aHUS Therapeutic Drug Pipeline – More ‘Ones to Watch’

NEW Information & Updates Available

Here’s the Latest:  Atypical HUS Drug Pipeline – 2017 Update from the aHUS Alliance
aHUS Therapeutic Drug Pipeline – More ‘Ones to Watch’
The medical community, healthcare policy makers, and patients alike are all concerned with aHUS treatment cost and how financial concerns impact access to treatment.  According to the 2016 aHUS Global Poll, with 233 respondents from 23 nations, 25% of patients outside the US & EU do not have access to the compliment inhibitor eculizumab in their nation even though it currently is the only drug approved for treatment of aHUS patients.   67% of poll respondents note their specialist or medical team has discussed aHUS treatment cost with the patient and their family, with 14% noting that cost concerns have impacted their aHUS care options.  (Source: aHUS Global Poll Questions: 21, 41, and 43)
Keeping abreast of orphan drug research and development is complex, as the landscape and information is continually changing.  The aHUS Alliance has offered a previous overview of potential aHUS drugs in the pipeline (24 May 2016), with links to both pharma corporate sites and press releases.  Here are 2 new “Ones to Watch” to add to our previous list of 7 biopharmaceutical companies with possible aHUS investigational drugs in development.**
EPIRUS Biopharmaceuticals of Boston MA announced in May that the company would reprioritization their pipeline to focus on biosimilars to treat rare diseases, with development and commercialization of biosimilar monoclonal antibodies (mAbs).  Our interest was this statement from their press release, although no further information was available on the Epirus Biopharmaceuticals website.  We look forward to having Epirus provide expanded information on their corporate site in the future.  BOW080 (eculizumab; reference biologic Soliris®) is a complement inhibitor being evaluated for the potential treatment of ultra-rare blood disorders such as, paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). “
Read the full Epirus Biopharmaceuticals’ 9 May 2016 press release:  http://ow.ly/McSp303Fo5J
 AMGEN has instituted a group study of its investigational drug ABP 959 to determine the biosimilarities of ABP 959 and eculizumab in healthy male subjects (both with intravenous delivery).  Registered with ANZCTR, an online registry of clinical trials being undertaken in Australia and New Zealand with the identifier ACTRN12616000509460, ABP 959 is not listed among its biosimilars in the pipeline on Amgen’s corporate site.  Quoted from Amgen source via the 21 June 2016 Pharma-Reporter.com article “Amgen Developing a Soliris Biosimilar” written by G. MacDonald:  “All we can say at this time is that Amgen has nine biosimilars in development, including a biosimilar candidate to Soliris®.  We look forward to providing further updates at the appropriate time.”
Read the article at BioPharma-Reporter.com:  http://ow.ly/SMEO303FtdS
What might the impact or response be regarding Alexion Pharmaceuticals’ product eculizumab?  Alexion Pharmaceuticals has a dual approach to potential competition with biosimilars, with a strengthening of their IP (patents protecting their intellectual property) as well as longer-acting versions of eculizumab called ALXN1210 expected to begin clinical trials within perhaps the next two months.
Read the article at BioPharma-Reporter.com:  http://ow.ly/D16f303Fric
Biosimilars versus Generic versions of Drugs:  In Brief
After a drug goes ‘off patent’, competition may arise for a branded therapy.  Generic drugs are chemically manufactured and go through a regulatory process, to demonstrate that they have the same active ingredients, and show no difference in safety or effectiveness.  Historically generic drugs have increased patient access and reduced treatment costs.
Biosimilars and biologic therapies are produced from living material, so making exact copies of such living things poses unique challenges.  As with generic drugs, biosimilars may increase patient access to treatment options, and may lower costs through competition with branded therapies within a field of other competitors.
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** Prior List “Ones to Watch” aHUS Therapeutic Drug Pipeline   
FMI and Links See: http://ow.ly/5Zx2303FodN

Achillion ACH-4471
Akari Therapeutics Coversin®
Alexion ALXN 1210, TT30
APELLIS Compstatin®/APL-2
Ra Pharma RA101495

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