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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: 2015-06-12

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


Pipeline Analysis on Glomerulonephritis Reviewed by Company, Profiled Drugs and Therapeutic Development Glomerulonephritis is also known as glomerular nephritis (GN) or glomerular disease. It is a disease of the kidney, characterized by inflammation of the glomeruli. Albany, NY, May 17, 2017 --( The report provides a detailed analysis of Glomerulonephritis by main industries and major drugs. Request Free Sample Report: http://www.marketresearchhub.com/enquiry.php?type=S&repid=1068016 Glomerulonephritis is medical disease that damages the part of the kidney that filters blood. It is a serious illness that can be life-threatening and requires immediate treatment, the condition is of two type that includes acute, and chronic. The early symptoms of acute glomerulonephritis are puffiness of face in the morning, blood in the urine and urinating less than usual. The chronic form may develop silently over several years that consequently leads to complete kidney failure. The chronic form symptoms include blood or protein in the urine, high blood pressure, swelling of your ankles or face, frequent urination during night time and very bubbly or foamy urine. The report starts with the Glomerulonephritis overview and its therapeutics development. Further, the companies and universities/institutes are considered for this pipeline analysis; and are overviewed along with the products under development by the companies and universities. The glomerulonephritis therapeutics are further assessed by the target, mechanism of action, route of administration and molecular type. Moving on, the leading companies involved in therapeutic development for glomerulonephritis are listed with top players including Achillion Pharmaceuticals Inc., Alexion Pharmaceuticals Inc., Anthera Pharmaceuticals Inc., Biogen Inc., BLR Bio LLC, ChemoCentryx Inc., Dimerix Bioscience Pty Ltd, GlaxoSmithKline Plc, Mallinckrodt Plc, Omeros Corp, Pfizer Inc., Pharmalink AB, Ra Pharmaceuticals Inc., Retrophin Inc., Rigel Pharmaceuticals Inc., Shire Plc and Visterra Inc. Browse Full Report with TOC - http://www.marketresearchhub.com/report/glomerulonephritis-pipeline-review-h1-2017-report.html This detailed study also includes the major drugs listed for glomerulonephritis that consist of (irbesartan + propagermanium), ACH-4471, AMY-101, AVX-002, BaxB-01, BaxG-03, belimumab, blisibimod, BLR-400, budesonide, CCX-140, corticotropin, eculizumab, fostamatinib disodium, iosmapimod, monoclonal antibodies to inhibit ITGAM for cardiovascular, immunology and Kidney Diseases. The other drugs are OMS-721, PF-1355, recombinant enzyme for immunoglobulin, rituximab, SHP-627, sparsentan, TM-5484, vaccine to target CD40 for membranous glomerulonephritis, VAR-200, VIS-649, small molecule to inhibit factor D for PNH and dense deposit disease, and other. The report is followed by the press release and news that further provide the facts andfigures for Glomerulonephritis and concluded with the list of tables that elaborate the research. About Market Research Hub Market Research Hub (MRH) is a next-generation reseller of research reports and analysis. MRH’s expansive collection of market research reports has been carefully curated to help key personnel and decision makers across industry verticals to clearly visualize their operating environment and take strategic steps. MRH functions as an integrated platform for the following products and services: Objective and sound market forecasts, qualitative and quantitative analysis, incisive insight into defining industry trends, and market share estimates. Our reputation lies in delivering value and world-class capabilities to our clients. Albany, NY, May 17, 2017 --( PR.com )-- According to a latest pipeline assessment on the Glomerulonephritis, it has been analysed that the main factors that result in glomerulonephritis are family history, strep throat, immune diseases, such as lupus, type 1 & type 2 diabetes and viruses, such as hepatitis B virus, HIV and hepatitis C virus. The rapid increase in these factors has directly augmented the glomerulonephritis disease. Recently, a detailed analysis has been added to Market Research Hub’s vast database (MRH), and titled as “Glomerulonephritis - Pipeline Review, H1 2017.”The report provides a detailed analysis of Glomerulonephritis by main industries and major drugs.Request Free Sample Report: http://www.marketresearchhub.com/enquiry.php?type=S&repid=1068016Glomerulonephritis is medical disease that damages the part of the kidney that filters blood. It is a serious illness that can be life-threatening and requires immediate treatment, the condition is of two type that includes acute, and chronic. The early symptoms of acute glomerulonephritis are puffiness of face in the morning, blood in the urine and urinating less than usual. The chronic form may develop silently over several years that consequently leads to complete kidney failure. The chronic form symptoms include blood or protein in the urine, high blood pressure, swelling of your ankles or face, frequent urination during night time and very bubbly or foamy urine.The report starts with the Glomerulonephritis overview and its therapeutics development. Further, the companies and universities/institutes are considered for this pipeline analysis; and are overviewed along with the products under development by the companies and universities. The glomerulonephritis therapeutics are further assessed by the target, mechanism of action, route of administration and molecular type. Moving on, the leading companies involved in therapeutic development for glomerulonephritis are listed with top players including Achillion Pharmaceuticals Inc., Alexion Pharmaceuticals Inc., Anthera Pharmaceuticals Inc., Biogen Inc., BLR Bio LLC, ChemoCentryx Inc., Dimerix Bioscience Pty Ltd, GlaxoSmithKline Plc, Mallinckrodt Plc, Omeros Corp, Pfizer Inc., Pharmalink AB, Ra Pharmaceuticals Inc., Retrophin Inc., Rigel Pharmaceuticals Inc., Shire Plc and Visterra Inc.Browse Full Report with TOC - http://www.marketresearchhub.com/report/glomerulonephritis-pipeline-review-h1-2017-report.htmlThis detailed study also includes the major drugs listed for glomerulonephritis that consist of (irbesartan + propagermanium), ACH-4471, AMY-101, AVX-002, BaxB-01, BaxG-03, belimumab, blisibimod, BLR-400, budesonide, CCX-140, corticotropin, eculizumab, fostamatinib disodium, iosmapimod, monoclonal antibodies to inhibit ITGAM for cardiovascular, immunology and Kidney Diseases. The other drugs are OMS-721, PF-1355, recombinant enzyme for immunoglobulin, rituximab, SHP-627, sparsentan, TM-5484, vaccine to target CD40 for membranous glomerulonephritis, VAR-200, VIS-649, small molecule to inhibit factor D for PNH and dense deposit disease, and other. The report is followed by the press release and news that further provide the facts andfigures for Glomerulonephritis and concluded with the list of tables that elaborate the research.About Market Research HubMarket Research Hub (MRH) is a next-generation reseller of research reports and analysis. MRH’s expansive collection of market research reports has been carefully curated to help key personnel and decision makers across industry verticals to clearly visualize their operating environment and take strategic steps.MRH functions as an integrated platform for the following products and services: Objective and sound market forecasts, qualitative and quantitative analysis, incisive insight into defining industry trends, and market share estimates. Our reputation lies in delivering value and world-class capabilities to our clients. Click here to view the list of recent Press Releases from Market Research Hub


News Article | May 16, 2017
Site: www.businesswire.com

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 that Doug Treco, PhD, President and Chief Executive Officer, will present at the UBS 2017 Global Healthcare Conference on Monday, May 22, 2017 at 10:00 a.m. E.T. The conference is being held at the Grand Hyatt New York. The presentation will be webcast live and can be accessed by visiting the investor relations section of the Company’s website, www.rapharma.com. A replay of the presentation will also be available and archived on the site for three weeks. 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.


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.


News Article | April 26, 2017
Site: www.businesswire.com

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced that it has initiated dosing in 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 and potent subcutaneously-administered (SC) inhibitor of complement component 5 (C5). Inhibition of C5 is a clinically validated approach for the control of complement-induced hemolysis, the process responsible for the rupture and destruction of red blood cells (RBCs) in patients with PNH. “Commencement of dosing in our Phase 2 clinical program for RA101495 in PNH is an important step in bringing a potential new therapy to patients with this rare and life-threatening blood disorder,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “Building on our promising Phase 1 data, our Phase 2 program is designed to demonstrate RA101495’s safety and preliminary efficacy in PNH patients. RA101495 is a convenient, once daily, self-administered subcutaneous dose and represents a significant advancement in the treatment of PNH. We look forward to sharing initial data from our Phase 2 program in mid-2017 and additional data by year-end.” The global Phase 2 program is designed to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetics, and pharmacodynamics of RA101495 in patients with PNH. The program is comprised of two open-label Phase 2 studies, one conducted outside the U.S. and one conducted in the U.S. All patients enrolled in the Phase 2 studies will receive a single, 0.3 mg/kg SC loading dose of RA101495, followed by 0.1 mg/kg SC daily thereafter. Following two weeks of treatment and based on a review of safety and efficacy data, patients will continue with 0.1 mg/kg daily or, if needed, the dose can be increased to 0.3 mg/kg daily to achieve adequate control of hemolysis. 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. 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 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 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.


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 joins forces with 30 million health care advocates around the world for the 10th annual Rare Disease Day®. Rare Disease Day, which is always the last day of February, is an annual awareness day dedicated to elevating public understanding of rare diseases and calling attention to the special challenges people living with rare diseases face. This year’s focus is on research and the global slogan for 2017 is “With Research, Possibilities are Limitless.” “A decade into its existence, the annual Rare Disease Day has achieved a noteworthy milestone in its efforts calling attention to the public health issues associated with rare diseases, a mission Ra Pharma is proud to support,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “As we prepare to initiate two Phase 2 clinical trials of our lead compound RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and refractory generalized myasthenia gravis (rMG), as well as a Phase 1b clinical trial supporting development in lupus nephritis (LN), we are confident in our ability to address the unmet need for people living with rare diseases where there are limited or no treatment options.” According to the National Institutes of Health (NIH), a disease is rare if it affects fewer than 200,000 people. Nearly 1 in 10 Americans live with a rare disease—affecting 30 million people—and nearly half of these patients are children. There are more than 7,000 rare diseases and only approximately 450 FDA-approved medical treatments. Rare Disease Day takes place every year on the last day of February (February 28 or February 29 in a leap year)—the rarest date on the calendar—to underscore the nature of rare diseases and what patients face. It was established in Europe in 2008 by EURORDIS, the organization representing rare disease patients in Europe, and is now observed in more than 80 nations. Rare Disease Day is sponsored in the U.S. by the National Organization for Rare Disorders (NORD)®, the largest and leading independent, nonprofit organization committed to the identification, treatment, and cure of rare diseases. For more information about Rare Disease Day in the U.S., go to www.rarediseaseday.us. For information about global activities, go to www.rarediseaseday.org. To search for information about rare diseases, visit NORD’s website, www.rarediseases.org. 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. 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. 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. Ra Pharma’s proprietary Extreme Diversity™ mRNA display platform allows us 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 our emerging pipeline projects in Factor D and C1. 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; as well as the other factors discussed in the “Risk Factors” section in Ra Pharma’s most recently filed Quarterly Report on Form 10-Q, 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.


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 that Doug Treco, PhD, President and Chief Executive Officer, will present at the Cowen and Company 37th Annual Health Care Conference on Tuesday, March 7, 2017 at 11:20 a.m. E.T. The conference is being held at the Boston Marriott Copley Place. The presentation will be webcast live and can be accessed by visiting the investor relations section of the Company’s website, www.rapharma.com. A replay of the presentation will also be available and archived on the site for three weeks. 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.

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