On August 31, 2020 Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, reported that data across the portfolio will be presented at the European Society for Blood and Marrow Transplantation (EBMT) annual meeting, held August 29 to September 1, 2020 (Press release, Magenta Therapeutics, AUG 31, 2020, View Source [SID1234564190]).

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"Our presentations at EBMT showcase Magenta’s integrated approach towards bringing the curative power of blood and immune system reset through stem cell transplant to more patients and across more therapeutic areas," said John Davis Jr., M.D., M.P.H., M.S., Head of Research & Development and Chief Medical Officer, Magenta Therapeutics. "We are making significant strides towards expanding eligibility and improving patient outcomes given the far-reaching potential of our programs. We are relentlessly focused on progressing our programs forward and further into the clinic and are encouraged by our steady progress. We look forward to our continued momentum across our pipeline."

All posters will be available to view on the EBMT website beginning Saturday, August 29 at 6:30am EDT / 12:30pm CET. Visitors can view the posters by accessing the Scientific Programme and selecting "ePoster viewing."

Clinical Data from MGTA-145 First-Line Stem Cell Mobilization Program:

Magenta is developing MGTA-145 as the new first-line standard of care for stem cell mobilization in a broad range of diseases, including autoimmune diseases, blood cancers and genetic diseases, such as sickle cell disease. MGTA-145, a CXCR2 agonist, works in combination with plerixafor, a CXCR4 antagonist, harnessing the physiological mechanism of stem cell mobilization to rapidly and robustly mobilize stem cells for collection and transplant.

Title: Phase 1 Clinical Study of MGTA-145 In Combination with Plerixafor Shows Rapid Single-Day Mobilization and Collection of CD34+ HSCs Without G-CSF (Abstract #B385)
Presenting Author: John DiPersio, M.D., Ph.D., Professor of Medicine and Chief of the Oncology Division, Washington University School of Medicine, St. Louis, Missouri

These data provide further confirmation that MGTA-145, in combination with plerixafor, enables the same-day mobilization and collection of highly functional hematopoietic stem cells (HSCs) for transplant. Earlier this year, Magenta completed dosing in this Phase 1 trial, achieving all primary and secondary endpoints. In the second half of 2020, the Company plans to move the MGTA-145 program into multiple Phase 2 clinical trials to include both allogeneic and autologous transplant settings across multiple diseases. These trials will evaluate mobilization and collection of functional HSCs and engraftment of these cells after transplant.

Preclinical Data from Magenta’s Antibody-Drug Conjugate Conditioning Programs

Targeted, disease-modifying antibody-drug conjugates (ADCs) are designed to selectively and rapidly remove disease-causing cells in the body and enable immune and blood system reset and long-term engraftment, without the need for aggressive chemotherapy or radiation.

MGTA-117, the clinical candidate for ADC-based conditioning for stem cell transplant and gene therapy and Magenta’s most advanced conditioning program, is on track with IND-enabling toxicology studies ongoing and progress in GMP manufacturing. Magenta expects to generate initial clinical data in 2021.

Title: A Single Dose of Short Half-Life CD117 Antibody Drug Conjugate Enables Hematopoietic Stem Cell-Based Gene Therapy in Non-Human Primates (Abstract #O076)
Presenting Author: Rahul Palchaudhuri, Ph.D., Magenta Therapeutics, Cambridge, Mass.

This collaborative study with the National Institutes of Health (NIH) demonstrates that a single dose of a tool CD117-ADC molecule in non-human primates enables successful transplant and long-term engraftment of HSCs modified with a lentiviral vector encoding the β-globin gene, the gene that causes sickle cell disease and β-thalassemia. The ADC was well tolerated with none of the significant side effects that are seen with myeloablative dosing of busulfan chemotherapy.

Title: A Novel Targeted Approach to Achieve Immune System Reset: CD45-Targeted Antibody-Drug Conjugates Ameliorate Disease in Preclinical Autoimmune Disease Models and Enable Auto HSCT (Abstract #O030)
Presenting Author: Geoff Gillard, Ph.D., Magenta Therapeutics, Cambridge, Mass.

Magenta’s CD45-ADC program targets CD45, a protein expressed on immune cells and blood stem cells, and is designed to remove the cells that cause autoimmune diseases to enable curative immune reset. Magenta has identified a lead antibody for this program and IND-enabling work on CD45-ADC is progressing in 2020.

Preclinical data in this abstract show that a single dose of CD45-ADC removed disease-causing T-cells, enabling successful immune reset to halt disease progression and was well tolerated in three models of autoimmune disease: multiple sclerosis, systemic sclerosis and inflammatory arthritis.

Title: A CD45-Targeted Antibody Drug Conjugate Enables Allogeneic Hematopoietic Stem Cell Transplantation as a Single Agent in Mice (Abstract #A174)
Presenting Author: Sharon Hyzy, M.S., Magenta Therapeutics, Cambridge, Mass.

Preclinical data in this abstract demonstrate that a single dose of CD45-ADC is fully myeloablative and enables complete chimerism in a full mismatch allogeneic hematopoietic stem cell transplant (HSCT), representing a substantial advance in establishing the potential of this targeted approach to conditioning to potently and safely enable immune reset in the allogeneic setting.

Title: High Dose Stem Cell Therapies, like MGTA-456, Enable Complete Neural and Peripheral Disease Cross-Correction Through Rapid and Robust Hematopoietic Engraftment (Abstract #A325)
Presenting Author: Kevin Goncalves, Ph.D., Magenta Therapeutics, Cambridge, Mass.

Preclinical data in this abstract demonstrate rapid and durable peripheral, central nervous system (CNS) and skeletal disease cross-correction after transplantation of a high HSC dose. Mechanistic studies demonstrated that CNS disease cross-correction was due to robust microglial engraftment in the brain. MGTA-456 led to more robust microglial engraftment in NSG mice, suggesting that MGTA-456 may lead to rapid and durable disease resolution in the central nervous system.

On August 31, 2020 Repertoire Immune Medicines, a clinical-stage biotechnology company focused on decoding and deploying the immune system across multiple major diseases, reported that it will present at Citi’s 15th Annual BioPharma Virtual Conference on Tuesday, September 8, 2020 at 9:30 am ET (Press release, Repertoire Genesis, AUG 31, 2020, View Source [SID1234564189]).

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On August 31, 2020 Seneca Therapeutics, Inc., a clinical-stage biopharmaceutical company dedicated to the development of oncolytic immune-therapeutics based on Seneca Valley Virus (SVV-001), reported the company has licensed TEM 8 intellectual property from Memorial Sloan Kettering Cancer Center (MSK) (Press release, Seneca Therapeutics, AUG 31, 2020, View Source [SID1234564188]). TEM8 may enable pre-screening of solid tumors to determine if SVV-001 might be effective in that patient. This discovery is based on research from Charles Rudin, MD., Ph.D. and colleagues at MSK. Researchers at MSK discovered that a protein called TEM 8 is the receptor of SVV. This discovery supports other work in the scientific field that TEM 8 is very selectively expressed on the surface of tumor cells in many solid cancer indications. Dr. Rudin and his laboratory demonstrated that the level of expression of TEM 8 and genes in the innate immune system could predict with a high degree of certainty what type of cancer cells would support SVV replication in and killing of the tumor cells. This research, originally published in 2017, will be utilized in upcoming SVV-001 clinical trials to pre-select patients that might best respond to SVV–mediated cancer therapy.

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Dr. Rudin is the Sylvia Hassenfeld Professor and Chief of Thoracic Oncology at Memorial Sloan Kettering Cancer Center. Dr. Rudin and his colleagues have been investigating SVV as a novel and promising cancer immunotherapeutic since 2005. Dr. Rudin said, "I couldn’t be more pleased to see this new exciting enabling technology head toward POC Phase II trials to demonstrate that SVV is efficacious and synergistic with checkpoint inhibitors."

Dr. Paul Hallenbeck, Founder, President, and Chief Scientific Officer at Seneca Therapeutics added, "Unlike most other oncolytic viruses SVV only replicates and kills cells that express TEM 8, namely solid tumor cells. MSK’s technology should also enable the wise selection of cancer indications best able to benefit from SVV therapy and enhance the number of patients that should respond."

On August 31, 2020 TRACON Pharmaceuticals (NASDAQ: TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported that following the closing of the private placement announced on August 27, 2020, it has entered into an additional definitive securities purchase agreement with multiple institutional health care focused funds to raise aggregate gross proceeds of approximately $5.0 million through a private placement of its common stock and pre-funded warrants (Press release, Tracon Pharmaceuticals, AUG 31, 2020, View Source [SID1234564187]). The closing of the private placement is expected to occur on or about August 31, 2020.

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TRACON will sell approximately 3.0 million shares of common stock, or in lieu of common stock, pre-funded warrants to purchase common stock, for aggregate gross proceeds of approximately $5.0 million. The purchase price of each share of common stock (or pre-funded warrant) is approximately $1.67. The pre-funded warrants will have a per share exercise price of $0.01 and will expire seven years from the date of issuance.

TRACON intends to use the net proceeds from the private placement to conduct the ENVASARC pivotal study of envafolimab in sarcoma and for working capital and general corporate purposes. As previously announced, the ENVASARC trial was cleared by the U.S. Food and Drug Administration on August 14, 2020.

The securities being sold in the private placement have not been registered under the Securities Act of 1933, as amended, or state securities laws and may not be offered or sold in the United States absent registration with the Securities and Exchange Commission ("SEC") or an applicable exemption from such registration requirements. TRACON has agreed to file a registration statement with the SEC covering the resale of the shares of common stock issuable in connection with the private placement, including upon exercise of the pre-funded warrants.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.

On August 31, 2020 Unum Therapeutics Inc. (Nasdaq: UMRX), a biopharmaceutical company focused on developing novel, best-in-class precision kinase inhibitors for a range of patients living with cancer and other unmet medical needs, reported the sale of its cell-based BOXR programs to SOTIO, a clinical stage immuno-oncology company owned by PPF Group (Press release, Unum Therapeutics, AUG 31, 2020, View Source [SID1234564184]). Under the terms of the agreement, SOTIO will make an upfront payment of $8.1 million for the BOXR technology and will assume development of Unum’s lead candidate, BOXR1030, which is on track for near-term entry into the clinic. In addition, Unum will be eligible to receive downstream milestones of up to $3.4 million. The sale to SOTIO will enable it to further advance its goal to develop the next generation of potent immunotherapies for patients with cancer. Unum will retain its antibody coupled T cell receptor (ACTR) technology and continues to explore strategic opportunities for the technology and assets. The sale is final as of August 28, 2020.

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"Within the past year, we have explored a range of strategic opportunities for our BOXR technology and programs, and we believe SOTIO has the clinical expertise to accelerate the development of this program, including BOXR1030, in multiple clinical trials for the benefit of patients," said Chuck Wilson, Ph.D., President and CEO of Unum. "We are extremely proud of the BOXR technology and programs and would like to thank all of our employees, and investors for not only supporting our vision, but advancing it as well. We look forward to SOTIO’s progress on the BOXR programs in the future."

SOTIO will assume responsibility for a portion of Unum’s facilities in Cambridge, MA to advance the BOXR programs, and certain Unum staff associated with the BOXR programs will transition to SOTIO to continue their work on the programs. Unum will retain certain staff and space in its Cambridge facilities as it refocuses on the development of small molecule precision kinase inhibitors, including its lead program PLX9486.

On July 6, 2020, Unum announced the completion of the acquisition of Kiq LLC, as part of a strategic pivot to focus on precision kinase inhibitors. Certain Unum stockholders of record at the time of the acquisition were granted a non-tradeable contingent value right (CVR). Holders of the CVR will be entitled to receive certain stock and/or cash payments from net proceeds received by Unum related to the disposition of Unum’s cell therapy assets for a period of three years following the closing of the transaction.

Net proceeds from the sale of BOXR will be reinvested into the development of PLX9486 as part of Unum’s focus on the development of small molecule precision kinase inhibitors to treat a range of genetically driven diseases. Additional operational updates will be provided in the upcoming months.

In addition, Unum appointed Peter Harwin, Co-founder and Managing Member, Fairmount Funds Management LLC as Board Chair. Harwin played an integral role in the recent acquisition of Kiq and will help guide the future vision of the company in delivering best-in-class therapies for patients with genetically defined diseases. Prior to founding Fairmount in 2016, Harwin served as a member of the investment team at Boxer Capital, a biotechnology unit of the Tavistock Group, and he currently serves as a strategic advisor to Quellis Biosciences Inc. and Dianthus Therapeutics, Inc. Harwin also serves on the board of directors of Viridian Therapeutics, Inc.

About BOXR and BOXR1030

Unum Therapeutics’ novel cell therapy programs address the major scientific obstacles in traditional T cell therapies. Scientists have discovered that the solid tumor microenvironment is highly immunosuppressive, blocking T cells from functioning as they should. The BOXR technology (Bolt-on Chimeric Receptor) addresses this issue by incorporating "bolt-on" transgenes to enhance intrinsic T cell functionality and overcome multiple mechanisms of immunosuppression in the solid tumor microenvironment. BOXR transgenes can then be engineered with therapeutic T cells, such as CAR T cells, to improve the functionality of T cell therapies, particularly in the solid tumor microenvironment. BOXR1030, the first product candidate from BOXR, contains the novel "bolt-on" enzyme called glutamic-oxaloacetic transaminase 2 (GOT2) that aims to improve intrinsic T cell function in the solid tumor microenvironment through enhanced metabolism. GOT2 plays a central metabolic role by linking multiple pathways involved in biosynthesis and cellular energy production.