Thrombocytopenia :: Positive clinical trial results of investigational thrombocytopenia agent

At the American Society of Hematology meeting in Orlando AkaRx, Inc. presented the positive results from two Phase I studies of AKR-501, an investigational compound for the treatment of thrombocytopenia.

AKR-501 is a promising member of a new class of agents called, ?TPO receptor agonists? that is now in Phase II clinical development. It is an investigational orally administered drug being developed by AkaRx, Inc. intended to mimic the biologic effect of thrombopoietin, a growth factor that stimulates production of platelets.

At the American Society of Hematology meeting, results from two Phase I clinical research trials were presented. These data in healthy volunteers showed that AKR-501 produced a 50% increase or greater over the baseline platelet count. AKR-501 is the first oral drug in its class to show these platelet increases with a single dose. In the single dose study this was achieved at the 100 mg dose. In all volunteers given multiple doses of either 10 mg or 20 mg for 10 ? 14 days a platelet effect was observed where increases were at least 50% over baseline.

The unmet medical need for AKR-501 is that there is no approved agent to specifically stimulate megakaryocytes to produce platelets to treat thrombocytopenia in the same way that there are products available to stimulate production of red and white blood cells. Severe thrombocytopenia is currently managed in some settings with platelet transfusions. However, this temporary solution in not suitable for long-term use in chronic settings and is often associated with serious complications when used in acute situations. AKR-501 imitates the body?s mechanism for stimulating platelet production by mimicking the action of thrombopoietin?the growth factor that modulates this process.

?AKR-501 is a very promising candidate for use in the prevention and treatment of thrombocytopenia in a host of diseases, among them, ITP, Chemotherapy Induced Thrombocytopenia, and Hepatitis C associated thrombocytopenia,? said Robert E. Desjardins, MD, President and CEO of the drug development company, AkaRx. ?These are the first clinical results of AKR-501 that show that it may be a safe thrombopoietic agent with an impressive ability to stimulate platelet production. This class may be a real breakthrough for patients. These results for AKR-501 give us confidence to envision a very broad clinical development program across the world for this compound that appears to have great medical and commercial potential.?

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