Data showed positive trend in multiple disease measures versus placebo
Results presented at the Annual
"While the outlook for patients with lupus has improved over the last 75
years, treatment options remain limited," said Dr.
The SLE-001 study randomized 42 patients who were diagnosed with lupus at least six months before and had a baseline SELENA-SLEDAI score greater than or equal to four. Participants were randomized to one of four escalating doses of CC-220 (CC-220 0.3 mg every other day, 0.3 mg once daily, 0.3 mg alternating with 0.6 mg once daily and 0.6 mg once daily) or placebo for 12 weeks, followed by 12 weeks of observational follow-up or long-term extension. The study evaluated safety, tolerability and pharmacokinetics. Efficacy endpoints, including changes in Cutaneous Lupus Area and Severity Activity Index (CLASI) and SELENA-SLEDAI score, were exploratory.
The most common adverse events (AEs) were nausea, diarrhea and maculopapular rash. Serious AEs were reported in two patients in the two highest CC-220 doses combined (pneumonia in both cases) and in two patients in the placebo group. Five patients in the two highest CC-220 dose groups and one patient in the placebo group discontinued due to AEs. No opportunistic infections or other systemic infections were reported in any CC-220 dose groups.
At day 85, reductions in skin-specific disease activity, as measured by CLASI, ranged from -4.3 to -7.8 in the CC-220 treatment groups, while a CLASI score increase of 0.4 was seen in the placebo group. Mean reductions in CLASI exceeded the minimal clinically important difference of -4.0 in the CC-220 groups. Additionally, more patients receiving CC-220 had at least a 4-point reduction in SELENA-SLEDAI score—an index used to assess lupus disease activity across 24 different disease descriptors (between 22.2 percent and 50.0 percent for CC-220 vs. 12.5 percent for placebo). A trend toward greater improvement in tender joint count and swollen joint count was seen in the CC-220 treatment groups compared with the placebo group at day 85. A trend toward improvement in the Physician's Global Assessment score was also seen at day 85 in the CC-220 groups (ranging from -0.5 to -0.9) compared with the placebo group (0.0). All data are as observed. The low number of study patients and some variability in baseline disease characteristics across treatment groups limit the interpretability of a dose response.
"Celgene is committed to addressing immunological diseases with serious
unmet needs and investigating compounds that we believe could have the
potential to improve the lives of patients. We're excited by the
possibility that CC-220 may offer a novel mechanism to address lupus, a
complex disease that has few effective treatment options," said
CC-220 is not approved for use in any indication in any country.
CC-220 is a novel, oral immunomodulatory compound that binds to and
modulates cereblon, a component of the E3 ubiquitin ligase complex.
CC-220 reduces levels of the transcription factors Ikaros and
About Systemic Lupus Erythematosus1,2,3
Systemic lupus erythematosus (SLE), commonly known as lupus, is an autoimmune disease in which the immune system creates antibodies that attack the body's healthy cells and tissues, including joints, skin, kidneys, heart, lungs, blood vessels and brain. Lupus affects five million people, mostly women, worldwide.
Symptoms of lupus may vary, but the most common include fatigue, pain or swelling in joints, muscle pain, skin rashes, hair loss, seizures, sun sensitivity, ulcers and fever as well as lung, kidney and heart problems. The disease may have periods without symptoms (known as remission) alternating with periods of disease flares. There is no cure for lupus, but medical interventions and lifestyle changes can help control it.
Celgene International Sàrl, located in Boudry,
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