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).

Start Date01 Aug 2012

Sponsor / Collaborator

Biogen, Inc.

100 Clinical Results associated with PRT-2607

100 Translational Medicine associated with PRT-2607

100 Patents (Medical) associated with PRT-2607

308

Literatures (Medical) associated with PRT-2607

31 Dec 2026·Hematology

Efficacy and mechanism of action of sovleplenib for aplastic anemia

Article

Author: Xin, Shan ; Sheng, Tianzi ; Liu, Hong ; Zhou, Chengcheng ; Sun, Wei

OBJECTIVE:

This study aimed to investigate the therapeutic potential of sovleplenib, a selective SYK inhibitor, in aplastic anemia (AA), focusing on its effects on immune modulation and the cGAS-STING-NF-κB inflammatory axis.

METHODS:

An immune-mediated AA mouse model was established and treated with sovleplenib. Hematopoietic function was assessed via peripheral blood counts and bone marrow mononuclear cell (BMNC) counts. Immune cell profiles were analyzed by flow cytometry. Cytokine levels were measured by ELISA. Underlying mechanisms were explored through proteomics and Western blotting.

RESULTS:

Sovleplenib treatment significantly improved peripheral blood counts (HGB, WBC, PLT) and BMNCs, and reduced bone marrow fat vacuolation. It corrected immune imbalance by increasing the CD4⁺/CD8⁺ T-cell ratio and decreasing the M1/M2 macrophage ratio, promoting an M2 phenotype. Concurrently, it elevated Arg-1 level while suppressing ROS, TNF-α, and IFN-β. Mechanistically, sovleplenib inhibited the activation of key pathway components (cGAS, STING, p-TBK1, p-p65).

DISCUSSION:

The results demonstrate sovleplenib's dual role in promoting hematopoiesis and immunomodulation. Its efficacy is mechanistically linked to the suppression of the overactivated cGAS-STING-NF-κB pathway, a key driver of inflammation in AA.

CONCLUSION:

Sovleplenib represents a promising targeted therapy for AA.

01 Mar 2026·INTERNATIONAL IMMUNOPHARMACOLOGY

TREM2 regulates neuroinflammation by SYK-dependent inhibition of BTK activation to improve perioperative neurocognitive dysfunction

Article

Author: Meng, Fanjun ; Guo, Xinlei ; Zhang, Kaiyun ; Huang, Yaru ; Chi, Wenying ; Guo, Junzuo ; Zhang, Bowen ; Zhang, Zhe

This study investigates the role of the TREM2-SYK-BTK signaling pathway in microglial activation and cognitive decline. In vivo and in vitro experiments demonstrate that surgical trauma induces BTK phosphorylation in hippocampal microglia, promoting their transition towards a pro-inflammatory M1 phenotype while suppressing the anti-inflammatory M2 phenotype. In a perioperative neurocognitive disorder (PND) mouse model, hippocampal injection of AAV-BTK siRNA suppressed BTK phosphorylation, promoting microglial transition from M1 to M2 phenotype. This shift manifested as reduced pro-inflammatory cytokines (IL-1β, IL-6) and increased anti-inflammatory cytokines (IL-4, IL-10), ultimately alleviating cognitive impairment. Further mechanistic analysis revealed that TREM2 regulates microglial inflammatory balance by enhancing SYK phosphorylation to inhibit BTK activity, an effect antagonized by the SYK inhibitor R406. Subsequent studies indicate this pathway modulates neuroinflammation by regulating NF-κB signaling and NLRP3 inflammasome activation. These findings illustrate that TREM2-SYK-BTK signaling pathway is the key internal mechanism to regulate microglial polarization and neuroinflammation, which provides a new theoretical basis and potential therapeutic strategy for PND.

01 Feb 2026·DRUG DEVELOPMENT RESEARCH

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

Review

Author: 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.

News (Medical) associated with PRT-2607

31 Dec 2024

·www.echemi.com

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.

Drug ApprovalPhase 3Clinical ResultADCImmunotherapy

21 Mar 2024

·www.prnewswire.com

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.

Phase 2Orphan DrugPhase 1Immunotherapy

25 Aug 2023

·www.fiercepharma.com

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

VaccineDrug ApprovalPhase 3Phase 1mRNA

100 Deals associated with PRT-2607

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Rheumatoid Arthritis	Phase 2	Canada	Biogen, Inc.	01 Aug 2012
Inflammation	Phase 1	United States	Portola Pharmaceuticals LLC	-
Inflammation	Phase 1	United Kingdom	Portola Pharmaceuticals LLC	-
Neoplasms	Phase 1	United States	Portola Pharmaceuticals LLC	-
Neoplasms	Phase 1	United Kingdom	Portola Pharmaceuticals LLC	-
Systemic Lupus Erythematosus	Phase 1	United States	-	-

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