{"id":141,"date":"2023-02-13T19:54:37","date_gmt":"2023-02-13T19:54:37","guid":{"rendered":"https:\/\/amiratx.com\/?page_id=141"},"modified":"2025-12-15T12:42:50","modified_gmt":"2025-12-15T12:42:50","slug":"ami463","status":"publish","type":"page","link":"https:\/\/amiratx.com\/index.php\/ami463\/","title":{"rendered":"AMI463"},"content":{"rendered":"

[et_pb_section fb_built=”1″ admin_label=”Header” _builder_version=”4.16″ custom_margin=”||” custom_padding=”6vw|||||” global_colors_info=”{}”][et_pb_row column_structure=”3_5,2_5″ _builder_version=”4.16″ custom_padding=”0px|||||” global_colors_info=”{}”][et_pb_column type=”3_5″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text _builder_version=”4.16″ text_font=”||||||||” text_font_size=”20px” text_line_height=”1.6em” header_font=”Gentium Basic|700|||||||” header_font_size=”70px” header_line_height=”1.2em” text_orientation=”center” header_font_size_tablet=”40px” header_font_size_phone=”25px” header_font_size_last_edited=”on|phone” locked=”off” global_colors_info=”{}”]<\/p>\n

AMI463<\/span><\/h1>\n

Investigational, brain-penetrant, first-in-class, highly selective small molecule inhibitor of the Cell adhesion molecule (CAM)-related downregulated by oncogenes (CDON)<\/span><\/span><\/span><\/span><\/p>\n

<\/span><\/p>\n

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AMI463 has received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) and from the US Food and Drug Administration (FDA) for the treatment of soft tissue sarcomas (STS). Additionally, AMI463 has obtained the Rare Pediatric Disease Designation (RPDD) from the FDA for the treatment of pediatric rhabdomyosarcoma, the most common STS in the pediatric population.<\/h3>\n

Soft tissue sarcomas <\/span>represent a rare and heterogeneous group of tumors, arising in embryologically derived mesenchymal connective tissues. Advanced STS is associated with a poor prognosis, and treatment options are limited to chemotherapy.<\/span><\/h3>\n

[\/et_pb_text][\/et_pb_column][et_pb_column type=”2_5″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/amiratx.com\/wp-content\/uploads\/2023\/02\/black-kid-enjoying-his-painting-2022-12-15-23-30-55-utc-scaled.jpg” title_text=”AMI463″ _builder_version=”4.19.5″ max_width_tablet=”400px” max_width_phone=”300px” max_width_last_edited=”on|desktop” custom_margin=”|-6vw||” custom_margin_tablet=”6vw|||0px” custom_margin_phone=”” custom_margin_last_edited=”on|tablet” box_shadow_style=”preset4″ box_shadow_horizontal=”6vw” box_shadow_vertical=”-6vw” box_shadow_color=”#ff5f24″ locked=”off” global_colors_info=”{}”][\/et_pb_image][\/et_pb_column][\/et_pb_row][\/et_pb_section][et_pb_section fb_built=”1″ admin_label=”Services” module_id=”AMI463MoA” _builder_version=”4.19.5″ global_colors_info=”{}”][et_pb_row _builder_version=”4.19.5″ _module_preset=”default” global_colors_info=”{}”][et_pb_column type=”4_4″ _builder_version=”4.19.5″ _module_preset=”default” global_colors_info=”{}”][et_pb_image src=”https:\/\/amiratx.com\/wp-content\/uploads\/2023\/02\/Captura-de-pantalla-2023-02-13-a-les-21.17.06.png” title_text=”Captura de pantalla 2023-02-13 a les 21.17.06″ _builder_version=”4.19.5″ _module_preset=”default” global_colors_info=”{}”][\/et_pb_image][\/et_pb_column][\/et_pb_row][et_pb_row column_structure=”1_4,1_4,1_4,1_4″ _builder_version=”4.16″ locked=”off” global_colors_info=”{}”][et_pb_column type=”1_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_blurb title=”First-in-class” content_max_width=”100%” _builder_version=”4.19.5″ header_font=”|700|||||||” header_font_size=”16px” header_line_height=”1.2em” body_font=”||||||||” body_line_height=”1.6em” animation=”off” locked=”off” global_colors_info=”{}”]<\/p>\n

AMI463 is the first selective\u00a0<\/span><\/span>inhibitor of the <\/span><\/span>Hedgehog (<\/span><\/span>Hh<\/span><\/span>) pathway <\/span><\/span>coreceptor<\/span><\/span>, CDO.<\/span><\/span><\/p>\n

[\/et_pb_blurb][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_blurb title=”New Target” content_max_width=”100%” _builder_version=”4.19.5″ header_font=”|700|||||||” header_font_size=”16px” header_line_height=”1.2em” body_font=”||||||||” body_line_height=”1.6em” animation=”off” locked=”off” global_colors_info=”{}”]<\/p>\n

CDO is a new therapeutic target against the Hedgehog pathway in rhabdomyosarcoma and other pediatric cancers.<\/p>\n

[\/et_pb_blurb][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_blurb title=”Specificity” content_max_width=”100%” _builder_version=”4.19.5″ header_font=”|700|||||||” header_font_size=”16px” header_line_height=”1.2em” body_font=”||||||||” body_line_height=”1.6em” animation=”off” locked=”off” global_colors_info=”{}”]<\/p>\n

CDO is a receptor with high tumor specificity in childhood cancers.<\/span><\/p>\n

[\/et_pb_blurb][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_blurb title=”Potency” content_max_width=”100%” _builder_version=”4.19.5″ header_font=”|700|||||||” header_font_size=”16px” header_line_height=”1.2em” body_font=”||||||||” body_line_height=”1.6em” animation=”off” locked=”off” global_colors_info=”{}”]<\/p>\n

AMI463 has much greater antiproliferative potency than current Hh inhibitors.<\/p>\n

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AMI463\u00a0arrests the cell cycle,\u00a0<\/span><\/span>induces apoptosis, <\/span><\/span>promotes <\/span><\/span>differentiation, and <\/span><\/span>reduces the <\/span><\/span>invasive <\/span><\/span>abilities of cancer cells.<\/span><\/span><\/p>\n

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AMI463 shows <\/span><\/span>antioncogenic<\/span><\/span> efficacy\u00a0in <\/span><\/span>several cancer cell lines, <\/span><\/span>standing out in the most <\/span><\/span>aggressive subtype <\/span><\/span>(PAX3\/FOXO1 <\/span><\/span>translocated<\/span><\/span>).<\/span><\/span><\/p>\n

[\/et_pb_blurb][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_blurb title=”Safety” content_max_width=”100%” _builder_version=”4.19.5″ header_font=”|700|||||||” header_font_size=”16px” header_line_height=”1.2em” body_font=”||||||||” body_line_height=”1.6em” animation=”off” locked=”off” global_colors_info=”{}”]<\/p>\n

AMI463 has no apparent <\/span><\/span>toxicity not in cell lines <\/span><\/span>nor in <\/span><\/span>murine<\/span><\/span> models.<\/span><\/span><\/p>\n

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AMI463 reduces <\/span><\/span>orthotopic<\/span><\/span> <\/span><\/span>primary <\/span><\/span>tumor<\/span><\/span> growth <\/span><\/span>by half in <\/span><\/span>murine<\/span><\/span> <\/span><\/span>in vivo<\/span><\/span> <\/span><\/span>models.<\/span><\/span><\/p>\n

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AMI463 is an investigational selective CDON inhibitor.<\/h2>\n

Dysregulation of the Hedgehog pathway has been shown to occur in many cancer types and it is one of the most commonly mutated oncogenic pathways in cancer.<\/span><\/p>\n

AMI463 is an investigational, brain-penetrant, highly-selective\u00a0Cell adhesion molecule (CAM)-related downregulated by oncogenes (CDON)<\/span> designed to target a key enzyme in the MAPK signaling pathway, which may offer an important alternative for people with primary brain tumors or brain metastases of solid tumors.<\/p>\n

AMI463 has been meticulously tested in over ten different animal models, showcasing promising outcomes. Amira Therapeutics is currently preparing for a pivotal Phase 1\/2 clinical trial of AMI463 to assess its safety and efficacy in treating Rhabdomyosarcoma (RMS), the most common form of soft tissue cancer in children.<\/span><\/p>\n

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AMI463 Investigational, brain-penetrant, first-in-class, highly selective small molecule inhibitor of the Cell adhesion molecule (CAM)-related downregulated by oncogenes (CDON) AMI463 has received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) and from the US Food and Drug Administration (FDA) for the treatment of soft tissue sarcomas (STS). Additionally, AMI463 has obtained the Rare […]<\/p>\n","protected":false},"author":5,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_et_pb_use_builder":"on","_et_pb_old_content":"","_et_gb_content_width":"","footnotes":""},"class_list":["post-141","page","type-page","status-publish","hentry"],"_links":{"self":[{"href":"https:\/\/amiratx.com\/index.php\/wp-json\/wp\/v2\/pages\/141","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/amiratx.com\/index.php\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/amiratx.com\/index.php\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/amiratx.com\/index.php\/wp-json\/wp\/v2\/users\/5"}],"replies":[{"embeddable":true,"href":"https:\/\/amiratx.com\/index.php\/wp-json\/wp\/v2\/comments?post=141"}],"version-history":[{"count":47,"href":"https:\/\/amiratx.com\/index.php\/wp-json\/wp\/v2\/pages\/141\/revisions"}],"predecessor-version":[{"id":630,"href":"https:\/\/amiratx.com\/index.php\/wp-json\/wp\/v2\/pages\/141\/revisions\/630"}],"wp:attachment":[{"href":"https:\/\/amiratx.com\/index.php\/wp-json\/wp\/v2\/media?parent=141"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}