January 7, 2020

2019 was a phenomenal, breakthrough year for lupus research on both the clinical development and basic science fronts. The Lupus Research Alliance (LRA) is incredibly proud that every new discovery leading to a brand new medicine for lupus had its origin in work funded by LRA years ago.

The past 12 months delivered many exciting “firsts” for lupus — three positive Phase III clinical trials, Fast Track and Breakthrough Therapy designations from the U.S. Food and Drug Administration including one in lupus nephritis (a serious form of lupus affecting the kidneys), and the first treatment approved for pediatric lupus, among other developments.

Watch this video to hear the highlights from LRA President & CEO Kenneth Farber while Chief Scientific Officer Dr. Teodora Staeva describes major break throughs in basic research science supported by the LRA.

2019 Was a Year of Many “Firsts” in Lupus Drug Development

1. First Positive Phase 3 Lupus Results in 8 Years — Phase 3 TULIP-2 trial demonstrated the effectiveness of Astra Zeneca’s treatment anifrolumab in reducing lupus disease activity. The LRA has supported numerous studies identifying and unraveling the type I interferon pathway that anifrolumab targets.
2. First Positive Phase 3 Data in Lupus Nephritis — Lupus nephritis, which affects the kidneys, is one of the most dangerous forms of lupus. A late stage trial (AURORA) conducted by Aurinia affirmed the safety and effectiveness of voclosporin. If approved by the FDA, this would be the first medicine specifically approved for this form of lupus. Because of the great need for a new treatment, the company received Fast Track designation from the U.S. FDA.
3. First and Only Lupus Treatment Approved for Use in Children — Benlysta (belimumab) was approved by the U.S. FDA to treat children with lupus who are five years old or above. Pediatric lupus typically hits children harder than adults and comes with extra health issues since children are affected by the disease longer, impacting organ damage.
4. First Breakthrough Therapy Designation for Gazyva Phase 2 Trials Show Effective in Treating Proliferative Lupus Nephritis, the Most Severe Form of Lupus Nephritis – The Phase 2 NOBILITY trial, conducted by Genentech, demonstrated the effectiveness of Gazyva® (obinutuzumab) in combination with mycophenyolate mofetil in treating proliferative lupus nephritis, the most severe form of lupus nephritis that affects the kidneys. The FDA recently granted obinutuzumab Breakthrough Therapy Designation which aims to speed up the development and review of drugs which may demonstrate substantial improvement over available therapy.
5. First Promising, Phase 2 Trial Demonstrates Effectiveness of a Second Interferon Inhibitor — Positive Phase 2 trial results for Biogen’s type I interferon inhibitor BIIB059 demonstrated that it met clinical endpoints in patients with cutaneous lupus (skin related lupus) as well as in systemic lupus.
6. Second Potential Lupus Nephritis Drug Fast Tracked by FDA — A new drug in development by Equillium for lupus nephritis, itolizumab, was just granted Fast Track Designation by the U.S. FDA. The LRA was involved in the design of the clinical trial through its affiliate Lupus Therapeutics and enlisted its patient advisory board to provide the patient perspective on clinical trial education materials.

2019 Discoveries that May Advance New Treatments and a Cure

1. Evidence that Diet May Offer a Treatment — Pieces of bacteria that escape from the intestines may trigger lupus and associated disease flares in some patients, according to a new study led by LRA-grantee Dr. Gregg Silverman. These findings may allow disease treatment with probiotics or diets that alter the mix of bacterial species in the intestines. In addition, a study partly funded by an LRA grant to Dr. Martin Kriegel showed how a diet of high fiber may suppress harmful bacteria and enrich good bacteria in the gut microbiome.
2. Novel Research on Biomarkers is Emerging — Novel research on biomarkers or molecules in the urine, skin and blood in lupus could minimize invasive kidney biopsies. This groundbreaking research is emerging from the Accelerating Medicines Partnership, a RA/SLE initiative involving the NIH, FNIH, industry and LRA.
3. New Targets for Drug Development — Researchers partly funded by the LRA have found possible new targets for drugs to treat lupus after discovering that stopping a molecule BRISC from connecting to another molecule SHMT2 can reduce inflammation.
4. Back to the Future with B Cells — Depleting the number of harmful B cells with a novel immunotherapy that employs modified T cells called CAR T cells may offer an effective strategy to treat lupus, according to results of a study led by LRA-grantee Dr. Marko Radic. These findings offer a renewed optimism for the elimination of B cells to provide a therapeutic option in lupus and pave the way for clinical research to test this new approach.
5. Why the Immune System Goes on the Attack — A new study partly funded by the LRA may help explain how the immune system attacks patients’ DNA in lupus. Together with the laboratory of Dr. David Raulet, Dr. Joshua Woodward and his colleagues discovered a protein door in cells that allows messenger molecules that may promote these attacks to spread.

The Lupus Research Alliance is the world’s leading private funder of lupus research, created to improve treatments for lupus while advancing toward a cure. We believe that scientific research is the most powerful way we can improve the lives of people living with lupus, today and over the long term. By pushing the limits of scientific exploration and shepherding new discoveries into potential treatments, we aim to seize every opportunity that will help ease the burden of people living with this difficult disease.

As part of the organization’s commitment to advance lupus research and find effective and safer treatments for people living with lupus, the Lupus Research Alliance established Lupus Therapeutics.  As the administrative and fiscal entity of the only Lupus Clinical Investigators Network (LuCIN), Lupus Therapeutics manages the Alliance’s clinical trial programs. LuCIN is comprised of more than 200 clinician-scientists at 57 academic medical centers and more than 20,000 active lupus patients. Supported by the Lupus Research Alliance and Lupus Therapeutics, LuCIN scientists work collaboratively to identify potentially transformative treatments and conduct related clinical trials.

The LRA celebrates this year’s achievements and looks forward to continued positive developments in 2020.