Conference Day 2

All times in EDT

MICROBIOME REGULATION & DRUG DISCOVERY CONSIDERATIONS

8:20 am Chair’s Opening Comments

8:30 am Accelerate & Transform Discovery Research & Capitalize on the Diverse Opportunities of the Microbiome

9:00 am PANEL DISCUSSION: Legislative & Regulatory Overview of the Microbiome Arena in the United States

9:30 am Regulatory Considerations for Microbiome-based Therapeutics

Synopsis

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

10:00 am Discussing the Role of CDMO’s in the LBP Industry

10:30 am Vedanta’s Live Biotherapeutic Product Development in the Clinic: VE303 & Beyond

  • Mary Ruisi Vice President Clinical Research, Vedanta

11:00 am Harness the Power of Metabolomics to Advance Microbiome Drug Development

  • Brian Ingram Director, Discovery & Translational Sciences, Metabolon

Synopsis

  • Find out how metabolomics can provide a functional readout of the microbiome which can be used to complement other approaches such as genomics and proteomics and provide a deeper understanding of host-microbiome interactions and how these are impacted by disease and novel therapeutics
  • Discover how the incorporation of metabolomics into drug discovery programs can assist in understanding the mechanism of action, safety, and efficacy of new and novel treatments as well as aiding in aspects important for consistent clinical outcomes such as patient stratification
  • Learn about several metabolomic solutions, ranging from broad global metabolomic profiling to targeted analysis of specific microbial
    analytes, that can provide actionable insights to advance drug development
  • Gain unique and valuable insights key to understanding the microbiome’s role in several therapeutic areas using metabolomics through discussion of select case studies

11:30 am Morning Break, Refreshments & Structured Speed Networking

TRACK A:
EMERGING TECHNOLOGY & UNCONVENTIONAL APPROACHES

12:00 pm Genetically Engineered Microbial Medicines (GEMMs) for the Treatment of Chronic Diseases

Synopsis

  • Novome has developed a suite of synthetic biology tools for engineering highly abundant gut commensal microbes
  • Establishing an exclusive niche through rare carbohydrate utilization enables robust and controllable engraftment of GEMMs in the gastrointestina l tract
  • The company’s lead asset is a GEMM designed to treat hyperoxaluria and prevent recurrent kidney stone formation through the breakdown of dietary oxalate
  • A recently completed first-in-human study in healthy volunteers demonstrated safety and tolerability of the therapy as well as platform proof-of-concept that strain engraftment can be precisely controlled by once-daily oral dosing of a prebiotic control molecule

12:30 pm Phage Therapies Targeting Pathogenic Bacteria in Chronic Indications

Synopsis

  • BOLT – a novel phage discovery platform allowing clinical data within 12-18 months
  • Cystic fibrosis – A phase 1b/2a study of BX004 targeting P. Aeruginosa
  • Atopic dermatitis – BX005, a topical phage cocktail targeting S. Aureus

1:00 pm How Metagen’s is Developing an Exploratory Pipeline for Gastrointestinal & Immunological Disease by Leveraging their FMT Platform

Synopsis

  • Fecal Microbiota Transplantation platform in Japan
  • Drug discovery

TRACK B:
CLINICAL TRANSLATION, PATIENT STRATIFICATION & BIOMARKERS

12:00 pm PANEL: Learn How to Advance Your Clinical Trial Recruitment & Progress Your Pipeline

Synopsis

  • Advice on how to perfectly design your clinical trials
  • Learn how to minimise the risk of a failed clinical trial
  • Hear why certain clinical trials have been successes
  • Designing first-in-human clinical trials for live bacterial therapeutics
  • Healthy volunteers or straight into patients?
  • Biomarkers in early microbiome clinical trials

12:30 pm Microbiome Therapeutics to Address the Connection Between the Mouth & Health

1:00 pm Causal Effect Relationship Between Stress Induced Depression in Alzheimer’s Disease (AD) Progression Through Single Bacteria Modulating Regulatory T cell (Treg) and Th17 cells (Th17) Immunity

  • Giulio Pasenetti Saunders Family Chair and Professor of Neurology, Icahn School of Medicine at Mount Sinai

Synopsis

  • Role of T cell regulation of IL-15 and IL-17 in the attenuation of Segmented Filamentous Bacteria (SFB) mediated cognitive deterioration through mitigation of mechanism associated to anxiety and depression
  • Investigation of the role of IL-17a production in the gut lamina propria in the regulation of Th17 responses in the meningeal compartment of SFB mice
  • Translational studies to alleviates anxiety and depression in SFB positive mice through modulation of adaptive immunity, implications for novel AD therapies

TRACK C:
FUNDING, PARTNERSHIPS & COMMERCIALIZATION

12:00 pm Learn About Scioto Biosciences Progression to Commercialization

Synopsis

  • Developing an activated bacterial therapeutic
  • Investment and scale through partnering
  • Outcomes of phase Ib clinical trial in patients with autistic disorder

12:30 pm Building Investor Confidence in Microbiome Drug Development; How to Promote & Restore Confidence

Synopsis

  • Numerous challenges from bench to bedside but overall industry progress
  • Selecting therapeutically-relevant indications beyond C. difficile
  • The impact of recent clinical setbacks – lessons learned on how and when to enter clinic with microbiome therapeutics

1:00 pm Increase Your Knowledge on Finding & Partnerships That Will Lead to Better Patient Treatment & Value Creation

1:30 pm Lunch & Networking

2:30 pm Function-based Approaches for Microbiome Research & Drug Development

  • Wei-Kai Wu Research Physician, Department of Medical Research, Division of Gastroenterology and Hepatology, Department of Internal Medicine, National Taiwan University Hospital, Department of Internal Medicine

Synopsis

  • An integrative platform of culturomics, metabolomics and gnotobiotic animals for translational microbiome research
  • Small molecule function-directed approaches-case, study 1
  • Large molecule function-directed approaches-case, study 2

3:00 pm Engineering the Skin Microbiome to Treat Skin Diseases

  • Travis Whitfill Co-Founder, Executive Director of Advanced Technology Chair, Scientific Advisory Board, Azitra

Synopsis

  • Engineering techniques to engineer skin commensals
  • Using engineered topical microbes to treat Netherton syndrome, a rare disease
  • Preclinical data and clinical plan of ATR-12, an engineered strain of S. epidermidis, for Netherton syndrome

3:30 pm Data-Driven Discovery of Microbiome Based Therapeutics

  • Trent Munro Senior Vice President of Therapeutics, Microba

Synopsis

  • Presenting how big data in combination with machine learning can be applied for the rational discovery of microbiome-derived therapeutics
  • Applying this novel approach to IBD which has helped identify new biological leads which have shown to suppress key inflammatory pathways
  • Developing new therapeutic leads for other indications, including metabolic diseases, GI conditions and psychiatric disorders

4:00 pm Unlocking Access to Secondary Metabolites Through Cell-free Systems

Synopsis

  • How to scale up a cell-free approach to expressing novel chemicals
  • What cell-free systems could mean for the gut microbiome field
  • Summary of comparable approaches and why cell-free stands to win

2:30 pm A Human-First Discovery Approach: Leveraging Clinical Data to Identify Microbial Communities Driving Positive Clinical Outcomes

Synopsis

  • Utilizing data from human microbiota transplantation studies and machine learning to develop novel investigational microbiome therapeutics and retire risk early
  • An update on Finch’s clinical and preclinical pipeline
  • An update on latest clinical data supporting Finch’s lead product candidate, CP101, for the prevention of recurrent C. difficile infection

3:00 pm Understand How an Interventional Strategy of Metabolite Removal is Being Translated into a Clinical Setting

Synopsis

  • Microbial metabolites have been implicated in many neurological conditions, from neurodevelopmental and behavioral disorders to neurodegenerative diseases
  • Axial, in collaboration with its scientific founder, has established that metabolites derived from the gut microbiome can reach the brain and alter oligodendrocyte maturation, and hence myelination capacity, which can be tied to behavioral changes (Nature, accepted)
  • A resulting interventional strategy of metabolite removal is being translated into a clinical setting; Axial is developing AB-2004, a gut-retained sequestrant molecule, to manage irritability associated with autism. We plan to tell this mechanistic and translational story

3:30 pm Starting with the End in Mind: Leveraging Microbiome Biomarkers to Optimize Development & Market Success of Microbiome Therapeutics

Synopsis

  • 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

4:00 pm Challenges in Lung Microbiome Modulation

Synopsis

  • Air liquid interface tissue culture work for translation in cystic fibrosis
  • Challenges of the murine pulmonary model
  • Addressing investor

2:30 pm PANEL Discussion: Advancing Innovation in Microbiome Therapeutics Development Through Partnerships

Synopsis

  • Developing an activated bacterial therapeutic
  • Investment and scale through partnering
  • Outcomes of phase Ib clinical trial in patients with autistic disorder

3:00 pm Deciphering the Gut-Brain Axis

Synopsis

  • Listen to how Stellate Therapeutics are developing microbiome-derived small molecules into first-in-class
    therapies for neurological disease
  • Learn how they have successfully created funding and ongoing partnerships

3:30 pm Listen to Anaerobe Systems Way to Maximize Anaerobic Culture Techniques

4:00 pm Listen to how Partnerships & Collaborations Have Helped My-Biotics Successfully Create an Expansive Product Pipeline

Synopsis

  • MyBiotics’ SuperDonor technology – a unique disruptive technology for scalable and standardized artificial FMT
  • MBX-SD-202 – successful completion of Phase I safety with artificial FMT based on the SuperDonor technology
  • MBX-SD-202 – planned clinical development for recurrent C. diff infection

4:30 pm Afternoon Break & Networking

5:00 pm Microbiome Therapeutics to Transform the Treatment of Antimicrobial Resistant Infections

5:30 pm Microbiota Associated Microbial Drivers of Inflammatory Disease

Synopsis

  • Platform to identify microbiota-associated microbial drivers of inflammatory disease
  • Identification of lead organism implicated in a subset of patients with inflammatory bowel disease (IBD)
  • Discovery of a lead therapeutic molecule for the treatment of these patients

6:00 pm Bacteria as Evolved Chemists: Microbial Bioactive Molecules are the Lexicon of Microbe-microbe & Microbe-host Communication

  • John Hambor Director, Research Beyond Boarders, Boehringer Ingleheim

Synopsis

  • Merging both an evolutionary and pharmaceutical perspective from the realization that bacteria have evolved the capacity to synthesize a staggering catalogue of pharmacologically active small molecules
  • Learn how new insights into promising drug discovery approaches, that functionally link the role of microbial ecosystems in human health and disease, are providing growth within this space
  • Like any harbored ecological niche, complex species diversity can be maintained with careful cultivation, but the dynamic balance is both susceptible

6:30 pm Close of the Conference