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Press Release

Ra Pharma Supports 11th Annual Rare Disease Day® and Joins Global Movement to Raise Awareness for Rare Diseases

Ra Pharmaceuticals,Inc
Posted on: 28 Feb 18

Ra Pharmaceuticals, Inc. (NASDAQ:RARX), a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for the treatment of complement-mediated diseases, today joins forces with 30 million health care advocates around the world for the 11th annual Rare Disease Day®. Rare Disease Day, which is always the last day of February, is an annual awareness day dedicated to elevating public understanding of rare diseases and calling attention to the special challenges people living with rare diseases face. This year’s theme is research, and the global slogan for 2018 is “Show Your Rare, Show You Care.”

“After more than a decade of raising awareness through the observance of Rare Disease Day and achieving great progress in rare disease research, there is still significant work to be done to develop treatments and Ra Pharma is committed to the fight against rare diseases,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “Since Rare Disease Day 2017, Ra Pharma has implemented and completed a global Phase 2 program for our lead compound RA101495 SC for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), as well as initiated a Phase 2 clinical trial in generalized myasthenia gravis (gMG). Additionally, we have begun a Phase 1b clinical trial supporting expansion into other rare, complement-mediated disorders, including atypical hemolytic uremic syndrome (aHUS) and lupus nephritis (LN). We remain committed to advancing the development of RA101495 SC across a broad range of indications to address the significant unmet need for these patients.”

Rare Disease Day 2018 offers participants the opportunity to be part of a global call on policy makers, researchers, companies, and healthcare professionals to increasingly and more effectively involve patients in rare disease research. According to the National Institutes of Health (NIH), a disease is rare if it affects fewer than 200,000 people. In the U.S., approximately 30 million people (nearly 1 in 10 Americans) live with a rare disease and more than half of them are children. There are more than 7,000 rare diseases and only approximately 450 FDA-approved medical treatments.

About Rare Disease Day

Rare Disease Day takes place every year on the last day of February (February 29 in a leap year—the rarest date on the calendar) to underscore the nature of rare diseases and what patients face. 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, nonprofit organization committed to the identification, treatment, and cure of rare diseases.

For more information about Rare Disease Day in the U.S., go to www.rarediseaseday.us. For information about global activities, go to www.rarediseaseday.org. To search for information about rare diseases, visit NORD’s website, www.rarediseases.org.

About RA101495 SC

Ra Pharma is developing RA101495 SC for paroxysmal nocturnal hemoglobinuria (PNH), generalized myasthenia gravis (gMG), atypical hemolytic uremic syndrome (aHUS), and lupus nephritis (LN). The product is designed for convenient, once-daily subcutaneous self-administration. RA101495 SC 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, RA101495 SC is designed to disrupt the interaction between C5b and C6 and prevent assembly of the membrane attack complex (MAC). 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 Statement

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 the safety, efficacy and regulatory and clinical progress of our product candidates, including RA101495. 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 RA101495, will not successfully be developed or commercialized; the risk that topline results as of February 7, 2017 from the Company’s global Phase 2 clinical program evaluating RA101495 for the treatment of PNH may not be indicative of final study results; 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: http://www.businesswire.com/news/home/20180228005484/en/

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Last updated on: 28/02/2018

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