News & Events
The 31st Annual Congress of the European Hematology Association (EHA) was held in Stockholm, Sweden, started on June 11, 2026. This year's congress brought together top experts and scholars in the global hematology field. For the first time， Lupeng Pharmaceutical reported the single-agent efficacy data of rocbrutinib for the treatment of relapsed or refractory Primary Central Nervous System lymphoma (R/R PCNSL). Lupeng Pharmaceutical also updated the efficacy and safety data of rocbrutinib after prolonged follow-up in the ROCK-1 study.
Poster 1．FIRST REPORT OF ROCBRUTINIB FOR THE TREATMENT OF PATIENTS WITH RELAPSED OR REFRACTORY PRIMARY CENTRAL NERVOUS SYSTEM LYMPHOMA
Primary central nervous system lymphoma (PCNSL) is a lymphoma arising in the brain, spinal cord, cerebrospinal fluid, or eyes. It represents 4%–6% of all extranodal lymphomas, with approximately 90% classified histopathologically as diffuse large B-cell lymphoma (DLBCL). This disease is highly aggressive, with limited treatment options and poor prognosis, particularly in relapsed or refractory (R/R) cases. Although Bruton’s tyrosine kinase inhibitors (BTKi) have demonstrated clinical activity, outcomes remain suboptimal. Rocbrutinib, a highly selective fourth-generation covalent and non-covalent BTKi, has shown promising efficacy in non-GCB DLBCL and exhibits good blood-brain barrier penetration in mice. This report presents the preliminary clinical data from rocbrutinib-treated patients with R/R PCNSL.
We report the PCNSL subgroup results from a rocbrutinib phase 1 study (NCT04993690) conducted in China.
In this open-label phase 1 study, eligible subjects had a PCNSL diagnosis and had received at least one prior therapy, including a high-dose methotrexate-based regimen; prior BTKi exposure was permitted, including patients who were refractory to covalent BTKi. Subjects received rocbrutinib tablets at 200 mg once daily as monotherapy until disease progression or unacceptable toxicity. Adverse events (AEs) were graded according to CTCAE v5.0. Cerebrospinal fluid (CSF) and peripheral blood were collected for pharmacokinetic analysis. Efficacy was assessed via gadolinium-enhanced magnetic resonance imaging (MRI) using the International PCNSL Collaborative Group Response Criteria.
As of 31 December 2025, nine subjects were enrolled, all with a pathological diagnosis of non-GCB DLBCL. The median age was 63 years (range, 49-71). The median number of prior lines of therapy was 2 (range, 1-4); eight (88.9%) subjects had refractory disease. Three subjects had prior covalent BTKi exposure, all of whom discontinued treatment due to disease progression. The median International Extranodal Lymphoma Study Group (IELSG) score was 3 (range, 1-4).
CSF samples were collected 1 to 5 hours post-dose at steady-state. The median rocbrutinib concentration was 5.71 ng/mL (range, 2.89–8.21 ng/mL), which is approximately 40- to 161-fold higher than its in vitro potency (IC50), confirming effective blood-brain barrier penetration by rocbrutinib.
After a median treatment duration of 3.0 months (range, 1.9-4.7), the overall response rate (ORR) was 88.9%, with a complete response (CR)/unconfirmed complete response (CRu) rate of 22.2%. One subject achieved stable disease, with a 34.8% reduction in target lesions after one month of treatment. Six patients are still on treatment.
The most common treatment-related AEs (TRAEs) (any grade; ≥grade 3) with an incidence ≥20% were neutrophil count decreased (33.3%; 22.2%), white blood cell count decreased (33.3%; 0), hypertriglyceridemia (22.2%; 0), and platelet count decreased (22.2%; 0); most events were grade 1. All TRAEs were grade 1 or 2 except for two cases of grade 3 neutrophil count decreased. No serious AEs (SAEs) or AEs leading to dose reduction, interruption, discontinuation, or death were reported.
Rocbrutinib demonstrated effective blood-brain barrier penetration and encouraging single agent activity in patients with PCNSL, along with a manageable safety profile. These findings support its potential for treating R/R PCNSL.
1st Author: Lin Fu：Beijing Tiantan Hospital, Capital Medical University, Beijing, China
Abstract number: EHA-5004, PF1033
Place and time of presentation: Poster Session 1, other aggressive lymphomas, on Friday, June 12 (18:45 - 19:45 CEST)
Abstract Link:
Poster 2. UPDATED EFFICACY AND SAFETY RESULTS OF ROCBRUTINIB FROM THE PHASE 2 ROCK-1 STUDY IN PATIENTS WITH RELAPSED OR REFRACTORY MANTLE CELL LYMPHOMA AND PREVIOUSLY TREATED WITH BTK INHIBITOR
Rocbrutinib is a novel, fourth generation BTK inhibitor that incorporates both covalent (non-reversible) and non-covalent (reversible) binding mechanisms. In the primary analysis of the Phase 2 ROCK-1 Trial (NCT05716087), rocbrutinib demonstrated clinically meaningful and durable efficacy with manageable safety profile in heavily pretreated, cBTKi-exposed Chinese patients with relapsed or refractory mantle cell lymphoma (R/R MCL) (ASH 2025, abs25-7939).
Here we present updated efficacy and safety results from the ROCK-1 trial after prolonged follow-up (median, 19.49 months; range, 0.62-28.65). Eligible patients received oral rocbrutinib (150 mg once daily) until disease progression or unacceptable toxicity. The primary endpoint was IRC-assessed overall response rate (ORR) per Lugano 2014 criteria. Key secondary endpoints included duration of response (DOR), progression-free survival (PFS), overall survival (OS), and safety. Adverse events (AEs) were graded according to CTCAE v5.0.
As of Jan 05, 2026, among the 61 R/R MCL patients, the median treatment duration was 168 days (range, 9-813), the maximum number of treatment cycles received was 29 (28 days per cycle). Per IRC’s assessment, ORR was 63.9% (95% CI, 50.6-75.8), including 23.0% complete response (CR). Median PFS and DOR were 7.39 months (95% CI, 3.71–18.30) and 16.46 months (95% CI, 8.25–not reached), respectively. The efficacy results per investigator were consistent with IRC’s results, showing an ORR of 62.3% (95% CI, 49.0–74.4) with CR rate of 26.2%, a median PFS of 5.52 months (95% CI, 3.71–12.68), and a median DOR of 16.46 months (95% CI, 5.55–22.14). After a median follow-up for OS of 23.36 months (range, 0.62-28.65), the median OS was not reached (95% CI, 15.97-not reached), corresponding to an estimated 24-month OS rate of 61.4%.
Treatment emergent adverse events (TEAEs) were predominantly Grade 1 or 2，and no atrial fibrillation or flutter was reported. No TEAEs leading to permanent treatment discontinuation or death.
With extended follow-up, rocbrutinib still showed durable efficacy and favorable safety in patients with R/R MCL. A randomized phase 3 confirmatory trial of rocbrutinib versus investigator’s choice of BTK inhibitors in patients with R/R MCL is ongoing (NCT07377578).
1st Author: Yuqin Song，Peking University Cancer Hospital, Beijing, China,
Abstract number: EHA-1354, PF945
Place and time of presentation: Poster Session 1, Indolent and mantle-cell non-Hodgkin lymphoma - Clinical, on Friday, June 12 (18:45 - 19:45 CEST)
Abstract Link: