Narsoplimab Demonstrates Significant Survival Benefit in Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy

Narsoplimab Efficacy:
Narsoplimab, a MASP-2 inhibitor, has shown a 68% improvement in overall survival in patients with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA)125.

Clinical Trial Results:
The pivotal clinical trial demonstrated a 61% complete response rate and a 100-day survival rate of 68% in the full analysis set, with 83% survival in the per-protocol population and 94% in complete responders5.

Regulatory Status:
The FDA granted Priority Review to narsoplimab for the treatment of HSCT-TMA, highlighting its potential as a breakthrough therapy2.

Mechanism of Action:
Narsoplimab inhibits MASP-2, the effector enzyme of the lectin pathway of the complement system, without interfering with the classical complement pathway, which is critical for the acquired immune response to infection1.

Future Developments:
Omeros is advancing a diagnostic kit to identify patients with acute and long COVID who could benefit from narsoplimab treatment, and is also developing small-molecule inhibitors of MASP-2 and MASP-3 as part of lifecycle planning1.

Sources:

1. https://www.omeros.com/pipeline/

2. https://www.targetedonc.com/view/fda-grants-priority-review-to-narsoplimab-for-treatment-of-post-transplant-complications

5. https://investor.omeros.com/news-releases/news-release-details/omeros-reports-final-efficacy-and-safety-data-narsoplimab