Essential thrombocyhemia (ET) is a chronic hematological disorder caused by the overproduction of platelets in the bone marrow. Common symptoms are bleeding, blood clots, headache, nausea, and numbness in extremities. In some extreme cases the disorder may lead to acute myeloid leukemia or myelofibrosis.
PharmaEssentia is in Phase III of testing P1101 for use in treatment of essential Thrombocythemia. Allogenic stem cell transplantation is currently the only known cure, but involves significant risks. Additional treatment options provide some support, but do not cure the condition. These include blood transfusions and administration of chemotherapeutics.
Chronic Myeloid Leukemia (CML) is a cancer of the white blood cells. CML specifically is characterized by unregulated growth of myeloid cells in the bone marrow. It accounts for 15-20% of all adult leukemias, with an age-adjusted incidence of about 1.6 per 100,000.
PharmaEssentia is currently engaged in Phase I clinical trials for P1101 in combination with imatinib to treat CML.
Polycythemia Vera (PV) is a neoplasm in which too many red blood cells are created on the bone marrow. It has a prevalence of 1 to 3 per 100,000. Patient experiences range from asymptomatic to mild conditions such as itching and tiredness to severe such as enlarged organs and blood clots with the potential for heart attacks and strokes.
There is currently no cure for PV. Without treatment, half of patients die within two years. With currently available treatments (regular phlebotomy or cytotoxic therapy), patients can live 15 to 20 years. P1101 aims to create a long-term treatment option with less severe side effects than current options. PharmaEssentia is currently engaged in Phase III clinical trials for the treatment of PV with P1101.
Myelofibrosis is a bone marrow cancer classified as a type of myeloproliferative neoplasm. Excessive proliferation of an abnormal clone of hematopoietic stem cells in the bone marrow results in the replacement of marrow with scar tissue, known as fibrosis.
Hepatitis C is an infectious disease caused by the hepatitis C virus (HCV), mainly affecting the liver. Over time, symptoms of the chronic infection can progress from largely asymptomatic to liver disease and cirrhosis. Sometimes this results in severe complications such as liver failure, liver cancer, or gastric varices.
A Phase II dose-finding clinical trial of P1101 in treatment of chronic Hepatitis C was begun in 2012. Various antiviral medications are the current standard of care depending
Hepatitis B is an infectious disease caused by the hepatitis B virus (HBV), mainly affecting the liver. Chronic infection can lead to inflammation of the liver and eventual cirrhosis. It also increases the risk of liver cancer. Hepatitis B and C are responsible for approximately half of hepatocellular carcinomas.
PharmaEssentia is conducting early-stage research into the use of P1101 to treat Hepatitis B. Current treatments including antiviral drugs cannot clear the infection, but can minimize liver damage and cancer risk by blocking viral replication. Treatment with interferon reduces viral replication and viral load, but response differs between viral genotypes.
Managed Care Segment
Selected Sample of Epidemiology Sources Used in This Analysis
Alvarez-larrán A, Cervantes F, Bellosillo B, et al. Essential thrombocythemia in young individuals: frequency and risk factors for vascular events and evolution to myelofibrosis in 126 patients. Leukemia. 2007;21(6):1218-23.
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