(Shared Conference with World Preclinical Congress and IO Pharma Congress)

Cambridge Healthtech Institute’s Inaugural

Small Molecules for Immuno-Oncology Therapeutics

Activating the Immune System with Drug-like Orally Bioavailable Compounds to Combat Cancer

June 19-20, 2019


Small molecule agents as opposed to biologics that steer or enable the immune system to attack cancer cells, represent an emerging area of R&D focus in the oncology drug development industry. Small molecules are being investigated as stand-alone agents and synergistically with approved biologics because of the ability of small molecules to reach intracellular targets and the greater patient convenience offered by their oral bioavailability. Targeting non-redundant and/or intracellular pathways with small molecules is highly desirable because there is still a large proportion of patients who are not responsive to the current IO biologics-based therapies. However, small molecule drug development has unique challenges such as chemistry lead optimization decisions and how to find molecules against non-enzymatic, less druggable ‘protein-protein interaction’ targets, which describe many of the IO targets. Join fellow medicinal chemists, discovery biologists, pharmacologists, immunologists and oncology researchers to stay abreast of this burgeoning field and share strategies for tackling common challenges.

Final Agenda

Wednesday, June 19

12:00 pm Registration Open

12:00 Enjoy Lunch on Your Own

12:30 Transition to Plenary


12:50 PLENARY KEYNOTE SESSION 

2:20 Booth Crawl and Dessert Break in the Exhibit Hall with Poster Viewing

2:25 Meet the Plenary Keynotes

SMALL MOLECULE INHIBITORS OF IMMUNO-ONCOLOGY CHECKPOINT BLOCKADE
Beacon Hill

3:05 Chairperson’s Remarks

Frank Parlati, PhD, Vice President, Research, Calithera Biosciences

3:10 FEATURED PRESENTATION: Small-Molecules Lead to Big Opportunities in Immuno-oncology Therapies

Huck_BayardBayard R. Huck, PhD, Vice President, Global Head of Medicinal Chemistry, Discovery Technologies, Merck KGaA 

The treatment paradigm in many types of cancer is being upended with the recent approval of immune-oncology checkpoint inhibitors.  These game changing approvals have yielded improvements for patients; however, not all patients benefit from these treatments.  Small-molecule drugs offer the promise of improving on the new standard of care treatments.  This talk will review the current immuno-oncology landscape of small-molecule inhibitors with guidance on future opportunities.

3:40 Tumor Associated Macrophage (TAM) RTKs as Innate Immune Checkpoints in the Tumor Microenvironment

Earp_SShelton Earp, MD, Director, University of North Carolina Lineberger Cancer Center and Co-Founder, Meryx Pharmaceuticals

MerTK, receptor tyrosine kinase is expressed in myeloid cells where it plays a physiologic role in ingesting apoptotic material and dampening inflammatory responses, preventing chronic inflammation and auto immunity. In the tumor microenvironment this role is subverted adding to the immunosuppressive environment. Our UNC team with NCI NeXT support developed and is testing small molecule MerTK inhibitors to reverse myeloid immunosuppression and augment T cell directed agents.

4:10 CA-170, a First-in-Class, Orally Available, Small Molecule Immune Checkpoint Inhibitor Dually targeting VISTA and PDL1

Eves_TaylorP. Taylor Eves, PhD, Director of Clinical Science, Curis, Inc.

VISTA and PD-1 are independent immune checkpoints that negatively regulate T-cell function. VISTA is expressed on immune cells and tumor cells and is found to be upregulated in tumors as a potential resistance mechanism after therapy with current immune checkpoint inhibitors. Pre-clinical studies demonstrated that dual blockade of both checkpoints can be synergistic.  CA-170 directly targets VISTA and PDL1/L2 and demonstrated significant anti-tumor activity in multiple preclinical models and is currently being investigated in ph1 and ph2 clinical studies.

4:40 T Cell Dysfunction in Cancer and Combination Immunotherapy

Skokos_DimitrisDimitris Skokos, PhD, Director, Immune & Inflammatory Diseases, Regeneron Pharmaceuticals

Most patients with cancer do not develop durable antitumor responses after programmed cell death protein 1 (PD-1) or programmed cell death ligand 1 (PD-L1) checkpoint inhibition monotherapy because of an ephemeral reversal of T cell dysfunction and failure to promote long-lasting immunological T cell memory. Activating costimulatory pathways to induce stronger T cell activation may improve the efficacy of checkpoint inhibition and lead to durable antitumor responses.

5:10 4th of July Celebration in the Exhibit Hall with Poster Viewing

5:30-5:45 Speed Networking: Oncology

6:05 Close of Day


5:45 Dinner Short Course Registration

6:15 Dinner Short Course*

*Separate registration required.

Thursday, June 20

7:15 am Registration Open

7:15 Breakout Discussion Groups with Continental Breakfast

INNATE IMMUNE SYSTEM modulators: targeting STING
beacon Hill

8:10 Chairperson’s Remarks

Claus Kremoser, PhD, CEO, Phenex

8:15 Discovery and Characterization of E7766, a Novel Macrocycle-bridged STING Agonist with Pan-genotypic and Potent Antitumor Activity

Huang_Kuan-ChunKuan-Chun Huang, PhD, Associate Director, H3 Biomedicine

In recent years, STING (Stimulator of interferon genes), a potent innate immunity stimulator is an emerging therapeutic agent in the field of Immuno-Oncology. Currently, two STING agonists have already completed early clinical evaluation. In my talk, the path that led us to the discovery of a novel macrocycle-bridged STING agonist and the detail pre-clinical characteristics of the compound will be described.

8:45 Characterization of Novel STING Ligands

Schroeder_GGottfried Schroeder, PhD, Senior Scientist, Department of Pharmacology, Merck Research Labs Boston

Modulation of the innate immune receptor STING is of pharmacological interest for both oncology and autoimmune indications. Binding of cyclic dinucleotide 2’3’-cGAMP to dimeric STING stabilizes a ‘lid-closed’ protein conformation, ultimately inducing interferon production. Biophysical characterization of different classes of STING ligands revealed significant differences in binding kinetics, stoichiometry and mode of action. Results of complimentary techniques further support these observed mechanistic differences.

9:15 Cyclic Dinucleotides that Self-Assemble into Nanostructures as Potent STING Agonists for Immuno-Therapy of Cancer

Iyer_KRadhakrishnan P. Iyer, PhD, CSO, Spring Bank Pharmaceuticals

The induction of innate and adaptive immunity via activation of Stimulator of Interferon Genes (STING) signaling is a potentially transformative immuno-therapeutic strategy in cancer. Using structure-based drug design and focused library synthesis, we have discovered novel cyclic dinucleotides (CDNs), that self-assemble into cell-permeable nanostructures for uptake by immune cells. The lead CDNs administered by i.v., i.p., and i.t., routes in subcutaneous and orthotopic syngeneic tumor models show potent STING-mediated induction of Type I IFNs and cytokines resulting in profound and durable anti-tumor activity, abscopal effects and induction of immune memory. The lead CDNs have been formulated into nanospheres for controlled and sustained delivery and have also been conjugated with antibodies to enable targeted delivery to tumor site.

Drummond_MichaelMichael Drummond, PhD, Scientific Applications Manager, Chemical Computing Group

Recently, bifunctional small molecules known as Proteolysis-Targeting Chimeras, or PROTACs, have been the focus of intense research. PROTACs have the potential to offer a new modality in drug discovery, as a PROTAC tags a targeted protein for degradation rather than inhibition. Despite the demonstration of numerous advantages, the eventual success of the PROTAC approach hinges upon, among other factors, the ability to rationally modify and eventually design new PROTAC molecules. In this work, we will discuss our suite of tools for predicting the structures of ternary complexes, which are at the heart of successful protein degradation. In addition to generating a large ensemble of possible structures, we propose metrics that have been developed based on available experimental knowledge to identify the structures that are likely to degrade. We demonstrate the utility of our methods in a number of scenarios, including across different targets and PROTAC molecules.

10:15 Coffee Break in the Exhibit Hall with Poster Viewing

10:45 Poster Winner Announced

TARGETING IMMUNO-METABOLISM FOR CANCER

11:00 A2AR Antagonists: Targeting the Adenosine Pathway to Reverse Immune Suppression in the Tumor Microenvironment

Michelle L. Lamb, PhD, Team Leader, Chemistry, Oncology, IMED Biotech Unit, AstraZeneca, Boston

11:30 Small Molecule Inhibitors of CD73

Rosen_BBrandon Rosen, PhD, Senior Scientist, Chemistry, Arcus Biosciences

CD73 is an enzyme that catalyzes the dephosphorylation of adenosine monophosphate (AMP) to adenosine. Given the upregulation of CD73 in the tumor microenvironment and the role of adenosine in immune suppression, inhibition of this enzyme increases immune cell infiltration into mouse tumors and results in decreased tumor growth, making it a promising immuno-oncology target. The inhibition of CD73 using small molecules will be discussed.

12:00 pm Arginase Inhibitors

Parlati_FFrank Parlati, PhD, Vice President, Research, Calithera Biosciences

Myeloid cells are an abundant leukocyte in many types of tumors and contribute to immune evasion. Expression of the enzyme arginase is a defining feature of immunosuppressive myeloid cells and leads to depletion of L-arginine, a nutrient required for T cell and natural killer (NK) cell proliferation. CB-1158 is a potent and orally-bioavailable small-molecule inhibitor of arginase that has anti-tumor activity in pre-clinical models and is currently in clinical trials for the treatment of cancer.

Advanced-Cell-Diagnostics 12:30 Biomarker Development with RNAscope® in situ Hybridization Assay Platform

Christopher BunkerChristopher Bunker, PhD, MBA, Senior Vice President, Business Development, Advanced Cell Diagnostics (ACD), a Bio-Techne brand</p> <p class=" abstract "=" " />RNAscope in situ hybridization platform is utilized widely for high resolution tissue-based target expression data in preclinical validation and safety studies, and for clinical biomarkers. RNAscope is clinically validated and enables quantitative, cell-specific expression analysis within the TME. Advances in RNAscope technologies will be discussed in relation to novel targets in immuno-metabolism and immuno-oncology: Multiplexed RNA ISH Dual ISH/IHC for target analysis combined with cell marker staining Isoform-specific assays with exon junction probes.

1:00 Enjoy Lunch on Your Own

1:35 Dessert and Coffee Break in the Exhibit Hall with Poster Viewing

1:45-2:00 Speed Networking: Last Chance to Meet Potential Partners and Collaborators!

2:20 Chairperson’s Remarks

Michelle L. Lamb, PhD, Team Leader, Chemistry, Oncology, IMED Biotech Unit, AstraZeneca, Boston


2:25 KEYNOTE PRESENTATION: Role of Aryl Hydrocarbon Receptor in Cancer Immuno-Metabolism

Sherr_D David H. Sherr, PhD, Professor, Pathology and Laboratory Medicine, Boston University

Our studies, and those of others, indicate that the Aryl Hydrocarbon Receptor (AHR) is responsible for dampening immune responses to a variety of cancers. Here, we will demonstrate the role of the AHR in regulating immunosuppressive macrophage and T cell subsets in representative tumor types. Using small molecule inhibitors, we will present data suggesting that the AHR is an immune checkpoint regulator that can be targeted for cancer immunotherapy.

2:55 Updates on IDO1 Inhibitor in Phase I Clinical Trials

Dorsey_Frank Frank Charles Dorsey, PhD, Principal Research Scientist, Immunology, Eli Lilly & Co.


3:25 Targeting the IDO1-KYN-AhR Pathway Using Arylhydrocarbon Receptor Antagonists for Cancer Immunotherapy

Pinto_S Sheena Pinto, PhD, Senior Scientist, Drug Discovery, Phenex Pharmaceuticals

The aryl hydrocarbon receptor (AhR) is a ligand-controlled transcription factor known as a sensor of xenobiotics and tumor-promoting activities of halogenated and polycyclic aromatic hydrocarbons. Metabolites of L-Tryptophan-produced under control of IDO1 & TDO2 namely L-Kynurenine and Kynurenic acid are known ligands that modulate the activity of AhR. Activation of the IDO1-Kyn-AhR pathway and accumulation of nuclear AhR protein is frequently seen in different tumors and possibly linked to the diminished anti-tumor immune response. In order to reduce AhR-mediated immune-suppression in cancer patients, Phenex Pharmaceuticals identified small molecule AhR antagonists of high potency, and in vivo efficacy to block activated downstream signaling of AhR and its targets, which will be presented herein.

3:55 Targeting Aryl Hydrocarbon Receptor to Reverse Tumor Immunosuppression

McGovern_K Karen McGovern, PhD, Vice President, Drug Discovery, Kyn Therapeutics

Aryl Hydrocarbon Receptor (AHR) is a transcription factor expressed in many cell types that when activated leads to an immunosuppressive microenvironment in tumors. Therefore, inhibition of AHR activation provides a novel approach to reverse immunosuppression in various tumor types. Kyn Therapeutics is developing novel and potent AHR antagonists that lead to pro-inflammatory phenotypes in human immune cells and inhibit murine tumor growth alone and in combination with other agents.

4:25 Close of Conference

Arrive early to attend Tuesday, June 18 - Wednesday, June 19

TS11A: Introductory Immunology for Cancer and Autoimmune Drug Discovery or
Immuno-Oncology Clinical Trials Strategy and Execution or
Combination Cancer Immunotherapy

Recommended Short Course

SC1: Introduction of GPCR-Based Drug Discovery
SC10: In vitro and in vivo Modeling for Cancer Immunotherapy