Leydig Cell Tumor

Rakuten Medical, Inc. (Rakuten Medical) announced that data from a phase 1/2a, open-label, multicenter study of RM-1929 photoimmunotherapy in patients with locoregional, recurrent head and neck squamous cell carcinoma (rHNSCC) (RM-1929-101 study, ClinicalTrials.gov Identifier: NCT02422979) was accepted for publication in Head and Neck and published online on October 9th, 2021. The manuscript describes the design and results of the 2-part study, which was aimed to elucidate the recommended dose, safety, pharmacokinetics, immunogenicity, and preliminary efficacy of RM-1929 photoimmunotherapy in patients with locoregional rHNSCC. Part 1* of the study (dose-escalation) determined the recommended drug dose of RM-1929 to be 640 mg/m2 with a fixed light dose of 50 J/cm2 for superficial tumors and 100 J/cm fiber diffuser length for interstitial tumors. Among Part 2* participants, 30 patients received RM-1929 photoimmunotherapy, and were included in the safety and treated population. Among Part 2* study participants, 13 (43.3%) patients reported a serious TEAE wherein 3 were considered to be treatment related (tumor pain, oral pain, and airway obstruction). Also, for patients enrolled in Part 2* of the study, the median OS was 9.30 months (95% CI 5.16–16.92 months). Confirmed and unconfirmed objective responses were achieved in 13 (43.3%, 95% CI 25.46%–62.57%) patients, with 4 (13%) patients achieving a complete response (CR) and 9 (30.0%) patients demonstrating a partial response (PR). Disease control [defined as CR, PR, or stable disease (SD)] was observed in 24 (80%, 95% CI 61.43%–92.29%) patients. The full text article is available at *Part 1: Phase 1 study part (safety and dose-finding) / Part 2: Phase 2a study part (safety and preliminary anticancer activity; patients were treated with up to 4 cycles of RM-1929 photoimmunotherapy) **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 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. About ASP-1929** Since 2013, Rakuten Medical, Inc. has been using an exclusively licensed antibody complex to develop new cancer therapies based on its IlluminoxTM technology platform. Rakuten Medical's first pipeline drug developed using its Illuminox 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 receptors (EGFR), a cancer antigen expressed in multiple types of solid tumors, including head and neck, cutaneous, esophageal, lung, colon and pancreatic cancers. After binding to cancer cells, ASP-1929 is locally activated by non-thermal red light (690 nm) illumination emitted by a laser device system. Pre-clinical data indicates that Illuminox technology induces a biophysical process that compromises cell membrane integrity, leading to cancer cell death and tumor necrosis. ASP-1929 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 and the laser device system have 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.