New drug for anemia in the pipeline

[Birgitta-A]

Hi All,
A new drug for anemia is now tested in a Phase II trial:
http://www.checkorphan.org/grid/news...stic-syndromes
Kind regards
Birgitta-A

[Neil Cuadra]

The Acceleron press release has these quotes from the investigators:

The anemia experienced by patients with MDS is clinically challenging because it is often unresponsive to administration of erythropoietin and many patients ultimately require red blood cell transfusions.

ACE-536 has the potential to make a significant impact on the treatment of anemia in MDS. Unlike erythropoietin, ACE-536 may target the specific defect in the erythropoietic maturation process in MDS patients and we are optimistic that it could become an important new therapeutic option for this underserved patient population.

An additional bit of information from the Acceleron product page:

In preclinical studies, ACE-536 promoted RBC formation in the absence of erythropoietin (EPO) signaling, had distinct effects from EPO on RBC differentiation, and acted on a different population of progenitor blood cells than EPO during RBC development.

[riccd2001]

Thanks Birgitta and Neil for the links. There's still more to do but these are very encouraging words in the PR...

"In a phase 1 clinical study of healthy volunteers, ACE-536 produced a dose-dependent increase in red blood cells and hemoglobin levels. "

[riccd2001]

Very good to see progress with FDA classification for ACE-536 !!

http://www.acceleronpharma.com/2013/...s-for-ace-536/