MaaT Pharma Announces First U.S. Patient Treated with MaaT013 for aGvHD Under Expanded Access Program

Key Insights

• MaaT Pharma has treated its first U.S. patient with MaaT013 for acute Graft-versus-Host Disease (aGvHD) under an FDA-approved compassionate access program.
• The patient, having undergone multiple prior therapies, received MaaT013 at City of Hope Hospital, a leading cancer institution in Los Angeles.
• This single-patient expanded access program allows for investigational drug use when standard therapeutic options are limited or unavailable.

MaaT Pharma has announced the treatment of the first patient in the United States with its investigational therapy, MaaT013, for acute Graft-versus-Host Disease (aGvHD). The treatment was administered under a single-patient expanded access program approved by the U.S. Food and Drug Administration (FDA). This marks a significant step for MaaT Pharma in its efforts to address unmet needs in aGvHD treatment.

The patient, who had previously undergone several lines of treatment, received MaaT013 at City of Hope Hospital in Los Angeles. City of Hope is recognized as one of the leading cancer research and treatment centers in the U.S.

Compassionate Use and Expanded Access

The FDA's expanded access program, often referred to as "compassionate use," provides a pathway for patients with serious or immediately life-threatening conditions to access investigational medical products outside of clinical trials. This option is available when there are no comparable or satisfactory alternative therapies. In the U.S., a treating physician can request compassionate use through a Single-Patient Investigational New Drug (IND) application.

Dr. Monzr M. Al Malki of City of Hope Hospital expressed confidence in MaaT Pharma's technology, which aims to improve survival in cancer patients by modulating the immune system.

Ongoing Clinical Development

MaaT Pharma is currently conducting the Phase 3 ARES trial in Europe (NCT - 04769895). Patient recruitment for the European study is complete, and topline results are anticipated in January 2025. Furthermore, the company plans to initiate a U.S. Phase 3 clinical trial to evaluate MaaT013 in aGvHD patients with gastrointestinal involvement who are refractory or intolerant to ruxolitinib. Clinical batches of MaaT013 have been manufactured to support both clinical trials and expanded access programs under FDA oversight.

Additional data on the efficacy, safety, and long-term follow-up from the Early Access Program in Europe will be presented at the upcoming ASH 2024 Annual Meeting in San Diego, California.