ALLSCHWIL, Switzerland and MARSEILLE, France—Actelion Ltd. and privately-held Trophos SA announced this week that they have entered into a binding agreement whereby Actelion has, for EUR 10 million, obtained an exclusive option to acquire privately-held Trophos SA, a clinical-stage pharmaceutical company.
Trophos' lead compound olesoxime has completed enrollment into a Phase III study in amyotrophic lateral sclerosis (ALS), the orphan disease that is more colloquially known as Lou Gehrig's disease. This study is expected to report data by the end of 2011; at this time Actelion may exercise the option for an acquisition price between EUR 125 and EUR 195 million in cash, contingent on different regulatory approvals and other clinical progress of Trophos' pipeline.
"Trophos has done an excellent job to enroll more than 500 ALS patients into a well-designed pivotal study. Once study results are available, Actelion is ideally positioned to leverage these achievements with our proven global regulatory and marketing expertise in the area of orphan drugs," says Simon Buckingham, president of global corporate and business development for Actelion.
Trophos is a clinical-stage company with a pipeline of new molecular entities in development for the motor neuron diseases ALS and spinal muscular atrophy (SMA) as well as a novel compound for cardiac ischemia-reperfusion injury.
"Since its inception, Trophos has made significant progress in turning its key expertise in neurodegenerative disorders and orphan diseases into achievements that include advancing our lead compound olesoxime into late stage clinical development," says Damian Marron, CEO of Trophos. "The development of olesoxime has benefited from significant support from patient communities, clinical investigators and the European Union (EU), including Trophos spearheading an EU-funded consortium dedicated to improving the treatment of ALS."
The two companies also agreed on a research collaboration to allow Actelion access to Trophos' proprietary CNS assay technology and compound library. The technology mimics neuronal degeneration processes in the test tube and is used to screen chemical compounds for their ability to block these processes.
"Trophos has a pioneering approach and proprietary expertise that has enabled the development of high throughput screens using primary neurons as well as the ability to broadly profile more advanced compounds." notes Dr. Martine Clozel, chief scientific officer at Actelion. "This is of great value to Actelion as we have developed a large in-house compound library and significant expertise in the field of neurological disorders."
Olesoxime is Trophos' lead compound of a proprietary mitochondrial pore modulator series, and preclinical studies have indicated that olesoxime may promote 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 been shown to be active in the SOD1 model of ALS.
Phase I studies in healthy volunteers and Phase Ib studies in ALS patients demonstrated that olesoxime is well-tolerated, according to Actelion and Trophos. These studies also helped to determine the dose regimen used in the pivotal Phase III study.
Trophos' lead compound olesoxime has completed enrollment into a Phase III study in amyotrophic lateral sclerosis (ALS), the orphan disease that is more colloquially known as Lou Gehrig's disease. This study is expected to report data by the end of 2011; at this time Actelion may exercise the option for an acquisition price between EUR 125 and EUR 195 million in cash, contingent on different regulatory approvals and other clinical progress of Trophos' pipeline.
"Trophos has done an excellent job to enroll more than 500 ALS patients into a well-designed pivotal study. Once study results are available, Actelion is ideally positioned to leverage these achievements with our proven global regulatory and marketing expertise in the area of orphan drugs," says Simon Buckingham, president of global corporate and business development for Actelion.
Trophos is a clinical-stage company with a pipeline of new molecular entities in development for the motor neuron diseases ALS and spinal muscular atrophy (SMA) as well as a novel compound for cardiac ischemia-reperfusion injury.
"Since its inception, Trophos has made significant progress in turning its key expertise in neurodegenerative disorders and orphan diseases into achievements that include advancing our lead compound olesoxime into late stage clinical development," says Damian Marron, CEO of Trophos. "The development of olesoxime has benefited from significant support from patient communities, clinical investigators and the European Union (EU), including Trophos spearheading an EU-funded consortium dedicated to improving the treatment of ALS."
The two companies also agreed on a research collaboration to allow Actelion access to Trophos' proprietary CNS assay technology and compound library. The technology mimics neuronal degeneration processes in the test tube and is used to screen chemical compounds for their ability to block these processes.
"Trophos has a pioneering approach and proprietary expertise that has enabled the development of high throughput screens using primary neurons as well as the ability to broadly profile more advanced compounds." notes Dr. Martine Clozel, chief scientific officer at Actelion. "This is of great value to Actelion as we have developed a large in-house compound library and significant expertise in the field of neurological disorders."
Olesoxime is Trophos' lead compound of a proprietary mitochondrial pore modulator series, and preclinical studies have indicated that olesoxime may promote 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 been shown to be active in the SOD1 model of ALS.
Phase I studies in healthy volunteers and Phase Ib studies in ALS patients demonstrated that olesoxime is well-tolerated, according to Actelion and Trophos. These studies also helped to determine the dose regimen used in the pivotal Phase III study.
