Inaugural

Small Molecules for Cancer Immunotherapy

Discovery and Development of Immune-Modulatory Small Molecules

April 27, 2017 | Sheraton San Diego Hotel & Marina


First-generation cancer immunotherapy agents being developed or approved are mainly monoclonal antibodies that block protein-protein interactions between T cell checkpoint receptors and their ligands. Recently, discovery efforts have dramatically expanded to focus on the development of immune-modulatory small molecules, particularly for synergistic combinations with checkpoint antibodies, and addressing a wide array of new immune-modulatory targets.

Cambridge Healthtech Institute introduces the Inaugural Small Molecules for Cancer Immunotherapy symposium, designed to convene leading developers and discovery scientists to share new targets, novel immune-modulatory inhibitors, as well as preclinical and clinical studies in combination with checkpoint antibodies.

Final Agenda

Thursday, April 27

7:25 am Registration and Morning Coffee

PD-1/PD-L1 SMALL MOLECULE INHIBITORS

7:55 Chairperson’s Opening Remarks

R.P. (Kris) Iyer, Ph.D., Co-Founder & CSO, Spring Bank Pharmaceuticals

8:00 FEATURED PRESENTATION: Small Molecule Antagonists Targeting PD-1/PD-L1 and Other Immune Checkpoint Pathways

Murali_RamachandraMurali Ramachandra, Ph.D., CSO, Aurigene Discovery Technologies Limited

We are developing small molecule oral agents dually targeting PD-L1 and another pathway to increase the response rate, and with a relatively shorter pharmacokinetic exposure for better management of irAEs. We have identified candidates potently targeting PD-L1 and VISTA or PD-L1 and TIM-3 pathways along with desirable physico-chemical profile, exposure upon oral dosing and pharmacological properties. CA-170, the first candidate from this approach dually targeting PD-L1 and VISTA, is now undergoing clinical trials.

9:00 Coffee Break

IDO AND TDO INHIBITORS

9:30 FEATURED PRESENTATION: Inventing INCB24360 (Epacadostat), a Highly Potent and Selective Indoleamine-2,3-Dioxygenase-1 (IDO1) Inhibitor for Immuno-Oncology

Andy_CombsAndrew P. Combs, Ph.D., Vice President, Discovery Chemistry, Incyte Corporation

INCB24360 was designed to potently and selectively inhibit IDO1 through a data-centric medicinal chemistry approach. The novel molecular structure of INCB24360 "breaks the rules associated with drug-likeness", but nevertheless demonstrates excellent ligand efficiency, cross-species PK, in vivo efficacy, and pre-clinical safety. Epacadostat (INCB24360) is the first small molecule I-O candidate to enter a phase 3 trial in combination with an anti-PD1 mAb (pembrolizumab) and underscores the important role small molecules, such as IDO1 inhibitors, will likely play in optimal combination I-O therapies to treat cancer patients.

10:00 Late Breaking Presentation: Dual PI3K/BRD4 Inhibitor (SF2523) for Immuno-Oncology to Suppress MYC and the Mϴ Immunosuppressive Tumor Microenvironment

Donald Durden, M.D., Professor, Department of Pediatrics, University of California, San Diego

10:30 Discovery of Novel Selective IDO1 Inhibitors

Lijun Sun, Ph.D., Director, Center for Drug Discovery and Translational Research, Beth Israel Deaconess Medical Center; Associate Professor, Harvard Medical School

We conducted in silico screening of a unique conformer library composed of 20 million entries. Using the IDO1 and TDO2 enzymatic assays, we identified 2 novel scaffolds IDO1 inhibitors. We will discuss the in silico process, the in vitro activity and ADME characterizations, and the optimizations of the inhibitors.

11:00 Enjoy Lunch on Your Own

TARGETING THE STING PATHWAY, A2A RECEPTORS AND TREGS

1:00 pm Chairperson’s Remarks

R.P. (Kris) Iyer, Ph.D., Co-Founder & CSO, Spring Bank Pharmaceuticals

1:05 Development of SB 11285, a Highly Potent STING Agonist for Application in Immune-Oncology

Kris_IyerR.P. (Kris) Iyer, Ph.D., Co-Founder & CSO, Spring Bank Pharmaceuticals

Immunotherapy has emerged as a transformative approach for the treatment of cancer. Evidence suggests that the activation of Stimulator of Interferon Genes (STING) pathway in tumor cells and/or immune cells induce type I Interferon production leading to apoptosis of tumor cells, as well as, induction of adaptive immune response thereby providing a powerful anti-cancer strategy. Herein, we describe the discovery of highly potent and selective first-in-class STING agonist SB 11285 for application in immuno-oncology.

1:35A2A Receptor Antagonists

Stephen_HatfieldStephen Hatfield, Ph.D., Associate Research Scientist, Sitkovsky Lab, New England Inflammation and Tissue Protection Institute, Northeastern University

During this presentation, I will provide the instructive overview of our discovery of cAMP elevating A2a adenosine receptor, a new paradigm in toxicology studies, and discuss why A2b is not as important as A2a receptor.

2:05 Inhibiting Treg Trafficking into the Tumor Microenvironment

David_WustrowDavid Wustrow, Vice President, Drug Discovery, FLX Bio, Inc.

Recent longitudinal studies in patients receiving IO agents demonstrate an influx of Treg in responding patients which may dampen optimal anti-tumor responses. Understanding the mechanisms of Treg recruitment into the TME thereby preventing their ability to induce immune tolerance. This talk will describe the discovery of the key mechanism of such Treg recruitment as well as in vitro and in vivo validation of this small molecule approach to selectively decreasing immune tolerance in the TME.

2:35 Refreshment Break

SMALL MOLECULE COMBINATION IMMUNOTHERAPY INHIBITORS

3:05 Small Molecules as Frontline Therapy for Immune Oncology: Experience from Ubiquitin Pathway Targets

Tauseef_ButtTauseef Butt, Ph.D., President and CEO, Progenra, Inc.

This presentation will describe de-ubiquitylase USP7 and ubiquitin ligases that promote tumor evasion by immune system. We will show data that certain tumor cells are dependent on these targets for growth and metastasis. Small molecules inhibitors that target ubiquitin pathway enzymes have dual effects, unleashing anti-tumor immune response as well as direct anti-tumor activity. We believe that small molecules that have dual properties can replace biologicals as frontline therapy for cancer.

3:35 Inhibition of Kinase-Mediated Signaling in Myeloid Cells Suppresses Peritumoral Immune Suppression in Pancreas Cancer

Michael_BurnetMichael Burnet, Ph.D., Managing Director, Oncology Discovery, Synovo GmbH

We have identified small molecule kinase inhibitors that act on myeloid cells infiltrating tumors. These compounds promote the tumor-specific local secretion of interferon gamma leading to activation of CD8+ and NK cells. Tumor specificity appears to be due to a reliance on tumor co-signalling for target pathways to be expressed. The agents have safety margins in the range of 15-30x and are effective in very low doses in mice in the order of 3 to 5 mg/kg/day. The compounds synergize both cytotoxic agents and checkpoint antibodies.

4:05 Targeting HDAC3 for Cancer Immunotherapy and Combinations

Ed_HolsonEdward Holson, Ph.D., CSO, KDAc Therapeutics


4:35 Close of Symposium