Trophos Starts Phase Ib Clinical Trial of TRO19622 in Spinal Muscular Atrophy

Neurodegenerative disease specialist begins Phase Ib study of lead molecule in patients with SMA

Marseille, September 28th, 2007 - Trophos SA, a biopharmaceutical company specializing in the discovery and development of drugs for neurological disorders, announced today that the company has begun enrolling Spinal Muscular Atrophy (SMA) patients in a Phase Ib clinical trial of its lead product, TRO19622. The clinical trial will involve 20 type 1b-3 SMA patients aged between 6 - 25 years of age and will assess the pharmacokinetics and safety of drug product after administration of single and multiple doses, once-daily, by the oral route. The study is being conducted at three centers in France. The clinical program in SMA is supported by the Association Française contre les Myopathies (AFM,  www.afm-france.org ), through a strategic partnership begun in 2000.

TRO19622 is representative of novel compounds identified using the proprietary neuronal cell screening platform developed at Trophos. Preclinical studies have demonstrated that these compounds promote the survival of a wide range of neurons under disease relevant stress conditions. TRO19622 has successfully completed Phase I/Ib studies in both healthy volunteers and ALS patients demonstrating the product is well tolerated, has an excellent safety profile and that once-a-day dosing achieves the predicted exposure level required for efficacy, based on preclinical models. The European Commission has granted the company an 'Orphan Medicinal Product' designation for TRO19622 as a treatment for SMA. Trophos is currently enrolling patients in a European Phase IIa trial to assess the efficacy of TRO19622 in painful diabetic neuropathy and expects to begin a Phase II/III trial in ALS in 2008.


"We are delighted to have begun enrolling patients in our ongoing clinical development program in SMA, a debilitating disease with tremendous unmet medical need," said Jean-Louis Abitbol, CMO at Trophos. "The excellent safety profile of TRO19622 in healthy volunteers and ALS patients supports further clinical development in juvenile/adolescent SMA patients. The successful completion of these Phase Ib studies will enable Trophos, subject to regulatory approvals, to initiate Phase II/III clinical trials to evaluate TRO19622 efficacy in the SMA indication."


"We at the AFM are an association which has for many years been actively encouraging the development of innovative therapies to treat long forgotten diseases on behalf of patients, including those suffering from SMA," said Laurence Tiennot-Herment, AFM president. "Now, barely four years after the identification of a candidate molecule, there is a clinical trial underway. This shows once again how a patient association can play a leading role in promoting drug development."

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.curesma.org

About Trophos: www.trophos.com
Trophos is a biopharmaceutical company committed to the discovery and development of novel therapeutic compounds to treat neurological disorders and other diseases with high unmet medical needs. The Trophos discovery strategy has enabled it to develop a proprietary portfolio of products, such as its lead product TRO19622, that confer a survival benefit upon both neuronal and non-neuronal cell types (such as cardiomyocytes & hepatocytes). This is achieved through a mitochondria-based mechanism of action with a robust therapeutic rationale predicted to exhibit a therapeutic benefit in diseases such as neuropathic pain, ischemia-reperfusion injury and hepatotoxicity. The company is focusing its efforts on the orphan indicates ALS, SMA and Huntington's disease. It is also seeking to establish clinical proof of concept and partnerships in other indications, such as neuropathic pain, ischemia-reperfusion injury and hepatotoxicity. The company is currently performing two clinical studies with TRO19622, a Phase IIa trial in painful diabetic neuropathy and a Phase Ib study in SMA.

Trophos was founded in 1999, is based in Marseille, France and currently has 32 employees.