Trophos starts trials on patients with neurological disorders

Neurodegenerative disease specialist Trophos is enrolling spinal muscular atrophy (SMA) patients in a Phase 1b clinical trial of its lead product TRO19622.

The clinical trial will involve 20 type 1b-3 SMA patients aged between six and 25 years of age. The trial will assess the pharmacokinetics and safety of the drug product after administration of single and multiple doses once daily by the oral route.

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 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 enrolling patients in a European Phase IIa trial to assess the efficacy of TRO19622 in painful diabetic neuropathy and expects to begin a PhaseII/III trial in ALS in 2008.

The study is being conducted at three centres in France and the programme is supported by the Association Francaise contre les Myopathies