– RM-1929 photoimmunotherapy is a novel therapy which was generally well tolerated in three heavily pre-treated Japanese patients with rHNSCC and showed a manageable safety profile –

SAN MATEO, Calif., July 8, 2021 /PRNewswire/ — Rakuten Medical, Inc. (Rakuten Medical) announced that data from a phase I, single-center, open-label study of RM-1929 photoimmunotherapy in Japanese patients with recurrent head and neck squamous cell carcinoma (rHNSCC) was accepted for publication in the International Journal of Clinical Oncology and published on June 24, 2021.

The manuscript describes the design of a Phase I study, where patients were given a single fixed dose (640 mg/m²) of RM-1929, an anti-EGFR antibody cetuximab conjugated with a light-activatable dye (IRDye®700DX), and a fixed light treatment dose (50 J/cm² for superficial illumination; 100 J/cm fiber diffuser length for interstitial illumination) in Japan. The study was designed to evaluate the safety, tumor response (modified RECIST v1.1 by central radiology review), pharmacokinetics, and immunogenicity administered in subjects with rHNSCC.

The data concluded that RM-1929 photoimmunotherapy showed a manageable safety profile in rHNSCC – and showed tumor response in the heavily pre-treated patient population was clinically meaningful and warranted further investigation.

RM-1929 is a first-in-class drug developed on the Illuminox™ platform based on photoimmunotherapy and received conditional marketing approval in Japan from the Ministry of Health, Labour and Welfare in September 2020.

A paper of full text is available at https://rd.springer.com/article/10.1007/s10147-021-01960-6

*RM-1929 and ASP-1929 are analogous. Extensive physiochemical studies show that they have comparable physical and chemical properties.

About Rakuten Medical, Inc.

Rakuten Medical, Inc. is a global biotechnology company developing precision, cell-targeting investigational therapies on its Illuminox™ 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. Rakuten Medical is committed to its mission to conquer cancer and aims to realize a society where cancer patients can lead fulfilling lives. With its headquarters, and research and development base in the United States, Rakuten Medical has 6 locations in 5 countries, Japan, the Netherlands, Taiwan and Switzerland. 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™ 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 received conditional early 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 has 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 products developed on the Illuminox™ platform such as ASP-1929, and other regulatory and marketing authorization efforts. The approval and commercial success of the products 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,” “generally well tolerated,” “manageable,” 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.

SOURCE Rakuten Medical, Inc.

Release: PR Newswire