Saint-Léger-du-Bourg-Denis, France
Saint-Léger-du-Bourg-Denis, France

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AB Science announces that ANSM requested the temporary suspension of clinical studies conducted in France following deviations from Good Clinical Practice (GCP) standards AB Science corrected these deficiencies and an audit plan confirming the GCP compliance will be performed in the coming months ANSM accepted the principle of restarting the clinical studies on the basis on the findings of this independent audit AB Science SA (NYSE Euronext - FR0010557264 - AB), a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), received yesterday the notification from the Agence Nationale de la Sécurité des Médicaments (ANSM) requesting the temporary suspension of the ongoing masitinib studies until their compliance is confirmed by an external audit. AB Science is actively collaborating with ANSM in order to restart the recruitment of patients in clinical studies in France. This decision follows the findings of the inspection that was carried out as part of the procedure for the marketing authorization of masitinib in mastocytosis, which showed deviations from the Good Clinical Practice (GCP) in the conduct of the mastocytosis pivotal study (AB06006) and deviations related to the pharmacovigilance system. The EMA inspection focused on study AB06006 conducted between 2009 and the beginning of 2015 and does not therefore reflect the quality system put into place since mid-2015. Indeed, since mid-2015, the company has implemented a new quality system to ensure compliance with the GCP requirements. As an evidence that other studies have no unreported adverse events, the last 11 site inspections carried out by national health authorities outside France since mid-2015 have not identified any serious or severe adverse events that would have been left unreported. In particular, a site inspection performed by the Canadian Health Authority on the amyotrophic lateral sclerosis phase 3 study concluded that the study is conducted under GCP compliance. Additionally, all the deficiencies identified in the pharmacovigilance system have been corrected following the inspection and the pharmacovigilance system currently in place is now GCP compliant. An independent audit will be performed in the coming months in order to provide ANSM with confirmation that the system meets the GCP standards. ANSM confirmed that the clinical studies could restart in France following the positive results of this audit plan. As part of the implementation of the new quality system, data from all studies, clinical sites and patients have been remonitored since mid-2015. This remonitoring of all studies concerned key efficacy data and adverse events. This full remonitoring was performed between mid-2015 and November 2016 for all studies except mastocytosis and following EMA inspection between January 2017 and March 2017 for mastocytosis study. The masitinib's safety profile was not modified following this full remonitoring. The 2017 Investigational Brochure will include the remonitored data and will be sent to health authorities in the coming months. Additionally, independent quality audits of key efficacy data and adverse events in mastocytosis and ALS studies will be conducted in order to ensure the data reliability and secure registration in both indications. Clinical batches records, including treatment number correspondence lists, have been received by AB Science's Pharmaceutical Operations department in preparation for an inspection, without AB Science being informed of the presence of these lists, and discovered during EMA inspection. These lists only concern a very small number of the patients included in the mastocytosis study and have no impact on other studies, including the ALS study. Regarding the mastocytosis study, the inspection showed that no modification of the protocol had occurred after the receipt of these lists and showed no use of these lists before unblinding. The masitinib safety profile is known on the basis of more than 5,000 patients, has not increased, and is acceptable in the targeted indications. The masitinib safety profile has been assessed as acceptable in all ongoing indications by the Independent Data Monitoring Committees (IDMC) of the studies, which have access to unblinded data, as well as by health agencies of more than 25 countries where masitinib is currently developed and finally by the ethics committees which have authorized the studies. Concerning the risk of severe skin toxicity, since the implementation of the new risk management plan in 2012, the suspected Stevens Johnson syndrome cases have been reviewed by two independent experts and no case of Stevens Johnson syndrome have occurred on the 3500 recruited patients, proving the control of this risk. 2 cases of possible or probable Stevens Johnson syndrome were reported before 2012 in cancer patients receiving masitinib at high doses (7,5 mg and 9 mg) in combination with chemotherapy. Similarly, the risk of severe neutropenia was minimized by the risk management plan in 2012, around 1% and remained stable. Clinical risk analysis does not indicate an identified risk of death or renal toxicity or cardiac toxicity. A vigilance plan has been implemented for this latter risk given the class effect, but masitinib appears to be unique because of its very selective kinase inhibition profile and no toxicity signal has been identified in clinical studies. A follow-up on 250 patients of the left ventricular ejection fraction showed no risk increase. Similarly, a QT/QTc study did not show an increase in this risk. Additionally, the carcinogenic risk observed in non-clinical studies in animals was evaluated in detail by the EMA during the procedure for the marketing authorization of masitinib in mastocytosis and this assessment shows that the risk of tumors is specific to evaluated animal species and not transposable to humans, and that the genotoxic risk is below the acceptable limits set in EMA guidelines for a lifetime treatment in a non-life-threatening indication. It is therefore now established that this residual risk is not an obstacle to the registration of masitinib in indications outside oncology. The clinical studies have not been stopped. The decision applies in France only. In France, studies are not prohibited, only suspended. The study suspension in France has a low impact on the recruitment of ongoing studies, France accounting for less than 5% of patient recruitment in these ongoing studies. Additionally, patients who could be discontinued account for less than 1% of the number of patients to be recruited in phase 3 studies, which does not threaten the data interpretation. For these patients, the discontinuation is not certain as ANSM informed AB Science that the decision to discontinue the treatment for ongoing patients may be subject to an appeal and a review of individual benefit for each patient. The findings identified during this inspection do not affect the ALS study for the following reasons: A webcall will be hosted on May 12, 2017 at 4.30pm (CET) for French speakers and at 6.30pm (CET) for English speakers. To participate please send an email at linda.carlet@ab-science.com. Masitinib is a new orally administered tyrosine kinase inhibitor that targets mast cells and macrophages, important cells for immunity, through inhibiting a limited number of kinases. Based on its unique mechanism of action, masitinib can be developed in a large number of conditions in oncology, in inflammatory diseases, and in certain diseases of the central nervous system. In oncology due to its immunotherapy effect, masitinib can have an effect on survival, alone or in combination with chemotherapy. Through its activity on mast cells and microglia and consequently the inhibition of the activation of the inflammatory process, masitinib can have an effect on the symptoms associated with some inflammatory and central nervous system diseases and the degeneration of these diseases. Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a  class of targeted proteins whose action are key in signaling pathways within cells. Our programs target only diseases with high unmet medical needs, often lethal with short term survival or rare or refractory to previous line of treatment in cancers, inflammatory diseases, and central nervous system diseases, both in humans and animal health. AB Science has developed a proprietary portfolio of molecules and the Company's lead compound, masitinib, has already been registered for veterinary medicine in Europe and in the USA. The company is currently pursuing thirteen phase 3 studies in human medicine in metastatic prostate cancer, metastatic pancreatic cancer, relapsing metastatic colorectal cancer, relapsing metastatic ovarian cancer, GIST, metastatic melanoma expressing JM mutation of c-Kit, relapsing T-cell lymphoma, severe asthma, amyotrophic lateral sclerosis, Alzheimer's disease and progressive forms of multiple sclerosis. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB). Further information is available on AB Science's website: www.ab-science.com. This press release contains forward-looking statements. These statements are not historical facts. These statements include projections and estimates as well as the assumptions on which they are based, statements based on projects, objectives, intentions and expectations regarding financial results, events, operations, future services, product development and their potential or future performance. These forward-looking statements can often be identified by the words "expect", "anticipate", "believe", "intend", "estimate" or "plan" as well as other similar terms. While AB Science believes these forward-looking statements are reasonable, investors are cautioned that these forward-looking statements are subject to numerous risks and uncertainties that are difficult to predict and generally beyond the control of AB Science and which may imply that results and actual events significantly differ from those expressed, induced or anticipated in the forward-looking information and statements. These risks and uncertainties include the uncertainties related to product development of the Company which may not be successful or to the marketing authorizations granted by competent authorities or, more generally, any factors that may affect marketing capacity of the products developed by AB Science, as well as those developed or identified in the public documents filed by AB Science with the Autorité des Marchés Financiers (AMF), including those listed in the Chapter 4 "Risk Factors" of AB Science reference document filed with the AMF on November 22, 2016, under the number R. 16-078. AB Science disclaims any obligation or undertaking to update the forward-looking information and statements, subject to the applicable regulations, in particular articles 223-1 et seq. of the AMF General Regulations.


Depuis mi-2015, nous avons mis en place un nouveau système qualité pour se conformer au référentiel BPC, avec l'accent mis prioritairement sur la re-collecte et la re-vérification des données de tolérance et des principales données d'efficacité. Nous avons aussi corrigé les écarts observés dans le système de pharmacovigilance, et AB Science estime que le système de pharmacovigilance est maintenant conforme aux BPC. Le « remonitoring » ou re-collecte et re-vérification des données consiste à effectuer un rapprochement entre les données du dossier médical du patient - les données sources - et les données saisies par le personnel hospitalier dans le cahier de recueil des données de l'étude. Il s'agit donc de vérifier l'exhaustivité et l'exactitude des données d'efficacité et de tolérance recueillies dans le cadre des études cliniques. Par ailleurs, les deux études de phase 3 dans la mastocytose et la SLA sont positives. Dans la SLA, les résultats seront présentés cette semaine à la réunion annuelle de l'ENCALS à Ljubiana en Slovénie. La prochaine étape sera la décision de l'EMA concernant l'enregistrement du masitinib dans la SLA, qui interviendra fin 2017. AB Science note qu'un produit concurrent a été approuvé très récemment par la FDA, sur la base d'une étude ayant recruté moins de patients et d'une amélioration du score fonctionnel plus faible par rapport au masitinib, ce qui permet à AB Science d'espérer un vote favorable du CHMP dans la SLA sur la base d'un système qualité aux normes BPC. Par ailleurs, 50,000 patients ont signé une pétition aux Etats-Unis demandant l'enregistrement du masitinib dans la SLA dans les plus brefs délais. Dans l'hypothèse où plusieurs autres agences européennes suspendraient les études, il faut noter que les essais cliniques du masitinib sont conduits en Europe de l'Ouest, en Amérique du Nord, en Amérique Latine, en Europe de l'est, en Asie et en Afrique du Nord et du Sud et au Moyen-Orient. Le développement du masitinib n'est donc pas dépendant d'une zone géographique en particulier et encore moins d'un pays. De plus, des audits indépendants de la qualité des variables clés d'efficacité et des effets indésirables dans la mastocytose et la SLA sont conduits pour garantir leur fiabilité et sécuriser l'enregistrement dans ces deux indications. Dans son courrier, l'ANSM fait référence à plusieurs inspections, dont certaines remontent à l'année 2006. Les écarts constatés sur les critères d'inclusion et d'exclusion ne concernent pas les études mastocytose et SLA et ont été résolus. Les études cliniques ne sont pas à refaire. AB Science doit apporter la preuve à l'ANSM que le système qualité est à présent conforme aux normes BPC. C'est pourquoi AB Science a proposé la conduite d'un audit indépendant des systèmes et des centres cliniques afin de le démontrer. L'ANSM a accepté le principe de reprendre les études cliniques sur la base de la réalisation de cet audit indépendant. Par ailleurs, AB Science précise que la re-vérification des données d'efficacité et de tolérance a été effectuée pour l'ensemble des études entre mi-2015 et mars 2017. L'audit à venir permettra de confirmer la fiabilité de ces données. AB Science conteste la position de l'ANSM concernant la sécurité du produit et rappelle que le profil de sécurité du masitinib a été évalué comme acceptable dans toutes les indications en cours par les IDMC (Independent Data Monitoring Committee) des études qui ont accès aux données après levée de l'aveugle, ainsi que par les agences de santé de plus de 25 pays dans lesquels le masitinib est développé et par les comités d'éthiques qui ont autorisé les études. Le vote du CHMP aura lieu cette semaine. Néanmoins, AB Science rappelle que la re-vérification des données d'efficacité et de tolérance pour l'étude mastocytose a été effectuée après l'inspection de l'EMA. Il est donc probable que la procédure d'autorisation de mise sur le marché dans cette indication soit impactée négativement. Dans tous les cas, conformément à la pratique en place et à sa pratique antérieure, AB Science ne communique pas sur les processus d'autorisation de mise sur le marché du masitinib et sur les échanges avec les autorités de santé relatifs à ce processus avant le vote du CHMP. Concernant le dossier d'enregistrement dans la SLA, il n'y pas d'impact à notre connaissance. L'inspection de l'EMA dans cette étude dans le cadre de la procédure d'enregistrement aura lieu au quatrième trimestre 2017, après la re-vérification des données d'efficacité et de tolérance qui a déjà été effectuée et après l'audit externe des systèmes et des centres cliniques qui aura lieu dans les prochains mois et qui apportera la preuve de la fiabilité des données. Le dossier d'enregistrement dans la SLA sera donc plus robuste d'un point de vue du respect des BPC. Le masitinib est un nouvel inhibiteur de tyrosine kinase, administré par voie orale, qui cible les mastocytes et les macrophages, cellules essentielles de l'immunité, par l'inhibition d'un nombre limité de kinases. En raison de son mode d'action unique, le masitinib peut être développé dans un grand nombre de pathologies, en oncologie, dans les maladies inflammatoires, et certaines maladies du système nerveux central. En oncologie, par son activité d'immunothérapie, le masitinib peut avoir un effet sur la survie, seul ou en association avec la chimiothérapie. Par son activité sur le mastocyte et les cellules microgliales et donc par son effet inhibiteur sur l'activation du processus inflammatoire, le masitinib peut avoir un effet sur les symptômes associés à certaines pathologies inflammatoires et du système nerveux central. Fondée en 2001, AB Science est une société pharmaceutique spécialisée dans la recherche, le développement, et la commercialisation d'inhibiteurs de protéines kinases (IPK), une classe de protéines ciblées dont l'action est déterminante dans la signalisation cellulaire. Nos programmes ne ciblent que des pathologies à fort besoin médical, souvent mortelles avec un faible taux de survie, rares, ou résistantes à une première ligne de traitement, dans les cancers, les maladies inflammatoires et les maladies du système nerveux central, en santé humaine et animale. AB Science a développé en propre un portefeuille d'inhibiteurs de protéines kinases (IPK), une nouvelle classe de molécules ciblées dont l'action consiste à modifier les voies de signalisation intracellulaire. La molécule phare d'AB Science, le masitinib, a déjà fait l'objet d'un enregistrement en médecine vétérinaire en Europe et aux États-Unis et est développée dans 13 phases 3 chez l'homme, dans le cancer de la prostate métastatique, le cancer du pancréas métastatique, le cancer colorectal métastatique en rechute, le cancer de l'ovaire métastatique en rechute, le GIST, le mélanome métastatique exprimant la mutation c-Kit JM, le lymphome périphérique à cellule T en rechute, l'asthme sévère, la sclérose latérale amyotrophique, la maladie d'Alzheimer et la sclérose en plaques progressive. La Société a son siège à Paris et est cotée sur Euronext Paris (Ticker : AB). Ce communiqué contient des déclarations prospectives. Ces déclarations ne constituent pas des faits historiques. Ces déclarations comprennent des projections et des estimations ainsi que les hypothèses sur lesquelles celles-ci reposent, des déclarations portant sur des projets, des objectifs, des intentions et des attentes concernant des résultats financiers, des événements, des opérations, des services futurs, le développement de produits et leur potentiel ou les performances futures. Ces déclarations prospectives peuvent souvent être identifiées par les mots « s'attendre à », « anticiper », « croire », « avoir l'intention de », « estimer » ou « planifier », ainsi que par d'autres termes similaires. Bien qu'AB Science estime que ces déclarations prospectives sont raisonnables, les investisseurs sont alertés sur le fait que ces déclarations prospectives sont soumises à de nombreux risques et incertitudes, difficilement prévisibles et généralement en dehors du contrôle d'AB Science qui peuvent impliquer que les résultats et événements effectifs réalisés diffèrent significativement de ceux qui sont exprimés, induits ou prévus dans les informations et déclarations prospectives. Ces risques et incertitudes comprennent notamment les incertitudes inhérentes aux développements des produits de la Société, qui pourraient ne pas aboutir, ou à la délivrance par les autorités compétentes des autorisations de mise sur le marché ou plus généralement tous facteurs qui peuvent affecter la capacité de commercialisation des produits développés par AB Science ainsi que ceux qui sont développés ou identifiés dans les documents publics déposés par AB Science auprès de l'AMF, y compris ceux énumérés dans le chapitre 4 « Facteurs de risques » du document de référence d'AB Science enregistré auprès de l'AMF le 22 novembre 2016, sous le numéro R. 16-078. AB Science ne prend aucun engagement de mettre à jour les informations et déclarations prospectives sous réserve de la réglementation applicable notamment les articles 223-1 et suivants du règlement général de l'AMF.


Summary of the conference call held on May 12, 2017 AB Science SA (NYSE Euronext - FR0010557264 - AB), a pharmaceutical company specialized in research, development and marketing of protein kinase inhibitors (PKIs), provides the summary of the questions and answers of both conference calls held on May 12, 2017, following ANSM decision requesting suspension of masitinib clinical studies conducted in France by AB Science, until their compliance is confirmed by an external audit. ANSM decision is related to an implementation problem and does not question the fundamentals of the masitinib clinical results or the fundamentals of its development. 1 - Questions and answers related to Good Clinical Practice (GCP) compliance of clinical studies and pharmacovigilance system deviations The ANSM decision follows the findings of the inspection that was carried out as part of the procedure for the marketing authorization of masitinib in mastocytosis, which showed GCP deviations in the conduct of the mastocytosis pivotal study (AB06006) and deviations related to the pharmacovigilance system. AB Science proposed to ANSM an action plan to demonstrate that the deviations have been corrected. This action plan is based on the implementation of independent audits in order to provide the ANSM with confirmation that the quality system is BPC compliant. Additionally to the audits, the investigator brochure will be updated on the basis of the latest safety data (2017 Investigator Brochure) and will integrate the full safety data remonitoring for all studies and will confirm that masitinib safety profile is acceptable (see section 2). ANSM validated this action plan. AB Science knows how to respond to ANSM's request. There is therefore no uncertainty about the actions to be taken to restart studies in France. AB Science is not in the situation where the benefit balance of masitinib is questioned, but is facing an implementation problem, with steps for its resolution clearly identified. The company is therefore confident about the prospect of being authorized to restart the studies in France within a reasonable period of a few months. What is the duration of the planned independent audits? As decided by AB Science, external audits of the different systems ensuring data quality will be initiated in May and will last for a few months. The ANSM accepted the principle of restarting the clinical studies on the basis on findings from these independent audits. Two different audits are planned: Why AB Science did not perform these audit before? Since mid-2015, AB Science implemented a new quality system to comply with the GCP requirements, with a priority focus on collection and remonitoring of all safety data and key efficacy data. AB Science also corrected the deficiencies in the pharmacovigilance system, which is now GCP compliant according to AB Science. An independent audit plan will now be launched as soon as possible. AB Science recognizes that these external audits should have been carried out previously, in order to ensure the GCP compliance of the quality system and clinical data reliability. What do you mean by data «remonitoring»? The data «remonitoring» consists in reconciling the data of the patient's medical record - the source data - and the data entered in the case report form by the hospital staff. The objective is therefore to check the completeness and the accuracy of the efficacy and safety data collected in the clinical studies. Does ANSM decision seem exaggerated or fully justified? Is this a political decision? AB Science does not want to comment on reasons for the ANSM decision. AB Science is actively collaborating with ANSM and focus on the implementation of the proposed action plan. The following points can be emphasized: AB Science does not consider this decision to be political. The reasons are related to quality procedure deviations from AB Science, the product benefit is not questioned. Why was the ANSM decision only made now? The ANSM decision follows the findings of an inspection that was carried out recently as part of the procedure for the marketing authorization of masitinib in mastocytosis. How did AB Science come to such a deadlock? How can AB Science rebuild its credibility? For AB Science this is not a deadlock, but a temporary decision that will be lifted following positive results of the audit plan. AB Science implemented an action plan validated with ANSM to cancel the suspension: The quality system upgrade to GCP requirements has already been done. This upgrade must now be demonstrated by an external audit. In addition, the two phase 3 studies in mastocytosis and ALS are positive. In ALS, the results will be presented this week at the ENCALS annual meeting in Ljubiana, Slovenia. The next step will be the EMA's decision related to masitinib registration in ALS, expected by the end of 2017. AB Science indicates that a competing product was recently approved by the FDA, following a study with a fewer patients and a lower functional score improvement compared with masitinib, allowing AB Science to hope for a favorable CHMP vote in ALS, on the basis of a GCP compliant quality system. In addition, 50,000 patients signed a petition in the United States requesting the registration of masitinib in ALS as soon as possible. What are the expected delays on the ongoing programs? AB Science would like to highlight that this decision applies in France only. AB Science's studies continue in more than 25 countries, which are not affected by the decision. In France, studies are not prohibited, only suspended. Moreover, this suspension has a low impact on the conduct of AB Science studies, since: The impact is therefore limited, especially because AB Science believes a resumption of recruitment on the basis of the conclusions of the external audit can be achieved within a reasonable period of a few months. Will other national agencies revise their position and follow the ANSM's decision? To date, AB Science did not receive any notification from another agency requesting the suspension of studies. In addition, AB Science informed all European health agencies about: To date, AB Science does not know what the position of the other agencies will be following the EMA inspection. It should be noted that in the past, ANSM decisions have not always been followed by other agencies, which remain independent. In the case other European agencies would suspend the clinical studies, it should be noted that the clinical studies of masitinib are conducted in Western Europe, North America, Latin America, Eastern Europe, Asia, North Africa, South Africa and the Middle East. The development of masitinib is therefore not dependent on a particular geographical area and even less on a country. 2- Questions and answers related to reliability of safety and efficacy data Data collected in clinical studies are not invalidated or lost. The 2017 investigator brochure, which will include data remonitoring, will be sent to the health authorities in the coming months. We already know that the collected and remonitored data do not change the masitinib safety profile. In addition, independent audits of data quality for key efficacy endpoints and adverse effects in mastocytosis and ALS are conducted to ensure their reliability and to secure registration in both indications. In summary, no efficacy and safety data have been lost. No study is irreversibly damaged. The safety profile of masitinib is not modified after this full remonitoring. Have side effects been hidden or underestimated? The efficacy and safety data remonitoring showed that masitinib safety profile has not been modified since the adverse events which had not been correctly reported were for their overwhelming majority mild or moderate adverse events. In the mastocytosis study, only four severe adverse events had not been reported: one peripheral edema in the masitinib arm and three severe adverse events in the placebo arm. The 2017 investigator brochure, which will include data remonitoring, will be sent to the ANSM and health authorities in the coming months. In its letter, ANSM notified that there were several deviations on inclusion and exclusion criteria. Is this problem fixed? Additionally, ANSM highlighted many protocol deviations that could have impacted the outcome on the primary endpoint, what do you think they refer to? In its letter, the ANSM refers to several inspections, some of which date back to 2006. The findings related to inclusion and exclusion criteria do not concern mastocytosis and ALS studies and have been resolved. Protocol deviations that could have impacted the outcome on the primary endpoint are related to clinical batches records, including treatment number correspondence lists that have been received by AB Science's Pharmaceutical Operations department in preparation for an inspection, without AB Science being informed of the presence of these lists, and discovered during EMA inspection. These lists only concern a very small number of the patients included in the mastocytosis study and have no impact on other studies, including the ALS study. Regarding the mastocytosis study, the inspection showed that no modification of the protocol had occurred after the receipt of these lists and showed no use of these lists before unblinding. What studies will have to be redone to comply with the requirements of the ANSM? Clinical studies are not to be redone. AB Science must provide evidence to the ANSM that the quality system is now GCP compliant. This is why AB Science proposed to conduct an independent audit of systems and clinical sites to demonstrate this. The ANSM accepted the principle of restarting clinical studies on the basis of the conduct of this independent audit. In addition, AB Science indicates that the efficacy and safety data remonitoring was carried out for all studies between mid-2015 and March 2017. The upcoming audit will confirm the reliability of these data. AB Science's position diverges from ANSM's position on this question. AB Science also notes that in its letter, ANSM conditions the restart of studies to GCP compliance confirmation, and not to a masitinib safety profile reassessment. Masitinib adverse events published by The Lancet do not correspond with ANSM's position. Why? AB Science does not agree with ANSM's position regarding masitinib safety profile and reiterates that the masitinib safety profile has been assessed as acceptable in all ongoing indications by the Independent Data Monitoring Committees (IDMC) of the studies, which have access to unblinded data, as well as by health agencies of more than 25 countries where masitinib is currently developed, and finally by the ethics committees which have authorized the studies. Regarding masitinib risks analysis, AB Science confirms the following: 4- Questions and answers related to ANSM decision impact on masitinib development in amyotrophic lateral sclerosis What is the impact of ANSM's decision on registration dossiers for mastocytosis and amyotrophic lateral sclerosis (ALS)? The CHMP vote will take place this week. Nevertheless, AB Science reiterates that efficacy and safety data remonitoring for the mastocytosis study was carried out after the EMA inspection. Therefore, the marketing authorization procedure is likely to be negatively impacted in this indication. In any case, in accordance with current practice and similarly to previous dossiers, AB Science does not communicate on the marketing authorization process for masitinib and on discussions related to this process with the health authorities before the CHMP. Concerning the registration dossier in the ALS, there is no impact according to AB Science. The EMA inspection for this study as part of the procedure for the marketing authorization will take place in the fourth quarter of 2017, after the efficacy and safety data remonitoring, which has already been carried out and after the external audit of systems and clinical sites that will take place in the coming months and which will demonstrate the reliability of the data. Therefore, the ALS registration dossier will be more robust in terms GCP compliance. Could the masitinib safety profile in ALS be a problem? The masitinib safety profile in systemic indolent mastocytosis is acceptable. Masitinib's safety profile can only be more acceptable in ALS, which is a life-threatening disease. In addition, due to a lower masitinib dose in the ALS study, the masitinib safety profile should be better in the ALS study and highly acceptable for EMA. Masitinib safety data in ALS will be presented at ENCALS this week (European Network for the Cure of ALS). Since ALS is a life-threatening disease like GIST or pancreatic cancer, how GCP compliance had been evaluated by CHMP in these two dossiers? There was no GCP finding in the masitinib registration dossier in pancreatic cancer, although the masitinib marketing authorization filing in pancreatic cancer occurred before the new quality system implementation in mid-2015. GCP deviations were observed in the GIST registration dossier. A data remonitoring was therefore carried out by AB Science and finally EMA did not object on GCP compliance in GIST. For how long the stock market trading will be suspended? Stock market trading recovery is planned on Tuesday 16 May at the opening. AB Science decided to suspend the stock market trading for two days (Friday May 12, 2017 and Monday May 15, 2017) in order to provide the necessary explanations to all shareholders, following the ANSM decision. Can you extend the stock market trading suspension until CHMP decision, the clarification of the situation with ANSM, or at least the availability of the external audit conclusions? This is not desirable to suspend the stock market trading for several weeks or months. The stock market trading recovery is scheduled for Tuesday, May 16. AB Science believes that the necessary clarifications will have been provided to shareholders in the meantime. Why was this information given before the markets closed? The ANSM made public its decision on its website on Thursday May 11, 2017 at 5pm, without having previously informed AB Science. AB Science was surprised by this decision of the agency and therefore issued a press release on Friday May 12, 2017 at 3 am to explain its position. In view of the current situation, is it possible to have details about the recent sale of shares by Alain Moussy? The sale of Alain Moussy's shares took place on March 31, 2017. This sale followed the announcement of positive results in amyotrophic lateral sclerosis, which allowed Alain Moussy to find a buyer for the sale of a block of shares, off-market. Moreover, this sale of shares was carried out with the sole purpose of repaying a maturing bank debt. This debt had been contracted in order to finance the acquisition of AB Science shares as part of stock option plans. The ANSM letter informing of a possible decision to suspend clinical trials was received by AB Science on April 19, 2017. Alain Moussy was therefore not aware of the ANSM intentions at the time of the sale of the shares. With these new delays, will you have new needs of cash in the coming months? The company recently raised 34 million euros through private placements, before knowing ANSM intentions to suspend clinical studies in France. As of March 31, 2017, AB Science had a cash position of 58.5 million euros, sufficient to continue the development of masitinib. AB Science does not need additional cash in the short term. Masitinib is a new orally administered tyrosine kinase inhibitor that targets mast cells and macrophages, important cells for immunity, through inhibiting a limited number of kinases. Based on its unique mechanism of action, masitinib can be developed in a large number of conditions in oncology, in inflammatory diseases, and in certain diseases of the central nervous system. In oncology due to its immunotherapy effect, masitinib can have an effect on survival, alone or in combination with chemotherapy. Through its activity on mast cells and microglia and consequently the inhibition of the activation of the inflammatory process, masitinib can have an effect on the symptoms associated with some inflammatory and central nervous system diseases and the degeneration of these diseases. Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a  class of targeted proteins whose action are key in signaling pathways within cells. Our programs target only diseases with high unmet medical needs, often lethal with short term survival or rare or refractory to previous line of treatment in cancers, inflammatory diseases, and central nervous system diseases, both in humans and animal health. AB Science has developed a proprietary portfolio of molecules and the Company's lead compound, masitinib, has already been registered for veterinary medicine in Europe and in the USA. The company is currently pursuing thirteen phase 3 studies in human medicine in metastatic prostate cancer, metastatic pancreatic cancer, relapsing metastatic colorectal cancer, relapsing metastatic ovarian cancer, GIST, metastatic melanoma expressing JM mutation of c-Kit, relapsing T-cell lymphoma, severe asthma, amyotrophic lateral sclerosis, Alzheimer's disease and progressive forms of multiple sclerosis. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB). Further information is available on AB Science's website: www.ab-science.com. This press release contains forward-looking statements. These statements are not historical facts. These statements include projections and estimates as well as the assumptions on which they are based, statements based on projects, objectives, intentions and expectations regarding financial results, events, operations, future services, product development and their potential or future performance. These forward-looking statements can often be identified by the words "expect", "anticipate", "believe", "intend", "estimate" or "plan" as well as other similar terms. While AB Science believes these forward-looking statements are reasonable, investors are cautioned that these forward-looking statements are subject to numerous risks and uncertainties that are difficult to predict and generally beyond the control of AB Science and which may imply that results and actual events significantly differ from those expressed, induced or anticipated in the forward-looking information and statements. These risks and uncertainties include the uncertainties related to product development of the Company which may not be successful or to the marketing authorizations granted by competent authorities or, more generally, any factors that may affect marketing capacity of the products developed by AB Science, as well as those developed or identified in the public documents filed by AB Science with the Autorité des Marchés Financiers (AMF), including those listed in the Chapter 4 "Risk Factors" of AB Science reference document filed with the AMF on November 22, 2016, under the number R. 16-078. AB Science disclaims any obligation or undertaking to update the forward-looking information and statements, subject to the applicable regulations, in particular articles 223-1 et seq. of the AMF General Regulations.


News Article | May 17, 2017
Site: www.prnewswire.com

How this report will benefit you Read on to discover how you can exploit the future business opportunities emerging in this sector. In this brand new 164-page report you will receive 84 tables and 93 figures - all unavailable elsewhere. The 164-page report provides clear detailed insight into the global erythropoietin market. Discover the key drivers and challenges affecting the market. By ordering and reading our brand new report today you stay better informed and ready to act. • Revenue forecasts of global erythropoietin market, segmented by product type: - Epoetin Alfa - Epoetin Beta - Darbepoetin Alfa - Biosimilars - Others This section also discusses the leading drugs as well as SWOT analysis of each submarket. • Revenue forecasts of global erythropoietin market, segmented by application: - Anemia (Cancer and HIV Treatment) - Kidney Disorders (ESRD and Dialysis) - Others Forecast for each regional market is further segmented by product type, application and country: - US - Canada - UK - Germany - France - Spain - Italy - Rest of Europe - China - Japan - India - Rest of Asia-Pacific - Brazil - Mexico - Rest of Latin America - Saudi Arabia - South Africa - Rest of Middle East and Africa • Assessment of selected leading companies that hold major market shares in the erythropoietin market: - Amgen - Johnson & Johnson - Roche - Biocon - 3SBio - Pfizer - Kyowa Hakko Kirin Co Ltd - LG Life Sciences Limited - Intas Pharmaceuticals Ltd - Novartis AG Visiongain's study is intended for anyone requiring commercial analyses for the erythropoietin market. You find data, trends and predictions. To request a report overview of this report please email Sara Peerun at sara.peerun@visiongain.com or call Tel: +44-(0)-20-7336-6100 3SBio Inc. Accord Healthcare Limited Actavis Akebia Therapeutics Amgen Astellas Pharma Inc AstraZeneca Bio Sidus SA Biocon Ltd Casa Marzam Casa Saba Chugai Pharmaceutical Claes-Göran Östenson Corvidia Therapeutics DaVita Inc Dr. Reddy's Laboratories FibroGen First Shanghai Investments Limited Fresenius Medical Care North America (FMCNA) Galenica Germans Trias i Pujol Hospital GlaxoSmithKline H. Lundbeck A/S Hoffmann-La Roche Hospira Janssen Biotech Japan Chemical Research Pharmaceuticals Co., Ltd. Johnson & Johnson Kissei pharmaceuticals Kosin University Gospel Hospital Kyowa Hakko Kirin Co Ltd LG Life Sciences Limited NADRO NovaQuest Co-Investment Fund I, L.P. Novartis Institutes for BioMedical Research (NIBR) Novartis Pharmaceuticals NOXXON Pharma AG Pfizer Proveedora de Medicamentos RPG Life Sciences Sandoz Sirton Pharma Stada Stem Cell Therapeutics Corp. Teva Pharmaceutical Industries Ltd. UBI Pharma Inc. Vifor Pharma List of Organizations Mentioned in the Report Agence Nationale de Sécurité du Médicament et des Produits de Santé (ANSM) Centers of Disease Control and Preventions China's Ministry of Health (MOH) Committee for Medicinal Products for Human Use (CHMP) Department of Health & Family Welfare of Karnataka Deutsches Ärzteblatt EMA European Commission (EC) FDA Indian Council of Medical Research (ICMR) International Agency for Research on Cancer (IARC) Ministry of Health, Malaysia National Cancer Institute National Center for Biotechnology Information National Kidney Foundation UK Nephrology - Renal Association World Cancer Research Fund International World Health Organizations To see a report overview please email Sara Peerun on sara.peerun@visiongain.com


News Article | May 17, 2017
Site: www.prnewswire.co.uk

How this report will benefit you Read on to discover how you can exploit the future business opportunities emerging in this sector. In this brand new 164-page report you will receive 84 tables and 93 figures - all unavailable elsewhere. The 164-page report provides clear detailed insight into the global erythropoietin market. Discover the key drivers and challenges affecting the market. By ordering and reading our brand new report today you stay better informed and ready to act. • Revenue forecasts of global erythropoietin market, segmented by product type: - Epoetin Alfa - Epoetin Beta - Darbepoetin Alfa - Biosimilars - Others This section also discusses the leading drugs as well as SWOT analysis of each submarket. • Revenue forecasts of global erythropoietin market, segmented by application: - Anemia (Cancer and HIV Treatment) - Kidney Disorders (ESRD and Dialysis) - Others Forecast for each regional market is further segmented by product type, application and country: - US - Canada - UK - Germany - France - Spain - Italy - Rest of Europe - China - Japan - India - Rest of Asia-Pacific - Brazil - Mexico - Rest of Latin America - Saudi Arabia - South Africa - Rest of Middle East and Africa • Assessment of selected leading companies that hold major market shares in the erythropoietin market: - Amgen - Johnson & Johnson - Roche - Biocon - 3SBio - Pfizer - Kyowa Hakko Kirin Co Ltd - LG Life Sciences Limited - Intas Pharmaceuticals Ltd - Novartis AG Visiongain's study is intended for anyone requiring commercial analyses for the erythropoietin market. You find data, trends and predictions. To request a report overview of this report please email Sara Peerun at sara.peerun@visiongain.com or call Tel: +44-(0)-20-7336-6100 3SBio Inc. Accord Healthcare Limited Actavis Akebia Therapeutics Amgen Astellas Pharma Inc AstraZeneca Bio Sidus SA Biocon Ltd Casa Marzam Casa Saba Chugai Pharmaceutical Claes-Göran Östenson Corvidia Therapeutics DaVita Inc Dr. Reddy's Laboratories FibroGen First Shanghai Investments Limited Fresenius Medical Care North America (FMCNA) Galenica Germans Trias i Pujol Hospital GlaxoSmithKline H. Lundbeck A/S Hoffmann-La Roche Hospira Janssen Biotech Japan Chemical Research Pharmaceuticals Co., Ltd. Johnson & Johnson Kissei pharmaceuticals Kosin University Gospel Hospital Kyowa Hakko Kirin Co Ltd LG Life Sciences Limited NADRO NovaQuest Co-Investment Fund I, L.P. Novartis Institutes for BioMedical Research (NIBR) Novartis Pharmaceuticals NOXXON Pharma AG Pfizer Proveedora de Medicamentos RPG Life Sciences Sandoz Sirton Pharma Stada Stem Cell Therapeutics Corp. Teva Pharmaceutical Industries Ltd. UBI Pharma Inc. Vifor Pharma List of Organizations Mentioned in the Report Agence Nationale de Sécurité du Médicament et des Produits de Santé (ANSM) Centers of Disease Control and Preventions China's Ministry of Health (MOH) Committee for Medicinal Products for Human Use (CHMP) Department of Health & Family Welfare of Karnataka Deutsches Ärzteblatt EMA European Commission (EC) FDA Indian Council of Medical Research (ICMR) International Agency for Research on Cancer (IARC) Ministry of Health, Malaysia National Cancer Institute National Center for Biotechnology Information National Kidney Foundation UK Nephrology - Renal Association World Cancer Research Fund International World Health Organizations To see a report overview please email Sara Peerun on sara.peerun@visiongain.com

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