Repligen Corporation (NASDAQ: RGEN) announced today that it has received approval from the Food and Drug Administration (FDA) to initiate a Phase 1 clinical trial of RG3039, a potential treatment for Spinal Muscular Atrophy (SMA). SMA is an inherited neurodegenerative disease in which a defect in the SMN1 (“survival motor neuron”) gene results in low levels of the protein SMN and leads to progressive damage to motor neurons, loss of muscle function and, in many patients, early death. This is a double-blind study to evaluate the pharmacokinetic and safety profile of escalating doses of RG3039 in up to 40 healthy volunteers. This will be the first clinical trial of a novel drug specifically designed to treat SMA and the first treatment approach which seeks to increase levels of the protein SMN.

“We are very pleased to have received approval to initiate human clinical trials with RG3039,” stated Walter C. Herlihy, President and Chief Executive Officer of Repligen Corporation. “RG3039 targets the core deficit of SMA and if this therapeutic approach is successful, it has the potential to arrest or slow disease progression and significantly improve patients’ lives.”

Patients lacking a functional SMN1 gene survive only because humans carry a second gene called SMN2 which produces low levels of SMN protein. RG3039, an orally bioavailable compound, is an inhibitor of an RNA processing enzyme which targets SMN2 and has been shown to increase production of SMN protein in cells derived from patients. In addition, RG3039 has been shown to improve mobility and lifespan in preclinical animal models of SMA. RG3039 is a new chemical entity, which is the subject of worldwide composition of matter patent applications which, if allowed, will remain in force until 2028 prior to any patent term extensions.

Spinal Muscular Atrophy is the second most common inherited neuromuscular disease. Symptoms of SMA typically emerge before the age of two and often progress to severe physical disability or loss of life. The prevalence of SMA in the United States and Europe is approximately 20,000 patients and there is currently no treatment or cure for the disease. This program was licensed in 2009 from Families of Spinal Muscular Atrophy, a non-profit organization with 30 chapters throughout the United States and over 70,000 members and supporters. Families of SMA fully funded and directed the preclinical development work with an investment of more than $13 million prior to licensing RG3039 to Repligen. This was the very first drug development program ever done for SMA.