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Forodesine

Forodesine (PNP Inhibitor)

Structure of PNP enzyme

Structure of PNP enzyme

Forodesine is in a pivotal study for the treatment of cutaneous T-cell lymphoma (CTCL):

  • The ongoing pivotal trial of forodesine in CTCL is being conducted under a special protocol assessment (SPA) with the FDA
  • Data from the pivotal trial of forodesine is expected in the second half 2010
  • Prevalence of CTCL in U.S.:18,000 patients/1,500 diagnosed year.

Forodesine is being studied in an exploratory Phase 2 study in chronic lymphocytic leukemia (CLL):

  • Prevalence of CLL in U.S.:90,000 patients/15,000 diagnosed year.

Forodesine is an orally-available transition-state analog inhibitor of purine nucleoside phosphorylase (PNP), a purine salvage pathway enzyme that is essential for the proliferation of T-cells and B-cells. Typically, T- and B-cells are an essential part of the body's immune system, but when they multiply uncontrollably they can cause various forms of cancer. Inhibiting PNP produces selective suppression of T- and B-cells, inducing apoptosis in both types of cells.

Forodesine has been granted Orphan Drug status by the FDA for three indications: T-cell non-Hodgkin's lymphoma, including CTCL; CLL and related leukemias including T-cell prolymphocytic leukemia, adult T-cell leukemia, and hairy cell leukemia; and for treatment of B-ALL. The FDA has also granted “fast track” status to the development of forodesine for the treatment of relapsed or refractory T-cell leukemia, and Special Protocol Assessment (SPA) from the FDA for forodesine to conduct a pivotal clinical trial in CTCL with an oral formulation.

About cutaneous T-cell lymphoma (CTCL)

CTCL affects approximately 18,000 people in the U.S. CTCL is a primary skin neoplasm and accounts for nearly 50% of all T-cell malignancies. Unlike most cancer patients, CTCL patients are treated chronically and could benefit from an oral agent that is well tolerated like forodesine. Long term treatment (>12 months) of CTCL with forodesine in the supportive Phase 1/2 study suggests a favorable safety profile. This data was presented at ASCO in 2009.

About chronic lymphocytic leukemia (CLL)

Lymphoma is a general term for a group of cancers that originate in the lymphatic system. About 58,870 Americans will be diagnosed with a non-Hodgkin's lymphoma in 2006 and approximately 15% of these will be considered T-cell lymphomas. T-cell lymphoma results when a T-lymphocyte (a type of white blood cell) undergoes a malignant change and begins to multiply, eventually crowding out healthy cells and creating tumors, which enlarge the lymph nodes and invade other sites in the body.

About acute lymphoblastic leukemia (ALL)

The most common form of leukemia in children is acute lymphoblastic leukemia (ALL). According to the National Cancer Institute, 23% of cancer diagnoses among children younger than 15 years of age are for ALL. This form of cancer, ALL, occurs in about one of every 29,000 children in the United States each year. ALL results from an acquired injury to the DNA of a single cell in the bone marrow.

Corporate Partnership

In February 2006, BioCryst announced an exclusive licensing agreement with Mundipharma International Holdings Limited to develop and commercialize forodesine for use in the treatment of cancer in markets across Europe, Asia, Australia and certain neighboring countries.

Clinical Trials

To learn more about our current studies visit our Clinical Trials page or visit clincialtrials.gov.

Resources

For more information on Cutaneous T-cell Lymphoma visit the National Cancer Institute:
http://www.cancer.gov/cancertopics/pdq/treatment/mycosisfungoides/Patient/page1

For more information on Chronic Lymphocytic Leukemia (CLL) visit The National Cancer Institute:
http://www.cancer.gov/cancertopics/pdq/treatment/CLL

For more information on Acute Lymphocytic Leukemia (ALL) visit the following websites: