Priothera cleared to begin Japanese arm of pivotal Phase 3 study with mocravimod in patients with Acute Myeloid Leukemia undergoing allogeneic Hematopoietic Cell Transplant

Priothera cleared to begin Japanese arm of pivotal Phase 3 study with mocravimod in patients with Acute Myeloid Leukemia undergoing allogeneic Hematopoietic Cell Transplant

NEWS

March 2023

Priothera cleared to begin Japanese arm of pivotal Phase 3 study with mocravimod in patients with Acute Myeloid Leukemia undergoing allogeneic Hematopoietic Cell Transplant

Mocravimod is the only S1PR modulator being developed as an adjunctive and maintenance treatment for blood cancers

Dublin, Ireland – February 27th, 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.” 

About mocravimod

Mocravimod (also known as KRP203) is a synthetic, sphingosine 1-phosphate receptor (S1PR) 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 Acute Myeloid Leukemia (AML) receiving allogeneic hematopoietic cell transplantation (HCT). Allogeneic HCT is the only potentially curative approach for AML patients, but current treatments have unacceptably high mortality and morbidity rates.

Priothera leverages mocravimod’s unique mode of action to maintain the beneficial graft-versus leukemia (GVL) activity, while reducing tissue damage resulting from graft-versus-host disease (GVHD), both a consequence of allogeneic HCT. This novel treatment approach – mocravimod being the only S1PR modulator treating blood cancers – tackles a high unmet medical need and aims to improve patients’ quality of life.

 

About Priothera

Priothera is leading the way in developing orally applied sphingosine-1-phosphate (S1P) receptor modulators for the treatment of hematological malignancies and for the improvement of CAR-T cell therapies. S1P receptor modulators are known to largely reduce egress of T cells from lymphatic tissues. Unlike immunosuppressive drugs, mocravimod does not suppress the graft-versus-leukemia (GVL) benefits in patients receiving HCT while inhibiting graft-versus-host-disease (GVHD).

Priothera was founded in 2020 by an experienced team of drug development experts and is headquartered in Dublin, Ireland, and with a subsidiary in Saint-Louis, France. The Company is backed by international founding investors Fountain Healthcare Partners (Dublin, Ireland), funds managed by Tekla Capital Management, LLC (Boston, Massachusetts), HealthCap (Stockholm, Sweden), EarlyBird Venture Capital (Berlin, Germany), as well as non-dilutive financing in the form of loans from the European Investment Bank under its Venture Debt Instrument and Bpifrance (Grand Est Bpifrance) in the form of a R&D innovation loan.

For more information please visit: www.priothera.com or follow Priothera on LinkedIn www.linkedin.com/company/priothera/

Contact

Priothera
Florent Gros, CEO
E: info@priothera.com

MEDiSTRAVA Consulting
Sylvie Berrebi, Sandi Greenwood, Frazer Hall
E: priothera@medistrava.com
T: +44 (0) 203 928 6900

Priothera announces first patients enrolled in pivotal MO-TRANS global Phase 2b/3 study with mocravimod as an adjunctive and maintenance therapy for patients with Acute Myeloid Leukemia (AML) undergoing allogeneic Hematopoietic Cell Transplant (HCT)

Priothera announces first patients enrolled in pivotal MO-TRANS global Phase 2b/3 study with mocravimod as an adjunctive and maintenance therapy for patients with Acute Myeloid Leukemia (AML) undergoing allogeneic Hematopoietic Cell Transplant (HCT)

NEWS

January 2023

Priothera announces first patients enrolled in pivotal MO-TRANS global Phase 2b/3 study with mocravimod as an adjunctive and maintenance therapy for patients with Acute Myeloid Leukemia (AML) undergoing allogeneic Hematopoietic Cell Transplant (HCT)

Mocravimod is the only S1PR modulator being developed to treat blood cancers and improve CAR-T therapy 

Phase 1b/2a data has shown mocravimod is safe and well tolerated

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.”  

About mocravimod

Mocravimod (also known as KRP203) is a synthetic, sphingosine 1-phosphate receptor (S1PR) 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 2b/3 study for patients with Acute Myeloid Leukemia (AML) receiving allogeneic hematopoietic cell transplantation (HCT). Allogeneic HCT is the only potentially curative approach for AML patients, but current treatments have unacceptably high mortality and morbidity rates.

Priothera leverages mocravimod’s unique mode of action to maintain the beneficial graft-versus leukemia (GVL) activity, while reducing tissue damage resulting from graft-versus-host disease (GVHD), both a consequence of allogeneic HCT. This novel treatment approach – mocravimod being the only S1PR modulator treating blood cancers – tackles a high unmet medical need and aims to improve patients’ quality of life.

About Priothera

Priothera is leading the way in developing orally applied sphingosine-1-phosphate (S1P) receptor modulators for the treatment of hematological malignancies and for the improvement of CAR-T cell therapies. S1P receptor modulators are known to largely reduce egress of T cells from lymphatic tissues. Unlike immunosuppressive drugs, mocravimod does not suppress the graft-versus-leukemia (GVL) benefits in patients receiving HCT while inhibiting graft-versus-host-disease (GVHD).

Priothera was founded in 2020 by an experienced team of drug development experts and is headquartered in Dublin, Ireland, and with a subsidiary in Saint-Louis, France. The Company is backed by international founding investors Fountain Healthcare Partners (Dublin, Ireland), funds managed by Tekla Capital Management, LLC (Boston, Massachusetts), HealthCap (Stockholm, Sweden), EarlyBird Venture Capital (Berlin, Germany), as well as non-dilutive financing in the form of loans from the European Investment Bank under its Venture Debt Instrument and Bpifrance (Grand Est Bpifrance) in the form of a R&D innovation loan.

For more information please visit: www.priothera.com or follow Priothera on LinkedIn www.linkedin.com/company/priothera/

Contact

Priothera
Florent Gros, CEO
E: info@priothera.com

MEDiSTRAVA Consulting
Sylvie Berrebi, Sandi Greenwood, Frazer Hall
E: priothera@medistrava.com
T: +44 (0) 203 928 6900

Priothera – Positive mocravimod Phase 1b clinical data published in Transplantation and Cellular Therapy

Priothera – Positive mocravimod Phase 1b clinical data published in Transplantation and Cellular Therapy

NEWS

November 2022

Priothera – Positive mocravimod Phase 1b clinical data published in Transplantation and Cellular Therapy

Mocravimod is safe and well-tolerated in allogeneic hematopoietic cell transplantation (HCT) patients

Mocravimod significantly reduced circulating lymphocyte numbers and did not affect engraftment

Dublin, Ireland – 14th November 2022 – Priothera, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator compound mocravimod, announces that positive data from the Phase 1b clinical trial evaluating mocravimod in allogeneic hematopoietic cell transplantation patients has been published in Transplantation and Cellular Therapy.

The study assessed the safety and tolerability of mocravimod in patients undergoing allogeneic hematopoietic cell transplantation (allo-HCT) for hematological malignancies. The secondary objectives were to determine the pharmacokinetic profile of mocravimod in this patient group as well as to assess GvHD-free, relapse-free survival at 6 months after last treatment.

The study found that mocravimod can safely be added to standard treatment regimens in patients with hematological malignancies requiring allo-HCT. CD4+ T cells were more sensitive to mocravimod treatment than CD8+ T cells. Mocravimod resulted in a significant reduction of circulating lymphocyte numbers and had no negative impact on engraftment and transplant outcomes.

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 HCT.

A global Phase 2b/3 study assessing the efficacy and safety of mocravimod as an adjunctive and maintenance therapy in acute myeloid leukemia (AML) patients undergoing allo-HCT is planned to start in the coming months. The trial design for this study will be published as an online abstract as part of the 2022 American Society of Hematology (ASH) conference (December 10-13th).

Florent Gros, Co-Founder and CEO of Priothera, said: “The positive Phase 1b data reinforce the potential for mocravimod to improve survival outcomes for patients with hematological malignancies requiring HCT. We’re looking forward to building upon this foundation with our upcoming pivotal Phase 2b/3 trial and advancing our lead asset towards its next significant milestones.”

Elisabeth Kueenburg, M.D., Chief Medical Officer at Priothera, commented: “Mocravimod’s mode of action has already been well established in autoimmune indications, and the Phase 1b trial shows that its potential also extends to hematology. We believe mocravimod has the potential to be a first-in-class therapy in maintaining graft-versus-leukemia responses, one of the most serious complications of allogeneic HCT, while preventing graft-versus-host disease.”

References
S. Dertschnig et al. Mocravimod, a selective S1PR modulator in allogeneic hematopoietic stem cell transplantation for malignancy, Transplantation and Cellular Therapy.

Online publication: https://doi.org/10.1016/j.jtct.2022.10.029

About Mocravimod

Mocravimod (also known as KRP203) is a synthetic, sphingosine 1-phosphate receptor (S1PR) 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 – commonly known as leukemias and lymphomas – led Priothera to further develop mocravimod for the treatment of blood cancers and the improvement of CAR-T cell therapy.

Mocravimod is being investigated as an adjunctive and maintenance treatment in a Phase 2b/3 study for patients with Acute Myeloid Leukemia (AML) receiving allogeneic hematopoietic cell transplantation (HCT). Allogeneic HCT is the only potentially curative approach for AML patients, but current treatments have unacceptably high mortality and morbidity rates.

Priothera leverages mocravimod’s unique mode of action to maintain the beneficial graft-versus leukemia (GVL) activity, while reducing tissue damage resulting from graft-versus-host disease (GVHD), both a consequence of allogeneic HCT. This novel treatment approach – mocravimod being the only S1PR modulator treating blood cancers – tackles a high unmet medical need and intends to add quality life to patients.

 

About Priothera

Priothera is leading the way in developing orally applied sphingosine-1-phosphate (S1P) receptor modulators for the treatment of hematological malignancies and for the improvement of CAR-T cell therapies. S1P receptor modulators are known to largely reduce egress of T cells from lymphatic tissues. Unlike immunosuppressive drugs, mocravimod does not suppress the graft-versus-leukemia (GVL) benefits in patients receiving HCT while inhibiting graft-versus-host-disease (GVHD).

Priothera was founded in 2020 by an experienced team of drug development experts and is headquartered in Dublin, Ireland, and with a subsidiary in Saint-Louis, France. The Company is backed by international founding investors Fountain Healthcare Partners (Dublin, Ireland), funds managed by Tekla Capital Management, LLC (Boston, Massachusetts), HealthCap (Stockholm, Sweden), EarlyBird Venture Capital (Berlin, Germany), as well as non-dilutive financing in the form of loans from the European Investment Bank under its Venture Debt Instrument and Bpifrance (Grand Est Bpifrance) in the form of a R&D innovation loan.

For more information please visit: www.priothera.com or follow Priothera on LinkedIn www.linkedin.com/company/priothera/

Contact

Priothera
Florent Gros, CEO
E: info@priothera.com

MEDiSTRAVA Consulting
Sylvie Berrebi, Sandi Greenwood, Frazer Hall
E: priothera@medistrava.com
T: +44 (0) 203 928 6900

Priothera – Clinical Mocravimod Data in Hematological Malignancies Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplant (aHSCT) Presented at the 2022 European Hematology Association (EHA) Congress

Priothera – Clinical Mocravimod Data in Hematological Malignancies Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplant (aHSCT) Presented at the 2022 European Hematology Association (EHA) Congress

NEWS

June 2022

Priothera – Clinical Mocravimod Data in Hematological Malignancies Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplant (aHSCT) Presented at the 2022 European Hematology Association (EHA) Congress

Phase 1b/2a data shows that mocravimod is safe and well tolerated

Dublin, Ireland – 13th June 2022 – Priothera, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator compound, mocravimod, presented first clinical data on mocravimod (also known as KRP203) in hematological malignancies patients undergoing allogeneic hematopoietic stem cell transplant (HSCT) at the European Hematology Association (EHA) 2022 Congress, which was held in Vienna, Austria, June 9-12th, 2022.

The oral presentation, which took place on 12th June, was entitled A two-part, single- and two-arm randomized, open-label study to evaluate the safety, tolerability and pharmacokinetics of KRP203 in subjects with hematological malignancies undergoing allogeneic hematopoietic stem cell transplantation. The study showed that in the Phase 1b/2a study mocravimod was safe and well tolerated with promising overall survival.

Key take aways from the data presentation were:

  • The study provided first ever human data of a S1P receptor modulator administered to patients undergoing allogeneic HSCT
  • S1PR modulator class effects, such as bradycardia, were of no clinical concern
  • Promising overall survival (OS) data
  • Limited number of relapses, acute and chronic graft-versus-host disease (GVHD)
  • Data supportive for conducting the planned MO-TRANS pivotal study to investigate efficacy (Relapse-free survival and OS) and GVHD in AML patients undergoing allogeneic HSCT

Priothera is initiating the pivotal MO-TRANS global Phase 2b/3 study in Europe, US and Japan, to assess the efficacy and safety of mocravimod as an adjunctive and maintenance therapy in adult AML patients undergoing allogeneic HSCT. The 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, said: “We are delighted to have been given the opportunity to present the full first human clinical data on mocravimod at the European Hematology Association Congress this year. Based on these results mocravimod has the potential to be a first-in-class therapy in maintaining graft-versus-leukemia responses while preventing graft-versus-host disease. This novel approach would provide a tremendous benefit to AML patients undergoing an allogeneic hematopoietic stem cell transplant. We are excited about mocravimod which leverages a well-described mode of action in a hematology/oncology setting and has been successfully used in autoimmune indications. Pre-clinical and clinical proof of concept studies have herewith demonstrated mocravimod’s ability to improve survival outcomes for this devastating disease. We look forward to advancing the global pivotal Phase 2/3 trial which is due to start in the second half of 2022.”  

Priothera’s abstract is available on EHA2022 Congress Abstract Book, here: https://journals.lww.com/hemasphere/Documents/EHA2022%20Congress%20Abstract%20Book.pdf

About Mocravimod

Mocravimod (also known as KRP203) is a synthetic, sphingosine 1-phosphate receptor (S1PR) 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 CART cell therapy.

Mocravimod will be investigated as an adjunctive and maintenance treatment in a Phase 2b/3 study as a potential treatment for patients with Acute Myeloid Leukemia (AML) receiving allogeneic hematopoietic stem cell transplantation (HSCT). Allogeneic HSCT is the only potentially curative approach for AML patients, but current treatments have unacceptably high mortality and morbidity rates.

Priothera leverages S1PR modulator’s unique mode of action to maintain anti-leukemia activity – graft-versus leukemia (GVL) while reducing tissue damage resulting from graft-versus-host disease (GVHD), a consequence of allogeneic HSCT. This novel treatment approach – mocravimod being the only S1PR modulator treating blood cancers – tackles a high unmet medical need and intends to add quality life to patients.

 

About Priothera

Priothera is leading the way in developing orally applied sphingosine-1-phosphate (S1P) receptor modulators for the treatment of hematological malignancies and for the improvement of CART cell therapies. S1P receptor modulators are known to largely reduce egress of T cells from lymphatic tissues. Not being an immunosuppressant, mocravimod maintains the graft-versus-leukemia (GVL) benefits in patients receiving HSCT while inhibiting graft-versus-host-disease (GVHD).

Priothera was founded in 2020 by an experienced team of drug development experts and is headquartered in Dublin, Ireland, and with a subsidiary in Saint-Louis, France. The Company is backed by international founding investors Fountain Healthcare Partners (Dublin, Ireland), funds managed by Tekla Capital Management, LLC (Boston, Massachusetts), HealthCap (Stockholm, Sweden) and EarlyBird Venture Capital (Berlin, Germany).

For more information please visit: www.priothera.com or follow Priothera on LinkedIn www.linkedin.com/company/priothera/

Contact

Priothera
Florent Gros, CEO
E: info@priothera.com

MEDiSTRAVA Consulting
Sylvie Berrebi, Sandi Greenwood, Frazer Hall
E: priothera@medistrava.com
T: +44 (0) 203 928 6900

Priothera Receives Fast Track Designation for mocravimod in Combination with Allogeneic Hematopoietic Stem Cell Transplant (HSCT) for Post Remission Therapy of Acute Myeloid Leukemia (AML) Patients

Priothera Receives Fast Track Designation for mocravimod in Combination with Allogeneic Hematopoietic Stem Cell Transplant (HSCT) for Post Remission Therapy of Acute Myeloid Leukemia (AML) Patients

NEWS

June 2022

Priothera Receives FDA clearance of Investigational New Drug (IND) to start Phase 2b/3 study with mocravimod in Acute Myeloid Leukemia (AML) Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplant (HSCT)

Global Phase 2b/3 trial (MO-TRANS) assessing the efficacy and safety of mocravimod, a novel S1P receptor modulator, as an adjunctive and maintenance therapy in AML patients undergoing allogeneic HSCT, planned to start in H2 2022

Dublin, Ireland – 31st May 2022Priothera, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator compound, mocravimod, today announces that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation (FTD) for mocravimod in combination with allogeneic Hematopoietic Stem Cell Transplant (HSCT) for post remission therapy of Acute Myeloid Leukemia (AML) patients. FDA’s Fast Track designation is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening diseases and that demonstrate the potential to address unmet medical needs.

Priothera is working to initiate the MO-TRANS global Phase 2b/3 study in Europe, US and Japan, to assess the efficacy and safety of mocravimod as an adjunctive and maintenance therapy in adult AML patients undergoing allogeneic 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.

Karen Von Graevenitz, Head of Regulatory Affair at Priothera, commentedThe Fast Track designation grant for mocravimod in combination with allogeneic HSCT is an important milestoneand underlines the significant unmet need in AML patients undergoing HSCT, a serious disease where currently no available therapy exists. The designation means mocravimod will be eligible for expedited review and we will work closely with the US FDA to advance the global Phase 2/3 trial which is due to start in the second half of 2022.

Florent Gros, Co-Founder and CEO of Priothera, added:Following being granted orphan drug designations for mocravimod in the US and Europe, we are pleased to have been granted Fast Track designation for this highly promising compound. This important regulatory milestone moves us a step closer to bringing mocravimod to patients with AML and other hematologic malignancies.”

About Mocravimod

Mocravimod (also known as KRP203), is a synthetic, sphingosine 1-phosphate receptor (S1PR) 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.

Mocravimod will be investigated as an adjunctive and maintenance treatment in a Phase 2b/3 study as a potential treatment for patients with Acute Myeloid Leukemia (AML) receiving allogeneic hematopoietic stem cell transplantation (HSCT). Allogeneic HSCT is the only potentially curative approach for AML patients, but current treatments have unacceptably high mortality and morbidity rates.

Priothera leverages S1PR modulator’s unique mode of action to maintain anti-leukemia activity – graft-versus leukemia (GVL) while reducing tissue damage resulting from graft-versus-host disease (GVHD), a consequence of allogeneic HSCT. This novel treatment approach – mocravimod being the only S1PR modulator treating blood cancers – tackles a high unmet medical need and intends to add quality life to patients.

About Priothera

Priothera is leading the way in developing orally applied sphingosine1phosphate (S1P) receptor modulators for the treatment of hematological malignancies. S1P receptor modulators are known to largely reduce egress of T cells from lymphatic tissues. Not being an immunosuppressant, mocravimod maintains the graft-versus-leukemia (GVL) benefits in patients receiving HSCT while inhibiting graft-versus-host-disease (GVHD).

Priothera was founded in 2020 by an experienced team of drug development experts and is headquartered in Dublin, Ireland, and with a subsidiary in Saint-Louis, France. The Company is backed by international founding investors Fountain Healthcare Partners (Dublin, Ireland), funds managed by Tekla Capital Management, LLC (Boston, Massachusetts), HealthCap (Stockholm, Sweden) and EarlyBird Venture Capital (Berlin, Germany).

For more information please visit: www.priothera.com or follow Priothera on LinkedIn www.linkedin.com/company/priothera/.

Contact

Priothera
Florent Gros, CEO
E: info@priothera.com

MEDiSTRAVA Consulting
Sylvie Berrebi, Sandi Greenwood, Frazer Hall
E: priothera@medistrava.com
T: +44 (0) 203 928 6900