SL-701 / Menarini - LARVOL DELTA

Study of Tagraxofusp for Post-Transplant Maintenance for Patients With CD 123+ AML, MF and CMML (HSCT 002) (clinicaltrials.gov) - P1 | N=44 | Recruiting | Sponsor: Karen Ballen, MD | Trial primary completion date: Jul 2024 ➔ Jul 2026

Post-transplantation • Trial primary completion date • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelofibrosis • Oncology • Transplantation • IL3RA

A Phase 2 Study of a Novel Immunotherapy SL-701 in Adults With Recurrent Glioblastoma: Exploring the Prognostic Value of Treatment-Induced CD8+CD57+ T-cells as a Marker for Survival. (SNO 2023) - P1/2 | "In this study, we present updated findings from a phase 2 clinical trial (NCT02078648) evaluating SL-701+poly-ICLC+bevacizumab, where the 12-month overall survival (OS) was 50%. These qualitative differences in the immune response, detectable as early as week 8 post treatment, may serve as biomarkers for monitoring and predicting survival. Deep sequencing of SL-701-specific T-cells is planned."

Clinical • IO biomarker • P2 data • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor • B3GAT1 • BIRC5 • CD4 • CD8 • LAMP1

Phase 2 study of a novel immunotherapy SL-701 in adults with recurrent GBM: Identification of treatment-induced CD8+CD107a+ CD57+ PD-1- memory T-cells that are associated with increased survival (SNO 2022) - P1/2 | "Here we describe an 18-color flow cytometry analysis from stage 2 of a Ph2 clinical trial of SL-701+poly-ICLC+bevacizumab (NCT02078648), in which 12-month overall survival (OS) was 50%. The final 6 core phenotypes identified activated CD4+ CD154+ SL-701-specific T-cells (5%-19%) that were enhanced 0.3- to 0.5-fold in patients with an OS < 12 months (P < 0.05), suggesting helper T-cell responses in the absence of cytotoxic T-cell responses are associated with an OS < 12 months. Deep sequencing of SL-701-specific T-cells using whole transcriptome-based molecular cytometry is planned."

Clinical • IO biomarker • P2 data • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor • B3GAT1 • BIRC5 • CD4 • CD40LG • CD8 • LAMP1 • PD-1

Study of Tagraxofusp for Post-Transplant Maintenance for Patients With CD 123+ AML, MF and CMML (HSCT 002) (clinicaltrials.gov) - P1 | N=44 | Recruiting | Sponsor: Karen Ballen, MD | Not yet recruiting ➔ Recruiting

Enrollment open • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelofibrosis • Oncology • Solid Tumor • Transplantation

Study of Tagraxofusp for Post-Transplant Maintenance for Patients With CD 123+ AML, MF and CMML (HSCT 002) (clinicaltrials.gov) - P1 | N=44 | Not yet recruiting | Sponsor: Karen Ballen, MD | Trial completion date: Jul 2026 ➔ Oct 2026 | Initiation date: Mar 2022 ➔ Jun 2022 | Trial primary completion date: Apr 2024 ➔ Jul 2024

Trial completion date • Trial initiation date • Trial primary completion date • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Cervical Cancer • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelofibrosis • Oncology • Solid Tumor • Transplantation

Study of Tagraxofusp for Post-Transplant Maintenance for Patients With CD 123+ AML, MF and CMML (HSCT 002) (clinicaltrials.gov) - P1 | N=44 | Not yet recruiting | Sponsor: Karen Ballen, MD

New P1 trial • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelofibrosis • Oncology • Transplantation

Phase 2 study of SL-701, a novel immunotherapy, in adults with recurrent GBM: A high parameter flow cytometry analysis of CD8+ T cells and potential implications for patient enrichment strategies (SNO 2021) - P1/2 | "In stage 2 (SL-701 + bevacizumab + poly-ICLC) the overall survival at 12 months was 50%. By week 24, SL-701-induced target-specific CD8+ T cells expressing IFNg were detected in 8 of 27 patients (30%) who had sufficient samples, with co-expression of PD-1, TIM3, and LAG3 detected in 4 patients. To further understand the T-cell response to SL-701, deep sequencing of target-specific CD8+ T cells using whole transcriptome-based molecular cytometry and high parameter (25+ color) flow cytometry is currently underway and updated data will be reported."

Clinical • IO biomarker • P2 data • Patient Enrichment • Brain Cancer • Glioblastoma • Oncology • Solid Tumor • BIRC5 • CD8 • HAVCR2 • IFNG • LAG3 • PD-1

Phase 2 trial of SL-701 in relapsed/refractory (r/r) glioblastoma (GBM): Correlation of immune response with longer-term survival. (ASCO 2018) - P1/2; "... Patients with r/r GBM HLA-A2+, bevacizumab (bev)-naive and KPS > 60, enrolled. Stage 1: SL-701 with adjuvants GM-CSF and imiquimod dosed biweekly for 6 months, then q28 days. Stage 2: SL-701 with adjuvant poly-ICLC dosed biweekly with bev (10 mg/kg) for 6 months, then q28 days... SL-701 with immunostimulants, alone and in combination with bev, was well-tolerated and demonstrated anti-tumor activity including multiple major responses. There is also a preliminarily promising survival tail in r/r GBM patients who received SL-701 with bev Further analyses ongoing; updated data to be presented."

P2 data • Solid Tumor

Results of Phase 2 Trial of SL-701, a novel immunotherapy targeting IL-13Ra2, EphA2, and survivin, in adults with second-line recurrent glioblastoma (GBM) (ESMO 2018) - P1/2; "...In Stage 1, SL-701 was administered with adjuvants GM-CSF and imiquimod biweekly for 6 months, then q28 days. In Stage 2, SL-701 and adjuvant poly-ICLC were administered biweekly with bev (10 mg/kg) for 6 months, then q28 days... SL-701 plus adjuvants with or without bevacizumab was well-tolerated and demonstrated anti-tumor activity, including multiple major responses and a preliminarily promising survival curve, warranting further study. Updated study data will be presented."

Clinical • P2 data • Brain Cancer

Safety and Efficacy Study of SL-701, a Glioma-Associated Antigen Vaccine To Treat Recurrent Glioblastoma Multiforme (clinicaltrials.gov) - P1/2; N=76; Recruiting; Sponsor: Stemline Therapeutics, Inc.; N=100 ➔ 76

Enrollment change • Biosimilar • Oncology

Stemline Therapeutics announces oral presentation of SL-701 phase 2 data in second-line glioblastoma at the 23rd Annual Meeting of the Society of Neuro-Oncology (SNO) (GlobeNewswire) - P2, N=74; NCT02078648; Sponsor: Stemline Therapeutics; "Stemline Therapeutics...announced today that data from the Phase 2 trial of SL-701 in patients with second-line glioblastoma (GBM) were selected for oral presentation at the 23rd Annual Meeting of the Society of Neuro-Oncology (SNO) being held November 15-18, 2018 in New Orleans, LA."

P2 data • Brain Cancer • Glioblastoma • Oncology • Solid Tumor

Stemline Therapeutics announces SL-701 and SL-801 clinical data to be featured, including an oral presentation, at upcoming ESMO 2018 Congress (GlobeNewswire) - "Stemline Therapeutics...announced...that clinical data from its SL-701 and SL-801 trials have been selected for presentation, including an oral presentation, at the upcoming European Society of Medical Oncology (ESMO) Annual Congress 2018 to be held from October 19-23, in Munich, Germany. The details of the data presentations...Results of Phase II Trial Of SL-701, a Novel Immunotherapy Targeting IL-13Ra2, Epha2, and Survivin, in Adults with Second-Line Recurrent Glioblastoma (GBM)....Interim Results from a Phase 1 Trial of SL-801, a Novel XPO-1 Inhibitor, in Patients with Advanced Solid Tumors."

P1 data • P2 data • Brain Cancer • Glioblastoma • Oncology • Solid Tumor

Stemline Therapeutics reports first quarter 2019 financial results (GlobeNewswire) - “First Quarter 2019 Financial Results Review: Net revenue for ELZONRIS was $5.05 million for the quarter ended March 31, 2019; ELZONRIS™ (tagraxofusp) – Blastic plasmacytoid dendritic cell neoplasm (BPDCN): We submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in January 2019 seeking marketing approval in Europe. The MAA was granted accelerated assessment and is currently under review; Other pipeline candidates: We expect to provide periodic updates on our other product candidates, SL-701, SL-801…later this year.”

Clinical data • European regulatory • Sales