CONFERENCE DAY TWO | THURSDAY APRIL 3, 2025
7:30 am Check In & Morning Coffee
8:20 am Chair’s Opening Remarks
Plotting the Course from Pipeline to Clinic to Inform Future Peptide Drug Discovery Efforts
8:30 am Invention of MK-0616 – Merck’s Macrocyclic Peptide Oral PCSK9 Inhibitor
Synopsis
- Highlighting the need for new modalities to drug challenging targets
- Elevating cyclic peptides as privileged scaffolds and a potential alternative to small molecules and large biopharmaceuticals
- Integrating various capabilities, including mRNA display and structure-based drug design, into drug discovery
9:00 am Turning Disordered Proteins “Druggable” with Intrametics TM
Synopsis
- Elevating that intrinsically disordered proteins (IDPs) represent half of the human proteins and perform a plethora of biological functions
- Demonstrating how, IntrameticsTM , IDP Pharma’s proprietary technology, enables for the first-time the inhibition and cellular degradation of IDPs with peptidomimetics
- Successfully delivering four peptide-derived drug candidates targeting novel IDP targets, with one at clinical stage
Combining Computational Approaches with Experimental Techniques to Investigate PeptideLigand Binding & Optimise for Solubility, Stability & Affinity
9:30 am Integrated Computational & Biochemical Cyclic Peptide Screening
Synopsis
- Uncovering how passive permeability is achievable even for relatively large cyclic peptides
- Understanding that consideration of PK from the start is critical
- Careful integration of the best technologies available is required
10:00 am Biological Evaluation of Therapeutics Engineered with De Novo Designed Miniproteins
Synopsis
- Engineering multivalent miniprotein immune cell engagers to design optimal immune synapses
- Designing and validating biological strategies to assess ligand-receptor interactions with miniproteins
- Evaluating pharmacokinetics parameters of miniprotein to engineer therapeutic products
10:30am Morning Refreshments, Networking Break & Scientific Poster Session
Weaving in Formulation Considerations to Inform Peptide Medicinal Chemistry Discovery, Design & Synthesis Requirements
11:30 am Delivering Orally Bioavailable Peptides with Increased Stability & Half-Life Through Formulation Innovation
Synopsis
- Collaborating with formulation teams to learn how to optimize formulation for peptides of different sizes • Discussing biopharmaceutics mechanisms for different oral formulation platforms
- Translating formulation considerations into early-stage design of peptides with clinical viability potential
- Sharing key learnings from the successful launch of oral semaglutide and continued success of subcutaneous peptide products
12:00 pm Realizing Needle-Free Protein Delivery: Formulation Strategies to Improve Stability & Efficacy
Synopsis
- Carefully selecting excipients to secure a backbone for robust oral protein formulations
- Addressing the multiple barriers impeding protein delivery through base formulations
- Exploiting physiological metabolic pathways to be applied for a wide range of proteinbased drugs and vaccines
12:30pm Lunch & Networking Break
1:30 pm Highlighting Challenges in Oral Peptide Delivery & Defining Approaches to Overcome Them
Synopsis
- Discussing limitations in oral peptide delivery and providing an overview of current advancements in the field
- Overcoming the challenge of transitioning from a liquid to solid dosage formulation for new oral peptide delivery systems
- Demonstrating species-related intestinal absorption and how we can ensure large animal to human translatability
2:00 pm Buccal Administration of Peptides: The EU BUCCAL-PEP Consortium
Synopsis
- Exploring why buccal administration has some advantages over other routes
- Designing buccal films to entrap peptides and permeation enhancers
- Discovering a GLP-1 analogue candidate for prototype films
Highlighting Expanded Cutting-Edge Applications of Peptides as Drug Delivery Vehicles & Diagnostic Tools
2:30 pm Warburg for Warheads: Leveraging the Cybrexa Alphalex PDC for Antigen Agnostic Targeting of Tumor Cells
Synopsis
- Introducing how to use pHLIP peptides for tumor targeting
- Reviewing the discovery and development of tumor microenvironment sensitive anticancer PDCs
- Optimizing the Alphalex-payload linker for delivering two different cytotoxic warhead classes
3:00pm Afternoon Networking Break
3:30 pm The Endosomal Escape Vehicle Platform Safely & Effectively Delivers Oligonucleotide Therapeutics to Skeletal and Cardiac Muscle Tissue for the Potential Treatment of Duchenne Muscular Dystrophy
Synopsis
- Developing the Endosomal Escape Vehicle (EEV™) family of cyclic cell-penetrating peptides that were developed to overcome limitations of intracellular therapeutics
- Delivering antisense oligonucleotides using EEV-PMO constructs to skeletal and cardiac muscle in preclinical models of Duchenne muscular dystrophy (DMD)
- Discussing how healthy human volunteers were administered a single dose of ENTR601-44, a DMD exon 44 skipping EEV-PMO construct, and showed dose-dependent exon 44 skipping with no adverse events related to study drug
- Demonstrating the therapeutic potential of the EEV platform and using findings to support further clinical development of EEV-PMO constructs in people living with DMD
4:00 pm Brain Delivery Using Peptide Shuttles: From Discovery to Application
Synopsis
- Highlighting peptides as promising tools for drug delivery to the brain
- Exploring potential applications delivering a broad range of cargoes in vitro and in vivo
- Defining the relevant hallmarks of an ideal peptide shuttle
4:30 pm Discovery & Optimization of High-Affinity Macrocyclic Peptides for PET Imaging of Human Granzyme-B
Synopsis
- Reviewing modality selection and the application of mRNA display screening to identify two high affinity hit classes of granzyme B binders
- Characterizing lead candidates in a mouse xeno-graft versus host disease model and rhesus monkey distribution studies
- Discussing prioritization, affinity optimization for a single hit class and identification of a preclinical candidate