Catch me in the Clinic
Capturing the Right Patients at the Right Time… as Quickly as Possible

8:30 am Design of IPF Clinical Trials in the Era of Approved Therapies:

  • Fernando Martinez Chief, Division of Pulmonary & Critical Care Medicine, Weill Cornell Medicine
  • Robert Kaner Associate Professor of Clinical Medicine, Weill Cornell
  • Majd Mouded Medical Director, Clinical Development, Biogen
  • Ganesh Raghu Director, Interstitial Lung Disease/ Sarcoid, Pulmonary Fibrosis Program, University of Washington

Synopsis

• Debating clinical trial design for Phase 1-3
• Endpoints to assess improvement in quality of life
• How to determine proof of concept from a phase 2 trial
• How is best to judge efficacy in phase 3

9:15 am Think Tank Roundtable Discussions:

Synopsis

More practical and highly interactive breakout roundtables where attendees can crowd-source solutions and share opinions around pre-assigned topic areas. A valuable chance for attendee to unite around hot topics, debate best practice and share experience to identify solutions.

• In Vitro & In Vivo for Target Identification & Validation

• Proof of Concept: Phase 2 Design

• Novel Intermediate Outcomes Including Circulating Biomarkers & Biomarkers of Response

9:45 am Roundtable Feedback Panel

10:15 am Morning Break & Networking

Preclinical Stream

Clinical Stream

Cellular Senescence as a New Frontier & Pathological Feature for IPF

Investigating Further Than IPF

Challenges in Dissecting Protective from Disease-Driving Cell Senescence in IPF

11:00 am

Progressive Fibrosing ILD: An Extension of IPF?

11:00 am

• The accumulation and persistence of senescent cells is now a well-established feature of IPF
• The role of senescence in IPF is complicated because cell senescence can be organ protective via mitigation of tumor development, etc.
• Discussing strategies around distinguishing protective from disease-driving cell senescence

Cory Hogaboam, Professor of Medicine,
Cedars-Sinai Medical Center

• Sharing the concept of progressive fibrosing ILD in the context of IPF clinical trials
• Updates on ongoing clinical trials with anti-fibrotics in non-IPF lung disease

Kevin Flaherty, Division of Pulmonary & Critical Care Medicine,
University of Michigan

Mechanisms of DNA Damage Response in the Lungs In Vivo

11:30 am

SSc-ILD Related Outcomes: Path to Proof of Concept in IPF

11:30 am

• Discussing the current paradigm between exposure to genotoxic stress, subsequent cellular senescence and lung fibrosis
• Assessing the relevance of in vitro findings to the in vivo context
• Revealing results and discussing relevance to human pathology and their translational opportunities

Andrei Gudkov, Professor of Oncology,
Roswell Park Comprehensive Cancer Center

• SSc presents in skin and other tissues, including lung. Pre/post treatment skin biopsies can inform on target engagement in tissue
• Treating patients earlier in disease course may prevent progression to significant ILD
• Potential to get informative clinical outcomes in smaller, shorter trials than in IPF

Joe Arron, Senior Scientist & Senior Director, Immunology Discovery,
Genentech

Targeting Cellular Senescence as a Therapeutic Approach for IPF

12:00 pm

Comparing Liver & Lung Fibrosis Trials

12:00 pm

• Analyzing cellular senescence: from academic research to clinical trials
• Discussing induced senescence cells in IPF
• Reviewing current senolytic drugs development for IPF

Marc Ramis-Castelltort, Co-Founder & Chief Executive Officer,
Senolytic Therapeutics

• Suggesting reasons for failures in both disease states
• Reviewing challenges in anti-fibrotic drug development for both liver and lung , reasons why a paradigm shift is needed, and FDA endpoints for both
• Discussing Genentech scleroderma trials, questioning whether we should be intervening in lung diseases at an earlier stage and evaluating how we can identify high risk pulmonary patients

Joanne Imperial, Vice President, Clinical Research,
Blade Therapeutics

12:30 pm Lunch & Networking

Preclinical Progress & Results

Sharing Preclinical Data on GLPG1205

1:30 pm

• The accumulation and persistence of senescent cells is now a well-established feature of IPF
• The role of senescence in IPF is complicated because cell senescence can be organ protective via mitigation of tumor development, etc.
• Discussing strategies around distinguishing protective from disease-driving cell senescence

Nick Vandeghinste,
Galapagos

IDL-2965: A Selective, Highly Potent, Clinical Stage Integrin Antagonist for the Treatment of IPF & NASH

1:30 pm

• Discussing IDL-2965: an oral small-molecule integrin antagonist that potently inhibits αvβ1, αvβ3, and αvβ6
• Presenting IDL-2965 potent antifibrotic activity at low, once-daily, oral doses in multiple animal models of lung and liver fibrosis
• Sharing investigations of IDL-2965 in an adaptive, biomarker driven Phase 1/2a clinical program, allowing rapid progression from healthy volunteers to IPF and NASH patients

Karl Kossen, Senior Vice President of Translational Science,
Galapagos

Next Generation miR-29 Mimics as a Therapy for Pulmonary Fibrosis

2:00 pm

• Developing second generation miR-29 mimics with targeting capability
• Understanding improved stability, retained miRNA activity and shown efficacy in vivo in bleomycin treated mice
• Reviewing clinical viability including systemic administration at viable doses unlike previous versions

Rusty Montgomery, Director, Research
Miragen Therapeutics

Targeting the Collagen-Specific Chaperone, HSP47, with ND-L02-s0201, a Novel Lipid Nanoparticle Containing HSP47 siRNA, for the Treatment of Fibrotic Diseases

2:00 pm

• Outlining HSP47 as a compelling anti-fibrotic target
• Sharing of preclinical data supporting ND-L02-s0201 clinical development
• Overview of on-going Phase 2 randomized, double blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics and biological activity of ND-L02-s0201 in subjects with Idiopathic Pulmonary Fibrosis

Sonya Zabludoff, Senior Director,
Nitto Denko

Targeting Lysyl Oxidase Like 2 (LOXL2) to Treat IPF

2:30 pm

• Scientific rationale for targeting LOXL2
• Presenting preclinical data supporting compound selection
• Sharing clinical data for LOXL2 inhibitors

Wolfgang Jarolimek, Head of Drug Discovery
Pharmaxis

Evaluating Partnership & Collaboration Opportunities in IPF

Perspectives for Partnerships Panel Discussion

2:30 pm

Panelists to include:

Yves Wyckmans, Senior Director, Corporate Business Development & Strategic Planning at Actelion,
Janssen Pharmaceuticals

3:00 pm Afternoon Break & Networking

3:30 pm PLN-74809, an Oral Dual Inhibitor of Integrins αVβ6 & αVβ1, for the Treatment of Idiopathic Pulmonary Fibrosis

Synopsis

• PLN-74809 was shown to be orally bioavailable and well tolerated in a Phase 1a single and multiple ascending dose study, with a long half-life supporting oncedaily dosing
• PLN-74809 is undergoing Phase 1b evaluation to assess its inhibitory effects on TGF-β activation, as measured by pSMAD levels in alveolar macrophages, collected via bronchoalveolar lavage
• Phase 2a trials in IPF patients are planned for 2H19 and will include: PET imaging to assess target engagement of PLN-74809 on αVβ6 in lungs; a multinational dose-ranging safety, tolerability and PK study with exploratory endpoints including pulmonary function, biomarkers and imaging

4:00 pm Directional Approach in Clinical Trials

  • Elias Kouchakji Senior Vice President, Clinical Development,
    Drug Safety & Pharmacovigilance, FibroGen

Synopsis

• Additional data demonstrating improvement in quantitative lung fibrosis
• Showing positive result in patient reported outcomes
• Continuing phase 3 studies with patient reported outcomes

4:30 pm Chair’s Closing Remarks

  • Fernando Martinez Chief, Division of Pulmonary & Critical Care Medicine, Weill Cornell Medicine