A Bold
Approach

A Bold
Approach

THERAPEUTIC FOCUS

We are focused exclusively on B-cell mediated diseases.

B-cells are white blood cells responsible for producing antibodies. Along with T-cells, they are the major cellular components of the adaptive immune response. Produced in the bone marrow, B-cells migrate to the spleen and other secondary lymphoid tissues where they mature and differentiate into immunocompetent B-cells.

B-cell diseases comprise a constellation of disorders that result from cancerous B-cells or aberrant B-cells. In the case of cancerous B-cells, the most common forms of B-cell cancers or B-cell malignancies are non-Hodgkin’s lymphoma (NHL) and chronic lymphocytic leukemia (CLL). Additionally, many autoimmune diseases are believed to result from aberrant B-cell activity, including, multiple sclerosis (MS), rheumatoid arthritis (RA), and systemic lupus erythematosus (SLE).

Our current pivotal clinical programs are focused on CLL, various subtypes of NHL including marginal zone lymphoma (MZL) follicular lymphoma (FL), and relapsing forms of multiple sclerosis (RMS).

More about our therapeutic areas of :

CLL

  • Chronic lymphocytic leukemia (CLL) is the most common type of adult leukemia, and in 2019, it is estimated there will be more than 20,000 new cases of CLL diagnosed in the United States1. Although signs of CLL may disappear for a period of time after initial treatment, the disease is considered incurable and many people will require additional treatment due to the return of malignant cells.

MZL

  • Marginal zone lymphoma (MZL) comprises a group of indolent (slow growing) B-cell non-Hodgkin lymphomas (NHLs) that begin forming in the marginal zone of lymphoid tissue. With an annual incidence of approximately 7,500 newly diagnosed patients in the United States2, MZL is the third most common B-cell NHL, accounting for approximately eight percent of all NHL cases. MZL consists of three different subtypes: extranodal MZL of the mucosal-associated lymphoid tissue (MALT), nodal marginal zone lymphoma (NMZL), and splenic marginal zone lymphoma (SMZL)3.

FL

  • Follicular lymphoma (FL) is typically a slow-growing or indolent form of NHL which accounts for 20 to 30 percent of all NHL cases4, with a prevalence of approximately 245,0005 and an incidence of approximately 31,0006 in the US, Japan, and 5 major EU markets. Advanced stage FL is usually not considered to be curable, but more of a chronic disease, with patients living for many years.

MS

  • Relapsing multiple sclerosis (RMS), is a chronic demyelinating disease of the central nervous system (CNS) and includes people with relapsing-remitting multiple sclerosis (RRMS) and people with secondary progressive multiple sclerosis (SPMS) who continue to experience relapses. RRMS is the most common form of multiple sclerosis (MS) and is characterized by episodes of new or worsening signs or symptoms (relapses) followed by periods of recovery. It is estimated that nearly 1,000,000 people are living with MS in the United States7 and approximately 85 percent are initially diagnosed with RRMS8. The majority of people who are diagnosed with RRMS will eventually transition to SPMS, in which they experience steadily worsening disability over time.

 

 

 

 

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1 Cancer Stat Facts: Leukemia – Chronic Lymphocytic Leukemia https://seer.cancer.gov/statfacts/html/clyl.html

2 2016 Lymphoid Malignancy Statistics by World Health Organization Subtypes VOLUME 66 _ NUMBER 6 _ NOVEMBER/DECEMBER 2016 https://onlinelibrary.wiley.com/doi/pdf/10.3322/caac.21357

3 Lymphoma Research Foundation: Marginal Zone Lymphoma https://lymphoma.org/aboutlymphoma/nhl/mzl/

4 Lymphoma Research Foundation: Follicular Lymphoma

https://lymphoma.org/aboutlymphoma/nhl/fl/

5 Decision Resources via Pharmacyclics Investor Presentation, December 2014

6 Global Data 2016 NHL Model

7 National MS Society https://www.nationalmssociety.org/About-the-Society/MS-Prevalence

8 Multiple Sclerosis International Federation, 2013 via Datamonitor p. 236