Olesoxime
Encyclopedia
Olesoxime is the lead compound of Trophos
' proprietary cholesterol-oxime compound family of mitochondrial pore modulators.
It is a molecule that has a cholesterol-like structure and displays strong neuroprotective properties. TRO19622 is as effective as a cocktail of three neurotrophic factors in keeping motor neurons alive in culture.
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 has successfully completed phase I studies in healthy volunteers and phase Ib studies in Amyotrophic Lateral Sclerosis
and Spinal Muscular Atrophy
patients. These clinical trials showed that the product is well tolerated and has an excellent safety profile. They also showed that once-a-day oral dosing achieves the predicted exposure levels, required for efficacy, based on preclinical models.
Drug interaction studies with riluzole, the only registered treatment for ALS, showed no interaction of TRO19622 on riluzole pharmacokinetics.
Olesoxime (TRO19622) is currently being developed for the treatment of two motor neuron diseases: Amyotrophic Lateral Sclerosis
(ALS) and Spinal Muscular Atrophy
(SMA).
Trophos
Trophos is a biopharmaceutical company specialising in the discovery and development of novel therapeutics to treat both orphan neurodegenerative diseases and more prevalent disorders....
' proprietary cholesterol-oxime compound family of mitochondrial pore modulators.
It is a molecule that has a cholesterol-like structure and displays strong neuroprotective properties. TRO19622 is as effective as a cocktail of three neurotrophic factors in keeping motor neurons alive in culture.
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 has successfully completed phase I studies in healthy volunteers and phase Ib studies in Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis
Amyotrophic lateral sclerosis , also referred to as Lou Gehrig's disease, is a form of motor neuron disease caused by the degeneration of upper and lower neurons, located in the ventral horn of the spinal cord and the cortical neurons that provide their efferent input...
and Spinal Muscular Atrophy
Spinal muscular atrophy
Spinal Muscular Atrophy is a neuromuscular disease characterized by degeneration of motor neurons, resulting in progressive muscular atrophy and weakness. The clinical spectrum of SMA ranges from early infant death to normal adult life with only mild weakness...
patients. These clinical trials showed that the product is well tolerated and has an excellent safety profile. They also showed that once-a-day oral dosing achieves the predicted exposure levels, required for efficacy, based on preclinical models.
Drug interaction studies with riluzole, the only registered treatment for ALS, showed no interaction of TRO19622 on riluzole pharmacokinetics.
Olesoxime (TRO19622) is currently being developed for the treatment of two motor neuron diseases: Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis
Amyotrophic lateral sclerosis , also referred to as Lou Gehrig's disease, is a form of motor neuron disease caused by the degeneration of upper and lower neurons, located in the ventral horn of the spinal cord and the cortical neurons that provide their efferent input...
(ALS) and Spinal Muscular Atrophy
Spinal muscular atrophy
Spinal Muscular Atrophy is a neuromuscular disease characterized by degeneration of motor neurons, resulting in progressive muscular atrophy and weakness. The clinical spectrum of SMA ranges from early infant death to normal adult life with only mild weakness...
(SMA).