Prosensa는 DMD 커뮤니티에 엑손 스키핑 프로그램 업데이트를 제공합니다

Prosensa는 DMD 커뮤니티에 엑손 스키핑 프로그램 업데이트를 제공합니다.

Drisapersen는 Prosensa의 수석 글락소 스미스 클라인은  엑손 스키핑 약물의 최근  실시한 3 상 시험에서 실망스러운 결과를 보여 주었다.

이들과 다른 drisapersen 시험 결과를 이해하고 Duchenne MD의 자연 역사 연구에서 데이터를 분석하는 Prosensa의 미래 엑손 - 건너 뛰는 약물 실험을 안내하는 데 사용됩니다,

이 회사는 2013년 11월 18일, 전화 회의 및 보도 자료에서 말했다 .

Prosensa는 세 개의 추가 DMD 엑손 - 건너 뛰는 임상 시험에서 약물 및 실험실 단계의 연구에서 두 가지가 있습니다.

이 뉴스도 DMD자연사연구 관련 뉴스입니다.

원문나갑니다.

The update, which covers the third quarter of 2013, comes two months after Prosensa announced disappointing results for a phase 3 trial of drisapersen, an experimental DMD drug it is developing in collaboration with GlaxoSmithKline (GSK).

Prosensa's pipeline

Drisapersen, originally known as PRO051, is designed to change how cells interpret genetic instructions for the dystrophin protein by targeting exon (section) 51 of the dystrophin gene. The dystrophin protein is missing in DMD-affected muscles because of any of a large number of mutations in the dystrophin gene.

Prosensa has three additional exon-skipping drugs for DMD now in clinical trials and two in preclinical (laboratory) testing.

PRO044 (targeting exon 44), PRO045 (targeting exon 45), and PRO053 (targeting exon 53) are being tested in early-stage clinical trials. PRO052 (targeting exon 52) and PRO055 (targeting exon 55) are still undergoing laboratory development.

Drisapersen analysis expected by end 2013

"We remain committed to drisapersen," said Hans Schikan, CEO of Prosensa on the Nov. 18 conference call. "And we will continue to work with GSK on [drisapersen] and our overall program, as well as working with patient groups in the U.S. and Europe to provide as much information as possible."

Schikan said they are now analyzing the results of all studies of drisapersen and expect to have the analysis completed by the end of 2013.

He also said Prosensa and GSK are making progress in their current study of the natural history (usual course) of DMD, and that this study and the drisapersen analysis will inform their current and future drug studies. "The timing of other studies may be affected" by new data, he said.

Seeking responders

Unfortunately, the phase 3 drisapersen trial (conducted at some 50 sites outside the U.S.) showed that the distance a trial participant could walk in six minutes ("six-minute walk distance") was not significantly different in those treated with drisapersen for 48 weeks compared to those treated with a placebo.

On the conference call, Schikan said the companies will look at how factors such as a participant's age, baseline six-minute walk distance and serum creatine kinase level (an indicator of muscle damage) may have correlated with six-minute walk test results. In so doing, they hope to identify possible subgroups of boys with DMD (for example, perhaps those below a certain age) who may have benefited from treatment with drisapersen.

He noted that it would be premature to discuss these analyses at this point and that the path forward for drisapersen is not known at this time.

Modifications to newer studies possible

Giles Campion, Prosensa's chief medical officer, said the drisapesen analysis "will help us gain insights into how we may need to adapt — if at all — our follow-on programs."

Campion also said that laboratory studies have shown the five exon-skipping compounds developed by Prosensa after drisapersen are more effective than drisapersen at causing exon skipping in cells.

출처:http://quest.mda.org/news/prosensa-provides-exon-skipping-program-update-dmd-community