aHUS Therapeutic Drugs

NEW Information & Updates Available

Here’s the Latest:  Atypical HUS Drug Pipeline – 2017 Update from the aHUS Alliance

aHUS Therapeutic Drugs – Potential for Advancements?

May 2016

Progress in complement therapeutics for clinical use is likely to expand in 2016 and beyond, given the increased interest in aHUS research and development. Since a hallmark of aHUS is chronic, uncontrolled complement activation, look forward to tracking future advancements in this broad and rapidly moving field.  Here are some key aspects that bear watching, although it’s likely that clinical trials for aHUS patients may occur secondarily to involvement of PNH or related rare disease patient populations.

Keeping pace with a drug development landscape that seems quite fluid can be difficult, with treatment research approaches that hinge on differences in how the drug works (current literature mentions these: monoclonal antibody, small molecule, peptide, protein or biologic, and nucleic acid-based therapy).  Differences in how the drug enters the patient’s body (drug delivery) also may have more options, ranging from intravenous (IV, or infused through ports) to subcutaneous (self-administered shots) and mention of an oral (pIll) delivery method for patients as well.

This is a complex topic, heavy with the technical medical terminology to wade through that often can be difficult for patients and families to grasp. We understand that some just wish to know names of the ‘ones to watch’, in terms of pharmaceutical companies with investigational drugs that may hold future potential for aHUS patients, while others want detailed information. The chart below provides a brief view of possible advancements in treatment for patients with complement mediated kidney disease, such as aHUS. (Looking to dig deeper? Click the chart’s hyperlinks and read the two supplementary articles.)

As research advances in areas that may be related to aHUS patient treatment, future updates offered here may help keep you connected and informed as circumstances and new information unfold.

Potential New Compliment Inhibitors Under Investigation

Company Molecule Mechanism Indications
Alexion ALXN1210, TT30** C3 inhibitor PNH, others
Achillion ACH-4471 Factor D inhibitor PNH, others
AKARI Therapeutics Coversin® C5 inhibitor PNH, aHUS, Guillain Barre syndrome
ALNYLAM ALN-CC5 C5 gene knockdown PNH, aHUS, others
APELLIS Compstatin®/APL-2 C3 inhibitor PNH, others
OMEROS OMS721®/MASP-2 Lectin pathway inhibitor TMAs including aHUS
Ra Pharma RA101495 C5 inhibitor PNH, others

** Alexion Acquistion, Taligen TT30

(This table should be considered only a partial listing of industry in the aHUS investigational drug space. Please connect with us at info@aHUSallianceaction.org to offer additional information or updates on aHUS drug R&D.)

NOTE: Most pharmaceuticals release informational press releases with updates on drug research and recent developments. Visit their corporate sites, or sign up to receive updates from these or other pharma.

FMI – Articles Regarding Complement and Drug Development (w/ R&D pipeline)

Complement, a target for therapy in inflammatory and degenerative diseases. Nature Reviews: Drug Discovery. Nature Reviews Drug Discovery: 14, 857–877 (2015) doi:10.1038/nrd4657. Published online: 23 October 2015.

Morgan, B. Paul and Harris, Claire L.

Complement in therapy and disease: Regulating the complement system with antibody-based therapeutics. Molecular Immunology. Volume 67, Issue 2, Part A, October 2015, Pages 117–130

Joost P.M. Melisa, Kristin Strumanea, Sigrid R. Ruulsa, Frank J. Beurskensa, Janine Schuurmana, Paul W.H.I. Parrena

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