Maxwell Score

“So beyond confirming powering, would the SSRE outcome change how you plan for RCC overall, for example, the target patient segment, phase three sizing, or whether to partner the program?”

“Could you expand a bit more on the profile of potentially eligible NHCM patients? For example, what share of those diagnosed would meet Acacia's entry criteria, and is that kind of the right starting point for sizing the market?”

“So, longer term, could you help us understand directionally where we can expect R&D spend to go? And then just one quick follow-up, if I could, on Angelman, can you provide any additional information on what drove the miss?”

“Regarding Votoplank, would an accelerated approval path change invests the Phase III design or enrollment, given that patients may be less willing to accept placebo if a commercial drug is available?”

“Just following up on the comorbid COPD and asthma opportunity, identifying these patients seem to be one piece of the puzzle. Maybe you can elaborate a bit more on how AI is contributing to that effort.”