On December 5, 2024 Sysmex Corporation (HQ: Kobe, Japan; President: Kaoru Asano) and Japan Tissue Engineering Co., Ltd. (HQ: Gamagori, Aichi Prefecture; President & CEO: Ken-ichiro Hata; hereinafter "J-TEC") reported that they signed a basic agreement ("the Agreement") on December 3, 2024, with the aim of advancing (i.e., mechanizing and automating) manufacturing capabilities for regenerative medical products by utilizing innovative technologies, in order to accelerate the industrialization of regenerative medicine and cell therapy and to enhance sustainability (Press release, Sysmex, DEC 5, 2024, View Source [SID1234648830]). Going forward, both companies will start concrete strategic collaboration based on this Agreement.
One of the major challenges in the industrialization of regenerative medicine and cell therapy is the difficulty of manufacturing products from living cells. In particular, autologous cell products1 derived from patients’ own cells need to be manufactured to accommodate the non-uniform character of the cells from each patient. As a result, it is extremely challenging to mechanize and automate the manufacturing processes in a standardized manner. This creates a bottleneck in expanding the scale of production and enhancing efficiency in the regenerative medicine and cell therapy industry, and the transfer of manual manufacturing techniques therefore becomes an issue that directly affects the sustainability of the business.

Sysmex has contributed to the evaluation of cell functions by providing quality control testing2 capable of non-destructively analyzing cell characteristics, as well as Internet of Things (IoT) and other robotic technologies, to academic institutions and pharmaceutical companies that develop regenerative medical products. The company has also been working on automating manufacturing processes for the pipeline of our group companies and strategic partners.

J-TEC was the first company in Japan to develop and obtain approval of regenerative medical products, making it a pioneer in regenerative medicine. In particular, it has been working to commercialize and utilize autologous cell products in society. Based on the experience and knowledge gained in this process, the company has established a platform to stably supply products while maintaining high quality.

Both companies have been discussing ways to leverage their individual strengths to solve issues in the regenerative medicine and cell therapy industry, based on the technology and know-how they have developed through their respective business activities. As part of this, a basic agreement was signed on December 3, 2024, with the aim of enhancing manufacturing capabilities for regenerative medicine and cell therapy through innovative technologies.

Through open innovation based on the strengths of both companies, they will contribute to the sustainable development of Japan’s regenerative medicine and cell therapy industry by realizing the mechanization and automation of manufacturing processes for regenerative medical products, which is a key issue in the regenerative medicine and cell therapy industry.
Terminology
1
Autologous cell products:
Regenerative medical products manufactured from patients’ own living cells.

2
Quality testing that allows non-destructive cell analysis:
Testing that enables the analysis of cell quality without destroying the cells themselves. Since the cells do not need to be destroyed, it allows for the efficient and accurate testing of cell products in a short period of time.

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On December 5, 2024 Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage biotechnology company focused on developing innovative treatments for patients with unmet medical needs, reported the first patient ever dosed with MNPR-101-Lu (Press release, Monopar Therapeutics, DEC 5, 2024, View Source [SID1234648854]). This novel therapeutic radiopharmaceutical combines MNPR-101, Monopar’s antibody that selectively targets the urokinase plasminogen activator receptor (uPAR), with the therapeutic radioisotope lutetium-177. uPAR is involved in tumor growth and metastasis, and is found in some of the most aggressive, deadly cancers, including pancreatic, ovarian, triple negative breast, and colorectal cancers.

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The MNPR-101-Lu intravenous infusion was well-tolerated with no serious adverse reactions reported. This patient, dosed under a compassionate use protocol in the US, has metastatic pancreatic cancer, and prior to dosing, the cancer was imaged using MNPR-101-Zr (a zirconium-89 imaging radioisotope conjugated to MNPR-101) with a PET/CT scanner and showed uPAR expression.

"As a result of encouraging biodistribution and dosimetry clinical data we recently reported (link) with our radiodiagnostic, MNPR-101-Zr, we have been eagerly looking forward to initiating treatment of patients with MNPR-101-Lu, hopeful it may provide an important therapeutic benefit to a group of cancer patients very much in need," said Chandler Robinson, MD, Monopar’s Chief Executive Officer.

"We are thrilled to have dosed this patient with MNPR-101-Lu, and believe this may be the world’s first dosing of a patient with a uPAR-targeted therapeutic radiopharmaceutical," said Andrew Cittadine, Monopar’s Chief Operating Officer.

Monopar is actively enrolling participants in two Phase 1 clinical studies in Australia, evaluating MNPR-101-Zr for imaging and MNPR-101-Lu for treatment of advanced solid tumors. Further information about the MNPR-101-Lu Phase 1a trial is available at www.ClinicalTrials.gov under study identifier NCT06617169. Further information about the MNPR-101-Zr Phase 1 imaging and dosimetry clinical trial is available at www.ClinicalTrials.gov under study identifier NCT06337084.

On December 5, 2024 Duality Biologics ("DualityBio") reported first data from a global Phase 1/2a clinical trial (NCT05914116, CTR20232835) evaluating BNT324/DB-1311, an investigational next-generation antibody-drug conjugate ("ADC") targeting the transmembrane glycoprotein B7-H3 (Press release, DualityBio, DEC 5, 2024, View Source [SID1234648840]). The data were presented in an oral session at the 2024 European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Asia Annual Meeting ("ESMO Asia") in Singapore and showed encouraging antitumor activity alongside a manageable safety profile in heavily pretreated patients with locally advanced or metastatic solid tumors. BNT324/DB-1311 is being co-developed by BioNTech SE (Nasdaq: BNTX, "BioNTech") and DualityBio.

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The analysis of the ongoing Phase 1/2a trial included 277 participants across various solid tumor types including small cell lung cancer ("SCLC"), non-small cell lung cancer ("NSCLC"), castration-resistant prostate cancer ("CRPC"), and squamous cell carcinoma of the head and neck ("SCCHN"). About 75% of participants had an Eastern Cooperative Oncology Group ("ECOG") performance status of 1, and approximately 61% had undergone two or more lines of therapy. The primary endpoints of the trial are safety and objective response rate ("ORR") as determined by investigator. The secondary endpoints include duration of response ("DoR"), disease control rate ("DCR"), progression-free survival ("PFS"), overall survival ("OS") among others. The data showed the following results:

Among all evaluable patients with at least one post-baseline tumor assessment (n=238), the overall uORR was 32.4%, and the DCR was 82.4%.
Among patients with SCLC (n=73), the uORR was 56.2%, and the DCR was 89.0%. The majority of patients with SCLC received 6 mg/kg and 9 mg/kg of BNT324/DB-1311, with no difference in uORR between the two dose groups (54.5% and 58.8%, respectively). Notably, at the 9 mg/kg dose level, the uORR in patients with SCLC who had prior immunotherapy but no treatment with topoisomerase I inhibitors reached 70.4%.
Most patients with NSCLC had non-squamous histology (n=41), exhibiting an uORR of 22.0%, while patients with squamous NSCLC (n=25) had an uORR of 16.0%.
Among patients with CRPC (n=32), BNT324/DB-1311 demonstrated early antitumor activity with an uORR of 28.0% and a DCR of 92.0%. With a median rPFS of 7.2 months, the rPFS data were not mature at the time of the analysis. The 6-month rPFS rate was 94.7%.
In other tumor types, including cervical cancer (n=4), hepatocellular carcinoma (n=12), head and neck squamous carcinoma (n=3), and melanoma (n=11), BNT324/DB-1311 also exhibited antitumor activity with uORRs of 75.0%, 25.0%, 100.0%, and 36.4%, respectively.
BNT324/DB-1311 showed a manageable safety profile across all evaluated patients and tumor types (n=277). The most common treatment-related adverse events (TRAEs) reported included nausea, neutrophil count decreased, anemia, white blood cell count decreased, decreased appetite, and platelet count decreased.
Dr. John Zhu, Founder and CEO of Duality Biologics, said, "BNT324/DB-1311 is an innovative ADC molecule co-developed by BioNTech and DualityBio, showing clinical data in the study phase. This early data supports DualityBio’s ADC technology platform, and the continued research and development of novel ADC therapies with the aim to improve the standard of care, and embodying DualityBio’s commitment to exploring innovative treatments while advancing the global ADC industry for patient benefit."

BNT324/DB-1311 is one of three clinical stage ADC candidates in BioNTech’s and DualityBio’s global strategic partnership aimed at advancing these novel ADC assets into late-stage development in multiple high unmet medical need cancer indications. Multiple clinical trials combining selected assets from BioNTech’s and DualityBio’s strategic partnership with BNT327/PM8002, a novel investigational bispecific antibody targeting PD-L1 and VEGF-A, which is being jointly developed by BioNTech and Biotheus are planned in various solid tumor indications. A Phase 1/2 clinical trial evaluating the combination of BNT325/DB-1305, a TROP2 targeting ADC candidate, and BNT327/PM8002 is currently ongoing. A Phase 1/2 trial evaluating BNT324/DB-1311 in combination with BNT327/PM8002 in patients with SCLC or NSCLC is planned to start in 2025.

About BNT324/DB-1311

BNT324/DB-1311 is a next-generation topoisomerase-I-inhibitor-based ADC candidate targeting the immune checkpoint protein B7-H3. The transmembrane glycoprotein B7-H3 plays a critical role in the anti-tumor immune response and the shaping of the tumor microenvironment. It is overexpressed in a range of solid tumors, with limited expression in healthy tissues, and has been associated with disease progression and very poor prognosis.[i] Preclinical studies have shown that BNT324/DB-1311 exhibits antitumor activity in various solid tumor models.[ii] Preliminary data from the ongoing Phase 1/2a trial (NCT05914116) has demonstrated antitumor activity and a manageable safety profile for BNT324/DB-1311 in patients with advanced solid tumors.

In June 2024, the U.S. Food and Drug Administration ("FDA") granted Fast Track designation for BNT324/DB-1311 for the treatment of patients with advanced/unresectable, or metastatic castration-resistant prostate cancer ("CRPC"). In July 2024, the FDA granted Orphan Drug Designation to BNT324/DB-1311 for the treatment of advanced or metastatic esophageal squamous cell carcinoma.

On December 5, 2024 Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President: Toichi Takino; "Ono") reported that it has entered into a drug discovery collaboration agreement with Congruence Therapeutics (Headquarters: Montreal, Quebec, Canada; CEO: Clarissa Desjardins; "Congruence") to generate novel small molecule correctors against multiple protein targets in the oncology area by leveraging Congruence’s proprietary drug discovery platform, Revenir (Press release, Ono, DEC 5, 2024, View Source [SID1234648841]).

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Under the terms of the agreement, Congruence will generate small molecule correctors by leveraging Congruence’s proprietary drug discovery engine called, Revenir. Ono will obtain an exclusive option right to develop, manufacture and commercialize the identified small molecule correctors worldwide. Congruence will be eligible to receive an upfront payment, research expenses, milestone payments based on research and development progress and sales, as well as tiered royalties based on net sales.

"We believe that this collaboration with Congruence may help generating novel small molecule correctors for validated targets in the oncology area by leveraging their own technologies in protein dynamics and computational biology, leading to our development pipeline," said Seishi Katsumata, Corporate Officer / Executive Director, Discovery & Research of Ono. "We will be committed to delivering innovative new drugs to cancer patients as soon as possible."

"Congruence is thrilled to partner with Ono, which has established itself as a global leader in drug development, particularly in the oncology space. We believe that our Revenir platform and capabilities in protein dynamics will accelerate the discovery of novel therapies for compelling targets of interest to both companies," said Sharath Hegde PhD, Chief Scientific Officer of Congruence.

About Revenir Drug Discovery Platform

Revenir, Congruence’s proprietary computational drug discovery platform, captures the dynamic biophysical changes caused by mutations in proteins, offering unique insights into protein defects and their correction. By examining surface features and a spectrum of biophysical descriptors across an ensemble of protein conformers, Revenir predicts small molecule induced correction of the underlying defect.

MEDSIR, a leading company dedicated to advancing independent clinical research in oncology on an international level and part of Oncoclínicas & Co, the largest oncology treatment group in Latin America with significant strength in the clinical and outpatient setting, reported it will participate in the prestigious San Antonio Breast Cancer Symposium 2024 (SABCS) with the presentation of five innovative studies, including one of particular significance in advanced breast cancer: the DEMETHER study (Press release, MedSIR, DEC 5, 2024, View Source [SID1234648842]). These landmark studies demonstrate remarkable progress in the development of more personalized and less invasive therapies, offering the potential to enhance patients’ quality of life and redefine the treatment approach for advanced breast cancer.

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The DEMETHER clinical trial explores an innovative treatment strategy for previously untreated HER2-positive advanced breast cancer. The study employs a dual-phase approach, beginning with an induction phase using trastuzumab deruxtecan (T-DXd), followed by a maintenance phase with subcutaneous administration of trastuzumab and pertuzumab. This strategy aims to prolong progression-free survival, improve overall survival rates at three years, and provide enhanced safety and quality of life compared to standard treatments.

By offering an alternative to conventional chemotherapy and prolonged T-DXd administration, DEMETHER aims to redefine therapeutic de-escalation. This strategy seeks to minimize the side effects associated with traditional therapies, thereby significantly improving patients’ quality of life. Furthermore, it underscores a commitment to making advanced breast cancer treatments more accessible, less invasive, and patient centered.

23 active centers in 4 countries and several Highly Cited Researchers 2024

The trial is currently being conducted at 23 active centers across the United States, Spain, Italy and Germany. Additionally, the study benefits from the involvement of leading international researchers renowned for their excellence. Among them are several scientists featured in the Highly Cited Researchers 2024 list by Clarivate, including Dr. Javier Cortés, Dr. Hope Rugo, Dr. Nadia Harbeck, Dr. Sara M. Tolaney, and Dr. Peter Schmid, whose expertise and contributions bring exceptional value to the project.

The DEMETHER study seeks to further advance personalized medicine, exploring less invasive, safer, and potential more effective alternatives to conventional treatments. "This study reflects the collaborative efforts of international experts, leading centers, the pharmaceutical industry and MEDSIR in designing therapeutic strategies that enhance patients’ quality of life through treatment de-escalation." highlights Dr. Javier Cortés, principal investigator of the DEMETHER study.

New therapeutic options for patients with metastatic breast cancer: exploring advances in brain metastases with PHENOMENAL and TUXEDO-4

Both the PHENOMENAL and TUXEDO-4 studies presented at SABCS are particularly relevant as they address a critical and historically underserved population: patients with metastatic breast cancer (MBC) and brain metastases (BMs). BMs occur in up to 25% of patients with MBC 1, significantly impacting prognosis and quality of life.

These studies stand out by exploring innovative therapeutic strategies tailored to overcome the unique challenges of treating brain metastases. The PHENOMENAL study focuses on nanoliposomal irinotecan, Nal-IRI, to enhance drug delivery to brain tumors while reducing systemic toxicity. Meanwhile, the TUXEDO-4 study investigates the use of T-DXd, a novel antibody-drug conjugate targeting HER2-low tumors, with the goal of achieving meaningful intracranial tumor shrinkage.

An innovative therapeutic regimen with the potential to expand treatment options and improve the quality of life for patients with advanced breast cancer

The Phase III ADELA study, in collaboration with the MENARINI Group, explores new perspectives in the treatment of advanced ER+ and HER2-negative breast cancer, specifically in patients with mutations in the ESR1 gene whose cancer has progressed after receiving standard first-line of therapy. This gene produces estrogen receptor proteins that can stimulate the growth of this type of breast cancer.

ADELA has the potential, if successful, to pave the way for regulatory approval of this therapeutic combination, making it accessible to a broader patient population.

In addition to this study, MEDSIR is presenting the PRIMED trial, which evaluates the efficacy of prophylactic administration of granulocyte colony-stimulating factor (G-CSF) and loperamide during the initial treatment cycles of sacituzumab govitecan (SG) therapy to mitigate the incidence of neutropenia and diarrhea, aiming to enhance treatment tolerability and patient safety.

The presentation of these results at an event of SABCS 2024’s magnitude not only positions MEDSIR as a leader in oncology research excellence but also highlights its ability to lead transformative projects addressing unmet needs in breast cancer treatment.