Product Pipeline - Stemline Therapeutics

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	Stage	Additional Detail	External Links
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.	Review prescribing information Important Safety Information MECHANISM OF ACTION
		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.
		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.
		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.
ELACESTRANT	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 in 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.	Review Prescribing Information
		Breast cancer monotherapy	Phase 2	ELCIN Trial is an open-label multicenter Ph 2 trials of Elacestrant in women and men with CDK4/6 inhibitor naïve ER+/HER2- metastatic breast cancer. The study investigates elacestrant as single agent without prior CDK4/6i exposure
		Breast cancer combination	Phase 2	ELECTRA Trial is An 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 is a Global Study with the aim of determining the activity of elacestrant in combination with Abemaciclib in BC EH+/HER2- with brain metastases
		Breast cancer combination	Phase 2	ELEVATE Trial is A Phase 1b/2, Open-Label Umbrella Study to Evaluate Safety and Efficacy of Elacestrant in various combination in patients with ER+/HER2- metastatic Breast Cancer. ELEVATE is a Global Study which the aim of determining safety and efficacy of combining elacestrant with other agents in the metastatic setting
NEXPOVIO® (selinexor)	XPO1 inhibitor	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. US – Fast Track Approval and Orphan Drug Designation NEXPOVIO® was approved in Europe on March 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 Important Safety Information
SL-701	Immunotherapy (IL-13Rα2, EphA2, Survivin)	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.
MEN1703	SME (PIM/FLT3i)	Acute myeloid leukemia	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 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 is continued in other hematologic and solid tumors indications.
MEN1611	SME (PI3Ki)	Colorectal cancer	Phase 2	MEN1611 is a potent and selective phosphatidylinositol 3-kinase (PI3K) inhibitor with a novel chemical structure targeting the α, β and γ isoforms while sparing the δ, with a novel chemical structure. The PI3K/Akt pathway regulates various cellular processes such as proliferation, growth, apoptosis and cytoskeletal rearrangement. Various types of cancers are thought to be “addicted” to PI3K/Akt pathway signaling and therefore inhibition of PI3K is a potent therapeutic strategy for fighting cancer. MEN1611 is being investigated as a potential treatment for patients with PIK3CA mutated, K-RAS, N-RAS and B-RAF wild-type metastatic colorectal cancer, failing irinotecan, oxaliplatin, 5-FU and anti-EGFR containing regimens.
		Breast cancer	Phase 1b	MEN1611 is a potent and selective phosphatidylinositol 3-kinase (PI3K) inhibitor with a novel chemical structure targeting the α, β and γ isoforms while sparing the δ, with a novel chemical structure. The PI3K/Akt pathway regulates various cellular processes such as proliferation, growth, apoptosis and cytoskeletal rearrangement. Various types of cancers are thought to be “addicted” to PI3K/Akt pathway signaling and therefore inhibition of PI3K is a potent therapeutic strategy for fighting cancer. MEN1611 is being investigated as a potential treatment for patients with PIK3CA-mutated, HER2-positive, advanced or metastatic breast cancer who have failed anti-HER2 based therapy.
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.
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.
MEN2312	SME (Kat6i)	Solid Tumors	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 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+/HER2- patients.

Mission/Beliefs/Values

To build a leading biopharmaceutical company focused on improving the lives of cancer patients by developing and marketing innovative oncology therapeutics that target difficult-to-treat cancers.