Rakuten Medical Announces FDA Acceptance of an Investigational New Drug (IND) Application for RM-1995, a Conjugate of IR700 and anti-CD25 Antibody

SAN DIEGO, Dec. 15, 2021 /PRNewswire/ -- Rakuten Medical, Inc today announced that the U.S. Food and Drug Administration (FDA) has accepted the company's Investigational New Drug (IND) application to begin clinical studies of RM-1995 photoimmunotherapy in patients with advanced cutaneous squamous cell carcinoma or with head and neck squamous cell carcinoma.

RM-1995 photoimmunotherapy treatment is an investigational drug-device combination being developed by Rakuten Medical, Inc. The drug portion of the treatment, RM-1995, is a conjugate of a photoactivatable dye (IRDye® 700DX [IR700]) and a monoclonal antibody specific for cell-surface interleukin 2 (IL-2) receptor α-chain (CD25). The device portion of the treatment is a laser device system (PIT690 Laser System) used to activate IR700 by illumination with 690nm nonthermal red light. Tumor-resident regulatory T cells (Tregs), that inhibit antitumor immune responses to promote tumor growth, can be specifically targeted with anti-CD25 antibodies1. Rakuten Medical, Inc is investigating RM-1995 as a potential new treatment to provide targeted, direct reduction of Tregs specifically within light-treated tumor lesions to induce systemic anticancer immune responses.

"We are very excited to have achieved this regulatory milestone and delighted to be moving RM-1995 into the clinic," said Mickey Mikitani, Chief Executive Officer of Rakuten Medical, Inc. "RM-1995 has the potential to attack the cancer in a novel way, targeting Tregs. Based on data from preclinical studies, RM-1995 photoimmunotherapy treatment is expected to result in acute killing of Tregs within the tumor. We are developing drugs using various antibodies in our drug discovery program based on Rakuten Medical's IlluminoxTM platform, and RM-1995 is the second pipeline drug developed on this platform. We will continue to advance our business with a sense of speed so that we can deliver these important treatments to as many patients as possible."

About Rakuten Medical, Inc.

Rakuten Medical, Inc. is a global clinical stage biotechnology company developing and commercializing precision, cell-targeting investigational therapies on its IlluminoxTM 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 RM-1995

RM-1995, developed by Rakuten Medical using its IlluminoxTM platform, is an antibody-dye conjugate comprised of a monoclonal antibody specific for cell-surface interleukin 2 (IL-2) receptor α-chain (CD25) and IRDye® 700DX (IR700), a light-activatable dye. RM-1995 photoimmunotherapy is designed to specifically kill CD25+ regulatory T cells (Tregs) within solid tumors, once illuminated with 690nm nonthermal red light.2 Rakuten Medical's preclinical data has suggested that RM-1995 photoimmunotherapy treatment can be utilized to specifically deplete intratumoral Tregs, thereby alleviating local Treg-mediated restraint within the tumor microenvironment, rapidly improving the CD8 T cell: Treg ratio, and reinvigorating effector CD8+ T cell responses.

1Arce Vargas F, Furness AJS, Solomon I, Joshi K, Mekkaoui L, Lesko MH, et al. Fc-Optimized Anti-CD25 Depletes Tumor-Infiltrating Regulatory T Cells and Synergizes with PD-1 Blockade to Eradicate Established Tumors. Immunity. 2017;46(4):577-586.

2 Sato, K. et al. Spatially selective depletion of tumor-associated regulatory T cells with near-infrared photoimmunotherapy. Sci Transl Med 8, 352ra110, doi:10.1126/scitranslmed.aaf6843 (2016).

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 RM-1995, the Illuminox platform and IRDye® 700DX, as well as other regulatory and marketing authorization efforts, the potential benefits, efficacy, and safety of RM-1995 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," "might," "suggest," "plan," "strategy," "will," "hopeful," "excited," "do," "aims," "develop" and similar expressions. Forward-looking statements are neither historical facts nor assurances of future performance. 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 "expected," "important," "notable," and "novel" 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 RM-1995 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 RM-1995 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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SOURCE Rakuten Medical, Inc.

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