척수성 근위축증(SMA) 치료제 Olesoxime 임상3단계 중간 검토 보고

[환장머슴]

안녕하세요...

추위와 잦은 눈으로 몸과 마음이 힘들었던 겨울도 이젠 그 기세가 꺽이고 따뜻한 봄의 길목에 들어선 3월입니다.

회원님들 모두 건강하신지요..

일전에 척수성 근위축증(Spinal Muscular Atrophy) 치료제 개발 진행현황 전해드렸었는데.. 그 중 개발 진행 정도가 가장 빨랐던

Trophos社의 Olesoxime 치료제 개발에 대한 중간 Report가 지난 주에 있었나 봅니다.

임상3단계로 11년 부터 3세부터 25세 까지의 160명이 넘는 SMA 환우를 대상으로 임상을 진행해 오고 있었는데요..

지난 1년간의 임상결과에 대해 "The independent Data Monitoring Committee (DMC)"에서 Review 한 결과 긍정적인 Output이

나왔다고 하는군요.. DMC에서 이런 검토를 했다는 건 Olesoxime이 SMA 치료제로서 유효한 물질이며, 남은 기간의 임상실험을

계속해서 진행 할 수 있게되는 중요한 의미를 지닌다고 하네요.

올해 말이 되면 정말 좋은 소식이 있으리라 믿어 의심치 않습니다..

마지막 까지 좋은 결과가 나오길 기대하면서..희망의 끈을 놓지 말도록 합시다....

해당 보고내용 원문은 다음을 참고해 주세요...

http://www.trophos.com/news/pr20130227.htm

Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for under-served medical needs in neurology and cardiology, announced the completion of the interim analysis of the pivotal efficacy study of olesoxime in the rare neurodegenerative condition Spinal Muscular Atrophy (SMA).

The independent Data Monitoring Committee (DMC) has reviewed the treatment effect at one year on the primary outcome measure of efficacy, change in motor function using the MFM scale, together with the latest safety report including electrocardiogram traces, periodic laboratory findings, haemostatic parameters and serious adverse events listings for all participants. Based on the trial stopping criteria as defined in the protocol as well as no safety concerns related to olesoxime treatment, their recommendation is to continue the study as planned.

An interim analysis of efficacy, as included in the study protocol, has been conducted after all participants have been treated for one year. Over 160 patients have been recruited into the trial between October 2010 and September 2011 (see press release of  September 8, 2011). Following the recommendations of the DMC, the study will continue until all participants have been treated for two years with the last patient out scheduled for September 2013. Top line results are expected by the end of 2013.

The trial is substantially funded by Trophos' partnership with the Association Francaise contre les Myopathies (AFM-Telethon) (see press release of March 19, 2009). The trial protocol has benefited from the EMA protocol advice procedure.

"The approval from the DMC to continue the trial based on the interim analysis for this innovative treatment of SMA is perfect news at this stage. We confirmed the good safety of olesoxime treatment and we look forward to completing the two year treatment period to report efficacy in these SMA patients," said Rebecca Pruss, chief scientific officer at Trophos. "SMA is a progressive and disabling neuromuscular disease. Treatments are desperately needed that slow down or prevent the loss of neuromuscular function in SMA patients. This study of olesoxime has been conducted successfully so far due to the enormous commitment of patients and clinicians to find a treatment for SMA. We believe the results due in fourth quarter 2013 could be an historic moment in the development of treatments for SMA."

"The recommendation by the DMC to proceed confirm‎s that olesoxime is a safe molecule and we look forward to quickly analysing results on the efficacy outcome of the trial after it reaches the planned conclusion," said principle investigator Dr Enrico Bertini. "Other interesting potential outcomes that will surely emerge from the results of this trial will be related to the issue of biomarker analysis and also to the reliability, variability and validity of electrophysiological data which have been collected longitudinally in patients. All the information from this study will be valuable for future clinical multi-centre trials in SMA. In the meantime we hope that olesoxime will have a therapeutic impact on the natural history of SMA."

"Thanks to the donations to the French telethon, we have been supporting the development of olesoxime since the first screening up to and including the ongoing clinical trial. This is an important clinical study and brings hope for a first potential treatment for SMA patients," said Christian Cottet, CEO, AFM-Telethon.

"Trophos and the AFM have been working together for over a decade. This crucial clinical study is the latest step in our long standing partnership," said Christine Placet, CEO, Trophos. "Olesoxime has a promising profile as a potential treatment for SMA. We are very hopeful that the results of this study will bring a much needed treatment option and new hope to SMA patients and their families. Trophos has a history of commitment to developing breakthrough therapies for rare and serious neurodegenerative diseases."

Trial design and end-points

The study is a 24-month randomized, parallel group, double-blind, placebo controlled trial comparing olesoxime against placebo in non-ambulant type II and type III SMA patients aged from 3 to 25 years old. Olesoxime is administered at the dose of 10 mg/kg/day using a specially developed liquid formulation; patients were randomized to receive olesoxime in a 2:1 ratio versus placebo.

The primary end-point of the study is the change from baseline in the Motor Function Measure (MFM) functional scale. Secondary endpoints include the Hammersmith Functional Motor Scale and electromyography (CMAP -Compound Muscle Action Potential - and MUNE - Motor Unit Number) as well as measure of safety, tolerance and quality of life. Trophos is also exploring changes in a panel of possible SMA biomarkers in collaboration with the Spinal Muscular Atrophy Foundation (http://www.smafoundation.org). The study is sponsored by Trophos and the principle investigator is Dr. Enrico Bertini, a key opinion leader with extensive experience in the natural history as well as the design and performance of clinical trials in SMA. The study is being conducted in 22 centers in France, Italy, Germany, UK, Belgium, the Netherlands and Poland by a consortium of prominent European clinical investigators, all of whom have extensive experience conducting and collaborating in SMA clinical trials.

[채호맘]

좋은 소식이네요. 올해안에 더 좋은 소식있길 바래요

[서연맘]

진짜 좋은 소식이예요.. 모든 근육병에 치료제가 나오길..

[부모회장후니맘]

기대하고 있습니다 일본근육병 협회에서도 올 가을즈음에 확실한 발표가 있을 예정이라고 하더군요 우리나라도 올해말쯤에 신약에 대한 발표가 있으리라 예상하고 있습니다 조금만 더 인내를 갖고 기다리면 머지않아 희망의 소식이 있으리라 생각됩니다

[한정훈]

좋은 정보 감사합니다

[윤아사랑해(태윤아빠)]

정확한 내용을 이해할순없지만 희망적 내용임에는 틀림없군요
긍정의 힘을 믿습니다 좋은글 올려주셔서 감사해요

[비담공]

하루라도 빨리 신약이 만들어졌으면 좋겠네요.. 부디....

[이명주]

다음세대에서라도 아픔이 없길! 약은 꼭 나올것입니다!

[김동호]

스크랩 합니다~

[환장머슴]

몇 달 사이 다리힘이 눈에 띄게 빠지고 있어 정말 걱정이었는데..신약 개발 소식에 기운이 납니다..

[울아들사랑해]

우리겸주 오늘 유치원 입학했답니다. 겸주도 다른 친구들처럼 여기저기 뛰어다니면서 말썽도 피우고^^ 개구쟁이 꼬맹이가 될 수 있겠죠? 제발 꼭 성공해주세요!

[욱하는웅]

좋은소식 이네요~

[민병준]

마음으로 기도합니다

[김명식]

간절한 마음으로 기다립니다..

[나르새]

좋은 소식 전해주셔서 감사합니다. 올해안에 치료제가 꼭 나왔으면 좋겠습니다

[섭맘]

빨리 좋은 소식 있었으면 좋겠어요. 울 지섭이 정말 잃고 싶지 않아요. 입에 붙었어요,,,우리 지섭이,,,,이 이름,,,평생 부르고 싶어요.