US FDA grants Orphan Drug Designation to mocravimod to improve the outcome of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with hematologic malignancies

• Mocravimod is the only S1P receptor modulator being developed as an adjunctive and maintenance treatment for blood cancers

• Mocravimod is being investigated in a global registration-enabling Phase 3 trial

• This is the second Orphan Drug designation for mocravimod

Priothera Ltd., a Phase 3 clinical stage biotechnology company pioneering the development of its S1P receptor modulator compound mocravimod, today 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 leukemia-free survival by enhancing a graft-versus-leukemia (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 hematological malignancies to potentially increase the leukemia free survival – graft-versus-leukemia 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)’ – see press release here.

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[1] in patients with hematologic malignancies undergoing allo-HSCT.

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

About mocravimod

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-leukemia/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.

About Priothera

Priothera is leading the way in developing orally applied 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 GvL benefits in patients receiving allogeneic HSCT while inhibiting 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/

Contacts

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

[1]*Ref: Dertschnig et al, 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 AML undergoing allogeneic 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

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 doses 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 towards 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/

Contacts

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

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

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

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/

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, 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 Affairs at Priothera, commented: “The Fast Track designation grant for mocravimod in combination with allogeneic HSCT is an important milestone and 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 sphingosine-1-phosphate (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/

Contacts

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 FDA clearance of 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

Priothera Ltd, 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 provided clearance to proceed with the Company's Investigational New Drug (IND) application to begin its pivotal Phase 2b/3 study of mocravimod (named MO-TRANS).

Priothera will initiate the MO-TRANS global Phase 2b/3 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.

Allogenic stem cell transplantation is the only potentially curative approach for AML patients, however current treatment options are still associated with a high number of side effects, and high mortality rates.

Florent Gros, Co-Founder and CEO of Priothera, commented "The FDA IND clearance to initiate the MO-TRANS study assessing mocravimod in AML patients undergoing allogeneic HSCT is another major milestone for Priothera. We are on track to initiate this pivotal Phase 2b/3 clinical trial and are looking forward to working alongside a large team of enthusiastic investigators across the US, Europe and Asia, who share our goal of bringing mocravimod to patients as an adjunctive and maintenance treatment for AML and potentially 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). Allogenic 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 allogenic 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. 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

Contacts

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 First Regulatory Approvals to Start a Global Pivotal Study with Mocravimod in Acute Myeloid Leukemia Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplant

Global Phase 2b/3 trial assessing the efficacy and safety of mocravimod as an adjunctive and maintenance therapy in AML patients undergoing allogeneic HSCT planned to start in H2 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.”

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). Allogenic 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 allogenic 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. 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

Priothera – FDA and EMA Grant Orphan Drug Designation to mocravimod for the treatment of Acute Myeloid Leukemia (AML) in patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT)

• Mocravimod is being developed as a potential best-in-class adjunctive and maintenance therapy to enhance the curative potential of HSCT for AML patients

• A global registration-enabling Phase 2b trial assessing mocravimod in AML patients undergoing allogeneic HSCT is planned in H2 2022

Priothera, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator drug, mocravimod, today announced that the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have both granted orphan drug designation (ODD) to mocravimod for the treatment of Acute Myeloid Leukemia (AML) in patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). EMA’s ODD follows a recommendation from the Committee for Orphan Medicinal Products (COMP).

Florent Gros, Co-Founder and CEO of Priothera, commented: “The orphan drug designations we received for mocravimod from both the FDA and EMA are important milestones towards addressing the urgent, unmet needs of AML patients. Allogenic stem cell transplantation is the only potentially curative approach for AML patients but has unacceptably high mortality rates with current treatments. We are looking forward to initiating the global Phase 2b clinical trial with mocravimod in multiple centers in the US, Europe and Asia in the coming months.”

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

A multicenter Phase 2b study evaluating the efficacy and safety of mocravimod as an adjunctive and maintenance therapy to HSCT in adult AML patients is planned for the second half of 2022. The study will include approximately 250 patients in several countries in Europe, the US and Asia, upon approvals from respective health authorities.

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

About mocravimod

Mocravimod (also known as KRP203), is a novel, synthetic, sphingosine 1-phosphate receptor (S1PR) modulator with a long duration in the body. Phase 1 and Phase 2 trials successfully assessed mocravimod for safety and tolerability 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 in a Phase 2b/3 study as a potential treatment for patients with Acute Myeloid Leukemia (AML) receiving allogeneic hematopoietic stem cell transplantation (HSCT). Allogenic HSCT is the only potentially curative approach for AML patients, but current treatments have unacceptably high mortality and morbidity rates. Priothera leverages S1PR’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 allogenic HSCT. This novel treatment approach – 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. S1P receptor modulators are known to largely reduce egress of T cells from lymphatic tissues and not being immunosuppressants, thereby allowing for inhibition of graft-versus-host-disease (GvHD) while maintaining graft-versus-leukemia benefits in patients receiving HSCT.

Priothera which was founded in 2020 by an experienced team of drug development experts is headquartered in Dublin, Ireland. 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

Contacts

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

MEDiSTRAVA Consulting
Sylvie Berrebi, Sandi Greenwood, Frazer Hall
E: priothera@medistrava.com
T: +44 (0)7714 306525

Priothera Enters Loan Agreement of €17.5 Million with the European Investment Bank

Loan will further support the conduct of a global registration-enabling clinical trial with mocravimod for the treatment of Acute Myeloid Leukemia, and for expansion into additional cancer indications

Priothera, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator drug, mocravimod, today announces that it has entered a Loan Agreement of €17.5 million with the European Investment Bank (EIB). This loan will further support a European, US and Asian registration-enabling clinical trial with mocravimod in Acute Myeloid Leukemia (AML) patients receiving hematopoietic stem cell transplant (HSCT).

The loan facility of €17.5 million is divided into two tranches, the first of which is €10 million and unconditional. Priothera will request payment of the first tranche in 2022 to expand the clinical development of mocravimod and prepare for its commercial drug supply. The second tranche of €7.5 million is available upon achievement of specific manufacturing, clinical and regulatory milestones and will be used to finance clinical validation in CAR-T cancer indications.

Florent Gros, Co-Founder and CEO of Priothera, comments: “We are very grateful for EIB’s support, a major and high quality European financial institution, as the Company is at an inflection point of its clinical development and potential commercialization path. This financing allows Priothera to extend its financial visibility to 2024, allowing us to complete a global registration-enabling clinical trial, as well as exploring other blood cancer indications for mocravimod. This new funding tool will help reinforce Priothera’s global lead in developing S1P receptor modulators in oncology.”

Christian Kettel Thomsen, Vice-President of the EIB, said: “The European Investment Bank Group supports development of innovative and pioneering treatments and medicine by leading biotech and medtech companies in Ireland and across Europe. Priothera’s new drugs offer an opportunity to revolutionise treatment of leukaemia and other cancers and the EIB is pleased to agree to €17.5 million of new financing to accelerate the clinical development and commercialization of mocravimod.”

About mocravimod

Mocravimod (also known as KRP203), a propane-1,3-diol derivative, is a novel, synthetic, sphingosine 1-phosphate receptor (S1PR) agonist with long duration in the body. Phase 1 and Phase 2 trials successfully assessed mocravimod for safety and tolerability in several autoimmune indications. Promising data from a Ph1b/Ph2a clinical study with patients with haematological malignancies led Priothera to further develop mocravimod for the treatment of blood cancers. Mocravimod will be investigated in a Phase 2b/3 study as a potential treatment for patients with Acute Myeloid Leukemia (AML) receiving HSCT. Allogenic HSCT is the only potentially curative approach for AML patients but remains having unacceptably high mortality and morbidity rates with current treatments.

Priothera leverages S1PRs unique mode of action to maintain anti-leukaemia activity while reducing tissue damage resulting from graft-versus-host disease (GVHD), a consequence of HSCT. This novel treatment approach - the only S1PR modulator treating blood cancers - tackles a high unmet medical need that 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 haematological malignancies. S1P receptor modulators are known to largely reduce egress of T cells from lymphatic tissues and not being immunosuppressants, thereby allowing for inhibition of graft-versus-host-disease (GvHD) while enhancing graft-versus-leukaemia benefits in patients receiving HSCT.

Headquartered in Dublin, Ireland, Priothera was founded in 2020 by an experienced team of drug development experts Drs. Florent Gros, Stephan Oehen, Dhaval Patel, Christoph Bucher, Simone Seiter, Philippe Lievre and Brice Suire. Founding investors are 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

About the European Investment Bank (EIB)

The EIB is the European Union (EU) long-term financing institution and its shareholders are the 27 EU Member States. Its mission is to contribute to the integration, balanced development and economic and social cohesion of EU Member States. It borrows large volumes of funds from the capital markets and lends them with very favourable terms to support projects which contribute to the achievement of EU objectives. The EIB is working to put the EU at the forefront of the next wave of innovation, especially in the health sector. In response to the Covid-19 health crisis, the EIB has released € 6 billion for investments in the health sector to support medical infrastructure, additional research activities or other financing related to vaccines and treatments. As a European bank supporting the climate, the EIB is one of the main fund providers in the green transition towards a more low-carbon and sustainable growth model.

Contacts

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

MEDiSTRAVA Consulting
Sylvie Berrebi, David Dible, Sandi Greenwood, Frazer Hall
E: priothera@medistrava.com
T: +44 (0)7714 306525

Priothera Appoints Elisabeth Kueenburg M.D., as Chief Medical Officer

Dr. Kueenburg, former Clinical Development Lead at Celgene, a Bristol Myers Squibb Company, to advance clinical development of mocravimod in Acute Myeloid Leukemia patients undergoing allogeneic hematopoietic stem cell transplant.

Priothera Limited, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator compound, mocravimod, announces the appointment of Elisabeth Kueenburg, M.D., as Chief Medical Officer. Dr. Kueenburg will lead the advancement of mocravimod into Phase 2b/3 clinical trials as a potential treatment for patients with Acute Myeloid Leukemia (AML) receiving hematopoietic stem cell transplantation (HSCT), and expansion of Priothera’s pipeline.

“The breadth of knowledge Elisabeth has gained working at Celgene, alongside her extensive clinical experience, makes her a crucial addition to our team,” said Florent Gros, Co-Founder and CEO of Priothera. “We are delighted to welcome Elisabeth during this exciting time as we look to progress mocravimod, into a Phase 2b/3 study as a potential treatment for patients with Acute Myeloid Leukemia receiving hematopoietic stem cell transplantation. The study is expected to begin in 2022.”

“I am pleased to join Priothera at such an important stage of its development,” said Dr. Kueenburg. “Mocravimod has the potential to address the significant unmet need of AML patients undergoing HSCT. I look forward to guiding mocravimod and future programs into the clinic and making an important contribution to Priothera’s future success.”

Dr. Kueenburg brings significant drug development and medical affairs experience from her years at Celgene where she most recently served as Clinical Development Lead. At Celgene she developed deep clinical development and medical affairs expertise, providing strategic insight and overseeing the coordination of multiple clinical trials, in the area of hematology and specifically in multiple myeloma. Furthermore, Dr. Kueenburg has supported the successful global launch of Celgene’s Revlimid.

Prior to her numerous roles at Celgene, Dr. Kueenburg spent more than 15 years in clinical practice and academic research specializing in oncology and hematology.

Dr. Kueenburg gained her Doctor of Medicine from the University of Vienna in Austria

About Priothera

Priothera is leading the way in developing orally applied sphingosine 1 phosphate (S1P) receptor modulators for hematological malignancies. S1P receptor modulators are known to largely reduce egress of T cells from lymphatic tissues and not being immunosuppressants, thereby allowing for inhibition of graft-versus-host-disease (GvHD) while enhancing graft-versus-leukemia benefits in patients receiving HSCT. Headquartered in Dublin, Ireland, Priothera was founded in 2020 by an experienced team of drug development and biotech experts.

Founding investors are 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

Priothera closes €30 million Series A financing to develop highly promising therapies for acute myeloid leukemia (AML)

Financing to advance mocravimod, a modulator of sphingosine 1 phosphate (S1P) receptors, and potential best-in-class therapy for AML patients

Priothera Limited, a clinical stage company developing orally applied sphingosine 1 phosphate (S1P) receptor modulators for haematological malignancies, today announced that it has successfully closed a Series A financing round of €30 million led by Fountain Healthcare Partners with participation from co-lead investor HealthCap and funds managed by Tekla Capital Management, LLC as well as EarlyBird Venture Capital.

Priothera will use the funds to progress the clinical development of mocravimod, a modulator of sphingosine 1 phosphate (S1P) receptors, to enhance the curative potential of allogenic hematopoietic stem cell transplantation (HSCT) for treating AML. Priothera expects to generate further randomized clinical data in high risk AML patients with this Series A funding round.

Mocravimod has already been extensively tested in multiple immunologic indications and has shown survival benefit in an early clinical study evaluating acute myeloid leukemia (AML) and acute lymphocytic leukemia (ALL) patients undergoing hematopoietic stem cell transplantation (HSCT). Priothera acquired mocravimod from KYORIN Pharmaceutical Co., Ltd.

Following the closing of the financing, Florent Gros (Priothera's Co-Founder and CEO), Dr. Dhaval Patel (Priothera's Co-Founder and CSO at UCB), Dr. Manus Rogan (Fountain Healthcare Partners Co-Founder and Managing Director), Dr. Mårten Steen (Partner at HealthCap), Dr. Henry Skinner (Senior Vice President at Tekla Capital Management, LLC) and Lionel Carnot (Partner at EarlyBird Venture Capital), have joined the Board of Directors.

Florent Gros, Co-Founder and CEO of Priothera, commented, "We are delighted to welcome this terrific syndicate of investors, who share our passion, commitment, and vision for advancing Priothera's potentially best-in-class new therapy, mocravimod, in patients with acute myeloid leukemia and other hematologic malignancies. Allogenic stem cell transplant is the only potentially curative approach for AML patients, but has unacceptably high mortality with current treatments. We are excited about mocravimod which has a unique mechanism of action and clinical proof of concept demonstrating its ability to improve survival outcomes for this devastating disease."

Dr. Dhaval Patel, Co-Founder and Board Director of Priothera, who previously led Research Europe and the Autoimmunity, Transplantation and Inflammation Disease Area at Novartis, known for its success in developing other S1P receptor modulators, stated, "Mocravimod is an outstanding, well-characterized and well-behaved S1P receptor modulator that is well-suited for clinical development and commercial manufacturing. Based on the clinical results to date, mocravimod has the potential to be a best-in-class therapy to decrease the morbidity and mortality associated with HSCT for AML. I have high hopes that Priothera can make an impactful difference for these patients."

Commenting on the launch of the company, Dr. Manus Rogan, Chairman of Priothera added, "The Priothera team has identified and validated a novel commercial opportunity for an S1P receptor modulator allowing the company to complete this significant Series A funding round. With their extensive expertise and capabilities I am confident that Florent and the team are uniquely placed to successfully generate further clinical data to highlight the significant potential of mocravimod."

Priothera
Priothera is leading the way in developing orally applied sphingosine 1 phosphate (S1P) receptor modulators for haematological malignancies. S1P receptor modulators have been suggested to largely reduce egress of T cell subsets from lymphatic tissues allowing for dual inhibition of graft-versus-host-disease (GvHD) and enhancing graft-versus-leukemia benefits in patients receiving allogenic stem cell transplant.

Headquartered in Dublin, Ireland, Priothera was founded in 2020 by Drs. Florent Gros and Dhaval Patel. Joining the founding team are the experienced industry executive, Dr. Christoph Bucher, Dr Simone Seiter, and Brice Suire.

Founding investors are 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

KYORIN Pharmaceutical Co., Ltd.

Trusted among patients and professionals in the medical industry, KYORIN Pharmaceutical strives to be a company that contributes to public health and is recognized as a key player with social significance, by improving its presence in specific therapeutic areas through global discovery programs of novel drugs. KYORIN Pharmaceutical uses a franchise customer strategy where its marketing efforts are focused on respiratory, otolaryngology and urology indications. In drug discovery, Kyorin is deploying 'selection and concentration' approach and is promoting activities aimed at identifying first-in-class drug candidates. Actively searching for and introducing external drug discovery themes as well as multi-tiered program development are at the center of Kyorin's efforts.

Fountain Healthcare Partners

Fountain Healthcare Partners is a life science focused venture capital fund with EUR 300 million (USD 354 million) under management. Within the life science sector, specific areas of interest to Fountain include specialty pharma, medical devices, biotechnology and diagnostics. The firm deploys the majority of its capital in Europe, with the balance in the United States. Fountain's main office is in Dublin, Ireland, with a second office in New York. fh-partners.com

HealthCap

HealthCap is a family of venture capital funds investing globally in life sciences. With more than EUR 1 billion raised since the start in 1996, HealthCap is one of the largest specialized providers of venture capital within life sciences in Europe. The investment strategy focuses on diseases with high-unmet medical needs and breakthrough therapies that have the potential to be transformative and change medical practice, and the lives of patients suffering these conditions. Over the years HealthCap has backed and built more than 100 companies, taken more than 40 companies public and done numerous trade sales.

Tekla

Tekla Capital Management LLC is a registered investment adviser based in Boston, Massachusetts and is the investment adviser for four closed-end funds, Tekla Healthcare Investors, Tekla Life Sciences Investors, Tekla Healthcare Opportunities Fund and Tekla World Healthcare Fund. The Funds predominately invest in the securities of public and private healthcare companies.

Earlybird

Earlybird is a venture capital investor focused on European technology companies. Founded in 1997, Earlybird invests in all growth and development phases of a company. Amongst the most experienced venture investors in Europe, Earlybird offers its portfolio companies not only financial resources, but also strategic and operational support as well as access to an international network and capital markets. Earlybird manages separate funds focussing on digital technologies in Eastern and Western Europe as well as healthcare across Europe. With over EUR 1 billion under management, seven IPOs and 24 trade sales, Earlybird is one of the most successful venture capital firms in Europe.

Contacts

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

Citigate Dewe Rogerson
Sylvie Berrebi, David Dible, Frazer Hall
E: priothera@citigatedewerogerson.com
T: +44 (0)7714 306525