BLINCYTO® (BLINATUMOMAB) ADDED TO CONSOLIDATION CHEMOTHERAPY SIGNIFICANTLY IMPROVES SURVIVAL IN ADULT PATIENTS WITH MEASURABLE RESIDUAL DISEASE-NEGATIVE B-LINEAGE ACUTE LYMPHOBLASTIC LEUKEMIA (B-ALL)

On December 13, 2022 Amgen (NASDAQ: AMGN) reported the ECOG-ACRIN Cancer Research Group (ECOG-ACRIN) reported that it will present results from the E1910 randomized Phase 3 trial (Press release, Amgen, DEC 13, 2022, View Source [SID1234625175]). This is the first study to demonstrate superior overall survival (OS) with BLINCYTO added to consolidation chemotherapy over current standard of care (multiagent consolidation chemotherapy) in newly diagnosed adult patients with Philadelphia chromosome-negative B-ALL who were measurable residual disease (MRD)-negative following induction and intensification chemotherapy. These results were featured in a press briefing on Monday, Dec. 12 at 8:30 a.m. CT and presented on Tuesday, Dec. 13 at 9 a.m. CT as a late breaking oral presentation (LBA1) at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition in New Orleans.

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"Treatment with BLINCYTO in addition to consolidation chemotherapy reduced the risk of death by 58% compared to chemotherapy alone. We are pleased by this remarkable improvement in overall survival, and we look forward to sharing these data with regulatory authorities as soon as possible," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "Amgen continues to advance a robust development program for BLINCYTO, with a focus on minimizing chemotherapy and a subcutaneous formulation to help address remaining unmet needs for patients with B-ALL."

The Phase 3 randomized trial (E1910), activated in December 2013, evaluated the safety and efficacy of BLINCYTO added to standard of care consolidation chemotherapy compared to chemotherapy alone in patients with newly diagnosed B-ALL with no MRD after induction and intensification chemotherapy. The primary endpoint was OS and key secondary endpoints included relapse-free survival, MRD status, and incidence of adverse events. Based on a recommendation by the ECOG-ACRIN Data Safety Monitoring Committee and consistent with the pre-defined efficacy threshold, results from the planned interim analysis are now reported due to overwhelming efficacy reported in the BLINCYTO arm.

With a median follow up of 43 months, the study met its primary endpoint with a significant improvement in overall survival favoring the BLINCYTO arm; median OS was not reached vs. 71.4 months in the control arm (hazard ratio [HR] = 0.42, 95% CI: 0.24 – 0.75; two-sided p=0.003). After about 3.5 years of follow-up, 83% of the patients who went on to receive additional standard consolidation chemotherapy plus experimental BLINCYTO were alive versus 65% of those who received chemotherapy only. No new safety signals were reported for the combination.

"Adults with newly diagnosed ALL can achieve a high rate of complete remission with chemotherapy, but frequently relapse and have disappointing survival rates.1,2 Historically, outcomes for newly diagnosed adults with ALL have been significantly worse than for children, where up to 90% of patients are cured with frontline therapy.3,4 In this study, survival rates for adults when blinatumomab was added to chemotherapy are significantly improved in patients with MRD-negative remission, approaching those we have seen in children," said Selina M. Luger, M.D., professor of hematology-oncology at the University of Pennsylvania’s Abramson Cancer Center and Perelman School of Medicine, chair of the ECOG-ACRIN Leukemia Committee and an investigator on the study. "Moreover, the data provide additional clinical evidence supporting the recent guideline updates for adult ALL recommending blinatumomab as consolidation in both MRD-positive and MRD-negative patients."

Data from the trial will be submitted to global regulatory authorities, including where BLINCYTO has been previously approved.

Study E1910 was designed and conducted independently from industry with public funding. The ECOG-ACRIN Cancer Research Group sponsored the trial with funding from the National Cancer Institute (NCI), part of the National Institutes of Health. Other NCI-funded network groups took part in the study. In addition, Amgen provided BLINCYTO and support through an NCI Cooperative Research and Development Agreement (CRADA).

E1910 Study Design
In the E1910 Phase 3 randomized trial, 488 patients aged 30-70 with newly diagnosed B-ALL were enrolled. All participants initially received 2.5 months of combination induction chemotherapy (step 1). After remission induction (step 1), if patients were in complete remission, they continued on-study and received an intensification course of high dose chemotherapy (step 2). Subsequently, their remission and MRD status were determined. All patients were then randomized/assigned to receive four cycles of consolidation chemotherapy with or without four 28-day cycles of BLINCYTO (step 3). Following FDA approval of blinatumomab for MRD-positive patients in March 2018, MRD-positive participants were assigned to the blinatumomab arm. MRD-negative patients continued to be randomized. After completion of consolidation chemo +/- BLINCYTO, patients were given 2.5 years of chemotherapy maintenance therapy timed from the start of the intensification cycle (step 4).

For more information, please visit ClinicalTrials.gov.

About BLINCYTO (blinatumomab)
BLINCYTO is a BiTE (bispecific T-cell engager) immuno-oncology therapy that targets CD19 surface antigens on B cells. BiTE molecules fight cancer by helping the body’s immune system detect and target malignant cells by engaging T cells (a type of white blood cell capable of killing other cells perceived as threats) to cancer cells. By bringing T cells near cancer cells, the T cells can inject toxins and trigger cancer cell death (apoptosis). BiTE immuno-oncology therapies are currently being investigated for their potential to treat a wide variety of cancers.

BLINCYTO was granted breakthrough therapy and priority review designations by the U.S. Food and Drug Administration and is approved in the U.S. for the treatment of:

relapsed or refractory CD-19 positive B-cell precursor ALL in adults and children.
CD-19 positive B-cell precursor ALL in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1% in adults and children. This indication is approved under accelerated approval based on MRD response rate and hematological relapse-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
In the European Union (EU), BLINCYTO is indicated as monotherapy for the treatment of:

adults with Philadelphia chromosome negative CD19 positive relapsed or refractory B-precursor acute lymphoblastic leukemia (ALL).
adults with Philadelphia chromosome negative CD19 positive B-precursor ALL in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1%.
pediatric patients aged 1 year or older with Philadelphia chromosome negative CD19 positive B-precursor ALL which is refractory or in relapse after receiving at least two prior therapies or in relapse after receiving prior allogeneic hematopoietic stem cell transplantation
IMPORTANT SAFETY INFORMATION

WARNING: CYTOKINE RELEASE SYNDROME and NEUROLOGICAL TOXICITIES

Cytokine Release Syndrome (CRS), which may be life-threatening or fatal, occurred in patients receiving BLINCYTO. Interrupt or discontinue BLINCYTO and treat with corticosteroids as recommended.
Neurological toxicities, which may be severe, life-threatening or fatal, occurred in patients receiving BLINCYTO. Interrupt or discontinue BLINCYTO as recommended.
Contraindications

BLINCYTO is contraindicated in patients with a known hypersensitivity to blinatumomab or to any component of the product formulation.

Warnings and Precautions

Cytokine Release Syndrome (CRS): CRS, which may be life-threatening or fatal, occurred in 15% of patients with R/R ALL and in 7% of patients with MRD-positive ALL. The median time to onset of CRS is 2 days after the start of infusion and the median time to resolution of CRS was 5 days among cases that resolved. Closely monitor and advise patients to contact their healthcare professional for signs and symptoms of serious adverse events such as fever, headache, nausea, asthenia, hypotension, increased alanine aminotransferase (ALT), increased aspartate aminotransferase (AST), increased total bilirubin (TBILI), and disseminated intravascular coagulation (DIC). The manifestations of CRS after treatment with BLINCYTO overlap with those of infusion reactions, capillary leak syndrome, and hemophagocytic histiocytosis/macrophage activation syndrome. If severe CRS occurs, interrupt BLINCYTO until CRS resolves. Discontinue BLINCYTO permanently if life-threatening CRS occurs. Administer corticosteroids for severe or life-threatening CRS.
Neurological Toxicities: Approximately 65% of patients receiving BLINCYTO in clinical trials experienced neurological toxicities. The median time to the first event was within the first 2 weeks of BLINCYTO treatment and the majority of events resolved. The most common (≥ 10%) manifestations of neurological toxicity were headache and tremor. Severe, life–threatening, or fatal neurological toxicities occurred in approximately 13% of patients, including encephalopathy, convulsions, speech disorders, disturbances in consciousness, confusion and disorientation, and coordination and balance disorders. Manifestations of neurological toxicity included cranial nerve disorders. Monitor patients for signs or symptoms and interrupt or discontinue BLINCYTO as outlined in the PI.
Infections: Approximately 25% of patients receiving BLINCYTO in clinical trials experienced serious infections such as sepsis, pneumonia, bacteremia, opportunistic infections, and catheter-site infections, some of which were life-threatening or fatal. Administer prophylactic antibiotics and employ surveillance testing as appropriate during treatment. Monitor patients for signs or symptoms of infection and treat appropriately, including interruption or discontinuation of BLINCYTO as needed.
Tumor Lysis Syndrome (TLS), which may be life-threatening or fatal, has been observed. Preventive measures, including pretreatment nontoxic cytoreduction and on-treatment hydration, should be used during BLINCYTO treatment. Monitor patients for signs and symptoms of TLS and interrupt or discontinue BLINCYTO as needed to manage these events.
Neutropenia and Febrile Neutropenia, including life-threatening cases, have been observed. Monitor appropriate laboratory parameters (including, but not limited to, white blood cell count and absolute neutrophil count) during BLINCYTO infusion and interrupt BLINCYTO if prolonged neutropenia occurs.
Effects on Ability to Drive and Use Machines: Due to the possibility of neurological events, including seizures, patients receiving BLINCYTO are at risk for loss of consciousness, and should be advised against driving and engaging in hazardous occupations or activities such as operating heavy or potentially dangerous machinery while BLINCYTO is being administered.
Elevated Liver Enzymes: Transient elevations in liver enzymes have been associated with BLINCYTO treatment with a median time to onset of 3 days. In patients receiving BLINCYTO, although the majority of these events were observed in the setting of CRS, some cases of elevated liver enzymes were observed outside the setting of CRS, with a median time to onset of 19 days. Grade 3 or greater elevations in liver enzymes occurred in approximately 7% of patients outside the setting of CRS and resulted in treatment discontinuation in less than 1% of patients. Monitor ALT, AST, gamma-glutamyl transferase, and TBILI prior to the start of and during BLINCYTO treatment. BLINCYTO treatment should be interrupted if transaminases rise to > 5 times the upper limit of normal (ULN) or if TBILI rises to > 3 times ULN.
Pancreatitis: Fatal pancreatitis has been reported in patients receiving BLINCYTO in combination with dexamethasone in clinical trials and the post-marketing setting. Evaluate patients who develop signs and symptoms of pancreatitis and interrupt or discontinue BLINCYTO and dexamethasone as needed.
Leukoencephalopathy: Although the clinical significance is unknown, cranial magnetic resonance imaging (MRI) changes showing leukoencephalopathy have been observed in patients receiving BLINCYTO, especially in patients previously treated with cranial irradiation and antileukemic chemotherapy.
Preparation and administration errors have occurred with BLINCYTO treatment. Follow instructions for preparation (including admixing) and administration in the PI strictly to minimize medication errors (including underdose and overdose).
Immunization: Vaccination with live virus vaccines is not recommended for at least 2 weeks prior to the start of BLINCYTO treatment, during treatment, and until immune recovery following last cycle of BLINCYTO.
Risk of Serious Adverse Reactions in Pediatric Patients due to Benzyl Alcohol Preservative: Serious and fatal adverse reactions including "gasping syndrome," which is characterized by central nervous system depression, metabolic acidosis, and gasping respirations, can occur in neonates and infants treated with benzyl alcohol-preserved drugs including BLINCYTO (with preservative). When prescribing BLINCYTO (with preservative) for pediatric patients, consider the combined daily metabolic load of benzyl alcohol from all sources including BLINCYTO (with preservative) and other drugs containing benzyl alcohol. The minimum amount of benzyl alcohol at which serious adverse reactions may occur is not known. Due to the addition of bacteriostatic saline, 7-day bags of BLINCYTO solution for infusion with preservative contain benzyl alcohol and are not recommended for use in any patients weighing < 22 kg.
Adverse Reactions

The most common adverse reactions (≥ 20%) in clinical trial experience of patients with MRD-positive B-cell precursor ALL (BLAST Study) treated with BLINCYTO were pyrexia (91%), infusion-related reactions (77%), headache (39%), infections (pathogen unspecified 39%), tremor (31%), and chills (28%). Serious adverse reactions were reported in 61% of patients. The most common serious adverse reactions (≥ 2%) included pyrexia, tremor, encephalopathy, aphasia, lymphopenia, neutropenia, overdose, device related infection, seizure, and staphylococcal infection.
The most common adverse reactions (≥ 20%) in clinical trial experience of patients with Philadelphia chromosome-negative relapsed or refractory B-cell precursor ALL (TOWER Study) treated with BLINCYTO were infections (bacterial and pathogen unspecified), pyrexia, headache, infusion-related reactions, anemia, febrile neutropenia, thrombocytopenia, and neutropenia. Serious adverse reactions were reported in 62% of patients. The most common serious adverse reactions (≥ 2%) included febrile neutropenia, pyrexia, sepsis, pneumonia, overdose, septic shock, CRS, bacterial sepsis, device related infection, and bacteremia.
Adverse reactions that were observed more frequently (≥ 10%) in the pediatric population compared to the adults with relapsed or refractory B-cell precursor ALL were pyrexia (80% vs. 61%), hypertension (26% vs. 8%), anemia (41% vs. 24%), infusion-related reaction (49% vs. 34%), thrombocytopenia (34% vs. 21%), leukopenia (24% vs. 11%), and weight increased (17% vs. 6%).
In pediatric patients less than 2 years old (infants), the incidence of neurologic toxicities was not significantly different than for the other age groups, but its manifestations were different; the only event terms reported were agitation, headache, insomnia, somnolence, and irritability. Infants also had an increased incidence of hypokalemia (50%) compared to other pediatric age cohorts (15-20%) or adults (17%).
Dosage and Administration Guidelines

BLINCYTO is administered as a continuous intravenous infusion at a constant flow rate using an infusion pump which should be programmable, lockable, non-elastomeric, and have an alarm.
It is very important that the instructions for preparation (including admixing) and administration provided in the full Prescribing Information are strictly followed to minimize medication errors (including underdose and overdose).

Alpha Tau to Present at Biotech Showcase and Participate in LifeSci Advisors Corporate Access Event During J.P. Morgan Healthcare Conference in San Francisco in January 2023

On December 13, 2022 Alpha Tau Medical Ltd. (Nasdaq: DRTS, DRTSW) ("Alpha Tau," or the "Company"), the developer of the innovative alpha-radiation cancer therapy Alpha DaRT, reported that the Company plans to present at the Biotech Showcase and host institutional investor meetings at this event, as well as at the LifeSci Partners Corporate Access Event (Press release, Alpha Tau Medical, DEC 13, 2022, View Source [SID1234625170]). Both in-person events are taking place in parallel with the 41st Annual J.P. Morgan Healthcare Conference which will be held January 9-12, 2023 in San Francisco, California.

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Biotech Showcase:

Raphi Levy, Chief Financial Officer, will provide an overview of the Company, including its Alpha DaRT technology, recent pre-clinical and clinical progress, and upcoming clinical trials including its U.S. multi-center pivotal trial in recurrent cutaneous squamous cell carcinoma. Uzi Sofer, CEO of Alpha Tau and Raphi Levy will also host 1×1 institutional investor meetings.

Date/Time: Monday January 9, 2023, at 3 pm PT

Location: Hilton San Francisco Union Square in San Francisco; Yosemite A (Ballroom Level)

To schedule a meeting, investors can register on the Biotech Showcase website (https://informaconnect.com/biotech-showcase/registration-options/)

LifeSci Partners Corporate Access Event:

Uzi Sofer, CEO and Raphi Levy, CFO will be hosting 1×1 institutional investor meetings.

Date: Tuesday, January 10, 2023

Location: Beacon Grand Hotel in San Francisco

To schedule a meeting on the online system managed by LifeSci Partners, please click here (View Source) to register for the conference.

About Alpha DaRT

Alpha DaRT (Diffusing Alpha-emitters Radiation Therapy) is designed to enable highly potent and conformal alpha-irradiation of solid tumors by intratumoral delivery of radium-224 impregnated sources. When the radium decays, its short-lived daughters are released from the sources and disperse while emitting high-energy alpha particles with the goal of destroying the tumor. Since the alpha-emitting atoms diffuse only a short distance, Alpha DaRT aims to mainly affect the tumor, and to spare the healthy tissue around it.

WuXiDiagnostics and Abcam expand existing collaboration to advance companion diagnostics for cancer therapies

On December 13, 2022 WuXiDiagnostics, an enabling platform company that implements integrated diagnostics and Abcam (AIM:ABC; NASDAQ:ABCM), a global life science company working together with researchers to advance science and enable faster breakthroughs, reported that a new supply and licensing agreement in the area of companion diagnostic reagents to support the development of innovative anti-cancer treatments in China (Press release, Abcam, DEC 13, 2022, View Source [SID1234625168]).

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The rapid development of targeted cancer immunotherapy in the past decade has accelerated the need for accurate and efficient companion diagnostic tests for biomarker status assessment. At patient selection, the detection of disease-related biomarkers helps to identify individuals that may respond to a certain targeted therapy, thereby supporting safe and effective treatment. During treatment, companion diagnostic tests provide vital pharmacodynamic data on how patients respond.

Building on their existing relationship, this new collaboration leverages Abcam’s industry-leading antibody development capabilities, combined with WuXiDiagnostics’ long-standing research and development expertise in the area of companion diagnostics. The two parties will join forces to comprehensively assist drug development and diagnostic tools, to continue to fuel the growth of the life science industry and enable the faster delivery of positive outcomes for science and health.

Yin Wang, Senior Director of pharma company service and Companion Diagnostics at WuXiDiagnostics, said: "Since its foundation, developing companion diagnostic reagents with pharmaceutical partners has been one of WuXiDiagnostics‘ major strategies. This whole process relies on excellent antibody quality and supply chain stability. I am very happy that WuXiDiagnostics has established a long-term strategic cooperation with Abcam, one of the world’s top antibody manufacturers. Together we are poised to deliver the best solutions for the clinical progression of anti-cancer drugs and the application of precision medicine."

Emma Sceats, SVP Sales, Service & Business Development at Abcam, said: "I am excited that we are further developing our cooperation with WuXiDiagnostics. With our combined expertise, Abcam and WuXiDiagnostics will enable the biopharma industry to achieve fast advances and develop innovative medicine that has the potential to benefit more people in China."

Biosion Announces Research Collaboration with ImmunoGen to Create Novel Antibody-Drug Conjugates

On December 13, 2022 Biosion USA, Inc. (Biosion), a global R&D biotechnology company, reported an exploratory research collaboration to create antibody-drug conjugates (ADCs) for the treatment of cancer with ImmunoGen, Inc. (Nasdaq: IMGN), a leader in developing next generation ADCs (Press release, Biosion, DEC 13, 2022, View Source [SID1234625164]). In the joint research effort, Biosion will leverage its proprietary SynTracer high-throughput (HT) endocytosis platform to generate highly selective antibodies to targets allocated by each company and ImmunoGen will provide their proprietary linker-payload technology to create novel ADCs.

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"We are excited about our new joint research effort with ImmunoGen, a world-renowned leader in ADCs," said Hugh Davis, Ph.D., Chief Operating Officer, and President of Biosion USA, Inc. "This collaboration will take advantage of the strengths of each company; ImmunoGen’s expertise in linkers and payloads combined with Biosion’s SynTracer HT endocytosis platform with the goal of identifying novel treatments for solid tumor cancers."

Ascentage Pharma Presents Latest Data of APG-2575, Including Encouraging Results of the Combination with BTKi in Patients with R/R CLL/SLL

On December 13, 2022 Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, reported that it has released preliminary results from a global Phase II study of lisaftoclax (APG-2575), a key member of the company’s apoptosis-targeting pipeline, as a monotherapy or in combination with CALQUENCE (acalabrutinib) or rituximab in patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (R/R CLL/SLL), in an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) 64th Annual Meeting & Exposition (New Orleans, LA) (Press release, Ascentage Pharma, DEC 13, 2022, View Source;ascentage-pharma-presents-latest-data-of-apg-2575-including-encouraging-results-of-the-combination-with-btki-in-patients-with-rr-cllsll-301701343.html [SID1234625163]).

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The ASH (Free ASH Whitepaper) Annual Meeting is one of the largest gatherings of the international hematology field, featuring world-class advances on cutting-edge scientific and clinical research in hematology. As a leading member of the Chinese hematology and oncology research community that has been increasingly active on the global stage, Ascentage Pharma had results from 5 of its clinical trials selected for 4 oral presentations at this year’s ASH (Free ASH Whitepaper) Annual Meeting, attracting widespread interest at the event. In total, Ascentage Pharma will have 8 presentations at ASH (Free ASH Whitepaper) 2022, including 4 oral and 4 poster presentations including 3 poster presentations submitted independently by investigators based on Real World Evidence.

In the results reported in the oral presentation, the Company’s investigational Bcl-2-selective inhibitor lisaftoclax showed promising therapeutic potential, both as a single agent and in combinations. In particular, the combinations showed high objective response rates (ORRs), on the order of 98% (56/57) when combined with acalabrutinib in patient with R/R CLL/SLL. In terms of tumor lysis syndrome (TLS), the combination regimens showed low incidences comparable to that of lisaftoclax monotherapy. In addition, unlike the 5-week dose-escalation applied in trials of other Bcl-2 inhibitors, this study of lisaftoclax adopted a daily dose ramp-up that allowed dose-escalation to be completed in only 4 to 6 days, allowing the patients to receive the full therapeutic dose earlier.

Acalabrutinib is a next-generation Bruton tyrosine kinase inhibitor (BTKi). In June 2020, Ascentage Pharma entered into a clinical collaboration with Acerta Pharma B.V., the hematology research and development center of excellence of AstraZeneca, to evaluate the combination of Ascentage Pharma’s investigational Bcl-2 inhibitor, lisaftoclax, and Acerta’s BTKi, acalabrutinib. The oral presentation at the 2022 ASH (Free ASH Whitepaper) Annual Meeting marks the first ever data release on the combination regimen.

The combination of lisaftoclax with the BTK inhibitor acalabrutinib resulted in an ORR of 98%," according to principal investigator Matthew S. Davids, MD, MMSc of Dana-Farber Cancer Institute (Boston, MA). "Given an encouraging safety profile, with limited TLS despite a daily dose ramp-up, these findings signal the potential clinical utility of this new Bcl-2 inhibitor in patients with CLL/SLL."

"Results reported at this year’s ASH (Free ASH Whitepaper) Annual Meeting have again shown lisaftoclax’s exciting therapeutic potential for the treatment of R/R CLL/SLL," according to Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "Combined use of Bcl-2 and BTK inhibitors has received much interest in recent years. For the first time, we announced the efficacy data of lisaftoclax plus acalabrutinib, with an ORR that is indeed very encouraging."

"At this year’s ASH (Free ASH Whitepaper) Annual Meeting, clinical researchers delivered four Company-sponsored oral presentations on lisaftoclax as well as our BCR-ABL1 inhibitor olverembatinib, which has been approved in China for the management of treatment-resistant chronic myeloid leukemia," according to Dr. Zhai. "We are very proud of this accomplishment as another validation of our robust global innovation capabilities. Ascentage Pharma remains committed to our founding mission of addressing unmet clinical needs in China and around the world for the benefit of more patients, and we will continue to accelerate our clinical development programs to bring well tolerated and effective therapeutics to patients as soon as possible."

These data of lisaftoclax reported in the oral presentation at this year’s ASH (Free ASH Whitepaper) Annual Meeting are as follows (for details of those oral presentations on olverembatinib, please refer to other two press releases to be published during ASH (Free ASH Whitepaper) 2022):

Lisaftoclax (APG-2575) Safety and Activity as Monotherapy or Combined with Acalabrutinib or Rituximab in Patients (pts) with Treatment-Naïve, Relapsed or Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (R/R CLL/SLL): Initial Data from a Phase 2 Global Study

Format: Oral Presentation
Abstract: 160386
Session: 642. Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Drugs in Development and COVID-19
Highlights:

Lisaftoclax, a specific Bcl-2 inhibitor, is active in patients with R/R CLL/SLL, including patients whose disease harbored del(17p) and had progressive disease (PD) after BTKi therapies. This is the first report of lisaftoclax combined with acalabrutinib or rituximab in patients with CLL/SLL.

Patients with R/R CLL/SLL were treated daily with oral lisaftoclax (400, 600, and
800 mg) alone or combined with continuous acalabrutinib or rituximab for six 28-day cycles. Primary objectives were to determine the recommended Phase II dose (RP2D), safety, and efficacy, including ORRs of lisaftoclax alone and combined with acalabrutinib or rituximab. Patients underwent lisaftoclax daily ramp-up over 4 to 6 days, with the monitoring of TLS. Dose ramp-up was followed by Cycle 1 Day 1 (C1D1) of lisaftoclax target doses of 400, 600, or 800 mg. Patients in the combination groups completed ramp-up, as well as an additional 7 days of lead-in of lisaftoclax at the target dose, before acalabrutinib or rituximab was added on C1D8, and then treated until PD or unacceptable toxicity was observed.

As of December 5, 2022, 164 patients had been enrolled. The lisaftoclax monotherapy cohort enrolled a total of 46 patients, with a median age of 60.5 (range, 41-80) years. The rituximab combination cohort enrolled a total of 39 patients, with a median age of 64 (34-75). The acalabrutinib combination cohort enrolled a total of 79 patients, with a median age of 64 (18-80). Of all patients, 16 (9.8%) were treatment-naïve and 19 (11.6%) had received prior treatment with BTKis. In the combination cohorts (n = 118), 25 patients had the TP53 mutation and/or del(17p), and 34 patients had unmutated IGHV. Median treatment duration with lisaftoclax monotherapy was 16.5 (range, 1-36) cycles, 11 (range, 0-21) cycles for the rituximab combination, and 11 (range, 1-24) cycles for the acalabrutinib combination.

Safety: Common adverse events (AEs) of any grade in all cohorts included neutropenia, diarrhea, and infections. Common AEs of grade ≥ 3 in the lisaftoclax monotherapy cohort included neutropenia (30.3%), COVID-19 infections (28%), anemia (15%), thrombocytopenia (6.5%), and pneumonia (6.5%). Common AEs of grade ≥ 3 in the rituximab combination cohort mainly included neutropenia (21%) and anemia (8%), thrombocytopenia (5%). Common AEs of grade ≥ 3 in the acalabrutinib combination cohort mainly included neutropenia (23%), COVID-19 infections (11.5%), anemia (10%), and thrombocytopenia (6.4%). First onset of grade ≥ 3 cytopenias mainly occurred during ramp-up or C1 and infrequently after C2. Grade ≥ 3 neutropenia was manageable with growth factor support. A total of 4 patients met Howard criteria for TLS (2 clinical TLS/2 laboratory TLS), and 2 with clinical TLS fully recovered and showed responses at 600 mg. No dose-limiting toxicities (DLTs) were observed, and no drug-drug interactions were observed in either combination group.

Preliminary efficacy: ORRs were 67% (29/43) in the monotherapy group, including 67% (4/6) in patients who were BTKi resistant or intolerant; 98.6% (72/73) in the acalabrutinib combination cohort, including 98% (56/57) in relapsed/refractory patients, 100% (16/16) in treatment-naïve patients,and 88% (7/8) in prior BTKi resistant or intolerant patients; and 79% (27/34) in the rituximab combination cohort.

Conclusions: Initiated with a daily dose ramp-up, lisaftoclax alone or combined with acalabrutinib or rituximab had a manageable safety profile and favorable clinical activity in patients with treatment-naïve or R/R CLL/SLL.