RARX - Ra Pharmaceuticals Supports 12th Annual Rare Disease Day® and Joins Global Movement to Raise Awareness for Rare Diseases

Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today joins forces with 30 million healthcare advocates around the world for the 12th 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.

“Despite important scientific advances in the treatment of rare diseases, there remains significant work to be done to help patients and families. At Ra Pharma, we are focused on developing medicines to treat patient populations with complement-mediated diseases and are committed to expanding patient access to more convenient life-saving medicines,” said Doug Treco, Ph.D., President and Chief Executive Officer of Ra Pharma. “In 2018, we made important progress in achieving this goal, as we successfully completed a Phase 2 clinical trial for our lead compound, zilucoplan, in generalized myasthenia gravis. As we prepare to initiate a Phase 3 clinical program for this indication, we also continue to advance our broader pipeline of therapeutic candidates for the treatment of rare, complement-mediated diseases.”

Dr. Treco added: “One of the privileges of being at a research-based company like ours is working side-by-side with families and patients who are battling rare diseases. This Rare Disease Day, we extend our appreciation to these families and patients for their bravery, tenacity, and strength. It’s an honor to be a part of their lives, and they inspire us to do our best work every day.”

According to the National Institutes of Health, there are more than 6,000 rare diseases known today that affect an estimated 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 amongst 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 (formerly RA101495 SC)

Ra Pharma is developing zilucoplan for generalized myasthenia gravis (gMG), paroxysmal nocturnal hemoglobinuria (PNH), and other complement-mediated disorders. The product candidate is designed for convenient, once-daily subcutaneous self-administration. Zilucoplan 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, zilucoplan is additionally designed to disrupt the interaction between C5b and C6 and prevent assembly of the membrane attack complex. This activity may define an additional, novel mechanism for the inhibition of C5 function.

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 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 more convenient life-saving medicines and our plans to initiate a Phase 3 clinical program for zilucoplan in generalized myasthenia gravis. 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.

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