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

Priothera Receives FDA clearance of Investigational New Drug (IND)

Priothera Receives FDA clearance of Investigational New Drug (IND)

NEWS

April 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 – 26th April 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

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 to present an abstract at the TANDEM Meetings / Transplantation & Cellular Therapy Meetings of ASTCT™ and CIBMTR – April 23-26 2022

Priothera to present an abstract at the TANDEM Meetings / Transplantation & Cellular Therapy Meetings of ASTCT™ and CIBMTR – April 23-26 2022

PRESS RELEASE

April 2022

Priothera to present an abstract at the TANDEM Meetings / Transplantation & Cellular Therapy Meetings of ASTCT™ and CIBMTR – April 23-26 2022

Simone Dertschnig1, Stephan Oehen, Dominik Heim, Jürgen Finke and Christoph Bucher.
Priothera SAS, St. Louis, France, 2University Hospital Basel, Basel, Switzerland, 3University of Freiburg, Freiburg, Germany.

Background

The success of allogeneic hematopoietic stem cell transplantation (HSCT) is limited by disease relapse. Alloreactive donor T cells have the potential to reject residual malignant cells and are essential to prevent relapse by graft-versus-leukemia (GVL) effect. This GVL response is very powerful, however, the same T cells cause acute graft-versus-host disease (aGVHD). T cell trafficking from lymphoid organs is a major step in the development of aGVHD and is regulated by sphingosine-1-phosphate (S1P) gradients. Pre-clinical data showed that pharmacological modulation of S1P receptor (S1PR) signaling by KRP203 sequesters T cells in lymphoid organs and prevents their egress to aGVHD target sites. In murine models, KRP203 efficiently reduced aGVHD without suppressing T cell function, while GVL was maintained and survival improved. Here, we show data of the first clinical trial investigating S1PR modulation by KRP203 as GVHD prophylaxis while maintaining GVL in patients undergoing HSCT for the tratement of hematological malignancies.

Study design

This multicentric, phase Ib, prospective, open label, two-part study evaluated safety, tolerability and pharmacokinetics of KRP203 in intermediate to high-risk patients undergoing allogeneic HSCT for hematological malignancies. Three treatment arms were investigated: 3 mg KRP203 + cyclosporine A (CsA)/methotrexate (MTX) (CsA3), 1 mg KRP203 + CsA/MTX (CsA1) and 3 mg KRP203 + tacrolimus (Tac)/MTX (Tac3). KRP203 was administered daily from day 1 until day 111 and HSCT was performed on day 11.

Results

Upon KRP203 treatment, absolute lymphocyte counts (ALC) stabilized above 0.2×109/L but were efficiently reduced in all three treatment arms below pre-HSCT level (<0.7×109/L). Tretment effect was resolved when KRP203 was stopped on day 111, which caused an increase in blood ALC to pre-HSCT level. CD4+ and CD8+ T cells, GVL mediators and major players in the pathophysiology of aGVHD, were reduced in peripheral blood in response to KRP203 treatment. Only CD4+ T cells remained below pre-HSCT level (<100 cells/μl).

Overall survival probaility was highest for subjects in the CsA3 arm with the first death reported on day 532. Overall survival at 1 year was 100% in the CsA3 arm, 67% in the CsA1 arm and 36% in the Tac3 arm. The median time to relapse was 749 days. 2/6 subjects in the CsA1 arm, 4/10 subjects in the CsA3 arm and 4/7 subjects in the Tac3 arm reported relapse. The median time to any aGVHD was 55 days and 21.7% of subjects presented with grade III-IV aGVHD. The median time to any chronic GVHD was 174 das and 56.5% of subjects presented with moderate and 8.7% with severe chronic GVHD. 52.2% of subjects did not experience a GRFS event and were alive at 6 months.

Conclusion

KRP203 shows promising early clinical outcome with a limited number of relapses, acute and chronic GVHD. The results of this small study support the further investigations of mocravimod in a homogeneous patient population undergoing allogeneic HSCT to confirm safety and assess clinical efficacy, such as overall survival, relapse-free survival and GVHD-free survival.

TANDEM Meetings / Transplantation & Cellular Therapy Meetings of ASTCT™ and CIBMTR

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

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

NEWS

April 2022

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 202

Dublin, Ireland – 11th 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.”

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

Contact

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 Receives R&D Innovation Loan from Bpifrance

Priothera Receives R&D Innovation Loan from Bpifrance

NEWS

April 2022

Priothera Receives R&D Innovation Loan from Bpifrance

€1.5 million loan will further support clinical manufacture of mocravimod for the adjunctive treatment of Acute Myeloid Leukemia (AML)

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.

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

 

About Bpifrance

Bpifrance is the one stop shop for entrepreneurs with a vastly comprehensive toolbox offered in the field to customers through 50 local branches. Our mission is simple: we believe in serving the future, by being entrepreneur-centric and heavily decentralized.

Contact

Priothera
Philippe Lievre, Chief Business Officer Priothera
T: +41 76 802 11 30
E : philippe.lievre@priothera.com

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