China Approves First-in-Class JAK1 Inhibitor Golidocitinib for Relapsed/Refractory Peripheral T-Cell Lymphoma

Dizal, a biopharmaceutical company, has announced that the National Medical Products Administration of China has approved golidocitinib for treating adult patients with relapsed or refractory (r/r) peripheral T-cell lymphoma (PTCL). This approval marks golidocitinib as the first and only Janus kinase 1 (JAK1) selective inhibitor available for r/r PTCL patients worldwide.

Peripheral T-cell lymphoma (PTCL) is an aggressive type of non-Hodgkin lymphoma that originates from T-cells and natural killer cells. Patients with PTCL often face a high likelihood of relapse even after achieving remission with initial treatments. The prognosis for patients with relapsed or refractory PTCL is generally poor, with a three-year survival rate of only 23% and a median overall survival of 5.8 months. Although some drugs have received conditional approval for relapsed or refractory PTCL, their effectiveness has been limited, with response rates below 30%.

The approval of golidocitinib was based on results from the multinational pivotal JACKPOT8B study, which evaluated the drug's efficacy and safety as a monotherapy for r/r PTCL. This study's primary endpoint was the objective response rate (ORR), assessed by an independent review committee. The study's findings were concurrently published in The Lancet Oncology and presented at the 65th American Society of Hematology Annual Meeting and Exposition.

In the JACKPOT8B study, golidocitinib showed superior and lasting antitumor efficacy with a favorable safety profile compared to existing treatment options for r/r PTCL patients. As of August 31, 2023, the ORR was 44.3%, with a complete response rate of 23.9%. Tumor responses were observed across various PTCL subtypes, and the median duration of response was 20.7 months, with 53.8% of patients still responding.

Dr. Jun Zhu from the Peking University Cancer Hospital and Institute, the study's leading principal investigator, highlighted golidocitinib's novel mechanism and unique molecular design. He emphasized that the drug achieved an ORR of 44.3% and a duration of response of 20.7 months in r/r PTCL patients. Its approval provides doctors with a much-needed treatment option for PTCL.

Dizal has been a pioneer in targeting the JAK/STAT pathway as a therapeutic approach for PTCL, leading to the development of golidocitinib. The drug's selectivity over other JAK family members and its favorable pharmacokinetic properties contribute to its potent antitumor efficacy.

CEO of Dizal, Xiaolin Zhang, expressed enthusiasm about bringing golidocitinib to patients in China and underscored the company's commitment to developing first-in-class and groundbreaking medicines to address unmet medical needs globally. Golidocitinib has also been granted Fast Track Designation by the U.S. FDA and is undergoing expedited global development.

The drug has gained international recognition at prestigious congresses such as ASCO, EHA, ICML, and ASH, with multiple oral presentations over consecutive years. The results of the JACKPOT8 study have been published in prominent journals like Annals of Oncology and The Lancet Oncology.

As of June 2024, golidocitinib remains the first and only JAK1 selective inhibitor approved for treating r/r PTCL. The drug's robust and durable anti-tumor efficacy, with an ORR of 44.3% and a complete response rate of 23.9%, highlights its significant clinical benefit for patients with this challenging condition.