Announced details
Treatment of pediatric patients with achondroplasia (ACH) aged 2–14 years
Safety margin of at least 15-fold. Cumulative administration experience in pediatric ACH patients: more than 600 administrations
Administration experience in pediatric ACH patients (more than 600 administrations in total); 5 subjects had begun home administration in the preceding Phase 2 long-term administration trial (as of 28 November 2025)
Discussing the design of the Phase 3 clinical trial
An independent administrative agency and public institution responsible for the review and safety measures of pharmaceuticals and other products. Other information, including its location and representative, is not disclosed.
Clinical trial protocol number RBM-007CS-ACH05
Target enrollment: 16 cases
No impact on full-year earnings for the fiscal year ending March 2027
The company does not address Contract Value / Offering price.
Nonclinical studies; domestic Phase 1 clinical trial; preceding Phase 2 clinical trial and long-term administration trial; long-term intermittent-dose toxicity study in juvenile cynomolgus monkeys
| Item | Details |
|---|---|
| Names of Products / Services Covered | umedaptanib pegol (anti-FGF2 aptamer) |
| Counterparty | Pharmaceuticals and Medical Devices Agency (PMDA) |
| Counterparty overview | An independent administrative agency and public institution responsible for the review and safety measures of pharmaceuticals and other products. Other information, including its location and representative, is not disclosed. |
| Relationship with the Company | Discussing the design of the Phase 3 clinical trial |
| Launch date | May 2026–April 2028 (planned trial period) |
| Applicable area | Treatment of pediatric patients with achondroplasia (ACH) aged 2–14 years |
| Registration number and expiry date | Clinical trial protocol number RBM-007CS-ACH05 |
| Location scale | Target enrollment: 16 cases |
| Quantitative Effects | Safety margin of at least 15-fold. Cumulative administration experience in pediatric ACH patients: more than 600 administrations |
| Usage history | Administration experience in pediatric ACH patients (more than 600 administrations in total); 5 subjects had begun home administration in the preceding Phase 2 long-term administration trial (as of 28 November 2025) |
| Impact on Business Performance | No impact on full-year earnings for the fiscal year ending March 2027 |
| Source | Nonclinical studies; domestic Phase 1 clinical trial; preceding Phase 2 clinical trial and long-term administration trial; long-term intermittent-dose toxicity study in juvenile cynomolgus monkeys |
Items this document does not state (6)
These are items the company does not state in this document. We do not leave them blank, nor fill them in from other sources or by inference.
The reasons the company gives
The company's own explanation, not something inferred from the numbers.Numerous inquiries were received from shareholders and investors regarding the detailed trial design and safety. The company therefore explained the rationale for the trial design, its approach to safety risks, and its management policy.