RIBOMIC Inc.
4591・Growth Market・Pharmaceuticals
RIBOMIC Inc. (Drug Discovery Business, Single Segment)
Biotech venture with a drug discovery platform specialized in aptamer nucleic acid medicines
| Period | Current | Previous | Change |
|---|---|---|---|
| Business revenue | ¥3 million | ¥2 million | ↑ |
| Operating loss | -¥1,207 million | -¥1,050 million | ↓ |
| Ordinary loss | -¥1,138 million | -¥1,014 million | ↓ |
| Net loss for the period | -¥1,145 million | -¥1,018 million | ↓ |
| Research and development expenses | ¥803 million | ¥667 million | ↓ |
| Selling, general and administrative expenses | ¥406 million | ¥385 million | ↓ |
| Total assets | ¥2,976 million | ¥3,185 million | ↓ |
| Net assets | ¥2,841 million | ¥3,043 million | ↓ |
| Equity ratio | 95.4% | 95.5% | — |
| Cash and cash equivalents at end of period | ¥1,927 million | ¥1,837 million | ↑ |
| Securities (bonds held to maturity) | ¥900 million | ¥1,200 million | ↓ |
| Total liquid assets (cash + securities) | ¥2,827 million | ¥3,037 million | ↓ |
| Net assets per share | ¥52.30 | ¥68.26 | ↓ |
| Net loss per share for the period | -¥23.07 | -¥25.21 | ↑ |
| Number of shares issued at end of period | 54,332,640 shares | 44,613,940 shares | ↓ |
| Cash flow from operating activities | -¥1,110 million | -¥996 million | ↓ |
Business Details
Utilizing its proprietary drug discovery platform "RiboART System®," the company conducts research and development of aptamers (a type of nucleic acid medicine), which are expected to be next-generation drugs following antibodies. Its focus areas are ophthalmic diseases and rare diseases. The business model rests on two pillars: licensing out its own drug discovery products (upfront payments, milestones, royalties) and joint research income with pharmaceutical companies. The company is currently in an upfront-investment stage, with business revenue near zero and continuing losses.
Recent Overview
Phase 2 ACH trial confirmed POC-supporting results; the Phase 3 clinical trial application and its approval represent the most significant progress
In March 2026, statistical analysis of the ACH Phase 2 clinical trial was completed, confirming efficacy with a mean ΔAHV of +1.4 cm/year (p=0.04) and a favorable safety profile. In the same month, the company filed a clinical trial application for a Phase 3 trial with the PMDA and obtained approval to conduct it, putting it in a position to start the clinical trial at any time from a regulatory standpoint. In May 2025, the company obtained orphan drug designation (ODD) and received subsidy income of ¥39,190 thousand during the period. All of the 18th series stock acquisition rights were exercised by December 10, 2025, raising ¥921 million. For FY2027 (ending March 2027), the company expects an operating loss of -¥1,547 million due to Phase 3 clinical trial costs and other expenses.
Key Products
Growth Drivers
- Expansion of licensing-out opportunities through the initiation and progress of the Phase 3 clinical trial for umedaptanib pegol in ACH (PMDA approval to conduct obtained; 16 subjects aged 2–14, 52 weeks)
- Utilization of PMDA preferential measures and accelerated development through orphan drug designation (ODD), along with continued subsidy income
- Expansion of indications to wet AMD, diabetic retinopathy, and diabetic macular edema (clinical POC already obtained; potential for differentiation as a first-line treatment and for scar suppression)
- Improved R&D efficiency through integration of AI drug discovery technologies such as RaptScore into the "RiboART System®"
- Early monetization through partnership and licensing agreements with pharmaceutical companies in the DDS Aptamer Business (photoimmunotherapy, siRNA delivery, LNP modification)
- Accumulation of drug discovery support income through three ongoing joint research projects with Leadpharma, Nissan Chemical, and SK Plasma
- Additional fundraising through exercise of the 19th series stock acquisition rights (for EVO FUND) (1,440,000 shares exercised as of May 13, 2026)
Risks
- Continuation of the upfront-investment stage with business revenue near zero, creating a risk of funding depletion if licensing-out is not realized (FY2027 (ending March 2027) operating loss forecast of -¥1,547 million; liquid assets of ¥2,827 million)
- Risk of failure or delay in the ACH Phase 3 clinical trial (the Phase 2 trial was an exploratory analysis, and efficacy confirmation in Phase 3 is essential)
- In wet AMD development, efficacy exceeding that of aflibercept monotherapy has not been demonstrated, and clinical proof of the differentiation strategy (first-line use, scar suppression) has not yet been achieved, requiring partnerships with other companies or fundraising from funds and other sources
- Dilution risk from exercise of the 19th series stock acquisition rights (22,000,000 potential shares; 55,772,640 shares issued as of May 13, 2026)
- Concentration risk from reliance on a single segment and single modality (specialization in aptamer drug discovery)
- Risk of renewed expansion of R&D expenses associated with the transition to Phase 3 clinical trials (total business expenses for FY2027 (ending March 2027) forecast at ¥1,565 million, an increase of ¥355 million year on year)
- Uncertainty in pipeline progress, as exemplified by the difficulty in establishing an effective animal model for the PVR indication of RBM-006, leading to the termination of joint research with Nihon University in May 2025
Last updated: June 19, 2026