News Release
Ra Pharmaceuticals Reports Fourth Quarter and Full Year 2018 Financial Results and Provides Corporate Update
Successful completion of Phase 2 trial of zilucoplan in generalized myasthenia gravis (gMG) with rapid, clinically meaningful, and statistically significant reductions in QMG and MG-ADL
Zilucoplan extended release (XR) achieves greater than one week of target drug concentrations in non-human primates following a single subcutaneous injection, supporting once weekly or less frequent dosing
“The fourth quarter of 2018 was transformational for Ra, with the successful completion of our Phase 2 clinical trial of zilucoplan for the treatment of gMG, the strengthening of our balance sheet with a
Dr. Treco continued: “The strong Phase 2 results in gMG and zilucoplan’s competitive profile as an accessible and convenient self-administered therapy support the potential to serve a broad population of patients with gMG. We plan to leverage the unique properties of our small peptide C5 inhibitor and expand development into other tissue-based, complement-mediated disorders with high unmet medical need.”
“Lastly, we are excited to announce significant progress in our zilucoplan extended release program, in which a single subcutaneous injection of zilucoplan rapidly achieved target drug concentrations in a non-human primate model, with near complete inhibition of an ex vivo hemolysis assay for greater than one week, supporting once weekly or less frequent dosing. Importantly, the peptide properties of zilucoplan enable extended interval subcutaneous dosing without the need for prior intravenous loading required by monoclonal antibody C5 inhibitor therapies. In addition to advancing the development of this and other XR formulations in 2019, we plan to progress our other pipeline programs, including our oral C5 small molecule inhibitor program, and anticipate entering human clinical studies in both programs in the first half of 2020.”
Fourth Quarter 2018 Highlights and Recent Developments
- In
December 2018 , Ra Pharma announced the successful completion of the Phase 2 clinical trial evaluating zilucoplan for the treatment of gMG, achieving rapid, clinically meaningful, and statistically significant reductions in pre-specified primary and key secondary endpoints for both zilucoplan dose groups tested versus placebo at 12 weeks.- Zilucoplan dosed at 0.3 mg/kg SC daily achieved a mean reduction from baseline of 6.0 points in the Quantitative Myasthenia Gravis (QMG) score (placebo-corrected change = -2.8; p=0.05) and a mean reduction from baseline of 3.4 points in the MG Activities of Daily Living (MG-ADL) score (placebo-corrected change = -2.3; p=0.04), with no patients treated with the 0.3 mg/kg dose of zilucoplan requiring rescue therapy.
- Based on these data, Ra Pharma plans to engage with regulatory agencies, including the
U.S. Food and Drug Administration (FDA ), in the first half of 2019 regarding the design of a Phase 3 registrational trial evaluating zilucoplan versus placebo in patients with gMG. Ra Pharma anticipates initiating the Phase 3 gMG program in the second half of 2019.
- Ra Pharma today announced significant progress on its first XR formulation of zilucoplan, with a single subcutaneous dose resulting in rapid attainment of target drug concentrations for greater than one week in non-human primates, supporting once weekly or less frequent dosing. Of note, near complete inhibition of terminal complement activity for greater than one week was achieved without the need for prior intravenous loading doses, as measured by an ex vivo hemolysis assay. Data from the XR development program will be presented at the 6th Annual
Peptides Congress ,April 24-25 , 2019, inLondon, UK . Ra Pharma continues to evaluate the pharmacokinetic profiles of this and other formulations to optimize the XR dosing regimen. Ra Pharma anticipates the XR program entering human clinical studies in the first half of 2020. - In
December 2018 , the Company strengthened its balance sheet with a follow-on offering, raising approximately$150 million in gross proceeds. - In
December 2018 , the Company announced the receipt of a$2.5 million development milestone payment under its collaboration agreement withMerck & Co. (known as MSD outside the U.S. andCanada ). The milestone payment is associated with the companies’ collaboration for a non-complement cardiovascular target with a large market opportunity. - In
December 2018 , Ra Pharma completed dosing in a Phase 1 ethno-bridging study in healthy subjects of Japanese and non-Japanese descent, enrolling 16 subjects in a multi-dose cohort and 20 subjects in a single-dose cohort. The Phase 1 study is designed to support development of zilucoplan inJapan . - In
September 2018 , based onFDA feedback, as well as advice provided by the Medicines and Healthcare products Regulatory Agency in theUK (MHRA) andHealth Canada , Ra Pharma announced the design of a single-arm Phase 3 study in treatment-naïve paroxysmal nocturnal hemoglobinuria (PNH) patients. InJanuary 2019 , Ra Pharma concluded Scientific Advice and protocol design discussions with theEuropean Medicines Agency (EMA). The EMA was not receptive to a single-arm study design to support approval of zilucoplan in PNH. Ra Pharma does not plan to alter the design of the Phase 3 PNH program, but is reassessing the likelihood of obtaining marketing authorization for PNH in theEuropean Union . - In
November 2018 , Ra Pharma electedBo Cumbo to its Board of Directors. Mr. Cumbo serves as Senior Vice President and Chief Commercial Officer ofSarepta Therapeutics, Inc. (Nasdaq:SRPT).
Fourth Quarter 2018 and Full Year Financial Results
For the fourth quarter of 2018, the Company reported a net loss of
Research and development (R&D) expenses for the fourth quarter of 2018 were
General and administrative (G&A) expenses for the fourth quarter of 2018 were
Total revenue earned in the fourth quarter and year ended
As of
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 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: 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 Ra Pharma's ability to expand patient access to important therapies, the potential, safety, efficacy and regulatory and clinical progress of Ra Pharma's product candidates, including without limitation zilucoplan, beliefs regarding clinical trial data, statements regarding trial design, timeline and enrollment of Ra Pharma's ongoing and planned clinical programs, including without limitation the Phase 3 trial of zilucoplan for the treatment of PNH, the Phase 3 trial of zilucoplan for the treatment of gMG and the XR and oral C5 small molecule inhibitor programs, pending End-of-Phase 2 discussions with regulatory agencies in the first half of 2019, plans to reassess the likelihood of obtaining marketing authorization for PNH in the
Ra Pharmaceuticals, Inc. | |||||||||||||||||
Condensed Consolidated Statements of Operations | |||||||||||||||||
(Unaudited) | |||||||||||||||||
(in thousands, except per share data) | |||||||||||||||||
Three Months Ended
December 31, |
Year Ended
December 31, |
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2018 | 2017 | 2018 | 2017 | ||||||||||||||
Revenue | $ | 2,500 | $ | - | $ | 2,500 | $ | - | |||||||||
Operating expenses: | |||||||||||||||||
Research and development | 15,357 | 12,645 | 54,449 | 45,251 | |||||||||||||
General and administrative | 3,801 | 2,677 | 14,439 | 9,778 | |||||||||||||
Total operating expenses | 19,158 | 15,322 | 68,888 | 55,029 | |||||||||||||
Loss from operations | (16,658) | (15,322) | (66,388) | (55,029) | |||||||||||||
Other income (expense), net | 444 | 162 | 1,426 | 571 | |||||||||||||
Benefit from income taxes | (19) | (19) | (19) | (19) | |||||||||||||
Net loss | $ | (16,195) | $ | (15,141) | $ | (64,943) | $ | (54,439) | |||||||||
Net loss per common share – basic and diluted | $ | (0.47) | $ | (0.67) | $ | (2.06) | $ | (2.41) | |||||||||
Weighted average number of common shares outstanding – basic and diluted | 34,185 | 22,626 | 31,542 | 22,591 | |||||||||||||
Ra Pharmaceuticals, Inc. | |||||||||
Condensed Consolidated Balance Sheets | |||||||||
(Unaudited) | |||||||||
(In thousands) | |||||||||
|
December 31, 2018 |
December 31, 2017 |
|||||||
Assets | |||||||||
Cash and cash equivalents | $ | 209,822 | $ | 70,381 | |||||
Prepaid expenses and other current assets | 2,585 | 2,496 | |||||||
Property and equipment, net | 5,165 | 5,606 | |||||||
Other noncurrent assets | 1,648 | 1,714 | |||||||
Total assets | $ | 219,220 | $ | 80,197 | |||||
Liabilities and Stockholders’ Equity | |||||||||
Accounts payable and accrued expenses | $ | 9,722 | $ | 8,285 | |||||
Deferred rent | 479 | 329 | |||||||
Noncurrent liabilities | 1,901 | 2,399 | |||||||
Stockholders' equity | 207,118 | 69,184 | |||||||
Total liabilities and stockholders’ equity | $ | 219,220 | $ | 80,197 | |||||
View source version on businesswire.com: https://www.businesswire.com/news/home/20190307005347/en/
Source:
Investors:
Ra Pharmaceuticals, Inc.
Natalie Wildenradt, 617-674-9874
nwildenradt@rapharma.com
Media:
Argot Partners
David Rosen, 212-600-1902
david.rosen@argotpartners.com