The Atypical HUS Foundation

Ra Pharma launches Phase 1 study of RA101495

December 2, 2015

     Ra Pharmaceuticals announced today the launch of a Phase 1 study of its C5 complement inhibitor RA101495.  From the Ra Pharmaceutical press release, “Ra Pharma is developing RA101495 for paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and other debilitating conditions. The product is designed for convenient, subcutaneous self-administration.”  This first step in the clinical development of RA101495 will focus on in-human study to help determine suitable dosing for treating patients within the paroxysmal nocturnal hemoglobinuria (PNH) patient population, which is a rare disease with similar red blood cell destruction as in aHUS patients.

     In just a few days clinicians and researchers in the field of hematology will gather for 57th American Society of Hematology Annual Meeting and Exposition (ASH), taking place December 5-8, 2014 in Orlando, Florida.  Ra Pharmaceuticals will be presenting preclinical data regarding C5 inhibition related to its suitability as treatment for various complement-mediated disease.   Details on the presentations at ASH are as follows:

Abstract Title: Preclinical Evaluation of RA101495, a Potent Cyclic Peptide Inhibitor of C5 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (#939)  Session 101, Saturday 12/5

Abstract Title: Complement C5 Inhibition Blocks the Cytokine Storm and Consumptive Coagulopathy By Decreasing Lipopolysaccharide (LPS) Release in E. coli Sepsis (#765)  Session 331, Monday 12/7

     The Atypical HUS Foundation is hosting an informational booth at this ASH event, to provide an outreach opportunity that expresses the voice and issues of aHUS patients, families, and caregivers.  We look forward to hearing more about potential new treatments that may become available in the future for aHUS patients, and to reporting that information to the aHUS community as events unfold in the drug development landscape.

 

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Comment by Cheryl Biermann on December 3, 2015 at 11:08am
Hey folks, please share this kind of. Info. With your specialists. Then they see you as competent, proactive and informed, which of course, you are. Plus, in some areas thus information lags and you are doing a service. In addition, this keeps aHUS at the forefront of thoughts in the medical world, which is a profound help to new cases presenting themselves in doctors' offices and ER rooms.

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The Atypical HUS Foundation encourages patients and investigators to share information and explore options/resources as we work together to gain insight into this rare complement disorder. By increasing contact opportunities with researchers and medical personnel interested in helping the aHUS community, our stories foster a better understanding of atypical hemolytic uremic syndrome.

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NEW DIAGNOSIS OF aHUS?
Be proactive! Get the medical basics of aHUS, what lab values to monitor, and areas of concern...check out the "aHUS Bootcamp" and "About aHUS" tabs at the top of this page!
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Did you know...

CFH (Serum Complement Factor H) is a regulatory protein. The secreted protein product of CFH consists of 20 repetitive units named "short consensus repeats" or SCRs (each approximately 60 amino acids). In patients with aHUS the last 5 "pearls" in the twenty pearl strand protein, SCR16 - SCR20, should bind to protect cells but do not- they are defective in one or more of the last 5 SCR locations. If they cannot bind or stick to the kidney to protect that tissue, the platelets clump into clots that affect the glomeruli of the kidney -potentially causing acute renal failure.
  
• • • • • • • • • • • •
  
It is estimated that there are about 2 cases of aHUS in the U.S. per 1,000,000 of population, and about 60% of aHUS patients are diagnosed as children. The condition is potentially life threatening, and either can be chronic or can recur at intervals.
  
more factoids...

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