7TH ANNUAL

Chief Medical Officer SummiT

The Annual Gathering for Biotech CMOs to Exchange Best Practices, Benchmark Ideas and Work Through R&D Challenges

EXECUTIVE SPONSORS

Organized by the Conference Forum | www.theconferenceforum.org | #CMOBoston

APRIL 4-5, 2019 HILTON BOSTON BACK BAY, BOSTON, MA

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CHIEF MEDICAL OFFICER SUMMIT AGENDA

DAY ONE - THURSDAY, APRIL 4, 2019

8:00 am Registration and Morning Coffee/Tea

8:30 am Co-Chairs’ Opening Remarks Zain Kassam, MD, MPH Co-Founder, EVP, Clinical Development and Translational Medicine, Finch Therapeutics

Julie Krop, MD CMO, EVP, Clinical Development & Regulatory Affairs, AMAG Pharmaceuticals, Inc

8:50 am Keynote: How the CMO of Sage Therapeutics Positioned a New Indication for FDA Approval In this session, hear from Dr Steve Kanes, CMO, Sage Therapeutics on how a company considered as an “up and comer” in the Cambridge biotech sector navigated the development and approval of the first treatment specifically designed for postpartum depression. Steve Kanes, MD, PhD CMO, Sage Therapeutics

9:25 am Case Study: Life of a CMO Pre and Post IPO Dr Tal Zaks shares his experience and lessons learned through one of the biggest transitions a CMO can make in biotech: • Expanding the role of the CMO to meet the requirements of a

public company• Insights into the experience• Managing growth• What role does the CMO have in articulating the data of clinical trials• What role does the CMO have in business development

opportunities• What are the personal implications of being a CMO in a publicly

traded company• Lessons learnedTal Zaks, MD, PhD CMO, Moderna Therapeutics

10:00 am Grand Opening of the Exhibit & Networking Café

10:45 am Annual Keynote Address: The Evolving Patient Engagement Movement and Its Anticipated Impact on Development Operations and Performance More specifically:• How the patient engagement movement is maturing and changing• The implications for patient data and how it is used and managed• The migration from traditional to flexible and virtual clinical trial

models• Anticipated impact on development performance and economicsKen Getz, MBA Director of Sponsored Research, Tufts CSDD / Founder, CISCRP

11:20 am Addressing the Talent Gap in Biotech – Recruit and Retain In an increasingly competitive market, there is a growing need for efficiency in attracting and retaining top talent. This session addresses: • How to source talent; pros and cons of recruiters versus word-of-

mouth• How to make the work environment optimal to keep employees

satisfied• How to compete with the competition banging on the door?• How to ease or address relocation concerns of potential hires• When is a good time to have consultants or contractors filling the

gaps?• How to access the untapped talent in academia, do they know we

exist?• Benefit/risk of having non-MDs fill the gaps and guided by a

physician• Does the industry need to evolve to attract and retain talent?• How do you decide whom to recruit first?• How to identify the right hireModerated by:Julie Krop, MD CMO, EVP, Clinical Development & Regulatory Affairs, AMAG Pharmaceuticals, Inc

Panelists:Kara Coluccio Bern Partner, Perspective Group

James Lewis Global Contract Director, Barrington James

Chris Poshka VP, Govig

11:55 am Experienced CMO Insights on Building the R&D Team This panel of experienced CMOs address:• What factors impact the decision of who to hire and when?• How to determine the order and priority of roles/functions• Setting a timeline for completing your team• What circumstances/factors might make you change your mind?• What is your approach to consultants vs vendors/CRPs vs head-

count hire?Moderated by:Jim Roach, MD EVP, R&D, Clementia Pharmaceuticals

Panelists:Christophe Arbet-Engels, MD, PhD CMO, Poxel Pharmaceuticals

Pedro Huertas, MD, PhD CMO, Sentien Biotechnologies Inc

Ramon Mohanlal, MD, PhD, MBA EVP, R&D and CMO, BeyondSpring Pharmaceuticals

John Paolini, MD, PhD SVP, CMO, Kiniksa Pharmaceuticals

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12:30 pm Optimizing Development with Model-Informed Decision-Making In this session, hear from Tina Checchio, Cytel, on the impact of pharmacometric modeling on R&D strategy. Highlights include:• Increasing team confidence at critical development milestones• Improving the probability of correct GO/NO GO decisionsTina Checchio, PhD Associate Director, Quantitative Pharmacology & Pharmacometrics, Cytel

12:45 pm Lunch

1:45 pm Track Session Choices – Addressing Unique Development Challenges in Novel Therapeutics Areas

3:10 pm Afternoon Networking Break

3:40 pm Transforming Decision Making in Early Clinical Development In this session, hear from Aiden Flynn, CEO, Exploristics and Mark Trusheim, Mark Trusheim, Visiting Scientist, MIT Sloan School of Management and Strategic Director, NEWDIGS, MIT Center for Biomedical Innovation on a unique approach to modelling and simulation in clinical trials. Highlights include:• Applications to study design• Using data to optimise clinical trial design

AGENDA CHIEF MEDICAL OFFICER SUMMIT

Track 1A: Immuno-Oncology

1:50 pm When and How to Approach Strategic Collaborations and Partnerships  How do you think about and approach a partnership as a small biotech when large pharma dominates the space? Here are a number of considerations:• What is the relationship between where your drug is in development

and the potential value you can obtain by partnering at that stage?

• Does your strategy change on partnering an asset depending on whether you have more than one asset and/or a platform technology to generate additional drug candidates?

• Timing; when do you partner/collaborate? After safety and efficacy data? Before phase 1? In between?

• Pros and Cons of supply agreements?

• Understanding your rights to negotiate for partnership of your compound

Moderated by:Beth Trehu, MD, FACP, CMO, Jounce TherapeuticsPanelists:Christina Coughlin, MD, PhD, EVP and CMO, TmunityIldiko Csiki, MD, PhD, CMO, Sensei Bio Wei Lin, MD, SVP, Clinical Development and Head of Oncology, Nektar Therapeutics Ian Walters, MD, CMO, Intensity Therapeutics

Track 1B: Microbiome

2:30 pm Addressing Unique Development Challenges in the Microbiome • What does PK/PD mean in the context and what is the playbook?

• Considerations for developing dose-ranging studies for microbiome products?   

• Determining suitable clinical endpoints and the most appropriate regulatory pathway

• How do you match traditional drug development structures with an emerging therapeutic area? Are there lessons from other technologies (e.g. CAR-T, etc)

Moderated by:Zain Kassam, MD, MPH, Co-Founder, EVP, Clinical Development and Translational Medicine, Finch Therapeutics Panelists:Jessica R Allegretti, MD, MPH, Attending Gastroenterologist,  Brigham Women’s Hospital Crohn’s and Colitis Center;  Director, Fecal Microbiota Transplant Program; Assistant Professor of Medicine, Harvard Medical School Aoife Brennan, MB, BCh, BAO, MRCPI, CEO and CMO, Synlogic

Track 2A: Gene Therapy

1:50 pm Designing Clinical Trials for Gene Therapy – A Paradigm Shift • How to utilize adaptive trial design within the specific context of

a gene therapy – are there any unique aspects of development conducive to this approach?

• How to ensure flexibility when pushing for an accelerated pathway?

• Communicating pros and cons of development options and your recommendation to your CEO

• De-risk identifying the primary efficacy endpoint without a proof of concept phase

• How to interpret the current FDA guidance• Factors to consider around in-vivo and ex-vivo regulatory guidance• How to use the guidance to direct the path of your program• Global regulatory considerations and how to overcome themModerated by:Martin Childers, DO, PhD, CMO, Asklepios BioPharmaceutical Panelists: Edward Conner, MD, CMO and SVP, Sangamo Therapeutics Kathleen Reape, MD, CMO, Spark Therapeutics

Track 2B: Rare Disease

2:30 pm The Unique Challenges of Developing an Innovative Product When No Approved Product Currently Exists • Establishing the epidemiology, unmet medical need and global

opportunity• How many patients are there really?• Yes this is a real and viable unmet medical need

• Developing a clinical and regulatory strategy• Mapping an uncharted development path – working in the data

desert• Identifying endpoints and trial design – to randomize or not to

randomize?• Finding quality investigators – finding a needle in the haystack

• How to estimate market size and forecasts• Choice of market research firm is critical

• Communicating with investors and acquirers• You need to be a teacher and plan your lessons carefully• Our data really does show efficacy and safety!• You think we are worth WHHAAT?

• Bottom line – Innovation sounds cool but be careful what you ask for!

Moderator:Cadmus Rich, MD, MBA, CMO, Aura Biosciences Panelists:Shefali Agarwal, MBBS, CMO, Epizyme Nerissa Kreher, MD, MBA, CMO, Tiburio Therapeutics Heather Paden, Head of Clinical Operations, PROMETRIKA

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CHIEF MEDICAL OFFICER SUMMIT AGENDA

• Mitigating risk around suboptimal study design• Providing regulators with a workable data packageModerated by:Robert Grundy, PhD Director of Business Development, Exploristics

Panelists:Aiden Flynn CEO, Exploristics

Mark Trusheim Visiting Scientist, MIT Sloan School of Management and Strategic Director, NEWDIGS, MIT Center for Biomedical Innovation

3:55 pm How to Make Clinical Operations as Lean as Possible in a Small Biotech Dr Krop comes with enormous experience from both large pharma to a biotech startup and brings her experience to help fellow CMOs best practices for sensible lean operations.Moderator:Julie Krop, MD CMO, EVP, Clinical Development & Regulatory Affairs, AMAG Pharmaceuticals, Inc

Panelists: Brandy Lind Senior Director, Operations, Rho

Murray Stewart, MD CMO, Rhythm Pharmaceuticals

4:30 pm The Dynamic Trial Design: The Missing Step Between Phase I and Phase II Jennifer Keppler, MBAVP, Translational Medicine, Translational Drug Development

4:45 pm How can CROs Best Meet the Needs of Small/Emerging Biotech CRO selection is enormously important for a small company. Many of the large CROs are evolving their approach to working with small companies. In this session, hear from the panel of CROs and CMOs on how the industry is reimagining this partnership.   • What are the key drivers of the evolution in these approaches and

relationships?• How are the new models different from traditional models?• How successful have these new models been? • What steps can both CROs and CMOs take to ensure success?Moderated by:Jim Roach, MD EVP, R&D, Clementia Pharmaceuticals Panelists:Lori Buckenmyer Head, Clinical Operations, InSeption GroupDaniel Burch, MD, MBA Global Medical Officer, PPD Biotech Zain Kassam, MD, MPH Co-Founder, EVP, Clinical Development and Translational Medicine, Finch Therapeutics

Stacy Lindley, PharmD VP, Head of North America Emerging Biopharma Solutions Alliance Management, IQVIA

5:30 pm Networking Reception

6:30 pm Day One Concludes

DAY TWO - FRIDAY, APRIL 5, 2019

8:00 am Morning Coffee/Tea

8:30 am Co-Chairs’ Opening Remarks Zain Kassam, MD, MPH Co-Founder, EVP, Clinical Development and Translational Medicine, Finch Therapeutics

Julie Krop, MD CMO, EVP, Clinical Development & Regulatory Affairs, AMAG Pharmaceuticals, Inc

8:40 am Keynote: Navigating Partnerships to Deliver Innovation - A Global Pharmaceutical CMO’s Perspective The path from first introduction to partnership or potential acquisition is a complex one, where the CMO plays a pivotal role. This keynote will follow the collaboration journey through the eyes of a global pharmaceutical CMO – providing in-depth perspectives on success stories, overcoming barriers and learnings from challenges. The session will also highlight the need for establishing mutual objectives early on to maximize value for patients and both partners.Bernie Zeiher, MD CMO, Astellas Pharma

9:10 am Fireside Chat with an R&D Veteran Dr Chodakewitz for the first time is joining the CMO Summit to share his incredible journey as a CMO.• The journey• Prioritizing resources• Successes and failures• Overcoming hurdles• Career decisions • Transitioning from large pharma to small biotech• Considerations for building your team in large and small

companies Jeffrey Chodakewitz, MD former EVP, Clinical Medicine and External Innovation, CMO, Vertex PharmaceuticalswithJulie Krop, MD CMO, EVP, Clinical Development & Regulatory Affairs, AMAG Pharmaceuticals, Inc

9:40 am Morning Networking Break

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AGENDA CHIEF MEDICAL OFFICER SUMMIT

10:10 am How to De-Risk a Development Program from a Regulatory Perspective The path to approval is notorious for hurdles from every angle from technical/scientific risk to competitive risk. The hurdles are even higher when there is limited or no precedent for the indication being sought typical in rare diseases and novel indications or technologies. This session will explore how to minimize these risks that translate into delays, weak ROI and in some cases non approval. We will probe the regulatory milestones during early development, late development and during Agency review. Similarities and differences in regulatory approaches in US and Europe and impact on program risk  will also be addressed.Moderated by:Laurie Smaldone, MD CMO, CSO, NDA GroupPanelists:Michael Needle, MD CMO, AVEO Oncology Shamim Ruff SVP Regulatory Affairs and Quality, Stoke Therapeutics Guest Panelist:Diego Cadavid, MD SVP, Clinical Development, Fulcrum Therapeutics

10:40 am Case Study: Why it is Crucial for a CMO to Have a Deep Understanding of Regulatory Affairs Adrian Senderowicz, MD SVP, CMO, Constellation Pharmaceuticals

11:00 am How to Navigate the Development Path from CMO to CEO This panel gives insights in what it takes and how to navigate and what to expect in the CEO role. • How can a CMO position themselves as a corporate leader?• How to build a network of advisors and who should it include?Moderated by:Zain Kassam, MD, MPH Co-Founder, EVP, Clinical Development and Translational Medicine, Finch Therapeutics Panelists:Aoife Brennan, MB, BCh, BAO, MRCPI CEO and CMO, Synlogic Greg Fiore, MD CEO, Sollis Therapeutics

11:30 am Mechanisms for CMO Performance Improvement Dr Kassam challenges the audience on performance improvement mechanisms for the CMO role. More specifically:• How do CMOs think about growing within their role? How do you

get better?• How to build a network of advisors and who should it include?• What are the structures/processes to assess your performance?

Peer review, self-assessment, managerial review, an executive coach etc.?

Zain Kassam, MD, MPH Co-Founder, EVP, Clinical Development and Translational Medicine, Finch Therapeutics

11:50 am Going from Big Pharma to Small Biotech Leadership In this session hear from Dr Wei Lin, SVP, Clinical Development and Head of Oncology, Nektar Therapeutics on his transition from established oncology giant Roche/Genentech to Nektar Therapeutics, a clinical-stage biotech with a pipeline of promising immune-oncology candidates.Wei Lin, MD SVP, Clinical Development & Head of Oncology, Nektar Therapeutics

12:10 pm Featured Session - Navigating Academia-Industry Partnerships: The View from Partners Clinical Trials Office In this session Dr Stephen Wiviott, Executive Director Clinical Trials Office, Partners HealthCare joins Dr Barry Ticho, CMO, Stoke Therapeutics for an in depth look at the realities of academia-industry partnerships.Considerations:• Developing a relationship with the PI• Contracting • Addressing IP clauses • Understanding academic IRBs• Innovative approaches to collaborationStephen Wiviott, MD, FACC Executive Director, Clinical Trials Office, Partners HealthCareSenior Investigator, TIMI Study GroupCardiovascular Division, Brigham and Women's HospitalAssociate Professor of Medicine, Harvard Medical SchoolwithBarry Ticho, MD, PhD CMO, Stoke Therapeutics

12:30 pm Lunch

1:30 pm The Role of the CMO when Interacting with the Board Join us to explore:• What are the different relationships? Direct contact, buddy

system, CEO link?• How to make sure the board understands the clinical objectives

and your perspective?Elliot Ehrich, MD CMO, Expansion Therapeutics

John Paolini, MD, PhD SVP, CMO, Kiniksa Pharmaceuticals

2:00 pm Best Practice for Preparing for Investors and Q&A This is an area of the CMO job that requires constant improvement and the purpose of this session is to give you the opportunity to hear about best practices on: • How to strategize with your CEO prior to an investor meeting on

topics to be covered• The do's and don'ts • What are appropriate materials to present?• How to navigate conversations around the FDA• What boundaries exist and how to overcome them?

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CHIEF MEDICAL OFFICER SUMMIT AGENDA

Christopher Heery, MD CMO, Bavarian Nordic Inc

Julie Krop, MD CMO, EVP, Clinical Development & Regulatory Affairs, AMAG Pharmaceuticals, Inc

2:30 pm Proving Value to Payors: Best Practices What additional measures are needed to show value to payors and to justify price? In this session where we will be joined by a payor, we discuss: • How can CMOs interact with payors to find out what info they want

from a drug program in order to make their payment decisions?    • Are we overloading trials with extra info? We’ve doubled the

number of assessments just to try and address needs of payors. How much is too much to put into a clinical trial?

• How does the CMO generate evidence and create value to present to payors?

• What kind of structural support is required to develop a value plan?• Is there a structural cost and how to decide on a financial

commitment to this process?• How to build a value story; structurally, operationally and a pathwayHarry Leider, MD, MBA EVP, CMO, Gelesis

3:00 pm Conference Concludes