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

- Rapid Recruitment into PNH Phase 2 Study with 17 Patients Currently Enrolled

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Aug. 9, 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 second quarter ended June 30, 2017, and provided an update on recent corporate and clinical developments, including enrollment in the Phase 2 program for RA101495 in paroxysmal nocturnal hemoglobinuria (PNH).

“We are pleased with the initial data from the first two eculizumab-naïve patients enrolled in our Phase 2 program evaluating RA101495 in PNH, which showed rapid declines in lactate dehydrogenase (LDH), near-complete inhibition of hemolytic activity, and no safety or tolerability concerns. The strength of these data enabled the opening of the eculizumab-switch cohort and generated strong interest from investigators and patients,” said Ramin Farzaneh-Far, MD, Chief Medical Officer of Ra Pharma. “Recruitment has since progressed rapidly, with a total of 17 patients currently enrolled, 14 of whom are already receiving study drug. Enrollment targets in both the eculizumab-naïve and eculizumab-switch cohorts have been met, and the first two patients receiving RA101495 have transitioned to a long-term extension study.”

Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma, added: “The initial Phase 2 data with this self-administered, subcutaneous product candidate support our goal of improving treatment options for patients suffering from PNH, as well as other complement-mediated diseases, including myasthenia gravis (MG) and lupus nephritis (LN). We plan to initiate a Phase 2 trial evaluating RA101495 in MG and a Phase 1b trial supporting development in LN in the second half of 2017. We look forward to providing updates on these and other pipeline programs later this year.”

Recent Developments

  • Announced initial data from Ra Pharma’s global Phase 2 clinical program evaluating the safety, tolerability, preliminary efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of RA101495 in patients with PNH. The initial data on two eculizumab-naïve patients with seven weeks of follow-up showed no safety or tolerability concerns, no injection site reactions, near-complete inhibition of hemolytic activity, rapid declines in LDH, and 100% compliance with the once daily, subcutaneous self-administration dosing regimen.
  • Commenced enrollment in Cohort B of Ra Pharma’s Phase 2 clinical program evaluating RA101495 in PNH, comprised of patients switching from eculizumab, the current standard of care. Cohort B was opened following review of the initial data reported in Cohort A, which consists of eculizumab-naïve patients. To date, 17 patients have been enrolled, including 9 patients in Cohort A representing an expanded range of elevated baseline LDH levels, and 8 patients in Cohort B, in whom LDH was well-controlled at baseline. A total of 14 patients have received RA101495, 8 patients in Cohort A and 6 patients in Cohort B. The first two patients in Cohort A successfully completed the Phase 2 study and have elected to continue treatment with RA101495 in a long-term extension study. The Company remains on track to report additional data around year-end.
  • Received Orphan Drug Designation from the US Food and Drug Administration (FDA) for RA101495 for the treatment of PNH. This designation provides various development incentives, including tax credits for qualified clinical testing, for drugs that treat a rare disease or condition, defined as affecting fewer than 200,000 people in the US.
  • Presented data on the Company’s oral small molecule complement inhibitor program at the 22nd Congress of the European Hematology Association on June 24, 2017, in Madrid. The presentation highlighted this novel class of orally bioavailable small molecules that bind to C5 with high affinity and inhibit its cleavage into C5a and C5b, demonstrating the feasibility of an orally-administered therapy for complement-mediated disorders.

Second Quarter 2017 Financial Results

For the second quarter of 2017, the Company reported a net loss of $12.7 million, or a net loss of $0.56 per share (basic and diluted), compared to a net loss of $4.8 million, or a net loss of $8.90 per share for the same period in 2016.

Research and development expenses for the second quarter of 2017 were $10.5 million, compared to $6.5 million for the same period in 2016. The increase in R&D expenses for the second quarter 2017 was primarily due to clinical development costs associated with our lead program, RA101495, for the treatment of PNH.

General and administrative expenses for the second quarter of 2017 were $2.3 million, compared to $1.1 million for the same period in 2016. The increase in G&A expenses for the second quarter 2017 was 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.

There was no revenue recorded in the three months ended June 30, 2017, compared to $3.0 million for the same period in 2016. The Company’s 2016 revenue was derived from its collaboration and licensing agreement with Merck, which expired in April 2016.

As of June 30, 2017, Ra Pharma reported total cash and equivalents of $96.6 million. The Company expects that its cash and cash equivalents will be sufficient to fund operations through the end of 2018.

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 subcutaneous (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 RA101495 Phase 2 Clinical Program

The global, dose-finding Phase 2 program is designed to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetics, and pharmacodynamics of RA101495 in patients with PNH. The study will evaluate RA101495 in three cohorts. Cohort A includes eculizumab-naïve patients, Cohort B includes patients switching from eculizumab to RA101495, and a third cohort includes patients who are currently treated with eculizumab, but have evidence of an inadequate response. Patients in all three cohorts will be eligible for a long-term extension study following the completion of the initial 12-week studies. The primary efficacy endpoint is change in LDH from baseline to the mean level from week 6 to week 12.

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; the risk that initial data from the Company’s global Phase 2 clinical program evaluating RA101495 for the treatment of PNH may not be indicative of final study results; the risk that initial data from a limited number of patients may not be indicative of results from the fully patient enrollment planned for such study; 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)
 
June 30, 2017 December 31, 2016
 
Assets
Cash and cash equivalents $ 96,605 $ 117,812
Prepaid expenses and other current assets 1,231 1,690
Property and equipment, net 6,239 5,537
Other noncurrent assets   1,746   1,779
Total assets $ 105,821 $ 126,818
 
Liabilities and Stockholders’ Equity
Accounts payable and accrued expenses $ 6,578 $ 6,434
Deferred rent 422 303
Noncurrent liabilities 2,645 2,859
Stockholders' equity   96,176   117,222
Total liabilities and stockholders’ equity $ 105,821 $ 126,818
 
                       
Ra Pharmaceuticals, Inc.
Condensed Consolidated Statements of Operations
(Unaudited)
(in thousands, except per share data)
 
Three Months Ended June 30 Six Months Ended June 30
2017 2016 2017 2016
Revenue $ - $ 3,044 $ - $ 4,928
Operating expenses:
Research and development 10,464 6,505 19,476 11,462
General and administrative   2,348     1,085     4,817     2,376  
Total operating expenses   12,812     7,590     24,293     13,838  
Loss from operations (12,812 ) (4,546 ) (24,293 ) (8,910 )
Other income (expense), net   149     (260 )   270     (952 )
Net loss $ (12,663 ) $ (4,806 ) $ (24,023 ) $ (9,862 )
 
 
Net loss per common share – basic and diluted $ (0.56 ) $ (8.90 ) $ (1.06 ) $ (18.32 )
Weighted average number of common shares outstanding – basic and diluted 22,575 540 22,562 538
 

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