Ra Pharmaceuticals Receives Orphan Drug Designation from the U.S. FDA for RA101495 for the Treatment of Paroxysmal Nocturnal ...
July 31 2017 - 7:12AM
Business Wire
Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced that the
U.S. Food and Drug Administration (FDA) has granted Orphan Drug
Designation to RA101495 for the treatment of paroxysmal nocturnal
hemoglobinuria (PNH). Ra Pharma is a clinical stage
biopharmaceutical company focusing on the development of
next-generation therapeutics for the treatment of
complement-mediated diseases. RA101495, the Company’s lead clinical
candidate, is a synthetic macrocyclic peptide inhibitor of
complement component 5 (C5). The molecule is currently in Phase 2
clinical development as a self-administered subcutaneous (SC)
injection for the treatment of PNH, a rare, chronic,
life-threatening blood disorder where red blood cells are attacked
and destroyed by the complement system.
“There is an urgent need for new treatment options for patients
suffering from PNH. The current standard of care requires biweekly
intravenous infusions, a dosing regimen that imposes a severe
burden on patients, providers, and caregivers,” said Doug Treco,
PhD, President and Chief Executive Officer of Ra Pharma. “We have
designed RA101495 for once-daily, subcutaneous self-administration,
an approach which has the potential to ease this burden, improve
convenience, and provide much-needed dosing flexibility. We are
encouraged by our initial Phase 2 data in PNH patients, which
showed near-complete inhibition of hemolysis and a favorable safety
and tolerability profile. We look forward to advancing our PNH
program and providing additional data updates around year-end.”
The FDA grants Orphan Drug Designation status to products that
treat rare diseases, providing incentives to sponsors developing
drugs or biologics. The FDA defines rare diseases as those
affecting fewer than 200,000 people in the United States at the
time of designation. Orphan Drug Designation qualifies the sponsor
certain development incentives, including tax credits for qualified
clinical testing.
RA101495 received Orphan Drug Designation for the treatment of
PNH in Europe in November 2016.
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, once daily SC self-administration. RA101495 is a
synthetic, macrocyclic peptide discovered using Ra Pharma’s
powerful proprietary drug discovery technology. The peptide binds
complement C5 with sub-nanomolar affinity and allosterically
inhibits its cleavage into C5a and C5b upon activation of the
classical, alternative or lectin pathways. By binding to a region
of C5 corresponding to C5b, RA101495 also disrupts the interaction
between C5b and C6 and prevents assembly of the membrane attack
complex (MAC). This activity defines an additional, 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. To learn more about RA101495, please visit:
http://rapharma.com/pipeline/ra101495/.
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 initial data from the
Company’s global Phase 2 clinical program evaluating RA101495 for
the treatment of PNH may not be indicative of final study results;
the risk that initial data from a limited number of patients may
not be indicative of results from the fully patient enrollment
planned for such study; 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, and 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/20170731005477/en/
Investors:Ra Pharmaceuticals, Inc.Jennifer Robinson,
617-674-9873jrobinson@rapharma.comorMedia:Argot PartnersEliza
Schleifstein, 917-763-8106eliza@argotpartners.com
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