The Atypical HUS Foundation

Drug Development Overview: aHUS Community Looks for Advancements in 2016

     As 2015 draws to a close, it seems likely that 2016 will bring progress in complement therapeutics development toward clinical use.  Since a hallmark of aHUS is chronic, uncontrolled complement activation, we’re looking forward to what happens in a rapidly moving field which seems promise advancement soon.  (FMI Click Here) As with eculizumab (Soliris ®, from Alexion Pharmaceuticals), we likely will see clinical trials for PNH patients prior to involvement of the aHUS patient population.

     To keep pace with what seems to be a drug development landscape that is rapidly changing,  the 2015 aHUS Alliance meeting in London produced the first overview of the expanding drug landscape in 2016 , with differences in anti-complement agent approaches (monoclonal antibody, small molecule, peptide, protein or biologic, or nucleic acid-based therapy).  Differences in drug delivery also seem in the works, from intravenous to subcutaneous (self-administered shots) and mention of an oral delivery method for patients as well.  In the article Complement, a Target for Therapy in Inflammatory and Degenerative D... by B. Paul Morgan and Claire L. Harris (Nature Reviews Drug Discovery | AOP, published online 23 October 2015; doi:10.1038/nrd4657):

 

     ”Among the monoclonal antibodies, TNT003 and TNT009 (True North Therapeutics) target complement fragment C1s; OMS721 (Omeros) targets MBL-associated serine protease 2 (MASP2); Lampalizumab (Roche/Genentech) targets Factor D (FD), Bikaciomab (Novelmed) targets FB; IFX‑1 (InflaRx) targets C5a; eculizumab (Soliris; Alexion Pharmaceuticals), LFG316 (Novartis), ALXN1210 and ALXN550 (Alexion) all target C5. Regenesance are developing an antibody that targets C6, and Novelmed are developing an antibody (NM9401) against properdin. Among the protein biologics, Berinert (CSL Behring), Ruconest (Salix Pharmaceuticals) and Cinryze (Shire Pharmaceuticals) are all C1INH preparations that target C1; AMY‑201 (Amyndas) is a mini FH; TT30 (ALXN1102; Alexion) is a CR2–FH hybrid; TP10 (CDX‑1135; Celldex Therapeutics) is a soluble form of complement receptor type 1 (CR1); Coversin (Volution Immuno Pharmaceuticals) is a C5‑binding protein; Mirococept is a targeted CR1 fragment developed by AdProTech and currently in clinical trials led by King’s College London; and the affibody SOBI002 (Swedish Orphan Biovitrum) targets C5 (programme recently terminated). Small-molecule inhibitors include CCX‑168 (ChemoCentryx) and DF2593A (Dompé Pharmaceutical), which target C5aR; FD inhibitors from Achillion and Novartis; and a properdin inhibitor from Novelmed. Among the peptide-based therapeutics, Cp40 (Amyndas), APL‑1 and APL‑2 (Apellis Pharmaceuticals) are compstatin derivatives targeting C3; and RA101348 (RaPharma) is a C5 blocking peptide. Nucleic acid-based drugs include ARC1905 (Zimura; Ophthotech), a C5 aptamer; ALN‑CC5 (Alnylam Pharmaceuticals), a C5 RNA interference (RNAi) molecule; and the Spiegelmers NOX‑D19 to NOX‑D21 (Noxxon Pharma), targeting C5a. Regenesance are developing a C6 antisense molecule.”

     The American Society of Hematology meeting held December 5-8, 2015 in Orlando Florida brought a wealth of new information about potential drugs in the orphan drug pipeline. Poster abstracts, research, and sessions available to medical professionals at the 2015 ASH conference gained a broader audience with these reports for the aHUS community, containing links to corporate sites, news releases and research about each company’s orphan drug in development.

               Achillion Pharmaceuticals presents data at ASH: Small-Molecule fact...

 

               Akari Therapeutics to present data on Coversin at ASH

 

               Alnylam Pharmaceuticals to present data on ALN-CC5 at ASH

 

               Ra Pharma launches Phase 1 study of RA101495

                FDA grants 'Fast Track' Status for Omeros OMS721, * July 2015, prior to ASH mtg.

*Update re OMS721:  March 8, 2016*  

Click Title Below to read the March 8, 2016 Business Wire Press Release:

Omeros Corporation Initiates its Phase 3 Program for OMS721 in aHUS...

 

 

     The Atypical HUS Foundation and the aHUS Alliance of nations looks forward to bringing you more information of interest to patients, caregivers, and aHUS families.  We’re aware of other developments, such as Alexion’s entry this November 2015 with clinical trial  NCT02598583 of ALXN1210 for PNH Patients, and will present factual information as events unfold and information becomes available.  With innovation that seems ready to burst on the horizon, we wish you a New Year filled with hope and new possibilities for a brighter future in 2016.

FMI about potential new treatments for aHUS, click the link below:

aHUS Alliance meeting:   London 2015

 

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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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