Webcast Morgan Stanley 17th Annual Global Healthcare Conference, September 9, 2019, 10:30 am ET, NY


ARGX-110 is a first-in-class SIMPLE Antibody™ with broad therapeutic potential across a range of cancers (blood and solid tumors). ARGX-110 has demonstrated a very promising safety profile and initial signs of biological activity in solid and blood tumors. Our current clinical development plan focuses on acute myeloid leukemia (AML) and cutaneous T-cell lymphoma (CTCL) two indications with a very high unmet medical need.

Clinical trials

  • Ongoing Phase 1/2 clinical trial in AML & MDS (combination therapy with azacitidine)
    • The preliminary data from the first set of patients suggest ARGX-110 is active both at the circulating and bone marrow blast levels and at the leukemic stem cell (LSC) level. 
    • Enroll up to 42 newly diagnosed AML or MDS patients
    • Phase 1: open-label, dose-escalating study
    • Phase 2: proof-of-concept cohort
    • Primary endpoints Phase 1: safety and tolerability
    • Primary endpoints Phase 2: efficacy
  • Ongoing Phase 2 clinical trial in CTCL (monotherapy)
    • Enroll up to 10 additional relapsed/refractory CTCL patients
    • Conducted at multiple centers in Europe
    • Primary endpoints: safety and efficacy
    • Secondary endpoints include: pharmacokinetics and immunogenicity
  • Concluding  Phase 1b clinical cohort in TCL patients
    • Phase 1b safety expansion study ongoing aiming to recruit up to 20 relapsed/refractory TCL patients
    • Conducted at multiple centers in Europe‚Äč
  • Ongoing Phase 1b cohort in NPC 
    • Safety expansion study ongoing
    • Active, not recruiting (UZ Gent, BE)
  • Clinical activity & safety demonstrated
    • Enrolled a total of 94 patients
    • Observed promising signs of biological activity in patients with a range of cancers including platinum refractory ovarian cancer, head-and-neck cancer, myoepithelial carcinoma, mesothelioma, gastric, adenoid cystic, pancreas, lung, and renal cell carcinomas, cervix adenocarcinoma, Hodgkin’s lymphoma, relapsed AML, Waldenstrom Macroglobulinemia and TCL, including in seven out of 13 cutaneous TCL, or CTCL, patients to date.

Preclinical data

  • ARGX-110 modes of action: killing tumor cells via enhanced ADCC and immune checkpoint blockade (MAbs 2014, Silence et al.)
  • ARGX-110 overcomes treatment resistance of leukemia stem cells in chronic myelogenous leukemia (Science Translational Medicine, Riether et al.)
 MAbs 2014, Silence et al.  Science Translational Medicine 2015, Riether et al.

Mode of action

  • Three potential modes of action: inhibition of tumor cell proliferation, elimination of tumor cells and prevention of tumor immune escape
  • SIMPLE Antibody™ potently blocking CD70
  • Highly potent cell killing properties through POTELLIGENT®


  • CD70: novel checkpoint
  • Highly specific to tumors, negligable expression on healthy cells
  • Therapeutic effect selective for cancer cells
 Adv Exp Med Biol 2009, Grewal et al.


  • T-cell Lymphoma (TCL) & cutaneous T-cell Lymphoma

    Lymphoma is the most common blood cancer. The two main forms of lymphoma are Hodgkin lymphoma and non-Hodgkin lymphoma (NHL). 

    Lymphoma is the most common type of blood cancer. The two main forms of lymphoma are Hodgkin’s lymphoma and non-Hodgkin’s lymphoma. Lymphoma occurs when lymphocytes, a type of white blood cell, grow and multiply uncontrollably. Cancerous lymphocytes can travel to many parts of the body, including the blood and bone marrow, giving rise to leukemias, and to lymph nodes, spleen, skin or other organs, forming a mass known as a tumor. The body has two main types of
    lymphocytes that can develop into lymphomas: B-cells and T-cells. Hodgkin’s lymphoma involves B-cells, while non-Hodgkin’s lymphoma may involve either B-cells or T-cells.

    TCL accounts for 6% of all cases of lymphoma and can be divided into subtypes such as PTCL, angioimmunoblastic TCL, anaplastic large cell lymphoma, or ALCL, and CTCL. These subtypes differ by location, distribution and aggressiveness of the primary tumor as well as by specific changes to the affected lymphocytes. Overall, there are approximately 7,900 new cases of TCL in the United States each year. According to the Cutaneous Lymphoma Foundation, the incidence of CTCL in the
    United States is approximately 3,000 new cases per year. The two most common types of CTCL are mycosis fungoides, representing approximately 50% of CTCL patients, and a more advanced form known as S´ ezary syndrome, representing
    approximately 15% of CTCL patients. In both mycosis fungoides and S´ ezary syndrome, visible skin lesions offer an ongoing means with which to monitor both the progression of disease and the impact of treatment. S´ ezary syndrome is distinguished by the presence of malignant lymphocytes in the blood, an extensive rash covering over 80% of the body and tumors visible on the skin.

  • AML & MDS

    Most cases of AML develop from cells that would turn into white blood cells (other than lymphocytes), but some cases of AML develop in other types of blood-forming cells.

    AML is a hematologic cancer characterized by excessive proliferation of myeloid stem cells and their failure to properly differentiate into mature white blood cells. AML is the second most common subtype of leukemia in adults. In the United States, AML has an incidence of approximately 22,000 new cases annually. AML is generally a disease of elderly people, with more than 60% of diagnosed patients being older than 60 years, and AML is uncommon before the age of 45. The average age of an AML patient is 67. The average five-year survival rate for patients with AML is 27%, but there are significant differences in prognosis depending on several factors, including the age of the patient at diagnosis. For patients under the age of 45, the five-year survival rate is approximately 57%, while for those over the age of 65 it is only 6%. There are likely multiple reasons for this discrepancy, including the ability of younger patients to tolerate more aggressive therapy.

    Current first-line treatments in AML typically involve aggressive chemotherapy, including alkylating agents and cytarabine potentially followed by stem cell transplantation, for younger patients with the aim to induce remission. This therapy is not recommended for older patients or patients with comorbidities, who are often treated with hypomethylating agents. We believe there is a significant need for safer, more effective AML treatments that can also be used in elderly patients. Because relapse is often due to leukemic stem cells present next to the bulk of malignant AML cells, or blasts, therapies targeting both blasts and leukemic stem cells may be more efficacious than chemotherapy only and could increase survival rates.

    MDS also affects bone marrow cells, reducing their ability to produce red and white blood cells or platelets. In the United States, MDS has an incidence of approximately 13,000 new cases annually. There are currently an estimated 60,000 MDS patients in the United States. Approximately 75% of MDS patients are older than 60 years of age when diagnosed, and, like with AML, as the
    population ages the disease prevalence is expected to rise. Some MDS patients are at high risk to develop AML and are treated in a similar way as AML patients.

  • Nasopharyngeal cancer (NPC)

    Nasopharyngeal carcinoma is cancer that occurs in the nasopharynx, which is located behind your nose and above the back of your throat. 

    Nasopharyngeal carcinoma is rare in the United States, where it accounts for approximately 1% of all childhood malignancies, in other parts of the world, specifically Southeast Asia, nasopharyngeal carcinoma occurs much more frequently. Whereas almost all adult nasopharyngeal cancers are carcinomas, only 35-50% of nasopharyngeal malignancies are carcinomas in children. (Source: American Cancer Society).