US FDA grants orphan drug designation to Priothera’s mocravimod to improve the outcome of allo-HSCT in patients with hematologic malignancies

Priothera Ltd., a phase 3 clinical stage biotechnology company pioneering the development of its S1P receptor modulator compound mocravimod, announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation (ODD) to mocravimod for the “treatment to improve outcome following hematopoietic stem cell transplantation in hematologic malignancies”. This ODD aims to potentially increase leukaemia-free survival by triggering a ‘graft-versus-leukaemia’ (GvL) response.

Florent Gros, co-founder and CEO of Priothera, commented: “We are very pleased that the US FDA granted mocravimod this Orphan Drug designation. This designation emphasizes the importance of developing novel therapeutic options to improve the outcome and success of maintenance therapy following allo-HSCT in blood cancer patients. This is an important milestone as this ODD complements the first ODD granted for prevention of ‘graft-versus-host disease’. 

“The two ODDs highlight mocravimod’s dual mode of action which for the first time is being leveraged to improve the allo-HSCT treatment outcomes in haematological malignancies to potentially increase the leukaemia free survival – ‘graft-versus-leukaemia’ response – while reducing tissue damage resulting from the ‘graft-versus-host disease’.”

The first ODD granted for mocravimod by the US FDA was for the “prevention of graft-versus-host disease (GvHD).”

Mocravimod, a sphingosine-1-phosphate (S1P) receptor modulator, is being investigated in a pivotal global phase 3 study – MO-TRANS (NCT05429632) – evaluating the efficacy and safety of mocravimod as an adjunctive and maintenance therapy to allo-HSCT. The study which is enrolling approximately 250 adult Acute Myeloid Leukemia (AML) patients, is ongoing in the US, Europe, Southeast Asia and Latin America.

Mocravimod, which has been previously tested in multiple autoimmune indications, is being developed to enhance the curative potential of allo-HSCT. Moreover, it has shown a clinically meaningful outcome in a phase 1b/2a study in patients with hematologic malignancies undergoing allo-HSCT.

The Orphan Drug designation is reserved for medicines treating rare, life-threatening or chronically debilitating diseases.

Mocravimod (also known as KRP203) is a synthetic, S1P receptor modulator. This novel investigational drug has been assessed in phase 1 and phase 2 trials for safety and tolerability, as well as for efficacy in several autoimmune indications. Promising data from a phase 1b/2a clinical study in patients with hematological malignancies led Priothera to further develop mocravimod for the treatment of blood cancers and the improvement of CAR-T cell therapy.

Mocravimod is currently being investigated as an adjunctive and maintenance treatment in a Phase 3 study for patients with AML receiving allogeneic HSCT. Allogeneic HSCT is the only potentially curative approach for AML patients, but current treatments have unacceptably high mortality and morbidity rates.

Priothera leverages mocravimod’s dual mode of action to maintain the beneficial graft-versus-leukaemia/lymphoma (GvL) activity, while reducing tissue damage resulting from graft-versus-host disease (GvHD), both a consequence of allo-HSCT. This novel treatment approach – mocravimod being the only S1P receptor modulator treating blood cancers – tackles a high unmet medical need and aims to improve patients’ quality of life.

Priothera is leading the way in developing orally applied S1P receptor modulators for the treatment of haematological malignancies and for the improvement of CAR-T cell therapies.

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