Jarwei Fang

Jarwei Fang asked if the early U.S. trends for Sephience, such as capturing 'lost to follow-up' patients and social media awareness, are expected to be similar in ex-U.S. geographies like Japan and Brazil. He also inquired about what the remaining 20% of U.S. Centers of Excellence that have not yet prescribed Sephience need to see to get on board.

Answer

Matthew Klein, CEO, stated it's early to tell if all trends will be identical globally, but noted that patients worldwide are networked through social media and patient organizations, driving awareness. He acknowledged that country-specific dynamics (e.g., concentrated centers in Japan) may differ, but PTC's experienced teams understand these regional nuances. Eric Pauwels, Chief Business Officer, added that for the remaining U.S. centers, ongoing medical education, peer-to-peer programs, newly published data, and increasing patient demand driven by social media testimonies are expected to accelerate adoption.

Jarwei Fang asked if the early Sephience launch trends observed in the U.S., such as capturing 'lost to follow-up' patients, early start trends, and social media awareness, are expected to be similar in ex-U.S. geographies like Japan and Brazil, or if it's too early to tell. She also inquired about what the remaining 20% of U.S. centers of excellence that have not yet prescribed Sephience need to see to get on board.

Answer

CEO Dr. Matthew Klein stated that while it's early to tell, global patient networks and social media are common, driving awareness and shared success stories. He noted that country-specific dynamics, such as patient attachment to centers, may differ. Chief Business Officer Eric Pauwels added that for the remaining U.S. centers, PTC is conducting medical education, peer-to-peer programs, and leveraging newly published data, with patient demand and testimonies expected to accelerate adoption.

Jarwei Fang, on behalf of Jeff Meacham, asked about overcoming the lack of an ICD-10 code to demonstrate the PBH market need to payers and about the level of patient community awareness for the PSP and Wolfram syndrome trials.

Answer

Co-CEO & Co-Founder Josh Cohen noted that work on an ICD-10 code is active and that claims analysis supports their market estimates. He highlighted the significant unmet need conveyed by physicians. Chief Medical Officer Dr. Camille Bedrosian added that the PSP patient community is very supportive, and Cohen mentioned growing awareness and referrals for Wolfram syndrome.