TREND CHART ON
INnovative bioindustries

Dear All,
Please, find below our new edition of TrendChart on Innovative Bioindustries. On the following pages you’ll find 8 to 10 selected items not older than 10 days. These news are placed in context with the latest relevant informations. In each issue, we also highlight a « feature story » that presents an accurate and meaningful information in the biotech area. This publication is part of the new Business Intelligence Services provided by BioPharmAnalyses and OctopusyX BioConsulting, well-recognised platforms with over twenty years of experience in highly competitive areas of pharma and biotech industries. Long-term follow-up of major strategic topics or specific requirements…. We are also able to build with you the tools you need to strategically address and maximize the opportunities available in your business area (competitive landscape, follow-up of selected companies, follow-up of product portfolio in a defined therapeutic area or in defined disorders….) Please don't hesitate to contact us by sending an email or by phone call We hope you’ll enjoy reading Trend Chart on Innovative Bioindustries Anne-Lise Berthier Olivier Revelant General Manager General Manager BioPharmanalyses OctopusyX BioConsulting alb@biopharmanalyses.fr olivier@octopusyx.fr Phone: 33 (0) 686 683 220 Phone: 33 (0) 668 071 907)

Dear All,

Please, find below our new edition of TrendChart on Innovative Bioindustries. On the following pages you’ll find 8 to 10 selected items not older than 10 days. These news are placed in context with the latest relevant informations. In each issue, we also highlight a « feature story » that presents an accurate and meaningful information in the biotech area.

This publication is part of the new Business Intelligence Services provided by BioPharmAnalyses and OctopusyX BioConsulting, well-recognised platforms with over twenty years of experience in highly competitive areas of pharma and biotech industries.

Long-term follow-up of major strategic topics or specific requirements…. We are also able to build with you the tools you need to strategically address and maximize the opportunities available in your business area (competitive landscape, follow-up of selected companies, follow-up of product portfolio in a defined therapeutic area or in defined disorders….)

Please don't hesitate to contact us by sending an email or by phone call

We hope you’ll enjoy reading Trend Chart on Innovative Bioindustries

Anne-Lise Berthier Olivier Revelant
General Manager General Manager
BioPharmanalyses OctopusyX BioConsulting
alb@biopharmanalyses.fr olivier@octopusyx.fr
Phone: 33 (0) 686 683 220 Phone: 33 (0) 668 071 907)

" Landscape in… " Gene Therapy Companies Soon-To-Be Published

BioPharmAnalyses and OctopusyX BioConsulting
are proud to announce the upcoming publication of their next report
on the dynamic and growing landscape of Gene Therapy Companies

This new report provides a deeply scrutinized mapping of
more than 280 companies involved in one of the
fastest-growing businesses in the health sector.

contents

September 2019,12th
FEATURE STORY ● The landscape of early clinical gene therapies outside oncology BASIC SCIENCE ● Enhanced CAR T cell expansion and prolonged persistence in pediatric patients with ALL CLINICAL TRIALS/DATA ● ATL1102 for DMD commencement of European regulatory interactions ● UniQure stays a step ahead in hemophilia B FINANCIAL DATA ● Passage Bio Closes $110 Million Series B Financing ● Achilles Therapeutics raises 100 M£ in oversubscribed series B financing ● Nkarta Therapeutics raises $114 Million in Series B financing to advance multiple programs into clinical trial M&A/AGREEMENTS ● Phio Pharmaceuticals announces research collaboration with Carisma Therapeutics to evaluate its self-delivering RNAi technology ● Vertex to acquire Semma Therapeutics with a goal of developing curative cell-based treatments for type 1 diabetes ● Sumitomo Dainippon buys five Roivant companies for $3 Billion with option to buy six more

September 2019,12th

FEATURE STORY
● The landscape of early clinical gene therapies outside oncology
BASIC SCIENCE
● Enhanced CAR T cell expansion and prolonged persistence in pediatric patients with ALL
CLINICAL TRIALS/DATA
● ATL1102 for DMD commencement of European regulatory interactions
● UniQure stays a step ahead in hemophilia B
FINANCIAL DATA
● Passage Bio Closes $110 Million Series B Financing
● Achilles Therapeutics raises 100 M£ in oversubscribed series B financing
● Nkarta Therapeutics raises $114 Million in Series B financing to advance multiple programs into clinical trial
M&A/AGREEMENTS
● Phio Pharmaceuticals announces research collaboration with Carisma Therapeutics to evaluate its self-delivering RNAi technology
● Vertex to acquire Semma Therapeutics with a goal of developing curative cell-based treatments for type 1 diabetes
● Sumitomo Dainippon buys five Roivant companies for $3 Billion with option to buy six more

FEATURE STORY

The landscape of early clinical gene therapies outside oncology

The field of cell and gene therapy is expanding rapidly and there is undoubtedly a wave of enthusiasm and anticipation for what these treatments could achieve next. Researchers (in collaboration with GSK / R&D Cell and Gene Therapy Discovery Research Unit) have assessed the worldwide landscape of gene therapy assets currently in early clinical development (clinical trial Phase 1/2 or about to enter clinical trial). They included all “gene therapies”, i.e. strategies that modify an individual’s protein make-up by introducing exogenous nucleic acid or nucleic acid modifiers, regardless of delivery. Unmodified cell therapies, Oncology therapies and vaccine programs (distinct therapeutic strategy) were not included. Using a December 31, 2018 cut-off date, they identified 336 gene therapies being developed for 138 different indications covering 165 genetic targets. In all, researchers have found that the early clinical gene therapy landscape comprises a very disparate group of drug candidates in terms of indications, organizations, and delivery methods. They highlight interesting trends revealing the evolution of the field towards in vivo therapies and adeno-associated viral vector-based delivery systems. It will be interesting to witness what proportion of this current list effectively translates into new medicines. The review appeared in September 06th online issue of Mol Ther

Related Informations/Publications

-Adv Biochem Eng Biotechnol. 2019 Sep 7. Gene Therapy. Del Pozo-Rodríguez A et al. University of the Basque Country UPV/EHU, Vitoria-Gasteiz, Spain
Results/Comments: Issues such as efficacy and safety of the gene delivery systems and manufacturing capacity of biotechnological companies to produce viral vectors are usually considered, but problems related to cost and patient affordability must be also faced to ensure the success of this emerging therapy. Link: Abstract
-Hum Gene Ther. 2019 Aug 9. Gene-based approaches to inherited neurometabolic diseases. Poletti et al. Dana-Farber/Children's Hospital Cancer Center, USA
Results/Comments: This review provides an overview of the gene therapy strategies currently under clinical investigation for neurometabolic Lysosomal and Peroxisomal Storage Diseases such as Adreno and Metachromatic Leukodystrophies, as well as novel emerging indications as Mucopolysaccharidoses, Gangliosidoses and Neuronal Ceroid Lipofuscinoses Link: Abstract
-Ther Deliv. 2019 Aug 19. Industry update: the latest developments in the field of therapeutic delivery, April 2019. Timmins P. University of Huddersfield, Queensgate, Huddersfield, HD1 3DH, UK
Link: Abstract

BASIC Science

Enhanced CAR T cell expansion and prolonged persistence in pediatric patients with ALL

Chimeric antigen receptor (CAR)-modified T cells targeting CD19 demonstrate unparalleled responses in relapsed/refractory acute lymphoblastic leukemia (ALL), but toxicity, including cytokine-release syndrome (CRS) and neurotoxicity, limits broader application. Moreover, 40–60% of patients relapse owing to poor CAR T cell persistence or emergence of CD19− clones. Some factors, including the choice of single-chain spacer and extracellular7 and costimulatory domains, have a profound effect on CAR T cell function and persistence. However, little is known about the impact of CAR binding affinity. There is evidence of a ceiling above which increased immunoreceptor affinity may adversely affect T cell responses. Autolus Therapeutics researchers have generated a novel CD19 CAR (CAT) with a lower affinity than FMC63, the high-affinity binder used in many clinical studies. CAT CAR T cells showed increased proliferation and cytotoxicity in vitro and had enhanced proliferative and in vivo antitumor activity compared with FMC63 CAR T cells. In a clinical study (CARPALL, NCT02443831), 12/14 patients with relapsed/refractory pediatric B cell acute lymphoblastic leukemia treated with CAT CAR T cells achieved molecular remission. The results appeared in September 02nd online issue of Nat Med or Autolus Therapeutics.

Related Informations / Publications

-Bone Marrow Transplant. 2019 Aug;54 (Suppl 2):810-814. The role of allogeneic HSCT after CAR T cells for acute lymphoblastic leukemia. Jacoby E. Tel Aviv University, Tel Aviv, Israel. Link: Abstract
-Hematol Oncol. 2019 Aug 29. Evidence of Long-Lasting Anti-CD19 Activity of Engrafted CD19 Chimeric Antigen Receptor Modified T Cells in A Phase I Study Targeting Pediatrics with Acute Lymphoblastic Leukemia. Ma F et al. The Second Hospital of Hebei Medical University, Shijiazhuang, Hebei, China Results/Comments: Monthly assessments of CD19+ minimal residual disease (MRD) and CAR-T engraftment demonstrated the anti-CD19 activity of long-term engrafted CAR-T cell clones in one patient for more than two years. Link: Abstract
-APR 2019: Autolus Therapeutics Receives FDA Orphan Drug Designation for AUTO3 for Treatment of Acute Lymphoblastic Leukemia
Link: Press Release

CLINICAL tRIALS - DATA

ATL1102 for DMD commencement of European regulatory interactions

Antisense Therapeutics announced on September 02nd that it has received confirmation of two of the three proposed Scientific Advice (SA) meetings for its planned interactions with regulatory authorities to progress the design and conduct of the next clinical trial of ATL1102 in Duchenne muscular dystrophy and the development path for product registration as previously foreshadowed by the company. The first meeting is confirmed for end of October with the second meeting scheduled for November and confirmation of the third meeting anticipated within the coming weeks. The Company had previously received advice from international regulatory consultants that, based on the existing preclinical and clinical data generated in the development of ATL1102, the Company could look to seek approval to conduct a Phase IIb clinical trial of the drug in DMD patients in Europe with this regulatory process to run in parallel with the conduct of the current Phase II study of ATL1102 in DMD patients at the Royal Children’s Hospital in Melbourne. ATL1102 is an antisense inhibitor of CD49d, a subunit of VLA-4 (Very Late Antigen-4). For further information, see ProActive Investors

Related Informations / Publications

-Antisense Therapeutics anticipates trial results for ATL1102 drug on Duchenne Muscular Dystrophy by year-end
Results/Comments: Antisense Therapeutics has completed enrolment for its phase two clinical trial using its immunomodulatory therapy ATL1102 drug on Duchenne Muscular Dystrophy (DMD), with results expected before the end of the year. The trial is underway at a neuromuscular centre which is part of Melbourne’s Royal Children’s Hospital. Link: Small Caps
-F1000Res. 2019 May 22;8. Antisense-mediated splice intervention to treat human disease: the odyssey continues. Pitout I et al. Murdoch University, Murdoch, WA, 6150, Australia. Link: Abstract - Full Text

UniQure stays a step ahead in hemophilia B

uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced that the planned enrollment of 56 patients has been achieved in the HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. Due to the high level of interest in the study from both patients and study investigators, uniQure expects to over-enroll up to six additional patients before the end of September. Etranacogene dezaparvovec has been granted Breakthrough Therapy Designation by the FDA and access to Priority Medicines (PRIME) regulatory initiative by the European Medicines Agency. The pivotal Phase III HOPE-B study builds on the success of the interim 36-week results of the Company’s ongoing Phase IIb study of etranacogene dezaparvovec, which demonstrated that a single administration of the investigational gene therapy resulted in sustained increases in Factor IX (FIX) levels up to 54% of normal, and a mean FIX level of 45% of normal. During that time, no patient reported any bleeding events or required any infusion of FIX replacement therapy for bleeds or experienced any material loss of FIX activity. Additionally, an ongoing Phase I/II study of AMT-060, the Company’s first-generation gene therapy for the treatment of hemophilia B, demonstrated that all 10 patients continue to show sustained and stable increases in FIX activity and long-term clinical benefits at up to 3.5 years of observation. For further information, see UniQure Press Release or BiopharmaDive

Related Informatiions/Publications

-JUL 2019: uniQure Announces 36 Weeks of Follow-Up Data from Phase IIb Study of AMT-061 and Long-Term Follow-Up Data for AMT-060 in Patients with Hemophilia B. Results/Comments: FIX Activity Up to 54% of Normal, with Mean of 45% of Normal, at 36 Weeks After Administration of AMT-061 in Phase IIb Study
Link: Press Release
-Hum Mol Genet. 2019 Jul 23. pii: ddz157. Advances and Challenges for Hemophilia Gene Therapy. Batty B et al. Richardson Laboratory, Queen's University, Kingston, Ontario, Canada
Results/Comments: Availability of these high-cost novel therapeutics will require evolution of both clinical and financial healthcare services to allow equitable personalization of care for persons with hemophilia. Link: Abstract
-MAY 2019: uniQure Announces Updated Clinical Data from Phase IIb Study of AMT-061 in Patients with Hemophilia B. Link: Press Release
-Mol Ther Methods Clin Dev. 2019 Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs. Majowicz A et al. uniQure N.V., Amsterdam, the Netherlands
Link: Abstract - Full Text

FINANCIAL DATA

Passage Bio Closes $110 Million Series B Financing

Passage Bio, a genetic medicines company developing AAV-delivered gene therapies for the treatment of rare monogenic central nervous system (CNS) diseases, announced on September 04th the closing of a $110.0 million Series B financing. The financing round was led by Access Biotechnology with participation from existing investors, including OrbiMed, Frazier Healthcare Partners, Versant Ventures, Lily Asia Ventures, New Leaf Venture Partners and Vivo Capital and new investors Boxer Capital of Tavistock Group, Highline Capital Management, Logos Capital and Sphera Funds Management. Proceeds from the financing will support the continued development of Passage Bio’s portfolio of AAV-delivered therapeutics for the treatment of rare monogenic CNS diseases.
For further information, see Passage Bio

Related Informations / Publications

-AUG 2019 : Passage Bio Announces Launch of Natural History Study to Evaluate Patients with GM1 Gangliosidosis
Results/Comments: The study is being conducted by the Orphan Disease Center (ODC) in the Perelman School of Medicine at the University of Pennsylvania . Link: Press Release
-JUL 2019: Passage Bio Announces New Dedicated Gene Therapy Manufacturing Suite
Results /Comments : Dedicated cGMP suite to be built under strategic partnership agreement with Paragon Gene Therapy to support future clinical and commercial production for Passage Bio’s AAV-delivered gene therapies Link: Press Release
-MAY 2019 : Passage Bio Announces Third Gene Therapy Development Program in Krabbe Disease and Supports Million Dreams Fundraising Gala Link: Press Release

Achilles Therapeutics raises 100 M£ in oversubscribed series B financing

Achilles Therapeutics, a biopharmaceutical company developing personalised cancer immunotherapies, announced on September 03rd that it has closed a £100 million Series B financing led by incoming U.S. investor RA Capital Management, corner-stoned by founding investor Syncona and joined by important new investors including Forbion, Invus, Perceptive Advisors and Redmile Group. Proceeds from this financing will deliver two human proof-of-concept studies using a unique personalised T cell therapy approach targeting clonal neoantigens in non-small cell lung cancer and melanoma. These programmes are expected to enter the clinic this year. In addition, the financing will enable the Company to continue building out its manufacturing capabilities as well as broadening its growing solid tumour pre-clinical product pipeline. Achilles is developing personalised T cell therapies for solid tumours targeting clonal neoantigens: protein markers unique to each patient that are present on the surface of all cancer cells. Using its PELEUS™ bioinformatics platform, Achilles can identify clonal neoantigens from each patient’s unique tumour profile which are present on every cancer cell. Achilles uses its proprietary process to manufacture T cells which seek to exquisitely target a specific set of clonal neoantigens in each patient. Targeting multiple clonal neoantigens that are present on all cancer cells, but not on healthy cells, reduces the risk that new mutations can induce immune evasion and therapeutic resistance, and allows individualised treatments to potentially target and destroy tumours without harming healthy tissue.
For further information see Achilles Therapeutics Press Release

Related Informations / Publications

-FEB 2019: Achilles Therapeutics receives CTA approval for phase I/II study in metastatic or recurrent melanoma – second CTA approval in 2019 (the first was for a NSCLC study). Link: Press Release
-Int J Mol Sci. 2019 Feb 11;20(3). mTOR Signaling in Cancer and mTOR Inhibitors in Solid Tumor Targeting Therapy. Tian T et al. Beijing Jiaotong University, Beijing 100044, China
Results/Comments: Innovative therapies with better efficacy and less drug resistance are still in great need, and new biomarkers and deep sequencing technologies will facilitate these mTOR targeting drugs benefit the cancer patients in personalized therapy . Link: Abstract - Full Text
-Science 25 Mar 2016:Vol. 351, Issue 6280, pp. 1463-1469. McGranahan et al. The Francis Crick Institute, London WC2A 3LY, UK
Results/Comments: In this study of lung cancer and melanoma, researchers found that a high burden of clonal tumor neoantigens correlated with improved patient survival, an increased presence of tumor-infiltrating lymphocytes, and a durable response to immunotherapy. Link: Abstract

Nkarta Therapeutics raises $114 Million in Series B financing to advance multiple programs into clinical trial

Nkarta Therapeutics, a privately-held biopharmaceutical company developing engineered natural killer (NK) cell therapies to fight cancer, announced on September 04th that it has closed an oversubscribed $114 million Series B financing, led by new investor Samsara Capital. Additional new investors include Amgen Ventures, Deerfield Management, Life Science Partners, Logos Capital and RA Capital Management, who were joined by existing investors including NEA, Novo Holdings, and SR One. In conjunction with the financing, Mike Dybbs, Ph.D., partner at Samsara, and Fouad Azzam, Ph.D., general partner at LSP, have joined the company's board of directors. This financing is intended to support two clinical trial programs of NKX101, Nkarta’s allogenic NK cell therapy targeting NKG2D in patients with either hematologic malignancies or solid tumors, as well as IND-enabling studies and clinical trials of its CAR-NK program targeting CD19 in B-cell malignancies. Nkarta is also developing additional product candidates based upon its broad NK cell engineering platform. Finally, in 2020, the company expects to complete a GMP manufacturing facility in South San Francisco to supply investigational product for its early-stage clinical trials.
For further information, see Nkarta Therapeutics Press Release

Related Informations / Publications

-Bone Marrow Transplant. 2019 Aug;54(Suppl 2):785-788. Adoptive cell therapy using engineered natural killer cells. Revzani K. The University of Texas MD Anderson Cancer Center, Houston, TX, USA
Results/Comments: The review discusses advantages and potential drawbacks of using NK cells as a novel cellular therapy against hematologic malignancies, as well as strategies to further enhance their effector function. Link: Abstract
-Front Pharmacol. 2019 Aug 13;10:898. Astragaloside III Enhances Anti-Tumor Response of NK Cells by Elevating NKG2D and IFN-γ. Chen X et al. School of Life Sciences, Tianjin University, Tianjin, China
Results/Comments: The effective anti-tumor function of Astragaloside III was achieved through up-regulation of the immune response of NK cells and elevation of NKG2D, Fas, and IFN-γ production. Link: Abstract
-Methods Mol Biol. 2019;2048:107-119. An Improved Method to Produce Clinical-Scale Natural Killer Cells from Human Pluripotent Stem Cells. Zhu H et al. University of California, San Diego, San Diego, CA, USA
Link: Abstract
-APR 2019: Nkarta Therapeutics presents data at AACR demonstrating proprietary NK cell engineering and expansion technology enhances potency and cytotoxicity of NK cells. Link Press Release

m&A-AGREEMENTS

Phio Pharmaceuticals announces research collaboration with Carisma Therapeutics to evaluate its self-delivering RNAi technology

Phio Pharmaceuticals, a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (sd-rxRNA®) therapeutic platform, announced on September 09th that it has entered into a research collaboration with Carisma Therapeutics to evaluate the potential of Phio's self-delivering RNAi compounds to synergistically modify Carisma's chimeric antigen receptor macrophages (CAR-M). Silencing of key genes using sd-rxRNA compounds may enhance the immune function of these cells as a novel adoptive cell therapy for use in cancer treatment. Carisma Therapeutics Inc. is biopharmaceutical company developing a differentiated cell therapy platform focused on engineered macrophages. The first applications of the platform are autologous chimeric antigen receptor macrophages for the treatment of solid tumors.
For further information, see Phio Pharmaceuticals Press Release

Related Informations / Publications

-AUG 2019: Phio Pharmaceuticals and Helmholtz Zentrum München to Collaborate on Novel Targets for the Use of Self-Delivering RNAi In T Cell and NK Cell Adoptive Cell Therapy Therapeutics
Results/Comments: The agreement covers the design and development of new targets based on Phio Pharmaceuticals' proprietary self-delivering RNAi platform for use in cancer immunotherapies. Link: Press Release
-MAR 2019: Phio and Glycostem to Collaborate on Use of its sd-rxRNA® platform and Glycostem's oNKord® Cell Therapy Products for the Next Generation of Natural Killer Cell-based Immunotherapy for Cancer Treatment Results/Comments: The companies' research teams will collaborate and examine the applicability of Phio's sd-rxRNA technology to be integrated into Glycostem's processes to produce NK-cells with the ultimate goal to further improve Glycostem's cellular immunotherapies for the treatment of cancer patients. Link: Press Release

Vertex to acquire Semma Therapeutics with a goal of developing curative cell-based treatments for type 1 diabetes

Vertex Pharmaceuticals announced on September 03rd that the company has entered into a definitive agreement under which Vertex will acquire Semma Therapeutics, a privately held biotechnology company pioneering the use of stem cell-derived human islets as a potentially curative treatment for type 1 diabetes, for $950 million in cash. Semma has demonstrated a differentiated approach to treat type 1 diabetes, a serious disease affecting over one million people in the United States alone. Semma has made two major scientific advances: the ability to produce large quantities of functional human pancreatic beta cells that restore insulin secretion and ameliorate hypoglycemia in animal models and a novel device that encapsulates and protects these cells from the immune system, enabling durable implantation without the need for ongoing immunosuppressive therapy.
For further information

Related Informations / Publications

-BMC Pulm Med. 2019 Aug 13;19(1):146. Patient-reported outcomes in patients with cystic fibrosis with a G551D mutation on ivacaftor treatment: results from a cross-sectional study. Bell Sc et al. The Prince Charles Hospital and QIMR Berghofer Medical Research Institute, Brisbane, QLD, Australia Results/Comments: After differences in patient demographic and clinical characteristics were controlled for, significantly better scores were observed in the G551D/IVA group than in the F508del/SOC group on multiple domains of the validated Cystic Fibrosis Questionnaire-Revised and the EuroQol 5-dimensions 5-level questionnaire. Link: Abstract - Full Text
-JUL 2019: Semma Therapeutics announces pre-clinical proof of concept in two lead programmes in type I diabetes. Link: Press Release
-JUN 2019: Vertex Expands into New Disease Areas and Enhances Gene Editing Capabilities Through Expanded Collaboration with CRISPR Therapeutics and Acquisition of Exonics Therapeutics. Link: Press Release

Sumitomo Dainippon buys five Roivant companies for $3 Billion with option to buy six more

Sumitomo Dainippon Pharma said on September 06th that it is set to pay $3 billion upfront to buy Roivant’s stake in five of its Vant startups. The big, broad deal could provide Dainippon with a series of new product launches to offset the upcoming loss of patent protection on bipolar depression drug Latuda. Latuda is Sumitomo Dainippon’s drug for schizophrenia, which will lose U.S. market exclusivity in 2023. The company is Japan’s seventh-largest pharmaceutical company by revenue, and like other Japanese drug companies, is struggling with a declining domestic population, forcing it to look outside the country for growth. In April, Dainippon sketched out a plan for how it will establish new growth engines ahead of the loss of exclusivity for Latuda, one of its primary revenue drivers. Efforts to mitigate the anticipated loss of sales through existing assets have hit setbacks, including the recent phase 3 failure of napabucasin in pancreatic cancer, ramping up the need for Dainippon to buy in new growth drivers. At Roivant, Dainippon found a single organization capable of meeting that need. Dainippon is set to pay $3 billion to take over Roivant’s stakes in five Vants: Myovant, Urovant, Enzyvant, Altavant and an as yet unidentified Vant. For further information

Related Informations / Publications

-Cancer Chemother Pharmacol. 2019 Aug 19. Pharmacokinetics of thiotepa in high-dose regimens for autologous hematopoietic stem cell transplant in Japanese patients with pediatric tumors or adult lymphoma. Kondo E et al. Okayama University Graduate School of Medicine, Dentistry and Pharmaceutical Sciences, Okayama, Japan Link : Abstract
-AUG 2019: Datavant Partners with the People-Centered Research Foundation to De-Identify and Link Data Across National Clinical Research Network Link : Press Release
-AUG 2019: Altavant Sciences Initiates Phase 2a Study of Rodatristat Ethyl in Pulmonary Arterial Hypertension
Link : Press Release

Please, add our e-mail (alb@biopharmanalyses.fr) to your e-mail address book.
To learn more about us:

LinkedIn BioPharmAnalyses
LinkedIn Anne-Lise Berthier
Twitter

Open in a browser/Ouvrir dans un navigateur
Unsubscribe/Se désinscrire
To unsubscribe from all further electronic notices from BioPharmAnalyses, you can also e-mail your name to alb@biopharmanalyses.fr entering "Unsubscribe" in the subject line.

Pour un panorama synthétique de l’actualité d’une société, d’une pathologie…

Pour identifier les acteurs impliqués dans une pathologie donnée

Adoption de la nouvelle réglementation européenne sur les essais cliniques

Le Parlement européen vient d’adopter à une très large majorité (594 voix pour, 17 contre et 13 abstentions) le rapport sur la proposition de règlement relatif aux essais cliniques de médicaments à usage humain. Le texte qui abroge l’actuelle directive 2001/20/CE a notamment vocation à stimuler la compétitivité de l’Europe pour la recherche clinique grâce à une harmonisation et une simplification des procédures entre les Etats-membres. Est notamment prévue la création d’un cadre uniforme pour l'autorisation d'essais cliniques avec le dépôt d’un seul dossier pour la réalisation d’une étude dans plusieurs Etats-membres Le dépôt de la demande d'autorisation se fera via un portail de l’Agence européenne du médicament et la procédure d’évaluation du protocole sera réalisée par un seul Etat-membre. Dès la demande soumise, ce dernier sera par ailleurs tenu de répondre dans les délais impartis.
Deux autres nouveautés majeures vont aussi faire leur apparition. La première concerne la possibilité pour la Commission de procéder à des contrôles dans les Etats-membres mais aussi dans des pays tiers pour vérifier le respect de la réglementation. Enfin, tous les promoteurs d’essais cliniques, groupes pharmaceutiques comme chercheurs académiques et associations, devront publier les résultats de leurs études dans une base de données accessible au public. Cette obligation devra être respectée dans un délai d’un an à compter de la fin de l’étude. Par ailleurs, les rapports d'études cliniques complets devront aussi être publiés dès qu'une décision de mise sur le marché est prise ou si l'autorisation de mise sur le marché est retirée. Des amendes sont prévues pour les promoteurs qui ne respecteraient pas ces exigences. dans les délais impartis.

Lien
Article du site du Parlement Européen