Particle-based Product Development Partnership Overview Phosphorex - - PowerPoint PPT Presentation

Particle-based Product Development

Partnership Overview

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Phosphorex Snapshot

• A Contract development and manufacturing (CDMO), founded in 2005
• 12,000 ft2 facility with GLP space in Hopkinton, MA

– Wet chemistry and Analytical labs – Pilot and process development with cleanroom suites – Capability of tech transfer to GMP

• Focus on particle technology and formulation development

– Pharmaceuticals – Medical devices – Diagnostics

• Experienced team composed of experts in particle technologies with broad cross-

functional experiences

• Work across all stages: innovation, early stage, optimization, scale up and GLP
• Flexible business model
• Mission is to help clients to reduce cost and shorten the time to clinic

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• A large variety

– Polymeric microspheres and nanoparticles (biodegradable and non-degradable) – Lipid and liposome nanoparticles – Functionalized nanoparticles

• Functionalize particle surface to enable antibody or ligand conjugation

– EDC chemistry – Click chemistry

• Various particle size range for selection

– 15 – 100 nm – 100 – 500 nm – 0.5 – 20 mm – 20 – 500 mm – can be uniform/monodispersed if required

• Can be color-stained, fluorescently labeled or API-loaded
• Robust process

– Reproducibility – Scalability – GLP manufacturing

• Applications

– diagnostic – pharmaceutical

Core Competency: Development and manufacturing of microparticles and nanoparticles

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Sustained Release, Long-Acting Injectables

Cumulative Release (%) Time (Days)

10 20 30 40 50 60 70 80 90 5 10 15 20 25 30

• Reduce dose frequency

– API is released to environment slowly to maintain therapeutic level for extended period (weeks to months) – Improve patient compliance and enhance efficacy

• Extend biological half-lives of API

– Protect protein/peptide from enzymatic degradation

• Biocompatible and biodegradable material

– PLGA, PLA and PCL are used extensively as drug delivery carriers – Low toxicity and high drug encapsulation capabilities enabled multiple sustained release products in marketplace

• Flexible administration method

– SC or IM – Topical administration within specific tissue/organ

• Suitable for delivery of broad therapeutics

– Small molecule, peptide, protein, nucleic acid, etc.

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Nanoparticles for Various Applications

– Prolong circulation and decrease dosing frequency – Deliver drug to desired organ, tissue or cell – Concentrate API within targeted tissue and extend half life – Enhance blood-brain barrier crossing – Protect drug from premature degradation – Increase potency and decrease systemic toxicity – Enhance intracellular uptake of macromolecular therapeutics

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Liposome and Lipid-Based Nanoparticles (LNP)

• LNP advantages

– High rate of transfer through cell membrane – Endosome escape capability via lipid-fusion mechanism

• Useful for delivery of

– Small-molecule chemotherapeutics – Peptides and proteins – ASO, siRNA, mRNA, pDNA - encapsulated with high loading efficiency

• Potential vaccine adjuvant
• Help cross blood-brain barrier
• Transfection agent for gene delivery

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GMP via Tech Transfer Formulation Design and Prototyping

Fully Integrated Approach: From Prototyping to Clinic

Scale Up and GLP

• Interactively work with partner

to identify critical parameters, discuss pros/cons

• Design and fabricate various

prototype particles

• Optimization of small-scale

formulation.

• Scale up, process development

and optimization of large- scale formulations

• GLP batches to support IND-

enabling, pre-clinical studies

• Pilot process at a scale

finalized for tech transfer

• Strategic partnership with

CMO

• Technology transfer to CMO
• Oversight and technical

support during manufacturing

• f clinical materials
• Troubleshooting as needed

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Partner in Pharmaceutical Development

• Over 15 years of experiences in developing

and manufacturing microparticles and nanoparticles, advancing products into the clinic

• Helped 65 life sciences companies (16

large pharma & biotech) develop their products

• Completed 35 preclinical and clinical

projects collaborating with Pharma/Biotech/Academia

• Integrated approach shortens the time to

clinic

• Experiences, know-hows and innovation