Marseille, France, March 19th, 2009 - Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics for indications with under-served needs in neurology and cardiology, announced today that it will receive EUR 6.7 million over the next three years from the Association Francaise contre les Myopathies (AFM), which will substantially cover the cost of the further clinical development of its lead drug candidate olesoxime (TRO19622) as a treatment for Spinal Muscular Atrophy (SMA). This funding will support an important clinical trial seeking to demonstrate the efficacy of olesoxime in SMA patients.
Trophos is currently undertaking a Protocol Assistance procedure with the EMEA in order to finalize the design of the clinical efficacy study and to determine the regulatory requirements for approval of olesoxime for the treatment of SMA. It is currently anticipated the trial will commence around the end of this year, subject to the outcome of the discussions with the EMEA.
“Trophos is very pleased to be renewing its long standing strategic partnership with the AFM for the continued development of olesoxime for SMA, which has been instrumental in advancing our SMA program” said Damian Marron, CEO of Trophos, “Olesoxime is uniquely suited for development for this devastating condition, for which no treatments exist today. This program is a key part of our ongoing commitment to rare and under served motor neuron diseases and this agreement underlines and strengthens our joint efforts to further the clinical development of olesoxime for SMA.”
Trophos further disclosed today that olesoxime has recently been granted Orphan Drug Designation (ODD) for the treatment of SMA by the US FDA, following the earlier, similar Orphan Medicinal Product designation by the EMEA. This is recognition of the potential of olesoxime for the treatment of SMA and brings a number of development, regulatory and marketing exclusivity benefits. Trophos has previously received orphan drug status for olesoxime for the treatment of Amyotrophic Lateral Sclerosis in the US and EU.
Trophos also announced that an oral presentation on its successful phase Ib safety and PK study in SMA patients will be made at the American Academy of Neurology meeting being held in Seattle in April. The title of the talk is “Safety and Pharmacokinetics (PK) of TRO19622 in Spinal Muscular Atrophy (SMA) Children and Adults” and it will be presented in Scientific Sessions: Anterior Horn: SMA and Kennedy’s Disease (3:45 PM-5:00 PM) on Wednesday April 29th.
Olesoxime is the lead drug candidate from Trophos’ novel and proprietary cholesterol-oxime based pipeline of drug candidates that enhance the function and survival of stressed cells via modulation of dysfunctional mitochondria, through interactions at the permeability transition pore (mPTP). Preclinical studies have demonstrated that these compounds promote the function and survival of neurons and other cell types under disease relevant stress conditions (Bordet et al., JPET 322:709-720, 2007). Olesoxime has successfully completed a phase 1b study in SMA patients (see above), having previously completed phase I/Ib studies in healthy volunteers and ALS patients. These clinical trials demonstrated that the product is well tolerated, has an excellent safety profile and that once-a-day oral dosing achieves the predicted exposure level required for efficacy, based on preclinical models.
About Spinal Muscular Atrophy:
Spinal Muscular Atrophy (SMA) is an autosomal recessive genetic disease that affects the motor neurons of the voluntary muscles that are used for activities such as crawling, walking, head and neck control, and swallowing. Approximately 1 in 6000 babies born are affected and about 1 in 40 people are genetic carriers. SMA patients are divided into three subtypes depending on disease onset and severity but all suffer from degeneration of motor neurons controlling voluntary muscles with proximal limb and trunk muscle weakness leading to respiratory distress and in the most severe cases, ultimately death. For further information, see www.afm-france.org or www.curesma.org
About Trophos: www.trophos.com
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, with a lead product, TRO19622, in phase II clinical trials. Trophos’ mitochondrial pore modulator compounds enhance the function and survival of stressed cells via modulation of dysfunctional mitochondria through interactions at the permeability transition pore (mPTP). Recently published clinical studies support the therapeutic rationale for mitochondria targeted drugs in neurology (Alzheimer’s disease) and cardiology (ischemia-reperfusion injury), which Trophos is uniquely placed to exploit.
