On February 8, 2021 The Sarah Cannon Transplant and Cellular Therapy Network, formerly the Sarah Cannon Blood Cancer Network, reported 19 abstracts and presentations have been accepted for presentation at the 2021 TCT | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, held virtually from February 8-12, 2021 (Press release, Sarah Cannon Research Institute, FEB 8, 2021, View Source [SID1234574742]). The meetings bring together thousands of healthcare professionals for a full scientific program that address the most timely issues in hematopoietic cell transplantation (HCT) and cellular therapy.

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"We look forward to discussing the important developments in our rapidly evolving field at this year’s TCT meetings," says Fred LeMaistre, MD, Physician-in-Chief of Blood Cancers, Sarah Cannon. "Sarah Cannon experts will share presentations focused on immune effector cell therapy and the efficacy and treatment standards of HCT for both adult and pediatric patients. Together, the advances we are making across the network will ultimately better serve our patients."

The Sarah Cannon Blood Cancer Network has evolved, as have the therapies it offers, to become the Sarah Cannon Transplant and Cellular Therapy Network in 2021. This transition reflects the latest advancements in the field and the broader options now available to patients. In 2020, the Sarah Cannon Transplant and Cellular Therapy Network performed ~1,200 hematopoietic cell transplants and treated more than 100 patients with various cellular therapies, including both FDA-approved and research options, across eight Foundation For The Accreditation of Cellular Therapy (FACT)/Joint Accreditation Committee ISCT-Europe & EBMT (JACIE) accredited sites.

2021 TCT | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR oral and poster presentations with Sarah Cannon Transplant and Cellular Therapy Network experts as first authors will be presented by:

Haydar Frangoul, MD, MS, Medical Director, Pediatric Hematology/Oncology, Sarah Cannon Pediatric Transplant and Cellular Therapy Program at TriStar Centennial, who will present "Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia (TDT) or Sickle Cell Disease (SCD): Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-Cas9-Modified CD34+ Hematopoietic Stem and Progenitor Cells (HSPCs)" in an oral presentation on February 10 from 3-5 p.m. CST.
Jesus G. Berdeja, MD, Director, Myeloma Research, Sarah Cannon Research Institute, who will present "Efficacy and Safety of Idecabtagene Vicleucel (ide-cel, bb2121) in Elderly Patients with Relapsed and Refractory Multiple Myeloma: KarMMa Subgroup Analysis" in a poster presentation on February 8 – February 12 from 8:30 a.m. – 5:30 p.m. CST.
Therese Dodd, BA, MBA, RN, CPHQ, Quality Improvement Director, Sarah Cannon Transplant and Cellular Therapy Network, who will present "Successful Virtual Mock FACT Inspection: How Sarah Cannon Blood Cancer Network and Texas Transplant Institute in San Antonio and Austin Made It Work" and "Resiliency During a COVID 19 Pandemic: The Quality Manager Perspective" in two poster presentations on February 8 – February 12 from 8:30 a.m. – 5:30 p.m. CST.
Alireza Eghtedar, MD, Associate Member Physician, Colorado Blood Cancer Institute at Presbyterian/St. Luke’s Medical Center- HealthONE, who will present "Non-Myeloablative Hematopoietic Stem Cell Transplantation (NMA HSCT) Utilizing Low-Dose Total Body Irradiation (TBI) Plus Fludarabine (Flu): A Comparison of Single-Center Based Flu 150 Mg/m2 Plus 400 Cgy TBI Versus Flu 90 Mg/m2 and 200 Cgy TBI Containing Regimens" in a poster presentation on February 8 – February 12 from 8:30 a.m. – 5:30 p.m. CST.
Racheal Peaytt, PharmD, PGY-2 Oncology Pharmacy Resident, Sarah Cannon Cancer Institute at TriStar Centennial Medical Center, who will present "The Impact of Early Versus Late Tocilizumab Use in Patients with Cytokine Release Syndrome Receiving Immune Effector Cell Therapy" in a poster presentation on February 8 – February 12 from 8:30 a.m. – 5:30 p.m. CST.

On February 8, 2021 Seneca Therapeutics, Inc. ("STI"), a clinical-stage biopharmaceutical company dedicated to the development of targeted oncolytic immunotherapeutics based on Seneca Valley Virus (SVV-001), reported the expansion of its R&D pipeline with six new armed gene therapy/oncolytic constructs directed against important cancer targets and indications (Press release, Seneca Therapeutics, FEB 8, 2021, View Source [SID1234574741]). STI also announced an SVV-001 Armed Construct program developing precision medicine constructs expressing patient-specific Neo-antigens. Each of these new gene therapy technologies uses SVV to exclusively target cancer cells with TEM 8 expression. Normal cells lack significant expression of TEM 8 and are therefore not infected by SVV-001, and so the armed transgene will not express in normal cells. Studies in multiple human tumor types indicates TEM 8 expression is an adverse indicator of long-term survival and is associated with cancer metastasis. STI intends to develop each combination as an IV product, taking advantage of the multiple IV dosing program currently underway at the company:

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SVV-012- SVV+ Anti PD-L1 – "Addition of a checkpoint inhibitor is designed to enhance the CD8+ T-cell response against tumors"
SVV-024 – SVV+ Enhanced IL-2 Gene – "This construct is designed to bind IL-2 receptors to activate the immune response in tumors without activating CD25 and thereby avoid toxicities observed with the IL-2 protein"
SVV-037 – SVV+ CXCL-9 – "This construct is designed to express the chemokine CXCL-9 in the tumor microenvironment and promote recruitment of immune cells into treated tumors"
SVV-044 – SVV+ TGF-beta decoy – "This construct is designed to block TGF-beta signaling in tumors and avoid immunosuppression"
SVV- 058 SVV+ Nitroreductase – "This nitroreductase construct is designed to function as a prodrug and convert cytotoxic metabolites selectively in the tumor microenvironment"
SVV- 069 – SVV+ IL-2/IL-15 Fusion Protein – "Vectors delivering IL-2/IL-15 agonists are designed to improve immune cell trafficking and infiltration into tumors and provide both T-cell and NK-cell signals"
Each of these products either has been or is being produced in STI’s SVV platform. Animal studies for each product are anticipated to begin in 2Q21.

The SVV-001 Armed Construct for Neo-antigens program plans to use patient specific neo-antigens to take advantage of the rapid two-week development cycle for SVV-001 Armed Constructs.

"SVV itself has many distinct advantages over other forms of cancer therapy. Chief among these is the exquisite cancer cell specificity of both SVV replication and the expression of a transgene. Together, these make SVV a unique and powerful multi-modal platform," said Dr. Paul Hallenbeck, Ph.D. Founder and President of STI.

On February 8, 2021 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a pioneer in T-cell immunotherapy, leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with serious diseases including solid tumors, hematologic cancers and autoimmune diseases, reported the presentation of data supporting the proposed mechanism of action of their lead product candidate, tabelecleucel (tab-cel), and five additional poster presentations at the Transplantation & Cellular Therapy (TCT) Meeting of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR), held virtually February 8-12, 2021 (Press release, Atara Biotherapeutics, FEB 8, 2021, View Source [SID1234574740]).

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"We are pleased to present key translational data advancing the clinical development of ATA188 and tab-cel, the most advanced allogeneic T-cell immunotherapy in development. Additional data being presented at TCT also support the further development of tab-cel in EBV-driven diseases, such as EBV+ acquired and primary immunodeficiency lymphoproliferative diseases (AID-LPD and PID-LPD) for which we’re currently enrolling patients for a clinical study," said Jakob Dupont, M.D., Head of Global Research & Development at Atara. "These are diseases with few treatment options and poor prognosis, and it is our goal to ultimately bring this potentially transformative therapy to patients in need. We look forward to advancing tab-cel, remaining on track to complete the Biologics License Application in the third quarter of this year based on very encouraging clinical data from an interim analysis of our pivotal trial."

Results presented at TCT detail new findings from a comprehensive multiomic analysis of modes of activation for tab-cel. Each component of the assessment contributes to building the overall understanding of therapeutic activity, mechanisms of action and extended characteristics.

In this analysis, a subset of tab-cel lots were used to confirm and establish the consistency of the product’s profile, regardless of donor.

These results demonstrated, that upon stimulation, tab-cel exhibits a consistent activation signature at the level of gene expression, T-cell receptor engagement (TCR), and secretion of factors associated with effective T cell responses. Results further illustrated that the tab-cel manufacturing process results in complementary clonal expansion and consistent enrichment of TCRs associated with productive engagement of EBV-driven diseases.

Atara will also report additional data sets related to tab-cel, as well as preclinical findings demonstrating the potent anti-tumor activity of the Company’s next-generation CD19-based CAR T, ATA3219.

Additionally, the Company is presenting data on an innovative testing solution to detect and quantify non-engineered allogeneic T-cell therapies such as ATA188. As demonstrated in partnership with CareDx, AlloCell is a precise and reproducible method that resolves the analytical barrier for non-engineered allogeneic cell therapies, which do not have transgenes available to support standard quantitative pharmacokinetic (PK) assay development. The novel testing solution offers potential applications in clinical development for determining PK profiles and correlating with response and other clinical endpoints, including the understanding of fundamental aspects of pharmacology such as presence and expansion in the patient’s body.

"Together these analyses represent a leading edge for allogeneic cell therapy research, leveraging state-of-the-art profiling technologies and innovating new methodologies in collaboration with CareDx to support development of Atara’s programs," said Blake Aftab, Head of Preclinical Science and Translational Medicine at Atara. "These methods not only support our novel allogeneic EBV T-cell development programs, but also have potential for wide-ranging applications for patients."

Atara Poster Presentations at TCT 2021:
All posters will be available for viewing at the start of the meeting on Monday, February 8, 2021.

Title: Comprehensive activation profiling of tabelecleucel, an off-the-shelf, allogeneic EBV-specific T cell therapy
Poster #: 206

Title: A sensitive and precise universal surveillance solution for pharmacokinetic monitoring of off-the-shelf cell therapies (in collaboration with CareDx)
Poster #: 204

Title: Clinical experience of tabelecleucel in patients with EBV+ primary (PID) or acquired immunodeficiency (AID) associated lymphoproliferative disease (encore from ASH (Free ASH Whitepaper) 2020)
Poster #: 219

Title: Clinical experience of tabelecleucel in patients with life-threatening complications of Epstein–Barr virus viremia (encore from ASH (Free ASH Whitepaper) 2020)
Poster #: 223

Title: A multicenter, multicohort, open-label, single arm per cohort, Phase II study to assess the efficacy and safety of tabelecleucel in patients with EBV-associated diseases using an adaptive two-stage study design (encore from ASH (Free ASH Whitepaper) 2020)
Poster #: 532

Title: ATA3219: A potent next-generation allogeneic off-the-shelf CD19 CAR-T therapy without the need for gene editing (encore from ASH (Free ASH Whitepaper) 2020)
Poster #: 203

On February 8, 2021 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, reported plans to release its financial results for the fourth quarter and full-year 2020 ended December 31, 2020, on Thursday, February 25, 2021, before the opening of the financial markets (Press release, Akebia, FEB 8, 2021, View Source [SID1234574739]).

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Akebia will host a conference call at 9:00 a.m. Eastern Time on Thursday, February 25, 2021 to discuss its fourth quarter and full-year 2020 financial results and recent business highlights. To listen to the conference call, please dial (877) 458-0977 (domestic) or (484) 653-6724 (international) using conference ID number 5455117. The call will also be webcast live and can be accessed via the Investors section of the Company’s website at View Source

A replay of the conference call will be available two hours after the completion of the call through March 3, 2021. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 5455117. An online archive of the conference call can be accessed via the Investors section of the Company’s website at View Source

On February 8, 2021 Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune and blood systems reset via stem cell transplant to more patients, reported data presentations across its stem cell mobilization and targeted conditioning programs at the Transplantation and Cellular Therapy (TCT) Annual Meeting, to be held virtually on February 8-12, 2021 (Press release, Magenta Therapeutics, FEB 8, 2021, View Source [SID1234574738]).

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"Magenta continues to generate encouraging data across our pipeline, furthering our commitment to patients to expand eligibility and improve the clinical outcomes with stem cell transplant," said John Davis Jr., M.D., M.P.H., M.S., Magenta’s Head of Research & Development and Chief Medical Officer. "Our presentations this year at TCT highlight the potential wide-ranging utility of our portfolio, and we are particularly excited to share these results, and to continue our progress in the year ahead."

Oral Presentations Showcasing Clinical Data of MGTA-145 Stem Cell Mobilization Program

Magenta is developing MGTA-145 in combination with plerixafor utilizing complementary mechanisms to mobilize hematopoietic stem cells (HSCs) for collection and transplantation. This combination has the potential to be the preferred mobilization regimen for rapid, reliable, predictable and safe collection of high numbers of functional blood stem cells to improve outcomes across autologous and allogeneic stem cell transplantation, which also includes stem cells necessary for all HSC-based gene therapies.

Title: MGTA-145 / Plerixafor-Mediated HSC Mobilization and Intravenous HDAd5/35++ Vector Injection into Mice Allows for Efficient in vivo HSC Transduction and Stable Gene Marking in Peripheral Blood Cells (Oral Abstract, #16)
Presenting Author: Chang Li, Ph.D., Division of Medical Genetics, Department of Medicine, University of Washington
Date and Time of Presentation: Session B – Transplantation for Non-Malignant Disease; Monday, February 8, 2021, 3:15PM CST / 4:15PM EST

Data from this preclinical study demonstrate the potential of MGTA-145 plus plerixafor to serve as an efficient, single-dose mobilization regimen for in vivo HSC gene therapy where stem cells could be gene corrected or edited without having to remove them from the body. This could potentially replace current mobilization regimens that rely on ex vivo gene therapy approaches to treat genetic diseases.

Title: MGTA-145, in Combination with Plerixafor in a Phase 1 Clinical Study, Mobilizes Large Numbers of Hematopoietic Stem Cells and a Graft with Potent Immunosuppressive Properties for Autologous and Allogeneic Transplant (Oral Abstract, #35)
Presenting Author: Kevin Goncalves, Ph.D., Magenta Therapeutics
Date and Time of Presentation: Session E – Consider the Source: Stem Cell Grafts and Donors; Tuesday, February 9, 2021, 4:00PM CST / 5:00PM EST

Data from this Phase 1 clinical trial with healthy volunteers further underscore the potential utility of MGTA-145 plus plerixafor as an effective, single-day mobilization and collection regimen for autologous and allogeneic HSC transplant. MGTA-145 plus plerixafor mobilized high numbers of HSCs and showed durable engraftment, successful gene-modification and immunosuppressive properties by reducing Graft-versus-Host disease (GvHD) in preclinical models.

Oral Presentation Showcasing Preclinical Study of MGTA-117 Targeted ADC Conditioning Program

Magenta is developing a suite of novel antibody-drug conjugates (ADCs) for conditioning, a step in the transplant process that currently relies on the use of systemic chemotherapy agents and radiation. Magenta’s targeted conditioning programs are designed to selectively eliminate stem cells and/or immune cells from a patient prior to transplant or gene therapy, and to reduce or potentially eliminate the need for high dose or high intensity chemotherapy-based treatments. These programs focus on developing targeted products that remove specific cell types, with an approach that is tailored to the patient’s disease and transplant requirements.

MGTA-117, Magenta’s most advanced conditioning program, is a CD117-targeted ADC designed to precisely deplete hematopoietic stem and progenitor cells to clear space in the bone marrow prior to transplant, and to support long-term engraftment and improved disease outcomes in patients. MGTA-117 has shown to be highly selective with potent activity, efficacy and tolerability in preclinical models.

Title: A Single Dose of a Novel Anti-Human CD117-Amanitin Antibody Drug Conjugate (ADC) Engineered for a Short Half-life Provides Dual Conditioning and Anti-Leukemia Activity and Extends Survival Compared to Standard of Care in Multiple Pre-clinical Models of Acute Myeloid Leukemia (AML) (Oral Abstract, #53)
Presenting Author: Leanne Lanieri, M.S., Magenta Therapeutics
Date and Time of Presentation: Session H – Novel Conditioning Regimens & Transplantation for Aged Populations, Wednesday, February 10, 2021, 4:00PM CST / 5:00PM EST

Hematopoietic stem cell transplant (HSCT) can often be a curative treatment for patients with acute myeloid leukemia (AML). There is currently a need for safer and more effective targeted conditioning agents, as current conditioning regimens are associated with severe toxicities and high post-transplant relapse or graft failure. MGTA-117 was studied in multiple human leukemic xenograft murine models to mimic untreated and refractory AML. In preclinical models, MGTA-117 significantly increased median survival versus a multi-day standard-of-care regimen using cytarabine. Data from this study demonstrate MGTA-117’s potential as a potent, targeted HSCT conditioning agent with anti-leukemic activity, emphasizing its potential to improve HSCT outcomes in AML by reducing the risk of post-transplant relapse.

Poster Presentation Highlighting Preclinical Data of CD45-ADC Targeted Conditioning Program

Magenta’s other ADC-based conditioning program, CD45-ADC, targets both patient HSCs and disease-causing immune cells. The program’s lead target is CD45, a cell surface molecule broadly expressed throughout the hematopoietic and immune systems. CD45-ADC has the potential to significantly increase the number of patients eligible to receive a stem cell transplant, particularly those patients with autoimmune diseases and acute leukemias.

Developing a broad targeting approach for safer patient conditioning prior to HSCT could bring the curative potential of allogeneic HSCT to more patients with both malignant and non-malignant disorders. Current conditioning regimens limit accessibility of this procedure due to toxicity.

Title: Targeted CD45 Antibody Drug Conjugate Enables Full Mismatch Allogeneic Hematopoietic Stem Cell Transplantation in a Murine HSCT Model as a Single Agent (AML) (Poster #242)
Lead Author: Sharon Hyzy, M.S., Magenta Therapeutics

Data from this study showed conditioning with single agent CD45-ADC enabled complete chimerism in a full mismatch allogeneic HSCT model.

Oral Presentation of MGTA-456 Stem Cell Therapy Expansion Program in Patients with Blood Cancer

Magenta is continuing long-term patient follow up to evaluate MGTA-456 in blood cancers through the investigator-initiated Phase 2 trial in blood cancers at the University of Minnesota and will assess best next steps for the program. Magenta previously announced in June 2020 it had discontinued enrollment in the Phase 2 trial of MGTA-456 in patients with inherited metabolic disorders.

Title: MGTA-456, A CD34 Expanded Cord Blood Product, Permits Selection of Better HLA Matched Units and Results in Rapid Hematopoietic Recovery, Uniform Engraftment and Reduced Graft-Versus-Host Disease in Adults with High-Risk Hematologic Malignancies (Oral Abstract, #31)
Presenting Author: Heather Stefanski, M.D., Ph.D., Assistant Professor, Department of Pediatrics, University of Minnesota
Date and Time of Oral Presentation: Session E – Consider the Source: Stem Cell Grafts and Donors; Tuesday, February 9, 2021, 3:00PM CST / 4:00PM EST

Twenty-two patients were enrolled in the study, with 18 transplanted with MGTA-456. Compared to transplant patients who had undergone the same conditioning, GvHD prophylaxis and supportive care, patients who received MGTA-456 showed faster neutrophil recovery (median of 17 days compared to 23 days) and better platelet recovery (median 36 days compared to 59 days). Additionally, incidence of grade 2-4 acute GvHD was lower (24% compared to 46%), likely because of the ability to find a better matched cord unit.