Companies
NeoStem’s CEO, Dr. Robin L. Smith, Letter to Shareholders
NEW YORK , Jan. 3, 2012 /PRNewswire/ –
Dear NeoStem Shareholders,
We would like to take a moment to both look back at 2011 – a transformative year for NeoStem (NYSE Amex: NBS) – and to look ahead to near term catalysts that we expect to move the company forward in 2012 and beyond.
We have closed two acquisitions – Progenitor Cell Therapy, LLC (“PCT”) and Amorcyte, LLC (“Amorcyte”).
We believe our therapeutic product development team is very close to accomplishing its aggressive goal of getting a first patient enrolled in our AMR-001 Phase 2 clinical trial for the treatment of AMI with the clinical sites beginning to open. This brings us closer to achieving our goal of enrollment of the targeted 160 patients in the study over the next year or so with first data follow-up six months after the last patient is enrolled (roughly mid-2013).
Our cell therapy manufacturing business is growing and client satisfaction confirms our belief and excitement that we have unique skills and people (expertise, quality and work ethic) to serve as a platform to be a global leader in the cell therapy space.
We raised $16.5 million in gross proceeds in 2011 for working capital, including research and development of our cell therapeutic candidates.
We received awards of over $1.7 million in Department of Defense funding for development of our VSELTM Technology to treat osteoporosis and $245,176 from the National Institutes of Health (NIH) with Excell Therapeutics to progress our T regulatory program in Lupus.
We co-hosted a spectacular international conference in partnership with the Vatican’s Pontifical Council for Culture on Adult Stem Cells: Science and the Future of Man and Culture, moving forward the public discussion of adult stem cells and adult stem cell research.
Our cord blood banking enrollment more than doubled over the previous year.
We have been marketing our ownership in Suzhou Erye Pharmaceutical Co. Ltd. subsidiary for possible sale.
We have positioned our intellectual property portfolio to expand beyond the current indications and give us a strong position in the cell therapy arena.
We continue to make great headway in integrating IT systems, legal, finance, and marketing for our multiple entities to achieve cost savings and maximize efficiencies.
NeoStem gained a significant pharmaceutical partnership with Becton Dickinson through our co-ownership of Athelos, Inc. (80% NeoStem, 20% BD). We are actively pursuing additional strategic relationships with major pharmaceutical and biotechnology companies in 2012.
We look forward to keeping you updated and encourage your questions via the contact information below. I also encourage you to learn more by visiting our company websites, www.neostem.com, www.amorcyte.com, and www.progenitorcelltherapy.com, our social media outlets, and our company blog at thechairmansblog.com/robin-l-smith. Thank you for your continued support of NeoStem and our ongoing transformation.
Sincerely,
Dr. Robin L. Smith
Chairman and CEO
About NeoStem, Inc.
NeoStem, Inc. (“NeoStem”) is a leader in the development and manufacture of cell therapies. NeoStem has a strategic combination of revenues, including that which is derived from the contract manufacturing services performed by Progenitor Cell Therapy, LLC, a NeoStem company. That manufacturing base is one of the few cGMP facilities available for contracting in the burgeoning cell therapy industry, and it is the combination of PCT’s core expertise in manufacturing and NeoStem’s extensive research capabilities that positions the company as a leader in cell therapy development. Amorcyte, Inc., also a NeoStem company, is developing a cell therapy for the treatment of cardiovascular disease. Amorcyte’s lead compound, AMR-001, represents NeoStem’s most clinically advanced therapeutic, poised to commence enrollment of patients in a Phase 2 trial for the preservation of heart function after a heart attack. Athelos Corporation, also a NeoStem company, is developing a T-cell therapy for a range of autoimmune conditions with our partner Becton-Dickinson. NeoStem’s pre-clinical assets include its VSEL™ Technology platform for regenerative medicine, which NeoStem believes is an endogenous pluripotent non-embryonic cell that has the potential to change the paradigm of cell therapy as we know it today.
Open Letter to Shareholders from Pluristem Therapeutics CEO Zami Aberman
Dear Shareholders,
As we close in on the end of the calendar year, it is important to look back upon all that we at Pluristem have accomplished over the last 12 months, but it is even more crucial to focus the majority of our energies on the challenges and opportunities awaiting Pluristem Therapeutics in 2012 and beyond.
Here is a recap of our recent achievements, which have put the company on course for a very successful future.
Phase I CLI Study Data
In early November we announced positive 12-month data from our Phase I open-label, dose-escalation clinical trials, conducted under protocols approved by the Food & Drug Administration (FDA) in the U.S. and the Paul-Ehrlich-Institute (PEI) in Germany. PLX-PAD cells met all the clinical studies’ protocol endpoints, demonstrating a safe immunologic profile at all dosage levels and suggesting that PLX-PAD is potentially effective in treating patients suffering from Critical Limb Ischemia (CLI). The results demonstrated no significant safety issues and proved that PLX-PAD cells can be safely given IM to patients without matching, even when the patients are dosed twice from the same placental source. These results, together with the great effort we invest in our manufacturing and operating capabilities, will provide us significant advantages when marketing PLX in the future.
United Therapeutics Update
As you know, during the recently-closed first quarter of our fiscal year we recognized our first revenues from our exclusive out-license agreement with United Therapeutics Corporation (NasdaqGS: UTHR) for the use of Pluristem’s PLacental eXpanded (PLX) cells to develop and commercialize a cell-based product for the treatment of Pulmonary Hypertension (PH). We are pleased with the collaboration to-date and are working closely with United Therapeutics on ensuring that we hit the upcoming key milestones in our quest to develop and receive regulatory approval for a product treating PH.
Manufacturing Facility Construction
One of the cornerstones of Pluristem’s strategy is to manufacture our PLacental eXpanded (PLX) based products and then enter into strategic partnerships with large pharmaceutical companies around the world for marketing and distribution, as we had done with United Therapeutics.
To that end, we reported in October that we had signed a deal with Biopharmax Group Ltd., a company specializing in the design and construction of biotechnological and pharmaceutical facilities, to handle the build-out of a new cGMP manufacturing facility in Israel that will meet regulatory authorities’ requirements. We expect the facility to be completed by the end of the 2012 calendar year. This facility will allow us to significantly scale up our manufacturing capacity and will enable us to produce PLX cells for the treatment of over 150,000 patients annually, meeting the expected near-term demand for our products.
Financial Status
In our recently released 10-Q quarterly report, we were pleased to record our first revenues. These revenues are the result of the licensing deal with United Therapeutics and they are a meaningful validation of our platform technology. We have a strong balance sheet with over $45 million in cash and cash equivalent and short term bank deposits. We have successfully maintained a controlled and modest burn rate, despite making significant progress. Although we expect our burn rate to increase in the coming year, resulting from initiation of advanced clinical trials, no significant increase in manufacturing expenses is foreseen, due to our efficient production process. This advantage will become more and more noticeable as we progress with our clinical trials.
Our Potential Impact
Before I close, I wanted to outline for you why our work at Pluristem is so important. Of course, we work hard on increasing shareholder value, but there is a different kind of impact to be felt if we are able to successfully develop our product candidates. The first therapy that Pluristem is focusing on is for CLI, where in the United States (U.S.) alone, patients undergo more than 160,000 major limb amputations per year. Current data suggest that approximately 20% of CLI patients will die within the first 6 to12 months after disease onset and many others will require amputation within six months of diagnosis. Approximately half of all diabetes patients diagnosed with CLI will undergo a major amputation following diagnosis, and many will require a second amputation within 3 to 5 years.
The overall U.S. hospitalization and amputation rate associated with CLI has increased since 1985, suggesting a growing incidence of CLI and a substantial unmet medical need to improve outcomes for these patients beyond conventional treatments.
All this adds up to a total annual cost of between $10 billion and $20 billion for treating U.S. CLI patients. Economic modeling indicates that even a 25% reduction in amputations has the potential to create annual savings for the U.S. health care system of up to $3 billion. This is just a conservative model and for one therapy only.
In conclusion, Pluristem has an opportunity to impact society, and it is important for our shareholders to understand that through the development of our CLI product, we are working towards fulfilling our mission of bringing significant changes to the lives of patients around the world.
We thank you for your continued support, and we look forward to providing you with more exciting news in the coming months on both the business and the technology aspects of Pluristem.
Thank you very much,
Zami Aberman
Chairman and CEO
About Pluristem Therapeutics
Pluristem Therapeutics Inc. (NasdaqCM: PSTI; TASE: PLTR) is a leading developer of standardized cell therapy products for the treatment of life threatening diseases. The company’s patented PLX (PLacentaleXpanded) cells drug delivery platform releases a cocktail of therapeutic proteins in response to a host of local and systemic inflammatory diseases. PLX cells are grown using the company’s proprietary 3D micro-environmental technology and are an off-the-shelf product that requires no tissue matching prior to administration. Data from two phase I studies indicate that Pluristem’s first PLX product candidate, PLX-PAD, is safe and potentially effective for the treatment of end stage peripheral artery disease. Pluristem’s pre-clinical animal models have demonstrated PLX cells are also potentially effective in nerve pain and muscle damage, when administered locally, and in inflammatory bowel disease, MS and stroke, when administered systemically.
Pluristem has a strong patent portfolio, GMP certified manufacturing and research facilities, strategic relationships with major research institutions and a seasoned management team.
For more information visit www.pluristem.com, or follow us on Twitter @Pluristem, the contents of which are not part of this press release.
3 Contenders To Be The First Profitable Stem Cell Company
By Ryan Pollack, SeekingAlpha.com
Every day is a day closer to a disease being treated, or even cured, by a stem cell therapy. The medical field is in a transition with clinical trials that are ongoing using adult, embryonic, and other forms of stem cells derived and differentiated using various laboratory techniques. These three companies have the potential to be the first self-sustaining, profitable regenerative medical companies.
See complete article at SeekingAlpha.com regarding Advanced Cell Technology (ACTC), Geron (GERN), and International Stem Cell Corporation (ISCO)
The Promising Cells Presented by International Stem Cell Corporation
Ken Aldrich of International Stem Cell Corporation (ISCO.OB) speaks on the latest research involving stem cell technology utilizing unfertilized eggs, as a basis for cell generation with a goal toward organ transplants which are immune to rejection by the body.
See complete article at the International Stem Cell Corporation blog
FDA Approves Fibrocell’s Stem Cell Wrinkle Smoother
Fibrocell Science Inc. (FCSC) won U.S. approval for an injectible wrinkle treatment that uses a patient’s own stem cells to restore elasticity to the skin. The Food and Drug Administration cleared azficel-T, the company said today in a statement. Fibrocell plans to sell the drug under the name LaViv. Shannon Pettypiece reports on Bloomberg Television’s “Fast Forward.” (Source: Bloomberg)
See Fibrocell Science Website for more information.
Stem cells from body fat promising
A San Diego-based company is breaking new ground in the field of regenerative medicine with a system that uses patients’ own body fat to generate stem cells and repair tissue and organs. Ben Gruber reports.
Neuralstem Announces First Patient Treated in ALS Stem Cell Trial
ROCKVILLE, Md., Jan. 21 /PRNewswire-FirstCall/ — Neuralstem, Inc. (NYSE Amex: CUR) announced that the first ALS patient was treated with its spinal cord stem cells yesterday at the Emory ALS Center at Emory University, in Atlanta, GA. A total of up to 18 patients is planned to be treated in this first U.S. clinical trial to evaluate human neural stem cells for the treatment of ALS (Amyotrophic Lateral Sclerosis, or Lou Gehrig’s disease). ALS affects roughly 30,000 people in the U.S., with about 5,600 new diagnoses per year, according to the ALS Association.
“The treatment of the first patient with our spinal cord stem cells represents a significant milestone for the Company,” said Neuralstem President and CEO, Richard Garr.
Click here for complete article.
International Stem Cell Corporation and Absorption Systems Announce Potential Stem Cell Alternative to Live Animal Testing for Corneal Damage
OCEANSIDE, Calif. & EXTON, Pa.–(BUSINESS WIRE)–International Stem Cell Corporation (OTCBB:ISCO)), the first company to perfect a method of creating human “parthenogenetic” stem cells from unfertilized eggs, announces positive results of its collaboration with Absorption Systems to study ISCO’s stem-cell-derived human corneal tissue as an alternative to live animals for drug testing. Initial results using non-animal or “in vitro” tests indicate an excellent correlation between the rates at which drugs pass through ISCO’s lab-grown corneal tissue and rabbit corneal tissue. The results offer great promise for reducing the use of living animals for eye safety testing. ISCO’s human corneal tissue is created from parthenogenetic stem cells in the laboratories of Lifeline Cell Technology (Walkersville, MD), ISCO’s wholly-owned subsidiary.
See Intlstemcell.blogspot.com for complete article.
Stem cell trial for ALS treatment gets FDA OK
From ChicagoTribune.com
A University of Michigan neurologist is the principal investigator for the first human clinical trial of a stem cell treatment for amyotrophic lateral sclerosis.
Dr. Eva Feldman has worked with a team of neurologists to develop the protocol for injecting neural stem into patients’ spinal cords. The cells are patented by Neuralstem Inc., a Rockville, Md.-based biotech company.
The U.S. Food and Drug Administration has approved Neuralstem’s plan to test the safety of the treatment for the fatal, untreatable neurodegenerative disorder commonly called Lou Gehrig’s Disease.
The initial phase to determine the safety of the treatment is to take place at Emory University in Atlanta.
Next Big Market: Adult Stem Cell Drugs and Therapies
Written by M.E.Garza, Biomedreports.com
According to a new report in Fortune Magazine, marketable therapies emerging from work in the (less controversial) adult stem space could be the next multi-billion dollar market.
Research on adult stem cells has generated a great deal of excitement. Adult stem cells have already been used successfully with patients: to treat cartilage defects in children; restore vision to patients who were legally blind; relieve systemic lupus, multiple sclerosis, and rheumatoid arthritis; and to serve as an aid in numerous cancer treatments.
Scientists have found adult stem cells in many more tissues than they once thought possible. This finding has led researchers and clinicians to ask whether adult stem cells could be used for transplants. In fact, adult hematopoietic, or blood-forming, stem cells from bone marrow have been used in transplants for 40 years. Scientists now have evidence that stem cells exist in the brain and the heart. If the differentiation of adult stem cells can be controlled in the laboratory, these cells may become the basis of transplantation-based therapies. These Adult stem cells can be harvested from many areas of the body, including the bone marrow, fat and peripheral blood. Once the cells have been harvested, they are sent to the lab where they are purified and assessed for quality before being reintroduced back in the patient. Since the stem cells come from the patient there is no possibility for rejection and they are used in transplants to treat diseases, such as cancers like leukemia.
According to various studies, stem cells isolated from a patient (i.e. from the bone marrow or fat) have the ability to become different cell types (i.e. nerve cells, liver cells, heart cells and cartilage cells). Studies have also shown that these are capable of “homing in” on and repairing damaged tissue. Researchers feel they are far closer to commercializing drugs based on adult stem cells than any product based on embryonic stem cells. In fact, many clinics outside of North America already tout stem cell based treatments to treat chronic diseases for which there are inadequate standard therapies. These clinics currently accept patients with Diabetes Type 2, Autoimmune Diseases, Multiple Sclerosis, Degenerative Joint Disease, Autoimmune Diseases as well as Rheumatoid Arthritis and Osteoarthritis. Unfortunately, patients seeking those treatments in other countries most often run the risk of parting with their money and being disappointed with the results.
Back in the states, Robin Young, a medical industry analyst from RRY Publications, estimates that gross sales of adult cellular therapies will be well over $100 million this year. By 2018, he says stem cell therapy revenues could grow to $8.2 billion.
“Adult derived cells are the ones that have been studied for the past 10 to 15 years and are ready for prime time,” says Debra Grega, the executive director of the Center for Stem Cell and Regenerative Medicine at Case Western Reserve University. “Large pharmaceutical companies are now wanting to get into the adult stem cell therapeutic area. That indicates to me that there is enough safety and enough efficacy that they are willing to put money in.”
Pharmaceutical giant Pfizer (NYSE:PFE) announced in November that it would invest up to $100 million in regenerative research, which would include both adult and embryonic stem cell research, over a three to five year period.
The overall stem cell market, however, is still quite small. The California-based outfit Geron (NASDAQ:GERN) dominates the embryonic stem cell market, and is perhaps 10 years away from commercializing a spinal cord treatment based on its research.
The frontrunner in the adult stem cell space, according to Forbes, is Osiris Therapeutics, Inc.(NASDAQ:OSIR)- currently trading at $14.20 per share. Genzyme Corp. (NASDAQ:GENZ) has signed a partnership alliance with Osiris Therapeutics to develop two late-stage adult stem cell treatments — Prochymal and Chondrogen — thought to be useful to treat a variety of diseases by controlling inflammation, promoting growth of new tissue and preventing scars. The deal will pay Osiris $130 million upfront ($75 million initially and the difference to be paid on July 1, 2009). Assuming the drugs reach the marketplace, Genzyme will pay up to $1.25 billion in development, regulatory and sales milestone payments.
Osiris is focused on developing and marketing products to treat medical conditions in the inflammatory, orthopedic, and cardiovascular areas. Their principal biologic drug candidate, Prochymal, is being evaluated in Phase III clinical trials for three indications, including acute and steroid refractory Graft versus Host Disease (GvHD), Crohn’s disease and for the repair of gastrointestinal injury resulting from radiation exposure, and is the only stem cell therapeutic granted both Orphan Drug and Fast Track status by the United States Food and Drug Administration (FDA). Prochymal is also being developed for the repair of heart tissue following a heart attack, for protection of pancreatic islet cells in patients with type I diabetes, and for the treatment of Chronic Obstructive Pulmonary Disease (COPD). The FDA could approve within a year which fights a painful illness called “graft-versus-host disease” which afflicts transplant recipients. If they succeed, Osiris would be the first company to gain approval for a stem cell drug. Osiris will commercialize both drugs in the U.S. and Canada, and Genzyme will sell the drugs in all other countries.
Investors should be aware that there are only a limited number of stocks which are pure plays or semi-pure plays in the stem cell industry. Below are some of the companies working in the adult stem cell medicine space:
StemCells, Inc.(NASDAQ:STEM) – a company is engaged in the discovery and development of cell-based therapeutics to treat damage to, or degeneration of, major organ systems. Currently trading at $1.60 with a market cap of $164.09M.
Cytori Therapeutics Inc. (NASDAQ:CYTX) which develops, manufactures, and sells medical technologies to enable the practice of regenerative medicine. The Company’s commercial activities are focused on cosmetic and reconstructive surgery in Europe and Asia-Pacific, and stem and regenerative cell banking (cell preservation) in worldwide. Its product pipeline includes the development of new treatments for cardiovascular disease, spinal disc degeneration, gastrointestinal disorders, liver and renal disease and pelvic health conditions. They currently trade at $3.55 with a market cap of $121.02M.
Aastrom Biosciences, Inc.(NASDAQ:ASTM) engaged in the development of autologous cell products for the repair or regeneration of human tissue. The Company’s tissue repair cell (TRC) technology involves the use of a patient’s own cells to manufacture products to treat a range of chronic diseases and serious injuries affecting vascular, bone, cardiac and neural tissues. Aastrom’s TRC-based products contain increased numbers of stem and early progenitor cells, produced from a small amount of bone marrow collected from the patient. Late last month, the company made headlines after temporarily suspending enrollment and patient treatment in its U.S. Phase II IMPACT-DCM clinical trial following a report that a patient died at home after being released from the hospital following treatment in the trial. The stock trades at $0.36 pps with a market cap of $58.44M.
ThermoGenesis Corp.(NASDAQ:KOOL) designs, manufactures and markets automated and semi-automated devices and single-use processing disposables that enable hospitals and blood banks to manufacture a therapeutic dose of stem cells, wound healing proteins or growth factors from a single unit of cord blood or the patient’s own blood in less than one hour. They currently trade at just under $0.70 and have a market cap of $37.02M.
Opexa Therapeutics, Inc. (Nasdaq:OPXA) is a biopharmaceutical company developing autologous cellular therapies with the potential to treat major illnesses, including multiple sclerosis (MS) and diabetes. The Company has a global license from Baylor College of Medicine (or Baylor) to an individualized T-cell therapeutic vaccine, Tovaxin, which is in clinical development for the treatment of MS. MS is the result of a person’s own T-cells attacking the myelin sheath that coats the nerve cells of the central nervous system (CNS). Shares currently trade under $0.60 with a market cap of $7.29M.
At BioMedReports, we will continue to explore this potentially bountiful market within the healthcare sector in coming weeks, as it sounds like investors would greatly benefit from insight into the current marketplace.
“From a Wall Street perspective, adult stem cells are a much better investment,” said Stephen Dunn of Dawson James Securities.”
