Question · Q4 2025
June Lee asked about the primary endpoint selection for the Phase 3 Angelman study (Bayley-IV cognition versus expressive communication), inquiring if the choice was based on higher success probability or parental priority. She also asked about the percentage of Phase 3 patients opting to roll over into the long-term extension study.
Answer
CEO Emil Kakkis explained that Bayley cognition is a fundamental activity intertwined with communication, which takes more time to develop. He noted that while expressive communication is evaluated, it was not chosen as a primary endpoint due to its heterogeneity and developmental complexities. Kakkis highlighted the Multidomain Responder Index as a broader assessment important to parents. CMO Eric Crombez added that the Phase 3 studies show a very high retention rate, with patients electing to continue in the long-term extension, similar to Phase 1/2.
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