Advanced Cell Technology’s Myoblast program for the treatment of heart failure has successfully completed Phase I human clinical trials utilizing the therapy safely in over forty patients. Safety of the company’s myoblast therapy has been demonstrated in four independent studies. Moreover, the company has been permitted to apply its myoblast therapy without the use of accompanying arrhythmia preventatives usually required for similar programs. The FDA has reviewed the “end-of-Phase I” data and will allow Advanced Cell Technology to proceed with a Phase II human clinical trial. The company plans to begin the Phase II human clinical trial for the treatment of heart failure in approximately 160 patients. If successful, the company expects to proceed with a pivotal Phase III trial. The Myoblast program may prove particularly beneficial for patients that have experienced a serious heart attack and have a high risk of heart failure.
ACT achieved a significant milestone in its retinal pigment epithelium (RPE) program by successfully restoring visual function in rats through the implantation of RPE cells derived from human embryonic stem cells. The study results were reported in a paper published in the journal Cloning and Stem Cells. Spurred by this positive data, the company formed a collaboration with the Casey Eye Institute at Oregon Health and Science University to conduct preclinical trials for its RPE program. In early 2008, the company announced that it had completed pre-IND meetings with the FDA for the RPE program. Advanced Cell Technology’s focus on age-related macular degeneration (AMD) represents a tremendous market opportunity given the size of the patient population and the lack of treatment alternatives. Existing therapies only treat the condition and do not regenerate cells of the macula that have been damaged. Plus, existing therapies only treat wet AMD, not dry AMD, leaving a significant unmet need for an effective treatment for the 90% of AMD patients who have the dry variety. The company expects to file an IND for its RPE program in 2008.
Advanced Cell Technology recently announced a significant milestone in its hemangioblast (HG) program by reporting for the first time that hemangioblast precursor cells derived from hESC’s can be used to achieve vascular repair. The research , which appeared in the journal Nature Methods, by the company and its collaborators, described an efficient method for generating large numbers of bipotential progenitors (hemangioblasts) from hESC’s that are capable of differentiating into blood vessels as well as into all blood and immune cell lineages. The ability to repair vascular damage using these cells could have a profound impact on a large number of diseases that are major human afflictions. The company is committed to moving this technology from the laboratory into the clinic and plans on filing an Investigational New Drug Application (IND) with the Food and Drug Administration for the first clinical application of these cells in 2008.