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Repligen Announces Favorable Results from Phase 1 Clinical Trial of Experimental Treatment for Spinal Muscular Atrophy

Apr 25, 2012, 03:55 ET from Muscular Dystrophy Association

Crucial clinical trial support provided by MDA

TUCSON, Ariz., April 25, 2012 /PRNewswire-USNewswire/ -- Repligen Corp., in Waltham, Mass., announced today that its experimental drug RG3039, designed to treat spinal muscular atrophy (SMA), was safe and well-tolerated in a phase 1 clinical trial. In addition, data suggested that the drug reached and acted on its biological target, an enzyme called DcpS.


The trial of RG3039 marked the first of a new drug specifically designed to treat SMA.

Data from the trial, in which 32 healthy volunteers received a single dose of the experimental therapy, may help determine the appropriate dose levels of RG3039 for later-stage trials.

"MDA is pleased to see that its support of Repligen's advancement of RG3039 to a phase 1 trial has resulted in such a positive outcome," said Jane Larkindale, MDA's director of translational research. "These results represent a concrete step forward in the development of a therapy for SMA."

MDA awarded Repligen $1.4 million in December 2010 to support the advancement of RG3039 into phase 1 clinical trials. The award was made through MDA Venture Philanthropy (MVP), the drug development arm of MDA's translational research program.

About RG3039

RG3039 is a small-molecule therapy designed to increase cellular levels of fully functional SMN protein, which is deficient in SMA. It has been shown to increase production of SMN protein in cells taken from people with SMA, and also to improve mobility, life span and the health of nerve cells (motor neurons) in animal models of the disease.

The U.S. Food and Drug administration (FDA) has granted RG3039 fast track and orphan drug designations. A fast track designation allows for faster review of drugs that treat serious diseases and fill an unmet need, while orphan drug status provides economic incentives to companies developing treatments for rare diseases. Repligen licensed RG3039 from the patient organization Families of SMA, which funded and directed its preclinical development.

For more on RG3039 and Repligen's phase 1 trial, see the Quest News Online article "RG3039 Proves Safe in Phase 1 Trial for SMA."

About spinal muscular atrophy

SMA is a genetic disease caused by a flawed or missing SMN1 gene, which leads to insufficient production of SMN protein. SMN, which stands for "survival of motor neuron," is necessary for normal neuromuscular function. In people with SMA, motor neurons in the spinal cord die off, resulting in weakness and eventual paralysis of voluntary muscles, including those involved in breathing. The scope and severity of SMA vary widely, correlated with the age when symptoms begin. Newborns with SMA symptoms often don't survive more than a few years, while those who develop symptoms later may live well into adulthood — although mobility and respiration may be significantly affected.

About MDA Venture Philanthropy (MVP)

MVP is the Muscular Dystrophy Association's drug development program, which operates within MDA's translational research program. MVP is exclusively focused on funding the discovery and clinical application of treatments and cures for neuromuscular diseases. For more information, visit and follow MDA on Facebook ( and Twitter (@MDAnews).

About Repligen Corporation

Repligen Corporation is a leading supplier of critical biologic products used to manufacture biologic drugs. Repligen also applies its expertise in biologic product development to SecreFlo™, a synthetic hormone being developed as a novel imaging agent for the diagnosis of a variety of pancreatic diseases. In addition, the company has two central nervous system (CNS) rare disease programs in phase 1 clinical trials. Repligen's corporate headquarters are located at 41 Seyon Street, Building 1, Suite 100, Waltham, MA 02453. Additional information may be requested at

SOURCE Muscular Dystrophy Association

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