PharmaEssentia Reports Positive Phase 3 Results for Ropeginterferon Alfa-2b in Essential Thrombocythemia Study

PharmaEssentia Corporation, a global leader in biopharmaceutical innovations, recently revealed promising results from its SURPASS-ET clinical trial. This trial evaluated the efficacy and safety of ropeginterferon alfa-2b-njft (P1101) for treating patients with essential thrombocythemia (ET), a rare blood disorder. The findings demonstrated that P1101 achieved its primary endpoint with significant clinical responses and a manageable safety profile.

P1101, marketed under the name BESREMi®, is already approved by the FDA for polycythemia vera (PV), and PharmaEssentia plans to expand its use to include ET. The SURPASS-ET trial, conducted globally, involved 174 participants who were randomized to receive either P1101 or Anagrelide as a second-line therapy over a 12-month period. The trial showed that 42.9% of patients in the P1101 group achieved durable responses at 9 and 12 months, compared to just 6.0% in the Anagrelide group, marking a significant advance in the treatment of ET.

Secondary outcomes of the trial focused on the JAK2 V617F allelic burden, a genetic marker associated with ET. Results showed a greater reduction in JAK2 allelic burden in the P1101 group than in the Anagrelide group, suggesting P1101’s potential to better address the underlying disease biology.

Dr. Ko-Chung Lin, the founder and CEO of PharmaEssentia, expressed pride in the trial's outcomes, emphasizing the potential of P1101 as a novel treatment for ET. By utilizing PharmaEssentia’s monopegylated, long-acting interferon technology, P1101 represents a shift towards non-chemotherapy treatments for myeloproliferative neoplasms like ET. The company aims to use these results to expand P1101's label and meet a significant global medical need.

In line with these results, PharmaEssentia plans to engage with the FDA for regulatory discussions to expand the existing label of P1101 for ET treatment. They anticipate submitting necessary documentation by the end of 2025. Albert Qin, Chief Medical Officer, highlighted the importance of the results, noting that ET poses significant treatment challenges due to its symptoms and risks. The positive findings from SURPASS-ET offer hope for a more effective and tolerable treatment option for ET patients.

In addition to the SURPASS-ET trial, PharmaEssentia is conducting the EXCEED-ET trial in North America. This Phase 2b study also evaluates P1101's efficacy and safety in adults with ET, with data expected to be presented by late 2025.

Essential thrombocythemia is a chronic condition characterized by excessive production of platelets due to genetic mutations, leading to increased risks of stroke, heart attack, and other complications. Affecting around 148,000 individuals in the U.S., ET significantly impacts patients' quality of life. Current treatment options are limited, underscoring the importance of new therapies like P1101 that can address thrombosis risk and disease progression.

BESREMi® (ropeginterferon alfa-2b-njft), approved for polycythemia vera, is known for its long-acting interferon formulation. It provides flexible dosing options and has been approved in over 40 countries, including the U.S., Europe, and Japan. PharmaEssentia retains global rights to BESREMi and produces it in its Taichung facility, which meets international regulatory standards.

PharmaEssentia, founded in 2003, is based in Taipei, Taiwan, and is expanding its global presence with operations in multiple regions. The company continues to leverage its scientific expertise to bring forward innovative treatments in hematology, oncology, and immunology.