ENVALITH
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RIBOMIC Inc.

4591・Growth Market・Pharmaceuticals

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RIBOMIC Inc.4591

Business

RIBOMIC Inc. is a drug discovery platform biotech venture established in 2003 based on research achievements from the Institute of Medical Science, the University of Tokyo. Utilizing its proprietary drug discovery platform technology, RiboART System®, the company conducts research and development of aptamers (a type of nucleic acid medicine) expected to become next-generation drugs following antibodies. The company focuses on ophthalmic diseases and rare diseases as priority areas, and is developing its lead pipeline candidate, umedaptanib pegol (anti-FGF2 aptamer), as a treatment for achondroplasia (ACH) and wet age-related macular degeneration (wet AMD). Its main objective is to generate revenue through licensing-out to pharmaceutical companies, while also concurrently operating a drug discovery support business. Its major customers are domestic and overseas pharmaceutical companies and research institutions, and it has a track record of licensing agreements with AJU Pharm of South Korea.

Business Model

In the Drug Discovery Business, aptamer drug candidates are developed in-house or through joint research with universities and others, generating upfront payments, milestone income, and royalties upon out-licensing to pharmaceutical companies. In the Drug Discovery Support Business, the company utilizes the "RiboART System®" to support pharmaceutical companies' drug discovery challenges, thereby securing research contract revenue and technology license fees at an early stage. By combining these two businesses, the model is designed to achieve both near-term revenue security and the creation of high-value-added revenue over the medium to long term.

Company Strengths

In the Phase 2 clinical trial of umedaptanib pegol for ACH (12 subjects), a statistically significant difference (p=0.04) was confirmed with a mean change in annualized height velocity (ΔAHV) of +1.4 cm/year, achieving clinical proof of concept. In March 2026, the company obtained approval from the PMDA to conduct a Phase 3 clinical trial (16 subjects aged 2–14 years, 52 weeks), representing clear progress in the development stage.

Substance patents for the lead pipeline asset RBM-007 (umedaptanib pegol) have been registered and maintained in 13 countries and regions, including Japan, the United States, Europe, China, South Korea, and India. RBM-011 (IL-21 aptamer) has also received patent grants in Japan and the United States. Formulation patents and use patents are likewise maintained in multiple countries, providing a solid intellectual property foundation to support license-out negotiations.

Through joint development with Waseda University, the company has established a three-stage AI aptamer evaluation technology comprising RaptRanker, RaptGen, and RaptScore, which has been published in academic journals. In addition, the company is developing DDS Aptamer technologies in three areas: photoimmunotherapy, siRNA delivery, and LNP modification. These technologies are integrated into the RiboART System®, serving as a differentiating factor for the drug discovery support business.

ENVALITH's Perspective

Total cash and securities at the end of FY2026 (ending March 2026) stood at ¥2,827 million (cash of ¥1,927 million plus securities of ¥900 million). Operating cash outflow has been expanding at an annual pace of ¥1,110 million, and an operating loss of ¥1,547 million is forecast for FY2027 (ending March 2027). Although exercises of the 19th series stock acquisition rights (allocated to EVO FUND) continue, cash consumption is expected to accelerate as Phase 3 clinical trial expenses ramp up in earnest, creating a risk that funds could be depleted within 2-3 years without additional fundraising. There is no note regarding going concern assumptions for the current period, but whether fundraising can be realized remains the most critical item to monitor.

While it is commendable that the ACH Phase 2 clinical trial confirmed a statistically significant difference of ΔAHV +1.4cm/year, the number of subjects was extremely small at 11, and reproducibility needs to be confirmed in Phase 3 (16 subjects, 52 weeks). For wet AMD, the TOFU trial failed to demonstrate efficacy superior to aflibercept monotherapy, and future development policy remains at the consideration stage, including partnerships with other companies and fundraising from funds, among other options. Business revenue for FY2026 (ending March 2026) was only ¥3 million, a slight increase from the previous fiscal year, and the realization of license income remains uncertain. The forecast business revenue for FY2027 (ending March 2027) also remains at a low level of ¥17 million.

As an external factor, the approval of IZERVAY™ (anti-complement C5 aptamer) in the US in August 2023 and in Japan in August 2025 has raised market appreciation for the aptamer modality, serving as a tailwind for the industry as a whole. The case of its originator, IVERIC Bio, being acquired by Astellas Pharma for approximately ¥800 billion in total demonstrates the high value of licensing out aptamer drugs. However, whether RIBOMIC can convert this tailwind into revenue depends entirely on the success of Phase 3 trials for umedaptanib pegol (RBM-007, anti-FGF2 aptamer) and the outcome of licensing negotiations, and at this point it remains only a potential opportunity.

Growth Strategy

Focusing on the transition of umedaptanib pegol to Phase 3 and realization of out-licensing, expanding the next-generation pipeline through AI and DDS technologies

A clinical trial application was filed with the PMDA in March 2026 and approval to conduct the trial has been obtained. A single-agent, 52-week trial is planned involving 16 pediatric patients aged 2 to 14, with subcutaneous administration of 1mg/kg once per week. Contract procedures with study sites are currently underway, and creating an out-licensing opportunity through the success of Phase 3 represents the greatest value realization scenario.

The TOFU trial confirmed a suppressive effect on scar formation. The company is aiming to demonstrate scar-suppressing effects in a clinical trial targeting treatment-naive wet AMD patients, and is considering partnerships with other companies and fundraising including from funds. Suppression of fundus hemorrhage was also confirmed in a diabetic retinopathy model, with expansion into diabetic macular edema also under consideration.

Vascular stabilizing effects were confirmed in a diabetic retinopathy model. A new substance patent was filed in January 2026, and the company succeeded in creating a novel aptamer with superior activity and shorter chain length compared to existing aptamers. Nonclinical trial and drug substance synthesis costs are planned to be recorded in FY2027 (ending March 2027), advancing preparations for the transition to clinical trials.

Drug substance synthesis and toxicity studies have been completed, and the trial is ready to enter Phase 1 clinical trial. Patents have been granted in Japan (December 2025) and the United States (March 2026), and the company is at a stage where the decision to initiate the clinical trial will be made depending on the fundraising status.

RaptScore (based on a large language model) has been established, enabling evaluation and optimization of arbitrary sequences. Three joint research projects are underway with Leadpharma, Nissan Chemical, and SK Plasma. The company is advancing relationship-building with multiple companies, including major domestic pharmaceutical companies, through business commissioning agreements and other arrangements, aiming to develop these into joint research agreements.

Regarding the 19th Series Stock Acquisition Rights issued on August 12, 2025, 14,400 units (1,440,000 shares) were exercised between April 1 and May 13, 2026, resulting in common stock of ¥933 million and additional paid-in capital of ¥5,217 million. Securing funds in preparation for the full-scale ramp-up of Phase 3 clinical trial costs is an urgent priority.

Last updated: July 19, 2026