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The present disclosure relates to pantothenate derivatives for the treatment of neurologic disorders (such as pantothenate kinase-associated neurodegeneration), pharmaceutical compositions containing such compounds, and their use in treatment of neurologic disorders.


News Article | May 11, 2017
Site: www.businesswire.com

WILMINGTON, N.C.--(BUSINESS WIRE)--Pharmaceutical Product Development, LLC, (PPD) today announced it recently has added two new leaders in product development, strengthening the global contract research organization’s therapeutic expertise and ability to aid biopharmaceutical clients in delivering life-changing therapies to patients. “PPD’s global team of scientific, medical and strategic experts provides our clients expertise that helps reduce the cost and time associated with bringing medicines to market,” said Rob Dow, senior vice president of medical affairs for PPD. “By joining this team, these leaders deepen our expertise in neuroscience, pediatrics and rare diseases – areas that are very important to PPD and our customers.” Stephen Peroutka, M.D., Ph.D., has joined PPD to serve as vice president of global product development and therapeutic area head for neuroscience. Peroutka has served as an advisor and consultant to several biopharmaceutical companies. He previously served as chief medical officer at Semnur Pharmaceuticals and as chief medical officer and executive vice president of NeurogesX Inc. Earlier, he served as vice president of neurosciences for a large contract research organization (CRO); chief medical officer of Zogenix Inc.; franchise leader for pain conditions at Johnson & Johnson Inc.; founder, CEO and president of Synergia Pharma Inc.; and founder, CEO and president of Spectra Biomedical Inc., a startup focused on identification of genes in migraine, later acquired by GSK. Peroutka served as chief of the neurology service at the Palo Alto Veteran’s Administration Hospital. He earned both a medical and doctoral degree in pharmacology and experimental therapeutics from Johns Hopkins University School of Medicine. Horacio Plotkin, M.D., FAAP, joined PPD as vice president of global product development in the pediatrics and rare diseases therapeutic area and also serves as medical lead for the Rare Disease and Pediatric Center of Excellence. Plotkin most recently served as global clinical development lead at Shire. Previously, he spent three years as chief medical officer of Retrophin, Inc., and held medical director positions at Alexion Pharmaceuticals, Enobia Pharma and Genzyme Corp. He spent 20 years as a practicing pediatrician in the field of pediatric rare diseases. Plotkin serves as adjunct associate professor of pediatrics and orthopedic surgery at the University of Nebraska School of Medicine. He earned his medical degree from the University of Buenos Aires School of Medicine. PPD is a leading global contract research organization providing comprehensive, integrated drug development, laboratory and lifecycle management services. Our clients and partners include pharmaceutical, biotechnology, medical device, academic and government organizations. With offices in 47 countries and more than 19,000 professionals worldwide, PPD applies innovative technologies, therapeutic expertise and a firm commitment to quality to help clients and partners bend the cost and time curve of drug development to deliver life-changing therapies that improve health. For more information, visit www.ppdi.com. Any statements made in this news release that are not statements of historical fact, including statements about the future performance of personnel, are forward-looking statements that involve a number of risks and uncertainties. These statements often include words such as “anticipate,” “expect,” “suggests,” “plan,” “believe,” “intend,” “estimates,” “targets,” “projects,” “should,” “could,” “would,” “may,” “might,” “will,” “forecast” and other similar expressions. The forward-looking statements contained in this news release are subject to and involve risks, uncertainties and assumptions, and therefore you should not place undue reliance on them. Although PPD believes these forward-looking statements are based on reasonable assumptions at the time they are made, many factors are beyond PPD’s ability to control or predict and could affect the outcome of the subject matter of this news release and our actual financial results, and therefore the outcome and results might differ materially from those expressed in the forward-looking statements. Additional factors that might materially affect the forward-looking statements include, but are not limited to: our ability to recruit, retain and motivate key personnel; the competitive nature of the drug development services industry; changes in trends in the biopharmaceutical industry; rapid technological changes that make our services less competitive or obsolete; the impacts of political, economic and/or regulatory changes on the health care industry; the fact that our backlog may not accurately predict or convert into service revenue; the termination, delay or change in scope of our contracts; industry, customer or therapeutic concentration; the pricing of and cost management of customer contracts; information and communication systems failures; contractual failures; regulatory and ethical standards failures; our ability to attract investigators and enroll patients in clinical trials; violations of laws governing privacy, conduct of clinical trials and/or other pharmaceutical research; competition between existing and potential customers; management of business restructurings and acquisitions; risk relating to the performance of drug development services and our insurance coverages, if any, for such risks; U.S. or international economic, currency, political and other risks; changes in existing or interpretations of tax laws; factors impacting the value of our goodwill and intangible assets; and other factors. PPD assumes no obligation and expressly disclaims any duty to revise or update any forward-looking statements, or make any new forward-looking statement, whether as a result of new information, future events or otherwise, except as required by applicable law. PPD is not responsible for updating the information contained in this news release beyond the published date, or for changes made to this news release by wire services or internet service providers or any other party.


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 4, 2017
Site: globenewswire.com

Phase 3 FORT study of RE-024 in PKAN to begin dosing mid-2017 Protocol development underway for pivotal Phase 3 trial of sparsentan in FSGS SAN DIEGO, May 04, 2017 (GLOBE NEWSWIRE) -- Retrophin, Inc. (NASDAQ:RTRX) today reported its first quarter 2017 financial results and provided a corporate update. “I’m very pleased with our progress to start the year,” said Stephen Aselage, chief executive officer of Retrophin. “Our meeting with the FDA in the first quarter helped define the path forward for sparsentan, and progress continues on the development of a protocol that will enable us to begin the pivotal trial in FSGS. In addition, our recent advancements with RE-024 have positioned us to begin dosing the first PKAN patients in our Phase 3 study mid-year. These clinical achievements, along with strong operational performance during the quarter, have built solid momentum for the remainder of 2017.” Net product sales for the first quarter of 2017 were $33.6 million, compared to $29.0 million for the same period in 2016. The increase in net product sales is attributable to growth across the Company’s commercial products: Thiola®, Cholbam®, and Chenodal®. The Company reiterates its full-year 2017 guidance of $150.0 to $160.0 million in net product sales. Research and development (R&D) expenses for the first quarter of 2017 were $20.9 million, compared to $14.7 million for the same period in 2016. The increase is largely attributable to enhanced clinical efforts related to sparsentan and RE-024. On a non-GAAP adjusted basis, R&D expenses were $18.1 million for the first quarter of 2017, compared to $12.1 million for the same period in 2016. Selling, general and administrative (SG&A) expenses for the first quarter of 2017 were $23.1 million, compared to $19.1 million for the same period in 2016. The difference is largely attributable to an increase in marketing expense related to the growth of the Company’s commercial portfolio, and a one-time benefit of $3.0 million in the first quarter of 2016 due to a settlement covering disputed legal fees. On a non-GAAP adjusted basis, SG&A expenses were $14.5 million for the first quarter of 2017, compared to $11.0 million for the same period in 2016. Total other income for the first quarter of 2017 was $1.3 million, compared to $14.4 million for the same period in 2016. The decrease is largely due to a change in the fair value of derivative instruments as a result of changes in the Company’s stock price. Net loss for the first quarter of 2017 was $11.1 million, or $0.29 per basic share, compared to net income of $11.2 million, or $0.31 per basic share for the same period in 2016. On a non-GAAP adjusted basis, net income for the first quarter of 2017 was $0.3 million, or $0.01 per basic share, compared to $5.2 million, or $0.14 per basic share for the same period in 2016. As of March 31, 2017, the Company had cash, cash equivalents, marketable securities and note receivable of $295.3 million. Retrophin will host a conference call and webcast today, Thursday, May 4, 2017 at 4:30 p.m. ET to discuss development updates and first quarter 2017 financial results. To participate in the conference call, dial +1-855-219-9219 (U.S.) or +1-315-625-6891 (International), confirmation code 10209780 shortly before 4:30 p.m. ET. The webcast can be accessed at www.retrophin.com, in the Events and Presentations section, and will be archived for at least 30 days. A replay of the call will be available starting at 7:30 p.m. ET, May 4, 2017 until 7:30 p.m. ET, May 11, 2017. The replay number is +1-855-859-2056 (U.S.) or +1-404-537-3406 (International), confirmation code 10209780. Use of Non-GAAP Financial Measures To supplement Retrophin’s financial results and guidance presented in accordance with U.S. generally accepted accounting principles (GAAP), the Company uses certain non-GAAP adjusted financial measures in this press release and the accompanying tables. The Company believes that these non-GAAP financial measures are helpful in understanding its past financial performance and potential future results. They are not meant to be considered in isolation or as a substitute for comparable GAAP measures, and should be read in conjunction with the consolidated financial statements prepared in accordance with GAAP. Retrophin’s management regularly uses these supplemental non-GAAP financial measures internally to understand, manage and evaluate its business and make operating decisions. In addition, Retrophin believes that the use of these non-GAAP measures enhances the ability of investors to compare its results from period to period and allows for greater transparency with respect to key financial metrics the Company uses in making operating decisions. Investors should note that these non-GAAP financial measures are not prepared under any comprehensive set of accounting rules or principles and do not reflect all of the amounts associated with the Company’s results of operations as determined in accordance with GAAP. Investors should also note that these non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future the Company may exclude other items, or cease to exclude items that it has historically excluded, for purposes of its non-GAAP financial measures; because of the non-standardized definitions, the non-GAAP financial measures as used by the Company in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by the Company’s competitors and other companies. As used in this press release, (i) the historical non-GAAP net income (loss) measures exclude from GAAP net income (loss), as applicable, revaluation of acquisition related contingent consideration, stock-based compensation expense, depreciation and amortization expense, change in fair value of derivative instruments; income tax benefit; (ii) the historical non-GAAP SG&A expense measures exclude from GAAP SG&A expenses, as applicable, stock-based compensation expense, and depreciation and amortization expense; (iii) the historical non-GAAP R&D expense measures exclude from GAAP R&D expenses, as applicable, stock-based compensation expense, and depreciation and amortization expense. Retrophin is a fully integrated biopharmaceutical company dedicated to delivering life-changing therapies to people living with rare diseases who have few, if any, treatment options. The Company’s approach centers on its pipeline featuring late-stage assets targeting rare diseases with significant unmet medical needs, including sparsentan for focal segmental glomerulosclerosis (FSGS), a disorder characterized by progressive scarring of the kidney often leading to end-stage renal disease, and RE-024 for pantothenate kinase-associated neurodegeneration (PKAN), a life-threatening neurological disorder that typically begins in early childhood. Research exploring the potential of early-stage assets in several rare diseases is also underway. Retrophin’s R&D efforts are supported by revenues from the Company’s commercial products Thiola®, Cholbam®, and Chenodal®. This press release contains "forward-looking statements" as that term is defined in the Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, these statements are often identified by the words "may", "might", "believes", "thinks", "anticipates", "plans", "expects", "intends" or similar expressions. In addition, expressions of our strategies, intentions or plans are also forward-looking statements. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties associated with the Company’s business and finances in general, success of its commercial products as well as risks and uncertainties associated with the Company's preclinical and clinical stage pipeline. Specifically, the Company faces risks associated with market acceptance of its marketed products including efficacy, safety, price, reimbursement and benefit over competing therapies. The risks and uncertainties the Company faces with respect to its preclinical and clinical stage pipeline include risk that the Company's clinical candidates will not be found to be safe or effective and that planned clinical trials will not proceed as planned. Specifically, the Company faces the risk that the planned Phase 3 clinical trial of sparsentan will not demonstrate that sparsentan is safe or effective or serve as a basis for accelerated approval of sparsentan as planned; risk that the Phase 3 clinical trial of RE-024 will not demonstrate that RE-024 is safe or effective or serve as the basis for an NDA filing as planned; and risk that the Company’s product candidates will not be approved for efficacy, safety, regulatory or other reasons, and for each of the programs, risk associated with enrollment of clinical trials for rare diseases and risk that ongoing or planned clinical trials may not succeed or may be delayed for safety, regulatory or other reasons. The Company faces risk that it will be unable to raise additional funding that may be required to complete development of any or all of its product candidates; risk relating to the Company's dependence on contractors for clinical drug supply and commercial manufacturing; uncertainties relating to patent protection and intellectual property rights of third parties; and risks and uncertainties relating to competitive products and technological changes that may limit demand for the Company's products. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Investors are referred to the full discussion of risks and uncertainties as included in the Company's most recent Form 10-K, Form 10-Q and other filings with the Securities and Exchange Commission.


News Article | April 20, 2017
Site: globenewswire.com

SAN DIEGO, April 20, 2017 (GLOBE NEWSWIRE) -- Retrophin, Inc. (Nasdaq:RTRX) today announced it will report first quarter 2017 financial results on Thursday, May 4, 2017 after the close of the U.S. financial markets. The Company will host a conference call and webcast to discuss the financial results and provide a general business update at 4:30 p.m. ET. A replay of the call will be available from 7:30 p.m. ET, May 4, 2017 to 7:30 p.m. ET, May 11, 2017. The replay number is +1 (855) 859-2056 (U.S.) or +1 (404) 537-3406 (International), confirmation code 10209780. Retrophin is a fully integrated biopharmaceutical company dedicated to delivering life-changing therapies to people living with rare diseases who have few, if any, treatment options. The Company's approach centers on its pipeline featuring late-stage candidates targeting rare diseases with significant unmet medical needs, including sparsentan for focal segmental glomerulosclerosis (FSGS), a disorder characterized by progressive scarring of the kidney often leading to end-stage renal disease, and RE-024 for pantothenate kinase-associated neurodegeneration (PKAN), a life-threatening neurological disorder that typically begins in early childhood. Research exploring the potential of early-stage assets in several rare diseases is also underway. Retrophin's R&D efforts are supported by revenues from the Company's commercial products, Thiola®, Cholbam®, and Chenodal®.


The present disclosure relates to pantothenate derivatives for the treatment of neurologic disorders (such as pantothenate kinase-associated neurodegeneration), pharmaceutical compositions containing such compounds, and their use in treatment of neurologic disorders.


Compounds having the following formula (E): Formula E or a pharmaceutically acceptable salt thereof, wherein R, R, R, X and n are as defined herein are provided. Methods comprising use of such compounds for the treatment of neurologic disorders, such as pantothenate kinase-associated neurodegeneration, and pharmaceutical compositions containing such compounds, and their use in treatment of neurologic disorders are also provided.


The present disclosure relates to pantothenate derivatives for the treatment of neurologic disorders (such as pantothenate kinase-associated neurodegeneration), pharmaceutical compositions containing such compounds, and their use in treatment of neurologic disorders.


The present disclosure relates to pantothenate derivatives for the treatment of neurologic disorders (such as pantothenate kinase-associated neurodegeneration), pharmaceutical compositions containing such compounds, and their use in treatment of neurologic disorders.


News Article | February 15, 2017
Site: globenewswire.com

SAN DIEGO, Feb. 15, 2017 (GLOBE NEWSWIRE) -- Retrophin, Inc. (Nasdaq:RTRX) today announced it will report fourth quarter and full year 2016 financial results on Wednesday, March 1, 2017 after the close of the U.S. financial markets. The Company will host a conference call and webcast to discuss the financial results and provide a general business update at 5:00 p.m. ET. A replay of the call will be available from 8:00 p.m. ET, March 1, 2017 to 8:00 p.m. ET, March 8, 2017. The replay number is +1 (855) 859-2056 (U.S.) or +1 (404) 537-3406 (International), confirmation code 70808962. Retrophin is a fully integrated biopharmaceutical company dedicated to delivering life-changing therapies to people living with rare diseases who have few, if any, treatment options. The Company’s approach centers on its pipeline featuring late-stage assets targeting rare diseases with significant unmet medical needs, including sparsentan for focal segmental glomerulosclerosis (FSGS), a disorder characterized by progressive scarring of the kidney often leading to end-stage renal disease, and RE-024 for pantothenate kinase-associated neurodegeneration (PKAN), a life-threatening neurological disorder that typically begins in early childhood. Research exploring the potential of early-stage assets in several rare diseases is also underway. Retrophin’s R&D efforts are supported by revenues from the Company’s commercial products Thiola®, Cholbam® and Chenodal®.

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