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 Learning from Nature: Genomic database standard alleviates search for novel antibiotics
02.09.2015 | Max-Planck-Institut für marine Mikrobiologie

nachricht Orang-utan females prefer cheek-padded males
02.09.2015 | Max Planck Institute for Evolutionary Anthropology, Leipzig

All articles from Life Sciences >>>

The most recent press releases about innovation >>>

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

Im Focus: How wind sculpted Earth's largest dust deposit

China's Loess Plateau was formed by wind alternately depositing dust or removing dust over the last 2.6 million years, according to a new report from University of Arizona geoscientists. The study is the first to explain how the steep-fronted plateau formed.

China's Loess Plateau was formed by wind alternately depositing dust or removing dust over the last 2.6 million years, according to a new report from...

Im Focus: An engineered surface unsticks sticky water droplets

The leaves of the lotus flower, and other natural surfaces that repel water and dirt, have been the model for many types of engineered liquid-repelling surfaces. As slippery as these surfaces are, however, tiny water droplets still stick to them. Now, Penn State researchers have developed nano/micro-textured, highly slippery surfaces able to outperform these naturally inspired coatings, particularly when the water is a vapor or tiny droplets.

Enhancing the mobility of liquid droplets on rough surfaces could improve condensation heat transfer for power-plant heat exchangers, create more efficient...

Im Focus: Increasingly severe disturbances weaken world's temperate forests

Longer, more severe, and hotter droughts and a myriad of other threats, including diseases and more extensive and severe wildfires, are threatening to transform some of the world's temperate forests, a new study published in Science has found. Without informed management, some forests could convert to shrublands or grasslands within the coming decades.

"While we have been trying to manage for resilience of 20th century conditions, we realize now that we must prepare for transformations and attempt to ease...

Im Focus: OU astrophysicist and collaborators find supermassive black holes in quasar nearest Earth

A University of Oklahoma astrophysicist and his Chinese collaborator have found two supermassive black holes in Markarian 231, the nearest quasar to Earth, using observations from NASA's Hubble Space Telescope.

The discovery of two supermassive black holes--one larger one and a second, smaller one--are evidence of a binary black hole and suggests that supermassive...

Im Focus: What would a tsunami in the Mediterranean look like?

A team of European researchers have developed a model to simulate the impact of tsunamis generated by earthquakes and applied it to the Eastern Mediterranean. The results show how tsunami waves could hit and inundate coastal areas in southern Italy and Greece. The study is published today (27 August) in Ocean Science, an open access journal of the European Geosciences Union (EGU).

Though not as frequent as in the Pacific and Indian oceans, tsunamis also occur in the Mediterranean, mainly due to earthquakes generated when the African...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

Networking conference in Heidelberg for outstanding mathematicians and computer scientists

20.08.2015 | Event News

Scientists meet in Münster for the world’s largest Chitin und Chitosan Conference

20.08.2015 | Event News

Large agribusiness management strategies

19.08.2015 | Event News

 
Latest News

Tiny Drops of Early Universe 'Perfect' Fluid

02.09.2015 | Physics and Astronomy

Learning from Nature: Genomic database standard alleviates search for novel antibiotics

02.09.2015 | Life Sciences

International research project gets high level of funding

02.09.2015 | Awards Funding

VideoLinks
B2B-VideoLinks
More VideoLinks >>>