Select 2015 Stem Cell Company Catalysts: Sagient Research's Edward Stopke

Under agreement with StreetWise Reports, I'm pleased to share their recent coverage of the regenerative medicine sector.

Original Source: Source: Peter Byrne of The Life Sciences Report (1/22/15)
"Convert Catalysts into Profits: Sagient Research's Edward Stopke"

The biotech sector is teeming with companies racing to bring the hot new drug or therapy to the marketplace. But realistically assessing the therapeutic potential of pipeline products is the healthy approach for selecting a winner. In this interview with The Life Sciences Report, Edward Stopke of Sagient Research unveils his list of companies with catalytic moments in the making.

The Life Sciences Report: What types of catalysts affect biotech stocks?

Edward Stopke: The primary catalyst affecting a biotech stock is trial data, especially large Phase 3 trials that test the compound against a comparator. The other main catalysts emerge from regulatory interactions with the U.S. Food and Drug Administration (FDA) or the European Medicines Agency. Product safety rulings can drastically affect the trajectory of a biotech stock, of course. But investors need to watch a company's interactions in meetings with the advisory committees serving the governmental agencies. These interactions can presage whether or not the agency will eventually approve the drug.

TLSR: Are there economic catalysts that affect the life sciences sector as a whole?

ES: Pending changes in pricing policies for therapies can catalyze shareholder actions. Historically, many companies have been able to price high without too much backlash from payers. Companies have typically charged less for treatments with large patient populations, and the more expensive treatments target smaller numbers of patients. But during the last year, some high pricing became less sustainable. I am thinking of Gilead Sciences Inc.'s (GILD:NASDAQ) pricing for Sovaldi, its treatment for hepatitis C virus (HCV), which caused quite a stir with insurers and governments alike. Companies generally argue that high prices are in line with previous treatments and that the products offer superior efficacy. But the sheer number of eligible patients for a high-priced drug can strain entire healthcare systems. I predict that during 2015 payers will force prices downward, not just for highly prevalent diseases such as HCV, but in all therapeutic markets, stem cells included.

TLSR: What catalysts are coming up for stem cell companies in 2015?

ES: Cytori Therapeutics Inc. (CYTX:NASDAQ) is developing a stem cell therapy from fat-derived stem and regenerative cells. It has Phase 1/2 data for heart disease slated for release in Q1/15. Vericel Corp. (VCEL:NASDAQ), which recently changed its name from Aastrom Biosciences Inc., has Phase 2 data expected in H1/15 on tissue repair cells for heart failure. Athersys Inc.
(ATHX:NASDAQ) is developing a stem cell product called MultiStem.

TLSR: What are the projected applications for MultiStem?

ES: Athersys is studying MultiStem in human trials for stroke, myocardial infarction (MI) and graft-versus-host disease (GvHD). It is in Phase 2 development for stroke and early Phase 1 for MI and GvHD. The firm completed enrollment in its Phase 2 stroke study before the new year, so the first 90-day results will emerge in Q1/15E. Athersys plans to progress its MI program into Phase 2 in Q1/15. The company is looking for a development partner to further its program. Announcing a partnership deal would signal investors that experts have faith in MultiStem, and Athersys' stock price would respond positively.

TLSR: Who else do you like in the stem cell space?

ES: bluebird bio Inc. (BLUE:NASDAQ) is developing its Lenti-D product, which is more of a gene therapy product, but does consist of the patient's own stem cells. These are modified by the lentiviral vector to deliver genetic material into the cells themselves. There is a lot of hype floating around this company. It is currently in Phase 2/3 studies for a rare genetic disease, adrenomyeloneuropathy, which is similar to multiple sclerosis. Hopefully, we will see data for that pipeline product next year.

TLSR: What other therapeutic spaces do you like for strong catalysts in early 2015?

ES: Neuralstem Inc. (CUR:NYSE.MKT) has a major depressive disorder product in clinical trials code named NSI-189. It is an oral compound designed to stimulate neurogenesis of the hippocampus, which could potentially reverse the atrophy seen in depression and schizophrenia. So far, we have only seen earlier preclinical and Phase 1 data, but those results have shown meaningful reductions in both cognitive and depressive symptoms in patients who are on active therapy. And the treatment has been well tolerated.

TLSR: How does Neuralstem's product differ from competitive products?

ES: There are many treatments on the market for treating depression, of course. Most of these products, however, are small molecules. They work by inhibiting the reuptake of a combination of norepinephrine, serotonin and/or dopamine. Neuralstem's treatment uses the patient's own neural stem cells to protect against damage to the nervous system itself, and to repair existing damage, too.
TLSR: What expertise do Neuralstem's managers bring to the marketplace?

ES: Neuralstem's chief scientific officer and senior vice president of research previously worked at the National Institutes of Health's Laboratory of Molecular Biology, where they researched the isolation of human neural stem cells.

TLSR: Do you see any other potential catalysts for Neuralstem?

ES: If all goes well, Neuralstem's NSI-189 product will move into Phase 2 development in Q2/15, so that is a good advancement opportunity. We should see topline results in the early part of this year for the firm's other stem cell products, including NSI-566. That treatment is currently being studied in a Phase 2 trial for Lou Gehrig's disease.

TLSR: Who is making waves in the cancer treatment space?

ES: There is a lot of excitement surrounding the chimeric antigen receptor (CAR) T-cell immunotherapies that engineer the patient's own immune cells to target tumor-specific molecules. Kite Pharma (KITE:NASDAQ) is developing its CAR T programs for hematologic cancers, and its KTE-C19 program has shown high response rates in earlier Phase 1/2 studies. Kite just bought a licensing deal with Amgen Inc. (AMGN:NASDAQ). The deal is related to the next generation of immunotherapies based on Kite's cell therapy platform. Celgene Corp. (CELG:NASDAQ), Novartis AG (NVS:NYSE) and Juno Therapeutics (JUNO:NASDAQ) are working on similar programs.
Rexahn Pharmaceuticals Inc. (RNN:NYSE.MKT) has a couple of clinical-stage oncology candidates. Its most advanced compound is known as Archexin. It targets the PI3K pathway and is being studied in a Phase 2 trial for renal cell cancer. While we have not yet seen data yet from this compound, last year the FDA approved Gilead's Zydelig for lymphoma, which has a similar mechanism of action. Other large pharma companies, such as Novartis and Merck and Co. Inc. (MRK:NYSE), are studying similar compounds in various oncology indications. The research synergy with these big firms could prove fruitful for Rexahn in terms of acquisition potential.

TLSR: I see that Rexahn recently appointed Richard Rodgers to its board. What does Rodgers bring to the company?

ES: He brings experience with in- and out-licensing, as well as mergers and acquisitions. He was previously with Abraxis BioScience Inc. until it was acquired by Celgene. He was also with MGI Pharma Inc., which was acquired by Eisai Inc. (ESALF:OTCPK).

TLSR: What types of specific catalysts are likely to affect Rexahn's stock price?

ES: If Rexahn can harness Rodgers' experience to secure a licensing deal for Archexin, its stock price would obviously respond positively. It has a few other early-stage compounds in various tumor types. If any of these products survive later-stage studies, the market will provide rewards. We could see some Phase 1 data from Rexahn's RX-5902 and RX-3117 compounds in the near future. These are being studied for very solid tumor indications.

TLSR: What other biotechs with emerging catalysts do you follow?

ES: I like quite a few smaller companies, like Raptor Pharmaceutical Corp. (RPTP:NASDAQ), Celator Pharmaceuticals (CPXX:NASDAQ), Sarepta Therapeutics Inc. (SRPT:NASDAQ), Celldex Therapeutics Inc. (CLDX:NASDAQ), PTC Therapeutics Inc. (PTCT:NASDAQ) and Rockwell Medical Inc. (RMTI:NASDAQ). Each of these has a unique pipeline.

TLSR: Can you synopsize where each of these companies is at in the pipeline?

ES: Raptor Pharmaceutical's main compound is Procysbi. It is already on the market. It was approved in 2013 to treat cystinosis, and it is one of only three such treatments approved in the U.S. Raptor is also studying Huntington's disease and nonalcoholic fatty liver disease, the latter being of notable interest due to its large market size and connection to obesity. Raptor is expecting results from its larger Phase 2b study in H1/15.

Celator Pharmaceuticals is developing CPX-351 for leukemia. It began Phase 3 development in late 2012 with about 300 patients. The first results from this study are expected sometime in Q2/15.
Sarepta Therapeutics has a number of compounds. The most advanced is eteplirsen, developed for a type of muscular dystrophy. We have seen clinical data on it, and it is fairly good, although taken from a small subset. There is a lot of speculation about whether the FDA will accept Sarepta's new drug application (NDA) and what kind of data the agency will require for approval. Sarepta expects to file around the middle of this year. A successful filing and acceptance could move the stock price nicely.

PTC Therapeutics is developing its ataluren compound for muscular dystrophy. The company submitted a rolling NDA to the agency in late December. It is not yet a complete application, but it does allow completed portions of the application to be submitted and reviewed on an ongoing basis. PTC hopes to complete the application in late 2015. We should see the first results from its larger Phase 3 study toward year-end 2015.

Celldex Therapeutics' most advanced candidate is rindopepimut, which is being developed for glioblastoma, brain cancer. Topline results for a large Phase 3 are expected in the middle of this year. If the results prove positive, Celdex shareholders will benefit, as rindopepimut is the firm's main compound in a large treatment space.

Rockwell Medical has a single compound called Triferic. It is being reviewed by the FDA as a treatment for iron deficiency in chronic kidney disease patients. The agency is expected to give a decision on provability by the end of this month.

TLSR: Aside from handicapping the pipeline products, what qualities do you look for in a firm?
ES: It is vitally important to understand the capital structure of a company. As far as a company's potential staying power, I look at the general platform. Is it a stem cell platform? Is it a genetic therapy type of platform? Is it a completely new mechanism of action? Is it something that could be used in various types of diseases?

TLSR: Sounds good, Edward. Thank you for speaking with us.

ES: Thank you, Peter.

Edward Stopke is a financial analyst with BioMedTracker and has been with Sagient Research for three years. He is responsible for day-to-day analysis with the BioMedTracker analyst team, and works with the scientific analysts to determine the financial and market impact of early-stage drugs. In his time with Sagient, Stopke has gained an understanding of the pharmaceutical and biotech development process to better develop revenue models for various indications. Stopke received a bachelor's degree in economics from San Diego State University.

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DISCLOSURE:
1) Peter Byrne 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 or his family own shares of the following companies mentioned in this interview: None.
2) The following companies mentioned in the interview are sponsors of Streetwise Reports: Rexahn Pharmaceuticals Inc., Neuralstem Inc., Athersys Inc. 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) Edward Stopke: I own, or my family owns, shares of the following companies mentioned in this interview: Gilead Sciences Inc., Sarepta Therapeutics Inc. 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.

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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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Japan Gives a Hug to the Cell Therapy Industry

Original Source: George S. Mack of The Life Sciences Report (10/22/14)

Last year, Japan rolled out the red carpet for cell therapy developers with new legislation designed to expedite development of regenerative medicine solutions for all manner of disease. In this interview with The Life Sciences Report, former regenerative medicine consultant R. Lee Buckler, now a vice president with RepliCel Life Sciences Inc., puts his consultant's hat back on to talk about Japan's hospitable stance on proposed cell therapies. He provides an update on the meaning of the new law and discusses a small cohort of companies poised to reap the advantages.

The Life Sciences Report: (TLSR) Lee, you've been around the stem cell and regenerative medicine industry for quite a long time, and you have a special interest in the new legislation enacted by the Japanese Diet (parliament) back in November 2013. The intention of the law is to hasten clinical development of cell therapies and regenerative medicine therapies. A new cell therapy can get conditional approval in Japan for a limited time with clinical (Phase 2) data showing safety in humans. This new law goes into effect in November. Tell me your thoughts.

R. Lee Buckler: The entire industry is watching Japan and this new regulatory model with an incredible amount of interest. We're on the brink of seeing more detail around the regulations.

This initiative sets up a paradigm of conditional approval, where a company can bring an application to the Japanese regulators, and if it shows sufficient safety evidence and some evidence of efficacy, the company could get conditional approval to sell its product in the Japanese marketplace. This conditional approval would be for a seven-year window, during which the sponsor or sponsors would have an obligation to continue to file clinical data toward an eventual final market approval—a biologics license application (BLA) or the Japanese equivalent thereof. There is also some suggestion that during the seven-year window, some reimbursement will be attached to the product being sold on the market.

TLSR: In mid-September, Pluristem Therapeutics Inc. (PSTI:NASDAQ), an Israeli company, said it was going to pursue a development strategy in Japan. It hired consultants to help the company get into that market. The company's lead indications are peripheral artery disease, intermittent claudication, critical limb ischemia and muscle injury. But otherwise, I don't see a lot of other companies initiating programs in Japan. Do you see companies from the U.S., Canada or Europe getting into that regulatory environment?

RLB: I think most companies in the regenerative medicine industry have a tremendous desire to have a Japanese strategy right now. The problem is that a lot of companies in this sector don't have enough bandwidth, or capital, to execute on that.

However, a few are making Japan a high priority. Currently, less than a handful of companies have a clearly articulated Japanese strategy, and only that many again have begun executing on the opportunity.

Pluristem has made the announcement about pursuing opportunities in Japan, and Chairman and CEO Zami Aberman has done an admirable job of executing deals. The company has the partnership with United Therapeutics Corp. (UTHR:NASDAQ), which is conducting a Phase 1 study of the company's PLX-PAD (full-term placenta-derived adherent stromal cells) product in pulmonary arterial hypertension, which is a fatal disease and happens to be a very large market. Pluristem also has a nice deal in place in Korea with CHA Biotech Co. Ltd. (CHA:KOSDAQ).

Going it alone all the way to market is not a good option for most cell therapy companies, and with the change in the Japanese regulation there is an appetite for regenerative medicine products in Japan that foreign companies in the sector need to capitalize on.

TLSR: You said there were a few others. Give me an overview, please.

RLB: Athersys Inc. (ATHX:NASDAQ) has announced a desire to move into Japan, and I know it is putting boots on the ground in the country to develop a strategy there. Back in January, the company announced some new Japanese patents in graft-versus-host disease (GvHD) and autoimmune diseases, such as inflammatory bowel diseases.

Cytori Therapeutics Inc. (CYTX:NASDAQ) has always had strong relationships in Japan, and has been there for a decade. It has a subsidiary in Tokyo called Cytori Therapeutics K.K.

Also, my own company, RepliCel Life Sciences Inc. (RP:TSX.V; REPCF:OTCQB), has an existing relationship with Shiseido Company Ltd. (4911:TSE), which will launch a clinical trial in Japan for pattern baldness in the next few months. This is one of the few cell therapy deals—perhaps the only one to date—that involves putting manufacturing capacity in Japan.

The company that is potentially in the pole position in Japan at the moment, however, may be Mesoblast Ltd. (MSB:ASE; MBLTY:OTCPK). The company inherited a relationship with Japan-based JCR Pharmaceuticals Co. Ltd. (4552:TKY) when, in 2013, it acquired the Prochymal (remestemcel-L or allogeneic, adult human mesenchymal stem cells) product portfolio from Osiris Therapeutics Inc. (OSIR:NASDAQ). The Osiris/JCR partnership, which was put into place a number of years ago, had been stagnant until the reemergence of the Japanese market as an important one for the regenerative medicine sector. Now Mesoblast has "regenerated" that relationship, and announced on Oct. 1 that JCR would be filing a market approval application for Prochymal in Japan for the treatment of pediatric GvHD, following similar approvals in Canada and New Zealand.

TLSR: Will a company be able to take Phase 2 data generated in the U.S. or Europe or Canada, including positive proof-of-concept and safety data, and submit that in Japan, potentially getting a seven-year conditional approval?

RLB: That's an important question. The best intelligence I have, based on interacting with people who have been working with the Japanese, including Professor Chris Mason from University College London, indicates the answer is "yes." The Japanese government is intentionally welcoming ex-Japan data for conditional market approval in Japan.

If a company gets conditional approval to market a product in Japan, it will also have to generate pivotal data outside of Japan, because no patients in Japan will want to risk being involved in a placebo trial when they have the option to buy the product in the marketplace. The end result of the paradigm is that there will continue to be data generated outside of Japan, which will be brought into Japan to support eventual final approval.

TLSR: Thank you for your time.

RLB: Many thanks to you.

[Editor's Note: Between the time this interview was scheduled and the time it was conducted, Buckler left his consultancy firm to become vice president of business and corporate development at Vancouver-based RepliCel Life Sciences Inc., a cell therapy company.]

R. Lee Buckler, vice president of business and corporate development at RepliCel Life Sciences Inc., has been an executive in the cell therapy sector since 2000, beginning with Malachite Management in the Stem Cell Technologies group of companies. Most recently he was the managing director of Cell Therapy Group, a firm he formed in 2008 to do business development consulting for companies and organizations working in or interested in the cell therapy sector. His work included deal-targeting, transactions, market intelligence, competitive analyses, strategic assessments and market profile planning for companies ranging from top-tier multinationals to start-ups. 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. Buckler has a bachelor's degree in education, and a law degree. He is on the editorial advisory boards of the journalRegenerative Medicine and the BioProcess International magazine. He is also co-chair of the Alliance for Regenerative Medicine's Communications and Education Committee. He is an active industry commentator in publications and in social media and serves on numerous industry advisory boards.

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 he provides services to Streetwise Reports as an independent contractor. He owns, or his family owns, shares of the following companies mentioned in this interview: None.

2) R. Lee Buckler: I own, or my family owns, shares of the following companies mentioned in this interview: RepliCel Life Sciences Inc. I personally am, or my family is, paid by the following companies mentioned in this interview: RepliCel Life Sciences Inc. 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.

3) The following companies mentioned in the interview are sponsors of Streetwise Reports: RepliCel Life Sciences Inc., Athersys 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.

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.

101 Second St., Suite 110

Petaluma, CA 94952

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Lessons from Toronto on the Future of Regenerative Medicine in Japan

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One of the early pioneers of cell therapy as we know it today is Professor Arnold Caplan commonly thought of as responsible for the identification and isolation of the mesenchymal stem cell.  Several years ago Dr. Caplan launched a summer course at Case Western Reserve University he called Business of Regenerative Medicine. This year it was hosted by the Centre for Commercialization of Regenerative Medicine (CCRM) in Toronto - the first time it's been hosted outside of Cleveland.  

I was fortunate to be invited to participate in this year's 7th Annual “Business of Regenerative Medicine: New Therapies, New Models”.  Signals blogger, Mark Curtis, has done a nice job of summarizing the highlights of this year's course.  

One of the most valuable take-aways for me was from a talk given by the inimitable Professor Chris Mason of University College London who has reportedly been advising Japanese lawmakers and regulators on their new proposed regulatory framework for regenerative medicine.  

Let me back up.  In June 2012, regenerative medicine was given priority status in the national Cabinet Secretariat’s Five-year Healthcare Innovation Strategy. In October 2012 Prof. Shinya Yamanaka of Kyoto University won a share in the Nobel Prize in Physiology or Medicine for his work in the development of iPS cell technology. This solidified regenerative medicine as an area of national pride and one of the areas in which Japan wanted to demonstrate innovative global leadership not just for scientific discovery but for the development of an industry around those discoveries. However, the Japanese regulatory framework which applied to cell therapies was widely recognized as being in desperate need of revision in order to support true commercialization of these products.  It made sense, then, that the Japanese started their innovation with the regulation. 

In November 2013, the Japanese legislature (the National Diet) passed legislation that revised Japan's Pharmaceutical Affairs Law and created the new Regenerative Medicine Law intended to be the groundwork for establishing Japan as a global leader in regenerative medicine in the years ahead. The import of this legislation was that it created a legislative mandate to create regulation that established an expedited development and commercialization pathway for regenerative medicine therapies while continuing to ensure and protect patient safety. The legislation passed with overwhelming support in both the lower and upper houses of the Diet.  Since then the policy makers and regulators at the MHLW and PMDA have been busily attempting to build a regulatory framework around this mandate.

There has been a lot written about this potentially ground-breaking approach to regulating cell therapies (see also here and here) but until now I have seen very little detail around how the mandate of the new law might be translated into operational regulation.  On that front, here is my brief second-hand synopsis of what I think I learned from Chris:

• conditional approval will be available to any cell therapies that provide evidence of safety and early signals of efficacy - this is reportedly not limited in any way by the indication or by product type 
• in other words there is no need to be addressing an orphan indication or for the product to be autologous in order to qualify
• at least in the early days of this implementation it is expected that this data will be 'imported' from trials conducted outside of Japan 
• once conditional approval is granted, a sponsor company will have 7 years to obtain final approval
• there is likely to be a requirement that a product be manufactured in Japan to obtain final approval 
• conditional approval will also come with reimbursement though there is no indication yet as to how pricing will be determined
• there is an expectation that the pivotal studies needed to obtain final approval will be done outside of Japan - since the product is already being sold in Japan, conducting a placebo-controlled trial would be next-to-impossible

There are a number of Japanese companies already in the regenerative medicine and cell therapy space that may be the initial and primary beneficiaries of this new regulatory paradigm.  Most of these are involved in the Forum for Innovative Regenerative Medicine (FIRM) established in 2011 to promote and support the commercialization of regenerative medicine in Japan. 

As largely an industry organization, FIRM really started emerging as a key national player with its involvement in the development of this newly emerging regulatory framework.  To quote FIRM, "Commercializing this state-of-the-art technology is not just a question of scientific and biotechnological prowess; the broad-ranging manufacturing capabilities nurtured by Japan’s industrial community will also be an indispensable element. Indeed, this is an opportunity for Japan to leverage its unique technologies to grow an internationally competitive industry."

Over the past months it has been interesting to observe how companies in Japan (even large multinational conglomerates) have responded to this new national mandate.  Many of them appear quite anxious to demonstrate to their shareholders, government, and peers that they have active programs in regenerative medicine and thus are supporting this national initiative.  This appears to be creating considerable appetite among Japanese companies, many of whom have not been in the sector to-date, for regenerative medicine technologies with which they can associate. 

As a result of this phenomenon, we expect there will be many regenerative medicine companies outside of Japan which will find wins for themselves as a result of this national initiative.  Naturally, companies in the regenerative medicine and cell therapy space outside of Japan are tripping over themselves to prioritize Japan as part of their corporate planning and to elucidate their Japanese strategy to investors and partners as a competitive advantage and potential earlier route to market if not return on investment.  Some of the companies distinguishing themselves early in this race are Cytori (NASDAQ:CYTX), Mesoblast (ASX:MSB), and Athersys (NASDAQ:ATHX). 

Cytori has long been in Japan and is making sure no one forgets that by press releasing the importance of their Japanese focus.  

Mesoblast inherited a Japanese licensee when they took over the Prochymal portfolio from Osiris.  JCR Pharmaceuticals has the license for the allogeneic mesenchymal stem cell (MSC) product in Japan to treat steroid refractory graft-versus-host disease (GvHD). Mesoblast has announced its intention to leverage this door to the Japanese market. Given that the product has already received approval in Canada and New Zealand for pediatric GvHD, it has been speculated that JCR may be the first company to secure conditional approval for a cell therapy in Japan.

Not to be outdone, Athersys announced in January, "We are actively evaluating the potential for accelerated development of MultiStem cells in a number of therapeutic areas in Japan, and will seek to establish strategic collaborations to assist in the rapid development of products that will help Japanese patients." 

One of the sleepers in this race may be an emerging regenerative medicine company named RepliCel Life Sciences (TSXV:RP).  In May 2013 RepliCel announced a Collaboration and Technology Development Transfer Framework Agreement with Shiseido Company, Limited (Tokyo Stock Exchange Code: 4911) for an exclusive geographic license for RepliCel’s RCH-01 hair regeneration technology.  Shiseido has an exclusive geographic license to use RCH-01 in Japan, China, South Korea, Taiwan and the ASEAN countries.

This deal garnered RepliCel ~$4M in upfront cash and was valued at over $30M in milestone payments and sales royalties.  Perhaps more importantly, since that time the companies have executed a tech transfer agreement, RepliCel was granted a key patent in Japan, and Shiseido recently celebrated the opening of its new cell-processing facility at the Kobe Biomedical Innovation Cluster (KBIC).  This facility will focus on the commercialization of RepliCel’s RCH-01 hair regeneration technology for Shiseido's markets.

In speaking with management, I understand Shiseido has plans to conduct its own clinical trial of the product in Japan and that the companies are collaborating closely (through a joint development committee) on the continued improvement of the technology to ensure a single product emerges.  This means there will be data from two concurrent trials (RepliCel's proposed phase 2 trial in Germany and Shiseido's proposed trial in Japan).

My own take on this is that it seems RepliCel/Shiseido may be well positioned to take advantage of early conditional approval of this technology in Japan in a way that generates near-term revenue while they continue to generate clinical data and optimize the treatment (dose, frequency) and manufacturing.  In due course this data will be available to RepliCel to use in licensing discussions for non-Shiseido markets.  Furthermore, RepliCel/Shiseido have already solved the anticipated hurdle of having manufacturing in the country as a predicate to eventual final approval.

The fact that their Shiseido collaboration may position RepliCel as a leader among foreign companies taking early advantage of this new regenerative medicine framework in Japan has not been lost on management (see here) but given RepliCel's expanded technology platform and product/clinical pipeline combined with the 3rd party validation and unique attributes of the Shiseido deal, I'm not sure the message has sunk in with investors yet given the ~$30M market cap.

I'll be watching the Japanese regenerative medicine race with great interest over the next few months.  It has been reported that there will be a MHLW-sponsored public comment period for the new regenerative medicine law announced soon. All stakeholders are encouraged to provide feedback on the new legislation aimed at regenerative medicine therapies. 

Also of note will be to monitor how other regulatory agencies respond.  Already we have seen moves toward expedited or conditional approvals (in limited circumstances) in Europe and potentially expedited pathways (e.g., breakthrough designation) in the U.S.  Other jurisdictions such as South Korea, Canada, and Australia have apparently taken a more functional approach to expediting certain cell therapy product approvals but on a case-by-case basis.

The Accuracy of Adipose Stem Cell Doses

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In August we published a blog post, "Are some cell counts too good to be true? Why some companies' product data may mislead", pointing people to a white paper released by INCELL Corporation.  That white paper appears now to have been pulled from their website (we are working to get a copy to make available again) but now they have published a paper providing more detailed data on aspects of their comparative cell count study.

The paper is introduced by the following abstract:

"Cell therapy products derived from adipose tissue have some unique processing issues with regard to obtaining accurate cell counts. This is because processing methods may not only show us the nucleated stromal vascular fraction (SVF) cells but also the micellular and microvesicle particles. This is true for both veterinary and human clinical products, and poses special concerns for in-clinic processing where the cell therapy dose is correlated with cell numbers and other QC data is not especially useful.

In this study, multiple cell counting methods were compared for SVF cell reparation that were derived from canine adipose tissue using commercially-available rocessing kits. The data clearly showed that many non-nucleated particles appear cell-like by size and shape, and can lead to counting errors with automated counters. In addition, certain reagents important to processing can have properties wherein the reagents alone (e.g., lecithin) may be counted as cells. The most accurate cell numbers were from hemocytometer-counting of cells stained with 4´,6-diamidino-2-phenylindole (DAPI) which shows the nuclei in concert with a viability stain such as trypan blue. The data clearly showed that care must be taken when counting cells used as a therapeutic dose."

This is an important issue particularly as it pertains to autologous cell-based treatments produced by point-of-care devices and/or kits.  I encourage you to read the paper.   

Morrison DG, Hunt DA, Garza I, Johnson RA, Moyer MP*. Counting and Processing Methods Impact Accuracy of Adipose Stem Cell Doses. BioProcess J, 2012; 11(4): 4-17.

* Dr. Moyer is CEO and Chief Science Officer for INCELL Corporation, 12734 Cimarron Path, San Antonio, Texas 78249 USA. www.incell.com

Industry-sponsored cardiovascular cell therapies. Some metrics.

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Cell therapies for cardiovascular-related conditions is a closely watched, much studied, oft-discussed, and hotly contested segment of the cell therapy industry.


The data to-date are admittedly confusing.  From a clinical perspective, the studies for which we have data have been relatively small involving a mish-mash of indications, endpoints, eligibility criterion, methods and/or route of administration, as well as the time of administration relative to event or disease progression.


Further compounding any interpretation of the data, from a technical perspective, is the fact the products have been widely varied in terms of being autologous vs allogeneic, expanded and not, genetically modified and not, from a plethora of different sources, and utilizing a wide variety of cell types from skelatal myoblasts, cardiomyocytes, mesenchymal stromal cells, mononuclear cells, etc. 


All this makes it extremely difficult to draw any conclusions with respect to what's working and what's not.  We will not attempt to do so.


All we do below is attempt to give a snapshot of the industry-sponsored cell therapy trials currently ongoing for cardiovascular-related conditions.  So here it is:


Commercial:
Pharmicell's Heartcelligram is the only cell therapy to have received regulatory approval for commercial distribution for the treatment of a cardiac-related indication.  Heartcelligram is an autologous cell therapy approved in 2011 by the Korean Food and Drug Administration (KFDA) for the treatment of Acute Mycardial Infarction (AMI).  The price is reportedly $19,000 and the trial data behind the approval has not yet been published in a peer-reviewed journal.


Phase III or II/III:
There are currently only 3 active and recruiting cardiac-related, industry-sponsored cell therapy trials.  Interestingly they all involve autologous products, two involve devices, two involve centralized manufacturing, two involve bone marrow cells as a source, two are only in European clinical sites, and two are targeting ischemic-related conditions.

• Baxter Therapeutics' Auto-CD34+ cells
• phase III trial actively recruiting
• Indication:  refractory angina and chronic myocardial ischemia
• Estimated enrollment:  444
• Estimated primary completion: June 2016 
• Cytori
• phase II/III trial actively recruiting
• Indication: ST-elevation acute myocardial infarction
• Estimated enrollment: 360
• Estimated primary completion: July 2014

• Miltenyi Biotec
• phase III trial actively recruiting
• Indication: myocardial ischemia or coronary artery disease
• Estimated enrollment: 142
• Estimated primary completion: July 2012

Two companies warrant particular mention at this stage as they appear to be in transition between phases II and III.

Cardio3 Biosciences initially designed a trial of their autologous C-Cure in heart failure secondary to ischemic cardiomyopathy to be a phase II/III trial enrolling 240 patients.  While the trial began in late 2008 and is still registered as active but no longer recruiting on ClinicalTrials.gov the entry has not been updated for almost a year.  

In 2010 the company announced that after enrolling 45 patients - of which 21 were in the treatment arm (24 in the control arm) - they decided to close the study to future enrollment and prepare for a phase III trial.  This decision was reportedly based on "very encouraging data". 

Dr. Christian Homsy, CEO of Cardio3 BioSciences provided the following guidance: “The highly promising data we report today build on the favourable safety profile we have observed through this Phase II trial and documents in patients our belief that we have with  C-Cure a product candidate with the potential to make a real difference in the treatment of heart failure... As noted in the company’s press release of 29 June 2010, with the Phase II stage completed and to allow for potential modifications to the trial protocol, Cardio3 BioSciences has not proceeded to Phase III recruitment into the trial but has continued to gather all data for the six month analysis. Through the Phase II trial, we gained significant  experience in working with a highly innovative stem cell therapy in a clinical setting, and we are using this acquired knowledge in the design of our planned Phase III programme."  The phase III trial of C-Cure is expected to commence in the second half of 2012.

Mesoblast has also announced with its strategic partner, Teva, that they are proceeding with plans to conduct a phase III study of its allogeneic cell therapy product, Revascor, in chronic heart failure.  Most anticipate this clinical trial application to be filed sometime in late 2012.

Phase I or II:
There are over 20 active, industry-sponsored earlier-stage trials (phase I, I/II or II) for cardiovascular-related conditions.  At least 5 of these are expected to have clinical readouts this year.   

Hope this is useful.

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This post has been brought to you by your friends at CTG.  All cell therapy. All the time. :)  

-- Lee @celltherapy

p.s.  As always we welcome your feedback, comments, and corrections.  

Cell Therapy Industry HiLites 2009-07-17

As Late, Late Show host, Craig Ferguson, says..."It's a great day for America. 'Why is it a great day for America,' you ask? Well, let me tell you".

The US House of Representatives has passed what promises to be (if approved by the Senate) the largest single budget increase for the FDA in the agency's history. Now many an economist or financial wizard may argue why this is fiscally irresponsible given America's economic woes but it certainly is promising for the biotech industry which has suffered due to a lack of resources in the Agency overseeing the sector. This lack of resources permeates into almost every interaction with the agency whether it be awaiting new Guidance, submission reviews, staff attrition, etc.

As it happens it is also a great day for the mother country from which America declared its independence because of their dastardly tea-taxing ways. "Oh, why ever it that so?", you might ask with a fake English accent. Well I'lltell you. It's because the UK Office for Life Sciences (OLS) has announced a "Life Sciences Blueprint" that will attempt to capitalise (well, that's how the English spell it) fully on its "leading global position in regenerative medicine". The OLS has announced £18 million funding from the Technology Strategy Board for a Regenerative Medicine programme (again with the English spelling) of investment to support key areas of commercial R&D and the development of R&D partnerships, supported by additional funding of £3.5 million from the Medical Research Council, the Engineering and Physical Sciences Research Council and the Biotechnology and Biological Sciences Research Council.
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I fell in love with this conference, the venue, and the charming city of Leipzig when it was held in 2007. For a unique and international take on the multi-disciplinary world of regenerative medicine, you should consider meeting at the WCRM 29-31 October 2009.

www.wcrm-leipzig.com

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Well I seem to be on a roll with anything related to Wake Forest recently so 'why stop now' I ask myself, without any compelling reply to the negative. This is just a little FYI. The Babcock Demon Incubator (BDI), a new venture incubator at WFU, promises to be a unique new venture incubator opportunity for regenerative medicine startups. The BDI has has fully equipped wet lab space with available equipment including chemical and tissue culture hoods, PCR, Gel systems, HPLC, microscopes, centrifuges, etc. In addition, BDI clients have access to collaborative opportunities at the Wake Forest Institute of Regenerative Medicine and to a wide range of services and specialized equipment. The incubator provides both residential and non-residential client models depending on client needs. All clients have access to office or lab space, can take advantage of business consulting, and access to an extensive services network designed to provide difficult-to-obtain resources to help young companies succeed. For more information, visit the BDI web site.

**

As regular readers of my Industry HiLites will know, from time-to-time I occasionally profile a company here in the front-end of the post providing some background, analysis, and recent developments. I'll formalize this a little by calling it a COMPANY PROFILE but won't promise to include one every time.

On another house-keeping matter you will note that I've changed the section title "FINANCIAL" to "FINANCIAL and DEALS".

COMPANY PROFILE

Bioheart, Inc., (OTC Bulletin Board: BHRT) has announced that they will proceed to the final phase of their clinical trial with muscle stem cells for treatment of cardiovascular disease.

The company convened a panel of experts to review pre-clinical data dating back to 1988 and clinical data from controlled studies which began in early 2000 (involving nearly 400 patients). Bioheart’s latest Phase II/III MARVEL study has undergone interim analysis. While the data has not been disclosed pending publication confidentiality, this panel of experts was reportedly asked to also review this data.

The panel is reported to have "unanimously and enthusiastically endorsed moving forward" to complete the final phase of clinical trials (MARVEL) to advance muscle stem cell (myoblast) therapy for treating heart failure with the goal of bringing the product to market with FDA approval and CMS reimbursement.

It's not entirely clear to me how or why this panel of experts was called together but a listen of the audio of the panel discussion frankly makes it sound a little bit like a love-in for Bioheart. In the accompanying slide deck, you will find an overview by Chairman and CEO, Howard J. Leonhardt listing an extremely aggressive list of deliverables for 2009 for a dizzying array of clinical products & programs.

On the financial front, Leonhardt states the company has to-date invested $125 million to support heart failure-related research - $85 million of which was in paid capital.

From its May 20, 2009 Form 10-Q Quarterly Report filed with the SEC, "The Company has incurred significant operating losses over the past several years and has a deficit accumulated during the development stage of $98.6 million as of March 31, 2009. In addition, as of March 31, 2009, the Company’s current liabilities exceed current assets by $12.7 million. Current liabilities include notes payable of $5.4 million. ...the Company also has an obligation of $3 million to the Company’s Chairman and spouse [they repaid the principal on a company loan and a pro rata portion of accrued interest ]. The Company does not have sufficient cash to support its operations through December 2009. The Company will need to secure significant additional sources of capital in June 2009 to develop its business and product candidates as planned."

On July 9, the company issued a press release saying that between October 1, 2008, and July 7, 2009, Bioheart received proceeds in the amount of $2,855,830 from the placement of restricted common stock and warrants under its current offering which it has now extended through October 2009. There's a little positive spinning here because this would appear to include the approximately $1.8 million raised in the sale of stocks and warrants which it announced back in October 2008. The new ~$1M appears to be coming in dribs and drabs from the sale of small chunks of stock or warrants including, if I'm reading it right, the granting of stock in repayment of an outstanding loan (see the May 2009 Quarterly Report for clarification and details).

FINANCIAL and DEALS

Bioheart, Inc., (OTCBB: BHRT) has raised approximately $1 million over the past few month in an ongoing offering (see details above).

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Continuing on its very active deal-making course to becoming one of the premier sources of stem cell lines, media, antibodies, cultureware, and characterization kits in the pre-clinical end of the cell therapy sector, Millipore Corporation has signed a co-marketing deal with BioTime, Inc. (OTCBB:BTIM) subsidiary Embryome Sciences, Inc whereby Millipore will become a worldwide distributor of the ACTCellerate™ human progenitor cell lines derived from human embryonic stem cells but not fully differentiated into specific cell types

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Not to be outdone by its sub, BioTime, Inc. (OTCBB:BTIM) announced that two of its shareholders have completed the funding of the second tranche of their equity investments in BioTime following the exercise of their rights to purchase additional shares and warrants under the terms of their May 2009 stock and warrant purchase agreements. Broadwood Partners, L.P. and George Karfunkel have each purchased an additional 1,100,000 BioTime common shares and 1,100,000 stock purchase warrants. BioTime received $4,000,000 from the sale of the additional shares and warrants. The warrants, which are substantially the same as BioTime’s publicly traded stock purchase warrants, entitle the investors to purchase additional common shares at an exercise price of $2.00 per share. The warrants will expire on October 31, 2010 and may not be exercised after that date. “The $8,000,000 of new equity capital we have raised since May from these investors, plus our $4,700,000 research grant from the California Institute for Regenerative Medicine, will be available to finance our strategic programs to build our product and technology portfolios in the emerging fields of stem cell research and regenerative medicine,” said Michael West, Ph.D., BioTime’s CEO.

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America Stem Cell, Inc. has been awarded $2.5 million from the Texas Emerging Technology Fund to use in developing its enzyme technology platforms (ASC-101 and ASC-201) designed to improve the homing and engraftment of stem cells to target organs and increase their therapeutic potential for cancer patients. Additionally, these platforms have the potential to enhance stem cell treatment of inflammation from chemotherapy/radiation, solid tumors, autoimmune diseases, and ischemic diseases including myocardial infarction and stroke.

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Aastrom Biosciences, Inc. (Nasdaq:ASTM) has gained a modest extension from NASDAQ to comply with the $1.00 minimum closing bid price rule in order to remain listed on the Nasdaq Capital Market. This means they now have until October 1, 2009 to regain compliance by achieving a $1.00 closing bid price for a minimum of 10 consecutive trading days anytime before that date. If not, they get kicked off the NASDAQ and forced to find another market for their listing. Absent a stock split it seems an unlikely goal give that the stock is currently trading in the region of .35 cents and the 52 range for the stock is 0.15 - 0.78.

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Quantum Immunologics Inc., a Tampa, Fla.-based developer of cancer immunotherpeutics, has raised $2.2 million in VC funding from Mentor Capital, Inc. (Pink Sheets:MNTR). In addition to providing $2.2 Million in funding to help support the FDA trials through approximately February 2010, Mentor Capital has agreed to assist QI in funding future strategic stock or cash acquisitions. Mentor will also stand by as a preferred funding source for QI during later stage or additional trials. This commitment, of course, stands to reason given that QI represents the majority of Mentor Capital’s portfolio assets.

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The good news for StemCyte, Inc recently bagged $1 million in funding; the not as good news for StemCyte, Inc. is that it is a debt funding round from debt financing firm Agility Capital. Agility said that the senior secured growth capital loan closed in June. The firm has previously received venture funding from Sycamore Venture Capital, Asia Star IT Fund and National Technology Enterprise Co. of Kuwait, and the W.I. Harper Group. Agility is a provider of venture debt to high technology and other companies. This blogger speculates the debt financing will be used to fund the company's commitments to a recently announced joint venture, StemCyte Therapeutics India Pvt. Ltd, which is expected to be revenue-generating in the near-term.

CLINICAL

Quantum Immunologics, Inc, a Tampa, Florida company focusing on the research and development of cancer immunotherapies, announced that the first patient in its breast cancer trial has begun to receive its dendritic cell therapy. QI is currently sponsoring and conducting an FDA-authorized Phase I/II clinical trial testing the safety and efficacy of its immunotherapy on 27 Stage IV breast cancer patients who have failed conventional therapy. Each patient will receive three monthly injections of the patient's own dendritic cells that have been sensitized to OFA (oncofetal antigen) - a cancer antigen (a protein found on cancer cells that can be targeted by the body's own immune system) found in many tumor cell lines or fetal tissue, but absent on normal, healthy tissue. When the sensitized cells are injected back into the patient, QI expects the patient's T-cells will locate the OFA found on the patient's cancer cells, thereby generating an immune response with the goal of killing the cancer cells and preventing further spread of the disease.

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Harvest Technologies Corp. announced that the company-sponsored 60-patient clinical trial conducted in Chennai, India using the company's BMAC point-of-care System to treat patients with non-reconstructable Critical Limb Ischemia (CLI) has completed enrollment. Over 300 CLI patients have been treated with the Harvest BMAC treatment protocol in an ongoing multi-center FDA study in the U.S. and other studies in India, Germany and the Czech Republic.

COMMERCIAL

Dendreon Inc (NASDAQ:DNDN) is currently eying Atlanta, GA as the location for a new $80 million, 300-person manufacturing facility. "Why Atlanta", you ask? "Ah, good question", I say. It all comes down to transportation logistics. An autologous cell therapy like Provenge requires the acquisition of a biological sample from the patient which is then used to manufacture a unit of the therapeutic that is then shipped back to the patient's clinical stie for administration. This makes logistics a primary consideration for the business model. Atlanta, has one of the world's busiest and most connected airports.

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Cord Blood America, Inc. (OTCBB:CBAI) has signed a lease for a 17,000 square foot building in Las Vegas, Nevada, for a state-of-the-art laboratory for the storage of multiple stem cell products including umbilical cord blood stem cells. Aggressively the company intents to start processing and storing its customer's umbilical cord blood stem cells in the facility in the fourth quarter of 2009. According the the company CEO, in 2010, CBAI intends to expand its cryogenic storage services to other forms of stem cells, including peripheral blood stem cell and adipose tissues. Presumably this move to self-manufacture will mean that its Corcell banking brand will no longer be able to claim the advantage of cGMP-quality processing and storage which it has made a part of its pitch since contracting with Progenitor Cell Therapy in 2007.

Meanwhile CBAI has picked the pockets of Bio-Matrix Scientific Group Inc (OTCBB:BMSN) by hiring its former Vice President and Chief Operating Officer Geoffrey John O’Neill as its new Laboratory Director and Brian Pockett as Vice President of Laboratory Operations.

In its May 2009 quarterly report, Bio-Matrix Scientific Group Inc (OTCBB:BMSN) reported they had all of $6,621 cash on hand with current liabilities of $1,371,834 such liabilities consisting of Accounts Payable, Notes Payable, Accrued Payroll Taxes, Amounts due to Shareholder, Accrued Expenses and Accrued Interest. Additional financing will be required to meet cash requirements over the next twelve months.

Meanwhile in a weird twist of almost Seinfeld-like interconnectedness, in researching SEC filings for this post I discovered that on August 11, 2008, MSN entered into a Letter of Intent with CBAI regarding the processing and storage by BMSN of cord blood specimens newly acquired by CBAI for consideration acceptable to BMSN. It would appear, however, that deal never materialized given CBAI's recent moves.

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Living Cell Technologies Limited (ASX:LCT; OTCQX:LVCLY) has announced the formation of a subsidiary, LCT Biomedical Limited in Russia, to facilitate the commercial development of DIABECELL®, its lead product for the treatment of type 1 diabetes. Reportedly the regulatory process has already been initiated and it is envisaged that the pivotal study and product would be registrable by late 2011. They recently implanted their 8th patients with DIABECELL, their porcine xenogeneic cell therapy product in clinical trial for type 1 diabetes. The plan is for the product to be manufactured and supplied from New Zealand, where the company is located, for the foreseeable future.

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Progenitor Cell Therapy, LLC has appointed a Vice President of Manufacturing Operations with extensive pharmaceutical production experience to head its two U.S. contract manufacturing facilities. PCT recent announced the recent appointment of Daryl LeSueur as Vice President of Manufacturing Operations. As head of Manufacturing Operations, Daryl is responsible for managing and supervising the day-to-day conduct of the manufacturing, packaging, and operational functions of PCT’s two North American contract manufacturing facilities.

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TheraCell, Inc. aims to develop a revolutionary point-of-care device for isolating and processing stem cells that will facilitate the broad use of autologous stem cells combined with an injectable, highly oxygenated scaffold gel for for advanced regenerative medicine procedures such as spinal fusion. The company recently licensed the underlying technology for the planned device from Yissum Research Development Company of the Hebrew University of Jerusalem Ltd., the technology transfer arm of the University. The technology was invented by a team headed by Professor Dan Gazit from the Faculty of Dental Medicine at the Hebrew University of Jerusalem.

Included in the license was a special scaffold with a particularly high oxygen-carrying capacity, which aims to increase fusion rates by increasing local oxygenation at the surgical site. Scaffolds derived from this technology will act to improve oxygenation and encourage the activity of stem cells and bone-forming cells, or osteoblasts. In preclinical experiments in rodents, accelerated spinal fusion and bone fracture healing were observed, as well as improved stem cell survival. The licensed technology also includes a novel and efficient methods for purifying and manipulating stem cells as well as an innovative, biologically compatible, oxygenated gel that improves bone regeneration and fusion of bone grafts

The company intends to pursue a medical devise status for the combined technologies which would be faster than if it were deems a biologic or combination product.

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In response to Cytori's Request for Designation, Cytori Therapeutics, Inc. (NASDAQ:CYTX) has been informed by the U.S. Food and Drug Administration (FDA) that its Celution(R) 700 System will be regulated as a medical device and reviewed by the FDA's Center for Biologics Evaluation and Research under the law applicable to medical devices. Cytori can now begin to compile and submit a marketing application to the FDA for the Celution(R) 700 System for use as a medical device in "aesthetic body contouring and/or filling of soft tissue voids" - in other words, "breasts". Before they get that far, however, there is still the unresolved issue of whether they will require further clinical trials. Clinical investigations, if required, would be conducted in accordance with the Investigational Device Exemption (IDE) regulations, not the Investigational New Drug regulations applicable to drugs and biologics

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New England Cord Blood Bank, Inc. (NECBB) announced that in August it will begin processing cord blood units using the fully closed and sterile AutoXpress™ System (AXP), developed by ThermoGenesis Corp. and distributed by GE Healthcare. They will, however, continue to operate their manual system, which will allow for processing of samples too small for the automated system. The company hopes that implementation of the automated system will bring NECBB one step closer to accreditation as a public cord blood bank in addition to offering its existing private banking services.

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Advanced Cell Technology, Inc. (Advanced Cell, ACT) (OTC: ACTC) announced that it has filed its Form 10-K for the year-ended December 31, 2008. The Company anticipates filing its Form 10-Q for its first quarter ended March 30, 2009 prior to the end of July. Upon completing that filing, the Company intends to file for relisting on the Over-the-Counter Bulletin Board as its financial statements will be current.

MISCELLANIA

The ERA Consulting Group is proud to announce the next intERActions seminars entitled:

The Regulation of “Advanced Therapy Medicinal Products*” in Europe, from the Clinic to Approval (*Cell Therapy, Gene Therapy and Tissue Engineered Products)

Topics to be covered:
* The EU regulatory framework for Advanced Therapy Medicinal Products (gene, cell therapy and tissue engineered products)
* Opportunities in the EU regulatory landscape, especially for rare diseases: How can they benefit your company?
* Current and future regulatory guidelines: Latest developments and implications for your company and product portfolio
* What are the EU CMC/quality issues/requirements at various stages of development?
* Preclinical development program: EU regulatory expectations - rational approach
* Clinical trials with Advanced Therapy Medicinal Products in the EU

Including Europe in your Strategy to Add Value to Early-Stage Biotech Product Development

Topics to be covered:
* The value of including an EU component into your overall regulatory strategy
* Optimizing interactions with European regulators and how this can aid development and add value
* The benefits of orphan medical product designation in the EU and an explanation of the procedure
* Clinical Development in the EU: The European Clinical Trials Directive
* Opportunities in the EU regulatory framework to add value and reduce time to approval, such as accelerated review, conditional approval or exceptional circumstances
* Compassionate use programs

The seminars will be held at The GreenV Sustainable Center of South San Francisco on 14th – 15th September 2009. For a complete program and registration information, please visit their website at: www.eraconsulting.com.

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Check out Daniel Kraft's TED talk on his MarrowMiner technology being developed by StemCor Systems, Inc.

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After 15 years in the biotech conference industry, the Williamsburg BioProcessing Foundation has shuttered its operation in financial distress. Hasta la vista, Wilbio.

Sign-off

Ok, this is a stretch for inclusion in this blog which is why I leave it to this humble location in the post but two fellow Canadians have been named among the six researchers to receive prestigious awards from the American Society of Hematology later this year. Congratulations to stem cell researchers Connie Eaves and John Dick.







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