Cell Therapy 2015: With Maturity Comes Promise

Under agreement with StreetWise Reports, I'm pleased to share their recent coverage of several companies in the cell therapy sector.

Original Source: Tracy Salcedo-Chourré of The Life Sciences Report (12/23/2014)
http://www.thelifesciencesreport.com/pub/na/cell-therapy-2015-with-maturity-comes-promise

A number of new and innovative medical paradigms are being explored by companies in the life sciences sector, with one of the front-runners being cell therapy—a disruptive technology that has, in the past few years, progressed out of preclinical studies and into clinical development. To learn more about the transformative promise of regenerative medicine, and what investors might expect from companies working in the sector in 2015, The Life Sciences Report asked analysts Jason Kolbert of Maxim Group and Dr. Christopher James of Brinson Patrick to discuss the advances that could generate robust returns on investment in coming years. 


Cell Therapy in a Nutshell
Cell therapies have been gaining momentum in the life sciences for more than half a century. The first cell therapies, bone marrow transplants, were performed in the 1960s, and since then the idea of using healthy cells to replace or regenerate diseased cells has expanded into a number of indications, from cancer to cardiac disease to cosmetic therapy. Today, a number of companies are engaged in both clinical and preclinical trials, and there is some consensus that cell therapies will be "mainstream" in the near future.

The new year could prove a bellwether for regenerative medicine. The sheer volume of companies, universities and financial institutions conducting or backing research and development in the field attests to its potential: The Alliance for Regenerative Medicine(ARM), an advocacy group working with legislators, the investment community and regulatory agencies to promote development of cell therapy technologies, has more than 170 members working in the field.

On the horizon: Australian cell-therapy company Mesoblast Ltd. (MSB:ASE; MBLTY:OTCPK) is positioning itself for expedited approval of several of its allogeneic (derived from the same species) cell therapy products in Japan in 2015, following passage of legislation in that country that expedites approval. And though it hit a significant speed bump last month, Dendreon Corp.'s (DNDN:NASDAQ) Provenge (sipuleucel-T), an autologous (derived from and returned to the same patient) cell therapy for metastatic castrate-resistant prostate cancer, was the first FDA-approved cell therapy, and has helped pave a path forward for additional approvals in the U.S.

To get a handle on what investors can expect, and an idea of which companies to keep an eye on, The Life Sciences Report turned to Dr. Christopher James, managing director and senior equity research analyst with Brinson Patrick Securities Corp. and Jason Kolbert, managing director, senior biotechnology analyst and head of healthcare research with Maxim Group. Here's what they had to say about what 2015 might bring in both the sector and for certain companies working in the space.

Great Expectations
Asked which specific indications could be most responsive to cell therapies, and about companies working in those therapy areas, Kolbert provided several names.

The next "major event" in the sector will be results in a Phase 2 trial in ischemic stroke being conducted by Athersys Inc. (ATHX:NASDAQ), with results expected in Q1/2015, the analyst said. A number of other trials are also slated to start in 2015, including NeoStem Inc.'s (NBS:NASDAQ) Phase 3 trial in acute myocardial infarction and Cesca Therapeutics Inc.'s (KOOL:NASDAQ) pivotal trial in critical limb ischemia.

Kolbert is also looking forward to two trials involving Mesoblast's own stem cell product, as well as results from Prochymal (remestemcel-L, human mesenchymal stem cells for intravenous infusion, acquired from Osiris Therapeutics Inc. [OSIR:NASDAQ]). The Mesoblast product is expected to enter a pivotal trial in back pain, and the company is currently enrolling a global trial in congestive heart failure with partner Teva Pharmaceutical Industries Ltd. (TEVA:NASDAQ). The Osiris product is expected to be redeveloped in the U.S. for graft-versus-host disease (GvHD). Prochymal has been conditionally approved in Canada and New Zealand for GvHD in children. In addition, Mesoblast is expected to release data in a Phase 3 Prochymal trial in Crohn's disease.

James believes the area where cell therapy—cell regeneration—could have the biggest impact is also one of the riskiest—in the central nervous system (CNS), treating disorders or injury of the brain or spinal cord. James sees applications in multiple sclerosis (MS) and spinal cord injury, to regenerate neuronal function, and in Parkinson's disease, to address the death of dopaminergic cells. He also likes ocular applications, for treatment of such conditions as age-related macular degeneration (AMD).

James has two cell therapy companies under coverage. Opexa Therapeutics Inc. (OPXA:NASDAQ), with its Tcelna platform technology, is developing therapies in both CNS and the eye. A Phase 2 trial in secondary progressive MS is expected to read out in mid-2016. The company is also developing OPX-212, an autologous T-cell immunotherapy, to treat neuromyelitis optica, an autoimmune condition that leads to blindness.

The second company, StemCells Inc. (STEM:NASDAQ), is in a Phase 2 trial in the treatment of cervical spinal cord injury. On Dec. 18, the company announced that it had transplanted its HuCNS-SC (purified human neural stem cells) into its first patient; James expects final data from that trial in late 2015. Results from the company's Phase 1/2 trial in dry AMD, are expected in Q1/2015—perhaps as early as January.
StemCells' Phase 2 trial in cervical spinal cord injury points out what James sees as one of the regulatory hurdles cell therapies will have to overcome as they progress through the U.S. Food and Drug Administration's (FDA's) approval process. The trial is a randomized, controlled, single-blind study, as opposed to the FDA standard of double-blind pivotal studies.

To treat central nervous system disease or injury, companies must inject cells into a patient, a "surgical" intervention, James explained. But clinicians "can't do a sham surgery on the brain or on the spine. . .that's not ethical." So companies like StemCells must work with the FDA to develop pivotal trial designs that are "less stringent" than the double-blind model.

The Bigger Picture
Kolbert is enthusiastic about the prospects for cell therapies in 2015, and doesn't see much in the way of headwinds. "Cell therapy ultimately lowers the cost of treating disease, and at a time when there is an outcry over high-priced therapies, cell therapy offers promise to lower the cost of treating disease. In terms of politics, the cell therapies we are focusing on are typically not controversial (using adult stem cells, not cells from embryos). As such, we don’t see any controversy. The big change in the landscape now is the acceptance that cell therapy is 'safe.'"

On the plus side of the spectrum is oncology. Using cell therapy in cancer immunotherapy "holds great promise," Kolbert continued. "The ability to rev up the immune system with checkpoint inhibitors, and couple that with therapeutics, like the therapies being developed by ImmunoCellular Therapeutics Ltd. (IMUC:OTCBB) and Agenus Inc. (AGEN:NASDAQ) targeting glioblastoma, and the work that OncoSec Medical Inc. (ONCS:OTCBB), Inovio Pharmaceuticals Inc. (INO:NYSE.MKT) and NeoStem are doing in melanoma, is exciting."

James and Kolbert agree that recent elections, in which Republicans gained control of both houses of Congress, shouldn't affect ongoing work in the cell therapy field. "They have bigger issues to deal with," James commented.

However, James believes cell therapy companies may suffer from an "overhang" following the recent bankruptcy of Dendreon Corp. And Kolbert cautions investors that second- and third-generation technologies always follow. Such is the case with Bavarian Nordic's (BAVA:OMX) Prostvac. Prostvac has the potential to be equivalent to, or even superior to, Provenge, but at a fraction of the cost. The Phase 3 Prostvac trial reports data next year.

Another concern for investors, according to James, is the question of whether cell therapy companies can manufacture their products on a commercial scale. But the analyst believes that concern can be addressed, and cites the StemCells model, in which, according to the company, "[c]ryopreserved lines of donor-derived cells can be reproduced at commercial scale as 'stem cells in a bottle,' then distributed for patient doses as needed, much like an off-the-shelf pharmaceutical product."

Christopher James, M.D., is a managing director and senior equity research analyst focusing on life sciences companies with strong growth potential and developing novel agents for serious diseases including cancer and infectious, neurological, inflammatory, metabolic and cardiovascular diseases. He was previously a senior equity research analyst at Rodman & Renshaw and MLV & Co. Prior to joining Brinson Patrick, Dr. James was chief medical officer and senior vice president of medical affairs at Retrophin, a biotechnology company focused on developing therapeutics for rare and devastating diseases. While at Retrophin, he played a pivotal role in the study design and subsequent acceptance of an investigational new drug application with the cardiorenal division of the FDA to initiate a Phase 2 clinical study in a rare kidney disease called focal segmental glomerulosclerosis. Dr. James has prior buyside experience working at Trivium Capital Management and MSMB Capital Management. Dr. James trained in neurological surgery at Cornell-New York Hospital and Memorial Sloan Kettering Cancer Center. He obtained a medical degree from Yale University School of Medicine and a bachelor of science in biology from Cornell University.

Jason Kolbert has worked extensively in the healthcare sector as product manager for a leading pharmaceutical company, as a fund manager and as an equity analyst. Prior to joining Maxim Group, where he is head of healthcare research, senior managing director and biotechnology analyst, Kolbert spent seven years at Susquehanna International Group, where he managed a healthcare fund and founded SIG's biotechnology team. Previously, Kolbert served as the healthcare strategist for Salomon Smith Barney. He is often quoted in the media and is a sought-out expert in the biotechnology field. Prior to beginning his Wall Street career, Kolbert served as a product manager for Schering-Plough in Osaka, Japan. He received a bachelor's degree in chemistry from State University of New York, New Paltz, and a master's degree in business administration from the University of New Haven.

Want to read more Life Sciences Report interviews like this? Sign up for our free e-newsletter, and you'll learn when new articles have been published. To see recent interviews with industry analysts and commentators, visit our Streetwise Interviews page.

DISCLOSURE:
1) Tracy Salcedo-Chourre compiled this article for Streetwise Reports LLC, publisher of The Gold Report, The Energy Report, The Life Sciences Report and The Mining Report, and provides services to Streetwise Reports as an employee. She owns, or her family owns, shares of the following companies mentioned in this interview: None.
2) The following companies mentioned in the interview are sponsors of Streetwise Reports: NeoStem Inc., Athersys Inc., StemCells Inc., Inovio Pharmaceuticals Inc. Mesoblast Ltd. is not affiliated with Streetwise Reports. The companies mentioned in this interview were not involved in any aspect of the interview preparation or post-interview editing so the expert could speak independently about the sector. Streetwise Reports does not accept stock in exchange for its services.
3) Christopher James: I own, or my family owns, shares of the following companies mentioned in this interview: None. I personally am, or my family is, paid by the following companies mentioned in this interview: None. My company has a financial relationship with the following companies mentioned in this interview: Opexa Therapeutics. I was not paid by Streetwise Reports for participating in this interview. Comments and opinions expressed are my own comments and opinions. I determined and had final say over which companies would be included in the interview based on my research, understanding of the sector and interview theme. I had the opportunity to review the interview for accuracy as of the date of the interview and am responsible for the content of the interview.
4) Jason Kolbert: I own, or my family owns, shares of the following companies mentioned in this interview: None. I personally am, or my family is, paid by the following companies mentioned in this interview: None. My company has a financial relationship with the following companies mentioned in this interview: None. I was not paid by Streetwise Reports for participating in this interview. Comments and opinions expressed are my own comments and opinions. I determined and had final say over which companies would be included in the interview based on my research, understanding of the sector and interview theme. I had the opportunity to review the interview for accuracy as of the date of the interview and am responsible for the content of the interview.
4) Interviews are edited for clarity. Streetwise Reports does not make editorial comments or change experts' statements without their consent.
5) The interview does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal disclaimer.
6) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their families are prohibited from making purchases and/or sales of those securities in the open market or otherwise during the up-to-four-week interval from the time of the interview until after it publishes.

Streetwise – The Life Sciences Report is Copyright © 2014 by Streetwise Reports LLC. All rights are reserved. Streetwise Reports LLC hereby grants an unrestricted license to use or disseminate this copyrighted material (i) only in whole (and always including this disclaimer), but (ii) never in part..
Streetwise Reports LLC does not guarantee the accuracy or thoroughness of the information reported.
Streetwise Reports LLC receives a fee from companies that are listed on the home page in the In This Issue section. Their sponsor pages may be considered advertising for the purposes of 18 U.S.C. 1734.
Participating companies provide the logos used in The Life Sciences Report. These logos are trademarks and are the property of the individual companies.
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Reprint from LSR: How RepliCel Is Harnessing the Awesome Power of Cell Therapy

I've interviewed a number of executives from several cell therapy companies recently and intend to post my interview of them in the coming weeks but in the interim, StreetWise Reports published this interview of me which I'm pleased to share.

Original Source: George S. Mack of The Life Sciences Report (11/07/2014) 

Regenerative medicine and cell therapies hold possibilities for achieving near miracles in a multitude of indications, from life-saving treatments to aesthetic applications. RepliCel Life Sciences Inc. (RP:TSX.V; REPCF:OTCQB) is tackling a mix of medical and cosmetic issues that include hair regeneration, repair of painful and debilitating tendon injuries and rejuvenation of damaged skin. In this interview with The Life Sciences Report, R. Lee Buckler, the company's new vice president of business and corporate development, discusses his firm's innovative technology platform and the upcoming milestones that could affect its shares.

The Life Sciences Report: Lee, you've made a recent career change and are now an executive at RepliCel. Tell us about that.

R. Lee Buckler: As of Oct. 1, I have been appointed vice president of business and corporate development for RepliCel Life Sciences Inc., which is engaged in development of cell-based regenerative medicine therapeutics in Canada, Europe and Japan through our licensing partner, Shiseido Company Ltd. (4911:TSE). I found RepliCel to be a very interesting company poised to go on an exciting run, and that enticed me to join the team.

TLSR: Prior to your work with RepliCel, what experience had you had in the cell therapy industry?

RLB: In 2000, I left the practice of law to join Allen Eaves in the Stem Cell Technologies group of companies, where I ran a company called Malachite Management Inc. In 2006, I was recruited by Progenitor Cell Therapy to run its business development, marketing, communication and sales before the NeoStem acquisition.

In 2008, I founded my own consulting firm, called the Cell Therapy Group (CTG), which focused exclusively on the cell therapy industry. In the early days, we did some communications work for clients, but for the bulk of my tenure with CTG, we were involved in a wide range of planning and business development work. Some of it was transactional, but other aspects included market and competitive intelligence, building strategies, identifying partners, targeting partners, engaging in partnership discussions on behalf of clients and the like. I also worked with fund managers and investors through education, namely technology and platform explanations.

Over the past year, I worked as a consultant and on the board of directors positioning TheraVitae Inc. (private) for a merger with a company listed on the Toronto Venture Exchange. The merger is expected to complete in early November, and the company will be renamed Hemostemix Inc. I helped TheraVitae raise several million dollars as part of that process. Being on the road giving presentations to prospective investors is a new skill set for me, but I've found I really enjoy this side of the business.

TLSR: You are an attorney by training, but I see from your curriculum vitae that you did a couple of stints as a medical laboratory technician while you were still in law school. Is that what led you to the life sciences field?

RLB: Yes. I always joke with people that I didn't get to the cell therapy/regenerative medicine industry through education—I got here more by osmosis. I was not a silver spoon kid; I had to work my way through school. So while I was studying to be an attorney, I ended up working in the lab of a leading cardiovascular investigator, who was involved in some clinical trials at the time. I was mainly doing grunt work, but it exposed me to an environment where people were extremely dedicated to their sciences and to doing something novel. I was exposed to the excitement that builds when people truly believe what they're doing could revolutionize the way people are treated.

I've always felt a little bit like an outsider in an industry of people who belong here. While that may feel uncomfortable from time to time, it also affords me a unique perspective that others don't have. While others in the industry tend to focus on vertical specialties, I've come to specialize in a macro view of this industry. My focus has been very horizontal, which gives me a perspective of the industry that not many people are able to see.

TLSR: What kind of work has RepliCel been doing in the cell therapy field?

RLB: When CEO and President David Hall took over the company in 2011, it was built around hair regeneration. That is still an important part of our portfolio; however, he had a vision for broadening the technology and building a platform, which the company has now executed.

We are preparing to launch a very significant Phase 2 trial using our RCH-01 (dermal sheath cup [DSC] cells) for hair regeneration. This is a cellular injection—a cell transplant rather than a hair transplant—and is an important evolution because hair transplant is limited by three very significant factors. First, when transplanting hair follicles from one location on your scalp to another, there are only so many follicles available to harvest. With a cell transplant, there is no limit to the number of cells we can grow to use in regenerating poorly functioning hair follicles. Second, hair transplantation only achieves a satisfactory result when performed by a gifted surgeon, of which there are few. A simple cell injection takes the art out of the procedure—particularly when combined with our proprietary injection device designed to optimally deliver the cells into the scalp. Finally, hair transplantation is not an option women find attractive for a number of reasons, and a significant population of women suffer from hair loss.

TLSR: How is RepliCel working to ensure this therapy will be effective in both the short and long term? What prevents the dermal sheath cells from ceasing to grow hair once they are in the locale where the original follicles quit producing hair?

RLB: The cells we are using to address pattern baldness (androgenetic alopecia) are taken from a cell population found at the base of the hair follicle. These DSC cells are used to produce our RCH-01 product. Research has demonstrated that these cells are responsible for the reorganization of the hair follicle, which is a mini-organ that organizes upon an unknown signal. Our research leads us to believe this cell population is responsible for hair regeneration.

We source our particular cells from hair follicles isolated from the back of the scalp, between the ears, because most balding people retain this area of hair. This hair is insensitive to the androgen hormone (DHT), which causes hair loss, making these hair follicles prime candidates for our hair regeneration product.

As to the question of whether this will be a durable response—how long the hair will last—this is one of several questions both we and Shiseido are targeting in our respective upcoming pattern baldness trials. We're designing this next phase to look at dosing. We're also looking at frequency of treatment: One cohort of the study gets a single treatment, another gets a second treatment at day 91.

But we'll also be following these patients for a considerable length of time, to see whether the intended effects are maintained or whether they diminish over time. Even though there are only a proposed 160 participants, utilizing different dosing and different injection points throughout the scalp, there will be 396 treatment sites, or data points, that we will be able to gather from those 160 patients, in addition to the data to be gleaned and shared from the trial Shiseido is funding in Japan. These questions are great—effectiveness and duration of effect—and we have an obligation to answer them, which is why the trials are designed the way they are.

TLSR: What else is RepliCel working on at the moment?

RLB: We have another population of cells derived from the hair follicle (the non-bulbar dermal sheath cells [NBDS cells]) that we believe is a platform capable of generating multiple products for various indications. These cells can be readily expanded, and it turns out they are highly expressive of type 1 collagen. Our first trial with these cells will be using our RCT-01 product for the treatment of chronic Achilles tendinosis.

Up to 90% of healthy tendon is comprised of well-constructed type 1 collagen, and a number of indications manifest in patients due to the loss of type 1 collagen production in the endogenous cells, one of which is Achilles tendinosis. Tendons often are not well vascularized, and after a series of injuries and as a patient ages, the endogenous fibroblasts are exhausted of their ability to continue to produce the type 1 collagen necessary to support healthy tendons. RepliCel's predicate science is built around the injection of autologous (harvested and administered back to the same patient) fibroblasts capable of producing the kind of collagen needed to restore the patient to healthier function and better pain scores.

TLSR: Do you have evidence of actual tendon regeneration?

RLB: In three tendinosis-related clinical trials performed using a similar cell type, which has now been licensed into the company, MRI imaging shows that tendon treated with this cell type was much more akin to healthy, young, functioning tendon than what the patients had prior to injection of cells. This is an exciting platform, and the company is about to launch a Phase 1/2 trial in chronic Achilles tendinosis. We believe the cells could also have application in other indications, including jumper's knee, golfer's elbow, tennis elbow and torn rotator cuffs, as well as in a number of dermatological applications. Late this year, we will launch a Phase 1 study in healthy volunteers to look at the ability to regenerate the underlying tissue of skin in patients who have aging or sun-damaged skin.

TLSR: Achilles tendinosis and androgenic alopecia are very different indications. Androgenic 

alopecia is a hormone-dependent condition, while Achilles tendinosis is trauma-related.

RLB: That's a great point. Even though both of these studies—tendon repair and hair regeneration—use cells derived from the hair follicle, we're working with two very different cell populations. As a result, they have the ability to elicit very different, targeted responses.

TLSR: RepliCel's shares have suffered considerably over the past six months. What caused the dip and what is the company doing to fix the issue?

RLB: The fact of the matter is the company was delayed in progressing to its Phase 2 trial for RCH-01 in hair regeneration because of an issue with the supply of a critical growth media. The new media wasn't producing the same results, so we had to go back to the drawing board and discover what the problem was. The comparability data is now coming in to support our belief that we've solved that problem. We have four trials expected to launch in the next few months (three of ours and one of Shiseido's). Two of these are expected to give clinical readouts late next year. Until we are a company executing clinical trials, we are a company talking about executing clinical trials, and certain investors grow understandably impatient.

I'm very pleased that in October we submitted an application to Health Canada for the proposed Phase 1/2 clinical trial for chronic Achilles tendinosis. This triggers a 30-day window during which Health Canada can provide a No Objection Letter allowing us to proceed with the trial. This event is the initial trigger for a cascade of catalysts anticipated over the following months related to this trial, as well as our proposed Phase 1 clinical trial in Germany for aging and sun-damaged skin, our proposed Phase 2 trial for pattern baldness (androgenetic alopecia) in Germany, and Shiseido's upcoming trial for pattern baldness in Japan. We've been on the road for several weeks, crisscrossing the U.S., Canada and Japan, talking to investors, analysts and potential partners. We are pleased with the level of interest being generated, and believe that once we demonstrate we are executing on schedule, we will generate increased support.

TLSR: Is it possible that all these milestones will be met by the end of 2014?

RLB: We are working very hard to make that happen. I believe we're on target to have our three clinical trial application filings submitted by year-end. We expect Shiseido to file its clinical trial application early in Q1/15.

We are, of course, dependent on regulatory clearance to initiate any trial, but we have had very active dialogues with the regulators overseeing all our proposed trials, and are submitting precisely what has been discussed. As mentioned, we have now filed the first of our three applications. The second trial application proposes to use a product (RCS-01) developed from the same platform technology, so the clinical-regulatory team can leverage much of the work already done to get the second application filed.

One thing to note is that the dermatology and tendinosis trials are relatively quick studies to enroll. We've been in constant dialogue with the principal investigators of the RCT-01 trial in chronic Achilles tendinosis, and they assure us there's a pipeline of patients waiting to enroll in the study. The RCT-01 tendon trial is going to be a 28-participant study, and the RCS-01 in skin rejuvenation is a proposed 28-participant study design as well, but using healthy volunteers. The RCH-01 hair regeneration study is going to extend over a longer period of time, because it targets 160 participants. But the RCT-01 and RCS-01 studies will be relatively quick to enroll and to follow up on, and we expect data in 2015 for both of those.

Both of these studies are randomized, placebo-controlled and specifically designed to provide measurable and material biologic and mechanistic data that we will use to drive partner discussions. Remember that the company's business model is to codevelop assets with partners who understand the markets and have proven commercialization capabilities.

We are excited about being in the position we are now in, poised to imminently execute on three clinical trials, finalize the development and validation of our propriety injection device (which has licensable applications for acellular injectables), capitalize on our partnership with Shiseido and the innovative regulatory pathway for regenerative medicines in Japan, which provides a window to early-market access for our pattern baldness treatment, and to execute on one or more additional licenses with co-development partners in the near term.

TLSR: Thank you very much for your insight, Lee.

RLB: Thank you.

R. Lee Buckler is vice president of business and corporate development with RepliCel Life Sciences Inc. Prior to working with RepliCel, he was the managing director of Cell Therapy Group, a firm he formed in 2008, where he did business development consulting for companies and organizations in or interested in the cell therapy sector. Buckler served six years as executive director of the International Society for Cellular Therapy, and just over two years as director of business development for Progenitor Cell Therapy. He is on the editorial advisory boards of the journal Regenerative Medicine and the BioProcess International magazine, as well as the co-chair of the Alliance for Regenerative Medicine's Communications and Education Committee. Buckler cofounded Cell Therapy News, founded Cell Therapy Blog, cofounded Regenerative Medicine Jobs, founded and continues to manage the LinkedIn Cell Therapy Industry Group, and is an active industry commentator in publications and in social media. He serves on numerous industry conference advisory boards, is an advisory board member for BioCision and RoosterBio, and is on the board of directors for Hemostemix. He has a bachelor's degree in education and a law degree.

For additional comments on RepliCel Life Sciences Inc., Shiseido Company Ltd. and Hemostemix Inc. from newsletter writers, money managers and analysts, click on their respective links or visit The Life Sciences Report.

Want to read more Life Sciences Report interviews like this? Sign up for our free e-newsletter, and you'll learn when new articles have been published. To see recent interviews with industry analysts and commentators, visit our Streetwise Interviews page.

DISCLOSURE: 

1) George S. Mack conducted this interview for Streetwise Reports LLC, publisher of The Gold Report, The Energy Report, The Life Sciences Report and The Mining Report, and provides services to Streetwise Reports as an independent contractor. He owns, or his family owns, shares of the company mentioned in this interview: None. 

2) RepliCel Life Sciences Inc. paid Streetwise Reports to conduct, produce and distribute the interview

3) R. Lee Buckler had final approval of the content and is wholly responsible for the validity of the statements. Opinions expressed are the opinions of R. Lee Buckler and not of Streetwise Reports or its officers. 

4) The interview does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal disclaimer.

5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their families are prohibited from making purchases and/or sales of those securities in the open market or otherwise during the up-to-four-week interval from the time of the interview until after it publishes.

Streetwise – The Life Sciences Report is Copyright © 2014 by Streetwise Reports LLC. All rights are reserved. Streetwise Reports LLC hereby grants an unrestricted license to use or disseminate this copyrighted material (i) only in whole (and always including this disclaimer), but (ii) never in part..

Streetwise Reports LLC does not guarantee the accuracy or thoroughness of the information reported.

Streetwise Reports LLC receives a fee from companies that are listed on the home page in the In This Issue section. Their sponsor pages may be considered advertising for the purposes of 18 U.S.C. 1734.

Participating companies provide the logos used in The Life Sciences Report. These logos are trademarks and are the property of the individual companies.

101 Second St., Suite 110

Petaluma, CA 94952

Tel.: (707) 981-8204

Fax: (707) 981-8998 

Email: jluther@streetwisereports.com

Cell Therapy Industry HiLites 2009-07-03

Last week I mentioned the Regenerative Medicine Foundation and the conference they have announced for Spring 2010. I promised to find out more and bring you what I learned.

I've discovered that the driver behind the RMF is Anthony Atala and Case Western. It is a 501(c)(3) charity, non-profit organization. It was formed in 2005 by Dr. Atala to host a meeting with the U.S. Food and Drug Administration (FDA) around the topic of regenerative medicine. In 2006, the Foundation was instrumental in the formation of STRAC, the Soldier Treatment and Rengeneration Consortium, a national partnership of leading military and academic research centers and industry. STRAC formed the basis for the Armed Forces Institute of Regenerative Medicine (AFIRM).

Now the Foundation announced the first annual "Translational Issues in Regenerative Medicine" Conference, to be held in Winston-Salem, NC, December 8-10. The conference promises to focus on best practices in clinical trials and GMP facilities, as well providing a forum to discuss regulatory and reimbursement challenges. In addition, the conference will feature a venture forum to introduce early-stage and later-stage companies to accredited and institutional investors.

As its name suggests, the organization functions like a "foundation" rather than a member-based organization. This is reinforced by the fact the "Participate" page on the website has no content.

All of this is interesting but nothing more than you would learn from the RMF's website. What I DID discover this week that isn't obvious from anything yet online is that Atala has also been a primary driver behind the creation of a newly announced organization, this one a 501(c)(4) organization intended to be much more about lobbying.

This week the Alliance for Regenerative Medicine was formerly launched. (no website yet). Headquartered in Washington, DC, the Alliance is "dedicated to promoting regulatory, research, and reimbursement policies that will foster innovation in regenerative medicine" in addition to serving as a "source of information about regenerative medicine for policy makers, the media, and the general public".

With Atala's 'encouragement', Michael Wener of DC law firm Holland & Knight and Morrie Ruffin of Adjuvant Global Advisors signed up to be the driving force in the creation and evelopment of the Alliance intented to bring together academic institutions, non-profit organziations, and companies that commit to supporting and participating in a lobbying organization to promote much-needed policies in support of the regenerative medicine sector.

The organization intends to announce its final list of charter members later this month. Thus far its a respectable list including Wake Forest Institute for Regenerative Medicine, Stanford University, the University of Washington, Georgia Tech University, the Genetics Policy Institute, Geron, Johnson & Johnson, Aldagen, iZumi, Fate Therapeutics, Maxcyte, Kleiner, Perkins, Caufield and Byers, and Proteus Ventures.

Werner and Ruffin both have a great deal of experience with Capitol Hill and the biotechnology sector. I had a great conversation with Werner this week and am very encouraged by the plans for the Alliance and their intended inclusiveness. In my opinion, the Alliance promises to fill a much-needed gap unmet by any existing organizations. I will dedicate a post to more information about the Alliance in the days to come.

_______________________________________________________________

More stakeholders united in one place at one time than any other.

_______________________________________________________________

FINANCIAL

Surprisingly, the past week or so - here in the beginning of summer with July 4th weekend being most everyone's primary focus in America - was a very big week for closing financing deals in the cell therapy sector.

In this financial environment the ability to raise any money is a marked accomplishment. Nevertheless four companies in this emerging field did just that: a technology company, a service-based one, a company developing a therapeutic, and one that sort of does all three.

Celsense, Inc. announced they recently raised just over $1.76 million in additional funds in their Series B.

The funds will reportedly be used to commence human clinical testing of its lead compound, Cell Sense which is a fluorocarbon tracer agent used to label cells ex vivo without the use of transfection agents. The technology reportedly enables researchers and clinicians to non-invasively track the administration and migration of the labeled and transplanted therapeutic and/or diagnostic cells using 19F MRI or MRS. Applications include tracking cells in immunotherapy or regenerative medicine as well as diagnosis of inflammatory sites by tracking selected populations of immune cells.

**

NeoStem, Inc. (NYSE Amex: NBS) has also bucked the trend recently by tapping into Asian investors and deal-making. NBS announced that it now has raised additional gross funds of over $4 million from institutional and private investors. These funds, together with the $11 million private placement from three Asia-based investors announced April 13, 2009, bring the total investment obtained by NeoStem in the past two and half months to over $15 million.

The funds are said to be tagged for sundry purposes including support of the company's continuing initiatives with their VSEL (very small embryonic-like) stem cell technology licensed from the University of Louisville, advance NeoStem's expansion activities in China, expand U.S.-based operations, add seasoned management to the NeoStem team with experience in clinical drug development in pharmaceutical and biotechnology industry, fund the administration of overseas based clinical trials, and other general corporate purposes. Suddenly that $15 million doesn't seem like nearly enough for a focus spread that broadly

**

Neuralstem, Inc. (NYSE Amex:CUR) announced that an investor fund has purchased $1 million worth of stock and purchased $3 million worth of warrants to be exercised in $1 million allotments to be exercised within a year, three years, five years from the date of issuance. Neuralstem plans to use these proceeds for "working capital" much of which - according to SEC-filings - is currently being spent on legal fees.

**

International Stem Cell Corporation (OTCBB:ISCO) entered into a definitive agreement for a $5 million financing investment commitment (the “Investment”) with a biotechnology-focused fund (the “Investor”) that has offices in New York and California. The Company may draw down funds from the Investor as it finds a need, but is not obligated to do so. Funds will be drawn down through the issuance of redeemable Series E Preferred Stock.

**

I usually don't track or include reports of grants being received by companies but this one is a good size and - I admit - more importantly I wanted to point you to their unique usage of social media.

**

Pluristem Therapeutics Inc. (NasdaqCM:PSTI) (DAX:PJT) has been awarded a $1.9 million government grant from the Office of the Chief Scientist (OCS) at the Ministry of Industry, Trade and Labor of Israel, as a government participation in R&D expenses for the period March 2009 to February 2010. The OCS awards grants to industry in Israel to foster technological innovations. This is the fourth consecutive year that Pluristem has received this grant. The funds will be designated and used by Pluristem to support the clinical trials of the allogeneic placental-derived adherent stromal cell product, termed PLX-PAD, for the treatment of critical limb ischemia (CLI), the end-stage of peripheral artery disease (PAD), as well as for other research and development activities of the Company.

Here's the cool part: Check out their product animation on YouTube.

CLINICAL

Ok, this isn't exactly a clinical story but it's the closest thing I've got this week and I hate leaving sections bare.

Geron Corporation (Nasdaq: GERN) has published in advance of print in the journal Regenerative Medicine a paper that reports that dendritic cells (DCs) scalably manufactured from human embryonic stem cells (hESCs) exhibit normal functions of naturally occurring human DCs found in the bloodstream. This, they say, supports their planned use of hESC-derived DCs in their therapeutic vaccine currently in Phased II for AML but employing autologous dendritic cells. (see GEN's summary here)

COMMERCIAL

The big deal of the week, of course, was between GE Healthcare, a unit of General Electric Company (NYSE: GE), and Geron Corporation (Nasdaq: GERN).

The companies announced an agreement to co-develop human embryonic stem cell (hESC)-derived assay products for in vitro drug screening with the aim of having products on the market in by "early 2010".

Under the worldwide, exclusive license and collaboration agreement, GE Healthcare obtained an exclusive license to Geron’s hESC IP and a sublicense under Geron’s rights to foundation hESC patients held by the Wisconsin Alumni Research Foundation. IP arising from the alliance will be shared between the companies, with GE Healthcare retaining rights to the resulting technologies for drug discovery applications and Geron for cell therapy applications.

When Geron announced that its IND had been accepted by the FDA earlier this year, Okarma made quite a bit about how they had figured out how to scale the production of their product " unlike adult stem cell therapies" and that their product was "scalable in the same way as a monoclonal antibody".

This theme continues in this announcement with Geron's David Earp saying "“Geron is intensely focused on developing hESC-based cell therapies, and the expertise that we have developed in scalable manufacturing and differentiation of hESCs to specific cell types is directly applicable to the production of these dells for drug discovery.”

Interestingly, GE claims it is bringing "expertise in cell manufacturing" to the deal. GE Healthcare will fund the R&D program and will be responsible for manufacturing, sales and distribution of products developed under the agreement.

Click here for more background information

**

As has been discussed here several times, ever since Cell Genesys Inc (NASDAQ: CEGE) pulled the plug on its late-stage trials of its GVAX immunotherapy triggering Takeda Pharmaceuticals to pull out of its co-development deal for the product, Cell Genesys has been busy dealing with downsizing, lawsuits, and shopping itself around to potential suitors looking primarily to take it on for its cash.

This week BioSante Pharmaceuticals, Inc. (NASDAQ: BPAX) anted up to announce it is merging with Cell Genesys in an all-stock transaction valued at about $38 million - pretty much the exact amount of cash CEGE has in its bank account. the surviving entity will be BioSante.

Cash value is actually a generous purchase price for biotech companies which usually come with a burn rate that makes then worth less than their cash. CEGE, however has no clinical program, had sold most of its assets, and had downsized to 9 staff in a rented office.

There are, of course, persistent rumors of back alley shenanigans that took place in CEGE's demise but we'll leave these on the street. What is interesting to me is that BioSante has suggested they will examine options for the future of Cell Genesys' GVAX Immunotherapies, including potential combination with BioVant, BioSante's vaccine adjuvant, as well as possible external collaborations. The combined entity is also on record saying they will try to outlicense other Cell Genesys technologies.

**

Pantheon China Acquisition Corp. ("Pantheon") (OTCBB:PCQC) announced that its shareholders approved its plan to purchase China Cord Blood Services Corporation ("CCBS"). Pantheon has changed its name to China Cord Blood Corporation and will continue to trade on the OTCBB under the symbol PCQC until its expected transition to a larger U.S. exchange is complete. Pantheon China Acquisition Corp. is a "special purpose acquisition company" formed for the purpose of acquiring businesses in the People's Republic of China. Pantheon China consummated its initial public offering on December 20, 2006 generating an aggregate gross proceeds of $34,500,000. China Cord Blood Corporation is currently the largest cord blood banking operator in China in terms of geographical coverage and is the only market player with two (i.e., Beijing and Guangdong) out of six exclusive cord blood bank licenses issued by the PRC government authorities to date. Under the current PRC government regulations, only one licensed cord blood bank operator is permitted to operate in each licensed region.

**

Former CEO, Ed Scott, retired late last year from Lifeblood Biological Services, LLC with a plan to buy it out. Scott has now successfully closed that transaction and has rebranded as Key Biologics, LLC. The beauty here is that the company has a steadily growing book of business since its 2005 insception and Scott bought the assets of LBS for $188,000 on previous 12-month revenues of close to $1.6 million.

Key Biologics is being positioned to service the burgeoning cell therapy market. For insight into just some of the market need the company is poised to address, click here to read an article by Scott, previously published a year ago.

The company produces nearly 300 products for researchers involved in cellular-based therapies. Among other types of products and custom services, Key Biologics provides researchers with products made from human blood collected at its office which it converts into customer-specific products like leukocytes, plasma or T-cells shippint them directly to its customers per their specification.

**

Living Cell Technologies Limited ASX:LCT; OTCQX:LVCLY) announced that it has now officially opened its new designated pathogen free pig breeding facility in New Zealand to support the imminent launch of its clinical trial in New Zealand of DIABECELL in subjects with type 1 diabes. DIABECELL is an encapsulated porcine insulin-producing cells which can be administered without the need to use immunosuppressive drugs.

**

GEN this week carries an interesting article on a company applying its existing technology to cell therapy bioprocessing scale-up. The article sites "...large-scale culturing of embryonic and induced pluripotent stem (ES, iPS) cell lines for use in regenerative medicine and drug discovery applications" as still a "major challenge" for the industry. Cyntellect, Inc. reports that its Stem Cell Manager "is a series of stem cell management processes including automated physical passage of stem cells, automated embryoid body generation, and purification of specialized cell types derived from stem cells" which can help alleviate some of the major challenges in large-scale automation of the processes required to manipulate sensitive therapeutic cells.

**

Bio-Matrix Scientific Group, Inc. (OTCBB:BMSN) announced the launch of a national marketing program aimed at hospitals "in need of storing umbilical cord blood specimens for future transplantation".

The business model is not entirely clear to me but it would appear BMSN believes hospital clients will outsource the processing and storage of cord blood units which parents donate to the hospital-owned public cord blood "bank" (or registry). I'm told BMSN may not have the appropriate license for processing of this sort but I'm sure they will be taking care of that. More importantly, I am keenly aware this hospital-based model for selling back-end cord blood processing and storage to hospitals is a very tough sell and not one that hospitals understand easily. For what it's worth, that didn't stop the WallStreetCorner.com from naming the company its “Stock Pick” for the second time since February of this year.

We will watch BMSN's future with interest.

**

Toronto-based bio-commercialization center, MaRS Innovation, has selected an umbilical cord stem cell technology as its first commercialization opportunity. The technology offers a proprietary method to create multi-potent stem cells (MPSCs) from human umbilical cord blood with several promising therapeutic applications but which they will initially focus on diabetes. Pre-clinical data has reportedly demonstrated that these cells uniquely secrete insulin in response to glucose, thereby mimicking the “normal” physiological state.

MISCELLANIA

ProChon Biotech, Ltd., a company working with tissue regenerative technologies announced that it recently received the Genzyme Award for Excellence in Cartilage Research for its poster presentation entitled, “Comparative Analysis of Cell Sources for Cartilage Cell Therapy.” The company reports that the research recognized by the award is incorporated into their development pipeline and will compliment its BioCart™ Cartilage Regeneration System.

Prochon's BioCart^™ Cartilage Regeneration System is an autologous chondrocyte implant system that - and here's why I love this story because this is just another example of a commercially-available cell therapy that people in the US never talk about - is commercially available in Italy, Greece and Israel and is undergoing an FDA Phase II multicenter clinical study in the United States. The company reports that to-date over 70 patients have received the BioCart^TM implant with some patients over five years post-implantation.

**

I came across a nice piece this week discussing Arteriocyte Medical Systems' work on its DARPA-funded project to create human blood from stem cells at a rate that could allow the military and hospitals to produce units of blood on site. The company claims that with a technology developed by a company called Nanex, they are able to grow stem cells at a rate 10 to 100 times faster than with more traditional methods. The Nanex technology mimics a bone marrow environment in which stem cells can grow quickly. The trick now is to automate and scale-up the system to meet the specs DARPA required in its project paramaters.

**

Luke Timmerman, a recent Twitter convert (@ldtimmerman), and one of the most prodigious reporters on the biotech scene, has posted another great article on Dendreon with this great opening line: "Dendreon, as part of its plan to morph into a full-fledged company that discovers, develops, and markets cancer drugs, has worked behind the scenes to transform its shareholder base from a Wild West gallery into a roster of backers you might expect from a blue-chip biotech." The article, "Dendreon Tames Short Sellers, Transforms Itself into Old-School Buy-and-Hold Stock", is - as usual - a good read.

**

The LA Times carried a very thorough and informative piece on the state (promise and challenges) of therapeutic cancer vaccines that is also well worth the read.

**

Another interesting article I uncovered this week was a rather poorly written but informative review of some the scientific, clinical and regulatory progress in the field of stem cell research/therapy in India.

Sign-off

The latest issue of the journal Regenerative Medicine has been released and it looks well worth the read. Here the table of contents.

Hope this was useful.

Cell Therapy Industry HiLites 2009-06-26

Let's get the public services announcement out of the way right up front.

• CellTherapy 2010. Save the date. Submit your session ideas. www.celltherapy2010.com

• While you're planning your calendar, plan on the 2009 World Stem Cell Summit September 21-23, 2009. Baltimore Convention Center Baltiomore, Maryland. (the 2008 World Stem Cell Summit webcast and downloadable World Stem Cell Report are at www.worldstemcellsummit.com).

• Curiously, the Regenerative Medicine Foundation (I know. I hadn't heard of it either until now.) has announced that its "Translational Issues in Regenerative Medicine" conference is the "premiere event for leaders in regenerative medicine translation, representing academic research, clinical, healthcare policy, regulatory, reimbursement, investment and biotechnology industry interests." Curious because it has yet to ever occur. That being said, if they do what they promise, it may just well be. I'm trying to learn more and when I do I'll let you know. Meantime, this is about all I know:
  

Spring 2010
Twin City Quarter
Winston-Salem, NC

"...to facilitate business development opportunities, the conference will feature a venture forum including 15 regenerative medicine companies, as well as partnering sessions."

____________________________________________________
Do yourself a favor. Stop monkeying around with home-made cryopreservation brew or, for than matter, any media not built to minimize cellular damage from freezing and thawing.

Do your cells a favor. Try CryoStore and HypoThermosol.

www.biolifesolutions.com

_____________________________________________________

I'm dedicating this issue of Cell Therapy Industry HiLites to TiGenix NV (NYSE EURONEXT: TIG). TiGenix wins the Cell Therapy Group "kick-ass cell therapy company of the week" award which is, of course, something I just made up but they had have a great week raising several million dollars, announcing plans for a new European facility, and receiving a positive opinion from the EMEA CHMP which all but assures them of an MAA within 90 days applicable in 30 European countries. See details below.

FINANCE

TiGenix (NYSE EURONEXT: TIG) has secured the necessary financing to fund the set-up of a GMP manufacturing facility in Europe through a private placement for a total amount of EUR 5,4 million. The investment agreement was subscribed by NV Industriebank LIOF, Particon BV, Limburg Ventures BV and LRM NV. The funds will be used to set up a new cell expansion facility in Europe for the commercial production of its cell based products in the Netherlands. The financing for the facility has been secured through a private placement. After carefully evaluating a number of options throughout Europe, taking into consideration technical, logistical, regulatory and financial criteria, TiGenix has selected a building on the Chemelot Campus, near to Maastricht, the Netherlands, to locate its new cell expansion facility. The site is centrally located in TiGenix’ key European markets, in a region that is strong in distribution and (bio)logistics and that is highly committed to develop as a transnational knowledge centre in life sciences and regenerative medicine.

This financing has now been completed following the execution of a long term lease agreement for the facility in question.

**

After raising $40+ million last year and over $14 million so far this year, Cytori Therapeutics Inc (NASDAQ: CYTX) has announced it has entered into an agreement to raise up to another $20 million over the next year. The company announced recently that it had raised $850,000 in what is described as an agreement by Orlando, FL-based fund, Seaside 88, to buy 7.15 million shares over the next year by buying a specified allotment every two weeks at an agreed discount of the then current stock price. The agreement could being in roughly $20 million for the company depending on share price. Cytori intends to use the proceeds to "accelerate marketing efforts and continue our investment in clinical trials to broaden the number of potential applications for which the Celution® System may be applied".

**

A bankruptcy court hearing is scheduled for July 6 in which Isolagen Inc. (AMEX:ILE) will request final approval of its debtor-in-possession financing plan under Chapter 11 bankruptcy reorganization protection.

**

Always a master at the art of spinning good profile, NeoStem, Inc. (NYSE: NBS) (AMEX: NBS), in the business of collection, processing and long-term storage of adult stem cells for future medical need, garnered a feature in Gene Marcial's prominent Business Week column, "Inside Wall Street," on June 18, 2009. The same week saw the company profiled in a piece on the investment potential for adult stem cell businesses in a Fortune Magazine article distributed by CNNMoney.com. The company has been on a deal-making, press release and publications tear the past few months. "Big plans by tiny NeoStem (NBS)," Mr. Marcial observed, "have helped catapult its stock to $2.27 a share from 50 cents on March 9." It must be the plans because the company which has been doing what it does since 2006, generated a mere $45,100 in total revenuefor the three months ended March 31, 2009. The good news is that is up from the $700 generated in the three months ended March 31, 2008.

The company also announced another deal saying it has signed an exclusive 10-year agreement with Enhance BioMedical Holdings Limited, a Shanghai corporation, to develop a Stem Cell Collection and Treatment Network using NeoStem's "technologies" Taiwan, Shanghai and the Chinese provinces of Jiangsu, Zhejiang, Fujian, Anhui and Jiangxi. Enhance BioMedical Holdings is a subsidiary of Enhance Holding Corporation, a multinational conglomerate whose CEO, Jackson Ling, recently invested $5 million in an $11-million private placement financing for NeoStem.

CLINICAL

Osiris Therapeutics, Inc. (NASDAQ:OSIR) announced six-month interim data from a Phase II clinical trial evaluating Prochymal, the Company's formulation of adult mesenchymal stem cells, for the treatment of chronic obstructive pulmonary disease (COPD). The trial failed to demonstrate an improvement in pulmonary function in COPD patients. The company, quickly becoming practised at spinning what might be considered bad news, said they were pleased the data did provide additional evidence of safety for the product in addition to demonstrating significantly decreased inflammation in the COPD patients. Sixty-two patients were enrolled and are being followed for two years in the placebo-controlled study.

**

Pluristem Therapeutics Inc. (NasdaqCM:PSTI) (DAX: PJT) received approval from the Paul Ehrlich Institute to begin clinical trials evaluating the company’s placental-derived adherent stromal cell product, PLX-PAD, for the treatment of critical limb ischemia.

**

Aastrom Biosciences’ (NASDAQ:ASTM) trial of its cell therapy for severe chronic heart failure was released from a clinical hold by the FDA. The the agency determined the therapy did not cause the death of a participant which resulted in the hold in May.

**

The University of Pittsburgh Cancer Institute began enrolling patients in a Phase III study evaluating the safety and efficacy of Gamida Cell Ltd's StemEx cord blood stem cell therapy for the treatment of blood cancers.

**

Having received regulatory approval late last year to proceed with its clinical trial in New Zealand, Living Cell Technologies Limited (ASX:LCT; OTCQX:LVCLY) CEO Paul Tan said enrolment of patients should be complete in about two months to begin testing their pig islet cells implanted in humans to treat diabetes.

**

Neuralstem, Inc. (NYSE Amex: CUR) has entered into a sponsored research agreement with the China Medical University & Hospital of Taiwan, in Taichung, Taiwan, to prepare for a human clinical trial using Neuralstem’s human spinal cord neural stem cells to treat stroke patients. The therapy will focus on patients whose post-stroke symptoms, including complete or partial paralysis, have stopped improving more than six months after an ischemic stroke.

COMMERCIAL

Not to be outdone by finance and operational teams, the regulatory team at TiGenix also announced reason to celebrate this week. TiGenix (NYSE EURONEXT: TIG) announced that the company received a positive opinion from the Committee for Advanced Therapies (CAT) and the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMEA) on the European Marketing Authorisation application (MAA) for
its lead product ChondroCelect.

CHMP opinions are forwarded to the European Commission for adoption of the final community Marketing Authorisation, which is typically issued between 60 and 90 days from adoption of the CHMP opinion. I'm told that European Commission generally follows the advice of the CHMP but, as Dendreon, there's little reason to celebrate until the formal approval is formalized. For the EMEA take and related info on the decision, click here.

Upon approval, ChondroCelect will be, I believe, the first product approved under the new ATMP regulatory framework. It will certainly be the first cell based product to be centrally approved in the 27 member states of the European Union as well as in Iceland, Lichtenstein and Norway under the new Advanced-Therapy Medicinal Products (ATMP) legislation.

**

Further to my March 31 speculation about who might be the buyer of York Pharma plc (and its two cell therapy products on the market) and my April 13 post that ULURU Inc. (NYSE Amex: ULU) had signed a non-binding offer letter to acquire the company, ULURU Inc. (NYSE Amex: ULU) has announced that at this time discussions to acquire York Pharma, plc have been terminated. ULURU has notified York that the $1 million USD line of credit it had extended York are due plus accrud interest. The revolving credit facility is secured by substantially all of the assets of York and its subsidiaries. York is now actively pursuing alternative funding facilities in order to repay the line of credit within 30 days and to meet its ongoing working capital requirements.

Meanwhile, rumor has it the company is already in talks with another potential suitor.

**

Here are a couple hints for your next press release. It's rarely newsworthy to have as the primary point of your release an announcement about what you are going to do, rather than what you've done; secondly, don't announce seemingly contradictory information. I'm just saying....

Stem Cell Therapy International, Inc. (OTCBB: SCII), through its wholly owned subsidiary AmStem International, Inc., announced that it has "started the process" to enable it distribute Histostem's products in anticipation of the finalization of the merger between Stem Cell Therapy International, Inc. and Histostem. This is intented, according to the SCTI President and CEO, to "enable AmStem International, Inc. to begin US distribution and immediately bolster the company's cash flow". However, the product has yet to be the subject of a clinical study in the U.S. for which they are seeking "investigators...of the highest caliber, to compare its Stem Cell Facial Cream with others on the market."

The companies reportedly continue "to work on the necessary steps to finalize the merger, and both sides are working diligently to manage its many details."

**

Community Blood Services has entered into a licensing agreement with AuxoCell Laboratories, Inc. to use AuxoCell’s exclusive patent rights and propriety process to extract fetal mesenchymal stem cells from the Wharton’s Jelly of donated umbilical cords, allowing Community Blood Services to operate the first Wharton’s Jelly derived stem cell bank in the United States.

**

After netting $221 million last month by nearly 12 million shares at $19.20 apiece, Dendreon Therapeutics Inc (NASDAQ:DNDN) expects to spend up to $50 million in a two-phase expansion of its manufacturing facility in Morris Plains, New Jersey. Monday in a regulatory filing. Additional quality control laboratories, data center, training areas, infrastructure and offices are scheduled to by done by mid-December. The final phase, with additional manufacturing clean-room work stations, production support areas, warehouse, infrastructure and offices, is to be substantially complete by April 23 of next year. Dendreon finished the initial build-out of its 158,000-square-foot facility in July 2006, before its FDA application was tabled to await additional clinical results.

**

Apparently yet another way has been invented to derive stem cells from placenta - reportedly a source much richer in stem cells than umbilical cord blood. The inventors are looking for commercial partners.

**

Cell Genesys, Inc. (NASDAQ:CEGE) - the company that just won't go away - is still pursuing "strategic alternatives". Meanwhile, it expects its cash balance will be approximately $36 million upon imminent completetion of its stock exchange offer.

MISCELLANIA

This week's issue of Science has a special focus on stem cells including a piece on "FDA Regulation of Stem Cell–Based Products" by Donald W. Fink, Jr. of FDA, CBER, an article on stem cell tourism and 3 reviews. 26 June 2009. Vol 324, Issue 5935, Pages 1603-1754

**

The Production Assistance for Cellular Therapies Group (PACT) Educational Web Seminar: Topic: "Deviation Management of Type 351 & 361 Cell Products" Date: Thursday, July 16, 2009 Time: 12:00pm-1:00pm (Eastern US Time) Register is now open for this Web Seminar at www.pactgroup.net.

Sign-off

Thanks again to BioLife Solutons. Congratulations to Tigenix. May the force be with all of you and for those going to ISSCR later this week....safe travels and please try not to enjoy Barcelona too much without me!

Cell Therapy Industry HiLites 2009-05-26

This week I'm ROTFLMAO loving this cartoon so I thought I'd share the chuckles (thanks to www.onstartups.com). I can't tell you how many times in the past years I've seen the inevitable 'hockey stick' market projections graph from companies, analysts, and investors when discussing the coming 5 years in the cell therapy sector. It's become a standing joke about how we just keep pushing the hockey stick out to the right but the curve is inevitably the same. This pokes a little fun at this much maligned tradition.

Maybe it's just because I'm Canadian that I love this so much but c'mon the beloved hockey stick has become almost more a marketing tool than the venerable piece of sports equipment every Canadian boy must own! Maybe we can adapt this new hockey stick model to give the over-used Gartner cycle a run for its money!

Down to business...

I was told last week that Senators have been inviting people to Capital Hill to speak to them about FDA regulation of adult stem cells perhaps at the instigation of Chris Centeno's lobbying through ASCTA (www.stemcelldocs.com) that the medical use of moderately expanded autologous adult stem cells should not be regulated by the FDA but rather self-regulated by physicians. This could, of course, have serious implications on business models for autologous cell therapy companies. I've heard nothing of the Senators asking for ISSCR or ISCT counsel to-date. It would, of course, likely be Centeno's position that anyone involved in those orgs are "pharma hacks" as he has accused me of being.

ASCTA which is comprised of physicians none of whom are acknowledged leaders in stem cell research recently proudly announced the release of their stem cell guidelines which they suggest should replace FDA regulation. In what appears to be simple ignorance of the years of work being done by AABB & FACT, ASCTA described their guidelines as "the world's first clinical guidelines to allow U.S. doctors to safely begin using the patient's own stem cells to treat disease." Their lab practice guidelines contain such gems as:

• Any facility used in the processing A-ASC’s must be of suitable size, construction, and location to prevent contamination.
• The facility should be in a good state of repair.
  
• Equipment used in the facility should be adequately maintained
• In humid areas, dehumidifiers should be used to control the spread of infectious agents in the lab.
• Any reagents used must be approved for human use (where practical).

In an online posting entitled "The FDA is Killing Adult Stem Cell Therapy Thus Killing Patients", ASCTA supporter, Don Margolis, states:

In a startling new interview on stem cell research and adult stem cells in the near future, Dr. Christopher Centeno warns that Big Pharma and the FDA are teaming to control the use of a person’s own stem cells thus slowing innovation and the use of Adult Stem Cells to help patients now.

FINANCIAL

ReNeuron Group plc (LSE: RENE.L) has raised the £3 million it announced it intended to raise. ReNeuron recently received regulatory approval to commence a Phase I clinical trial in the UK with its lead ReN001 stem cell therapy for disabled stroke patients. The Company is developing stem cell therapies for a number of other conditions, including peripheral arterial disease and diseases of the retina. ReNeuron has also developed a range of stem cell lines for non-therapeutic applications – its ReNcell® products for use in academic and commercial research. The Company’s ReNcell®CX and ReNcell®VM neural cell lines are marketed worldwide under license by USA-based Millipore Corporation.

**

Pfizer expects to pump $100 million into its international stem cell development program which aims to focus both on small molecules that work by modifying cells in the body and cell therapy where the cells are manipulated outside the body. The first major use of the Pfizer money is for a collaboration with University College London (UCL) for the treatment of certain forms of blindness through the London Project to Cure Blindness. The collaboration will attempt to develop stem cell-based therapies primarily for wet and dry macular degeneration (AMD), which Pfizer has the rights to progress through clinical trials and then commercialize. Pfizer’s work on this could potentially provide a boost for the cash-strapped and searching-for-a-buyer, Intercytex Group plc (AIM: ICX), whose involvement on the London Project could yield cash royalties. But that's not where the Pfizer-Intercytex connection may end, apparently. Reports indicate that Pfizer is in fact eyeing the troubled Intercytex as a potential acquisition or investment. The company is on the auction block and actively seeking suitors.

**

Cytori Therapeutics, Inc. (NASDAQ: CYTX) expected to close a private placement for $4.2 million by May 11. No word yet...

**

Cord Blood America, Inc. (OTCBB:CBAI) has received a commitment for up to $2.3 million of new funding to acquire or build its own state-of-the-art laboratory for the storage of multiple stem cell products including umbilical cord blood stem cells. Less than two years ago the company signed a deal with Progenitor Cell Therapy LLC to process and store their stem cell units on a contract basis. Now CBAI says having their own facility will over the long term reduce their costs for both the processing and storage and additionally allow CBAI the flexibility and capacity to pursue new sources of revenue such as the storage of adipose tissue and peripheral blood stem cells processing and storage much easier than under the current structure of outsourced relationships.

**

Core Dynamics announced it has recently closed on a new financing round of US$17.9 million institutional and private investors. The money will be used to further develop the company's process for freeze drying human stem cells to be stored at room temperature.

**

Bioheart, Inc. (OTCBB:BHRT) announced today that its common stock is now being quoted on the OTC Bulletin Board (OTCBB) under the trading symbol "BHRT," the company's original ticker symbol.

CLINICAL

Aastrom Biosciences, Inc. (Nasdaq:ASTM) temporarily suspended 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. IMPACT-DCM is a clinical trial to evaluate the surgical delivery of autologous cells directly into the human heart muscle for the treatment of congestive heart failure associated with dilated cardiomyopathy (DCM) in both ischemic and non-ischemic patients. The patient's cause of death has not yet been determined and is the subject of a pending investigation at the clinical site. An independent Data Safety Monitoring Board (DSMB) will also assess the circumstances of the event. The Company has voluntarily suspended patient enrollment and treatment in the trial and the FDA placed the trial on temporary clinical hold pending an investigation.

**

ImmunoCellular Therapeutics, Ltd. (OTC: IMUC.OB ) (IMUC) announced that clinical data from the company's Phase 1 trial of its antigen-pulsed dendritic cell immunotherapeutic cancer vaccine ICT-107 for patients with brain stem glioma and glioblastoma will be presented at this year's ASCO. Having said that, they also said that while "encouraging data" was observed from the phase I trial of ICT-107, IMUC's primary focus going forward will be on its lead product candidate, ICT-121, which is an peptide-based "off-the-shelf" cancer vaccine that targets cancer stem cells and may have applicability to multiple types of cancer. IMUC anticipates filing an Investigational New Drug (IND) application in the third quarter of 2009 for a Phase I trial of ICT 121 in the treatment of glioblastoma (brain cancer).

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Little-known, Memgen, LLC will be presenting results from a recently completed phase I clinical trial of its novel active immunotherapy product, ISF35, in patients with high-risk, progressive chronic lymphocytic leukemia (CLL), at next week's ASCO meeting. According to the study's principle investigator, Dr. Castro, one injection with no chemotherapy resulted in durable reductions in lymph nodes and spleen size in most patients and three of fifteen patients with rapidly progressing disease did not require any further treatment for more than one year. Based on the results from this single injection study, a phase II trial evaluating multiple intranodal injections of ISF35 in CLL has begun funded by - of all things! - the Food and Drug Administration at UCSD. The company believes the product is also positioned for use in lymphomas and solid tumor cancers.

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Aderans Research Institute Inc. (ARI) announced it has launched Phase 2 of its clinical study on cell-based hair regeneration for men and women. The company's research is focused on developing a state-of-the-art tissue engineering solution to the regeneration of hair, taking actual hair cells--fibroblasts and keratinocytes, the two primary cell types within hair follicles--and promoting their growth in controlled laboratory environments. The research teams then recombine them and these "combined hair cells" are then placed in the patient's skin, where they are expected to elicit hair growth.

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Cytori Therapeutics, Inc. (NASDAQ:CYTX) completed enrollment in the first study to investigate adipose derived stem and regenerative cells in chronic heart disease. The trial, named the PRECISE study, was designed as a double-blind, randomized, placebo controlled, dose escalation study specifically enrolling patients suffering from an advanced form of chronic heart disease, known as chronic myocardial ischemia, for which there is no generally accepted treatment. The trial enrolled 27 patients in a study involving the patients’ own cells extracted from adipose tissue and processed for delivery at the point of care using Cytori’s Celution® System. The cells were then injected back into the patients using the NOGA® XP System (Biologics Delivery Systems, Cordis Corp., a Johnson and Johnson company). Primary endpoints were safety and feasibility of Cytori’s Celution® System as part of a novel procedure for chronic heart disease.

A major attribute of the Celution® output is that it is comprised of multiple cells types. This, the company believe, contributes to multiple mechanisms of action, and thus increases the potential to restore heart function. The Celution® System used in PRECISE was configured specifically for cardiovascular disease, including proprietary processes and enzymes needed to achieve a level of purity required for vascular delivery.

COMMERCIAL

Cytori Therapeutics (NASDAQ:CYTX) and GE Healthcare, a unit of General Electric Company (NYSE:GE) announced an agreement by which GE Healthcare will commercialize Cytori's StemSource(R) technology in the North American stem cell banking and research markets. The StemSource technology includes automated equipment to process stem and regenerative cells found in adipose tissue, cryopreserve them or use them directly for research purposes. In January 2009, Cytori and GE Healthcare formed a separate agreement to commercialize Cytori's products in ten European countries. This includes selling the Celution(R) 800/CRS System in the European cosmetic and reconstructive surgery market as well as selling StemSource products in the European cell banking and research markets. The recent partnership is similar in nature to the European agreement, but is limited to the sale of StemSource banking and research products in the U.S., Canada and Mexico for 18 months starting in the second quarter of 2009. The agreement does not include U.S. commercialization of Cytori's Celution System, which is currently under review by the FDA.

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Celsense, Inc. has announced the formal opening of its new and expanded R&D facility said to be a key component to Celsense’s commercialization strategy for its pre-clinical and clinical-grade molecular imaging agents used to non-invasively visualize and measure cellular activity and biodistribution. The company is now actively bringing both their lead research and clinical-grade cellular and molecular imaging agents to market over the coming months and is said to be collaboration with several academic and industry parties currently evaluating our products for potential use in their discovery research or clinical protocols.

Their clinical-grade Cell Sense reagent is a fluorocarbon tracer agent used to label cells in vitro then transplanted thus enabling investigators and clinicians to non-invasively track the administration and delivery of therapeutic and diagnostic cells in vivo using MRI. Applications include tracking therapeutic cells in regenerative medicine and immunotherapy applications, and observing localized immune system response by tracking populations of immune system cells. The research-grade V-Sense is an injectable fluorocarbon MRI tracer agent that labels leukocytes in situ, enabling the direct, non-invasive observation of immune system response and localized inflammation. Applications include observing change in immune system response to therapeutics and mapping localized disease such as certain cancers and infections.

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After securing a $12M equity financing facility and closing a $1.5M private placement, formerly on-the-rocks Melbourne-based Prima BioMed (ASX: PRR), which had less than $500,000 in the bank at the end of December, is now preparing IND filing in the US for a phase IIb / III pivotal trial of CVac for ovarian cancer. As previously reported here, the company has also commenced CVac™ ovarian cancer treatments on selected patients in Australia made possible through the Australian Government’s Australian Regulatory Control Mechanism’s Special Access Scheme under the Therapeutic Goods Administration. Meanwhile the company is looking at taking the product to other countries as a means of generating cash flows in the short-term through the sale of CVac™ treatments. Now the company's executive director, Mr Martin Rogers, is touring North America in discussions with a number of hedge funds and other biotech focused investment funds about new investment and partnering opportunities for the commercialization of CVac.

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Progenitor Cell Therapy, LLC has done a deal with NeoStem, Inc. (NYSE Amex: NBS) as their exclusive provider of commercial adult stem cells processing and storage. Progenitor founder, Andrew L. Pecora, has also joined NeoStem's Advisory Board.

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NeoStem, meanwhile, - the new favorite company of alternative "medical" products promoter, Suzanne Sommers - has been busy the last few weeks creating a curious empire of sundry types of bedfellows. Recently the company announced it had signed an agreement to license the exclusive worldwide rights to a technology, with pending patent applications, developed by Vincent Falanga, M.D., Chairman of the Department of Dermatology and Skin Surgery at Roger Williams Medical Center, Providence, R.I. Dr. Falanga's stem cell product under development, Primcel, is a mesenchymal stem cell (MSC) product. Early clinical studies are said to indicate that Primcel can be used to accelerate or jump start healing of chronic wounds. Dr. Falanga's work seeks to develop a prepackaged product created from a patient's own cells, ready for physician use in a clinical setting, consisting of three applications over a 12-week period.

Almost in the same breath, the company filed a patent application claiming the proprietary stem cell technology of Vincent C. Giampapa, M.D., F.A.C.S. relating to cosmetic facial rejuvenation, which NeoStem first licensed in February 2009. Dr. Giampapa is director of the Giampapa Institute for Anti-Aging Medical Therapy, a board-certified plastic reconstructive surgeon and Assistant Clinical Professor of Plastic and Reconstructive Surgery at the University of Medicine and Dentistry of New Jersey. His cosmetic stem cell face lift technology is said to comprise a "non-surgical procedure for complete facial rejuvenation that involves injecting pluripotent cells, including stem cells, into the skin of individuals whose skin has lost its firmness and texture due to age".

Days later NeoStem announced it had signed an exclusive royalty-bearing license agreement for the Asia territory for a procedure developed by Regenerative Sciences, LLC and marketed for the treatment of chronic orthopedic conditions, under the name, Regenexx. Additionally, Regenerative Sciences, through its founder and CEO, Christopher Centeno, M.D., will serve as a consultant to NeoStem, Inc. in the area of stem cell therapy in orthopedics.

Undoubtedly Centeno will also embroil and leverage NeoStem in his fight with the FDA about the marketing of Regenexx in direct flagrance of FDA regulation. In any event this is clearly a solid "plan B" for Centeno should the FDA ever get around to shutting him down in the U.S. In the interim, Centeno continues to strengthen his network of supporters and is reportedly actively working Capital Hill for support for his fight.

To mix it up a little, NeoStem then announced they had signed a deal to promote and glean royalties from the sale of a liquid, nutritional supplement - AIO Premium Cellular Health. Not kidding. They have signed an agreement for Ceres Living, Inc., a developer and direct marketer of health and wellness products. AIO Premium Cellular Health was apparently developed in conjunction with NeoStem's scientists and Advisory Board members, based on certain nutraceuticals that have been shown to "optimize stem cell functions". The promotion of AIO is said to be "part of NeoStem's corporate commitment to advancing anti-aging and regenerative medicine therapies and technologies."

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Novocell, Inc. announced that it has received U.S. Patent # 7,534,608 with with wide-ranging method claims covering the Company’s innovative stem cell therapy for the production of functional pancreatic, insulin-producing cells from human embryonic stem cells (hES). Novocell’s therapy is being developed as a method for the use of hES cells to replace insulin-producing pancreatic cells that are destroyed in people with diabetes.

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Neuralstem, Inc. (NYSEAmex:CUR) recently published a reaction to a press release by StemCells, Inc. (STEM) refuting many of their statements in what has become a very public dispute. Included in the reaction is the claim that "the PTO upheld the patentability of Neuralstem’s core technology in May, 2006, in response to a challenge from STEM" and that the Neuralstem "patents are not being challenged in the PTO or in any of the suits with STEM".

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This is not a cell therapy story but it could be. Takeda Pharmaceutical Company Limited (TSE: 4502) and IDM Pharma, Inc. (Nasdaq: IDMI) today announced that Takeda America Holdings, Inc., a wholly-owned subsidiary of Takeda (Takeda America), and IDM Pharma have entered into an agreement for Takeda America to acquire IDM Pharma. Takeda America has established Jade Subsidiary Corporation as a wholly-owned subsidiary to effect that transaction. Under the agreement, Takeda America will purchase all of IDM Pharma's outstanding shares for US$2.64 per share in an all cash tender offer followed by a merger.

You may recall that on April 1 last year (omen perhaps?), Cell Genesys, Inc. (“Cell Genesys”, Nasdaq: CEGE) and Takeda announced that the companies had formed a global alliance for the development and commercialization of GVAX immunotherapy for prostate cancer, Cell Genesys' lead product candidate then in Phase 3 clinical development. Under the agreement, in exchange for exclusive worldwide commercial rights to GVAX immunotherapy for prostate cancer, Takeda was to pay Cell Genesys an upfront payment of $50 million and additional milestone payments totaling up to $270 million relating to regulatory approval and commercialization of GVAX immunotherapy for prostate cancer in the United States, European Union and Japan.

This deal quickly unraveled when they decided to mothball both phase 3 studies of GVAX on statistical likelihood of failure. This was Takeda's big venture into cell therapy and it is an ugly, recent memory. Now they have bought IDM Pharma. They 've bought it for the company's primary asset, MEPACT, (mifamurtide), a macrophage activator drug therapy indicated for the treatment of non-metastatic osteosarcoma (malignant bone cancer) following surgical removal of the tumor (resection) in children, adolescents and young adults.

Here's the cell therapy part: IDM Pharma has 3 autologous cell therapy products currently on developmental hold as a means of perserving resources to get them to the home run on MEPACT. BEXIDEM is activated macrophages (Monocyte-derived Activated Killer cells or MAK® cells) for bladder cancer. UVIDEM, for melanoma, and COLLIDEM, for colorectal cancer, are both therapies using dendritophages (specialized immune cells derived from the patient's own white blood cells). It will be very interesting to see what Takeda does with these products, if anything.

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Veterinary stem cell company, Vet-Stem, Inc, has now expanded from its US base into Canada, providing commercially available autologous, adipose-derived stem cell tranplants for horses and dogs.

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According to the Seattle Times which played a critical role in exposing what is now alleged to be potential fraud, CellCyte Genetics, "left a cashless zombie after the stock-touting campaign that lifted its value to $400 million faded away, says it has reached a tentative settlement in the Securities and Exchange Commission (SEC) inquiry into its activities" but it still faces a criminal probe and the "number of lawyers embroiled in the company's legal troubles have now outnumbered the company's peak work force".

MISCELLANEA

International Stem Cell Corporation (OTCBB:ISCO), the company behind human stem cells from unfertilized eggs (called “parthenogenetic stem cells”), submitted comments to NIH on their draft stem cell research guidelines issued by the National Institutes of Health (NIH) on April 17, 2009. ISCO then published its comments in a press release and then their CEO published a blog to address questions regarding their comments.

Genetics Policy Institute also submitted a letter to the National Institutes of Health (NIH) commenting on the Draft Guidelines for Human Stem Cell Research. The complete letter is available at www.genpol.org for public viewing.

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The California Institute for Regenerative Medicine is working its way through Congress seeking $500 million in federal funds it hopes to apply toward guaranteeing the long-planned small-business loan program.

Forbes published what is actually quite an informative article on cell therapy medical tourism for a change.

Sign-off

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Thank you all for all your good wishes about the new addition to my family - my own little sample of regenerative medicine! Now I gotta go and spend some time with her before I'm enrolling her in college...

Remember. Cell Therapy Means Business.