Biosyngen’s BRG01 enters Phase II clinical trial, a first-in-kind autologous EBV-Specific CAR-T Therapy for Solid Tumors on Recurrent/Metastatic Nasopharyngeal Carcinoma

On July 16, 2024 Biosyngen, a leading biotechnology company focused on the development of innovative cell therapies, reported that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China has approved the initiation of a pivotal Phase ll clinical trial evaluating BRG01, the company’s autologous Epstein-Barr virus (EBV) specific chimeric antigen receptor T-cell (CAR-T) therapy, for the treatment of patients with recurrent or metastatic EBV-positive nasopharyngeal carcinoma (Press release, BioSyngen, JUL 16, 2024, View Source [SID1234644898]). BRG01 is the world’s first CAR-T therapy for solid tumor to obtain clinical trial approvals from China and the U.S. and to advance to a pivotal Phase ll clinical trial, representing a significant milestone in the field of cell-based immuno-therapies for solid cancers.

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"The approval of the Phase ll clinical trial for BRG01 is a testament to the robust preclinical data and strong early clinical results observed with this innovative therapy" said Professor Zhang Li, Director of the Phase l Ward at the Sun Yat-Sen University Cancer Center and Deputy Director of the Lung Cancer Research Institute at Sun Yat-sen University, who is serving as the principal investigator for the BRG01 clinical trial. "BRG01 has the potential to be a first-in-class T-cell therapy for EBV-positive tumors, and we are confident in its ability to deliver meaningful clinical benefits to patients with this difficult-to-treat malignancy."

The Phase l clinical trial of BRG01 in China and the U.S. has completed patient enrollment in January this year. All participants have received BRG01 infusion as part of this registered clinical trial. The Phase l study has successfully completed assessments of dose-limiting toxicity (DLT) and preliminary efficacy in nine patients with advanced nasopharyngeal carcinoma who had previously been treated with at least one immune checkpoint inhibitor, such as a PD-1antibody.

Initial data from the study have demonstrated excellent safety and encouraging signs of clinical activity, with 75% of high-dose patients experiencing local tumor shrinkage and reduced metabolic activity on PET-CT scans, and some patients achieving complete remission of their tumor lesions. Additionally, BRG01 has shown potent anti-EBV activity, with significant reductions in peripheral blood EBV viral load observed following treatment.

These findings underscore BRG01’s potential in tumor therapy and highlight its dual benefits in antiviral treatment, establishing a robust foundation for future clinical applications. These results are likely pivotal in the CDE’s decision to advance BRG01 to phase II clinical trials.

BRG01 is an autologous T cell immunotherapy product that has been engineered to express chimeric receptors targeting the Epstein-Barr virus (EBV) antigen on the surface of T cells. This innovative therapy represents a new generation of CAR-T cell treatment specifically designed to target EBV. BRG01 received phase I clinical trial approval from the CDE in China in December 2022 and from the FDA in the United States in February 2023. Subsequently, it was granted orphan drug designation (ODD) and fast track designation (FTD) by the FDA in June and July 2023, respectively.

At the annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) in May, Biosyngen presented preclinical research on the therapy, and the data was published as an original study in Frontiers in lmmunology. In addition, at the Gordon Research Conference (GRC) on Nasopharyngeal Carcinoma held in Switzerland in early June this year, Biosyngen’s scientist and pipeline inventor, Chair of Translational Immuno-Oncology at the University of Cologne, Professor Renata Stripecke also presented the results of preclinical research and the latest clinical data of this therapy.

The development of cell therapy drugs for hematological malignancies has seen substantial progress, yet advancements in solid tumor treatments have lagged behind. The FDA’s accelerated approval of lovance’s tumor-infiltrating lymphocyte (TlL) therapy, lifileucel, for the treatment of advanced melanoma in February represents a significant milestone in the commercialization of solid tumor cell therapies. This milestone has bolstered confidence among companies engaged in the development of cell therapies for solid tumors. However, overcoming the challenges posed by solid tumors requires a unique and comprehensive approach from the outset. This approach must encompass considerations regarding technology, target selection, and indication, ensuring satisfaction of clinical needs and druggability of the therapeutic interventions.

As one of the most common head and neck tumors, nasopharyngeal carcinoma – an epithelial carcinoma arising from the nasopharyngeal mucosal lining, is closely related to EBV infection. According to WHO, an estimated number of 133,000 new cases of nasopharyngeal carcinoma worldwide was reported in 2020; 50% of which was diagnosed in China. South China provinces such as Guangdong and Guangxi provinces make up for more than 60% of nasopharyngeal carcinoma patient population in China. Though existing practice such as immune checkpoint inhibitor has been applied in second-line treatment of nasopharyngeal carcinoma, overall response rates were generally below 30%. In another words, more than 70% patients did not benefit from existing therapy. Therefore, it is imperative to explore new approaches to improve efficacy and satisfy unmet medical needs.

The emergence and progression of nasopharyngeal carcinoma are closely associated with Epstein-Barr virus (EBV) infection. EBV has infected about 95% of population worldwide. It has been listed as Group 1 carcinogen ("Carcinogenic to humans") by World Health Organization (WHO) and proved to be associated with a range of diseases including nasopharyngeal carcinoma, EBV-positive gastric cancers, lymphoma and lymphoproliferative diseases. Research indicates that tumor cells in nasopharyngeal carcinoma frequently exhibit EBV antigen expression, and EBV-positive tumor cells display target proteins for CD4+T cells and CD8+T cells, facilitating the infiltration of EBV-targeting CAR T cells into tumor tissue to exert a cytotoxic effect.

Leveraging this specific EBV target, CAR T-cell therapy developed by Biosyngen is also being explored for treating EBV-positive lymphoma. In April 2023, BRG01 obtained approval from the Drug Evaluation Center (CDE) and the Food and Drug Administration (FDA) for clinical trials in this new indication, with Phase I clinical studies currently ongoing.

Biosyngen has evolved into a biotechnology company specializing in three major cell therapies for solid tumors and hematologic malignancies, including CAR-T, TCR-T, and TIL. The company’s therapies utilizing these strategies have all been granted approvals for clinical trials, with its TCR-T and TIL therapies targeting various solid tumors such as lung and liver cancer.

"The approval of BRG01 for a pivotal Phase ll clinical trial is a major milestone for Biosyngen and underscores our commitment to developing innovative cell therapies to address significant unmet needs in solid tumors," said Dr. Michelle Chen, Co-Founder and CEO of Biosyngen. "We plan to continue investing in research and development to expedite the clinical progress and market availability of BRG01, offering more effective treatment options for patients worldwide."

With Biosyngen’s efficient operations and rapid research outcomes, there is optimism for significant advancements in solid tumor cell therapies within a shorter timeframe, potentially bringing new treatment possibilities and hope to patients.

FDA Grants Orphan Drug Designation to 7MW3711

On July 16, 2024 Mabwell (688062.SH), an innovation-driven biopharmaceutical company with entire industry chain, reported that its self-developed novel B7-H3-targeting ADC (R&D code: 7MW3711) has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA), for the treatment of small cell lung cancer (Press release, Mabwell Biotech, JUL 16, 2024, View Source [SID1234644897]).

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The FDA grants Orphan Drug Designation to treatments for rare diseases in the United States that affect fewer than 200,000 patients. Orphan Drug Designation offers policy benefits to drug developers, including as aid with medication development, tax credits for a part of clinical trial expenditures, and seven years of market exclusivity upon approval.

7MW3711 is a novel B7-H3-targeting ADC developed by Mabwell’s IDDC platform. It is composed of innovative antibody molecule, novel linker, and novel payload Mtoxin (TOP1i). When 7MW3711 enters human body, it specifically binds to antigens on the tumor cell membrane surface, be internalized and trafficked to the lysosome, release cytotoxic drug, and induce the apoptosis of tumor cells.

7MW3711 is pharmaceutical characterized as stable structure, homogeneous composition, high purity, and it is suitable for industrial scale-up. Compared with ADCs in the same class at home and abroad, 7MW3711 has shown better tumor killing effects in multiple animal tumor models. In the safety evaluation model of animals including cynomolgus monkeys, the on-target and off-target toxicities of 7MW3711 are effectively controlled, showing its good safety profile and pharmacokinetic properties. The above research results indicate that 7MW3711 has clinical differentiation characteristics and a promising future of clinical development.

About the next generation ADC platform – IDDC

IDDC is a next generation ADC site-specific conjugation technology platform independently developed by Mabwell, consisting of multiple systematic core patented technologies including the site-specific conjugation process DARfinity, the site-specific linker IDconnect, the novel payload Mtoxin, and the conditional release structure LysOnly. The next generation ADCs developed based on the above systematic patented technologies have better structural uniformity, quality stability, efficacy, and tolerability. The novel payload Mtoxin (MF6) demonstrates good pharmacodynamics, bystander killing efficacy, and anti-multidrug resistance.

The IDDC platform has been validated in multiple drug candidates under development. Mabwell currently has several ADCs in clinical development stages. Among them, the novel Nectin-4-targeting ADC, 9MW2821, is in Phase III clinical trial for the indication of urothelial carcinoma. The novel B7-H3-targeting ADC, 7MW3711, and Trop-2-targeting ADC, 9MW2921, are both in clinical trial stages.

Full-Life Technologies, SK Biopharmaceuticals Enter Licensing Agreement for Novel Therapeutic Targeting Multiple Solid Tumors

On July 16, 2024 Full-Life Technologies ("Full-Life"), a fully integrated global radiotherapeutics company, reported that it has entered into a license agreement with SK Biopharmaceuticals, a global biotech company, for exclusive worldwide clinical research, development, manufacturing, and commercialization rights to Full-Life’s "FL-091" radiopharmaceutical compound targeting neurotensin receptor 1 (NTSR1) positive cancers (Press release, Full-Life Technologies, JUL 16, 2024, View Source [SID1234644896]).

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This licensing deal worth $571.5 million includes an upfront payment, and development and commercial milestones, separate from royalties. Under the terms of the agreement, SK Biopharmaceuticals will in-license the NTSR1-targeting Radionuclide Drug Conjugate (RDC) program FL-091 – as well as its back-up compounds – aimed at developing and commercializing it as an innovative anti-cancer drug.

FL-091 is a small-molecule radioligand vector designed to deliver targeted radiation therapy to cancer cells by binding specifically to NTSR1, a receptor protein, which is selectively overexpressed in various types of solid tumors, including colorectal cancer, prostate cancer, and pancreatic cancer.

SK Biopharmaceuticals also has a right of first negotiation to license other pre-selected RDC programs of Full-Life.

Lanny Sun, Chief Executive Officer of Full-Life, said, "This agreement with SK Biopharmaceuticals highlights the potential of FL-091 in advancing cancer treatment and demonstrates SK Biopharmaceuticals’ unwavering commitment to building an oncology business around medical innovation. We look forward to future collaborations with SK Biopharmaceuticals, and to leveraging its expertise and resources to advance radiopharmaceutical therapy. The agreement is aligned with our strategic vision of fostering global partnerships and making a meaningful impact on patients worldwide."

Donghoon Lee, Chief Executive Officer and President of SK Biopharmaceuticals, said, "The licensing agreement with Full-Life not only brings the two companies closer together for future collaborations in the fastest rising biotech sector, but also most importantly, pushes SK Biopharmaceuticals forward to become a ‘Big Biotech’. Since the introduction of the company’s strategy roadmap to venture into radiopharmaceuticals last year, we have been on track toward our envisioned goal. We expect to further unveil and implement business plans for RPT (radiopharmaceutical therapy) this year, and actively pursue clinical development and commercialization in the near future to provide treatment options and create new value worldwide."

About FL-091

FL-091 is a novel small-molecule radioligand vector targeting NTSR1 positive solid tumors. Overexpression of NTSR1 has been associated with disease progression of multiple types of cancers, including colorectal, breast, pancreatic, and head and neck cancers. FL-091 radioligands have demonstrated favorable biodistribution profiles and enhanced binding affinity to NTSR1, as well as encouraging anti-tumor activities in preclinical studies. The development of the alpha-emitter therapy candidate 225Ac-FL-091 targeting NTSR1-positive tumors is currently in progress.

Beyond Air® To Participate in the BTIG Virtual Biotechnology Conference 2024

On July 16, 2024 Beyond Air, Inc. (NASDAQ: XAIR) ("Beyond Air" or the "Company") a commercial stage medical device and biopharmaceutical company focused on harnessing the power of endogenous and exogenous nitric oxide (NO) to improve the lives of patients suffering from respiratory illnesses, neurological disorders and solid tumors (through its affiliate Beyond Cancer, Ltd. ("Beyond Cancer")), reported that Steve Lisi, Chairman and Chief Executive Officer of Beyond Air, will participate in one-on-one meetings at the BTIG Virtual Biotechnology Conference being held August 5-6, 2024 (Press release, Beyond Air, JUL 16, 2024, View Source [SID1234644895]).

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If you are interested in meeting with the Beyond Air team during the conference, please contact your BTIG representative.

SELLAS Announces U.S. FDA Rare Pediatric Disease Designation (RPDD) Granted to SLS009 for the Treatment of Pediatric Acute Myeloid Leukemia

On July 16, 2024 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to SLS009, a highly selective CDK9 inhibitor, for the treatment of pediatric acute myeloid leukemia (AML) (Press release, Sellas Life Sciences, JUL 16, 2024, View Source [SID1234644894]). The FDA previously granted Orphan Drug and Fast Track Designations to SLS009 for the treatment of AML.

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"Receiving our second Rare Pediatric Disease Designation, following pediatric acute lymphoblastic leukemia last month, is another acknowledgment of SLS009’s novel transformational treatment potential to improve the lives of patients, including children with AML," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "This designation reinforces our dedication to addressing the urgent needs of children with AML, including those with treatment-resistant mutations, highlighting the promise of SLS009 to offer the specialized care and support they require, especially considering the limited treatment options for rare pediatric diseases. We look forward to continued SLS009 development and enrolling pediatric AML patients in our Phase 2 clinical trial."

AML prognosis with currently available treatments in the refractory and/or relapsed pediatric patient population remains poor. In a representative study, the 5-year overall survival (OS) rate in relapsed pediatric AML was 33% for all patients, and in patients whose remission lasted less than 12 months only 15.7%. In patients who did not achieve complete remission after one course of chemotherapy 5-year overall survival was 0%. About 50% of children with pediatric AML relapse. Generally, the only therapy considered curative in relapsed and refractory patients is a bone marrow transplant and the primary goal of chemotherapy is to achieve remission so that pediatric patients can be transplanted.

Rare Pediatric Disease (RPD) Designation is granted by the FDA for serious or life-threatening diseases that affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If, in the future, a New Drug Application (NDA) for SLS009 for the treatment of pediatric AML is approved by the FDA, SELLAS might be eligible to receive a Priority Review Voucher (PRV) that could be redeemed to receive a priority review for any subsequent marketing application. PRVs may be used by the sponsor or sold to another sponsor for their use and have recently sold for approximately $100 million.