With our stated mission to create new standards of care for unmet medical needs, Biogen Idec is focused on five therapeutic areas: Neurology, Immunology, Oncology, Cardiopulmonary and Hemophilia. These are areas in which we have both proven expertise and products in our pipeline that we believe can lead to first-in-class or best-in-class molecules. Within each area, we have selected targets based on both biology and the pathophysiology of disease.
Neurology
Biogen Idec is an established leader in the treatment of multiple sclerosis (MS); we produce and market both AVONEX® and TYSABRI®. Our goal in neurology is to maintain our leadership position in treating MS and expand that expertise into other areas of neurology, such as Parkinson's disease (which hasn't had a breakthrough treatment in nearly 40 years) and neuropathic pain.
There are two primary opportunities to improve treatment for MS. One is to develop more convenient therapies. We are working with several treatments that would be much easier to administer to MS patients and increase the likelihood that they will adhere to treatment routines and thus benefit from the therapies.
Second is to address a devastating unmet need for MS patients. AVONEX and TYSABRI are effective at silencing the inflammation that is characteristic of MS. But, neither have shown the ability to repair damage or cure the disease. We have several products in our pipeline that are designed to provide neuro-protection, or perhaps even reverse the damage caused by inflammation.
Another promising area of research in neurology is repairing or regenerating nerve fibers, which could fundamentally change the treatment of several conditions, such as MS, Parkinson's disease and Alzheimer's.
Immunology
Immunology is a core strength at Biogen Idec. RITUXAN® is approved for use in combination with methotrexate for those with moderate- to severe-active rheumatoid arthritis (RA). Our pipeline also contains a number of compounds targeted towards the treatment of RA.
Rheumatoid arthritis is a common disease that affects 1 in 100 people, with women 2-3 times more vulnerable than men. There are several treatment goals for RA patients: managing disease symptoms, improving the convenience of therapies, delaying X-ray damage, and, of course, arresting or perhaps even preventing the disease.
Our pipeline has molecules targeted toward each of these goals.
Oncology
Biogen Idec is a pioneer in oncology; RITUXAN® was the first monoclonal antibody approved to treat cancer and has transformed the treatment of non-Hodgkin's leukemia or NHL.
Currently, we are investigating how RITUXAN can be used in combination with other drugs for even greater effectiveness and against other blood cancers such as chronic lymphocytic leukemia. There are a number of examples of this in our pipeline. In addition, we are developing another molecule in collaboration with Genentech/Roche, GA101, which is the next generation of anti-CD20 molecule. Our goal is to develop a best-in-class anti-CD20 therapy that improves clinical outcomes for patients with B-cell malignancies.
For more information, please visit www.biogenideconcology.com.
Hemophilia
With the 2007 acquisition of Syntonix Pharmaceuticals, Biogen Idec formed our Hemophilia therapeutic area, and is working to develop innovative therapies that enable better treatment options for people with hemophilia.
Hemophilia is a rare hereditary disorder in which the ability of patients' blood to clot is impaired. As a result, patients with severe disease suffer from excessive bleeding and uncontrolled internal bleeding, leading to pain and eventual permanent damage to joints and muscles. The preferred mode of treatment for younger patients is a prophylaxis regimen where patients are infused, on average, two to three times per week to maintain an increased circulating level of coagulation factor. Long-term studies demonstrate that such regimens greatly reduce, if not eliminate, progressive joint deterioration.
Using proprietary fusion protein technology, Biogen Idec aims to develop long-acting recombinant replacement factors that may improve the lives of people with hemophilia. Our lead product under development, rFIXFc, is a long-acting, fully-recombinant Factor IX Fc fusion protein, which is poised to enter a registrational clinical trial to explore the potential to prolong protection from bleeding and reduce the frequency of intravenous injections required to manage disease in people with severe hemophilia B.
For more information, please visit www.biogenidechemophilia.com.
Cardiopulmonary
Biogen Idec is pursuing opportunities in the cardiopulmonary therapeutic area. One main area of focus is heart failure, which, despite a number of effective therapies, remains among the most devastating diseases across all populations.
The outcomes for heart failure patients are dismal. The one-year mortality is 25%; five-year mortality is about 50%. For perspective, these rates are comparable to lung cancer and worse than breast cancer. The estimated cost in healthcare dollars to treat heart failure is $35 billion in the United States alone.
Our approach to new drugs for heart failure is to address two co-morbidities of the condition, hyponatremia and renal insufficiency. Renal function is the single most predictive factor for heart failure death.
We have two programs addressing these co-morbidities of heart failure. Lixivaptan is designed to treat hyponatremia and is in a Phase 3 clinical trial. ADENTRI®, in Phase 2, is designed to allow physicians to treat the patient's heart congestion without the accompanying renal failure.
