Overcoming the Challenges of Microbiome Drug Development

8:00 am Morning Coffee

8:55 am Chair’s Opening Remarks

9:00 am Regulatory Considerations for Microbiome-based Therapeutics


• Overview of investigational new drug protocol requirements
• CMC considerations for live biotherapeutic products
• CMC considerations for fecal transplant based products

9:30 am Solving LBP Manufacturing Challenges: Introducing BacThera & Industry Changing Downstream Technologies

10:00am Lightning Networking: Regulation


This is the final lightning networking session, enabling you to have three, five minute video calls at random, to discuss:
• What is the biggest regulatory problem that you are facing?
• What are regulatory challenges are you expecting to have over the next 12 months?

10:15 am Panel Discussion: How Has Late Stage Clinical Success Impacted Investment Within the Microbiome Space?


• What areas are investors actively looking in?
• What are companies hearing from investors?

10:45 am Deeper Understanding Of the Microbiome Composition and Interactions Using the Alternative Microbiological Methods FISH and Flow Cytometry


• How the alternative method Fluorescence In Situ Hybridization (FISH) provides new and unexpected insights into the real microbiology of the microbiome
• How flow cytometry assays can improve quality control of LBP and probiotic products
• Case studies demonstrate rapid and specific monitoring of bacteria directly in the microbiome, improving quality control in LBP production and assessing the viability of probiotic bacteria in the colon of human probands

11:15am Speed Networking Session


12:00 pm Understanding High Resolution Metabolomics in Microbiome Studies


•Understanding the influence of the microbiome is largely not understood
•Complex data requires complex solutions
•Field is growing exponentially, but research takes time.
•How can your CRO best help your research questions?

12:10 pm Phage Therapies to Eradicate Pathogenic Bacteria in Chronic Indications


• BOLT platform – Rapid clinical proof of concept of phage therapy within 12-18 months from project initiation
• Acne – Phase 1 data on statistically significant eradication of C. acnes in acne patients using an optimized phage cocktail
• IBD – Phase 1 pharmaco-kinetic data for an orally administered phage cocktail for IBD

12:40 pm Phages as Microbiome-Editing Tools and Therapeutics


  • Phages can target and eliminate specific microbial traits in patient microbiomes
  • Using machine learning, we can predict potential genetic determinants of phage host range and other phenotypes
  • Genome engineering enables development of custom phages with modified host range
  • Felix is seeking additional partners to develop novel therapeutic solutions for critical unmet needs in the microbiome space


Scaling-Up Microbiome Manufacturing to Reach Clinical Demands Continued

12:00 pm Low-temperature Drying & Microencapsulation of Microbiomes – Advancements of Electrostatic Spray Drying PolarDry


  • Low-temperature drying in nitrogen for thermal and oxidative sensitive products
  • Microencapsulation of API and actives to eliminate degradation or denaturalization
  • A continuous process for time and energy saving; and a full production line for your choice of scale

12:10 pm Effectively Scaling Up Microbiome Manufacturing


•What is the future of GMP infrastructure?
•What does comparability mean in this space?
•What are the new or improved QC methods or potency to replace microscopy and CFU count?
•What factors affect the decision to manufacture internally or partner with a CDMO?

1:00 pm Building a Strategic Partnership to Effectively Scale & Manufacture Microbiome Based Therapeutics


• Discussing working with microorganisms including anaerobic, GMO & non-GO bacteria
• Also discussing both the manufacturing of both single and mixed strain products


Bugs & Drugs – Understanding Microbiome Metabolism

12:10 pm Xenobiotic Metabolism: The Role of the Gut Microbiome

  • Joe Roesner Senior Scientist, Merck Exploratory Science


• Bi-directional interaction between the gut microbiome and xenobiotics is critical for PK/PD of various drugs
• Ex vivo gut microbiome model was established to investigate microbial metabolism of investigational drugs
• Employing microbial metabolism liability in early drug discovery process enables comprehensive understanding of drug candidates

12:40 pm Exploring Gut Microbiota as a Source of Potential Biomarkers


• One of the hallmarks of aging comprises of decreased gut microbiota diversity
• Disturbances in gut microbiota communities have been lined with several (age-related) neurological conditions, including depression, Alzheimer’s disease, and Parkinson’s disease
• Initial results from the ANAVEX the ANAVEX®2-73 Alzheimer’s Disease Clinical Study

1:10pm Lunch / Demo Area


Use this break in proceedings to make some lunch and coffee and head across to the Demo Area to connect with our Event Partners

2:15 pm Modulation of the Gut Microbiota to Improve Vaccine- Induced Immunity


• A gnotobiotic mouse model of childhood undernutrition has varied vaccine responses to cholera toxin (CT)
• Co-housing mice with responsive and hyporesponsive microbiota improves CT vaccine responses
• Combined prebiotic and microbial intervention promote oral cholera vaccine efficacy in a preclinical model

2:45 pm A New Microbiome-Host Symbiosis, Lactobacillus Plantarum Wjl, a Precibiomic™ Strain Promoting Growth by Enhancing Host Capacity for Protein Degradation


• Introducing a new Precibiomic™ strain
• Describing the mechanism of action resulting in promoting amino acids absorption and growth factors
• Presenting the preclinical studies on juvenile rodent growth

Harnessing Engineered Microbes for Improved Function

3:15 pm Engineering Colonizing Microbes for Therapeutic Function


• Novome has built the first platform for the controlled colonization of therapeutically engineered bacteria in the gut
• There are significant advantages to engineering therapeutic microbes over delivering natural strains
• A proprietary synthetic biology toolset enables precise targeting of relevant disease biology in a wide range of diseases

2:15 pm LBP Scale-Up Challenges from Cell Bank to IMPs

2:45 pm Disruptive Approaches to Treat CNS Disease Via the Gut-Brain Axis


• Evidencing the role of the GBA in neurological disorders
• Describing Axial’ approach and therapies to treat these disorders

Overcoming the Legal Challenges of Working Within the Microbiome Space

3:15 pm Achieving IP on a Microbiome Therapeutics


• Discussing tends in issuing microbiome patents
• Exploring the IP questions investors are asking
• What are the trends in challenges to issued patents
• Providing suggestions for stronger protection going forward

End of Track B

Microbiome Biomarkers, Patient Stratification & Diagnostics

2:15 pm Starting With the End in Mind: Leveraging Microbiome Biomarkers to Optimize Development & Market Success of Microbiome Therapeutics


•How microbiome biomarkers can support product development and market acceptance
•Required properties of microbiome biomarkers for clinical applications
•Examples of applications of biomarker discovery and use of biomarkers in clinical trials

2:35 pm Exploring Microbiome Diagnostics


• Exploring how diagnostics can be incorporated into the drug discovery and development process
• Discussing the use of bioinformatics within diagnostics

2:55 pm Tissue & Circulating Microbiomes – The New Frontier


• We need to think beyond stool, oral and skin microbiomes
• Tissue and circulating microbiomes are a very promising sources of new drug discovery
• These can be analysed to improve drug trials and disease detection and monitoring as seen with our work in Oncology

3:45 pm Afternoon Refreshment Break

4:15 pm Development of a Synthetic Biotic for the Treatment of Enteric Hyperoxaluria


• Enteric hyperoxaluria results in significant kidney damage and has limited available treatment options
• Our team at Synlogic has developed SYNB8802, an engineered Synthetic Biotic to consume oxalate in the GI tract
• Preclinical studies in vitro, in vivo and mathematical modelling indicate that SYN8802 could lower urinary oxalate to meet the needs of patients

4:45 pm Toward Universal Functional Genomics: Enabling Efficient Recombineering in Diverse Bacteria


• Genetic manipulation of most bacteria remains difficult
• Efficient recombineering can be made to work in most bacterial species
• Recombineering is a gateway to many advanced techniques

Harnessing Systems Biology to Understand Microbiome Function

4:15 pm InterSystems Biology: The Next Frontier in Drug Discovery


• Defining intersystem biology
• Describe how cross talk between different species enables novel small molecule discovery
• Discuss “evolutionary” approaches to deploy large molecules

4:45 pm Towards Rational Engineering of Gut Microbiome Metabolic Outputs

  • Sean Gibbons Assistant Professor, Institute Systems Biology


• The gut microbiome is an anaerobic bioreactor that produces a variety of bioactive small molecules, many of which are absorbed by the host and circulate throughout the bloodstream
• Metagenome-scale metabolic models (i.e. Flux Balance Analysis for complex communities) provide a systems-level tool for predicting metabolic outputs of the gut microbiome for a given input
• We have built a metagenome-scale metabolic model (MICOM) that allows for personalized predictions of gut microbiome metabolic outputs and in silico design of personalized dietary and probiotic interventions. Recent experimental data indicates that many of these personalized predictions are accurate

5:15 pm Bringing it Back to the Patient – The Importance of Microbiome Therapeutics for the Treatment of C.difficile infection

  • Nancy Caralla Founding President & Executive Director, C.diff Foundation


• Hearing the viewpoint of a C. difficile survivor and microbiome advocate
• Showcasing the importance of developing microbiome therapeutics for recurrent CDIs

5:30 pm Closing Remarks

5:35 pm End of Day Two