News Release

<< Back
Ra Pharmaceuticals Reports Fourth Quarter and Full Year 2016 Financial Results and Provides Corporate Update

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Mar. 6, 2017-- 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 fourth quarter and year ended December 31, 2016 and provided an update on recent corporate and clinical developments.

“Following our successful initial public offering in October 2016, we are well positioned to advance our lead complement C5 inhibitor, RA101495, and other pipeline programs in 2017,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “Having secured approval from the FDA and other ex-U.S. regulatory agencies to initiate our Phase 2 program in PNH patients, we look forward to starting these studies by the end of the first quarter. RA101495 is designed as a convenient self-administered subcutaneous injection, with highly predictable pharmacokinetics and robust pharmacodynamic effects. We believe that it represents an attractive alternative to the current standard of care, which requires regular intravenous infusions and does not adequately address the needs of all PNH patients. We expect to share data from these studies in the second half of this year.”

Dr. Treco added: “In addition to our PNH program, we plan to initiate a Phase 2 clinical trial of RA101495 in myasthenia gravis and a Phase 1b clinical trial supporting development in lupus nephritis in the second half of 2017. We will also advance our once-weekly formulation of RA101495, our orally-available small molecule C5 inhibitors and other pipeline programs. We look forward to reporting on our continued progress.”

Recent Developments

  • Successfully completed our initial public offering which 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.
  • Granted Orphan Drug Designation by the European Commission for RA101495 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.
  • Presented data from the Company’s RA101495 and Oral C5 Inhibitor programs at the International PNH Interest Group Annual Scientific Assembly on December 2, 2016 in San Diego. The presentation highlighted a completed Phase 1 study of RA101495 in healthy volunteers, which demonstrated that it is a well-tolerated, potent, subcutaneously-administered inhibitor of C5 capable of achieving rapid, complete and sustained inhibition of hemolysis. The presentation also included a description of the planned Phase 2 study for RA101495 in PNH, and highlighted new molecules discovered through the Company’s oral small molecule C5 inhibitor program.

Upcoming Milestones

  • In the first quarter of 2017, the Company expects to initiate a global Phase 2 development program of RA101495 in PNH. The program is comprised of two open-label studies, one in the U.S. and one ex-U.S. Data is expected in the second half of 2017.
  • 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 to support development in lupus nephritis (LN).

Fourth Quarter and Full Year 2016 Financial Results

As of December 31, 2016, Ra Pharma reported total cash and equivalents of $117.8 million.

For the fourth quarter of 2016, the Company reported a net loss attributable to common shareholders of $10.9 million, or a net loss of $0.74 per share (basic and diluted), compared to net loss of $4.6 million, or a net loss of $8.63 per share, for the same period in 2015. For the full year 2016, Ra Pharma reported a net loss attributable to common shareholders of $28.9 million, or a net loss of $6.98 per share (basic and diluted), compared to a net loss of $12.3 million, or a net loss of $24.68 per share, for the full year 2015.

Research and development expenses for the fourth quarter of 2016 were $9.4 million compared to $4.6 million for the same period in 2015. Research and development expenses for the full year 2016 were $27.9 million compared to $15.2 million for the full year 2015. The increase in R&D expenses for both the fourth quarter and full year were 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 fourth quarter of 2016 were $1.6 million, compared to $0.7 million for the same period in 2015. General and administrative expenses for the full year 2016 were $5.0 million, compared to $2.2 million for the full year 2015. The increase in G&A expenses for both the fourth quarter and the full year were 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.

The Company’s revenue is derived from its collaboration and licensing agreement with Merck. Revenue for the three months ended December 31, 2016 was $0 compared to $0.8 million for the same period in 2015. The decrease was due to the expiration of the research term of the Merck Agreement in April 2016. Total revenue for the full year 2016 was $4.9 million compared to $4.1 million for the full year 2015, and included a $3.0 million milestone payment for the delivery and transfer of novel, orally-available cyclic peptides for a non-complement cardiovascular target with a large market opportunity, to Merck for further development.

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 Pharma’s powerful proprietary drug discovery technology. 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. 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.

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.

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 (unaudited)
(In thousands)
 
December 31, December 31,
2016 2015
 
Assets
Cash and cash equivalents $ 117,812 $ 19,386
Prepaid expenses and other current assets 1,690 800
Property and equipment, net 5,537 2,143
Other assets, noncurrent   1,779   2,013  
Total assets $ 126,818 $ 24,342  
 
Liabilities and stockholders’ equity (deficit)
Accounts payable and accrued expenses $ 6,434 $ 2,593
Deferred revenue - 1,621
Other liabilities, current 303 2,724
Other liabilities, noncurrent 2,859 928
Redeemable convertible preferred stock - 53,675
Stockholders' equity (deficit) 117,222

(37,199

)

Total liabilities, redeemable convertible    
preferred stock and stockholders’ equity (deficit) $ 126,818 $ 24,342
               
Ra Pharmaceuticals, Inc.
Condensed Consolidated Statements of Operations (Unaudited)
(In thousands, except share and per share data)
 
Three Months Ended

Twelve Months Ended

December 31, December 31,
2016 2015 2016 2015
 
Revenue $ -   $ 821   $ 4,928   $ 4,094  
Operating expenses
Research and development 9,387 4,624 27,928 15,217
General and administrative   1,605     684     5,024     2,233  
Total operating expenses   10,992     5,308     32,952     17,450  
Loss from operations (10,992 ) (4,487 ) (28,024 ) (13,356 )
Other income (expense), net and benefit from income taxes   104     (76 )   (840 )   (587 )
Net loss $ (10,888 ) $ (4,563 ) $ (28,864 ) $ (13,943 )
Gain on extinguishment of redeemable convertible preferred shares   -     -     -     1,673  
Net loss attributable to common shareholders $ (10,888 ) $ (4,563 ) $ (28,864 ) $ (12,270 )
 
Net loss per share attributable to common shareholders-basic and diluted $ (0.73 ) $ (8.63 ) $ (6.98 ) $ (24.68 )
Weighted-average number of common shares oustanding-basic and diluted 14,815,949 528,511 4,135,331 497,073

Source: Ra Pharmaceuticals, Inc.

Investors:
Ra Pharmaceuticals, Inc.
Jennifer Robinson, 617-674-9873
jrobinson@rapharma.com
or
Media:
Argot Partners
Eliza Schleifstein, 917-763-8106
eliza@argotpartners.com