Celgene Corporation (NASDAQ:CELG) announced results from the phase 2a SLE-001 trial evaluating CC-220, the Company’s investigational, oral immunomodulatory compound, at the Annual European Congress of Rheumatology (EULAR) 2017 in Madrid. A trend toward greater improvement with CC-220 treatment compared with placebo in multiple measures of disease activity, as measured by standard scores employed in clinical trials, was observed in patients with systemic lupus erythematosus (SLE), commonly known as lupus.

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

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 Terrie Curran, President, Celgene Inflammation & Immunology. “Our work with CC-220 will also help to diversify and deepen our Inflammation and Immunology franchise as we continue to advance CC-220 in the clinic.”

CC-220 is not approved for use in any indication in any country.

Shares of Celgene closed yesterday at $119.62, up $0.27 or 0.23%. CELG has a 1-year high of $127.64 and a 1-year low of $94.42. The stock’s 50-day moving average is $119.41 and its 200-day moving average is $119.60.

Sentiment on the street is mostly bullish on CELG stock. Out of 13 analysts who cover the stock, 11 suggest a Buy rating and 2 recommend to Hold the stock. The 12-month average price target assigned to the stock is $146.91, which represents a potential upside of 23% from where the stock is currently trading.

Celgene Corp. is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation. Its targeting areas include intracellular signaling pathways, protein homeostasis and epigenetics in cancer and immune cells, immunomodulation in cancer and autoimmune diseases and therapeutic application of cell therapies.