Bioheart (OTC: BHRT; Twitter: $BHRT)


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Bioheart (OTC: BHRT; Twitter: $BHRT) seeks to be the "go to technology partner for heart failure specialists and their patients".

  • This is far from an unbiased presentation, but a corporate pitch for investment. It's important to recognize and discriminate an impartial review from a sales job. There are excellent results from clinical trials using mesenchymal stem cells for allogenic therapy as well as cardiac stem cells for autologous therapy currently in clinical trials for heart failure. Also, the issues of myoblast engraftment, electrical coupling, durability of repair, and contribution to contractile performance of the heart remain. It is certainly possible that recruitment and activation of endogenous repair mechanisms plays a significant role in the observed effects that are, in fact, achievable with a variety of cell types other than myoblasts. The concept of myoblast therapy has had a decade-long head start in research because it began long before stem endogenous stem cell pools were recognized as participants in active ongoing tissue repair throughout life. Other tissue-specific stem cell types are catching up fast.
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Bioheart (OTC: BHRT; Twitter: $BHRT)

  1. 1. <ul><li>BIOHEART INC. </li></ul><ul><li>April 2011 </li></ul><ul><li>OTC: BHRT.OB </li></ul>
  2. 2. Forward-Looking Statement <ul><li>Except for historical matters contained herein, statements made in this presentation are forward-looking and are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Without limiting the generality of the foregoing, words such as “may”, “will”, “to”, “plan”, “expect”, “believe”, “anticipate”, “intend”, “could”, “would”, “estimate”, or “continue” or the negative other variations thereof or comparable terminology are intended to identify forward-looking statements. </li></ul><ul><li>  </li></ul><ul><li>Investors and others are cautioned that a variety of factors, including certain risks, may affect our business and cause actual results to differ materially from those set forth in the forward-looking statements. These risk factors include, without limitation, (i) our ability to obtain additional financing; (ii) our ability to control and reduce our expenses; (iii) our ability to establish a distribution network for and commence distribution of certain products for which we have acquired distribution rights; (iv) our ability to timely and successfully complete our clinical trials; (v) the occurrence of any unacceptable side effects during or after preclinical and clinical testing of our product candidates; (vi) the timing of and our ability to obtain and maintain regulatory approvals for our product candidates; (vii) our dependence on the success of our lead product candidate; (viii) our inability to predict the extent of our future losses or if or when we will become profitable; (ix) our ability to protect our intellectual property rights; and (x) intense competition. The Company is also subject to the risks and uncertainties described in its filings with the Securities and Exchange Commission, including the section entitled &quot;Risk Factors&quot; in its Annual Report on Form 10-K for the year ended December 31, 2010. </li></ul>
  3. 3. Company Thesis <ul><li>Bioheart is dedicated to the discovery and development of myoblast and stem cell therapies for heart damage: </li></ul><ul><li>MyoCell ® </li></ul><ul><ul><li>An autologous cell therapy comprised of muscle derived stem cells for congestive heart failure. </li></ul></ul><ul><li>MyoCell ® SDF-1 </li></ul><ul><ul><li>An autologous cell and gene therapy comprised of muscle derived stem cells transduced to express the protein stromal derived factor – 1 for congestive heart failure. </li></ul></ul>
  4. 4. Heart Disease is the Leading Cause of Death Worldwide <ul><li>According to the American Heart Association, approximately 5.7 million Americans suffer from congestive heart failure (CHF). </li></ul><ul><li>The cost of hospitalization and treatment for heart failure is twice that of all forms of cancer combined. </li></ul><ul><li>Treatment with drugs and devices is available but not all patients return to a normal lifestyle. </li></ul><ul><li>Myoblasts represent the most advanced cell therapy that can effectively be targeted to this patient population. </li></ul>
  5. 5. MyoCell ® Cell Therapy Overview <ul><li>Muscle stem cell-based therapy designed to treat heart damage by growing new muscle in damaged heart tissue. </li></ul><ul><li>Uses myoblasts (muscle stem cells) </li></ul><ul><ul><li>Patient-derived; reduces risk of tissue rejection </li></ul></ul><ul><ul><li>Committed to forming muscle, will not differentiate into other cell types or over-proliferate </li></ul></ul><ul><ul><li>Tolerates low-oxygen conditions present in scar tissue </li></ul></ul><ul><li>Large potential savings in healthcare costs </li></ul>
  6. 6. Myoblast Engraftment Post-Transplantation Contractile muscle tissue growing in the scarred portion of the heart following treatment with myoblast injections. Human Heart, Proof of Concept * Hagege et al., Viability and Differentiation of Autologous Skeletal Myoblast Grafts in Ischemic Cardiomyopathy, Lancet, Vol. 361, 2003: 491-492
  7. 7. MyoCell: Heart Failure Treatment Process Scar tissue following heart attack Injection of skeletal myoblasts into scar tissue using deflecting-tip catheter Cell manufacturing following thigh muscle biopsy 1 3 2
  8. 8. Bioheart Has Addressed CHF with Myocell Including third-party studies, myoblasts have been evaluated in at least 11 clinical trials involving more than 345 enrollees and more than 255 treated patients Trial # of Patients Status Primary Endpoints EU Phase I/II 5 Phase I 15 Phase I/II Completed Q2 2003 <ul><li>Serious adverse events </li></ul><ul><li>Global ventricular function </li></ul>MYOHEART (US, Phase I) 20 Completed Final Patient Treated Q4 2006 <ul><li>Serious adverse events </li></ul>SEISMIC (EU, Phase II-a with Control) 40 (14 control) Completed Final Patient Treated Q3 2007 <ul><li>Serious adverse events </li></ul><ul><li>LVEF </li></ul>MARVEL Trial (N. Am./EU: Phase II/III, Double-blind, Placebo-controlled) 150 anticipated (50 controls) Stage 1 Results Announced First Patient Enrolled and Treated Q4 2007 <ul><li>Serious adverse events </li></ul><ul><li>6-minute walk test </li></ul><ul><li>Quality of life score </li></ul>
  9. 9. Journal of the American College of Cardiology, Vol. 42, No. 12, 2003, Serruys, Smits et al. Study sponsored by Bioheart, Inc. LVEF Measured via LAO LV Angio. 3 month, p=0.009; 6 month, p=0.23. N = 5. Percutaneous Intramyocardial Transplantation of Autologous Myoblasts: Bioheart First-In-Man Experience LVEF (%)
  10. 10. Percutaneous Intramyocardial Transplantation of Autologous Myoblasts – BIOHEART Phase I/II Trial Results for these trials were not statistically significant due, in part, to the limited number of patients treated. Baseline 6 Months 12 Months N = 13 N = 13 N = 13 LVEF: Mean Baseline = 34.4% + 7.4 Mean 12-Month Follow-up = 36.6% + 9.6 NYHA Class Improvement
  11. 11. Measurements via PV Loop, n=5. 33 41 190 150 4.6 5.6 Percutaneous Intramyocardial Transplantation of Autologous Myoblasts – BIOHEART Phase I/II Trial LVEF CO LVESV
  12. 12. Bioheart Percutaneously Delivered Myoblasts Clinical Trial Data Summary EU FIM, Phase I/II Adjudicated Data (n = 5+15) Safety • 2 patient deaths (10%) Efficacy • 4/5 of patients improved one heart failure class • 1/3 of patients improved two heart failure classes • 1/3 of patients experience ~ 35% relative improvement in LVEF • 15.3% improvement in Wall Motion Score Index via stress echo • 13% reduction of LVED volume <ul><li>7 arrhythmic events reported </li></ul><ul><ul><li>5 possibly related to cell </li></ul></ul><ul><ul><li>therapy (25%) </li></ul></ul><ul><ul><li>All occurred during first </li></ul></ul><ul><ul><li>3 mos. post implant </li></ul></ul><ul><ul><li>Anti-arrhythmic medication </li></ul></ul><ul><ul><li>not prophylactically prescribed </li></ul></ul><ul><li>PVCs peaked at 3-weeks but were </li></ul><ul><li>reduced from 1.89% at baseline </li></ul><ul><li>to 0.04% at 12 mo. follow-up </li></ul>Bioheart Phase I/II Trial (005/006).
  13. 13. * All 5 treated patients withdrew due to changes in German biopsy regulations. ** Both control patients withdrew after knowledge of randomization allocation. Bioheart Percutaneously Delivered Myoblasts EU Phase II Trial (SEISMIC) 47 Patients Randomized: ICD Patients: 31 MyoCell ® , 16 Standard Medical Therapy Treatment Arm ( MyoCell ® 150 - 800 x 10 6 ) 26 ICD Patients Control Arm (Standard Medical Therapy) 14 ICD Patients Baseline Evaluation Screening: 62 ICD Patients 15 Screen Fails 5 Withdrawals* 2 Withdrawals**
  14. 14. SEISMIC NYHA heart failure class Improvement Myoblast therapy Control therapy N=26 N=23 N=22 N=20 N=14 N=9 N=12 N=13 BL 1 mo 3 mo 6 mo BL 1 mo 3 mo 6 mo
  15. 15. SEISMIC +60.3 m + 54.1 -0.2 m ± 177.1 Treatment N=26 N=21 N=19 Control Control N=14 N=12 N=13 448 m 441 m 406 m 466 m 6-Minute Walk Test Improvement Difference Between Baseline and 6 Months
  16. 16. SEISMIC Response to Treatment NYHA HF / 6MWT / MLFQ / LVEF
  17. 17. MYOHEART US Phase I Study Flow Clinical Sites: Columbia Presbyterian, Cleveland Clinic, Mayo Clinic, Minneapolis Heart, St. Joseph ’ s / ACRI Core Lab: Gentiae Clinical Research 2 injections (.25 cc) 6 injections (.25 cc) 18 injections (.25 cc) First Cohort (n=5) 25 x 10 6 cells 30 - Day Safety Evaluation Second Cohort (n=5) 75 x 10 6 cells 30 - Day Safety Evaluation Third Cohort (n=5) 225 x 10 6 cells 30 - Day Safety Evaluation Fourth Cohort (n=5) 675 x 10 6 cells 27 injections (.50 cc)
  18. 18. MYOHEART 6MWT Average 6 Minute Walk Test – All Cohorts 6MWT (meters) N=20 422 474 471 N=18 N=15 47 36 N=15 6MWT (meters) N=18 All Patients Paired Analysis p = .0074 p = .2126 474 N=17 42 p = .1367 N=17
  19. 19. MYOHEART MLHFQ Average Minnesota Living with Heart Failure Score All Cohorts MLHF Score N=20 51 34 33 N=19 N=18 -14 -16 N=19 N=18 All Patients Paired Analysis p = .0016 p = .0004 MLHF Score 30 N=18 N=18 -18 p = .0006
  20. 20. Bioheart MARVEL Program Unique Features and Goals
  21. 21. MARVEL Program Study Objectives / End Points <ul><li>Overall objective </li></ul><ul><ul><li>Assess the safety and efficacy of MyoCell ® in CHF patients post myocardial infarction(s) using the MyoStar ® and NOGA XP ® </li></ul></ul><ul><li>Primary safety end point </li></ul><ul><ul><li>Incidence of SAEs at 3 and 6 months post implantation. </li></ul></ul><ul><li>Co-Primary efficacy end point </li></ul><ul><ul><li>Changes in 6-minute walk distance or quality of life. </li></ul></ul><ul><li>Secondary efficacy end points </li></ul><ul><ul><li>Changes in LVEF, LV volume, wall motion indices, BNP, NYHA class, and frequency and nature of re-hospitalizations. </li></ul></ul>
  22. 22. p=0.50 MARVEL-1: Mean Change in 6-Minute Walk Distance (meters) n=6 n=7 n=6
  23. 23. MARVEL-1 Arrhythmia Analysis: Myocell ® Patients Amiodarone started Amiodarone continued Myocell ® Txt 14 Patients No Amiodarone Txt n= 7 4 Patients 4 Episodes VT Amiodarone Started at Implant or Stopped n=3 Amiodarone Started at Bx n=4 2 Patients 2 Episodes VT 0 Patients 0 Episodes VT 0 Episodes VT 1 Patient 1 Episode VT
  24. 24. Competitive Advantage <ul><li>Time to market </li></ul><ul><ul><li>Bioheart ’s technology is authorized for Phase II/III US trials </li></ul></ul><ul><li>Bioheart has Intellectual Property and technological exclusivity that protects freedom of operation </li></ul><ul><ul><li>Extensive Patent portfolio and 10+ years of development of proprietary methods </li></ul></ul><ul><li>Bioheart ’s product focuses on a unique mechanism of action </li></ul><ul><ul><li>Myoblasts have been shown to be the only effective cell-based therapy to grow new muscle for treatment of CHF </li></ul></ul>
  25. 25. Competition – Congestive Heart Failure
  26. 26. Myoblast – Cell culture Process Development Manufacturing process is protected, scalable and reliable
  27. 27. Myoblasts vs. Bone Marrow Stem Cells for Cardiac <ul><li>Characteristic of the bone marrow cells (mainly MSCs): </li></ul><ul><ul><li>cells in contact with bone tend towards differentiating into bone…when cells transplanted into chronic myocardial infarct, tend towards differentiating into fibrosis. </li></ul></ul><ul><ul><li>“ it is hard to believe that bone marrow cells could recolonize the chronically infarcted myocardium with new cardiac muscle fibers.” </li></ul></ul>Combined Transplantation of Skeletal Myoblasts and Mesenchymal Cells (Cocultivation) in Ventricular Dysfunction After Myocardial Infarction. Souza et al., Arg Bras Cardiol. 2004 Oct; 83(4):294-9; 288-93. <ul><li>“ The transplantation of skeletal myoblasts has proved to be effective in the infarcted myocardium, because those cells can differentiate into viable muscle amidst myocardial fibrosis. ” </li></ul>
  28. 28. Bone Marrow Mesenchymal Stem Cell Study <ul><li>“ The labeled MSC population trapped within the implantation site surrounded by fibrous tissue appeared poorly differentiated, with high nucleus/cytoplasm ratio.” </li></ul><ul><li>“ Within the scar, they (MSC’s) appeared unorganized and had fibroblast-like morphology.” </li></ul><ul><li>“ cells appeared to have differentiated into fibroblasts, cardiomyocytes, endothelial cells and even adipocytes.” </li></ul><ul><ul><li>Ray Chiu, M.D., Ph.D. </li></ul></ul>
  29. 29. Bone Marrow Stem Cells Do Not Convert to Muscle <ul><li>&quot;Our studies show that only the satellite cells, located near muscle fibers, can give rise to new muscle cells. Contrary to previous studies, precursor cells from bone marrow or other blood-forming tissues did not change their destiny to become muscle cells.&quot; </li></ul><ul><li>“ The results show that adult stem cells that are committed to the blood lineage do not normally differentiate into muscle cells.” </li></ul><ul><li>“ The only cells that had full potential to generate muscle cells were derived from muscle, not from transplanted bone-marrow or blood-forming stem cells.” </li></ul><ul><ul><li>Amy J. Wagers, Ph.D., Investigator in the Developmental and Stem Cell Biology Research Section at Joslin Diabetes Center and Assistant Professor of Pathology at Harvard Medical School. November 11, 2004 . </li></ul></ul>
  30. 30. Bioheart: The FDA Approved REGEN Trial Next Generation: MyoCell ® SDF-1 Increased muscle formation Increased blood vessel supply Endogenous circulating stem cells attracted to injury by chemokine proteins Damaged area secretes chemokine proteins Adult stem cells SDF-1 Cells Four years of sponsored animal studies have demonstrated that 2nd generation MyoCell ® SDF-1 provides significantly higher levels of improvement than the first generation MyoCell ® composition.
  31. 31. REGEN Trial – MyoCell SDF-1 <ul><li>Preclinical testing in animals has been both safe and efficacious with double the improvement over MyoCell </li></ul><ul><li>FDA clearance received for a 15 patient, 3 center Phase I study </li></ul><ul><ul><li>Dose Escalation – 3 cohorts: 200 million, 400 million and 800 million cells </li></ul></ul><ul><li>First ever approval for a combination gene and cell therapy for the treatment of cardiovascular disease. </li></ul>
  32. 32. Manufacturing MyoCell <ul><li>Currently at Bioheart ’s Headquarters in Florida </li></ul><ul><ul><li>Current facility sufficient for next two years and able to be expanded </li></ul></ul><ul><ul><li>Capacity </li></ul></ul><ul><ul><ul><li>One Shift: 15 patients/month </li></ul></ul></ul><ul><ul><ul><li>Two Shifts: 25 patients/month </li></ul></ul></ul><ul><ul><ul><li>Three Shifts: 30-60 patients/month </li></ul></ul></ul><ul><ul><li>Ability to add capacity in Florida if expanded with additional suites </li></ul></ul><ul><li>Other Locations to be Ramped Up as OUS business grows </li></ul><ul><ul><li>Korea </li></ul></ul><ul><ul><li>The Netherlands </li></ul></ul>
  33. 37. <ul><li>BIOHEART INC. </li></ul><ul><li>April 2011 </li></ul><ul><li>OTC: BHRT.OB </li></ul>