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Rakuten Medical, Inc. (Rakuten Medical) and Shimadzu Corporation (Shimadzu) today announced enrollment of the first patient in the phase 2 clinical trial of ASP-1929 photoimmunotherapy with fluorescence imaging (Study number: ASP-1929-103) at the National Cancer Institute of the National Institutes of Health in the United States.
This is a phase 2, open-label, single-arm, window of opportunity study* of an antibody-dye conjugate, ASP-1929 photoimmunotherapy with fluorescence imaging in patients with operable primary or recurrent head and neck squamous cell carcinoma (HNSCC) or cutaneous squamous cell carcinoma (cuSCC) and will enroll 22 patients. This trial, conducted under Cooperative Research and Development Agreements with the National Cancer Institute, will assess the efficacy and safety of a single ASP-1929 photoimmunotherapy treatment administered prior to standard of care surgical tumor resection in patients with operable primary or recurrent HNSCC or cuSCC.  Additionally, the feasibility of the Shimadzu fluorescence imaging system for real-time monitoring and recording of fluorescence of the IRDye® 700DX (IR700) portion of ASP-1929 will also be studied. 

“Currently, we are conducting monotherapy clinical trials with ASP-1929 photoimmunotherapy and a combination therapy trial with anti-PD1 in patients with unresectable recurrent HNSCC and cuSCC. This clinical trial further expands the studied patient groups by targeting primary operable HNSCC or cuSCC. I hope the results of this trial will lead to Rakuten Medical’s photoimmunotherapy treatment reaching as many patients as possible,” said Mickey Mikitani, Chief Executive Officer of Rakuten Medical, Inc. 

“Shimadzu in partnership with Rakuten Medical, has developed a fluorescence imaging system for photoimmunotherapy treatment by using fluorescence visualization technology. In this clinical trial, we will observe and record the reaction of IR700 in real time during the treatment with the fluorescence imaging system developed by our technology to visualize the condition of the tumor. We hope that through the use of our fluorescence visualization technology, we can contribute to a future where this treatment can be delivered to as many patients as possible without delay including those with early-stage cancer,” said Teruhisa Ueda, President & CEO of Shimadzu.
Other trials being conducted by Rakuten Medical, Inc. with ASP-1929 include a global phase 3 clinical trial for recurrent HNSCC and a U.S. phase 1b/2 clinical trial for recurrent HNSCC and cuSCC using combination therapy with anti-PD1. Moreover, in September 2020, the combined drug and a laser device system received marketing approval in Japan from the Ministry of Health, Labour and Welfare for ASP-1929 photoimmunotherapy to treat unresectable locally advanced or recurrent head and neck cancer.  

*A window of opportunity study takes place in the period between a cancer diagnosis and the subsequent initiation of standard treatment, during which, the patient receives a non-standard drug or treatment of interest over a short period of time ^1.2. 

About Rakuten Medical, Inc.

Rakuten Medical, Inc. is a global clinical stage biotechnology company developing and commercializing precision, cell-targeting investigational therapies on its Illuminox^TM platform, which, in pre-clinical studies have been shown to induce rapid and selective cell killing and tumor necrosis. Outside of Japan, Illuminox therapies have not yet been approved as safe or effective by any regulatory authority. The company’s first drug developed on the Illuminox platform, ASP-1929, has received approval from the Japanese Ministry of Health, Labour, and Welfare, and is currently the subject of a global phase 3 clinical trial for recurrent head and neck cancer. Rakuten Medical is committed to its mission to conquer cancer and aims to realize a society where cancer patients can lead fulfilling lives. The company has 6 locations in 5 countries, including the United States, where it is headquartered, Japan, the Netherlands, Taiwan, and Switzerland. For more information, visit www.rakuten-med.com .

About Shimadzu Corporation

With “Contributing to Society through Science and Technology” as the corporate philosophy and “Realizing Our Wishes for the Well-being of Mankind and the Earth” as the management principle, Shimadzu Corporation operates in various fields such as testing and measuring instruments, medical systems, aircraft equipment, and industrial machinery and equipment. For the development of photoimmunotherapy, Shimadzu has also signed a separate Cooperative Research and Development Agreement with the National Cancer Institute and a joint development agreement with the National Cancer Center Hospital East to advance the development of measurement technologies for photoimmunotherapy.

About ASP-1929

Since 2013, Rakuten Medical, Inc. has been using an exclusively licensed antibody complex to develop new cancer therapies based on its IlluminoxTM technology platform. Rakuten Medical’s first pipeline drug developed using its Illuminox platform is ASP-1929, an antibody-dye conjugate comprised of the antibody cetuximab and IRDye® 700DX, a light activatable dye. ASP-1929 binds to epidermal growth factor receptors (EGFR), a cancer antigen expressed in multiple types of solid tumors, including head and neck, cutaneous, esophageal, lung, colon and pancreatic cancers. After binding to cancer cells, ASP-1929 is locally activated by non-thermal red light (690 nm) illumination emitted by a laser device system.  Pre-clinical data indicates that Illuminox technology induces a biophysical process that compromises cell membrane integrity, leading to cancer cell death and tumor necrosis. ASP-1929 received marketing approval from the Japanese Ministry of Health, Labor, and Welfare in September 2020, and is currently under investigation in a global phase 3 clinical trial for recurrent head and neck cancer. Rakuten Medical, Inc. is moving forward with product development by conducting clinical trials of monotherapy and combination therapy with other drugs. Outside of Japan, ASP-1929 and the laser device system have not yet been approved by any regulatory authority.

About Illuminox™ platform

The Illuminox platform is an investigational platform based on a cancer therapy called photoimmunotherapy, which was developed by Dr. Hisataka Kobayashi and team from the National Cancer Institute in the United States. Rakuten Medical is developing the Illuminox platform as a technology consisting of a drug, device, and other related components. The drug component of the platform consists of a targeting moiety conjugated with one or more dyes leading to selective cell surface binding. The device component consists of a light source that locally illuminates the targeted cells with non-thermal light to transiently activate the drug.  Pre-clinical data have shown that this activation elicits rapid and selective necrosis of targeted cells through a biophysical process that compromises the membrane integrity of the targeted cells. Therapies developed on Illuminox may also result in local and systemic innate and adaptive immune activation due to immunogenic cell death of the targeted cells and/or the removal of immunosuppressive elements within the microenvironment. Outside of Japan, Illuminox therapies have not yet been approved as safe or effective by any regulatory authority.

Forward Looking Statements

This press release contains forward looking statements that correspond to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements include various risks, uncertainties, and assumptions that may cause Rakuten Medical’s business plans and results to differ from the anticipated results and expectations expressed in these statements. These “forward looking statements” contain information about the status and development of our products, including ASP-1929, the Illuminox platform and IRDye® 700DX, as well as other regulatory and marketing authorization efforts, the potential benefits, efficacy, and safety of ASP-1929 and other therapies created using the Illuminox platform, and the status of regulatory filings. The approval and commercial success of such therapies may not be achieved, and the results and effects shown in pre-clinical studies and data may not be replicated in clinical trials. Such statements may include words such as “expect,” “believe,” “hope,” “estimate,” “looks as though,” “anticipate,” “intend,” “may,” “suggest,” “plan,” “strategy,” “will,” “hopeful,” “excited,” “do,” “aims,” “develop” and similar expressions.  Forward-looking statements are neither historical facts nor assurances of future performance.  Furthermore, Rakuten Medical’s partnership with Shimadzu does not infer any particular result or achievement for Rakuten Medical. Such forward-looking statements are based upon Rakuten Medical’s current beliefs, expectations, and assumptions regarding the future of its business, future plans and strategies, projections, anticipated events and trends, the economy and other future conditions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of Rakuten Medical’s control. Rakuten Medical’s actual results and financial condition may differ materially from those indicated in the forward-looking statements. Therefore, you should not rely on any of these forward-looking statements. In addition, this press release uses terms such as ”hope,” “contribute,” and “expand” to express opinions about clinical trial data. Ongoing clinical trial studies include various risks and uncertainties, in particular, problems that arise during the manufacturing stage of ASP-1929 or other product candidates, the occurrence of adverse safety events, situations in failure to demonstrate therapeutic benefits, and other various risks and uncertainties, both reasonable and unreasonable. For this reason, actual results, including regulatory approvals and uncertainties in the commercialization process of ASP-1929 or other product candidates, may differ from published information. Except to the extent required by applicable law, we undertake no obligation to publicly update this or any other forward-looking statement, whether because of new information, future developments or events, changes in assumptions, changes in the factors affecting forward-looking statements. If one or more forward-looking statement(s) is updated, no inference should be drawn that additional updates will be made to those or other forward-looking statements.

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References:

1. Aroldi F, Lord SR. Window of opportunity clinical trial designs to study cancer metabolism. Br J Cancer. 2020;122(1):45-51. doi:10.1038/s41416-019-0621-4
2. Schmitz S, Duhoux F, Machiels JP. Window of opportunity studies: Do they fulfil our expectations? Cancer Treat Rev. 2016 Feb;43:50-7. doi: 10.1016/j.ctrv.2015.12.005. Epub 2015 Dec 31. PMID: 26827692