Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Trophos announces top-line results in pivotal trial of olesoxime in spinal muscular atrophy

11.03.2014

Promising results for the maintenance of motor function in spinal muscular atrophy (SMA) - a rare, serious and debilitating pediatric-onset neurodegenerative disease

Trophos today announces that top-line results from a pivotal clinical trial of its lead product candidate olesoxime in spinal muscular atrophy (SMA) show a beneficial effect on the maintenance of motor function in SMA patients. If approved, olesoxime could be the first treatment specifically developed for SMA patients.

SMA is an autosomal recessive genetic disease that affects the motor neurons of the voluntary muscles used for activities such as crawling, walking, head and neck control and swallowing. SMA affects approximately 20,000 people worldwide. One in every 6,000 babies is born with SMA. It is the number one genetic cause of death in children under the age of two.

The mutated gene responsible for SMA is carried by up to 20 million potential parents in the US and EU. Most of them are unaware that they are carriers. SMA patients are divided into four subtypes depending on disease onset and severity, but they all suffer from the degeneration of motor neurons controlling voluntary muscles, with proximal limb and trunk muscle weakness leading to respiratory distress and in the most severe cases, death.

... more about:
»SMA »atrophy »death »mitochondrial »muscles »muscular »neurons »spinal

The pivotal study was conducted in seven European countries. It was a double-blind, placebo-controlled study in 165 type II and non-ambulatory type III SMA patients, ranging in age from 3 to 25 years old. Patients were randomized to treatment (10 mg/kg olesoxime dosed daily as a liquid oral suspension or matching placebo in a 2:1 ratio).

They were evaluated every three months for two years. The primary outcome measure was the change in motor function at two years using a standardized neuromuscular disease-specific functional scale, the MFM. The secondary outcome measures included an additional scale, the Hammersmith Functional Motor Scale for Spinal Muscular Atrophy, as well as electromyography measures, pulmonary function, patient-reported outcomes such as clinical global impression (CGI), quality of life measures (PedsQL), typical SMA complications and product safety.

Results from this pivotal study showed a progressive loss of motor function in the placebo arm, as described for the typical disease progression and similarly documented in other observational and interventional trials. This loss of function, assessed as the primary endpoint, was prevented for two years in olesoxime-treated patients, with fewer disease-related adverse events. Other secondary endpoints were consistent with these effects. Detailed results will be published and presented at upcoming conferences in Europe and the USA. The discovery of olesoxime and its development for SMA was mainly supported by AFM-Telethon, the French muscular dystrophy association.

“Spinal muscular atrophy is a devastating condition and a leading cause of death in babies and infants under two years old. Children with a less severe form of SMA suffer progressive muscle wasting and loss of mobility and motor function,” said Dr. Enrico Bertini, the principle investigator of the study. “Olesoxime has the potential to be the first ever approved treatment specifically developed for SMA patients. Its neuroprotective effect combined with fewer adverse events caused by the disease itself is encouraging. The results and data collected in this study on outcome measures and biomarkers will establish standards for future clinical trials in SMA patients.”

“We are grateful for the long-term financial support and commitment of AFM-Telethon and our other shareholders, to the patients, to their families and clinicians who have enabled us to complete this pivotal trial with olesoxime,” said Christine Placet, chief executive officer of Trophos. “Our focus now is on the regulatory steps needed to bring this important product to patients as quickly as possible. We are currently exploring a number of options, including potential industry partnerships and identifying new sources of funding.”
 
More information about spinal muscular atrophy
http://www.afm-telethon.fr
http://www.curesma.org
http://www.smafoundation.org/about-sma/
 
About olesoxime
Olesoxime (TRO19622) is the lead compound in Trophos' proprietary cholesterol-oxime family of compounds that target and preserve mitochondrial integrity and function in stressed cells. Preclinical studies have demonstrated that olesoxime promotes the function and survival of neurons and other cell types under disease-relevant stress conditions. It has been shown to be active in multiple preclinical neurodegeneration models including the NSE-Cre F7/F7 model of SMA.
Trophos has been granted 'Orphan Medicinal Product' designation for olesoxime for the treatment of SMA by the European Commission and orphan drug designation by the US Food and Drug Administration.

About Trophos
Trophos is a clinical stage pharmaceutical company developing innovative therapeutics for indications with under-served needs in neurology and cardiology. The company has a novel and proprietary cholesterol-oxime based chemistry platform generating a pipeline of drug candidates. The lead product, olesoxime (TRO19622), is being developed for SMA and multiple sclerosis. Other indications are in the selection process. Trophos' mitochondrial targeted compounds enhance the function and survival of stressed cells by preventing mitochondrial permeability transition, a key determinant of cell death or survival. There is growing support for the therapeutic rationale for such mitochondria targeted drugs, which Trophos is uniquely placed to exploit.
Trophos was founded in 1999 and is based in Marseille, France. It is supported by a syndicate of private equity funds among which Viveris Management, OTC Asset Management, Amundi PEF, Turenne Capital and Vesale Partners.
http://www.trophos.com

To view the original press release: http://www.ala.com/article.php?id=354

Alexandra Virey | ANDREW LLOYD & ASSOCIATES

Further reports about: SMA atrophy death mitochondrial muscles muscular neurons spinal

More articles from Life Sciences:

nachricht Molecular Spies Sabotage a Protein's Activities in Specific Cellular Compartments
20.04.2015 | Johns Hopkins Medicine

nachricht Evolution puts checks on virgin births
20.04.2015 | Okinawa Institute of Science and Technology (OIST) Graduate University

All articles from Life Sciences >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Advances in Molecular Electronics: Lights On – Molecule On

Scientists at the Helmholtz-Zentrum Dresden-Rossendorf (HZDR) and the University of Konstanz are working on storing and processing information on the level of single molecules to create the smallest possible components that will combine autonomously to form a circuit. As recently reported in the academic journal Advanced Science, the researchers can switch on the current flow through a single molecule for the first time with the help of light.

Dr. Artur Erbe, physicist at the HZDR, is convinced that in the future molecular electronics will open the door for novel and increasingly smaller – while also...

Im Focus: Pruning of Blood Vessels: Cells Can Fuse With Themselves

Cells of the vascular system of vertebrates can fuse with themselves. This process, which occurs when a blood vessel is no longer necessary and pruned, has now been described on the cellular level by Prof. Markus Affolter from the Biozentrum of the University of Basel. The findings of this study have been published in the journal “PLoS Biology”.

The vascular system is the supply network of the human organism and delivers oxygen and nutrients to the last corners of the body. So far, research on the...

Im Focus: Astronomers reveal supermassive black hole's intense magnetic field

Astronomers from Chalmers University of Technology have used the giant telescope Alma to reveal an extremely powerful magnetic field very close to a supermassive black hole in a distant galaxy

Astronomers from Chalmers University of Technology have used the giant telescope Alma to reveal an extremely powerful magnetic field very close to a...

Im Focus: A “pin ball machine” for atoms and photons

A team of physicists from MPQ, Caltech, and ICFO proposes the combination of nano-photonics with ultracold atoms for simulating quantum many-body systems and creating new states of matter.

Ultracold atoms in the so-called optical lattices, that are generated by crosswise superposition of laser beams, have been proven to be one of the most...

Im Focus: UV light robot to clean hospital rooms could help stop spread of 'superbugs'

Can a robot clean a hospital room just as well as a person?

According to new research out of the Texas A&M Health Science Center College of Medicine, that is indeed the case. Chetan Jinadatha, M.D., M.P.H., assistant...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

HHL's Entrepreneurship Conference on FinTech

13.04.2015 | Event News

World Conference On Regenerative Medicine 2015: Registration And Abstract Submission Now Open

25.03.2015 | Event News

University presidents from all over the world meet in Hamburg

19.03.2015 | Event News

 
Latest News

High-Power Laser Spinoff Proves Versatility Is Strength

20.04.2015 | Physics and Astronomy

New “Cool Roof Time Machine” Will Accelerate Cool Roof Deployment

20.04.2015 | Architecture and Construction

STAR Heavy Flavor Tracker Detects Signs of Charm at RHIC

20.04.2015 | Physics and Astronomy

VideoLinks
B2B-VideoLinks
More VideoLinks >>>