Ra Pharmaceuticals Reports Third Quarter 2016 Financial Results and Provides Corporate Update

Ra Pharmaceuticals (NASDAQ:RARX), a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for diseases of complement dysregulation, today announced financial results for the three months ended September 30, 2016 and provided an update on recent corporate and clinical developments.

“Our successful initial public offering in October strengthened our balance sheet and allows us to initiate our Phase 2 clinical program evaluating our lead synthetic macrocyclic peptide candidate, RA101495, in paroxysmal nocturnal hemoglobinuria, or PNH, in the first quarter of 2017,” said Doug Treco, PhD, Founder and CEO of Ra Pharmaceuticals. “In addition, we look forward to leveraging our work in PNH and advancing RA101495 in other complement-mediated indications, including refractory generalized myasthenia gravis and lupus nephritis, as well as advancing our earlier stage complement inhibitors derived from our proprietary Extreme Diversity™ platform. I look forward to providing future updates on our progress as we execute our clinical strategy.”

Recent Developments

  • Completed a successful initial public offering that raised approximately $105.4 million in gross proceeds, including the full exercise by the underwriters of their over-allotment option. Net proceeds to Ra Pharma, after deducting underwriting discounts and commissions and offering expenses, were approximately $95.7 million.
  • Announced that RA101495 has been granted Orphan Drug Designation by the European Commission for the treatment of PNH. This designation provides certain regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union, and for which no satisfactory treatment is available, or for which the product under consideration is expected to provide significant benefits to patients versus existing therapies.

Upcoming Milestones

  • In the first quarter of 2017, the Company expects to initiate its Phase 2 clinical trial of RA101495 in PNH patients.
  • In the second half of 2017, the Company plans to initiate a Phase 2 clinical trial of RA101495 in refractory generalized myasthenia gravis (rMG) and a Phase 1b clinical trial in lupus nephritis (LN).
  • In the second half of 2017, the Company expects to report top-line data from its Phase 2 trial of RA101495 in PNH.

Third Quarter 2016 Financial Results

As of September 30, 2016, Ra Pharma reported total cash and equivalents of $31.2 million. Subsequent to the end of the third quarter, the Company completed an initial public offering of common stock that raised total net proceeds of approximately $95.7 million, including overallotments.

For the third quarter of 2016, Ra Pharma reported a net loss attributable to common shareholders of $8.1 million, or $14.22 per share (basic and diluted), compared to $2.2 million, or $4.57 per share, for the same period in 2015.

Research and development expenses for the third quarter of 2016 were $7.1 million compared to $4.0 million for the same period in 2015. The increase was primarily due to pre-clinical and clinical development costs associated with our lead program, RA101495, for the treatment of PNH.

General and administrative expenses for the third quarter of 2016 were $1.0 million, compared to $0.5 million for the same period in 2015. The increase was due 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.

Total revenue for the three months ended September 30, 2016 was $0 compared to $1.1 million for the same period in 2015. Revenue is comprised primarily of collaboration revenue from Merck. 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, subcutaneous self-administration. RA101495 is a synthetic, macrocyclic peptide discovered using Ra’s Proprietary Extreme Diversity™ platform. 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. 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™ platform allows us to produce synthetic macrocyclic peptides that combine the diversity and specificity of antibodies with the pharmacological properties of small molecules. We utilize a process called ‘‘mRNA display’’ to produce extremely large and diverse libraries of peptides from which to select potential product candidates. 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.

About Ra Pharmaceuticals

Ra Pharmaceuticals is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for diseases of complement dysregulation and other orphan indications. 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” and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections in Ra Pharma’s Registration Statement on Form S-1 (File No. 333-214242), as amended, which is on file with the Securities and Exchange Commission and was declared effective on October 25, 2016, 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 (Unaudited)
(In thousands)
September 30,December 31,
Cash and cash equivalents $ 31,196 $ 19,386
Prepaid expenses and other current assets 669 800
Property and equipment, net 5,673 2,143
Other assets, noncurrent 3,110 2,013
Total assets $ 40,648 $ 24,342
Liabilities and stockholders’ deficit
Accounts payable and accrued expenses $ 5,510 $ 2,593
Total deferred revenue - 1,862
Other liabilities, current 219 2,724
Other liabilities, noncurrent 2,981 687
Redeemable convertible preferred stock 86,483 53,675
Stockholder's deficit (54,545 ) (37,199 )
Total liabilities and stockholders’ deficit $ 40,648 $ 24,342
Ra Pharmaceuticals, Inc.
Condensed Consolidated Statements of Operations (Unaudited)
(In thousands, except per share data)
Three Months EndedNine Months Ended
September 30,September 30,
Revenues $ - $ 1,096 $ 4,928 $ 3,273
Operating expenses
Research and development 7,079 4,027 18,541 10,593
General and administrative 1,042 539 3,418 1,549
Total operating expenses 8,121 4,566 21,959 12,142
Loss from operations (8,121 ) (3,470 ) (17,031 ) (8,869 )
Other expense, net 7 (462 ) (945 ) (511 )
Net loss $ (8,114 ) $ (3,932 )




$ (9,380 )
Gain on extinguishment of redeemable convertible preferred shares - 1,673 - 1,673
Net loss attributable to common shareholders $ (8,114 ) $ (2,259 )




$ (7,707 )
Net loss per share-basic and diluted $ (14.22 ) $ (4.57 ) $ (32.73 ) $ (15.84 )
Weighted-average number of common shares used in net loss per share
applicable to common stockholders-basic and diluted 570,558 494,302 549,138 486,479


Argot Partners
Susan Kim, 212-600-1902
Eliza Schleifstein, 917-763-8106

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