Golidocitinib approved in China as first-in-class JAK1 only inhibitor for the treatment of relapsed or refractory peripheral T-cell lymphoma

PTCL is a heterogeneous group of aggressive T-cell/natural killer (NK) cell non-Hodgkin lymphomas (NHL). Patients with PTCL face an extremely high risk of disease relapse even if they achieved tumor remission following first-line conventional therapy. The outcome for relapsed or refractory patients is extremely poor, with a 3-year survival rate of 23% and a median overall survival (mos.) of 5.8 months. Although couple of drugs have been granted conditional approval by regulatory agencies in the relapsed or refractory setting, their single agent activities have been modest, with objective response rates (ORRs) lower than 30%.

Golidocitinib was approved based on findings from JACKPOT8 Part B (JACKPOT8B) study, the multinational pivotal study to evaluate the efficacy and safety of golidocitinib in r/r PTCL as a monotherapy. The primary endpoint was the objective response rate (ORR), assessed by an independent review committee (IRC). Full analysis of the study was simultaneously published in The Lancet Oncology and presented in oral session at the 65th American Society of Hematology Annual Meeting and Exposition.

In JACKPOT8B study, golidocitinib demonstrated superior and durable antitumor efficacy and a favorable safety profile in r/r PTCL patients, compared with existing treatment options. At the data cut-off date August 31, 2023, the ORR was 44.3% including a complete response (CR) rate of 23.9% per IRC. Tumor responses were observed across various PTCL subtypes. The median duration of response (mDoR) was 20.7 months and 53.8% of patients were still responding.

Dizal was the first to identify and validate targeting the JAK/STAT pathway as a promising therapeutic approach for PTCL, leading to the development of golidocitinib as the world’s first JAK1 only inhibitor. With greater than 200 to 400-fold selectivity over other JAK family members and ideal pharmacokinetic properties, golidocitinib exerts potent antitumor efficacy with a favorable safety profile.

With superior efficacy and safety profile, golidocitinib has been widely acknowledged at prestigious international congresses including ASCO, EHA, ICML, and ASH, with six oral presentations over four consecutive years. The results of the multinational study JACKPOT8 were published in Annals of Oncology and The Lancet Oncology.

At the data cut-off date of August 31, 2023, golidocitinib has demonstrated robust and durable anti-tumor efficacy, with an ORR of 44.3%. More than 50% of the patients with tumor remission achieved a complete response with a CRR of 23.9%. Per IRC assessment, mDoR reached 20.7 months. Golidocitinib was granted Fast Track Designation by the FDA for the treatment of r/r PTCL in February 2022. In September 2023, the CDE accepted its NDA and granted Priority Review for the treatment of r/r PTCL. The Phase I clinical data of golidocitinib (JACKPOT8 PART A) was published in Annals of Oncology (Impact Factor: 51.8), and global pivotal trial data of golidocitinib for the treatment of r/r PTCL (JACKPOT PART B) was published in The Lancet Oncology (Impact Factor: 54.4).

See-Annals of Oncology{ VOLUME 34, ISSUE 11, P1055-1063, NOVEMBER 2023,."Phase I dose escalation and expansion study of golidocitinib, a highly selective JAK1 inhibitor, in relapsed or refractory peripheral T-cell lymphomas". Y. Song, D.H. Yoon, H. Yang, .Z. Yang. W, S. Kim, J. Zhu. Published:September 04, 2023DOI:https://doi.org/10.1016/j.annonc.2023.08.013.

See- The Lancet: VOLUME 25, ISSUE 1, P117-125, JANUARY 2024 "Golidocitinib, a selective JAK1 tyrosine-kinase inhibitor, in patients with refractory or relapsed peripheral T-cell lymphoma (JACKPOT8 Part B): a single-arm, multinational, phase II study". Prof Yuqin Song, MD ,Luis Malpica, MD , Prof Qingqing Cai, MD, Prof Weili Zhao, MD, Prof Keshu Zhou, MD, Prof Jianqiu Wu, MD , et al. Published: December 09, 2023DOI:https://doi.org/10.1016/S1470-2045(23)00589-2.