2Q26 Update: Sephience-Driven Increase in FY Sales

Research evidence excerpt

2Q26 Update: Sephience-Driven Increase in FY Sales

UpdateMof the DMD franchise. Meaningful revenue for these products was maintained in

2Q26 despite continued generic competition for Emflaza and expiration of the

Translarna conditional marketing authorization in the EU. Management also noted a

government purchase order from Russia for Translarna during the quarter.

US Sephience growth solid, ex-US contribution expected to increase well into

2027. As of the end of 2Q, there were 1,647 patients on commercial Sephience

globally. US and ex-US Sephience revenue for 2Q26 was $127.6M and $23.8M,

respectively. Within the US, management noted that 100% of previously identified

PKU centers of excellence have now prescribed Sephience (in addition to other

centers). Uptake remains broad across patients of different ages, severities, and

treatment histories, with adoption supported not only by Phe reductions and diet

liberalization, but also cognitive and QoL benefits. Management noted a

discontinuation rate of ~20%, which is believed to be near steady state (and

somewhat below management's expectations heading into the launch). The focus for

the US launch going forward will be on achieving deeper penetration into each

center of excellence. Momentum is continuing to build ex-US as well, with JPN sales

ramping in 2Q reflecting similarly broad uptake and pricing on par with the US. In

the EU, PTC is starting expanded access programs in a number of countries. In

France, the HTA assessment has been completed and the early access program was

approved in Q2. PTC is also seeing accelerated demand in Italy and Spain and is also

leveraging early access programs in Latin America and the Middle East. Management