Estonia Develops Personalized CAR-T Cell Therapy for Blood Cancer Patients

On May 27, 2024 The Tartu University Hospital, North Estonia Medical Centre, and Icosagen reported to have joined forces to develop and introduce an innovative personalized cell therapy (CAR-T cell therapy) for patients in Estonia, offering hope to those with incurable blood cancer (Press release, Icosagen Cell Factory, MAY 27, 2024, View Source [SID1234643715]).

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The Issue:

Each year, over 600 individuals in Estonia are diagnosed with blood cancer, with many succumbing within the first few years post-diagnosis.

Dr. Ain Kaare from Tartu University Hospital explains: "Significant progress has been made in cancer treatment. However, despite rapid advancements, we can only offer long-term disease-free periods to a few blood cancer patients. This means that many still experience disease progression. The new personalized CAR-T cell therapy could potentially save up to 50% of patients with certain hematological malignancies who currently have no curative options."

Dr. Annett Vapper from North Estonia Medical Centre adds: "CAR-T is a groundbreaking treatment that has revolutionized the approach to malignant blood diseases worldwide, offering a chance for recovery or longer disease-free survival for many patient groups for whom other treatments have been exhausted or proved ineffective. In the future, the potential of CAR-T technology could extend beyond blood diseases, providing therapeutic options for various oncological and rheumatological conditions based on ongoing research. This is a crucial step towards personalized medicine and more effective cancer treatment. Additionally, we can modernize treatment centers, train staff, and build medical infrastructure that enables clinical trials and enhances scientific competence."

Innovative Solution:

Tanel Mahlakõiv, PhD, from Icosagen, elaborates: "CAR-T cell therapy is an innovative treatment where a patient’s own immune cells (T-cells) are genetically modified to identify and destroy cancer cells. Unfortunately, this therapy is not yet available in Estonia due to its complexity, limited production capacity by pharmaceutical companies, and high cost. We aim to make this personalized cell therapy accessible to Estonian patients within a few years. Initially, we will focus on leukemia and lymphoma patients, followed by other types of cancer and certain autoimmune diseases."

The Plan:

The collaboration project to launch CAR-T cell therapy involves Tartu University Hospital, North Estonia Medical Centre, and Icosagen. Icosagen will develop the manufacturing processes, conduct preclinical trials, and produce therapy-grade cells meeting all quality standards. Tartu University Hospital and North Estonia Medical Centre will jointly prepare the clinical protocol and related procedures, select patients in need of treatment, collect and isolate patient cells, and conduct cell therapy. Both medical institutions will ensure treatment quality and monitor patients post-treatment through coordinated efforts.

Fresenius Announces FDA Acceptance for Review of Denosumab Biosimilar Application

On May 27, 2024 Fresenius, via its Operating Company Fresenius Kabi, reported that the U.S. Food and Drug Administration (FDA) has accepted for review the company’s Biologics License Application (BLA) for its biosimilar candidate of Prolia (denosumab) and Xgeva (denosumab) (Press release, Fresenius, MAY 27, 2024, View Source [SID1234643714]).

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The denosumab biosimilar is indicated for the treatment of osteoporosis in men and women, including glucocorticoid-induced osteoporosis, and bone loss due to prostate or breast cancer.

This BLA submission acceptance is the latest development in Fresenius Kabi’s continuing commitment to improving patient access to high-quality biological products through expanding its biosimilars development capabilities and product portfolio. Expanding the company’s Biopharma platform is a substantial cornerstone of #FutureFresenius.

Ferring Pharmaceuticals successfully launches its third Swiss Franc Bond offering for CHF 330 million

On May 27, 2024 Ferring Pharmaceuticals reported the successful launch of its third Swiss Franc Bond offering for an amount totalling CHF 330 million (Press release, Ferring, MAY 27, 2024, View Source [SID1234643713]). This senior unsecured bond transaction issued by Ferring Holding SA comprised two tranches, CHF 210 million with 5-year maturity to 28th June 2029 at a fixed coupon rate of 2.25% per annum, and CHF 120 million with 9-year maturity to 28th June 2033 at a fixed coupon rate of 2.50% per annum. The bonds will be listed on the SIX Swiss Exchange.

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The offering attracted interest from high-quality institutional investors and banks, demonstrating continued recognition of the company’s successful track record and solid cash generation from its core business. The company is rated as BBB (UBS) and Baa (Fedafin), both with a stable outlook.

Dominic Moorhead, Chief Financial Officer of Ferring Pharmaceuticals, said: "We are pleased with the successful outcome of our third bond offering and the proceeds will be used for general corporate purposes, as we continue to invest in the launch and development of our new product opportunities."

The issue was lead-managed by UBS AG and Basler Kantonalbank, with Banque Cantonale Vaudoise acting as co-manager.

Verastem Oncology Announces Updates on Avutometinib Development

On May 27, 2024 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that Verastem Oncology (Nasdaq: VSTM) issued a press release on May 24 that Verastem Oncology has initiated the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for the combination of avutometinib, a RAF/MEK inhibitor, and defactinib, a selective FAK inhibitor, for adult patients with recurrent KRAS mutant low-grade serous ovarian cancer, who received at least one prior systemic therapy (Press release, Chugai, MAY 27, 2024, View Source [SID1234643712]). Avutometinib was created by Chugai, and its clinical development is being conducted by Verastem Oncology.

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Please refer to the link below for details of the Verastem Oncology’s press release:
Verastem Oncology Announces the Initiation of a Rolling Submission of NDA to FDA Seeking Accelerated Approval of Avutometinib and Defactinib Combination for the Treatment of Adult Patients with Recurrent KRAS Mutant Low-Grade Serous Ovarian Cancer
View Source

Chugai Obtains Approval for FoundationOne Liquid CDx Cancer Genomic Profile to Provide Information on Detection of Copy Number Alterations of Cancer-Related Genes and bTMB Score

On May 27, 2024 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that it has obtained approval from the Ministry of Health, Labour and Welfare (MHLW) on May 23, 2024, for FoundationOneLiquid CDx Cancer Genomic Profile to provide information on detection of copy number alterations of cancer-related genes and blood tumor mutational burden (bTMB) score (Press release, Chugai, MAY 27, 2024, View Source [SID1234643711]). Copy number alterations and bTMB score in plasma samples are important indicators for predicting the efficacy of molecular target therapeutics such as immune checkpoint inhibitors. With this approval, our two comprehensive genomic profiling (CGP) tests using tissue and blood specimens, including FoundationOneCDx Cancer Genomic Profile, can support diagnosis and treatment decisions based on regulatory-approved information.

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"We are pleased that two important additions that support cancer treatment decisions have been approved for FoundationOne Liquid CDx Cancer Genome Profile based on blood specimens in addition to the FoundationOne CDx cancer genomic profile using tissue specimens," said Chugai’s President and CEO, Dr. Osamu Okuda. "For patients with solid tumors for whom it is difficult to collect tissue samples, cancer genome profiling tests using blood specimens provide information on predicting the effectiveness of anticancer drug treatment, allowing for smooth consideration of treatment plans. We will continue our efforts to realize advanced personalized healthcare."

As a leading company in the field of oncology, Chugai is committed to realizing advanced personalized healthcare in oncology and contributing to patients through the expansion of CGP.

About FoundationOne Liquid CDx Cancer Genomic Profile
Developed by Foundation Medicine Inc. based in Boston, USA, FoundationOne Liquid CDx Cancer Genomic Profile is a next-generation sequencing-based in vitro diagnostic device using blood samples for advanced cancer patients with solid tumors. By using circulating tumor DNA (ctDNA) in the blood of patients with advanced solid tumors, it is intended to identify genomic alterations (base substitutions, insertions/deletions, rearrangements, copy numbers alterations) in 324 cancer-related genes and calculate the bTMB score. It has been approved by the MHLW as a medical device program that has a genome profiling function for cancer-related genes and a companion diagnostic function for multiple molecular target drugs.
For the latest information about FoundationOne Liquid CDx Cancer Genomic Profile, please refer to the approval information.

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