Question · Q4 2025
Joe Heidt asked for details on the Votoplam Phase III study design, potential approval pathways, and scenarios that could enable approval prior to the primary readout. He also requested a comment on Sephience's net pricing expectations.
Answer
Matthew Klein, CEO, described the INVEST-HD Phase III study as a placebo-controlled, 3:2 randomization trial targeting 770 participants across 30+ countries, with a primary endpoint of change in cUHDRS. He noted an interim analysis for efficacy and futility could potentially lead to earlier approval. Pierre Gravier, CFO, stated that Sephience's gross-to-net is expected to be at the lower end of the 15%-25% range initially, gradually increasing over time, but not reaching the top end this year, given that two-thirds of patients are covered by commercial plans.
Ask follow-up questions
Fintool can predict
PTCT's earnings beat/miss a week before the call