Rakuten Medical Releases Phase 2a Study Data during ASCO Annual Meeting Highlighting Positive Safety Profile and Clinically Meaningful Anti-tumor Activity

• Clinically meaningful activity of RM-1929 Photoimmunotherapy in patients with locoregional, recurrent head and neck cancer
• Generally well-tolerated treatment results and an overall response rate of 43 percent (95 percent CI 25.5-62.6)
• Initiates Phase 3 clinical trial to evaluate efficacy and safety of ASP-1929 Photoimmunotherapy versus standard-of-care

CHICAGO, June 1, 2019 /PRNewswire/ -- Rakuten Medical, a clinical-stage biotechnology company developing precision-targeted cancer therapies based on its proprietary Photoimmunotherapy (PIT) platform, announces positive data results from its Phase 2a study, evaluating the safety and anti-tumor activity of RM-1929 (an EGFR-targeted antibody conjugate) PIT in patients with locoregional, recurrent head and neck squamous cell carcinoma (rHNSCC). Details of its global Phase 3 study trial design were also presented.

The Phase 2a positive data, presented during a poster presentation discussion during the American Society of Clinical Oncology (ASCO) annual meeting in Chicago on Saturday, June 1, 2019, showed that treatment with RM-1929 PIT resulted in clinically meaningful activity in 30, heavily pre-treated patients with rHNSCC.

• The overall response rate by independent review was 43 percent (95 percent CI 25.5‒62.6), including four (13 percent) complete responses and nine (30 percent) partial responses.
• The median, progression-free survival was 5.2 months (95 percent CI 2.1‒5.5), and median overall survival was 9.3 months (95 percent CI 5.2‒16.9).
• Treatment with RM-1929 PIT was generally well-tolerated.
• Out of the 13 patients (43.3 percent) who experienced a serious adverse event, only three (10 percent) were considered treatment-related.

"Rakuten Medical is committed to addressing the unmet medical needs of patients with cancer, and we are encouraged by the Phase 2a trial data, which demonstrated clinically meaningful anti-tumor activity in patients with late-stage head and neck cancer," said Jeannie Hou, VP of Clinical Development at Rakuten Medical. "We look forward to enrolling patients in our global Phase 3 trial in head and neck cancer."

Phase 3 Trial Overview
Rakuten Medical has initiated its Phase 3, multicenter, randomized, double-arm, open-label trial of ASP-1929 (an EGFR-targeted antibody conjugate; analogous to RM-1929) PIT versus a physician's choice of standard-of-care for locoregional rHNSCC patients who have failed after at least two lines of therapy, of which at least one line must be systemic, platinum-based chemotherapy for HNSCC.

An overview of the trial background, design and criteria was also presented at ASCO during a poster session. The primary endpoints of the study are progression-free survival and overall survival, with the key secondary endpoint being objective response rate. Rakuten Medical expects to enroll approximately 275 patients in the U.S., EU and Asia Pacific.

For more information about the Phase 3 clinical trial (NCT03769506), visit www.clinicaltrials.gov.

About Photoimmunotherapy
Photoimmunotherapy (PIT) is an investigational, anti-cancer treatment platform that is comprised of a drug and device combination that utilizes monoclonal antibodies conjugated to a dye (IRDye^® 700DX). Transient excitation of IRDye 700Dx with non-thermal red light (690 nm) is believed to result in anti-cancer activity, which is mediated by biophysical processes that may compromise the membrane integrity of cells. The requirement of targeted binding of antibody-IR700 conjugate to a specific antigen on the cell surface and subsequent illumination is believed to result in rapid and selective cell killing and tumor necrosis with minimal effects on surrounding normal tissue. PIT may also lead to the systemic induction of innate and adaptive immunity.

About Rakuten Medical
Rakuten Medical is a privately funded, clinical-stage biotechnology company with offices in the U.S., Japan, Taiwan, Germany and Netherlands. Rakuten Medical aims to become a leader in precision-targeted medicines through its proprietary photoimmunotherapy platform, which is being studied in ongoing clinical trials, evaluating its safety and effectiveness as a cancer-treatment option that can be administered independently, or in combination with other drugs. Rakuten Medical's lead product, ASP-1929, has achieved Fast Track designation from both the FDA and the Japanese Ministry of Health, Labour, and Welfare; and is conducting a global Phase 3 clinical trial for recurrent head and neck squamous cell carcinoma. Additional Phase 2 studies for ASP-1929 PIT are also underway to evaluate the safety in patients with other cancer types. For more information, visit www.rakuten-med.com.

Forward Looking Statement
This press release contains forward-looking statements made pursuant to the safe harbor provisions of the (United States) Private Securities Litigation Reform Act of 1995 that involve risks, uncertainties, and assumptions that could cause Rakuten Medical's actual results and experience to differ materially from anticipated results and expectations expressed in these forward-looking statements. These forward-looking statements include information concerning the commercialization of services related to Rakuten Medical products such as ASP-1929 and other initiatives toward regulatory approval to be sold and marketed. These products may not successfully be granted regulatory approval to be sold and marketed, or may not be commercially successful. Forward-looking statements suggest potential profitability, efficacy and safety, and the status of the application for approval. Rakuten Medical has in some cases identified forward-looking statements by using words such as "anticipates," "believes," "hopes," "estimates," "looks," "expects," "intends," "potential," "may," "suggest," "plans," "strategizes," "likely", "will", and similar expressions. Such forward-looking statements are based upon Rakuten Medical's current beliefs. Moreover, this press release states an opinion related to clinical research data, hence the use of expressions such as "important," "notable" and "abnormal." Ongoing clinical studies involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including uncertainty of success in regulatory approval or commercialization of ASP-1929 which may be impacted by, among other things, problems with the manufacturing process for ASP-1929, the occurrence of adverse safety events, failure to demonstrate therapeutic benefit, and the other risks and uncertainties, both reasonable and unreasonable. Rakuten Medical undertakes no obligation to release publicly the results of any revisions to any such forward-looking statements that may be made to reflect new information obtained, events or circumstances after the date of this press release or to reflect the occurrence of unanticipated events, except as required by applicable law or regulation. In the event of one or more revisions to Rakuten Medical's forward-looking statement, it should not be inferred that such revisions or other forward-looking statements will be further revised.

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SOURCE Rakuten Medical, Inc.