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Wednesday, May 6, 2015

An Interview with RegMedNet. Bringing value to the online regenerative medicine community.

As anyone who has followed my activity over any period of time will know, I'm a big fan of the power of social media to power business and careers if not entire industries, trends, movements etc.  I'm a participant (some more, some less) in many different platforms for different purposes (some personal, some business). Consequently when a new online network comes across my radar it piques my interest. 

Recently I had the pleasure of sitting down with Alexandra Thompson who is the Webinar & Community Manager for future science group in the UK to talk about the new online social network they are building around regenerative medicine.  

Describe RegMedNet to us.

RegMedNet is a social network designed especially for the regenerative medicine community, aiming to help them showcase their news, views and insights to the rest of the community, and easily access those of others. By talking to authors and editorial board members of our journal and members of the regenerative medicine field at conferences, we realised there is not really one place where the whole community, including all disciplines and nationalities, could easily connect and communicate: we wanted to design a site that would act like an online, international, multidisciplinary conference, where people can showcase the latest news, share insights, debate hot topics, make connections and learn – but at any time and from any place. 

What is lacking in other online activities or forums that you hope RegMedNet will address?

There are a lot of existing blog sites, websites and social media sites out there already, but with RegMedNet we have done three things. First, we have made it extremely easy to join and use, knowing time is a constraint for many in the community. Sign-up is free and has no lengthy forms or processes, and posting your own content and following others requires only a couple of clicks. Second, we have all the best features of various other sites together in this one network; blog posts, video posts, video chat, following, etc. Lastly, we have structured the site to cut out the noise you can get with social networks, making it easy for you to get the precise content you are interested in. You only get email alerts about people you choose to follow, but you can still visit anyone’s profile and view their content. Also, the site is organised into different ‘channels’ of interest such as ‘Research’ and ‘Industry developments’, so it is easy to find content relevant to you.

How does RegMedNet interact with people’s existing social media activity?

The idea for RegMedNet is that often people have more to say than say a Tweet but less than a published paper, which can also take a fair amount of time from submission to publication and therefore sharing! A lot of people have blogs already, and you can post those on RegMedNet or link over to these existing blogs, so RegMedNet becomes the hub of knowledge for all things regenerative medicine. You can also easily share content from RegMedNet to Facebook, LinkedIn, Google+ and Twitter, so you still get positive feedback into your social media streams.

Are you looking at using RegMedNet to supply additional interactive media content that supplement published papers in the journal?

We certainly are. A disadvantage with the traditional publishing model is the lack of showcasing of large numbers of video or image files. You can not only share these on RegMedNet, but also encourage further discussion of articles. You could show video tours of your lab or institution discussed in an institutional profile, a conference talk to supplement your conference report or a video of a protocol discussed in your research article. The possibilities are endless!

For those new to your site, how would you propose they start?

By signing up of course! Followed by completing their profile to showcase to the rest of the community who they are and what they know. There are various how-to guides in the RegMedNet channel to help users, but I hope that everyone will find the site fairly intuitive as it is designed with ease and simplicity in mind. If members are entirely new to blogging and vlogging, so am I! It is just about sharing what you know or think; as long as you are being respectful to others you can approach both however you like. Of course, as the Community Manager I will always be happy to help by offering advice and answering any questions.

What are you hoping people will say about RegMedNet and how it has helped them?

We have designed the site because we truly believe in the power of regenerative medicine. It is not only a priority investment for the future of healthcare, but offers hope to indications with no cure such as Huntingdon’s disease. Therefore, if people say that RegMedNet is a useful tool helping them to learn, connect and work together, which should help move the field forward more quickly and effectively, we would be delighted. I hope that down the line people say that it has helped international collaboration and standardization, as well as the unification of different disciplines encompassed by regenerative medicine.

What would be a successful first year look like for RegMedNet?

Community sites can take a long time to set up, so if we have a steady number of people signing up and then using the site over the next year we will be happy. By RegMedNet’s first birthday we are consistently getting positive feedback about the network, hearing that it is a useful tool that is helping its members to connect and collaborate etc. as hoped, I will be thrilled.

Friday, April 24, 2015

How to Grow a Regenerative Medicine Industry in Canada: Michael May of CCRM


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

Original Source: Source: George S. Mack of The Life Sciences Report (4/24/15)
"How to Grow a Regenerative Medicine Industry in Canada: Michael May of CCRM"

There's a new kind of incubator in town. The Centre for Commercialization of Regenerative Medicine (CCRM) is a Canadian nonprofit that fosters hands-on association between academia, government, industry and investors to grow stem cell and regenerative medicine companies from the ground up. In this interview with The Life Sciences Report, CCRM President and CEO Michael May guides us through the process that brings ideas out of academia and adds the essential nurturing elements to get startups off the ground and into commercial development. Along the way, he mentions a few names that may interest investors.


The Life Sciences Report: You are the president and CEO of Centre for Commercialization of Regenerative Medicine (CCRM), a Canadian not-for-profit organization supporting development of technologies that will hopefully hasten the journey of stem cell and related therapeutics through regulatory pathways and to the market. How does it work?

Michael May: CCRM was founded through a unique government program called the Networks of Centres of Excellence. It was built around the concept of creating the right kind of ecosystem and network to drive commercialization. The government of Canada invests in three types of networks: academic; commercialization, which is where CCRM fits in; and what is called business-led networks—consortia of established companies that work together in an open innovation model.

CCRM actually followed on an academic network that was around for 14 years. That network had built a very strong culture of collaboration among the stem cell and regenerative medicine scientists in Canada, and prepared them to commercialize their technologies. CCRM was established to integrate elements over and above the discovery phase—access to industry, company creation and access to investors. And now CCRM has built an industry consortium of more than 40 companies from around the world. You could almost envision CCRM spinning off a business-led network one day to take full advantage of the government's strategy for building the right kind of ecosystem for commercialization.

TLSR: Does CCRM recruit innovators to Canada?

MM: Our model is to build a global network and to be a not-for-profit, neutral, safe place to bring together the right resources, elements and leaders, including innovators, to drive technologies forward. We, of course, need to look at a benefit for Canada, but we don't see a benefit to Canada as being mutually exclusive from the benefit to any investor or innovator or stakeholder around the world.

TLSR: How does CCRM differ from other industry advocacy organizations—for instance, from the Alliance for Regenerative Medicine (ARM), which was formed in the U.S. in 2009. I recently spoke with ARM Chairman Edward Lanphier about ARM. Are you familiar with that organization?

MM: Indeed I am. I'm on the executive committee for ARM.

CCRM is not just an advocacy group. It's actually built around tangible facilities and infrastructure that can drive not only technology development for out-licensing to our industry partners, but can also enable company creation in a very different way than has been done here in the past.

We have a tie-in to a network of academics and in-house facilities that develop technologies the way a startup company would. With our founding academic network of Canadian institutions and scientists, we have access to global innovators. We have been able to negotiate access to their intellectual property (IP). When we find IP, and after doing due diligence on it and bundling it with other IP, we can bring that technology into our own facility, where we have a staff of 35 people, including about 20 scientists, who can conduct wet diligence and product development. We fill a gap—we are doing work that academics typically don't like to do or don't always do very well. By bringing together all these capabilities, along with some specialized platforms, like cellular reprogramming, gene editing, cell manufacturing and biomaterial synthesis, we can complement what a standard academic network would bring to the table.

We are also driven by a government mandate to be sustainable, so when we invest in promising technologies or bundles of technologies, we expect to take some equity, or some position, in those opportunities. That way, over time, we can grow and sustain an industry around our network.

TLSR: Regenerative medicine appears to have been left behind in terms of development, compared to other biotechnologies. Why do you think these powerful breakthrough technologies have languished on the lab bench?

MM: The bottom line is that regenerative medicine is complicated. It's an expensive process to take on from start to finish.

Our model at CCRM is built around a couple of assumptions. One is that you have to do more with less these days, and so you need capital efficiency, especially at the very early stages, where investors really have abandoned projects. Although today they say there's a lot of investment in early-stage development, that typically means venture capitalists providing follow-on investment to their current companies. New startups are still having trouble getting financed. Regenerative medicine is also a new industry, so I think this "left-behind" feeling is symptomatic of that fact.

TLSR: You just used an important term: capital efficiency. How do you get around, or solve, this problem of doing more with less?

MM: I think it can be solved through collaborative vehicles. CCRM started with an academic network that was building collaboration and getting access to IP. We then leveraged the reputation of our academic network to attract industry. Then, of course, industry brought its ever-important validation and marketing savvy to the table, which represents expertise that is largely absent in the academic environment.

After three years of operation, with significant deal flow and two networks in place, CCRM is now at a stage where it's building the third key element of its model: an investor network. The system was primed with a very modest amount of money, but now we want to fuel our model with risk capital. Global networks, access to deal flow, sufficient funding and coordination of specialized infrastructure/expertise are all key elements of CCRM’s model for filling the gap between discovery and the market.

Investors are looking for good investments. If we can get companies over some of the early hurdles with unique resources, I think we will demonstrate accelerated commercialization.

TLSR: Michael, is it hard to get investors to invest in science projects? They really need to see the path to commercialization, don't they?

MM: Absolutely. Financiers don't like to take scientific risks. They are more comfortable with commercial risk. Despite what people think, scientists are also risk takers, but they don't like to take financial risk. The role of an entrepreneur is to sit in the middle and manage everyone's comfort or discomfort with different risks. That's an important role for an organization like CCRM and the entrepreneurs that it brings to bear.

TLSR: You have said that Japan and Canada would lead the way in regenerative medicine. Does that imply that the U.S. has lagged?

MM: I would never discount the ability of the U.S. to muster resources and drive innovation in any field. There are strengths in regenerative medicine across the U.S., but regenerative medicine has a unique status in Canada. Stem cells were actually discovered in Canada prior to the bone marrow transplants of the 1960s. Of course, Japan's Shinya Yamanaka received the 2012 Nobel Prize in Physiology or Medicine for discovering that mature cells could be reprogrammed to become "pluripotent." There is a nationalistic element to regenerative medicine in both Canada and Japan. That's one difference.

Japan has taken it one step further. It has taken a novel approach in its regulatory policy by acknowledging that cell-based products may require a different regulatory pathway than drugs or devices. In November 2013, the Japanese Diet (parliament) approved new legislation, the Regenerative Medicine Law, to provide for conditional approvals of cell-based products that demonstrate very strong safety data. These products can go to the market and be sold. Once enough patients have been treated to demonstrate proof of efficacy, then they get full approval. This is a very different model. In fact, if you think about it, if a company can sell its product after Phase 2 development with proof of safety, it changes the return on investment (ROI) model entirely for early-stage programs.

Canada has been the first to approve regenerative medicine products. It was the first country to approve the early skin substitutes from Organogenesis (private) and Advanced Tissue Sciences (Chapter 11 liquidation under bankruptcy in March 2009). Canada was also the first to approve Osiris Therapeutics Inc.'s (OSIR:NASDAQ)(OSIR:NASDAQ) Prochymal (remestemcel-L). Where Canada needs to be innovative now is with reimbursement. None of those products has been reimbursed in Canada.

TLSR: Is CCRM working with regulators on reimbursement?

MM: Yes, it's one of the areas of focus for CCRM. Who is going to pay for a therapy is typically the last question people ask in the development process. We are working with government agencies in Canada so that the reimbursement issue gets addressed early in development. That would put Japan and Canada at the leading edge of innovation in the commercialization of these products.

TLSR: You have referenced Japan's new regulatory legislation. Does Canada propose to do anything like that, so that companies could effectively get a drug on the market with strong Phase 2 safety data?

MM: Canada is not proposing legislation like that—at least that I'm aware of. What the Canadian regulatory authority, Health Canada, has demonstrated in the past is that it will approve cell-based products on a conditional basis, just like Japan. Health Canada is focusing more on risk/benefit, and is therefore flexible. It has also demonstrated that it will approve cell-based products much faster than other jurisdictions. So although Canada doesn't appear to be going down the same regulatory route as Japan in creating a formal Phase 2 approval policy, it is being very clear about its willingness to be open-minded and to be both a cost-and time-effective regulator of these new therapies.

TLSR: Risk/benefit implies efficacy, not just safety.

MM: It does. It also implies that if patients have no other option, then having an experimental therapy available quickly fits into the risk/benefit equation. That's part of the flexibility embedded in the Canadian system. About six months ago Health Canada produced a guidance document for cell-based clinical trials to try to provide not only flexibility in its regulation but also some clarity on what it's expecting in terms of clinical data.

TLSR: Tell me about some of the interesting companies developing regenerative medicine technologies in Canada?

MM: One of our leading company creations (private) concerns the expansion of hematopoietic stem cells from cord blood. Getting the levels of stem cells to treat leukemias in adults, versus children, has been a clinical development goal for some time. We have brought together a couple of technologies from different institutions, along with some bioreactor technology from one of our industry consortium partners, to deliver best-in-class hematopoietic stem cell expansion, gene therapy and potentially the ability to produce mature blood products.

Another area where Canadian researchers have been leading is in cancer stem cells and the thesis that cancer is actually driven by a stem cell that's gone awry. If you can find and target that cancer stem cell, you can target the cancer. One of the companies that CCRM has funded in the past year is Actium Research (private), which is raising money now. Its technology is based on the work of Dr. Mick Bhatia, a senior scientist at McMaster University in Hamilton, Ontario. Actium has created a unique platform to screen molecules that will attack cancer stem cells as opposed to normal stem cells.

TLSR: Is Actium's technology about identifying cell membrane antigens that might be exclusive to a cancer stem cell?

MM: That could be part of it. The company is identifying ways to attack a cancer stem cell versus a normal cell. For instance, if you can push a stem cell to differentiate, you can kill it with standard treatments. Because a stem cell is in a pluripotent state, it's more resistant to drug therapy. There are a number of different mechanisms there, but Actium is a good example of a very young Canadian company that is focused on the country's strength in stem cell research.

TLSR: What about another name? Do you have a public name or two that investors could access?

MM: Yes. RepliCel Life Sciences Inc. (RP:TSX.V; REPCF:OTCQB) is focused on cell therapy for orthopedic and hair restoration applications. It too is a Canadian company, and it has been aggressively developing ties to Japan, in association with its partnership with Shiseido Company Ltd. (4911:TSE), around transplanting cells for hair growth. The company has a diversified portfolio of technologies, is very focused and is growing rapidly as a public company.

TLSR: Many cell therapy companies have not participated in this tremendous biotech bull market of the past few years. RepliCel's stock has done quite well over the past 12 weeks, while most cell therapy companies have not. Do you have a thought on why that might be?

MM: I think, in part, that comes down to the leadership of the company and its understanding of what it takes to not only advance a technology, but to also tell its story. RepliCel has made some key hires in the last year to augment and strengthen its offering. The company has experienced management, and we all know that's a key piece of the puzzle in moving these companies forward.

TLSR: Is there another public company you might mention?

MM: Canadian company Sernova Corp. (SVA:TSX.V) is an interesting name. It has developed a unique vascularized pouch for cells, and it is working in the same area as San Diego-based ViaCyte Inc. (private) in the U.S., which has been funded by the California Institute for Regenerative Medicine. Sernova is working with Dr. James Shapiro, at the University of Alberta in Edmonton, around transplanting islets, or islet-like cells, to treat diabetes. Sernova is focused on the device to transplant those cells.

TLSR: Thanks for your insights, Michael.

Michael May is president and chief executive officer of the Centre for the Commercialization of Regenerative Medicine (CCRM). Prior to CCRM, he was president, chief operating officer and cofounder of Rimon Therapeutics Ltd., a Toronto-based regenerative medicine company developing novel medical polymers that possess druglike activity. May completed his Ph.D. in chemical engineering at the University of Toronto in 1998 as a NSERC (Natural Sciences and Engineering Research Council of Canada) Scholar, and was awarded the Martin Walmsley Fellowship for Technological Entrepreneurship. May sits on a number of boards and advisory committees, including MaRS Innovation, the Alliance for Regenerative Medicine, 20/20 Vision and the Department of Chemical Engineering and Applied Chemistry at the University of Toronto.

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 a list of recent interviews with industry analysts and commentators, visit our Interviews page.

DISCLOSURE: 

1) George S. Mack conducted this interview for Streetwise Reports LLC, publisher ofThe 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) The following companies mentioned in the interview are sponsors of Streetwise Reports: RepliCel Life Sciences 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) Michael May: 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. Senova Corp. and RepliCel Life Sciences Inc. are fee-paying members of CCRM industry consortium. CCRM is a shareholder of Actium Research. 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.
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Thursday, January 22, 2015

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.

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 a list of recent interviews with industry analysts and commentators, visit our Interviews page.

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.

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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Friday, January 9, 2015

Where Do Cures Reside? ARM Executives Think the Answer Is in Regenerative Medicine

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

Original Source: George S. Mack of The Life Sciences Report (01/18/2015)
http://www.thelifesciencesreport.com/pub/na/where-do-cures-reside-morrie-ruffin-and-michael-werner-of-the-alliance-for-regenerative-medicine-think-the-answer-is-in-regenerative-medicine

Where Do Cures Reside? Morrie Ruffin and Michael Werner of the Alliance for Regenerative Medicine Think the Answer Is in Regenerative Medicine

The practice of medicine is being transformed. The journey has been arduous, but revolutionary stem cell, gene and immunocellular therapies are rapidly moving toward pivotal milestones—and investors in the space should strap in for a rewarding joy ride. In this interview with The Life Sciences Report, Alliance for Regenerative Medicine cofounders Morrie Ruffin and Michael Werner consider how new federal guidance might enable new standards of care in cardiovascular, neurologic and oncologic medicine, and offer a preview of next week's Biotech Showcase in San Francisco.

The Life Sciences Report: On Dec. 22, the stem cell and regenerative medicine space saw a very significant development. The U.S. Food and Drug Administration (FDA) published a new draft guidance paper on what constitutes minimal manipulation of human tissues and cells. Although this document is not binding, it does tell us what the agency is thinking. In effect, tissues and cells that have been processed in some way will be treated as drugs, and will come under the regulatory umbrella of the FDA. What does this mean for the industry?

Michael Werner: As sometimes happens with government agencies, major regulatory documents are released right before the holidays. As far as the Alliance for Regenerative Medicine (ARM) is concerned, our member companies are currently looking at the paper and trying to digest it. This draft guidance is open for comment for 60 days after its publication in the Federal Register. We will go through it with a fine-tooth comb, and we will prepare formal comments, which we will submit to the FDA early this year.

Generally speaking, I think the most significant aspect of the guidance is that it provides greater clarity on the FDA's definition of minimal manipulation and, therefore, which products can qualify as minimally manipulated, and which will be regulated as biologics or drugs.

What ARM has said, from its inception many years ago, is that this industry needs a clear and predictable regulatory pathway. The regulations for minimal manipulation have been around for a while, but as this field has expanded, and as the technology has changed, questions have arisen about how the FDA is applying its regulations, and what “minimally manipulated” means in the context of new, tissue-engineered products.

At the very least, it's important that the FDA has, through this document, been transparent about its views.

TLSR: Michael, will you explain further why this transparency is important?

MW: Companies need to know what the FDA is thinking. We've had conversations with individual companies that think the FDA will regulate them as a 361 HCT/P, but are not totally sure, which makes it hard to plan clinical programs. The fact that the FDA has come forward with a document like this is great. As always, the devil is in the details, and we will certainly be going over the document carefully. But published guidelines are a good thing.

TLSR: Could this guidance, in effect, run retail or storefront stem cell therapeutic clinics out of business?

MW: Time will tell. In terms of its impact on what you describe as storefront, I think it really comes down to this: If you are running one of those kinds of operations, or if you perform so-called stem cell tourism, you are now on notice about what kinds of technologies, processes and products are subject to FDA regulation. If you're a storefront clinic, you now know that the FDA not only believes it has jurisdiction to regulate, but how it's going to regulate in the days ahead. It lays the foundation for FDA's enforcement actions going forward.

I think we can expect the agency to use its enforcement discretion in a more consistent way. How aggressive it will be in terms of going after storefront clinics remains to be seen.

TLSR: Do you feel that this, in the long run, is going to be positive for the regenerative medicine and stem cell industry?

MW: I think it will. If you're a product developer, what you want is clarity. You want to know where the goalposts are, and you don't want the goalposts to keep moving. If the FDA is transparent, then that's 80% of the ballgame for the product developer.

Now, in terms of specific policies, the specific ways FDA is going to apply the regulations—that remains to be seen. Companies can—and will—go back to the FDA and say "Here are our thoughts about some of the specifics." Of course, each individual company must go through an iterative process with the agency regardless.

TLSR: It feels like the track to progress, and to the market, for the regenerative medicine industry has been even more difficult than it was for monoclonal antibodies, for lack of a better comparator. What do you think has held the industry back?

MW: One thing has been the clarity of the regulatory pathway. You describe the industry as being held back, but I'd like to say, more positively, that more clarity and predictability in the pathway would enable more products to move forward.

Another key issue involves standards, which has been acknowledged by the FDA and industry as important in terms of enabling more products to come to market. We also need changes to the reimbursement and payment systems, both public and private, to reward innovation. These are different products and models, differentiated by how they treat diseases at the underlying molecular and cellular level, and they could lead not only to treatments, but also to cures.

We need support from the U.S. government in terms of policies, in the same way that governments in Japan, the United Kingdom and elsewhere around the world have been enacting policies specifically designed to support the sector. The U.S. government needs to do the same.

The field is now poised to make a significant impact on healthcare. It's hard to say what's held the field back, but reimbursement is certainly a challenge that we need to get our hands around, to make sure the sector reaches its potential.

TLSR: Morrie, did you want to comment? The cell therapy/regenerative medicine sector has been in development now for about two decades-plus, yet I see only a couple of stem cell companies with a market cap above $1 billion ($1B), and all the rest are in the penny-stock to $500 million category. Why have cell technology companies lagged biotech?

Morrie Ruffin: We actually don't see it that way. That's one of the things we will show in the data that will be coming out in our Regenerative Medicine and Advanced Therapies State of the Industry Briefing on Jan. 12 at the Biotech Showcase in San Francisco.

Clearly, as in any sector, there are going to be successes and failures, but we do see a very significant and recognizable upward trend in the amount of money being raised in the sector. The cancer immunotherapy space is drawing a lot of interest right now.

But if you look across the board at cell therapy, gene therapy and immunocellular therapy companies, we count nine to 10 public companies with $1B-plus market caps in the sector right now. And there are obviously a number of private companies raising significant amounts from private investors as well.

TLSR: Morrie, are we on the verge of seeing pivotal data come out of companies in the sector?

MR: We are about to see a number of very significant events in the sector. Clearly, both those working in the industry and investors are looking for positive clinical events—milestones—that signal we are making progress in the clinic, and that we understand how we might commercialize these therapies. Over the next year or two, you will see a number of very significant data events around cell therapy. We are already seeing this in the oncology space, which is why there is so much interest in what's happening with cell-based or immunotherapies. This has led to a significant interest in cellular therapies across the board. Investors are going to hear a lot in the near future, as people begin to understand the clinical milestones that are going to drive this sector over the next 12–18 months.

TLSR: What do you think is the greatest obstacle to this industry moving forward? Is that issue being addressed currently?

MR: One of the challenges in this space—and I think this is to be expected in any evolving sector—is the issue of commercialization, which concerns manufacturing, scale-up and all the things required to make these new products available and successful. I believe that we have made tremendous progress just in the last few years in understanding what this will take, whether it's an allogeneic therapy or an autologous therapy. I think this is one of the reasons we see manufacturing and tool companies making significant investments in the sector. Companies like GE Healthcare (a unit of General Electric Co. [GE:NYSE]), Thermo Fisher Scientific Inc. (TMO:NYSE), the Lonza Group AG (LONN:SIX; LO3:FSE; LZAGF:OTCPK) and others are heavily engaged in this space. They see the huge opportunity here.

The cell therapy companies also understand that infrastructure is going to be an important part of how these therapies are brought to the market, and to the patient. One of the things we have been anticipating is having the processes of manufacturing, handling, and scale-up catch up with the lab bench science, and then duplicating that science on a large, profitable scale. I think we are on the cusp of that.

TLSR: Michael, you raised a point about government being involved in regenerative medicine, and you mentioned Japan, which has a new regulatory pathway in place whereby companies, in effect, have to show safety and the equivalent of Phase 2 proof of concept to get conditional approval for a cell therapy. Do you see the U.S. doing anything like that?

MW: I'll put it this way: I think it's possible the U.S. will take steps analogous to what other countries are doing to support the field. Is the FDA going to give conditional approval if a company shows certain kinds of safety data? I'm not sure about that, but I do think it is possible to take steps to smooth the pathway forward. For instance, we've talked with the FDA about standards in product development and manufacture. This is an idea that the FDA itself has said is critical to its product review process. Other ideas could include expedited approval programs. Such a program was recently created in the U.S. to support development of antibiotics.

The question is whether the kinds of programs that already exist can be applied or adapted to regenerative medicine, cell therapy and gene therapy products. I think you're more likely to see changes in existing U.S. policy, rather than copying Japan's policy or copying what other countries are doing.

TLSR: What do you think will be the motivating factor for policymakers to accelerate cell therapy development?

MW: There is more recognition that this sector could, potentially, cure diseases that have gone untreated or have been deemed incurable. In diseases like stroke or heart disease, we are basically treating symptoms. We are certainly helping people, but we are not actually curing disease. We're not getting people all the way back to health.

I think there's a greater recognition on the part of policymakers, as well as others, that regenerative medicine—and we use that term very broadly—is where cures and treatments are going to reside. Therefore, you're starting to see more acknowledgment of that. The U.S. government needs to make sure there are policies in place that will allow this industry to thrive.

TLSR: Morrie, you and Michael are going to be at the Biotech Showcase in San Francisco. What will you and ARM members be doing for attendees?

MR: One of the things we're doing, at the kick-off for the showcase at 8 a.m. on Monday, Jan. 12, is presenting our Regenerative Medicine and Advanced Therapies State of the Industry Briefing. This will be our fifth briefing at the Showcase. In the two-hour briefing, we will provide information on the performance of the sector. We will look back at 2014 and 2013, highlighting the progress made in the sector. Then we will look forward to what we anticipate to be major milestones and major events over the coming two years.

TLSR: Will there be an emphasis on any particular topic, or will it be a very broad briefing?

MR: We will be talking about several different things, but we plan to spend a lot of time talking about the progress being made in the gene therapy sector and genetically modified cell therapies.
When we talk about advanced therapies in regenerative medicine, we are being inclusive of all the companies working in the in vivo and ex vivo gene therapy areas, whether that's cancer immunotherapy, or CAR (chimeric antigen receptor) T-cell receptors, or other strategies being employed to harness the immune system to attack cancer. This will be a big part of the briefing. We will also look forward to a number of the major data events that we anticipate in 2015, in a number of indications, including stroke, cardiovascular disease and a number of neurodegenerative disorders.

TLSR: Thank you both for your insights.

Morrie Ruffin has more than 20 years of experience in the biotech and healthcare industries. He is a founder and managing director of the Alliance for Regenerative Medicine, the global organization representing the interests of the regenerative medicine community. Ruffin is also the managing partner of Adjuvant Partners, a boutique regenerative medicine and advanced therapies business consulting firm. Prior to joining Adjuvant Partners, he was the chief executive officer of LifeTech Innovations LLC (LTI), a business development consulting firm. Prior to his position at LTI, he was executive vice president of capital formation and business development at the Biotechnology Industry Organization (BIO), the largest trade organization representing the biotech and drug development industries. Prior to joining BIO, Ruffin worked for U.S. Senator Arlen Specter for five years as his senior legislative assistant. He received his master's degree in international studies and economics from the Johns Hopkins School for Advanced International Studies and his bachelor's degree from the University of Virginia.

Michael Werner is a partner in Holland & Knight's Washington, D.C. office. He has almost three decades of healthcare law, lobbying, regulatory, reimbursement and policy development experience in Washington. He is also is the cofounder and executive director of the Alliance for Regenerative Medicine, a Washington, D.C.-based organization whose mission is to advocate for federal funding, regulatory and reimbursement policies that will advance regenerative medicine research and product development. Before joining Holland & Knight, Werner was president of The Werner Group, a Washington, D.C.-based firm that provided lobbying, regulatory, and bioethics consulting services for biotechnology and pharmaceutical companies, physicians, health plans, investors, and patient advocacy groups. Prior to founding The Werner Group, he was chief of policy for the Biotechnology Industry Organization (BIO), representing over 1,000 biotechnology companies in the U.S. and other countries. Werner is also a founding member of the Board of Directors of the Coalition for the Advancement of Medical Research. He was senior healthcare advisor to U.S. Senate Majority Leader George Mitchell, a congressional investigator for the U.S. Senate Special Committee on Aging, and senior advisor to Maryland Governor William Donald Schaefer. Werner is a frequent media commentator and has appeared in the Wall Street Journal, Science, Scientific American, the Washington Post, BIOWorld, Congressional Quarterly and the Baltimore Sun, as well as on many TV and radio news programs. In 2013, he was named one of the Top 50 Global Stem Cell Influencers by Total BioPharma.

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) The following companies mentioned in the interview are sponsors of Streetwise Reports: None. 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) Morrie Ruffin: 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) Michael Werner: 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.
5) Interviews are edited for clarity. Streetwise Reports does not make editorial comments or change experts' statements without their consent.
6) 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.
7) 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


Tuesday, December 23, 2014

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.
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