Agios' top-notch results to grant relief from sickle cell disease

Announcement

Agios Pharmaceuticals, Inc., a marketing-level biopharmaceutical company attentive on serving the best innovative medicines for patient groups with rare diseases, have unveiled its promising top results from the long-term 52-week global RISE UP Phase 3 trial of an oral pyruvate kinase (PK) activator, mitapivat, in patients aged ranging from 16 years or older suffering from sickle cell disease.

The RISE UP was engineered with the two initial endpoints and five major secondary endpoints to further examine the objective volume of hemolysis enhancement and integrated parameters of sickle cell disease. The robust design allows an extensive examination of the best benefit of mitapivat throughout various aspects of the disease.

The trial successfully reached its primary endpoint of haemoglobin response along with the mitapivat, demonstrating a measured prominent improvement as compared to placebo. Mitapivat showed a fall in the primary endpoint of yearly volume of sickle cell pain crises (SCPCs) as compared to placebo, as this trend didn’t meet the statistical excellence.

Additional specification of Mitapivat

The mitapivat described a statistically best-in-class improvement in the first two major secondary endpoints. The drastic change from Week 24 to Week 52 regarding its ground study in haemoglobin concentration and stages of indirect bilirubin, an outline of hemolysis in comparison to placebo. The third crucial secondary endpoint of Week 24 stretched through Week 52 showed a normal fluctuation from baseline on Patient Reported Outcome Measurement Information System Fatigue 13a (PROMIS Fatigue) was not achieved.

The patient group under the mitapivat arm that qualified for haemoglobin response also witnessed a clinically valuable benefit in the endpoints of overall rate of SCPCs, PROMIS Fatigue and annualised rate of hospitalisations for SCPCs (subjected to the fourth major endpoint of the trial). Overall, this analysis is based on a post hoc analysis.

MD, Professor of Medicine and American Red Cross Endowed Chair in Transfusion Medicine, University of Connecticut Health and a RISE UP trial investigator, Biree Andemariam, said, “Sickle cell disease is a crucial and destructive condition that has a very limited space for innovation. Due to this, the patient group doesn’t have effective, valuable treatments that lead to short lifespans and poor outcomes. The data from RISE UP elaborates that treatment, along with mitapivat, prominently enhanced haemoglobin concentration and mitigated hemolysis.”

Biree added, “These topline results are bound with a commendable safety profile, contributing to the potential of mitapivat as a novel treatment alternative for sickle cell disease patients.”