Aptose Presents Highlights from Clinical Update

On June 10, 2023 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral kinase inhibitors to treat hematologic malignancies, reported highlights from a clinical update event held today, June 10, 2023, in conjunction with EHA (Free EHA Whitepaper) 2023 International Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in Frankfurt, Germany (Press release, Aptose Biosciences, JUN 10, 2023, View Source [SID1234632619]). The event included an up-to-date review of clinical data for Aptose’s two investigational products under development for hematologic malignancies: tuspetinib, an oral, myeloid kinase inhibitor in the Phase 1/2 APTIVATE trial in patients with relapsed or refractory acute myeloid leukemia (AML); and luxeptinib, an oral, dual lymphoid and myeloid kinase inhibitor in Phase 1 a/b stage development for the treatment of patients with relapsed or refractory hematologic malignancies. The webcast of the presentation is available on Aptose’s website here.

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Aptose provided updated clinical findings with tuspetinib, a potent suppressor of FLT3, SYK, JAK 1/2, mutant forms of cKIT, and the RSK1/2 kinases operative in AML:

Completed tuspetinib dose escalation and dose exploration Phase 1/2 trial in 77 R/R AML patients.
Tuspetinib demonstrated a favorable safety profile.
Tuspetinib delivered monotherapy responses across four dose levels with no DLT in mutationally diverse and difficult to treat R/R AML populations, including TP53-mutated patients with a CR/CRh = 20% and RAS-mutated patients with a CR/CRh = 22%.
Completed successful End of Phase 1 (EOP1) Meeting with US FDA for tuspetinib, and a monotherapy RP2D was selected as 80mg daily, and all development paths remain open, including the single arm accelerated path.
Initiated tuspetinib APTIVATE expansion trial with R/R AML patients.
Tuspetinib is being administered as a monotherapy and as a combination doublet with tuspetinib + venetoclax (TUS/VEN), and enrollment has been brisk.
TUS/VEN doublet has been well tolerated, all patients remain on study, and preliminary CR activity has already been reported in patients previously treated with VEN.
Aptose also reviewed clinical findings with the new G3 formulation of luxeptinib (Lux):

50mg G3 formulation with continuous dosing achieves roughly equivalent PK profile as 900mg original G1 formulation.
Expect to dose escalate G3 formulation with continuous dosing in patients soon.
"We are delighted to have finalized our dose escalation and dose exploration Phase 1/2 trial with tuspetinib (TUS), to have demonstrated single agent responses across four dose levels that had no DLTs and across a range of R/R AML populations with a diversity of adverse mutations," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer. "As part of our EOP1 meeting with the FDA, we designated 80mg daily as our monotherapy recommended phase 2 dose (RP2D), and now we focus all attention on our APTIVATE expansion trial to place monotherapy TUS in more patients with highly adverse mutations and to evaluate TUS in combination with venetoclax (VEN) as a doublet in R/R AML patients. Notably, to date, the doublet appears to be well tolerated, with all patients remaining on study, including a preliminary CR in a R/R AML patient who previously failed venetoclax. We look forward to accelerating testing of the doublet, adding MDS patients to our APTIVATE trial, and with the aim of moving into triplet therapy (TUS/VEN/HMA) in 1L AML patients."

Nkarta Presents NKX019 Clinical Data at the European Hematology Association 2023 Congress and 17th International Conference on Malignant Lymphoma

On June 10, 2023 Nkarta, Inc. (Nasdaq: NKTX), a biopharmaceutical company developing engineered natural killer (NK) cell therapies, reported presentations highlighting preliminary data based on a November 2022 data cut-off from its Phase 1 dose escalation clinical trial of NKX019 at two scientific conferences: the European Hematology Association (EHA) (Free EHA Whitepaper) 2023 Hybrid Congress and the 17TH International Conference on Malignant Lymphoma (17-ICML) (Press release, Nkarta, JUN 10, 2023, View Source [SID1234632614]). NKX019 is an allogeneic, off-the-shelf NK cell therapy candidate derived from healthy donors and engineered to target CD19.

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Seven of ten patients with relapsed/ refractory non-Hodgkin lymphoma (NHL) treated at the higher dose levels showed a complete response (70% CR), including two patients with aggressive large B cell lymphoma (LBCL) as well as other difficult histologies, including mantle cell lymphoma (MCL), high-risk follicular lymphoma (FL) and marginal zone lymphoma (MZL). No dose limiting toxicity, neurotoxicity / ICANS, graft versus host disease (GvHD), or >Grade 3 cytokine release syndrome (CRS) were observed in the study.

"Autologous CAR-T cell therapies set a standard for responses in patients with relapsed/ refractory B-cell malignancies. However, potential for toxicity and logistic challenges have limited access to these therapies, and many patients could still benefit from a safe, on-demand treatment," said Michael Dickinson, M.D., Lead, Aggressive Lymphoma disease group, Clinical Haematology, Peter MacCallum Cancer Centre and Royal Melbourne Hospital, and investigator in the NKX019 trial. "In this early evaluation of an allogeneic CAR-NK cell therapy candidate, NKX019 had a manageable safety profile with encouraging anti-tumor activity as well as the option for retreatment after relapse. Based on these early data, NKX019 merits further study as a potential outpatient cell therapy approach."

"These data highlight the encouraging safety profile and clinical activity across different histologies in the dose escalation portion of the NKX019 study," said David R. Shook, M.D., Nkarta’s Chief Medical Officer. "We continue to explore the potential of allogeneic CAR NK cells, leveraging their biology to create a differentiated cellular therapy, and we look forward to the next update on the NKX019 program later this year."

Nkarta’s presentation materials from EHA (Free EHA Whitepaper) and ICML will be available for download on the Nkarta website (View Source). The presentations will ensure that the broader clinical and academic community has the opportunity to assess the NKX019 clinical data in a peer-reviewed format. All data were previously disclosed at a company event in December 2022. Nkarta plans to provide an update from the NKX019 program, including data from dose expansion cohorts, in 2023.

About NKX019
NKX019 is an allogeneic, cryopreserved, off-the-shelf cancer immunotherapy candidate that uses natural killer (NK) cells derived from the peripheral blood of healthy adult donors. It is engineered with a humanized CD19-directed CAR for enhanced tumor cell targeting and a proprietary, membrane-bound form of interleukin-15 (IL-15) for greater persistence and activity without exogenous cytokine support. CD19 is a biomarker for normal and malignant B cells, and it is a validated target for B cell cancer therapies. To learn more about the NKX019 clinical trial in adults with advanced B cell malignancies, please visit ClinicalTrials.gov.

Theolytics presents data on THEO-260 and THEO-310 at ASCO and EHA

On June 9, 2023 Theolytics, a biotechnology company harnessing viruses to combat disease, reported data presentations at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, which was held 2 to 6 June, 2023 in Chicago and at the European Hematology Association (EHA) (Free EHA Whitepaper) 2023 Congress, which is being held 8 to 11 June in Frankfurt (Press release, Theolytics, JUN 9, 2023, View Source [SID1234635716]).

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ASCO Abstract

Title: Evaluation of THEO-260 as a novel oncolytic virus therapy for treating stromal rich tumours

LINK to abstract

Stromal rich cancers pose a challenge to many cancer therapies due to the abundance of cancer associated fibroblasts (CAFs). This compartment of the tumour microenvironment physically blocks the penetration of drugs, immune cells and promotes resistance to many therapies. THEO-260 is a new oncolytic virus in development that is intrinsically capable of targeted killing of both cancer cells and CAFs.

The efficacy and safety of THEO-260 was assessed in multicellular spheroids, in patient-derived ex vivo fresh ovarian samples and in mouse models of human stromal tumours. Unpassaged ascites and tumour samples collected from cytoreductive surgeries for advanced ovarian cancer were treated with a clinically-relevant dose of THEO-260. THEO-260 effectively killed the cancer cell and CAF populations, being efficacious in treatment-naive and platinum-resistant patient samples. Importantly, the T cell population in these patients, in addition to primary normal fibroblasts from healthy donors, were not damaged. Ex vivo studies show THEO-260 stimulates effector T cell responses upon tumour lysis and demonstrates suitability as an IV

delivered agent. THEO-260 excels in low nutrient, low proliferative environments, typical of human solid tumours and has been developed with a view to persist in the tumour microenvironment without the need for repeated cycles of delivery. In ovarian cancer murine models containing human CAFs and cancer cells, THEO-260 shows antitumor efficacy, with a complete reduction in tumour volume in subcutaneous and intraperitoneal systems, and superiority to standard of care. Virus activity is not restricted to ovarian cancer, with cell line data demonstrating killing in additional solid tumour types, including pancreatic, colorectal, lung, and breast cancer. THEO-260 exhibits genetic and temperature based stability, and GMP manufacturing to high yields is underway.

"The positive THEO-260 data presented at ASCO (Free ASCO Whitepaper) come at an exciting time for Theolytics as we progress this lead product towards the clinic in a first-in-human study in ovarian cancer," said Charlotte Casebourne Stock, Chief Executive Officer and Co-Founder of Theolytics. "THEO-260 is a virotherapy that potentially could demonstrate something that neither chemotherapy, nor immunotherapeutic approaches have been capable of to date and has the potential to deliver a step-change for the oncolytic virus field."

EHA Poster Presentation

Title: Development of a Novel Oncolytic Virus – THEO-310 – Specialised for Targeting the Bone Marrow Tumour Microenvironment of Multiple Myeloma Patients

LINK to abstract

Session Title: 24. Gene therapy, cellular immunotherapy and vaccination – Biology & Translational Research

Poster ID: P1362

Session date and time: Friday, 9 June, 18:00 – 19:00 CEST

Session room: Poster area

Presenting Author: Dr Margaret Duffy

THEO-310 is an oncolytic virus developed for tackling the bone marrow tumour microenvironment (TME) of advanced late-stage myeloma patients. THEO-310 demonstrates efficacy in >80% of ex vivo bone marrow aspirates, across newly diagnosed, relapsed and refractory myeloma patients. In these experiments, TME immune modulation and superiority to ‘best-in-class’ oncolytic virus strains for myeloma, including the measles virus, was observed.

"These exciting set of results differentiates Theolytics’ approach to other oncolytic virus candidates. In addition to showing increased efficacy in patients ex vivo, the candidate demonstrates systemic safety as shown by cytokines levels post IV injection in vivo, whilst exhibiting >40-fold better circulation compared to a clinically validated oncolytic virus strain, thus supporting effective intravenous delivery," said Margaret Duffy, Chief Scientific Officer and Co-Founder of Theolytics. "Taken together, THEO-310 represents a new therapeutic option, with a highly differentiated mechanism independent of BCMA, for myeloma patients with relapsed refractory disease.

NovalGen announces two oral presentations at upcoming conferences

On June 9, 2023 NovalGen Ltd ("NovalGen"), a biopharmaceutical company developing disruptive therapies, based on a proprietary autoregulation (AR) platform, reported the presentation of preclinical data on this novel technology in two separate indications at the International Society on Thrombosis and Haemostasis Congress (ISTH), 24-28 June, 2023 in Toronto and the Immuno-Oncology Summit Boston 2023, 7-9 August, 2023 in Boston (Press release, NovalGen, JUN 9, 2023, View Source [SID1234633763]).

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"We are delighted to be showcasing our proprietary platform, which has the potential to improve the safety profile and therapeutic index of a range of immunotherapies. Preclinical data with NVG-444 shows a reduction in thrombotic complications with a bispecific Factor VIII mimetic. These data will be presented in an oral presentation at ISTH. The AR platform is target agnostic as illustrated by its application to an extended half-life T cell engager (TCE) targeting ROR1, resulting in a dramatic reduction in toxicity, thus addressing the "Achilles heel" of TCEs allowing more patients to benefit from this off the shelf, T cell activating technology. The AR technology can be applied to a wide range of immunotherapies and heralds a new and exciting class of self-regulating drugs that respond to biological cues to avoid life-threatening toxicities," said Professor Amit Nathwani, Founder and CEO of NovalGen.

Both abstracts will be delivered as oral presentations at the conferences.

Abstracts to be presented:

Conference: ISTH 2023 Congress
Abstract Title: Thrombin-mediated autoregulation restores sensitivity to the coagulation feedback loop and reduces the risk of prothrombotic events associated with FVIII mimetic antibodies.

Abstract Presentation Number: OC 69.1

Session Title: Hemophilia – Novel therapies II

Session Date: Wednesday, 28 June, 2023
Presentation Time: 10:15 am – 10:30 am

Presenting Author: Vincent Muczynski

Conference: Immuno-Oncology Summit Boston 2023
Abstract Title: NVG-222: A ROR1-Targeting T Cell Engager with Integral Autoregulating Capability Designed to Reduce the Risk of Serious Adverse Events Related to T Cell Activation

Session Title: Bispecific Antibodies for Cancer Immunotherapy: Overcoming Obstacles to Efficacy

Session Date: Tuesday, 8 August, 2023

Presentation Time: 8:05am – 8:35am

Presenting Author: David Granger

AbbVie’s VENCLYXTO®/VENCLEXTA® (venetoclax) Continues to Show Sustained Progression-Free Survival (PFS) in Chronic Lymphocytic Leukemia (CLL) Patients

On June 9, 2023 AbbVie (NYSE: ABBV) reported new findings demonstrating sustained long-term safety and efficacy of VENCLYXTO/ VENCLEXTA (venetoclax)-based combination therapies in patients with previously untreated CLL with co-existing conditions, as well as R/R CLL (Press release, AbbVie, JUN 9, 2023, View Source [SID1234632624]). The results are being presented during oral sessions at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress in Frankfurt, Germany.

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"Results from the CLL14 and MURANO studies demonstrate the long-term benefits of fixed-duration venetoclax combinations for patients living with CLL," said Mariana Cota Stirner, M.D., Ph.D., vice president, hematology, AbbVie. "These results underscore our commitment to transform how blood cancers are treated today and show that venetoclax can give patients lasting results with time off treatment."

CLL14 Long-Term Analysis

New six-year follow-up results from the Phase 3 CLL14 study showcase updated outcomes in previously untreated patients with CLL and co-existing conditions. Patients treated with fixed-duration venetoclax plus obinutuzumab continued to experience improved PFS (95% Confidence Interval (CI) 0.31-0.52; Hazard Ratio (HR) 0.40) and higher rates of undetectable minimal residual disease (uMRD) when treated with fixed-duration venetoclax plus obinutuzumab compared to those who received chlorambucil plus obinutuzumab (53.1% vs 21.7%, respectively).

The data also showed significantly improved rates of time to next treatment (TTNT) with venetoclax plus obinutuzumab at 65.2 percent (95% CI 0.33-0.58; HR 0.44) compared to chlorambucil plus obinutuzumab at 37.1 percent.1 The observed differences in PFS and TTNT benefits between venetoclax-based treatment and chemoimmunotherapy were maintained across all risk groups, including patients with high-risk molecular features of CLL.

No new safety signals were observed in this six-year analysis. The most frequently occurring Grade 3 (≥2%) adverse events (AEs) in patients receiving the venetoclax-based combination were neutropenia, thrombocytopenia, infusion-related reaction (during treatment), anemia, febrile neutropenia, pneumonia and leukopenia.1

"The latest findings show that patients can experience long-term disease control, five years after stopping treatment," said Othman Al-Sawaf, M.D., investigator in the CLL14 study, hematologist-oncologist at the University Hospital Cologne in Germany, and study physician at the German CLL Study Group. "These results confirm the treatment benefits of fixed-duration venetoclax and obinutuzumab for previously untreated CLL patients with co-existing conditions."

Results will also be featured at EHA (Free EHA Whitepaper)’s Press Briefing.

MURANO Long-Term Analysis

Final data from the Phase 3 MURANO trial showcase that R/R CLL patients treated with two-year fixed-duration venetoclax plus rituximab sustained significantly longer median PFS at 54.7 months (95% CI 52.3, 59.9), the study’s primary endpoint, compared to 17.0 months (95% CI 15.5, 21.7; HR 0.23) with bendamustine plus rituximab after 7 years of median follow-up.2

Seven-year OS rates were 69.6 percent (95% CI 62.8, 76.5) for patients treated with the venetoclax-based combination versus 51 percent (95% CI 43.3, 58.7) for study participants who received bendamustine-based combination (HR 0.53).2 Furthermore, most of the patients treated with the full two-year venetoclax-based combination achieved uMRD (70.3%) at the end of their treatment course, and those patients were shown to have improved PFS and OS compared to patients with detectable MRD (29.7%).

The safety profile of the venetoclax-rituximab combination is consistent with the known safety profile of each individual therapy alone. No new serious safety issues were observed in the MURANO updated analysis. The most common adverse reactions (ARs) (≥20%) of any grade were neutropenia, diarrhea, upper respiratory tract infection, fatigue, and nausea.

"We are pleased to find that uMRD was associated with prolonged PFS in R/R CLL patients after seven years," said study investigator Prof. John Seymour, Director of the Integrated Haematology Department of the Peter MacCallum Cancer Center and the Royal Melbourne Hospital in Melbourne. "Overall, these findings continue to support the use of treatment with venetoclax plus rituximab in this patient population."

VENCLYXTO/VENCLEXTA is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S.

About the CLL14 Phase 3 Trial1,3
The prospective, multicenter, open-label, randomized Phase 3 CLL14 trial, which was conducted in close collaboration with the German CLL Study Group (GCLLSG), evaluated the efficacy and safety of a combined regimen of VENCLYXTO/VENCLEXTA and obinutuzumab (n=216) versus obinutuzumab and chlorambucil (n=216) in previously untreated patients with CLL and co-existing medical conditions (total Cumulative Illness Rating Scale [CIRS] score >6 or creatinine clearance <70 mL/min). The therapies were administered for a fixed-duration of 12 months for VENCLYXTO/VENCLEXTA in combination with six cycles of obinutuzumab. The trial enrolled 432 patients, all of whom were previously untreated, according to the International Workshop on Chronic Lymphocytic Leukemia (iwCLL) criteria. Efficacy was based on PFS, as assessed by an independent review committee.

Key secondary endpoints were rates of MRD in peripheral blood and bone marrow, overall and complete response rates, MRD in complete response in peripheral blood and bone marrow, and OS.

In patients with CLL receiving venetoclax combination therapy with obinutuzumab, the most frequently occurring ≥ Grade 3 AEs (≥2%) were neutropenia (51.9%), thrombocytopenia (14.2%), infusion-related reaction (9.0%), anemia (7.5%), febrile neutropenia (4.2%), pneumonia (3.8%) and leukopenia (2.4%). Serious ARs were most often due to febrile neutropenia and pneumonia (5% each). The most common ARs (≥20%) of any grade were neutropenia (60%), diarrhea (28%), and fatigue (21%). Fatal ARs that occurred in the absence of disease progression and with onset within 28 days of the last study treatment were reported in 2 percent (4/212) of patients, most often from infection.

About the MURANO Phase 3 Trial2,4
A total of 389 patients with R/R CLL who had received at least one prior therapy were enrolled in the international, multicenter, open-label, randomized Phase 3 MURANO trial. The trial was designed to evaluate the efficacy and safety of VENCLYXTO/VENCLEXTA and rituximab (n=194) compared with bendamustine and rituximab (n=195). The median age of patients in the trial was 65 years (range: 22 to 85).

The trial met its primary efficacy endpoint of investigator (INV)-assessed PFS. At the time of the primary analysis, median PFS with VENCLYXTO/VENCLEXTA and rituximab was not reached compared with 17.0 months for bendamustine and rituximab (HR: 0.17; 95% CI: 0.11- 0.25; p<0.0001). In the primary efficacy analysis, the median follow-up for PFS was 23.8 months (range: 0 to 37.4). Additional efficacy endpoints included independent review committee (IRC)-assessed PFS, INV- and IRC-assessed overall response rate (defined as complete response + complete response with incomplete marrow recovery + partial response + nodular partial response), OS and rates of MRD-negativity.

In patients with CLL receiving combination therapy with rituximab, the most frequent serious adverse reaction (AR; ≥5%) was pneumonia (9%). The most common ARs (≥20%) of any grade were neutropenia (65%), diarrhea (40%), upper respiratory tract infection (39%), fatigue (22%), and nausea (21%). Fatal ARs that occurred in the absence of disease progression and within 30 days of the last venetoclax treatment and/or 90 days of the last rituximab were reported in 2% (4/194) of patients.

About VENCLYXTO (venetoclax)

VENCLYXTO (venetoclax) is a first-in-class medicine that selectively binds and inhibits the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers, BCL-2 prevents cancer cells from undergoing their natural death or self-destruction process, called apoptosis. VENCLYXTO targets the BCL-2 protein and works to help restore the process of apoptosis.

VENCLYXTO is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S. Together, the companies are committed to BCL-2 research and to studying venetoclax in clinical trials across several blood and other cancers. Venetoclax is approved in more than 80 countries, including the U.S.

Indication and Important VENCLYXTO (venetoclax) EU Safety Information5

Indications

Venclyxto in combination with obinutuzumab is indicated for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL).

Venclyxto in combination with rituximab is indicated for the treatment of adult patients with CLL who have received at least one prior therapy.

Venclyxto monotherapy is indicated for the treatment of CLL:

In the presence of 17p deletion or TP53 mutation in adult patients who are unsuitable for or have failed a B-cell receptor pathway inhibitor, or
In the absence of 17p deletion or TP53 mutation in adult patients who have failed both chemoimmunotherapy and a B-cell receptor pathway inhibitor.
Venclyxto in combination with a hypomethylating agent is indicated for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) who are ineligible for intensive chemotherapy.

Contraindications

Hypersensitivity to the active substance or to any of the excipients is contraindicated. Concomitant use of strong CYP3A inhibitors at initiation and during the dose-titration phase due to increased risk for tumour lysis syndrome (TLS). Concomitant use of preparations containing St. John’s wort as Venclyxto efficacy may be reduced.

Special Warnings & Precautions for Use

Tumour Lysis syndrome, including fatal events, has occurred in patients when treated with Venclyxto. For CLL and AML, please refer to the indication-specific recommendations for prevention of TLS in the Venclyxto summary of product characteristic (SmPC).

Patients should be assessed for risk and should receive appropriate prophylaxis, monitoring, and management for TLS. The risk of TLS is a continuum based on multiple factors, including comorbidities. Venclyxto poses a risk for TLS at initiation and during the dose-titration phase. Changes in electrolytes consistent with TLS that require prompt management can occur as early as 6 to 8 hours following the first dose of Venclyxto and at each dose increase.

Neutropenia (grade 3 or 4) has been reported. Complete blood counts should be monitored throughout the treatment period.

In patients with AML, neutropenia (grade 3 or 4) is common before starting treatment. The neutrophil counts can worsen with Venetoclax in combination with a hypomethylating agent. Neutropenia can recur with subsequent cycles of therapy. Dose modification and interruptions for cytopenias are dependent on remission status.

For CLL and AML, please refer to the indication-specific recommendations for dose modifications for toxicities in the Venclyxto SmPC.

Serious infections including sepsis with fatal outcome have been reported. Monitoring of any signs and symptoms of infection is required. Suspected infections should receive prompt treatment including antimicrobials and dose interruption or reduction as appropriate.

Live vaccines should not be administered during treatment or thereafter until B-cell recovery.

Drug Interactions

In CLL and AML CYP3A inhibitors may increase Venclyxto plasma concentrations.

In CLL, at initiation and dose-titration phase, Strong CYP3A inhibitors are contraindicated due to increased risk for TLS and moderate CYP3A inhibitors should be avoided. If moderate CYP3A inhibitors must be used, please refer to the recommendations for dose modifications in the Venclyxto SmPC.

In AML, please refer to the AML-specific recommendation for dose modifications for potential interactions with CYP3A inhibitors, in the VENCLYXTO SmPC.

Avoid concomitant use of P-gp and BCRP inhibitors at initiation and during the dose titration phase.

CYP3A4 inducers may decrease Venclyxto plasma concentrations. Avoid coadministration with strong or moderate CYP3A inducers. These agents may decrease venetoclax plasma concentrations.

Co-administration of bile acid sequestrants with VENCLYXTO is not recommended as this may reduce the absorption of VENCLYXTO.

Adverse Reactions

CLL

The most commonly occurring adverse reactions (>=20%) of any grade in patients receiving venetoclax in the combination studies with obinutuzumab or rituximab were neutropenia, diarrhoea, and upper respiratory tract infection. In the monotherapy studies, the most common adverse reactions were neutropenia/neutrophil count decreased, diarrhoea, nausea, anaemia, fatigue, and upper respiratory tract infection.

The most frequently occurring serious adverse reactions (>=2%) in patients receiving venetoclax in combination with obinutuzumab or rituximab were pneumonia, sepsis, febrile neutropenia, and TLS. In the monotherapy studies, the most frequently reported serious adverse reactions (>=2%) were pneumonia and febrile neutropenia.

Discontinuations due to adverse reactions occurred in 16% of patients treated with venetoclax in combination with obinutuzumab or rituximab in the CLL14 and Murano studies, respectively. In the monotherapy studies with venetoclax, 11% of patients discontinued due to adverse reactions.

Dosage reductions due to adverse reactions occurred in 21% of patients treated with the combination of venetoclax and obinutuzumab in CLL14, in 15% of patients treated with the combination of venetoclax and rituximab in Murano, and in 14% of patients treated with venetoclax in the monotherapy studies. The most common adverse reaction that led to dose interruptions was neutropenia.

AML

The most commonly occurring adverse reactions (>=20%) of any grade in patients receiving venetoclax in combination with azacitidine or decitabine in the VIALE-A and M14-358, respectively, were thrombocytopenia, neutropenia, febrile neutropenia, nausea, diarrhoea, vomiting, anaemia, fatigue, pneumonia, hypokalaemia, and decreased appetite, haemorrhage, dizziness/syncope, hypotension, headache, abdominal pain, and anaemia.

The most frequently reported serious adverse reactions (≥5%) in patients receiving venetoclax in combination with azacitidine were febrile neutropenia, pneumonia, sepsis and haemorrhage. In M14-358, the most frequently reported serious adverse reactions (≥5%) were febrile neutropenia, pneumonia, bacteraemia and sepsis.

Discontinuations due to adverse reactions occurred in 24% of patients treated with venetoclax in combination with azacitidine in the VIALE-A study, and 26% of patients treated with venetoclax in combination with decitabine in the M14-358 study, respectively.

Dosage reductions due to adverse reactions occurred in 2% of patients in VIALE-A, and in 6% of patients in M14-358. Venetoclax dose interruptions due to adverse reactions occurred in 72% and 65% of patients, respectively. The most common adverse reaction that led to dose interruption (>10%) of Venetoclax in VIALE-A, were febrile neutropenia, neutropenia, pneumonia, and thrombocytopenia. The most common adverse reactions that led to dose interruption (≥5%) of venetoclax in M14-358 were febrile neutropenia, neutropenia/neutrophil count decreased, pneumonia, platelet count decreased, and white blood cell count decreased.

Special Populations

Patients with reduced renal function (CrCl <80 mL/min) may require more intensive prophylaxis and monitoring to reduce the risk of TLS at initiation and during the dose-titration phase. Safety in patients with severe renal impairment (CrCl <30 mL/min) or on dialysis has not been established, and a recommended dose for these patients has not been determined.

For patients with severe (Child-Pugh C) hepatic impairment, a dose reduction of at least 50% throughout treatment is recommended.

Venclyxto may cause embryo-fetal harm when administered to a pregnant woman. Advise nursing women to discontinue breastfeeding during treatment.

This is not a complete summary of all safety information. See Venclyxto (venetoclax) SmPC at www.ema.europa.eu. Globally, prescribing information varies; refer to the individual country product label for complete information.