Regeneron showcased promising results for its investigational therapies Veopoz in paroxysmal nocturnal hemoglobinuria (PNH) and Ordspono in lymphoma at a recent hematology meeting. These findings highlight Regeneron's commitment to addressing difficult-to-treat diseases through innovative scientific approaches.
Veopoz Shows Superior Disease Control in PNH
In a head-to-head Phase 3 trial against Alexion Pharmaceuticals’ Ultomiris, Regeneron's combination of Veopoz, an antibody, with an investigational siRNA drug developed with Alnylam Pharmaceuticals, demonstrated better disease control in patients with PNH. According to Dr. L. Andres Sirulnik, senior vice president of translational and clinical sciences in hematology at Regeneron, this combination leverages an antibody coupled with an siRNA to bring a differentiated approach and potential improvement in PNH treatment. The siRNA inhibits the synthesis of the C5 protein in the liver, reducing the burden on the antibody. For five patients who never achieved control on Ultomiris, four of them achieved immediate disease control with Veopoz. A pivotal study comparing the combination to Soliris, the gold standard in PNH treatment, is currently enrolling.
Ordspono Demonstrates High Response Rates in Lymphoma
Regeneron's bispecific antibody Ordspono also showed remarkable efficacy in a Phase 3 study for follicular lymphoma. In previously untreated patients, Ordspono led to a 100% complete response rate in 12 out of 12 patients who received the full dose. In the last line of treatment for follicular lymphoma, Ordspono achieved an 80% objective response rate, with 73% achieving complete remission. These results suggest that Ordspono has the potential to be a best-in-class treatment option for patients with lymphoma, potentially offering a chemotherapy-free option.
Challenges and Future Directions
While Ordspono has demonstrated impressive efficacy, safety remains a challenge, particularly in earlier lines of therapy where patients have intact immune systems. Cytokine release syndrome (CRS) was observed, but it was better tolerated in earlier lines. Regeneron is also developing other drugs for PNH, with the potential to disrupt the market with improved efficacy and convenient administration. The company's disease-based approach and focus on scientific innovation are driving its success in hematology.
