Families of Spinal Muscular Atrophy Announces Repligen Corporation Receives FDA Approval to Begin Phase I Safety Study

May 23, 2011

Families of Spinal Muscular Atrophy Announces Repligen Corporation Receives FDA Approval to Begin Phase I Safety Study

Press Release | May 19, 2011

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.

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MotorGraft Clinical Trial for SMA Placed on FDA Clinical Hold

February 8, 2011

MotorGraft Clinical Trial for SMA Placed on FDA Clinical Hold

FSMA Press Release | February 8, 2011

In December, California Stem Cell, Inc. (CSC) announced that CSC filed an investigational new drug (IND) application with the US Food and Drug Administration (FDA) for approval to commence a Phase I safety study on a jointly-developed stem cell-derived motor neuron transplantation therapy for Spinal Muscular Atrophy (SMA) Type I.  Recently, CSC received an official response from the FDA.  As anticipated the trial is on clinical hold. A summary of that FDA response from CSC is shown below.

California Stem Cell’s MotorGraft trial for the treatment of spinal muscular atrophy (SMA) has been placed on clinical hold by the Food and Drug Administration (FDA). A clinical hold is an order that the FDA issues to a sponsor to delay a proposed trial. Clinical holds are not uncommon, especially in cases of novel treatments such as this one. Similar holds were issued by the FDA to each of the previous applications for pluripotent stem cell therapeutics, such as those submitted by NeuralStem (ALS trial), Geron Corporation (spinal cord injury trial) and Advanced Cell Technology (macular degeneration trial), and all have since been lifted and advanced to clinical trials.

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FSMA Research Funding Leads to the Publication of 17 Journal Articles in 2010

December 17, 2010

FSMA Research Funding Leads to the Publication of 17 Journal Articles in 2010

Press Release | December 16, 2010

The research goal at Families of Spinal Muscular Atrophy is to accelerate the discovery and development of a treatment and cure for SMA. To help achieve this we fund basic research grants, along with later-stage drug discovery programs and clinical trials. FSMA research funding contributed to the results in 17 published articles in 2010.

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California Stem Cell Inc. Files IND to Commence Phase I Clinical Trial in Spinal Muscular Atrophy

December 1, 2010

California Stem Cell Inc. Files IND to Commence Phase I Clinical Trial in Spinal Muscular Atrophy

FSMA Press Release | December 1, 2010

IRVINE, Calif. (December 1, 2010) – California Stem Cell, Inc. (CSC) and Families of Spinal Muscular Atrophy (FSMA) announced today that CSC has filed an investigational new drug (IND) application with the US Food and Drug Administration (FDA) for approval to commence a Phase I safety study on a jointly-developed stem cell-derived motor neuron transplantation therapy for Spinal Muscular Atrophy (SMA) Type I.

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