Regeneron’s combination therapy of Veopoz and the siRNA drug developed with Alnylam Pharmaceuticals has shown promising results in achieving better disease control for patients with paroxysmal nocturnal hemoglobinuria (PNH) when compared to Alexion Pharmaceuticals’ standard-of-care medicine, Ultomiris. This is a significant achievement for Regeneron, as Alexion has been a dominant player in the PNH market for a long time.
Dr. L. Andres Sirulnik, senior vice president of translational and clinical sciences in hematology at Regeneron, attributes the company’s success to its science-driven approach and focus on understanding the biology of diseases. Regeneron’s unique approach to drug development involves using different methodologies and technologies to address challenging medical problems. In the case of complement inhibitors for PNH, Regeneron has combined an antibody with an siRNA to achieve a more constant, deeper, and prolonged inhibition of the target protein, leading to improved disease control.
In addition to their success in PNH, Regeneron has also made significant progress in the treatment of follicular lymphoma with their bispecific antibody, Ordspono. A phase 3 study of Ordspono resulted in a complete response in all 12 previously untreated patients, setting the stage for further clinical trials and potential head-to-head comparisons with existing lymphoma treatments.
Regeneron’s innovative approach to drug development, focus on the underlying biology of diseases, and willingness to explore new technologies have led to these groundbreaking achievements. By staying true to their scientific roots and continuing to push the boundaries of medical research, Regeneron is poised to disrupt treatment paradigms and bring new hope to patients with challenging medical conditions.
Looking ahead to the future, we are excited to introduce a potentially groundbreaking drug that could revolutionize the PNH market. With a more convenient method of administration and promising results in clinical trials, we believe that this new drug has the potential to disrupt the current landscape of PNH treatment. In addition to this upcoming release, we also have other drugs in development that have not yet been disclosed, but we anticipate they will have a significant impact on the PNH market as well.
Shifting our focus to lymphoma, let’s discuss the results of the bispecific Ordspono that we will be presenting at ASH. What sets this drug apart and what challenges do we face moving forward?
Our research in lymphoma aims to address key scientific questions that could potentially reshape the way we treat this disease. The efficacy of Ordspono, particularly in follicular lymphoma, has been nothing short of remarkable. In the last line of treatment for follicular lymphoma, all 12 patients who received the full dose experienced a complete response, with over 75% achieving complete remission. This level of efficacy is truly exceptional and offers a chemotherapy-free option for patients.
One of the challenges we face is ensuring the safety of the drug, especially as we consider moving it to earlier lines of therapy where patients have a more intact immune system. In later lines of treatment, we observed cytokine release syndrome, but we are seeing improved permeability and reduced cytokine release with a single agent in earlier stages of the disease.
Additionally, the lymphoma market is highly competitive, but based on our promising results in late lines of therapy, we believe that Ordspono has the potential to be a best-in-class treatment for follicular lymphoma.
With our commitment to innovation and cutting-edge research, we are confident that our upcoming drug releases and ongoing clinical trials will continue to push the boundaries of what is possible in the treatment of PNH and lymphoma. Stay tuned for more updates on our progress in the coming months.
