RARITAN, N.J., Dec. 8, 2013 /PRNewswire/ -- Janssen Research & Development, LLC ("Janssen") today announced positive results from a pivotal Phase 2 global registration study (MCD2001) suggesting siltuximab, an investigational compound, along with best supportive care (BSC), exhibited statistically significant efficacy and a tolerable safety profile compared with placebo and BSC in treating patients with the rare disorder Multicentric Castleman's Disease (MCD) who are HIV-negative and human herpes virus-8 (HHV-8)-negative. 1 MCD is a disorder in which lymphocytes, a certain type of white blood cells, are over-produced and lead to enlargement of lymph nodes. 2 ,3
These data supported the recent regulatory filings of siltuximab in the United States and European Union. Interim findings from a separate Phase 2 study (MCD2002) reinforce the safety profile of siltuximab. 4 The studies were featured in an oral presentation (MCD2001) and poster presentation (MCD2002) at the 55 th American Society of Hematology (ASH) Annual Meeting in New Orleans, USA.
"MCD is a devastating disease that weakens the immune system and may lead to life-threatening infections," said Raymond S. Wong, MBChB, M.D., Prince of Wales Hospital, The Chinese University of Hong Kong, Hong Kong and lead study investigator. "These results are encouraging and from my perspective support the potential for siltuximab as a new treatment for these patients who previously had no approved treatment options."
Oral PresentationThe MCD2001 study found more than one-third of patients in the siltuximab arm had a durable tumor and symptomatic response to treatment, compared to none of the patients who received placebo plus BSC (34 percent versus 0 percent). The median time to treatment failure was not reached for patients who received siltuximab plus BSC versus 134 days for who received placebo plus BSC. In looking at the response rate to treat MCD-related symptoms, 25 percent of patients who received siltuximab plus BSC had durable complete symptom resolution, defined as 100 percent of reduction of baseline overall symptom scores for at least 18 weeks, compared to none of the patients who received placebo plus BSC. 1 The safety profile, defined by frequencies of treatment-emergent adverse events (AEs), Grade 3 or higher AEs and serious adverse events (SAEs), was similar between siltuximab and placebo even with the duration of treatment being twice as long for those in the siltuximab arm. The most frequently reported Grade 3 or higher AEs with siltuximab were fatigue (9 percent), night sweats (8 percent), hyperkalemia (high levels of potassium in blood), hyperuricemia (high levels of uric acid in blood), localized edema (swelling at a specific site in the body), hyperhidrosis (excessive sweating), neutropenia (an abnormally low number of neutrophils, a type of white blood cell), thrombocytopenia (a decrease of platelets in the blood), hypertension (high blood pressure), and weight increase (4 percent each). 1 Poster Presentation In addition, an interim analysis of the Phase 2 study MCD2002 was presented on December 7 in a poster titled:
- An Open-Label, Phase 2, Multicenter Study Of The Safety Of Long-Term Treatment With Siltuximab (an Anti-Interleukin-6 Monoclonal Antibody) In Patients With Multicentric Castleman's Disease (Abstract #1806)