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 Cryo-electron microscopy achieves unprecedented resolution using new computational methods
24.03.2017 | DOE/Lawrence Berkeley National Laboratory

nachricht How cheetahs stay fit and healthy
24.03.2017 | Forschungsverbund Berlin e.V.

All articles from Life Sciences >>>

The most recent press releases about innovation >>>

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

Im Focus: Giant Magnetic Fields in the Universe

Astronomers from Bonn and Tautenburg in Thuringia (Germany) used the 100-m radio telescope at Effelsberg to observe several galaxy clusters. At the edges of these large accumulations of dark matter, stellar systems (galaxies), hot gas, and charged particles, they found magnetic fields that are exceptionally ordered over distances of many million light years. This makes them the most extended magnetic fields in the universe known so far.

The results will be published on March 22 in the journal „Astronomy & Astrophysics“.

Galaxy clusters are the largest gravitationally bound structures in the universe. With a typical extent of about 10 million light years, i.e. 100 times the...

Im Focus: Tracing down linear ubiquitination

Researchers at the Goethe University Frankfurt, together with partners from the University of Tübingen in Germany and Queen Mary University as well as Francis Crick Institute from London (UK) have developed a novel technology to decipher the secret ubiquitin code.

Ubiquitin is a small protein that can be linked to other cellular proteins, thereby controlling and modulating their functions. The attachment occurs in many...

Im Focus: Perovskite edges can be tuned for optoelectronic performance

Layered 2D material improves efficiency for solar cells and LEDs

In the eternal search for next generation high-efficiency solar cells and LEDs, scientists at Los Alamos National Laboratory and their partners are creating...

Im Focus: Polymer-coated silicon nanosheets as alternative to graphene: A perfect team for nanoelectronics

Silicon nanosheets are thin, two-dimensional layers with exceptional optoelectronic properties very similar to those of graphene. Albeit, the nanosheets are less stable. Now researchers at the Technical University of Munich (TUM) have, for the first time ever, produced a composite material combining silicon nanosheets and a polymer that is both UV-resistant and easy to process. This brings the scientists a significant step closer to industrial applications like flexible displays and photosensors.

Silicon nanosheets are thin, two-dimensional layers with exceptional optoelectronic properties very similar to those of graphene. Albeit, the nanosheets are...

Im Focus: Researchers Imitate Molecular Crowding in Cells

Enzymes behave differently in a test tube compared with the molecular scrum of a living cell. Chemists from the University of Basel have now been able to simulate these confined natural conditions in artificial vesicles for the first time. As reported in the academic journal Small, the results are offering better insight into the development of nanoreactors and artificial organelles.

Enzymes behave differently in a test tube compared with the molecular scrum of a living cell. Chemists from the University of Basel have now been able to...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

International Land Use Symposium ILUS 2017: Call for Abstracts and Registration open

20.03.2017 | Event News

CONNECT 2017: International congress on connective tissue

14.03.2017 | Event News

ICTM Conference: Turbine Construction between Big Data and Additive Manufacturing

07.03.2017 | Event News

 
Latest News

Gravitational wave kicks monster black hole out of galactic core

24.03.2017 | Physics and Astronomy

Researchers make flexible glass for tiny medical devices

24.03.2017 | Materials Sciences

Laser activated gold pyramids could deliver drugs, DNA into cells without harm

24.03.2017 | Health and Medicine

VideoLinks
B2B-VideoLinks
More VideoLinks >>>