Serve BioPharma Obtains Orphan Regenerative Medicine Product Designation for Primary Malignant Bone Tumor Therapeutic Drug Using Oncolytic Virus Surv.m-CRA-1

Serve BioPharma Inc. (Headquarters: Kagoshima City, Kagoshima Prefecture; Representative Director & President: Masaki Yamada; Founder, Director, Chairman & Chief Scientific Officer: Kenichiro Kozai; hereinafter "the Company") is pleased to announce that its therapeutic drug for primary malignant bone tumors (hereinafter "this therapeutic drug"), which utilizes the uniquely developed oncolytic adenovirus "Surv.m-CRA-1 (Survivin-responsive, multifactorial proliferation-controlled adenovirus-1)," received designation as an "Orphan Regenerative Medicine Product" (Orphan Drug) from the Minister of Health, Labour and Welfare on May 8, 2026.

Background and Significance

Oncolytic viruses are attracting global attention as a next-generation cancer treatment that selectively destroys cancer cells with excellent safety and therapeutic efficacy. However, in Japan, the development of regenerative medicine products, including oncolytic viruses, has significantly lagged, and the development and practical application of innovative Japanese-origin oncolytic viruses are highly anticipated.

Against this backdrop, the Company is promoting research and development towards the social implementation of the oncolytic virus Surv.m-CRA (Survivin-responsive, multifactorial proliferation-controlled adenovirus) series, uniquely developed by the Company's founder, Director, Chairman & Chief Scientific Officer Kenichiro Kozai (Professor at Kagoshima University).

"Surv.m-CRA-1" is an oncolytic virus equipped with a "survivin promoter" that is specifically activated in cancer cells. It can proliferate only in cancer cells and selectively kill them without harming normal cells. Therefore, it combines high therapeutic efficacy with safety, and is expected to be a new cancer therapeutic drug (regenerative medicine product) that also acts on cancer stem cells resistant to existing treatments.

Aiming to overcome intractable cancers for which effective treatments have not yet been established, the Company is advancing commercialization for primary malignant bone tumors, a rare cancer, as its first target. Positive results were obtained in a Phase II investigator-initiated clinical trial, and a Phase III investigator-initiated clinical trial is currently underway, aiming for official approval for primary malignant bone tumors.

This time, the efficacy and effects of this therapeutic drug were recognized through review by the Pharmaceuticals and Medical Devices Agency (PMDA) and deliberations by the Pharmaceutical Affairs and Food Sanitation Council, an advisory body to the Minister of Health, Labour and Welfare, leading to its designation as an "Orphan Regenerative Medicine Product" by the Minister of Health, Labour and Welfare.

The Orphan Regenerative Medicine Product (Orphan Drug) Designation System is established under Article 77-2 of the Pharmaceuticals and Medical Devices Act to promote the development of therapeutic drugs (regenerative medicine products) for diseases with a small number of patients (rare diseases). The Minister of Health, Labour and Welfare designates such products after consulting with the Pharmaceutical Affairs and Food Sanitation Council, provided they meet criteria related to "number of target patients," "medical necessity," and "development potential." Specifically, it is necessary to meet conditions such as the number of target patients in Japan being less than 50,000, and there being a particularly high medical necessity for the target disease.

If designated as an Orphan Drug, preferential measures such as subsidies for development costs, priority review, and extension of the re-examination period can be received.

Starting with this Orphan Drug designation in Japan, the Company plans to obtain Orphan Drug designation for this therapeutic drug in the United States and Europe in the future. We will concurrently advance clinical development so that we can deliver this innovative therapeutic drug to patients suffering from the rare disease of primary malignant bone tumors on a global scale, not just in Japan, as soon as possible.

To meet unmet medical needs, the Company will continue to promote research and development, pioneering new possibilities in cancer treatment and gene therapy by utilizing viral therapeutic drugs as a new modality, and aiming to become a global leader in gene therapy.

Supplementary Information

About the Orphan Drug Designation System

This system promotes the research and development of drugs for intractable diseases with high medical necessity and a domestic target patient population of less than 50,000. Based on Article 77-2 of the Pharmaceuticals and Medical Devices Act, the Minister of Health, Labour and Welfare designates such drugs after consulting with the Pharmaceutical Affairs and Food Sanitation Council, provided they meet conditions such as the number of target patients in Japan being less than 50,000 and there being a particularly high medical necessity. Depending on the type of target drug, there are three categories: orphan drugs, orphan medical devices, and orphan regenerative medicine products.

If designated as an Orphan Drug, preferential measures such as subsidies for development costs, tax incentives, priority review, and extension of the re-examination period can be received.

Source: Ministry of Health, Labour and Welfare Overview of the Orphan Drug, Orphan Medical Device, and Orphan Regenerative Medicine Product Designation Systems

https://www.mhlw.go.jp/stf/seisakunitsuite