Not sure who sent this to me or where it originated, it appears this article will be out in BioWorld on Monday. Thanks to whomever.
ACT Awaits Details of FDA Hold on Embryonic Stem Cell Study
BioWorld Today
11 January 2010
By Catherine Hollingsworth,, Staff Writer
Phase I and II studies of Advanced Cell Technology Inc.'s retinal pigment epithelium cells are at a standstill due to a clinical hold imposed by the FDA.
But the company has the funds in place to conduct those studies, using the more than $15 million in definitive commitments it received last year through three private placements with institutional and accredited investors.
The Worcester, Mass.-based company said that it is awaiting a response from the FDA relating to an investigational new drug (IND) application filed late last year to obtain clearance to start the Phase I/II study of its embryonic stem cell-derived retinal cells in patients with Stargardt's macular dystrophy.
The FDA did not provide specifics about the clinical hold but indicated that it would do so in a follow-up letter expected within a few weeks, according to ACT, which declined to comment further on the matter.
ACT Chairman and CEO William M. Caldwell IV said in a statement, "Although ACT has taken important steps to advance our RPE program during the last several years, we anticipate the need for further discussions with the agency before clinical trials can proceed."
If the hold is lifted, much of the $15 million in proceeds expected from the private placements would go toward the retinal pigment epithelial (RPE) program, ACT has said. Those financings, detailed in a Securities & Exchange Commission filing in November 2009, gave ACT enough cash to fund the company's general operations and working capital well into 2011.
ACT has published positive data from animal studies for the RPE cell program and also has reported promising results from safety studies and long-term efficacy studies for the RPE therapy. Those studies were completed in collaboration with the Casey Eye Institute at Oregon Health and Science University.
The company's other programs include myoblasts, an autologous adult stem cell therapy, aimed at heart disease that successfully completed several Phase I trials.
...The program has clearance from the FDA to begin Phase II trials, and ACT is seeking funding to start that study.
In addition, ACT has an hemangioblast cell program in preclinical research for possible use of the cells to treat cardiovascular disease, stroke and cancer. ACT's research, published in Nature Methods, has demonstrated success in treating vascular indications such as heart attack and limb loss due to vascular damage. An IND for that program is expected to be filed in 2010.
ACT and Geron Corp. are the furthest along in development of embryonic stem cell-based products, and both have had holds placed on their INDs. However Geron recently reached agreement with the FDA on a plan for restarting a Phase I trial of its human ESC-based product, GRNOPC1, in spinal cord injury.
The agency placed a hold on the Geron study after a preclinical study showed a higher frequency of animals developing cysts in the injury site than had been seen in previous preclinical trials.
Geron agreed to complete a confirmatory preclinical study using new markers and assays that it has developed for testing in an animal model of cervical injury.
If data from that study are positive, the FDA agreed that those results could be used to support the release of the clinical hold, as well as the expansion of the study into cervical patients. Geron anticipates data from the study to enable reinitiation of the Phase I trial in the third quarter of 2010.
Shares in ACT (OTC OB:ACTC) were down 1 cent Friday, closing at 11 cents.
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