Anemia

A Gene Therapy Opportunity Utilizing Mirus Bio's Pathway IV ™ Delivery Technology

Medical Need

Anemia is a condition in which the number of red blood cells drops in the bloodstream, resulting in insufficient oxygen being delivered to tissues and organs. The condition is commonly associated with renal failure, cancer and HIV infection. Patients presenting with severe anemia receive emergency red blood cell transfusions to quickly rebuild the cell population. When there is an underlying long term medical condition like chronic renal failure in which the natural production of erythropoietin (EPO) is impaired, patients often receive supplemental injections of recombinant human EPO (rEPO) protein to elevate and maintain their red blood cell levels within a normal range. The prevalence of severe anemia (defined as hematocrit <30%) in patients with chronic kidney disease at the onset of dialysis is high, approximately 67%¹. In cancer patients, the prevalence in solid tumors and hematological tumors ranges from 28% to 48%².

The market for treating anemia exceeds $8 billion annually. Recombinant EPO products, marketed by Amgen, Johnson & Johnson, and Roche have been and continue to be among the largest selling products in the pharmaceutical industry³.

The erythropoiesis market is ripe for a product that can provide a cost effective, long-term treatment option for severe anemia that would displace or reduce the need for frequent rEPO injections.

Product Description and Rationale

Mirus’ proposed therapy will utilize the company’s proprietary Pathway IV ™ protocol. Patients will be injected with plasmid DNA (pDNA) molecules that carry the genetic instructions to produce EPO. Once the DNA is inside target muscle cells, it is anticipated that the patient’s own body will naturally produce sufficient EPO to regulate and maintain a stable volume of red blood cells in the bloodstream. It is envisioned that patients will receive a series of escalating doses in a single treatment course until the desired therapeutic threshold is achieved. Thereafter, long term gene expression will maintain a steady physiological state, reducing or eliminating the need for chronic injections.

Unlike other delivery platforms, our breakthrough Pathway IV ™ platform provides a unique ability to pursue a repetitive dosing regimen to gradually restore the red blood cell population in a stepwise, controlled procedure, without the risk of overproducing EPO.

Partners

Independent

Development Status

Current studies, supported by a SBIR Phase II grant from the NIH, are directed at refining product dosing and delivery parameters in large animals, which should enable the initiation of a Phase I dose escalation study in humans for the treatment of severe anemia. The initial target group for such a gene therapy approach will most likely be end-stage renal failure patients. Please contact us for more information.

References

¹ Thacker, Paul D. 2002 Posters and Abstracts: Effectively Managing Patients with Renal Disease. Renal Week 2002: American Society of Nephrology 35th Annual Meeting
² Mocharnuk, Robert S. Anemia and Erythropoietin: Latest Results and Clinical Guidelines. 44th annual Meeting of the American Society of HematologyEuropean Journal of Clinical Investigation 31, 651-666
³ "Buzz – 2004 Product Sales Will Easily Clear the $50B Mark". Signals Magazine August 27, 2004