お知らせ • Jan 09
Maat Pharma Announces Positive Topline Results from the Pivotal Phase 3 Ares Study Evaluating Maat013 in Acute Graft-Versus-Host Disease
MaaT Pharma SA announced topline results from ARES, a pivotal, single-arm, open-label, multicenter European Phase 3 study evaluating the efficacy and safety of MaaT013 in acute Graft-versus-Host Disease patients with gastrointestinal involvement (GI-aGvHD) in third-line treatment, meaning refractory to steroids and refractory or intolerant to ruxolitinib. Notably, the study met its primary endpoint, with asignificant gastrointestinal overall response rate (GI-ORR) at 28 Days of 62%, exceeding the expected 38% response rate. Responses reviewed by an Independent Review Committee (IRC), exceed the per-protocol prespecified threshold and confirm the unprecedented clinical efficacy of MaaT013 for the treatment of third-line GI-aGvHD. The therapeutic options for patients with GI-aGvHD refractory to steroids and refractory or intolerant to ruxolitinib remain very limited despite the poor prognosis for this condition, which has 1-year survival rates of just 15% (Abedin et al., 2021). MaaT013 has the potential to be the first approved third-line treatment option and thereby transform the survival outcomes and redefine long-term prospects for approximately 3,000 third-line GI-aGvHD patients per year in the U.S., Canada and Europe. Top-line Data Highlights: In the single-arm ARES study, 66 adult patients with GI-aGvHD refractory to steroids and refractory or intolerant to ruxolitinib were treated with MaaT013 as third-line treatment across 50 European sites (Austria, Belgium, France, Germany, Italy and Spain). Patients’ characteristics: Gender: 47% females, 53% males. Median age: 55.5 years (24-76) At baseline, aGvHD grading (according to both IRC and investigators’ assessments): Grade II: 9.1%, Grade III: 57.6%, Grade IV: 33.3%, Steroid refractory: 86.4%, Steroid dependent: 13.6%, Ruxolitinib refractory: 100%, Ruxolitinib intolerant: 0, The study met its primary endpoint of GI-ORR at Day 28 of treatment with MaaT013 (p <0.0001), as assessed by the Independent Review Committee (IRC). Frequent, strong and durable response rates translating into prolonged survival. GI-ORR at Day 28 occurred in 41/66 patients (62%) and prevalently consisted of complete response (CR) (25/66 patients, 38%) and very good partial response (VGPR) (13/66 patients, 20%). ORR in all evaluable organs occurred in 42/66 patients (64%) patients and was similarly driven by high rates of CR (24/66 patients, 36%) and VGPR (12/66 patients, 18%). The 12-month probability of survival was 54% (median survival not reached). The 12-month probability was significantly higher in patients who responded at Day 28 than those who did not respond (67% vs 28% respectively, p <0.0001), demonstrating MaaT013’s significant survival benefit in refractory GI-aGvHD. Enrolled patients will continue to be followed for secondary and exploratory endpoints for the duration of the study. Results are expected to be presented at future scientific conferences. MaaT013’s safety has already been confirmed by the ARES Data Safety Monitoring Board (DSMB) in October 2023 for the first 30 patients, showing it was well tolerated with no increased infection risk or treatment-related fatal events (Full details here). Pharmacovigilance and DSMB surveillance for the study remain ongoing. With robust data supporting efficacy and safety, MaaT Pharma is pursuing the regulatory submission of MaaT013, in Europe, as a treatment for GI-aGvHD in third-line, aiming for a Centralized Marketing Authorization Application (MAA) submission to the European Medicines Agency (EMA) in mid-2025, earlier than previously expected. The centralized procedure enables a single authorization across the EU (27 members), thus facilitating patient access and market launch. In line with its mission to offer new treatment options for high unmet medical needs, MaaT Pharma will continue to ensure availability of MaaT013 in Europe, for patients with aGvHD (and other indications) as part of its Early Access Program (EAP), which exceeded 100 requests for patients in 2024. The EAP will continue during the regulatory evaluation phase and up to commercialization, anticipated for end of 2026. The Early Access Program, has been expanded to the United States in December 2024, will continue as the Company advances readiness for the U.S. Phase 3 clinical trial, which is expected to be launched in 2025, upon securing financing.