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.
Read the full press release