ApoPharma Inc.

Toronto, Canada

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News Article | May 9, 2017
Site: marketersmedia.com

— The Psoriasis market report titled “Psoriasis - Pipeline Review, H1 2017" report provides an overview of Psoriasis clinical trials scenario. Browse the 209 Tables and 10 Figures, 150 Company Profiles, Spread across 648 Pages Report Available at http://www.reportsnreports.com/reports/983978-psoriasis-pipeline-review-h1-2017.html Psoriasis market companies are 3SBio Inc,AbbVie Inc,Abeome Corp,AbGenomics International Inc, Addex Therapeutics Ltd, Adello Biologics LLC, Advinus Therapeutics Ltd, Affibody AB, Albireo Pharma Inc, Alfacyte Ltd, Allergan Plc, Almirall SA, Alteogen Inc, Alvotech Iceland, Amgen Inc, Anacor Pharmaceuticals Inc, AnaptysBio Inc, ApoPharma Inc, Arbor Pharmaceuticals LLC, Arena Pharmaceuticals Inc, Argos Therapeutics Inc, Arrien Pharmaceuticals LLC, AstraZeneca Plc, Athenex Inc, Atlantic Bio Sci LLC, Aurigene Discovery Technologies Ltd, Aurinia Pharmaceuticals Inc, Bayer AG, Beta Pharma Inc, BioApex sro, Biocad, Biocon Ltd, BioLingus AG, BioMAS Ltd, Biomics Biotechnologies Co Ltd,Bionomics Ltd, Bionovis SA, BirchBioMed Inc,Boehringer Ingelheim GmbH,Brickell Biotech Inc,Bristol-Myers Squibb Company,C4X Discovery Holdings PLC,CalciMedica Inc,Can-Fite BioPharma Ltd,Celgene Corp,Cell Medica Ltd,Cellceutix Corp,ChemoCentryx Inc,Chipscreen Biosciences Ltd,ChironWells GmbH,Coherus BioSciences Inc,Compugen Ltd,Concenter BioPharma Silkim Ltd,Crescita Therapeutics Inc,CritiTech Inc,Curapel Ltd,Dermala Inc,Dr. August Wolff GmbH & Co KG Arzneimittle,DURECT Corp,EA Pharma Co Ltd,Eli Lilly and Company,ELORAC Inc,Exicure Inc,Foamix Pharmaceuticals Ltd,Forward Pharma A/S,Galapagos NV,Galderma SA many more.. Place Order to This Report at http://www.reportsnreports.com/purchase.aspx?name=983978 . The Psoriasis market pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages. The pipeline guide reviews pipeline therapeutics for Psoriasis (Immunology) by companies and universities/research institutes based on information derived from company and industry-specific sources. The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages. The pipeline guide encapsulates all the dormant and discontinued pipeline projects. The pipeline guide reviews latest news related to pipeline therapeutics for Psoriasis (Immunology). The Psoriasis (Immunology) pipeline guide also reviews of key players involved in therapeutic development for Psoriasis and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Filing rejected/Withdrawn, Phase III, Phase II, Phase I, IND/CTA Filed, Preclinical, Discovery and Unknown stages are 14, 1, 21, 44, 42, 1, 103, 32 and 9 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 8 and 6 molecules, respectively. Psoriasis (Immunology) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. Reasons to buy • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies. • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage. • Find and recognize significant and varied types of therapeutics under development for Psoriasis (Immunology). • Classify potential new clients or partners in the target demographic. • Develop tactical initiatives by understanding the focus areas of leading companies. • Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics. • Formulate corrective measures for pipeline projects by understanding Psoriasis (Immunology) pipeline depth and focus of Indication therapeutics. • Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope. • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline. About Us: ReportsnReports.com is your single source for all market research needs. Our database includes 500,000+ market research reports from over 95 leading global publishers & in-depth market research studies of over 5000 micro markets. With comprehensive information about the publishers and the industries for which they publish market research reports, we help you in your purchase decision by mapping your information needs with our huge collection of reports. For more information, please visit http://www.reportsnreports.com/reports/983978-psoriasis-pipeline-review-h1-2017.html


News Article | June 12, 2017
Site: www.prnewswire.com

"This ruling is of critical importance, as it will allow biosimilars to reach patients and physicians sooner." said Steve Lydeamore, president, Apobiologix. The Supreme Court invited the parent company of Apobiologix to submit this briefing, based on the company's earlier submission of a Petitionfor Certiorari, which was denied in December.  The Petition for Certiorari filed by the company questioned appropriate timing for the currently-required 180-days' notice to brand companies from biosimilar companies who have followed the patent dance. The patent dance is a formal procedure with strict timing and sequencing requirements for resolving patent disputes between the biosimilar company and the company marketing the reference product. In today's ruling, the Supreme Court has allowed the timing of the 180-days' notice for companies to come before regardless of if they had participated in the "patent dance" or not. About Biosimilars Biosimilar medicines are biologic therapies developed to offer patients additional treatment options with similar therapeutic value to existing products. Biosimilars are required by the FDA to demonstrate that there are no clinically meaningful differences in terms of the safety, purity, and potency between the biosimilar product and the previously licensed biologic drug. About Apobiologix Apobiologix is a leader in the field of biosimilar development. As a division of ApoPharma USA, Inc. which is part of the Apotex group of companies, Apobiologix is focused on developing innovative biologic products, aimed at providing patients and providers with high-quality medicines. For more information about Apobiologix, go to www.apobiologix.com. To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/apobiologix-applauds-supreme-court-decision-in-sandoz-biosimilar-case-300472675.html


Latest report "Amyotrophic Lateral Sclerosis - Pipeline Review, H1 2017", provides comprehensive information on the therapeutics under development for Amyotrophic Lateral Sclerosis (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Complete report on Amyotrophic Lateral Sclerosis - Pipeline Review, H1 2017 spread across 455 pages is available at http://www.rnrmarketresearch.com/amyotrophic-lateral-sclerosis-pipeline-review-h1-2017-market-report.html . Amyotrophic lateral sclerosis (ALS) is the most common degenerative disease of the motor neuron system. The disorder is named for its underlying pathophysiology, with amyotrophy referring to the atrophy of muscle fibers, which are denervated as their corresponding anterior horn cells degenerate. Lateral sclerosis refers to the changes seen in the lateral columns of the spinal cord as upper motor neuron (UMN) axons in these areas degenerate and are replaced by fibrous astrocytes (gliosis). Symptoms include difficulty breathing, difficulty swallowing, head drop due to weakness of the neck muscles, muscle cramps, speech problems, such as a slow or abnormal speech pattern (slurring of words), voice changes, hoarseness, weight loss. Amyotrophic Lateral Sclerosis (Central Nervous System) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. Order a copy of this research report at http://www.rnrmarketresearch.com/contacts/purchase?rname=1019641. (This report is available at up to 25% Discount till June 02nd 2017.) Main key players of Amyotrophic Lateral Sclerosis pipeline are 2-BBB Medicines BV, AB Science SA, Anavex Life Sciences Corp, Angion Biomedica Corp, Apogenix GmbH, ApoPharma Inc, ArmaGen Inc, Avanir Pharmaceuticals Inc, BioCrea GmbH, Biogen Inc, BioHealthonomics Inc, BrainStorm Cell Therapeutics Inc, Catabasis Pharmaceuticals Inc, Chronos Therapeutics Ltd, ContraVir Pharmaceuticals Inc, Corcept Therapeutics Inc, Corestem Inc, Cytokinetics Inc, Daval International Ltd, Edison Pharmaceuticals Inc, Eisai Co Ltd, Ensemble Therapeutics Corp, Evotec AG, F. Hoffmann-La Roche Ltd, Flex Pharma Inc, FPRT Bio Inc, Gemac SA, Genentech Inc, Genervon Biopharmaceuticals LLC, GeNeuro SA more.. The Amyotrophic Lateral Sclerosis pipeline guide provides a snapshot of the global therapeutic landscape of Amyotrophic Lateral Sclerosis (Central Nervous System). The pipeline guide reviews pipeline therapeutics for Amyotrophic Lateral Sclerosis (Central Nervous System) by companies and universities/research institutes based on information derived from company and industry-specific sources. The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages. The Report "Amyotrophic Lateral Sclerosis Global Clinical Trials Review, H1, 2017" provides an overview of Amyotrophic Lateral Sclerosis clinical trials scenario. This report provides top line data relating to the clinical trials on Amyotrophic Lateral Sclerosis. Report includes an overview of trial numbers and their average enrollment in top countries conducted across the globe. The report offers coverage of disease clinical trials by region, country (G7 & E7), phase, trial status, end points status and sponsor type. Report also provides prominent drugs for in-progress trials (based on number of ongoing trials). Clinical Trial Reports are generated using proprietary database - Pharma eTrack Clinical trials database. Clinical trials are collated from 80+ different clinical trial registries, conferences, journals, news etc across the globe. Clinical trials database undergoes periodic update by dynamic process. Explore more reports of Therapeutics Market Research at http://www.rnrmarketresearch.com/reports/life-sciences/pharmaceuticals/therapeutics RnRMarketResearch.com is your one stop market research and industry analysis reports' library providing business data and intelligence information on thousands of micro markets with global as well as regional coverage. Category focused research, country reports, company profiles, regional and global industry profiles and guides to premium reports offering extensive coverage of other 20+ industries are all available in our library of syndicated market research reports.


Latest report "Amyotrophic Lateral Sclerosis - Pipeline Review, H1 2017", provides comprehensive information on the therapeutics under development for Amyotrophic Lateral Sclerosis (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Complete report on Amyotrophic Lateral Sclerosis - Pipeline Review, H1 2017 spread across 455 pages is available at http://www.rnrmarketresearch.com/amyotrophic-lateral-sclerosis-pipeline-review-h1-2017-market-report.html . Amyotrophic lateral sclerosis (ALS) is the most common degenerative disease of the motor neuron system. The disorder is named for its underlying pathophysiology, with amyotrophy referring to the atrophy of muscle fibers, which are denervated as their corresponding anterior horn cells degenerate. Lateral sclerosis refers to the changes seen in the lateral columns of the spinal cord as upper motor neuron (UMN) axons in these areas degenerate and are replaced by fibrous astrocytes (gliosis). Symptoms include difficulty breathing, difficulty swallowing, head drop due to weakness of the neck muscles, muscle cramps, speech problems, such as a slow or abnormal speech pattern (slurring of words), voice changes, hoarseness, weight loss. Amyotrophic Lateral Sclerosis (Central Nervous System) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. Order a copy of this research report at http://www.rnrmarketresearch.com/contacts/purchase?rname=1019641. (This report is available at up to 25% Discount till June 02nd 2017.) Main key players of Amyotrophic Lateral Sclerosis pipeline are 2-BBB Medicines BV, AB Science SA, Anavex Life Sciences Corp, Angion Biomedica Corp, Apogenix GmbH, ApoPharma Inc, ArmaGen Inc, Avanir Pharmaceuticals Inc, BioCrea GmbH, Biogen Inc, BioHealthonomics Inc, BrainStorm Cell Therapeutics Inc, Catabasis Pharmaceuticals Inc, Chronos Therapeutics Ltd, ContraVir Pharmaceuticals Inc, Corcept Therapeutics Inc, Corestem Inc, Cytokinetics Inc, Daval International Ltd, Edison Pharmaceuticals Inc, Eisai Co Ltd, Ensemble Therapeutics Corp, Evotec AG, F. Hoffmann-La Roche Ltd, Flex Pharma Inc, FPRT Bio Inc, Gemac SA, Genentech Inc, Genervon Biopharmaceuticals LLC, GeNeuro SA more.. The Amyotrophic Lateral Sclerosis pipeline guide provides a snapshot of the global therapeutic landscape of Amyotrophic Lateral Sclerosis (Central Nervous System). The pipeline guide reviews pipeline therapeutics for Amyotrophic Lateral Sclerosis (Central Nervous System) by companies and universities/research institutes based on information derived from company and industry-specific sources. The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages. The Report "Amyotrophic Lateral Sclerosis Global Clinical Trials Review, H1, 2017" provides an overview of Amyotrophic Lateral Sclerosis clinical trials scenario. This report provides top line data relating to the clinical trials on Amyotrophic Lateral Sclerosis. Report includes an overview of trial numbers and their average enrollment in top countries conducted across the globe. The report offers coverage of disease clinical trials by region, country (G7 & E7), phase, trial status, end points status and sponsor type. Report also provides prominent drugs for in-progress trials (based on number of ongoing trials). Clinical Trial Reports are generated using proprietary database - Pharma eTrack Clinical trials database. Clinical trials are collated from 80+ different clinical trial registries, conferences, journals, news etc across the globe. Clinical trials database undergoes periodic update by dynamic process. Explore more reports of Therapeutics Market Research at http://www.rnrmarketresearch.com/reports/life-sciences/pharmaceuticals/therapeutics RnRMarketResearch.com is your one stop market research and industry analysis reports' library providing business data and intelligence information on thousands of micro markets with global as well as regional coverage. Category focused research, country reports, company profiles, regional and global industry profiles and guides to premium reports offering extensive coverage of other 20+ industries are all available in our library of syndicated market research reports.


El-Beshlawy A.M.,Cairo University | El-Alfy M.S.,Ain Shams University | Sari T.T.,Cipto Mangunkusumo Hospital | Chan L.L.,University of Malaya | Tricta F.,ApoPharma Inc.
European Journal of Haematology | Year: 2014

Approximately 6% of patients with thalassemia receiving deferiprone develop neutropenia. Present practice is to monitor absolute neutrophil count (ANC) weekly and to interrupt treatment at the first sign of neutropenia, lest continuation lead to progressive neutrophil reduction. In a 6-month study evaluating the safety and efficacy of a liquid form of deferiprone in 100 children, ANC was initially checked weekly for all patients. For individuals experiencing mild neutropenia, deferiprone was continued but monitoring was increased to daily until resolution. Therapy was to be suspended only if the episode was prolonged or if it worsened. Four patients experienced single episodes of mild neutropenia, and two others each experienced two episodes. All eight episodes resolved within 4-7 d despite continued therapy. (One patient later developed agranulocytosis and had treatment terminated.) This study showed that not all cases of mild neutropenia during deferiprone therapy develop into agranulocytosis, and suggests that many may not be caused by deferiprone. Transient declines in ANC to levels defined as neutropenic are common even in healthy individuals, particularly children; and it could be that the frequent monitoring of ANC mandated during deferiprone therapy may reveal cases of transient neutropenia that would otherwise have gone undetected and resolved on their own without clinical consequences. In patients with thalassemia, several factors increase the probability of a transient fall in ANC. These findings raise the question of whether deferiprone should be routinely stopped in cases of mild neutropenia, provided that such patients have their ANC monitored more frequently during the neutropenic episode. © 2013 John Wiley & Sons A/S.


El Alfy M.,Ain Shams University | Sari T.T.,University of Malaya | Lee C.L.,Cipto Mangunkusumo National Hospital | Tricta F.,ApoPharma Inc. | El-Beshlawy A.,Cairo University
Journal of Pediatric Hematology/Oncology | Year: 2010

Limited data are available on the use of deferiprone in children younger than 10 years of age. This study evaluated the safety and efficacy of a new liquid formulation of deferiprone for the treatment of transfusional iron overload in children 1-10 years old. One hundred children (91 thalassemia major, 8? Hb E-β thalassemia, and 1 sickle cell disease) were enrolled for a 6-month treatment with deferiprone (50 to 100 mg/kg/d). The safety profile was similar to or better than that reported in earlier studies with deferiprone tablets in older children and adults. No unexpected adverse reactions were observed. Gastrointestinal intolerance (GI) was observed in 11% and an increased serum ALT in 12% of the children. Both events were transient. Mild neutropenia, observed in 6% of patients, did not progress to agranulocytosis and resolved despite continuous deferiprone treatment. Two patients experienced agranulocytosis that resolved without complications upon discontinuation of therapy. Deferiprone use was associated with a significant decline in mean serum ferritin level from 2532±1463 μg/L at baseline to 2176±1144? μg/L (P<0.0005). The results of this study show a favorable benefit/risk ratio of deferiprone oral solution for the treatment of young children with transfusional iron overload. © 2010 by Lippincott Williams & Wilkins.


Bentley A.,Abacus International 6 Talisman Business Center | Gillard S.,Abacus International 6 Talisman Business Center | Spino M.,ApoPharma Inc. | Spino M.,University of Toronto | And 2 more authors.
PharmacoEconomics | Year: 2013

Background: Patients with β-thalassaemia major experience chronic iron overload due to regular blood transfusions. Chronic iron overload can be treated using iron-chelating therapies such as desferrioxamine (DFO), deferiprone (DFP) and deferasirox (DFX) monotherapy, or DFO-DFP combination therapy. Objectives: This study evaluated the relative cost effectiveness of these regimens over a 5-year timeframe from a UK National Health Service (NHS) perspective, including personal and social services. Methods: A Markov model was constructed to evaluate the cost effectiveness of the treatment regimens over 5 years. Based on published randomized controlled trial evidence, it was assumed that all four treatment regimens had a comparable effect on serum ferritin concentration (SFC) and liver iron concentration (LIC), and that DFP was more effective for reducing cardiac morbidity and mortality. Published utility scores for route of administration were used, with subcutaneously administered DFO assumed to incur a greater quality of life (QoL) burden than the oral chelators DFP and DFX. Healthcare resource use, drug costs (2010/2011 costs), and utilities associated with adverse events were also considered, with the effect of varying all parameters assessed in sensitivity analysis. Incremental costs and quality-adjusted life-years (QALYs) were calculated for each treatment, with cost effectiveness expressed as incremental cost per QALY. Assumptions that DFP conferred no cardiac morbidity, mortality, or morbidity and mortality benefit were also explored in scenario analysis. Results: DFP was the dominant strategy in all scenarios modelled, providing greater QALY gains at a lower cost. Sensitivity analysis showed that DFP dominated all other treatments unless the QoL burden associated with the route of administration was greater for DFP than for DFO, which is unlikely to be the case. DFP had >99 % likelihood of being cost effective against all comparators at a willingness-to-pay threshold of £20,000 per QALY. Conclusions: In this analysis, DFP appeared to be the most cost-effective treatment available for managing chronic iron overload in β-thalassaemia patients. Use of DFP in these patients could therefore result in substantial cost savings. © 2013 The Author(s).


Grant
Agency: European Commission | Branch: FP7 | Program: CP-FP | Phase: HEALTH.2010.4.2-1 | Award Amount: 7.63M | Year: 2011

-thalassaemia major is one of the most severe forms of chronic congenital anaemia. The recommended treatment consists in regular blood transfusions combined with chelating therapy to remove harmful iron accumulation in the body. The use of deferoxamine, the first chelating agent only available for subcutaneous administration is limited due to toxicity and the lack of compliance, despite its satisfactory therapeutic effects. An oral iron chelating agent, deferiprone, was authorised in Europe in August 1999 and recommended for the treatment of iron overload in patients with thalassaemia major when deferoxamine is contraindicated or inadequate. Despite a wide experience of the administration of deferiprone for thalassaemic patients, limited data are available on its use in children below 10 years and the need for additional data in this age subset was clearly indicated in the 2009 priority list approved by the Paediatric Committee at the European Medicines Agency (PDCO). In addition, according to the recent scientific advancements and in consideration of the anticipated benefit of this chelator in controlling cardiac iron overload, studies evaluating the effects of the deferiprone in all the paediatric ages and in all transfusion-dependent chronic congenital anaemia (including Sickle Cell Diseases) were also considered a critical therapeutic need. The DEEP project, in line with these premises, has been funded with the specific aim to produce a new oral liquid formulation of deferiprone suitable for the paediatric use and to provide evidences for the use of this chelator as first line therapy in the whole paediatric population (from 1 month to 18 years) affected by transfusion-dependent chronic anaemia. The condition under study in the DEEP project is rare. This poses special difficulties in the conduct of the studies due to the small patient population and the need to involve a large number of recruiting centres . However, being dedicated to develop an orphan drug, DEEP has been also recognised in the context of IRDiRC, the International Rare Diseases Research Consortium devoted to repurpose/develop 200 new drugs for Rare Diseases by the end of 2020. Main features of the DEEP project are: -The innovative design of the clinical studies including pharmacokinetic modelling for the definition of the most appropriate dosage of deferiprone in younger children, the cardiac MRI T2* evaluation as primary endpoint, a three years safety study aimed at evaluating deferiprone, in monotherapy or in combination, in the real worlds setting and, for the first time, a comparative efficacy-safety trial to compare the two existing oral chelators: deferiprone and deferasirox. -The DEEP Consortium including European and non-European Countries from the Mediterranean region where the transfusion-dependent congenital anaemia, in particular -thalassemia major, is particularly widespread: the collaboration within a multinational and multicultural network makes the Project extremely challenging due to many different ethical, methodological and social approaches to be explored and positively addressed.


Grant
Agency: European Commission | Branch: FP7 | Program: CP-FP | Phase: HEALTH-2011.2.2.1-1 | Award Amount: 6.98M | Year: 2011

Neurodegeneration with brain iron accumulation (NBIA) is a heterogeneous group of rare hereditary neurodegenerative disorders characterized by high levels of brain iron. The most common form is pantothenate kinase-associated neurodegeneration (PKAN). Classic PKAN and most other NBIA cases are characterised by early childhood onset and rapid progression. Currently, there is no proven therapy to halt or reverse PKAN or any other NBIA. This is especially unfortunate as both the iron accumulation in NBIA and the biochemical defect in PKAN are predicted to be amenable to drug-based treatment. Thus, the current absence of clinical trials is not due to lack of therapeutic options but to rarity of the disease, lack of patient registries and fragmentation of therapeutic research worldwide. For example, the iron-chelating drug deferiprone has been administered to PKAN patients on an individual basis or in pilot trials, both precluding firm conclusions about its efficacy. With TIRCON, we will address this urgent and unmet need for NBIA/PKAN therapy with an ambitious and highly collaborative plan that leverages worldwide expertise. We propose a large investigator-driven randomized clinical trial of deferiprone in PKAN, bringing together leading centres and patient advocacy groups from Europe and the US to reach the required patient cohort size. In addition, together with a European SME, we propose to pursue preclinical development of pantethine and its derivatives which have shown promising efficacy in a Drosophila PKAN model. To facilitate future research, we will develop a harmonized patient registry and biomaterial bank to allow for natural history studies and biomarker development, two critical needs in NBIA research. TIRCON partners, apart from their unique clinical and basic science expertise in NBIA, have longstanding experience in investigator-driven and industry-driven randomized clinical trials. Importantly, they have been closely collaborating in recent years.


Parkinsons disease (PD) is a major, chronic, non-communicable disease and the 2nd most frequent neurodegenerative disorder worldwide. Excess iron is primarily detected in the substantia nigra pars compacta, where dopaminergic neurons are exposed to high levels of oxidative stress produced by mitochondrial disorders and dopamine metabolism. Our previous preclinical, translational and pilot clinical studies demonstrated that novel iron chelation therapy with the prototypic drug deferiprone (DFP) (i) induces neuroprotection in cell models of PD via a powerful antioxidant effect, (ii) reduces regional siderosis of the brain, (iii) reduces motor handicap via inhibition of catechol-o-methyl transferase, and (iv) slows the progression of motor handicap in the 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine mouse model and in early PD patients. This project now seeks to demonstrate that conservative iron chelation therapy with moderate-dose DFP (30 mg/kg/day) slows the progression of handicap in de novo PD patients while not affecting systemic parameters. The 9-month, parallel-group, randomized, placebo-controlled, multicentre trial will be followed by a 1-month wash-out period. The primary efficacy criterion will be the change in motor and non-motor handicap scores on the Total Movement Disorders Society Unified Parkinsons Disease Rating Scale to identify disease-modifying and symptomatic effects. The secondary efficacy criterion will be the change in score between baseline and 40 weeks (i.e. probing the disease-modifying effect only). Potential surrogate radiological and biological biomarkers, health economics and societal impacts will be assessed. 17 national, European and international research and innovation activities will be linked with the project. The study results should prompt academic and industrial research on iron chelation as a disease-modifying treatment in neurodegenerative diseases.

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