BioCryst Pharmaceuticals designs, optimizes and develops novel small-molecule pharmaceuticals that block key enzymes involved in infectious diseases, cancer and inflammatory diseases.
BioCryst uses structure-based drug design, which incorporates multiple scientific disciplines including biology, crystallography, medicinal chemistry and computer modeling, in order to most efficiently develop new therapeutic candidates.
BioCryst has progressed two novel compounds into late-stage pivotal clinical trials:
- Peramivir, an anti-viral for influenza
- Forodesine, a purine nucleoside phosphorylase (PNP) inhibitor for cutaneous T-cell lymphoma (CTCL)
BioCryst’s Approach to Building its Product Pipeline
BioCryst designs drug candidates using detailed knowledge of the structures of active sites of targeted enzymes. The active site of an enzyme is the area into which a chemical or biological molecule fits to initiate a biochemical reaction. Enzymes make ideal drug targets as they mediate biochemical processes along disease pathways, are relatively easy to crystallize, and have small active sites. BioCryst aims to license or design compounds that will fit in the active sites of enzymes and thereby interfere with the progression of disease. Using this approach, the company has successfully both licensed and designed product candidates for infectious diseases, cancer and inflammatory diseases.
Our therapeutic product pipeline contains two advanced clinical programs:
Corporate & Academic Partnerships
The Company uses strategic partnerships early in the drug identification process through in-licensing of drug targets. BioCryst currently has collaborations with leading academic organizations including The University of Alabama at Birmingham and Albert Einstein College of Medicine of Yeshiva University and Industrial Research, Ltd., New Zealand. We have established collaborative relationships for development and commercialization of product candidates in their respective territories as follows:
- Mundipharma International Holdings Limited for forodesine in Europe, Asia and Australia
- Shionogi & Co., Ltd. for peramivir in Japan and Taiwan
- Green Cross Corporation for peramivir in South Korea
For more information regarding corporate and academic partnerships visit our Partners & Business Development page.
Peramivir Stockpiling Partnerships
In addition, BioCryst has established agreements with partners who will exclusively represent BioCryst and its anti-viral peramivir for influenza stockpiling opportunities for their territories outside of the U.S.
- Hikma Pharmaceuticals PLC for Middle East and North Africa (MENA) region, excluding Israel
- Merck Serono for Europe, Russia, Canada and Singapore
- moksha8 Pharmaceuticals, Inc. for Brazil and Mexico
- Neopharm Group for Israel
- NT Pharma Co., Ltd. for China
For more information regarding these peramivir stockpiling partnerships visit our Partners & Business Development page.
Neuraminidase Inhibition
Peramivir
Peramivir is a potent, intravenously administered anti-viral agent that rapidly delivers high plasma concentrations to the sites of infection. Discovered by BioCryst, peramivir inhibits the interactions of influenza neuraminidase, an enzyme that is critical to the spread of influenza within the host.
According to the U.S. Centers for Disease Control and Prevention (CDC), an estimated 5% to 20% of the American population suffers from influenza annually, more than 200,000 people are hospitalized from flu-related complications and approximately 36,000 people die. For hospitalized patients, there are no anti-virals approved for treatment in patients with severe or complicated influenza. Currently, the only approved products are oral or inhaled which are not suitable for a large portion of hospitalized patients. Peramivir has the potential to address unmet needs in the treatment of influenza.
Peramivir currently is being developed under a $180 million contract from the Biomedical Advanced Research and Development Authority (BARDA) within the United States Department of Health and Human Services (HHS).
BioCryst has licensed peramivir to Shionogi & Co., Ltd. for development and commercialization in Japan and Taiwan. On January 13, 2010, Shionogi announced that it had received marketing and manufacturing approval for i.v. peramivir to treat patients with influenza in Japan. Additionally, BioCryst has licensed peramivir to Green Cross Corp. for development and commercialization in South Korea. In January 2010, Green Cross announced that it had filed a New Drug Application (NDA) in South Korea to seek regulatory approval for i.v. peramivir to treat patients with influenza. For more information visit our peramivir page.
Purine Nucleoside Phosphorylase (PNP) Inhibitors
Forodesine
Forodesine is an investigational purine nucleoside phosphorylase (PNP) inhibitor for the treatment of T- and B-cell mediated disorders. PNP is an enzyme that plays a key role in T- and B-cell proliferation and inhibiting this enzyme provides a novel mechanism for inhibiting T- and B-cell proliferation. 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.
We have received Special Protocol Assessments (SPA) from the U.S. Food and Drug Administration (FDA) for forodesine to conduct pivotal clinical trials in CTCL with an oral formulation. Forodesine also has been granted Orphan Drug status by the FDA for three indications: T-cell non-Hodgkin's lymphoma (including CTCL), CLL and B-ALL. CTCL impacts the lives of 18,000 people in the U.S. with an additional 1,500 people diagnosed each year. Nearly 90,000 people in the U.S. have chronic lymphocytic leukemia (CLL) with 15,000 newly diagnosed every year. Acute lymphoblastic leukemia (ALL) is the most common form of leukemia in children, and nearly 4,000 new cases in both adults and children are diagnosed each year.
BioCryst has signed an exclusive licensing agreement with Mundipharma International Holdings Limited to develop and commercialize forodesine for oncology indications in markets across Europe, Asia and Australia. For more information visit our forodesine page.
BCX4208
BCX4208 is a next generation PNP inhibitor with the potential for once-a day dosing suitable for chronic administration. Recent studies have shown that BCX4208 affects T-cells, B-cells and uric acid with broad applications in inflammatory diseases. For more information visit our BCX4208 page.
