Cambridge Healthtech Institute’s Inaugural

Immuno-Oncology Clinical Trials Strategy and Execution

Improving Clinical Success Rates for IO Agents and Combinations

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. There are specific limitations in clinical development in IO such as treatment of patients not sufficiently represented in clinical trials, uncertainty of the optimal treatment dosing and duration, and lack of understanding regarding long-term immune related toxicities and atypical tumor responses. Moreover, combination immunotherapy brings additional levels of complexity and requires novel approaches to trial design and execution. Cambridge Healthtech Institute’s Inaugural Immuno-Oncology Clinical Trials Strategy and Execution conference is designed to bring together clinical research executives from biopharma companies and CROs, leading academic PIs, and regulatory experts to discuss the next generation of IO clinical trials.

Final Agenda

Tuesday, June 18

7:00 am Registration Open and Morning Coffee


8:00 Chairperson’s 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.

8:40 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.

9:10 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

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.

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

10:25 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.

10:55 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.

BostonHealthcare 11:25 Market Access Challenges and Opportunities for Orphan and Ultra-Orphan Drugs

Thomas 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.

11:55 Transition to Lunch

12:00 pm Enjoy Lunch on Your Own

12:30 Session Break


1:05 Chairperson’s Remarks

Bruno Gomes, DVM, PhD, Global Head of Biomarkers Oncology, Roche

1:10  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.

1:35 Challenges and Opportunities for Biomarkers in Early Clinical Development

Gomes_BrunoBruno Gomes, DVM, PhD, Global Head of Biomarkers Oncology, Roche

The presentation will present the opportunities and challenges in developing impactful biomarker strategies for early clinical development of new immuno-oncology drugs. We will focus on the design of fit-for-purpose entry into human clinical trials, proof or dis-proof of mechanism of action and optimal biological dose finding.

2:00 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.

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


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 combos 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 Welcome Reception in the Exhibit Hall with Poster Viewing

6:25 Find Your Table, Meet Your Moderator

6:30 Breakout Discussion Groups View details

7:30 Close of Day

Wednesday, June 19

7:00 am Registration Open and Morning Coffee


8:00 Chairperson’s Remarks

Justin Fairchild, Executive Director, Clinical Development, Parker Institute for Cancer Immunotherapy

8:05 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

8:35 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.

9:05 Sponsored Presentation (Opportunity Available)

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


10:20 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.

10:45 The Parker Institute’s Collaborative and Integrated Approach to Immuno-Oncology: Innovative Clinical Trials

Fairchild_JustinJustin Fairchild, Executive Director, Clinical Development, Parker Institute for Cancer Immunotherapy

The Parker Institute for Cancer Immunotherapy’s mission is to accelerate the development of breakthrough immune therapies to turn cancer into a curable disease. Through collaborative partnerships in both industry and academia, innovative trial designs, and integration of clinical and correlative datasets, Parker is advancing the understanding of novel immuno-oncology combinations.

11:10 PANEL DISCUSSION: Combination Collaborations in Immuno-Oncology Clinical Trials

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

Panelists: Justin Fairchild, Executive Director, Clinical Development, Parker Institute for Cancer Immunotherapy

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

Jill Loftiss, Head, Clinical Operations & Oncology, MedImmune

  • How do combination collaborations require different thinking from a relationship that involves a single compound?
  • What potential strategies should be reviewed prior to entering into a combination collaboration?
  • What specific areas will potentially need new best practices as compared to monotherapy collaborations?

11:50 Transition to Lunch

12:00 pm Enjoy Lunch on Your Own

12:30 Transition to Plenary


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