RIBOMIC Inc. Q4 FY2025 earnings call

• Core Pipeline Development (Lead Candidate RBM-007)

  • RBM-007 for achondroplasia (ACH): Completed Phase 2a Cohort 1 testing in pediatric patients aged 5-14. No safety issues were identified across over 400 total subcutaneous administrations. The trial recorded an average +1.46 cm/year increase in growth velocity, matching the efficacy of the already approved drug VOXZOGO at RBM-007's lowest tested dose. RBM-007 recently received Orphan Drug Designation (ODD) from Japan's Ministry of Health, Labour and Welfare, qualifying the program for up to 50% coverage of clinical trial costs, expected to amount to hundreds of millions of yen in financial support. The company is currently conducting license out negotiations and preparing for late-stage trials, with a possible direct move to Phase 3 instead of an intermediate Phase 2b.
  • RBM-007 for wet age-related macular degeneration (wet AMD): Completed Phase 2 clinical trials (TOFU, RAMEN, TEMPURA studies) and established Proof of Concept (PoC). RBM-007 demonstrated non-inferior efficacy compared to the standard of care drug Eylea, and showed potential to inhibit fibrosis, an unmet need in current anti-VEGF standard treatments. Marked treatment effects were observed in 5 treatment-naïve patients. Two papers on these results were published in the journal of the Royal College of Ophthalmologists.

• Second Pipeline Candidate RBM-006

  • RBM-006 is an anti-Autotaxin aptamer being developed for fibrotic ophthalmic conditions including proliferative vitreoretinopathy (PVR), glaucoma, and diabetic retinopathy. Preclinical testing in porcine PVR models has yielded strong positive results, and ongoing analysis is being conducted via a comprehensive collaborative research agreement with the University of Tokyo Department of Ophthalmology.

• Platform Technology Advances

  • Co-development with Ajinomoto resulted in breakthrough improvements to aptamer pharmacokinetics: a new PEOZ polymer alternative to PEG for aptamer stabilization, and a novel method of conjugating antibody Fc domains directly to aptamers to extend in vivo half-life. Both new technologies have had patent applications filed, and the company expects these could become global standard technologies for nucleic acid and aptamer stabilization.
  • AI-enabled aptamer development collaboration with Waseda University produced two novel proprietary algorithms: RaptRanker for sequence sorting and RaptGen for generating novel non-experimental aptamer sequences. The company holds a leading global position in AI application for aptamer development, with applications including COVID-19 aptamer discovery.
  • New targeted DDS (Drug Delivery System) aptamer technology development is ongoing with two programs: one for photoimmunotherapy of cancer in collaboration with Tokyo Jikei University School of Medicine, and aptamer-functionalized lipid nanoparticles (LNP) for targeted drug delivery to hard-to-reach organs including the brain, lungs, and kidneys. The company is in discussions with multiple firms to advance this proprietary platform technology.

• External Partnerships & Collaborations

  • Signed a memorandum of understanding (MOU) with King Abdullah International Medical Research Center (KAIMRC) in Saudi Arabia, as part of Saudi Arabia's Vision 2030 initiative to build a leading regional advanced healthcare hub. Negotiations are ongoing to conduct Phase 3 trials of RBM-007 for wet AMD in Saudi Arabia with KAIMRC funding, plus joint aptamer research collaborations. The company aims to structure this as a mutually beneficial partnership.
  • Entered a comprehensive collaborative research agreement with the University of Tokyo Department of Ophthalmology to advance development of RBM-007 and RBM-006 for ophthalmic indications.
  • The company's 13-year endowed department (RNA Medical Science Social Collaboration Research Division) at the Institute of Medical Science, University of Tokyo concluded at the end of its term, having produced 36 academic papers and 4 patent applications.

As a clinical-stage biotech startup focused on aptamer drug development, Ribomic does not report revenue-generating product segments. Total operating expenditures for the full fiscal year were held at 1 billion yen. Cash and cash equivalents on hand total just over 3 billion yen, sufficient to support 2-3 years of ongoing operations. The company raised 690 million yen via the 17th new stock subscription rights allocation in March 2024, and also receives public research grants from NEDO to support development activities.

• Lead candidate RBM-007 for ACH: The company will continue active licensing negotiations, advance preparations for late-stage clinical development, and leverage the financial and regulatory benefits of Orphan Drug Designation to speed progress.
• RBM-007 for wet AMD: The company aims to initiate a Phase 3 trial in treatment-naïve patients in Saudi Arabia, with support from KAIMRC and the Saudi government, if partnership terms are finalized.
• RBM-006 for ophthalmic indications: The company will prioritize preclinical development and efficacy validation in collaboration with the University of Tokyo.
• Platform technology: The company will continue advancing next-generation aptamer stabilization and DDS platform technologies, with the goal of establishing these as global proprietary standards and expanding external partnering opportunities.
• Current cash on hand is sufficient to support 2-3 years of ongoing operations, with no immediate funding gaps expected.

As a clinical-stage biotech, the primary risks are standard to early drug development: potential failure of late-stage clinical trials to confirm efficacy and safety observed in early trials, delays in regulatory review and approval, difficulty finding development and commercialization partners, uncertainty around the successful execution of the planned Saudi Arabian collaboration, and competition from existing and emerging marketed drugs for ACH and wet AMD.

No question and answer section was included in the provided transcript.