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AZ, 윌슨병 3상 '1차 충족'.."알렉시온 인수 성과" - 바이오스펙테이터

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AZ, 윌슨병 3상 '1차 충족'.."알렉시온 인수 성과"

입력 2021-08-31 07:17 수정 2021-08-31 13:52

바이오스펙테이터 윤소영 기자

이 기사는 '프리미엄 뉴스서비스 BioS+' 기사입니다.
'ALXN1840', 조직내 구리와 결합해 체외로 배출..기존 치료제 대비 3배 효능

아스트라제네카(AstraZeneca)가 알렉시온(Alexion)을 인수하면서 얻은 윌슨병(Wilson disease) 치료제 후보물질 ‘ALXN1840’이 임상 3상에서 기존 치료제 대비 좋은 효능을 냈다. 아스트라제네카는 이번 긍정적인 임상 결과를 바탕으로 규제당국에 승인심사 서류를 제출할 예정이다.

아스트라제네카는 윌슨병 약물 ‘ALXN1840’으로 진행한 임상3상 결과를 26일(현지시간) 발표했다. 윌슨병은 ATP7B(ATPase Copper Transporting Beta) 유전자의 변이로 다양한 장기에 구리(copper)가 쌓여 발병하는 희귀 유전질환이다. 윌슨병은 미국에서 3만명에 한명 꼴로 발병하는 비교적 흔한 희귀질환이다.

ATP7B는 간과 신장 등의 조직에서 구리를 세룰로플라스민(ceruloplasmin)과 결합시켜 혈액으로 내보내는 역할을 한다. 이러한 기전으로 혈액내 구리의 95% 이상은 세룰로플라스민과 결합해 있다. 일반적으로 구리 섭취량은 체내에서 필요로 하는 양보다 많기 때문에 지속적으로 구리를 배출시켜 체내 구리 농도를 일정하게 유지하는 것이 중요하다. 하지만 ATP7B에 변이가 생겨 ATP7B가 정상 기능을 하지 못하는 경우 구리는 조직 밖으로 배출이 되지 않는데, 이로인해 구리가 간, 뇌, 안구 등에 쌓이면 장기 손상을 일으킨다.

ALXN1840(bis-choline tetrathiomolybdate)은 구리와 높은 친화도를 갖도록 설계된 경구용 약물이다. 조직내의 구리와 결합한 ALXN1840이 체내에 존재하는 알부민(albumin)과 결합하게 되면 안정적인 형태의 3중결합체를 형성한다. 이렇게 형성된 3중결합체는 담즙 배설(biliary excretion)을 통해 체외로 배출된다....

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