Rakuten Medical Releases New, Post-hoc Analysis of Safety, Pharmacokinetic (PK), Immunogenicity and Biomarker Data from its Phase 1 & 1/2a Clinical Trials

• Two posters were presented during the European Society for Medical Oncology (ESMO) Congress 2019 on its RM-1929 photoimmunotherapy (PIT) treatment in patients with locoregional, recurrent head and neck squamous cell carcinoma (rHNSCC)
• Based on the preliminary safety profile, PK, and previously reported, clinically meaningful anti-tumor activity, a global Phase 3 clinical trial of ASP-1929 Illuminox™ in patients with rHNSCC is underway

BARCELONA, Spain, Sept. 30, 2019 /PRNewswire/ -- Rakuten Medical, a clinical-stage, global biotechnology company developing precision-targeted cancer therapies based on its proprietary, anti-cancer treatment platform, Illuminox™, released post-hoc analysis of safety, pharmacokinetic (PK), immunogenicity and exploratory biomarker data from its RM-1929 photoimmunotherapy (PIT) Phase 1 & 1/2a studies. These early clinical trials evaluated the safety and anti-tumor activity of RM-1929 PIT in patients with locoregional, recurrent head and neck squamous cell carcinoma (rHNSCC).

The key findings were presented during two poster presentations at the 2019 ESMO Congress held this week in Barcelona:

"Integrated safety, pharmacokinetics (PK) and immunogenicity data of RM-1929 photoimmunotherapy (PIT) in subjects with locoregional, recurrent head and neck squamous cell carcinoma (rHNSCC)" (Abstract 2954), presented by Jennifer M. Johnson, MD, PhD, FACP; Assistant Professor, Department of Medical Oncology and Associate Program Director, Hematology and Medical Oncology Fellowship, Thomas Jefferson University.

The assessment of safety and PK across the Phase 1 & 1/2a studies, when considered in connection with preliminary Phase 2a clinically meaningful anti-tumor activity reported at ASCO 2019, support the current global Phase 3 study of ASP-1929 Illuminox treatment in locoregional rHNSCC patients. Specifically:

• Out of the 41 patients in the trial, 17 experienced at least one serious adverse event (SAE), and five experienced SAE considered related to study treatment; the majority being local to the treatment site.
• RM-1929 PIT 640 mg/m² dose achieved an AUC and T_1/2, which is expected to achieve saturation of the epidermal growth factor receptors (EGFR). Repeated administration of RM-1929 PIT (cycle 2-4) given approximately four weeks apart, were comparable to those in cycle one, with a low presence of anti-drug antibodies (ADA).

"Intratumoral and peripheral exploratory biomarker analysis in patients with locoregional, recurrent head and neck squamous cell carcinoma (rHNSCC) treated with RM-1929 photoimmunotherapy" (Abstract 3725), presented by Jack Bui, MD, PhD, Pathology; University of California San Diego Health.

Immune-activation biomarkers following RM-1929 PIT treatment were evaluated in the Phase 1/2a study in patients with rHNSCC; conclusions indicated that certain subsets of patients displayed potential signs of immune activation following treatment, regardless of best target tumor response. More specifically:

• RM-1929 PIT was associated with induction of PD-L1 expression in tumor and immune cells in eight out of 12 patients.
• EGFR expression was maintained in residual tumors in 12 patients with pre- and post-treatment biopsies, indicating that target lesions may be candidates for additional PIT treatment cycles.
• Peripheral blood immunophenotyping suggests possible activation of innate and adaptive immunity following treatment in 15 of 18 patients.

"Rakuten Medical is encouraged by these post-hoc analyses across our available early studies, as they further characterize the safety and PK, and provide insights into potential immune activation by RM-1929 PIT that could potentially enable further development in combination with other anti-cancer, immunotherapy treatment options," said Jeannie Hou, VP of Clinical Development at Rakuten Medical. "Our ASP-1929 Illuminox Phase 3 clinical trial is currently underway, which is another important step in the development of a possible new therapeutic option for head and neck cancer patients; and may provide those suffering from this devastating disease with an additional therapeutic choice."

About Rakuten Medical
Rakuten Medical is a privately funded, clinical-stage biotechnology company with offices in the U.S., Japan, Taiwan, Germany and the Netherlands. Rakuten Medical aims to become a leader in precision-targeted medicines through its proprietary, anti-cancer treatment platform, Illuminox™. Rakuten Medical's lead product, ASP-1929 Illuminox, 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 Illuminox are also underway to evaluate the safety in patients with other cancer types. For more information, visit www.rakuten-med.com.

About Iluminox™
Illuminox™ is a proprietary, anti-cancer treatment platform of Rakuten Medical that is comprised of drug, device and other related technologies. Transient excitation of this drug, including antibodies conjugated to light-excitable dyes, with non-thermal light is believed to result in anti-cancer activity and in rapid and selective cell killing and tumor necrosis with minimal effects on surrounding normal tissue. It may also lead to the systemic induction of innate and adaptive immunity. Illuminox is based upon the photoimmunotherapy treatment platform developed at the National Institutes of Health (NIH) and licensed exclusively to Rakuten Medical for commercialization. Illuminox is the Rakuten Medical platform used for the development of specific therapies using a combination of drug, device and other technologies. None of these therapies based on this platform have been approved by any regulatory authority. The Illuminox platform is not a drug nor device on its own. The Illuminox name is not the brand name of a drug nor device.

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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 Illuminox™ 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.