Ra Pharmaceuticals Announces Selection for the First ALS Platform Trial by the Sean M. Healey & AMG Center for ALS at Mass Ge...
18 Settembre 2019 - 1:00PM
Business Wire
Multi-center platform trial sponsored by the
Healey Center will simultaneously assess multiple drug regimens
Zilucoplan selected by leading experts in ALS
as one of the first therapeutic candidates to be tested
Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today announced the
selection of zilucoplan as one of the first clinical candidates to
be evaluated in a pioneering platform trial for amyotrophic lateral
sclerosis (ALS), led by the Sean M. Healey & AMG Center for ALS
at Mass General. The Healey Center has agreed to provide funding
for the execution of the platform trial to support its goal of
accelerating the development of effective treatments for patients
with ALS.
“The Healey Center brought together top leaders from around the
world to dramatically re-think how we design ALS trials and create
a groundbreaking platform trial program,” says Merit Cudkowicz,
M.D., M.Sc., director of the Sean M. Healey & AMG Center for
ALS at Mass General. “With research suggesting a key role for
central and peripheral complement activation and deposition in ALS,
we look forward to improving patient access to promising therapies
and contributing important knowledge to the ALS community.”
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, aims to greatly accelerate development of therapies
by allowing investigators to test more drugs, increase patient
access to trials, and reduce costs by quickly and efficiently
evaluating the effectiveness of multiple therapies. The Healey
Center issued a call for the best therapeutic ideas to enter the
HEALEY ALS Platform Trial, and the first drug candidates have been
chosen by a group of expert ALS scientists and members of the
Healey Center Science Advisory Committee.
“Robust pre-clinical data support the evaluation of a C5
inhibitor in ALS. Zilucoplan is a small peptide inhibitor with
enhanced tissue distribution as compared with therapeutic
monoclonal antibodies and has demonstrated efficacy in gMG, a
complement-mediated disease of the neuromuscular junction,” said
Simon Read, Ph.D., Chief Scientific Officer of Ra Pharma. “The ALS
platform trial design, together with a close relationship with the
ALS patient community, has the potential to enable rapid
recruitment and robust assessment of the therapeutic effect of
zilucoplan in this disease.”
“We’re grateful to the Healey Center for its sponsorship of this
trial and are honored to participate in this important and
innovative study. We look forward to working closely together in
this collaboration,” said Doug Treco, Ph.D., President and Chief
Executive Officer of Ra Pharma. “Participation in the ALS platform
trial represents a further expansion of our neurologic pipeline for
zilucoplan, which leverages the unique properties of a small
peptide C5 inhibitor in complement-mediated neurologic
diseases.”
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 candidates are
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.
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-led 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/20190918005167/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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