Ra Pharmaceuticals Reports Third Quarter 2018 Financial Results and Provides Corporate Update
Dosing completed in Phase 2 trial of zilucoplan in generalized myasthenia gravis
On track for topline data read-out around year-end 2018
Announces issuance of U.S. patent covering zilucoplan composition of matter and methods of use
“We are pleased to report that dosing in our Phase 2 trial of zilucoplan for gMG is now complete and we look forward to reporting top-line data around year-end,” said
Third Quarter 2018 Highlights and Recent Developments
- In August, Ra Pharma completed enrollment in the Phase 2 trial evaluating zilucoplan for the treatment of gMG. The multi-center, randomized, double-blind, placebo-controlled trial enrolled a total of 44 patients. At the outset of the 12-week treatment period, patients were randomized in a 1:1:1 ratio and received daily SC doses of 0.1 mg/kg of zilucoplan, 0.3 mg/kg of zilucoplan, or matching placebo. Dosing was completed at the end of
October 2018and the Company plans to report top-line data around year-end 2018. All 44 patients completed the 12-week study and, of these, 43 (98%) elected to enter a long-term extension to receive active study drug.
- In September, Ra Pharma announced the completion of End-of-Phase 2 interactions with the
U.S. Food and Drug Administration( FDA) for its global Phase 3 program of zilucoplan for the treatment of PNH. Based on FDAfeedback and advice provided by the Medicines and Healthcare products Regulatory Agency in the UK(MHRA) and Health Canada, and pending discussions with the European Medicines Agency(EMA) in the fourth quarter of 2018, Ra Pharma plans to initiate a global, pivotal, single-arm Phase 3 trial to evaluate the safety and efficacy of zilucoplan in approximately 40 treatment-naïve PNH patients in the first half of 2019. In addition, the Company expects to initiate a supportive trial in approximately 40 transfusion-independent patients switching from eculizumab to zilucoplan. In a separate CMC Type C meeting, Ra Pharma has also reached agreement with the FDAon the pharmaceutical development program required to support the Phase 3 clinical program.
- In September, Ra Pharma reported positive results from the Phase 1b, multi-center, open-label, pharmacokinetic (PK) study of zilucoplan in patients with renal disease. The trial enrolled 16 patients, eight with severe renal impairment matched with eight healthy control subjects. The PK profile of zilucoplan was consistent and similar across both groups. These results support the expansion of zilucoplan into complement-mediated renal disorders without the need for dose adjustment.
- In September, Ra Pharma elected
Aoife M. Brennan, M.B., B.Ch., to its Board of Directors. Dr. Brennan serves as President, Chief Executive Officer, and Chief Medical Officer of Synlogic, Inc.(Nasdaq:SYBX).
- Ra Pharma announced today that the U.S. Patent and Trademark Office (USPTO) has issued U.S. Patent No. 10,106,579 which covers a family of molecules, including zilucoplan. The patent provides composition of matter protection of zilucoplan and methods of use in the treatment of complement-mediated disorders. This patent is the first in a series of composition of matter and treatment filings that is designed to protect zilucoplan through at least 2035.
Third Quarter 2018 Financial Results
For the third quarter of 2018, the Company reported a net loss of
Research and development expenses for the third quarter of 2018 were
General and administrative expenses for the third quarter of 2018 were
There was no revenue earned in the three months ended
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.
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.
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 potential safety, efficacy and regulatory and clinical progress of our product candidates, including without limitation zilucoplan, beliefs regarding clinical trial data, statements regarding trial design, timeline and enrollment of our ongoing and planned clinical programs, including without limitation our Phase 3 trial of zilucoplan for the treatment of PNH, pending discussions with the EMA in the fourth quarter of 2018, and upcoming milestones, including without limitation the release of top-line data in gMG around year-end 2018. 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 topline results as of
|Ra Pharmaceuticals, Inc.|
|Condensed Consolidated Statements of Operations|
|(in thousands, except per share data)|
Three Months Ended
Nine Months Ended
|Research and development||$||13,375||$||13,130||$||39,092||$||32,606|
|General and administrative||3,504||2,284||10,637||7,101|
|Total operating expenses||16,879||15,414||49,729||39,707|
|Loss from operations||(16,879||)||(15,414||)||(49,729||)||(39,707||)|
|Other income (expense), net||375||139||981||409|
|Net loss per common share – basic and diluted||$||(0.51||)||$||(0.68||)||$||(1.60||)||$||(1.74||)|
|Weighted average number of common shares outstanding – basic and diluted||32,349||22,614||30,652||22,579|
|Ra Pharmaceuticals, Inc.|
|Condensed Consolidated Balance Sheets|
|Cash and cash equivalents||$||81,059||$||70,381|
|Prepaid expenses and other current assets||2,502||2,496|
|Property and equipment, net||5,394||5,606|
|Other noncurrent assets||1,664||1,714|
|Liabilities and Stockholders’ Equity|
|Accounts payable and accrued expenses||$||7,648||$||8,285|
|Total liabilities and stockholders’ equity||$||90,619||$||80,197|