Cambridge Healthtech Institute’s 3rd Annual

Clinical Innovation for Combination Immunotherapy

Combination Strategies and Clinical Trials in Immuno-Oncology

June 18-19, 2019


Recent advances in immuno-oncology (IO) and regulatory approvals of several new agents have led to a spike in the number of IO clinical trials. Most of the current trials are small, and they are evaluating combination cancer therapy. Combination immunotherapy promises to deliver long-term survival benefits that may be unavailable with current approaches. Cambridge Healthtech Institute’s Clinical Innovation for Combination Immunotherapy conference is designed to bring together translational scientists and clinical research executives from biopharma companies and CROs, leading academic PIs, and regulatory experts to discuss innovative approaches to clinical development strategies for immunotherapy combinations.

Final Agenda

Tuesday, June 18

7:00 am Registration Open and Morning Coffee

Beacon Hill

8:00 Chairperson’s Opening Remarks

Nikolas T. Weissmueller, PhD, Associate Director, Strategic Options & Assessment, Business Insights & Analytics, R&D, Bristol-Myers Squibb

8:10 KEYNOTE PRESENTATION: Harnessing the Power of the Immune System: Lessons Learned from the Pembrolizumab Story

Cheng_JonathanJonathan Cheng, MD, Vice President & Oncology Therapeutic Area Head, Merck

Drug development in oncology has seen a rush of clinical trials given the emergence of checkpoint inhibitors in the oncologist’s armamentarium and the continued promise of immunotherapy. This has led to unprecedented opportunities resulting in a plethora of clinical trials with various designs and approaches. The lessons learned from the development of pembrolizumab will be discussed, in particular learnings in how to register a new drug quickly, the potential uses of a biomarker, and how to approach combinations for future development.

8:30 Value-Based Clinical Trials in Immuno-Oncology

Weissmueller_NikolasNikolas T. Weissmueller, PhD, Associate Director, Strategic Options & Assessment, Business Insights & Analytics, R&D, Bristol-Myers Squibb

The development of IO combination therapies requires making multiple complex design choices at speed. Throughout development, many key value drivers are uncertain, interconnected, and hinge on the broader market. A structured evaluation of the evidence and the strategic options can quantify value trade-offs and help generate clarity of action for the decision maker.

8:50 Registration Strategies for IO Agents

Witman_PhilipPhilip A. Witman, MPH, MPhil, Director, Global Product Development, Regulatory – Oncology, Pfizer Inc.

In only a few years, the advent of immuno-oncology agents has exploded the options available to patients with cancer. Some improvements have been significant while some have been incremental. Study designs include specific tumor indications or markers across tumor types, all patients vs. groups defined by biomarkers, monotherapy vs. combination therapy. The permutations are staggering, and the regulatory considerations and strategies are evolving along with the science.

9:10 Market Access Challenges and Opportunities for Orphan and Ultra-Orphan Drugs

BostonHealthcareGoss_ThomasThomas Goss, PharmD, Senior Vice President, Boston Healthcare Associates, Inc.

Topics to be discussed include:1) Looking into the global demand for an evidence-based approach toward orphan drug pricing and reimbursement 2) Current landscape on pharmaceutical practices: undertaking the burden of Illness studies, focusing on baseline quality of life, and direct and indirect costs to evaluate orphan drugs and assist with reimbursement 3) Tools to overcome the challenges faced by industry when identifying relevant data sources to document the cost and burden of orphan diseases 4) Strengths and limitations of available methods of data capture.

9:40 Grand Opening Coffee Break in the Exhibit Hall with Poster Viewing


10:25 KEYNOTE PRESENTATION: PD-1 Antibody Is Foundational Anti-Cancer Therapy Both as Single Agent and with Additional Modalities

Baynes_RoyRoy Baynes, MD, PhD, Senior Vice President and Head, Global Clinical Development, CMO, Merck Research Laboratories

PD-1 antibodies have shown important therapeutic activity and clinically meaningful benefit across a number of major cancer types and lines of treatment as monotherapy. Precision medicine tools have enriched for monotherapy activity, identified potential resistance biology and provided rationale for modalities to be explored as combination partners. Certain combinations have demonstrated clinically meaningful therapeutic benefit.

10:45 Combinatorial Treatment with Oncolytic Adenovirotherapy and CAR T Cell Therapy for Solid Tumor Treatment

Suzuki_MasatakaMasataka Suzuki, PhD, Assistant Professor, Center for Cell & Gene Therapy, Baylor College of Medicine

This presentation will cover: 1) Local treatment of oncolytic adenovirus is safe but is insufficient to eradicate advanced/metastasized tumors; 2) Successful CAR T cell therapy for solid tumors needs to overcome multiple barriers; 3) Combination with oncolytic adenovirus and CAR T cell overcomes the inherent limitations of each agent.

11:05 Reversing Resistance to Definitive Anti-PD-1 Failures with Intratumoral IL-12 and Pembrolizumab Combination Therapy

Twitty_ChrisChris Twitty, PhD, CSO, OncoSec

This talk will discuss approaches to overcome immune resistance mechanisms in cancer, namely by reinvigorating T cell exhaustion with engineered cytokines, checkpoint bispecifics and T cell engaging CD3 bispecifics.


11:25 Development of Novel Combination Immunotherapies for Ovarian Cancer and Mesothelioma

Poznansky_MarkMark C. Poznansky, MD, PhD, Director, Vaccine & Immunotherapy Center, Massachusetts General Hospital; Associate Professor, Harvard Medical School

Ovarian cancer and mesothelioma are considered immunogenic tumors and yet the impact of immunotherapy on these diseases has been limited. We set out to design novel combination immune therapies that both reverse the immune suppressive intratumoral microenvironment while arming and activating the tumor antigen specific T cell response against the cancer. We will present preclinical data in immune competent syngeneic and orthotopic mouse models that support our combinatorial approach.

11:45 The Art of War: Combating Recurrent Glioblastoma with MDNA55, an IL4 Guided Toxin - Interim Top-Line Phase 2b Results

Merchant_FaharFahar Merchant, PhD, President & CEO, Medicenna Biopharma

IL4 receptor, highly expressed in GBM and tumor microenvironment, is associated with poor survival outcomes. MDNA55 is a novel IL4-targeted fusion toxin administered in combination with MRI contrast agent for precision delivery as single treatment for rGBM. This talk focuses on the recently completed Phase2b trial of MDNA55 in rGBM.

12:00 pm Enjoy Lunch on Your Own



12:40 Chairperson’s Remarks

Rakesh Dixit, PhD, DABT, President & CEO, Bionavigen

12:45 Optimization of Clinical Trials in Immuno-Oncology and Implementation of Precision Medicine

Tsimberidou_ApostoliaApostolia-Maria Tsimberidou, MD, PhD, Professor, Investigational Cancer Therapeutics, University of Texas MD Anderson Cancer Center

The introduction of new, promising immuno-oncology treatments has significantly improved the outcomes of selected patients with cancer. Advances in technology, understanding of immune mechanisms of tumorigenesis, carefully designed clinical trials and use of standardized reporting guidelines are expected to accelerate the implementation of precision medicine in immuno-oncology.

1:05 Drivers in the Clinical Development of Cancer Combination Therapies

Schmidt_EmmettEmmett Schmidt, PhD, Distinguished Scientist & Executive Director, Merck Research Labs

More than 3,000 clinical trials are evaluating the clinical activity of the PD-1/PD-L1 checkpoint inhibitors as monotherapies and in combination with other cancer therapies. The PD-1/PD-L1 checkpoint inhibitors are remarkable for their clinical activities in shrinking tumors across a wide range of tumor types, in causing durable responses, and in their tolerability. These attributes position them as favorable agents in clinical combinations. Dr. Schmidt will update the current landscape of emerging results of PD-1 checkpoint inhibitor combinations and consider potential options for “Bedside to Bench” translation.

1:25 Immunotherapy Combinations: Friends or Foes

Dixit_RakeshRakesh Dixit, PhD, DABT, President & CEO, Bionavigen

This presentation will cover: 1) similarities and differences in outcome of immune activation with antagonists vs. agonists/combinations, etc.; 2) strategies for inducing immunologically hot vs. cold tumors; 3) single agents vs. combinations: pros and cons; 4) personalized medicine approaches to immune-oncology; 5) a case of unique bispecific of anti-PD-1:CTA4 with a potential for better TI than combination.

1:45 Implementing Patient-Specific Immunotherapy Using an Adaptive Clinical Study Design

Ferguson_AndrewAndrew Ferguson, PhD, Director, Clinical Science, Gritstone Oncology, Inc.

Development of patient-specific immunotherapies to generate neoantigen-specific T cells that control tumor growth requires unique study design considerations. These include the choice of immunotherapy platform, treatment setting to enable manufacturing of patient-specific therapies, dose selection, and combination treatments. Gritstone’s first clinical study uses an adaptive design for dose selection in combination with checkpoint blockade to provide an efficient assessment of the safety, immunogenicity, and clinical activity of patient-specific immunotherapy.

2:05 Characterization of the Safety Profile of Immune-Cancer Therapies in a Changing Environment

Adessi_CélineCéline Adessi, PhD, Safety Science Senior Group Director, pRED Oncology PDS Lead, Roche

The presentation will cover key aspects of the current paradigm shift in cancer therapy. The requirements of the characterization of the safety profile of immune therapies, particularly used in combination, are evolving. The emergent novel toxicities and the need of long term follow up of toxicities are impacting the dose finding process compare to conventional chemotherapies. In this context the benefit-risk balance evaluation will be addressed.

2:25 Refreshment Break in the Exhibit Hall with Poster Viewing

2:30-2:45 Speed Networking: Young Professionals 



3:10 PANEL DISCUSSION: Partnering, Preclinical Strategies and Tools, IO Clinical Trials; Capital Invested in IO Relative to Other Oncology Areas

Zawel_LeighModerator: Leigh Zawel, PhD, CSO, Cullinan Oncology; Executive Partner, MPM Capital


Woo_MichaelMichael Woo, PhD, Head, Search & Evaluation, Immuno-Oncology, Business Development & Licensing, Novartis Institutes for BioMedical Research, Inc.

Williams_SybilSybil Williams, PhD, Director, Biology Oncology Discovery, Merck

Young_PaulPaul Young, PhD, Executive Director, Business Development & Licensing, Merck

Adam_StaceyStacey J. Adam, PhD, Director, Cancer Research Partnerships, Foundation for the National Institutes of Health

  • Has the IO bubble popped? Why have no other IO drugs emerged with PD1/PDL1-like efficacy?
  • What combination strategies are being explored to convert poorly PD1 responsive tumors to more responsive ones?
  • Are there new IO monotherapies or combo’s that appear promising?
  • Is patient stratification practical with IO therapies?
  • What partnering strategies are most effective?
  • What is a recent deal your firm signed that you’re excited about?

4:10 Transition to Keynote


5:20 Taste of New England Welcome Reception in the Exhibit Hall with Poster Viewing

5:25 Meet the Plenary Keynotes

6:25 Find Your Table, Meet Your Moderator

6:30 Breakout Discussion Groups

7:30 Close of Day

Wednesday, June 19


7:00 am Registration Open and Morning Coffee

Beacon Hill

8:05 Chairperson’s Opening Remarks

Lena Frank, Executive Director, Alliance & Program Management, Eisai

8:15 Oncology Immunotherapy Combinations: Relationships between Companies

Frank_LenaLena Frank, Executive Director, Alliance & Program Management, Eisai

With the huge number of oncology combination studies currently ongoing, many companies are still in the process of figuring out the best practices for these combination studies and resulting filings. The management for combination studies with a collaborator may require different thinking, especially in the areas of study conduct, regulatory interactions and submissions, and commercialization.

8:35 Maximizing Specimen Assets in Immuno-Oncology Clinical Trials

Tanen_MichaelMichael Tanen, MBA, Director, Clinical Biomarker Specimen Management, Merck Research Laboratories

Immuno-oncology clinical trials have shifted toward more innovative clinical trial designs such as; Basket, Umbrella, and Adaptive methodologies that create increased complexity and demands on supporting functions. These trial designs are frequently biomarker intensive and need to be managed with a specimen-centric perspective to maximize collected bio-specimen assets. Innovative bio-specimen management approaches are becoming an essential part of empowering clinicians and researchers to better understand the individualized connections between biomarkers and human disease.

8:55 Digitalization of Clinical Trials in Immuno-Oncology

Loftiss_JillJill Loftiss, Head, Clinical Operations & Oncology, MedImmune

It is becoming increasingly valuable to leverage digital technology in a clinical trial environment to differentiate therapy, provide rapid insights and provide patientcentric solutions. This talk will aim to discuss how to move beyond ongoing digital health “pilot-itis” and describe the ecosystem of functions and capabilities that are needed to scale the use of digital technologies in clinical operations

9:15 Real World Data in Immuno-Oncology

Zhang_YingYing Zhang, Associate Director, Oncology, Center for Observational Research and Data Sciences (CORDS), Business Insights & Analytics (BI&A), Bristol-Myers Squibb

Will briefly introduce the concept of real-world data, different types of real-world data, pros and cons for each type of data, and share experience using real world data for FDA filing support.

9:35 Coffee Break in the Exhibit Hall with Poster Viewing

10:05 Poster Winner Announced


10:15 Chairperson’s Opening Remarks

Raphael Clynes, MD, PhD, Vice President, Translational Biology, Xencor

10:20 Bispecifics to the Rescue: Enabling T Cell Activation in Immune Deserts

Clynes_RaphaelRaphael Clynes, MD, PhD, Vice President, Translational Biology, Xencor

This talk will discuss approaches to overcome immune resistance mechanisms in cancer, namely by reinvigorating exhausted T cells by treatment with engineered cytokines, checkpoint bispecifics and/or T cell engaging CD3 bispecifics.

10:40 Understanding Immune Checkpoint TIM-3 Biology and Anti-TIM-3 Antibody for Cancer Immunotherapy

Jiang_XiaomoXiaomo Jiang, PhD, Investigator, Immuno-Oncology, Novartis Institutes for BioMedical Research

TIM-3 has critical roles in tumor-induced immune suppression. Blockade of TIM-3, alone or in combination with PD-1 pathway blockade, has shown anti-tumor efficacy in several preclinical cancer models. TIM-3/PD-1 pathway co-blockade to activate immune response and control tumor growth could reflect the combined effects on modulating not only the functional phenotype of dysfunctional effector T cells, but also inhibiting the suppressive activity of various suppressor cells.

11:00 NEW: Supercharging the Tumor Microenvironment with the Engineered Cytokines NKTR-214 and KNTR-255

Madakamutil _LouiLoui Madakamutil, PhD, Senior Vice President and Head of Preclinical Development, Nektar Therapeutics

This presentation will cover: 1) the idea of combining the immune-modulating properties of checkpoint inhibitors and other immunological medicines with the immune-stimulating function of engineered cytokines being conceptually powerful; 2) engineered cytokines can more effectively stimulate cytokine receptor pathways, while controlling adverse events; 3) the combination of NKTR-214 with Opdivo has demonstrated powerful anti-tumor effects and profoundly alters the tumor microenviroment, increasing effector T cell counts, increasing PD-1 expression on tumor T cells, and converting PD-L1 negative tumors to positive, while maintaining a more tolerable AE profile than traditional cytokine therapies.

11:20 Rational Combinations with an ICOS Agonist Based on Reverse Translational Biomarker Analysis

Trehu_ElizabethElizabeth Trehu, MD, CMO, Jounce Therapeutics

Inducible T cell Co-stimulator (ICOS) is a costimulatory molecule expressed on T cells upon activation. JTX-2011 is an ICOS agonist with a primary mechanism of action of activation and proliferation of primed CD4 T effector cells. Peripheral blood and tumor biomarkers collected in ICONIC, the first-in-human clinical trial of JTX-2011, have produced new insights into the mechanism of action and have informed combination strategies for future development.

11:40 Q&A with Speakers

12:00 pm Enjoy Lunch on Your Own



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

2:25 Meet the Plenary Keynotes

3:05 Close of Conference