Innovation & Research

TAGRAXOFUSP is a novel targeted therapy directed to the IL-3 receptor α (CD123), a target present on a wide range of malignancies. It comprises human IL-3 recombinantly fused to a truncated diphtheria toxin (DT) payload engineered such that IL-3 replaces the native DT receptor-binding domain. The IL-3 domain of TAGRAXOFUSP directs the cytotoxic DT payload to cells expressing CD123. Upon internalization, TAGRAXOFUSP irreversibly inhibits protein synthesis and induces apoptosis of the target cell.
TAGRAXOFUSP is now being investigated for the treatment of Myelofibrosis.

SUMIT-ELA Trial is an open-label, Phase 1b/2 study, to confirm safety and efficacy of samuraciclib in combination with elacestrant in adult participants with metastatic or locally advanced Hormone Receptor (HR) positive and Human Epidermal Growth Factor Receptor (HER)2-negative breast cancer.

NEXPOVIO® is a first-in-class, oral exportin 1 (XPO1) inhibitor. XPO1 is a nuclear exporter protein that transport from the nucleus to the cytoplasm several proteins, including tumor suppressors, oncogenes, and proteins involved in governing cell growth; it is often overexpressed, and its function mis-regulated in several types of cancers.

By inhibiting the XPO1 protein, tumor suppressors proteins buildup in the nucleus of malignant cells and reduce levels of oncogene products which drive cell proliferation. This ultimately leads to cell cycle arrest and death of cancer cells by apoptosis

NEXPOVIO® was approved by the U.S. FDA in 2019 for the treatment of adult patients with relapsed refractory multiple myeloma (RRMM) who have received at least four prior therapies and whose disease is resistant to several other forms of treatment, including at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. 

Important Safety Information

 US – Fast Track Approval and Orphan Drug Designation

NEXPOVIO® was approved in Europe in 2021 by the EMA as combination therapy with:

• bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy and combination therapy
• dexamethasone for the treatment of multiple myeloma in adult patients who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, two immunomodulatory agents and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy.

Important Safety Information

SENTRY is a Phase 3 study to evaluate efficacy and safety of selinexor, a selective inhibitor of nuclear export, in combination with ruxolitinib in JAK inhibitor (JAKi) treatment-naïve myelofibrosis (MF) participants.

SL-701 is an off-the-shelf, systemically delivered (subcutaneous) immunotherapy designed to direct the immune system to attack targets present on certain malignancies, including brain cancer.
SL-701 is composed of three short peptides (IL-13Rα2, EphA2, and Survivin), targeting glioma-associated antigens and designed to generate an antigen specific CD8+ T cell response.
SL-701 is currently being investigated for the treatment of recurrent glioblastoma multiforme.

MEN1703 is a clinical-stage first-in-class, dual PIM/FLT3 kinase inhibitor. Preclinical data suggests therapeutic potential in both hematological malignancies and in solid tumors.
Based on strong activity data generated in diffuse large B-cell lymphoma (DLBCL) preclinical models, MEN1703 is being evaluated in combination with standard-of-care therapy and as a single agent in relapsed/refractory DLBCL patients (Phase II trial).
In parallel the preclinical characterization of MEN1703 will continue in other hematologic indications.

Orphan Drug Designation

MEN1309 is an antibody-drug conjugate (ADC) constituted by a fully human IgG1 antibody conjugated through a cleavable linker to a cytotoxic agent (a maytansin derivative, DM4), responsible for its antitumor activity. The target antigen, CD205, is highly expressed in a wide range of both solid and hematological cancer cells. Once MEN1309 binds its target, the MEN1309/CD205 complex is rapidly internalized in antigen expressing cells where it can exert its cytotoxic activity. MEN1309 is currently being tested as a potential treatment for patients with solid tumors.

FELEZONEXOR (SL-801) is a novel, oral, small-molecule reversible inhibitor of exportin-1 (XPO1), a key nuclear transport protein. XPO1 is overexpressed by many cancers, leading to mislocalization of key tumor suppressors and growth-regulatory proteins in the cytoplasm. FELEZONEXOR is currently being tested as a potential treatment for patients with solid tumors.

MEN2312 is a potent and selective orally bioavailable KAT6A/B inhibitor. KAT6A/B belong to the MYST family of acetyltransferases (HAT) that acetylates histone H3K23 and also non-histone proteins, such as p53 exerting an oncogenic role in several tumor types, including breast cancer, where KAT6A is frequently amplified/overexpressed. Inhibiting KAT6A, MEN2312 blocks the Estrogen receptor (ER) at the transcriptional level, giving it the potential to overcome resistance to endocrine therapies due to mutation or ligand-independent constitutive activation of ER. MEN2312 is being tested as single agent and in combination with SoC in metastatic Breast Cancer ER+/HER- patients.

MEN2501 is an orally bioavailable, potent and highly selective small molecule of novel oncology molecular target which is being developed in Phase I in Solid Tumors

Phase: IND ready*

ELCIN Trial is a global, Open-label, multicenter Phase 2 study of eacestrant in women and men with ER+/HER2- metastatic breast cancer who received 1 or 2 prior hormonal therapies and no prior CDK4/6i exposure in the metastatic setting

ENGOT-EN20/GOG-3083/xport-EC-042 is a Phase 3, Randomized, Placebo-Controlled, Double-Blind, Multicenter Trial of Selinexor in Maintenance Therapy After Systemic Therapy for Patients With p53 Wild-Type, Advanced or Recurrent Endometrial Carcinoma

ADELA Trial is a global, double-blind, multicenter, randomized Phase 3 study of elacestrant plus everolimus versus elacestrant in patients with ER+/HER2-, ESR1-mutated, advanced breast cancer progressing to endocrine therapy and CDK4/6 inhibitors.

ELEGANT Trial is a global, Multicenter, randomized, open label, Phase 3 study of elacestrant vs standard endocrine therapy in women and men with node positive, ER+, HER2-negative, early breast cancer with high risk of recurrence.

SUMIT-ELA Trial is an open-label, Phase 1b/2 study, to confirm safety and efficacy of samuraciclib in combination with elacestrant in adult participants with metastatic or locally advanced Hormone Receptor (HR) positive and Human Epidermal Growth Factor Receptor (HER)2-negative breast cancer.

TAGRAXOFUSP is a novel targeted therapy directed to the IL-3 receptor α (CD123), a target present on a wide range of malignancies including Blastic Plasmacytoid Dendritic Nell Neoplasm (BPDCN). It comprises human IL-3 recombinantly fused to a truncated diphtheria toxin (DT) payload engineered such that IL-3 replaces the native DT receptor-binding domain. The IL-3 domain of TAGRAXOFUSP directs the cytotoxic DT payload to cells expressing CD123. Upon internalization, TAGRAXOFUSP irreversibly inhibits protein synthesis and induces apoptosis of the target cell.

TAGRAXOFUSP was approved by the U.S. FDA in 2018 for the treatment of adult and pediatric patients, 2 years or older, with BPDCN.

Important Safety Information

Breakthrough Therapy Designation and Orphan Drug Designation

In Europe, on January 2021 TAGRAXOFUSP was approved by the EMA as monotherapy for the first-line treatment of adult patients with BPDCN.

Important Safety Information

Orphan Drug Designation

NEXPOVIO® is a first-in-class, oral exportin 1 (XPO1) inhibitor. XPO1 is a nuclear exporter protein that transport from the nucleus to the cytoplasm several proteins, including tumor suppressors, oncogenes, and proteins involved in governing cell growth; it is often overexpressed, and its function mis-regulated in several types of cancers.

By inhibiting the XPO1 protein, tumor suppressors proteins buildup in the nucleus of malignant cells and reduce levels of oncogene products which drive cell proliferation. This ultimately leads to cell cycle arrest and death of cancer cells by apoptosis

NEXPOVIO® was approved by the U.S. FDA in 2019 for the treatment of adult patients with relapsed refractory multiple myeloma (RRMM) who have received at least four prior therapies and whose disease is resistant to several other forms of treatment, including at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. 

Important Safety Information

 US – Fast Track Approval and Orphan Drug Designation

NEXPOVIO® was approved in Europe in 2021 by the EMA as combination therapy with:

• bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy and combination therapy
• dexamethasone for the treatment of multiple myeloma in adult patients who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, two immunomodulatory agents and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy.

Important Safety Information

SL-701 is an off-the-shelf, systemically delivered (subcutaneous) immunotherapy designed to direct the immune system to attack targets present on certain malignancies, including brain cancer.
SL-701 is composed of three short peptides (IL-13Rα2, EphA2, and Survivin), targeting glioma-associated antigens and designed to generate an antigen specific CD8+ T cell response.
SL-701 is currently being investigated for the treatment of recurrent glioblastoma multiforme.

TAGRAXOFUSP is a novel targeted therapy directed to the IL-3 receptor α (CD123), a target present on a wide range of malignancies. It comprises human IL-3 recombinantly fused to a truncated diphtheria toxin (DT) payload engineered such that IL-3 replaces the native DT receptor-binding domain. The IL-3 domain of TAGRAXOFUSP directs the cytotoxic DT payload to cells expressing CD123. Upon internalization, TAGRAXOFUSP irreversibly inhibits protein synthesis and induces apoptosis of the target cell.

TAGRAXOFUSP is now being investigated for the treatment of Acute Myeloid Leukemia.

TAGRAXOFUSP is a novel targeted therapy directed to the IL-3 receptor α (CD123), a target present on a wide range of malignancies. It comprises human IL-3 recombinantly fused to a truncated diphtheria toxin (DT) payload engineered such that IL-3 replaces the native DT receptor-binding domain. The IL-3 domain of TAGRAXOFUSP directs the cytotoxic DT payload to cells expressing CD123. Upon internalization, TAGRAXOFUSP irreversibly inhibits protein synthesis and induces apoptosis of the target cell.
TAGRAXOFUSP is now being investigated for the treatment of chronic myelomonocytic leukemia.

TAGRAXOFUSP is a novel targeted therapy directed to the IL-3 receptor α (CD123), a target present on a wide range of malignancies. It comprises human IL-3 recombinantly fused to a truncated diphtheria toxin (DT) payload engineered such that IL-3 replaces the native DT receptor-binding domain. The IL-3 domain of TAGRAXOFUSP directs the cytotoxic DT payload to cells expressing CD123. Upon internalization, TAGRAXOFUSP irreversibly inhibits protein synthesis and induces apoptosis of the target cell.
TAGRAXOFUSP is now being investigated for the treatment of Myelofibrosis.

FELEZONEXOR (SL-801) is a novel, oral, small-molecule reversible inhibitor of exportin-1 (XPO1), a key nuclear transport protein. XPO1 is overexpressed by many cancers, leading to mislocalization of key tumor suppressors and growth-regulatory proteins in the cytoplasm. FELEZONEXOR is currently being tested as a potential treatment for patients with solid tumors.

MEN1703 is a clinical-stage first-in-class, dual PIM/FLT3 kinase inhibitor. Preclinical data suggests therapeutic potential in both hematological malignancies and in solid tumors.
Based on strong activity data generated in diffuse large B-cell lymphoma (DLBCL) preclinical models, MEN1703 is being evaluated in combination with standard-of-care therapy and as a single agent in relapsed/refractory DLBCL patients (Phase II trial).
In parallel the preclinical characterization of MEN1703 will continue in other hematologic indications.

Orphan Drug Designation

MEN2312 is a potent and selective orally bioavailable KAT6A/B inhibitor. KAT6A/B belong to the MYST family of acetyltransferases (HAT) that acetylates histone H3K23 and also non-histone proteins, such as p53 exerting an oncogenic role in several tumor types, including breast cancer, where KAT6A is frequently amplified/overexpressed. Inhibiting KAT6A, MEN2312 blocks the Estrogen receptor (ER) at the transcriptional level, giving it the potential to overcome resistance to endocrine therapies due to mutation or ligand-independent constitutive activation of ER. MEN2312 is being tested as single agent and in combination with SoC in metastatic Breast Cancer ER+/HER- patients.

Meropenem + Vaborbactam is a fixed dose combination of a carbapenem and a novel boronic acid β-lactamase inhibitor of class A and class C serine β-lactamases, that was specifically developed to inhibit Carbapenem-Resistant Enterobacterales (CRE), including Klebsiella Pneumoniae Carbapenemase (KPC)-producing bacteria. Meropenem exerts bactericidal activity by inhibiting peptidoglycan cell wall synthesis as a result of binding to and inhibition of activity of essential penicillin-binding proteins (PBPs). Vaborbactam forms covalent adduct with β-lactamases and is stable to β-lactamase-mediated hydrolysis.

Delafloxacin is an anionic fluoroquinolone (FQ), with improved characteristics and safety profile in comparison to other FQs It acts through a balanced inhibition of bacterial topoisomerase IV and DNA gyrase (topoisomerase II), enzymes required for bacterial DNA replication, transcription, repair, and recombination. Delafloxacin has been approved in the US and in Europe for the treatment of the Community Acquired Bacterial Pneumonia (CABP). 

Oritavancin is a semisynthetic glycopeptidic antibiotic with a long half-life allowing the administration as single dose (1200 mg) by intravenous infusion over 3 hours. It has three mechanisms of action: (i) inhibition of the transglycosylation (polymerization) step of cell wall biosynthesis by binding to the stem peptide of peptidoglycan precursors; (ii) inhibition of the transpeptidation (crosslinking) step of cell wall biosynthesis by binding to the peptide bridging segments of the cell wall; and (iii) disruption of bacterial membrane integrity, leading to depolarization, permeabilization, and rapid cell death. Menarini developed a new Oritavancin formulation to favor the reconstitution of the medical solution and to be administered in shorter times.

New formulation

Cefepime + Taniborbactam is a combination of a carbapenem and a β-lactamases, that is under development specifically against difficult-to-treat drug resistant gram-negative bacteria, most notably carbapenem-resistant Enterobacterales (CRE) and carbapenem-resistant Pseudomonas aeruginosa (CRPA).
Cefepime is a fourth-generation cephalosporin with a well proven safety and efficacy profile against Gram positive and negative bacteria. Taniborbactam is a beta-lactamse inhibitor that increases the antimicrobial activity of cefepime against resistant and multi-resistant bacteria.
Cefepime-Taniborbactam completed the clinical investigation in complicated Urinary Tract Infection, including acute pyelonephritis; additional clinical investigation will include hospital acquired pneumonia, including ventilator associated pneumonia

Meropenem + Vaborbactam is a fixed dose combination of a carbapenem and a novel boronic acid β-lactamase inhibitor of class A and class C serine β-lactamases, that was specifically developed to inhibit Carbapenem-Resistant Enterobacterales (CRE), including Klebsiella Pneumoniae Carbapenemase (KPC)-producing bacteria. Meropenem exerts bactericidal activity by inhibiting peptidoglycan cell wall synthesis as a result of binding to and inhibition of activity of essential penicillin-binding proteins (PBPs). Vaborbactam forms covalent adduct with β-lactamases and is stable to β-lactamase-mediated hydrolysis.

Oritavancin is a semisynthetic glycopeptidic antibiotic with a long half-life allowing the administration as single dose (1200 mg) by intravenous infusion over 3 hours. It has three mechanisms of action: (i) inhibition of the transglycosylation (polymerization) step of cell wall biosynthesis by binding to the stem peptide of peptidoglycan precursors; (ii) inhibition of the transpeptidation (crosslinking) step of cell wall biosynthesis by binding to the peptide bridging segments of the cell wall; and (iii) disruption of bacterial membrane integrity, leading to depolarization, permeabilization, and rapid cell death. Menarini developed a new Oritavancin formulation to favor the reconstitution of the medical solution and to be administered in shorter times.

New formulation

Delafloxacin is an anionic fluoroquinolone (FQ), with improved characteristics and safety profile in comparison to other FQs It acts through a balanced inhibition of bacterial topoisomerase IV and DNA gyrase (topoisomerase II), enzymes required for bacterial DNA replication, transcription, repair, and recombination. Delafloxacin has been approved in the US and in Europe for the treatment of the Community Acquired Bacterial Pneumonia (CABP).