Ra Pharmaceuticals Presents Data on Oral Small Molecule Complement Inhibitors at the 22nd Congress of the European Hematology...
June 24 2017 - 10:00AM
Business Wire
Ra Pharmaceuticals, Inc. (NASDAQ:RARX), a clinical stage
biopharmaceutical company focusing on the development of
next-generation therapeutics for the treatment of complement
mediated diseases, today announced the presentation of scientific
data at the 22nd Congress of the European Hematology Association
(EHA), June 22-25, 2017 in Madrid, Spain.
“The data presented at EHA showcase a novel class of orally
bioavailable small molecules that bind to C5 with high affinity and
inhibit its cleavage into C5a and C5b,” said Alonso Ricardo, Senior
Vice President and Head of Research at Ra Pharma. “These findings
demonstrate the feasibility of an orally-administered therapy for
complement-mediated disorders and support the continued advancement
of these molecules.”
Inhibition of C5 is a clinically-validated approach for the
therapeutic treatment of complement-mediated disorders. Ra Pharma’s
portfolio includes drug candidates for several such disorders,
including paroxysmal nocturnal hemoglobinuria (PNH), myasthenia
gravis (MG) and lupus nephritis (LN). The Company is pursuing
the identification of orally-available inhibitors of C5 with the
goal of providing additional options for patients diagnosed with
these and other complement-mediated disorders.
“We are encouraged by our scientists’ ability to discover and
characterize a small molecule inhibitor of complement C5,” said
Doug Treco, PhD, President and Chief Executive Officer of Ra
Pharma. “These data show that C5 inhibition can be accomplished
with an orally-bioavailable small molecule, and we hope that
compounds from this class can be advanced to provide a new
treatment option for a broader spectrum of patients.”
The presentation is available at
http://rapharma.com/science/presentations-and-publications/. A
summary of the data presented by Ra Pharma at the conference is as
follows:
Title: Discovery of Orally Bioavailable Small Molecules
for Inhibition of Complement C5Session Title: Bone marrow
failure and PNH (oral presentation)Presenter: Alonso
Ricardo, PhD, Senior Vice President and Head of Research, Ra
PharmaDate/Time: Saturday, June 24, 5:00- 5:15 PM
CESTLocation: Room N104Abstract Code:
S500Summary: These initial studies describe a series of
first-in-class, orally bioavailable small molecules that bind to
C5, inhibiting the protein’s cleavage into C5a and C5b. These
molecules possess favorable pharmacokinetic properties, exhibiting
up to 50% oral availability in preclinical species. Molecules from
this class were found to bind to a unique site on complement C5
with a 1:1 stoichiometry. Notably, binding to this site also
inhibited the cleavage of C5 containing the R885H polymorphism, a
mutation that confers resistance to eculizumab, today’s standard of
care in PNH. The extensive in vitro and in vivo data, along with a
high-resolution crystal structure, support the feasibility of
developing alternatives to injectable treatment of disorders of
complement regulation.
About Ra Pharmaceuticals
Ra Pharmaceuticals is a clinical stage biopharmaceutical company
focusing on the development of next-generation therapeutics for
complement-mediated diseases. The Company discovers and develops
peptides and small molecules to target key components of the
complement cascade. For more information, please visit:
www.rapharma.com.
Forward-Looking Statement
This press release contains "forward-looking statements" within
the meaning of the Private Securities Litigation Reform Act of
1995, including, but not limited to, statements regarding the
safety, efficacy and regulatory and clinical progress of our
product candidates, including RA101495. All such forward-looking
statements are based on management's current expectations of future
events and are subject to a number of risks and uncertainties that
could cause actual results to differ materially and adversely from
those set forth in or implied by such forward-looking statements.
These risks and uncertainties include the risks that Ra Pharma’s
product candidates, including RA101495, will not successfully be
developed or commercialized; the risk that the results of
preclinical studies may not be indicative of the results of
clinical studies; as well as the other factors discussed in the
“Risk Factors” section in Ra Pharma’s most recently filed Annual
Report on Form 10-K, as well as other risks detailed in Ra Pharma’s
subsequent filings with the Securities and Exchange Commission.
There can be no assurance that the actual results or developments
anticipated by Ra Pharma will be realized or, even if substantially
realized, that they will have the expected consequences to, or
effects on, Ra Pharma. All information in this press release is as
of the date of the release, and Ra Pharma undertakes no duty to
update this information unless required by law.
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version on businesswire.com: http://www.businesswire.com/news/home/20170624005012/en/
Investors:Ra Pharmaceuticals, Inc.Jennifer Robinson,
617-674-9873jrobinson@rapharma.comorMedia:Argot PartnersEliza
Schleifstein, 917-763-8106eliza@argotpartners.com
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