[Translation] A multicenter, open-label, single-arm phase II clinical trial evaluating oral infigratinib monotherapy in patients with locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma with fibroblast growth factor receptor 2 (FGFR2) gene amplification

主要目的：评价Infigratinib单药治疗伴有FGFR2基因扩增的GC/GEJ患者的有效性次要目的：评价Infigratinib单药治疗伴有FGFR2基因扩增的GC/GEJ患者的有效性；评价Infigratinib单药治疗伴有FGFR2基因扩增的GC/GEJ患者的安全性和耐受性描述在伴有FGFR2基因扩增的GC/GEJ患者中Infigratinib和活性代谢产物的药代动力学（PK）特征探索性目的： 1.探索采用不同检测方法检测患者肿瘤组织中FGFR2基因扩增状态的结果。 2.在接受至少一次Infigratinib治疗的患者中，探索肿瘤生物标志物的变化及其与有效性和/或耐药性的潜在关系。

[Translation]

Primary purpose: To evaluate the efficacy of Infigratinib monotherapy in patients with GC/GEJ with FGFR2 gene amplification Secondary purpose: To evaluate the efficacy of Infigratinib monotherapy in patients with GC/GEJ with FGFR2 gene amplification; To evaluate the safety and tolerability of Infigratinib monotherapy in patients with GC/GEJ with FGFR2 gene amplification; To describe the pharmacokinetic (PK) characteristics of Infigratinib and active metabolites in patients with GC/GEJ with FGFR2 gene amplification Exploratory purpose: 1. To explore the results of different detection methods for detecting the FGFR2 gene amplification status in patient tumor tissues. 2. To explore the changes in tumor biomarkers and their potential relationship with efficacy and/or resistance in patients who have received at least one Infigratinib treatment.

Start Date07 Nov 2023

Sponsor / Collaborator

Alcami Carolinas Corp.

[+2]

CTR20232013 / TerminatedPhase 1

一项评估BBP-398联合奥希替尼治疗EGFR突变的局部晚期或转移性NSCLC患者的有效性和安全性的开放标签、非随机、多队列、多中心的Ia/Ib 期临床研究

[Translation] An open-label, non-randomized, multi-cohort, multi-center Phase Ia/Ib clinical study to evaluate the efficacy and safety of BBP-398 combined with osimertinib in the treatment of patients with locally advanced or metastatic NSCLC with EGFR mutations

Ia 期的目的是评估BBP-398 联合奥希替尼治疗在既往接受第三代EGFR TKIs后出现EGFR 突变的局部晚期或转移性非小细胞肺癌受试者的安全性、耐受性、药代动力学和抗癌活性。 Ib 的目的是研究BBP-398 联合奥希替尼治疗伴有EGFR突变的局部晚期或转移性NSCLC 受试者的疗效和安全性。

[Translation]

The purpose of Phase Ia is to evaluate the safety, tolerability, pharmacokinetics, and anticancer activity of BBP-398 combined with osimertinib in subjects with locally advanced or metastatic NSCLC who have EGFR mutations after previous treatment with third-generation EGFR TKIs. The purpose of Phase Ib is to study the efficacy and safety of BBP-398 combined with osimertinib in subjects with locally advanced or metastatic NSCLC who have EGFR mutations.

Start Date12 Jul 2023

Sponsor / Collaborator

Shanghai Lianbio Development Co., Ltd.

[+2]

CTR20222749 / TerminatedPhase 1

评价BBP-398 在中国晚期实体瘤受试者中的安全性、耐受性、药代动力学和初步抗肿瘤活性的I 期临床试验

[Translation] A Phase I clinical trial to evaluate the safety, tolerability, pharmacokinetics and preliminary anti-tumor activity of BBP-398 in Chinese subjects with advanced solid tumors

主要目的： -A部分（剂量递增）：评估BBP-398（SHP2抑制剂）在中国晚期实体瘤受试者中的安全性和耐受性；评估BBP-398在中国晚期实体瘤受试者中的最大耐受剂量（MTD）和/或II期推荐剂量（RP2D）。 -B部分（剂量扩展）：评估BBP-398（MTD或RP2D）在NSCLC受试者中的初步抗肿瘤活性。次要目的： -A部分（剂量递增）：评估中国晚期实体瘤受试者接受单/多次BBP-398给药后血浆和尿液的药代动力学（PK）特征；研究者评估BBP-398在中国晚期实体瘤受试者中的初步抗肿瘤活性。 -B部分（剂量扩展）：TEAE和SAE的发生率及严重程度；

[Translation]

Primary objectives: -Part A (dose escalation): evaluate the safety and tolerability of BBP-398 (SHP2 inhibitor) in Chinese subjects with advanced solid tumors; evaluate the maximum tolerated dose (MTD) and/or Phase II recommended dose (RP2D) of BBP-398 in Chinese subjects with advanced solid tumors. -Part B (dose expansion): evaluate the preliminary anti-tumor activity of BBP-398 (MTD or RP2D) in NSCLC subjects. Secondary objectives: -Part A (dose escalation): evaluate the pharmacokinetic (PK) characteristics of plasma and urine in Chinese subjects with advanced solid tumors after single/multiple administrations of BBP-398; investigators evaluate the preliminary anti-tumor activity of BBP-398 in Chinese subjects with advanced solid tumors. -Part B (dose expansion): incidence and severity of TEAEs and SAEs;

Start Date18 Oct 2022

Sponsor / Collaborator

Shanghai Lianbio Development Co., Ltd.

[+2]

100 Clinical Results associated with Shanghai Lianbio Development Co., Ltd.

0 Patents (Medical) associated with Shanghai Lianbio Development Co., Ltd.

Literatures (Medical) associated with Shanghai Lianbio Development Co., Ltd.

01 Oct 2023·JAMA Cardiology

Effect of Mavacamten on Chinese Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy

Article

Author: Ma, Wei ; Tian, Zhuang ; Zhang, Qing ; Jin, Wei ; Zhang, Shuyang ; Lyu, Cheng ; Li, Zhanquan ; Cheng, Xiang ; Wang, Fang ; Li, Xiaoyan ; Sun, Jing ; Fu, Yiqun ; Wang, Jian'an ; Yang, Ping ; Li, Liwen ; Peng, Daoquan

ImportanceMavacamten has shown clinical benefits in global studies for patients with obstructive hypertrophic cardiomyopathy (oHCM), but evidence in the Asian population is lacking.ObjectiveTo evaluate the safety and efficacy of mavacamten compared with placebo for Chinese patients with symptomatic oHCM.Design, Setting, and ParticipantsThis phase 3, randomized, double-blind, placebo-controlled clinical trial was conducted at 12 hospitals in China. Between January 4 and August 5, 2022, patients with oHCM and a left ventricular outflow tract (LVOT) gradient of 50 mm Hg or more and New York Heart Association (NYHA) class II or III symptoms were enrolled and received treatment for 30 weeks.InterventionsPatients were randomized 2:1 to receive mavacamten (starting at 2.5 mg once daily) or placebo for 30 weeks.Main Outcomes and MeasuresThe primary end point was change in Valsalva LVOT peak gradient from baseline to week 30. Left ventricular outflow tract gradients and left ventricular ejection fraction (LVEF) were assessed by echocardiography, while left ventricular mass index (LVMI) was determined by cardiac magnetic resonance imaging. Analysis was performed on an intention-to-treat basis.ResultsA total of 81 patients (mean [SD] age, 51.9 [11.9] years; 58 men [71.6%]) were randomized. Mavacamten demonstrated a significant improvement in the primary end point compared with placebo (least-squares mean [LSM] difference, −70.3 mm Hg; 95% CI, −89.6 to −50.9 mm Hg; 1-sided P &lt; .001). Similar trends were demonstrated for resting LVOT peak gradient (LSM difference, −55.0 mm Hg; 95% CI, −69.1 to −40.9 mm Hg). At week 30, more patients receiving mavacamten than placebo achieved a Valsalva LVOT peak gradient less than 30 mm Hg (48.1% [26 of 54] vs 3.7% [1 of 27]), less than 50 mm Hg (59.3% [32 of 54] vs 7.4% [2 of 27]), and NYHA class improvement (59.3% [32 of 54] vs 14.8% [4 of 27]). Greater improvements were also observed with mavacamten regarding the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (LSM difference, 10.2; 95% CI, 4.4-16.1), N-terminal pro-B-type natriuretic peptide level (proportion of geometric mean ratio, 0.18; 95% CI, 0.13-0.24), high-sensitivity cardiac troponin I level (proportion of geometric mean ratio, 0.34; 95% CI, 0.27-0.42), and LVMI (mean difference, −30.8 g/m2; 95% CI, −41.6 to −20.1 g/m2). Safety and tolerability were similar between mavacamten and placebo. No patients experienced LVEF less than 50%.ConclusionsMavacamten significantly improved Valsalva LVOT gradient vs placebo for Chinese patients. All secondary efficacy end points were also improved. Mavacamten was well tolerated with no new safety signals. This study supports the efficacy and safety of mavacamten in diverse populations, including Chinese patients.Trial RegistrationClinicalTrials.gov Identifier: NCT05174416

01 Jun 2023·BMJ Open

Study design and rationale of EXPLORER-CN: a phase III, randomised, double-blind, placebo-controlled clinical study to evaluate the efficacy and safety of mavacamten in Chinese adults with symptomatic obstructive hypertrophic cardiomyopathy

Article

Author: Wang, Fang ; Sun, Jing ; Han, Yaling ; Tian, Zhuang ; Zhou, Jingmin ; Hsu, Peiwen ; Zhang, Qing ; Zhang, Shuyang ; Yang, Ping ; Jin, Wei ; Wang, Geng

IntroductionHypertrophic cardiomyopathy (HCM) is a primary myocardial disease commonly caused by pathogenic genetic variants encoding sarcomere proteins. Mavacamten, a first-in-class allosteric inhibitor of cardiac-specific myosin, has demonstrated efficacy and safety in international clinical trials of patients with symptomatic obstructive HCM (oHCM) but clinical evidence for mavacamten in the Chinese population is lacking.Methods and analysisEXPLORER-CN is a multicentre, phase III, randomised, double-blind, placebo-controlled registration trial to evaluate the efficacy and safety of mavacamten in Chinese adults with symptomatic oHCM. The study will enrol approximately 81 participants with symptomatic oHCM. Eligible participants are randomised 2:1 to receive once-daily, oral mavacamten (starting dose 2.5 mg/day), or matching placebo, for 30 weeks, followed by a long-term extension (LTE) period of 48 weeks with active treatment for all subjects. The mavacamten dose will be adjusted by pharmacokinetic (PK)/pharmacodynamic (PD) parameters during the double-blinded, placebo-controlled period and PD-only during the LTE period. The primary efficacy endpoint is change from baseline to week 30 in Valsalva left ventricular outflow tract (LVOT) peak gradient determined by Doppler echocardiography. Secondary efficacy endpoints are change in resting LVOT peak gradient, proportion of participants achieving a Valsalva LVOT peak gradient <30 or < 50 mm Hg, New York Heart Association functional class improvement, change in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score, cardiac biomarkers and left ventricular mass index evaluated by cardiac magnetic resonance. LTE endpoints will characterise the long-term safety and efficacy of mavacamten.Ethics and disseminationThis clinical study has been approved by the Drug Clinical Trial Ethics Committee of the Chinese Academy of Medical Sciences & Peking Union Medical College Hospital (reference number: HS2021089). Written informed consent will be obtained from each participant. The results will be published in peer-reviewed journals and presented during national and international conferences.Trial registration numberNCT05174416.

Focus of Regulatory Concerns Regarding Pharmacometric Review in the Development and Evaluation of Antimicrobial Agents

Author: Qiu, Weiyi ; Yang, Dong ; Bian, Xingchen ; Lyu, Cheng ; Hu, Zijian ; Zhang, Jing ; Li, Xin ; Huang, Zhiwei ; Chen, Yuancheng ; Zhao, Ming

Pharmacometrics is an emerging science that interprets drug, disease and trial information in a mathematical fashion to inform and facilitate efficient drug development and/or regulatory decisions. Pharmacometric study is increasingly adopted in the regulatory review of new antimicrobial agents. We summarized and reviewed the 31 antimicrobial agents approved by the US Food and Drug Administration (FDA) and the 26 antimicrobial agents approved by European Medicines Agency (EMA) from January 2001 to May 2019. Regulatory concerns in pharmacometric review of antimicrobial agents were expounded with examples, including modeling and simulation methods, effects of internal/external factors on PK parameters, safety and efficacy evaluation in terms of exposure-response analysis, refinement of the wording of product labeling and package leaflet, and possible post-marketing clinical trial. Ongoing communication among regulators, academia, and industry regarding pharmacometrics is encouraged to streamline and facilitate the development of new antimicrobial agents. The industry can maximize its benefit in drug development through continued pharmacometric education/training.

100 Deals associated with Shanghai Lianbio Development Co., Ltd.

100 Translational Medicine associated with Shanghai Lianbio Development Co., Ltd.

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Pipeline

Pipeline Snapshot as of 07 Nov 2024

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

Phase 1 Clinical

Phase 2 Clinical

Phase 3 Clinical

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Current Projects

Drug(Targets)	Indications	Global Highest Phase

Mavacamten ( Cardiac myosin )	Hypertrophic obstructive cardiomyopathy More	Approved
Hafnium oxide nanoparticles	Locally Advanced Head and Neck Squamous Cell Carcinoma More	Phase 3
Infigratinib Phosphate ( FGFR1 x FGFR2 x FGFR3 x FGFR4 )	FGFR2 positive Gastrooesophageal Junction Cancer More	Phase 2
Mometasone Furoate ( GR )	Chronic sinusitis More	Phase 2
Omilancor ( LANCL2 )	Crohn Disease More	Phase 2

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