Autologous cell therapy for severe ocular surface disease enters a new stage of global development, with the United States as the top priority

• Approved and reimbursed in Japan: SAKRACY is made from the patient’s own oral mucosal cells, so it does not require healthy limbal tissue—an important advantage for patients with disease in both eyes.
• Phased grant support: GRiT provides up to ¥1.7 billion per project (up to ¥200 million for a feasibility study and up to ¥1.5 billion toward international clinical trial costs). Actual funding is subject to final grant terms and the outcome of a stage-gate review.
• U.S.-focused next steps: The Company will advance regulatory preparations, assess clinical trial sites in multiple countries and validate international product logistics, laying the groundwork for the planned international Phase 3 trial.

TOKYO, October 7, 2026 — CynosBio Inc. (“CynosBio” or the “Company”) today announced that its research and development project for the global development of SAKRACY® has been selected by the Japan Agency for Medical Research and Development (“AMED”) under the Global Type of the Alliance program for Innovative Medical/healthcare research by Government-Academia-INdustry Collaboration (AIMGAIN), known as the Global Research and Development Support Project for the Treatment of Intractable/Rare Diseases (“GRiT”). The project will support preparations for an international Phase 3 trial in patients with severe ocular surface disease associated with Stevens-Johnson syndrome (SJS) or ocular cicatricial pemphigoid (OCP).

SAKRACY received marketing approval in Japan in January 2022 for the reduction of ocular surface adhesions in limbal stem cell deficiency, and was added to Japan’s National Health Insurance reimbursement list in September of the same year. The therapy builds on clinical research that began at Kyoto Prefectural University of Medicine in 2002 and, since its approval, has been used in clinical practice in Japan. CynosBio will draw on this clinical track record and its established business foundation in Japan to advance international development.

Shinji Uga, President and Chief Executive Officer of CynosBio, commented:

“SAKRACY grew out of academic research and is already part of clinical practice in Japan. Our next goal is to extend its reach worldwide. AMED’s selection further strengthens our foundation as we prepare for development in the United States. By combining our clinical experience with partners who bring complementary strengths in development and commercialization, we aim to bring this therapy to many more patients.”

Addressing a clinical challenge that extends beyond the cornea

In severe ocular surface disease associated with SJS/toxic epidermal necrolysis (TEN) and pemphigoid—both designated intractable diseases in Japan—extensive scarring and adhesions can develop across both the cornea and the conjunctiva, causing pain and severe visual impairment. Because the ocular surface itself is so badly damaged in these patients, conventional corneal transplantation may be unsuitable or, even when performed, may have a poor prognosis.

SAKRACY is an epithelial cell sheet made by culturing cells from a small sample of the patient’s own oral mucosa on a human amniotic membrane substrate. Because the cells are taken from the oral mucosa rather than the corneal limbus, there is no need to harvest limbal tissue from the patient’s healthy fellow eye—a particularly important advantage for patients with disease in both eyes. After the adhesions are surgically released, the sheet can be shaped to fit the affected area and transplanted onto adhesion sites on the cornea and conjunctiva.

SAKRACY is not approved by the FDA and remains investigational in the United States.

Chie Sotozono, M.D., Ph.D., Professor, Department of Ophthalmology, Kyoto Prefectural University of Medicine, commented:

“SJS/TEN and pemphigoid, both designated intractable diseases in Japan, can damage the mucosa of the ocular surface and lead to severe visual impairment in both eyes, and until now there has been no effective surgical treatment for these patients. SAKRACY is built on more than 20 years of research and clinical experience at Kyoto Prefectural University of Medicine with transplantation of cultivated autologous oral mucosal epithelial sheets, work we pursued to develop new treatments for these intractable diseases. SAKRACY is a regenerative medicine product intended to reduce ocular surface adhesions in limbal stem cell deficiency. There is no comparable product in Japan or elsewhere, and patients around the world are waiting for this treatment. Working together with experts overseas, we hope to bring this technology developed in Japan to those patients.”

Building the clinical and operational foundation for an international Phase 3 trial

The feasibility study will focus on regulatory consultations with the U.S. Food and Drug Administration (FDA) and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA); assessment of eligible patients and clinical trial sites in the United States and Asia; and validation of international product logistics. Through this work, the Company aims to define not only the trial protocol but also the clinical, quality and supply requirements for international development.

CynosBio plans to seek feedback from the FDA through the Pre-IND process in the fourth quarter of 2026. Trial design, enrollment targets and analysis methods will be finalized based on consultations with the FDA and PMDA.

CynosBio will lead the program as sponsor, working with Kyoto Prefectural University of Medicine and expert advisers in clinical development, regulatory affairs and manufacturing. The international clinical trial is intended to generate evidence to support a future Biologics License Application (BLA) in the United States, and the Company also plans regulatory submissions in selected countries and regions in Asia.

A phased funding framework for global development

Under GRiT, funding of up to ¥200 million is available for the feasibility study and up to ¥1.5 billion for the international clinical trial.

The final award amount and payment schedule will be determined in accordance with AMED’s grant documentation. In addition, funding for the clinical trial phase is contingent on passing a stage-gate review conducted by AMED after the feasibility study is completed. This cost-sharing framework will complement the Company’s own funds and potential future strategic partnerships, helping CynosBio advance its international development.

About AMED and GRiT

The Japan Agency for Medical Research and Development (AMED) is Japan’s national funding agency for medical research and development. Launched in fiscal year 2026, GRiT supports Japanese companies pursuing international clinical development for intractable and rare diseases by funding feasibility studies and sharing eligible costs of international clinical trials, including trials with participating sites in Japan.

Forward-Looking Statements

This press release contains forward-looking statements regarding AMED funding, including its amount, timing and conditions; regulatory interactions; the design, timing and conduct of clinical trials; potential regulatory submissions and approvals; strategic collaborations; and the potential of SAKRACY. These statements reflect current expectations and involve risks and uncertainties. Actual results may differ materially due to factors including grant decisions and stage-gate outcomes; regulatory requirements; clinical trial results, enrollment and timing; manufacturing and international logistics; and the availability of additional funding. CynosBio undertakes no obligation to update these statements except as required by law.

This press release is intended to provide corporate information and is not intended to advertise or promote any product or to provide medical advice.