4588.T

Company Transition Milestone

• The company is transitioning from a drug discovery venture to a fully-fledged pharmaceutical company, with 2026 marked as the culminating year for 22 years of development of lead candidate Telomelysin (OBP-301).
• Completed all planned 2025 commitments for Telomelysin: initiated prior comprehensive evaluation consultation, submitted domestic approval application, completed domestic orphan drug application, obtained regenerative medicine product sales business license, passed 1-year stability testing for final product, and secured the key patent for endoscopic administration of Telomelysin.
• OBP-601 (Censavudine) development milestones planned for 2025 were not achieved, due to external funding constraints facing licensee Transposon.

Telomelysin (OBP-301) Development and Approval Status

• Completed the approval application submission to PMDA (Japan Pharmaceuticals and Medical Devices Agency) on December 15, 2025 after addressing all detailed regulatory inquiries. PMDA's review is expected to conclude within the current month, with review by the Ministry of Health, Labour and Welfare (MHLW) expert committee to follow, and approval expected between May and July 2026.
• OBP101JP trial results: 41% of patients achieved complete response (total tumor disappearance) at 24 weeks post-treatment, rising to 50% complete response in Stage 2/3 esophageal cancer patients at 1 year, compared to just 22% complete response with radiation alone. No cancer progression was observed in complete response patients for at least 1.5 years. The product has only mild side effects (fever, minor nerve numbness, temporary lymphopenia without pain or impaired immune function).
• In the U.S., the Phase 1 trial of Telomelysin combined with chemoradiotherapy for esophageal cancer at Memorial Sloan Kettering Cancer Center saw local recurrence-free survival in 12 of 13 patients (92%), with a 60% 1-year overall survival (including non-cancer related deaths). A manuscript is currently under review, and planning is ongoing for a larger 150-200 patient trial pending funding. The pembrolizumab combination trial for gastric cancer has completed 12 of 13 planned patient doses, with an observed case of complete disappearance of a brain metastasis in a patient unresponsive to checkpoint inhibitor monotherapy; final results are still pending.
• No other company globally is developing an oncolytic virus for esophageal cancer, so approval would make Telomelysin the world's first local treatment for esophageal cancer, and the first approved adenovirus-based oncolytic virus product in Japan.

Pre-Commercialization and Supply Chain Preparation

• A full end-to-end cold chain supply chain has been built: virus is manufactured by Henogen in Belgium, bottled, air freighted to Japan, repackaged in domestic packaging, and shipped to clinical sites at -80°C. The full supply chain (supported by Mitsui Warehousing, Fujifilm Toyama Chemical, and domestic pharmaceutical wholesalers) is nearly complete, awaiting approval for launch.
• 18-month product stability has been confirmed, with 24-month stability data expected by fall 2026. Post-approval, the company will first roll out to 80 core esophageal cancer treatment centers, before expanding to over 300 centers to cover most Japanese esophageal cancer patients. The product can now be administered on an outpatient basis (same-day discharge) with standard COVID-level infection control, eliminating the need for 1-week inpatient observation.
• A three-department quality and safety management system has been established to handle quality assurance, safety monitoring, and post-marketing surveillance. The company plans to add a second manufacturing site to support future sales expansion and global development, which will also support manufacturing of next-gen candidate OBP-702, with the goal of establishing domestic manufacturing capacity by 2030 for full in-house production earlier than the original 2035 target.

Pipeline Status

• OBP-601 (Censavudine), licensed out fully to U.S.-based Transposon for neurodegenerative disease development, has demonstrated efficacy against progressive supranuclear palsy (PSP) and ALS, and has been selected for the HEALEY ALS Platform Trial. It has received $5 million USD from the Alzheimer's Drug Discovery Foundation for Alzheimer's trial planning, and $22 million USD from ARPA-H for anti-healthy aging research. Transposon is currently advancing fundraising, and management expects fundraising to complete in the first half of 2026 to initiate clinical trials.
• OBP-702, the next-generation oncolytic adenovirus carrying the p53 tumor suppressor gene, is planned to initiate investigator-led first-in-human clinical trials in 2026 at Okayama University. The company will manufacture the candidate for this trial in Asia.
• The company plans to initiate indication expansion trials for Telomelysin in 2026: anal cancer (to reduce the need for permanent colostomy after chemoradiotherapy) and laryngeal cancer (to preserve vocal function), both expected to start in 2026 (early 2027 at the latest for laryngeal cancer).

Global Expansion Strategy

• 70% of global esophageal cancer cases are in Asia, so the company prioritizes Asian markets outside of China (which would require costly additional domestic trials and manufacturing). A partnership with Medigen for Taiwan was already signed in December 2024, and the company is in active partnership discussions for South Korea, Singapore, Hong Kong, and Thailand.
• The company will also prioritize the UK and EU, leveraging the UK's regulatory pathway that accepts Japanese clinical data to pursue early approval, and is in discussions with local partners. The U.S. market is deprioritized for now due to the high cost of large-scale pivotal trials.
• The company targets signing at least one new global partnership agreement in 2026.

Human Resources Strategy

• The company plans to expand its development team from fewer than 10 to ~15 employees, and its biomanufacturing team to 15 employees, to support pipeline and commercial growth. Key attractions for talent include the innovative pipeline, the social mission of curing cancer without surgery, the opportunity to build a new pharmaceutical company from a venture, and founder CEO's unique long-term experience in oncolytic virus research.
• Talent development focuses on deepening technical knowledge of R&D staff through regular internal lectures from external clinicians and scientists, and developing next-generation internal leaders with combined technical and management skills to strengthen organizational capacity.

Corporate Development

• The company's share price has performed solidly since the December 2025 approval submission, with growing market capitalization driven by investor recognition of the company's progress towards full commercialization. The company meets the new TOPIX inclusion criteria as of early 2026, with the final decision based on August 2026 price levels; inclusion would improve visibility, increase institutional shareholding, and reduce share price volatility.

• Telomelysin is expected to receive full (unconditional) approval from MHLW between May and July 2026. PMDA has not raised any major concerns, and the company has completed all required regulatory checks and established a stable supply chain, so conditional approval (which would require costly post-approval trials) is unlikely.
• 24-month (2-year) stability data for Telomelysin will be available by fall 2026, further strengthening supply reliability.
• Indication expansion trials for Telomelysin for anal cancer and laryngeal cancer will start in 2026, with the company targeting at least one approved indication expansion by 2030, with an expected timeline of 3 to 4 years from trial initiation to approval.
• The first-in-human clinical trial for next-generation candidate OBP-702 will start in 2026.
• Transposon is expected to complete fundraising for OBP-601 in the first half of 2026, and initiate the pivotal neurodegenerative disease trial and Alzheimer's disease trial in 2026, with potential updates available to shareholders by the end of 2026.
• The company targets signing at least one new overseas partnership agreement for regional commercialization of Telomelysin in 2026.
• The company is on track to meet the criteria for TOPIX index inclusion, with the final decision expected in late 2026 ahead of the October 2026 index reconstitution.
• The company aims to achieve early profitability following Telomelysin launch, through successful pricing and planned market share expansion in the esophageal cancer market.

No financial performance data for product segments (absolute revenue amounts or revenue contribution percentages) is disclosed in this transcript. The call focuses on pipeline development and pre-commercialization activities for the company's lead candidate, rather than reporting historical segment financial results.

• OBP-601 development is entirely dependent on Transposon, which has faced prolonged difficulty securing funding due to broader market trends that have shifted biotech investment away from neurodegenerative disease towards higher-margin indications like obesity and psychiatric disorders. While fundraising is expected to complete in 2026, there is no guarantee it will be completed on schedule.
• Large-scale clinical trials for Telomelysin in the U.S. require substantial external funding, which has not yet been secured, creating uncertainty around the timeline for U.S. development.
• Chinese market expansion is not currently feasible due to the high cost of required additional local clinical trials and domestic manufacturing requirements, creating a risk of foregone revenue from the largest single national esophageal cancer market.
• TOPIX index inclusion is not guaranteed, as it depends on relative free-float market capitalization rankings compared to other listed companies, which is outside of the company's direct control.
• While the company expects full unconditional approval for Telomelysin, there remains residual risk of conditional approval if supply stability is questioned, which would require additional large post-approval clinical trial costs that would divert revenue from other pipeline activities.
• The company faces intense competition for talent in the biotech and biomanufacturing sectors, which creates a risk that it may not be able to hire required staff to support its planned growth and pipeline expansion on schedule.

Q: For indication expansion of Telomelysin, how long would you expect it to take from trial initiation to approval for indications like anal cancer and laryngeal cancer?

A: PMDA has already established that for Telomelysin's local treatment approach, approval can be granted based on local tumor response endpoints rather than overall survival endpoints, which greatly shortens the development timeline. A survival endpoint would require 2-3 years of patient follow-up per patient, while local response endpoints can be assessed within 6-18 months. For anal cancer, the company is targeting an endpoint of reducing the need for permanent colostomy, and plans to build a new trial protocol, complete pilot testing, and initiate a pivotal trial, with an expected total timeline of 3-4 years to approval. The same timeline applies to laryngeal cancer, where the endpoint is preserving vocal function. The company is targeting at least one approved indication expansion by 2030, and is working with PMDA to keep the timeline as short as possible.