Ra Pharmaceuticals Reports First Quarter 2017 Financial Results and Provides Corporate Update

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 announced financial results for the first quarter ended March 31, 2017 and provided an update on recent corporate and clinical developments.

“We continue to advance our lead compound, RA101495, and we recently commenced dosing in our Phase 2 program in paroxysmal nocturnal hemoglobinuria, or PNH,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “The goal of our program in PNH is to provide sustained and improved disease control, which reduces the risk of breakthrough hemolysis, while simultaneously offering patients a more convenient and flexible subcutaneous treatment regimen as a substitute for intravenous administration, today’s standard of care. We look forward to sharing initial data from these Phase 2 studies mid-year 2017 and additional results at year-end.”

Dr. Treco added: “We are also investigating RA101495 in other complement-mediated diseases such as myasthenia gravis (MG) and lupus nephritis (LN). We plan to initiate a Phase 2 trial in MG and a Phase 1b clinical trial supporting development in LN in the second half of 2017. We look forward to updating you on our RA101495 development plans, our orally-available small molecule C5 inhibitors and other pipeline programs.”

First Quarter 2017 Financial Results

As of March 31, 2017, Ra Pharma reported total cash and equivalents of $105.4 million.

For the first quarter of 2017, the Company reported a net loss of $11.4 million, or a net loss of $0.50 per share (basic and diluted), compared to net loss of $5.1 million, or a net loss of $9.42 per share for the same period in 2016.

Research and development expenses for the first quarter of 2017 were $9.0 million compared to $5.0 million for the same period in 2016. The increase in R&D expenses for the first quarter 2016 were primarily due to clinical development costs associated with our lead program, RA101495, for the treatment of PNH.

General and administrative expenses for the first quarter of 2017 were $2.5 million, compared to $1.3 million for the same period in 2016. The increase in G&A expenses for the first quarter 2017 were due primarily to employee-related costs, including salary, benefits and stock-based compensation due to the increase in G&A headcount to support the growth of the Company.

The Company’s revenue is derived from its collaboration and licensing agreement with Merck. Revenue for the three months ended March 31, 2017 was $0 compared to $1.9 million for the same period in 2016. The decrease was due to the expiration of the research term of the Merck Agreement in April 2016.

About RA101495
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, once daily SC self-administration. RA101495 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 also disrupts the interaction between C5b and C6 and prevents assembly of the membrane attack complex (MAC). This activity defines an additional, 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. To learn more about RA101495, please visit: http://rapharma.com/pipeline/ra101495/.

About the Extreme Diversity™ Platform
Ra Pharma’s proprietary Extreme Diversity™ mRNA display platform allows the Company 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 the Company’s emerging pipeline projects in Factor D and C1. To learn more about the Extreme Diversity platform, please visit: http://rapharma.com/science/extreme-diversity/.

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 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.

Ra Pharmaceuticals, Inc.
Condensed Consolidated Balance Sheets
(In thousands)
March 31, 2017December 31, 2016
Cash and cash equivalents $ 105,373 $ 117,812
Prepaid expenses and other current assets 1,632 1,690
Property and equipment, net 6,215 5,537
Other assets, noncurrent 1,763 1,779
Total assets $ 114,983 $ 126,818
Liabilities and Stockholders’ Equity
Accounts payable and accrued expenses $ 4,919 $ 6,434
Deferred rent 388 303
Noncurrent liabilities 2,755 2,859
Stockholders' equity 106,921 117,222
Total liabilities and stockholders’ equity $ 114,983 $ 126,818
Ra Pharmaceuticals, Inc.
Condensed Consolidated Statements of Operations
(in thousands, except per share data)
Three Months Ended March 31
Revenue $ - $ 1,884
Operating expenses:
Research and development 9,012 4,957
General and administrative 2,469 1,291

Total operating expenses

$ 11,481 $ 6,248
Loss from operations (11,481 ) (4,364 )
Other income (expense), net 121 (692 )
Net loss $ (11,360 ) $ (5,056 )
Net loss per common share – basic and diluted $ (0.50 ) $ (9.42 )
Weighted average number of common shares outstanding – basic and diluted 22,549 537


Ra Pharmaceuticals, Inc.
Jennifer Robinson, 617-674-9873
Argot Partners
Eliza Schleifstein, 917-763-8106

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