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.

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

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.

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

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

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

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

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

101 Second St., Suite 110

Petaluma, CA 94952

Tel.: (707) 981-8204

Fax: (707) 981-8998 

Email: jluther@streetwisereports.com

Reprint from LSR: How RepliCel Is Harnessing the Awesome Power of Cell Therapy

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

TLSR: Do you have evidence of actual tendon regeneration?

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

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

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

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

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

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

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

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

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

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

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

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

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

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

RLB: Thank you.

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

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

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

DISCLOSURE: 

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

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

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

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

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

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

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

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

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

101 Second St., Suite 110

Petaluma, CA 94952

Tel.: (707) 981-8204

Fax: (707) 981-8998 

Email: jluther@streetwisereports.com