Healthcare Investors, Take Note of These Stem Cell Gems

Regenerative Medicine is no longer a term that the investment community is unaware of. The industry has gained substantial traction and exposure, reaching its tipping point with numerous products in late stage clinical development utilizing stem cells, whether allogeneic or autologous, to treat diseases and conditions at the cellular or molecular level. What investors may not be aware of is the robust number of applications for regenerative medicines that are no longer philosophic in nature. Years of dedicated research by a strong community of companies are culminating in an industry growing at exponential speed to target countless areas of great unmet medical need as stand-alone therapies, diagnostic tools and adjunctive treatments that can be employed with new or existing therapies to enhance efficacy and minimize side effects.

Wall Street is slowly starting to embrace regenerative medicine companies after years of a wait and see strategy amid controversy originally surrounding the source of some stem cells used in research more than a decade ago, and regulatory policy that somewhat hamstrung public acceptance. Through the concerted efforts of organizations such as the Alliance for Regenerative Medicine, the health care space has gone through a bit of a paradigm shift to see companies and other organizations standing shoulder-to-shoulder to advance this promising industry for the good of patients worldwide. This is presenting once-in-a-lifetime investment opportunities to be part of the genesis of many game-changing technologies.

The upcoming Stem Cell Meeting on the Mesa, co-hosted by ARM, the California Institute for Regenerative Medicine, and the Sanford Consortium for Regenerative Medicine, in La Jolla, California on October 14-16 is a premium opportunity for investors to learn more about individual companies as well as gain a more comprehensive knowledge of the immense potential of regenerative medicine as a whole. ARM launched just four years ago with 17 charter companies and organizations has now blossomed into more than 150 member companies and institutions representing promising therapeutics in development for a broad array of indications.

For example, Athersys (NASDAQ:ATHX), a charter member of ARM, is developing a diverse portfolio of products centered around its MultiStem cell therapy product, an off-the-shelf allogeneic regenerative medicine therapy for indications in the cardiovascular, neurological, inflammatory and immune disease areas. Athersys has partnered with Pfizer (NYSE:PFE) in a Phase 2 clinical trial of MultiStem for inflammatory bowel disease/ulcerative colitis; with top-line results expected in the first half of 2014.

MultiStem is also in separate Phase 2 clinical trials for ischemic stroke, and is preparing for a Phase 2/3 trial to address Graph versus Host Disease in bone marrow transplant patients, where it is being developed under an Orphan Drug designation from the FDA. Athersys also recently announced that it received a SBIR Fast Track grant from the National Heart, Lung and Blood Institute to support a Phase 2 clinical trial further evaluating MultiStem in acute myocardial infarction patients to build upon promising data collected in a Phase 1 trial.

NeoStem (NASDAQ:NBS) is emerging as a leader in autologous, or user-derived, cellular therapy and the introduction of new cells into tissue to prevent or treat disease or regenerate damaged or aged tissue. NeoStem is unique compared to many other small-cap companies through a diversified business model, which includes its core efforts developing therapeutics as well as its wholly-owned subsidiary, Progenitor Cell Therapy, a contract development and manufacturing organization serving the entire regenerative medicine industry.

NeoStem is developing three proprietary cell therapeutics, with AMR-001 for the treatment of cardiovascular disease being the furthest advanced. AMR-001 is a bone marrow derived product being studied in a Phase 2 clinical trial treating 160 patients for its effect in preserving heart function in patients that have suffered a severe heart attack. Patient enrollment is on schedule with the final patient expected to be enrolled this year, and top line-data to be reported in the second or third quarter of 2014. With the end of enrollment nearing and a series of grants to study indications with its VSEL technology, shares have been on the rise in the past three months, surging 80 percent.

Osiris Therapeutics (NASDAQ:OSIR) has a pipeline targeting several therapeutic areas, including inflammatory, autoimmune, orthopedic and cardiovascular conditions and diseases. The company has a bevy of clinical trials in mid-to-late stages, with Prochymal, one of the worlds first approved stem cell drugs, pacing the way and six trials for other indications currently in either late Phase 2 or Phase 3. Prochymal for Graft versus Host Disease and for Crohns Disease are being developed under Fast Track designations from the FDA.

Shares of Osiris jumped in August when the company reported top-line data from its Protocol 302 trial comparing the safety and efficacy of Grafix, a human cellular repair matrix containing living stem cells, to standard of care in patients with chronic diabetic foot ulcers. The 131-patient trial showed 62 percent of patients receiving Grafix having complete wound closure, far exceeding the 21 percent of patients receiving the standard of care, representing a record 191 percent relative improvement. Grafix demonstrated a statistical improvement in all top-line secondary endpoints as well. A fully integrated company, Osiris generates revenue by marketing Grafix and Ovation for wound and tissue repair and Cartiform for cartilage repair.

Bluebird bio (NASDAQ:BLUE) made a splash on the public regenerative medicine scene with its initial public offering in June. The company is addressing indications with serious need of new therapies, such as Parkinsons disease, Beta-thalassemia, chronic lymphocytic leukemia and adrenoleukodystrophy. bluebirds cell therapy platform is focused on developing first-in-class products that deliver high expression levels of missing or dysfunctional proteins that are scalable for commercial production and application.


Bluebird has formed a global strategic collaboration with Celgene Corp. (NASDAQ:CELG) to develop novel gene therapies in oncology, emphasizing gene therapy technology to modify a patients chimeric antigen receptor T-cells to target and destroy cancer cells. In a new potential therapy for childhood cerebral adrenoleukodystrophy by bluebird, a functional copy of the ABCD1 gene is inserted ex vivo into autologous hematopoietic stem cells with safety and therapeutic benefit being sustained for more than five years. The FDA and the European Medicines Agency have granted bluebird an Orphan Drug designation for CCALD. The company is developing Lenti-D, a next generation iteration of its gene therapy, and plans to initiate a larger scale Phase 2/3 clinical study for CCALD this year.

Taking a different approach, Organovo Holdings (NYSE MKT:ONVO) has garnered plenty of industry attention for bioprinting, designing and creating functional, scaffold-free, three-dimensional human tissues for medical research and therapeutic applications. The company collaborates with pioneering companies, such as those in the stem cell space, to develop human biological disease models in three dimensions that have the potential to accelerate the drug discovery process and at a significantly lower cost.

Among other partnerships, Organovo is working with engineering software expert Autodesk to create the worlds first 3D design software for bioprinting, which should not only improve functionality for 3D human tissue design, but potentially expand the scope of bioprinting capacities. Following some volatile trading after going public in February 2012, shares have been steadily rising as investors realize the benefits of tissue engineering to the health care industry that Organovo is bringing to the table.

This is just a glimpse of the more than 50 leading companies from the regenerative medicine industry that will be presenting at the Stem Cell Meeting on the Mesa conference next month. Collectively, the companies have more than 30 products on the market and another 100 in various phases of clinical development. The conference provides companies the opportunity to showcase their products and technologies and pursue partnering opportunities with the many pharma companies and other potential collaborators in attendance.


For the active investor, the conference is a one-of-a-kind opportunity to get an inside look at the rapidly blossoming regenerative medicine industry. The cold, hard fact is that biotechnology is teetering unbalanced on three cornerstones of pharmaceuticals, medical devices and biologics with a dire need for novel technologies to augment current therapeutics. Regenerative medicine is delivering that fourth cornerstone to reinvigorate health care and the Meeting on the Mesa is the chance for investors to put their finger on the pulse of the space.

Originally written for, a leading provider of SEC filings, real-time alerts, and in-depth analysis, with a team of experienced financial writers that cover quarterly/annual reports, insider trading/hedge fund activity, and IPOs, spin-offs, and other disclosures of interest identified from time to time within documents filed with U.S. regulatory agencies. may be compensated for its services in the form of cashed-based compensation or equity securities in the companies they write about, or a combination of the two. For a full disclaimer, click here

Investing Insights: Can Pfizer Stock Keep Going?