CAMBRIDGE -
'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
According to the
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
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
About
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
Contact:
Tel: 617-674-9874
Email: nwildenradt@rapharma.com
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