Product Candidates

 

Myocell®

myocell heart diagram

The human heart does not have cells that naturally repair or replace damaged heart muscle. Accordingly, the human body cannot, without medical assistance, repopulate regions of scar tissue within the heart with functioning muscle. MyoCell is a clinical therapy designed to improve cardiac function by populating regions of scar tissue within a patient's heart with muscle stem cells (myoblasts) derived from a biopsy of a patient's thigh muscle. Myoblasts are precursors to muscle cells that have the capacity to fuse with other myoblasts or with damaged muscle fibers to regenerate muscle. When injected into scar tissue within the heart wall, myoblasts have been shown to be capable of engrafting in the damaged tissue and differentiating into mature muscle cells. In a number of clinical and animal studies, the engrafted muscle cells have been shown to express various proteins that are important components of contractile function. The use of myoblasts obtained from a patient's own body to treat chronic heart damage has several distinct advantages to the use of other cell types including but not limited to the following:

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Since the myoblasts are patient-derived it reduces the risk of tissue rejection;

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Myoblasts are committed to forming muscle and will not differentiate into other cell types in the heart;

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Myoblasts do not over-proliferate so there is no risk of forming tumors in the heart;

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Myoblasts better tolerate the low-oxygen environment of scar tissue (therefore surviving better in the heart) as it is not unlike the environment in which myoblasts naturally reside in the thigh muscle;

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Myoblasts are the only progenitor cells in the human body that normally develop a contractile apparatus, which makes them more likely to restore contractile function in the damaged fibrous regions of the heart;

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Myoblasts normal function is to regenerate muscle after injury or trauma;

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Myoblasts are easily expandable in culture allowing for high yields to be obtained in order to replace the many cells that have been lost in the damaged heart.

Our preclinical and clinical research to date suggests that MyoCell may improve the contractile function of the heart. Some theories as to why contractile function improves include:

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The engrafted myoblasts can contract in unison with the other muscle in the heart by stretching;

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The myoblasts acquire certain characteristics of the heart muscle cells or fuse with them;
• The injected myoblasts release various proteins that indirectly result in limiting scar tissue formation;

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The myoblasts may limit the remodeling (or enlargement) of the heart.

As part of the MyoCell therapy, a general surgeon removes approximately five to ten grams of thigh muscle tissue from the patient utilizing local anesthesia, typically on an outpatient basis. The muscle tissue is then shipped to Bioheart’s cell culturing site. At the cell culturing site, our proprietary techniques are used to isolate and remove myoblasts from the muscle tissue. We typically produce enough cells to treat a patient within approximately 14 to 21 days of his or her biopsy. Such production time is expected to continue to decrease as we continue to refine our cell culturing processes. After the cells are subjected to a variety of tests, the cultured cells are packaged in injectate media and shipped to the interventional cardiologist. Within four days of packaging, the cultured myoblasts are injected via a needle injection catheter directly into the scar tissue of the patient's heart. The injection procedure takes on average about one hour and can be performed with or without general anesthesia. Following treatment, patients generally remain in the hospital for approximately 48-72 hours for monitoring. The MyoCell injection procedure is minimally invasive which presents less risk and considerably less trauma to a patient than conventional (open) heart surgery. Patients are able to walk immediately following the injection process and require significantly less time in the hospital compared with surgically treated patients.

We use a number of proprietary processes to create therapeutic quantities of myoblasts from a patient's thigh muscle biopsy. We have developed and/or licensed what we believe are proprietary or patented techniques to:

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Transport muscle tissue and cultured cells;
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 Disassociate muscle tissue with manual and chemical processes;
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 Separate myoblasts from other non-useful cells;
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 Identify a cell population with the propensity to engraft, proliferate and adapt to the cardiac environment; and
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 Maintain and test the cell quality and purity.

Assuming we secure regulatory approval of MyoCell for the treatment of all NYHA Class II and NYHA Class III patients, we believe MyoCell will provide a treatment alternative for the millions of these patients in the United States and Europe who either do not qualify for or do not have access to a heart transplant. Furthermore, we anticipate that the time incurred and cost of identifying patients qualified to receive MyoCell as well as the cost of MyoCell, including any ICD, drug and bi-ventricular pacer therapies that are simultaneously prescribed, if any, will be less expensive than the current cost of a heart transplant. Moreover, MyoCell is less invasive than a heart transplant and is not subject to the tissue rejection and immune system suppression issues associated with heart transplants.

We believe there is still a large population of patients exhibiting symptoms consistent with NYHA Class II and NYHA Class III heart failure that is seeking an effective or more effective therapy for chronic heart damage than ICDs, bi-ventricular pacers and drug therapies. We hope to demonstrate that MyoCell is complementary to various therapies including ICDs, bi-ventricular pacers and pharmaceuticals.

Candidate
  
Proposed Use or Indication
  
Status/Phase
MyoCell®   Autologous muscle stem cell therapy for the treatment of severe heart damage in heart failure patients   Phase I, Phase I/II and Phase II trials completed. Phase II/III underway
 
Comments: Prior MyoCell clinical trials include MYOHEART (20-patient U.S. Phase I dose-finding study) and SEISMIC (40-patient European Phase II-a safety study). MYOHEART and SEISMIC demonstrated no unexpected safety issues in this very sick patient population and suggested trends toward improvement in efficacy parameters.

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