Corporate Banner
Satellite Banner
Pharma Outsourcing
Scientific Community
 
Become a Member | Sign in
Home>News>This Article
  News
Return

Trophos Announces Positive Interim Review in Pivotal Study of Olesoxime in SMA

Published: Monday, March 04, 2013
Last Updated: Sunday, March 03, 2013
Bookmark and Share
Topline results expected in the fourth quarter of 2013.

Trophos SA has 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.

Pruss continued, "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 analyzing results on the efficacy outcome of the trial after it reaches the planned conclusion," said principle investigator Dr Enrico Bertini.

Dr Bertini continued, "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."


Further Information
Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 2,500+ scientific posters on ePosters
  • More than 3,700+ scientific videos on LabTube
  • 35 community eNewsletters


Sign In



Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into TechnologyNetworks.com you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.

Related Content

Trophos Announces Top-Line Results in Pivotal Trial of Olesoxime in SMA
Promising results for the maintenance of motor function in spinal muscular atrophy - a rare, serious and debilitating pediatric-onset neurodegenerative disease.
Thursday, March 20, 2014
Trophos Completes Pivotal Phase II/III Study of Olesoxime in SMA
The French patients’ association AFM-Telethon supported the study which evaluates the ability of olesoxime to preserve the neuromuscular function of SMA patients.
Friday, November 08, 2013
Trophos Announces Final Patient Completion of Phase II Study of TRO40303
The study, a critical part of the EU funded MitoCare Project, will evaluate the reduction of reperfusion injury by TRO40303 in acute ST-elevation myocardial infarction (STEMI) patients.
Thursday, October 03, 2013
Trophos Launches First Study of Olesoxime in Multiple Sclerosis Patients
A Phase 1b study in RRMS patients is starting in three MS reference centers in France.
Tuesday, March 19, 2013
Trophos Appoints JSB Partners as Global Partnership Advisor
Life science advisor to advise Trophos on strategic partnerships for orphan disease and niche indication candidates.
Monday, December 31, 2012
Trophos Program Boosted by EUR 1 Million Grant for Olesoxime in Progressive MS
Agence Nationale de la Recherche awards second grant to Trophos led consortium for Phase Ib/IIa clinical study.
Friday, October 12, 2012
Marceline Clementine Appointed Chief Financial Officer
Ms. Clementine is now responsible for all administrative and financial services at Trophos.
Wednesday, September 19, 2012
Trophos Announces Conclusion of MitoTarget Consortium
EU funded 3-year project spearheaded by orphan disease specialist Trophos reports results from neurodegenerative program to the European Commission.
Wednesday, August 01, 2012
Trophos Appoints Christine Placet as Chief Executive Officer
Christine becomes CEO under a succession plan designed to provide continuity for Trophos.
Thursday, June 14, 2012
Trophos Announces Results of Phase III Study of Olesoxime in ALS
Company continues its ongoing programs and financed until at least the end of 2013.
Wednesday, December 14, 2011
Trophos Initiates Phase II Study for Novel Cardioprotective Compound, TRO40303, in Acute MI
European study being performed as part of Trophos-led, EU funded MitoCare project.
Tuesday, November 01, 2011
Trophos Completes Patient Enrolment in Pivotal Efficacy Study of Olesoxime in SMA
Topline results expected in second half of 2013.
Friday, September 09, 2011
Trophos Successfully Completes Phase 1 Study for Novel Cardioprotective Compound, TRO40303
Phase 2 proof-of-concept study in acute myocardial infarction planned for second half 2011 with MitoCare EU consortium.
Friday, February 25, 2011
Trophos to Lead new MitoCare Consortium Awarded EUR 6 Million to Study Cardioprotective Compound, TRO40303
Program of translational research to study innovative approaches to reducing cardiac ischemia-reperfusion injury.
Wednesday, December 15, 2010
Trophos Initiates Pivotal Efficacy Study of Olesoxime in Spinal Muscular Atrophy
European study supported by partnership with the Association Française contre les Myopathies.
Monday, October 18, 2010
Scientific News
NIH Study Shows No Benefit of Omega-3 Supplements for Cognitive Decline
Research was published in the Journal of the American Medical Association.
NIH Launches Human RSV Study
Study aims to understand infection in healthy adults to aid development of RSV medicines, vaccines.
Computerized Flexible Needles Prove Themselves in Biological Tissue
The advantage of the system is that you can avoid obstacles with the needles or critical tissues and that the system during the insertion of the needle in real time can adjust the path if, for example, the tissue deforms.
DARWIN 2 24-week Monotherapy Data in RA Confirm Previous Results
Safety profile in DARWIN 2 consistent with previous filgotinib RA studies.
Researchers Publish Landmark “Basket Study”
Researchers from Memorial Sloan Kettering Cancer Center (MSK) have announced results from the first published basket study, a new form of clinical trial design that explores responses to drugs based on the specific mutations in patients’ tumors rather than where their cancer originated.
Agricultural Intervention Improves HIV Outcomes
A multifaceted farming intervention can reduce food insecurity while improving HIV outcomes in patients in Kenya, according to a randomized, controlled trial led by researchers at UC San Francisco.
Overdose of Vitamin D in Teenagers May Lead to Increased Cholesterol Levels
Dosing obese teens with vitamin D shows no benefits for their heart health or diabetes risk, and could have the unintended consequences of increasing cholesterol and fat-storing triglycerides. These are the latest findings in a series of Mayo Clinic studies in childhood obesity.
Phase 2 Trials Underway for New Single Dose Malaria Treatment
The new drug, which prevents the malaria parasite from reproducing and spreading, is now undergoing Phase II clinical trials in humans.
Promising Drug for Parkinson's Disease
A drug which has already been in use for decades to treat liver disease could be an effective treatment to slow down progression of Parkinson’s disease, scientists from the University of Sheffield have discovered.
Benefits of Early Antiretroviral Therapy in HIV Infection
Scientists have explored the clinical importance of starting treatment early in individuals suffering with HIV.
Scroll Up
Scroll Down
SELECTBIO

Skyscraper Banner
Go to LabTube
Go to eposters
 
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
2,500+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
3,700+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FREE!