Geron and ACTC leading the stem cell revolution

This is from Michael J. Ray's blog on Seeking Alpha. I'm long both Geron and ACTC.

 One cannot initiate a discussion of stem cell companies without starting with Geron (GERN). Geron is a company that develops biopharmaceuticals for the treatment of cancer and chronic degenerative diseases, including spinal cord injury, heart failure, and diabetes. GERN’s pipelines run fairly deep and besides stem cell technologies it has multiple Phase 2 trials dealing with cancer therapies. That being the case, the real catalyst for the company is the Phase 1 clinical trial dealing with treatment of spinal cord injuries with stem cells. If GERN is going to be the catalyst that starts this medical revolution then it will begin in this trial.

paralysis in many patients. In animal models, Geron was able to show improved functional behavior using GRNOPC1 at the injury site seven days after injury. After much review, the FDA then granted approval for a clinical trial and Phase 1 testing of GRNOPC1 in patients with spinal cord injury began in October 2010. Two clinical sites were opened for patient enrollment in 2010, and the remaining 5 were opened in 2011.

What GERN has done is to derived Oligodendrocyte progenitor cells (GRNOPC1) from Human Embryonic Stem Cell (hESCs). Oligodendrocytes are naturally occurring cells in the nervous system that have several functions. They produce myelin (insulating layers of cell membrane) that enables the cells to conduct electrical impulses, plus they also produce neurotrophic factors that support the maintenance of nerve cells. When these Oligodendrocytes are lost in spinal cord injuries, the results are a loss of the myelin and neuronal function that in the end will cause 

In late July 2011, Geron stated that a second subject was enrolled and received GRNOPC1 in the ongoing Phase 1 clinical trial. The treatment was administered by investigators at Northwestern Memorial Hospital and Northwestern University Feinberg School of Medicine. The company also went on to state that the preliminary data from the first two patients showed no safety concerns or serious adverse events. That being the case, Geron received clearance from the FDA to expand the eligibility criteria for the trial to allow inclusion of a wider range of patients.

million. The company also stated that the cash reserve plus the income Geron receives will be sufficient to fund the current level of operations through at least December 2012. Geron also states that future capital requirements will be substantial as several trials are moving forward.

For the second quarter of 2011, the company reported a net loss of $21.1 million. Net loss for the first six months of 2011 was $45.5 million and the company ended the quarter with $192.2 million in cash and investments. The total operating expenses for the second quarter of 2011 were $21.9 million which broke down to research and development expenses of $16.5 million and general and administrative expenses of $5.3 

So will Geron be the one who will strike first with its stem cell therapy? Phase 1 trials have been going on for awhile and so far all we have really heard is that the therapies are safe but we are still unsure as to how effective they are. It is obvious that the trial is very ambitious and if successful will be a game changer, but one must worry if the company will run out of funding before they get to that point. It will be most interesting to watch as events unfold in this company’s future.

commercialization of human embryonic and adult stem cell technology in the field of regenerative medicine. In early 2010 the FDA granted orphan drug status to Advanced Cell for an embryonic stem cell derived treatment for a specific form of blindness, Stargardt’s Macular Dystrophy.

If Geron is not moving at the pace you want, then you might want to consider fast acting Advanced Cell Technology (ACTC). Advanced Cell is a biotechnology company focused on the development and 

Later that same year the company was cleared by U.S. regulators to test a therapy made from embryonic stem cells in patients with macular degeneration. Advanced Cell wasted little time and by July 2011 the company announced the dosing of the first patients in each of its two Phase 1/2 clinical trials for Stargardt's macular dystrophy and dry age-related macular degeneration (dry AMD). These patients were treated at the David Geffen School of Medicine at UCLA and at UCLA's Jules Stein Eye Institute. Notice that the trial is Phase1/2 thereby speeding up the trials. Both the Stargardt's trial and the dry AMD trial will enroll 12 patients each, with cohorts of three patients each receiving an ascending dosage of the stem cells as the trials move forward. The initial dosing was relatively small (50,000 cells) and the early indications were that the patients tolerated the surgical procedures well. In late September 2011, the independent Data and Safety Monitoring Board (DSMB) overseeing the Company’s two trials authorized ACTC to move forward with enrolling and treating the next set of patients in each of the trials.

what is going on in Europe. At the same time the DSMB made its announcement, Advanced Cell received clearance from the U.K. Medicines and Healthcare products Regulatory Agency to begin similar Phase 1/2 clinical trial for Stargardt’s Macular Dystrophy. It should also be known that the European Medicines Agency previously granted Orphan Drug designation for the company's RPE cell product for use in treating SMD. At this point it should be noted that macular degeneration in the U.S. is a $30 billion market with relatively no competition. Add a European market to the mix and the numbers go even higher. To add fuel to the fire one has to consider a recent statement from the CEO when he stated,What should be taken away from this is that the pre-clinical animal findings were extremely successful and if this carries forward in humans then the sky is the limit for the company. Also this is the first time that any testing in Asia had been mentioned. And if all that is not enough, one must also consider the Advanced Cell’s Phase II-approved Myoblast autologous adult stem cell therapy for the treatment of chronic heart failure advanced cardiac disease, myocardial infarction, and ischemia; as well as the Hemangioblast (HG) program for the treatment of blood and cardiovascular diseases which is currently in preclinical development.

If that was not enough, then on top of the U.S. trials one must consider 

As wonderful as all Advanced Cell’s programs sound, there are still lots of risks with the company. The company basically has no revenue and for the second quarter of 2011; net cash used in operations was $3.2 million. Needless to say the expenses will only increase as the company pushes forward on its clinical trials. Advanced Cell ended the 2011 second quarter with cash and cash equivalents of $16.1 million and will burn through that rather quickly. Add to this a couple newly filed lawsuits and a 1.7 billion share count and we find Advanced Cell in a race for funding. Advanced Cell has stated that several top pharmaceutical companies are closely following the trials and if the company can prove it can eliminate macular degeneration, it is almost certain it will get a joint venture or even a buyout. Until such time though, investors will have to wait and see if this company will be the spark that lights the fires of stem cell revolution.

For now, it will have to suffice to say that we are extremely pleased and believe that these trials will validate our earlier pre-clinical findings. In the meantime, we have begun to set our sights on expanding our clinical trials to in Europe, having been recently approved to begin our SMD trial in the United Kingdom, and various countries in Asia.

                                     Good luck in this rigged casino,