On May 17, 2023 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported clinical data on its Phase 1 AMELI-01 clinical trial (evaluating UCART123) that were unveiled in an oral presentation at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting, as well as preclinical data on multiplex engineering for superior generation of CAR T-cells, at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2023 Annual Meeting (Press release, Cellectis, MAY 17, 2023, View Source [SID1234631803]).

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Oral presentation:

AMELI-01, a study evaluating UCART123, an allogeneic CAR T-cell product candidate, in relapsed/refractory acute myeloid leukemia (r/r AML)

The oral presentation highlights the following clinical data:

Preliminary Clinical Data from the AMELI-01 Study Presented at ASH (Free ASH Whitepaper) 2022

AMELI-01 is a Phase 1 open-label dose-escalation trial evaluating the safety, tolerability, expansion and preliminary activity of UCART123 given at escalating dose levels after lymphodepletion (LD) with either fludarabine and cyclophosphamide (FC) or FC with alemtuzumab (FCA) in patients with relapsed or refractory acute myeloid leukemia (r/r AML).

The oral presentation reviewed preliminary data from patients who received UCART123 at one of the following dose levels: dose level 1 (DL1) 2.5×105 cells/kg; dose level 2 (DL2) 6.25×105 cells/kg; intermediate dose level 2 (DL2i) 1.5×106 cells/kg; or dose level 3 (DL3) 3.30×106 cells/kg after lymphodepletion with FC ([n=8], DL1 – DL3) or FCA ([n=9], DL2 & DL2i).

Preliminary Safety Data

The FCA LD regimen resulted in robust lymphodepletion for greater than 28 days in all patients. Seven out of nine patients demonstrated UCART123 expansion, with maximum concentration (Cmax) ranging from 13,177 to 330,530 copies/µg DNA, an almost nine-fold increase compared with FC LD, and a significant increase in area under the curve (AUC) (0-28 days) (p=0.04; FC 10.2 vs. FCA 34.9).

Cytokine release syndrome (CRS) occurred in eight patients in the FC arm and nine patients in the FCA arm. In the FC arm, one patient experienced Grade 3 immune effector cell-associated neurotoxicity syndrome (ICANS) and two patients experienced Grade 4 protocol-defined dose limiting toxicities (DLTs) secondary to CRS. In the FCA arm, two patients experienced Grade 5 DLTs secondary to CRS.

Preliminary Efficacy Data

Evidence of UCART123 anti-tumor activity was observed in four patients out of fifteen at DL2 or above with best overall responses in the FCA arm. Two out of eight patients (25%) at DL2 in the FCA arm achieved meaningful response:

A patient who failed five prior lines of therapy experienced a durable minimal residual disease (MRD) negative complete response (CR) with full count recovery at Day 56 that continues beyond one year.
A patient with stable disease achieved greater than 90% bone marrow blast reduction (60% to 5%) at Day 28.
The preliminary data show that adding alemtuzumab to the FC LD regimen was associated with sustained lymphodepletion and significantly higher UCART123 cell expansion, which correlated with improved anti-tumor activity.

Patient Enrollment in a 2-Dose Regimen Arm

Overall, these preliminary data support the continued administration of UCART123 after FCA lymphodepletion in patients with r/r AML. Based on observed UCART123 expansion patterns and cytokine profiles, pursuant to an amended protocol, a second dose of UCART123 is given after 10-14 days to allow for additional UCART123 expansion and clinical activity without the use of additional lymphodepletion. The UCART123 cell expansion from the second dose of UCART123, in the setting of reduced disease burden, is expected to be safe and allow for clearance of residual disease.

"These clinically meaningful preliminary data from the AMELI-01 study are very encouraging for patients and for the future of allogeneic CART-cell therapy. AML is a disease with an urgent need for alternative treatment options for patients, and we are excited to be moving the study forward," said Dr. Mark Frattini, M.D., Ph.D., Chief Medical Officer at Cellectis. "We have now implemented a two-dose regimen arm for our AMELI-01 trial and we look forward to sharing future updates as they become available."

Title: AMELI-01: A Phase I Trial of UCART123v1.2, an Anti-CD123 Allogeneic CAR-T Cell Product, in Adult Patients with Relapsed or Refractory (R/R) CD123+ Acute Myeloid Leukemia (AML)

Presenter: Daniel Lee, M.D., Director, Clinical Sciences at Cellectis

Session Date/Time: 5/17/2023 – 3:45 PM – 5:30PM PDT
Session Title: CAR Engineering and Production Advances for Targeting Hematologic and Solid Tumor Malignancies
Session Room: 502 AB

Final Abstract Number: 94

A copy of the ASGCT (Free ASGCT Whitepaper) oral presentation will be available on Cellectis’ website after the presentation: View Source

Poster Presentation:

Expanding the scope of multiplex engineering for superior generation of efficient CAR T-cells

In recent years, advances in genomic-based cellular engineering are bringing us a step closer to conquering solid tumors. This glimpse of success also demonstrated that we need to be able to creatively customize and equip CAR T-cells to target these tumors.

In this presentation, Cellectis shows that we can use the state-of-the-art TALEN technology to precisely edit up to four loci simultaneously while delivering several additional payloads to increase the efficacy and persistence of CAR T-cells.

The preclinical data demonstrate that multiplexed engineering does not compromise CAR T-cell function, which can even be enhanced and display improved anti-tumor activity. Thus, multiplexed engineering at superior efficiency rates while preserving genomic integrity has the potential to generate highly functional CAR T-cells to advance in the fight against solid tumors.

Cellectis takes it a step further and uses a curated combination of genome engineering technologies including TALE base editors (TALE-BE) to increase the efficiency of multiplexed gene editing while protecting genomic integrity.

"The immunosuppressive barriers of the tumor microenvironment antagonize CAR T-cells and have limited our ability to target solid tumors. These preclinical data show that we can precisely select and combine an array of gene and cell engineering approaches to produce armored CAR T-cells with high efficiency rates. With this strategy, we can focus on unmeet clinical needs and equip CAR T-cells with enhanced activity to help us in our quest to defeat solid tumors," said Beatriz Aranda Orgilles, Ph.D., Team Leader at Cellectis.

The poster presentation at ASGCT (Free ASGCT Whitepaper) highlights the following preclinical data:

Optimization of delivery timings and selection of compatible TALEN pairs provides high editing efficiency while attenuating potential TALEN crosstalk.

TALEN and TALE-BE technologies can be integrated in the generation of CAR T-cells to provide high gene editing rates while preserving genomic safety.

CAR T-cells can be engineered to carry multiple edits and simultaneously exhibit several key features to combat solid tumors: immuno-evasive properties, secretion of the pro-inflammatory cytokine IL-12, resistance to the immunosuppressive pathways PD-1 and TGFB1.

Multi-equipped CAR T-cells can efficiently target in vivo and in vitro models of triple negative breast cancer, an aggressive tumor that to date has limited therapeutic possibilities.
Title: Expanding the Scope of Multiplex Engineering for Superior Generation of Efficient CAR T-cells

Presenter: Beatriz Aranda Orgilles, Ph.D., Team Leader at Cellectis

Session Date/Time: 5/17/2023 12:00 PM PDT
Session Title: Wednesday Poster Session
Poster Board Number: 604

Final Abstract Number: 604

Poster of the presentation will be available on Cellectis’ website after the presentation: View Source

On May 17, 2023 Almac Diagnostic Services, a member of the Almac Group, reported significant updates to claraT, its unique software-driven solution for gene expression data analysis, which gives cancer researchers access to the most comprehensive and robust biomarker discovery support available (Press release, Almac, MAY 17, 2023, View Source [SID1234631802]).

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Almac’s claraT, which has supported over 60 oncology studies to date for multiple Biopharma and Academic clients, classifies the most biologically relevant gene expression signatures into a comprehensive easy-to-interpret report that covers all 10 Hallmarks of Cancer.

The updates include 16 new gene expression signatures across the Hallmarks of Cancer, improved functionality with online data access and simplified reporting through an interactive dashboard. Clients receive a unique, interactive report that allows the easy visualisation of the key discriminating biologies within either a large cohort or an individual tumour sample.

Toshimitsu Uenaka, President EPAT (Epochal Precision Anti-Cancer Therapeutics), at Eisai Inc., whose translational team have used Almac claraTstated: "We really found the claraT analysis of our studies to be very informative and a useful tool for our future drug discovery."

Adding these new gene expression signatures ensures that claraT remains current in the market with the most up to date content available. Almac claraT offers a unique solution in the market for biomarker discovery from either RNASeq data generated from sample profiling within Almac labs or from gene expression data sets generated elsewhere that require further bioinformatics analysis.

Professor Richard Kennedy, Global VP and Medical Director, Almac Diagnostic Services said: "Being able to equip leading Biopharma and academic cancer researchers globally with a powerful gene expression analysis platform has supported the identification of exciting new data.

"Conventionally gene expression signatures are applied individually to classify patients into molecular subgroups. Signatures designed to identify the same biology are often developed independently using different bioinformatics algorithms and therefore may not classify identically. To address this, we developed the claraT software solution which uses consensus between multiple published signatures, categorised into the Hallmarks of Cancer, to classify cancers in a more robust manner. Importantly, this standardised bioinformatics approach enables the analysis of datasets in a highly reproducible and cost-effective offering.

"Our proprietary claraT report has demonstrated its ability to accelerate mRNA-based research, enabling deeper insights into translational cohorts which leads to better drug development and trial stratification, potentially contributing to better patient outcomes in the future."

Almac Diagnostic Services is hosting a GenomeWeb Webinar on Tuesday 13th June alongside leading cancer researchers from the University of Kansas, USA. The webinar will focus on how claraT was utilised effectively within breast cancer studies.

The LIVE webinar will be held at 11am EDT / 4pm BST and interested parties can sign up here.

On May 17, 2023 2seventy bio, Inc. (Nasdaq: TSVT), a leading immuno-oncology cell therapy company, reported new data featuring novel approaches combining the company’s platform CAR T cell and T cell receptor technology and unique cell therapy engineering capabilities to potentially enhance treatment potency in a range of cancers (Press release, 2seventy bio, MAY 17, 2023, View Source [SID1234631801]). The data were shared in three presentations at this year’s American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting in Los Angeles, California.

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"We’re excited to present new data related to our programs in acute myeloid leukemia, non-Hodgkin lymphoma and solid tumors," said Philip Gregory, D.Phil., chief scientific officer, 2seventy bio. "These presentations provide further evidence of our focus on generating potent and targeted anti-tumor activity while limiting toxicity through innovative approaches to controlling T cell responses. With this approach, we believe we can augment the tremendous potential of cell-based immunotherapy, with the goal of advancing next-generation treatments targeting a broad range of cancer types."

bbT369, a dual-targeted and CBLB gene edited autologous CAR T product for non-Hodgkin lymphoma, showed edit-driven enhanced activity in preclinical in vitro and in vivo models

bbT369 is 2seventy’s novel investigational CD79a/CD20 dual-targeting CBLB gene edited CAR T cell therapy for the treatment of patients with relapsed and/or refractory B-cell non-Hodgkin lymphoma (B-NHL). This preclinical study evaluated the role of the CBLB edit across multiple aspects of CAR T cell function compared to unedited controls. In the study, in vitro assays were designed to model known challenges to maximal CAR T cell activity within the tumor, such as chronic activation, antigen downregulation, and immunosuppression.

The data demonstrated that the CBLB gene edit significantly enhanced the activity of the bbT369 CAR T cells across each of the in vitro model systems tested.
bbT369 T cells also drove greater T cell expansion and durable tumor control compared with unedited controls in vivo using a xenograft NSG mouse model.
RESET, a novel TCR coupled antigen receptor architecture, showed targeting sensitivity and pharmacologically- controlled anti-tumor activity in models of acute myeloid leukemia

For some tumors, available target antigens lack ideal expression, necessitating improvements in targeting sensitivity, potency, control, and/or exhaustion mitigation to achieve robust anti-tumor efficacy. While classical CAR T cells require high levels of target antigen for maximum activity, the endogenous T cell receptor (TCR) is significantly more sensitive to low target antigen abundance. To address this gap, a novel receptor architecture called RESET (rapamycin-enabled, switchable endogenous TCR) was developed to more closely mimic TCR activation, thus potentially improving T cell killing of low antigen expression tumor cells. RESET combines the antibody-based targeting of CARs, the drug-regulated activity of the DARIC (a rapamycin-regulated CAR) technology and the natural signaling proficiency and inherent sensitivity of TCRs. The RESET receptor comprises two functional units that are designed to enable toggling of RESET T cells between inert and activated states through rapamycin-induced dimerization:

When challenged with low antigen density tumor cells in vitro, CD33-targeted RESET T cells (RESET33) were more potently reactive to CD33+ tumor cells after rapamycin dimerization, secreting approximately three times more cytokine than CD33-targeted regulated CAR T cells.
This enhancement was also observed in preclinical NSG mouse models in vivo, where RESET33 T cells more deeply controlled systemic CD33+ AML tumor models, providing a significant survival benefit in comparison to DARIC33 T cells.
A novel TGFβ switch receptor drove robust MAGE-A4 TCR anti-tumor activity with a favorable safety profile

Solid tumors frequently secrete TGFβ, a pleiotropic cytokine that suppresses T cell responses. To counteract the immunosuppressive effects of TGFβ within engineered T cells, a synthetic receptor (CTBR12) was developed to leverage an immunosuppressive cytokine to convert TGFβ binding into pro-inflammatory signaling events that enhance anti-tumor function:

T cells co-expressing a pairing enhanced MAGE-A4 TCR with the CTBR12 switch receptor demonstrated robust antigen-dependent responses that were significantly enhanced upon exposure to TGFβ, whereas MAGE-A4 TCR controls lacking CTBR12 were suppressed by this anti-inflammatory cytokine.
CTBR12 co-expression did not alter the safety profile of the MAGE-A4 TCR.
In two murine xenograft tumor models with abundant TGFβ, T cells co-expressing the MAGE-A4 TCR and CTBR12 both controlled tumors at T cell doses that elicited only minimal responses without CTBR12 co-expression and exhibited superior durability of response overall.
About SC-DARIC33

SC-DARIC33 is an investigational CD33-specific cell therapy that utilizes 2seventy bio’s proprietary Dimerizing Agent Regulated Immunoreceptor Complex (DARIC) T cell platform. SC-DARIC33 is designed as a regulatable, potentially first-in-class autologous T cell therapy and is now being studied at Seattle Children’s in a Phase 1 trial, PLAT-08 (NCT05105152), as a first-in-human investigation of the DARIC T cell platform in relapsed/refractory pediatric and young adult AML.

DARIC separates the antigen binding and signaling functions of a CAR, with the intent that these two components are brought together by the small molecule rapamycin, resulting in a functional CAR construct. In preclinical studies, SC-DARIC33 has shown robust drug-dependent anti-tumor activity (similar to CD19 CAR T controls). Importantly, SC-DARIC33 has been shown to be activated by low non-immunosuppressive concentrations of rapamycin in the blood and, when rapamycin is removed, DARIC returns to an inactive state. SC-DARIC33 tests the hypothesis that a pharmacologically regulated CAR can enable potent AML targeting while limiting toxicities associated with normal myeloid and myeloid progenitor cell targeting.

The investigation of SC-DARIC33 in the Phase 1 PLAT-08 study of pediatric and young adult AML patients and the scientific translation of these data are intended to establish the safety profile of SC-DARIC33 and evaluate feasibility of the reversable modulation (OFF-ON-OFF) of SC-DARIC33.

SC-DARIC33 is not approved for any indication in any geography.

About bbT369

bbT369 is an investigational dual-targeting CAR T cell therapy with a gene edit being evaluated for the treatment of patients with relapsed and/or refractory B-NHL currently being evaluated in a first-in-human Phase 1/2 trial (CRC-403 NCT05169489). bbT369 was purposely designed with three layers of innovation to address the potential mechanisms of anti-CD19 CAR T cell therapy failure, including dual targeting of CD79a/CD20, which is a novel combination of antigens highly expressed in B cell lymphomas, a split co-stimulation signaling technology intended to drive more robust T cell activation, and a gene edit to remove the function of CBLB, a known negative regulator of T cells.

In the 3L+ relapsed and/or refractory B-NHL setting, 60-70% of patients treated with commercially available CAR T cell therapies do not achieve a long-term remission, highlighting a significant unmet clinical need.

In December 2021, the FDA cleared the Investigational New Drug (IND) application for bbT369.

The clinical development program for bbT369 includes the Phase 1/2 CRC-403 study (NCT05169489). Safety and potential efficacy of bbT369 in patients with specific subtypes of relapsed and/or refractory B-NHL will be assessed, including patients who relapsed after CD19 CAR T cell therapy as well as patients who are CAR-naïve.

bbT369 is not approved for any indication in any geography.

About the MAGE-A4 Program

MAGE-A4 is a member of the MAGE family of cancer-testis antigens expressed in a number of solid tumor types. The program employs a highly potent TCR discovered in our MediGene collaboration that recognizes HLA-presented MAGE-A4 peptides and further enhances the potency of these re-directed T cells using our CTBR12 TGFβ "flip" receptor technology — which converts the immunosuppressive effects of TGFβ into an activation signal for the T cells. Regeneron and 2seventy bio are co-developing the program under their collaboration entered into in 2018.

On May 16, 2023 Astrazeneca reported Positive high-level results from the FLAURA2 Phase III trial showed Tagrisso (osimertinib) in combination with chemotherapy demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared to Tagrisso alone for patients with locally advanced (Stage IIIB-IIIC) or metastatic (Stage IV) epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC) (Press release, AstraZeneca, MAY 17, 2023, View Source [SID1234631785]).

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Safety results and discontinuation rates due to adverse events were consistent with the established profiles of each medicine. At the time of this analysis, the overall survival (OS) data were immature and will be formally assessed at a subsequent analysis.

Each year, there are an estimated 2.2 million people diagnosed with lung cancer globally with 80-85% of patients diagnosed with NSCLC, the most common form of lung cancer.1-3 Approximately 70% of people are diagnosed with advanced NSCLC. Additionally, about 10-15% of NSCLC patients in the US and Europe, and 30-40% of patients in Asia have EGFRm NSCLC.4-6

Pasi A. Jänne, MD, PhD, medical oncologist at Dana-Farber Cancer Institute and principal investigator for the FLAURA2 trial, said: "As the global standard of care for EGFR-mutated non-small cell lung cancer, osimertinib monotherapy has transformed the treatment landscape allowing many patients the opportunity to achieve improved survival. FLAURA2 provides compelling evidence that the addition of chemotherapy to osimertinib can provide a new option for patients and clinicians that further improves outcomes compared to osimertinib alone and as such, can further delay treatment resistance and disease progression."

Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: "These significant FLAURA2 results show Tagrisso has the potential to offer patients in the first-line setting a new treatment option that can extend the time they live without their disease progressing. This meaningfully builds on successive trials which have demonstrated improved clinical benefit with Tagrisso in patients with EGFR-mutated lung cancer."​

The data will be presented at a forthcoming medical meeting and shared with global health authorities.

These results add to the extensive body of evidence for Tagrisso in EGFRm NSCLC, which has improved patient outcomes in both early-stage disease in the ADAURA Phase III trial and late-stage disease in the FLAURA Phase III trial. Tagrisso has also shown proven clinical activity in treating central nervous system (CNS) metastases across settings.

As part of AstraZeneca’s ongoing commitment to treating patients as early as possible in lung cancer, Tagrisso is also being investigated in unresectable NSCLC in the pivotal LAURA Phase III trial, with results expected later this year.

Notes

Lung cancer
Lung cancer is the leading cause of cancer death among both men and women, accounting for about one-fifth of all cancer deaths.1 Lung cancer is broadly split into NSCLC and small cell lung cancer.2 The majority of all NSCLC patients are diagnosed with advanced disease.7

Patients with EGFRm NSCLC are particularly sensitive to treatment with an EGFR-tyrosine kinase inhibitor (EGFR-TKI) which blocks the cell-signaling pathways that drive the growth of tumour cells.8

FLAURA2
FLAURA2 is a randomised, open-label, multi-centre, global Phase III trial in the 1st-line treatment of 586 patients with locally advanced (Stage IIIB-IIIC) or metastatic (Stage IV) EGFRm NSCLC. Patients were treated with Tagrisso 80mg once daily oral tablets in combination with chemotherapy (pemetrexed (500mg/m2) plus cisplatin (75mg/m2) or carboplatin (AUC5)) every three weeks for four cycles, followed by Tagrisso with pemetrexed maintenance every three weeks.

The trial was enrolled in more than 150 centres across more than 20 countries, including in the US, Europe, South America and Asia. This is the final analysis of the primary endpoint of PFS. The trial is ongoing and will continue to assess the secondary endpoint of OS.

Tagrisso
Tagrisso (osimertinib) is a third-generation, irreversible EGFR-TKI with proven clinical activity in NSCLC, including against CNS metastases. AstraZeneca continues to explore Tagrisso as a treatment for patients across multiple stages of EGFRm NSCLC.

Tagrisso is approved as monotherapy in more than 100 countries including in the US, EU, China and Japan. These include for 1st-line treatment of patients with locally advanced or metastatic EGFRm NSCLC, locally advanced or metastatic EGFR T790M mutation-positive NSCLC, and adjuvant treatment of early-stage (IB, II and IIIA) EGFRm NSCLC, where Tagrisso recently demonstrated a statistically significant and clinically meaningful OS benefit.

In addition to investigating Tagrisso and chemotherapy in late-stage disease (FLAURA2), AstraZeneca has several ongoing Phase III trials focused on earlier stages of lung cancer. Tagrisso is being tested in the neoadjuvant resectable setting (NeoADAURA), in the Stage IA2-IA3 adjuvant resectable setting (ADAURA2), and in the Stage III locally advanced unresectable setting (LAURA).

The Company is also researching ways to address tumour mechanisms of resistance through the SAVANNAH and ORCHARD Phase II trials, and the SAFFRON Phase III trial, which test Tagrisso given concomitantly with savolitinib, an oral, potent and highly selective MET TKI, as well as other potential new medicines.

On May 16, 2023 Tyligand Bioscience, a clinical stage biotech company focused on the development of innovative drugs to treat drug resistant tumors, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to TSN084, the company’s multi-kinase inhibitor for the treatment of Acute Myeloid Lymphoma (AML) (Press release, Tyligand Bioscience, MAY 16, 2023, View Source [SID1234644986]).

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The orphan drug designation process ("ODD") begins with an application to the US Food and Drug Administration ("FDA") Orphan Drug Designation office, and is accompanied by a comprehensive package that includes the basic science, analysis of addressable number of individuals with Acute Myeloid Lymphoma (AML) and description of the patients projected to be helped by a therapy.

Dr. Tony Zhang, cofounder and CEO of Tyligand Bioscience stated, "Orphan drug designation represents a major milestone for Tyligand in pursuit of accelerated marketing approval for TSN084, our front runner of innovative molecules designed to treat drug resistant malignancies."

The company developed TSN084 leveraging a novel Type II kinase binding mode that targets CDK8/19, FLT3, AXL & cMet etc., for the treatment of both solid tumors and hematological malignancies. Drugs intended to treat orphan diseases (rare diseases that affect less than 200,000 people in the US) are eligible to apply for ODD, which provides multiple benefits to the sponsor during development and after approval.

About Acute myeloid Leukemia :
Acute myeloid leukemia (AML) is the most common type of acute leukemia in adults. The average age at diagnosis is around 68 years—though it can also occur in adolescents and children. According to the National Cancer Institute, AML accounts for 1% of all cancer cases in the U.S., and an estimated 20,050 people in the U.S. were newly diagnosed with it in 2022. Despite currently available treatments for AML, the five-year overall survival rate for patients remains less than 30%. Novel therapies are needed to improve patient survival and quality of life.