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Ra Pharmaceuticals Supports 13th Annual Rare Disease Day® and Joins Global Movement to Raise Awareness for Rare Diseases

CAMBRIDGE, Mass.–(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, For information about global activities, go to

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:

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.



Ra Pharmaceuticals, Inc.

Natalie Wildenradt, 617-674-9874


Argot Partners

David Rosen, 212-600-1902


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