On September 28, 2021 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported that Christine Brown, Ph.D., Deputy Director, T Cell Therapeutics Research Laboratory and The Heritage Provider Network Professor in Immunotherapy, will present updated Phase 1 clinical data regarding MB-101 (IL13Rα2‐targeted CAR T cells) at two upcoming scientific conferences in October 2021 (Press release, Mustang Bio, SEP 28, 2021, View Source [SID1234590406]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Details of the virtual presentations are as follows:

First Annual Conference on CNS Clinical Trials, Co-sponsored by the Society for Neuro-Oncology and American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)

Title: CAR T Cells in Primary Brain Tumors
Session: Session 3: Immunotherapy – Hype and Hope
Date and Time: Friday, October 1, 2021 at 2:30 PM ET
For more information, please click here.

American Association for Cancer Research Virtual Special Conference: Brain Cancer

Title: Advancing CAR T Cell Therapy for Glioblastoma
Session: Plenary Session 2: Novel Therapeutics
Date and Time: Monday, October 25, 2021 at 1:15 PM ET
For more information, please click here.

Mustang is collaborating with City of Hope on several chimeric antigen receptor T cell clinical trials, including MB‐101 (IL13Rα2‐targeted CAR T cells) clinical trials for brain cancer. Additional information about the brain cancer clinical trials can be found on clinicaltrials.gov using identifiers NCT04661384, NCT02208362 and NCT04003649.

About MB‐101 (IL13Rα2‐targeted CAR T cells)
IL13Rα2 is an attractive target for CAR T therapy as it has limited expression in normal tissue but is overexpressed on the surface of the majority of malignant glioma cells, including glioblastoma multiforme, ependymoma and medulloblastoma. CAR T cells are designed to express a membrane‐tethered IL‐13 receptor ligand (IL‐13) incorporating a single‐point mutation that provides high affinity for IL13Rα2 and reduces binding to IL13Rα1 in order to reduce healthy tissue targeting. Mustang is developing MB‐101 as an optimized CAR T product incorporating enhancements in CAR design and T cell engineering to improve antitumor potency and T cell persistence. MB‐101 includes a second‐generation hinge optimized CAR containing mutations in the IgG4 linker to reduce off‐target Fc interactions, the 4-1BB (CD137) co‐stimulatory signaling domain for improved persistence of CAR T cells and the extracellular domain of CD19 as a selection/safety marker. To further improve persistence, central memory T cells are enriched and genetically engineered using a manufacturing process that limits ex vivo expansion to reduce T cell exhaustion and maintain a memory T cell phenotype.

On September 28, 2021 Moderna, Inc. (Nasdaq: MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, reported that Stephen Hoge, M.D., President, will participate in a fireside chat at Chardan’s Virtual 5th Annual Genetic Medicines Conference on Tuesday, October 5th at 1:00 p.m. ET (Press release, Moderna Therapeutics, SEP 28, 2021, View Source [SID1234590405]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A live webcast of each presentation will be available under "Events and Presentations" in the Investors section of the Moderna website at investors.modernatx.com. A replay of the webcast will be archived on Moderna’s website for 30 days following the presentation.

On September 28, 2021 Aptevo Therapeutics Inc. ("Aptevo") (NASDAQ:APVO), a clinical-stage biotechnology company focused on developing novel immuno-oncology therapeutics based on its proprietary ADAPTIR and ADAPTIR-FLEX platform technologies, reported that the prestigious medical journal Frontiers in Aging, published a peer-reviewed article by Fatih Uckun, M.D., Ph.D., Chief Clinical Advisor at Aptevo who is coordinating the APVO436 clinical development program (Press release, Aptevo Therapeutics, SEP 28, 2021, View Source [SID1234590404]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The article discusses the clinical impact potential of bispecific antibodies (BiAB) capable of redirecting host T-cell cytotoxicity to malignant clones as well as immunosuppressive myeloid-derived suppressor cells (MDSC) as a new class of anti-MDS drug candidates.

The article, "CD123-Directed Bispecific Antibodies for Targeting MDS Clones and Immunosuppressive Myeloid-Derived Suppressor Cells (MDSC) in High-Risk MDS Patients," has been published in Frontiers in Aging, section "Neoplastic Pathologies of Aging,"

and it is available online. To view the online publication, click here:
http://journal.frontiersin.org/article/10.3389/fragi.2021.757276/full?&utm_source=Email_to_authors_&utm_medium=Email&utm_content=T1_11.5e1_author&utm_campaign=Email_publication&field=&journalName=Frontiers_in_Aging&id=757276.

Citation Reference: Uckun FM and Watts J (2021) CD123-Directed Bispecific Antibodies for Targeting MDS Clones and Immunosuppressive Myeloid-Derived Suppressor Cells (MDSC) in High-Risk Adult MDS Patients. Front. Aging 2:757276. doi: 10.3389/fragi.2021.757276

Adult myelodysplastic syndrome (MDS), a heterogeneous group of clonal malignant hematologic disorders with an incidence rate of 4.5 per 100,000 persons per year, is characterized by an enhanced risk of transformation to acute myeloid leukemia (AML). There is no effective standard treatment that will prevent the leukemic transformation or result in sustained deep remissions in high-risk adult MDS patients.

The immunosuppressive bone marrow microenvironment (BMME) in adult MDS has been implicated in clonal evolution and disease progression. Expanded populations of myeloid-derived suppressor cells (MDSC) contribute to the immunosuppressive tumor microenvironment (TME) by inhibiting both memory and cytotoxic effector T-cell populations as well as natural killer (NK) cells, thereby promoting the immune evasion of MDS clones. The abundance of MDSC is associated with a higher risk of rapidly progressive disease and poor survival outcomes in adult MDS.

The expression of CD123 on MDSC as well as MDS clones provides a compelling rationale for targeting CD123 antigen on the malignant clones as well as the MDSC in the immunosuppressive BMME of adult MDS patients in an effort to delay disease progression and transformation to AML.

In a recently completed APTEVO study the results of which have been published in the respected oncology journal, Cancers, APVO436 induced bone marrow complete remissions in 3 of 6 evaluable high-risk MDS patients, providing the first proof of concept that APVO436 is a new candidate anti-MDS drug.

"There is an urgent need to identify effective strategies to prevent leukemic transformation and induce sustained deep remissions in adult high-risk myelodysplastic syndrome (MDS) patients," explained Fatih Uckun, M.D. Ph.D., the study’s lead author. "T-cell engaging bispecific antibodies targeting the CD123 antigen like our lead drug candidate APVO436 may help delay disease progression in high-risk adult MDS and potentially reduce the risk of transformation to secondary AML."

"Emerging data that show APVO436 can sufficiently empower dysfunctional and exhausted T-cells to induce remissions in relapsed AML and MDS patients is the driving motivation behind our current clinical development plan for our lead clinical candidate," said Marvin White, CEO of Aptevo.

About APVO436

Overexpression of CD123 is the hallmark of many forms of leukemia. Aptevo’s lead proprietary drug candidate, APVO436 is a bispecific ADAPTIR that targets CD123 x CD3 and is designed to redirect the immune system of the patient to destroy leukemia cells expressing the target CD123 molecule on their surface. This antibody-like recombinant protein therapeutic is designed to engage both leukemia cells and T-cells of the immune system and bring them closely together to trigger a rapid and complete destruction of leukemia cells. APVO436 has been engineered using Aptevo’s proprietary and enabling bioengineering methods and is designed to reduce the likelihood and severity of an unintended and potentially harmful activation of the immune system. APVO436 has been engineered to stay in the blood circulation long enough to locate, bind with and destroy target leukemia cells. APVO436 has received orphan drug designation ("orphan status") for AML according to the Orphan Drug Act.

On September 28, 2021 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported that it earned a $10 million milestone payment from Janssen Pharmaceuticals, Inc., (Janssen) part of the Janssen Pharmaceutical Companies of Johnson & Johnson (Press release, Arrowhead Pharmaceuticals, SEP 28, 2021, View Source [SID1234590403]). The milestone payment was earned after Janssen dosed the fifth patient in a Phase 1 clinical study of ARO-JNJ1, an investigational RNAi therapeutic candidate which utilizes Arrowhead’s proprietary Targeted RNAi Molecule (TRiM) platform.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Arrowhead entered into a license and collaboration agreement, and into a research collaboration and option agreement, with Janssen in October 2018. Arrowhead is eligible for additional payments when future milestones are met and is also eligible for royalties on commercial sales of ARO-JNJ1.

On September 28, 2021 Cartesian Therapeutics, a fully integrated, clinical-stage biopharmaceutical company pioneering RNA cell therapy in and beyond oncology, reported the hire of Miloš Miljković, M.D., as Vice President and Chief Medical Officer (Press release, Cartesian Therapeutics, SEP 28, 2021, View Source [SID1234590401]). Dr. Miljković will oversee the strategy and execution of the Company’s growing clinical pipeline of RNA cell therapies. In addition, the Cartesian appointed co-founder Metin Kurtoglu, M.D., Ph.D., as the Company’s first Chief Operating Officer, responsible for bridging clinical operations with the cGMP manufacturing and translational medicine groups. Cartesian also announced that it has expanded its cGMP manufacturing facility in Gaithersburg, MD to support its growing clinical pipeline.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are delighted to have Miloš join our leadership team at Cartesian," said Murat Kalayoglu, M.D., Ph.D., President and Chief Executive Officer at Cartesian Therapeutics. "His experience in developing and executing clinical programs will be a valuable addition to our team as we accelerate development of our three clinical-stage RNA cell therapies and matriculate new therapies into clinical trials."

Dr. Miljković added, "I am eager to lead Cartesian’s engagement with the medical community and regulators throughout clinical development. I am impressed by the pace of progress at Cartesian to date, highlighted by recent positive clinical data from the ongoing Phase 1/2a clinical trial of Descartes-08 in generalized Myasthenia Gravis (gMG). Cartesian’s focus on using the cell as both a factory for producing, as well as a vehicle for delivering, RNA therapeutics is a novel, highly differentiated strategy, one that has the potential to change the treatment paradigm for a spectrum of indications."

Dr. Miljković is board-certified in hematology, medical oncology, and internal medicine. Prior to joining Cartesian, he was a Staff Clinician at the National Cancer Institute for over seven years, where he specialized in early-stage trials in immuno-oncology. Dr. Miljković has served as Principal Investigator on 6 clinical trials of immunotherapy and targeted agents and as Associate Investigator on more than 20 others. He completed residency at the Johns Hopkins University and Sinai Hospital of Baltimore program in internal medicine, followed by clinical and research fellowships in the joint NCI/NHLBI program at the NIH, where he also served as Chief Fellow.

Meanwhile, Cartesian is nearly doubling its footprint in Gaithersburg to accommodate the Company’s growing need for GMP manufacturing and clinical operations space. As part of this expansion, Cartesian expects to double its headcount in 2022.

Dr. Kurtoglu stated, "Our in-house cGMP manufacturing capabilities are essential to our long-term strategy: to pioneer RNA cell therapy as a new therapeutic modality. As COO, I look forward to working closely with Miloš to create an optimal environment for cross-functional development of our RNA cell therapies."