Trophos is a biopharmaceutical company specialising in the discovery and development of novel therapeutics to treat both orphan neurodegenerative diseases and more prevalent disorders.Trophos was founded in 1999 in Marseille by three scientists, Christopher Henderson, Olivier Pourquie and Jean-Louis Kraus and two entrepreneurs, Antoine Beret and Michel Delaage. Trophos' lead compound is TRO19622 / olesoxime,.. Olesoxime is a mitochondrial targeted compound, which will be established as first of a new class of therapeutic agents aimed at bringing significant therapeutic benefit to patients suffering from devastating neurological diseases. Spinal Muscular Atrophy is an autosomal recessive genetic disease that affects the motor neurons of the voluntary muscles used for activities such as crawling, walking, head and neck control and swallowing. SMA affects approximately 20,000 people worldwide. One in every 6,000 babies is born with SMA. It is the number one genetic cause of death in children under the age of two. Results from an international, double-blind, placebo-controlled phase II study involving 165 type II and non-ambulatory type III SMA patients, completed in February 2014 to assess safety and efficacy of olesoxime were recently released. The data shows that patients treated with olesoxime were able to maintain motor function over the two-year period of the study and that typical health complications associated with SMA occurred less frequently than in patients treated with a placebo, leading to better well being.In January 2015, the firm was acquired by Hoffmann-La Roche. Wikipedia.

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Gosselies, Belgique, le 29 mai 2017, 7h00 - BONE THERAPEUTICS (code Euronext Bruxelles et Paris : BOTHE, éligible PEA-PME) société de thérapie cellulaire osseuse qui répond à d'importants besoins médicaux non satisfaits dans les domaines de l'orthopédie et des maladies osseuses, annonce aujourd'hui la nomination de Steve Swinson et de Damian Marron en qualité d'administrateurs indépendants. Leurs nominations ont pris effet le 26 mai 2017. Steve Swinson et Damian Marron, de par leur connaissance et leur expertise spécifiques des sociétés cotées dans le secteur de l'orthopédie et de la thérapie cellulaire, viennent compléter la solide expertise du Conseil d'administration de Bone Therapeutics, tout en apportant leurs compétences à l'équipe de direction. Ils remplaceront Jacques Reymann et Jean-Jacques Verdickt. Steve Swinson a occupé de nombreux postes à responsabilité dans des sociétés de technologies médicales en orthopédie, et a notamment été en charge de la direction générale, de la stratégie, des ventes, du marketing et des opérations commerciales de Medtronic International, leader international des technologies médicales. Chez Medtronic, Steve Swinson a dirigé la division Spine & Biologics pour le Canada ainsi que l'Europe occidentale, et a été Vice-Président et Directeur Général des activités internationales liées à la colonne vertébrale avec la responsabilité d'un chiffre d'affaires substantiel. Durant sa carrière internationale, riche de 30 années passées en Asie, aux États-Unis, en Europe et en Afrique, il a également occupé des postes à responsabilité dans des départements médicaux et de diagnostic au sein de multinationales majeures de l'ingénierie, telles que General Electric et Hewlett Packard. Steve est titulaire d'un doctorat en ingénierie électrique de l'Université de Manchester et d'un MBA de l'Université de Chicago. Damian Marron est un dirigeant expérimenté du domaine des sciences de la vie, doté d'un solide bilan en termes de création de valeur. Il a mené avec succès des projets dans le financement de sociétés cotées et privées, dans la gestion de programmes de développement et de redressement, dans les fusions-acquisitions, les accords de licence ainsi que les partenariats de recherche et de commercialisation. Il dispose de compétences spécifiques dans les domaines de la thérapie cellulaire, de l'immuno-oncologie et des maladies orphelines. Dernièrement, il a été Directeur Général d'Agalimmune et de TxCell, société de biotechnologie française spécialisée dans les immunothérapies cellulaires personnalisées à partir de cellules T, dont il a piloté l'introduction en bourse sur Euronext Paris. En tant que Directeur Général de Trophos, France, il a contribué à réunir 34 M€ de financements pour accélérer le développement de la société, qui sera finalement acquise par Roche pour un prix de 700 M€. Damian a également été Vice-Président Exécutif en charge du développement chez NicOx, assistant le Directeur Général dans le cadre de diverses levées de fonds pour un montant total dépassant les 175 M€. Michel Helbig de Balzac, Président du Conseil d'administration de Bone Therapeutics, a déclaré : « Nous sommes très heureux d'accueillir Steve et Damian au sein du Conseil d'administration de Bone Therapeutics. Leur bilan cumulé en termes de management et de création de valeur dans le domaine de la santé, leur connaissance du secteur ainsi que leurs compétences respectives dans les technologies médicales orthopédiques et la thérapie cellulaire, constitueront un atout majeur pour la Société. Leurs recommandations stratégiques seront précieuses pour l'équipe de direction à l'heure où les efforts se concentrent sur l'avancée de notre plateforme innovante de thérapie cellulaire allogénique vers la commercialisation. Nous adressons nos remerciements à Jacques Reymann et Jean-Jacques Verdickt pour leur dévouement au cours des nombreuses années passées chez Bone Therapeutics, et nous leur souhaitons le meilleur pour la suite ». Damian Marron a ajouté : « Je me réjouis de rejoindre Bone Therapeutics. J'ai hâte d'apporter mon expérience du développement stratégique et mes compétences en thérapie cellulaire au profit de la Société et de sa plateforme innovante de thérapie cellulaire allogénique, à un moment clé avant l'atteinte potentielle de points d'inflexion importants ». Bone Therapeutics est une société leader dans la thérapie cellulaire osseuse qui répond à d'importants besoins médicaux non satisfaits dans le domaine de l'orthopédie et des pathologies osseuses. Basée à Gosselies, Belgique, la Société dispose d'un vaste portefeuille diversifié de produits de thérapie cellulaire osseuse, en développement clinique dans divers domaines thérapeutiques ciblant des marchés caractérisés par d'importants besoins médicaux non satisfaits et des innovations limitées. Les produits de thérapie cellulaire de Bone Therapeutics sont fabriqués selon les normes des BPF les plus strictes, et protégés par un vaste portefeuille PI couvrant neuf familles de brevets. De plus amples informations sont disponibles sur notre site www.bonetherapeutics.com/fr. Certaines déclarations, croyances ou opinions du communiqué de presse sont des déclarations prospectives, et reflètent les attentes actuelles et les projections futures relatives à des événements futurs de la Société ou, le cas échéant, de ses administrateurs. De par leur nature, les déclarations prospectives impliquent un certain nombre de risques, d'incertitudes et de suppositions qui pourraient entraîner des résultats ou événements effectifs substantiellement différents de ceux exprimés de manière explicite ou implicite dans les déclarations prospectives. Ces risques, incertitudes et suppositions peuvent affecter de manière négative les résultats et effets financiers des plans et événements décrits dans le communiqué. Une multitude de facteurs, notamment, sans s'y limiter, des modifications intervenant en matière de demande, de concurrence et de technologie, peuvent avoir pour conséquence que les événements, performances ou résultats diffèrent de manière importante des développements anticipés. Les déclarations prospectives contenues dans ce communiqué de presse qui se basent sur des tendances ou des activités passées ne constituent pas des garanties que ces tendances ou activités se poursuivront à l'avenir. En conséquence, la Société rejette expressément toute obligation ou engagement de publier des mises à jour ou révisions des déclarations prospectives de ce communiqué de presse suite à une modification des prévisions ou à une modification des événements, des conditions, des suppositions ou des circonstances sur lesquelles ces déclarations prospectives sont basées. Ni la Société ni ses conseillers ou représentants, ni aucune de ses filiales, ni aucun cadre ou employé de ces personnes ne garantit que les hypothèses sous-jacentes à ces déclarations prospectives sont exemptes d'erreurs et aucun de ceux-ci n'accepte la moindre responsabilité en ce qui concerne l'exactitude future des déclarations prospectives contenues dans ce communiqué de presse ou la survenance effective des événements prévus. Il ne faut pas placer une confiance indue dans les déclarations prospectives, qui ne concernent que la situation telle qu'elle se présente à la date de ce communiqué de presse. Pour les médias et investisseurs français: NewCap Investor Relations & Financial Communications Pierre Laurent, Louis-Victor Delouvrier et Nicolas Merigeau Tél: + 33 (0)1 44 71 94 94 bone@newcap.eu


Bone Therapeutics strengthens Board with the appointments of Steve Swinson and Damian Marron as Non-Executive Directors Senior executives with international experience in orthopaedics and cell therapy Gosselies, Belgium, 29 May 2017, 7am CEST - BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the bone cell therapy company addressing high unmet medical needs in orthopaedics and bone diseases, today announces the appointment of Steve Swinson and Damian Marron to its Board of Directors as Non-Executive Directors. The appointments of Steve Swinson and Damian Marron, effective 26 May 2017, complement an already strong Board of Bone Therapeutics bringing in specific public company, orthopaedic and cell therapy understanding and expertise and will provide ongoing support to the leadership team. Steve Swinson and Damian Marron will replace Jacques Reymann and Jean-Jacques Verdickt. Steve Swinson has served in a number of senior roles in orthopaedic medical technology and electronics companies, including general management, senior strategy, sales, marketing and commercial operation positions at Medtronic International, a global leader in medical technology. At Medtronic, he led the Spine and Biologics division for Canada and Western Europe, and was Vice President and General Manager for the international spine divison with substantial revenue responsibility. In a 30 year international business career covering Asia, US, Europe and Africa, he has also held senior positions at the diagnostic and medical departments of the blue chip engineering multinationals, General Electric and Hewlett Packard. Steve has a PhD in electrical engineering from the University of Manchester and a MBA from the University of Chicago. Damian Marron is an experienced life sciences executive with a successful track record of value creation through public and venture capital financing, portfolio planning and turnaround, M&A, licensing agreements and research and marketing collaborations. He has particular competencies in cell therapy, immuno-oncology and orphan diseases. Damian served most recently as Chief Executive Officer of Agalimmune and has also served as Chief Executive Officer of TxCell, a France-based specialist in personalised T-cell immunotherapies, where he led the Company's IPO on Euronext Paris. As Chief Executive Officer of Trophos, France, he helped raise EUR 34 million in financing and positioned the company for a subsequent acquisition by Roche for EUR 700 million. Damian also served as Executive Vice President, Corporate Development, for NiCox, where he supported the CEO in financing rounds raising over EUR 175 million. Michel Helbig de Balzac, Chairman of Bone Therapeutics, commented: "We are delighted to welcome Steve and Damian to the Board of Bone Therapeutics. Their collective track record in leadership and value creation in the healthcare sector and their industry knowledge and expertise in orthopaedic medical technology and cell therapy respectively will be a major asset to the Company. They will be a valuable sounding board to the leadership team as it focuses on advancing our innovative allogeneic cell therapy platform towards commercialization. We would like to thank Jacques Reymann and Jean-Jacques Verdickt for their many years of dedication to Bone Therapeutics and wish them the best in their well-deserved retirement." Commenting on his appointment, Steve Swinson said: "Bone Therapeutics leads the field in regenerative approaches to orthopaedics and bone diseases, and its allogeneic cell therapy platform has the potential to transform medicine in these areas. I'm delighted to have the opportunity to use my deep experience in orthopaedic medical technology to help support this Company as it advances its technology towards commercialization." Damian Marron added: "I am very excited to be joining Bone Therapeutics. I look forward to bringing my experience in strategic development and my expertise in cell therapy to support the Company as it approaches key value inflection points with its innovative allogeneic cell therapy platform." Bone Therapeutics is a leading cell therapy company addressing high unmet needs in orthopaedics and bone diseases. Based in Gosselies, Belgium, the Company has a broad, diversified portfolio of bone cell therapy products in clinical development across a number of disease areas targeting markets with large unmet medical needs and limited innovation. Our technology is based on a unique, proprietary approach to bone regeneration which turns undifferentiated stem cells into "osteoblastic", or bone-forming cells. These cells can be administered via a minimally invasive procedure, avoiding the need for invasive surgery. Our primary clinical focus is ALLOB®, an allogeneic "off-the-shelf" cell therapy product derived from stem cells of healthy donors, which is in Phase II studies for the treatment of delayed-union fractures and spinal fusion. The Company also has an autologous bone cell therapy product, PREOB®, obtained from patient`s own bone marrow and currently in Phase III development for osteonecrosis and non-union fractures. Bone Therapeutics` cell therapy products are manufactured to the highest GMP standards and are protected by a rich IP estate covering nine patent families. Further information is available at: www.bonetherapeutics.com. Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors` current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such person`s officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release. For Belgium and International Media Enquiries: Consilium Strategic Communications Amber Fennell, Jessica Hodgson and Hendrik Thys Tel: +44 (0) 20 3709 5701 bonetherapeutics@consilium-comms.com For French Media and Investor Enquiries: NewCap Investor Relations & Financial Communications Pierre Laurent, Louis-Victor Delouvrier and Nicolas Merigeau Tel: + 33 (0)1 44 71 94 94 bone@newcap.eu


Target-directed drug design, although a conceptually rational approach, is only one strategy for drug discovery. In the case of neurodegenerative diseases where molecular targets and disease mechanisms are unknown, even when specific genes are known to trigger the disease, phenotypic screening offers another approach. This review describes the establishment of phenotypic screening assays using primary neurons subjected to a disease-relevant pathophysiological stress and measuring the most important functional outcome, survival. Although a challenge both to screening teams to reproducibly produce the cells and chemists to interpret structure-activity relationships, such systems have historically identified or produced effective drugs. The primary screening assay is only the start; once hits are validated, they must be characterized using traditional target-directed or mechanism-based secondary assays to establish their selectivity, lack of side-effect liability, and eventually be shown to produce the desired effects in a preclinical animal model of the disease. These compounds then provide valuable pharmacological tools to identify neurodegenerative disease targets and mechanisms, whether or not they have all the properties required of a drug candidate. © 2010 Bentham Science Publishers.


Grant
Agency: European Commission | Branch: FP7 | Program: CP-FP | Phase: HEALTH-2009-2.4.4-1 | Award Amount: 8.05M | Year: 2010

The Collaborative Project on Mendelian Forms of Parkinsons Disease (MEFOPA) will bring together the major groups in Europe with a track-record in basic and clinical research on rare Mendelian forms of Parkinsons disease (PD) in order to identify and validate relevant disease-related molecular pathways, drug-targets and biomarkers for disease susceptibility and progression.. Over the last years it has become increasingly clear that progress in the understanding of the molecular basis of PD, the second most common neurodegenerative disorder, and hence the chance to develop effective disease-modifying treatments, will most likely be brought about by focusing on the rare variants of the disease with known genetic defects. The groups forming the MEFOPA-consortium will therefore analyze the molecular pathways underlying inherited forms of PD with autosomal-dominant and autosomal-recessive inheritance in an integrative way, using cellular and animal models and cutting-edge technology. These two subprojects will provide targets for novel, disease-modifying treatment strategies. In a third subproject, a European registry and biobank for patients with rare Mendelian forms of PD will be established. Body fluids will be collected and systematically analyzed by unbiased proteomic techniques as well as by focussed analysis of candidate proteins, and ex vivo cellular models will be generated, in order to allow validation of disease-related alterations detected in the models analyzed in subprojects 1 and 2. Through this integrated, translational approach combining basic and clinical research groups, the project aims to achieve measurable progress in defining the relevant targets and readouts for disease-modifying therapies and will set the stage for rationally designed drug trials in carefully selected groups of patients and even presymptomatic mutation carriers.


Grant
Agency: European Commission | Branch: FP7 | Program: CP-FP | Phase: HEALTH.2010.2.4.2-2 | Award Amount: 8.19M | Year: 2011

Current interventional treatment of acute myocardial infarction (AMI) focused on re-establishing cardiac reperfusion has significantly improved clinical outcome by reducing infarct size and mortality due to cardiac ischemia.It is now recognized that events triggered at reperfusion also result in cell death and may account for as much as 50% of the infarct volume, this being termed ischemia-reperfusion injury (IRI). Mitochondrial permeability transition pore (mPTP) opening appears to be a responsible for IRI and a recent small clinical trial with cyclosporine A shows that it is a feasible target for the development of new therapies to treat it. Since total infarct size is a major determinant of a patients risk to develop heart failure, treating IRI is expected to further reduce morbidity, mortality and the need for regenerative medicine following cardiac ischemia. By harnessing a multi-disciplinary consortium of clinical and basic scientists along with four SMEs, MitoCare brings state-of-the art expertise together to 1) better understand IRI pathophysiology and factors directly or indirectly influencing patients recovery or response to treatment; 2) investigate the translational usefulness of preclinical models; and 3) compare selected treatments. These objectives will be reached through the following work plan: A) a medium-scale phase II clinical study will evaluate the efficacy of a novel complementary therapy to PCI, the new mPTP modulator TRO40303, while at the same time 1) perform extensive sampling from subjects in the study for analysis of standard and emerging biomarkers; 2) identify confounding factors influencing patients outcomes. B) Parallel investigations in preclinical in vitro and in vivo AMI models. C) Statistical analysis of data from clinical and preclinical studies, to identify better diagnostic and prognostic endpoints in man and assess predictive utility of preclinical models.


Grant
Agency: European Commission | Branch: FP7 | Program: CP-FP | Phase: HEALTH-2009-2.4.4-1 | Award Amount: 9.25M | Year: 2010

Leukodystrophies (LDs) are inherited rare neurodegenerative diseases of the white matter and its main component, the myelin, that are affecting predominantly children. Severity of the disease is related to the axonal dysfunction due to myelin deficiency or destruction. Despite the achievement of remarkable advances made in the past decade, there is no current curative therapy. The development of therapeutic approaches for myelin repair and neuroprotection constitutes the main objective of the LeukoTreat project. Indeed LDs constitute prototypic pathologies to tackle myelin formation/destruction issues as well as glial cells dysfunctions in neurodegeneration. The global aim is to promote the development of therapeutic strategies for the largest number of LD affected patients and further applications to more common white matter disorders and finally neurodegenerative diseases. For this purpose, the project will combine the expertise of (i) recognized European research teams in the field of White Matter diseases (COST Myelinet), (ii) high-technology SMEs, (iii) experts in medical ethics and (iv) LD patients and families associations. To develop efficient therapies, the LeukoTreat project is based on 5 complementary approaches consisting in: (i) collecting information on the epidemiology, the natural history, the genotype/phenotype correlation of LDs for at least 500 patients; (ii) validating/identifying biomarkers for therapeutic decisions/follow up to isolate new therapeutic targets; (iii) developing pharmacological strategies with the ultimate objective to launch at least 4 pharmacological clinical trials during 5 years following the project; (iv) developing innovative gene and cell therapies with the ultimate objective to launch at least 3 clinical trials during the next 5 years; (v) tackling ethical impacts of the proposed therapeutic challenges by integrating the participation of patients driven by a well-experienced research team strongly skilled in ethics


The object of the invention is the use as a drug of a compound fitting formula I


A method for providing neuroprotection to a patient in need of neuroprotection, comprising administering a neuroprotective-effective amount of a compound of formula I


The present invention relates to a novel galenic form of cholesterol derivative. More particularly the invention relates to liposomes comprising at least one cholesterol derivative and compositions comprising said liposomes.


The object of the invention is the use as a drug of a compound fitting formula I wherein X represents an oxygen atom or a NOH group and R represents a group selected from

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