Dublin, Ireland – 11^th April 2022 – Priothera Ltd, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator mocravimod, today announces it has received the two first European country approvals from the Swiss and French national health authorities (Swissmedic and ANSM) to begin its planned pivotal study of mocravimod. The company has also received encouraging feedback from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) on the clinical study design that is closely aligned with earlier feedback from the US Food and Drug Administration (FDA).

Priothera will initiate a global Phase 2b/3 study (MO-TRANS study) in Europe, US and Japan, assessing the efficacy and safety of mocravimod as an adjunctive and maintenance therapy in adult Acute Myeloid Leukemia (AML) patients undergoing allogenic hematopoietic stem cell transplant (HSCT). The MO-TRANS study is expected to start in the second half of 2022 and preliminary data from this study are expected by the end of 2024. 

Florent Gros, Co-Founder and CEO of Priothera, commented “Following on from our recent receipt of orphan drug designations for mocravimod in the US and Europe, we are pleased to have received our first approvals to initiate this key global Phase 2b/3 trial with this highly promising compound. Moreover, the CHMP feedback is encouraging, and is also closely aligned with the feedback we received from the FDA. These first approvals are important regulatory and clinical milestones for Priothera and move us a step closer to bringing mocravimod, an adjunctive and maintenance treatment, to patients with AML and other hematologic malignancies, for whom there remains a significant unmet medical need.”

Dublin, Ireland and Saint-Louis, France – Monday 4 th April 2022 – Priothera, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator drug, mocravimod, today announces that it has entered a €1.5 million Loan Agreement with the regional Bpifrance in Strasbourg (Grand Est Bpifrance), via Priothera SAS, its French affiliate.

This R&D innovation loan will be used to further support the clinical manufacture of mocravimod for a European, US and Asian registration-enabling clinical trial with mocravimod in Acute Myeloid Leukemia (AML) patients undergoing allogeneic hematopoietic stem cell transplant (HSCT).

Brice Suire, Co-Founder and Chief Financial Officer of Priothera, said: “This non-dilutive financing, alongside the funding from the European Investment Bank that we announced recently, will play an important role in financing the development of mocravimod. It will allow us to strengthen our local French team and accelerate delivery of the clinical supply of mocravimod needed for our upcoming registration-enabling global clinical trial. We are very pleased with the confidence shown by Grand Est Bpifrance in supporting the Company in its development program.”

Alban Stamm, Innovation Manager at Bpifrance Alsace commented: “We believe that Priothera, with its drug candidate mocravimod, has the potential to enable a major breakthrough in preventing transplant rejection which would provide a tremendous benefit to AML patients undergoing a stem cell transplant. The Bpifrance loan will support Priothera’s key objective of establishing human proof of concept and generating registrational data, allowing the company to create significant socio-economic value in France and beyond that for patients globally.”

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 HSCT in patients with AML. Moreover, it has shown clinically relevant benefits in an early clinical study in patients with hematologic malignancies undergoing HSCT.

In recent decades, the incidence of some hematologi- cal malignancies (HMs), such as acute lymphocytic leukemia (ALL) and chronic myeloid leukemia (CML), has decreased, while the incidence of others, includ- ing chronic lymphocytic leukemia (CLL) and acute myeloid leukemia (AML), have increased in most countries worldwide. At the same time, the number of allogeneic hematopoietic stem cell transplants (HSCT), an established treatment for HMs following chemotherapy, has also increased significantly.

Yet HMs remain a major cause of cancer deaths, with overall mortality rising in recent decades—a fact that points to the unsatisfactory curative poten- tial of HSCT. Priothera is addressing this clear unmet medical need with the development of mocravimod, a small-molecule immune-modulating adjunctive and maintenance therapy for HMs that is initially being developed for AML with the first study in adult AML patients to be initiated in 2022.

For many intermediate and high-risk AML patients, as well as those with other HMs, chemotherapy fol- lowed by HSCT is the only potentially curative treat- ment option, but one that comes with a significant risk of serious and sometimes fatal graft-versus- host-disease (GvHD). HSCT is also limited in efficacy, due to immune-suppressive comedications that aim at controlling GvHD: 40% of post-HSCT AML patients relapse, with a dismal prognosis of 2-year survival less than 20%.

Mediating graft-versus-host disease