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NEWARK, Calif., Jan. 6, 2014 (GLOBE NEWSWIRE) -- StemCells, Inc. (Nasdaq:STEM), a leading stem cell company developing novel cell-based therapeutics for disorders of the central nervous system, posted today the following Letter to Shareholders from its President and CEO, Martin McGlynn.
Dear Fellow Shareholders,
Every day, the news media brings us word of some new advancement in the development of stem cell therapies. While you clearly comprehend the vast potential of stem cell therapeutics to transform the practice of medicine, it can be understandably difficult to assess which companies to bank on amidst so much hoopla and hyperbole. As CEO, one of my easiest and most gratifying jobs is to report our persistent progress to you — real evidence that StemCells, Inc. continues to be a clear frontrunner.
What is more difficult is trying to understand our Company's stock performance for the year just ended. We have reached out to usually well-informed third parties in the analyst and investor communities to help us understand this dynamic, but have been unable to identify anything unusual or particularly enlightening. Perhaps we have not adequately explained to the market the progress we have made in advancing the science and the greatly enhanced organizational capabilities that we have put in place to enable StemCells, Inc. to effectively progress our technology to significant value-creating milestones. Clearly, we feel compelled to remedy any misconceptions about the Company's true value. I trust this letter will be a first step in the right direction.
Continuing to Lead the Industry
Our Company's guiding mission has been, and remains, to harness the regenerative power of stem cells to restore the healthy functioning of organs and other tissues impaired by the ravages of disease or injury. Focusing on uses for our proprietary platform technology consisting of purified and expandable human neural stem cells (HuCNS-SC
® cells), StemCells, Inc. has emerged as a leader in the quest for cellular therapies designed to treat diseases and disorders of the central nervous system (CNS). StemCells, Inc. was the first company to complete a U.S. clinical trial using human neural stem cells and is the only company with a pipeline currently addressing disorders in all three regions of the CNS: the spinal cord, the eye and the brain. Before this year is out, we intend to initiate Phase II multicenter, controlled efficacy clinical studies in both spinal cord injury (SCI) and dry age-related macular degeneration (AMD); in 2016, we expect to file an IND to evaluate the safety and efficacy of our HuCNS-SC cells to restore lost memory in Alzheimer's patients. No other company has so successfully and so broadly advanced R&D in stem cell-based therapies for such pervasive CNS disorders.
Clinical Trials Currently Underway and CompletedSpinal Cord Injury. In September 2011, we transplanted the first patient in our Phase I/II clinical trial in chronic spinal cord injury in Switzerland. To date, we have dosed eight of the twelve patients planned for this study. We have reported very encouraging results from the first three spinal cord injury patients who have already completed the trial. Twelve months post-transplant, two of the three patients regained lost sensation below the site of injury, and one of them improved from an Asia A classification (complete injury with no function below the site of the injury) to Asia B (partial sensation below the site of injury). Using the encouraging safety and efficacy data obtained from the Swiss trial, in 2013 we sought and obtained approval from Health Canada and the FDA to expand the study into North America, the largest market in the world for spinal cord injury. We did so to accelerate completion of the current study, and so we are confident that we will wrap up enrollment in Q1 2014, which should pave the way for the controlled Phase II efficacy trial that we plan to initiate by the middle of 2014.
Age Related Macular Degeneration. In October 2012, we transplanted the first patient in our FDA-authorized Phase I clinical trial for dry age-related macular degeneration (AMD), the leading cause of blindness in people over the age of 65. There is no approved treatment for dry AMD. To date, we have dosed seven of the sixteen patients planned for the study. Initially, out of an abundance of caution for patient safety, the Company agreed with the FDA to three major restrictions for the conduct of the trial: (i) we would restrict recruitment to two clinical sites in an effort to minimize the potential for adverse outcome due to differences in surgical skills; (ii) the first four patients would have little visual acuity,
i.e., would be "legally blind;" and (iii) we would limit the number of cells to be transplanted into the back of the eye to 200,000 cells per patient. Upon successful completion of the first four transplants at the Retina Foundation of the Southwest in Dallas, Texas, and upon full review of all the available data by an independent data-monitoring committee comprised of physicians with well-recognized expertise in ophthalmology, macular disease and vitreal-retinal surgery, the FDA agreed to increase the number of clinical sites from two to five, allowed for the enrollment of patients at a much earlier stage of the disease, and allowed us to increase the dose five-fold to one million cells per patient. As a result of this encouraging action by FDA, the Company is confident that it will be able to complete enrollment in its current study and initiate a controlled Phase II efficacy study later this year.
Genetic Disorders. We have completed and published the results of two Phase I trials evaluating the safety and preliminary efficacy of our HuCNS-SC cells in two rare genetic disorders: Neuronal Ceroid Lipofusinosis (Batten's Disease) and Pelizaeus Merzbacher's Disease (PMD). These studies have shown that we can safely transplant up to one billion cells into the brain of an infant or a child. We also learned that the cells engraft robustly, migrate deep into the brain structure, remain biologically active post withdrawal of the immunosuppression regime and, in the case of the PMD trial, we showed by MRI imaging that the cells had shown signs of myelination in the transplanted regions of all four children who received the cells. The hallmark of PMD, a fatal genetic disease affecting mostly male children, is that from birth these children are unable to produce sufficient protective myelin sheathing, which is essential for the survival of the neurons in the white matter tract of the brain. In severe cases, this characteristically results in critical neurological and intellectual deficits leading to the death of the child within the second decade of life.
Phase II controlled efficacy Proof of Concept Trials Planned this Year
Proof-of-Concept (PoC) studies are the definitive gate before initiating pivotal registration trials. Contrary to safety studies that are typically open label (where all patients receive the same treatment and there is no "control" procedure), PoCs are "controlled" because some patients receive the experimental treatment while others receive either a placebo or no treatment. Such methodology enables a definitive assessment of the efficacy of the experimental procedure. In the world of biotech, these results usually represent the inflection point determining a "go/no-go" status for the product candidate.