Wednesday, October 22, 2014

Japan Gives a Hug to the Cell Therapy Industry

Japan Gives a Hug to the Cell Therapy Industry: R. Lee Buckler

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

http://www.thelifesciencesreport.com/pub/na/japan-gives-a-hug-to-the-cell-therapy-industry-r-lee-buckler

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

TLSR: Thank you for your time.

RLB: Many thanks to you.

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

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

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DISCLOSURE:
1) George S. Mack conducted this interview for Streetwise Reports LLC, publisher of The Gold Report, The Energy Report, The Life Sciences Report and The Mining Report, and he provides services to Streetwise Reports as an independent contractor. He owns, or his family owns, shares of the following companies mentioned in this interview: None.
2) R. Lee Buckler: I own, or my family owns, shares of the following companies mentioned in this interview: RepliCel Life Sciences Inc. I personally am, or my family is, paid by the following companies mentioned in this interview: RepliCel Life Sciences Inc. My company has a financial relationship with the following companies mentioned in this interview: None. I was not paid by Streetwise Reports for participating in this interview. Comments and opinions expressed are my own comments and opinions. I determined and had final say over which companies would be included in the interview based on my research, understanding of the sector and interview theme. I had the opportunity to review the interview for accuracy as of the date of the interview and am responsible for the content of the interview.
3) The following companies mentioned in the interview are sponsors of Streetwise Reports: RepliCel Life Sciences Inc., Athersys Inc. Mesoblast Ltd. is not affiliated with Streetwise Reports. The companies mentioned in this interview were not involved in any aspect of the interview preparation or post-interview editing so the expert could speak independently about the sector. Streetwise Reports does not accept stock in exchange for its services.
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Tuesday, October 21, 2014

Follow the Baby Boom to Profits

Follow the Baby Boom to Profits: Agility Health's Steven Davidson

http://www.thelifesciencesreport.com/pub/na/follow-the-baby-boom-to-profits-agility-healths-steven-davidson

In recent years, entrepreneurial therapists have comprised the fast-growing sector in outpatient physical therapy. But companies like Agility Health are beginning to seize acquisition opportunities and market share in this growing sector. Steven Davidson, CEO of Agility Health Inc., tells The Life Sciences Report how his company is innovating in the physical therapy space, improving patient care and clients' bottom lines through its management expertise and proprietary software, AgileRPM. With baby boomers aging and the Affordable Care Act increasing access to services, physical therapy just might be the safe haven sector to shield your portfolio from macroeconomic uncertainty.

The Life Sciences Report: What is the structure of the national physical therapy market?

Steven Davidson: Physical therapy is a $31 billion ($31B) market. During the last 20 years, the sector has steadily experienced a 6% annual growth rate. One reason for the growth is that baby boomers are getting older. But in addition to that, there is a strong national trend toward evidence-based medicine. The market is sorting out what types of services provide the best value for the dollar. Studies report that rehab is a cost-saver, especially when compared to surgery and other procedures. For example, early therapeutic intervention for workers' compensation claims saves as much as 79% of the cost versus waiting before starting rehab. These quantifiable outcomes bode well for the financial health of the industry.

TLSR: What is the acquisition history of your company, Agility Health Inc. (AHI:TSX.V)?

SD: Physical therapy has long been a fragmented industry. Even today, the top four players represent only 9% of the overall market. Agility Health started in 1968. In 2003, my business partner and I engineered a management buyout. We self-funded acquisitions until 2010, then we took on some leverage to do eight buys. One of our two key growth strategies is to continue to acquire small- to medium-size outpatient clinic groups with two to four clinics. That is our sweet spot: It is big enough to be meaningful, but too small for the much larger players. We tell the clinic groups that we will buy a super majority of the business. "We want you to continue to manage it," we tell them. "And we will absorb human resources, revenue cycle management, IT services, practice management software and accounting functions."

TLSR: What is your other key growth strategy?

SD: Our other key strategy is to target organic growth in our hospital business line. Partnering with hospitals in joint ventures is a big part of our strategy. We are talking with three hospital systems right now about collaboratively managing their hospital services. We also have a proprietary software system designed to manage hospital-based rehab businesses. That software, combined with our management expertise, improves the quality of service and overall performance metrics for all our clients.

TLSR: What portion of the outpatient business is workers' compensation-related?

SD: Worker's comp is less than 10% of our business. Most of our accounts receivable are for traditional insurance product—health management organizations, preferred provider organizations. About 20% is Medicare.

TLSR: Has the Affordable Care Act impacted your sector?

SD: It has generally affected our space positively. The Affordable Care Act has quietly improved healthcare access for some folks who did not have such access in the past. It has also reinforced the evidence that rehab is a service that is required for all insurance plans.

TLSR: What makes Agility Health unique in your industry?

SD: Most of our competitors stick to providing just one or two service lines. For example, U.S. Physical Therapy Inc. (USPH:NYSE) sticks to outpatient clinics and industrial rehab. Kindred Healthcare Inc. (KND:NYSE) sticks to the nursing home business and outsourcing to hospital and nursing homes. Concentra Inc., which is a subsidiary of Humana Inc. (HUM:NYSE), focuses on occupational medicine and rehabilitation. Agility Health provides all these services and more. We are in the acute care hospital business, outpatient services, senior care, long-term care, orthotics and industrial rehab, not to mention the software business.

When we acquired the company in 2003, our goal was to diversify geographically. Originally, most of our business was in Michigan, and now less than half of it is in that state. Healthcare services in the U.S. are very decentralized and regional. For example, here in Michigan, the system in Grand Rapids is very different from the healthcare environment an hour away in Kalamazoo.

Geographical diversification is like creating a stock portfolio that spreads the risks and benefits over a large space. We provide all these services, and then we tie them together with software and management policies. We are the only rehab company that has developed its own propriety practice management software.

TLSR: Are you putting the proprietary software on the market?

SD: We have just announced that we will begin selling the AgileRPM software service early next year to hospitals and outpatient clinics.

TLSR: Who are your major clients?

SD: In the national hospital business, we work with large providers such as Trinity Health Corp (private). We service a lot of regional healthcare companies. On the industrial side, our long-term clients include Ford Motor Co. and Pratt & Whitney, which is a subsidiary of United Technologies Corp. (UTC:NYSE). We have American Airlines Group Inc. (AAL:NASDAQ) and Medtronic Inc. (MDT:NYSE). In the orthotics business, we are the exclusive provider for the Women's Tennis Association, for which we make handcrafted orthotics.

TLSR: Do you contract directly with Ford?

SD: Like many large corporations, Ford Motor is self-insured. We have physical therapists located inside Ford's plants who provide direct rehab services in their assembly facilities. We provide a broad range of preventative and rehabilitation services for our manufacturing clients. We provide ergonomic assessments, early intervention on injuries, and coaching on how to do the job properly without getting injured. Our goal is to reduce the number of workers' compensation claims and promote good health practices.

TLSR: Are you taking on debt to support your revenue growth curve?

SD: We have taken on debt to support acquisitions, as well as a bit of equity. Moving forward requires a combination of debt, equity and internal cash flows. We went public last October to accelerate growth. This created an opportunity for us to reward our 1,100 employees with a stock purchase plan and managers with stock options. Our clinicians are our key asset. It is smart to provide them with stock options and stock bonus incentives.

TLSR: What was your operating revenue last year?

SD: Our revenue in 2013 was $63 million ($63M).

TLSR: Are you operating at a net profit?

SD: We are EBITDA (earnings before interest, taxes, depreciation and amortization) profitable, but not yet net income profitable, which is a near-term goal, of course. Our adjusted in 2013 EBITDA was roughly $3M.

TLSR: Are you going to continue to make acquisitions to grow revenue?

SD: Our target is to grow revenue by 25% per year, with roughly 20% of the 25% through acquisitions of small outpatient clinic groups. Each acquisition has its own twist, but we look to finance a buy with a combination of 50–60% cash and the balance in vendor take-backs or seller debt. We are looking to acquire clinic groups in the 4–5X range on a multiple of trailing 12 months EBITDA.

TLSR: Do you structure acquisitions as partnerships?

SD: Our goal is to partner with sellers. These entrepreneurs retain 20–30% as a synthetic or real equity for three to five years.

TLSR: Is there a limit to Agility's growth?

SD: Our goal is to become one of the largest physical therapy operators in North America.

TLSR: Who are your main competitors?

SD: U.S. Physical Therapy is New York Stock Exchange-listed. It is the best pure-play rehab comparable for a public company. Select Medical (SEM:NYSE ) is the largest provider of rehab in the country, with numerous hospital assets. Gentiva Health Services Inc. (GTIV:NASDAQ) provides predominantly post-acute healthcare. Kindred Healthcare is one of the more aggressive acquirers. It is in the long-term care and the post-acute care business. It also has substantial nursing home assets. On the whole, there is a very dynamic and competitive acquisition market. Unlike resource stocks, macroeconomic events do not generally affect our industry.

TLSR: Is the corporate future of physical rehab emerging from these acquisitions, or will there still be a majority of mom-and-pop operations?

SD: There is more consolidation now than there has been in the past. Running a rehab clinic is becoming more difficult. Fewer people are jumping into the industry. There is a lot of private equity money in the space. There is no single emerging leader; everybody wants to consolidate to grow.

TLSR: How has Agility Health's stock fared since your initial public offering?

SD: We came out at $0.20/share. We peaked at $1/share. We are at about $0.20/share today. Our business management team and our directors are very bullish. A number of us have been actively buying our own stock. It is at a good value, and we are all quite positive about the long-term prospects.

TLSR: As you continue to make acquisitions, will there be splits in the stock?

SD: Because we are a growth company, most of our energy is focused on creating shareholder value and reinvesting.

TLSR: What catalysts could put you into the net income profitability?

SD: We are aggressively shifting our energy toward our higher margin business lines. Our second quarter was 10% EBITDA over revenue, and our long-term vision is to be over 10%. We have been reengineering within the organization to improve our overall margins, by acquiring high-margin businesses and picking up high-margin contracts.

TLSR: Where would you like to be in five years?

SD: I see Agility Health with revenue of $100M within a year or two, and $300M within five years.

TLSR: What business weaknesses do you need to address to get to that goal?

SD: We need to make sure that we aren't capital-constrained. That is one reason why we went public. We aim to keep our executive talent, adding more of that talent as we grow by acquiring organizations that share our corporate culture.

TLSR: How do you describe your corporate culture?

SD: Our culture is a major asset. We started with one therapist in 1968. Our managers are clinically oriented, with strong healthcare backgrounds. Our clinical focus makes us unique in the rehab marketplace. People looking to sell their clinical businesses tend to migrate toward our strong, clinically based culture because they are comfortable with our priorities.

TLSR: Thank you, Steven.

SD: You are welcome.

Steven Davidson is CEO of Agility Health, where he directs the company's care delivery and quality initiatives, financial performance and strategic acquisitions. Davidson has served as CEO since 2003, when he and partner Ken Scholten led the management buyout of Agility Health's predecessor, GNA. Davidson's 14-year tenure at GNA included a series of leadership positions covering regional business development. Davidson has also served in leadership roles at Health East Corp. and PT Management Consultants, where he provided specialized rehabilitation consulting and contracting services to nursing homes and hospitals across a five-state region. Davidson is a licensed physical therapist and member of the American Physical Therapy Association. He earned a bachelor's degree in physical therapy from the College of St. Scholastica and a master's degree in business administration from the University of Chicago.

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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 owns, or his family owns, shares of the company mentioned in this interview: None.
2) Agility Health Inc. paid Streetwise Reports to conduct, produce and distribute the interview.
3) Steven Davidson had final approval of the content and is wholly responsible for the validity of the statements. Opinions expressed are the opinions of Steven Davidson 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.

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Sunday, October 19, 2014

Amicus Therapeutics (FOLD) Announces Additional Positive Phase 3 Data Late Sunday Night

Amicus (FOLD) announced additional positive phase 3 results from its Phase 3 trial in Fabry disease using its lead compound Migalastat late Sunday night. I've provided an insert of the release below and expect shares to move up Monday morning due to the additional positive data. Across All Subgroups, Patients Treated with Migalastat Compare Favorably to Natural History on Kidney Function (eGFR) Additional GL-3 Data Further Validate Assay for Identifying Patients with Amenable Mutations CRANBURY, N.J., and SAN DIEGO, Oct. 19, 2014 (GLOBE NEWSWIRE) -- Amicus Therapeutics (FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today announced additional positive data from a Phase 3 study (Study 011) of the oral small molecule chaperone migalastat HCl ("migalastat") in Fabry disease patients with amenable mutations. In a poster at the American Society of Human Genetics (ASHG) Annual Meeting, Daniel G. Bichet, M.D., M.Sc., Professor, Department of Physiology, University of Montreal, presented results from patients in Study 011 including those who continued on migalastat in an open-label extension (Study 041). Assessment of kidney function by various measures of glomerular filtration rate (GFR) for patients receiving migalastat in Study 011 for at least 18 months and continuing migalastat treatment in Study 041 showed continued stability of kidney function for an average of 32 months. Decline in kidney function is a key cause of morbidity and mortality in patients with Fabry disease. Measured (iohexol) GFR (mGFR) showed stability over 18-24 months in Study 011 but was not collected in Study 041; mGFR was previously reported with topline Study 011 results. Mean Annualized Change in GFR (ml/min/m2/yr) (SEM) Over an Average of 32 Months with Migalastat in 011 and 041 Estimated GFR (eGFR) (CKD-EPI) (n=41) -0.20 (0.60) eGFR (MDRD) (n=41) +0.63 (0.08) Stratifying patients for gender and baseline proteinuria demonstrated that patients treated with migalastat experienced less decline in kidney function than untreated patients from a previously published natural history study1. Dr. Daniel Bichet, Full Professor and Section Head, Renal Function & Transport Physiology, University of Montreal, said, "Baseline proteinuria levels are among the most predictive indicators of disease prognosis and kidney function decline in Fabry patients. The data presented today show that when comparing patients with similar levels of proteinuria, patients treated with migalastat are more stable in their kidney function versus untreated patients. These results are very encouraging for migalastat as a treatment for Fabry patients with amenable mutations." Data from a subgroup analysis comparing the change in GL-3 substrate levels between amenable patients and non-amenable patients based on the GLP HEK cell assay provided additional validation of the sensitivity of the GLP HEK assay for identifying patients who will respond to migalastat monotherapy. Overall, patients with amenable mutations had declining levels of GL-3 when treated for six months with migalastat. In contrast, patients with non-amenable mutations had no change or increasing levels of GL-3 after six months of migalastat treatment. "We are pleased to present these additional Phase 3 results in a scientific forum. With 32 months of data, Fabry patients treated with migalastat exhibit long-term stability in their kidney function. These results contrast with the decline in kidney function reported in natural history studies. Decline in kidney function is one of the primary causes of morbidity and mortality in Fabry patients," stated Dr. Jay A. Barth, Chief Medical Officer of Amicus Therapeutics, Inc. "The additional data on substrate reduction show that we can accurately identify patients who may benefit from migalastat. We look forward to meeting with regulatory agencies starting this quarter as we work to make migalastat available for all amenable Fabry patients as quickly as possible."