First Safety and Efficacy Data of Rakuten Medical's Alluminox Treatment using ASP-1929 in Combination with anti-PD-1 for Recurrent and/or Metastatic Head and Neck Cancer Presented at AHNS 2023

• Generally tolerated treatment with overall survival rate at 18 months of 53.5% at data cutoff ¹⁾
• A potential, new, innovative treatment option for recurrent head and neck cancer

SAN DIEGO, July 10, 2023 /PRNewswire/ -- Rakuten Medical, Inc., a global biotechnology company developing and commercializing precision, cell targeting therapies based on its proprietary Alluminox™ platform, today announced that new data from the ASP-1929-181 study was presented at the American Head and Neck Society's (AHNS) 11^th International Conference on Head and Neck Cancer (AHNS 2023) on July 10, 2023.

The early evaluation of 19 enrolled patients in ASP-1929-181 study for recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) presented during the meeting suggested that the novel treatment approach of Alluminox treatment using ASP-1929 in combination with anti-PD-1 therapy showed promising early results in patients with locoregional and/or metastatic HNSCC.

Ann M. Gillenwater, Professor, Department of Head and Neck Surgery, Division of Surgery, The University of Texas MD Anderson Cancer Center said at the podium presentation that "Patients with advanced head and neck cancer lack many treatment options and experience overall low survival and high locoregional recurrence. PD-1 therapy is used to treat various tumor types including head and neck cancer. The preliminary results seen in this study warrant further study to evaluate its potential as a new innovative treatment for recurrent head and neck cancer. Targeting both EGFR+ tumors and PD-1 blockade, the antitumor immune response is anticipated to result in improved tumor shrinkage that is durable compared to either monotherapy alone."

Key findings presented at AHNS 2023
"Safety and efficacy findings from a phase 1b/2 open-label study of a novel combination of ASP-1929 photoimmunotherapy with anti-PD-1 therapy in EGFR-expressing advanced head and neck squamous cell carcinoma" (Abstract: S252)

• The objective response rate (ORR) was 29.4% (5 patients, 95% CI 10.3‒56.0), including 17.6% (3 patients) complete responses (CR) and 11.8% (2 patients) partial responses (PR) ²⁾
• The overall survival (OS) rate at 18 months was 53.5% (95% CI 18.5‒79.3) ^{1) 2)}
• Alluminox treatment using ASP-1929 in combination with anti-PD-1 therapy was generally tolerated, and adverse events were manageable
• The encouraging early efficacy and safety outcomes presented at AHNS 2023 warrant additional clinical studies to substantiate and reinforce these preliminary findings

1) Median OS has not been reached at data cutoff
2) Only 17 out of 19 enrolled patients were considered as efficacy evaluable population

"We are truly honored to present the promising data from our ASP-1929-181 study at AHNS," said Mickey Mikitani, Co-CEO of Rakuten Medical. "We are excited by the possibility that the addition of Alluminox treatment using ASP-1929 to standard of care anti-PD-1 therapy may provide meaningful progress in the treatment of head and neck cancer and potentially beyond."

Disclaimer: These preliminary findings may change upon completion of follow up of and final data analysis.

ASP-1929-181 overview
ASP-1929-181 is a Phase 1b/2, open-label study of Alluminox treatment using ASP-1929 in combination with anti-PD-1 therapy in EGFR expressing advanced solid tumors (ClinicalTrials.gov Identifier: NCT04305795). The trial will enroll three cohorts of patients within distinct populations – 1) recurrent locally advanced and/or metastatic HNSCC 2) locally advanced or metastatic cutaneous squamous cell carcinoma (CuSCC), and 3) locally advanced or metastatic CuSCC with progressive disease despite at least 2 months of anti-PD1 therapy. Primary endpoints of this study include safety, tolerability, and tumor response of the Alluminox treatment using ASP-1929 in combination with anti-PD1 therapy.

About Rakuten Medical, Inc. 
Rakuten Medical, Inc. is a global biotechnology company developing and commercializing precision, cell targeting therapies based on its proprietary Alluminox™ platform, which, in pre-clinical studies, has been shown to induce rapid and selective cell killing and tumor necrosis. Alluminox therapies have not yet been approved outside of Japan. Rakuten Medical is committed to its mission to conquer cancer by delivering our innovative treatments as quickly as possible to as many patients as possible all over the world. The company has offices in 6 countries, including the United States, where it is headquartered, Japan, the Netherlands, Taiwan, Switzerland and India. For more information, visit www.rakuten-med.com.

About Alluminox™ platform  
The Alluminox™ platform is an investigational technology 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 Alluminox 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 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 the Alluminox platform may also result in local and systemic innate and adaptive immune activation due to immunogenic cell death of the targeted cancer cells and/or the removal of targeted immunosuppressive cells within the tumor microenvironment. Outside of Japan, Alluminox therapies have not yet been approved by any regulatory authority.

About ASP-1929
Rakuten Medical's first pipeline drug developed on its Alluminox™ 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 receptor (EGFR), a cancer antigen expressed in multiple types of solid tumors, including head and neck, breast, lung, colorectal, prostate and pancreatic cancers. After binding to cancer cells, ASP-1929 is locally activated by   illumination with red light (690 nm), emitted by a laser device system to produce a photochemical reaction. This reaction is believed to cause damage to the membrane of cancer cells, leading to selective necrosis of cancer cells. ASP-1929 received Fast Track designation from the U.S. Food and Drug Administration (FDA) in January 2018, and is currently under investigation in a global Phase 3 clinical trial for recurrent head and neck cancer. In Japan, ASP-1929 received marketing approval from the Japanese Ministry of Health, Labor, and Welfare for unresectable locally advanced or recurrent head and neck cancer in September 2020, under the Sakigake Designation System and the Conditional Early Approval System. Outside of Japan, ASP-1929 has not yet been approved 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 the Alluminox™ platform, as well as other regulatory and marketing authorization efforts, the potential benefits, efficacy, and safety of therapies created using the Alluminox platform, and the status of regulatory filings. The approval and commercial success of the product may not be achieved. Forward looking statements relate to the potential benefits, efficacy, and safety of our therapies, 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 our therapies, 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 our therapies, 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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