4591.T

• Core Business Strategy: Develop novel aptamer nucleic acid drugs targeting unmet medical needs, focusing on two core areas: rare disease treatments (ACH) and retinal ophthalmology drugs. A new third pipeline candidate (DME) was added in this quarter.
• ACH (RBM-007) Phase 2 Clinical Trial Results: Phase 2 trial met all primary objectives; 500+ total subcutaneous doses administered with zero reported safety adverse events. The trial tested two dosing regimens: 0.3mg weekly (Cohort 1) and 0.6mg every 2 weeks (Cohort 2), with equal total drug exposure across arms. Mean 6-month height growth was +1.46cm in Cohort 1 and +1.39cm in Cohort 2, comparable to the approved competitor VOXZOGO's Phase 3 result of +1.7cm per 6 months. Responders in both arms achieved growth exceeding VOXZOGO's average. Cohort 2 (higher dose every 2 weeks) had a higher responder rate, and prior VOXZOGO non-responders who switched to RBM-007 achieved good efficacy. Long-term extension data (up to 2 years for one patient) shows sustained growth of ~3cm per year, which is expected to deliver a total 20cm gain over 7 years of treatment, potentially increasing average adult male height from 130cm to 150cm and improving quality of life.
• Retinal Disease Pipeline Progress: RBM-007 completed clinical Proof of Concept (PoC) for wet AMD in a physician-led trial of 5 patients, demonstrating non-inferiority to standard anti-VEGF treatments, with 4 out of 5 patients showing no scar fibrosis formation (an unmet need unaddressed by current standard treatments). Preclinical testing in diabetic mouse models confirmed RBM-007 significantly inhibited fundus hemorrhage progression for DME, supporting further clinical development.
• Novel DDS Technology Progress: Ribomic developed aptamer-modified lipid nanoparticle (LNP) delivery technology targeting the transferrin receptor 1 (TfR1) for brain delivery. In vivo mouse testing confirmed that encapsulated siRNA successfully reduced target mRNA concentration in a dose-dependent manner in the brain, with superior targeted nucleic acid delivery performance compared to existing leading technology (JCR-Brain Cargo). Multiple pharmaceutical companies are currently in partnership negotiations for this technology.

• RBM-007 ACH Phase 3 trial is planned to start in 2026 fiscal year and complete in 2028 fiscal year. A high 1mg/kg once-weekly dose is planned (3.3x higher total exposure than Phase 2), with PK simulation projecting annual height growth of +3cm to +4cm, which would exceed the leading Phase 3 competitor Infigratinib's +2.5cm result. Final dose setting will be confirmed by regulatory authorities by March 2026, with no current signs of pushback on the proposed dose. The trial will be a single-arm study (no placebo/active comparator) with lower age eligibility reduced from 5 to 2 years, and targets early approval under Japan's rare disease drug (ODD) designation.
• Management targets a partnership deal for RBM-007 ACH during Phase 3 development (prior to trial completion) to achieve early revenue via option or collaboration agreements, citing comparable comparable upfront deals of 15 billion yen for Infigratinib Japanese rights and 10 billion yen for TransCon CNP Japanese rights as a benchmark for potential value.
• Positive partnership announcement for the novel brain delivery DDS technology is expected in the near term.
• Positive clinical updates for the retinal disease pipeline are planned for next year's earnings call.

Ribomic is a clinical-stage biotech focused on aptamer-based drug development, with three main pipeline segments: 1) Achondroplasia (ACH) pipeline (lead candidate RBM-007/umedaptanib pegol): No revenue generated as the candidate is in late clinical development; 2) Retinal disease pipeline (RBM-007 for wet AMD and DME): No revenue, with preclinical/early clinical progress; 3) Novel DDS Aptamer Technology: No revenue, with ongoing collaborative development discussions. Total income for 2025 includes 45 million yen (4.5 million yen, 0.045 billion yen) in public research grants. Cash on hand is just over 3.3 billion yen (33亿日元 converted to 3.3 billion yen), with 0.7 billion yen (7亿日元 converted to 0.7 billion yen) raised from exercised stock warrants to date for ACH Phase 3 trials. R&D spending decreased for the RBM-007 ACH program (as manufacturing development is completed) and increased for RBM-006 (Autotaxin-targeting aptamer) GMP synthesis and formulation work.

• There is market and investor uncertainty regarding the Phase 2 trial results, which management attributes to insufficient prior communication and misunderstandings of trial design, that has not yet been fully reflected in the company's stock price.
• Phase 3 efficacy projections are based on PK simulation, and actual trial results may not meet the projected efficacy benchmarks required to secure a partnership or approval.
• Final dose approval for Phase 3 could be delayed or rejected by regulatory authorities, which would delay trial initiation.
• The company is dependent on external partnership for funding and commercialization of all pipeline candidates, and may fail to secure favorable partnership terms on timeline.

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