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Ra Pharmaceuticals Announces Acceptance of gMG Phase 2 and Open-Label, Long-Term Extension Data for Emerging Science Dual Presentation at the 2019 AAN Annual Meeting

April 1, 2019 Off By BusinessWire

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today announced that data from
its Phase 2 clinical trial of zilucoplan for the treatment of
generalized myasthenia gravis (gMG) have been selected for an Emerging
Science dual oral and poster presentation at the 2019 American Academy
of Neurology (AAN) Annual Meeting in Philadelphia, PA, from May 4 to 10,
2019. The presentation will feature data from the Phase 2, randomized,
double-blind, placebo-controlled clinical trial, as well as data from
the open-label, long-term extension study.

Details of the presentation are as follows:

Abstract Title: Zilucoplan, a Subcutaneously Self-Administered
Peptide Inhibitor of Complement Component 5 (C5), for the Treatment of
Generalized Myasthenia Gravis: Results of a Phase 2 Randomized,
Double-Blind, Placebo-Controlled Trial and Open-Label Long-Term Extension
Session
Title: Emerging Science
Date/Time: Tuesday, May 7,
Poster Presentation: 11:30 a.m.-12:45 p.m. E.T., Data Blitz Oral
Presentation: 12:06 p.m. E.T.

About Zilucoplan

Ra Pharma is developing zilucoplan for generalized myasthenia gravis
(gMG), paroxysmal nocturnal hemoglobinuria (PNH), and other
complement-mediated disorders. The product candidate is designed for
convenient, once-daily subcutaneous 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 Ra Pharmaceuticals

Ra Pharmaceuticals is a clinical-stage biopharmaceutical company focused
on leading the field of complement biology to bring innovative and
accessible therapies to patients with rare 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 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 upcoming
presentations, development of product candidates, 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 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.