Ra Pharmaceuticals Announces Clearance of IND Application for the HEALEY ALS Platform Trial
January 22 2020 - 9:00AM
Business Wire
Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today announced the U.S.
Food and Drug Administration’s (FDA) clearance of the
Investigational New Drug (IND) application for the HEALEY ALS
Platform Trial for the treatment of amyotrophic lateral sclerosis
(ALS). Zilucoplan was selected as one of the first clinical
candidates to be evaluated in this platform trial for ALS led by
the Sean M. Healey & AMG Center for ALS at Mass General.
“We are pleased that the FDA has authorized the initiation of
this pioneering platform trial, which, through the pooling of
placebo arms, optimizes the chance a patient receives active drug
and enables the efficient evaluation of multiple investigational
therapies,” said Doug Treco, Ph.D., President and Chief Executive
Officer of Ra Pharma. “With substantial data supporting the role of
central and peripheral complement activation and membrane attack
complex deposition in ALS, we look forward to assessing the
therapeutic potential of zilucoplan, an investigational small
peptide inhibitor of complement component 5 (C5), in this disease.
We’re grateful to the Healey Center for its sponsorship of this
trial and share its commitment to improving the lives of patients
with ALS, a debilitating disease with limited treatment
options.”
The HEALEY ALS Platform trial will be conducted across
approximately 54 U.S. centers and is designed to initially evaluate
three treatment regimens in parallel. Following enrollment,
patients will be randomized to one of three treatment regimens, one
of which will be the zilucoplan regimen. The zilucoplan regimen
will evaluate the efficacy and safety of zilucoplan versus placebo
on ALS disease progression, and the primary endpoint will be change
in ALS Functional Rating Scale-Revised (ALSFRS-R) score. Following
the initial randomization to this regimen, patients will be
randomized in a 3:1 ratio to receive daily subcutaneous doses of
0.3 mg/kg zilucoplan or placebo. The zilucoplan regimen is expected
to enroll 160 patients for a 24-week treatment period. Following
completion of the trial, patients will have the option to enter
into an open-label extension study with zilucoplan.
About the HEALEY ALS Platform Trial
The ALS platform trial is designed to disrupt the standard pace
of ALS therapy development by testing and evaluating multiple
treatments simultaneously. This model, which has had success in the
cancer field, is designed to accelerate development of therapies by
allowing investigators to test more drug candidates, increase
patient access to trials, and reduce costs by quickly and
efficiently evaluating the effectiveness of multiple
investigational therapies. Following the Healey Center’s call for
the best therapeutic ideas to enter the HEALEY ALS Platform Trial,
the first drug candidates were chosen by a group of expert ALS
scientists and members of the Healey Center Science Advisory
Committee.
About ALS
Amyotrophic lateral sclerosis (ALS) is the most prevalent
adult-onset progressive motor neuron disease, affecting
approximately 30,000 people in the U.S. and an estimated 500,000
people worldwide. ALS causes the progressive degeneration of motor
neurons, resulting in progressive muscle weakness and atrophy that
eventually lead to partial or total paralysis. Therapeutic options
for ALS are currently limited to two approved products.
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
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 potential, safety, efficacy, and regulatory and clinical
progress of Ra Pharma's product candidates, including without
limitation zilucoplan, beliefs regarding clinical trial data, and
statements regarding trial design, timeline, and enrollment of Ra
Pharma's ongoing and planned clinical programs, including without
limitation the Healey Center-sponsored ALS platform trial. 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; 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 be
unable to obtain orphan drug designation or to maintain the
benefits associated with orphan drug status, including market
exclusivity; 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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version on businesswire.com: https://www.businesswire.com/news/home/20200122005198/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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