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버텍스, AATD 'VX-814' 2상 중단.."간독성 이슈" - 바이오스펙테이터

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버텍스, AATD 'VX-814' 2상 중단.."간독성 이슈"

입력 2020-10-16 07:41 수정 2020-10-16 07:41

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AATD(Alpha-1 AntiTrypsin Deficiency) 치료제 후보물질 ‘VX-814’, 일부 환자들에게서 8배 높은 간수치.. ‘VX-864’로 AATD 치료제 개발 진행

버텍스(Vertex)는 AATD(Alpha-1 AntiTrypsin Deficiency) 치료제 후보물질 ‘VX-814’ 임상 2상에 참여한 일부 환자들에게서 높은 간수치가 나타나 임상을 중단했다. 버텍스는 다른 AATD 치료제 후보물질인 ‘VX-864’로 임상을 진행하며 AATD 치료제에 대한 개발을 이어나갈 예정이다.

버텍스는 지난 14일(현지시간) 보도자료를 통해 AATD 치료제 후보물질 ‘VX-814’에 대한 임상2상을 중단한다고 발표했다.

VX-814는 버텍스의 AATD 치료제 후보물질로서 50명의 환자를 대상으로 임상2상(NCT04167345)을 진행했던 약물이다. 버텍스는 이번 임상을 통해 VX-814의 효능과 안전성, 약물동태(Pharmacokinetic, PK)에 대한 평가를 목표로 했다. 그 결과 일부 환자들의 간수치(AST/ALT)가 높게 나왔는데, 특히 서로 다른 용량의 VX-814를 투약했던 4명의 환자들에게서 간수치가 정상범위보다 8배 높게 나왔다.

이에 버텍스는 VX-814가 효능을 낼 수 있는 투약 용량으로는 안전성에 도달할 수 없다고 판단, VX-814에 대한 임상을 중단했다. 버텍스는 간수치가 높아졌던 환자들은 치료를 받고 있으며, 그 환자들에게서 황달의 정도를 나타내는 빌리루빈(bilirubin) 수치의 증가는 나타나지 않았고 모두 다른 증상이 없었다고 밝혔다.

Carmen Bozic 버텍스 CMO(Chief Medical Officer)는 “VX-814 임상에 참여한 환자들과 조사관들에게 감사를 표하며 우리는 이 질병을 치료하기 위한 다른 치료제를 고려해야 한다”며 “AATD를 타깃하는 다른 치료제 후보물질 'VX-864'에 대한 임상을 계속 진행할 것”이라 말했다.

VX-864는 VX-814와 구조적으로 다른 약물로, 현재 40명의 AATD환자를 대상으로 임상2상을 진행하고 있다(NCT04474197). 버텍스는 VX-864로 AATD 치료제 개발을 이어나갈 계획이다.

알파-1-항트립신결핍(Alpha-1 AntiTrypsin Deficiency, AATD)은 SERPINA1 유전자의 변이로 알파-1항트립신(Alpha-1 AntiTrypsin, A1AT) 단백질이 잘못 접히면서(misfolding) 나타나는 질환이다. A1AT는 감염이나 염증이 발생했을 때 면역반응에 의해 분비되는 단백질 분해 효소로부터 폐와 장기들을 보호한다. A1AT의 비정상적인 접힘은 A1AT가 생성되는 간을 떠나지 못하게 해 폐와 같은 장기들을 보호할 수 없게 한다.

VX-814와 VX-864는 A1AT의 비정상적인 접힘을 바로잡는 저분자 화합물(small molecules)로 A1AT 분비를 돕고, A1AT가 정상적인 기능을 할 수 있게 한다.

한편, 지난 8일 다케다(Takeda Pharmaceutical)는 애로우헤드(Arrowhead Pharmaceuticals)의 임상2상 단계 AATD 타깃 RNAi 치료제인 ‘ARO-AAT’에 대해 10억4000만달러 규모의 공동 개발·상업화 및 라이선스 계약을 체결했다.

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