The TurnAround in Dendrion ? DNDN Presents at JP Morgan in SFO

Last week Dendreon NASDAQ: DNDN reported stronger than expected sales for Provenge sales at $82 Million generally above "street" consensus expectations. The company has a strong cash balance (approx. $600M) to fund the Provenge launch through to profitability. The stock rallied sharply starting the week at $8 and closing north of $12. This sets the stage for continued expansion for Dendrion and is welcome news for earlier stage cancer immune based therapies in development.

This week the JP Morgan Healthcare Conference begins in San Fransico, Monday through Thursday. Healthcare companies from all over the world converge for a week in meetings with sell side analysts, bankers and institutional portfolio managers.

We have been highlighting the Alliance for Regenerative Medicine (ARM) efforts as an industry group to highlight the progress that cell based companies have made over the past decade. To this end, ARM has organized a two day mini-conference (we have previously posted the schedule). What's special about the ARM event is that the companies will all present in a panels style format.

Industry leaders like Dendrion (presenting on a panel Wed) will be alongside other earlier stage companies in their market niche such as Immunocellular (IMUC), Coronado (CNDO), PrimaBioMed (PRR.AX) as well as other players in the space. We believe this will be a great venue for investors to learn not only about where Dendrion may be going as a stock, but what's next in the cancer-immunotherapy space.

We will be present throughout the day and blogging on the various panels as we go through the motions of comparing and contrasting the various approaches of the companies. For example we are keenly focused to listen to the cardiology panel where industry leader MesoBlast (MSB) will present alongside NeoStem (Amorcyte-NBS), Athersys (ATHX) and Cytori (CYTX).  Baxter (BAX) will not be present for the panel but investors should take note of their progress (P3 trial in cardiac ischemia).

We believe the next major breakthroughs on the regen side will likely be found in the cardiology space.

The Alliance for Regenerative Medicine Announces Agenda for Regenerative Medicine Insight Track at Biotech Showcase on January 10 and 11 in San Francisco

Speakers include Advanced Cell Technology, Athersys, Cytori, Dendreon,

Genzyme-Sanofi, Johnson & Johnson, Mesoblast and Shire

The Alliance for Regenerative Medicine (ARM), the international organization representing the interests of the regenerative medicine (RM) community, today released the agenda for its second annual State of the Industry Briefing and RM Insight Track. The meeting will take place on January 10 - 11, 2012 at the Park 55 Wyndham in San Francisco.

The Regenerative Medicine State of the Industry Briefing will focus on recent developments and the future outlook for regenerative medicine, cell therapy and other advanced therapeutics. A full day of panels focused on several disease areas will follow the briefing as part of the Regenerative Medicine Insight track at the Biotech Showcase 2012. The panels will be moderated by key analysts in the sector. Please see agenda below:

Tuesday, January 10, 2012

8:00 – 9:30 AM Regenerative Medicine State of the Industry Briefing

• Gil Van Bokkelen, CEO, Athersys (Moderator)
• Silviu Itescu, CEO, Mesoblast
• Tim Mayleben, CEO of Aaastrom
• Gary Rabin, CEO, Advanced Cell Technology
• Robin Smith, CEO, NeoStem
• Leanna Caron, VP & GM of Cell Therapy & Regenerative Medicine, Genzyme-Sanofi
• Ed Field, COO, Aldagen
• Jay Seigal, CBO, Johnson & Johnson
• Dean Tozer, Senior VP of Corporate Development, ABH-Shire
• Susan Solomon, CEO, New York Stem Cell Foundation
  

Therapeutic Focus Panels

9:30 - 10:45 AM Cardiovascular, AMI and PAD—Part 1

Analyst: Jason Butler, JMP Securities

• Amorcyte, Andrew Pecora, CSO
• Cytori, Marc Hendrick, President
• Athersys, Gil Van Bokkelen, CEO                               
• Mesoblast, Silviu Itescu, CEO                              

10:50AM - 12:15 PM Cardiovascular, CLI and Stroke—Part 2

Analyst: Ren Benjamin, Rodman and Renshaw

• Aastrom, Brian Gibson, VP Finance
• ReNeuron, Michael Hunt, CEO                             
• Aldagen, Ed Field, COO                            
• Tissue Genesis, Anton Krucky, CEO                                      
• SanBio, Keita Mori, CEO

1:45 – 3:00 PM Neurodegenerative Disease, Ophthalmology and Spinal Injury  

Analyst: Steve Brozak, WBB Securities

• StemCells, Inc., Martin McGlynn, CEO 
• NeuralStem, Richard Garr, CEO                           
• InVivo, Frank Reynolds, Chairman of the Board, CEO, CFO,
• Advanced Cell Technology, Gary Rabin, CEO

3:00 – 4:15 PM Renal and AutoImmune Disease

Analyst: Kai Makay, Roth Capital

• Tengion, Mark Stejbach, VP, CCO
• Allocure, John Wirthlin, COO
• Tigenix, Eduardo Bravo, CEO
• Q Therapeutics, Deborah Eppstein, CEO 

4:20 – 5:45 PM Tissue Engineering, Orthobiologics and Artificial Conduits to Wound Care and BioPrinting

Analyst: Jeffery Cohen, Ladenburg Thalmann & Co.

• ISTO, Scott Gill, CFO
• Organovo, Keith Murphy, CEO                   
• ABH - Shire, Dean Tozer, Senior VP of Corporate Development
• Healthpoint, Rob Bancroft, VP, Strategic and Corporate Development
• Axogen, Karen Zaderej, CEO
• ·      Harvard Biosciences, David Green, CEO

Wednesday, January 11, 2012

9:00 - 10:30 AM Hematology &  Immunology

Analyst: Stephen Dunn, LifeTech Capital

• Dendreon, David L. Urdal, Executive VP, CSO                                         
• Prima BioMed, Martin Rogers, CEO                               
• ImmunoCellular, Manish Singh, CEO
• Coronado, Bobby Sandage, CEO
• bluebird bio, Jeffrey Walsh, COO
• NexImmune, Ken Carter, CEO                                           
• Maxcyte, Doug Doerfler, CEO

To learn more and to register for the conference, please contact Rob Margolin at (646) 201-4192.  Registration is complimentary for credentialed members of the media and the investment community. The State of the Industry briefing is open to the public, but attendance at the disease panels will require registration for individuals without press or investor credentials. 

About Biotech Showcase™ (Innovation – Opportunity – Collaboration):

Now in its fourth year, Biotech Showcase™ will feature corporate presentations by 200+ innovative life science companies to an audience of public and private investors, business development executives and professional advisors who are interested in investment opportunities and collaboration.  The showcase takes place during the week of the annual JP Morgan Healthcare Conference, one of the most important healthcare industry events of the year which attracts thousands of healthcare and life science business executives to San Francisco. Biotech Showcase was co-founded by Demy-Colton Life Science Advisors and EBD Group.  Please visit their website for more information:  www.biotechshowcase.com.

About The Alliance for Regenerative Medicine (ARM)

ARM is a Washington, DC-based non-profit organization that promotes legislative, regulatory, reimbursement, and financing initiatives necessary to facilitate access to life-giving advances in regenerative medicine. ARM also works to increase public understanding of the field and its potential to transform human healthcare, and provides services to support the growth of its member companies and organizations. Prior to the formation of ARM in September 2009, there was no advocacy organization operating in Washington, DC to specifically represent the interests of regenerative medicine companies, research institutions, investors, and patient groups supporting more rapid adoption of technologies in our field. To learn more about ARM or to become a member, visit www.alliancerm.org.

Ventrus Biosciences (NASDAQ: VTUS) works to provide prescription alternatives for treatment of gastrointestinal disorders

Ventrus Biosciences (NASDAQ: VTUS) is a development stage specialty pharmaceutical company focused on the development of late-stage prescription drugs for gastrointestinal disorders, specifically hemorrhoids, anal fissures and fecal incontinence.

Last week, Ventrus announced that S.L.A. Pharma (Private), its EU partner, is ahead of schedule and has completed enrollment in the first of two pivotal Phase III studies with Diltiazem (VEN 307) for the treatment of anal fissures. Diltiazem is an off-label calcium channel blocker that is commonly used to treat anal fissures. 

Although Diltiazem has been shown to be effective, it remains difficult for consumers to access. Because it is off-label, patients need to find a special pharmacy that can make it, which is a hassle for pharmacies, doctors and patients. 

Ventrus hopes to resolve the situation by bringing a quality controlled, clinically trial proven product to the market. Avoiding surgery and having a prescription (ie, reimbursed) alternative is a genuine patient need.

The company’s other lead product is VEN 309, Iferanserin. This product, like VEN 307, is a topical treatment for hemorrhoids. The first Phase III trial started in August 2011 and top-line data is expected in June 2012. VEN 309 can be thought of as a prescription alternative to Preparation H, an over-the-counter cream.

Given that an increased risk of hemorrhoids is yet another consequence of leading a sedentary lifestyle, the market opportunity for a new Rx product for hemorrhoids is significant.

Alliance for Regenerative Medicine, January 10, 2012, San Francisco

The Alliance for Regenerative Medicine Announces Presenting Companies and Second Annual State of the Industry Briefing and Regenerative Medicine Insight Track at Biotech Showcase

Event Takes Place on January 10, 2012 in San Francisco; Expanded Agenda Includes Panel Sessions on Major Regenerative Medicine Target Indications

PRESENTING COMPANIES: Aastrom Biosciences, Advanced BioHealing, Advanced Cell Technology, Aldagen, Allocure, Amorcyte, Athersys, Axogen, Coronado Biosciences, Cytori Therapeutics, Dendreon, Harvard Biosciences, Healthpoint, ImmunoCellular Therapeutics, InVivo Therapeutics, ISTO Technologies, Maxcyte, Mesoblast, MolMed, Neostem, Neuralstem, NexImmune, Organovo, Prima Biomed, Q Therapeutics, ReNeuron, SanBio, StemCells, Inc., Tengion, TiGenix, Tissue Genesis

The Alliance for Regenerative Medicine (ARM), the international organization representing the interests of the regenerative medicine community, today announced that the second annual State of the Industry Briefing will take place on January 10, 2012 in San Francisco. A full day of panels focused on several disease areas will follow the briefing as part of the Regenerative Medicine Insight track at the Biotech Showcase 2012. The panels will be moderated by key analysts in the sector.

The Regenerative Medicine State of the Industry Briefing will focus on recent developments and the future outlook for the cell therapy and regenerative medicine industries. Speakers will include Gil Van Bokkelen, CEO of Athersys; Silviu Itescu, CEO of Mesoblast; Tim Mayleben, CEO of Aaastrom; Gary Rabin, CEO of Advanced Cell Therapy; Dr. Robin Smith, CEO of NeoStem and senior executives from large pharmaceutical companies. The panels will cover the outlook for the industry, a review of pipelines and clinical trials, and a general discussion of critical success factors including technology advancements, regulatory issues and the financial outlook for the sector.

A series of analyst-led, therapeutic area focused panels will follow the briefing. The panels will discuss therapeutic categories where regenerative medicine holds great promise including cardiology and ischemic related disease, spinal injury, ophthalmology, metabolic disease, and central nervous system disorders as well as an exploration in cell based solutions in immunology and oncology. The panels will be moderated by analysts from Rodman & Renshaw, Piper Jaffray, JMP Securities, ROTH Capital Partners, and WBB Securities and feature participation from more than 25 companies including:

Aastrom, Advanced BioHealing/Shire, Advanced Cell Technology, Aldagen, Athersys, Cytori, Healthpoint, ISTO Technologies, Maxcyte, Mesoblast, Neostem, Organovo, ReNeuron, SanBio, StemCells, Inc., Tengion and Tigenix.

"We are excited to once again bring together many of the leading experts in the regenerative medicine field to report on the progress made during 2011 and provide an outlook for 2012 and beyond," said Gil Van Bokkelen, PhD, Chairman of ARM and CEO of Athersys. "Based on the significant response to the meeting last year, it's clear that recognition of the near-term therapeutic promise of regenerative medicine is growing, and this event represents a great opportunity for investors and others to hear from industry leaders and gain insight into the tremendous potential of the field."

This event was developed by the Alliance for Regenerative Medicine, and will be held as part of the Biotech Showcase 2012 conference taking place at the Park 55 Wyndham in San Francisco beginning at 8:00 a.m. To learn more and to register for the conference, please contact Rob Margolin at (646) 201-4192. Registration is complimentary for credentialed members of the media and the investment community. The State of the Industry briefing is open to the public, but attendance at the disease panels will require registration for individuals without press or investor credentials.

The Alliance for Regenerative Medicine (ARM) is a Washington, DC-based non-profit organization that promotes legislative, regulatory, reimbursement, and financing initiatives necessary to facilitate access to life-giving advances in regenerative medicine. ARM also works to increase public understanding of the field and its potential to transform human healthcare, and provides services to support the growth of its member companies and organizations. Prior to the formation of ARM, there was no advocacy organization operating in Washington, DC to specifically represent the interests of regenerative medicine companies, research institutions, investors, and patient groups supporting more rapid adoption of technologies in our field. To learn more about ARM or to become a member, visit www.alliancerm.org .

Media Contact:
      Michelle Linn
      Linnden Communications
      Phone: 508-362-3087

Oncothyreon (ONTY): Files IND for ONT-10 - Vaccine for Ovarian Cancer: Can you say PrimaBioMed ?

News on the tape today that Oncothyreon (ONTY) has filed an investigational new drug (IND) application for ONT-10. ONT-10, a therapeutic vaccine directed at cancers expressing MUC1. Upon completion of the FDA's review of the IND, Oncothyreon expects to initiate a Phase 1 trial of ONT-10. We recently wrote on this and discussed its implications for PrimaBioMed. Please see that note here.

Coronado: CNDO - Makes the NASDAQ

Coronado began trading on the NASDAQ as CNDO, and was off in its first day of trading, $6 (down $0.50). Coronado is a fascinating story in immunology on two fronts, on the cell therapy side with an NK cell that rebuilds patients immunity and the other product CNDO-201 is a "whip worm" product. Normally we would consider this a parasite but in this case, the ova from the porcine helminth Trichuris suis may work symbiotically in man, to help re-balance out of balance "auto-immune" system. A phase IIb trial is scheduled to begin in 1Q-2012 to treat Crohn's disease patients but the implications here could reach as far as Multiple Sclerosis (MS).

Please see our write-ups here on Daily Dose and also on the ProActive Coronado page. We know that National Securities and their army of 750 retail brokers helped raise the initial capital, (as they did for Ventrus -VTUS) last year to bring this company public. As of September, the company has $26.7 million in cash. Coronado starts trading with a strong retail following as management now works to create an institutional following.

Insider Buying at NeoStem ($NBS) supports a Solid Therapeutics Story build on a Manufacturing Platform- PCT

There is nothing we like to see more than insiders putting their money with shareholders. We took note of several insiders from Neostem (NYSE AMEX: NBS) including CEO Dr Robin Smith, CMO Dr Andrew Pecora, and VP Business Development, Jason Kolbert, all making inside purchases.

NeoStem has been popping up on a radar screen a lot these days. First with the acquisition of Progenitor Cell Therapy earlier this year, and now the acquisition of Amorcyte (P2 asset for AMI). This trial is expected to enroll its first patient in Q1-2012 and according to management is ready to go. We expect the trial to enroll the target 160 patients within 12 months with data read-out 6 months afterwards.

We do know that the Amrocyte product is differentiated. It is an enriched cell population, bone marrow derived of CD 34+ cells, and that the company established a biologically effective threshold dose that must be met (>10 mln cells) to show efficacy. NeoStem talks in great definition about the mechanism of action, the dose, the biological (angiogenesis) and clinical effect they intend to measure, perfusion. There is ample historical evidence to suggest that CD34+ cells are potent ones for neoangiogenesis. A recent paper published by Baxter principal investigator and thought leader, Dr. Douglas Losordo suggests that among all the cells types, CD34+ are the most potent. (Baxter is moving forward in a P3 trial with a CD 34+ cell) for cardiac ischemia. The Baxter trial harvests the cells from peripheral blood after delivering a mobilizing agent (GCSF). Our understanding is that these cells are not as effective at homing, but that fine as Baxter delivers them locally using direct injection to the heart itself (where NeoStem injects into the infarct related artery) and allows the cells to home along the ischemic (oxygen starved) gradient travelling to where they are needed. We believe that the historical literature does not lie and that Baxter offers proof of concept for NeoStem.

Some question if the autologous model is viable in the wake of Dendrion. We would remind folks that NeoStem acquired both Progenitor Cell therapy (PCT) and Amorcyte for a 1-2 punch. PCT did the majority of clinical autologous manufacturing for Dendrion's Provenge. So PCT knows how to manufacture. We also know that autologous has lots of advantages over allogeneic.

While Bears are quick to point out the advantages of pills in a bottle model (allogeneic) the data suggests that only autologous cells (your own cells) will truly integrate into the target (your own heart) and continue to modulate neoangiogenesis. NeoStem has publically declared that they plan to divest their majority ownership in the China generic company they own. That should be a catalyst for the stock too. So by comparison to the rest of the field we see NeoStem with a solid manufacturing base of operations in PCT (several contract manufacturing clients in Phase 3, Phase 2 and Phase 1 generating revenues and creating options for commercial manufacturing down the road as one of the better positioned companies in the space. Apparently the insiders agree.

Geron versus PluriStem @ Seeking Alpha? That's the wrong question......

We saw an article at Seeking Alpha talking about Pluristem versus Geron. The PluriStem CEO does a compare and contrast. While PSTI CEO Zami makes some good points regarding market size, were not sure that any comparison to Geron makes any sense.

Allogeneic cells (other peoples cells) do fit the pills in a bottle model that Pharma craves but as we have heard Zami himself say at multiple conferences, these allogeneic cells (even though they are from a placenta) do not integrate long term to the host. Therefore the effect is likely moderate, and that may be fine depending on the indication.

CLI (Critical Limb Ischemia) itself is a very variable disease and PluriStem has Phase 1 data and is just beginning their Phase 2 trial. So its early days for sure. Aastrom is also pursuing CLI and now is in a Phase 3 trial. As we have said in the past we are hopeful that the results will be good for both companies but neither company has definitive data sets yet and the disease itself is very variable. As such investors can't be sure what the outcome of these trials might be. In the case of Aastrom the P2 first interim analysis looked great but the second did not (the amputation rate in the placebo group improved and narrowed the difference between active and control). CLI is a variable disease and this happens. Aastrom is smart, they expanded the P3 trial (made it larger), which gives them a greater chance of success but it also means it will be more expensive and probably take longer to complete.

We would push the concepts here back to basics and get away from a comparison between an off the shelf allogeneic cell that is targeting angiogenesis (new blood vessel formation), improving circulation versus an embryonic approach that it was hoped could repair damaged signal conduits (nerves), paralysis.

Rather we ask how did CLI patients become ill in the first place? Poor circulation, diabetes, and other co-morbidities are likely part of the problem. So while fixing circulation, locally, may stave off an amputation, it seems like it is not addressing the underlying cause. Why inject intra-muscular versus intra-arterial? Basic science questions such as dose, biological mechanism of action, clinical effect, even product variability remain issues with many of the institutional investors who are following these cell therapy companies.

We note that Biotech giant is Celgene (CELG) is also working on placental based cell therapy but here it is for down-regulation inflammation, in Crohns Disease for patients who have failed steroids. We also know that Athersys and Pfizer are pursuing this approach also with an allogeneic therapy (MultiStem). Proof of Concept from a Celgene or a Pfizer would be a big deal for all in the space.

We certainly believe that there is a role for allogeneic therapy as well as autologous and that indications should dictate, which therapy is best for which indication. The lower cost of goods and the multiple treatments likely for CLI do favor allogeneic players where the opposite is true in cardiovascular disease (heart attacks, CHF and cardiac ischemia). For example news today that Cytori has filed its Investigational Device Exemption (IDE) application to begin a clinical trial of the Celution® System for chronic myocardial ischemia (CMI). Cytori's ATHENA represents a device-based (Phase I/II) to investigate the use of autologous, clinical-grade adipose tissue (fat) derived stem and regenerative cells (ADRCs). This all happens at the patients point-of-care with Cytori’s Celution® System.

The institutional community evidenced by stock prices is not excited about CLI (long and expensive trials, variable disease), but granted, very much an unmet medical need. So while Geron's departure in the space is (was) a disappointment the clinical advances of other companies like MesoBlast, Athersys, Cytori, Aastrom, NeoStem, and yes PluriStem are positive.

In fact we are excited to see what happens with the work that PluriStem is doing with United Therapeutics in the COPD (airways) space.