Ra Pharmaceuticals Supports 13th Annual Rare Disease Day® and Joins Global Movement to Raise Awareness for Rare Diseases
February 28 2020 - 07:00AM
Business Wire
Ra Pharmaceuticals, Inc. (Nasdaq:RARX) is joining forces with 30
million healthcare advocates around the world for the 13th Annual
Rare Disease Day®, an awareness day dedicated to elevating public
understanding of rare diseases and calling attention to the special
challenges people living with rare diseases face.
“At Ra Pharma, our primary focus is the patients and communities
we serve. These patients, families, and caregivers are at the core
of our effort to develop innovative and accessible therapies for
rare complement-mediated diseases,” said Doug Treco, Ph.D.,
President and Chief Executive Officer of Ra Pharma. “We achieved
progress across our rare disease programs in 2019, including the
initiation of a pivotal Phase 3 study in generalized myasthenia
gravis and a Phase 2 study in immune-mediated necrotizing myopathy,
yet significant work remains in the global effort to improve the
lives of people living with rare diseases. This Rare Disease Day,
we extend our great appreciation to these families and patients for
their bravery and strength.”
According to the National Institutes of Health, there are more
than 6,000 rare diseases known today that affect more than 25
million people in the United States. In the U.S., a disease is
defined as rare if it affects fewer than 200,000 people.
About Rare Disease Day
Rare Disease Day takes place every year on the last day of
February to raise awareness among the general public and
decision-makers about rare diseases and their impact on patients’
lives. It was established in 2008 by EURORDIS, the organization
representing rare disease patients in Europe, and is now observed
in more than 80 nations. In the U.S., Rare Disease Day is sponsored
by the National Organization for Rare Disorders (NORD®), the
largest and leading independent, non-profit organization committed
to the identification, treatment, and cure of rare diseases.
For more information about Rare Disease Day in the U.S. and to
search for information about rare diseases, visit NORD’s website,
www.rarediseaseday.us. For information about global activities, go
to www.rarediseaseday.org.
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. 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 expanding
patient access to therapies for rare complement-mediated diseases
and the safety, efficacy, regulatory and clinical progress, and
therapeutic potential of Ra Pharma’s product candidates. 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 USAN does not
approve the name zilucoplan; 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20200228005034/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
Ra Pharmaceuticals (NASDAQ:RARX)
Historical Stock Chart
From Feb 2024 to Mar 2024
Ra Pharmaceuticals (NASDAQ:RARX)
Historical Stock Chart
From Mar 2023 to Mar 2024