以说是无辜的"被肥胖"。在临床研究中，科学家发现一些脑部（特别是下丘脑）受到损害的患者经常会出现肥胖的并发症状，而这种伴生的肥胖相比于正常的肥胖症更为复杂。

最近，Zafgen医药公司宣布，公司开发的专门用于治疗下丘脑损伤引起的肥胖症（hypothalamic injury-associated obesity，HIAO）药物beloranib在又一次临床二期研究中取得了显著进展。

在这项二期研究中，研究人员招募了十四名HIAO患者，实验组患者接受两周一次的beloranib注射治疗。相对于对照组患者，实验组患者在四周治疗和八周治疗后分别平均减重7.5磅和13.7磅。达到了之前研究人员的首要实验预期终点。另一方面，这次临床二期研究又一次验证了beloranib的安全性。

研究人员称接受beloranib的患者血脂含量和炎症情况均获得了明显改善，而后者则与心血管疾病的发病密切相关。但Zagen公司并未因此而满足，公司表示目前研究人员正在着手进行beloranib治疗普达-威利综合症的临床三期研究。普达-威利综合症是一种罕见疾病，会导致患者产生不必要的饥饿感从而暴饮暴食，进而肥胖。

Zafgen公司计划于2015年向美国和欧洲的医药部门提交beloranib的相关数据以获得上市许可。beloranib主要是通过作用于一种名为metaP2的酶而达到治疗作用。metaP2主要在脂肪酸的代谢过程中起作用。HIAO是一种罕见病，进行手术治疗移除脑癌时患者脑部受损会引发这一疾病。据统计，美国每年有超过五百例HIAO新增病例。

详细英文报道：

Zafgen ($ZFGN) charted a third major victory for its in-development obesity drug, spurring weight loss for patients who have endured brain injuries and stoking hopes for success in Phase III.

Treatment with the drug, beloranib, helped sufferers of hypothalamic injury-associated obesity (HIAO) lose significantly more weight than with placebo in a Phase II trial, Zafgen said. Enrolling 14 patients, the company found that those treated with the twice-weekly injection lost an average 7.5 pounds at four weeks and 13.7 pounds at 8 weeks, beating out the placebo group's 0.7 pounds and meeting the study's primary endpoint. Beloranib also proved itself safe and well-tolerated, Zafgen said, and the drug led to demonstrable improvements lipid levels and inflammation, which correlate with cardiovascular disease risk.

With the success in HIAO, Zafgen has now gone 3-for-3 in beloranib's Phase II program. The subcutaneous treatment previously came through in a midstage trial on the rare Prader-Willi syndrome (PWS), which leads to insatiable hunger, and posted excellent results against severe obesity. Now Zafgen is working through a Phase III trial in PWS with eyes on a future FDA filing.

"We are extremely pleased with these results, which differentiate beloranib from other weight-loss agents," Zafgen Chief Medical Officer Dennis Kim said in a statement. "... We believe beloranib shows tremendous potential to improve the lives of those impacted by HIAO and PWS, and for whom there are limited effective pharmaceutical alternatives. In 2015, we aim to establish the regulatory path for a registration program with U.S. and EU regulatory authorities."

Beloranib works by blocking metaP2, an enzyme involved in fatty acid production, thereby decreasing lipid production in the liver and fat storage while increasing metabolism. The drug's promise helped Zafgen pull off a $103 million IPO last year, and a steady stream of good news has helped the biotech's shares more than double since its debut.

HIAO, an orphan indication, is most commonly the result of surgeries to remove brain tumors, Zafgen said, with as many as 500 new cases cropping up in the U.S. each year.

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