Delving into the Latest Updates on LCL-161 with Synapse

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

Target

IAP

Action

inhibitors

Mechanism

Inhibitors of apoptosis (IAP) protein family inhibitors

Therapeutic Areas

NeoplasmsSkin and Musculoskeletal DiseasesImmune System Diseases+ [6]

Active Indication-

Inactive Indication

Advanced Malignant Solid NeoplasmColorectal CancerNon-Small Cell Lung Cancer+ [6]

Originator Organization

Novartis Pharma AG

Active Organization-

Inactive Organization

Novartis Pharmaceuticals Corp.US Oncology Research LLC

License Organization-

Drug Highest PhaseDiscontinuedPhase 2

First Approval Date-

Regulation-

Login to view timeline

Structure/Sequence

Molecular FormulaC26H33FN4O3S

InChIKeyUFPFGVNKHCLJJO-SSKFGXFMSA-N

CAS Registry1005342-46-0

This trial will investigate the combination of two anti-cancer agents to treat patients with relapsed/refractory small cell lung cancer (SCLC) and ovarian cancers. Oral topotecan has US FDA approval for treating select gynecological cancers and SCLC. LCL161 is an investigational product that has been shown in clinical trials to work together with other anti-cancer agents. In this trial, investigators will determine the optimal dose of LCL161 and topotecan to administer to patients with relapsed/refractory SCLC and ovarian cancers, and examine the safety profile of the drug combination.

Start Date23 Mar 2016

Sponsor / Collaborator

SCRI Development Innovations, LLC

[+1]

100 Clinical Results associated with LCL-161

Login to view more data

100 Translational Medicine associated with LCL-161

Login to view more data

100 Patents (Medical) associated with LCL-161

Login to view more data

179

Literatures (Medical) associated with LCL-161

24 May 2025·Journal of Biomolecular Structure and Dynamics

Integration analysis of PLAUR as a sunitinib resistance and macrophage related biomarker in ccRCC, an in silicon and experimental study

Article

Author: Tian, Yijun ; Li, Lin ; Yang, Qiwei ; Jiang, Aimin ; Chu, Jian ; Gan, Sishun ; Cao, Jianwei ; Ye, Fangdie

Sunitinib remains the preferred systemic treatment option for specific patients with advanced RCC who are ineligible for immune therapy. However, it's essential to recognize that Sunitinib fails to elicit a favourable response in all patients. Moreover, most patients eventually develop resistance to Sunitinib. Therefore, identifying new targets associated with Sunitinib resistance is crucial. Utilizing multiple datasets from public cohorts, we conducted an exhaustive analysis and identified a total of 8 microRNAs and 112 mRNAs displaying significant expression differences between Sunitinib responsive and resistant groups. A particular set of six genes, specifically NIPSNAP1, STK40, SDC4, NEU1, TBC1D9, and PLAUR, were identified as highly significant via WGCNA. To delve deeper into the resistance mechanisms, we performed additional investigations using cell, molecular, and flow cytometry tests. These studies confirmed PLAUR's pivotal role in fostering Sunitinib resistance, both in vitro and in vivo. Our findings suggest that PLAUR could be a promising therapeutic target across various cancer types. In conclusion, this investigation not only uncovers vital genes and microRNAs associated with Sunitinib resistance in RCC but also introduces PLAUR as a prospective therapeutic target for diverse cancers. The outcomes contribute to advancing personalized healthcare and developing superior therapeutic strategies.

01 Mar 2025·Hematological Oncology

Managing Myelofibrosis: Matching Advances in Treatments With Clinical Unmet Needs

Review

Author: Chan, Tze Wei ; Lee, Lai Heng ; Huang, Feng‐Ju ; Billa, Gauri ; Than, Hein

ABSTRACTMyelofibrosis (MF) is characterized by anemia, constitutional symptoms, hepatosplenomegaly and bone marrow fibrosis, and is associated with poor survival. The janus kinase inhibitor (JAKi) ruxolitinib has been the mainstay of treatment for over a decade. Despite demonstrated symptomatic and quality of life improvement, unmet clinical needs persist. A literature review identified promising novel targeted treatment options in MF using pre‐set selection criteria (available Phase 2 or 3 data, minimum enrollment of 50 patients, trial end date within the last 5 years). Available data for novel and approved therapies were extracted, tabulated, and analyzed for clinical relevancy. From an initial shortlist of 48, 16 retained molecules were selected for inclusion. Other JAKi (pacritinib, momelotinib, jaktinib) address treatment‐related cytopenia, expanding the therapeutic utility of this class of agents to patients with baseline anemia or thrombocytopenia. Novel candidates exploit multiple molecular pathways, and offer the potential to improve the management of MF‐associated cytopenia (imetelstat, pelabresib, navitoclax, selinexor, luspatercept, sotatercept, elritercept, LCL161, bomedemstat) and recover bone marrow fibrosis (imetelstat, pelabresib, navitoclax and bomedemstat). It remains to be seen if these newer agents can induce any remission in MF and enable patients to come off therapy, but the future is beginning to look much brighter.

01 Mar 2025·Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease

NetSDR: Drug repurposing for cancers based on subtype-specific network modularization and perturbation analysis

Article

Author: Li, Wanshi ; Xu, Zhen ; Li, Wei ; Hu, Guang ; Yang, Bin

Cancer, a heterogeneous disease, presents significant challenges for drug development due to its complex etiology. Drug repurposing, particularly through network medicine approaches, offers a promising avenue for cancer treatment by analyzing how drugs influence cellular networks on a systemic scale. The advent of large-scale proteomics data provides new opportunities to elucidate regulatory mechanisms specific to cancer subtypes. Herein, we present NetSDR, a Network-based Subtype-specific Drug Repurposing framework for prioritizing repurposed drugs specific to certain cancer subtypes, guided by subtype-specific proteomic signatures and network perturbations. First, by integrating cancer subtype information into a network-based method, we developed a pipeline to recognize subtype-specific functional modules. Next, we conducted drug response analysis for each module to identify the "therapeutic module" and then used deep learning to construct weighted drug response network for the particular subtype. Finally, we employed a perturbation response scanning-based drug repurposing method, which incorporates dynamic information, to facilitate the prioritization of candidate drugs. Applying the framework to gastric cancer, we attested the significance of the extracellular matrix module in treatment strategies and discovered a promising potential drug target, LAMB2, as well as a series of possible repurposed drugs. This study demonstrates a systems biology framework for precise drug repurposing in cancer and other complex diseases.

100 Deals associated with LCL-161

Login to view more data

External Link

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB12085

R&D Status

10 top R&D records.

Login

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Triple Negative Breast Cancer	Phase 2	Italy	Novartis Pharmaceuticals Corp.	01 Aug 2012
Triple Negative Breast Cancer	Phase 2	Germany	Novartis Pharmaceuticals Corp.	01 Aug 2012
Triple Negative Breast Cancer	Phase 2	Russia	Novartis Pharmaceuticals Corp.	01 Aug 2012
Triple Negative Breast Cancer	Phase 2	Czechia	Novartis Pharmaceuticals Corp.	01 Aug 2012
Triple Negative Breast Cancer	Phase 2	United States	Novartis Pharmaceuticals Corp.	01 Aug 2012
Triple Negative Breast Cancer	Phase 2	Spain	Novartis Pharmaceuticals Corp.	01 Aug 2012
Triple Negative Breast Cancer	Phase 2	South Korea	Novartis Pharmaceuticals Corp.	01 Aug 2012
Triple Negative Breast Cancer	Phase 2	Brazil	Novartis Pharmaceuticals Corp.	01 Aug 2012
Triple Negative Breast Cancer	Phase 2	Taiwan Province	Novartis Pharmaceuticals Corp.	01 Aug 2012
Triple Negative Breast Cancer	Phase 2	Ireland	Novartis Pharmaceuticals Corp.	01 Aug 2012

Login to view more data

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02649673 (Pubmed \| Oncologist)	Phase 1	Female Genital Neoplasms \| Small Cell Lung Cancer	35	LCL161 plus Oral Topotecan	(bdkdcfrzdn) = ygxvzmbmlb ujusspjuky (uxdohnvfuz ) View more	Negative	02 May 2023
NCT01617668 (Pubmed) Manual	Phase 2	Triple Negative Breast Cancer ER Negative \| PR Negative \| HER2 Negative	209	Paclitaxel+LCL 161	(wqqkjxlmuh) = bjuqytwier gaznolkwud (kudcinqbpv ) View more	Positive	20 Sep 2018
NCT01617668 (Pubmed) Manual	Phase 2		209	Paclitaxel	(wqqkjxlmuh) = swfjccdxwc gaznolkwud (kudcinqbpv ) View more	Positive	20 Sep 2018
NCT01617668 (ASCO2015)	Phase 2	Triple Negative Breast Cancer Neoadjuvant	209	LCL161 with paclitaxel	(cnjljhjlsf) = piacqceadb dqasfcohwg (almxtnjfkd )	-	20 May 2015
NCT01617668 (ASCO2015)	Phase 2	Triple Negative Breast Cancer Neoadjuvant	209	Paclitaxel alone	(cnjljhjlsf) = hhhqjfcwmn dqasfcohwg (almxtnjfkd )	-	20 May 2015
AACR2010	Phase 1	Advanced cancer	27	LCL161	lqsnwhssyo(dtbnporejc) = sigzibzmcd fawmgoqngp (lkweoljvtq )	-	15 Apr 2010