News Release
Ra Pharmaceuticals Presents Pre-Clinical Data for Zilucoplan XR at the 6th Annual Peptides Congress
Zilucoplan XR achieved rapid and sustained pharmacodynamic inhibition of complement C5 in non-human primates, supporting once weekly or less frequent dosing
This XR formulation of zilucoplan employs poly(D,L-lactic-co-glycolic acid) (PLGA) technology, an established delivery platform for sustained release of peptides. The PLGA XR formulation of zilucoplan is designed to produce uniform particles with homogenous distribution of zilucoplan and a predictable release profile. In pre-clinical studies, the PLGA XR formulation of zilucoplan achieved and maintained therapeutic drug levels necessary to treat complement-mediated diseases, including generalized myasthenia gravis.
“We’re excited to present data for the PLGA XR formulation of
zilucoplan, in which target drug concentrations were achieved and
maintained with once-weekly subcutaneous dosing,” said
Weekly subcutaneous doses of the PLGA XR formulation of zilucoplan in non-human primates rapidly achieved and maintained target drug concentrations for 14 days. Near-complete inhibition of complement component 5 (C5) in an ex vivo hemolysis assay was sustained throughout this 14 day period.
“Consistent with our patient-centric approach to drug development, the
progress of the PLGA XR formulation of zilucoplan is an important step
in our efforts to develop and expand patient access to simple and
convenient treatment options for a wide range of C5-mediated diseases,”
said
The presentation from the 6th Annual
About Zilucoplan
Ra Pharma is developing zilucoplan for generalized myasthenia gravis (gMG) and other tissue-based, complement-mediated disorders with high unmet medical need. The product candidate is designed for convenient, once-daily, subcutaneous (SC) self-administration. Zilucoplan is a synthetic, macrocyclic peptide discovered using Ra Pharma's powerful proprietary drug discovery technology. The peptide binds complement component 5 (C5) with sub-nanomolar affinity and allosterically inhibits its cleavage into C5a and C5b upon activation of the classical, alternative, or lectin pathways.
About
Forward-Looking Statements
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 potential and
progress of the PLGA XR formulation of zilucoplan and other XR
formulations, our expectations surrounding the initiation of clinical
trials of the XR formulation of zilucoplan and timing thereof, our
efforts to develop and expand patient access to treatment options for a
wide range of C5-mediated diseases, and bringing innovative and
accessible therapies to patients with rare diseases. 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 zilucoplan, will not successfully
be developed or commercialized, in the timeframe we expect or at all; 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
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Source:
Investors:
Ra Pharmaceuticals, Inc.
Natalie Wildenradt,
617-674-9874
nwildenradt@rapharma.com
Media:
Argot Partners
David Rosen, 212-600-1902
david.rosen@argotpartners.com