Rakuten Medical Receives Conditional Early Approval Designation from Japanese Health Ministry for its Lead Drug, ASP-1929, to Treat Recurrent Head & Neck Cancer

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Jun 29, 2020, 09:00 ET

SAN MATEO, Calif., June 29, 2020 /PRNewswire/ -- Rakuten Medical, Inc. (Rakuten Medical) today announced that the Japanese Ministry of Health, Labor, and Welfare (MHLW) has notified Rakuten Medical that its lead drug ASP-1929 will be reviewed under the Conditional Early Approval System (CEAS). Compared to standard review the CEAS speeds up the approval process, bringing potentially life-saving treatments to patients faster.

Rakuten Medical is developing precision cell-targeting investigational therapies on its Illuminox™ technology platform. ASP-1929, the first investigational drug developed on this platform, is currently undergoing Phase III clinical development for the treatment of recurrent head and neck cancer.

Rakuten Medical submitted a Japanese Biological License Application (JBLA) for this drug on March 26, 2020 - based on results of Phase I / II clinical trials. In addition – on March 19, 2020 – the company applied for Japan approval of the laser illumination system which is utilized in conjunction with ASP-1929.

"This prestigious designation confirms the potential of our technology to bring the light of hope to cancer patients as quickly as possible," said Hiroshi Mikitani, Chairman and CEO of Rakuten Medical regarding the Conditional Early Approval designation of ASP-1929. "This designation further validates the success of our ongoing work and inspires us to rapidly develop additional innovative therapies on the Illuminox platform for the benefit of patients suffering from cancer."

About Rakuten Medical, Inc.

Rakuten Medical, Inc. is a global biotechnology company developing precision, cell-targeting investigational therapies on its Illuminox^TM platform, which, in pre-clinical studies have shown to lead to rapid and selective cell killing and tumor necrosis. Illuminox therapies have not yet been approved as safe or effective by any regulatory authority. Rakuten Medical is committed to its mission to conquer cancer and aims to realize a society where cancer patients can lead fulfilling lives. Headquartered in the United States, Rakuten Medical has 6 locations in 5 countries, including Japan, the Netherlands, Germany and Taiwan. For more information, visit www.rakuten-med.com.

About ASP-1929

Since 2013, Rakuten Medical, Inc. has been using an exclusively licensed antibody complex to develop new cancer therapies based on its Illuminox^TM technology platform. Rakuten Medical's first pipeline drug developed on Illuminox is ASP-1929, an antibody-drug 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 cancer, esophageal cancer, lung cancer, colon cancer, and pancreatic cancer. After binding to cancer cells, ASP-1929 is locally activated by non-thermal red light (690 nm) illumination emitted by an investigational laser device system. Pre-clinical data indicate that Illuminox technology induces a biophysical process that compromises cell membrane integrity, leading to cancer cell death and tumor necrosis. ASP-1929, has achieved Fast Track designation from the FDA and Sakigake Designation from the Japanese Ministry of Health, Labor, and Welfare 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. ASP-1929 has not yet been approved by any regulatory authority.

About Illuminox^TM

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. 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, Illuminox technology and other regulatory and marketing authorization efforts. The approval and commercial success of the product may not be achieved. Forward looking statements relate to the potential benefits, efficacy, and safety of ASP-1929, and the status of regulatory filings. Such statements may include words such as "expect," "believe," "hope," "estimate," "looks as though," "anticipate," "intend," "may," "suggest," "plan," "strategy," "will," and "do", and are based on our current beliefs. In addition, this press release uses terms such as "important," "notable," and "abnormal" 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, 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, 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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SOURCE Rakuten Medical, Inc.