On August 27, 2024 Illumina, Inc. (NASDAQ: ILMN), a global leader in DNA sequencing and array-based technologies, reported Food and Drug Administration (FDA) approval of its in vitro diagnostic (IVD) TruSight Oncology (TSO) Comprehensive test and its first two companion diagnostic (CDx) indications (Press release, Illumina, AUG 27, 2024, View Source [SID1234646130]). This single test interrogates over 500 genes to profile a patient’s solid tumor, helping to increase the likelihood of identifying an immuno-oncology biomarker or clinically actionable biomarkers that enable targeted therapy options or clinical trial enrollment. TSO Comprehensive is FDA approved as a CDx to identify adult and pediatric patients with solid tumors who are positive for neurotrophic tyrosine receptor kinase (NTRK) gene fusions that may benefit from treatment with Bayer’s VITRAKVI (larotrectinib). The test is also approved to identify adult patients with locally advanced or metastatic rearranged during transfection (RET) fusion-positive non-small-cell lung cancer (NSCLC) that may benefit from treatment with Lilly’s RETEVMO (selpercatinib).

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"FDA approval for TruSight Oncology Comprehensive with accompanying companion diagnostics marks an awaited milestone for our oncology customers and community," said Everett Cunningham, chief commercial officer of Illumina. "We are committed to partnering with industry leaders like Bayer and Lilly to advance cancer diagnostics and help broaden access to precision oncology for more patients."

A CDx test may identify whether a patient’s tumor has a specific gene change or biomarker that can be targeted by a therapy, helping to determine if a patient should receive the therapy. Most CDx tests are specific to one type of cancer, but TSO Comprehensive is approved for use across solid tumor indications for the NTRK CDx, helping to maximize the chances of finding actionable information from each patient’s biopsy.

NTRK gene fusions are rare across most solid cancer tumor types (~0.1%–0.3%), and can be challenging to detect, given that these genes can fuse with different partners, many of which were previously unknown. TSO Comprehensive also interrogates RNA and thus can identify a broad range of known and novel gene fusion partners across all three NTRK gene fusions, NTRK1, NTRK2, and NTRK3. Bayer’s VITRAKVI (larotrectinib) is a highly selective TRK inhibitor approved for use in patients with TRK fusion cancer, in accordance with therapeutic labeling.

NSCLC is one of the most common types of lung cancer and the leading cause of cancer-related deaths globally. The expansive actionable biomarker landscape in NSCLC has driven the need for broad molecular profiling to enable a complete view of a patient’s disease to better guide clinical management. The oncogenic activation of RET fusion-positive NSCLC by gene fusions is a primary driver in NSCLC, occurring in up to 2% of cases. Lilly’s RETEVMO (selpercatinib) is a highly selective and potent RET kinase inhibitor in locally advanced or metastatic NSCLC. TSO Comprehensive enables broad characterization and simultaneous detection of multiple prognostic and predictive biomarkers such as RET, genomic signatures such as tumor mutational burden, and emerging biomarkers within NSCLC in a single test.

"Through research conducted globally, there is a significant body of evidence demonstrating the clinical utility of comprehensive genomic profiling for patients with advanced cancer," said Vivek Subbiah, MD, chief, Early-Phase Drug Development at Sarah Cannon Research Institute. "Illumina’s newest distributable IVD kit for comprehensive genomic profiling and accompanying CDx enable another valuable clinical tool for the oncology community to match patients with targeted therapies that can vastly improve their journey and outcomes."

TSO Comprehensive will begin shipping to customers this year. Comprehensive genomic profiling assays with CDx claims for solid tumors, like TSO Comprehensive, are reimbursable under a Centers for Medicare & Medicaid Service national coverage determination.

Illumina has a growing pipeline of CDx claims under development through partnerships with pharmaceutical companies, which will be added to TSO Comprehensive following appropriate regulatory approvals. These CDx claims will help unlock groundbreaking targeted therapies and immunotherapies to make a difference in the lives of patients with cancer.

A separate CE-marked version of TSO Comprehensive is already available in Europe, launched in 2022. To learn more about TruSight Oncology Comprehensive.About TruSight Oncology Comprehensive

TruSight Oncology Comprehensive is a qualitative in vitro diagnostic test that uses targeted next-generation sequencing to detect variants in 517 genes using nucleic acids extracted from formalin-fixed, paraffin-embedded (FFPE) tumor tissue samples from cancer patients with solid malignant neoplasms using the Illumina NextSeq 550Dx Instrument. The test can be used to detect single nucleotide variants, multi-nucleotide variants, insertions, and deletions from DNA, and fusions in 24 genes and splice variants in one gene from RNA. The test also reports a Tumor Mutational Burden (TMB) score.

The test is intended to be used as a companion diagnostic to identify cancer patients who may benefit from treatment with the targeted therapies listed in Table 1, in accordance with the approved therapeutic product labeling.

In addition, the test is intended to provide tumor profiling information for use by qualified health care professionals in accordance with professional guidelines in oncology for patients with solid malignant neoplasms. Genomic findings other than those listed in Table 1 of the intended use statement are not conclusive or prescriptive for labeled use of any specific therapeutic product.

Table 1: Companion diagnostic indications

Tumor Type

Biomarker(s) Detected

Therapy

Solid Tumors

NTRK1/2/3 fusions

VITRAKVI (larotrectinib)

Non-Small Cell Lung Cancer

RET fusions

RETEVMO (selpercatinib)

On August 27, 2024 Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) reported that it has submitted a New Drug Application (NDA) to the United States (U.S.) Food and Drug Administration (FDA) for TLX101-CDx, (Pixclara[1], 18F-floretyrosine or 18F-FET), an investigational PET[2] agent for the characterisation of progressive or recurrent glioma (brain cancer) from treatment related changes in both adult and pediatric patients (Press release, Telix Pharmaceuticals, AUG 27, 2024, View Source [SID1234646129]).

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Given the potential to address significant unmet medical need, Pixclara1 has been granted Orphan Drug[3] and Fast Track[4] designation by the FDA, which facilitates expedited review and closer consultation with the agency during the review process. FET PET (Pixclara1) is already included in international clinical practice guidelines for the imaging of gliomas[5], however there is currently no FDA-approved targeted amino acid PET agent for adult and pediatric brain cancer imaging commercially available in the U.S.

There is a critical unmet need to improve the diagnosis and management of glioma, particularly in the post-treatment setting. With low survival rates and the need to make rapid decisions, precision imaging is paramount. Subject to regulatory approval, Pixclara1 has the potential to address this need, enabling patients to receive greater clarity in their diagnosis and treatment decision making. Pixclara1 is also being developed as the "companion" theranostic imaging agent for TLX101, Telix’s investigational neuro-oncology drug candidate, which targets the same amino acid transporter mechanism with therapeutic targeted radiation.

Kevin Richardson, Chief Executive Officer, Telix Precision Medicine, stated, "Gliomas are the most common primary brain tumours of the central nervous system. Conventional imaging with MRI[6] often yields inconclusive results in characterising recurrent disease and therefore delays time-sensitive decision making4. Limitations of conventional imaging techniques include the lack of biological specificity, dependency on blood-brain barrier disruption, and an inherent inability to differentiate between tumour progression or treatment-related causes[7]. Telix’s filing of this NDA for Pixclara1 is an important milestone, reflecting our commitment to improved and accessible neuro-oncology imaging in the U.S., and taking us one step closer to commercial availability in 2025, subject to FDA approval."

About TLX101-CDx

TLX101-CDx (Pixclara1) is a PET imaging agent, which has been granted fast track and orphan drug designations by the FDA as an imaging agent for the characterisation of glioma. TLX101-CDx targets membrane transport proteins known as LAT1 and LAT2[8]. This enables TLX101-CDx to be potentially utilised as a companion diagnostic agent to TLX101 (4-L-[131I] iodo-phenylalanine, or 131I-IPA), Telix’s LAT1-targeting investigational glioblastoma (GBM) therapy, currently under investigation in the IPAX-2[9] and IPAX-Linz[10] studies.

About gliomas in the U.S.

Gliomas are very diffusely infiltrative tumours that affect the surrounding brain tissue. They are the most common form of central nervous system (CNS) neoplasm that originates from glial cells, accounting for approximately 30% of all brain and CNS tumours and 80% of all malignant brain tumours[11]. In the U.S., there are six cases of gliomas diagnosed per 100,000 people every year. GBM is a high-grade glioma and the most common and aggressive form of primary brain cancer, with approximately 22,000 new cases diagnosed annually in the U.S.[12]. The mainstay of treatment for GBM comprises surgical resection, followed by combined radiotherapy and chemotherapy. Despite such treatment, recurrence occurs in almost all patients[13], with an expected survival duration of 12-15 months from diagnosis.

On August 27, 2024 argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, reported that members of the management team will participate in the investor conferences in September (Press release, argenx, AUG 27, 2024, View Source [SID1234646127]).

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2024 Wells Fargo Healthcare Conference. Fireside chat on Wednesday, September 4, 2024 at 1:30 p.m. ET in Boston, MA.

Morgan Stanley 22nd Annual Global Healthcare Conference. Fireside chat on Thursday, September 5, 2024 at 4:50 p.m. ET in New York, NY.

Baird 2024 Global Healthcare Conference. Fireside chat on Tuesday, September 10, 2024 at 9:40 a.m. ET in New York, NY.

Additional information regarding these events will be available on the Investors section of the argenx website at argenx.com/investors.

On August 27, 2024 Rezolute, Inc. (Nasdaq: RZLT) ("Rezolute" or the "Company"), a late-stage rare disease company focused on significantly improving outcomes for individuals with hypoglycemia caused by hyperinsulinism (HI), reported that management will participate in the following investor conferences (Press release, Rezolute, AUG 27, 2024, View Source [SID1234646126]):

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Event: H.C. Wainwright 26th Annual Global Investment Conference
Date: September 9-11, 2024

Event: Cantor Global Healthcare Conference
Date: September 17-19, 2024

Management will be participating in one-on-one investor meetings throughout the conferences. Investors interested in scheduling a meeting with the Rezolute management team should contact their H.C. Wainwright and Cantor representatives.

On August 27, 2024 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported a poster presentation at the Society of Hematologic Oncology (SOHO) Annual Meeting being held September 4-7, 2024 in Houston, Texas (Press release, Autolus, AUG 27, 2024, View Source [SID1234646125]). These data demonstrate the rationale for tumor burden (TB)-guided dosing in adult patients with Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia (r/r B-ALL) by analyzing the impact of bone marrow (BM) blast percentage prior to lymphodepletion in patients treated with obe-cel in the FELIX Phase 1b/2 study.

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"Unlike other CD19 CAR T-cell therapies, obe-cel is administered to patients as two infusions using a tumor burden-guided dosing schedule," said Dr. Christian Itin, Chief Executive Officer of Autolus. "The data demonstrate the importance of administering both split doses of obe-cel to patients with r/r B-ALL and highlight the differentiation of obe-cel based on its unique binding properties and tumor burden-guided dosing approach."

Poster presentation:
Title: Obecabtagene autoleucel (obe-cel) for Relapsed/Refractory Adult B-Cell Acute Lymphoblastic Leukemia (r/r B-ALL): Impact of Chimeric Antigen Receptor T-Cell (CAR T) and Tumor Burden-Guided Dosing in the FELIX Phase 1b/2 Study

Session date and time: Wednesday, September 4, 6:15 pm
Session room: Hall B3
Poster Number: ALL-502
Presenting Author: Dr. Elias Jabbour, Professor, Department of Leukemia, Division of Cancer Medicine, MD Anderson Cancer Center, Houston, TX

Summary:
Tumor burden (TB) is a recognized driver of immunotoxicity from CAR T therapy for B-ALL. Obe-cel is a novel autologous CAR T with a differentiated fast off-rate CD19 binding domain and a 4-1BB co-stimulatory domain, designed to improve CAR T persistence and immunotoxicity. Obe-cel is an investigational therapy being evaluated in adult r/r B-ALL in the FELIX Phase Ib/II study (NCT04404660), utilizing TB-guided dosing based on bone marrow (BM) blast percentage prior to lymphodepletion. We report outcomes by TB, demonstrating the rationale for TB-guided dosing in adult r/r B-ALL.

Of 127 patients infused with obe-cel, 120 (94%) received the planned two doses (low TB: n=48 [40%]; high TB: n=72 [60%]). Overall, high CAR T expansion was observed, which progressively increased with TB; a TB increase of 50% was associated with a 1.754-fold increase in Cmax and a 2.068-fold increase in AUC0–28days. In both groups, peak expansion was reached after Dose 2 (low/high TB: Day 15 [6–55]/Day 11 [2–28]), demonstrating the need for both doses regardless of TB. Overall remission rate was 90% and 75% in low and high TB groups, respectively.

In summary, TB-guided dosing was associated with high CAR-T cell expansion and while providing a manageable adverse event profile. This supports the TB guided-dosing approach in adult r/r B-ALL.