Ra Pharmaceuticals Initiates Dosing in Phase 2 Clinical Trial Evaluating RA101495 SC in Generalized Myasthenia Gravis Patients
December 21 2017 - 3:01PM
Business Wire
Topline Data from Second Clinical Indication
Expected First Half of 2019
Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced
that it has initiated dosing in the Company’s Phase 2 clinical
trial evaluating RA101495 SC for the treatment of generalized
myasthenia gravis (gMG). gMG is a rare, complement-mediated,
autoimmune disease that causes weakness in skeletal muscles. Ra
Pharma is a clinical stage biopharmaceutical company focusing on
the development of next-generation therapeutics for the treatment
of complement-mediated diseases and is developing RA101495 as a
novel, subcutaneously-administered (SC) inhibitor of complement
component 5 (C5). RA101495 SC is currently in clinical development
for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
“Following promising Phase 1 data demonstrating a favorable
pharmacokinetic and pharmacodynamic profile for RA101495, this
Phase 2 trial is designed to evaluate the safety, tolerability, and
preliminary efficacy of RA101495 in gMG,” said James F. Howard, MD,
University of North Carolina School of Medicine. “RA101495 potently
inhibits C5, a validated therapeutic target in myasthenia. The
convenient, once-daily, SC, self-administration schedule of
RA101495 may enable treatment of a broad population of gMG
patients.”
The Phase 2, multicenter, randomized, double-blind,
placebo-controlled trial is designed to evaluate the safety,
tolerability, and preliminary efficacy of RA101495 SC in patients
with gMG. The trial will enroll approximately 36 patients. At the
outset of the 12-week treatment period, patients will be randomized
in a 1:1:1 ratio and will receive daily, SC doses of 0.1 mg/kg of
RA101495, 0.3 mg/kg of RA101495, or matching placebo. The primary
efficacy endpoint is change in Quantitative Myasthenia Gravis (QMG)
score from baseline to week 12. All patients will have the
opportunity to receive RA101495 SC in a long-term extension
study.
“gMG is a rare, autoimmune disease characterized by
autoantibodies targeting proteins critical for the normal
transmission of electrical signals from nerves to muscles,” said
Nancy Law, CEO of the Myasthenia Gravis Foundation of America.
“Patients with gMG have limited treatment options. Given the
significant unmet need in this patient population, we are
encouraged by the initiation of this Phase 2 trial, and we look
forward to determining the potential clinical benefit of this
novel, once-daily, subcutaneously-administered C5 inhibitor.”
Myasthenia gravis (MG) is a chronic, autoimmune, neuromuscular
disease characterized by weakness and fatigue of voluntary 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 over 80,000 individuals
worldwide.
“Initiation of dosing in our Phase 2 trial evaluating RA101495
in gMG is a critical step in the advancement of a potential novel
therapy for patients with this disabling autoimmune disorder, and
we expect topline data from this trial in the first half of 2019,”
said Doug Treco, PhD, President and Chief Executive Officer of Ra
Pharma. “We continue to be encouraged by the positive interim data
from our ongoing Phase 2 program evaluating RA101495 in patients
with PNH, and we remain focused on fully maximizing the potential
of RA101495’s safety, efficacy, and ease of administration to
expand patient access across a range of C5-mediated disorders.”
About RA101495 SC
Ra Pharma is developing RA101495 SC for paroxysmal nocturnal
hemoglobinuria (PNH), generalized myasthenia gravis (gMG), atypical
hemolytic uremic syndrome (aHUS), and lupus nephritis (LN). The
product is designed for convenient, once-daily, subcutaneous (SC)
self-administration. RA101495 SC 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. By binding to a region of C5
corresponding to C5b, RA101495 SC 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. To learn more about RA101495 SC,
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 of delay in enrollment or
release of topline results from that expected; 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 full
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, 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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Investors:Ra Pharmaceuticals, Inc.Jennifer Robinson,
617-674-9873jrobinson@rapharma.comorMedia:Argot PartnersEliza
Schleifstein, 917-763-8106eliza@argotpartners.com
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