Cell Therapy in Cardiology: Conference Begins in NYC Tomorrow

Just a reminder that tomorrow through Friday a very important conference in NYC takes place: The Conference on Cell Therapy for Cardiovascular Disease.

Web site: http://celltherapy.crf.org/conference/overview.html

This is an off-wall street conference that is focused on reaching the medical community: Clinicians and clinical investigators, interventional cardiologists, noninvasive cardiologists, cardiac surgeons, research associates, basic science investigators (cell and molecular biologists), hospital administrators, program directors, legislators, and regulatory agency personnel to name a few. We will be attending.

A few key panels to be aware of:

• Wednesday: Industry Session I. Focus on Acute Injury Technologies: 4:30 pm - 5:30 pm. The participants include Linda Marban, Alexander M Milstein, Andrew Pecora (NeoStem CMO), Kai Pinkernell, Anthony Ting, Christine Wallrapp
• Thursday: Acute Injury: STEMI and NSTEMI, 8:45 am - 10:15 am. Moderators: Timothy D. Henry, Andreas M. Zeiher. Participants: Henricus J. Duckers, Raj Makkar, Marc S. Penn, Arshed A. Quyyumi (PI in NeoStem’ s Amorcyte trial) , Warren Sherman, Wojciech Wojakowski. We note that Dr. Zeiher is a moderator. Dr Zeiher is concerned one of the most prominent thought leaders in cell therapy and cardiology.
• Thursday 10:45-12:15: CLI/CMI. Moderators: Douglas W. Losordo, Amit Patel. Discussants: Todd J. Brinton, Timothy D. Henry, Thomas F. O'Donnell, Emerson C. Perin, Christof Stamm, Gustav Steinhoff

On this panel we note the presence of Doug Losordo who is also one of the most respected key opinion leaders in cell therapy and is also a pioneer on CD34+ cells and cardiology. Dr. Losordo is the PI in Baxter's CD34+ trial. NeoStem also is running a CD 34+ trial.

• Trial 1: CD34+ Cells for Refractory Angina: ACT 34 CMI Update and Phase 3 Study Design. Timothy D. Henry
• Trial 2: Adipose-Derived Cells for Chronic Ischemia: The PRECISE Trial, Emerson C. Perin
• Trial 3: Update on Standardization of CD133 Intramyocardial BMSC for Intramyocardial Delivery, Gustav Steinhoff
• Trial 4: BMAC for CLI: A Phase III Trial, Thomas F. O'Donnell
• Trial 5: Placenta-Derived Cells for CLI, Christof Stamm

Immunocellular (IMUC) - Update(s) Phase II trial of ICT-107 for Glioblastoma

ImmunoCellular (OTCBB: IMUC) provided an update on the site and patient enrollment for ICT-107, currently in a Phase II trial for Brain Cancer. Recall that this is a dendritic cell based cancer vaccine targeting multiple tumor antigens for the treatment of glioblastoma multiforme (GBM or brain cancer).

• The company initiated the trial in 23 centers to enroll up to 160-200 patients, (100 with the HLA-A1/A2 immunological subtypes). 115 patients are enrolled in the study to date, ahead of schedule.
• Enrollment for the trial is expected to be completed by Q2 of 2012 and an interim analysis is expected when 50% of events (32 deaths) have been observed.
• NeoStem's, Progenitor Cell Therapy (PCT) is working with Immunocellular on the "manufacturing".

Conclusion: We discussed IMUC last week and we believe that this dendritic approach is showing compelling data in this unmet medical need. PCT (NeoStem) eliminates any manufacturing concerns, (PCT did most of the clinical manufacturing for Dendreon's Provenge). IMUC looks like an attractive play, and the company is financed through to proof of concept, given that capital was raised earlier in January 2012.

See all Daily Dose articles on IMUC.

WBB Upgrades NeoStem ($NBS), Target = $2.00

WBB Securities upgraded NeoStem (NASDAQ: NBS) to a Buy with a 12 months price target of $2.00 per share. The report cited a few reasons which include:

1. Expansion of the intellectual property associated with Amorcyte beyond STEMI (Heart attacks) to all Vascular Insufficiency.
2. New PCT clients on-board such as Immunocellular (IMUC) - (working on a dendritic cell based approach to brain cancer), and Islet BioSciences (microencapsulated insulin-producing, pathogen free swine (pigs) for type 1 diabetes’s. NeoStem actually gets a royalty on commercial sales (utilizing PCT's manufacturing currency to participate in clients downstream revenues).
   
3. Divestiture of China Generic company (WBB estimates $25 million).
4. The start of the Amorcyte Phase II trial.

ARM Panel shines light on the Cell Therapy Cardiovascular Space

This morning in SanFransico the ARM panel on cardiovascular medicine is taking place. NeoStem (Amorcyte), MesoBlast (MSB), Athersys (ATHX) and Cytori (CYTX) are presenting their cell therapy approaches. We will follow up with comments from this panel and the panels that follow throughout the day. In other news NeoStem announced the expansion of their IP for AMR-001 (their CD34+ cell therapy product) beyond STEMI (heart attacks) and to all ischemic (vascular insufficiency) diseases. We looked at the patent and we see this as a landmark expansion in the space. Immunocelluar (IMUC) also had news today, raising capital (aprox $9.4 mln) which will be used to fund the development of their dendritic approach to brain cancer (glioblastoma). We note that IMUC is working with NeoStem's CMO subsidiary, Progenitor Cell Therapy (PCT).

Athersys (ATHX): New IP that Protects MultiStem (but does not block others) !

Athersys (ATHX) announced earlier in the week a new patent has been granted (U.S. patent 8,075,881) that covers the use of "non-embryonic multipotent stem cells for the treatment of cardiovascular conditions including myocardial infarction, or heart attack, congestive heart failure, myocardial ischemia and other heart conditions".

From the company's press release: "The patent also provides protection for multiple techniques for delivering the stem cells to treat the conditions. Athersys also announced the issuance in 2011 of more than 25 patents in the U.S. and other countries covering non-embryonic multipotent stem cells, their production, and usage.

The issued patents cover Athersys' proprietary MultiStem has demonstrated therapeutic potential to treat a broad range of indications in the cardiovascular, neurological, and inflammatory and immune condition disease areas. Athersys currently has four clinical stage programs in acute myocardial infarction, ulcerative colitis, hematopoietic stem cell transplant support and ischemic stroke using its MultiStem product candidate."

We reviewed the IP claims as many questions were raised about how this IP impacts other companies also pursuing the use of adult stem cells in cardiology, MesoBlast, Cytori, NeoStem, Baxter and we found a few claims that narrow the Athersys IP around a specific cell type where the IP specifies this is for cells that are "mononuclear, centrifuged, and depleted (of CD45 and GlyA). The remaining cells are plated and, with successive doublings in culture, becoming more homogenous - by 10 doublings. So what does this all mean? It means that Athersys has IP to protect MultiStem but this is really not "blocking IP" that locks anyone else out of the 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.

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

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.