Trophos Successfully Completes Phase Ib ALS Clinical Trials for TRO19622, a Novel Compound to Treat Neurodegenerative Disease
One-month study shows good tolerance and exposure levels to drug, together with an excellent safety profile with once a day oral dosing, and paves the way for a Phase II/III pivotal trial in ALS
MARSEILLE, France, Dec. 7, 2006 - Trophos, a biopharmaceutical company specializing in the discovery and development of drugs for neurodegenerative diseases, announced today the successful completion of Phase Ib clinical studies for its lead compound TRO19622. The completed studies include a one month compliance, tolerance and safety study conducted in 36 ALS patients at doses of 125 mg, 250 mg and 500 mg administered orally, once a day, plus two drug-drug interaction studies conducted in healthy volunteers. TRO19622 is in clinical development for motoneuron diseases, amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) and spinal muscular atrophy (SMA), as well as for painful diabetic neuropathy.
The studies demonstrated that, in ALS patients, TRO19622 is well tolerated and exceeded the exposure level predicted to achieve efficacy via the oral route at doses superior or equal to 250mg per day. In healthy volunteers, TRO19622 exposure levels were approximately 30% higher after repeated dosing with riluzole for which bioequivalence was shown before and after repeated dosing with TRO19622. In these three Phase Ib studies, TRO19622 continues to demonstrate an excellent clinical safety profile in all subjects receiving drug substance.
The successful completion of these Phase Ib studies will enable Trophos, subject to regulatory approvals, to initiate a pivotal Phase II/III clinical trial to evaluate TRO19622 efficacy (survival and functional benefits) in the ALS indication, to be conducted at approximately 10 centers in Europe. Trophos is currently seeking a co-development/licensing partner for this program in the US. In addition, Trophos now expects to initiate a European Phase Ib clinical study to assess the tolerance and safety of TRO19622 in juvenile SMA patients in the first half 2007.
TRO19622 is representative of a class of novel compounds identified using the proprietary neuronal cell-screening platform developed at Trophos. In preclinical studies these 'first-in-class' compounds have been demonstrated to promote the survival of a wide range of neurons in vitro, as well as in several in vivo models of neurodegenerative diseases.
"We are excited that TRO19622, the lead molecule discovered using our innovative phenotypic screening platform has successfully completed these Phase Ib trials and continues to demonstrate an excellent clinical safety profile," said Dr Jean-Louis Abitbol, Chief Medical Officer at Trophos. "Completion of these studies will allow Trophos to proceed to assess the clinical efficacy of TRO19622 in ALS patients, in terms of both survival and functional benefits. In addition, the excellent safety profile of TRO19622 paves the way for further clinical studies to assess the efficacy of TRO19622 in juvenile SMA patients."
About ALS (Amyotrophic Lateral Sclerosis): ALS, more commonly known as Lou Gehrig's disease in the USA, is a progressive and fatal neurological disease that is estimated to affect over 100,000 people worldwide. There is no cure for ALS. The only drug approved for ALS is riluzole (Sanofi-Aventis), which has been demonstrated to give some survival benefit to ALS patients. For more information about ALS, see www.alsa.org
About SMA (Spinal Muscular Atrophy): SMA is a genetic disorder that affects muscle movement in approximately 1 in 6000 live births. It causes the motor neurons in the anterior horn of the spinal cord to deteriorate thereby gradually weakening the voluntary muscles used for activities such as crawling, walking, sitting and moving the head. SMA is classified into four types, based on the age at which it develops and the severity of the symptoms. Types I, II and III develop in childhood while Type IV starts in adulthood. There is no cure for SMA. For more information about SMA, see www.fsma.org
About Trophos: www.trophos.com
Trophos is a biopharmaceutical company developing drugs to treat neurodegenerative diseases. These are severely debilitating, often fatal, disorders with few effective drugs and tremendous unmet medical needs. In preclinical studies, compounds with similar effects as TRO19622 have been shown to prevent the neuronal degeneration that is involved in the onset or progression of motor neuron disease. The motoneuron disease program at Trophos is supported, in part, by the Association Francaise contre les Myopathies (AFM, for more information see www.afm-france.org)
. Trophos was founded in 1999, is based in Marseille, France and currently has 32 employees.
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