Menarini Group Shares NEXPOVIO® (selinexor) Subgroup Data from BOSTON trial at the European Hematology Association Congress, Highlighting Clinical Potential in Relapsed/Refractory Multiple Myeloma

On June 9, 2023 The Menarini Group ("Menarini"), a leading Italian pharmaceutical and diagnostics company, and Stemline Therapeutics, Inc. ("Stemline"), a wholly-owned subsidiary of the Menarini Group, reported important new data on NEXPOVIO (selinexor) at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress 2023 (Press release, Menarini, JUN 9, 2023, View Source [SID1234632621]).

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The two abstracts at EHA (Free EHA Whitepaper) bring new subgroup data from the Phase 3 BOSTON study (NCT03110562) in relapsed refractory multiple myeloma (RRMM). In patients with just one prior line of treatment, median progression-free survival (mPFS) was 21 months for those treated with SVd, versus 10.7 months for those treated with Vd alone (HR 0.62). In proteasome inhibitor (PI)-naïve patients, the mPFS was 29.5 months with SVd compared to 9.7 months with Vd alone (HR 0.29). In patients who were refractory to lenalidomide, overall survival was 26.7 months for the SVd arm, compared to 18.6 months for the Vd arm (HR 0.53). These efficacy analyses were based on the final data cut from the BOSTON trial in February 2021, representing a one-year update of previously presented data from 2020.

"The data presented today emphasize the synergy between selinexor and bortezomib, highlighting the importance of a double mode of action switch. These results are particularly relevant considering the increased use of the daratumumab lenalidomide dexamethasone combination in clinical practice today," said Professor Maria-Victoria Mateos, MD, PhD, University Hospital Salamanca, Spain. "These findings further support the use of selinexor in combination with bortezomib in PI / bortezomib-naïve or lenalidomide-refractory RRMM patients, as well as for patients at first relapse."

"We are proud to present these new subgroup data on selinexor," said Elcin Barker Ergun, CEO of the Menarini Group. "We are committed to bringing transformative new therapeutic options to the patients and families affected by cancer, including multiple myeloma."

The most common (≥25%) treatment-emergent adverse events (AEs) with SVd versus Vd in patients with one prior line of treatment were thrombocytopenia (61.6% vs 28.6%), nausea (52.5% vs 11.2%), fatigue (45.4% vs 17.3%), peripheral neuropathy (38.4% vs 52.0%), diarrhea (34.3% vs 24.5%), and anemia (30.3% vs 26.5%). Safety findings were similar in the PI-naïve and bortezomib-naïve subgroups, as well as the lenalidomide refractory subgroup. Safety data in these subgroups were consistent with those observed in the overall BOSTON study population.

2023 EHA (Free EHA Whitepaper) Annual Meeting Presentation Details
Abstract Title: Efficacy, Survival and Safety of Selinexor, Bortezomib and Dexamethasone (SVd) in Patients with Lenalidomide-Refractory Multiple Myeloma: Subgroup Data from the BOSTON Trial
Poster #: P886
Session Title: Myeloma and other monoclonal gammopathies – Clinical
Session Date and Time: June 9, 2023 18:00-19:00 CET
Presentation Type: Poster
Presenting Author: Dr. Maria-Victoria Mateos

Abstract Title: Selinexor, bortezomib, and dexamethasone in patients with previously treated multiple myeloma: updated results of BOSTON trial by prior therapies
Poster #: P917
Session Title: Myeloma and other monoclonal gammopathies – Clinical
Session Date and Time: June 9, 2023 18:00-19:00 CET
Presentation Type: Poster
Presenting Author: Dr. Maria-Victoria Mateos

In December 2021, Karyopharm Therapeutics Inc. and Menarini established an exclusive licensing partnership. Under this agreement, which was later amended in May 2022 and in March 2023, Menarini holds the responsibility for commercializing all existing and upcoming indications of NEXPOVIO in the European Economic Area, United Kingdom, Switzerland, CIS countries, Turkey, Middle East & Africa, and Latin America. Stemline Therapeutics B.V., a wholly-owned subsidiary of Menarini, is leading all commercialization activities in Europe.

About Multiple Myeloma in Europe
Multiple myeloma is an incurable cancer with significant morbidity and the second most common hematologic malignancy. According to the World Health Organization, in 2020, there were approximately 51,000 new cases and 32,000 deaths from multiple myeloma in Europe1. While the treatment of multiple myeloma has improved over the last 20 years, and overall survival has increased considerably, the disease remains incurable, and nearly all patients will eventually relapse and develop disease that is refractory to all approved anti-myeloma therapies. Therefore, there continues to be a high unmet medical need for new therapies, particularly those with novel mechanisms of action.

About the BOSTON study
The FDA and EMA approvals of selinexor with bortezomib and dexamethasone are based on the Phase 3 BOSTON study, which was a multi-center, randomized study that evaluated 402 adult patients with relapsed or refractory multiple myeloma who had received one to three prior lines of therapy. The study was designed to compare the efficacy, safety, and certain health-related quality of life parameters of once-weekly SVd versus twice-weekly Vd. The primary endpoint of the study was progression-free survival and key secondary endpoints included overall response rate, rate of peripheral neuropathy, and others. To learn more about this study, please refer to the Karyopharm press release announcing the publication of the BOSTON study results in The Lancet, issued on November 12, 2020.

About NEXPOVIO (selinexor)
NEXPOVIOhas been approved in the following oncology indications by the European Commission: (i) in combination with dexamethasone for the treatment of multiple myeloma in adult patients who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, two immunomodulatory agents and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy; and (ii) in combination with bortezomib and dexamethasone for the treatment of adults with multiple myeloma who have received at least one prior therapy. The marketing authorization of NEXPOVIO is valid in the EU Member States as well as Iceland, Liechtenstein, Norway, and Northern Ireland. NEXPOVIO has been commercially available in Germany and Austria since Q4 2022.

NEXPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor. NEXPOVIO functions by selectively binding to and inhibiting the nuclear export protein exportin 1 (XPO1, also called CRM1). NEXPOVIO blocks the nuclear export of tumor suppressor, growth regulatory and anti-inflammatory proteins, leading to accumulation of these proteins in the nucleus and enhancing their anti-cancer activity in the cell. The forced nuclear retention of these proteins can counteract a multitude of the oncogenic pathways that, unchecked, allow cancer cells with severe DNA damage to continue to grow and divide in an unrestrained fashion.

Please see NEXPOVIO Summary of Product Characteristics (SmPC) and European Public Assessment Report at www.ec.europa.eu

Please refer to local prescribing information where NEXPOVIO is approved for full information.

IMPORTANT SAFETY INFORMATION
Contraindications: Hypersensitivity to selinexor or to any of the excipients listed in the SmPC.

Special warnings and precautions for use:

Recommended concomitant treatments
Patients should be advised to maintain adequate fluid and caloric intake throughout treatment. Intravenous hydration should be considered for patients at risk of dehydration.
Prophylactic concomitant treatment with a 5-HT3 antagonist and/or other anti-nausea agents should be provided prior to and during treatment with NEXPOVIO.

Haematology:
Patients should have their complete blood counts (CBC) assessed at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first two months of treatment.

Thrombocytopenia:
Thrombocytopenic events (thrombocytopenia and platelet count decreased) were frequently reported in adult patients receiving selinexor, which can be severe (Grade 3/4).
Grade 3/4 thrombocytopenia can sometimes lead to clinically significant bleeding and in rare cases may lead to potentially fatal haemorrhage. Thrombocytopenia can be managed with dose interruptions, modifications, platelet transfusions, and/or other treatments as clinically indicated.
Patients should be monitored for signs and symptoms of bleeding and evaluated promptly.

Neutropenia:
Severe neutropenia (Grade 3/4) has been reported with selinexor. In a few cases concurrent infections occurred in patients with Grade 3/4 neutropenia. Patients with neutropenia should be monitored for signs of infection and evaluated promptly.

Gastrointestinal toxicity:
Nausea, vomiting, diarrhoea, which sometimes can be severe and may require the use of anti-emetic and anti-diarrhoeal medicinal products. Prophylaxis with 5HT3 antagonists and/or other anti-nausea agents should be provided prior to and during treatment with selinexor. Fluids with electrolytes should be administered to prevent dehydration in patients at risk. Nausea/vomiting can be managed by dose interruptions, modifications, and/or initiation of other antiemetics medicinal products as clinically indicated. Diarrhoea can be managed with dose interruptions, modifications and/or administration of anti-diarrhoea medicinal products.

Weight loss and anorexia:
Patients should have their body weight, nutritional status and volume checked at baseline, during treatment, and as clinically indicated. Monitoring should be more frequent during the first two months of treatment. Patients experiencing new or worsening decreased appetite and weight may require dose modification, appetite stimulants, and nutritional consultations.

Confusional state and dizziness:
Patients should be instructed to avoid situations where dizziness or confusional state may be a problem and to not take other medicinal products that may cause dizziness or confusional state without adequate medical advice. Patients should be advised not to drive or operate heavy machinery until symptoms resolve.

Hyponatraemia:
Patients should have their sodium levels checked at baseline, during treatment, and as clinically indicated. Monitoring should be more frequent during the first two months of treatment. Hyponatraemia should be treated as per medical guidelines (intravenous sodium chloride solution and/or salt tablets), including dietary review.

Cataract:
Selinexor can cause new onset or exacerbation of cataract. Ophthalmologic evaluation may be performed as clinically indicated. Cataract should be treated as per medical guidelines, including surgery if warranted.

Tumour lysis syndrome (TLS):
TLS has been reported in patients receiving therapy with selinexor. Patients at a high risk for TLS should be monitored closely. Treat TLS promptly in accordance with institutional guidelines.

Fertility, pregnancy and lactation
Women of childbearing potential should be advised to avoid becoming pregnant or abstain from sexual intercourse while being treated with selinexor and for at least 1 week following the last dose of selinexor.

Women of childbearing potential/contraception in males and females:
Women of childbearing potential and male adult patients of reproductive potential should be advised to use effective contraceptive measures or abstain from sexual intercourse while being treated with selinexor and for at least 1 week following the last dose of selinexor.

Pregnancy:
There are no data from the use of selinexor in pregnant women. Selinexor is not recommended during pregnancy and in women of childbearing potential not using contraception.
If the patient becomes pregnant while taking selinexor, selinexor should be immediately discontinued, and the patient should be apprised of the potential hazard to the foetus.

Breast-feeding:
It is unknown whether selinexor or its metabolites are excreted in human milk. A risk to breast-fed children cannot be excluded. Breast-feeding should be discontinued during treatment with selinexor and for 1 week after the last dose.

Undesirable effects
Summary of the safety profile
The most frequent adverse reactions (≥30%) of selinexor in combination with bortezomib and dexamethasone were nausea, thrombocytopenia, fatigue, anaemia, decreased weight, diarrhea, and peripheral neuropathy.

The most commonly reported serious adverse reactions (≥3%) were pneumonia, cataract, sepsis, diarrhoea, vomiting and anaemia.

The most frequent adverse reactions (≥30%) of selinexor in combination with dexamethasone were nausea, thrombocytopenia, fatigue, anaemia, decreased appetite, decreased weight, diarrhoea, vomiting, hyponatraemia, neutropenia and leukopenia.

The most commonly reported serious adverse reactions were pneumonia, sepsis thrombocytopenia, acute kidney injury, and anaemia.

Description of selected adverse reactions
Infections: Infection was the most common non-haematological toxicity.
In patients who received selinexor in combination with bortezomib and dexamethasone, upper respiratory tract infection and pneumonia were the most commonly reported infections in 21% and 15% of patients, respectively.

In patients who received selinexor in combination with dexamethasone, upper respiratory tract infection and pneumonia were the most commonly reported infections (in 15% and 13% of patients, respectively) with 25% of reported infections being serious and fatal infections occurring in 3% of treated patients.

Elderly population
Patients 75 years and older had a higher incidence of discontinuation due to an adverse reaction, higher incidence of serious adverse reactions, and higher incidence of fatal adverse reactions.

Reporting of suspected adverse reactions
Reporting of suspected adverse reactions after authorisation of the medicinal product is important. It allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system listed in Appendix V.

Sumitomo Pharma Oncology, Inc. Presents Updated Preliminary Data from Phase 1/2 Clinical Study Evaluating Investigational Agent TP-3654 in Patients with Myelofibrosis at European Hematology Association 2023 Hybrid Congress

On June 9, 2023 Sumitomo Pharma Oncology, Inc., a clinical-stage company focused on novel cancer therapeutics, reported updated preliminary data from the ongoing Phase 1/2 study evaluating TP-3654, an investigational selective oral PIM1 kinase inhibitor, in patients with myelofibrosis (MF) previously treated with or ineligible for JAK inhibitor therapy (Press release, Sumitomo Pharmaceuticals, JUN 9, 2023, View Source [SID1234632620]). Initial preliminary data was presented at American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition 2022. Updated results were presented in a poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2023 Hybrid Congress, being held June 8-11, 2023 in Frankfurt, Germany as well as virtually on the Congress platform from June 8-11, 2023.

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Preliminary data of TP-3654 monotherapy in relapsed/refractory myelofibrosis (MF) patients showed spleen volume reduction (SVR), and total symptom score (TSS) improvement.1 Further, TP-3654 may prompt early cytokine changes that may correlate with symptoms response.

"We are encouraged by these preliminary clinical data and are pleased that in dose ranges evaluated to date, TP-3654 has been well-tolerated with no myelosuppressive treatment-related adverse events (TRAEs). Our commitment remains to continue to advance this program with additional clinical sites and contributing to the progress of potential treatment options, which may improve outcomes for patients with myelofibrosis," said Patricia S. Andrews, Chief Executive Officer and Global Head of Oncology, Sumitomo Pharma Oncology, Inc.

As of February 9, 2023, 15 patients were enrolled across 5 dose levels from 480mg QD to 720 mg BID. The results showed SVR observed in 7 of 10 evaluable patients treated for ≥ 12 weeks. TSS improvements were observed in 9 of 10 evaluable patients. Broad reductions in cytokines were observed after TP-3654 treatment. At week 12 analysis, patients with higher cytokine reductions correlated with higher TSS improvement. BM fibrosis reduction from grade 3 to 2 was seen in one patient who also achieved spleen and symptoms responses and showed reductions in MF associated cytokines: IL6 (68%), IL12p40 (83%), MMP9 (56%), and EN-RAGE (68%), and is on active treatment for more than 18 months.1 Overall, TP-3654 appears to be well tolerated with no dose limiting toxicity (DLT) observed to date. The most common adverse events are Grade 1 and 2 diarrhea, nausea, and vomiting.1

"These updated preliminary data of oral TP-3654 as a monotherapy in patients with MF presented at EHA (Free EHA Whitepaper) 2023 are encouraging as we evaluate the pharmacodynamic markers changes in MF patients treated with TP-3654," said Jatin J. Shah, M.D., Chief Medical Officer and Global Head of Development, Sumitomo Pharma Oncology (SMP Oncology). "We look forward to continuing to advance this study to evaluate the potential role of TP-3654 as a monotherapy, in addition to exploring combination opportunities with JAK inhibitors, for patients with myelofibrosis." Below are the details for the SMP Oncology presentation:

Abstract Title

Details

Presenter

Preliminary Data From the Phase 1/2 Study of TP-3654, an Investigational Selective PIM1 Kinase
Inhibitor, Showed Cytokine Reduction and Clinical Responses in Relapsed/Refractory Myelofibrosis

Abstract P1031

June 9, 2023 at 6:00 PM CEST

Poster Presentation

Firas El Chaer, MD, Division of Hematology/Oncology,
University of Virginia Health System,
Charlottesville, VA

About TP-3654

TP-3654 is an oral investigational inhibitor of PIM 1 kinase, which has shown potential antitumor and anti-fibrotic activity through multiple pathways, including induction of apoptosis in preclinical models.2,3 TP-3654 was observed to inhibit proliferation and increased apoptosis in murine and human hematopoietic cells expressing clinically relevant JAK2V617F mutation.3 TP-3654 alone and in combination with ruxolitinib also showed normalized WBC and neutrophil counts, and reduced spleen size and bone marrow fibrosis in JAK2V617F and MPLW515L murine models of myelofibrosis.3 TP-3654 is currently being evaluated in a Phase 1/2 study of oral TP-3654 in patients with intermediate and high-risk myelofibrosis (NCT04176198). The Food and Drug Administration (FDA) granted Orphan Drug Designation for TP-3654 for the indication of myelofibrosis in May 2022.

About Myelofibrosis

Myelofibrosis is rare hematological malignancy with debilitating symptoms, splenic enlargement, cytopenia, and bone marrow fibrosis. The expression of PIM 1, which is known to contribute to cancer activation, is significantly elevated in hematopoietic cells from patients with myelofibrosis. It has been suggested to be a therapeutic target for myelofibrosis.

AFFIMED ANNOUNCES ACCEPTANCE OF ABSTRACTS AT THE 17TH INTERNATIONAL CONFERENCE ON MALIGNANT LYMPHOMA (17-ICML)

On June 9, 2023 Affimed N.V. (Nasdaq: AFMD) ("Affimed", or the "Company"), a clinical-stage immuno-oncology company committed to giving patients back their innate ability to fight cancer, reported that two abstracts have been accepted for presentation at the 17th International Conference on Malignant Lymphoma (17-ICML) taking place in Lugano, Switzerland on June 13-17, 2023 (Press release, Affimed, JUN 9, 2023, View Source [SID1234632618]). A poster presentation will share a preclinical data set of Affimed’s innate cell engager (ICE) AFM13 in combination with Artiva Biotherapeutics Inc.’s ("Artiva") off-the-shelf NK cell AB-101. An additional encore oral presentation will show the final results of the phase 2 REDIRECT study with AFM13 monotherapy in a r/r PTCL patient population.

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The preclinical data set shows that AFM13 binds homogenously to thawed AB-101, directs the NK cells to CD30-positive tumor cells and enhances the cytotoxic activity of AB-101 against the tumor cells. Associated with the AFM13-induced cytotoxic activity was an increased functional activation status of AB-101 demonstrated through degranulation and IFN-γ production. Importantly, in a mouse xenograft model, adoptive transfer of AB-101 in combination with AFM13 conferred tumor growth control.

The data of AFM13 in combination with the allogeneic, cryopreserved, off-the-shelf, cord blood-derived AB-101 NK cells demonstrate synergistic anti-tumor activity in vivo. Building on the unprecedented efficacy results of the phase 1 study with AFM13 in combination with fresh cord blood-derived NK cells (NCT04074746), as reported at ASH (Free ASH Whitepaper) 2022, the Company recently received Food and Drug Administration (FDA) IND clearance and is expecting to initiate a phase 2 study, LuminICE-203, with AFM13 and AB-101 in patients with r/r classical Hodgkin lymphoma in Q3 2023. The study will also include a cohort of 20 PTCL patients.

"These preclinical data of AFM13 + AB-101 as well as the clinical data from the phase 1 combination study demonstrate that AFM13 plus NK cells can achieve remarkable cytotoxicity against CD30-positive cancers," said Dr. Arndt Schottelius, Chief Scientific Officer at Affimed. "As a next step we want to bring this therapeutic approach to more patients in need and we look forward to initiate the LuminICE-203 study."

Details of the AFM13 + AB-101 poster presentation are as follows:

Title: AFM13 enhances the anti-tumor activity of AB-101 towards CD30+ tumors, conferring tumor growth control in vivo
Presenting Author: Jens Pahl
Poster Presentation Time: Thursday, 15 June 2023, 12:30 – 13:00 CET
Poster and Abstract Book Code: 419

In addition, an encore presentation of the final results of the phase 2 REDIRECT study will be given by Dr. Won Seog Kim, Professor of Hematology-Oncology at Samsung Medical Center in Seoul and a principal investigator of the study. AFM13 monotherapy exhibited clinical efficacy in a heavily pre-treated CD30-positive r/r PTCL population and a well-managed safety profile.

Details of the REDIRECT oral presentation are as follows:

Title: AFM13 in patients with CD30-positive relapsed or refractory (R/R) peripheral T cell lymphoma (PTCL): Results from the Phase 2 REDIRECT study
Presenting Author: Won Seog Kim
Session: Focus on… Session: T-Cell Lymphomas
Presentation Time: Thursday, 15 June 2023, 17:00 – 18:00 CET
Poster and Abstract Book Code: 126

More details about the 17-ICML conference are available online at View Source

About AFM13
AFM13 is a first-in-class tetravalent bispecific innate cell engager (ICE) that uniquely activates the innate immune system to destroy CD30-positive hematologic tumors. AFM13 induces specific and selective killing of CD30-positive tumor cells, leveraging the power of the innate immune system by engaging and activating natural killer (NK) cells and macrophages. AFM13 is Affimed’s most advanced ICE clinical program and was evaluated as monotherapy in a phase 2 trial in patients with relapsed/refractory peripheral T-cell lymphoma (REDIRECT, NCT04101331). In addition, The University of Texas MD Anderson Cancer Center is studying AFM13 in an investigator-sponsored Phase 1 trial in combination with cord blood-derived allogeneic NK cells in patients with recurrent or refractory CD30-positive lymphomas (NCT04074746). The company reported data from this study at ASH (Free ASH Whitepaper) 2022 annual meeting. To find out more about AFM13 and the studies, please visit: www.affimed.com.

About AB-101
AB-101 is Artiva’s non-genetically modified, cord blood-derived, allogeneic, cryopreserved, ADCC-enhancing NK cell therapy candidate for use in combination with monoclonal antibodies or innate-cell engagers in the out-patient setting. Artiva selects cord blood units with the high affinity variant of the receptor CD16 and a KIR-B haplotype for enhanced product activity. Using Artiva’s AlloNK platform, Artiva can generate thousands of doses of pure, cryopreserved, infusion-ready NK cells from a single umbilical cord blood unit while retaining high and consistent expression of CD16 and other tumor-engaging receptors, without the need for engineering. Artiva is conducting a Phase 1/2 multicenter clinical trial (ClinicalTrials.gov Identifier: NCT04673617) to assess the safety and clinical activity of AB-101 alone and in combination with the anti-CD20 monoclonal antibody, rituximab, in patients with relapsed or refractory B-cell-non-Hodgkin lymphoma (B-NHL) who have progressed beyond two or more prior lines of therapy. This study is progressing at multiple clinical sites across the U.S., and AB-101 is administered weekly in the out-patient setting over one-month cycles and with up to four cycles to assess therapeutic efficacy and durability. Artiva presented data from the first-in-human phase 1/2 clinical trial of AB-101 in combination with rituximab in R/R non-Hodgkin lymphoma at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting.

Wugen Presents Initial Data from First-in-Human Phase 1/2 Trial of WU-CART-007 at the European Hematology Association (EHA) 2023 Congress

On June 9, 2023 Wugen, Inc., a clinical-stage biotechnology company developing a pipeline of allogeneic cell therapies to treat a broad range of hematological and solid tumor malignancies, reported data for its two lead programs, WU-CART-007 and WU-NK-101, in separate presentations at the European Hematology Association (EHA) (Free EHA Whitepaper) 2023 Congress, taking place from June 8 – 11, 2023 in Frankfurt, Germany (Press release, Wugen, JUN 9, 2023, View Source [SID1234632617]).

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"We are very encouraged by these initial safety and efficacy data from our ongoing Phase 1/2 trial of WU-CART-007 in patients with R/R T-ALL/LBL," said Jan Davidson-Moncada, M.D., Ph.D., Chief Medical Officer of Wugen. "T-ALL/LBL are very challenging hematologic cancers. Many children and adults relapse after first line therapy and are left with very limited treatment options thereafter, often leading to high mortality. We are hopeful today’s data can be an early step to advance our goal of harnessing the power of innovative CAR-T cell therapies to address this stark unmet need."

In a presentation titled "Phase 1/2 Dose-Escalation/Dose-Expansion Study of Anti-CD7 Allogeneic CAR-T Cell in Relapsed or Refractory (R/R) T-Cell Acute Lymphoblastic Leukemia/ Lymphoblastic Lymphoma (T-ALL/LBL)," Wugen highlighted:

As of the January 30, 2023, data cut-off, 12 patients had been enrolled in the dose escalation phase of the study (Dose Levels (DL) 1-3), all of whom received WU-CART-007. Prior to enrollment, all patients had been heavily pretreated, with a median of 5 prior lines of therapy. Disease assessment at baseline (BL) showed extensive disease with high disease burden, median bone marrow (BM) blast count of 61.5% (range 43-85%), and extramedullary disease (EMD) in 67% (8/12) of patients. Nine patients had R/R T-ALL and three patients had R/R T-LBL.
Overall, WU-CART-007 was well-tolerated across all dose levels.
No cases of Graft versus Host Disease (GvHD), prolonged T-cell aplasia, or pancytopenia in the absence of disease were reported. One unrelated dose-limited toxicity (DLT) of encephalopathy, secondary to an intracranial bleed associated with a Rhizomucor infection, was reported.
All reports of cytokine release syndrome (CRS) were low-grade, Grade 1 or 2, (86%, 7/8) except for one Grade 3 report which resolved within 72 hours after treatment with tocilizumab, dexamethasone, and low dose vasopressors.
One instance of Grade 1 immune effector cell-associated neurotoxicity syndrome (ICANS), which resolved spontaneously, was reported in a patient at DL3.
The Objective Response Rate (ORR) in efficacy evaluable patients was 57% overall (4/7), including 2 Complete Responses (CR), 1 morphological leukemia-free state (MLFS), and 1 Partial Response (PR). At the time of the data cut, with a median follow-up of 107 days, the duration of response extending to 86 days had been reported.
In a presentation titled "Adoptively Infused Memory-Like (ML) Natural Killer (NK) Cells Elicit Adaptive Immune Responses in Patients with Acute Myeloid Leukemia (AML)," Wugen highlighted:

The infusion of ML-NK cells was associated with modulation of the AML tumor microenvironment and engagement of the endogenous adaptive immune response.
Post-infusion of ML-NK cells, high T-cell infiltration to the tumor site signaled the recruitment of the adaptive immune system and the potential for durable effectiveness in patients with R/R AML.
Additional meeting information can be found at View Source

About WU-CART-007

WU-CART-007 is an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR-T cell therapy engineered to overcome the technological challenges of harnessing CAR-T cells to treat CD7+ hematological malignancies. Wugen is deploying CRISPR/Cas9 gene editing technology to delete CD7 and the T-cell receptor alpha constant (TRAC), preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host-disease (GvHD). WU-CART-007 is manufactured using healthy donor-derived T-cells to eliminate the risk of malignant cell contamination historically observed in the autologous CAR-T setting. WU-CART-007 is currently being evaluated in a global Phase 1/2 clinical trial for the treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL). Additional information is available on clinicaltrials.gov, identifier NCT# 04984356. WU-CART-007 has received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration for the treatment of R/R T-ALL/LBL.

About WU-NK-101

WU-NK-101 is a novel immunotherapy harnessing the power of memory natural killer (NK) cells to treat liquid and solid tumors. Memory NK cells are hyper-functional, long-lasting immune cells that exhibit enhanced anti-tumor activity and a cytokine-induced memory-like (CIML) phenotype. This rare cell population has a superior phenotype, proliferation capacity, and metabolic fitness that makes it better suited for cancer therapy than other NK cell therapies. Wugen is applying its proprietary MonetaTM platform to advance WU-NK-101 as a commercially scalable, off-the-shelf cell therapy for cancer. WU-NK-101 is currently in development for acute myelogenous leukemia (AML) and solid tumors.

Notable Labs Presents Data Demonstrating PPMP’s Potential to Identify Novel Drug Combinations in JMML at the 2023 EHA Hybrid Congress

On June 9, 2023 Notable Labs, Inc. ("Notable"), a clinical stage therapeutic platform company developing predictive precision medicines for cancer patients, reported data from an ex vivo study demonstrating the potential for its Predictive Precision Medicine Platform (PPMP) to identify more active investigational pre-hematopoietic stem cell therapy (pre-HCST) regimens for the treatment of juvenile myelomonocytic leukemia (JMML) at the European Hematology Association (EHA) (Free EHA Whitepaper) Hybrid Congress being held in Frankfurt, Germany on June 8 – 15, 2023 (Press release, Notable Labs, JUN 9, 2023, View Sourceppmp-jmml-2023-eha-hybrid/" target="_blank" title="View Sourceppmp-jmml-2023-eha-hybrid/" rel="nofollow">View Source [SID1234632615]).

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"In collaboration with leading experts at University of California San Francisco, Benioff Children’s Hospital, we screened more than 100 prospective drug combinations on one of the largest collections of primary JMML samples and identified potentially more potent drug combinations for the treatment of JMML than currently used regimens," said Thomas Bock, M.D., Chief Executive Officer of Notable. "Specifically, this study makes a compelling case for the addition of tretinoin (TRET) to the conventional, but often suboptimal, anti-leukemic combination of fludarabine (FLU) with cytarabine (ARA-C), or 5-azacitidine (5-AZA) alone, and similarly for the addition of calcitriol (CALC) to 5-AZA. Overall, this data provides strong reason to further investigate these potentially more effective drug combinations as there remains a persistent need to develop better pre-HSCT regimens in JMML."

The study aimed to identify novel anti-leukemic drug combinations potentially more effective than currently used regimens in JMML pre-HSCT by employing Notable’s PPMP. Of 130 drug combinations, 27 were shown to be more active in killing leukemic cells ex vivo than compounds represented in these combinations on their own. In particular, TRET, a differentiating agent, enhanced the anti-leukemic effects of FLU + ARA-C in 90% (19/21) of patient samples and of 5-AZA in 76% (19/25) of patient samples. Similarly, CALC, another differentiating agent, enhanced the anti-leukemic effect of 5-AZA in 84% (21/25) of patient samples.

These results highlight PPMP as a potential tool for identifying superior drug combinations for patients with high medical needs, including with JMML.

Abstract title: Tretinoin and calcitriol enhance treatment regimens for juvenile myelomonocytic leukemia in ex vivo drug sensitivity assays.
Presenting Author: Associate Prof. Elliot Stieglitz
Abstract Code: P998
Session Type/Title: Poster Presentation
Date and Time: June 9, 18:00 – 19:00 CEST

Additional meeting information can be found on the 2023 EHA (Free EHA Whitepaper) Congress website, View Source The poster will be available on the Company’s website at View Source shortly after the event.