Our Products and Pipeline
Stemline is investing in advanced science and technology to develop a pipeline of new products that make a difference to the lives of patients affected by cancer.
Our pipeline refers to drugs in development, including those in preclinical testing, clinical development, regulatory approval and lifecycle management.
The information in the table reflects Stemline’s development pipeline and is not intended for promotional or marketing purposes. The safety and efficacy of the investigational compounds, or investigational uses of marketed products have not been established and marketing approval has yet to be granted by regulatory authorities.
| Compound | Mechanism of Action | Indication | Development Stage | Additional Detail | Clinical Trials |
|---|---|---|---|---|---|
| TAGRAXOFUSP | Targeted Biologic (CD123) | Blastic plasmacytoid dendritic cell neoplasm | Approved (US) | 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 US - 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 EU - Orphan Drug Designation | https://stemline.com/pipeline/clinical-trials/ NCT02113982 - Tagraxofusp in Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm or Acute Myeloid Leukemia |
| Acute myeloid leukemia | Phase 2 | 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. | NCT06456463 TRILLIUM: A Phase 2, Multicenter Open-label Trial of Tagraxofusp (TAG) in Combination with Venetoclax and Azacitidine (Ven/Aza) in Adults with Previously Untreated CD123+ Acute Myeloid Leukemia (AML) who are Ineligible for Intensive Chemotherapy | ||
| Chronic myelomonocytic leukemia | Phase 2 | 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. | NCT02268253 - Tagraxofusp (SL-401) in Participants With Chronic Myelomonocytic Leukemia (CMML) and Myelofibrosis (MF) | ||
| Myelofibrosis | Phase 2 | 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. | NCT02268253 - Tagraxofusp (SL-401) in Participants With Chronic Myelomonocytic Leukemia (CMML) and Myelofibrosis (MF) | ||
| ELACESTRANT | SME (SERD -ER) | Breast cancer monotherapy | Approved (US) | Elacestrant is a selective estrogen receptor degrader (SERD) evaluated for potential use as a once daily oral treatment in patients with estrogen receptor positive (ER+)/HER2- advanced or metastatic breast cancer. Studies completed to date, including the positive results from the EMERALD phase 3 trial, indicate that the compound has the potential for use as a single agent or in combination with other therapies for the treatment of breast cancer. Indeed, Elacestrant has been the first oral SERD to show positive results in a pivotal, phase III trial as a monotherapy versus standard of care for the treatment of ER+/ HER2- advanced or metastatic breast cancer. ORSERDU was approved by the U.S. FDA on January 27th, 2023 for the treatment of postmenopausal women or adult men, with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy and, by EMA on September 15th 2023 as a monotherapy for the treatment of postmenopausal women, and men, with estrogen receptor (ER)‑positive, HER2-negative, locally advanced or metastatic breast cancer (mBC) with an activating ESR1 mutation who have disease progression following at least one line of endocrine therapy including a CDK 4/6 inhibitor. Elacestrant has also been approved in other countries. | EMERALD - NCT03778931 – Phase 3 Trial of Elacestrant vs. Standard of Care for the Treatment of Patients With ER+/ HER2- Advanced Breast Cancer (EMERALD) |
| Breast cancer Combination | Phase 2 | ELECTRA Trial is a global, open-label, multicenter phase 1b/2 study of elacestrant in combination with abemaciclib in women and men with brain metastasis from estrogen receptor positive, HER-2 negative breast cancer. | ELECTRA - NCT05386108 – Combination with Abemaciclib in ER+/HER2- mBC with brain mets | ||
| Breast cancer combination | Phase 2 | ELEVATE Trial is a global, A Phase 1b/2, Oopen-Llabel, mmulticenter Phase 1b/2 Uumbrella Sstudy of Eelacestrant in various combinations in patients with ER+/HER2- metastatic Bbreast Ccancer. | ELEVATE - NCT05563220 - Combination with Alpelisib, Everolimus, Abemaciclib, Ribociclib, Palbociclib and Capivasertib) in ER+/HER2- mBC | ||
| Breast cancer Monotherapy | Phase 2 | 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 | ELCIN - NCT05596409 - Monotherapy in CDK4/6i naïve ER+/HER2- mBC | ||
| Breast cancer Combination | Phase 3 | 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 andCDK4/6 inhibitors. | ADELA- NCT06382948 -Combination with Everolimus in ER+/HER2- adv BC, ESR-1 mut. | ||
| Breast cancer Monotherapy | Phase 3 | ELEGANT tTrial is a global, mMulticenter, randomized, open label, Phase 3, study of Eelacestrant vs Sstandard Eendocrine Ttherapy in Wwomen and Mmen with Nnode positive, ER+, HER2-negative, Eearly Bbreast Ccancer with Hhigh Rrisk of Rrecurrence. | ELEGANT – NCT06492616 Monotherapy in ER+/HER2-, early BC | ||
| Breast cancer Combination | Phase 2 | 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. | SUMIT-ELA – NCT05963997 Combination with Samuraciclib in Metastatic or Locally Advanced HR+/ HER2-negative BC | ||
| NEXPOVIO® (selinexor) | SME (XPO1i) | Multiple myeloma | Approved (US) | 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 on 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 | NCT05028348 - A Study of Combination of Selinexor, Pomalidomide, and Dexamethasone (SPd) Versus Elotuzumab, Pomalidomide, and Dexamethasone (EloPd) in Subject With Previously Treated Multiple Myeloma |
| SELINEXOR | SME (XPO1ii) | Endometrial cancer | Phase 3 | 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 | NCT05611931 - Selinexor in Maintenance Therapy After Systemic Therapy for Participants With p53 Wild-Type, Advanced or Recurrent Endometrial Carcinoma (XPORT-EC-042) |
| SME (XPO1i) | Myelofibrosis | Phase 3 | 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. | NCT04562389 - Study of Selinexor in Combination with Ruxolitinib in Myelofibrosis (SENTRY) | |
| SL-701 | Immunotherapy (IL-13Rα2, EphA2, Survivin) | Recurrent Glioblastoma | Phase 2 | 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. | NCT02078648 - A Phase 1/2 Study of SL-701, a Subcutaneously Injected Multivalent Glioma-Associated Antigen Vaccine, in Adult Patients with Recurrent Glioblastoma Multiforme (GBM) |
| MEN1703 | SME (PIM/FLT3i) | DLBCL | Phase 2 | 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. US Orphan Drug Designation | NCT06534437 - Relapsed or Refractory Aggressive B-cell Non-Hodgkin Lymphoma (JASPIS-01) (JASPIS-01) |
| MEN1309 | ADC (Anti-CD205) | Solid Tumors | Phase 1b | 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. | NCT04064359 - Safety and Preliminary Efficacy of OBT076 in Recurrent/ Metastatic CD205+ Solid Tumors |
| FELEZONEXOR | SME (XPO1i) | Solid Tumors | Phase 1b | 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. | NCT02667873 – Phase 1 Trial of a Novel XPO1 Inhibitor in Patients with Advanced Solid Tumors |
| MEN2312 | SME (Kat6i) | Solid Tumors and Heme malignancies | Phase 1a | 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. | NCT06638307 - A First-in-Human Study of MEN2312 in Adults With Advanced Breast Cancer |
| MEN2501 | Solid Tumors | Solid Tumors | IND ready* | 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 | N/A |