On April 2, 2024 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported that the European Medicines Agency (EMA) has accepted the Company’s Marketing Authorization Application (MAA) for obecabtagene autoleucel (obe-cel) (Press release, Autolus, APR 2, 2024, View Source [SID1234641707]). Obe-cel is Autolus’ lead investigational chimeric antigen receptor (CAR) T cell therapy, for the treatment of patients with relapsed/refractory (r/r) adult B-cell Acute Lymphoblastic Leukemia (ALL). The MAA submission was based on data from the pivotal Phase 2 FELIX study of obe-cel in adult r/r B-ALL.

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Autolus’ Nucleus site has recently received the formal certification from the MHRA following a full inspection of the site in February 2024. The MHRA issued two new GMP certificates to cover both clinical and commercial manufacture from the site.

"Along with the recent acceptance of the BLA by the FDA, the acceptance of our EU marketing application is an important milestone in expanding into the European market and delivering this potentially transformative therapy to B-ALL patients," commented Dr. Christian Itin, Chief Executive Officer of Autolus. "We look forward to working with the EMA throughout the evaluation process of obe-cel, and thank the internal team at Autolus for their work on the submission and Nucleus site inspection."

Obe-cel has been granted Orphan Drug Designation by the FDA, Orphan Medical Product Designation by the EMA, Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA and PRIority MEdicines (PRIME) designation by the EMA for adult r/r B-ALL.

On April 2, 2024 BostonGene, a leading provider of AI-driven molecular and immune profiling solutions, reported that eight abstracts were selected for poster presentations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024, which will be held from April 5 – 10, at the San Diego Convention Center in San Diego, CA (Press release, BostonGene, APR 2, 2024, View Source [SID1234641723]). BostonGene will exhibit at booth #847.

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BostonGene, together with its collaborators, will deliver presentations on various topics, including the feasibility and utility of using comprehensive genomic and transcriptomic analysis for patients with blood and solid cancers, RNA-based models for TLS prediction in both lung and pancreatic cancers, a transcriptomic-based model to distinguish sarcomatoid features in kidney cancer and the use of comprehensive molecular profiling to evaluate the impact of race and HIV status on the genetic landscape of patients with diffuse large B-cell lymphoma (DLBCL) in southern Africa.

Details of BostonGene’s poster presentations at the AACR (Free AACR Whitepaper) Annual Meeting are below:

1) Title: Comprehensive Genomic and Transcriptomic Analysis to Guide Therapy for Patients with Metastatic Solid Tumors
Abstract number: 939
Date and time: Sunday, April 7, 2024 | 1:30 PM – 5:00 PM
Session title: Application of Precision Medicine for Cancer Care
Location: Poster section 39
Speaker: Burak Uzunparmak, MD, PhD, The University of Texas MD Anderson Cancer Center

This study shows the feasibility and utility of comprehensive molecular profiling to match patients to WES-informed treatments within a clinically relevant time frame. The high rate of additional actionable findings identified by BostonGene’s analytical platform suggest comprehensive testing may offer benefit over traditional targeted panels in solid tumor patients.

Research conducted in collaboration with MD Anderson Cancer Center

2) Title: Test-the-test: Clinical utility of comprehensive whole exome sequencing (WES) and RNA-seq for lymphoma patients
Abstract number: 1754
Date and time: Monday, April 8, 2024 | 9:00 AM – 12:30 PM
Session title: Genomic Characterization of Cancers and Cancer Subgroups
Location: Poster section 17
Speaker: Dai Chihara, MD, PhD, MD Anderson Cancer Center

This study shows an acceptable turnaround time for delivering BostonGene Tumor PortraitTM test reports that include clinically relevant findings and potential clinical trial matches, demonstrating the feasibility of using integrated WES and RNA-seq to guide clinical decision-making in lymphoma patients.

Research conducted in collaboration with MD Anderson Cancer Center

3) Title: Comparative analysis of a predictive transcriptomic model and functional gene expression signatures (FGES) for tertiary lymphoid structure (TLS) detection in lung adenocarcinoma (LUAD)
Abstract number: 6826
Date and time: Wednesday, April 10, 2024 | 9:00 AM – 12:30 PM
Session title: Gene Expression Regulation in the Tumor Microenvironment
Location: Poster section 8
Speaker: Alexander Bagaev, PhD, BostonGene

BostonGene developed a transcriptomic model for TLS detection in LUAD samples that demonstrated improved metrics and prognostic value compared to previously reported TLS FGES, highlighting its potential to guide therapeutic decision-making.

4) Title: Unveiling Heterogeneity of Cancer-Associated Fibroblasts (CAFs) Across Multiple Solid Cancer Types
Abstract number: 291
Date and time: Sunday, April 7, 2024 | 1:30 PM – 5:00 PM
Session title: Stroma Interactions
Location: Poster section 11
Speaker: Boris Shpak, MSc, BostonGene

To evaluate the heterogeneity of stromal cells in various solid cancer diagnoses, single-cell RNA sequencing and bulk RNA-seq data was used. Three consistent cancer-associated fibroblast subtypes were defined, laying the groundwork for a pan-cancer model to characterize stromal cell diversity that could guide efforts in targeting CAFs with anti-tumor therapies.

5) Title: Unraveling sarcomatoid features in clear cell renal cell carcinoma with RNA-seq
Abstract number: 4922
Date and time: Tuesday, April 9, 2024 | 9:00 AM – 12:30 PM
Session title: Artificial Intelligence and Machine/Deep Learning 3
Location: Poster section 36
Speaker: Andrey Shubin, PhD, BostonGene

This presentation describes the robust performance of a BostonGene-developed transcriptomic model in effectively identifying sarcomatoid features in ccRCC samples. With additional investigation, the sarcomatoid ccRCC transcriptomic model can be leveraged to guide checkpoint inhibitor selection for ccRCC patients.

6) Title: An RNA-based model for tertiary lymphoid structure (TLS) prediction and classification in pancreatic adenocarcinoma (PDAC)
Abstract number: 4909
Date and time: Tuesday, April 9, 2024 | 9:00 AM – 12:30 PM
Session title: Artificial Intelligence and Machine/Deep Learning 3
Location: Poster section 36
Speaker: Andrey Tyshevich, MSc, BostonGene

This presentation describes BostonGene’s development of an RNA-based model that stratifies PDAC samples based on TLS status. The results demonstrate an association between TLS-low groups and worse overall survival, offering a method to predict prognoses of PDAC patients based on TLS status.

7) Title: Improved survival and unique mutational signatures in small cell lung cancer arising in patients with limited tobacco use
Abstract number: 3867
Date and time: Tuesday, April 9, 2024 | 9:00 AM – 12:30 PM
Session title: Molecular Biology in Clinical Oncology: Genetics and Beyond
Location: Poster section 41
Speaker: Kyle Concannon, MD, MD Anderson Cancer Center

This presentation describes the use of whole exome sequencing (WES) to uncover targetable mutations in light-smokers with small cell lung cancer (SCLC), highlighting the use of molecular profiling in SCLC patients with minimal tobacco use.

Research conducted in collaboration with MD Anderson Cancer Center

8) Title: Genomic distinctions of HIV- and EBV-associated DLBCL in a diverse African cohort
Abstract number: 786
Date and time: Sunday, April 7, 2024 | 1:30 PM – 5:00 PM
Session title: Epidemiology
Location: Poster section 32
Speaker: Katherine Antel, MD, Dana Farber Cancer Institute

In this study, whole exome and whole transcriptome sequencing was utilized to evaluate the impact of HIV status and Epstein-Barr Virus (EBV) on the genetic landscape, molecular subtypes, and survival outcomes of diffuse large B-cell lymphoma (DLBCL) in a diverse African cohort.

Research conducted in collaboration with Dana Farber Cancer Institute

The abstracts will be published in an online-only Proceedings supplement to the AACR (Free AACR Whitepaper) journal Cancer Research after the conclusion of the AACR (Free AACR Whitepaper) Annual Meeting.

On April 2, 2024 EpicentRx, Inc, a clinical-stage biotechnology drug and device company with two therapeutic platforms that address cancer and inflammatory diseases of unmet clinical need, reported that an abstract on its lead therapy, AdAPT-001, will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting to be held in San Diego, CA from April 5-10, 2024 (Press release, EpicentRx, APR 2, 2024, View Source [SID1234641777]).

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AdAPT-001 constitutes a novel strategy to deliver a transforming growth factor-beta (TGF-β) trap to tumors. Importantly, data from a Phase 1/2 clinical trial demonstrate that administration of AdAPT-001 successfully converted immunologically cold tumors, such as sarcomas and triple negative breast cancer, into immunologically hot tumors. Furthermore, AdAPT-001 administration also demonstrates reversion of established resistance to checkpoint inhibitors.

"We’re excited to share groundbreaking clinical data with our lead therapy, AdAPT-001, against several treatment- and checkpoint inhibitor resistant solid tumors," said Tony R. Reid, MD, PhD, CEO of EpicentRx. "We’re also thrilled to be working with top-notch investigators like Dr. Anthony P. Conley from the MD Anderson Cancer Center and Dr. Lucy B. Kennedy from the Cleveland Clinic."

"Immune checkpoint inhibitors are largely ineffective against sarcomas. TGF-β is a soluble protein that suppresses immune responses," said treating study investigator and sarcoma oncologist, Dr. Conley from the MD Anderson Cancer Center. "Based on the several unprecedented responses that I have seen with AdAPT-001, which expresses a TGF-β trap, this TGF-β blockade from AdAPT-001 synergizes with PD-1/PD-L1 inhibition in checkpoint-resistant sarcoma."

Poster Presentation:
Title: Improved clinical outcomes in patients that received treatment beyond tumor progression with AdAPT-001 +/- a checkpoint inhibitor
Presenters: Dr. Anthony P. Conley of MD Anderson Cancer Center and Drs Tony Reid (CEO) and Chris Larson (VP) of EpicentRx.
Date and Time: Tuesday, April 9, 2024, 1:30 PM – 5:00 PM PDT
Location: Poster Section 48
Poster Number: 23

About AdAPT-001
AdAPT-001 is an investigational immunotherapy with a TGF-β receptor-immunoglobulin Fc fusion trap, designed to neutralize isoforms 1 and 3 of the profibrotic, proangiogenic, prohypoxic, and immunosuppressive cytokine, TGF-β, and to sensitize resistant tumors to checkpoint blockade.

In the ongoing Phase 1/2 BETA PRIME trial, AdAPT-001 was administered as single-agent and in combination with checkpoint inhibitors to patients with treatment-refractory tumors.

Importantly, AdAPT-001 plus checkpoint inhibitors improved toxicity and AE profile over what is typically observed with checkpoint inhibitors. No dose limiting toxicities, AdAPT-001 related serious adverse events, or dose reductions have been observed to date.

On April 2, 2024 MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the Standard Market of the Tokyo Stock Exchange (Code Number: 4875), reported that an abstract regarding results of a clinical trial of MN-166 (ibudilast) in glioblastoma (GBM) has been selected for an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (2024 ASCO (Free ASCO Whitepaper)) Annual Meeting to be held May 31 – June 4, 2024 in Chicago (Press release, MediciNova, APR 2, 2024, View Source [SID1234641708]). The oral presentation will be presented by one of the investigators of this clinical trial, Gilbert Youssef, M.D., Attending Physician at Harvard Medical School, Center for Neuro-Oncology at Dana-Farber Cancer Institute and Brigham and Women’s Hospital.

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The presentation details are as follows:

Abstract Number: 2106

Title: Phase 1b/2a study evaluating the combination of MN-166 (ibudilast) and temozolomide in patients with newly diagnosed and recurrent glioblastoma (GBM)

Session Title: Rapid Oral Abstract – Central Nervous System Tumors
Session Date: June 2, 2024
Session Time: 11:30 AM – 1:00 PM

About MN-166 (ibudilast)

MN-166 (ibudilast) is a small molecule compound that inhibits phosphodiesterase type-4 (PDE4) and inflammatory cytokines, including macrophage migration inhibitory factor (MIF). It is in late-stage clinical development for the treatment of neurodegenerative diseases such as ALS (amyotrophic lateral sclerosis), progressive MS (multiple sclerosis), and DCM (degenerative cervical myelopathy); and is also in development for glioblastoma, Long COVID, CIPN (chemotherapy-induced peripheral neuropathy), and substance use disorder. In addition, MN-166 (ibudilast) was evaluated in patients that are at risk for developing acute respiratory distress syndrome (ARDS).

On April 2, 2024 Kelonia Therapeutics, a biotech company revolutionizing in vivo gene delivery, reported new preclinical data from its lead program KLN-1010, which will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024 in San Diego, California (Press release, Kelonia Therapeutics, APR 2, 2024, View Source [SID1234641724]). KLN-1010, a novel, in vivo CAR-T cell therapy candidate for the treatment of multiple myeloma, which leverages the company’s in vivo Gene Placement System (iGPS) technology, demonstrated that it is safe and effective in preclinical animal models.

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"These data reinforce the best-in-class potential of our in vivo CAR-T cell therapies," said Kevin Friedman, Ph.D., Chief Executive Officer and Founder of Kelonia. "By eliminating the need for ex vivo manufacturing and toxic lymphodepleting chemotherapy, we believe our iGPS technology will remove barriers that currently prevent patients from accessing transformative genetic medicines. We are excited by the preclinical profile of KLN-1010, and are looking forward to advancing this program towards the clinic in the near future."

Diverse T cell lineages, including memory and effector CD4 and CD8 T cells were modified in vivo by KLN-1010 to express a fully human anti-BCMA CAR, without detectable transduction of multiple myeloma tumor cells. A single intravenous injection of KLN-1010 displayed potent anti-tumor efficacy and caused complete tumor regression at multiple dose levels in several preclinical animal models. In other studies, treatment of non-human primates with an iGPS particle expressing an anti-CD20 CAR resulted in potent CAR-T cell activity lasting several months without the need for additional treatments or conditioning chemotherapy.

Details for the poster presentation are as follows:

Title: T cell-specific in vivo transduction with preclinical candidate KLN-1010 generates BCMA directed CAR T cells with potent anti-multiple myeloma activity
Poster Session: Adoptive Cell Therapies 2: CAR-T Cells
Date and Time: April 7, 2024 at 1:30-5:00 pm PT
Location: Section 2
Poster Number: 16