Trophos, a biopharmaceutical company specializing in the discovery and development of novel therapeutics to treat orphan neurodegenerative diseases, has completed patient enrolment in the pivotal efficacy study of olesoxime in patients with spinal muscular atrophy (SMA). Over 160 patients have been recruited into the trial since its initiation in October 2010. Efficacy results are expected in the second half of 2013.
Olesoxime had previously successfully completed a phase 1b study in SMA patients and is undergoing a pivotal efficacy study in Amyotrophic Lateral Sclerosis (ALS) patients. Preclinical studies have demonstrated that the compound promotes the function and survival of neurons and other cell types under disease-relevant stress conditions through interactions with the mitochondrial permeability transition pore (mPTP).
Olesoxime had previously successfully completed a phase 1b study in SMA patients and is undergoing a pivotal efficacy study in Amyotrophic Lateral Sclerosis (ALS) patients. Preclinical studies have demonstrated that the compound promotes the function and survival of neurons and other cell types under disease-relevant stress conditions through interactions with the mitochondrial permeability transition pore (mPTP).
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ReplyDeletePreliminary results of the study have just been published and are positive: http://www.pharmalive.com/news/index.cfm?articleid=880729.
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