Legend Biotech Showcases Updated and New Data from Comprehensive BCMA CAR-T, Cilta-Cel, Program and First Preclinical Results for Tri-specific CAR-T at 2021 ASH

On November 4, 2021 Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global, clinical-stage biotechnology company developing and manufacturing novel therapies, reported that 12 company-sponsored studies were accepted for presentation at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (Press release, Legend Biotech, NOV 4, 2021, View Source [SID1234594523]). These include two oral presentations and 10 poster presentations .

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Presentation highlights include updates from the CARTITUDE clinical development program for the investigational B-cell maturation antigen (BCMA) directed chimeric antigen receptor T cell (CAR-T) therapy, ciltacabtagene autoleucel (cilta-cel), for the treatment of patients with relapsed or refractory multiple myeloma (RRMM). Presentations will detail longer-term follow-up data and new sub-group analysis results from the Phase 1b/2 CARTITUDE-1 study as well as adjusted indirect comparison of CARTITUDE-1 patient outcomes relative to standard-of-care therapies in real-world clinical practice from the LocoMMotion study. First data release from Cohort B and longer-term follow-up data from Cohort A of the CARTITUDE-2 study in earlier lines of treatments will be presented.

Additionally, Legend will share the first preclinical in vivo data on its novel tri-specific single-domain antibody (VHH) CAR-T (LCAR-AIO). LCAR-AIO targets three antigens—CD19, CD20 and CD22—with the potential for development as a treatment for patients with relapsed B cell lymphoma and prior CD19 CAR-T therapies.

"The new and updated data from the CARTITUDE-1 and CARTITUDE-2 studies show that cilta-cel continues to provide early, deep and durable responses, even in high-risk patients," said Ying Huang, PhD, CEO and CFO of Legend Biotech. "What’s also encouraging is the new preclinical data from our novel tri-specific VHH CAR-T, which was designed and developed by Legend. This trispecific CAR-T exemplifies our team’s ability to discover novel mechanisms of action by screening and optimizing antibodies in house."

A select list of abstracts from the meeting can be found below.

ASH Presentations (December 11-14, 2021)

Abstract No.

Title

INFO

Abstract #549
Oral

Updated Results From CARTITUDE-1: Phase 1b/2 Study of Ciltacabtagene Autoleucel, a B-cell Maturation Antigen–Directed Chimeric Antigen Receptor T Cell Therapy, in Patients with Relapsed/Refractory Multiple Myeloma

Session Title: 704. Cellular Immunotherapies: Cellular Therapies for Myeloma

Date/Time: Sunday, December 12, 2021 4:30 PM – 6:00 PM EST

Presentation time: 5:00 PM EST

Room: Georgia World Congress Center, Hall C2-C3

Abstract #550

Oral

Ciltacabtagene Autoleucel for Triple-Class Exposed Multiple Myeloma: Adjusted Comparisons of CARTITUDE-1 Patient Outcomes Versus Therapies from Real-World Clinical Practice from the LocoMMotion Prospective Study

Session Title: 704. Cellular Immunotherapies: Cellular Therapies for Myeloma

Date/Time: Sunday, December 12, 2021 4:30 PM – 6:00 PM EST

Presentation time: 5:15 PM EST

Location: Georgia World Congress Center, Hall C2-C3

Abstract#3938

Poster

Efficacy and Safety of Ciltacabtagene Autoleucel in Patients with Relapsed/Refractory Multiple Myeloma: CARTITUDE-1 Subgroup Analysis

Session Title: 731. Autologous Transplantation: Clinical and Epidemiological: Poster III

Date/Time: Monday, December 13, 2021 6:00 PM – 8:00 PM EST

Location: Georgia World Congress Center, Hall B5

Abstract #2812

Poster

Anakinra Targeting Cytokine Release Syndrome Associated with Chimeric Antigen Receptor T-cell Therapies

Session Title: 704. Cellular Immunotherapies: Clinical: Poster II

Date/Time: Sunday, December 12, 2021 6:00 PM – 8:00 PM EST

Location: Georgia World Congress Center, Hall B5

Abstract #3866

Poster

Efficacy and Safety of Ciltacabtagene Autoleucel (Cilta-cel), a B-cell Maturation Antigen–Directed Chimeric Antigen Receptor T-cell Therapy, in Lenalidomide-Refractory Patients with Progressive Multiple Myeloma After 1–3 Prior Lines of Therapy: Updated

Results From CARTITUDE-2

Session Title: 704. Cellular Immunotherapies: Clinical: Poster III

Date/Time: Monday, December 13, 2021 6:00 PM – 8:00 PM EST

Location: Georgia World Congress Center, Hall B5

Abstract #2910

Poster

CARTITUDE-2: Efficacy and Safety of Ciltacabtagene Autoleucel (Cilta-cel), a B-cell Maturation Antigen (BCMA)-Directed Chimeric Antigen Receptor T Cell (CAR T) Therapy, in Patients with Multiple Myeloma and Early Relapse After Initial Therapy

Session Title: 731. Autologous Transplantation: Clinical and Epidemiological: Poster II

Date/Time: Sunday, December 12, 2021 6:00 PM – 8:00 PM EST

Location: Georgia World Congress Center, Hall B5

Abstract #1835

Poster

Bortezomib, Lenalidomide, and Dexamethasone (VRd) Followed by Ciltacabtagene Autoleucel vs VRd Followed by Lenalidomide and Dexamethasone (Rd) Maintenance in Patients with Newly Diagnosed Multiple Myeloma Not Intended for Transplant: A Randomized, Phase 3 Study (CARTITUDE-5)

Session Title: 731. Autologous Transplantation: Clinical and Epidemiological: Poster I

Date/Time: Saturday, December 11, 2021 5:30 PM – 7:30 PM EST

Location: Georgia World Congress Center, Hall B5

Abstract #3057

Poster

LocoMMotion: A Prospective, Non-interventional, Multinational Study of Real-life Current Standards of Care in Patients with Relapsed/Refractory Multiple Myeloma Who Received ≥3 Prior Lines of Therapy

Session Title: 905. Outcomes Research—Lymphoid Malignancies: Poster II

Date/Time: Sunday, December 12, 2021 6:00 PM – 8:00 PM EST

Location: Georgia World Congress Center, Hall B5

Abstract #1676

Poster

Meta-analysis of Ciltacabtagene Autoleucel versus Physician’s Choice in the Treatment of Patients with Relapsed or Refractory Multiple Myeloma

Session Title: 653. Myeloma and Plasma Cell Dyscrasias: Clinical-Prospective Therapeutic Trials: Poster I

Date/Time: Saturday, December 11, 2021 5:30 PM – 7:30 PM

Location: Georgia World Congress Center, Hall B5

Abstract #4075

Poster

Real-World Outcomes for Standard-Of-Care Treatments in Patients with Relapsed/Refractory Multiple Myeloma

Session Title: 905. Outcomes Research—Lymphoid Malignancies: Poster III

Date/Time: Monday, December 13, 2021 6:00 PM – 8:00 PM EST

Location: Georgia World Congress Center, Hall B5

Abstract #1932

Poster

Considerations for optimal administration of Chimeric Antigen Receptor (CAR) T-Cell therapy programs: a multi-stakeholder qualitative analysis

Session Title: 902. Health Services Research—Lymphoid Malignancies: Poster I

Date/Time: Saturday, December 11, 2021 5:30 PM – 7:30 PM

Location: Georgia World Congress Center, Hall B5

Abstract #1700

Poster

Tri-specific CD19xCD20xCD22 VHH CAR-T cells (LCAR-AIO) eradicate antigen-heterogeneous B cell tumors, enhance expansion, and prolong persistence in preclinical in vivo models

Session Title: 703. Cellular Immunotherapies: Basic and Translational: Poster I

Date: Saturday, December 11, 2021 5:30-7:30 PM

Location: Georgia World Congress Center, Hall B5

About CARTITUDE-1

CARTITUDE-1 (NCT03548207) is a Phase 1b/2, open-label, multicenter study evaluating the safety and efficacy of cilta-cel in adults with relapsed or refractory with multiple myeloma, who previously received a proteasome inhibitor (PI), an immunomodulatory agent (IMiD) and an anti-CD38 antibody, and who had disease progression on or after the last regimen.1 The primary objective of the Phase 1b portion of the study was to characterize the safety and confirm the recommended Phase 2 dose of cilta-cel, informed by the first-in-human study with LCAR-B38M CAR-T cells (LEGEND-2). The Phase 2 portion further evaluated the efficacy of cilta-cel with overall response rate as the primary endpoint. Of the 97 patients enrolled in the trial, 99 percent were refractory to the last line of treatment and 88 percent were triple-class refractory, meaning their cancer did not respond, or no longer responds, to an IMiD, a PI and an anti-CD38 antibody.

About CARTITUDE-2

CARTITUDE-2 (NCT04133636) is an ongoing Phase 2 multicohort study evaluating the safety and efficacy of cilta-cel in various clinical settings. Cohort A included patients who had progressive multiple myeloma after 1–3 prior lines of therapy, including PI and IMiD, were lenalidomide refractory, and had no prior exposure to BCMA-targeting agents. Cohort B included patients with early relapse after initial therapy that included a PI and IMiD. The primary objective was percentage of patients with negative minimal residual disease (MRD).2

About CARTITUDE-5

CARTITUDE-5 (NCT04923893) is a Phase 3 open-label study of bortezomib, lenalidomide, and dexamethasone (VRd) followed by cilta-cel vs. VRd followed by Rd maintenance, in patients with newly diagnosed MM for whom autologous stem cell transplant (ASCT) is not planned as initial therapy.

About LocoMMotion

LocoMMotion (NCT04035226) is a prospective non-interventional study evaluating the safety and efficacy of real-life standard-of-care treatments under routine clinical practice over a 24-month period in patients with RRMM. This study aims to understand the effectiveness of current standards of care in heavily pretreated patients with RRMM (reflecting real-world practice in the patient population progressing after PIs, IMiDs and anti-CD38 antibodies).

About Multiple Myeloma
Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excessive proliferation of plasma cells.3 Although treatment may result in remission, unfortunately, patients will most likely relapse.4 Relapsed myeloma is when the disease has returned after a period of initial, partial or complete remission and does not meet the definition of being refractory.5 Refractory multiple myeloma is when a patient’s disease is non-responsive or progresses within 60 days of their last therapy.6,7 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections. 8 Patients who relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents, have poor prognoses and few treatment options available.9

About Cilta-cel
Cilta-cel is an investigational chimeric antigen receptor T cell (CAR-T) therapy, formerly identified as JNJ-4528 in the U.S. and Europe and LCAR-B38M CAR-T cells in China, that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed or refractory multiple myeloma and in earlier lines of treatment. The design consists of a structurally differentiated CAR-T with two BCMA-targeting single domain antibodies. In December 2017, Legend Biotech, Inc. entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. (Janssen) to develop and commercialize cilta-cel. In addition to a Breakthrough Therapy Designation (BTD) granted in the U.S. in December 2019, cilta-cel received a Priority Medicines (PRiME) designation from the European Commission in April 2019, and a BTD in China in August 2020. In addition, Orphan Drug Designation was granted for cilta-cel by the U.S. FDA in February 2019, and by the European Commission in February 2020. A Biologics License Application seeking approval of cilta-cel was submitted to the U.S. FDA and a Marketing Authorization Application was submitted to the European Medicines Agency.

Dendreon Pharmaceuticals and Shoreline Biosciences Announce CMC and Manufacturing Alliance to Advance the Future of iPSC Cellular Therapy

On November 4, 2021 Dendreon Pharmaceuticals, a commercial-stage biopharmaceutical company and pioneer in the development of cellular immunotherapy, and Shoreline Biosciences, a biotechnology company developing allogeneic off-the-shelf, standardized, and targeted natural killer (NK) and macrophage cellular immunotherapies derived from induced pluripotent stem cells (iPSC) for cancer, reported an alliance to advance the future of iPSC-derived cellular therapies (Press release, Shoreline Biosciences, NOV 4, 2021, View Source [SID1234594522]).

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The alliance leverages Dendreon’s extensive manufacturing, process development and end-to-end logistics expertise for the advancement of Shoreline’s pipeline of iPSC-derived cellular therapies. Dendreon is providing scalable cGMP manufacturing support for certain programs through clinical development and launch, enabling Shoreline to rapidly advance multiple products in parallel.

"With more than a decade of proven expertise in cell therapy manufacturing and an established supply chain and logistics infrastructure, Dendreon is well positioned to support Shoreline in manufacturing from Phase I clinical trials through commercialization," said Maria Cho, Vice President of Business Development and Corporate Strategy. "We are thrilled to partner with Shoreline to enable the future of cell therapy and change the way serious diseases are treated."

"We are excited to partner with Dendreon, a leader in cell therapy, to manufacture cost-efficient, highly-scalable product candidates," said Mohammad El-Kalay, Ph.D., Senior VP & Head of CMC for Shoreline. "Through our partnership with Dendreon, we are accelerating the commercialization of our next generation NK cell and macrophage products to bring scalable, allogeneic, "off the shelf" therapies to more patients in need."

ArsenalBio Announces Participation in Upcoming Society for Immunotherapy of Cancer’s (SITC) 36th Annual Meeting

On November 4, 2021 ArsenalBio, a privately held programmable cell therapy company focused on building advanced CAR T therapies for solid tumors, reported that it will present pre-clinical data from AB-X, the company’s integrated circuit T cell therapy program for the treatment of ovarian cancer (OC), at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 36th Annual Meeting taking place November 10-14, 2021 in Washington D.C., and virtually (Press release, Arsenal Bio, NOV 4, 2021, View Source [SID1234594521]).

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The accepted abstract titles are now available on the SITC (Free SITC Whitepaper) website. Details of the poster are as follows:

Title: AB-X integrated circuit T cells demonstrate improved potency, expansion, and specificity compared to unaugmented MSLN CAR T cells
Poster Number: 213
Presenter: Stephen Santoro, Ph.D., Senior Director, Program Lead, ArsenalBio
Date and Time: The ePoster will be released virtually on Friday, Nov. 12, 2021 at 7:00 a.m. ET. Full text of the abstract will be released on the SITC (Free SITC Whitepaper) website on Tuesday, Nov. 9, 2021 at 8:00 a.m. ET.

About AB-X

AB-X is ArsenalBio’s lead discovery program for ovarian cancer. In the United States, ovarian cancer ranks fifth in cancer deaths among women and accounts for more deaths than any other cancer of the female reproductive system. T cell infiltration into tumors correlates with improved survival, but existing CAR T cell therapies have demonstrated modest benefits, suggesting Arsenal’s approach could transform the treatment paradigm. AB-X leverages a dual antigen sensing logic gate approach, targeting ALPG/P and MSLN, which are co-expressed in over 70% of primary ovarian cancers, for enhanced tumor specificity and improved safety. This dual logic gate ensures that the T cell killing is only activated at the site of the tumor. In addition, AB-X is engineered to knockdown FAS and PTPN2, two critical regulators of T cell function and persistence. Knockdown of FAS and PTPN2 results in CAR T cells that are resistant to FAS-mediated apoptosis, demonstrate enhanced expansion in vivo and show greater efficacy compared with unaugmented MSLN CAR T cells. As such, AB-X integrated circuit T cells are expected to be more specific and more potent than conventional CAR T cell approaches. We intend to file an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) for AB-X in 2022.

Inspirna Announces Clinical Collaboration with Bristol Myers Squibb to Study RGX-104 in Combination with Yervoy® (ipilimumab) for Treatment of Metastatic Endometrial Cancer

On November 4, 2021 Inspirna, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and biologic cancer therapeutics, reported a collaboration with Bristol Myers Squibb on a clinical trial of a combination therapy using RGX-104 (abequolixron), the Company’s small molecule agonist of the Liver X Receptor/Apolipoprotein E ("LXR/APOE") pathway, and Yervoy (ipilimumab), Bristol Myers Squibb’s Cytotoxic T-Lymphocyte Associated protein 4 (CTLA-4) inhibitor (Press release, Inspirna, NOV 4, 2021, View Source [SID1234594520]). Under the terms of the agreement, Bristol Myers Squibb will provide ipilimumab for a Phase 1b/2 expansion study investigating the combination therapy for the 2nd and 3rd line treatment of patients with metastatic endometrial cancer whose genomes possess the E2 or E4 APOE genetic biomarker, including those patients who have progressed on prior checkpoint inhibitor therapy. Inspirna will sponsor the study and be responsible for study costs.

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RGX-104 is an orally administered LXR agonist that potently activates APOE protein expression in tumors to inhibit cancer progression. Approximately 40% of the human population harbors either the E2 or E4 variants of the APOE gene in their genomes. These APOE genetic variants can be readily identified by analyzing DNA from blood samples. The presence of either of the E2 or E4 variants has been shown to increase the likelihood of a favorable clinical response to RGX-104 treatment in some cancers, including endometrial cancer, in a Phase 1 clinical trial, thus providing a potential biomarker for RGX-104 therapy. RGX-104 is currently being investigated in a Phase 1b/2 clinical trial in combination with standard-of-care regimens in several lung cancer indications that are enriched for dysregulation of the APOE gene, including small cell lung cancer (SCLC) and non-small cell lung cancer (NSCLC).

"We are proud to embark on this collaboration with Bristol Myers Squibb to uncover an additional possible application of RGX-104 beyond our current areas of study," said Masoud Tavazoie, M.D., Ph.D., and Chief Executive Officer of Inspirna. "This collaboration validates the work we are doing with RGX-104, and follows our recent phase 1 combination dose escalation trial results, in which treatment with RGX-104 combination regimens resulted in a 60% response rate in patients with relapsed or refractory cancers that had progressed on prior checkpoint inhibitor therapy and possessed the E2 or E4 APOE genetic biomarkers. We look forward to working with Bristol Myers Squibb and unlocking the potential of RGX-104 as we aim to improve the lives of people suffering with cancer."

Yervoy is a trademark of Bristol Myers Squibb Company.

About RGX-104 (abequolixron)

RGX-104 (abequolixron) is an orally administered small molecule agonist of the Liver X Receptor (LXR) which activates expression of the APOE tumor suppressor protein. APOE expression becomes dysregulated (silenced) in the tumors of select patients with solid cancers. APOE dysregulation results in increased tumor angiogenesis (tumor blood vessel growth) as well as a shifting of the tumor myeloid cell population from immune-stimulatory to immune-suppressive, which are both counteracted by RGX-104. RGX-104 is currently being tested in a Phase 1b/2 clinical trial in combination with standard-of-care regimens in several lung cancer indications that are enriched for APOE dysregulation, including SCLC and NSCLC. Inspirna expects to present data from this clinical study in 2H 2022.

Ambrx Biopharma Inc. Announces First Patient Dosed in its Global Phase 2 ACE-Breast-03 Clinical Study of ARX788 for the Treatment of HER2-Positive Metastatic Breast Cancer

On November 4, 2021 Ambrx Biopharma Inc., or Ambrx, (NYSE: AMAM), a clinical stage biopharmaceutical company using an expanded genetic code technology platform to create Engineered Precision Biologics, reported the first patient has been dosed in its global ACE-Breast-03 Phase 2 clinical study of ARX788 in patients with HER2-positive metastatic breast cancer (Press release, Ambrx, NOV 4, 2021, View Source [SID1234594519]).

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ARX788 is an anti-HER2 antibody drug conjugate (ADC) that is being studied broadly in breast cancer, gastric/GEJ cancer and other solid tumors. The FDA has granted ARX788 Fast-Track Designation for the treatment of HER2-positive metastatic breast cancer in December 2020.

"Dosing the first patient in this Phase 2 study of ARX788 in patients with HER2-positive metastatic breast cancer marks an important milestone for Ambrx," said Feng Tian, Ph.D., Chairman of the Board, President, and CEO of Ambrx. "We have made excellent progress with our clinical development pipeline over the last few months, highlighted by our positive data of ARX788 for HER2-positive gastric cancer, as well as the dosing of the first patient in a Phase 1 trial of ARX517 for PSMA expressing tumors. Our growing clinical programs, coupled with an influx of capital from our IPO in June 2021, leaves Ambrx well-positioned to potentially attain several near-term clinical and corporate milestones."

The global ACE-Breast-03 Phase 2 clinical study is a multicenter study to evaluate the efficacy and safety of ARX788 in HER2-positive, metastatic breast cancer patients whose disease is resistant or refractory to T-DM1, and/or T-DXd, and/or tucatinib-containing regimens. The primary outcome measure of the study will be the objective response rate.