Celgene’s CC-220 Seen to Benefit Lupus Patients, But Researchers Urge Caution

A Phase 2a trial (NCT02185040) evaluating Celgene International‘s CC-220 in systemic lupus erythematosus (SLE) reports promising results, according to data presented at the Annual European Congress of Rheumatology (EULAR) 2017, held June 14-17 in Madrid.

“While the outlook for patients with lupus has improved over the last 75 years, treatment options remain limited,” Richard Furie, chief of rheumatology at New York’s Northwell Health, said in a press release. “New treatment options are greatly needed for people who are dealing with this often disabling autoimmune condition.”

Data showed that patients’ skin symptoms improved in a clinically meaningful way after treatment with CC-220, while overall lupus activity also decreased. In addition, those who received CC-220 tended to have fewer swollen or tender joints compared to patients on placebo.

But researchers said the data was difficult to interpret since the study was small and patients differed in their characteristics before beginning treatment.

The study randomly split 42 patients into five groups — four that received escalating doses of CC-220 and one placebo-treated control group. Participants had a lupus diagnosis for at least six months and had to have a lupus activity score — measured by the SELENA-SLEDAI tool — of four or higher.

After 12 weeks of treatment, researchers analyzed the drug’s safety and tolerability, as well as drug properties such as absorption and metabolism. The most common adverse events were nausea, diarrhea and skin rash. Two patients receiving the highest doses of CC-220 and two patients in the placebo group experienced serious adverse events. Those who received active treatment developed pneumonia.

Five patients in the two groups receiving the highest CC-220 doses quit the study because of adverse events, as did one patient in the placebo group.

CC-220 is an oral immunomodulatory drug that acts on a factor called cereblon. The molecule is also involved in cancer, and Celgene, based in Summit, New Jersey, is running a clinical trial to explore the drug in multiple myeloma.

“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,” said Terrie Curran, president of Celgene Inflammation & Immunology. “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.”