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news.Shares in development stage biotech firm StemCells Inc have fallen over 7% after the FDA put its proposed phase I trial of a Batten disease treatment candidate on hold.
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The company’s investigational new drug application filing was announced on January 4, for a phase I clinical trial of a proprietary neural cell therapy product, HuCNS, in Batten disease.
The FDA informed StemCells that it has suggestions and questions related to the proposed trial that require additional information. Consistent with normal FDA procedures, the FDA is expected to formally communicate the precise nature of these issues in writing to StemCells within 30 days.
In the interim, StemCells expects to continue talks with the FDA to address the outstanding issues.
The company pointed out that the FDA’s questions do not relate to contaminated embryonic stem cells that have received media attention recently. StemCells does not use embryonic stem cells or use mouse feeder cells in preparing its stem cells. All cells prepared by StemCells are grown in serum-free media and do not come into contact with cells from animals.
If approved by the FDA, the proposed study would mark the first-ever FDA-approved clinical trial to use a purified composition of human neural stem cells as the potential therapeutic agent.
The phase I trial is designed to investigate the safety of HuCNS-SC in the treatment of infantile and late-infantile neuronal ceroid lipofuscinosis (NCL), the most severe forms of a group of disorders commonly referred to as Batten disease.