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Families of Spinal Muscular Atrophy Announces Repligen Corporation Receives FDA Approval to Begin Phase I Clinical Trial in SMA
Date:5/19/2011

CHICAGO, May 19, 2011 /PRNewswire-USNewswire/ -- Families of Spinal Muscular Atrophy announced today that Repligen Corporation has received approval from the U.S. Food and Drug Administration to commence a Phase I safety study in healthy volunteers for RG3039, formerly called Quinazoline495, which is being developed for Spinal Muscular Atrophy.

FSMA began the Quinazoline program in 2000 at the very initial stages of drug development, when risk is the highest.  It was the very first industrial drug program for SMA ever conducted.  FSMA fully funded the program with investments of $13 Million.  The direction from FSMA provided the positive results necessary to license the program to Repligen Corporation and leverage larger funding for clinical development.

This clinical trial will be a double-blind, single ascending dose, Phase I study in healthy, adult volunteers to evaluate the pharmacokinetic and safety profile of RG3039 in up to 40 subjects.  The study will be the first step in the clinical development of RG3039 as a potential treatment for SMA.

"It is very exciting after 10 years of hard work by FSMA leading this program, including a significant financial investment, for the drug candidate to be licensed to Repligen in 2009 and to now be able to announce the approval to advance into human clinical trials.  This is a tremendous milestone for our community to have reached as we make progress toward a treatment for SMA," stated Jill Jarecki, Ph.D.  Research Director at FSMA.  "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.  

SMA, the leading genetic killer of children under the age of two, is typically marked by the degeneration of muscle movement including the muscles that control crawling, walking, swallowing and breathing.  There are no approved therap
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SOURCE Families of Spinal Muscular Atrophy
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