DMD : SMT C1100에 대한 놀라운 결과

SMT C1100, 성숙한 근육 utrophin 단백질 수준을 높여DMD,BMD를 치료하기 위해 개발 된 실험적인 약은 임상 1B 시험에서 놀라운 결과를 보여 주었다; 새로운 시도는 나중에 2014 년에 계획되어있다.

약은 MDA의 지원으로, 옥스퍼드, 영국, 생명 공학 회사 ,Summit PLC에 의해 개발되고 테스트되고 있다.

DMD 12 소년의 위상 1B 시험은 SMT C1100 안전하고 잘 용납이 경구 약물의 단기 투여는 혈청 CK 수준의 감소, 근육 건강의 긍정적 인 지표와 연관된 것으로 나타났다.

2014년 4분기에 다음 임상이 시작된다네요.

원문나갑니다.

출처:http://quest.mda.org/news/dmd-encouraging-results-smt-c1100

원문

Preliminary results of a phase 1b trial of the experimental drug SMT C1100 in 12 boys with Duchenne muscular dystrophy (DMD) show the oral compound was safe and well-tolerated at all doses tested but that blood levels of the drug varied among trial participants. The company says the variability in blood levels of the drug may be related to dietary intake in the participants or to other factors related to DMD.

In addition and encouragingly, most of the study participants showed a reduction in blood levels of the enzyme creatine kinase (CK), which leaks out of damaged muscle fibers and is considered an indication of ongoing muscle damage.

Summit PLC, an Oxford, United Kingdom, biotechnology company that has received significant funding from MDA to support the development of SMT C1100, announced these results in a May 21, 2014, press release.

The company says it expects to revise future clinical trials plans to determine the optimal way, either through dietary means or drug formulation changes, to address the blood level variability. Summit plans to start the next clinical trial of SMT C1100 in the fourth quarter of 2014.

Utrophin may help compensate for loss of dystrophin

SMT C1100, in development at Summit with MDA support, is designed to maintain levels of a protein known as utrophin in muscle fibers. This protein is normally made during early muscle development and regeneration but nearly disappears and is replaced by dystrophin as muscle fibers mature.

MDA-supported studies conducted in dystrophin-deficient mice have previously shown that raising utrophin protein levels may help compensate for a lack of dystrophin, which is missing in patients with DMD and diminished in patients with the related disorder Becker muscular dystrophy (BMD). (SMT C1100 may also have benefit in BMD patients, but tests in BMD have not been conducted to date.)

About the phase 1b trial

Trial participants were between 5 and 11 years old, and all received SMT C1100 in this open-label study, conducted in the United Kingdom. (There was no placebo group.)

The 12 participants were divided into three dosage groups, with each group receiving a higher daily dosage than the previous group. Participants received SMT C1100 for 10 days.

Unexpected impact on muscle health markers

"This first-ever trial of a utrophin modulator in DMD patients demonstrated the excellent safety profile of SMT C1100 with the study successfully achieving its primary endpoint," said Glyn Edwards, CEO of Summit. "In addition, the positive impact on the enzyme markers of muscle health was both unexpected and exciting, with these data potentially representing SMT C1100's first signs of activity in DMD patients."

He continued, "Our goal with utrophin modulation is to treat all patients with DMD. Armed with a greater understanding of the importance of diet and other disease-related factors, our future clinical trial plans will be modified and will increase the potential likelihood of achieving this goal."

For more information

To learn more, see the following.

• Phase 1b Clinical Trial in DMD Patients: Preliminary Results, a May 21, 2014, 10-minute webcast presented by Summit CEO Glyn Edwards
• Phase 1b Study of SMT C1100 in Subjects with Duchenne Muscular Dystrophy, which can also be accessed by entering NCT02056808 in the search box at ClinicalTrials.gov