adoptive cell therapy practice

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1 Adoptive Cell Therapy Practice Expertise & Experience Selected Case Studies

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Page 1: Adoptive Cell Therapy Practice

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Adoptive Cell Therapy PracticeExpertise & ExperienceSelected Case Studies

Page 2: Adoptive Cell Therapy Practice

2ACT/Oncology Practice Capabilities © Cello Health BioConsulting 2019

Adoptive Cell TherapyExperience & Expertise

Practice Leads:Joel Sandler, PhD, Associate PrincipalMike Rice, MBA, PrincipalAndrew Ng, PhD, Associate Consultant

Appreciation of the Science

• Our team of PhD-trained consultants and analysts dig deep into the biology of our clients’ technology and the diseases they aim to address. Target expression, immune infiltration, cell properties, resistance mechanisms, multiplexing and enabling technologies are among considerations we ponder daily.

Experience & Insight

• Completed numerous recent engagements with ACT-focused innovator (Biotech) and consolidator (Pharma) clients to provide strategic guidance informed by the science and value proposition of ACT technology within the context of rapidly evolving technical, clinical, competitive, and commercial parameters.

Robust & Innovative Resources

• In order quickly and robustly identify strategic implications of the science underlying ACT modalities, targets, and indications, our talented team of analysts have built and continually maintain a series of in-house databases that collectively characterize the competitive and technology landscapes and provide our clients with actionable tools and guidance.

Adoptive Cell Therapy Team

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3ACT/Oncology Practice Capabilities © Cello Health BioConsulting 2019

Adoptive Cell TherapyExperience & Expertise

Key Value Drivers for Our Clientele

⧫ Experience: 30 years of experience in understanding the oncology market across all tumor types

⧫ Prescience: Tracking and forecasting events and trends in the intensely competitive and increasingly technical oncology development space; prediction of future changes in treatment algorithms

⧫ Sense of Urgency: Appreciation for the small window of opportunity to make an impact, how important initial development choices are, and how that impacts the financing and partnering strategy

⧫ Supply Chain: Understanding of different manufacturing processes and potential barriers to the market pull and vested interests of healthcare systems. Early manufacturing choices are essential before products get locked into a competitively disadvantaged regulatory and commercial route to market.

⧫ Market Access: extensive expertise and project work focused on market access, pricing and risk sharing for ACT products, details of which are still playing out/evolving in real-time

⧫ Expert Network: extensive network of relationships with ACT-focused KOLs forged over many years through our primary research, collaborations with LLS and other advocacy/investor groups, and our annual Cancer Progress meeting in NYC (31st Annual Meeting in 2020 featuring a ½ day session dedicated solely to ACT)

⧫ Platform Adjacencies: ability to field opportunities in relevant therapy areas (e.g. AIID, monogenetic diseases)

⧫ Results-Oriented: as exemplified by case studies that follow, our project work produces not only actionable deliverables but has often helped trigger key value inflection events for clients

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4ACT/Oncology Practice Capabilities © Cello Health BioConsulting 2019

Adoptive Cell TherapyStrategic Insight

Evaluate Ltd.; CHBC analysis; CPIs: Checkpoint Inhibitors, ACTs: Adoptive Cell Therapies

ACT Sector Driven to Expand Beyond First Approvals With Improved Options in B-Cell Malignancies, Penetrance Into Additional Heme and Solid Tumors, and ‘One-Pot’ Solutions

Penetration into solids considered

mission critical for ACT sector

to succeed

ACTs lagging CPIs in early IO boom; ACT upside contingent on broader utility

via technical innovation and

strategic alignment

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5ACT/Oncology Practice Capabilities © Cello Health BioConsulting 2019

Adoptive Cell TherapyStrategic Insight

Strategy Will be Critical to Achieving Optimal Positioning, Value Inflection, and Partnering/Commercial Success in Dynamic ACT Field

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6ACT/Oncology Practice Capabilities © Cello Health BioConsulting 2019

Adoptive Cell TherapyExperience & Expertise

CHBC’s ACT Group Leverages Insights & Resources from Our Two Most Successful Practices

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7ACT/Oncology Practice Capabilities © Cello Health BioConsulting 2019

Public Preclinical Biotech With Differentiated ACT Platform in Need of Strategic Focus; Assessment/Prioritization of Putative Targets, Indications, and Enabling Tech

Adoptive Cell TherapyRecent Case Study #1

The Client

• Well-financed public Biotech company with a differentiated preclinical cell-based platform capable of generating homogenous off-the-shelf engineered adoptive cell therapies (e.g. CART-, CAR-NK, etc.) that could be positioned across a range of cancer types and settings

Business Challenge

• Platform technology positioned as “something for everyone”, lacking clear strategic or messaging direction

• Lacking internal consensus around optimal positioning (targets, indications) and product profiles (cell types, enabling edits) informed by differentiating platform features, unmet need and the competitive landscape

Our Approach

• Developed a robust approach to prioritize among tumor targets based on scientific, clinical & commercial criteria

• Tailored user-friendly Excel tools to generate rank-ordered lists of targets and enabling edits to inform TPPs

• Identified develop best-, last-in-class ACT opportunities capable of addressing early-mover shortcomings

Valued Outcome

• Provided client with supporting insight, rationale, and resources to inform internal decision-making and build consensus among key stakeholders (investors, partners)

• Company market cap has since more than doubled, and client has advanced multiple candidates into the clinic

• CHBC has remained a trusted partner for ongoing work

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8ACT/Oncology Practice Capabilities © Cello Health BioConsulting 2019

Venture-Backed Biotech With ACT-Enabling Cell Engineering Modalities Struggling to Align on Strategic Focus and Capture Unrealized Value from a Differentiated Platform

Adoptive Cell TherapyRecent Case Study #2

The Client

• Venture-backed Biotech that has assembled a core suite of ACT-enabling technologies positioned to inflect value

• Has secured several Pharma partnerships, advanced two ACT programs into the clinic, and multiple additional preclinical-stage assets generated by a prolific discovery engine

Business Challenge

• Despite fundraising, partnering, and R&D success, client had struggled to inflect value in line with its competitive peers due to a lack of strategic focus needed to support a clear and compelling positioning and messaging strategy that is informed not only by the platform tech but within context of the rapidly evolving ACT technology landscape

Our Approach

• Conducted comprehensive landscape assessment which provides necessary context for strategic decision-making

• In parallel, CHBC conducted an assessment of the client’s oncology platform and product portfolio, leveraging our extensive oncology knowledgebase, secondary research, KOL discussions, and internal stakeholder feedback

Valued Outcome

• Findings helped to build internal consensus and inform positioning, partnering, and messaging strategy

• Client secured a sizeable private round led by strategic investor ascribing value to both the lead program and broader utility/applications underscored by strategic alignment of platform technology with market gaps

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9ACT/Oncology Practice Capabilities © Cello Health BioConsulting 2019

Top Multinational Pharma Interested in Assessing/Onboarding ACT and/or Other IO Modalities; IO Landscape, Portfolio Gap Analysis, and BD Strategy Assessment

Adoptive Cell TherapyRecent Case Study #3

The Client

• Top 10 multinational Biopharma, historically a leading oncology player with a strong cash position and biologics-based clinical-regulatory and commercial infrastructure

• Looking to understand how/where to be positioned within the rapidly evolving competitive IO landscape

Business Challenge

• Client missed the first IO wave (anti-PD1 mAbs), now seeking to ensure their current/future R&D and BD strategy is optimally aligned with emerging competitive and commercial opportunities in solid tumors

• Lacking consensus needed to address looming oncology portfolio gap (early/mid-stage clinical candidates)

Our Approach

• Provided a comprehensive and multifactorial assessment of the evolving scientific, clinical, competitive, regulatory, reimbursement, and commercial landscape

• Individual discussions and multiple on-site workshops

• Deep-dive on technical details and strategic implications for emerging modalities including ACT, bsAbs, and OVs

Valued Outcome

• Provide recommendations on prioritization of in-house assets, ongoing partnerships, and external opportunities within attractive and feasible settings and platforms

• Recommendations supported by extensive competitive and scientific analyses, CHBC expertise, and discussions with key client-company stakeholders

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10ACT/Oncology Practice Capabilities © Cello Health BioConsulting 2019

TAA Database at a glance: characterization of ~500 clinical-stage WW antigen-targeted ACT and antibody (mAb, bsAb, ADC) assets characterized by development phase, region, modality, cell type (for ACT), antigen target (intra- and extracellular), and lead indication(s); continually maintained and updated by analysts and automation; user-friendly interface for push-button filtering; output includes frequency histogram and tables showing targets with desired attributes set according to ratings across metrics for validation, clinical performance, biological properties, and partnering/commercial attractiveness.

Adoptive Cell TherapyCHBC’s Proprietary Tumor Associated Antigen (TAA) Database

*Sources include syndicated pipeline databases (Clarivate, Adis Insight), informatics databases (TCGA/GTEx, GEPIA2, ProteinAtlas, COSMIC, etc.), and an extensive review of peer-reviewed literature

What is CHBC’s TAA Database? How can it help you?

⧫ CHBC maintains an in-house curated Excel database to address client demand for a rapid, robust means to track TAA candidates.

⧫ A common question among clients is how best to identify and rank-order among potential antigen targets best-suited for their ACT platform within the rapidly evolving competitive landscape.

⧫ Output from this tool combined with CHBC’s strategic insight supports our clients’ efforts to quickly identify, cross-compare, and rank-order among potential therapeutic targets supported by a robust and intuitive methodology needed for internal decision-making and stakeholder alignment.

Detailed, robustly-supported & QC’ed input

Intuitive, tailored interface

Actionable output (e.g. target prioritization)

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11ACT/Oncology Practice Capabilities © Cello Health BioConsulting 2019

ACT Technology Database at a glance: characterization of ~700 adoptive cell therapy programs (preclin to marketed; US and ex-US) categorized by Phase, region, presence/absence of edits, cell type, cell source (auto/allo), antigen target (if relevant), and a host of parameters (switches, payloads, dual targeting, editing vehicle, manufacturing, etc.); continually maintained and updated by analysts and automation; user-friendly interface to facilitate push-button filtering for rapid identification of programs in desired categories

Adoptive Cell TherapyCHBC’s Proprietary ACT Technology Database

*Sources include syndicated pipeline databases (Clarivate, Adis Insight), informatics databases (TCGA/GTEx, GEPIA2, ProteinAtlas, COSMIC, etc.), and an extensive review of peer-reviewed literature

What is CHBC’s ACT Tech Database? How can it help you?

⧫ CHBC maintains an in-house curated Excel database to track the diversity of ACT technologies currently in preclinical and clinical development both in the US/EU and RoW including China.

⧫ Clients commonly ask about the breadth of companies and assets that could be regarded as in-kind or otherwise competitive with their own programs.

⧫ Output from this tool includes rapid characterization of relevant programs and platforms (which could alternatively be regarded as competitive or complementary targets for onboarding), along with summative charts and tables which identify high-level emerging (often non-intuitive) trends in the ACT landscape.

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Thank You!: Joel Sandler, PhD : [email protected]

: 917-392-1149