MDA supports Repligen with $1.4M grant for RG3039 Spinal Muscular Atrophy development program

Dec 15 2010

Repligen Corporation (Nasdaq: RGEN) today announced that the Company has received $1,400,000 in research funding from the Muscular Dystrophy Association ("MDA") to support the ongoing development of RG3039 for Spinal Muscular Atrophy ("SMA").  RG3039, our lead compound, is an inhibitor of an RNA processing enzyme which targets increased production of SMN, a protein of deficient levels in patients with SMA.  The goal of this grant is to support the advancement of RG3039 into human clinical testing, including continued evaluation in preclinical models of SMA, GMP manufacturing for human clinical trials and early clinical evaluation in healthy volunteers and patients.  Funding from the Muscular Dystrophy Association provides important support for Repligen's development programs and access to a global network of scientists, physicians and patients.  Repligen has previously been awarded two research grants from the MDA to support its Friedreich's ataxia program.

"We are very pleased to receive additional support from the Muscular Dystrophy Association, a national organization recognized for its commitment to innovative research aimed at providing new therapeutic options for patients," stated Walter C. Herlihy, President and Chief Executive Officer of Repligen Corporation.  "We plan to complete preclinical studies and advance RG3039 into human clinical testing in 2011."

"MDA is pleased to be working with Repligen to advance a truly promising therapeutic approach for SMA," stated R. Rodney Howell, M.D., Chairman of the MDA Board of Directors.  "Having invested some $22 million on SMA research since 1999, MDA is pleased to support the vital preclinical work now being undertaken for the planned human clinical trial."

Spinal Muscular Atrophy 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.  Symptoms of SMA often emerge before the age of 2 and progress to severe physical disability or loss of life.  Patients lacking a functional SMN1 gene survive only because humans carry a second gene, known as SMN2 which produces low levels of SMN protein.  RG3039, our lead compound, was licensed in 2009 from Families of Spinal Muscular Atrophy who invested more than $10 million to discover and conduct the initial development work.  RG3039 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 and to improve mobility and lifespan in a preclinical model of SMA.  Genetic analysis of SMA patients has revealed a striking correlation between disease severity and the number of copies of SMN2 carried by the patient.  Patients with 2 copies of the SMN2 gene are usually unable to ever sit without assistance while patients with 4 copies develop few symptoms before adulthood.  Thus, a doubling of the SMN2 gene copy number dramatically alters disease course and suggests that a therapy which increases the level of SMN protein may provide a significant clinical benefit.  If this therapeutic approach is successful, it has the potential to change the progression of the disease and significantly impact patients' lives.  SMA is diagnosed in approximately one in every 6,000 births in the United States and Europe where the estimated prevalence is approximately 20,000 patients.