Cytori (CYTX) : FDA Green Lights the Start of a Trial based on an IDE

Cytori Therapeutics (NASDAQ: CYTX) received an Investigational Device Exemption (IDE) approval from the U.S. FDA to start their Phase 1 trial; "ATHENA". ATHENA will investigate the use of the Celution® System, (this is the "bedside centrifuge" that processes stem cells from fat (adipose-derived stem and regenerative cells ADRCs) to treat a form of coronary heart disease, chronic myocardial ischemia (CMI).

This IDE approval comes pretty quickly given that the application was originally submitted to the FDA in December 2011.

ATHENA is a multi-center, randomized, double blind, placebo controlled, pilot trial to investigate the use of autologous, clinical-grade ADRCs, processed at the point-of-care with the Celution® System. The trial will enroll up to N=45 patients with no-option CMI who have limited therapeutic options.

It will evaluate a variety of clinical and functional outcomes, including safety, peak oxygen consumption (mVO2), and clinical outcomes at 12-months.

Previously, Cytori reported six and 18-month trial data from PRECISE, a European clinical trial for this same indication showing improvement in mVO2. In Europe, Cytori has applied to expand its Celution® System CE Mark to include no-option CMI claims based on data from the PRECISE trial. Cytori is also enrolling ADVANCE, a European pivotal trial investigating the Celution® System for acute myocardial infarction (heart attacks).

Daily Dose Take-Away: This is an exciting and interesting approach. If the trial shows strong efficacy signals it has big implications for the space. The key issues here will be:

• Understanding what the cell population here actually is ?
  
• What is its effective dose ?
  
• How is biologic variability accounted for ?
  

For example, Baxter has completed a PII trial in the same indication, but with an enriched population of marrow derived cells (CD 34+) ? So the cell type, the active ingredient, the dose, the timing and delivery are critical variables (to name a few) for a large indication like this one.

NeoStem's ($NBS) First Patient is Enrolled in Amorcyte Trial

Wow, what a long way NeoStem (NYSE AMEX: NBS) has come. In the past year this company has been transformed from one focused on pre-clinical VSEL's which hold promise of being a naturally pluripotent adult stem cell to a Phase II clinical company with a well-designed, well vetted therapy (CD34+/CXCR4+ Cell derived from a patient’s own bone marrow) for the preservation of Heart function. The name of the trial is PreServe!

From the press release:

"The study is a multicenter, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety and efficacy of infarct-related artery infusion of AMR-001, an autologous bone marrow derived cell therapy enriched for CD34+ cells. AMR-001 is administered 5 to 11 days post-stent placement in patients diagnosed with an ST segment elevation myocardial infarction with ejection fraction less than or equal to 48%, as determined by cardiac magnetic resonance imaging."

This is a critical element of the NeoStem story. Unlike most of the other companies who are also working in the heart attack space, NeoStem is based on their understanding of the underlying biology of heart attacks. It typically takes 5-6 days for the ischemic signal from the healthy but overworked myocytes (heart cells) that surround the infarct tissue to build up to a point where the CD34 cells can migrate locally via their cell receptor (CXCR4). They then modulate the process of angiogenesis where it’s needed.

In other words, if you give cells too soon, it becomes cellular Advil (calming down inflammation) but not really modulating the localized needs around the peri-infarct zone, and if you give cells too late, the myocytes are not rescued. This is the only cell therapy company we are aware of that has been so explicit with their understanding of the active cell type (it’s not a gamish of stem cells, its CD 34+ cells), delivered at a specific dose, at a specific time.

Conclusion: Look for news from NeoStem on this trial which has the potential to transform the way heart attacks are treated. This will take 12 months to complete enrollment with data 6 months after last patient is treated. Also keep an eye out for the company to divest their 51% ownership in the China Generics company. This should be a welcome event by The Street that could unlock significant shareholder value.

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Today, January 25, 2011, NeoStem Senior Management, Subsidiaries Invited to Speak at Seventh International Conference on Cell Therapy for Cardiovascular Disease; $NBS

NeoStem, Inc. (AMEX: NBS; Stock Twits: $NBS) is an emerging biotechnology company engaged in the development and manufacturing of cell-based therapies in the U.S. Its January 2011 acquisition of Progenitor Cell Therapy, LLC ("PCT") is central to the Company's strategic mission of capturing the paradigm shift to cell therapy. The acquisition of PCT gives NeoStem not only access to a world class contract manufacturing cell therapy company but provides a platform and expertise around the evaluation, development and regulatory requirements to develop autologous, allogeneic, immunomodulatory and vaccine-based therapeutics. 

NeoStem announced that several of its representatives have been invited to present at the Seventh International Conference on Cell Therapy for Cardiovascular Disease held from January 25-27, 2012 in New York City. The invited include NeoStem’s Chief Medical Officer, the lead investigator for its PreSERVE Phase 2 clinical trial for AMR-001 for preservation of heart function in post acute myocardial infarction patients, and the President of Progenitor Cell Therapy, LLC, a NeoStem subsidiary. 

The Cardiovascular Research Foundation is organizing the conference, which will be held at the Myrna L. Daniels Auditorium in the Vivien and Seymour Milstein Family Heart Center at NYPH/Columbia University Medical Center.

The program “will be dedicated to the evolving field of cell-based therapies being developed for the repair and regeneration of cardiac and vascular disease, as well as related diseases such as diabetes and stroke.” The conference this year “will focus on preclinical and clinical studies on the path to commercialization, highlighting the status of molecular, cell, and tissue products in addition to delivery systems. Leaders from the international community will convene to present their work, experiences, observations, and opinions on the benefits and unmet challenges of cell-based therapies.”

NeoStem will present as follows:

• At 4:30 PM on Wednesday, January 25, Andrew L. Pecora, MD, FACP, Chief Medical Officer of NeoStem, will speak on an industry session entitled "Focus on Acute Injury Technologies."
• At 9:20 AM on Thursday, January 26, Arshed A. Quyyumi, MD, FRCP, FACC, principal investigator of the PreSERVE AMI Phase 2 trial and Professor of Medicine and Cardiology at Emory University, will speak on a panel entitled "Early and Next Phase Studies" within a session entitled "Acute Injury: STEMI and NSTEMI".
• From 7:30 to 8:30 AM on Friday, January 27, Robert A. Preti, PhD, President of Progenitor Cell Therapy will speak as part of Industry Session III entitled "Focus on Procedure, Systems Management, and Advanced Technologies."

Dr. Pecora, CMO of NeoStem, commented,

"As a presenter at the first industry session, I look forward to informing industry leaders about the PreSERVE Phase 2 clinical trial for AMI which is now open for enrollment and may provide data readout 18 months from the first patient enrollment, as well as discussing the recent patent issued to use the Amorcyte technology beyond cardiovascular disease." 

Chairman and CEO of NeoStem Dr. Robin L. Smith added,

"This conference represents validation that the development of cell therapies for diseases outside of bone marrow transplantation is progressing as represented by their current clinical data. Our acquisition of Amorcyte last year has positioned NeoStem to be a leader in the cell therapy cardiovascular space. We are excited to be a part of the developing science represented by this important conference and share our expertise represented by the progress with Amorcyte and our capabilities to assist the field with the manufacturing services of PCT."

Read the full news release at PR Newswire. 

Which Cell Therapy companies present the best value?

Which Cell Therapy companies present the best value?

We developed an analysis using public filings and annualizing the most recent quarters as of 12/31/11. As such, the accuracy is subjective to our proprietary algorithm, and this list is not mean to be all comprehensive.

We conclude that ex leaders Dendreon and MesoBlast make the space very inexpensive. The sample below represents just over $500 million in market cap for eight of the selected public companies (ex DNDN and MSB). These companies have 3 - PIII trials, 5 PII trials and several P1 trials (based on these crude metrics).

We conclude that is worth investors time to understand among these companies in terms of what they are doing and the associated probabilities of success of their trials. Recent data peaks from the Cardiology space suggests that Baxter (BAX) and their CD34 cell type may be very viable. NeoStem has a very similar approach. The retrenchment in MesoBlast shares since that company presented data at AHA in November is of concern. Cytori, Aastrom, Athersys all have active programs in cardiology while Athersys lead program is partnered with Pfizer for Ulcerative Colitis and Aastrom is entering a Phase 3 trial in CLI.

By price ($), Osiris, MesoBlast, and Dendreon are the highest $ priced stocks:

But that starts to change as we migrate to market cap:

And if we eliminate Mesoblast and Dendreon we get a better picture of the rest of the field:

And that picture changes as we adjust for debt and cash: (enterprise value). In all fairness here, we know that NeoStem with $20-$30 million in value from their China generic company, could be the cheapest name and others are / will raise capital but ex China, Athersys is the “cheapest name”.

Looking at R&D spending: IMUC, Prima and NeoStem are among the most efficient but in fairness one must adjust for the fact that Aastrom is embarking on a pivotal trial versus Prima just starting their P3 and NeoStem their P2.

Revenues: Here we have excluded NeoStem’s $65 million from China which means PCT (Cell Therapy CMO) is running at close to $10 million in annual revenues (currently).

Cytori (CYTX) Publishes Apollo (P1/2a Heart Attack) Data

Cytori – (CYTX):  Publishes Appolo Results

Cytori Therapeutics (NASDAQ: CYTX) published previously reported six-month outcomes from their Apollo trial. This was an EU trial evaluating adipose (fat) derived stem cells (ADCs) in patients with acute myocardial infarction (heart attack or AMI).

Apollo was small at N=14-patients but it was a prospective, randomized, double-blind, placebo-controlled, feasibility trial (Phase I/IIA).

In the Apollo trial all patients were treated with standard-of-care and subsequently underwent an abdominal liposuction. Each patient's adipose tissue was processed by the Celution® System where ADRCs were extracted, washed and concentrated into a syringe of clinical grade cells. Within 36 hours of the myocardial infarction and no longer than 24 hours after undergoing percutaneous coronary intervention, patients received an injection of either 20 million ADRCs (n=10) or a placebo (n=4). Improvement in cardiac function by SPECT  and an improvement in blood flow  as well as a reduction in scar formation (infarct size).

Cytori claims that the “advantage of adipose tissue as a cell source is that it allows physicians to get a meaningful dose of a patient's own cells at the point-of-care when using the Celution® System without cell culture or use of donor cells". The paper’s author also states, "We believe delivering cells within the first 24 to 36 hours takes advantage of the body's signaling and initiates the repair process before irreparable damage occurs".

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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

CBS NY - Applied DNA's ($APDN) Digital DNA’ May Soon Be Required To Take SAT And ACT Exams

Applied DNA Sciences Inc. (OTCBB: APDN; Twitter: $APDN) sells patented DNA security solutions to protect products, brands and intellectual property from counterfeiting and diversion. SigNature DNA is a botanical mark used to authenticate products in a unique manner that essentially cannot be copied, and provide a forensic chain of evidence that can be used to prosecute perpetrators.

On Monday, CBS New York’s Jennifer McLogan reported on what politicians will see first hand in Albany this afternoon, and what could soon be implemented in New York and at high schools around the country.

Inside the Applied DNA Sciences lab at Stony Brook University researchers are hard at work inventing and perfecting a system that can prevent cheating on SAT and ACT exams.

“A novel system that’s absolutely unbreakable for securing the identity of a student taking the SAT exam,” said Dr. James Hayward.

Applied DNA put out a press release this morning in anticipation of today's hearing which states:

Dr. James A. Hayward, President and CEO of Applied DNA Sciences, Inc. (OTC.BB: APDN.OB - News), has been invited by the Chairman of the New York Senate Higher Education Committee to speak about APDN's digitalDNA™ technology to help block cheating on standardized tests such as the SAT college entrance exam. The Chairman, State Senator Kenneth LaValle, has been pressing for a solution since dozens of individuals were arrested on Long Island last November for paying others to take the tests for them.

At least four test-takers have been accused of accepting cash payments of $500 to $3,600 to take the test for students, largely from Long Island's North Shore schools.

In his testimony, Dr. Hayward will detail how APDN's digitalDNA can aid in preventing a reoccurrence of the cheating scandal.

In a TV interview with CBS News on January 23, Dr. Hayward demonstrated the technology he called "a novel system that is absolutely unbreakable for students taking the SAT exams."

(A video clip of the interview may be found here, the CBS news story here).

The digitalDNA system creates a counterfeit-proof, secure ID card. The card is embedded with infinitesimal molecules of plant DNA segments that authenticate a student's identity in a way that is absolutely uncopyable. The anti-counterfeiting technology also features a scannable printed code, which visually represents the same identity information. Embedded within the iconic code, and in covert locations on the card, is the physical code of digitalDNA. A scan, using a Smartphone or other mobile device, may be done instantly for exam-screening purposes, or at any other point. If deemed necessary, a second level of screening is available: the plant DNA on the card may be swabbed and forensically evaluated; a system well-recognized by courts globally.

All information is sent wirelessly to, and stored in, an ultra-secure internet database in "a private cloud." Applied DNA Sciences is closely affiliated with Stony Brook University's Center of Excellence in Wireless & Information Technology.

In his planned testimony, Dr. Hayward comments:

"Botanical DNA markers transform SAT identification cards into robust evidentiary tools that can assist in criminal prosecution efforts. The challenge is to ensure that actual, eligible, registered participants complete their exams. The goal is preserving exam integrity and maintaining a level playing field for all participants, and the solution is enhanced preregistration in tandem with identity verification before and after exams. We are confident that digitalDNA can assist in achieving the goal of an effective, easy-to-use and affordable system for preventing testing fraud."

Applied DNA Sciences is well-known throughout Europe and the U.S. for using botanical DNA to create very high security anti-counterfeiting systems. Such a system, now being piloted by an agency of the Department of Defense, identifies counterfeit microchips before they make their way into the U.S. military supply chain. Product authentication using botanical DNA has proved to be virtually uncopyable and resistant to the most highly stressed environment -- a must for the military.

APDN has installed its products in Europe and the U.S., protecting banks and ATMs (U.S.), jewelry stores (Sweden), and cash-in-transit strong boxes (UK). Its Signature DNA cash-protection system has won two top policing prizes in the United Kingdom over the past two years for successful work with police in helping secure convictions of cash thieves.

The State Senate hearing on SAT cheating is scheduled for January 24th, between the hours of 12:00PM and 3:00PM Eastern Time in the Legislative Office Building, Hearing Room A in Albany, New York. This event will also be streamed live on the NY Senate, Committee on Higher Education webpage found at: http://www.nysenate.gov/committee/higher-education

SmartDNA to protect banks and ATMs, turn criminals into chumps; $APDN

Applied DNA Sciences Inc. (OTCBB: APDN; Twitter: $APDN) sells patented DNA security solutions to protect products, brands and intellectual property from counterfeiting and diversion. SigNature DNA is a botanical mark used to authenticate products in a unique manner that essentially cannot be copied.

One of APDN's products is SmartDNA, an intruder/offender tagging system. SmartDNA intruder/offender tagging devices are installed in retail locations, banks, industrial facilities - any place where the goal is to protect against theft or vandalism. If a crime is committed, the SmartDNAsystem is armed manually or through a central security system. When the intruder passes near the device, he is tagged with a DNA-marked spray that makes establishing crime linkage definitive. The DNA-marked spray provides a forensic chain of evidence that can be used to prosecute perpetrators...

...like this guy:

Criminals, you've been warned: hooded jackets and furtive moves are no match for DNA authentication.