Benchmarking the Specific Diseases the Inflammasome can Target

8:00 am Pharmacology Inhibition of the NLRP3 Inflammasome

  • Matt Cooper Chief Executive Officer & Co-Founder, Inflazome

Synopsis

  • The early breakthroughs
  • Innate immunity, infection & chronic inflammation
  • Drug development
  • Clinical utility in CNS & peripheral diseases
  • Inzomelid & Somalix

8:30 am Fbx03 Inhibitor KT-1002 for the Treatment of Inflammatory Bowel Disease

Synopsis

• Fbx03 involvement in the release of cytokines
• Connection of Fbx03 to IBD
• Development of KT-1002
• Challenges faced with First-in-Class E3 Ligase inhibitor

9:00 am Inflammasome Modulation to Treat NASH & Liver Fibrosis

  • Ariel Feldstein Professor & Chief, Pediatric Gastroenterology & Founder, UC San Diego & Jecure Therapeutics

Synopsis

• Describe experimental and human evidence for the role of NLRP3 inflammasome in NASH development
• Review mechanisms of cell specific activation of the NLRP3 inflammasome in liver inflammation and fibrosis
• Dissect the IL-1 dependent and -independent mechanisms of liver injury triggered by NLRP3 activation

9:30 am Characterizing NLRP3 in Parkinson’s Disease

  • Matthew Havrda Assistant Professor of Molecular & Systems Biology, Dartmouth School of Medicine

Synopsis

• Evidence supporting a role for NLRP3 in toxicant based mouse models of Parkinson’s disease
• Histopathologic analysis of NLRP3 expression in Parkinson’s patients
• Evaluation of plasma-born inflammasome related proteins in Parkinson’s patients

10:00 am Morning Refreshments & Networking

Too Much Choice? Delving into Target Identification for the Inflammasome

11:00 am Targeting Ion Channels to Trigger Inflammasome-Dependent Tumor Immunity

  • Marcelo Hill Chief Scientific Officer & Founder, ARDAN ImmunoPharma

Synopsis

• The cation channel TMEM176B inhibits the NLRP3 inflammasome by controlling cytosolic Ca2+.
• Lack of Tmem176b enhances antitumor immunity via the caspase-1/IL-1b pathway
• Human tumors responding to immune checkpoint blockade display an inflammasome-activated signature
• A TMEM176B inhibitor improves the antitumor activity of immune checkpoint blockade

11:30 am NLRP3 Inflammasome, Chemotaxis & Autoimmune Disease

Synopsis

• NLRP3 inflammasome and human diseases
• Current therapeutic targeting NLRP3 inflammasome
• NLRP3 inflammasome controls autoimmunity through regulating the chemotaxis of autoreactive immune cells.
• Perspective on NLRP3 inflammasome pathway as a therapeutic target for autoimmune diseases

12:00 pm Approaching Neurodegenerative Disease as an Immunological Disease

  • CJ Barnum Director of Neurosciences, INmune Bio

Synopsis

  • Chronic inflammation starts early, is the root cause of sickness syndromes, and
    promulgates the proteinopathies observed in nuerodegenerative diseases such as
    Parkinson’s and Alzheimer’s disease
  •  Effectively targeting chronic inflammation requires threading the needle
    between a sufficiently potent anti-inflammatory treatment that is not immunonor
    glial- suppressive
  •  Inflammatory biomarkers will play an important role in determining when and
    how these therapies should be used

Improving Innovation in Clinical Trial Design to Enhance Execution

12:30 pm Roundtable Session: Designing Phase II Studies

Synopsis

This round table session will host interactive discussions amongst your tables and allow you to share your opinions with your peers on determining effective, it will involve assessing first indications for putting new compounds in to a phase II Study

1:00 pm Lunch & Networking

Looking Outside of the NLRP3 Inflammasome

2:00 pm Activation of the NLRP1 Inflammasome

Synopsis

• Insights into the mechanism of DPP8/9 inhibitor-induced pyroptosis
• ASC-independence of CARD8 and ASC-dependence of NLRP1
• Requirement of human caspase-1 autoproteolysis for inflammasome

2:30 pm Targeting the Inflammasome through P2X7 receptor & Its Potential Implications for NASH Therapy

Synopsis

• Exploring the role of P2RX7 in NASH
• Effects of P2RX7 inhibition in liver cells and animal models with liver injury
• Clinical development and challenges of the first-in-class P2RX7 inhibitor, SGM-
1019

3:00 pm Inflammasome Proteins as Biomarkers

Synopsis

• Inflammasome proteins can be used as diagnostic biomarkers in a variety of diseases and conditions such as mild cognitive impairment, multiple sclerosis, traumatic brain injury and stroke
• Inflammasome proteins can be used as biomarkers to monitor disease severity
• Inflammasome proteins can be used as prognostic biomarkers of acute injury outcome

3:30 pm Afternoon Break & Networking

3:32 pm

Looking Outside of the NLRP3 Inflammasome

4:00 pm The Immune Response in Duchenne Muscular Dystrophy: Current Therapeutic Approaches & Potential Translational Opportunities

  • John Wing Senior Medical Science Liaison , PTC Therapeutics

Synopsis

• Discussion of the immune response in the pathogenesis of Duchenne Muscular Dystrophy
• The role of immunomodulation in DMD: the benefits of corticosteroids and the development of future and combination therapies
• What are the current translational opportunities that would support future therapies in DMD?

4:30 pm Mastermind Session: Discussing the Potential of Inflammasome Therapeutics in Combination

  • John Wing Senior Medical Science Liaison , PTC Therapeutics

Synopsis

• The complexity of the immune response in DMD: understanding the balance in the regulation of immunity
• Current therapeutic approaches in DMD
• Lessons learned from other inflammatory disease: Translational Medicine

5:00 pm Chair’s Closing Remarks

  • Alan Watt Chief Scientific Officer, Nodthera