Pivotal Phase 3 trial of zilucoplan in gMG
ongoing, with top-line results expected in early 2021
Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today announced that full
results from its Phase 2 clinical trial of zilucoplan in patients
with generalized myasthenia gravis (gMG) were published online in
JAMA Neurology. The JAMA Neurology publication can be accessed
here.
“This publication in JAMA Neurology recognizes the impact and
significance of the findings from our Phase 2 clinical trial
demonstrating zilucoplan’s potential in gMG, a chronic and
debilitating neuromuscular disease that affects more than 60,000
patients in the U.S.,” said Doug Treco, Ph.D., President and Chief
Executive Officer of Ra Pharma. “These results support our further
evaluation of zilucoplan in the ongoing RAISE study, a pivotal
Phase 3 trial of zilucoplan in gMG, with top-line results expected
in early 2021. If successfully developed and approved, zilucoplan
has the potential to expand access to convenient and effective
complement inhibition therapy to a broad range of patients with
gMG.”
As reported in the JAMA Neurology article, “Clinical Effects of
the Self-administered Subcutaneous Complement Inhibitor Zilucoplan
in Patients with Moderate to Severe Generalized Myasthenia Gravis:
Results of a Phase 2 Randomized, Double-Blind, Placebo-Controlled,
Multicenter Clinical Trial,” the study demonstrated that zilucoplan
resulted in rapid, clinically meaningful, statistically
significant, and sustained improvements in the primary and key
secondary endpoints, namely, change from baseline to week 12 in
Quantitative Myasthenia Gravis (QMG) and MG Activities of Daily
Living (MG-ADL) scores, respectively. Further, near-complete
complement inhibition was associated with a faster onset and
greater magnitude of benefit than submaximal complement inhibition,
with a favorable safety and tolerability profile.
Ra Pharma is currently evaluating zilucoplan in the RAISE study,
a single, global, pivotal, randomized, double-blind,
placebo-controlled, Phase 3 clinical trial, for the treatment of
gMG. The trial, which incorporates feedback from the U.S. Food and
Drug Administration (FDA), the European Medicines Agency (EMA), and
Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), is
designed to evaluate the efficacy of a once-daily, subcutaneously
(SC) self-administered dose of 0.3 mg/kg of zilucoplan versus
placebo. Top-line results from the RAISE study are expected in
early 2021.
About Zilucoplan Phase 2 gMG Clinical Trial
The Phase 2, multi-center, randomized, double-blind,
placebo-controlled trial was designed to evaluate the safety,
tolerability, and preliminary efficacy of zilucoplan in patients
with generalized myasthenia gravis (gMG), regardless of prior
therapies, who had an MGFA Disease Class of II-IVa at screening and
a Quantitative Myasthenia Gravis (QMG) score, a
physician-administered assessment of MG-related muscle weakness, of
≥ 12 at screening and randomization. The trial enrolled 44 patients
in the U.S. and Canada. At the outset of the 12-week treatment
period, patients were randomized in a 1:1:1 ratio to receive daily,
subcutaneous (SC) doses of 0.1 mg/kg of zilucoplan, 0.3 mg/kg of
zilucoplan, or matching placebo. The pre-specified primary efficacy
endpoint was the change in QMG score from baseline to week 12. The
key secondary efficacy endpoint was the change in MG Activities of
Daily Living (MG-ADL) score, a patient-reported outcome measure,
from baseline to week 12. Significance testing was pre-specified at
a 1-sided alpha of 0.1. All 44 patients completed the 12-week study
and, of these, 42 (95%) entered a long-term extension to receive
active study drug.
About gMG
Myasthenia gravis (MG) is a chronic, autoimmune, neuromuscular
disease characterized by weakness and fatigue of skeletal muscles.
Patients with MG present with muscle weakness that becomes
increasingly severe with repeated use and recovers with rest.
Weakness can be localized to specific muscles, such as those
responsible for eye movements, but often progresses to affect a
broader range, including head, limb, and respiratory muscles. This
progression is often described as the generalized, or severe, form
of the disease. gMG is estimated to affect approximately 60,000
people in the U.S. alone.
About Zilucoplan
Ra Pharma is developing zilucoplan and zilucoplan extended
release (XR) for generalized myasthenia gravis (gMG),
immune-mediated necrotizing myopathy (IMNM), amyotrophic lateral
sclerosis (ALS), and other tissue-based complement-mediated
disorders with high unmet medical need. The product candidate is
designed for convenient subcutaneous (SC) self-administration.
Zilucoplan is an investigational, synthetic, macrocyclic peptide
discovered using Ra Pharma's powerful proprietary drug discovery
technology. The peptide is designed to bind complement component 5
(C5) with sub-nanomolar affinity and allosterically inhibit its
cleavage into C5a and C5b upon activation of the classical,
alternative, or lectin pathways. The U.S. Food and Drug
Administration (FDA) has granted Orphan Drug Designation to
zilucoplan for the treatment of MG.
About Ra Pharmaceuticals, Inc.
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 Ra
Pharma’s ability to expand patient access to important therapies,
the safety, efficacy, regulatory and clinical progress, and
therapeutic potential of Ra Pharma’s product candidates, including
without limitation zilucoplan and zilucoplan XR, statements
regarding trial design, timeline, and enrollment of Ra Pharma’s
ongoing and planned clinical programs, including without limitation
the Phase 3 trial of zilucoplan for the treatment of gMG, and plans
and timing for the presentation of and beliefs regarding clinical
trial data, including top-line results for the RAISE study. 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
and zilucoplan XR, will not successfully be developed or
commercialized, in the timeframe we expect or at all; the risk that
Ra Pharma may fail to enroll patients in its clinical trials, which
may cause delays or other adverse effects; the risk that Ra Pharma
may fail to obtain additional financing on favorable terms or at
all; risks relating to the Merger, including: that Ra Pharma may be
unable to obtain stockholder approval as required for the Merger;
conditions to the closing of the Merger may not be satisfied and
required regulatory approvals may be delayed or not be obtained;
the Merger may involve unexpected costs, liabilities or delays; the
business of Ra Pharma may suffer as a result of uncertainty
surrounding the Merger; the outcome of any legal proceedings
related to the Merger; Ra Pharma may be adversely affected by other
economic, business, and/or competitive factors; the occurrence of
any event, change or other circumstances that could give rise to
the termination of the Merger Agreement; and other risks to the
consummation of the Merger, including the risk that the Merger will
not be consummated within the expected time period or at all. If
the Merger is consummated, Ra Pharma stockholders will cease to
have any equity interest in Ra Pharma and will have no right to
participate in its earnings and future growth. Additional factors
that may affect the future results of Ra Pharma are discussed in
the “Risk Factors” section in Ra Pharma’s most recently filed
Annual Report on Form 10-K and Quarterly Report on Form 10-Q for
the period ending September 30, 2019, 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: https://www.businesswire.com/news/home/20200218005177/en/
Investors: Ra Pharmaceuticals, Inc. Natalie Wildenradt,
617-674-9874 nwildenradt@rapharma.com Media: Argot Partners David
Rosen, 212-600-1902 david.rosen@argotpartners.com
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