jaktinib / Suzhou Zelgen - LARVOL DELTA

Long-term treatment continuation of gecacitinib in myelofibrosis patients after ruxolitinib failure (ASH 2025) - "Thirty-nine patients (46.4%) entered a compassionate use program after study completion.As of June 25, 2025, with a median follow-up of 3.8 years, 31 patients (36.9%) remained on gecacitinib.The median treatment duration was 22.4 months (range 0.1–60.3) for the entire cohort; 23.0 months(range 0.1–53.0) for patients starting at 100 mg BID; 23.0 months (range 0.1–60.3) for ruxolitinib-intolerant patients; and 18.3 months (range 3.2-47.0) for ruxolitinib-refractory/relapsed patients.Additionally, regression analysis demonstrated that treatment duration was significantly positivelycorrelated with the percentage reduction in spleen volume from baseline at week 24.ConclusionsGecacitinib demonstrates durable treatment continuation (median >18 months across subgroups) in MFpatients after ruxolitinib failure. These long-term data support its viability as a therapeutic option forruxolitinib-intolerant or refractory/relapsed MF patients."

Clinical • Myelofibrosis • Myeloproliferative Neoplasm • CALR

Effects of golidocitinib and gecacitinib in mouse models of primary and secondary hemophagocytic lymphohistiocytosis (ASH 2025) - "Golidocitinib and gecacitinib demonstrate favorable preclinical tolerability and efficacy inHLH. The highly selective JAK1 inhibitor golidocitinib exhibits broadly superior therapeutic effects togecacitinib and ruxolitinib in both HLH subtypes."

Preclinical • Cytomegalovirus Infection • Hematological Disorders • Hemophagocytic lymphohistiocytosis • Immunology • Rare Diseases • CD8 • IFNG • IL10 • PRF1 • TNFA • TYK2

Ferritin change and predictors of anemia response in ruxolitinib-intolerant myelofibrosis patients treated with gecacitinib (ASH 2025) - "Themedian treatment duration was 3.8 years (range 0.3 to 5.0) for responders and 1.5 years (range 0.0 to 4.3)for non-responders (P = 0.0039).ConclusionsIn our study, JAK2 mutation was significantly associated with anemia response to gecacitinib inruxolitinib-intolerant MF patients. Ferritin decreased rapidly in patients achieving anemia response.Anemia responders showed significantly higher spleen response and longer treatment duration, withnumerically higher symptom response."

Clinical • Anemia • Hematological Disorders • Myelofibrosis • ACVR1 • JAK2

Efficacy and safety of gecacitinib in thrombocytopenic myelofibrosis patients (ASH 2025) - "Amatching-adjusted indirect comparison of GCA versus ruxolitinb (presented on 2025 EHA congress; PS1839) reported GCA offered a trend of higher splenic benefits and was associated with significantly lessgrade 3/4 anemia and neutropenia compared with ruxolitinib (RUX). To evaluate the efficacy and safetyof GCA in patients with MF and thrombocytopenia (platelet counts <100 × 109/L), we conducted a post-hoc analyses with pooled data sets from 4 clinical studies: ZGJAK016 (phase 3; GCA versus hydroxyurea; JAK inhibitor naïve), ZGJAK002 (phase 2; GCA optimal dosing frequency exploration; JAK inhibitor naïve),ZGJAK006 (phase 2; GCA single arm; intolerant to RUX), and ZGJAK017 (phase 2; GCA single arm; refractory or relapsed to RUX).MethodsDetailed study designs for these studies have been published...A median duration of treatment was 17.4months (range, 0.5-69.4 months).The most common (occurring in ≥10% of patients) treatment-emergent adverse events during..."

Clinical • Hematological Disorders • Myelofibrosis • Neutropenia • Thrombocytopenia • ACVR1

Efficacy, Safety, and Survival Findings after Long-Term Follow-up from ZGJAK002, a Phase 2 Study Comparing Jaktinib 100 Mg Twice Daily with 200 Mg Once Daily for Myelofibrosis (MF) (ASH 2023) - "Background: Jaktinib, a novel Janus kinase (JAK) and ACVR1 inhibitor, demonstrated promising clinical benefit with good tolerability, not only efficiently shrinking the spleen and reducing symptom burdens, but also improving anemia, in JAK-inhibitor naïve, ruxolitinib-intolerant or -resistant MF patients. With over 30-month follow-up, jaktinib at doses of 100 mg BID and 200 mg QD demonstrated durable high effective rates and improved survival rates without new safety issue. The long-term data further supports the recommendation of 100 mg BID over 200 mg QD administration. It also concluded that jaktinib remains the treatment strategy that offers excellent chances for improving survival in MF patients."

Clinical • P2 data • Anemia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Myelofibrosis • Oncology • Pneumonia • Respiratory Diseases • Thrombocytopenia • ACVR1

Post Hoc Analysis of Phase III Gecacitinib Clinical Trial Outcomes: Enhanced Understanding and Implications (ASH 2024) - "Regarding anemia, gecacitinib enhanced transfusion independence as evidenced by a higher transfusion independence rate at Week 24 compared to hydroxyurea, with comparable baseline characteristics. The ZGJAK016 study validates the critical role of gecacitinib in addressing the core treatment needs of myelofibrosis patients."

Clinical • P3 data • Retrospective data • Anemia • Fatigue • Hematological Disorders • Musculoskeletal Pain • Myelofibrosis • Pain • Pruritus

A prospective, single-center, single-arm, pilot study evaluating the efficacy and safety of jaktinib combined with dexamethasone in adult patients with newly diagnosed hemophagocytic lymphohistiocytosis (ChiCTR) - P2 | N=20 | Not yet recruiting | Sponsor: The FIrst Affiliated Hospital, College of Medicine, Zhejiang University; The First Affiliated Hospital of Zhejiang University School of Medicine

New P2 trial • Hemophagocytic lymphohistiocytosis • Immunology • Rare Diseases

GRIT: Study of Gecacitinib in the Treatment of Acute Graft-Versus-Host Disease After Failure of Ruxolitinib-containing Second-line Therapy (clinicaltrials.gov) - P2 | N=15 | Not yet recruiting | Sponsor: Ruijin Hospital

New P2 trial • Acute Graft versus Host Disease • Graft versus Host Disease • Immunology

Efficacy and safety of Jaktinib tablet in Chinese adults with severe alopecia areata: a randomized, double-blind, multi-center, phase 3 trial (EADV 2025) - "Jaktinib presents a promising treatment option for adults with severe AA."

Clinical • P3 data • Alopecia • Cardiovascular • Dyslipidemia • Hepatology • Immunology • Infectious Disease • Liver Failure • Respiratory Diseases

Study of Gecacitinib-corticosteroid as First-line Therapy for Grade II-IV Acute Graft Versus Host Disease (clinicaltrials.gov) - P2 | N=25 | Not yet recruiting | Sponsor: Bin Gu

New P2 trial • Acute Graft versus Host Disease • Bone Marrow Transplantation • Graft versus Host Disease • Immunology • Transplantation

Gecacitinib Combined With Donafenib and PD-1 Inhibitor as Immune Rechallenge Therapy for Unresectable Hepatocellular Carcinoma (clinicaltrials.gov) - P2 | N=35 | Not yet recruiting | Sponsor: Tianjin Medical University Cancer Institute and Hospital

New P2 trial • Hepatocellular Cancer • Oncology • Solid Tumor

Suzhou Zelgen Biopharmaceuticals…announced that the…NMPA has approved its clinical trial for ZG005 in combination with gecacitinib hydrochloride tablets and chemotherapy for advanced solid tumors (flcube.com)

New trial • Solid Tumor

Study of ZG005 in Combination With Gecacitinib and Chemotherapy for Participants With Advanced Solid Tumors (clinicaltrials.gov) - P1/2 | N=153 | Not yet recruiting | Sponsor: Suzhou Zelgen Biopharmaceuticals Co.,Ltd

New P1/2 trial • Solid Tumor

Effect of gecacitinib hydrochloride tablets on immune function in patients with myelofibrosis (ChiCTR) - P=N/A | N=50 | Not yet recruiting | Sponsor: West China Hospital of Sichuan University; West China Hospital of Sichuan University

New trial • Myelofibrosis

Study of ZG005 in Combination With Gecacitinib in Participants With Advanced Cervical Cancer (clinicaltrials.gov) - P1/2 | N=120 | Not yet recruiting | Sponsor: Suzhou Zelgen Biopharmaceuticals Co.,Ltd

New P1/2 trial • Cervical Cancer • Oncology • Solid Tumor

INDIRECT TREATMENT COMPARISON OF GECACITINIB VS RUXOLITINIB IN MYELOFIBROSIS (EHA 2025) - "Overall, gecacitinib appears to show potential benefits in efficacy and safety compared to ruxolitinib in preliminary analyses of COMFORT-I, and SIMPLIFY-I studies. These findings suggest that gecacitinib may offer advantages in reducing AEs such as anemia and neutrophil count reduction, as well as lower discontinuation rates in JAK inhibitor-naïve patients with MF. However, these observations require confirmation through direct head-to-head studies."

Anemia • Hematological Disorders • Myelofibrosis • Thrombocytopenia

GECACITINIB IN JAKI-NAÏVE AND JAKI-EXPOSED MYELOFIBROSIS PATIENTS: IMPROVED TRANSFUSION INDEPENDENCE (EHA 2025) - "A total of 137 JAKi-naïve patients and 79 JAKi-exposed patients (45 ruxolitinib-intolerant and 34 ruxolitinib-relapsed/refractory) were included in the study. Gecacitinib demonstrated clinically meaningful TI conversion rates and prolonged transfusion-free intervals in both JAKi-naïve and JAKi-exposed patients with MF, with higher efficacy observed in JAKi-naïve patients. These outcomes were consistent regardless of the hemoglobin threshold used. This study supports gecacitinib as a promising therapy for anemia management in MF, highlighting the importance of early intervention."

Clinical • Anemia • Hematological Disorders • Inflammation • Myelofibrosis • ACVR1 • JAK1 • JAK2 • JAK3 • TYK2

EXPLORATORY ANALYSIS OF GECACITINIB IN RUXOLITINIB-INTOLERANT MYELOFIBROSIS PATIENTS: IMPACT OF BASELINE ANEMIA AND PRIOR RUXOLITINIB TREATMENT DURATION ON RESPONSE TO GECACITINIB (EHA 2025) - P2 | "In RUX-intolerant patients with anemia, GCA treatment resulted in comparable splenic benefits across anemia subgroups and led to a reduced transfusion rate. Notably, patients with shorter durations of prior RUX exhibited a numerically higher SVR35 rate at week 24. Furthermore, GCA demonstrated a dual therapeutic effect, improving anemia and reducing spleen volume."

Clinical • Anemia • Hematological Disorders • Myelofibrosis • ACVR1

AN EXPLORATORY ANALYSIS OF SYMPTOMATIC BENEFIT IN PATIENTS TREATED WITH GECACITINIB: A FOCUS ON THOSE INTOLERANT OR REFRACTORY/RELAPSED TO RUXOLITINIB (EHA 2025) - "These results suggest that patients who were intolerant or refractory/relapsed to RUX experienced symptomatic improvement and a dual response of SVR35 and TSS50 after treatment with gecacitinib."

Clinical • Anemia • Fatigue • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Thrombocytosis

GECACITINIB-INDUCED SPLEEN VOLUME REDUCTION (SVR) AND ITS PROGNOSTIC VALUE FOR OVERALL SURVIVAL (OS) IN MYELOFIBROSIS (EHA 2025) - "In patients with MF treated with gecacitinib, achieving a certain degree of splenic response is associated with improved survival outcomes. Additionally, lower risk and higher baseline hemoglobin values are also linked to better survival."

Clinical • Fibrosis • Hematological Disorders • Immunology • Myelofibrosis • ACVR1 • JAK2

GECACITINIB THERAPY'S IMPACT ON SYMPTOM BURDEN IN THE ZGJAK016 STUDY (EHA 2025) - "The study enrolled 105 patients with intermediate-2 or high risk MF, 71 on gecacitinib and 34 on hydroxyurea (HU). Gecacitinib has shown promising results in maintaining a longer duration of therapeutic effects compared to HU. The benefits of gecacitinib are observed across different subgroups and at various time points, providing a consistent therapeutic advantage. Analysis results reveal that gecacitinib treatment has yielded substantial and clinically significant improvements in MF symptom burden and severity in patients with intermediate or high-risk MF."

Myelofibrosis • ACVR1

Gecacitinib for cGVHD: Safety and Efficacy in Patients After ≥2 Lines of Prior Therapy (clinicaltrials.gov) - P1/2 | N=24 | Recruiting | Sponsor: Yujun DONG

New P1/2 trial • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology

An exploratory analysis of myelofibrosis (MF) patient subgroups by baseline hemoglobin levels in the gecacitinib phase 3 trial. (ASCO 2025) - P3 | "In the double-blind, randomized phase 3 ZGJAK016 trial, gecacitinib (GCA), a dual JAK/ACVR1 inhibitor, demonstrated superior spleen response over hydroxyurea, and a trend toward improvement in constitutional symptom and anemia associated with MF in JAK inhibitor-naive patients (pts). In summary, GCA delivers a threefold benefit in terms of spleen and symptom management, as well as anemia, to JAK inhibitor-naive pts with MF who have mild, moderate/severe, or no anemia at baseline, particularly those with Hb levels below LLN."

Clinical • P3 data • Anemia • Hematological Disorders • Myelofibrosis • ACVR1

The Safety and Efficacy of Gecacitinib in the Long-term Treatment of Idiopathic Pulmonary Fibrosis (ATS 2025) - "The main TEAEs occurred in long-term treatment period (with incidence >10% in any group) were anemia , hepatic function abnormal ,blood lactate dehydrogenase increased ,pneumonia, hypercholesterolemia, hyperuricemia, upper respiratory tract infection, sinus tachycardia, proteinuria etc. Conclusions The long-term administration of gecacitinib demonstrated sustained efficacy results, with notably better outcomes observed in patients who continued its use compared to those who switched from placebo to gecacitinib. Moreover, gecacitinib has an excellent tolerability and safety profile."

Clinical • Anemia • Cardiovascular • Dyslipidemia • Hematological Disorders • Idiopathic Pulmonary Fibrosis • Immunology • Infectious Disease • Metabolic Disorders • Otorhinolaryngology • Pneumonia • Pulmonary Disease • Renal Disease • Respiratory Diseases • Sinusitis

Managing Myelofibrosis: Matching Advances in Treatments With Clinical Unmet Needs. (PubMed, Hematol Oncol) - "The janus kinase inhibitor (JAKi) ruxolitinib has been the mainstay of treatment for over a decade...Other JAKi (pacritinib, momelotinib, jaktinib) address treatment-related cytopenia, expanding the therapeutic utility of this class of agents to patients with baseline anemia or thrombocytopenia. Novel candidates exploit multiple molecular pathways, and offer the potential to improve the management of MF-associated cytopenia (imetelstat, pelabresib, navitoclax, selinexor, luspatercept, sotatercept, elritercept, LCL161, bomedemstat) and recover bone marrow fibrosis (imetelstat, pelabresib, navitoclax and bomedemstat). It remains to be seen if these newer agents can induce any remission in MF and enable patients to come off therapy, but the future is beginning to look much brighter."

Journal • Review • Fibrosis • Hematological Disorders • Immunology • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Thrombocytopenia