On June 15, 2026 TuHURA Biosciences, Inc. (NASDAQ: HURA) ("TuHURA" or the "Company"), a Phase 3 immuno-oncology company developing novel therapeutics to overcome resistance to cancer immunotherapy, reported that it has filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for the study of its TBS-2025 VISTA inhibiting antibody for the treatment of molecularly defined subsets of AML and other blood related cancers. The IND is being filed following detailed feedback and guidance from the FDA on the IND filed in February 2026.

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"We are excited to be the first company to advance a VISTA-inhibiting antibody for investigation in hematologic cancers, targeting molecularly defined subsets of AML, including those with NPM1 and in the future, FLT3-ITD mutations, two of the most common mutations present in approximately 60-70% of patients with AML. We also plan to include patients with relapsed/refractory (r/r) high-risk myelodysplasia (MDS), where VISTA expression, like in AML, generally correlates with low response rates and poor survival outcomes," said Dr. James Bianco, President and Chief Executive Officer of TuHURA Biosciences. "Currently, there are no approved or effective treatment options for these patient populations, representing a significant unmet medical need."

Dr. Craig Tendler, Chief Medical Officer consultant overseeing the TBS-2025 development program and Board member of TuHURA Biosciences, added, "The FDA provided valuable feedback and comprehensive guidance on trial design, which we incorporated into our proposed Phase1b dose optimization trial. Our plan is to combine the Phase 1b with the Phase 2 study for a combination study design that is much more efficient and could potentially save 4-6 months in development time. We look forward to FDA’s written responses anticipated to be received next month and, depending on the responses, are targeting initiating the Phase 1b/2 trial of TBS-2025 in the second half of 2026."

The Phase 1b portion of the study will examine the safety and potential efficacy of monotherapy dose levels in relapsed/refractory (r/r) AML patients, most of whom will harbor the NPM1 mutation and have failed to respond or relapsed after menin inhibitor therapy. In the planned protocol, if a safe and biologically effective dose is identified, the Company will review the data with the FDA to discuss the potential to expand the study at a recommended Phase 2 dose determined in the Phase 1b, to pursue a potential accelerated approval pathway, a development path similar to that of menin inhibitors in this molecular subset of patients with r/r mutNPM1 AML.

It is anticipated that the Phase 2 portion of the study will explore the potential of TBS-2025 to improve complete response rates and duration of response when used in combination with menin inhibitors in patients with mutNPM1 r/r AML.

About TBS-2025
TBS-2025 is a unique VISTA-inhibiting monoclonal antibody. VISTA is a novel checkpoint expressed on quiescent (resting) T cells and highly expressed on myeloid cells, notably myeloid derived suppressor cells (MDSCs). Scientific evidence demonstrates that mutNPM1 has demonstrated the mutation drives the expression of VISTA on leukemic blasts, which is reported to be the primary mechanisms by which AML escapes recognition by the patient’s immune system, resulting in low response rates of short duration following current therapies, including recently approved menin inhibitors. When VSIR, the gene that encodes for VISTA, is removed in murine models of mutNPM1 AML, an immune response is observed and survival is enhanced. Similarly, in a murine model of AML, TBS-2025 resulted in an increase in survival comparable to intensive chemotherapy regimen that is currently used in front line treatment of patients with AML. When combined with intensive chemotherapy, survival was markedly improved. Collectively, these data underscore the potential for TBS-2025 in the treatment of patients with AML

TBS 2025 was initially investigated in a large Phase 1 trial as either monotherapy (n=24) or in combination with pembrolizumab (n=15) among patients with advanced, therapy refractory cancers, including breast, lung, colorectal, and ovarian cancer. The purpose of the study was to investigate its safety profile and determine the recommended Phase 2 dose for solid tumors. The drug demonstrated a favorable safety profile even at the highest dose level of 1,000mg administered every two weeks. Safety and pharmacokinetic data from this trial was helpful in designing the Phase 1b segment of the planned trial in AML.

(Press release, TuHURA Biosciences, JUN 15, 2026, View Source [SID1234668741])

On June 14, 2026 CEL-SCI Corporation ("CEL-SCI" or the "Company") (NYSE American: CVM), a clinical stage cancer immunotherapy company, reported the pricing of a best-efforts offering of 2,500,000 shares of common stock at an offering price of $1.00 per share. Total gross proceeds from the offering, before deducting the placement agent’s fees and offering expenses, are expected to be $2.5 million. The offering is expected to close on June 16, 2026, subject to satisfaction of customary closing conditions.

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The Company intends to use the proceeds for the continued development of Multikine*, general corporate purposes, and working capital.

ThinkEquity is acting as the sole placement agent for the offering.

The securities will be offered and sold pursuant to a shelf registration statement on Form S-3 (File No. 333-288515), including a base prospectus, filed with the U.S. Securities and Exchange Commission (the "SEC") on July 3, 2025, and declared effective on August 12, 2025. The offering will be made only by means of a written prospectus. A final prospectus supplement and accompanying prospectus describing the terms of the offering will be filed with the SEC on its website at www.sec.gov. Copies of the prospectus supplement and the accompanying prospectus relating to the offering may also be obtained, when available, from the offices of ThinkEquity, 17 State Street, 41st Floor, New York, New York 10004.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

(Press release, Cel-Sci, JUN 14, 2026, View Source [SID1234668727])

On June 14, 2026 Zymeworks Inc. (Nasdaq: ZYME), a biotechnology company managing a portfolio of licensed healthcare assets while developing a diverse pipeline of novel, multifunctional biotherapeutics, reported new clinical data from the dose-escalation portion of its ongoing Phase 1 study evaluating ZW191, a folate receptor alpha (FRα)-targeting antibody-drug conjugate (ADC), at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Gynaecological Cancers Congress 2026.

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The presentation highlights compelling anti-tumor activity in patients with platinum-resistant ovarian cancer (PROC) and in patients with endometrial cancer, including patients with both positive and negative FRα expression. The results further support the potential of ZW191 to address limitations of currently available and investigational FRα-targeted therapies and broaden treatment opportunities for patients with gynecologic cancers.

"We are highly encouraged by these data, which continue to demonstrate the differentiated profile of ZW191 and its potential to deliver meaningful clinical benefit across a broad population of patients with ovarian and endometrial cancers," said Sabeen Mekan, M.D., Senior Vice President and Chief Medical Officer of Zymeworks. "The response rate observed in FRα-positive ovarian cancer patients compares favorably with currently available therapies, while maintaining meaningful activity in patients with negative FRα expression. Together these encouraging data on durability, progression-free survival, and manageable safety profile further strengthen our confidence in the program and reinforce the ability of our ADC platform to generate differentiated therapeutics."

The analysis included efficacy data by FRα expression level from Part 1 of the ongoing Phase 1 study as of the March 9, 2026, data cutoff. FRα expression was assessed by immunohistochemistry and categorized using the Proportion and Staining Intensity, defined as the percentage of cells with 2+/3+ staining. Tumors were categorized as FRα-negative (<75%) or FRα-positive (≥75%).

Key Findings

Among response-evaluable PROC patients, ZW191 demonstrated a cORR of 78.6% in patients with FRα-positive tumors and 47.4% in patients with FRα-negative tumors across all dose levels. Disease control rates were 100.0% and 89.5%, respectively. Confirmed ORR in the overall PROC population was 58.8% across all dose levels studied and 65.2% in dose range of 6.4-9.6 mg/kg. These findings demonstrate meaningful anti-tumor activity across both FRα-positive and FRα-negative tumors as well as in the overall population.

In endometrial cancer patients with FRα-negative tumors, ZW191 demonstrated a cORR of 40.0% and a disease control rate of 80.0% across all doses evaluated.

Across both ovarian and endometrial cancer cohorts, responses were observed early and continued to deepen over time. Median duration of response was not reached at the time of data cutoff, and median progression-free survival was 7.6 months.

ZW191 continued to demonstrate a favorable tolerability profile. Treatment-emergent adverse events (TEAEs) occurred in 98% of patients, with grade ≥3 TEAEs reported in 55% of patients. The most common grade ≥3 adverse events were neutropenia (24%), anemia (20%), and thrombocytopenia (12%). Serious TEAEs occurred in 35% of patients, and 20% of patients discontinued treatment due to adverse events. Overall, the safety profile was manageable and consistent with continued clinical development.

"These results further demonstrate the potential of ZW191 to provide meaningful clinical benefit for patients with difficult-to-treat gynecologic cancers," said Kosei Hasegawa, M.D., Ph.D., Saitama Medical University International Medical Center and presenting author. "The robust activity observed in FRα-positive ovarian cancer, together with encouraging responses in tumors with lower FRα expression, suggests ZW191 could expand the benefit of FRα-targeted therapy to a broader patient population."

Part 2a, the dose optimization portion of the Phase 1 study evaluating ZW191 in ~60 PROC patients randomized to either 6.4 mg/kg every three weeks or 9.6 mg/kg every three weeks, has completed enrollment. Data from Part 2a will be presented at a future medical meeting and are expected to inform dose selection for potential future development.

ZW191’s differentiated profile, including its novel FRα-targeting antibody, ZD06519 payload, and high drug-to-antibody ratio, supports its potential to address key limitations of current therapies and broaden the applicability of FRα-targeted treatment approaches across multiple tumor types.

About ZW191

ZW191 is an antibody-drug conjugate engineered to target a protein called folate receptor-⍺ found in ~75% of high-grade serous ovarian carcinomas1, over 50% of endometrial cancers2,3 and ~70% of lung adenocarcinomas4. ZW191’s differentiated design strongly supports its ability to internalize into FR⍺-expressing cells with the potential to release bystander active topoisomerase-1 inhibitor (ZD06519), a novel proprietary payload developed by Zymeworks to kill tumor cells.

(Press release, Zymeworks, JUN 14, 2026, View Source [SID1234668728])

On June 14, 2026 Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global leader in cell therapy, reported first clinical proof-of-concept data for LB2501, its investigational in vivo CD19/CD20 dual-targeting CAR-T cell therapy, in patients with relapsed or refractory B-cell non-Hodgkin lymphoma (R/R B-NHL). The results are being presented today in a late-breaking session at the European Hematology Association (EHA) (Free EHA Whitepaper) 2026 Congress (Abstract #LB5006).

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In the ongoing Phase 1 study, a single infusion of LB2501 generated dose-dependent in vivo CAR-T expansion without lymphodepletion. At the higher dose level (DL2), LB2501 achieved a 100% objective response rate (ORR) (6/6) and an 83.3% complete response rate (CR) (5/6), with all responses ongoing at the time of data cutoff. LB2501 also showed a favorable safety profile, with no dose-limiting toxicities (DLTs), serious adverse events (SAEs), immune effector cell-associated neurotoxicity syndrome (ICANS), or deaths reported.

"In vivo CAR-T represents a compelling frontier in cell therapy, enabling the generation of CAR-T cells directly within the patient, with the potential to simplify treatment and expand access over time," said Ying Huang, Ph.D., Chief Executive Officer of Legend Biotech. "LB2501 is our step toward realizing that vision and reflects further progress toward our goal of leading the future of cell therapy. Backed by the commercial and scientific foundation we have built with CARVYKTI, we are well-positioned to advance this next generation of CAR-T delivery. These early data, with deep responses from a single infusion across patients, give us confidence in the path ahead."

LB2501 Demonstrates In Vivo CAR-T Generation and Early Clinical Activity

In an ongoing Phase 1 study, 12 patients with R/R B-NHL received LB2501 across two dose levels, DL1 (n=6) and DL2 (n=6). Patients had received a median of three prior lines of therapy, and 58.3% were refractory to their most recent treatment. The open-label, multi-center, dose-escalation study is evaluating safety, recommended Phase 2 dose, pharmacokinetics, and preliminary efficacy in adults with R/R B-NHL. The study was conducted without lymphodepletion.

At DL2, LB2501 achieved a 100% ORR (6/6) and an 83.3% CR rate (5/6), with responses observed across patients with diffuse large B-cell lymphoma (DLBCL), mantle cell lymphoma (MCL), and follicular lymphoma (FL). Across both dose levels, the ORR was 50.0% (6/12), and the CR rate was 41.7% (5/12). At the time of data cutoff, all responses at DL2 were ongoing.

LB2501 showed a favorable safety profile. No DLTs, SAEs, ICANS, or deaths were reported. Infusion-related reactions (IRR) and cytokine release syndrome (CRS) were the most common adverse events of special interest and were all Grade 1–2. Infusion-related reactions occurred in 75.0% (9/12) of patients overall, with a median onset of 1.4 hours after infusion and a median recovery time of 18.6 hours. CRS occurred in 66.7% (8/12) of patients overall, with a median onset at Day 11 and a median duration of 4.5 days. IRR and CRS were all Grade 1–2, no patients required glucocorticoids for CRS management. Four patients received tocilizumab.

Pharmacokinetic analyses showed dose-dependent in vivo CAR-T expansion in 100% (6/6) of patients at DL2 and 83% (5/6) of patients at DL1. CAR-T cells remained detectable in peripheral blood for up to 116 days. Viral copy number in peripheral blood peaked immediately after infusion and decreased to undetectable concentrations within 24 hours.

Additional translational analyses further characterized the in vivo profile of LB2501. No evidence of non-specific transduction was detected in NK cells or other non-T/B/NK lymphocyte populations. Vector integrations were highly polyclonal and diverse. These findings support proof-of-concept for in vivo T-cell engineering, with polyclonal vector integration and rapid vector clearance.

"These early clinical findings are encouraging in a heavily pretreated relapsed or refractory B-cell non-Hodgkin lymphoma population," said Lei Fan, M.D., Ph.D., Professor, Doctoral Supervisor, and Administrative Director, Hematology Department, Jiangsu Province Hospital, Nanjing, China. "The responses observed at the higher dose level achieved a 100% objective response rate, together with a favorable safety profile and the absence of lymphodepletion, support further investigation of LB2501 as a novel in vivo CAR-T approach. The additional pharmacokinetic and translational findings presented at EHA (Free EHA Whitepaper) further support the feasibility of generating CAR-T cells directly within the patient." ‡

ABOUT LB2501
LB2501 is an investigational, potential first-in-class CD19/CD20 dual-targeting in vivo CAR-T therapy designed to generate CAR-T cells directly within the patient following a single intravenous infusion. It is being evaluated in an ongoing Phase 1, open-label study (NCT07002112) in patients with relapsed/refractory B-cell malignanciesi to assess safety, tolerability, and preliminary efficacy.[i]

ABOUT B-CELL NON-HODGKIN LYMPHOMA
Non-Hodgkin lymphoma (NHL) is a group of cancers that originate in lymphocytes, a type of white blood cell that plays a key role in the body’s immune system.ii B-cell lymphomas account for approximately 85% of NHL cases and arise from abnormal growth of B lymphocytes (B cells), which are responsible for producing antibodies. These malignancies include a range of subtypes that vary in aggressiveness, from slow-growing to highly aggressive disease.iii

While treatment advances have improved outcomes for some patients, those with relapsed or refractory B-cell NHL, particularly after multiple lines of therapy, often face limited options.

(Press release, Legend Biotech, JUN 14, 2026, View Source [SID1234668730])

On June 14, 2026 Ascentage Pharma Group International (NASDAQ: AAPG; HKEX: 6855), a global, commercial-stage, integrated biopharmaceutical company engaged in the discovery, development and commercialization of novel therapies to address unmet medical needs in cancer, reported that seventeen clinical updates from its core assets were presented at the 31st Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) (EHA2026), including eight poster presentations. The presentations featured data from ongoing clinical studies of olverembatinib (HQP1351), the first third-generation BCR-ABL1 inhibitor approved in China, and lisaftoclax (APG-2575), the first approved China-developed Bcl-2 selective inhibitor. The EHA (Free EHA Whitepaper)2026 Congress convened in Stockholm, Sweden, from June 11 to 14, 2026.

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At the EHA (Free EHA Whitepaper)2026 Congress, presentations on olverembatinib comprised key concurrent updates across two therapeutic areas: chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). In CML, olverembatinib demonstrated deep and durable responses in patients with chronic-phase CML (CML-CP) without the T315I mutation, in whom the disease was resistant and/or intolerant to first-line TKI therapy, supporting its potential as a second-line treatment option. For CML-CP patients who have failed treatment with at least two prior TKIs, olverembatinib can be used as a standard treatment option. In addition, olverembatinib showed positive clinical data in CML patients with multiline TKI resistance and co-occurring high-risk genetic mutations. In Ph+ ALL, olverembatinib continued to demonstrate robust clinical response, with updated data from the first-line global registrational Phase III study (POLARIS-1) further validating its deep response rate and manageable safety profile. The chemotherapy-free combination regimen with lisaftoclax yielded encouraging clinical data in subpopulations such as pediatric patients with relapsed/refractory Ph+ ALL.

Updated data from the registrational Phase II study of lisaftoclax in chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) were also presented. Stratified analyses correlating baseline characteristics with prognosis provided important insights that will inform refinement of treatment strategies and optimization of individualized dosing regimens across different patient populations. The real-world data on lisaftoclax in myeloid neoplasms also provides robust evidence for its clinical utility.

Yifan Zhai, MD, Chief Medical Officer of Ascentage Pharma, said: "The clinical updates on olverembatinib and lisaftoclax presented at EHA (Free EHA Whitepaper)2026 further validate the therapeutic value of these two key assets in the global hematologic malignancy field. Olverembatinib has the potential to become an important treatment option for CML, while offering new treatment possibilities for Ph+ ALL patients. The updated data from the registrational Phase II study of lisaftoclax in CLL/SLL will help guide individualized dosing strategies across different patient populations. Its real-world data in myeloid malignancies further strengthens our confidence in its clinical application among such patients. We are particularly encouraged by the promising combination data. Looking ahead, we will continue to accelerate the global clinical development of our key assets, while actively exploring more innovative combination treatment strategies to provide meaningful treatment options for patients around the world."

Key highlights of the selected poster presentations at EHA (Free EHA Whitepaper)2026 are as follows (for more information of the Company’s candidates, please visit the official EHA (Free EHA Whitepaper) website):

EFFICACY OF OLVEREMBATINIB IN PATIENTS WITH CHRONIC-PHASE CHRONIC MYELOID LEUKEMIA (CP-CML) WITH PRIOR RESISTANCE TO PONATINIB OR ASCIMINIB AND ASXL1 MUTATIONS

● Abstract Number: EHA (Free EHA Whitepaper)-3991 (PS1727)

● First Author: Elias Jabbour, MD, Department of Leukemia, The University of Texas MD Anderson Cancer Center

● Key Highlights: This Phase Ib study performed mutational analyses on 22 patients with ponatinib- and/or asciminib-resistant CP-CML and evaluated the antileukemic activity of olverembatinib across different mutational backgrounds. ASXL1 mutations were present in 40.9% (9/22) of patients. After olverembatinib treatment, 44.4% (4/9) of patients with ASXL1 mutations achieved clinical responses, including 22.2% (2/9) who achieved MMR, one of whom achieved MR4.5. This study provided the first evidence that olverembatinib is active in patients with ponatinib- and/or asciminib-resistant CP-CML, including those harboring challenging genotypes such as ASXL1 mutations, highlighting its potential as a treatment option for patients with multi-line TKI-resistant disease.

UPDATED EFFICACY AND SAFETY OF OLVEREMBATINIB (HQP1351) AS SECOND-LINE THERAPY IN PATIENTS WITH CHRONIC-PHASE CHRONIC MYELOID LEUKEMIA (CP-CML)

● Abstract Number: EHA (Free EHA Whitepaper)-3388 (PS1733)

● First Author: Weiming Li, MD, Department of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology

● Key Highlights: This single-arm, multicenter, open-label Phase II study evaluated olverembatinib as a second-line treatment in patients with CP-CML without the T315I mutation. Among 42 evaluable patients, the complete cytogenetic response (CCyR) rate was 76.2% and the major molecular response (MMR) rate was 47.6%. Responses deepened over time with longer treatment duration, and olverembatinib exhibited manageable tolerability with no treatment-related deaths. These findings support the potential of olverembatinib to induce durable and deep responses in patients with CP-CML without the T315I mutation whose disease was resistant and/or intolerant to first-line TKI therapy, further supporting its role as a second-line treatment option.

THE EFFICACY AND SAFETY OF SWITCHING TO OLVEREMBATINIB OR CONTINUING ORIGINAL TKI THERAPY IN CML-CP PATIENTS TREATED WITH AT LEAST TWO PRIOR TKIS: A PROSPECTIVE, MULTICENTER, CONTROLLED TRIAL

● Abstract Number: EHA (Free EHA Whitepaper)-4595 (PS1728)

● First Author: Bingbing Wen, the Second People’s Hospital of Shenzhen

● Key Highlights: This prospective, multicenter, controlled trial enrolled 105 patients with CML-CP who had received at least two prior TKIs for ≥18 months and failed to achieve MMR. Patients were assigned in a 1:2 ratio to either switch to olverembatinib (40 mg orally every other day) or continue their most recent TKI therapy. Results showed that the 6-month MMR rate was significantly higher in the olverembatinib group than in the control group (54.3% vs 10.0%; P<0.001). At 12 months, the cumulative incidence of MMR was 57.14% in the olverembatinib group compared with 21.43% in the control group (P<0.0001). Common grade 3/4 hematologic treatment-emergent adverse events (TEAEs) included thrombocytopenia (42.86%) and anemia (17.14%). Grade 3/4 non-hematologic adverse events were rare. Notably, 78.57% of adverse events related to prior TKI therapy improved after patients switched to olverembatinib. These findings support olverembatinib as a potential standard of care for patients with CML-CP previously treated with at least two TKIs.

UPDATED RESULTS OF POLARIS-1 (PART 1), A GLOBAL REGISTRATIONAL PHASE 3 STUDY: OLVEREMBATINIB COMBINED WITH LOW-INTENSITY CHEMOTHERAPY IN NEWLY DIAGNOSED PH+ ALL

● Abstract Number: EHA (Free EHA Whitepaper)-3437 (PS1479)

● First Author: Suning Chen, The First Affiliated Hospital of Soochow University

● Key Highlights: This global, multicenter, registrational Phase III study (POLARIS-1 Part 1) is evaluating the efficacy and safety of olverembatinib in combination with reduced-intensity chemotherapy in patients with newly diagnosed Ph+ ALL. A total of 55 patients were enrolled. By the end of induction, the CR/CRi rate reached 94.4% and the MRD-negative CR rate reached 63.0%. MRD-negativity rates increased over time, reaching 93.1% by the end of cycle 9. The regimen demonstrated a manageable safety profile, with no meaningful differences in efficacy or safety observed between the 30 and 40mg dose groups. Encouraging activity was also observed in patients with adverse prognostic genotypes, including IKZF1plus. These findings suggest that olverembatinib combined with low-intensity chemotherapy may induce rapid, deep, and sustained MRD-negative responses in patients with newly diagnosed Ph+ ALL, while maintaining a favorable safety profile, and provide important evidence supporting its use in the frontline setting.

SAFETY AND PRELIMINARY EFFICACY OF OLVEREMBATINIB (HQP1351) COMBINED WITH LISAFTOCLAX (APG-2575) IN PEDIATRIC PATIENTS WITH RELAPSED/REFRACTORY (R/R PH+ ALL): RESULTS OF A PHASE 1B STUDY

● Abstract Number: EHA (Free EHA Whitepaper)-4691 (PS1473)

● First Author: Jingliao Zhang, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College

● Key Highlights: This open-label, dose-escalation Phase Ib study evaluated olverembatinib in combination with lisaftoclax in pediatric patients with relapsed/refractory (R/R) Ph+ ALL who are resistant or intolerant to at least one prior TKI. A total of 17 patients were enrolled, with a median age of 13 years, and 40% harbored ABL1 mutations, including T315I. Among nine efficacy-evaluable patients, the combination achieved an overall response rate (ORR) of 88.9%, an MRD-negativity rate of 66.7% (8/12 at cycle 2 day 28), and 93.3% of patients (14/15 at cycle 2 day 28) achieved MMR or better. Both agents were detectable in cerebrospinal fluid (CSF), providing evidence of CNS penetration, and demonstrated activity across ABL1 mutation subgroups. The regimen showed a manageable safety profile with no treatment-related deaths. These findings support the potential of this chemotherapy-free oral dual-targeted regimen to induce rapid and deep remissions, and may provide a novel treatment option for pediatric patients with R/R Ph+ ALL.

REAL-WORLD EFFICACY AND SAFETY OF LISAFTOCLAX IN MYELOID NEOPLASMS: A MULTICENTER STUDY

● Abstract Number: EHA (Free EHA Whitepaper)-5454 (PF562)

● First Author: Chen Cao, Qilu Hospital of Shandong University

● Key Highlights: This multicenter real-world (retrospective) study evaluated the efficacy and safety of the novel BCL-2 inhibitor lisaftoclax in patients with myeloid neoplasms. A total of 30 patients (median age 63) were enrolled, including 25 patients with acute myeloid leukemia (AML, 83%), 3 with myelodysplastic syndromes (MDS, 10%), and 2 with chronic myelomonocytic leukemia (CMML, 7%). In patients with AML, the CR/CRi rate reached 72%, with the highest response observed in the ELN low-risk subgroup (87%). Among patients achieving CR/CRi, the MRD-negativity rate was 61%. The CR/CRi rate was 100% in patients with NPM1 mutations and 83% in patients with IDH2 mutations. Among the three patients with MDS, two achieved CRi. Regarding safety, grade≥3 treatment-emergent adverse events (TEAEs) were primarily hematologic adverse events, including thrombocytopenia (27%), anemia (23%), and neutropenia (20%). Overall safety was manageable. These findings demonstrate promising efficacy and manageable safety of lisaftoclax in the real-world treatment of myeloid neoplasms, particularly AML.

CORRELATION OF BASELINE CHARACTERISTICS WITH PROGNOSIS IN PATIENTS WITH CHRONIC LYMPHOCYTIC LEUKEMIA/SMALL LYMPHOCYTIC LYMPHOMA (CLL/SLL) TREATED WITH LISAFTOCLAX (APG-2575) IN A PIVOTAL PHASE 2 STUDY

● Abstract Number: EHA (Free EHA Whitepaper)-3984 (PS1713)

● First Author: Keshu Zhou, Henan Cancer Hospital

● Key Highlights: This correlative analysis from the pivotal Phase II study (NCT05147467) evaluated associations between baseline characteristics and prognosis in patients with R/R CLL/SLL treated with lisaftoclax. The study enrolled 77 patients with R/R CLL/SLL refractory to BTKis who received lisaftoclax 600 mg once daily. Among 72 evaluable patients, the median progression-free survival (PFS) was 23.9 months and the Independent Review Committee (IRC)-assessed ORR was 62.5%. Further analyses showed that TP53 mutation/del(17p), complex karyotype (CK), and mutations in SF3B1, KIT, BLM, and SETD2 were associated with significantly shorter PFS. Complex karyotype and tumor size were identified as independent risk factors for shorter PFS. These findings demonstrate that lisaftoclax has clinical activity in patients with R/R CLL/SLL refractory to BTKi therapy. Additionally, these data may help to identify patients with poorer prognosis based on baseline risk characteristics, supporting future risk stratification and risk-adapted combination treatment strategies.

(Press release, Ascentage Pharma, JUN 14, 2026, View Source [SID1234668732])