Dublin, Ireland – February 27^th, 2023 – Priothera, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator compound mocravimod, today announced that it received clearance from the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan for the pivotal global MO-TRANS Phase 3 study evaluating mocravimod in Acute Myeloid Leukemia (AML) patients undergoing allogeneic hematopoietic cell transplant (HCT).

Elisabeth Kueenburg, M.D., Chief Medical Officer at Priothera, said: “We are grateful for the clearance from the PMDA to start our MO-TRANS Phase 3 study in Japan as we believe mocravimod has the potential to address a significant unmet need for Japanese patients with AML undergoing allogeneic HCT. We look forward to enrolling the first patients in several sites across Japan in the coming months.”

Priothera is conducting the pivotal MO-TRANS study – now classified as a global Phase 3 trial following acceptance by regulatory authorities – in 15 countries, including the US, Japan, Israel, France, and in additional European, Asian and Latin American countries, to assess the efficacy and safety of mocravimod as an adjunctive and maintenance therapy in AML patients undergoing allogeneic HCT. The double-blind, placebo-controlled study assesses relapse-free and overall survival of two dose levels of mocravimod in comparison to placebo. Topline data from this study are expected in 2025.

Dr. Takanori Teshima, M.D., is the Principal Investigator in Japan for the MO-TRANS global Phase 3 trial. Dr. Teshima is Professor of Medicine in the Division of Hematology and Deputy-director of Hokkaido University Hospital. He serves as a president of the Japanese Society of Hematopoietic Cell Transplantation (JSHCT), an executive director of Japanese Society of Hematology (JSH) and Japan Society of Transfusion Medicine and Cell Therapy (JSTMCT).

Dr. Teshima, M.D., commented: “AML is the most common acute leukemia in Japan accounting for approximately 70% of all myeloid leukemias. Despite advances in the use of HCT in Japan, patients are still at risk of relapse with resulting poor prognosis. Any new treatment that can enhance the curative potential of this approach would offer a significant benefit to patients. I am therefore delighted that Japan will be part of the MO-TRANS global Phase 3 study investigating mocravimod as an innovative potential new adjunctive and maintenance therapy for patients with AML undergoing allogeneic Hematopoietic Cell Transplant.”

Mocravimod, a sphingosine 1 phosphate (S1P) receptor modulator which has been previously tested in multiple autoimmune indications, is being developed to enhance the curative potential of allogeneic HCT. Mocravimod has shown a clinically relevant benefit in an early clinical study in patients with hematologic malignancies undergoing allogeneic HCT.

Florent Gros, Co-Founder and CEO of Priothera, added: “Japan represents an important potential market for us and one we see as a priority as we advance the development of mocravimod in AML patients undergoing allogeneic HCT globally. With this clearance we anticipate a further uptake in patient enrollment in our MO-TRANS trial. We look forward to seeing topline results from MO-TRANS in 2025.” 

Dublin, Ireland – January 23, 2023 – Priothera, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator compound mocravimod today announced that the first patients have been enrolled in the pivotal MO-TRANS global Phase 2b/3 study evaluating mocravimod in AML patients undergoing allogeneic hematopoietic cell transplant (HCT).

Mocravimod, a sphingosine 1 phosphate (S1P) receptor modulator which has been previously tested in multiple autoimmune indications, is being developed to enhance the curative potential of allogeneic HCT. Mocravimod has shown a clinically relevant benefit in an early clinical study in patients with hematologic malignancies undergoing allogeneic HCT.

Priothera is initiating the pivotal MO-TRANS global Phase 2b/3 study in Europe, Israel, the US and in additional Asian and Latin American countries, to assess the efficacy and safety of mocravimod as an adjunctive and maintenance therapy in AML patients undergoing allogeneic HCT. The double-blind, placebo-controlled study assesses relapse-free and overall survival of two dose levels of mocravimod in comparison to placebo. Topline data from this study are expected in 2025.

Marcos de Lima, M.D., is the Principal Investigator for the MO-TRANS global Phase 2b/3 trial. Dr. de Lima is professor of medicine at The Ohio State University College of Medicine and a hematologist-oncologist at The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute.

Prof de Lima said: “We are excited to be part of the important MO-TRANS global Phase 2b/3 study to investigate mocravimod, a potential new adjunctive and maintenance therapy for patients with Acute Myeloid Leukemia undergoing allogeneic Hematopoietic Cell Transplant. Maintenance therapy is fast becoming the new frontier in the treatment of AML and we are committed to bringing forth new innovative therapies to AML patients.”

Elisabeth Kueenburg, M.D., Chief Medical Officer at Priothera, commented: “This MO-TRANS global Phase 2b/3 study builds on pre-clinical and clinical proof of concept studies which demonstrated mocravimod’s ability to improve survival outcomes for patients with hematological malignancies requiring allogeneic HCT. The mode of action has been well-established in autoimmune indications, but never in hematology. Mocravimod has the potential to be a first-in-class therapy in maintaining the graft-versus-leukemia effect, while preventing graft-versus-host disease, one of the most serious complications of allogeneic HCT. We expect this trial to deliver important clinical data supporting the registration of mocravimod in this indication.”

Florent Gros, Co-Founder and CEO of Priothera, said: “Having successfully enrolled the first AML patients undergoing allogeneic HCT in our MO-TRANS global study represents a significant milestone for Priothera as we believe mocravimod has the potential to address a significant unmet need. Furthermore, we anticipate a strong uptake in patient enrollment with a significant number of patients currently being identified. We look forward to seeing topline results in 2025.”