On August 1, 2022 Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA), an oncology-focused drug development company, reported a progress update on the GBM AGILE pivotal study (NCT03970447), a global adaptive clinical trial platform designed to evaluate multiple therapies for glioblastoma, to which paxalisib commenced enrolment in January 2021 (Press release, Kazia Therapeutics, AUG 1, 2022, View Source [SID1234617178]).

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Kazia has been advised by the Global Coalition for Adaptive Research (GCAR), the sponsor of the study, that the first stage of the paxalisib arm has completed recruitment. The treatment arm did not meet pre-defined criteria for continuing to a second stage, and patients enrolled in the first stage of the paxalisib arm will therefore continue on treatment as per protocol, and in follow-up, until completion of the final analysis, which Kazia anticipates receiving in 2H CY2023, as previously disclosed.

Given that completion of recruitment has now occurred, the study will not open to the paxalisib arm in Germany or China. Kazia will work with its licensing partner to determine the way forward in China, given that country’s general requirement for local data to register a new pharmaceutical product.

All Kazia personnel continue to be blinded to efficacy and safety data from the ongoing study, as required by regulatory authorities, and so the company remains unable to provide analysis or interpretation of the study until follow-up is complete and final data is available.

Kazia CEO, Dr James Garner, commented, "GBM AGILE was designed as an adaptive study, with the potential to follow a range of different paths to completion. Today’s news defines the remaining trajectory of the study, with modestly positive implications for both costs and timelines, and with some specific consequences for regulatory strategy in China. It does not allow us to draw any meaningful inferences about the outcomes of the study, and indeed it is critical for regulatory purposes that we remain blinded to the evolving data. We look forward to reporting final results in 2H CY2023, as currently planned. In the meantime, we are excited by some of the emerging data in diffuse intrinsic pontine glioma (DIPG) and brain metastases, which have become increasingly important areas of focus for the company and look forward to sharing more detail on those activities in due course."

On August 1, 2022 Fresenius reported that The U.S. Food and Drug Administration (FDA) has accepted for review Fresenius Kabi’s Biologics License Application (BLA) for MSB11456, a biosimilar candidate of Actemra*(tocilizumab) (Press release, Fresenius, AUG 1, 2022, View Source [SID1234617195]). This is an important achievement in the development of Fresenius Kabi’s biosimilar pipeline in the US. The BLA includes presentations for both subcutaneous (prefilled syringe and autoinjector) and intravenous administrations.

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*Actemra is a registered trademark of Chugai Seiyaku Kabushiki Kaisha Corp., a member of the Roche Group.

On August 1, 2022 NanOlogy LLC, a clinical-stage interventional oncology drug company, reported that results from a clinical trial of Large Surface Area Microparticle Docetaxel (LSAM-DTX) in High-Risk Non-Muscle Invasive Bladder Cancer (HR-NMIBC) have been published in The Journal of Urology (Press release, NanOlogy, AUG 1, 2022, View Source [SID1234617211]).

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The research article entitled Phase 1/2 Trial Results of a Large Surface Area Microparticle Docetaxel for the Treatment of High-Risk Non-Muscle Invasive Bladder Cancer presents safety and response data from the multi-site study (NCT03636256). Clinical investigators included Max Kates, MD (Johns Hopkins Medical Institutions), Ahmed Mansour, MD (UT Health San Antonio), Donald Lamm, MD (BCG Oncology), and Neal Shore, MD (Carolina Urologic Research Institute).

The trial followed an open-label 3+3 dose-escalation design with enrollment expansion at the highest dose. After transurethral resection of the bladder tumor (TURBT), subjects received direct-injection LSAM-DTX into the resection site and intravesical LSAM-DTX, followed by six-week induction and three-week maintenance intravesical LSAM-DTX courses. The first-in-human trial was initially designed as a 7-month study with treatment limited to 4 months to establish safety but later amended to follow patients to 12 months despite the treatment limitation. Tumor recurrence was evaluated by cytology, cystoscopy, or biopsy. Pharmacokinetic analysis of blood and multiplex immunofluorescence analysis of tumor microenvironment were conducted pre/post treatment.

Nineteen subjects were enrolled, 14 with prior bacillus Calmette-Guérin (BCG) exposure, and 16 with one or more prior TURBTs. Direct-injection and intravesical LSAM-DTX were well-tolerated with minor recorded treatment-related local and systemic adverse events. Median recurrence free survival was 12.2 months in the high-dose and expansion cohorts and was significantly increased compared to the low dose cohorts at 5.4 months. Bladder biopsies show an increase in tumor microenvironment immunogenicity, including increases in adaptive (T cells) and innate (NK cells) effector cells. Notably, immune checkpoint receptor expression was increased across multiple cell types, suggesting LSAM-DTX in combination with immune checkpoint inhibitor therapy may provide additional benefit in treatment of HR-NMIBC.

Worldwide, an estimated 573,000 people were diagnosed with bladder cancer in 2020 and 81,000 people will be diagnosed with bladder cancer in the United States in 2022. About 40% of patients are at higher risk for disease progression at time of diagnosis (GLOBOCAN 2020; SEER). Recurrence rates are high in these patients with currently available treatments, potentially leading to disease progression and cystectomy. Bladder cancer has among the highest life-time treatment costs of all cancers and the negative impact to patient quality of life in patients facing cystectomy is severe. A significant need exists for better drug therapies to stave off disease progression.

"Bladder cancer at high risk for progression needs better treatment options," said Max Kates, MD, Director, Bladder Cancer Program and Associate Professor of Urology of the Brady Urological Institute at Johns Hopkins Medicine. "In this Phase 1/2 clinical trial, LSAM-DTX showed promising signs of preventing disease progression and interesting immunogenic effects with minimal adverse events. Further clinical research is warranted to confirm these findings."

NanOlogy is planning further clinical development of LSAM-DTX, which it believes has therapeutic potential in NMIBC and muscle invasive bladder cancer, which also showed promising results in a separate arm of the trial and will be reported once finalized.

In addition to LSAM-DTX, NanOlogy clinical programs have advanced tumor-directed investigational drugs in pancreas, lung, peritoneal, ovarian, prostate, and dermal cancers.

The NanOlogy therapeutic platform is based on a proprietary supercritical precipitation technology that converts active ingredients into stable large surface area microparticles (LSAMs) of pure drug optimized for tumor-directed therapy and continuous drug release to maximize drug delivered to the tumor and minimize systemic toxicity.

Taxane particles are covered by composition of matter patents issued in the US (US 9,814,685, US 10,507,195, US 10,993,927, and US 11,123,322), Canada, Europe, Japan, China, Hong Kong, South Korea, Australia, Indonesia, and Russia valid through June 2036. The composition patents form the foundation of an extensive intellectual property portfolio protecting NanOlogy investigational drugs, formulations, methods, and technology.

On August 1, 2022 Gracell Biotechnologies Inc. ("Gracell" or the "Company",NASDAQ: GRCL), a global clinical-stage biopharmaceutical company dedicated to developing highly efficacious and affordable cell therapies for the treatment of cancer, reported the appointment of Wendy Li, M.D., as its Chief Medical Officer (CMO) (Press release, Gracell Biotechnologies, AUG 1, 2022, View Source [SID1234617227]). In this role, Dr. Li will oversee Gracell’s clinical development activities, including the advancement of its pipeline of autologous and allogeneic product candidates across the Company’s multiple proprietary technology platforms.

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Dr. Li brings to Gracell over 20 years of experience leading all critical aspects of clinical and medical operations at early-stage and large pharmaceutical organizations in the U.S. and China. Her expertise spans both clinical development and medical affairs, including leading early- and late-stage clinical trials for several therapeutic candidates for the treatment of hematological malignancies and solid tumors, and overseeing over 30 successful Investigational New Drug applications (INDs), New Drug Applications (NDAs) and Biologics License Applications (BLAs) that led to several multi-billion-dollar blockbuster drugs.

"Dr. Li is a distinguished and enthusiastic clinical leader and shares our commitment to discovering and developing breakthrough cell therapies. We are excited to add her experience to Gracell’s global leadership team," said Dr. William (Wei) Cao, Founder, Chairman and CEO of Gracell. "Dr. Li’s decades of experience in leading clinical development and strategy across many types of cancers and treatment modalities, as well as expertise in working with regulatory agencies, will be invaluable as we continue to advance our pipeline of breakthrough cell therapies and approach the filing of our U.S. IND application for GC012F later this year."

Prior to joining Gracell, Dr. Li served as CMO of EXUMA Biotech, where she provided strategic medical and clinical leadership for the advancement of its cell therapy pipeline in the U.S. and Asia. She has also held clinical development and medical affairs leadership positions at Pfizer, Sanofi, Genentech, and Sihuan Pharmaceutical. Dr. Li holds an M.D. from Sun Yat-Sen University of Medical Sciences.

"With years of experience in CAR-T, I recognize this therapy’s potential to fulfill patients’ unmet needs and am eager to join a company with a pipeline as promising as Gracell’s," Dr. Li said. "I believe the company’s FasTCAR and TruUCAR platforms have vast potential to solve some of the greatest challenges facing CAR-T therapy. The lead candidate, GC012F, is a highly differentiated CAR-T therapy that potentially affords competitive efficacy, combined with a favorable safety profile and faster delivery to patients. I look forward to joining Gracell’s leadership team and bringing the company to its next stage of growth."

On August 1, 2022 GluBio Therapeutics, a biotech company focused on developing novel targeted protein degradation (TPD) drugs, reported the completion of Series A+ financing of $22 million (Press release, GluBio Therapeutics, AUG 1, 2022, View Source [SID1234638030]). This round is led by Qiming Venture Partners and joined by Lilly Asia Ventures and Kaitai Capital, bringing the total capital raised to nearly $90 million since its establishment in March, 2021.

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The investment will help advance two molecular glue degraders with best-in-class potential into the clinic for hematological malignancies, accelerate the lead optimization entry of three first-in-class TPD drugs for solid tumors and inflammatory diseases, and further upgrade GluBio’s proprietary TPD discovery platform and screening capabilities tailored for rapid discovery and optimization of small molecule protein degraders for ‘undruggable’ therapeutic targets.

"We are thrilled to advance molecular-glue and heterobifunctional protein degraders, which hold a great promise of eliminating disease-causing proteins that are not amenable to traditional therapeutic approaches," said Gang Lu, Ph.D., GluBio Founder and CEO. "In the past 16 months, GluBio made tremendous progress in developing best-in-class and/or first-in-class TPD assets and state-of-the-art TPD platform, fully maximizing the therapeutic and commercial potential of TPD technologies. We completed this round within two months of Series A financing. We are grateful for the unwavering confidence investors have in our assets and platform, and confident that this round will accelerate a smooth and successful execution of our pipeline and platform."

Dr. Kan Chen, Partner of Qiming Venture Parners, said, "Targeted protein degradation is a revolutionary technology in the biopharmaceutical field. GluBio team has extensive experience in targeted protein degradation and has built a strong platform for molecular glue and new target discovery technologies, making it a promising leader in TPD field. Qiming Venture Partners is pleased to continue our support to the company’s development and hopes that GluBio will accelerate its R&D and industrialization process to bring more unmet need targets to the clinical stage and new treatment options to patients worldwide."