A Phase 1b Open-label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Tinlarebant in Japanese Subjects with Stargardt Disease and a Phase 2/3 Randomized, Double-masked, and Placebo-controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Tinlarebant in Subjects with Stargardt Disease

The goal of this clinical trial is to evaluate the safety, tolerability, and efficacy of tinlarebant in subjects with Stargardt Disease

开始日期2024-07-31

申办/合作机构

Belite Bio, Inc.

CTR20241357

/ Recruiting临床3期

一项旨在探究tinlarebant治疗地图样萎缩的安全性和疗效的III期、多中心、随机、双盲、安慰剂对照研究（PHOENIX研究）

主要目的 ◆ 通过眼底自发荧光（FAF）成像评估地图样萎缩（GA）病变大小，自基线至第24个月的变化率（增长率的斜率）。关键次要目的 ◆ 采用早期治疗糖尿病视网膜病变研究（ETDRS）视力表评估最佳矫正视力（BCVA），分别在标准照度和低照度条件下测定自基线至第24个月的变化； ◆ 通过谱域光学相干断层扫描（SD-OCT）评估光感受器内/外节段连接处面积和大小（即椭圆体带[EZ]损伤），自基线至第24个月的变化； ◆ 通过SD-OCT评估视网膜平均厚度（即，外侧、中间和中央区域），自基线至第24个月的变化。

开始日期2024-07-22

申办/合作机构

倍亮生技医药（上海）有限公司

ACTRN12623001328662

/ Recruiting临床2期

An Open-Label, Single-Arm, 2-Year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Subjects with Stargardt Disease

开始日期2023-11-14

申办/合作机构

RBP4 Pty Ltd

100 项与 Tinlarebant 相关的临床结果

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100 项与 Tinlarebant 相关的转化医学

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100 项与 Tinlarebant 相关的专利（医药）

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项与 Tinlarebant 相关的文献（医药）

2021-12-01·European journal of medicinal chemistry1区 · 医学

Retinol binding protein 4 antagonists and protein synthesis inhibitors: Potential for therapeutic development

1区 · 医学

Review

作者: Kim, Noheul ; Priefer, Ronny

Retinol-binding protein 4 (RBP4) is a serum protein that transports Vitamin A. RBP4 is correlated with numerous diseases and metabolic syndromes, including insulin resistance in type 2 diabetes, cardiovascular diseases, obesity, and macular degeneration. Recently, RBP4 antagonists and protein synthesis inhibitors are under development to regulate the effect of RBP4. Several RBP4 antagonists, especially BPN-14136, have demonstrated promising safety profiles and potential therapeutic benefits in animal studies. Two RBP4 antagonists, specifically tinlarebant (Belite Bio) and STG-001 (Stargazer) are currently undergoing clinical trials. Some antidiabetic drugs and nutraceuticals have been reported to reduce RBP4 expression, but more clinical data is needed to evaluate their therapeutical benefits. As regulating RBP4 levels or its activities would benefit a wide range of patients, further research is highly recommended to develop clinically useful RBP4 antagonists or protein synthesis inhibitors.

2020-11-01·Doklady. Biochemistry and biophysics4区 · 生物学

Prostate Cancer Growth Inhibition by 1-(3,5-Dimethylphenyl)–6-methyl-1H-pyrazolo[4,3-c]pyridin-4(5H)-one via Down-regulation of Phosphorylation PI3K/AKT and STA3/JAK2

4区 · 生物学

Article

作者: Zhang, Zhenwei ; Xue, Jingxin ; Hu, Heyi

In the present study, 1-(3,5-dimethylphenyl)-6-methyl-1H-pyrazolo[4,3-c]pyridin-4 (5H)-one (DPMPP) was investigated as an antiproliferative agent for prostate cancer cells and the mechanism of its action was studied. Cell lines 22Rv1 and SGC‑7901 were used as in vitro models of prostate cancer. The DPMPP treatment inhibited proliferation of 22Rv1 and SGC‑7901 cells in dose-depended manner. The viability of 22Rv1 and SGC‑7901 cells was reduced to 21 and 19%, respectively after treatment with 32 µM DPMPP. In DPMPP treated (16 µM) 22Rv1 and SGC‑7901 cells apoptosis increased to 62.78 and 68.51%, respectively. Moreover, DPMPP treatment caused cell cycle arrest in S phase and inhibition of PI3K/AKT activation. In the same time ROS production showed elevation and MMP (Matrix MetalloProteinase) decreased in the cells. Apparently DPMPP induces cytotoxicity through induction of oxidative response and apoptosis in prostate cancer cells in vitro. The PI3K/Akt/ERK phosphorylation was inhibited, while p21 and p53, death receptor, expression was promoted by DPMPP treatment. Therefore, DPMPP has a potential to be used as a therapeutic agent for treatment of prostate cancer.

2020-05-01·European journal of medicinal chemistry

Structure-activity relationships of triazole-benzodioxine inhibitors of cathepsin X

Article

作者: Fonović, Urša Pečar ; Gobec, Stanislav ; Hrast, Martina ; Proj, Matic ; Kos, Janko ; Knez, Damijan ; Zidar, Nace

Cathepsin X is a cysteine carboxypeptidase that is involved in various physiological and pathological processes. In particular, highly elevated expression and activity of cathepsin X has been observed in cancers and neurodegenerative diseases. Previously, we identified compound Z9 (1-(2,3-dihydrobenzo[b][1,4]dioxin-6-yl)-2-((4-isopropyl-4H-1,2,4-triazol-3-yl)thio)ethan-1-one) as a potent and specific reversible cathepsin X inhibitor. Here, we have explored the effects of chemical variations to Z9 of either benzodioxine or triazol moieties, and the importance of the central ketomethylenethio linker. The ketomethylenethio linker was crucial for cathepsin X inhibition, whereas changes of the triazole heterocycle did not alter the inhibitory potencies to a greater extent. Replacement of benzodioxine moiety with substituted benzenes reduced cathepsin X inhibition. Overall, several synthesized compounds showed similar or improved inhibitory potencies against cathepsin X compared to Z9, with IC₅₀ values of 7.1 μM-13.6 μM. Additionally, 25 inhibited prostate cancer cell migration by 21%, which is under the control of cathepsin X.

项与 Tinlarebant 相关的新闻（医药）

2025-01-23

·PRNewswire

70+ Key Companies Advancing in Dry Age-Related Macular Degeneration Clinical Trial Therapeutic Space | DelveInsight

Dry macular degeneration is a common eye disorder among people aged above 50 years. It causes blurred or reduced central vision due to thinning of the macula. The macula is the part of the retina responsible for clear vision in a direct line of sight. The pharmaceutical industry is increasingly focused on developing pathway-based therapies for dry AMD, with promising treatments like ALK-001 and LBS-008 in trials, though a combination approach may be needed to fully inhibit disease progression. LAS VEGAS, Jan. 23, 2025 /PRNewswire/ -- DelveInsight's ' Dry Age-Related Macular Degeneration Pipeline Insight 2025 ' report provides comprehensive global coverage of pipeline dry AMD therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the dry AMD pipeline domain. Key Takeaways from the Dry Age-Related Macular Degeneration Pipeline Report DelveInsight's dry AMD pipeline report depicts a robust space with 70+ active players working to develop 80+ pipeline therapies for dry AMD treatment. Key dry AMD companies such as Alkeus Pharmaceuticals, Dobecure, Belite Bio, Cognition Therapeutics, Aviceda Therapeutics, Stealth BioTherapeutics, Allergo Ophthalmics, Annexon, Inc., Johnson & Johnson, InflammX Therapeutics, Lineage Cell Therapeutics, Ionis Pharmaceuticals, Evergreen Therapeutics, Inc., Alexion, Luxa Biotechnology, Astellas Pharma, OliX Pharmaceuticals, Hoffmann-La Roche, Boehringer Ingelheim, Eyevensys and others are evaluating new dry AMD drugs to improve the treatment landscape. Promising dry AMD pipeline therapies such as ALK-001, Etamsylate, Tinlarebant (LBS-008), CT1812, AVD-104, Elamipretide, Risuteganib, ANX 007, JNJ 81201887, Xiflam, OpRegen, IONIS-FB-LRx, EG-301, ALXN2040, RPESC-RPE-4W, MA09-hRPE, OLX301A, RO7303359, BI 771716, EYS 611 and others are under different phases of dry AMD clinical trials. In December 2024, Galimedix Therapeutics announced the initiation of its Phase II eDREAM study to assess the safety and efficacy of GAL-101 eye drops in patients with dry age-related macular degeneration. In December 2024, Ocugen, Inc. announced that the Data and Safety Monitoring Board (DSMB) for the OCU410 ArMaDa clinical trial recently convened and approved continuation of the second phase of the Phase 1/2 study. OCU410 (AAV5-hRORA) is a novel modifier gene therapy candidate being developed for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). In November 2024, Ocugen, Inc. announced positive preliminary efficacy and safety data from the Phase I dose-escalation portion of the Phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy (GA), secondary to dry age-related macular degeneration (dAMD). Key findings include: no drug-related serious adverse events, reduced lesion growth, preservation of retinal tissue, and most importantly there was a positive effect on the functional visual measure of low luminance visual acuity (LLVA). In October 2024, Eyestem Research, focused on creating scalable cell therapies for incurable diseases, had announced a positive outcome of the Drug Safety Monitoring Board (DSMB) review for the first cohort of its Phase one trial to treat Geographic Atrophy secondary to Dry Age-related Macular Degeneration, marking a milestone for Indian science and medical research. In September 2024, Stealth BioTherapeutics Inc. announced the presentation of new bevemipretide (SBT-272) preclinical data demonstrating topical ocular delivery to the retina with protective effects observed in models of AMD. In June 2024, Stealth BioTherapeutics Inc. announced that it has enrolled and dosed its first patient in the ReNEW trial as part of its Phase III clinical program for elamipretide in patients with dry age-related macular degeneration (dry AMD). The program consists of the two Phase III trials, ReNEW and ReGAIN. Both trials will evaluate the efficacy, safety, and pharmacokinetics of daily subcutaneous injections of elamipretide in participants with dry AMD. In April 2024, Eyestem received approval from India's Central Drugs Standards Control Organisation (CDSCO) to commence human trials for Eyecyte RPE, a potential treatment for geographic atrophy arising from dry age-related macular degeneration (AMD). Request a sample and discover the recent advances in dry AMD treatment drugs @ Dry Age-Related Macular Degeneration Pipeline Report The dry AMD pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage dry AMD drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the dry AMD clinical trial landscape. Dry Age-Related Macular Degeneration Overview Age-related macular degeneration (AMD) is one of the top four causes of blindness in older adults and the leading cause of irreversible blindness in developed countries. Around 170 million people globally are affected by AMD, making it the third most common cause of vision loss worldwide. Dry AMD typically progresses slowly, but wet AMD can cause severe visual impairment within days or weeks of onset. In the early stages of AMD, there are no noticeable symptoms, though an ophthalmologist can detect drusen before symptoms arise. Some individuals experience slight vision changes during the intermediate stage, but many remain symptom-free. Others may notice dark or grey spots in their central vision or have difficulty transitioning from bright to dim lighting. Clinicians currently use various imaging techniques to monitor changes in the retina, retinal pigment epithelium (RPE), and choroid, including fundus exams, color fundus photography, fundus autofluorescence (FAF), optical coherence tomography (OCT), and infrared reflectance imaging. Over the past two decades, OCT has significantly advanced the early detection, monitoring, and treatment assessment of dry AMD. Modern spectral-domain OCT (SD-OCT) systems provide detailed cross-sectional and volumetric views of the retina, enabling clinicians to visualize, measure, and monitor retinal layers, RPE, hyperreflective foci, geographic atrophy (GA), and drusen. While SD-OCT remains the standard of care, emerging technologies like polarization-sensitive OCT (PS-OCT), which offers tissue-selective imaging, may revolutionize AMD research and clinical practices. Currently, no approved treatments exist for dry AMD. Management involves regular monitoring, timely detection of visual deterioration, appropriate rehabilitation, and early identification of choroidal neovascularization (CNV). Research is underway to explore therapeutic strategies to prevent AMD, slow its progression, or restore vision. Potential treatments include lifestyle and behavioral changes, age-related eye disease study (AREDS) supplements, visual cycle modulators, anti-inflammatory agents, complement inhibitors, gene therapy, cell-based therapies, and approaches to reduce toxic by-products. Find out more about dry AMD treatment drugs @ Drugs for Dry Age-Related Macular Degeneration Treatment A snapshot of the Dry Age-Related Macular Degeneration Pipeline Drugs mentioned in the report: Learn more about the emerging dry AMD pipeline therapies @ Dry Age-Related Macular Degeneration Clinical Trials Dry Age-Related Macular Degeneration Therapeutics Assessment The dry AMD pipeline report proffers an integral view of the dry AMD emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Dry Age-Related Macular Degeneration Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Dimerisation inhibitors, Vitamin A replacements, RBP4 protein inhibitors, Sigma-2 receptor antagonists, Complement factor H stimulants, Macrophage inhibitors, Cell replacements, RNA interference, Key Dry Age-Related Macular Degeneration Companies: Alkeus Pharmaceuticals, Dobecure, Belite Bio, Cognition Therapeutics, Aviceda Therapeutics, Stealth BioTherapeutics, Allergo Ophthalmics, Annexon, Inc., Johnson & Johnson, InflammX Therapeutics, Lineage Cell Therapeutics, Ionis Pharmaceuticals, Evergreen Therapeutics, Inc., Alexion, Luxa Biotechnology, Astellas Pharma, OliX Pharmaceuticals, Hoffmann-La Roche, Boehringer Ingelheim, Eyevensys and others Key Dry Age-Related Macular Degeneration Pipeline Therapies: ALK-001, Etamsylate, Tinlarebant (LBS-008), CT1812, AVD-104, Elamipretide, Risuteganib, ANX 007, JNJ 81201887, Xiflam, OpRegen, IONIS-FB-LRx, EG-301, ALXN2040, RPESC-RPE-4W, MA09-hRPE, OLX301A, RO7303359, BI 771716, EYS 611 and others. Dive deep into rich insights for new drugs for dry AMD treatment, visit @ Dry Age-Related Macular Degeneration Drugs Table of Contents For further information on the dry AMD pipeline therapeutics, reach out @ Dry Age-Related Macular Degeneration Treatment Drugs Related Reports Dry Age-Related Macular Degeneration Market Dry Age-Related Macular Degeneration Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key dry AMD companies including Alkeus Pharmaceuticals, Belite Bio, Aviceda Therapeutics, Johnson & Johnson Innovative Medicine, Allegro Ophthalmics, Lineage Cell Therapeutics (CellCure Neurosciences), Roche, Cognition Therapeutics, Stealth BioTherapeutics, Evergreen Therapeutics, Annexon Biosciences, NGM Biopharmaceuticals, AstraZeneca, Alexion Pharmaceuticals, Ionis Pharmaceuticals, Novartis, Regenerative Patch Technologies, among others. Dry Age-related Macular Degeneration Epidemiology Forecast Dry Age-related Macular Degeneration Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted dry AMD epidemiology in the 7MM, i.e., the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan. Age-related Macular Degeneration Market Age-related Macular Degeneration Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key AMD companies including Regeneron Pharmaceuticals, Novartis, Roche, Opthea Limited, Kodiak Sciences Inc., REGENXBIO, Alkahest Inc, Graybug Vision, Ribomic USA Inc, Outlook Therapeutics, Inc., Unity Biotechnology, Inc, PanOptica, Inc., Clearside Biomedical, Alexion Pharmaceuticals, AstraZeneca, Evergreen Therapeutics, Alkeus Pharmaceuticals, Stealth BioTherapeutics, CellCure Neurosciences, Regenerative Patch Technologies, Allegro Ophthalmics, Annexon Biosciences, NGM Biopharmaceuticals, Ionis Pharmaceuticals, Apellis Pharmaceuticals, Iveric Bio, Gyroscope Therapeutics, Luxa Biotechnology, Gemini Therapeutics , among others. Wet Age-related Macular Degeneration Market Wet Age-related Macular Degeneration Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Wet Age-related Macular Degeneration companies including EyePoint Pharmaceuticals, Inc., AbbVie, Caregen Co. Ltd., Exegenesis Bio, Shanghai Henlius Biotech, Skyline Therapeutics, 4D Molecular Therapeutics, Ocugenix Corporation, Adverum Biotechnologies, Inc., Ashvattha Therapeutics, Inc., AiViva BioPharma, Inc., Ocular Therapeutix, Inc., Clearside Biomedical, Inc., Hoffmann-La Roche, Kyowa Kirin, Inc., Opthea Limited, AffaMed Therapeutics Limited, EyeBiotech Ltd., Novartis , among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床1期临床3期临床2期

2024-11-28

·GlobeNewswire-Health Care

Lin BioScience Receives U.S. FDA Fast Track Designation For LBS-007

Fast Track designation indicates LBS-007’s potential in filling the unmet medical need of acute leukemia LBS-007 is the Company’s investigational medicinal product for the treatment of acute leukemia A Phase 1/2 trial in patients with relapsed or resistant acute leukemias is ongoing LBS-007, the Company’s lead asset, has been granted orphan drug designations for acute myeloid leukemia and acute lymphocytic leukemia by the U.S. FDA Nov. 27, 2024 -- Lin BioScience (6696. Taiwan OTC), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting acute leukemia that have significant unmet medical needs, today announced that its lead pipeline, LBS-007, has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) for the treatment of acute myeloid leukemia. Lin BioScience is currently conducting a phase 1/2 trial in patients with relapsed or resistant acute leukemias in the US, Australia, and Taiwan, and has been granted for orphan drug designation (ODD) for acute myeloid leukemia and acute lymphocytic leukemia by the U.S. FDA. “We are thrilled to see LBS-007 showing signs of early treatment response and potential efficacy in addressing this critical unmet medical need,” said Dr. Tom Lin, Chairman of Lin BioScience. “Receiving FDA Fast Track Designation is a significant milestone, offering an expedited pathway to advance the development of this promising therapy. We remain deeply committed to delivering transformative solutions for patients in need.” “During the phase 1 dose escalation, we have not observed any Definitely or Probably Related Adverse Effects in all patients who have received low to high doses, demonstrating the safety profile is very tolerable and promising,” said Dr. Irene Wang, President & CSO of Lin BioScience. Fast Track is a process designed to facilitate the development, and expedite the review, of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious conditions. Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may be potentially better than available therapies. Once a drug receives Fast Track designation, it is eligible for more frequent meetings with the FDA, more frequent written communication from the FDA, Accelerated Approval and Priority Review if relevant criteria are met, and Rolling Review. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients. LBS-007 is a natural, non-ATP cell cycle inhibitor targeting a broad array of cancers. LBS-007 functions by blocking the kinase activity of CDC7, a key regulator of the cancer cell cycle. Inhibiting CDC7 stops the proliferation of tumor cells and results in cancer cell death. LBS-007 has demonstrated very potent activity against leukemia and multiple solid tumors in preclinical studies. Lin BioScience (6696. Taiwan OTC), founded in 2016, is a drug development company focused on unmet medical needs through developing first-in-class therapies for cancer, ophthalmology, and metabolic diseases. The Company currently has four primary drug candidates under development, including LBS-008 (for Geographic Atrophy [GA] in advanced dry age-related macular degeneration [AMD] and Stargardt Disease type 1 [STGD1]) and LBS-009 (for non-alcoholic steatohepatitis [NASH]), both developed by its subsidiary Belite Bio (NASDAQ: BLTE), as well as LBS-007 (for acute leukemia and solid tumors) and LBS-002 (for primary and metastatic brain cancers) developed by Lin BioScience. Significantly, LBS-008 has been granted Orphan Drug Designation (ODD) for the treatment of STGD1 from the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan. It has also received Fast Track Designation and Rare Pediatric Disease (RPD) designation from the U.S. FDA, along with Sakigake Designation from the Ministry of Health, Labour and Welfare (MHLW) in Japan. Additionally, LBS-007 has been granted ODD for the treatment of acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), as well as Fast Track Designation for the treatment of AML, from the U.S. FDA. The content above comes from the network. if any infringement, please contact us to modify.

快速通道孤儿药

2024-11-12

·GlobeNewswire-Health Care

Belite Bio Reports Third Quarter 2024 Financial Results and Provides a Corporate Update

Dosed first patient in Phase 2/3 DRAGON II trial of Tinlarebant for the treatment of Stargardt disease (STGD1)Pivotal global Phase 3 PHOENIX trial of Tinlarebant in geographic atrophy (GA) subjects is ongoing with more than 280 subjects enrolledAppointed Hendrik P.N. Scholl, MD, MA, a globally recognized leader in the field of ophthalmology and the coordinating principal investigator of the largest natural history study of Stargardt disease, as Chief Medical OfficerInterim analysis from the pivotal global Phase 3 DRAGON trial of Tinlarebant in adolescent STGD1 subjects anticipated by end of 2024 or early 2025Company to host webcast on Tuesday, November 12, 2024, at 4:30 p.m. EST SAN DIEGO, Nov. 12, 2024 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) (“Belite” or the “Company”), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced its financial results for the third quarter ended September 30, 2024, and provided a general business update. “I am pleased with the continued progress we made across our clinical programs. The pace of enrollment for the PHOENIX trial remains strong, and in the quarter, we completed the Phase 1b DRAGON II trial of Tinlarebant in Stargardt disease in Japan and dosed the first patient in the Phase 2/3 portion of the trial. We are well-positioned to achieve critical milestones and look forward to providing an update on the interim analysis from our pivotal Phase 3 DRAGON trial toward the end of 2024 or early 2025,” said Dr. Tom Lin, Chairman and CEO of Belite. “In the quarter, we were also excited to welcome Dr. Scholl, a leading global expert in Stargardt disease and age-related macular degeneration, as our Chief Medical Officer. Dr. Scholl’s decision to join Belite following his experience as Chair of the Data and Safety Monitoring Board for both our Phase 2 and Phase 3 Stargardt disease trials further validates our belief in Tinlarebant’s immense potential.” Third Quarter 2024 Business Highlights and Upcoming Milestones: Clinical Highlights Tinlarebant (LBS-008) is an oral, potent, once daily retinol binding protein 4 (RBP4) antagonist that decreases RBP4 levels in the blood and reduces vitamin A (retinol) delivery to the eye without disrupting systemic retinol delivery to other tissues. Vitamin A is critical to normal vision but can accumulate as toxic byproducts in individuals affected with STGD1 and GA (the advanced form of dry age-related macular degeneration (dry AMD) leading to retinal cell death and loss of vision. Stargardt disease (STGD1): Accumulation of cytotoxic vitamin A byproducts (bisretinoids) has been implicated in the onset and progression of STGD1. Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease Designation in the U.S., Orphan Drug Designation in the U.S., Europe, and Japan, and Sakigake (Pioneer Drug) Designation in Japan for the treatment of STGD1. DRAGON Trial: Ongoing, 24-month, randomized (2:1, active: placebo), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in adolescent STGD1 subjects Completed enrollment with 104 subjects in 11 countriesPrimary efficacy endpoint is slowing of atrophic lesion growth rate; safety and tolerability will also be assessedCompany expects to report interim analysis by end of 2024 or early 2025 DRAGON II Trial: Combination of Phase 1b open-label trial to evaluate the pharmacokinetics and pharmacodynamics of Tinlarebant in Japanese adolescent STGD1 subjects and a Phase 2/3, double-masked, placebo-controlled, multicenter, trial in adolescent STGD1 subjects Completed Phase 1b portion of the trial with six subjects evaluated in JapanDosed first patient in Phase 2/3 trial, with a target enrollment of approximately 60 subjects, aged 12 to 20 years old, including approximately 10 Japanese subjects; data from the Japanese subjects is intended to facilitate a future new drug application in JapanPrimary efficacy endpoint is slowing of atrophic lesion growth rate; safety and tolerability will also be assessed Geographic Atrophy (GA): GA is a chronic degenerative disease of the retina that leads to blindness in the elderly. Accumulation of toxic vitamin A byproducts (bisretinoids) has been implicated in the progression of GA. There are currently no FDA-approved, orally administered treatments for GA. PHOENIX Trial: Ongoing, 24-month, randomized (2:1, active: placebo; n~430 subjects), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in patients with GA More than 280 subjects have been enrolled as of November 11, 2024Primary efficacy endpoint is slowing of atrophic lesion growth rate; safety and tolerability will also be assessedCompany expects to conduct interim analysis at the mid-point of the trial Corporate Highlights Hendrik P. N. Scholl, MD, MA, was appointed as the Company’s Chief Medical Officer. Dr. Scholl is the foremost globally recognized authority on Stargardt disease and age-related macular degeneration and brings to Belite decades of expertise in treating retinal diseases, including the two key indications targeted by Tinlarebant. Belite also recently announced the exercise of warrants by certain holder of Belite’s outstanding warrants, pursuant to which Belite received total gross proceeds of approximately $28.75 million, which are expected to be used for general corporate purposes. Third Quarter 2024 Financial Results: Current Assets: As of September 30, 2024, the Company had $109.0 million in cash, money market fund, time deposits and U.S treasury bills. R&D Expenses: For the three months ended September 30, 2024, research and development expenses were $6.8 million compared to $8.7 million for the same period in 2023. This decrease was mainly attributed to fewer contract research organization milestone payments related to the DRAGON trial, partially offset by the increase in the DRAGON II trial expenses. For the nine months ended September 30, 2024, research and development expenses were $22.7 million compared to $20.0 million for the same period in 2023. The increase in research and development expenses was primarily attributable to (i) a royalty payment for completion of a Phase 2 trial, and (ii) share-based compensation granted in the third quarter of 2024. G&A Expenses: For the three months ended September 30, 2024, general and administrative expenses were $2.9 million compared to $2.2 million for the same period in 2023. For the nine months ended September 30, 2024, general and administration expenses were $5.9 million compared to $4.7 million for the same period in 2023. The increase in G&A expenses was primarily due to an increase in share-based compensation granted in the third quarter of 2024. Other Income: For the three months ended September 30, 2024, other income was $1.1 million compared to $0.03 million for the same period in 2023. For the nine months ended September 30, 2024, other income was $2.5 million compared to $0.08 million for the same period in 2023. The increase in other income was attributable to accrued interest from time deposits and U.S. treasury bills. Net Loss: For the three months ended September 30, 2024, the Company reported a net loss of $8.7 million, compared to a net loss of $10.9 million for the same period in 2023. For the nine months ended September 30, 2024, the Company reported a net loss of $26.0 million, compared to a net loss of $24.6 million for the same period in 2023. Webcast Information Belite Bio will host a webcast on Tuesday, November 12, 2024, at 4:30 p.m. Eastern Time to discuss the Company’s financial results and provide a business update. To join the webcast, please visit https://wsw.com/webcast/cc/blte5/1423080. A replay will be available following the event. About Belite Bio Belite Bio is a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting retinal degenerative eye diseases that have significant unmet medical needs such as Stargardt disease type 1, or STGD1 and Geographic Atrophy (GA) in advanced dry age-related macular degeneration (AMD), in addition to specific metabolic diseases. Belite’s lead candidate, Tinlarebant, an oral therapy intended to reduce the accumulation of toxins in the eye, and is currently being evaluated in a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) in adolescent STGD1 subjects and a Phase 3 study (PHOENIX) in subjects with GA. For more information, follow us on Twitter, Instagram, LinkedIn, Facebook, or visit us at http://www.belitebio.com. Important Cautions Regarding Forward Looking Statements This press release contains forward-looking statements about future expectations and plans, as well as other statements regarding matters that are not historical facts. These statements include but are not limited to statements regarding the potential implications of clinical data for patients, and Belite Bio’s advancement of, and anticipated preclinical activities, clinical development, regulatory milestones, and commercialization of its product candidates, and any other statements containing the words “expect”, “hope”, and similar expressions. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including but not limited to Belite Bio’s ability to demonstrate the safety and efficacy of its drug candidates; the clinical results for its drug candidates, which may not support further development or regulatory approval; the timing to complete relevant clinical trials and/or to receive the interim/final data of such clinical trials; the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approval of Belite Bio’s drug candidates; the potential efficacy of Tinlarebant, as well as those risks more fully discussed in the “Risk Factors” section in Belite Bio’s filings with the U.S. Securities and Exchange Commission. All forward-looking statements are based on information currently available to Belite Bio, and Belite Bio undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law. BELITE BIO, INCUNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS(Amounts in thousands of US Dollars, except share and per share amounts) For the Three Months For the Nine Months Ended September 30, Ended September 30, 2023 2024 2023 2024 Expenses Research and development 8,743 6,842 19,982 22,685 General and administrative 2,218 2,898 4,731 5,854 Total operating expenses 10,961 9,740 24,713 28,539 Loss from operations (10,961) (9,740) (24,713) (28,539)Other income: Total other income, net 27 1,061 81 2,501 Loss before income tax (10,934) (8,679) (24,632) (26,038)Income tax expense 1 - 10 6 Net loss (10,935) (8,679) (24,642) (26,044)Other comprehensive income (loss) Foreign currency translation adjustments, net of nil tax (55) 79 (115) (27)Total comprehensive loss (10,990) (8,600) (24,757) (26,071)Weighted average number of ordinary shares used in per share calculation: - Basic and Diluted 27,315,550 30,687,305 26,013,012 30,231,207 Net loss per ordinary share - Basic and Diluted $(0.40) $(0.28) $(0.95) $(0.86) BELITE BIO, INCUNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS(Amounts in thousands of US Dollars, except share amounts) December 31, September 30, 2023 2024 Current assets $89,940 $111,268 Other assets 4,702 4,553 TOTAL ASSETS $94,642 $115,821 TOTAL LIABILITIES $4,211 $3,621 TOTAL SHAREHOLDERS’ EQUITY 90,431 112,200 TOTAL LIABILITIES AND SHAREHOLDERS’ EQUITY $94,642 $115,821 Ordinary shares authorized 400,000,000 400,000,000 Ordinary shares issued 29,184,475 30,931,247 Ordinary shares outstanding 29,149,444 30,879,332 Media and Investor Relations Contact: Jennifer Wu /ir@belitebio.com Julie Fallon /belite@argotpartners.com

临床3期临床2期临床1期快速通道财报

100 项与 Tinlarebant 相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
地图样萎缩	临床3期	美国	Belite Bio, Inc.	2023-07-27
地图样萎缩	临床3期	中国	Belite Bio, Inc.	2023-07-27
地图样萎缩	临床3期	澳大利亚	Belite Bio, Inc.	2023-07-27
地图样萎缩	临床3期	捷克	Belite Bio, Inc.	2023-07-27
地图样萎缩	临床3期	法国	Belite Bio, Inc.	2023-07-27
地图样萎缩	临床3期	瑞士	Belite Bio, Inc.	2023-07-27
地图样萎缩	临床3期	中国台湾	Belite Bio, Inc.	2023-07-27
地图样萎缩	临床3期	英国	Belite Bio, Inc.	2023-07-27
斯塔加特病	临床3期	美国	Belite Bio, Inc.	2022-03-28
斯塔加特病	临床3期	中国	Belite Bio, Inc.	2022-03-28

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


EURETINA2024	N/A	斯塔加特病	-	Tinlarebant 5 mg/day	艱築齋獵繭範選壓築範(鬱餘壓積選遞簾網蓋壓) = Delayed dark adaptation (9 of 13 subjects [69.2%]) were most frequently reported 齋顧觸繭衊膚築簾壓選 (遞餘淵繭觸構糧蓋鏇鹹 ) 更多	-	2024-09-19
NCT05266014 (LBS-008-CT02, ARVO 12023 \| ARVO 22023) 人工标引	临床1/2期	斯塔加特病	13	Tinlarebant	觸遞鹽淵繭顧遞獵鹽顧(襯範觸膚廠夢窪淵網構) = 衊壓糧鹹夢夢製艱壓鹽觸齋鑰衊淵壓衊積窪蓋 (願艱鑰繭齋簾糧艱觸遞 ) 更多	积极	2023-04-23
NCT05266014 (Corporate Publications) 人工标引	临床1/2期	斯塔加特病	13	LBS-008	網網衊鑰窪鏇觸願網繭(簾獵醖鏇膚艱糧鹽艱獵) = 鑰襯獵遞範鑰膚積餘蓋遞遞積窪獵夢積糧鏇淵 (觸餘網鏇淵網壓齋願範 ) 更多	积极	2022-10-01