RESEARCH TRIANGLE PARK, N.C., Feb. 6, 2012 /PRNewswire/ -- Chimerix, Inc., a biotechnology company developing novel antiviral therapeutics, today announced positive results from CMX001 Study 201, a Phase 2 study evaluating CMX001 for the prevention of cytomegalovirus (CMV) disease in hematopoietic stem cell transplant (HCT) recipients. CMX001 is a broad spectrum Lipid-Antiviral-Conjugate completing Phase 2 clinical development for the prevention of CMV in HCT recipients. In CMX001 Study 201, a double-blind, placebo-controlled trial which enrolled 230 HCT recipients, CMX001 had a statistically significant benefit versus placebo in preventing CMV viremia and/or CMV disease 13 weeks post-transplant.
Francisco Marty, MD, Assistant Professor of Medicine at Dana-Farber Cancer Institute and Brigham and Women’s Hospital’s Division of Infectious Disease, and a lead investigator in Chimerix’s CMX001 Phase 2 CMV study, presented the data during the “Best Abstracts Plenary Session” at the 2012 BMT Tandem Meetings on February 3, 2012 in San Diego, California. “This study provides positive data supporting the antiviral activity of CMX001 at different dose levels, and a better understanding of CMX001’s safety and tolerability as a prophylactic agent against CMV infection, a major cause of morbidity and mortality in bone marrow transplant recipients,” said Dr. Marty. “There is a substantial unmet medical need for safer and effective therapies against CMV. If approved, many patients have the potential to benefit from the future availability of CMX001.”
“These results exceeded our high expectations, and we are thrilled to share such positive CMX001 data with the transplant community,” said Wendy P. Painter, MD, MPH, Chimerix’s Chief Medical Officer. “We look forward to initiating the Phase 3 CMV program later this year. This study reinforces our belief that CMX001’s broad spectrum application against multiple viral infections, its safety profile and convenient oral dosing will enable it to become a new standard of care for transplant recipients.”
CMX001 Study 201 Results Presented at BMT Tandem Meetings
Results from subjects receiving CMX001 100 mg twice weekly met the primary endpoint, a statistically significant reduction in CMV viremia (CMV > 200 copies/mL) or disease at the end of treatment in CMX001-treated subjects versus those who received placebo (p=0.001). Moreover, CMX001 Study 201 showed that three different doses of CMX001 demonstrated statistically significant reductions in the proportion of subjects with CMV viremia 1000 copies/mL at any time during treatment when compared to placebo (p=0.002, <0.001, <0.001, respectively; see Table 1 below). In subjects who were CMV viremia negative prior to treatment, four different CMX001 dose regimens demonstrated statistically significant reduction versus placebo (see Table 2 below).
Table 1 | ||||
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Dose | Enrolled (N) | CMV Viremia (N) | % | P |
40 mg QW(1) | 25 | 10 | 40% | 0.43 |
100 mg QW | 27 | 6 | 22% | 0.06 |
200 mg QW | 39 | 7 | 18% | 0.002 |
200 mg BIW(2) | 30 | 2 | 7% | < 0.001 |
100 mg BIW | 50 | 4 | 8% | < 0.001 |
Pooled Placebo | 59 | 25 | 42% | - |
(1)QW: Once weekly. (2)BIW: Twice weekly.
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Table 2 | ||||
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Dose | Enrolled (N) | CMV Viremia (N) | % | P |
40 mg QW | 18 | 4 | 22% | 0.55 |
100 mg QW | 23 | 2 | 9% | 0.04 |
200 mg QW | 29 | 2 | 7% | 0.02 |
200 mg BIW | 22 | 0 | 0 | 0.002 |
100 mg BIW | 41 | 0 | 0 | < 0.001 |
Pooled Placebo | 48 | 15 | 31% | - |
There was no difference versus placebo across CMX001 treatment groups in measurements of renal function and hematologic parameters. Diarrhea was the most common adverse event seen in the CMX001 treatment groups and was dose-limiting at the highest dose of CMX001 (200 mg twice weekly).
CMX001 Study 201 Design
CMX001-201 was a randomized, double-blind, placebo-controlled, dose-escalation, multi-center trial evaluating the safety, tolerability, and ability of CMX001 to prevent or control CMV disease in 230 evaluable CMV seropositive allogeneic stem cell transplant recipients. Following engraftment (Days 14-30 post-transplant), subjects were stratified based on the presence or absence of acute GVHD requiring systemic therapy and the presence or absence of CMV DNA in plasma and randomized (3:1, CMX001 versus placebo) into five sequential, dose-escalating cohorts. Subjects were treated once weekly or twice weekly for 9 to 11 weeks through post-transplant Week 13, after which subjects were followed for an additional 4 to 8 weeks. Placebo patient results were pooled for endpoint analysis.
