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Friday, August 6, 2021

Foreign company pursuit of cell therapy commercialization in Japan: strategies and considerations

 I'm thrilled to announce that I will be presenting at the upcoming 5th Annual Cell & Gene Therapy World Asia, live streaming on 15th and 16th September 2021.

I will be presenting on "Foreign company pursuit of cell therapy commercialization in Japan: strategies and considerations" at 9:30AM (Singapore Time) on 16th September.

My presentation will cover the following topics:

·       Unique constraints and opportunity for regenerative medicine in Japan

·       Practical strategies for building a virtual team to support real progress

·       PMDA conditional approval -- not the only expedited commercialization pathway in Japan

In preparing for this presentation, I had opportunity to speak with Gladys Koh of IMAPAC to discuss

1.    key trends shaping the cell therapy industry moving forward in a post-pandemic world in Asia
2.    exciting technologies or developments that have caught my attention in the past year
3.    thoughts on how regulations pertaining to cell therapies can be evolved to allow the progress of the industry
4.    current struggles with respect to commercialization of cell therapies and how would they might be overcome
5.    key challenges this industry faces and is currently trying to overcome
6.    developments should the industry look out for from RepliCel Life Sciences Inc. in the coming year
8.    some of the key takeaways the audience can expect from my presentation

To view the interview, https://youtu.be/p2oZPbgFkMQ


Click here and here for a written advance peak at a few thoughts from some of the following speakers involved in the event such as:

Gregory Fiore, President & CEO, Exacis Biotherapeutics, USA

Zhimei Du, Global Head Process Cell Sciences, Cell/Gene Therapy, Merck & Co, USA

William Hung, Assistant General Manager, MariaVon, Taiwan

Hardy TS Kagimoto, Chairman & CEO, Healios, Japan

Pascal Touchon, President & CEO, Atara Biotherapeutics, USA 

Joy (Shuxia) Zhou, Head of Drug Product Development, Cell Therapy, Takeda, USA

Magali Taiel, Chief Medical Officer, GenSight Biologics, France

Andreas Weiler, Global Head of External Supply Cell & Gene Therapy, Novartis,  Switzerland

Seong-Wook Lee, President & CEO, Rznomics Inc., Korea

Yu Zhang, SVP & Chief Scientific Officer, VCANBIO Cell & Gene Engineering, China

Shi-Jiang (John) Lu, President and CEO, HebeCell Corporation

Devyn Smith, CEO, Arbor Biotechnologies, USA

Lee Buckler, President & CEO, RepliCel Life Sciences Inc.



Thursday, July 15, 2021

Cell and gene data innovation and multi-stakeholder data transfer

Hello readers. It's been awhile. I'm pleased to be working with Reuters on some events including this upcoming webinar on how to utilize RWD in cell and gene therapies with speakers from Novartis, BMS and Salesforce. Click here for more info.

The requirements of complex and delicate value chains within cell and gene therapies (CGT) is something pharma has traditionally never seen before. Leaders in CGT are innovating data sharing approaches by utilizing RWE and RWD across the value chain, and achieving multi-stakeholder alignment, to ensure commercial success.












Join C&GT delivery trailblazers BMS, Novartis and Salesforce to learn how to turn data in to true insights: that enhance the science, reduce the cost of deliver, improve commercial decision-making and elevate the patient experience.

Sign up for the webinar “Cell and gene data innovation and multi-stakeholder data transfer” here.

When you join, you will learn how to:

                 Reimagine the technology: digitally transform existing data integration models for cell and gene therapies

                 Reach for the future together: develop a multi-stakeholder strategy collaboratively, that aligns on needs and capabilities

                 Cross-capture and utilize data flow from R&D through to launch to win the marketplace with science and data

                 Prepare for and address the major barriers to success: such as infrastructure non-readiness, digitally siloed stakeholders, underdeveloped partnerships, and data illiteracy

                 Turn data in to true insights: that enhance the science, reduce the cost of deliver, improve commercial decision making and elevate the patient experience

Tune in live to the free webinar on July 29 at 11am ET/4pm BST, or sign up to receive the recording here.

Please also feel free to share with your colleagues working in C&G therapy, so they can benefit too. 

Lee

 

Tuesday, January 24, 2017

Will 2017 be the year of Cell Therapies?

Here is the intro to a piece I published earlier this week on the Huffington post (click here for the whole article)

While 2016 may not go down as a lot of people’s favorite year, one development most people will agree to put a positive check mark beside is the near-unanimous passing of the 21st Century Cures Act in the United States.

In the last breaths of its tenure, the Obama Administration was able to bring both sides of Congress, the patient community and industry lobbyists together in a consensus on what was needed to drive better, faster medical innovation in the years to come.  Faster cures to future patients.

There are a lot of reasons to be pleased with the many provisions of the 21st Century Cures Act but as the CEO of a cell therapy company, RepliCel Life Sciences Inc., I’ll focus this brief post on the area in which I’ve dedicated most of my professional life.

Out of the spotlight of the more prominent provisions of the Act, is a section pertaining to what I predict will be a growing but eventually radical boost to the development of regenerative medicines such as “cell therapy, therapeutic tissue engineering products, human cell and tissue products, and combination products using any such therapies or products”.

Regenerative medicine is the idea that we can use cells or tissue from a patient or donor and do something more therapeutically significant for patients suffering from disease or conditions than we can do with drugs or other therapies.  This next pillar of medicine dares to cure not just slow disease progression or reduce symptoms.

The 21st Century Cures Act promises not only foster the development of such therapeutic products but presents, I believe, exciting reasons to re-look at investing in cell therapy and other regenerative medicine companies.  

 Here are four such reasons:

Click here for the remaining article on Huffington Post

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. 
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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.

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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.
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