“We have previously observed evidence of biological activity and a promising safety profile in several CD70-positive CTCL patients treated in an ongoing Phase 1 safety-expansion cohort, resulting in several patients with partial response or stable disease,” Nicolas Leupin, CMO of argenx, said in a news release.
“Based on these preliminary results, we opted to further evaluate ARGX-110 as a monotherapy in an exploratory Phase 2 study to demonstrate the intrinsic activity of the drug in relapsed/refractory CTCL patients and to broaden our efficacy database,” he said.
Leupin said this is the second Phase 1/2 study of ARGX-110; the first was launched in December 2016 as a combination therapy with standard of care in acute myeloid leukemia (AML).
Argenx uses its SIMPLE Antibody platform that allows researchers to identify novel and complex targets that could be used to treat patients with cancer and severe autoimmune diseases. Argenx then takes advantage of the diversity of the llama immune system to generate high-affinity antibodies with longer duration effects.
ARGX-110 is one of argenx’s antibodies developed using this technology. It targets the CD70 protein, which is thought to hamper the immune cell’s activity in hematological malignancies.
But in addition to restoring the immune surveillance against tumors, preclinical studies also showed that ARGX-110 blocks tumor growth and directly kills cancer cells.
The new Phase 2 trial evaluating ARGX-110 in relapsed or refractory cutaneous T-cell lymphoma will enroll up to 10 patients, and will be conducted in multiple centers in Europe.
The study’s primary endpoints are safety and effectiveness. Secondary goals include pharmacokinetics (how a drug is processed in the body, including its distribution, metabolism, and excretion), and immunogenicity. The company expects to collect interim data by the end of the year, and report topline data in the second half of 2018.
Argenx is also evaluating ARGX-110 in a Phase 2 trial (NCT03030612) for newly diagnosed acute myeloid leukemia (AML) and high-risk myelodisplastic syndrome (MDS). The study will enroll up to 36 patients who will receive ARGX-110 in combination with Vidaza (azacytidine).
Another Phase 1 trial (NCT02759250) is testing ARGX-110 as either monotherapy or combination therapy in patients with nasopharyngeal carcinoma, in various stages of disease. The study is still enrolling and is expected to recruit up to 15 patients.