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 10th 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 focus is on research and the global slogan for 2017 is “With Research, Possibilities are Limitless.”
“A decade into its existence, the annual Rare Disease Day has achieved a noteworthy milestone in its efforts calling attention to the public health issues associated with rare diseases, a mission Ra Pharma is proud to support,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “As we prepare to initiate two Phase 2 clinical trials of our lead compound RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and refractory generalized myasthenia gravis (rMG), as well as a Phase 1b clinical trial supporting development in lupus nephritis (LN), we are confident in our ability to address the unmet need for people living with rare diseases where there are limited or no treatment options.”
According to the National Institutes of Health (NIH), a disease is rare if it affects fewer than 200,000 people. Nearly 1 in 10 Americans live with a rare disease—affecting 30 million people—and nearly half of these patients 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 28 or 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 Europe in 2008 by EURORDIS, the organization representing rare disease patients in Europe, and is now observed in more than 80 nations. Rare Disease Day is sponsored in the U.S. 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.
Ra Pharma is developing RA101495 for paroxysmal nocturnal hemoglobinuria (PNH), refractory generalized myasthenia gravis (rMG), and lupus nephritis (LN). The product is designed for convenient, subcutaneous self-administration. RA101495 is a synthetic, macrocyclic peptide discovered using Ra Pharma’s powerful proprietary drug discovery technology. The peptide binds complement C5 with subnanomolar affinity and allosterically inhibits its cleavage into C5a and C5b upon activation of the classical, alternative or lectin pathways. RA101495 also directly binds to C5b, disrupting the interaction between C5b and C6 and preventing assembly of the membrane attack complex (MAC). This activity defines an additional and novel mechanism for the inhibition of C5 function. In Phase 1 studies, dosing of RA101495 was well tolerated in healthy volunteers and demonstrated sustained and near complete suppression of hemolysis and complement activity.
About the Extreme Diversity™ Platform
Ra Pharma’s proprietary Extreme Diversity™ mRNA display platform allows us to produce synthetic macrocyclic peptides that combine the diversity and specificity of antibodies with the pharmacological properties of small molecules. The platform generates highly specific and stable peptide-like molecules with the potential for greatly increased bioavailability, improved cell permeability, and the opportunity to address protein-protein interactions including previously undruggable targets. It can produce libraries of 10 to 100 trillion members, allowing for the rapid discovery of highly potent candidate molecules. The platform is being leveraged for our emerging pipeline projects in Factor D and C1.
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; as well as the other factors discussed in the “Risk Factors” section in Ra Pharma’s most recently filed Quarterly Report on Form 10-Q, 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.