Ra Pharma Supports 10th Annual Rare Disease Day® and Joins Global Movement to Raise Awareness for Rare Diseases
February 28 2017 - 7:00AM
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 joins forces with 30 million
health care advocates around the world for the 10th annual Rare
Disease Day®. Rare Disease Day, which is always the last day of
February, is an annual awareness day dedicated to elevating public
understanding of rare diseases and calling attention to the special
challenges people living with rare diseases face. This year’s focus
is on research and the global slogan for 2017 is “With Research,
Possibilities are Limitless.”
“A decade into its existence, the annual Rare Disease Day has
achieved a noteworthy milestone in its efforts calling attention to
the public health issues associated with rare diseases, a mission
Ra Pharma is proud to support,” said Doug Treco, PhD, President and
Chief Executive Officer of Ra Pharma. “As we prepare to initiate
two Phase 2 clinical trials of our lead compound RA101495 for the
treatment of paroxysmal nocturnal hemoglobinuria (PNH) and
refractory generalized myasthenia gravis (rMG), as well as a Phase
1b clinical trial supporting development in lupus nephritis (LN),
we are confident in our ability to address the unmet need for
people living with rare diseases where there are limited or no
treatment options.”
According to the National Institutes of Health (NIH), a disease
is rare if it affects fewer than 200,000 people. Nearly 1 in 10
Americans live with a rare disease—affecting 30 million people—and
nearly half of these patients are children. There are more than
7,000 rare diseases and only approximately 450 FDA-approved medical
treatments.
About Rare Disease Day
Rare Disease Day takes place every year on the last day of
February (February 28 or February 29 in a leap year)—the rarest
date on the calendar—to underscore the nature of rare diseases and
what patients face. It was established in Europe in 2008 by
EURORDIS, the organization representing rare disease patients in
Europe, and is now observed in more than 80 nations. Rare Disease
Day is sponsored in the U.S. by the National Organization for Rare
Disorders (NORD)®, the largest and leading independent, nonprofit
organization committed to the identification, treatment, and cure
of rare diseases.
For more information about Rare Disease Day in the U.S., go to
www.rarediseaseday.us. For information about global activities, go
to www.rarediseaseday.org. To search for information about rare
diseases, visit NORD’s website, www.rarediseases.org.
About RA101495
Ra Pharma is developing RA101495 for paroxysmal nocturnal
hemoglobinuria (PNH), refractory generalized myasthenia gravis
(rMG), and lupus nephritis (LN). The product is designed for
convenient, subcutaneous self-administration. RA101495 is a
synthetic, macrocyclic peptide discovered using Ra Pharma’s
powerful proprietary drug discovery technology. The peptide binds
complement C5 with subnanomolar affinity and allosterically
inhibits its cleavage into C5a and C5b upon activation of the
classical, alternative or lectin pathways. RA101495 also directly
binds to C5b, disrupting the interaction between C5b and C6 and
preventing assembly of the membrane attack complex (MAC). This
activity defines an additional and novel mechanism for the
inhibition of C5 function. In Phase 1 studies, dosing of RA101495
was well tolerated in healthy volunteers and demonstrated sustained
and near complete suppression of hemolysis and complement
activity.
About the Extreme Diversity™ Platform
Ra Pharma’s proprietary Extreme Diversity™ mRNA display platform
allows us to produce synthetic macrocyclic peptides that combine
the diversity and specificity of antibodies with the
pharmacological properties of small molecules. The platform
generates highly specific and stable peptide-like molecules with
the potential for greatly increased bioavailability, improved cell
permeability, and the opportunity to address protein-protein
interactions including previously undruggable targets. It can
produce libraries of 10 to 100 trillion members, allowing for the
rapid discovery of highly potent candidate molecules. The platform
is being leveraged for our emerging pipeline projects in Factor D
and C1.
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; as well as the other factors discussed
in the “Risk Factors” section in Ra Pharma’s most recently filed
Quarterly Report on Form 10-Q, 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/20170228005424/en/
Investors:Ra Pharmaceuticals, Inc.Jennifer Robinson,
617-674-9873jrobinson@rapharma.comorMedia:Argot PartnersEliza
Schleifstein, 917-763-8106eliza@argotpartners.com
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