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Last update 24 Oct 2025
BMS-986263
Last update 24 Oct 2025
Overview
Basic Info
Drug Type siRNA |
Synonyms BMS 986263, ND-L02-S0201 |
Target |
Action inhibitors |
Mechanism SERPINH1 gene inhibitors |
Therapeutic Areas |
Active Indication- |
Inactive Indication |
Originator Organization |
Active Organization- |
Inactive Organization |
License Organization |
Drug Highest PhaseDiscontinuedPhase 2 |
First Approval Date- |
Regulation- |
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Structure/Sequence
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Related
9
Clinical Trials associated with BMS-986263NCT04267393
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multiple-Dose Phase 2 Study to Evaluate the Efficacy and Safety of BMS-986263 in Adults With Compensated Cirrhosis From Nonalcoholic Steatohepatitis (NASH)
NCT04225936
An Open-label, Two-Part, Single-dose Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of BMS-986263 in Participants With Varying Degrees of Hepatic Impairment
NCT03538301
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Biological Activity, and PK of ND-L02-s0201 in Subjects With Idiopathic Pulmonary Fibrosis (IPF)
100 Clinical Results associated with BMS-986263
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100 Translational Medicine associated with BMS-986263
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100 Patents (Medical) associated with BMS-986263
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3
Literatures (Medical) associated with BMS-98626301 Oct 2023Clinical and translational science
Pharmacokinetics, safety, and tolerability of BMS-986263, a lipid nanoparticle containing HSP47 siRNA, in participants with hepatic impairment.
Article
Author: Charles, Edgar D ; de Oliveira, Claudia H M C ; Lawitz, Eric J ; Wetherington, Jeffrey ; Cizza, Giovanni ; Qosa, Hisham ; Colletti, Nicholas ; Tirucherai, Giridhar S
BMS-986263 is a retinoid-conjugated lipid nanoparticle delivering small interfering RNA designed to inhibit synthesis of HSP47 protein, a collagen-specific chaperone protein involved in fibrosis development. This is a phase I, open-label, two-part study evaluating pharmacokinetics and safety of BMS-986263 in participants with hepatic impairment (HI). Part 1 (n = 24) of this study enrolled two cohorts with mild and moderate HI and a separate cohort of age- and body mass index (BMI)-matched participants with normal hepatic function. Part 2 enrolled eight participants with severe HI and eight age- and BMI-matched participants with normal hepatic function. All participants received a single intravenous 90 mg BMS-986263 infusion. Compared with normal-matched participants, geometric mean area under the plasma concentration-time curve time zero to the time of the last quantifiable concentration (AUC(0-T) ) and AUC from zero to infinity (AUC(INF) ) of HSP47 siRNA were similar in participants with mild HI and 34% and 163% greater in those with moderate and severe HI, respectively, whereas the maximum plasma concentration was ~25% lower in mild and moderate HI groups but 58% higher in the severe HI group than in the normal group. Adverse events were reported by two of eight, four of eight, and three of eight participants with mild, moderate, or severe HI, respectively; none were reported in the normal-matched group. Overall, single-dose BMS-986263 was generally safe and well-tolerated and dose adjustment is not considered necessary for participants with mild or moderate HI. Although available data do not indicate that dose adjustment should be performed in patients with severe HI; the optimal posology of BMS-986263 in patients with severe HI may be determined later in its clinical development when additional data to establish exposure-safety/efficacy relationship becomes available.
01 Apr 2021ERJ open research
Anti-HSP47 siRNA lipid nanoparticle ND-L02-s0201 reverses interstitial pulmonary fibrosis in preclinical rat models
Article
Author: Zabludoff, Sonya ; Quimbo, Alistair ; Ying, Wenbin ; Clamme, Jean-Pierre ; Liu, Jian ; Yao, Jiping ; Liu, Yun ; Zhang, Jun ; Xia, Fengcheng
ND-L02-s0201 is a lipid nanoparticle encapsulating an siRNA which inhibits expression of heat shock protein 47 (HSP47), a collagen-specific chaperone. Accumulated evidence demonstrates a close association between increased level of HSP47 and excessive accumulation of collagen in fibrotic diseases. Our objective was to test ND-L02-s0201 efficacy in preclinical lung fibrosis models and characterise the downstream histological and functional consequences of inhibiting the expression of HSP47.Comprehensive optimisation and characterisation of bleomycin (BLM) and silica-induced rat lung fibrosis models were conducted, which ensured progressive pathological changes were sustained throughout the study during evaluation of the anti-fibrotic potential of ND-L02-s0201.In the BLM model, we demonstrated dose-dependent and statistically significant reduction in the relative lung weight, collagen deposition and histology, and fibrosis scores following ND-L02-s0201 treatment. Lung tissue mRNA profiling demonstrated that 11 out of 84 fibrosis-relevant genes were upregulated following BLM induction and were downregulated by approximately 4.5-fold following ND-L02-s0201 treatment. Epithelial–mesenchymal transition was characterised in the BLM model following ND-L02-s0201 treatment. Cell enrichment demonstrated that myofibroblasts contained the highest HSP47 mRNA expression. BLM led to more than a five-fold increase in myofibroblasts and ND-L02-s0201 treatment reduced the myofibroblasts to sham levels. Statistically significant improvement in lung function was noted in the BLM model which was determined by running endurance capacity using a 7-minute treadmill test. Comparable anti-fibrotic efficacy was also observed in the silica model.Results from two robust chronic rodent models of pulmonary fibrosis demonstrated significant anti-fibrotic effects and improved lung function which support the evaluation of ND-L02-s0201 in subjects with idiopathic pulmonary fibrosis.
01 Sep 2019AAPS Journal
A Fit-for-Purpose Method for the Detection of Human Antibodies to Surface-Exposed Components of BMS-986263, a Lipid Nanoparticle-Based Drug Product Containing a siRNA Drug Substance
Article
Author: Kavita, Uma ; Ji, Qin C ; Pillutla, Renuka C ; Miller, Wendy
ND-L02-s0201/BMS-986263 is a lipid nanoparticle (LNP) drug product containing a heat shock protein 47 (HSP47)-specific small interfering ribonucleic acid (siRNA) and being developed for the treatment of liver and idiopathic pulmonary fibrosis. To address immunogenicity-related issues, we developed a robust, fit-for-purpose (FFP) three-tier electrochemiluminescent (ECL) anti-drug antibody (ADA) assay for the detection of antibodies (Abs) generated to surface-exposed components of BMS-986263. The drug was coated directly on plates, and several Abs specific for polyethylene glycol (PEG) and other surface components were tested for use as positive quality controls (QCs). Following selection of a rabbit monoclonal anti-PEG Ab, the assay was optimized, and various method development challenges specific to the modality and pseudo surrogate rabbit control were addressed. Screening, confirmatory, and titer cut points were validated following a statistical evaluation of 41 individual K2EDTA human plasma samples at a minimum required dilution (MRD) of 100. Assay precision, sensitivity, selectivity, drug tolerance, and hook effect were determined for the rabbit Ab prepared in human K2EDTA plasma matrix. The assay was used to interrogate anti-drug Ab (ADA) responses in normal human subjects who were administered 90 mg of the drug intravenously (IV) once every week for 3 weeks in phase I clinical trials. All pre- and post-dose samples were found to be negative for ADA. Based on these results, we concluded that BMS-986263 is not immunogenic. To the best of our knowledge, this work represents the first ADA method developed and reported for an LNP-based drug product.
5
News (Medical) associated with BMS-98626303 Jan 2024
AcquisitionPhase 2
14 Sep 2023
Phase 1Phase 2PROTACsClinical Trial TerminationLicense out/in
25 May 2023
100 Deals associated with BMS-986263
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R&D Status
10 top R&D records. to view more data
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Metabolic Dysfunction Associated Steatohepatitis | Phase 2 | United States | 17 Mar 2021 | |
| Metabolic Dysfunction Associated Steatohepatitis | Phase 2 | Japan | 17 Mar 2021 | |
| Metabolic Dysfunction Associated Steatohepatitis | Phase 2 | Argentina | 17 Mar 2021 | |
| Metabolic Dysfunction Associated Steatohepatitis | Phase 2 | Belgium | 17 Mar 2021 | |
| Metabolic Dysfunction Associated Steatohepatitis | Phase 2 | Brazil | 17 Mar 2021 | |
| Metabolic Dysfunction Associated Steatohepatitis | Phase 2 | Canada | 17 Mar 2021 | |
| Metabolic Dysfunction Associated Steatohepatitis | Phase 2 | France | 17 Mar 2021 | |
| Metabolic Dysfunction Associated Steatohepatitis | Phase 2 | Germany | 17 Mar 2021 | |
| Metabolic Dysfunction Associated Steatohepatitis | Phase 2 | Israel | 17 Mar 2021 | |
| Metabolic Dysfunction Associated Steatohepatitis | Phase 2 | Italy | 17 Mar 2021 |
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Clinical Result
Clinical Result
Indication
Phase
Evaluation
View All Results
Phase 2 | 124 | Placebo | ruasmnkxnm = ldnudzixst rufrlazkca (kpxabuowmd, qhoskdiaei - evqctdchoq) View more | - | 04 Sep 2024 | ||
Phase 2 | 123 | Other: Placebo | pmijokrkcg = cwimkcoxgs jwzjuospfn (ysnofrdexn, zcoqzczwxa - ubkulbnavt) View more | - | 11 Dec 2023 | ||
Phase 1 | 8 | cruotadxci(ztmgdjujra) = 1 in BMS-986263, 0 in normal-matched jipxchdasq (nsygiyibzq ) View more | Positive | 25 Jun 2022 | |||
Phase 2 | 61 | (BMS-986263 45 mg QW (Once Weekly)) | skskhqjamc = rjfdkbmzkj jtyoeerqwx (aajzmzduwe, msmiqvywko - conzlgvquj) View more | - | 04 Feb 2022 | ||
(BMS-986263 90 mg QW (Once Weekly)) | skskhqjamc = ukynyqkjsm jtyoeerqwx (aajzmzduwe, nqhgqiqodq - uumultaaol) View more | ||||||
NCT03420768 (Pubmed) Manual  | Phase 2 | 61 | llafxtfnde(dyegbakmth) = tvbnhtigjv eqibjtgrgl (hdggfazwtv ) View more | Positive | 04 Oct 2021 | ||
Placebo | llafxtfnde(dyegbakmth) = nzyldhibya eqibjtgrgl (hdggfazwtv ) |
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