Announced details
Achondroplasia treatment, the FGF-FGFR growth factor axis, genetic diseases and regenerative medicine
Difference in mean annualized height velocity: +1.43 cm/year; proportion with increased AHV: 72.7% (8 of 11 cases); comparative response rate versus Voxzogo: 72.7% vs. 50%; AHV improved in 2 of 3 patients with prior Voxzogo treatment
11 cases included in the Phase II clinical trial analysis, excluding 1 case with fewer than 50% of the specified number of doses administered
The Company's Representative Director and President has been invited to give a presentation.
A biennial international research meeting held in St Julian's, Malta, covering embryology, genetic diseases, tissue homeostasis, tissue regeneration and related fields. It provides a forum for researchers from academia and industry, clinicians and other experts to exchange views and engage in discussion.
St Julian's, Malta (venue for Fusion Conference 2026)
No impact on full-year results for the fiscal year ending March 2027
The company does not address Contract Value / Offering price.
Savarirayan R, Tofts L, Irving M, et al., Lancet 2020; 396: 684–92 (comparative values for Voxzogo)
| Item | Details |
|---|---|
| Names of Products / Services Covered | umedaptanib pegol |
| Counterparty | Fusion Conference 2026 |
| Counterparty overview | A biennial international research meeting held in St Julian's, Malta, covering embryology, genetic diseases, tissue homeostasis, tissue regeneration and related fields. It provides a forum for researchers from academia and industry, clinicians and other experts to exchange views and engage in discussion. |
| Relationship with the Company | The Company's Representative Director and President has been invited to give a presentation. |
| Launch date | 9 May 2026 (scheduled presentation date) |
| Applicable area | Achondroplasia treatment, the FGF-FGFR growth factor axis, genetic diseases and regenerative medicine |
| Location | St Julian's, Malta (venue for Fusion Conference 2026) |
| Quantitative Effects | Difference in mean annualized height velocity: +1.43 cm/year; proportion with increased AHV: 72.7% (8 of 11 cases); comparative response rate versus Voxzogo: 72.7% vs. 50%; AHV improved in 2 of 3 patients with prior Voxzogo treatment |
| Usage history | 11 cases included in the Phase II clinical trial analysis, excluding 1 case with fewer than 50% of the specified number of doses administered |
| Impact on Business Performance | No impact on full-year results for the fiscal year ending March 2027 |
| Source | Savarirayan R, Tofts L, Irving M, et al., Lancet 2020; 396: 684–92 (comparative values for Voxzogo) |
Items this document does not state (7)
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.To present at Fusion Conference 2026 and facilitate in-depth expert discussion on the research's significance and future potential in the treatment of achondroplasia.