AXT-1003 / Axter Therap, Novartis - LARVOL DELTA

A first-in-class potent EZH2 degrader, AXT-1003, exhibits robust anti-tumor activity across multiple lymphomas and solid tumors (AACR 2026) - "Several S-adenosylmethionine (SAM)-competitive EZH2 inhibitors, which suppress methyltransferase activity of EZH2, have clinically shown promising results in treating sarcoma and lymphoma, including the FDA-approved tazemetostat (EPZ-6438)...Meanwhile, AXT-1003 had no significant effect on EZH2 at transcriptional levels, and its protein expression was restored by MG-132 (proteasome inhibitor)...Notably, in the patient-derived organoid (PDO) models from ovarian clear cell carcinoma (OCCC) samples, AXT-1003 displayed superior efficacy compared with cisplatin or mevrometostat (PF-06821497), regardless of ARID1A status...Furthermore, the in vivo combination treatment of AXT-1003 with enzalutamide showed synergistic efficacy in a prostate cancer (LNCaP) CDX mice model. In conclusion, our preclinical data highlight the therapeutic potential of AXT-1003 and support its ongoing clinical evaluation in patients with EZH2-driven cancers."

Clear Cell Carcinoma • Genito-urinary Cancer • Hematological Malignancies • Lymphoma • Oncology • Ovarian Cancer • Prostate Cancer • Sarcoma • Solid Tumor • ARID1A • EZH2

Study of AXT-1003 in Subjects With Advanced Malignant Tumors. (clinicaltrials.gov) - P1 | N=78 | Recruiting | Sponsor: Axter Therapeutics (Beijing) Co., Ltd | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Solid Tumor

Study of AXT-1003 in Adult Subjects With Relapsed/Refractory Non-Hodgkin Lymphomas (clinicaltrials.gov) - P1 | N=1 | Terminated | Sponsor: Axter Therapeutics (Beijing) Co., Ltd | Trial completion date: Jun 2024 ➔ Aug 2023 | Trial primary completion date: Jun 2024 ➔ Aug 2023

Trial completion date • Trial primary completion date • Hematological Malignancies • Non-Hodgkin’s Lymphoma • Oncology • ALK

Study of AXT-1003 in Adult Subjects With Relapsed/Refractory Non-Hodgkin Lymphomas (clinicaltrials.gov) - P1 | N=1 | Terminated | Sponsor: Axter Therapeutics (Beijing) Co., Ltd | N=79 ➔ 1 | Trial completion date: Jan 2026 ➔ Jun 2024 | Recruiting ➔ Terminated | Trial primary completion date: Dec 2025 ➔ Jun 2024; Due to the adjustment of the protocol and expansion of the indication population, a new clinical trial protocol was submitted to the Health Authority, so the registration was conducted as a new clinical trial and this trial was terminated.

Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Hematological Malignancies • Non-Hodgkin’s Lymphoma • Oncology • ALK

Study of AXT-1003 in Subjects With Advanced Malignant Tumors. (clinicaltrials.gov) - P1 | N=78 | Not yet recruiting | Sponsor: Axter Therapeutics (Beijing) Co., Ltd

Metastases • New P1 trial • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Solid Tumor

Study of AXT-1003 in Adult Subjects With Relapsed/Refractory Non-Hodgkin Lymphomas (clinicaltrials.gov) - P1 | N=79 | Recruiting | Sponsor: Axter Therapeutics (Beijing) Co., Ltd | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Malignancies • Non-Hodgkin’s Lymphoma • Oncology • ALK

Study of AXT-1003 in Adult Subjects With Relapsed/Refractory Non-Hodgkin Lymphomas (clinicaltrials.gov) - P1 | N=79 | Not yet recruiting | Sponsor: Axter Therapeutics (Beijing) Co., Ltd

New P1 trial • Hematological Malignancies • Non-Hodgkin’s Lymphoma • Oncology • ALK