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Leading the way to a cure

Turning Promising Ideas into Treatments


There has not been a new therapy specifically designed to treat lupus in nearly 50 years. Have you ever wondered why it takes so long for new therapeutic options to be available for treating the 1.4 million Americans living with this disease?

“Every step of the long and intensive drug research and development process is crucial to ensure safe and effective therapies are brought to the market,” explains Mary K. Crow, M.D., Scientific Advisory Board Chair of the Alliance for Lupus Research (ALR).  “While it has been decades since there has been a new drug available for people with lupus, it is extremely promising to witness the recent explosion of lupus research and development.  Never before have there been so many potential new treatments on the horizon.  At the ALR, we are hopeful that these efforts will soon lead to new therapeutic options for the thousands of people who are impacted by this debilitating disease.”

It can take a decade - and sometimes even longer - to develop a new drug from laboratory concept to pharmacy shelf.  The search for solutions begins in the lab, where pilot experiments are conducted by researchers across the world and can involve potentially thousands of compounds.  Most of these compounds will be eliminated, leaving only a handful that may be considered viable candidates for further study.  Next, studies are conducted to determine whether the potential new drug provides a real health benefit - and if it is safe.  An application must be made to the U.S. Food and Drug Administration (FDA) before it can be tested in humans in clinical trials.  The entire drug approval process can cost hundreds of millions of dollars, and includes a series of stages, as outlined in the below graph.

  

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Even once a drug candidate moves beyond the drug discovery/preclinical stages and enters into the clinical trial stage (stage 3 on the graph), there are still three phases of testing needed that can take anywhere from three to seven years.  

  • Phase I: The clinical studies in this phase represent the first time that an investigational new drug is tested on healthy volunteers with the aim to determine safety and dosage.  Phase I studies are usually conducted on 20 to 100 volunteers.
  • Phase II:  In the second phase, the clinical studies are designed to evaluate effectiveness and side effects.  Phase II trials involve a considerably larger number of patient volunteers than in Phase I - hundreds of patient volunteers participate in Phase II trials.
  • Phase III:  In the final phase, clinical studies are longer in duration with the aim to verify the effectiveness of potential new drugs and allow scientists to extrapolate the results to the general population.  Researchers monitor adverse and long-term use during this period.  Thousands of patient volunteers are involved in Phase III studies.

Upon successful completion of these three phases of trial, an application for approval to market the potential new drug is submitted to the FDA.  If the FDA approval is granted, the new drug is ready for production.

The Landscape of Lupus Drugs in Development

For more than 10 years, the ALR has been working to fund biomedical research with the mission to prevent, treat and cure lupus.  As a result of these efforts, along with those of others, more is known today about lupus than ever before and many potential new drugs are now in development.  In fact, the ALR is proud to have supported some of the important basic and translational research on molecules targeted by many of the lupus drugs in the development process.

Two examples of potential new lupus therapies currently in the drug development pipeline include BENLYSTA® (belimumab), a drug in development by Human Genome Sciences (HGS) and GlaxoSmithKline (GSK), and EPRATUZUMAB, a drug in development by UCB and its partner Immunomedics. 

A closer look at these two drug candidates…

BENLYSTA (belimumab)

The pharmaceutical companies who co-develop BENLYSTA, Human Genome Sciences (HGS) - based in Rockville, MD - and GlaxoSmithKline (GSK) - headquartered in the UK with operations based in the US - recently announced that they submitted a Biologics License Application (BLA) to the FDA for approval to market BENLYSTA for the treatment of systemic lupus erythematosus (SLE) or lupus in seropositive patients.  This means that the FDA will now move forward reviewing BENLYSTA’s clinical trial findings to determine if it is a safe and effective drug to treat lupus.  This is very exciting, long-awaited news for those touched by lupus, and an important milestone for BENLYSTA in the drug development process.

BENLYSTA specifically recognizes and inhibits the biological activity of B-lymphocyte stimulator, or BLyS, a naturally occurring protein that promotes the development of B-lymphocyte cells into mature plasma cells in SLE.  Plasma cells are a type of white blood cell that normally produces antibodies, an important component of the body’s defense against infection.  In autoimmune diseases such as lupus, elevated levels of BLyS are believed to contribute to the production of auto-antibodies that attack and destroy the body’s own healthy tissues, causing the inflammation that is a characteristic of the disease.

The BENLYSTA Phase III development program is the largest clinical trial program ever conducted in lupus patients.  The program included two double-blind, placebo controlled, multi-center Phase 3 superiority trials - BLISS-52, which randomized and treated 865 patients at 90 clinical sites in 13 countries and BLISS-76, which randomized and treated 819 patients at 136 clinical sites in 19 countries.  The results of the BENLYSTA Phase III trials show that BENLYSTA can not only reduce autoantibody levels, but it can also reduce disease activity.  Findings from these studies were previously released last year at the American College of Rheumatology (ACR) meeting, and were recently presented at the American League Against Rheumatism (EULAR) meeting in Rome, Italy and the World Lupus Congress meeting in Vancouver, BC.

It can take several months or even years for a new drug candidate to be reviewed and approved by the FDA - and then large-scale production is needed to stock pharmacy shelves with the new product.  If approved, BENLYSTA will be the first in a new class of drugs called BLyS-specific inhibitors.

While there is so much excitement surrounding the potential for the first new lupus drug in decades, if approved, it will be important for people with lupus to talk with their healthcare provider to determine if BENLYSTA may be a good option for them - not all therapies are right for everyone, even if approved by the FDA and available.

More information about BENLYSTA and its clinical development program can be found by clicking here

EPRATUZUMAB

Exciting new developments in lupus research are also underway as a result of efforts from the Belgian drug company UCB and Immunomedics, a New Jersey-based biopharmaceutical company.  The companies recently announced promising new results garnered from Phase IIb of a clinical investigation of its new drug EPRATUZUMAB in people with SLE or lupus.

The clinical development program of EPRATUZUMAB has been specifically designed to study moderate-to-severe lupus symptoms.  The recent data from the study demonstrate a meaningful treatment effect over placebo in 227 patients who had moderate to severe disease activity in multiple organ systems.  The data from this study was also recently presented at EULAR and the World Lupus Congress meeting.

With the success of Phase IIb, a Phase III study of EPRATUZUMAB is being initiated to evaluate the drug’s effectiveness on a larger patient population.  As we have discussed in this article, a Phase III study can take several years, especially as long-term safety and effectiveness of patient volunteers need to be evaluated.  As such, it is still too early in the drug’s development to fully understand the potential role of EPRATUZUMAB in treating lupus.  However, we look forward to bringing you more in the months and years ahead as results from EPRATUZUMAB’s PHASE III studies are released.

EPRATUZUMAB is an anti-CD22 monoclonal antibody with the potential to modulate B-cell activity.  Although the exact role of CD22 is not fully understood, it is considered to be a negative regulator of B-cell function.  These cells are known to contribute to lupus by producing antibodies that cause the immune system to turn on itself resulting in inflammation and tissue damage.  

The ALR has funded more than five million dollars in B-cell research.

More information about EPRATUZUMAB and its clinical development program can be found by clicking here.  

To learn more about other lupus drugs in development, click here.

The ALR: A Catalyst for Increased Attention to Lupus Research

The ALR has supported some of the important and innovative research leading to an increased understanding of this most complex autoimmune disease.  The diverse research programs developed by the ALR, including its international Systemic Lupus Erythematosus Genetics (SLEGEN) Consortium and its Functional Genomics and Genetic Pathways grants, along with the Target Identification in Lupus grants, are helping identify new drug targets, many of which may ultimately contribute to the development of directed therapies. 

As Joseph Craft, M.D., former Scientific Advisory Board Chair of the Alliance for Lupus Research states, “The ALR brought together many efforts to better our understanding of lupus – resulting in an explosion in knowledge… and an explosion in therapeutic approaches, many of which are in clinical trials today.”  These research endeavors, along with the steadfast commitment of lupus patients who participate in important clinical trials, will hopefully result in new therapeutic options for people living with lupus.

Together, we will find a cure!

The Alliance for Lupus Research (ALR) is the world’s largest charitable funder of lupus research.  The mission of the ALR is to prevent, treat and cure systemic lupus erythematosus (SLE or lupus), a debilitating autoimmune disease, through medical research.  Because the Board of Directors funds all administrative and fundraising expenses, 100% of every contribution received goes directly to support lupus research programs.



1.5 million

people in the U.S. have Lupus.

90 million

dollars committed to lupus research by the Alliance for Lupus Research.


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