TME Pharma Receives US FDA Fast Track Designation for Lead Asset NOX-A12 in Brain Cancer

On April 2, 2024 TME Pharma N.V. (Euronext Growth Paris: ALTME), a biotechnology company focused on developing novel therapies for treatment of cancer by targeting the tumor microenvironment (TME), reported that the US Food and Drug Administration (FDA) has granted Fast Track designation for NOX-A12 (olaptesed pegol), TME Pharma’s CXCL12 inhibitor, in combination with radiotherapy and bevacizumab for use in the treatment of the aggressive adult brain cancer, glioblastoma, in the newly diagnosed setting where the tumor is resistant to chemotherapy and measurable tumor remains after surgery (Press release, TME Pharma, APR 2, 2024, View Source [SID1234641726]).

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The FDA’s Fast Track designation aims to bring important new drugs to patients more quickly, facilitating the development and expediting the review of therapies intended to treat serious conditions and address unmet medical needs. Companies whose programs are granted Fast Track designation can benefit from more frequent interactions with the FDA during the clinical development process and potentially "accelerated approval" and "priority review" if the relevant criteria are met.

TME Pharma continuously evaluates ways to advance the clinical development of NOX-A12 while remaining focused on identifying and securing financial resources from multiple sources, including those having no or minimal dilutive effect on its shareholders, such as governmental grants or free supply of combination drugs. In addition to engaging with industry partners and specialized healthcare investors, TME Pharma will also explore the eligibility of NOX-A12-based therapy for compassionate use programs once sufficient Phase 2 data have been generated. The company would prioritize such programs that support financial compensation for therapies leading to revenue generation, thus potentially reducing the financial needs of late-stage clinical development and also helping to generate real-world clinical evidence.

Recently announced clearance by the FDA of TME Pharma’s Investigational New Drug (IND) application for a Phase 2 study with NOX-A12 in glioblastoma, that the company plans to initiate later this year, was a prerequisite to having Fast Track designation granted by the FDA. Having Fast Track designation in addition to an open IND with an FDA-approved study design that addresses questions of dosing and contribution of components optimizes late phase development and offers an economically efficient model which further de-risks TME Pharma’s glioblastoma program. Following IND approval, this Fast Track designation is an external validation of NOX-A12’s potential to address the unmet need for glioblastoma patients.

The necessary preparatory steps for the NOX-A12 Phase 2 in glioblastoma are ongoing, and TME Pharma is aiming to initiate the new Phase 2 study as soon as the necessary resources from financial and industrial partners have been secured. TME Pharma is prioritizing discussions with partners willing to support the company over the long term and having their financial interests aligned with current stakeholders. While discussions are ongoing, and until longer-term agreements with partners are reached, TME Pharma is determined to keep operational costs low to extend the financial visibility as far as possible and increase the chance of success.

TME Pharma’s latest regulatory milestones were supported by recent survival data from the GLORIA Phase 1/2 study in which NOX-A12 demonstrated an unprecedented median Overall Survival of 19.9 months in chemotherapy resistant patients with residual tumor after surgery, which compared very favorably to a matched standard of care reference cohort and exceeds what TME Pharma believes to be all relevant competitor therapy trials in newly diagnosed glioblastoma patients resistant to standard chemotherapy.

"At the start of this year, we announced the next phase of our development of NOX-A12 by targeting IND approval and an expedited regulatory pathway in the US and we are very proud to have successfully achieved these milestones within the timeframe we set out," said Aram Mangasarian, CEO of TME Pharma. "While advancing discussions with potential industrial and financial partners may require some time to materialize, the open IND and Fast Track designation awarded by the FDA are well-received signals by these partners. We now have a clear clinical development roadmap with which to take NOX-A12 forward in the treatment of glioblastoma and to support engagement with potential partners. We expect our new Phase 2 study will build on the unprecedented results of our GLORIA trial, which strengthens the potential of NOX-A12 to become the treatment option of choice for newly diagnosed chemotherapy-resistant glioblastoma. We look forward to working closely with the FDA as we advance NOX-A12 to market as quickly as possible for the benefit of patients suffering from this devastating and aggressive cancer for which there is extremely poor prognosis."

Eureka Therapeutics Announces UCSF Benioff Children’s Hospitals as First California Site for ARYA-2 Clinical Trial Targeting Pediatric Liver Cancers

On April 2, 2024 Eureka Therapeutics, Inc., a clinical-stage biotechnology company developing novel T-cell therapies to treat cancer, reported the activation of UCSF Benioff Children’s Hospitals as the first California site to join the ARYA-2 clinical trial (NCT04634357) (Press release, Eureka Therapeutics, APR 2, 2024, View Source [SID1234641725]).

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The ARYA-2 trial is evaluating Eureka’s investigational ARTEMIS ET140203 T-cell therapy which targets the alpha-fetoprotein (AFP)-peptide/HLA-A2 complex. This therapy is designed to treat pediatric subjects (1 to 21 years) with relapsed or refractory liver cancer, including hepatoblastoma (HB), hepatocellular neoplasm not otherwise specified (HCN-NOS), and hepatocellular carcinoma (HCC).

"Multiply relapsed or refractory liver cancers in children and adolescents are highly complex and lack effective treatment options, especially given the rarity of these conditions," said Dr. Arun Rangaswami, MD, Professor of Clinical Pediatrics and a senior solid tumor faculty member at UCSF. "We are excited to collaborate with Eureka to bring the promise of next-generation engineered T-cell therapy to this pediatric population with high unmet medical needs."

"We are proud to contribute to the collaborative effort aimed at developing cutting edge therapies for treating pediatric cancer," said Dr. Abla Creasey, PhD, Vice President of Therapeutics Development, at the California Institute for Regenerative Medicine (CIRM). "Our recent $10.6 million grant towards Eureka’s ARYA-2 program underscores our commitment to improving the lives of young patients battling cancer."

"Our ARTEMIS T-cells are designed to overcome challenges in safely and effectively treating solid tumors," said Dr. Cheng Liu, President and CEO of Eureka Therapeutics. "UCSF’s participation significantly accelerates progress in bringing this potentially life-saving therapy to young patients with liver cancer."

This expansion builds upon Eureka’s ongoing efforts with the ARYA-2 trial, which is also actively recruiting patients at the Dana-Farber Cancer Institute. For more information on the ARYA-2 trial, Eureka’s T-cell therapy platform, and the potential of this treatment, please visit eurekaconnectme.com.

Kelonia Therapeutics to Present Preclinical Data Highlighting Therapeutic Potential of in vivo CAR-T Cell Therapy in Multiple Myeloma

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

BostonGene Announces Acceptance of Eight Abstracts to be Presented at the American Association for Cancer Research Annual Meeting 2024

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.

Invenra Inc. Announces a Strategic Collaboration with Catalent to Co-Discover Novel Bispecific ADCs

On April 2, 2024 Invenra Inc., an innovative leader in bispecific antibody technology, reported a strategic collaboration with Catalent, a global Contract Development and Manufacturing Organization (CDMO) specializing in innovative drug development solutions (Press release, Invenra, APR 2, 2024, View Source [SID1234641722]). The collaboration will harness the combined expertise and proprietary technologies of Invenra and Catalent to co-discover novel bispecific antibody-drug conjugates.

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Dr. Roland Green, CEO of Invenra, remarks, "We are excited to collaborate with the innovative team at Catalent’s Redwood Bioscience subsidiary. We believe that the combination of Invenra’s B-Body bispecific antibody platform with Catalent’s SMARTag ADC expertise has the potential to unlock synergies and accelerate the development of next-generation cancer therapeutics."

Both companies share a commitment to developing novel therapeutic solutions with the potential to make a meaningful impact in the lives of patients.