Lyophilization stabilizes clinical-stage core-crosslinked polymeric micelles to overcome cold chain supply challenges.

drug delivery lyophilization nanomedicine polymeric micelles tumor targeting

Journal

Biotechnology journal
ISSN: 1860-7314
Titre abrégé: Biotechnol J
Pays: Germany
ID NLM: 101265833

Informations de publication

Date de publication:
Jun 2021
Historique:
revised: 15 01 2021
received: 31 08 2020
accepted: 21 01 2021
pubmed: 24 1 2021
medline: 8 6 2021
entrez: 23 1 2021
Statut: ppublish

Résumé

CriPec technology enables the generation of drug-entrapped biodegradable core-crosslinked polymeric micelles (CCPM) with high drug loading capacity, tailorable size, and drug release kinetics. Docetaxel (DTX)-entrapped CCPM, also referred to as CPC634, have demonstrated favorable pharmacokinetics, tolerability, and enhanced tumor uptake in patients. Clinical efficacy evaluation is ongoing. CPC634 is currently stored (shelf life > 5 years) and shipped as a frozen aqueous dispersion at temperatures below -60°C, in order to prevent premature release of DTX and hydrolysis of the core-crosslinks. Consequently, like other aqueous nanomedicine formulations, CPC634 relies on cold chain supply, which is unfavorable for commercialization. Lyophilization can help to bypass this issue. Freeze-drying methodology for CCPM was developed by employing CPC634 as a model formulation, and sucrose and trehalose as cryoprotectants. We studied the residual moisture content and reconstitution behavior of the CPC634 freeze-dried cake, as well as the size, polydispersity index, morphology, drug retention, and release kinetics of reconstituted CPC634. Subsequently, the freeze-drying methodology was validated in an industrial setting, yielding a CPC634 freeze-dried cake with a moisture content of less than 0.1 wt%. It was found that trehalose-cryoprotected CPC634 could be rapidly reconstituted in less than 5 min at room temperature. Critical quality attributes such as size, morphology, drug retention, and release kinetics of trehalose-cryoprotected freeze-dried CPC634 upon reconstitution were identical to those of non-freeze-dried CPC634. Our findings provide proof-of-concept for the lyophilization of drug-containing CCPM and our methodology is readily translatable to large-scale manufacturing for future commercialization.

Sections du résumé

BACKGROUND BACKGROUND
CriPec technology enables the generation of drug-entrapped biodegradable core-crosslinked polymeric micelles (CCPM) with high drug loading capacity, tailorable size, and drug release kinetics. Docetaxel (DTX)-entrapped CCPM, also referred to as CPC634, have demonstrated favorable pharmacokinetics, tolerability, and enhanced tumor uptake in patients. Clinical efficacy evaluation is ongoing. CPC634 is currently stored (shelf life > 5 years) and shipped as a frozen aqueous dispersion at temperatures below -60°C, in order to prevent premature release of DTX and hydrolysis of the core-crosslinks. Consequently, like other aqueous nanomedicine formulations, CPC634 relies on cold chain supply, which is unfavorable for commercialization. Lyophilization can help to bypass this issue.
METHODS AND RESULTS RESULTS
Freeze-drying methodology for CCPM was developed by employing CPC634 as a model formulation, and sucrose and trehalose as cryoprotectants. We studied the residual moisture content and reconstitution behavior of the CPC634 freeze-dried cake, as well as the size, polydispersity index, morphology, drug retention, and release kinetics of reconstituted CPC634. Subsequently, the freeze-drying methodology was validated in an industrial setting, yielding a CPC634 freeze-dried cake with a moisture content of less than 0.1 wt%. It was found that trehalose-cryoprotected CPC634 could be rapidly reconstituted in less than 5 min at room temperature. Critical quality attributes such as size, morphology, drug retention, and release kinetics of trehalose-cryoprotected freeze-dried CPC634 upon reconstitution were identical to those of non-freeze-dried CPC634.
CONCLUSION CONCLUSIONS
Our findings provide proof-of-concept for the lyophilization of drug-containing CCPM and our methodology is readily translatable to large-scale manufacturing for future commercialization.

Identifiants

pubmed: 33484630
doi: 10.1002/biot.202000212
pmc: PMC7611944
mid: EMS137163
doi:

Substances chimiques

Micelles 0
Polymers 0
Sucrose 57-50-1

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

e2000212

Subventions

Organisme : Deutsche Forschungsgemeinschaft
ID : SFB1066
Organisme : Deutsche Forschungsgemeinschaft
ID : LA2937/4-1
Organisme : H2020 European Research Council
ID : 864121
Organisme : H2020 Marie Skłodowska-Curie Actions
ID : 642028
Organisme : Bundesministerium für Bildung und Forschung
ID : 16GW0319K
Organisme : ERANET-EuroNanoMed-III
ID : NSC4DIPG
Organisme : European Research Council
ID : 864121
Pays : International
Organisme : Deutsche Forschungsgemeinschaft
ID : 331065168
Organisme : Phospholipid Research Center
ID : RBA-2019-076/1-1

Informations de copyright

© 2021 The Authors. Biotechnology Journal published by Wiley-VCH GmbH.

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Auteurs

Tarun Ojha (T)

Department of Nanomedicine and Theranostics, Institute for Experimental Molecular Imaging, Faculty of Medicine, RWTH Aachen University, Aachen, Germany.
Department of Pharmaceutics, Utrecht University, Utrecht, The Netherlands.
Department of Medical Cell BioPhysics, Faculty of Science and Technology, University of Twente, Translational Liver Research, Enschede, The Netherlands.

Qizhi Hu (Q)

Cristal Therapeutics, Maastricht, The Netherlands.

Claudio Colombo (C)

Cristal Therapeutics, Maastricht, The Netherlands.

Jan Wit (J)

Saudade Pharma Consultancy, Eijsden, The Netherlands.

Michiel van Geijn (M)

Cristal Therapeutics, Maastricht, The Netherlands.

Mies J van Steenbergen (MJ)

Department of Pharmaceutics, Utrecht University, Utrecht, The Netherlands.

Mahsa Bagheri (M)

Department of Pharmaceutics, Utrecht University, Utrecht, The Netherlands.

Hiltrud Königs-Werner (H)

Electron Microscope Facility, University Hospital RWTH, RWTH Aachen University, Aachen, Germany.

Eva Miriam Buhl (EM)

Electron Microscope Facility, University Hospital RWTH, RWTH Aachen University, Aachen, Germany.

Ruchi Bansal (R)

Department of Nanomedicine and Theranostics, Institute for Experimental Molecular Imaging, Faculty of Medicine, RWTH Aachen University, Aachen, Germany.
Department of Medical Cell BioPhysics, Faculty of Science and Technology, University of Twente, Translational Liver Research, Enschede, The Netherlands.

Yang Shi (Y)

Department of Nanomedicine and Theranostics, Institute for Experimental Molecular Imaging, Faculty of Medicine, RWTH Aachen University, Aachen, Germany.

Wim E Hennink (WE)

Department of Pharmaceutics, Utrecht University, Utrecht, The Netherlands.

Gert Storm (G)

Department of Pharmaceutics, Utrecht University, Utrecht, The Netherlands.
Department of Biomaterials, Science and Technology, Faculty of Science and Technology, University of Twente, Enschede, The Netherlands.

Cristianne J F Rijcken (CJF)

Cristal Therapeutics, Maastricht, The Netherlands.

Twan Lammers (T)

Department of Nanomedicine and Theranostics, Institute for Experimental Molecular Imaging, Faculty of Medicine, RWTH Aachen University, Aachen, Germany.
Department of Pharmaceutics, Utrecht University, Utrecht, The Netherlands.
Department of Biomaterials, Science and Technology, Faculty of Science and Technology, University of Twente, Enschede, The Netherlands.

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Classifications MeSH