That's you Keep_TheDooshbagLoser. Your husband is going to give you a real plugging tonight for spending the family fortune (2 shares) with no hope in sight of getting even for 2 more years! lol
STEM's Phase I AMD interim results were disappointing.
A former employee is making serious allegations about improper manufacturing practices in a lawsuit.
After granting STEM $19.4 million in the form of a forgivable loan, the former president of CIRM joined STEM’s board. Questions of graft ensued.
Politics, cost and scalability are working against STEM and their method of deriving stem cells from the brains of aborted fetuses.
StemCells Inc. (NASDAQ:STEM) is a small cap biotech company primarily involved in stem cell research. Its stock is currently trading at $1.16, which is 50% of its recent highs.
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In June of this year, STEM issued a press release, touting interim results for their Food and Drug Administration (FDA) Phase I clinical trial testing a treatment for Age-related Macular Degeneration (NYSE:AMD). According to the press release: "Interim results for the current AMD trial show a 70 percent reduction in the rate of geographic atrophy (GA) as compared to the control eye…"
Slowing the progression of a disease that currently has no FDA approved cure was initially seen as great news by the market. After rising steadily for a month prior to the news release of promised stellar results, STEM's stock shot up from $1.75 to $2.34. Those gains quickly faded as the public began to realize STEM's results were not as impressive as the company indicated they would be.
It would have been great news to slow the rate of AMD progression by 70% if one of their competitors, Advanced Cell Technology (OTCQB:ACTC), had not completely halted the disease in patients enrolled in their Phase I trial. Many of ACT's patients even experienced persistent gains in visual acuity. In my previous article, I postulated that Phase I results in stem cell trials were likely to persist throughout the ensuing trial phases. That won't be good enough for STEM. They're going to need to show better efficacy to keep up with the competition.
But all hope is not lost. STEM's Phase II trial should commence soon and include patients with better vision and more recoverable photoreceptors. They'll certainly have a better chance to halt the progression of the disease in healthier eyes, but I'm not holding out hope for any meaningful improvement over their disappointing Phase I results.
How much they choose to spend on this expensive and seemingly fruitless endeavor may soon determine the fate of this company.
Rob Williams, a former senior manager at STEM, made some serious allegations against the company in a lawsuit filed in July 2014. Specifically, he accuses the firm is manufacturing human cells in a process that puts "patients at risk of infection or death."
Ken Stratton, general counsel for STEM, responded in part, "We believe our processes, procedures and controls, as fully described in our regulatory filings, are appropriate for a company at our early stage of clinical development and comport with applicable guidelines and regulations."
This seems to me more like a confession than a denial. Any way you look at this, it's a weak response to a serious charge that desperate patients, condemned with a prognosis of encroaching, incurable blindness, were subjected to life-threatening risks due to STEM's poor manufacturing practices. Their response is not encouraging, to say the least.
Along with the obvious mortality and morbidity risks that go along with tumor formation, one contaminated assay from STEM could derail the progress of all stem cell companies. The sloppy tactics alleged by the former employee are highly irresponsible on many social and scientific levels.
They haven't been found guilty yet, and perhaps they never will, but let's get back to their weak response. They seem to believe, or want the public to believe, that the FDA's Current Good Manufacturing Practice (cGMP) regulations on processes, procedures and controls change based on the stage of clinical trials. This is absolutely false. Being a small company doesn't absolve STEM of the responsibility to protect their patients. Hopefully, Mr. Stratton just wrote a poorly worded sentence that is not indicative of their true beliefs. I look forward to clarification from the company as soon as possible.
How should they respond? If this lawsuit is simply filled with lies from a disgruntled employee, they should file a countersuit. They should explain to the world that these allegations could not be true and present evidence to back it up. Their weak defense is very troubling. This could have disastrous effects for patients, STEM, the doctors and hospitals involved who trusted STEM's methods and even for the stem cell industry as a whole.
STEM's relationship to Alan Trounson, Ph.D., former president of the California Institute for Regenerative Medicine (CIRM) - a taxpayer funded organization that governs the allocation of $3 billion authorized to fund stem cell research in California - is raising serious questions. The firestorm began after Dr. Trounson accepted a position on STEM's Board of Directors in June of this year, one week after resigning from his post at CIRM.
This relationship is troubling because in many ways CIRM acts as an arbiter of which small cap struggling biotech organizations are thrown a lifeline and which are condemned to suffer the slings and arrows of raising money on the free market. Notably, when Trounson was at the helm CIRM issued a forgivable loan to STEM for $19.4 million. His new appointment to STEM's board has raised questions of graft.
According to CIRM's new president and CEO, C. Randal Mills, "Finally, in the interests of transparency and good governance we will be conducting a full review of all CIRM activities relating to Stem Cells Inc." The press release went on to state, Dr. Alan Trounson's appointment to STEM's board, "… raises concerns on a number of fronts …"
If CIRM's internal investigation uncovers evidence of corruption and leads to a legal battle, that could finally doom this fledgling biotech company for good.
With the Republicans set to take over the US Senate this January, STEM may face some unexpected headwinds. STEM derives their HuCNS-SC® human neural stem cells from the brains of aborted fetuses. Why they chose this route, considering their poor pre-clinical and Phase I results, is unclear. Whatever benefit they thought this method would generate hasn't shown up clinically. There are certainly more ethical choices for a source of stem cells than the brains of aborted fetuses, and they may be in for a rude awakening as unexpected political hurdles arise.
Importantly, the cost and scalability of these human neural stem cells will become major issues if these trials ever progress to a large-scale Phase III.
After announcing a $20 million offering in July, I estimate STEM has approximately $27 million cash and cash equivalents on hand. I estimated this using their 10-Q quarterly report which was filed August 12, 2014. This report shows an average quarterly burn rate of $9,867,740 for 2014. At this rate, they'll need to dilute shareholders further in the summer of 2015 in order to keep operating, or they'll have to somehow come up with a joint venture, grant or some other means of non-dilutive funds. At this point, another forgivable CIRM loan is improbable.
If the stock price continues to drop they may get delisted from the Nasdaq exchange. To remedy this, a reverse split is likely necessary in 2015 in order to maintain the stock price at a level that meets Nasdaq's continued listing requirements.
On a more positive note, stem cells in general have shown great promise recently. If STEM could manage to publish their clinical results in a high impact, peer-reviewed journal, its small market cap of $80 million could see explosive growth. STEM needs some positive news to propel a rise in their stock price. They have shown some wild swings in the past. Whether or not they are able to achieve good enough results to warrant publication is a question that may not be answered for several more years, if ever.
More good news that should be considered is that STEM does have other programs in their pipeline, and they completed enrollment in their Phase I clinical trial treating spinal cord injury in April of this year. Results are expected mid-2015 and may warrant a bigger Phase II trial if the company sees promising results.
This stock has stunk like hot garbage as of late, losing 50% of its recent value. The small company is in a precarious financial position, and many dangerous pitfalls lie ahead with only a few rays of hope on the distant horizon. Sadly, in my opinion, even at these low levels, STEM is a resounding sell.
I see you like the name I gave him weeks ago! LOL Fits perfect JSHITTY...another DOOSHBAGLOSER!
Efficacy gains are much more likely to persist for stem cell trials than for drug trials.
The worldwide age-related macular degeneration patient population is approximately 200 million. This presents as a $2 trillion market opportunity for a cure.
In the summer of 2015, ACT's stock should begin an exponential rise as the company’s trials progress toward commercialization.
At the time this article was written, Advanced Cell Technology (OTCQB:ACTC) had a market cap of $220 million. For a typical pharmaceutical company with a few Phase I and several preclinical trials, this may seem to be quite a large valuation. But ACT is not a pharmaceutical company.
Next month, they will begin Food and Drug Administration Phase II clinical trials using Retinal Pigment Epithelium (RPE) cells derived from human embryonic stem cells. Widely viewed as equally ethical as adult stem cells, ACT's patented method used to derive these cells does not require the destruction of the embryo.
Why Is ACT Undervalued?
The FDA approval process is long and costly. In a 1997 study, Drs. Klees and Jones found that for "approximately every 5,000 to 10,000 compounds that enter preclinical testing, only one is approved for marketing." According to Martin S. Lipsky, MD, and Lisa K. Sharp, PhD, Department of Family Medicine, Northwestern University Medical School, Chicago, "on average, about two thirds of Phase I compounds will be found safe enough to progress to Phase II." Furthermore, a Tufts Center study in 2003 found that, "only 21.5% of drugs that begin Phase I trials are eventually approved for marketing."
Two of the most common reasons for the high failure rate of drugs in the rigorous FDA clinical trial process are low efficacy and intolerable side effects. To a large degree, these problems can be attributed to differences in human DNA. Even drugs that gain FDA approval can have harmful side effects or muted benefits in many people. This is because most drugs on the market are not active until metabolized in the body -- i.e., they are inert until chemically altered by the liver, kidneys, etc. The metabolic rate is determined by a patient's DNA. A drug may treat some people optimally, but others will metabolize it too quickly, too slowly or not at all. This can have disastrous effects for many people. For instance, metabolizing a common anticoagulant like warfarin at too rapid of a rate can cause internal bleeding.
These adverse drug events cause 7,000 deaths and $21 billion per year in the U.S., according to the New England Health Institute. And this doesn't include the 78.5% of drugs which start but don't make it through the FDA approval process. Luckily, stem cells are not drugs.
ACT's dry AMD (age-related macular degeneration) and Stargardt's Macular Dystrophy (SMD) trials are being treated like drug trials, going through the same clinical phases. And the investor community is valuing their chance of approval like they would for typical drug applications. But stem cell-derived RPE cells are not drugs. The same uncertainty and random effectiveness does not exist. ACT's RPE replenishment is more akin to an organ transplant than a drug treatment. Dosages need to be defined and procedures refined, but at the core these cells work or they do not. A large, diverse patient population is required by the FDA, but it should not be required to convince you of when to invest.
ACT's treatments have already shown efficacy in Phase I, with the treatment halting disease progression in at least 17 out of 18 patients. One patient's vision worsened due to developing wet AMD. We don't know if the patient's SMD progression halted, so all we can say right now is the efficacy rate is either 94.4% or 100%. I suspect the latter. After all, if a patient were to stick a fork in her eye, we certainly wouldn't call the SMD treatment in-efficacious simply because her vision worsened. This patient developed a different disease than was being treated. Perhaps at the upcoming Annual Shareholders Meeting the company officers will elaborate on this topic.
With this Phase I data now published in a high-impact peer-reviewed journal as noted above, investors should feel confident that the efficacy data will persist or improve throughout the trials. Of course, FDA approval is not a certainty. But it is much more likely than a typical drug application in early stages of clinical trials.
So, How Should ACTC Be Valued?
The company recently announced a $100 million shelf offering. This will be piecemealed out over the next couple of years and should be more than enough to fund the company through Phase II of the RPE trials. Looking ahead two years and adding the full amount (although, I suspect they will only raise about $30 million in the near term) of the shelf to the market cap at today's price per share gives ACT a market cap of $320 million.
According to a study by Wan Ling Wong and colleagues published in The Lancet in February 2014, "The projected number of people with age-related macular degeneration in 2020 is 196 million (95% CrI 140-261), increasing to 288 million in 2040 (205-399)." Clearly, two years from now a company about to enter Phase III and soon thereafter gain access to a $2 trillion worldwide market (200 million patients at $10,000 per treatment) will be worth considerably more than $320 million. But when?
Upon commercialization, revenue will be limited by the number of doses they are able to manufacture and the number of doctors trained in the procedure. Also, a majority of patients worldwide will not be able to afford the treatment. (There are advantages to pricing this therapy on a country to country basis, but this is beyond the scope of this article.) For now, I'll assume the viable market is 20% of the overall market. Eventually, other companies may gain approval to compete for this market as well. Although no other company has shown efficacy anywhere close to as good as ACT's, for the sake of argument and conservatism I'll assume ACT only has access to 25% of the 20% of recruitable patients able to pay for therapy. That leaves about 10,000,000 patients for ACT to begin to treat. At $10,000 per treatment, potential revenues are $100 billion. Right now, the market is saying ACT has a 0.3% (0.0032) chance of bringing this therapy to commercialization.
Phase II results are what typically convince the investing community to take a chance on a stock. With ACT's stellar efficacy shown in Phase I that will likely persist throughout the trials, these next few months may be the last time the stock will be able to be had at such a bargain. On a recent conference call, Dr. Eddie Anglade, ACT's chief medical officer, assured stakeholders Phase II would proceed much more rapidly than did Phase I and they would give regular updates on their progress. I expect the first Phase II update in the summer of 2015 and predict the stock price will then begin its exponential rise.
The year 2020 is the worst case estimate for how long it might take before commercializing this treatment. The best case would be 2017. If efficacy continues to track at anything close to 100%, the trials will definitely be stopped early. My guess is that these efficacious results persist and ACT gains FDA approval within three years of today, (10% completion of Phase III) at which time approximately 200 physicians at 100 hospitals in the U.S. and Europe will have been trained to perform this procedure. This means that my best case scenario for ACT in 2017 is $1 billion in revenue (100,000 treatments). The worst case scenario is that they don't generate this $1 billion in revenue until a full Phase III trial is completed and commercialization occurs in 2020.
The procedure is relatively simple, so physician training and patient recruitment around the world will ramp up quickly. ACT should be able to reach $10 billion in revenue within three years of commercialization. It costs ACT $100 per dosage of cells for each treatment. There will almost definitely be a joint venture for the purposes of marketing and distribution at some point, but how much should ACT give up for these services? My guess is 25%. So, on $10 billion of revenue ACT should profit about $7.5 billion.
How much of a P/E multiple will the market assign to a company with such incredible growth potential? (I am projecting that, at three years post commercialization, ACT will be treating one million patients per year. This is 0.5% or 1/200th of the patient population.) Conservatively, I will estimate 30:1 for a P/E multiple. This would give ACT a market cap of $225 billion, and this will occur between the years 2020-23. If there are 50 million shares outstanding, this gives us a stock price of $4,500 per share.
Currently, the stock trades at $6.30. So, over the next five to eight years I predict quite a healthy return. (For the purpose of this exercise I am assigning a zero value to all other programs in the company's pipeline. The valuations will ramp up quicker if there is a great deal of off-label usage for the orphan designated SMD treatment.)
ACT's quarterly burn rate absent of litigation expenses is averaging $5.4 million per quarter for 2014, as can been seen in the company's latest quarterly report here. Their burn rate will likely increase considerably next year. The number of hospitals involved in the RPE trials will rise from five to 30 in Phase II. A Phase I Myopic Macular Degeneration trial is projected to commence soon at UCLA, and ACT may begin a Photoreceptor Progenitor Cell Therapy Phase I trial in the first quarter of 2015. These endeavors may deplete the $100 million shelf offering sooner than anticipated. This could lead to further dilution and may limit any potential near-term gains in the stock price.
There will definitely be some peaks and troughs in the stock price, but the trend will be decisively up after the first Phase II update as the company runs their trials inexorably toward commercialization. These treatments have already shown efficacy and long-term safety. The uncertainty of eventual FDA approval should be minimal, and this is a great opportunity to invest in this stock before the rest of the world figures out stem cells are not drugs.
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Keep_TheDooshbagLoser.....another ID???? Geez....and then having your husband JShitty post to it? Sad little fluffer clown.