A Phase 2a, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Safety and Efficacy of BIIB057 in Subjects With Rheumatoid Arthritis Who Have Had an Inadequate Response to Disease-Modifying Antirheumatic Drugs

Phase II study designed to evaluate the safety and efficacy of BIIB057 in Subjects with Rheumatoid Arthritis who have experienced an Inadequate Response to Disease-Modifying Antirheumatic Drugs (DMARDs).

开始日期2012-08-01

申办/合作机构

Biogen, Inc.

100 项与 PRT-2607 相关的临床结果

登录后查看更多信息

100 项与 PRT-2607 相关的转化医学

登录后查看更多信息

100 项与 PRT-2607 相关的专利（医药）

登录后查看更多信息

283

项与 PRT-2607 相关的文献（医药）

2026-02-01·DRUG DEVELOPMENT RESEARCH

Recent Progress of Molecular Design and Activities of Spleen Tyrosine Kinase (SYK) Inhibitors

Review

作者: Chen, Sheng ; Li, Yongdong ; Lin, Jieman ; Shen, Jiayi ; Kong, Yunyi ; Li, Anzhi ; Li, Gonghui

ABSTRACT:

Spleen tyrosine kinase (SYK), a non‐receptor tyrosine kinase, serves as a pivotal regulator in multiple intracellular signaling pathways, particularly those involved in immune responses. Dysregulated SYK activation has been strongly implicated in the pathogenesis of various hematological malignancies and autoimmune disorders, making it an attractive therapeutic target. Consequently, significant progress has been made in developing SYK inhibitors as a targeted treatment strategy. This article comprehensively reviews recent advances in SYK inhibitor research, focusing on their therapeutic potential in autoimmune diseases and hematologic cancers. Furthermore, we highlight representative SYK inhibitors, discuss their clinical applications, outline current challenges, and future directions in this evolving field.

2026-02-01·Blood vessels, thrombosis & hemostasis

The small-molecule Syk inhibitor R788 inhibits hematopoiesis and worsens anemia in sickle cell disease mice

Article

作者: Parachalil Gopalan, Bindu ; Kamimura, Sayuri ; Shet, Arun S ; Shah, Niharika ; Quezado, Martha ; Lee, Duck-Yeon ; Dagur, Pradeep ; Quezado, Zenaide

Vaso-occlusive crises, thrombosis, inflammation, and immune dysregulation contribute to organ damage and poor outcomes in sickle cell disease (SCD). Because neutrophils and dysregulated extracellular trap formation (NETosis) contribute to sickle pathophysiology, and the spleen tyrosine kinase (Syk) signaling pathway is a key driver of NETosis, we investigated the effect of targeting Syk with fostamatinib (R788). Specifically, we studied the effect of a selective Syk inhibitor, R788, on hematologic and biochemical parameters, NETosis, platelet P-selectin expression, and platelet-neutrophil aggregate formation in Townes sickle mice at baseline and after exposure to pathophysiological stressors (tumor necrosis factor α [TNF-α] and hypoxia-reoxygenation). Our results showed that at baseline R788 impaired hematopoiesis, and worsened anemia and neutropenia in sickle mice. Additionally, R788 at nontoxic doses had little, if any, effect on NETosis and platelet activation induced by TNF-α or hypoxia-reoxygenation. Severe anemia and neutropenia induced by R788 in the sickle mouse model suggests that concomitant use of Syk inhibitors with hydroxyurea in patients with SCD should be approached cautiously. Further research is required to clarify the benefits and risks of selective Syk inhibition in SCD and other hemolytic conditions exhibiting stress hematopoiesis.

2025-12-01·CANCER GENE THERAPY

Spleen tyrosine kinase (SYK) inhibition suppresses growth of gastrointestinal neuroendocrine tumor cells: a pilot study in two cell lines

Article

作者: Toffoli, Lara ; D'Este, Francesca ; Vella, Viviana ; Ditsiou, Angeliki ; Gagliano, Teresa

Abstract:

Gastrointestinal neuroendocrine tumors (GI-NETs) lack effective targeted options beyond somatostatin analogs and mTOR inhibitors. Spleen tyrosine kinase (SYK) is a non-receptor kinase with emerging roles in solid tumors and available small-molecule inhibitors. We explored whether SYK is a plausible therapeutic target in GI-NET using two human cell lines. SYK expression in GI-NET cells was confirmed by immunofluorescence. Cells were exposed to a selective SYK inhibitor (BI-1002494), and proliferation was quantified using both 2D and 3D models. Both GI-NET models expressed SYK and exhibited reduced growth upon SYK blockade, with dose-dependent suppression of viability and increased cytotoxicity relative to vehicle. In spheroid assays, morphologic changes and reduced size were observed. These pilot data suggest SYK as a targetable vulnerability in GI-NET and support formal dose–response studies, genetic validation, and combination strategies with standard-of-care agents. Given the clinical availability of SYK inhibitors, these findings provide a rationale for translational studies in GI-NET.

项与 PRT-2607 相关的新闻（医药）

2024-12-31

·echemi

7 New Drugs Expected to Be Approved in 2025

In 2024, China approved several new drugs, and more blockbuster drugs are expected to be launched in 2025, covering oncology, autoimmune diseases, rare diseases, and other therapeutic areas. Drug Name Mechanism of Action Indications Development Institutions Evantuzumab c-Met/EGFR bispecific antibody Non-small cell lung cancer Johnson & Johnson; Genmab Taqetuzumab CD3/GPRC5D bispecific antibody Multiple myeloma Johnson & Johnson; Genmab Zenidatuzumab Anti-HER2 bispecific antibody Biliary tract cancer Zymeworks; Jazz Pharmaceuticals; BeiGene Inalises PI3Kα inhibitor HR-positive breast cancer Roche; Chugai Pharmaceutical Asunitinib STAMP allosteric inhibitor Speculated use for chronic myeloid leukemia treatment Novartis Tazemetostat EZH2 inhibitor Follicular lymphoma Epizyme; Huadong Medicine; Eisai Relatuzumab-alpha PDL1/TGF-β bispecific antibody Stomach cancer; gastroesophageal junction cancer Hengrui Medicine; Dong-A Pharma Vabysmo EGFR antibody-drug conjugate Nasopharyngeal cancer Lepu Biopharma Tenapanor NHE3 inhibitor Chronic kidney disease hyperphosphatemia Ardelyx; Kyowa Kirin; Fosun Pharma; AstraZeneca Mersutide OXM analog; GLP-1R/GCGR agonist Obesity Eli Lilly; Innovent Biologics Vusirumab IL-1β monoclonal antibody Gouty arthritis Junshi Biosciences Romosozumab SOST monoclonal antibody Osteoporosis Astellas; Amgen; UCB Teplizumab CD3 monoclonal antibody Type 1 diabetes Ligand Pharmaceuticals; Johnson & Johnson; Sanofi Solrepinib Syk inhibitor Immune thrombocytopenic purpura Huadong Medicine BBM-H901 Factor IX gene therapy Hemophilia B Gene Therapy Fitusiran RNAi therapy Hemophilia A; Hemophilia B Sanofi; Alnylam Andexanet alfa Recombinant factor Xa Factor Xa inhibitor antidote Portola Pharmaceuticals Lisocabtagene maraleucel IL-23p19 monoclonal antibody Crohn's disease Boehringer Ingelheim; AbbVie Lemborexant & Daridorexant OX1R antagonist; OX2R antagonist Insomnia Eisai & Idorsia; Sihuan Pharmaceutical; Nxera Pharma Highlights of new drug approvals in 2024 include: 1. Oncology: The world’s first antibody-drug conjugate (ADC) targeting folate receptor alpha (FRα) for ovarian cancer: Mirvetuximab soravtansine. PD-1/VEGF bispecific antibody envafolimab and Nectin-4 ADC enfortumab vedotin. 2. Autoimmune diseases: IL-17A/F monoclonal antibody bimekizumab for ankylosing spondylitis. 3. Rare diseases: Novartis’ complement factor B inhibitor for paroxysmal nocturnal hemoglobinuria (PNH). Blockbuster drugs expected to be approved in 2025 include: Oncology: Amivantamab (Q1) The world’s first c-Met/EGFR bispecific antibody. Highlight: Phase III study showed significant extension of median progression-free survival (mPFS). Talquetamab (Q2) A CD3/GPRC5D bispecific antibody and the world’s first drug targeting GPRC5D. Clinical results: Objective response rate (ORR) in Chinese patients was 69.0%-73.6%. Zanidatamab (Q2) A HER2-targeting bispecific antibody based on Azymetric™ technology. Indication: HER2-positive biliary tract cancer (BTC) with an ORR of up to 52%. Inavolisib (Q3) Roche’s highly selective PI3Kα inhibitor for PIK3CA-mutated breast cancer. Asciminib (Q3) Novartis’ Bcr-Abl allosteric inhibitor for Philadelphia chromosome-positive chronic myeloid leukemia (CML). SHR-1701 (Q4) A PD-L1/TGF-βRII bifunctional fusion protein for first-line treatment of gastric cancer. Phase III study demonstrated significant extension in median overall survival. MRG003 (Q4) China’s first EGFR-targeting ADC with remarkable efficacy in nasopharyngeal carcinoma. Endocrinology and Metabolism: Tenapanor (Q1) The world’s only approved NHE3 inhibitor for controlling hyperphosphatemia in chronic kidney disease patients. Trend Analysis: Continued Innovation in Oncology: The emergence of multiple bispecific antibodies and ADC drugs is making targets and treatment options more precise and diverse. Breakthroughs in Autoimmune and Rare Diseases: New biologics are gradually addressing drug resistance and limited treatment options in previous therapies. Emergence of Domestic Innovative Drugs: Examples include SHR-1701 and MRG003, highlighting the growing research and development capabilities of Chinese pharmaceutical companies. The sequential launch of these drugs will provide new therapeutic options for Chinese patients and enhance China’s position in the global pharmaceutical market.

上市批准临床3期临床结果抗体药物偶联物免疫疗法

2024-03-21

·PRNewswire

FDA Grants Orphan Drug Designation to Cevidoplenib for ITP

PANGYO, South Korea, March 21, 2024 /PRNewswire/ -- Oscotec Inc. has secured orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for its SYK inhibitor, Cevidoplenib, to treat immune thrombocytopenia (ITP). Oscotec has successfully completed phase 2 study in patients with chronic ITP last year and are currently seeking partners for further development and global commercialization. "Potential partners are drawing attention not only to the efficacy of cevidoplenib but also its exceptional safety profile and the convenience of oral dosing," said Dr. Taeyoung Yoon, CEO/CSO of Oscotec. "Obtaining ODD is an important milestone in the development of cevidoplenib and ultimately will benefit patients. We will explore every available option including partnership to deliver our drug as quickly as we can to those who suffers from the potentially crippling disease." The orphan drug designation is granted to agents that prevent, diagnose, or treat a rare disease. Drug sponsors are eligible for incentives including tax credits for clinical trials, exemption from user fees, and a potential of 7 years of market exclusivity after approval. About Immune Thrombocytopenia (ITP) Immune thrombocytopenia (ITP) is a disorder that can lead to easy or excessive bruising and bleeding. The bleeding results from unusually low levels of platelets. Formerly known as idiopathic thrombocytopenic purpura, ITP can cause purple bruises, as well as tiny reddish-purple dots that look like a rash. The prevalence (how many have ITP at any time) is 9.5 per 100,000. ITP patients experience chronic fatigue. The most concerning, but rare, form of bleeding due to ITP is an intracranial hemorrhage (bleeding in the brain). ITP treatment consists of the administration of corticosteroids, immunoglobulins, thrombopoietin receptor agonists, etc. However, sustained remission in the absence of treatment suggests the optimization of ITP management is necessary. About Oscotec Inc. Oscotec is a clinical stage drug discovery and development company pursuing translation of rigorous science into innovative medicine for clinically unmet needs.. The Company's clinical pipeline consists of targeted therapeutics in immunology and oncology. Oscotec is developing cevidoplenib (SYK inhibitor) for ITP (Phase 2, completed) and FLT3/AXL inhibitor for AML (Phase 1) as well as solid tumors (Phase 1). The company is also the originator of Lazertinib (LECLAZA®), a 3rd generation EGFR inhibitor being developed by Janssen Pharma and Yuhan Corp. for lung cancers. Contacts Jihyun Park, IR/PR [email protected] SOURCE Oscotec Inc.

临床2期孤儿药临床1期免疫疗法

2023-08-25

·FiercePharma

Fierce Pharma Asia—Moderna's cancer combo plan; GSK's trial win; Glenmark's DOJ deal

Moderna and CARsgen's cancer deal, GSK's shingles trial in China and Glenmark's settlement with the U.S. Justice Department made our news this week. Moderna is considering pairing one of its mRNA cancer vaccine candidates with a CAR-T therapy from China's CARsgen. GSK's Shingrix showed 100% efficacy in a postmarketing trial in China. Glenmark and Teva have reached a settlement with U.S. prosecutors over price-fixing charges. 1. Moderna reveals Claudin18.2 ambitions via cancer vaccine, solid tumor CAR-T combo plans Chinese company CARsgen Therapeutics has reached a deal with Moderna for the two companies to “contemplate conducting preclinical studies and a phase 1 clinical trial” for a combination of Moderna’s Claudin18.2 mRNA cancer vaccine and CARsgen’s CAR-T therapy directed at the same target. Astellas, AstraZeneca, BioNTech and Legend Biotech are also working on Claudin18.2 therapies. 2. In GSK's trial of shingles vaccine in China, Shingrix keeps it 100 In a postmarketing study of GSK’s Shingrix in China, the shingles vaccine demonstrated 100% efficacy in nearly 6,000 people ages 50 and up. No Shingrix recipients got shingles, compared with 31 cases in the placebo group. GSK has attributed much of Shingrix’s recent sales growth to the shot's expansion in China. 3. Teva, Glenmark ⁠reach $255M price-fixing settlement with DOJ, agree to offload certain meds Teva and Glenmark have reached a settlement with the U.S. Department of Justice to resolve a price-fixing case. In the deferred prosecution agreement, Glenmark agreed to pay a fine of $30 million. Glenmark also admitted to participating in a conspiracy over the price of pravastatin. Teva will pay $225 million in the deal. 4. Hutchmed eyes Chinese approval for autoimmune disorder drug after phase 3 success Hutchmed said a phase 3 trial in China testing its Syk inhibitor sovleplenib as a primary immune thrombocytopenia treatment has met its goals. Compared with placebo, sovleplenib showed a significant increase in durable response rate compared with placebo in previously treated patients. The company plans to file for approval in China by the end of the year. 5. Alembic and Aurobindo issue US drug recalls, citing quality and production issues Two Indian companies, Alembic and Aurobindo, issued voluntary recalls in the U.S. Alembic pulled bottles of the antibiotic tobramycin because of quality issues. Aurobindo recalled some rufinamide tablets for treating seizure disorders because the products were distributed without being approved. 6. With health panel backing, Eisai one step away from Leqembi approval in Japan (Fierce Pharma regulatory tracker) A Japanese drug review panel has recommended approval of Eisai’s Alzheimer’s disease drug Leqembi. The decision sets up an official go-ahead from the local health ministry. A Jefferies analyst said he expected the Japanese government will reimburse the drug at around $13,700 per year, according to Bloomberg. Other News of Note: 7. Chinese CRO Tigermed debuts new HQ den in Hong Kong 8. Crown Bioscience, HanX pact shows promise of lymphoma drug

疫苗上市批准临床3期临床1期信使RNA

100 项与 PRT-2607 相关的药物交易

登录后查看更多信息

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
类风湿关节炎	临床2期	加拿大	Biogen, Inc.	2012-08-01
炎症	临床1期	美国	Portola Pharmaceuticals LLC	-
炎症	临床1期	英国	Portola Pharmaceuticals LLC	-
肿瘤	临床1期	美国	Portola Pharmaceuticals LLC	-
肿瘤	临床1期	英国	Portola Pharmaceuticals LLC	-
系统性红斑狼疮	临床1期	美国	-	-