1. Why the Paradigm Shift at the FDA, over the Past 5 Years?

During past month, many important life science events were FDA driven : Dendreon (DNDN) with the Provenge approval, Intermune (INTM) with Pierfenedone’s rejection, the discontinuation of Merck’s biosimilar EPO program after the company received feedback from the agency, NicOx failed to gain FDA panel’s endorsement for Naproxcinod.. Hence this FDA SPECIAL of Biotech in the Money. Marina Bozilenko and Ed Allera (DC Attorney, FDA expert) discuss the tremendous change at the FDA in the past 5 years, as it underwent a complete philosophical change. Contributing factors are the Medicare Monetization Act (MMA, Medicare Part D), the change of control of Congress to the Democrats, the Food & Drug Amendments Act, the new (Obama) Administration and its Healthcare Reform. Ed argues that the perception of government was that “the pendulum had swing too far to the approval side.” Now, in the post-Vioxx-era, with Congressional oversight on the FDA, there is a much greater risk aversion.

2. Is Anything Binding With the FDA Anymore, or Are Companies Just Not Listening?

One of the biggest complaints companies have with FDA is that it changes regulations on a whim, which results in missed timelines and significant increases in clinical development spending. FDA’s primary complaint is that companies dont listen. Ed Allera explains FDA gives nuanced answers, and too often, companies hear what they want to hear. Breaking new ground (with FDA) in this day and age is very difficult. Is the FDA’s fault or the companies’ fault? Ed argues companies should learn to listen, and since FDA is “holding all the cards” it is healthy to get as many people as possible (Congress, key opinion leaders, media) looking at the trial to add more transparency and accountability to the FDA reviewal process. Ed also comments on the recent Dendreon Provenge approval.

3. Chronic Disease vs. Critical Needs: the FDA Fast Track, and Recourses for Companies

Is there still such a thing as “significant clinical need” or is everything a “chronic disease” now? While it used to be easier to develop drugs for cancer or infectious diseases, that doesn’t seem to be the case anymore. Are there any legal recourses to an “unfair” FDA decision? Can companies sue the FDA? How does sunlight bring sanity?

4. Biosimilars are Without a Champion at the FDA

Will all the talk about biosimilars finally result in concrete guidelines? Ed Allera explains that every initiative needs a champion, and that biosimilars have never had their champion inside FDA. Is there a chance they will?

5. FDA Very Actively Enforcing Compliance Regulation

The FDA has been very actively enforcing compliance. Curiously, FDA inspections are more stringent in the U.S. than abroad. Ed Allera talks about the FDA;s “vendetta” against pre-natal vitamins, and the failure of their transparency initiative.

6. If You Had Unlimited Powers, How Would You Reform the FDA?

If the FDA were to be reformed, Ed Allera would start by ensuring a more fair risk benefit calculation (current concerns being over rare events that are hard to detect), and re-initiating the dialogue on what is acceptable risk. As we move to more sophisticated technology platforms that can gather information on adverse events FDA should leverage this data. Additionally, Ed believes that the effort to control the message drug companies put out is ill considered given the amount of information that is available to patients online.

7. Combination Therapy in the Eyes of the FDA. Can We Let FDA Off the Hook? The System Return to a More Rational Process

Does FDA understand the critical need for combination therapies, and will they help the industry get some of these approved? Ed Allera believes FDA has an abstract understanding of combination therapy, and is being cautions because “they’ve already gotten beaten up on antibiotics.” Ed argues that given the amount of off label prescriptions and phase 4 breakthroughs, FDA could be held to a less stringent critique if something does go wrong. Ed also brings up the issues of demonstrating value add and comparative effectiveness. To the question “is there any good news?” Ed answers that the state of the current regulatory environment is just a phase, and that as more people raise issues, the system will return to a more rational process.

This interview was conducted at the NASDAQ Marketsite, on May 26th, 2010, in New York City.

Featuring:

• Marina Bozilenko, Executive Producer, BioBusiness.TV
• Edward John Allera, Chairman, FDA/Biotechnology Section, Buchanan Ingersoll & Rooney

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. DNA Vaccines, a Fast and Efficient ‘Plug-N-Play’ Technology

Vical uses closed loops of DNA (called plasmids or pDNA) as a DNA delivery technology, useful for protein therapy, immuno-therapy, cancer vaccines, and infectious disease vaccines. DNA vaccines use portions of the genetic code of a pathogen to cause the host to produce proteins of the pathogen that may induce an immune response. Vical CEO Vijay Samant explains the technology is ‘plug-n-play’ : it is extremely fast, and requires only the sequence for a pathogen (not the actual pathogen).

2.Allovectin-7, a Phase 3 Cancer Immunotherapy for Head & Neck Tumors

Vical’s lead compound, Allovectin-7, is a Phase 3, cancer immunotherapeutic, first-line treatment for metastatic melanoma. For its phase 3 trial, Vical has selected patients with normal LDH levels, an intact immune system (chemonaive), and no brain or liver metastases. The primary endpoint secured in a Special Protocal Assessment (SPA) with the FDA is response rate at 6mo. The secondary endpoint is survival. The recent approval of Provenge, Dendrion’s prostate cancer vaccine, has helped buy-side investors look at cancer immunotherapy as a reality, and has paved the way with regulators. Allovectin-7 is partnered with Japanese company AnGes in Asia for Head & Neck solid tumors. AnGes is covering the cost of the Phase 3 trial. Vical has retained the US and EU rights. The next milestone expected for Allovectin-7 is a safety review, by year end.

3.TransVax, a Phase 2 DNA Vaccine to Prevent Cytomegalovirus (CMV) Reactivation

Cytomegalovirus (CMV) is a nasty little bug that comes from the herpes family. 80% of people are infected by age 40 in the US. CMV hides in a synergistic balance in the immune system. When the immune system is compromised, it attacks multiple organs and can be deadly. In the immuno-suppressive state, the trial is in bone marrow transplant, where TransVax vaccine teaches the immune system to control the CMV virus. The data for bone marrow could be applicable to liver and kidney, which are very large indications. The entire Phase 2 data for Transvax – a 12 month follow up showing safety, immune response, and virological endpoints – is expected in september. CMV is also one of the leading causes of birth defects and mental retardation from an infectious agent, for childbearing women. For Vical’s pre-clinical CyMVectin, the opportunity (in childbearing women) could be comparable to that of Merck’s Gardasil for HCV, explains Vijay Samant. Vijay expects successful Phase 1/2 proof of concepts to attract development and manufacturing partners, given the succes of infectious disease vaccines in Phase 3 trials.

4.Vical’s Speed and Adaptability Play on Pandemic Influenza Appeals to Department of Defense for Military Vaccines

During the swine flu pandemic, Vical was the first company to develop a vaccine, and to test it in animals. Vical’s technology doesn’t require a pathogen, only the genetic code. The speed of study and adaptability are advantages Vical has on larger companies that dominate the space, such as GSK. That is why Vical is working with the Department of Defense’s Technology Transformation Initiative on rapidly manufacturing large quantities of DNA vaccines.

5.Vical Licensors, Sanofi and Angios, in Phase 3 Trials on Multi-Billion Dollar PAD Market

Data on the two Phase 3 PAD trials run by Vical licensors is expected in in the second half of 2010. Peripheral Artery Disease (PAD) can cause blockages in arteries, pain in the legs, and for diabetics it can lead to ulcers and amputations. The results of these trials can provide technology validation and mid-single digit royalties in a multi-billion dollar market. We could also see the first gene therapy concept to come to fruition in these trials.

This interview was conducted at the NASDAQ Marketsite, on May 25th, 2010, in New York City.

Featuring:

• Vijay Samant, President & CEO, Vical Incorporated
• Stephen Willey, Director, Equity Research – Biotechnology, Thomas Weisel Partners

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. Meeting Update: Hepatitis C – 2010 EASL

This month on we review headlines from EASL, AAN, discuss the healthcare reform, the implications of the Myriad Genetics suit for the biotech industry, and review the state of the financial markets with a focus on IPOs and Follow-ons.

The annual meeting of the European Association for the Study of Liver (a.k.a. EASL), is one of the most important meetings of the year for Hepatitis C developers, and it was held last week in Vienna.

Here are some of the headlines from EASL 2010 : – There seems to be continued interest from the investment community in the HCV space, and the meeting was reasonable well attended this year. Not as much data from the Streets’s favorite, Vertex, so perhaps not as much ado as EASL 2009. – EASL 2010 illuminated the profiles of agents in different drug classes, including polymerase inhibitors, various types of protease inhibitors, and NS5A inhibitors. Of particular interest were data from Vertex, Idenix, and Bristol Myers. – Many expect HCV treatment to evolve toward oral combination therapy to maximize cure rates, which is why investor focus on these new classes of compounds is high. – There seems to be a growing level of data and interest regarding drug responsiveness to specific genetic variants of the hepatitis C virus, as well as the genetic composition of the patient, which could play a role in guiding therapy over time.

With over 3M Americans infected with the disease, and mid and late stage drug development candidates that appear to be highly curative, HCV has potential to become one of the largest therapeutic classes in Pharma history. Hepatitis C is becoming the new battle ground for novel anti-viral treatments; quite similar to what was happening in HIV during the last decade.

2.Meeting Update: Neurology – 2010 AAN

Back from one of the most important neurology meetings of the year, Joel Sendek shares some recent developments. The American Academy of Neurology (a.k.a. AAN) meeting was important for many biotech and pharma companies (in particular those that have programs in Multiple Sclerosis). Joel talks about : – Acorda’s Amypra extension study, launch, and Lazard’s predictions for sales this year – Disease modifying therapy that can compete with traditional ABCR therapies, Biogen Idec’s Avonex, Cladribine, and FTY720.

3.Obama’s Healthcare Reform, Good for Pharma, Biotech, and Generics?

On March 23, President Obama signed the narrowly passed healthcare bill, which will extend coverage to over 32 million people, eliminating discrimination against people with pre-existing conditions and establishing a set time period of market exclusivity relating to drug patents.

At first glance, it seems that the healthcare overhaul announced by the Obama administration will not be terribly detrimental to the healthcare industry.

Pharma stocks rallied, given that there were no apparent pricing caps introduced into the system, rather, just a dramatic increase in the number of people who will be using drugs and subsequently paying for them. It is a bit too good to be true? How does the generics factor play into all this? Will the US follow the European model that would stipulate greater use of generics?

Joel Sendek comments on the outlook “from the income statement.” Companies seem to be taking charge. The impact is transitory, and while stocks may be volatile as a result, after adjustment we should be back to the previous trajectory, with the added benefit of 30M additional patients in the system.

Looking at Biotech, it seems the industry got what it wanted. Does this curb some of the investor concerns regarding generic biologics? Will these decisions taken in Washington have an impact on biotech innovation?

Dennis Purcell comments on the decreased interest he is seeing, of venture capitalist in generic companies, after the passing of the healthcare bill. Joel Sendek presents some of the benefits for biotech, namely the potential reinvestment of cash flows from increased sales of branded drugs to R&D, as well as some of the drawbacks, such as the excise tax.

4.Implications of the MYGN Suit, the Future of Genetic Testing

Federal judge Robert Sweet recently invalidated some of the patents held by Myriad Genetics relating to the identification of mutations and sequencing of the 2 genes (BRCA 1 and 2) whose mutations have been associated with breast cancer risk, classification and treatment.

In a telephone interview, David Resnick of Nixon Peabody explains that it puts a cloud over some patents until the federal circuit speaks, and that more than likely it will be appealed and reversed by the court of appeals, and could potentially be picked up by the Supreme Court. Myriad has invested a lot of money in figuring out what these mutations mean. While the tests may not be expensive to perform, the cost to validate them is significant.. and someone is going to need to invest that money. With the patent system and some exclusivity you can recoup your investment. Some commentators have said this is a place where the Federal Government should step in

Denis Purcell and Joel Sendek comment on the promise of personalized and the importance of diagnostics in the future healthcare system. Incentive structures should be put in place to encourage people to work with genetic testing, while making research is not limited by excessive patent estates.

5.Status of the Financial Markets, IPOs and FOs in the YTD

Here are some YTD numbers from DealLogic:
- 4 IPOs for 2010, vs. none in 2009, and 4 in 2008 – 27 follow ons in 2010, vs. 7 in 2009, and 12 in 2008 – in terms of total $ raised, that’s over $2.2bn in 2010, vs. $625M in 2009, and a bit over $1bn in 2008

Marina, Dennis, and Joel talk about the activity and the challenges in the public markets. Public buyers want the private guys to spend money de-risking programs, and yet they are not willing to pay for it. With regard to acquisitions, Pharma “bailing-out” Biotech, Dennis remains unconvinced, and shares the following data point :”If half of all biotech companies were for sale, any one company has less than a 1% chance of being acquired.” On a more positive note, Joel comments on how the success of mid-cap companies could help smaller companies enhance their appeal to investors, and their value as a result.

This interview was conducted at the NASDAQ Marketsite, on March 17th, 2010, in New York City.

Featuring:

• Marina Bozilenko, Executive Producer, BioBusiness.TV
• Dennis Purcell, Senior Managing Partner, Aisling Capital
• Joel Sendek, Managing Director Equity Research, Biotechnology, Lazard Capital Markets

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. New Blood Test Predicts Efficacy of Multiple Sclerosis Drug Beta Interferon

A scientific breakthrough recently published in Nature Medicine may help patients with Multiple Sclerosis find out, with a simple blood test, whether they will respond to one of the leading drugs on the market today (beta interferon) or not. Dr. Larry Steinman, one of the co-authors of the paper, tells BioBusiness.TV how he and his team came to this discovery. When asked by analyst Michael King, about the EAE model used for the test, Dr. Steinman explains the difference between the IL1 and IL17 pathways that caused paralysis. When beta interferon was administered, paralysis was reversed in animals with IL1, and was made worse in animals with IL17. An independant study from the University of Amsterdam showed a correlation between the occurrence of relapses in MS and the presence of IL17 in the blood, which is fully congruent with the data found here in animal models.

2. 1-in-4 MS Patients Could Be IL17 Non-Responders to the $4Bn Revenue Beta Interferon Drug

Dr. Larry Steinman and Mike King discuss some of the supporting research that shows Beta Interferon worsens the condition of Multiple Sclerosis (MS) patients with high levels of IL17 (in Neuromyelitis Optica a prevalent form of MS in Asia). Dr. Larry Steinman outlines the potential benefits of a screening test for the “IL17″ non-responder group (25% of patients), and for the “IL1″ responder group. Taking these different pathways in consideration, Beta Interferon may prove to work as well as the more powerful second line MS drugs such as Tysabri. Having said that, Dr. Steinman insists that other groups will have to repeat this observation in order to validate it, and the FDA will have to approve this finding before any test being available for patients.

3. IL17 to Multiple Sclerosis as KRAS to Colon Cancer?

Analyst Mike King compares the discovery of Dr. Steinman’s team to that of the KRAS gene mutation and anti-EGFR monoclonal antibody drugs like Erbitux for the treatment of metastatic colon cancer. The implication are two-fold, explains Dr. Larry Steinman. First, Multiple Sclerosis may not be a single disease, but a collection of diseases. Second, knowing whom a drug will benefit and whom it won’t is one of the most efficient ways to deliver medicine. Mike asks if insurance companies will demand this test to be performed prior to treat MS patients, and if many more tests are to be expected in the future in many other disease treatments, to which Larry responds that there are too many scientific, economic and common sense arguments in favor of this type of gating tests to be developed for all major drugs in the future.

4. When Will the 300,000+ Patients on Beta Interferon Benefit From Dr Steinman’s Breakthrough Study?

An expedited FDA study on IL17 would be welcome, says Dr. Steinman, given the potential impact of the IL17 pathway discovery on current treatments. The Multiple Sclerosis society will, hopefully, try to expedite the process. Mike King compares it again to KRAS, which took off like wildfire after it’s discovery with a very proactive FDA. Dr. Steinman insists we should not take shortcuts and make sure it’s “absolutely solid!” Dr. Steinman’s has also begun working on Teva’s Copaxone, and other experimental drugs in the pipeline.

5. Impact of IL17 on MS Drugs in the Pipeline, and Potential Applicability in Progressive Multifocal Leukoencephalopathy (PML)

Looking at some of the drugs in the Multiple Sclerosis pipeline (e.g. Ocrelizumab, Daclizumab), we could expect them to subdivide within the patient population, as with Beta Interferon. Dr. Steinman also tells us that a number of people are working to create a predictive test that can tell who is at risk of getting PML if they take Tsyabri, and that this test could bring great hope if announced and validated. “It’s going to be a wonderful frontier to watch as genetic medicine really comes to the forefront in neurology, as it has in so many other diseases” comments analyst Mike King.

This interview was conducted at the NASDAQ Marketsite, and is being broadcasted in conjunction with the release of Dr. Laurence Steinman’s paper, published in Nature Medicine on March 28th, 2010

Featuring:

• Professor Laurence Steinman, Professor, Neurology & Neurological Sciences, Pediatrics, and (by courtesy) Genetics
• Michael G. King Jr., Wedbush PacGrow Life Sciences, Wedbush Morgan Securities, Managing Director – Equity Research, Biotechnology/Biopharmaceuticals

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. The OSIP / Astellas Saga

In a hostile bid, Astellas offered a 40% premium for OSI Pharmaceuticals’s stock or $52.00 per share. It became known that Astellas made its first offer at $55-57 per share in February 2009, which OSIP Board rejected. As of the day of this interview, the OSIP stock is trading around $58. What is the market telling us? What is the investor sentiment? How will things unfold? In this interview Marina Bozilenko, Geoff Meacham, and Joel Sendek discuss the duration of assets in the value equation, arbitrage opportunities, and the possibility of another bidder stepping in.

2. The 2010 Biotech IPO Review, 1st Quarter

What can we say about the health of the IPO market today? Ironwood (IRWD), Anthera (ANTH), and AVEO (AVEO) conducted their IPO in 1Q10. They have all have been completed with a discount of 25-50% to their initial filing range. How are these stocks performing post-IPO? Marina Bozilenko, Geoff Meacham, and Joel Sendek dicuss the impact of buy side leverage, and IPO not necessarily being the best exit for early investors anymore and the current IPO stories.

3. Medivation’s Phase 3 is a Total Bust. Will Dimebon get a Second Life?

Despite great Phase 2 data and a huge Pfizer deal ($225M upfront, $500M in milestones), Medivation’s drug for Alzheimer’s disease, Dimebon, failed in Phase 3. This came as a surprise to many, but “you have to be in it to win it” as Geoff Meacham puts it. Marina, Geoff and Joel also discuss the possible application for the drug in Huntington’s disease, and the possibility of fraudulent Russian data from earlier trials.

4. Xenoport’s Phase 3, Also a Bust. An Eleventh-hour FDA Decision?

Did Xenoport “miss something” in discussions with the FDA, or did this come out of nowhere at the 11th hour? The approval of Horizant (restless leg syndrome, GSK partnership) was denied by the FDA because of pancreatic cancer in rats in the pre-clinical studies. Joel Sendek argues that values will be held in check until companies can bring products on the market and start selling.

5. Good News of the Month : Acorda and Viropharma, Stories with Upside

Intermune, Amylin, Acorda, and Viropharma all saw some good news this past month. Intermune has had favorable panel results for perfinedone. Amylin has had relatively good new on Byetta LAR. Acorda and Viropharma’s product launches are going well, they have moved beyond regulatory risk, and both offer lot’s of upside potential. Marina Bozilenko, Geoff Meacham and Joel Sendek discuss the positive news of this past month.

6. Big Pharma’s Cancer Setbacks, Arrr You Hungry for More Biotech?

Two big cancer drugs failed late-stage trials this month (Roche Avastin and Pfizer Sutent). These trials could have broadened the use of these drugs considerably, and constituted a big win for Pharma. Instead more of the “lost revenue” has to be made up for, with an ever closer patent cliff and a weaker pipeline. Must Pharma still prove they can generate growth from their biotech acquisitions? Should we expect changing behaviors from Pharma, and will there any fallout for Biotech? In this interview by Marina Bozilenko, Geoff Meacham comments on Pharma’s appetite for large companies vs. smaller 1 product “plug’n'play” stories. Joel points out that Pharma may not be better at hedging clinical and regulatory risk, than the investors betting on biotech. From that rationale, they may let others take these risks, and pursue hostile take-overs thereafter.

7. Shareholder Activism : What Carl Icahn Can and Cannot Do with Genzyme

Carl Icahn announced he was planning on nominating for Genzyme’s Board of Advisors, touting his coup with ImClone. Marina Bozilenko asks: what do investors think about his activities? Have we had enough or is he still a hero? Geoff Meacham and Joel Sendek believe there will be more, but each one will be observed on a case by case basis. Biotechnology is very different from other sectors in that you can’t force a trial to be successful, there is no “magic wand”. In the case of Genzyme, the value added of a shareholder activist is not so clear cut, they argue. Still, many companies are not that well managed, and could use restructuring. Joel points out that where shareholder activist may consider a sale at a premium a win, they might not be capitalizing on the full potential of the company – case in point : Acorda.

8. Next Month in Biotech, Events to Look Out For

Marina, Geoff, and Joel review the list of events to watch-out for next month, including: EASL, MEDCAC meeting for reimbursement of dialysis drugs, Viropharma’s analyst meeting, Genentech’s old analyst meeting, a Neurology meeting, the IMS data, and the early ASCO trade.

This interview was conducted at the NASDAQ Marketsite, on March 17th, 2010, in New York City.

Featuring:

• Marina Bozilenko, Executive Producer, BioBusiness.TV
• Geoff Meacham, Senior Biotechnology Analyst, JP Morgan
• Joel Sendek, Managing Director Equity Research, Biotechnology, Lazard Capital Markets

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. Adapting to Change, an Interview with Steve Burrill

Renown industry pundit Steve Burrill is publishing his annual state of the biotechnology industry report entitled “Biotech 2010 Life Sciences: Adapting for Success”. In this interview Steve Burrill shares with John Mohr some of the insights from this new report. Steve explains that everything has changed in the past 18 months: investors are less interested in company with less than $1bn market cap, and the regulatory and reimbursement authorities are no longer favoring “one size fits all” drugs. In the past 18 months, we’ve seen dramatic restructuring and refocusing of companies, and we are increasingly looking for ways to identify the patients with who our drugs will work.

2. No Real Healthcare Reform, Only Insurance Reform

How will Obama’s healthcare reform impact the fundamentals of the biotech industry? Steve Burrill argues we have a sickness care system, not a healthcare system; and that the debate in Washington is not about how we fix this dysfunctional system, but how we get more people in and pay for it. There is some industry concern around biosimilars and the effect they will ultimately have on innovation. Additionally, companies will have to demonstrate they create more value and reduction in cost in order to get paid by the buyers (Medicare and Medicaid).

3. Financing Biotech in 2010

Biotech raised more money in 2009 than it ever did before, despite the turmoil in capital markets, explains Steve Burrill. Why, you might ask? Because we had to in order to survive. Companies need cash and Pharma needs a pipeline. Steve expects we will raise a lot more money in 2010 for the same reasons. There are still thousands of diseases in the world that are poorly treated, and plenty of capital in the markets for good things. IPOs have turned out to be extremely expensive for companies. Steve predicts 15 IPOs this year in the US, that have mitigated technology, regulatory, and reimbursement risk.

4. From Personalized Medicine to Preemptive Medicine

We have technology today that can help us understand the differences that are hardwired within us: what drugs will work for each patient, what diseases we are at risk of getting, and how to prevent them. Steve Burrill explains we are moving from a “one size fits all” to personalized medicine, to predictive medicine, to a preemptive medicine. Our definitions for diseases are changing. With personalized medicine we’ll have more patients that are responsive to treatments in markets that are just as large. Investors will be looking for diagnostic companies, and therapeutic companies that have access to diagnostics to get through regulators and reimbursement.

5. Stem Cells and Other Promising New Technologies

Stem Cell research has been decriminalized, and funding has loosened up, but there has been a brain-drain, explains Steve Burrill. We are still doing a lot of science, we are not quite at the product level, and regulators are still a little uncomfortable. But there is no question we will live in a world of regenerative medicine, and the companies that are developing these treatments will be fabulous. We are also seeing enormous interest in vaccines, algorithms for biomarkers, and other technologies that will get us to more personalized and preemptive treatments.

6. FAST Word Association Game, with Steve Burrill

Steve Burrill share his spontaneous reaction to key buzz-words, in this classic game presented here by John Mohr and introduced by Needham’s Mark Monane. The day’s words are : ‘Consolidation’ ‘Biosimilars’ ‘Innovation’ ‘China’ ‘FDA’ ‘Big Pharma’ ‘Globalization’ ‘Carl Icahn’.

This interview was conducted at the NASDAQ Marketsite, on March 11th, 2010, in New York City.

Featuring:

• G. Steven Burrill, Chief Executive Officer, Burrill & Company
• John Mohr, Executive Producer, BioBusiness.TV

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. A Brief History of Myriad Genetics

Peter Meldrum, CEO of Myriad gives a brief history of Myriad Genetics. Led in this discussion by JMP Securities analyst Charles Duncan, Peter explains what made him want to start the company, and what differentiated it from all the other companies that sought to build on the genomic revolution. And even though Myriad Genetics has been a pioneer and a leader in the field of molecular diagnostics, it seems there is still a long way to go.

2. BRAC Analysis, Myriad Genetics’ Primary Revenue Driver

Two genes are responsible for the vast majority of hereditary breast and ovarian cancer. The BRAC Analysis diagnostic test can identify these genes and help physicians evaluate the breast and ovarian cancer risks with their patients. Peter Meldrum, discusses the significance of this test, the segments of patient population it serves, and the market size for the test. When Charles Duncan puts forward Myriad’s marketing strategy, Peter presents the rationale for his Direct To Consumer (DTC) television advertising, its effectiveness, and the regions of its future expansion.

3. Is a European Expansion to be Expected from Myriad Genetics? Checklist and Roadmap

Has the European expansion been delayed because of intellectual property or business question? When questioned by Charles Duncan, Peter Meldrum, CEO of Myriad Genetics explains the strategy which has been to establish a significant presence in the US, and then build the infrastructure necessary to capture the European market. We should expect a strong presence in the next 3 years.

4. Optimal Chemotherapy Dosing with OnDose, from Myriad Genetics

Myriad Genetics’ most recent personalized medicine product, OnDose, optimizes chemotherapy dosing for 5-FU regime. Charles Duncan inquires about the safety and efficacy gains from using OnDose vs. Body Surface Area (BSA) dosing. Peter Meldrum explains that OnDose uses pharmacokinetic analysis to measure the exposure each individual gets to the drug. This information can then be used by physicians to tailor the dosing and ensure their patients are in the optimal range. Quoting a Journal of Clinical Oncology study, an 80% reduction in toxic side effects and a doubling of response in terms of tumors shrinking were observed. Additionally, those who receive the OnDose dosing lived on average 6 months longer.

5. Myriad Genetics’ Latest Diagnostic (PROLARIS) Predicts Prostate Cancer Recurrence

On March 2nd, 2010, Myriad Genetics unveiled a diagnostic that can assess the risk of post-prostatectomy recurrence in men with prostate cancer. Charles Duncan asks Peter Meldrum about the clinical utility of the test, and the outlook for a PRE-prostatectomy diagnostic. It does indeed seem that too many prostatectomies are performed in the US (according to a NEJM study). Peter explains that Myriad Genetics just completed a study of its own that showed the diagnostic to be as predictive in pre-prostatectomy as in the post-prostatectomy. The pre-prostatectomy data was submitted to a peer reviewed journal and is expected to be published in the last quarter of 2010. As for reimbursement, PROLARIS has almost 100M lives under coverage, even though it is only a day old.

6. Patenting Genes: What if the ACLU Wins the Case Against Myriad Genetics?

In May of 2009 the ACLU filed a lawsuit against Myriad Genetics, challenging their patents on breast cancer genes. Charles Duncan asks Peter Meldrum of the implications of this case, and the impact on Myriad Genetics’ business model if the case is lost. From this point on Charles and Peter continue to discuss the numerous other competitive advantages of the company.

7. Financials: How is Myriad Genetics Planning to Return Cash to Investors?

Myriad Genetics is a profitable company that generates $150M cash per year, and has a great balance sheet. It would seem the lion’s share will be reinvested in the development of new products and expansion to new markets. Still, Charles Duncan asks how some of this cash will be returned to shareholders. Peter Meldrum explains the board of directors is currently evaluating sustainable cash dividends and sustainable stock repurchase programs.

This interview was conducted at the NASDAQ Marketsite, on March 3rd, 2010, in New York City.

Featuring:

• Peter D. Meldrum, President & CEO, Myriad Genetics
• Charles Duncan, Managing Director and Senior Biotechnology Analyst, JMP Securities

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. Batman is Back, Busting Clots with Desmotoplase

Paion’s lead compound is derived from the saliva of the vampire bat, and being tested as an anti-thrombotic in stroke. After an unsuccessful Phase 3 in 2007, a meta analysis revealed 50% of the patients had no clots to bust, and as Wolfgang Soehngen points out in this interview: “if there is no clot to bust, Desmotaplase has no job to do.” Batman is back, as it has recently been said, because of Paion’s recent turnaround. Desmotoplase is in 2 Phase 3s with Lundbeck, and added new drugs to portfolio. The partnership with Lundbeck includes 100% funding of products development, 63M Euros in upcoming milestones, and double digit royalties.

2. Paion De-Risks Pipeline with Acquisition of New Value Driver CNS 7056

The 12.3M Euro acquisition of CeNeS in 2008, and successful Phase 2b development of it’s CNS 7056 (short-acting sedative and general anaesthetic) has contributed to more than doubling the value of Paion shares in the second half of 2009. Wolfgang Seohngen explains the rationale was to de-risk Paion’s pipeline, and re-gain investor confidence, after the failed Phase 3 for Demostoplase. With the re-licencing of Desmoteplase, CNS 7056 is now Paion’s main priority, and Wolfgang expects to partner it within the year. Japanese company ONO Pharmaceuticals recently initiated clinical studies for CNS 7056 / ONO-2745 in Japan.

3. Paion’s Search & Develop Strategy: Get the Proof of Concept Faster and Cheaper than Pharma

Taking a product from discovery to market is a challenging proposition, with Wolfgang Seohngen, CEO of Paion addresses with the Search & Develop (S&D) strategy. S&D consists of taking some of Pharma’s assets that would otherwise “disappear in the basement” and developing them, knowing that they have already robust toxicology and CMC profiles. Paion’s R&D team being fully dedicated to get the proof of concept done can do the work faster and cheaper than Pharma would. Wolfgang suggests that S&D is a much safer way to generate value for shareholders. Wolfgang explains he wants Paion to be viewed as a proof of concept company and be very attractive to big pharma as future partner. In this segment, are also discussed Paion’s Phase 3 M6G morphine metabolite, and Wolfgang’s favored structure for partnership deals – a good upfront payment, with major coverage of Phase 3 costs.

This interview was conducted at the NASDAQ Marketsite, on February 10th, 2010, in New York City, on the occasion of BioCEO 2010

Featuring:

• Wolfgang Seohgnen, PhD Chief Executive Officer, PAION AG
• Swati Kumar, Research Associate, Specialty Pharma, Collins Stewart, LLC

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. Dim That Receptor Activity with Addex’s Allosteric Modulation

Ram Selvaraju asks Vincent Mutel, CEO of Addex, about allosteric modulation technology. In essence, it offers regulation of the efficiency of receptors, as opposed to the on / off switch of more traditional drugs. Ram and Vincent discuss the safety profile, and the potential not to induce desensitization. Finally, the business model is reviewed, with Merck and Johnson & Johnson collaborations, and value creation objectives for shareholders through commercialization of their own molecule

2. Addex’s New Lead Molecule ADX48621, Expected To Be Partnered Soon?

ADX48621 is the lead molecule in Addex’s pipeline. Ram Selvaraju challenges Vincent Mutel on upcoming clinical news and strategic objectives. With 110+ patients enrolled across 3 completed phase 1 trials, ADX48621 is expected to initialized it’s Phase 2B in Q410. Results are expected for late 2011 / early 2012. Vincent plans to partner ADX48621, perhaps in the near term, before getting his Phase 2 data. ADX48621’s lead indication is Parkinson’s disease levodopa associated dyskinesia (PD-LID), with potential in multiple indications.

3. December 2009, A Major Setback for Addex. Use of Cash, and Other Pipeline Molecules

The clinical trials for Addex’s former lead molecule ADX10059 were brought to halt in December 2009, after findings of hepatotoxicity. Can ADX10059 be salvaged, or is it a “dead horse”? Ram Selvaraju inquires about the future of ADX10059, Addex’s cash runway, and projected use of funds, as well the status of other pipeline molecules. In addition to the new lead ADX48621, Addex plans to develop and co-promote a GABA-B positive allosteric modulator for osteoarthritis, and develop and out-license the first fish follicle stimulating hormone receptor (FSHR) negative allosteric modulators first for benign prostatic hyperplasia (BPH), a known precursor to prostate cancer.

4. ADDEX Undervalued? What Could Drive Stock Price In The Next 6 Months

Finally, Ram Selvaraju and Vincent Mutel discuss the current valuation of Addex. Reviewed are the platform’s value and the near term catalysts. With a strong cash position, several partnerships, business development activity, and an expected Phase 2 by the end of the year, Vincent believes in a significant upside for the long term. Additionally, with the obvious product differentiation and innovation, Vincent presents a future for Addex that will undoubtedly be brighter.

Featuring:

• Vincent Mutel, PhD Chief Executive Officer, Addex Pharmaceuticals
• Raghuram “Ram” Selvaraju, Ph.D. MBA, Senior Vice President, Head of Healthcare Research, Hapoalim Securities USA, Inc.

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. Major Breakthrough in $5bn Osteoporosis Market

Everything started with this observation: patients with High Bone Mass Syndrome have an abnormally low level of serotonin in their blood, and menopausal women with this disease never develop osteoporosis. Gerard Karsenty’s team tested gut serotonin synthesis inhibitors in animal models. They discovered a way to stimulate osteoblast proliferation, which in turn leads to bone formation. This could be the first anabolic treatment of osteoporosis, and it could be administered orally. This breakthrough discovery suggests a new approach to the treatment osteoporosis, a major metabolic disease representing a $5bn market in the US. Study of Rare Genetic Disease Leads to New Target for the Treatment of Most Common Bone Disease

2. Inhibiting Serotonin Synthesis in the Gut, Rationale for Treating Osteoporosis

Gerard Karsenty studied LP533401 in animal models and found no side effects. This molecule was also tested in early clinical trials, for irritable bowel syndrome, and no side-effects were reported. The next step would be to perform additional animal testing at different doses in different treatment regiments.

3. How Does an Anabolic Treatment Compare to Other Approaches in Development for Osteoporosis?

Amgen’s Denosumab, expected to lead in the treatment of osteoporosis when approved, works by reducing bone resorption. LP533401 stimulates bone formation. This is the first report of gut derived serotonin being used as a target to treat osteporosis. Still, more work is needed to optimize the molecule. This will be the task of the Biotech and Pharma industry, but first they will have to call Columbia University’s patent office (212-854-8444 – no pressure, just there if you need it).

4. Study of Rare Genetic Disease Leads to New Target for the Treatment of Most Common Bone Disease

If the mandatory human clinical trials confirm the relevance of this new target, a inhibiting the gut serotonin synthesis may lead to a very convenient way to prevent and treat osteoporosis, and to a blockbuster drug.

This interview was conducted at the NASDAQ Marketsite, and is being broadcasted in conjunction with the release of Gerard Karsenty’s paper, published in Nature Medicine on February 7th, 2010

Featuring:

• Gerard Karsenty, Columbia University, Professor and Chair, Department of Genetics and Development
• Joel Sendek, Lazard Capital Markets, Managing Director and Senior Biotechnology Analyst

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. Who is Investing in Biotech Today? Kris Jenner

Marina Bozilenko interviews Dr. Kris Jenner of T. Rowe Price. What is the mood this year at the J.P. Morgan Healthcare Conference? What business models are investors looking for nowadays? How is the tone cautiously optimistic? Who is investing in Biotech today? Are we looking at the “core 100″ or has the base expanded?

2. Reconsidering Small and Mid Caps, IPOs, and Value Drivers. Kris Jenner

Are small and mid-cap Biotech companies now being favored by investors? Many IPO have been filed; what will make T. Rowe buy? What will be driving the value of biotechnology stocks in 2010? An interview of Dr. Kris Jenner of T. Rowe Price, by Marina Bozilenko.

3. Shareholder Activism in 2010, Good or Bad? Kris Jenner

Will shareholder activism increase this year? How will that affect Biotech? What are your market predictions for 2010? An interview of Dr. Kris Jenner of T. Rowe Price, by Marina Bozilenko.

4. A Transformative Crisis, or Just Another Cycle? Ansbert Gadicke

Marina Bozilenko interviews Dr. Ansbert Gadicke of MPM Capital. What is different this year at the J.P. Morgan Healthcare Conference? Does it look like we just went through another cycle, or are we seeing transformative change in the industry? From the venture capital perspective, how can this change be characterized? Where are we seeing increased pressure? What are the VCs looking at in new investments?

5. Key Issues for Venture Capital Investments and Fund Raising. Ansbert Gadicke

Are the key issues for venture capitalists in the current market environment? prediction for new funds trying to raise K? effect on the community? How will it affect early stage technology? An interview of Dr. Ansbert Gadicke of MPM Capital, by Marina Bozilenko.

6. Pharma Opportunities, Novel Technologies, and Diseases of the Decade. Ansbert Gadicke

Will Pharma consolidation create attractive opportunities for Venture Capitalists? What will be the next decade in technology? Which disease therapies will define this coming decade? An interview of Dr. Ansbert Gadicke of MPM Capital, by Marina Bozilenko.

7. The Insider Perspective, with Geoff Meacham (Part 1)

Marina Bozilenko interviews Geoff Meacham, Senior Biotechnology Analyst at JP Morgan. What were the companies that gave the tone, and what was the major news that came out during the week? What are the major milestones that could make or break 2010 for biotech? Have the under-served small cap become a good opportunity?

8. The Insider Perspective, with Geoff Meacham (Part 2)

At the end of the 2010 JPM conf, what is the best company-profile for a successful IPO? Is it true that Pharma’s validation is no longer sought? Is there a new trend in business models for biotech? What is the take home message of the 2010 conference? An interview of Geoff Meacham of JP Morgan, by Marina Bozilenko.

This interview was conducted at the JP Morgan Conference, on January 11th, 12th, and 13th 2010, in San Francisco.

Featuring:

• Marina Bozilenko, Executive Producer, BioBusiness.TV
• Dr. Kris Jenner, Portfolio Manager, T. Rowe Price
• Dr. Anbert Gadicke, Managing Director, MPM Capital
• Geoff Meacham, Senior Biotechnology Analyst, JP Morgan

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. 2009 in Review, the M&A and Venture Perspectives

Fred Frank and Dennis Purcell share their perspective on the transformative events of 2009 that are indicative of the future. With 3 mega-deal (Pfizer + Wyeth, Merck + Schering Plough, Roche + Genetech), the pharma landscape is changing – unfavorably for biotech. The follow-on market was strong for large cap, although we’ve seen less pharma aquiring, and a challenging IPO market. Dennis Purcell comments “we’ve reset the bar in terms of what you get paid for.” Fred Frank notes that while pharma’s mega-deals often diminish value and are motivated by fear of patent expiration, the Pfizer Wyeth deal seems to have a more solid strategic rationale. The structure of M&A deals appears to be converging with licensing deals – with smaller upfront and larger milestone payments.

2. 2010, Where Do We Go From Here?

Kicking off 2010, Fred Frank advises pharma CEOs to curb those silly stock repurchase programs, and Dennis Purcell advises investors to take capital when it’s there and not be overly concerned with dilution. Fred and Dennis continue to discuss consolidation, predominant deal structures, the focus of biotech and pharma, as well as their natural evolution and partnership. Finally, Fred and Dennis share some thoughts on the upcoming biotech IPOs. Many filings are expected but the strength of the post-IPO market is the most critical point to be considered – one which Dennis Purcell says might be victim of an excessive supply from companies trying to raise capital and boutique investment banks pushing them out. Fred remarks that while success breeds success, the later stage companies with approved phase 3 that complete successful IPOs might be encouraging to earlier stage companies, that might not perform as well in the aftermarkets and kill the IPO market. Fred warns that “failure also breeds failure”.

3. FAST Word Association Game, with Fred Frank and Dennis Purcell

Fred Frank and Dennis Purcell share with us their spontaneous reaction to key buzz-words, in this classic game presented here by John Mohr and introduced by Needham’s Mark Monane. The day’s words are : ‘Biotech’ ‘Personalized Medicine’ ‘Regulatory Environment’ ‘PharmacoEconomics’ ‘Innovation’ ‘Investors’.

This interview was conducted at the NASDAQ Marketsite, on January 7th, 2010, in New York City.

Featuring:

• Frederick Frank, Vice Chairman, Peter J. Solomon Company
• Dennis J. Purcell, Senior Managing Partner, Aisling Capital
• John Mohr, Executive Producer, BioBusiness.TV

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. Biotech Markets in 2009

While the perspectives might have looked grim early in the year, throughout the year, we’ve seen numerous signs of recovery and a newfound health in the markets. The Biotech IPO market reopened with Telacris (TLCR) the 4th largest deal of the year, and followed by Cumberland (CPIX) and Nuokang (NKBP) showing signs of an increasingly robust healthcare market, specifically for biotechnology. Since September 15th, the backlog started filling up with a dozen companies filing for IPOs.

2. Expectations for Biotech in 2010

How will Biotech perform in 2010? With a Q409 setting us up for a positive 2010, the return of the IPO market means new exits for private equity and venture capital investors, who will in turn be able to reinvest in new companies. A dozen Biotech IPOs could be expected in the first 4months of this year. The international outlook is also very positive with Israeli, European, and Chinese companies filing for NASDAQ IPOs. Indicators of success to look for are the strength of the overall market, and the pricing of the issues to come market.

3. Helping Smaller Biotech Companies Stay Listed

It is hard to get listed, and sometimes just as hard to stay listed. Just as we began to understand the magnitude of the financial crisis in September 2008, NASDAQ asked the SEC that companies shouldn’t be immediately delisted, if they began trading under $1. This request was shortly thereafter granted, helping keep many companies on the market. Additionally, NASDAQ has been developing the services it provides to issuers (e.g. risk management, corporate governance, investor relations, intelligence on stock trading) in an effort to drive new efficiencies. Finally, we’ve seen some success with Sarbanes-Oxley, further reducing the cost of trading for Biotechnology companies listed on the NASDAQ.

This interview was conducted at the NASDAQ Marketsite, on January 5th, 2010, in New York City.

Featuring:

• Robert H. McCooey, Jr., SVP Capital Markets and New Listings, The NASDAQ OMX Group
• John Mohr, Executive Producer, BioBusiness.TV

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

What are Hematopoietic Stem Cells (HSCs)? Where can we we find them? Where do they originate? Hanna Mikkola of UCLA helps us understand HSC, the current limitations in cell expansion that are limiting clinical use, as well as the direction of the efforts being made today to overcome this challenge (better harvesting, in vitro expansion, better engraftment, generating HSCs from human embryonic stem cells or induced pluripotent stem cells). Hanna reviews some of the clinical indications in which HSCs are being used, including leukemia, inherited immune deficiencies, aplastic anemia, and autoimmune diseases. New discoveries in the placenta show it is a niche in which HSCs can proliferate without differentiating, one we can study to understand HSCs expansion. Finally, Hanna gives a future outlook for HSCs.

Celgene Derives Stem Cells from the Placenta (EP9)

Celgene Cellular Therapeutics CEO Bob Hariri presents the company’s placental derived adherent cells (PDAC). These are stem cells derived from the placenta. The placenta is nature’s perfect allograft, as Bob points out. Placental cells have indeed been found to regulate the immune system. That is why Celgene’s PDACs are being administered to shut down auto-reactive destructions of the body. Celgene Cellular Therapeutics’ (CCT) principal clinical program is in Crohn’s Disease. Additionally CCT’s cells might be useful in other auto-immune diseases (such as Multiple Sclerosis), CNS, Diabetes, Cardiac, Oncology, Orthopeics, or Wound Healing. The immune advantage that the placenta means there is no need to match donor to recepient, allowing a true allogeneic / pharma business model. Bob Hariri’s long term strategy is to apply the understanding of the fundamental biology to specific diseases, and develop cells a regenerative product, with an extremely rigorous manufacturing program.

Fate Therapeutics Modulates Cell Fate (EP9)

Fate Therapeutic’s CEO Paul Grayson presents the company’s modulation of endogenous stem cells (stem cell fate). Fate can induce proliferation, differentiation, or modification of the function of a cell. Fate is using iPSCs to recapitulate a cell type in discovery, and see how to modulate that stem cell as a therapy for particular diseases. Fate’s products are currently in the clinic treating hematological disorders (such as leukemia, lymphoma) with Hematopoietic Stem Cells (HSC) from cord blood. Fate’s unique position in stem cell modulation and with iPSCs give it an IP and a competitive advantage. Financially, the company is sound, and backed by top tier VC including Arch Ventures, Venrock, and Polaris.

Alan Lewis of the Juvenile Diabetes Research Foundation distinguishes type 1 and type 2 diabetes, and continues to explain how stem cells are being used today to develop new treatments for type 1 diabetes (a.k.a. juvenile diabetes). Human embryonic stem cells (hESC) are being differentiated to the beta (insulin producing) cells that type 1 diabetics lack, and are being transplanted , in animal models. Since type 1 diabetes is an auto-immune disease, the transplanted cells must be protect from destruction by the immune system. Currently, researchers are working towards that goal with encapsulating technologies and a “gentle” immuno-modulation. In order to treat a diabetic patient, access to an unlimited number of cells is necessary. Alan compares embryonic stem cells, adult stem cells, and iPS as source of cells. And finally, in a future outlook, Alan comments on the FDA’s concern for safety, the risk of creating a tumor, artificial pancreas (as an alternative approach), and cell therapy’s potential to CURE diseases.

Novocell Leading Type-1 Diabetes Stem Cell Research (EP8)

Novocell CSO Ed Baetge presents the company’s pancreatic progenitor cell based approach to the treatment of diabetes type 1. Embryonic stem cells are differentiated into pancreatic progenitor cells and then transplanted in a capsule, where the beta (insulin) cells and islets are formed (invivo). The capsule used must be highly vascularized, and protect the cells from immune destruction. The combination of a cell therapy and a device is how Novocell plans to achieve a superior safety profile in it’s future human clinical trials. Ed mentions some of the issues that need to be overcome (including teratoma formation, purity, efficient cell differentiation, encapsulation). Future milestones include the development of a manufacturing for the cells, and a GMP process for the capsules, followed by an FDA pre-registration (expected late 2010), as well as safety, efficacy, and dosing studies for an NDA filing in 2011-2012.

Pfizer’s Broad Use of Stem Cells, in Search of New Cures (EP8)

How is Pfizer involved in stem cell research? Joe Hammang, traces back Pfizer’s long history in animal based stem cell research for drug screening, and their current use of embryonic stem cells, adult stem cells, and induced pluripotent stem cells (iPS). Pfizer’s focus was initially in tools, but now also includes a significant effort in diabetes, cardiovascular, and ophthalmology, where there is a significant unmet need. Joe explains Pfizer’s particular interest in diabetes, and their collaboration with Novocell. Additionally he underscores Pfizer’s research and leadership’s commitment to finding therapies that can CURE diseases.

What is a neural stem cell? How can they help treat neurological disorders such as Alzheimer’s disease, Parkinson’s disease, spinal cord injury, stroke, ALS (Lou Gehrig’s Disease)? Evan Snyder of the Burnham Institute helps define neural stem cells (NSC), explaining that they are relatively inaccessible in the adult patient, but that they seem to benefit from some immunotolerance. It follows that we may be able to use readily available lines of embryonic stem cells for therapy in neurodegenerative disease. Evan reviews current clinical applications for neural stem cells (including as a vehicle for small molecule delivery), and gives his future outlook for neural stem cells. Evan forsees NSCs being used in anti-inflammatory and neuroprotective functions, to deliver tumor killing genes, and to build iPS drug discovery models.

StemCells Inc. using Neural Stem Cells for their Neuroprotective Effect (EP7)

Discover StemCells, Inc (NASDAQ: STEM). A pioneer in the field of neural stem cells, StemCells Inc was founded by the renown Irving Weissman, Fred Gage, and David Anderson. Martin McGlynn, CEO, presents StemCells Inc’s use of neural stem cells, and clinical development strategy based on the neuroprotective effect of neural stem cells. Stem Cells Inc is preparing a BLA for NCL (a.k.a. Batten’s Disease). Martin reveals the source of the stem cells used, as well as the quantity of cells needed for the treatment of various diseases. StemCells Inc’s next clinical trials (PMD, spinal cord, macular degeneration) and milestones are also presented here. We conclude this interview with Martin’s comments on successfully dealing with the complexity of regularity matters, beyond FDA approval.

Q Therapeutics and the Role of Neuronal Support Cells (EP7)

Discover the Q cells of Q Therapeutics (Private, Salt Lake City, UT). Deborah Eppstein, CEO presents her company, which is focused on the creation of the support cells for the neurons that make up 90% of the central nervous system (CNS). Q Therapeutic’s glial progenitor cells – dubbed Q-Cells – form oligodendrocytes and astrocytes. The Q-Cells are “mini-factories” for neuron support cells that help keep neurons healthy and restore their function before they die. Q Therapeutics is targeting demylenating diseases (multiple sclerosis, transverse myelitis, and cerebral palsy), but also other neurodegenerative diseases and conditions (ALS / Lou Garrig’s Disease, Alzheimer’s Disease, Parkinson’s Disease, and spinal cord injury). Q’s clinical trial design is centered around a non-systemic therapy (for safety), and objective measure of improvement (for efficacy). In a competitive universe where most companies are focused on neural regeneration, Q-Cells could come in as a good therapeutic complement.

Gordana Vunjak-Novakovic of Columbia University gives us an explanation of what mesenchymal stem cells are; adult stem cells that are present in many organs and tissues, and that drive tissue repair. The main sources of mesenchymal stem cells are bone marrow and fat (adipose tissue), although they can be found lesser quantities in muscle, blood, heart, liver, and other tissues. Mesenchymal stem cells can be used to regenerate many different types of tissue, but the most promising clinical applications are in bone, cartilage, ligaments, muscle, neural cells, and striated muscles. Mesenchymal stem cells are thought to repair the heart muscles through revascularization and recruitment of repair cells, but the data does not yet show that they can convert into heart muscle. In a future outlook, Gordana shares with us what she thinks is in the near future of mesenchymal stem cells: well defined cells that can be differentiated predictably, understanding the importance of cell origin on clinical outcome, new FDA friendly delivery routes, more effective functional markers and monitoring, and increasing interdisciplinary collaboration.

Angioblast, Mesoblast, and the Medical Applications of Mesenchymal Precursor Cells (EP6)

AngioBlast Systems (Private, NY, USA) and Mesoblast (ASX:MSB), Melbourne, Australia) are the two companies featured in this episode of the Stem Cell Review. Michael Schuster presents the companies’ mesenchymal precursor cells isolated from bone marrow, for allogeneic treatment. On the one hand, AngioBlast focuses on cardio vascular indications (heart failure, heart attack) with a phase 2 trial that is 2/3rds of the way, and has an encouraging efficacy profile. On the other hand, Mesoblast is focused on orthopedic indications (mainly bone regeneration and cartilage repair) with an equally promising pilot study in long bone fractures that don’t heal over time.

1. XIAFLEX Driving Stock, Dupuytren Indication

Armando Anido, CEO of Auxilium Pharmaceuticals (AUXL) is interviewed by Salveen Kochnover of Collins Stewart. Salveen asks about the enzyme know as XIAFLEX that breaks down the collagen causing Dupuytren’s disease. Armando explains Dupuytren’s disease is present in 3-6% of adult caucasian males. Salveen and Armando discuss the clinical data, which has been favorably reviewed by the FDA for safety and efficacy. The results of the XIAFLEX treatment seem to be comparable to those of surgery.

2. The CEO’s Strategy to Bring XIAFLEX to the Dupuytren Market

With upcoming Phase 3 results, Armando Anido, CEO has ramped up Auxilium Pharmaceuticals’ commercial infrastructure. The interaction FDA has been positive although they missed their PDUFA date last August. Salveen Kochnover of Collins Stewart inquires about the physician training program and the 5year, 50% recurrence of Dupuytren’s disease.

3. Collagenese XIAFLEX Could be a Pipeline in a Product, Treat Multiple Indications

XIAFLEX attacks the collagen deposits that cause Dupuytren’s contracture (a fixed flexion contracture of the hand where the fingers bend towards the palm and cannot be fully extended), Peyronie’s disease (abnormal curvature of the penis), cellulite, keloidal scar, and frozen shoulder (restricting motion and causing chronic pain). Results are expected in Peyronie’s in December, with a breaking of the blind and release of top line data. Armando Anido, CEO of Auxilium Pharma is planning an FDA Phase 3 proposal in early 2010. Salveen asks Armando about access to treating physicians for Peyronie’s disease (since they are not the same as those treating Dupuytren). Armando explains that Auxilium is well implanted in the urology community with TESTIM, a product that has been treating men with hypogonadism, or low testosterone since Feb 2003. With a Phase 3 approval in Dupuytren, Auxilium will launch new programs, in new indications.

4. Pfizer Deal, Financials, Business Development, and Upcoming Milestones

$75M upfront, with a total of $500M in milestones, and double digit royalties. Those are the terms of Auxilium Pharmaceutical’s deal with Pfizer. The synergy has increased with Pfizer’s recent aquisition of Wyeth. Salveen Kochnover of Collins Stewart and Armando Andio of Auxilium discuss the commercial rights, with an outlook on geography, indications, and other partnership opportunities. Salveen and Armando also review the expected TESTIM revenues for 2009, and the upcoming milestones (Dec 09, data in Dupuytren’s; early 2010, submission of XIAFLEX in EU by Pfizer). 5 years from now, Armando expects XIAFLEX will be blockbuster drug.

This interview was conducted at the NASDAQ Marketsite, on November 4th, 2009, in New York City.

Featuring:

• Armando Anido, Chief Executive Officer and President, Auxilium Pharmaceuticals
• Salveen J. Kochnover, CFA Managing Director, Biotechnology Equity Research, Collins Stewart, LLC

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

How will stem cells change the way we think about treating diseases? Here is the 5 year forward look at the world of Stem Cells, from some of the greatest experts in the field.

What are the diseases we’ll be treating, and the tools we’ll be using in 2015? Where will we be in terms of clinical trials? What are the dangers in the stem cell hype, and medical tourism? How will stem cells pave the way for personalized medicine, and more rational treatments? How important will stem cells become in the drug discovery process? Discussed in the episode are the eye (macular degeneration), the skin, diabetes (type 1 & 2), blood and autoimmune diseases, glioblastoma, HIV, and more.

This is the Episode 5 of the Stem Cell Review, aired on October 29nd, 2009

Researchers are using stem cells as tools for disease study, drug screening, clinical trial strategy, and personalized medicine. The induced Pluripotent Stem cell (iPS) is giving us a chance to rethink the way we are developing new drugs. These iPS cells are usually created from somatic cells (such as skin), and not embryos or adult stem cells. In creating iPS from patients’ diseased cells, scientists can study the disease in vitro, looking for disease phenotypes, applying microenvironmental stress, and testing new drugs. Compared to animal model testing (e.g. mice), this represents a significant breakthrough, that can be used to validate clinical development strategy and test efficacy in specific groups of patients. iPS is bringing a revolution in drug discovery methodology which is being used to bridge genetics, cell biology, and physiology.

This is the Episode 4 of the Stem Cell Review, aired on October 22nd, 2009

What are the biological processes that stem cells go through? What are the industrial processes we need to manufacturing? What do we know about cancer stem cells? How do iPS cells fit into the picture vs. embryonic stem cells? In this episode we investigate how the science and research of stem cells is being translated into industrial cell processes to create FDA approvable, and commercializable products. Differentiation, proliferation, migration, retro-differentiation, trans-differentiation, transformation into cancer cells, the role of tumors’ micro environments and epigenetics and all reviewed here by the field’s foremost experts.

This is the Episode 3 of the Stem Cell Review, aired on October 15st, 2009

Reviewing the technology, business models, intellectual property, regulatory concerns, transplantation and immune rejection.

This is the Episode 2 of the Stem Cell Review, aired on October 8st, 2009

1. Can ISIS Pharma’s Antisense Turn Drug Discovery on its Head?

ISIS CEO Dr. Stanley Crooke presents the Antisense platform technology as the natural successor of small molecules and monoclonal antibodies as a drug discovery platform. This platform is fully controlled by ISIS and made available to partners. Needham Analyst Mark Monane and Dr. Crooke discuss the increased speed in drug discovery and reduced risk of failure that Antisense brings. Partnerships and competition are also brought into the discussion. Dr. Crooke affirms a couple billion dollars in partnerships over the past 20 years. With such promising technology, ISIS Pharmaceuticals might strike fear into the hearts of the “old school” biotech companies.

2. Genzyme and ISIS Partnership on Lead Product “Mipomersen” for LDL-Cholesterol Reduction

Dr. Stanley Crooke of ISIS Pharmaceuticals and Mark Monane of Needham & Company discuss ISIS’ pipeline, starting with Lead Product Mipomersen, antisense drug being developed with Genzyme, currently in Phase 3 studies in patients who cannot adequately control their cholesterol levels. Mark & Stanley talk through the relationship with Genzyme, the subcutaneous administration of the drug, and the merits of a study in cholesterol, where measurable targets provide an unequivocal test of success or failure. In a 19 product strong pipeline, are many other promising worth discussing. Stanley highlights a few of the next generation drugs in development at ISIS pharmaceuticals.

3. ISIS Pharma’s Development Strategy, Capital Needs, and Upcoming Milestones to Look Out For

How does ISIS Pharmaceuticals decide to partner a product or develop it internally? How far in the development will ISIS go? What is ISIS pharma’s financial position? What are its growth prospects? What are the important upcoming milestones that will have an impact on the stock? Mark Monane of Needham & Company inquires about all of the above with Dr. Stanley Crooke, CEO of ISIS Pharmaceuticals.

4. ISIS Pharmaceuticals CEO Comments on Valuation, Plays the “Word Association” Game with Mark Monane

With 19 Products in it’s pipeline, what may be a groundbreaking proprietary platform technology, and numerous partnerships, ISIS Pharmaceuticals is hard company to value. And Dr. Stanley Crooke, CEO doesn’t apologize for it. He then agrees to play Needham Analyst Mark Monane’s favorite game, the fun and incisive “Word Association”.

This interview was conducted at the NASDAQ Marketsite, on October 6th, 2009, in New York City.

Featuring:

• Stanley T. Crooke, MD, PhD, Founder, Chairman and Chief Executive Officer of Isis Pharmaceuticals.
• Mark Monane, MD, MS Managing Director, Equity Research, Biotechnology and Life Sciences, Needham & Company, LLC

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

What are the differences between embryonic, adult and induced pluripotent stem cells? Where do the experts expect the next medical application will be for stem cells? Do stem cells promote regeneration? How are cell types interacting? How can induced pluripotent stem (iPS) cells revolutionize drug discovery? Can cell therapies be made into a viable business? How close are we to finding the right business model? Are investors interested in stem cells today? What is the political and ethical landscape like now that the Obama administration has taken over the White House?

This is the Episode 1 of the Stem Cell Review, aired on October 1st, 2009

Exciting things we’ve seen in 2008 are:

• the involvement of pharma in cell therapy, investing like they haven’t before, developing cell therapy products as drugs, and not just tools (including companies such as Pfizer, GSK, Roche, and Novartis)
• the number of late stage companies in the sector such as Osiris (see: Osteocel / NuVasis, and Prochymal + Chondrogen / Genzyme deals)
• the financing, partnerships and M&A activity with $400M private equity investment, $100M M&A, and $1.8bn non M&A deals.

Things to watch for in 2009 are:

• some of the late stage companies (Osiris and Dendrion – i.e. Provange results in April)
• the struggling cell therapy companies and how they will find cash (M&A, Government, VC?)
• the role of the new Obama administration and the public pressure, interest, and enthusiasm for cell therapy

2. Harvard Steps Into the Cell Therapy Funding Gap with a Mission for Academic Research to Get to Patients

Brock Reeve explains the functioning of the Harvard Stem Cell Institute (HSCI). The HSCI is looking to make the bedside-to-bench and bench-to-bench loop in a way that unequaled anywhere, with the university, its 11 affiliated hospitals, drawing resources from the various schools (law, business, government, divinity). Brock tell us about about the HSCI partnership with GSK, as well as the other relationships they are seeking and building at the moment.

3. Roche’s Vertes on Big Pharma’s Interest in Cell Therapy

Alain Vertes of Roche presents the interest of big pharma in the cell therapy. He highlights the main points of his panel presentation:

1. Cell therapy is not a passing fad
2. Cell are not just transplants, they can also be drugs
3. Paradigm shifting applications for cell therapy
4. Protecting intellectual property
5. Potential for stunning efficacy and low risk of side effects

4. US and EU Regulatory Synergies, and EMEA Committee for Advanced Therapies

Christopher Bravery comments on the new committee for advanced therapies of the EMEA, and its impact on the development of cell therapy in Europe. Christopher also reflects on the similarity between US and European regulatory environments, and the importance of considering that in planning pivotal studies. He also gives us his definition of Cell Therapy and explains the business of ERA Consulting.

Featuring:

• Christopher A. Bravery, Director of Regulatory Affairs, ERA Consulting
• Lee Buckler, Principal, Cell Therapy Group

DISCLAIMER:

VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

We’ve asked guests and panelists of the 2009 Phacilitate Cell & Gene Therapy forum to answer the following question:

Click on the question to see their answers, MASHED UP!

1. How Would You Define Cell Therapy?

2. Are We Going to See New Cell Therapy Products in the Next 2-3 Years?

3. How Many Cell Therapy Products Are On the Market Today?

4. What Are Some of the Exciting Things Happening in Cell Therapy?

This interview was conducted at the Phacilitate Cell & Gene Therapy Forum 2009, on January 26th, 2009, in Washington DC.

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. Lee Buckler’s Cell Therapy ‘08 Review and ‘09 Outlook

Exciting things we’ve seen in 2008 are:

• the involvement of pharma in cell therapy, investing like they haven’t before, developing cell therapy products as drugs, and not just tools (including companies such as Pfizer, GSK, Roche, and Novartis)
• the number of late stage companies in the sector such as Osiris (see: Osteocel / NuVasis, and Prochymal + Chondrogen / Genzyme deals)
• the financing, partnerships and M&A activity with $400M private equity investment, $100M M&A, and $1.8bn non M&A deals.

Things to watch for in 2009 are:

• some of the late stage companies (Osiris and Dendrion – i.e. Provange results in April)
• the struggling cell therapy companies and how they will find cash (M&A, Government, VC?)
• the role of the new Obama administration and the public pressure, interest, and enthusiasm for cell therapy

2. Harvard Steps Into the Cell Therapy Funding Gap with a Mission for Academic Research to Get to Patients

Brock Reeve explains the functioning of the Harvard Stem Cell Institute (HSCI). The HSCI is looking to make the bedside-to-bench and bench-to-bench loop in a way that unequaled anywhere, with the university, its 11 affiliated hospitals, drawing resources from the various schools (law, business, government, divinity). Brock tell us about about the HSCI partnership with GSK, as well as the other relationships they are seeking and building at the moment.

3. Roche’s Vertes on Big Pharma’s Interest in Cell Therapy

Alain Vertes of Roche presents the interest of big pharma in the cell therapy. He highlights the main points of his panel presentation:

1. Cell therapy is not a passing fad
2. Cell are not just transplants, they can also be drugs
3. Paradigm shifting applications for cell therapy
4. Protecting intellectual property
5. Potential for stunning efficacy and low risk of side effects

4. US and EU Regulatory Synergies, and EMEA Committee for Advanced Therapies

Christopher Bravery comments on the new committee for advanced therapies of the EMEA, and its impact on the development of cell therapy in Europe. Christopher also reflects on the similarity between US and European regulatory environments, and the importance of considering that in planning pivotal studies. He also gives us his definition of Cell Therapy and explains the business of ERA Consulting.

This interview was conducted at the Phacilitate Cell & Gene Therapy Forum 2009, on January 26th, 2009, in Washington DC.

Featuring:

• Christopher A. Bravery, Director of Regulatory Affairs, ERA Consulting
• Lee Buckler, Principal, Cell Therapy Group

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. Molecular Diagnostics As The Next Major Conceptual Transition

Jan Buck welcomes Dr. George Poste onto his show to discuss his uniquely rich view of the life science industry and highlights of his distinguished career. George tells us about his time at SmithKline Beecham, where he witnessed the dramatic transformations driven by molecular biology, genomics, and genetic engineering. Furthermore Jan and George look into molecular diagnostics and how they will allow us to transcend symptoms based empirical approaches and more rigorously characterize the underlying molecular pathologies.

2. Mapping the Human Genome, And The Innovator’s Dilemma In The Pharma Industry

On this latest edition of the Jan Buck Show, Dr. George Poste tells the story of one of the greatest broad-scale industry collaborations, the SNP Consortium (which became the HapMap Project). Aimed to study population genomics in relation with the analysis of susceptibility to disease and responsive to treatment, it harness the power of a large number of companies to create this body of data, furthering the research capabilities of all, while defraying risk and cost. Jan and George continue to discuss radical innovation, the innovator’s dilemma, and how it has applied to pharma and it’s blockbuster drugs.

3. Taking Out A Small Number of Pharmaceutical Manufacturing Plants Could Dislocate Medicine Supply For Large Numbers of the Global Population

Dr. George Poste tells the story of his involvement with the Antibioterrorism Task Force and the Office of the Secretary of Defense. When the DoD started looking at global infectious diseases – trying to measure the social, economic and military risk – they brought George in. Jan and George then discuss the post 9/11 era, and securing the medicine production and supply chain.

4. The Biodesign Institute, A Radical Experiment To Extract The Low Intensity Signal That May Be Indicative Of Disease

Dr. George Poste tells Jan Buck about his beginnings as veterinarian in the south of England, his escape from Europe to the United States in pursuit of greater scientific opportunity, and his recruiting at SmithKlein Beecham, taking genetic engineering technologies into the industry. George and Jan comment on the contribution the pharmaceutical industry has made towards human life. They continue to discuss his role in the creation of the Biodesign Institute of Arizona State University, where the life sciences are integrated with high level engineering, computing, and mathematical analysis. They measure hundreds or thousands of genes simultaneously, defining patterns of expression of multiple markers in the body, that may be indicative of disease.

5. A Look to the Future: Personalized Medicine, Consumer Driven Health, Synthetic Biology

Jan Buck and Dr. George Poste take a look at the blockbuster based business model of the Pharma industry, and how it might evolve in the years to come. They identify two emerging trends. First, is personalized medicine. Diagnostics and therapy will be increasingly linked. Clinical trials will become smaller, more effective, and cheaper to run. Second is consumer driven health, in which we all take greater responsibility for our own health. Finally, George tells us of his latest governmental responsibility, heading a new task force on synthetic biology – genetically engineering microorganisms to develop a new industrial ecology that can create alternative energy, novel materials, and molecular species that would otherwise be impossible or prohibitively expensive to produce.

This interview was conducted at the Nassau Inn, Princeton NJ, on December 10th, 2008

Featuring:

• George Poste, Chief Scientist, Complex Adaptive Systems Initiative; Director, The Biodesign Institute; Arizona State University
• Jan Buck, Commentator & Celebrity

DISCLAIMER: BIOBUSINESS.TV IS AN INDEPENDENT MEDIA, AND A PLATFORM FOR THE EXCHANGE OF IDEAS AND OPINIONS. BIOBUSINESS.TV STRIVES TO ENHANCE THE QUALITY AND ACCESSIBILITY OF INDUSTRY DATA, AND TO DELIVER INFORMATION THAT INVESTORS AND EXECUTIVES CAN RELY UPON TO MAKE SOUND, INFORMED FINANCIAL AND MANAGERIAL DECISIONS. VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

Randy Mills of Osiris and Bill Kirdel discuss the Osiris pipeline and technology. Bill asks Randy about the difference between Osiris’ mesenchymal stem cells (MSC) and embryonic stem cells (ESC). Randy explains that MSC are persistent in the human body and that they contribute to our natural healing process. Moreover, the MSC, traditionally associated with connective tissue, have tremendous applicability due to their inflammation regulation, tissue regeneration, and tissue protection abilities. Randy continues to explain Osiris’ business development strategy, following the prodigious $1.4 partnering deal with Genzyme for lead compounds in ex-US/Canada markets. Randy and Bill continue to review the execution focused future of Osiris, potential consolidations in the field of cell therapy, and the future of regenerative medicine beyond Osiris.

This interview was conducted at the NASDAQ Marketsite, on December 8th, 2008, in New York City.

Featuring:

• C. Randal Mills, Ph.D., President and CEO at Osiris Therapeutics
• William Kridel, Managing Director at Ferghana Partners Group

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

Beyond Tests, Lab Service Provides Comprehensive Diagnostics, New Efficiencies for Healthcare System.

Genoptix CEO, Dr. Tina Nova is interviewed by Adam Feinstein of Barclays Capital. Tina and Adam discuss the origination of the business, and how their comprehensive diagnostics services fit into existing medical paradigms. Tina explains Genoptix holds some of the best hempaths among its ranks, and it focuses on quality diagnostics and quality service. This has been the fuel for its rapid growth. Adam challenges Tina about her plans for managing growth. Given the pricing is fixed, Genoptix will raise its bottom line from increased revenue and greater efficiencies. Tina and Adam continue to discuss the current macro-enviroment and its implications for Genoptix, the impact of President-elect Obama’s future healthcare reforms on diagnostics, as well as exciting new developments to come.

This interview was conducted at the NASDAQ Marketsite, on November 14th, 2008, in New York City.

Featuring:

• Dr. Tina Nova, President, CEO & Co-Founder, Genoptix Medical Laboratory
• Adam Feinstein, Senior Healthcare Services Analyst, Barclays Capital

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. Cephalon’s Growth Drivers and Promising Market Opportunities for Amrix, Treanda

Frank Baldino, CEO of Cephalon gives a quick overview of the company’s financial status and comments on Treanda’s recent approval for non-Hodgkin lymphoma (NHL). Interviewed by Eric Schmidt, Senior Biotechnology Analyst at Cowen, Frank comments on the high pricing of Treanda, the implication with respect to patients and payors, and the use of Treanda in earlier lines of treatment. Eric also asks Frank about the opportunity for Amrix, how it compares to Flexeril in the musculoskeletal pain market, and what the plans are for marketing the drug Amrix (given Cephalon has limited marketing experience in competitive markets).

2. Cephalon’s Provigil Cash Cow Running Out of Milk, But Growth Continues With New Products, Indications, and Geographical Markets

Once Provigil reaches the end of it’s patent, how will Nuvigil compete against it’s mother compound in the wakefulness market? Frank Baldino and Eric Schmidt discuss Nuvigil’s improved formula as well as the new markets it plans to reach. Frank and Eric also discuss the management of Cephalon’s substantial net earnings in investment vs. EPS. Frank comments on the future reduction of growth in US markets, as healthcare cost are being curbed. However, Cephalon is building an infrastructure to gain rapid access to the Chinese marketplace, which could be a strong growth driver in the years to come.

This interview was conducted at Cowen and Company, on November 5th, 2008, in New York City.

Featuring:

• Frank Baldino, Jr. Chairman & Chief Executive Officer
• Eric Schmidt, Biotechnology Analyst & Managing Director, Cowen and Company

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

DISCLOSURES FOR SCHMIDT, CEPH: – 1) Any financial interest held by the analyst, member(s) of his team, or his household members: No – 2) Firm and affiliates 1% or more ownership interest: No – 3) Any other material conflict of interest: No – 4) Subject company a client of the firms or its affiliates: No – 5) Position as officer or director of the company: No – 6) Analyst received compensation from company: No – 7) Cowen and Company received compensation from company: No

1. CoNCERT’s Leading Portfolio Asset to step into GSK Paroxetine’s $3Bn Shoes

CoNCERT’s CTP-347 is a Phase 1 treatment for hot flashes that acts as a substitute for GSK’s Paroxetine, with the potential added benefit of little to no drug-drug interaction. Roger Tung and Eric Schmidt discuss future studies vs. Paroxetine or placebo. They also review CTP-518, an HIV protease inhibitor that could offer higher tolerability without a combination use with Ritonavir / Norvir (Abbott), simplifying drug use, and reducing cost to payors.

2. CoNCERT’s IPO, Partnering Strategies and Full Integration for it’s Deuterium Substitution Platform & Pipeline Assets

The applicability of CoNCERT’s pipeline extends beyond what it can accomplish as a small biotech. To what extent is CoNCERT willing to partner its platform and assets? Is full integration a future possibility? As Eric Schmidt interviews Roger Tung, he asks him about taking the company public. Roger and Eric also discuss the use of this platform beyond the current focus in what could a number of spin-outs. Finally, they take a look at the how CoNCERT’s technology can help drugs going off patent, and their parent companies that may be staring at the abyss.

3. CoNCERT’s Deuterium Substitution Platform to Create Better, Faster, Cheaper Drugs

Deuterium has the same properties as hydrogen, only it has the power to make molecules more stable. This creates a number of interesting applications for existing drugs. CoNCERT has developed a technological platform that substitutes hydrogen for deuterium, and has applied this process to a number of marketed drugs in the hopes of creating first or best in class drugs. Roger Tung, CEO of CoNCERT is interviewed by Eric Schmidt, Senior Biotech Analyst at Cowen on the properties of the modified drugs, and their patentability. Eric continues to ask Roger about the efficiency drug delivery process and it’s starting points. Finally Roger answers questions regarding CoNCERT’s cash position, and potential for growth.

This interview was conducted at the NASDAQ Marketsite, on November 3rd, 2008, in New York City.

Featuring:

• Roger Tung, President and CEO, CoNCERT Pharmaceuticals
• Eric Schmidt, Biotechnology Analyst & Managing Director, Cowen and Company

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. Portrait of Wall Street’s Pioneer Drug Analysts

The first biotech investment banker tells Jan how he got started in the business, as an analyst and a dealmaker, and continued to build the first formal life science focused research program at Lehman Brothers. Fred Frank, Vice-Chairman of Lehman Brothers (Barclays) discusses the tsunamis of change that are affecting the industry, and why Biotech and Pharma form natural partnerships. One is opportunity long and capital short, the other is opportunity short and capital. Fred illustrated this point by comparing the cash and innovation coming from the 100 largest pharma on one hand, and the 100 largest biotech on the other, demonstrating the need they have for one another.

2. The Future of the Industry, and the Lehman / Barclays Deal

Jan Buck asks Fred Frank where the industry is heading, and if we should be expecting radical change in the near future. Fred Frank looks at the assumptions that fueled the growth of the industry from its earliest onset, and their relevance today. He then asks Fred about the Lehman / Barclays transaction. Is it a fit? Will there be a change in activities? What will determine the success of the acquisition?

3. Blockbuster Drugs, and the 3 Eras of the Biotech Industry

Fred and Jan review the changing dynamics of the industry (pre-76, 1976 to 2000, and post-2000). They discuss the monumental changes faced by Pharma, the danger of stock buybacks, and the impact of blockbusters on the development of drugs and the valuation of companies. Fred and Jan also discuss some of the more successful business models today, including the Genentech / Roche agreement – although the recent capital acquisition does come under question.

This interview was conducted at the New York Stock Exchange, on September 23rd, 2008, in New York City.

Featuring:

• Frederick Frank, Vice-Chairman, Lehman Brothers (Barclays)
• Jan Buck, Commentator & Celebrity

1. Ablynx introduces Nanobody Technologies, discusses Pharma Partnering Strategies

How does a Small Cap Biotech make it through the current macro environment? Edwin Moses of Ablynx says choice is no longer a luxury but a necessity. Edwin reviews some of Ablynx’s partnership agreements, and explains how he brought Pharma in. Beyond the cash, Ablynx is looking for experience and leverage in partners. He continues to explains how Ablynx’s Nanobody Technology was discovered in camels, developed in llamas, and how it could potentially offer significant clinical and commercial benefits. Geoff Meacham of JPMorgan continues to ask him about the marketplace differentiation it offers, and the technological challenges that Nanobodies could present in the future. Edwin and Jeff then discuss Pharma’s R&D productivity model and partnering models, capturing the entrepreneurial spirit of Biotech, and key drivers of M&A deals today.

2. Ablynx’s Pipeline, Technology Advantage over MABs, and Financial Risk

Edwin and Jeff discuss the planned evolution of Ablynx’s pipeline, taking products to the clinic and improving patient care. Edwin explains everything his team has learned and gained from working with industry partners. Geoff asks about Nanobodies’ advantages over Monoclonal AntiBodies (MABs). Finally Edwin and Geoff review Ablynx’s $170M cash position and their burn rate, as well as their execution focus for the years to come.

3. Ablynx’s Antithrombotic Safety and Efficacy Profile, Clinical Development Strategy, and Business Development Objectives

Edwin Moses tells us what he is most excited about in the pipeline, and where he will focus the company’s energy in the years to come. Geoff Meacham asks him about his plans for scaling up Ablynx’s clinical development. Edwin and Geoff continue to discuss the target specific and generic based issues that could present a challenge for Ablynx. Finally, they review the antithrombotic’s safety and efficacy profile with respect to currently marketed products, and take a look at Ablynx’s regulatory approval strategy.

This interview was conducted at the NASDAQ Marketsite, on September 23rd, 2008, in New York City.

Featuring:

• Edwin Moses, Chief Executive Officer and Chairman of the Board, Ablynx
• Geoff Meacham, Senior Biotech Analyst, JP Morgan

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. Targanta’s Antibiotic Shows Promise, And Overcomes the Challenges it Faced When Previously Developed at Lilly and InterMune

Oritavancin is Targanta’s lead compound. Initially developped at Lilly, it was then sold to InterMune, and picked up by Targanta in an equity deal. Mark Leuchtenberger explains the history of the drug, and in doing so faces further probe from Joel Sendek as to the corporate and medical challenges the drug had to face in its earlier days. Mark explains how they were overcome in Targanta’s hands, and the true potential of the was drug unlocked.

2. Targanta Attacking $1.2Bn Antibiotic Market with Resistance Adverse Drug, Do-or-Die!

Mark and Joel review the competitive landscape for Targanta’s Antibiotic and Lead Compound, Oritavancin. Key differentiators include shorter course of treatment, lack of monitoring and dosing adjustment, efficacy and coverage. With a growing $1.2bn market, Mark still sees an unmet medical need in resistance adverse antibiotics, which Targanta is looking to address with focus, passion, and a Do-or-Die attitude. A Phase 3 trial, validating a single dose treatment against a standard of care competitor is expected to run next year.

3. Targanta Confident and Prepared for FDA Panel Review of Lead Compound Oritavancin

Mark and Joel delve into the clincal trials and regulatory process, around Targanta’s Lead compound Oritavancin. Mark describes the current response of the FDA, and completed clinial site audits. He then continues to explain his team is expecting a panel meeting and is fully prepared to assit the panel’s review of the drug. This panel could be expected as early as November of this year. When Joel inquires about the release of simplified data, which might differentiate Oritavancin for its competitors, Mark tells him about the drug profile (administration, safety, efficacy), but choses to keep quiet about the release date, telling us he the abstract and data will be revealed at an upcoming, yet undisclosed scientific conference.

This interview was conducted at the NASDAQ Marketsite, on September 18th, 2008, in New York City.

Featuring:

• Mark Leuchtenberger, President and CEO, Targanta Therapeutics
• Joel Sendek, Managing Director and Senior Biotechnology Analyst, Lazard Capital

1. Alexza’s Eulogistic Symphony Deal, Partnering and Sales Strategies, and Cash ’till the Summer of 2010

Ted Tenthoff asks Tom King of Alexza’s partnering strategy. Alexza will develop its own compounds for Psychiatry but will seek to partner in other areas, such as sleep – where Alexza is working with Endo Pharmaceuticals. Tom explains Alexza’s strong cash position, and the value of the Symphony transaction, which ultimately led to the development of 3 phase 3 products. Tom and Ted review other financing vehicles used by Alexza, and the production capabilities of the new Mountain View, CA GMP facility. Finally Ted asks Tom about Alexza’s discovery process, and method for prioritization of new drug development.

2. Alexza’s Staccato: Vaporized Drugs in Multiple Indications, with a Safe and Effective Profile

The Staccato system combines drug and device to provide a quick and convenient relief for patients with acute and intermittent conditions. Tom King (Alexza) and Ted Tenthoff (Piper Jaffray) explore the technology, mechanism of action, and safety profile. Tom and Ted to discuss the drugs in the pipeline and the indications, which include acute agitation associated with schizophrenia or bipolar disorders, breakthrough pain, panic attacks, and insomnia. They review the clinical data, and path for regulatory approval, in what Tom King calls a “crisp NDA plan” for early 2010. Tom and Ted also discuss the marketing opportunity and marketing challenges for these drugs and their innovative delivery system.

This interview was conducted at the NASDAQ Marketsite, on September 17th, 2008, in New York City.

Featuring:

• Thomas B. King, President and CEO, Alexza Pharmaceuticals
• Edward Tenthoff, Managing Director and Senior Biotech Research Analyst, Piper Jaffray

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

DISCLOSURES FOR THE UNIVERSE OF TED TENTHOFF

1. I or a household member have a financial interest in the securities of the following companies: none
2. I or a household member is an officer, director, or advisory board member of the following companies: none
3. I have received compensation within the past 12 months from the following companies : none
4. Piper Jaffray or its affiliates beneficially own 1% or more of any class of common equities of the following companies: none
5. The following companies have been investment banking clients of Piper Jaffray during the past 12 months: SGMO
6. Piper Jaffray expects to have the following companies as investment banking clients within the next three months: none
7. Other material conflicts of interest for Ted Tenthoff or Piper Jaffray regarding companies in my universe for which I am aware include: none
8. Piper Jaffray received non-investment banking securities-related compensation from the following companies during the past 12 months: none
9. Piper Jaffray makes a market in the securities of the following companies, and will buy and sell the securities of these companies on a principal basis: ACAD, ALNY, ALXA, ANDS, ARRY, CRXX, CYTX, DCGN, EXEL, HGSI, INFI, ISIS, LXRX, MYGN, OSIR, SGMO, ZGEN

1. Poniard’s Platinum Compound for Oncology, A Winning Design for its Clinical Trial

How does one go about creating value with a platinum compound? In this interview Mike King asks Jerry McMahon of his rather broad use of Picoplatin in Oncology. They review the SPEAR clinical trial in Small Cell Lung Cancer, its design, endpoints, timeline, competitive landscape, and favorability with the FDA.

2. Picoplatin in Colorectal, Prostate, Ovarian Cancer and NSCLC, and Poniard’s Cash Position

Poniard has begun clinical trials of it’s Picoplatin in combination with other standard of care therapies. Jerry McMahon and Mike King discuss these indications and the different strategies for each one. Jerry tells us about the data presented in these trials at the latest ASCO meetings. Finally they review Poniard’s very attractive cash position, and its plans for partnering in the years to come.

This interview was conducted at the NASDAQ Marketsite, on September 8th, 2008, in New York City.

Featuring:

• Jerry McMahon, Chairman and Chief Executive Officer, Poniard Pharmaceuticals
• Michael King, Director of Research, Rodman & Renshaw

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. Nile’s Peptide for Heart Failure to Achieve Superiority through Hemodynamic and Renal Action

Peter Strumph describes Nile Therapeutics’ CD-NP peptide for the treatment of Acute Heart Failure, and the competitive landscape. Matt Kaplan asks him about the market size, existing products, and unmet needs. In answering these questions, Peter reviews the mechanisms of action of the peptides on the market and compares their mechanisms of action to Nile’s CDNP which is expected to have more potent Hemodynamic and Renal effects.

2. Nile’s CD-NP Phase 3 Strategy, and Secondary Pipeline Products, and 8 Person Team

Peter Strumph lines up the milestones to come for Nile’s lead compound CD-NP and gives us a closer look at his strategy for determining the right dosage in Phase 2, in order to adequately prepare his Phase 3. Peter and Matt then discuss CU-NP and 2NTX-99, other pipeline products, Nile’s 8 person team and leverage model, as well as future in-licensing.

This interview was conducted at the NASDAQ Marketsite, on September 3rd, 2008, in New York City.

Featuring:

• Peter M. Strumph, CEO, Nile Therapeutics
• Matthew L. Kaplan, Managing Director, Healthcare Group, Ladenburg Thalmann

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. Financing, Productivity, and the Cost of Innovation

Stelios Papadopoulos, veteran of the Biotech Industry, looks at the financing environment today, and the “windowless paradigm” we’ve been operating in since 2003. He emphasizes the importance of changing investor sophistication and investment horizons. Stelios also addresses the cost of innovation and overall sector productivity. Finally Stelios looks back on his expectations for the market today, when he prepared them as an analyst 20 years ago.

2. Biotech in 20 Years, Fundamental Science, Personalized Medicine, and Energy

Stelios shares his vision for Biotech in the next 20 years, comparing technical and fundamental problems, how they will be funded and solved. He also addresses the issue of energy, for which he thinks we will find biologically driven solutions. Stelios discusses the relevance of personalized medicine, and his choice of investment today.

This interview was conducted at the NASDAQ Marketsite, on June 25th, 2008, in New York City.

Featuring:

• Stelios Papadopoulos, Chairman of the Board/Director/Founder at Exelixis, Inc.
• Jean-Loup Romet-Lemonne, President & CEO of BioBusiness.TV

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

Vivalis uses Embryonic Stem Cell Technology to Enable Vaccine and Antibody Production.

Chicken eggs revolutionized the production of vaccines. Could animal stem cells be the new chicken egg? Vivalis has created a technology platform based on embryonic stem cells that enables the production of vaccines and antibodies. Vivalis’ CSO Majid Mehtali is interviewed by Reni Benjamin, analyst at Rodman & Renshaw. The business model is based on technology licensing, with future ambitions for own product development. Majid and Reni review comparables, Vivalis’ cash position, Euronext vs. NASDAQ listing, and the potential for acquisitions.

This interview was conducted at the NASDAQ Marketsite, on July 17th, 2008, in New York City.

Featuring:

• Dr. Majid Mehtali, CSO of Vivalis Reni Benjamin, Managing Director & Senior Biotechnology Analyst at Rodman & Renshaw
• Jean-Loup Romet-Lemonne, President & CEO of BioBusiness.TV

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

1. Standardizing Ways to Create New Treatments for Under-served Disease Markets, with Stem Cell Technology

Can stem cell technology be used to make under-served disease markets commercially relevant? Professor Marc Peschanski is working with stem cells (embryonic and adult) to create therapeutics for monogenic diseases. His goal and that of his institute are to standardize new ways of creating treatments for monogenic diseases, developing R&D with an emphasis on the technology platform.

2. Is Europe attractive for stem cell research? Which countries are the most advanced and have the most favorable legislation?

Professor Marc Peschanski discusses the different european legislations, looking at the countries that have taken the lead, and those quickly progressing – including the U.K., Germany, and Spain. He then continues to explain changes in French law that are making stem cell research possible.

3. Stem Cell Research Financing, Products, Business Models, and Regulatory Concerns

Why is stem cell financing limited, and how does I-Stem finance its research? Professor Marc Peschanski looks at the capital question, and potential applications of stem cell research. He then reviews the concerns of the regulators and compares the FDA and EMEA with respect to stem cells.

4. Will Adult Stem Cells Resolve Our Moral Quandaries? Do They Offer The Same Benefits As Embryonic Stem Cells?

Is the public opinion antagonistic towards, divided on, or supportive of stem cells? Professor Marc Peschanski explains the state of the debate, now that stem cells can be derived from adult tissue. He also comments on the applicability of human embryonic stem cell technology towards adult stem cell technology.

This interview was conducted at the NASDAQ Marketsite, on June 10th, 2008, in New York City.

Featuring:

• Marc Peschanski, Scientific Director for I-Stem, the Institute for Stem Cell therapy and Exploration of Monogenic Diseases
• Jean-Loup Romet-Lemonne, President & CEO of BioBusiness.TV

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.