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Patent
Ra Pharmaceuticals | Date: 2015-06-12

The present invention provides modulators of complement activity. Also provided are methods of utilizing such modulators as therapeutics.


Patent
Ra Pharmaceuticals | Date: 2017-04-19

The present invention provides modulators of complement activity. Also provided are methods of utilizing such modulators as therapeutics.


CAMBRIDGE, Mass.--(BUSINESS WIRE)--Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Ra Pharma is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for the treatment of complement-mediated diseases. RA101495, the Company’s lead clinical candidate, is a synthetic macrocyclic peptide inhibitor of complement component 5 (C5). The molecule is currently in Phase 2 clinical development as a self-administered subcutaneous (SC) injection for the treatment of PNH, a rare, chronic, life-threatening blood disorder where red blood cells are attacked and destroyed by the complement system. “There is an urgent need for new treatment options for patients suffering from PNH. The current standard of care requires biweekly intravenous infusions, a dosing regimen that imposes a severe burden on patients, providers, and caregivers,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “We have designed RA101495 for once-daily, subcutaneous self-administration, an approach which has the potential to ease this burden, improve convenience, and provide much-needed dosing flexibility. We are encouraged by our initial Phase 2 data in PNH patients, which showed near-complete inhibition of hemolysis and a favorable safety and tolerability profile. We look forward to advancing our PNH program and providing additional data updates around year-end.” The FDA grants Orphan Drug Designation status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States at the time of designation. Orphan Drug Designation qualifies the sponsor certain development incentives, including tax credits for qualified clinical testing. RA101495 received Orphan Drug Designation for the treatment of PNH in Europe in November 2016. 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 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/. 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 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, and 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.


CAMBRIDGE, Mass.--(BUSINESS WIRE)--Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced initial data from the Company’s global Phase 2 clinical program evaluating RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Ra Pharma is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for the treatment of complement-mediated diseases and is developing RA101495 as a novel, subcutaneously-administered (SC) inhibitor of complement component 5 (C5). The dose-finding Phase 2 clinical program is designed to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of RA101495 in patients with PNH (see About RA101495 Phase 2 Clinical Program, below). Currently, the study has three eculizumab-naïve patients enrolled, of which two have completed seven weeks of follow-up and are included in the Company’s initial data set. Review of these initial data enables the opening of Cohort B, which will enroll patients switching from eculizumab, the current standard of care, for PNH. At the week 6 visit, one patient reported recurrence of PNH symptoms associated with hemoglobinuria and an elevated LDH level. This episode of breakthrough hemolysis occurred in the setting of an intercurrent illness, judged by the investigator to be most likely due to a viral infection. The episode was transient and resolved rapidly with LDH levels returning to 1.3X ULN by the week 7 visit with resolution of hemolytic symptoms. “Currently, the only approved treatment for PNH is a monoclonal antibody which must be administered intravenously every two weeks by healthcare professionals,” said Principal Investigator Anita Hill, M.D., PhD, MRCP, FRCPath, Consultant Haematologist for Leeds Teaching Hospitals NHS Trust, UK, and Lead Clinician for the National PNH Service in England. “As a once daily, self-administered, subcutaneous therapy, RA101495 has the potential to offer a convenient option for patients with PNH. These initial data on eculizumab-naïve patients are encouraging, and I look forward to seeing data from patients switching from eculizumab to RA101495.” “We are pleased by the initial results from this study, our first in PNH patients, whose primary purpose is to gain important safety, preliminary efficacy, PK and PD data in the target patient population,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “These data will help inform dose selection for pivotal trials and will help refine our clinical development strategy. We look forward to giving you an update on our progress around year-end.” 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/. 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 lactate dehydrogenase from baseline to the mean level from Week 6 to week 12. 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 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.


CAMBRIDGE, Mass.--(BUSINESS WIRE)--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.” 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. 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/. 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. 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 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.


CAMBRIDGE, Mass.--(BUSINESS WIRE)--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 the presentation of scientific data at the 22nd Congress of the European Hematology Association (EHA), June 22-25, 2017 in Madrid, Spain. “The data presented at EHA showcase a novel class of orally bioavailable small molecules that bind to C5 with high affinity and inhibit its cleavage into C5a and C5b,” said Alonso Ricardo, Senior Vice President and Head of Research at Ra Pharma. “These findings demonstrate the feasibility of an orally-administered therapy for complement-mediated disorders and support the continued advancement of these molecules.” Inhibition of C5 is a clinically-validated approach for the therapeutic treatment of complement-mediated disorders. Ra Pharma’s portfolio includes drug candidates for several such disorders, including paroxysmal nocturnal hemoglobinuria (PNH), myasthenia gravis (MG) and lupus nephritis (LN). The Company is pursuing the identification of orally-available inhibitors of C5 with the goal of providing additional options for patients diagnosed with these and other complement-mediated disorders. “We are encouraged by our scientists’ ability to discover and characterize a small molecule inhibitor of complement C5,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “These data show that C5 inhibition can be accomplished with an orally-bioavailable small molecule, and we hope that compounds from this class can be advanced to provide a new treatment option for a broader spectrum of patients.” The presentation is available at http://rapharma.com/science/presentations-and-publications/. A summary of the data presented by Ra Pharma at the conference is as follows: Title: Discovery of Orally Bioavailable Small Molecules for Inhibition of Complement C5 Session Title: Bone marrow failure and PNH (oral presentation) Presenter: Alonso Ricardo, PhD, Senior Vice President and Head of Research, Ra Pharma Date/Time: Saturday, June 24, 5:00- 5:15 PM CEST Location: Room N104 Abstract Code: S500 Summary: These initial studies describe a series of first-in-class, orally bioavailable small molecules that bind to C5, inhibiting the protein’s cleavage into C5a and C5b. These molecules possess favorable pharmacokinetic properties, exhibiting up to 50% oral availability in preclinical species. Molecules from this class were found to bind to a unique site on complement C5 with a 1:1 stoichiometry. Notably, binding to this site also inhibited the cleavage of C5 containing the R885H polymorphism, a mutation that confers resistance to eculizumab, today’s standard of care in PNH. The extensive in vitro and in vivo data, along with a high-resolution crystal structure, support the feasibility of developing alternatives to injectable treatment of disorders of complement regulation. 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 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 the results of preclinical studies may not be indicative of the results of clinical studies; 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.


News Article | June 21, 2017
Site: www.businesswire.com

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced that it will host a conference call and webcast on Tuesday, June 27, 2017 to discuss initial data from the Company’s global Phase 2 clinical program evaluating RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Early safety, tolerability, preliminary efficacy and pharmacodynamic data on the first two treatment naïve patients enrolled will be presented. Ra Pharma is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for the treatment of complement-mediated diseases and is developing RA101495 as a novel, subcutaneously-administered (SC) inhibitor of complement component 5 (C5). Details of the conference call and webcast are as follows: 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/. 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 Cohort C 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 lactate dehydrogenase from baseline to the mean level from Week 6 to week 12. 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 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.


CAMBRIDGE, Mass.--(BUSINESS WIRE)--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.” 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.


Patent
Ra Pharmaceuticals | Date: 2015-12-08

The present invention provides inhibitors and/or antagonists of plasma kallikrein. Also provided are methods of utilizing the inhibitors as therapeutics.


Patent
Ra Pharmaceuticals | Date: 2013-03-14

The present invention provides inhibitors and/or antagonists of plasma kallikrein. Also provided are methods of utilizing the inhibitors as therapeutics.

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