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Treatment of beta-thalassemia using actrii ligand traps

A technology for thalassemia and activin receptors, which can be used in medical preparations containing active ingredients, blood diseases, pharmaceutical formulations, etc., and can solve problems such as unmet medical needs and untreated β-thalassemia.

Pending Publication Date: 2018-03-27
CELGENE CORP +1
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

[0008] Given that there are currently no safe and effective drug therapies for beta-thalassemias (eg, transfusion-dependent and non-transfusion-dependent There is a significant unmet medical need for new treatments for the underlying pathophysiology of

Method used

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  • Treatment of beta-thalassemia using actrii ligand traps
  • Treatment of beta-thalassemia using actrii ligand traps
  • Treatment of beta-thalassemia using actrii ligand traps

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 2

[0394] 8.2 Example 2: A phase 3 double-blind randomized placebo-controlled multicenter study to determine the efficacy and safety of ACTRIIB-HFC (SEQ ID NO: 25) in adults with transfusion-independent beta thalassemia

[0395] This example provides an overview of a phase 3 double-blind randomized placebo-controlled multicenter study to determine the efficacy and safety of ACTRIIB-HFC (SEQ ID NO: 25) in adults with transfusion-independent beta thalassemia. The indications for the phase 3 study are adults with transfusion-independent beta thalassemia, who are diagnosed with β-thalassemia or hemoglobin E / β-thalassemia.

[0396] 8.2.1 Goal

[0397] The main goal of the Phase 3 study is to determine if ActRIIB-hFc (SEQ ID NO: 25) is diagnosed as transfusion-independent β-thalassemia, documented β-thalassemia or hemoglobin E / β-thalassemia diagnosis, age ≥18 Years old and have received 0-6 red blood cell units and average baseline hemoglobin level within 24 weeks prior to random assignment ...

Embodiment 3

[0423] 8.3 Example 3: ACTRIIB-HFC (SEQ ID NO: 25) signal transduction inhibitor increases hemoglobin and reduces the burden of blood transfusion and reduces liver iron concentration in adults with β thalassemia

[0424] 8.3.1 Introduction

[0425] ActRIIB-hFc (SEQ ID NO: 25), a fusion protein containing a modified activin receptor, is being developed for the treatment of β-thalassemia. In β-thalassemia, anemia and complications are caused by ineffective erythropoiesis driven by excess α-globin. ActRIIB-hFc (SEQ ID NO: 25) binds to other ligands in the GDF11 and TGF-β superfamily to promote late red blood cell differentiation. Non-clinical and clinical studies have shown that ActRIIB-hFc (SEQ ID NO: 25) has a well-tolerated and corrective effect on ineffective erythropoiesis (Suragani R, Blood 2014, Attie K, Am JHematol 2014).

[0426] This example provides data from an ongoing Phase 2 multicenter open-label dose exploration study to evaluate ActRIIB-hFc (SEQ ID NO: 25) in adults wi...

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Abstract

Provided herein are methods of treating beta-thalassemia by subcutaneous administration of about 0.8 mg / kg of an ActRII signaling inhibitor. Also provided herein are methods of adjusting the dose of the ActRII signaling inhibitor administered to the subject.

Description

[0001] 1. Cross-reference of related applications [0002] This application requires U.S. Provisional Patent Application No. 62 / 161,136 filed on May 13, 2015, U.S. Provisional Patent Application No. 62 / 173,836 filed on June 10, 2015, and U.S. Provisional Patent Application No. filed on October 19, 2015 62 / 243,457 priority rights, the complete content of each of which is incorporated herein by reference and used for all purposes. [0003] 2. Sequence Listing [0004] This application is being submitted together with the sequence listing submitted under the file name "12827_952_228_SeqListing.txt", which is 97 kilobytes in size and was created on May 4, 2016. The sequence listing is incorporated herein by reference in its entirety and used for all purposes. 3. Field of Invention [0005] Provided herein are methods for the treatment and / or prevention of β-thalassemia, such as transfusion-dependent and non-transfusion-dependent β-thalassemia, the method comprising administering to a sub...

Claims

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Application Information

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IPC IPC(8): A61K38/18A61P7/00A61P7/06
CPCC07K14/495C07K14/71C07K2319/30A61K38/179A61P7/06A61P7/00A61K9/0019
Inventor 肯尼思·M·阿蒂阿伯达拉曼恩·拉亚德姆拉杰什·乔普拉杰伊·巴克斯托姆
Owner CELGENE CORP
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