PharmaEssentia (藥華藥) announced on the 11th that its long-acting interferon, Ropeginterferon alfa-2b (Ropeg), achieved positive results in the EXCEED-ET trial—a North American single-arm study encompassing a broad spectrum of Essential Thrombocythemia (ET) patients. The data demonstrates robust efficacy across the total ET population, with a particularly significant response in treatment-naïve (first-line) patients. This success lays a foundation for expanding Ropeg's Total Addressable Market (TAM) beyond its current Polycythemia Vera (PV) indication.

Clinical Landscape & Competitive Edge:

• Current Standards: Clinical management of ET relies heavily on off-label Hydroxyurea (HU), though ~20% of patients are intolerant or resistant. The only FDA-approved second-line option is Anagrelide (ANA), approved in 1997, which often faces tolerability issues.
• EXCEED-ET vs. SURPASS-ET: While the global Phase 3 SURPASS-ET study (comparing Ropeg to ANA) already showed superior clinical response (42.9% vs. 6.0%, p=0.0001), EXCEED-ET serves as confirmatory evidence using a high-dose titration regimen (250-350-500 mcg).
• Key Data Points: EXCEED-ET reported a response rate of 60.2% at months 10 and 13. Notably, the response rate for first-line patients reached 68.0%, compared to 33.4% for those previously treated with HU. Additionally, 100% of patients remained free from thromboembolic or hemorrhagic events during the evaluation period.

CEO Dr. Ko-Chung Lin noted that the US FDA has recognized SURPASS-ET as the pivotal study and EXCEED-ET as supportive evidence. The company anticipates receiving ET approvals in Taiwan, the US, Japan, and China in 2026, positioning ET as the "second engine" for corporate growth.

Resource: 藥華藥Ropeg擴大ET戰線再報捷 北美全族群試驗奏效、一線病患反應更亮眼