Ventrus (VTUS): Hemorrhoid Drug-309- PIIb hits its Primary Endpoint

Last week Ventrus Biosciences (NASDAQ: VTUS) reported that the final results from its PIIb study of VEN 309 (Iferanserin ointment) for the treatment of internal hemorrhoid disease (HD) were published. This study was conducted in Europe (Germany) and was randomized, double-blind, t=14-day placebo-controlled trial evaluating the efficacy and tolerability of BID (twice-daily) VEN 309 for the cessation of bleeding and other symptoms associated with Hemorrhoids.

The study hit its primary endpoint demonstrating efficacy and safety. The primary efficacy endpoints was a severity scores for bleeding on days 7 & 14. Secondary efficacy endpoints were itching, pain and other related symptoms also on day 7 & 14. The study was well powered at n= 111 patients split between control (placebo) and active VEN 309 groups.

VEN 309 was associated with a statisically significant reduction in daily bleeding &  itching on both days seven and 14 (bleeding, p<0.0001 and p=0.0075, respectively; itching, p=0.0008 and p=0.027, respectively). In terms of pain relief the data showed a favorable trend but was not statisically significant. Given that this is a Phase II trial and not a Phase III trial we are not concerned, a larger Phase III (P3 n=600) should add enough pwer to see a significant result. 

VEN 309 is on track to be the first new treatment (FDA Approved) for hemorrhoids. VEN 309 is in a Phase 3 trial now with top-line results expected this spring to summer. 

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 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.

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

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.

Celladon is focused in Heart Failure...gets Fast Track Designation (private)

Cell Therapy fan's know that on the Regenerative Medicine front the next major breakthrough may very well be in cardiovascular medicine. Several companies are in trials now with a wide range of cell therapies to stabilize failing hearts. What caught our eye today is news that private biotech company Celladon received fast track designation for Mydicar.

Celladon's MYDICAR® is a genetically targeted enzyme replacement therapy that is designed to restore functional levels of "SERCA2a", (a regulator of calcium cycling and contractility). In other words, this enzyme is required for the heart to beat properly and in patients whose heart is failing leading the enzyme levels to decline. MYDICAR® is a gene that is delivered using a recombinant adeno-associated virus (AAV) as the vector. This is really high tech gene therapy at a cellular level.

So what does this have to do with regenerative cell therapy ?

The phase 2 trial results (previously announced) showed the therapy met its primary safety and efficacy endpoints at 6 months for high dose MYDICAR® versus placebo. Additionally, 12 months after receiving a single infusion of MYDICAR®, patients treated with the highest dose versus placebo had an 88% risk reduction (Hazard Ratio = 0.12, P=0.003) of major cardiovascular events such as death, or a need for a left ventricular assist device (LVAD), cardiac transplant, or episodes of worsening heart failure and the related heart failure hospitalizations.

So now we are starting to see cardiac trials with therapies that are reporting a preservation of heart function and risk reduction as the therapeutic goals. We believe that in the cell therapy space, we will see a host of therapies developed along these same lines. We would take note on the autologous side of Baxter (P3) trial, NeoStem (NBS) (P2) trial, Aastrom (P2) and on the allogeneic side, MesoBlast (MSB), Cytori (CYTX), Athersys (ATHX), and others.

Critical Limb Ischemia (CLI) gets a lot of attention with Aastrom (ASTM), PluriStem (PSTI) and Aldagen (private) all talking about the potential but investors have not been convinced. We would watch closely the developments on the cardiac side as the unmet medical need here remains large. 

Why? The historical data sets and trial designs are well understood, so the feasibility of a successful trial based on what's known about the space is positive. News like Celladon, and a close examination of the trial goals bode well for the space.

Tengion (TNGN) : NeoUrinary Conduit

Tengion had a press release earlier today, announcing that they will have several presentations on the company's Neo-Urinary Conduit™ (NUC) and Neo-Kidney Augment™ (NKA) programs at the TERMIS North America Annual Conference now being held (December 11-14), in Houston, TX. Reading the press release tells us that the data set is building for both the NUC and NKA programs. We believe that the market size on the kidney program is off the charts, but acknowledge that the program is still pre-clinical so it’s not a driver for the stock yet.

What we find striking here is the fact that Tengion was a rising star. IPO'd by two of the best healthcare Wall Street firms and since that time, the stock has plunged, now trading at a little over a $10 million market cap with some trading liquidity. According to the September SEC filing, the Company had $9 mln in cash and $11 mln in short term investments versus $1.9 mln ST debt and $3.3 LT debt.

We have seen the NUC device and the technology is impressive. We see a company like Tengion, and fast followers in the space like Organovo as breakthrough technology plays. Perhaps we will see interest from Medical technology companies who are more familiar with the intense surgical procedures and specialized markets for devices that take interest in a product like NUC.

Our View: We would not give up on Tengion here.

See all TNGN articles @ Daily Dose Equities.

Neo-Urinary Conduit: Oral Presentation

• Genetic Stability of Autologous Human Smooth Muscle Cells (http://www.tengion.com/news/documents/Justewicz-SMC-Stability-Tengion-TERMIS-NA-Houston-14Dec11.pdf)
• Characterization of Chemotherapy and/or Radiation Treated Human Smooth Muscle Cells for Use in Regenerative Medicine (http://www.tengion.com/news/documents/Justewicz-Cancer-Secretome-Tengion-TERMIS-NA-13Dec11.pdf)

Neo-Kidney Augment : Oral Presentations

• Selected Regenerative Renal Cells combined with Bioresorbable Natural Biomaterials Catalyze in vivo Kidney Tissue Regeneration (http://www.tengion.com/news/documents/2011-Jain-TERMIS-NA-Presentation.pdf)

• Multivariate Analysis of Preclinical Data from the Tengion Neo-Kidney Augment (http://www.tengion.com/news/documents/Spencer-TERMIS-Presentation_121411_rev2.pdf)