Yesterday, Geron Corp. (GERN) provided an update on its experimental candidate, GRNOPC1, which is currently under clinical hold. On Aug. 18, Geron suffered a setback when the US Food and Drug Administration (FDA) put its Investigational New Drug (IND) application for the product on hold.
Earlier this year, the FDA cleared the company to begin clinical trials of GRNOPC1 for acute spinal cord injury. However, later the agency placed the trial on hold pending a review of new non-clinical animal study data submitted by the company.
Yesterday, Geron announced that the human study is being delayed as some animals treated with the injection developed microscopic spinal cysts in the regenerating injury site. It said that the cysts were non-proliferative, confined to the injury site and had no adverse effect on the animals.
Importantly, the animals did not develop teratomas (a type of tumor) or any other ectopic structure. According to the company, cysts of much larger size appear in the spinal cord scar tissue in more than 50% of patients with spinal cord injury.
Another animal study showed higher frequency of cysts, although their characteristics were similar to those in earlier studies. Meanwhile, Geron has identified new candidate markers and assays and reported that a recently concluded animal study showed no cysts in rats with spinal cord injuries.
Geron has submitted the above data to the FDA and is in discussions regarding the commencement of the clinical study. We expect investor focus to remain on the resumption of the spinal cord injury study. However, currently there is low visibility as to when the agency will allow the company to resume the phase I trials of the candidate.
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