Acceleron's ACE-536 Corrects Anemia and Iron Overload in Preclinical Model

Just announced at the Conference in Cyprus.

http://pharmalive.com/News/Index.cfm?articleid=863603

Acceleron's ACE-536 Corrects Anemia and Iron Overload in Preclinical Model of Beta-Thalassemia
Positive effects also demonstrated on reducing spleen size and life-threatening iron overload in the spleen, kidney and liver
 

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Oct 25, 2012 - Acceleron Pharma, Inc., a biopharmaceutical company developing protein therapeutics for cancer and orphan diseases, presented data today at the 3rd Pan-European Conference on Hemoglobinopathies and Rare Anemias in Limassol, Cyprus, demonstrating positive effects of ACE-536 to treat both the severe anemia and serious organ damage due to iron overload in a preclinical model of beta-thalassemia. Acceleron is developing ACE-536 in collaboration with Celgene.

Beta-thalassemias are a group of hereditary blood disorders characterized by anemia that results from defective hemoglobin beta chain synthesis and ineffective red blood cell (RBC) production. Hemoglobin is the iron-containing molecule in red blood cells that carries oxygen to cells throughout the body. Depending on disease severity and treatment, beta-thalassemic individuals often experience pale skin, weakness, fatigue as well as additional serious health effects, including splenomegaly (enlargement of the spleen), reduced growth, skeletal changes that result from expansion of the bone marrow and organ damage due to iron overload.

In a preclinical mouse model of beta-thalassemia intermedia, ACE-536 significantly increased levels of RBCs and hemoglobin by promoting the differentiation of late stage RBC precursors. Consistent with its ability to correct ineffective RBC production and anemia, ACE-536 reduced levels of erythropoietin (EPO) in the blood. ACE-536 also substantially reduced the size of the enlarged spleens of the beta thalassemic mice. Additionally, ACE-536 restored iron metabolism as evidenced by reduced serum ferritin and increased hepcidin levels, leading to reduced iron deposition in the spleen, liver and kidney.

“These beta-thalassemic mice are an excellent preclinical model of beta-thalassemia, reproducing the numerous, life-threatening clinical complications experienced by patients who suffer from this disease”, said Ravi Kumar, Ph.D., Chief Scientific Officer of Acceleron. “We are excited by these data which show the potential of ACE-536 to substantially alleviate the anemia and iron overload characteristic of the disease and look forward to initiating a Phase 2 trial of ACE-536 in patients with beta-thalassemia in the next few months.”

About ACE-536

ACE-536 is a modified type II activin receptor fusion protein that acts as a ligand trap for members in the TGF-β superfamily involved in late stages of erythropoiesis. ACE-536 promotes late-stage erythrocyte precursor cell differentiation, distinct from erythropoietin (EPO) which acts during early, proliferative stages of erythropoiesis. This mechanism of action to increase red blood cells has the potential to treat anemia in diseases such as Myelodysplastic Syndromes (MDS) and beta-Thalassemia that are poorly managed with current therapy.

About Acceleron

Acceleron is a privately-held biopharmaceutical company committed to discover, develop, manufacture and commercialize novel protein therapeutics for orphan diseases and cancer. Acceleron's scientific approach takes advantage of its unique insight to discover first-in-class therapies based on the TGF-β protein superfamily. Acceleron utilizes proven biotherapeutic technologies and capitalizes on the company's internal GMP manufacturing capability to advance its therapeutic programs rapidly and efficiently. The investors in Acceleron include Advanced Technology Ventures, Alkermes, Avalon Ventures, Bessemer Ventures, Celgene, Flagship Ventures, MPM BioEquities, OrbiMed Advisors, Polaris Ventures, QVT Financial, Sutter Hill Ventures and Venrock. For further information on Acceleron, please visit www.acceleronpharma.com.

 

Contact: Acceleron Pharma:
Steven Ertel, 617-649-9234
Chief Business Officer
or
Suda Communications LLC
Maureen L. Suda, 585-387-9248 (Media)

Our moderator, Manal is attending this conference. I hope she was able to see this presentation. Manal also send hers regards.

Wow Andy, this is the best news of the year that you gave.

Are there any trials going to be happening anytime soon on humans (Thal majors)?

I am excited to hear more because as you said that this medicine while increasing RBC (which in turn increases Hb levels) also decreases the deposition of iron in vital organs.

So glad that someone is spending their time behind improving our lives.

God bless them.

Also thanks a lot to Manal for attending this conference and letting us know, my keen regards to Manal, as well.

-P.

Andy,

This is amazing!  It sounds like this can be potentially helpful to all thals!  Would beta zero thals be able to benefit from this - or would the patient need to have some ability to produce red cells? 

This would be of great interest to Manal -- but I haven't heard from her in a very long time, I hope that she and her family are well.

Sharmin

Yes I believe that thal majors will also benefit from this, considering as they're stating such as "people with high iron overload" which is of course in thal majors and other transfused patients (unless you're stating thal minor? which I also believe is benefited from this drug).

On a side note, I've also contacted them today in the morning to their clinical trial department from the given email address on their website for more information. If I hear anything from them, I shall update it here.

-P.

I wish I could answer the questions, but I only know what the article says. I am not at the conference to ask the questions, and it looks doubtful that conferences will ever be in my future again.