NIH Brings Back Stem Cell Interest (STEM, GERN, ASTM, NBS, KOOL, OSIR, CYTX)
The National Institutes of Health has just today announced the approval of the first 13 human embryonic stem cell lines for use in NIH-funded research under the NIH Guidelines for Human Stem Cell Research adopted in July 2009. Children’s Hospital Boston developed 11 of the approved lines and Rockefeller University in New York City developed two of the approved lines. While those are private and non-public, this has some excitement around the other embryonic stem cell line companies because the NIH said that an additional 96 lines have been submitted to NIH for either internal administrative review or consideration, including more than 20 that will be considered on December 4, 2009. We are seeing strength as follows:
- StemCells Inc. (NASDAQ: STEM) up 5.5% at $1.14; 52-week range $0.89 to $3.07; market cap $135 million
- Geron Corporation (NASDAQ: GERN) up 4.8% at $5.70; 52-week range $3.00 to $9.24; market cap $522 million
- Aastrom Biosciences, Inc. (NASDAQ: ASTM) up 5.5% at $0.288; 52-week range $0.24 to $0.78; market cap $50 million
Neostem, Inc. (AMEX: NBS) is actually in adult stem cells, and shares are down 2.3% at $1.68; 52-week trading range is $0.41 to $2.72; market cap $61.3 million. ThermoGenesis Corp. (NASDAQ: KOOL) is also tied to adult stem cell lines but in products targeting stem cell companies; shares are up 3.4% at $0.61, its market cap is $34 million, and the 52-week range is $0.31 to $0.89.
Elsewhere in companies tied to stem cells… Osiris Therapeutics, Inc. (NASDAQ: OSIR) is up 0.4% at $6.89; 52-week range $5.35 to $21.65; market cap $225 million. Cytori Therapeutics, Inc. (NASDAQ: CYTX) is down 2.2% at $5.75, its market cap is $222 million, and the 52-week range is $1.42 to $6.04.
For a full data and explanation from the NIH website on what was approved, that can be found here.
JON C. OGG
DECEMBER 2, 2009
Stem Cell Study Starts in PMD (STEM)
StemCells Inc (NASD: STEM) has announced this morning that it and collaborator UCSF have initiated the enrollment of patients in the first ever clinical study of neural stem cells as a potential treatment in Pelizaeus-Merzbacher Disease (PMD), a neurological disorder that primarily afflicts children.
The company noted that the nature of this study is important on its own, but equally important is the fact that the findings from this trial may help also lead to potential stem cell treatments of other myelination disorders including multiple sclerosis and cerebral palsy.
The two have begun patient recruitment for a Phase I clinical trial designed to test the safety and preliminary efficacy of its HuCNS-SC® purified human neural stem cells. Also noted was that the study is being conducted at the University of California, San Francisco Children’s Hospital.
PMD results from a defective gene and is characterized by a lack of myelin, a substance that surrounds and insulates nerve cells’ communications fibers, which then makes the body unable to properly transmit nerve impulses. This leads to the loss of neurological function and eventually death in the most severe forms of PMD, and there are currently no effective treatment options for patients with PMD.
So far there is still a lukewarm reception. Shares are up 1%, but this is only a $1.00 stock. Starting trials is still a far cry from having positive trial results.
JON C. OGG
NOVEMBER 23, 2009
StemCells & PMD, Enters Pelizaeus-Merzbacher Disease Study (STEM)
StemCells, Inc. (NASDAQ: STEM) is trading higher this morning on a new study initiation. The stem cell player has announced that it will soon begin a study with the University of California, San Francisco Children’s Hospital. This will be in a Phase I clinical trial, and the goal will be to evaluate the therapeutic potential of StemCells’ proprietary HuCNS-SC® product candidate of purified human neural stem cells as a possible treatment for Pelizaeus-Merzbacher Disease (PMD).
PMD is a myelination disorder that primarily affects infants and young children. Myelin is the substance which surrounds and insulates nerve fibers and enables nerve cells to communicate with one another. Patients suffering from PMD have insufficient myelination of nerve cells in the brain, which leads to loss of neurological function and eventually death in the most severe forms of the disease.
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Geron Delay Dulls Stem Cell Sector (GERN, ASTM, CYTX, NBS, STEM, KOOL, OSIR, LIFE, CCEL)
Geron Corporation (NASDAQ: GERN) has some bad news in the land of stem cell investors. The stem cell leader has announced that its Investigational New Drug application for a cell therapy for neurologically complete, subacute spinal cord injury has been placed on clinical hold by the FDA pending the agency’s review of new nonclinical animal study data submitted by the company. This is an order that the FDA issues to a sponsor to delay a proposed trial or to suspend an ongoing trial.
As a reminder, no patients have yet been treated in this study. But this has investors spooked, even if it is just for a review of additional data. Stem Cell stocks often move hand in hand, and these are the other issues to watch on the news:
- Aastrom Biosciences, Inc. (NASDAQ: ASTM)
- Cytori Therapeutics, Inc. (NASDAQ: CYTX)
- Neostem, Inc. (NYSE: NBS)
- StemCells Inc. (NASDAQ: STEM)
- Thermogenesis Corp. (NASDAQ: KOOL)
- Osiris Therapeutics, Inc. (NASDAQ: OSIR)
- Life Technologies Corporation (NASDAQ: LIFE)
- Cryo-Cell International, Inc. (OTC Bulletin Board: CCEL)
Geron noted that the company has been undertaking studies to enable dose escalation of its spinal cord injury product and has also been investigating application of the product to other neurodegenerative diseases.
The company has also been performing additional product characterization and conducting further animal studies and the data from this work has been submitted to the FDA. Geron said that it will work closely with the FDA to facilitate the review of the new data and to release the clinical hold.
JON C. OGG
AUGUST 18, 2009
Diabetes Drug War Heats Up (NVO, AMLN, LLY, MNKD, PFE, SPEX, VVUS, GNBT, BMY, AZN, ARNA, GERN, STEM, OREX, HDIX, PODD)
Over the last couple of weeks, there has been quite a bit of new data in the drug war in the fight against diabetes. New studies have been updated, earnings projections have been made, FDA dates have been telegraphed and more. While these are still far short of ultimate cures, the war against diabetes may have many new or improved treatments out sooner rather than later. We originally discussed one or two of the key upcoming treatments pending for the eight major diseases and conditions as “the next $170 billion opportunity” and this is a much deeper dig into that broad initiation. We have included many of the recent developments in the potential treatments for obesity as well, considering that Type II diabetes and obesity are frequently conditions tied directly to each other.
According to the Journal of Health Affairs, the figure on obesity for Americans is a whopping $147 billion per year in total medical costs. This comes to 10% of all healthcare spending. The figure from the U.S. Centers for Disease Control was some $116 billion spent domestically on treating diabetes in 2007. As this is a lengthy bit, we have not included some of the other treatments that have been in use or that were recently flagged because of reports of higher chances of cancer rates associated by the long-term use of these.
FDA & IMMEDIATE ACTION
There is a new diabetes hopeful that is supposed to be coming sooner rather than later. Novo Nordisk (NYSE: NVO) reported a 21% gain in earnings in the last week and said that it expects the FDA to make a decision on its next-generation diabetes drug Victoza (liraglutide) in a matter of weeks. The company’s CFO and CEO both indicated that the Danish company does expects a positive response from the FDA and we heard a August to September expectation. Novo Nordisk has already launched Victoza in England, Germany and Denmark last month and expects to release it in other European Union countries throughout 2009 and into 2010. The benefit is that this one doesn’t risk pushing blood glucose levels to counts which are dangerously low and it also helps users lose weight. Novo Nordisk said it has priced Victoza competitively with Byetta from Amylin Pharmaceutical, Inc. (NASDAQ: AMLN) and Eli Lilly (NYSE: LLY). After the earnings and after shares were still close to 52-week highs, we saw analyst downgrades on Friday for Novo Nordisk by both UBS and by J.P. Morgan.
The drug still expected the next big new release with Blockbuster potential is an inhalable insulin from MannKind Corp. (NASDAQ: MNKD). Afresa is to be its name. Despite past woes of inhalable insulin, MannKind shares were hitting 52-week highs in June and its shares are still up 20% from three months ago. A late-stage study showed that Afresa’s performance was similar to injectable insulin. The company recently sold a 7.4 million shares secondary offering to raise cash for this launch, and its CEO took 1 million shares of the offering. The thought was that MannKind would secure a partner for marketing and development, but the recent stock offering gives it more internal options ahead of what is believed to be a Spring-2010 FDA approval action. Pfizer Inc (NYSE: PFE) has been thought of as a partner as it moved Exubera inhaled-insulin patients to MannKind’s experimental product. The two companies had been partners until Pfizer pulled Exubera from the market in 2007.
There is also a huge opportunity for the once per week dosing. We are not yet convinced that this can be a universal next generation treatment, however this might be fine for many of the lower grade cases if you can refer to any diabetes cases as lower grade. Amylin Pharmaceuticals, Inc. (NASDAQ: AMLN), Eli Lilly (NYSE: LLY) and Alkermes Inc. (NASDAQ: ALKS) have had a recent New Drug Application accepted by the FDA for review. Exenatide is an investigational sustained release medication for type 2 diabetes that would be injected once per week and is the active ingredient in BYETTA. We are not alone in this thought, but Amylin is a company which many have thought would be acquired for years now when considering the link of diabetes and obesity.
VIVUS, Inc. (NASDAQ: VVUS) has a substantial shot here with Qnexa, its Type 2 diabetes treatment through weight loss assistance. The stock recently came off on worries of its risk factor language that may have to be disclosed, but it showed a 9.4% weight loss or over 20 pounds observed in patients. The DM-230 study was a 56-week study assessing the impact of Qnexa on glycemic management in 130 obese patients. The 10-site study was comprised of 90 females and 40 males with an average age of 50 who had Type 2 diabetes, and a majority of the patients had been diagnosed with diabetes for more the five years and were taking two or more oral diabetes medications. In the phase II and phase III clinical trials, Qnexa demonstrated glycemic control, significant weight loss, and an improvement in cardiovascular risk factors. VIVUS is also presenting data at a brokerage firm conference this coming Thursday. The company’s market cap is still just under $500 million and its most recent balance sheet had north of $144 million in cash and equivalents with very little long term debt.
Bristol-Myers Squibb (NYSE: BMY) and AstraZeneca (NYSE: AZN) have recently received FDA approval for Onglyza as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes. This Onglyza is a once-daily dipeptidyl peptidase-4 (DPP4) inhibitor that can be used in combination with commonly prescribed anti-diabetic medications or on a standalone basis as a monotherapy to significantly reduce glycosylated hemoglobin levels.
MORE OBESITY CANDIDATES COMING
Several biotechnology companies are working on the next wave of obesity candidates, as noted above in VIVUS’s Qnexa. Arena Pharmaceuticals, Inc. (NASDAQ: ARNA) has Lorcaserin, Orexigen Therapeutics, Inc. (NASDAQ: OREX) has Contrave, and Amylin Pharmaceuticals Inc. (NASDAQ: AMLN) has pramlintide. Orexigen’s Contrave has completed phase III trials and our time line for when the company will file for approval is in early 2010. The company is presenting data this Thursday at the Canaccord Adams Global Growth Conference. VIVUS’s Qnexa is currently in two phase III programs with a new drug application expected around the middle of 2010. Amylin’s pramlintide and metreleptin are currently in phase IIb.
We have the expectation that Arena will have a first-mover advantage with an NDA planned before the end of 2009. Certainly, any delays or advances could change the status of the front-runner category leadership. Arena’s near-term catalyst is the release of the phase III BLOSSOM data out in September 2009, which will be used as part of a supplemental NDA in late 2009 or into 2010. This still leaves a year or more for final FDA action from now. Arena shares surged in late-July after reporting that its obesity results met the three endpoints.
STEM CELLS
Stem Cell therapy offers a huge promise, but so far that looks to be years out and the promise is actually more of a hope for the time being. Geron Corporation (NASDAQ: GERN) is in the research stage of using stem cells in evaluation of Type 1 diabetes. The exact level of this study is not as far as along as some of its cancer and spinal studies, but this is one of the few stem cell companies that have dedicated part of their mission to diabetes. StemCells Inc. (NASDAQ: STEM) also has a Pancreatic Program concentrating its efforts on Type-I diabetes. Its goals are to identify, isolate, and culture pancreatic stem and progenitor cells, and to test their therapeutic potential.
While we at BioHealthInvestor would love to hold hope and promise for stem cells, we would not be hoping for stem cell treatments any time in the near future. While some positive notions have been noted in the stem cell sector, the National Institute of Health noted, “Over the past several years, doctors have attempted to cure diabetes by injecting patients with pancreatic islet cells—the cells of the pancreas that secrete insulin and other hormones. However, the requirement for steroid immunosuppressant therapy to prevent rejection of the cells increases the metabolic demand on insulin-producing cells and eventually they may exhaust their capacity to produce insulin. The deleterious effect of steroids is greater for islet cell transplants than for whole-organ transplants. As a result, less than 8 percent of islet cell transplants performed before last year had been successful.”
Five Fast-Growing Biotechs To Watch Right Now (ALNY, FOLD, HALO, AOB, STEM)
Alnylam (Nasdaq: ALNY) leads a short list of some of the fastest-growing biotech stocks where analyst estimate revisions continue to rise.
BioHealth Investor began by analyzing 171 stocks in the biotech sector based on revenue growth over the trailing four quarters, identifying 30 stocks in the sector with better than 50 percent revenue growth over the past 12 months.
We then screened those 30 stocks to look for those very few names that have seen both strong growth in at least the past two years and positive analyst estimate revisions in recent months, in an effort to find stocks with strong trends that still have potentially improving operations going forward.
The work resulted in growth biotech stocks to watch: Alnylam Pharmaceuticals Inc. (Nasdaq: ALNY), Amicus Therapeutics Inc. (Nasdaq: FOLD), Halozyme Therapeutics Inc. (Nasdaq: HALO), American Oriental Bioengineering Inc. (NYSE: AOB), and Stemcells Inc. (Nasdaq: STEM).
1) Alnylam:
With a new class of potential biotech medicines, aggressive goals for development and partnerships, cash on the books and a large addressable market, Alnylam tops our short list of biotechs with strong growth and increasing analyst estimates.
Stem cell companies get endorsement from industry heavyweight (GERN, STEM, ASTM, GE)
Geron Corp. (Nasdaq: GERN) are up more than 25% in premarket trading after announcing an exclusive global licensing agreement with General Electric Co.’s (NYSE: GE) GE Healthcare.
The announcement is seen as an endorsement of the research done over the years by small-cap stem cell research companies. Rival stem cell biotechs including Stem Cells Inc. (Nasdaq: STEM) and Aastrom Biosciences Inc. (ASTM) also are rising on the news.
The GE Healthcare announcement is likely to be seen as an endorsement of the work that Geron had done on its own in bringing human embryonic stem cell-based therapies to market. The company plans to begin a Phase I trail of a therapy called GRNOPC1 for patients with thoratic injury, potentially in coming weeks.
by YE09.
The two companies have established a multi-year alliance program under which their respective scientists will work together to develop products for drug discovery.
GE Healthcarewill fund the R&D program and will be responsible for manufacturing, sales and distribution of products developed under the agreement. — Mike Tarsala
BioHealth & Healthcare Score Major Win in Russell Index Rebalance (AVII, CTIC, CEGE, IPXL, STEM, SUPG, VICL, XOMA)
This year’s Russell 3000 Index rebalancing and reconstitution may end up being the mother of all index rebalances. Because of the billions managed by Russell and because of the billions of outside funds that benchmark to the various Russell indexes, this year has many changes. All in all, there are over 400 additions and deletions to the Russell 3000 alone. The count was overwhelmingly “good” for healthcare. We screened out a few stocks under the Russell “Health Care” sector as they were not classic companies in the “BioHealth” space. But even after we screened those out, the ratio was huge by our own individual count:
- Healthcare additions were 57 and deletions were only 12… Yep a ratio of almost 5:1 for Health Care in the Russell 3000 alone.
Many of these additions or deletions were very surprising and based mostly on recent share price changes. Some of those standouts are are AVI Biopharma, Inc. (NASDAQ: AVII), Cell Therapeutics, Inc. (CTIC), Cell Genesys, Inc. (CEGE), Impax Laboratories Inc. (IPXL), StemCells Inc. (STEM), SuperGen Inc. (SUPG), Vical Inc. (VICL), and XOMA Ltd. (XOMA). We have broken out the fullist of additions and deletions below, and we started with the additions first as they may see more benefit between now and next Friday’s adjustments.
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Stem Cell Trial Positive in Batten Disease (STEM)
StemCells, Inc. (NASDAQ: STEM) has announced positive results from the first Phase I clinical trial of its proprietary HuCNS-SC product candidate of purified human neural stem cells, including demonstration of a favorable safety profile along with evidence of engraftment and long-term survival of the HuCNS-SC cells. This trial was in Neuronal ceroid lipofuscinosis, or Batten Disease, a fatal neurodegenerative disorder that afflicts infants and young children and is caused by genetic mutations. The Phase I trial was designed primarily to assess the safety of HuCNS-SC cells as a potential cell-based therapeutic. Six patients with advanced stages of infantile and late infantile neuronal ceroid lipofuscinosis were transplanted with HuCNS-SC cells and followed for 12 months.
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Persistent Merger & Acquisition Talk In Stem Cell Sector (GERN, ASTM, CYTX, NBS, STEM, KOOL, OSIR, LIFE)
It is not uncommon at all to have takeover rumors in the drug or biotech sector. That is also true in the speculative groups inside this. But we keep hearing the notorious “takeover chatter” in the stem cell sector, and particularly in Geron Corporation (NASDAQ: GERN). This is out there again, but be advised that we have seen the same notes getting passed around on multiple occasions with nothing that has ever come from it.
What we wanted to show you was where these stocks are now compared to the date of March 9, 2009 when the Obama administration was signing the executive order breaking the Bush ban on embryonic stem cell funding. Some of the other stem cell companies are as follows:
Aastrom Biosciences, Inc. (NASDAQ: ASTM) is at $0.40, and was at $0.62 when we covered it on March 9; market cap today is $69 million.
Cytori Therapeutics, Inc. (NASDAQ: CYTX) is at $2.83 and was at $3.20 when we covered it on March 9; market cap today is $97 million.
Neostem, Inc. (NBS) is at $2.16 and was at $0.69 when we covered it on March 9; market cap today is almost $17 million.
StemCells Inc. (NASDAQ: STEM) is at $1.75 and was at $2.15 when we covered it on March 9; market cap today is $182 million.
Thermogenesis Corp. (NASDAQ: KOOL) is at $0.55 today, was at $0.57 when we covered it on March 9; market cap today is $31 million.
Osiris Therapeutics, Inc. (NASDAQ: OSIR) is in a stem cell pact with Genzyme, but for adult stem cells. This is a $12.19 stock, and shares were at $17.97 when we covered it on March 9; market cap today is $399 million.
There is also a much larger company called Life Technologies Corporation (NASDAQ: LIFE) and it has the Invitrogen unit that has many efforts, including a focus on creating unique primary and stem cell models for better and more efficient drug screening.
We would describe takeover chatter in this sector as something that feels like more fiction or a stretch rather than something we’d consider as any documentary evidence. The stem cell sector offers wonderful promises and wonderful cures for many diseases and conditions. But that is also after the ethical argument has been made. Despite the positive studies and the positive indications, the group overall is still in its infancy and there are now many international patent issues in the sector because this was largely off-limits for about seven years on the new development front from new lines of stem cells.
