mesutoclax (ICP-248) / InnoCare - LARVOL DELTA

BTK and BCL2 targeting with orelabrutinib (ICP-022) and mesutoclax (ICP-248): single-agent and combination activity in marginal zone lymphoma, including drug-resistant derivatives (EORTC-NCI-AACR 2026) - "Abstract will be available as of 4 November (with consent of the author)"

Hematological Malignancies • Lymphoma • Marginal Zone Lymphoma • Oncology • BCL2

InnoCare Announces Approval of Head-to-Head Registrational Phase III Trial of Mesutoclax with Azacitidine versus Venetoclax with Azacitidine in Treatment-Naive AML in China (GlobeNewswire) - "The head-to-head randomized, open-label, multicenter registrational Phase III clinical trial is designed to demonstrate the superiority of mesutoclax in combination with azacitidine over venetoclax with azacitidine in elderly or unfit patients with treatment naive AML. The primary endpoint is overall survival (OS)."

New P3 trial • Acute Myelogenous Leukemia

InnoCare Announces Approval of Head-to-Head Registrational Phase III Trial of Mesutoclax with Azacitidine versus Venetoclax with Azacitidine in Treatment-Naïve AML in China (GlobeNewswire) - "The head-to-head randomized, open-label, multicenter registrational Phase III clinical trial is designed to demonstrate the superiority of mesutoclax in combination with azacitidine over venetoclax with azacitidine in elderly or unfit patients with treatment naive AML. The primary endpoint is overall survival (OS)."

New P3 trial • Acute Myelogenous Leukemia

A Phase 3 Study to Compare the Efficacy of ICP-248 in Combination With Orelabrutinib Versus Pirtobrutinib in Participants With Relapsed or Refractory Mantle Cell Lymphoma (r/r MCL). (clinicaltrials.gov) - P3 | N=280 | Not yet recruiting | Sponsor: Beijing InnoCare Pharma Tech Co., Ltd.

New P3 trial • Hematological Malignancies • Lymphoma • Mantle Cell Lymphoma • Oncology

InnoCare Pharma…announced today that orelabrutinib, in combination with mesutoclax (ICP-248), has been granted Breakthrough Therapy Designation (BTD) by the Center for Drug Evaluation (CDE) of the…NMPA for the treatment of patients with marginal zone lymphoma (MZL) who have received at least one prior therapy (PRNewswire) - "Data presented at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting demonstrated that orelabrutinib in combination with mesutoclax achieved excellent efficacy and safety in patients with MZL who had received at least one prior therapy, with an overall response rate (ORR) of 100%."

Breakthrough therapy • Non-US regulatory • Marginal Zone Lymphoma

Safety, tolerability, and efficacy of mesutoclax (ICP-248) in combination with azacitidine in patients with myeloid malignancies. (ASCO 2026) - P1 | "Clinical Trial Registration Number: NCT06656494 Background: Venetoclax has been approved for the treatment of newly diagnosed acute myeloid leukemia (AML) in elderly or unfit pts but failed in the high-risk myelodysplastic syndromes (MDS). The combination of mesutoclax and azacitidine demonstrated a favorable safety profile and encouraging anti-tumor activity not only in AML but also in MDS pts, supporting its continued development for the treatment of myeloid malignancies. These preliminary results warrant further investigation in larger, randomized trials."

Clinical • Combination therapy • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology • Thrombocytopenia

Efficacy and safety of mesutoclax (ICP-248) in combination with orelabrutinib in patients with B-cell malignancies: A pooled analysis. (ASCO 2026) - P1, P2/3 | "Mesutoclax in combination with orelabrutinib demonstrated a tolerable safety profile across B cell malignancy subtypes (MCL, MZL, CLL/SLL). Significant 100% ORR and deep response were observed in patients receiving mesutoclax 125mg combined with orelabrutinib. This all oral, chemo-free regimen has the potential to establish a novel therapeutic option for B-NHLs."

Combination therapy • IO biomarker • Retrospective data • Chronic Lymphocytic Leukemia • Hematological Malignancies • Marginal Zone Lymphoma • Oncology • TP53

ASCO 2026 | Updated Data from an Oral Presentation of InnoCare's Novel BCL2 Inhibitor Mesutoclax in MDS and AML Released (GlobeNewswire) - "As of April 20, 2026, among evaluable treatment-naïve (TN) MDS patients, the overall response rate (ORR) per IWG 2006 criteria was 100%, including a complete response (CR) rate of 40%, and marrow CR rate of 60%. The composite CR rate was 90% per IWG 2023 criteria, including a CR rate of 60%. As of April 13, 2026, among the evaluable TN AML patients, 81.8% achieved composite CR (cCR, CR+CRi), and 86.5% were MRD (Minimal Residual Disease) negative. Among cCR responders, 83% achieved cCR in the first treatment cycle, demonstrating that the mesutoclax regimen enables rapid and deep remissions. In the 125mg mesutoclax group, the 6-month duration of response (DOR) rate and overall survival (OS) rate were 93.3% and 90.5% respectively."

P1 data • Acute Myelogenous Leukemia • Myelodysplastic Syndrome

Efficacy and safety of mesutoclax (ICP-248) in combination with orelabrutinib in patients with B-cell malignancies: A pooled analysis (Abstract No.: 7073) (GlobeNewswire) - "The study enrolled patients with R/R mantle cell lymphoma (MCL), R/R marginal zone lymphoma (MZL), and TN chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL)...All treatment groups achieved an overall response rate (ORR) of 100% and a high complete response rate (CRR). Deep response was observed in patients receiving mesutoclax 125mg combined with orelabrutinib...Among MCL and MZL patients who had at least one disease evaluation, the CRR was 100% and 50% respectively. 38.5% of patients achieved peripheral blood (PB) undetectable MRD (uMRD)....For TN CLL/SLL, 76.2% of patients had moderate or high TLS risk, and 14.3% had TP53 mutation or del (17p). In the CLL/SLL patients receiving mesutoclax 125 mg, the CRR was 38.1%, and the peripheral blood uMRD rate at 36-week was 65%. The 12-month progression-free survival (PFS) rate was 100%."

Retrospective data • Chronic Lymphocytic Leukemia • Mantle Cell Lymphoma • Marginal Zone Lymphoma • Small Lymphocytic Lymphoma

Safety, tolerability, and efficacy of mesutoclax (ICP-248) in combination with azacitidine in patients with myeloid malignancies (Abstract No.: 6506) (GlobeNewswire) - "The study enrolled patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Mesutoclax has demonstrated favorable safety and efficacy in the treatment of both MDS and AML...Among evaluable treatment-naïve (TN) MDS patients, the overall response rate (ORR) per IWG 2006 criteria was 100%, including complete response (CR) in 20%, and marrow CR in 80%. The composite CR rate was 70% per IWG 2023 criteria including 30% CR even when most patients only received 1 cycle treatment...Among the evaluable TN AML patients, 85.7% achieved composite CR (cCR, CR+CRi). Among those who achieved cCR, 86.7% were MRD (Minimal Residual Disease) negative per flow cytometry. cCR was 75% in adverse risk per 2017 ELN classification. The duration of response (DOR) rate at 3 months was 91.7%. The 6-month overall survival (OS) rate was 94.1%."

P1 data • Acute Myelogenous Leukemia • Myelodysplastic Syndrome

Continued Breakthroughs Across Multiple Pipelines (The Manila Times) - "...mesutoclax’s latest data will be presented at the 2026 American Society of Clinical Oncology (ASCO) annual meeting...The novel B7-H3 targeted ADC ICP-B794 showed promising preclinical data and was selected for presentation at the 2026 American Association for Cancer Research (AACR) annual meeting. The Phase I ascending dose trial is progressing....The IND application for the novel CDH17-targeted ADC ICP-B208 has been submitted which will be developed for the treatment of gastrointestinal cancers, including gastric, colorectal, pancreatic ductal adenocarcinoma, and cholangiocarcinoma."

IND • P1 data • Preclinical • Acute Myelogenous Leukemia • Cholangiocarcinoma • Colorectal Cancer • Gastric Cancer • Myelodysplastic Syndrome • Pancreatic Ductal Adenocarcinoma

In the ongoing clinical development of mesutoclax in AML and MDS, preliminary results have been encouraging. (InnoCare Pharma Press Release) - "As of 12 January 2026, a total of 59 patients were enrolled, including 8 r/r AML, 39 TN AML and 12 TN MDS. Among the 35 evaluable TN AML patients, 85.7% achieved cCR. The DoR rate at 3-months was 91.7%. The 6-month OS rate was 94.1%. Preliminary data in MDS patients were also promising. No dose-limiting toxicities (“DLT”) or TLS events were observed. Detailed data will be presented at ASCO 2026."

P1 data • Acute Myelogenous Leukemia • Myelodysplastic Syndrome

In a Phase II study of 42 treatment-naïve patients receiving mesutoclax (ICP-248) in combination with orelabrutinib, no tumor lysis syndrome (“TLS”) was observed. (InnoCare Pharma Press Release) - "Preliminary results demonstrated an ORR of 100%, a target lesion CRR of 57.1%, and an undetectable minimal residual disease ('uMRD') rate of 65% at 36 weeks, supporting the advancement of the combination into a Phase III registrational trial, which has now completed patient enrollment."

P2 data • Chronic Lymphocytic Leukemia • Small Lymphocytic Lymphoma

Business Highlights: Mesutoclax (ICP-248) (InnoCare Pharma Press Release) - "A Phase III fixed-duration combination regimen with orelabrutinib for first-line CLL/SLL, which began patient enrollment in April 2025 and completed enrollment in February 2026, demonstrating the Company’s strong clinical execution capability. A Phase II registrational trial in BTK inhibitor-treated MCL, approved for initiation in June 2025, with patient enrollment expected to complete around mid-2026."

Trial status • Chronic Lymphocytic Leukemia • Mantle Cell Lymphoma • Small Lymphocytic Lymphoma

Mesutoclax (ICP-248) monotherapy or combined with orelabrutinib demonstrates encouraging activity and safety in patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) (ASH 2025) - P1, P2/3 | "Mesutoclax monotherapy or in combination with orelabrutinib demonstrated a tolerablesafety profile across all dose levels tested. Substantial efficacy and deep remission were observed in bothTN CLL/SLL patients receiving mesutoclax 125mg combined with orelabrutinib and R/R CLL/SLL treatedwith mesutoclax alone."

Clinical • Monotherapy • Chronic Lymphocytic Leukemia • Hematological Malignancies • Leukemia • Lymphoma • Non-Hodgkin’s Lymphoma • Small Lymphocytic Lymphoma • TP53

Title: Mesutoclax monotherapy or combined with orelabrutinib demonstrates encouraging activity and safety in patients with CLL/SLL (Publication No.: 5677) (GlobeNewswire) - "Latest Data of InnoCare’s Novel BCL2 Inhibitor Presented at the 67th Annual Meeting of the American Society of Hematology (ASH)....Substantial efficacy and deep remission were observed in both treatment-naive (TN) CLL/SLL patients receiving mesutoclax 125mg combined with orelabrutinib and R/R CLL/SLL treated with mesutoclax alone. In both TN CLL/SLL and R/R CLL/SLL patients (including those who had previously failed BTK inhibitor), the ORR was 100% in the 125 mg mesutoclax dose group. At week 36, when combined with orelabrutinib, the peripheral blood (PB) uMRD rate was 65% at 125 mg."

Clinical data • Chronic Lymphocytic Leukemia • Small Lymphocytic Lymphoma

Title: Safety, tolerability and efficacy of mesutoclax to treat patients with myeloid malignancies (Publication No.: 3417) (GlobeNewswire) - "Latest Data of InnoCare’s Novel BCL2 Inhibitor Presented at the 67th Annual Meeting of the American Society of Hematology (ASH)....The combination of mesutoclax and azacitidine (AZA) demonstrated encouraging antitumor activity. The composite CR (CR+CRi, cCR) rate was achieved in 92% of TN AML patients. 82.6% of the patients achieved uMR. Most CR+CRi in TN AML was achieved by the end of cycle 1. Notably, 44% of the treatment naive AML were classified as adverse risk group and median age is 68 years old."

P1 data • Acute Myelogenous Leukemia

Title: Efficacy and tolerability of mesutoclax monotherapy in Relapsed/Refractory MCL patients: High remission rates even in prior BTKi-refractory patients (Publication No.: 887) (GlobeNewswire) - "Latest Data of InnoCare’s Novel BCL2 Inhibitor Presented at the 67th Annual Meeting of the American Society of Hematology (ASH)....The clinical data from mesutoclax (ICP-248) monotherapy demonstrated potential best in class efficacy in MCL patients, particularly in heavily treated patients with BTK inhibitors refractory. The overall response rate (ORR) of MCL patients treated with 125 mg mesutoclax monotherapy was 87.5%, with a complete response rate (CRR) of 46.9%. Among MCL patients who were BTK inhibitor refractory, the ORR was 84.0% and the CRR was 36.0%."

P1 data • Mantle Cell Lymphoma

Preliminary Safety, Pharmacological and Efficacy Data from Patients with Relapsed or Refractory B-Cell Malignancies Treated with the ICP-248, a Next Generation BCL2 Inhibitor (ASH 2023) - P1 | "Venetoclax, the first and only approved BCL2 inhibitor, is associated with gastrointestinal toxicities and tumor lysis syndrome (TLS)...7 months); median of prior regimen numbers was 3 (range, 1-3; 2 out 3 patients failed on zanubrutinib or pirtobrutinib)... Preliminary results suggest that ICP-248 is safe and well tolerated in relapsed or refractory B-cell malignancies. The favorable clinical pharmacological and safety profiles support current dose ramp up strategy. Preliminary efficacy data demonstrated good response with deep remission."

Clinical • IO biomarker • Acute Myelogenous Leukemia • B Cell Non-Hodgkin Lymphoma • Chronic Lymphocytic Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Lymphoma • Mantle Cell Lymphoma • Neutropenia • Non-Hodgkin’s Lymphoma • Oncology • Small Lymphocytic Lymphoma

A Study to Evaluate the Efficacy and Safety of ICP-248 in Subjects With Relapsed or Refractory Mantle Cell Lymphoma (clinicaltrials.gov) - P2 | N=68 | Recruiting | Sponsor: Beijing InnoCare Pharma Tech Co., Ltd. | Not yet recruiting ➔ Recruiting | Trial primary completion date: Dec 2025 ➔ Dec 2027

Enrollment open • Trial primary completion date • Hematological Malignancies • Lymphoma • Mantle Cell Lymphoma • Oncology

InnoCare Announces First Patient Dosed in the Registrational Trial of BCL2 Inhibitor Mesutoclax for Treatment of BTKi-treated MCL Patients in China (GlobeNewswire) - "This registrational trial is designed to evaluate the safety and efficacy of mesutoclax in treating BTKi-treated MCL patients."

Trial status • Mantle Cell Lymphoma

A Study to Evaluate the Efficacy and Safety of ICP-248 in Subjects With Relapsed or Refractory Mantle Cell Lymphoma (clinicaltrials.gov) - P2 | N=68 | Not yet recruiting | Sponsor: Beijing InnoCare Pharma Tech Co., Ltd.

New P2 trial • Hematological Malignancies • Lymphoma • Mantle Cell Lymphoma • Oncology

InnoCare Announces FDA Approval of Clinical Trial for BCL2 Inhibitor Mesutoclax in AML and MDS (InnoCare Pharma Press Release) - "InnoCare Pharma...announced today that the U.S. Food & Drug Administration has approved the Investigational New Drug (IND) application to conduct the clinical trial of B-cell lymphoma-2 (BCL2) inhibitor mesutoclax (ICP-248) in combination with azacitidine for the treatment of myeloid malignancies, such as acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS)."

IND • Acute Myelogenous Leukemia • Myelodysplastic Syndrome

ICP-CL-01205: ICP-248 in Combination With Azacitidine for the Treatment in Patients With Myeloid Malignancies (clinicaltrials.gov) - P1 | N=206 | Recruiting | Sponsor: Beijing InnoCare Pharma Tech Co., Ltd. | N=102 ➔ 206

Enrollment change • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of ICP-248 as Monotherapy or in Combination With Anti-CD20 Monoclonal Antibody in Mature B-cell Malignancies (clinicaltrials.gov) - P1 | N=78 | Recruiting | Sponsor: InnoCare Pharma Inc. | Not yet recruiting ➔ Recruiting | N=40 ➔ 78

Enrollment change • Enrollment open • Monotherapy • Chronic Lymphocytic Leukemia • Hematological Malignancies • Oncology