NanOlogy Publishes Results from a Phase 1/2 Clinical Trial of Large Surface Area Microparticle Docetaxel in High-Risk Non-Muscle Invasive Bladder Cancer

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.

About NanOlogy

NanOlogy, LLC (www.nanology.us) is a private clinical-stage interventional oncology drug company formed in 2015 to improve the treatment of solid tumors based on a proprietary particle engineering technology platform for tumor-directed drug therapy to maximize drug in tumor and minimize systemic toxicity.

Disclaimers

This announcement contains forward-looking statements defined in the Private Securities Litigation Reform Act of 1995, as amended, including statements about our product development, business, and other activities. Such statements are subject to risks and uncertainties inherent in any pharmaceutical development program which may cause actual results to differ materially due to developmental, clinical, regulatory, market, competitive, technological, or other factors. All forward-looking statements are made as of the date of this announcement and the company disclaims any intent or obligation to update these statements. NanOlogy investigational drugs have not been proven safe and effective as required by U.S. FDA and have not been approved by FDA or any other jurisdiction for commercial distribution. NanOlogy is a trademark of NanOlogy LLC.

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