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CHBC ADVANCED THERAPEUTICS CAPABILITIES:Rare Diseases, Regenerative Medicine and Oncology

October 2019

2Capabilities deck© Cello Health BioConsulting 2019

Cello Health BioConsulting Overview

Leading business development strategy consulting firm, assisting clients in the pharmaceutical, biotech and healthcare investment industries for more than 25 years

Three core lines of business, each focused on helping companies build and strengthen development-stage assets: compounds, portfolios and platforms


Cello Health BioConsulting Core Services

▪ Assess commercial value in markets of interest

▪ Evaluate mechanistic approach and scientific rationale

▪ Position and differentiation from evolving SoC

▪ Define target patient populations, clinical endpoints and target product profiles

▪ Determine payer perspective

▪ Establish criteria for identifying programs that fit with strategy

▪ Identify assets and/or companies that best match criteria

▪ Work with our client to prioritize opportunities

▪ Provide deeper analysis of priority targets

▪ Identify value inflection points to optimize partnering strategy

Opportunity Assessments

Opportunity Search & Evaluation

▪ Analyze and prioritize pipeline portfolio

▪ Identify and assess novel indications for development

▪ Prioritize and sequence potential indications

▪ Define value inflection strategy for clinical candidates

▪ Advise on optimal time and stage of development for partnering

Portfolio and Platform Strategy


Cello Health BioConsultingDeep Knowledge Across Therapeutic Areas

• Cello Health BioConsulting has a deep knowledge base within and across all therapeutic categories.

• The focus of our work closely mirrors the therapeutic area focus of today’s biopharmaceutical pipeline.

• Cello Health BioConsulting’s research is conducted by its trained consultants, all of whom have PhD or MS degrees and/or significant relevant experience in pharma and the targeted scientific areas.

• Our consultants possess a combination of commercial, scientific, and clinical knowledge that enable comprehensive analyses and actionable recommendations.

OncologyCardiovascular

& Metabolic DiseaseCNS

Autoimmune &Inflammatory

Respiratory Ophthalmology Orphan Diseases Gastro-Intestinal

And other emerging areas of interest such as NASH & fibrosis

DermatologyGene & Cell Therapy PlatformTechnologies

InfectiousDisease


Cello Health BioConsultingInsight Across the Clinical Development Value Chain, with Particular Focus on Early-Stage Assets

Cello Health BioConsulting’s combination of scientific sophistication and strategic/commercial insight provides a competitive advantage for its core offerings across the clinical development value chain, with particular emphasis on assets in early phases of development.

Preclinical Clinical Launch

Opportunity Assessments, Indication Prioritization, and Strategy

Identify Value Inflection Points, Forecasting and Valuation

Payer Research, Pricing Analysis and Optimization

Opportunity Search and Evaluation

Scientific Focus: Translation of mechanism to potential clinical value proposition

Clinical and Commercial Experience:Guidance on strategies for differentiation


Insight from Key Stakeholders

• Cello Health BioConsulting has developed an extensive network of relationships with key stakeholders.

• Our consultants conduct in-depth collegial discussions with physicians and payers to understand their perspective on the value of development-stage assets as well as product portfolios and platforms.

• We also have the ability to do patient research to gain key insights into patient needs and willingness to pay for new products.

PayerPriceFormulary

PatientWillingness to Pay

PhysicianTreatment Preference


CHBC Brings Unique Perspective to Defining Value Proposition

Cello Health BioConsulting believes that the key to driving value inflection for early stage compounds is to go beyond mechanistic proof-of-concept to demonstrate clear potential for clinical differentiation and commercial value.

To capture this sentiment, CHBC has coined the phrase “proof-of-relevance.”

PoR =Proofof

Relevance

To survive, biotech companies must embark on clinical programs that go beyond scientific proof-of-concept (demonstrating that the science is applicable to the disease) to prove that its approach provides a clinically and commercially relevant value proposition that makes the program attractive to all stakeholders

• In its opportunity assessments, Cello Health BioConsulting provides unique perspective on clinical and commercial attributes that can (and must) be addressed in early stage clinical trials in order to demonstrate PoR.

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Commercial assessments of development-stage assets are the nucleus of Cello Health BioConsulting’s continuously growing experience, insight and knowledge, across therapeutic areas.

• The methodology includes a combination of primary and secondary research whereby a target product profile is created, refined, and evaluated within the context of the product’s unique market and competitive environments.

• The output typically includes insight into the product’s market potential (in the form of high-level analysis or full revenue/NPV models) as well as recommendations for value optimization at various points in development.

Commercial Assessment Framework

REFINED TARGET PRODUCT PROFILE

Client’s internal view of compound

Preliminary TPP

Treatment, Epidemiology, Pipeline, Market Overviews, Benchmarks

Secondary Research

KOL, Front-line physicians, payers

Primary Research



Cello Health BioConsulting employs the following information resources when conducting opportunity assessments for clients:

Provides the data to support the analysis and primary research

Sources include, but are not limited to:

• company pipelines• prescription & pipeline databases• analyst reports & SEC filings• medical info sites• scientific literature and conference

proceedings• industry publications• information gathered from publicly

available resources

One-to-one in-depth telephone discussions with the following across the US & EU

• Key Opinion Leaders (KOLs)• Front-line physicians• Payers (Medical Directors, Pharmacy

Directors, Hospital Pharmacists)

Prior to these in-depth discussions, CHBC drafts a discussion guide(s) and a target product profile which are refined with the client to meet the project objectives

Web-based surveys with US and EU physicians

Provides insight from a larger sample (typical projects range from 50-200 respondents)

Survey questionnaire is drafted and viewed with the client before launch

Results from the surveys are analyzed and presented graphically along with key takeaways from the physician research

Secondary Research

WebSurveys

TelephoneInterviews


Primary and Secondary Research

Through the combination of primary and secondary research, Cello Health BioConsulting’s opportunity assessments evaluate four key areas:

Commercial

Unmet Need

• Epidemiology, patient segments, disease and treatment landscape – current and future

• Level of clinical or patient need

• Key remaining unmet needs

• Value proposition within current and future treatment algorithms

• Pipeline quantity, quality, and novelty

• Market access and pricing environment and challenges

Scientific

• Scientific basis/validation for the mechanism

• Degree of differentiation and ability to address unmet needs

• Potential limitations associated with the product’s profile

Development

• Clinical development path and critical endpoints for success

• Expected regulatory challenges or roadblocks

• Anticipated clinical development risk

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Additional Components of Opportunity Assessments

Opportunity assessments also frequently include the payer and financial components below. We view these as additional core competencies of our business.

Cost-effectivenessModels

Payer Research

Forecasting and Valuation

▪ Determine current cost of care and quality of life metrics

▪ Develop a value-based pricing model that takes into consideration a willingness to pay threshold, improvements to QoL and costs savings provided

▪ Identify relevant pricing analogs

▪ Assess market access, reimbursement and pricing environment

▪ Determine potential coverage and formulary status/ restrictions

▪ Evaluate what additional clinical/ pharmacoeconomic data is needed

▪ Pressure-test price and establish pricing benchmarks

▪ Create dynamic and adaptive patient-based and prescription-based forecasts

▪ Benchmark valuations using comparable deal analysis

▪ Produce risk-adjusted NPV models of companies, products, and deals

▪ Perform sensitivity analysis of inputs to determine key value drivers


Cost-Effectiveness Models

• For price testing/optimization, Cello Health BioConsulting uses a comprehensive approach to arrive at a price that is derived from value-based pricing models, health economic/payer guidance, and relevant pricing analogs.

• CHBC develops dynamic value-based pricing models based on the NICE model. Our consultants then pressure-test the VBP during one-on-one discussions with key stakeholders in the market access space and triangulate it with pricing benchmarks of relevant analogs.

• We believe this multi-pronged approach ensures that the price attained is reasonable and justifiable.

PayerResearch

Value-BasedPrice

PricingAnalogs


Payer Research

PatientSegmentation

Coverage & Formulary

Status

Restrictions on Use

Pricing

Trial Design & Data Required

♦ Desired improvements in outcomes

♦ Key drug characteristics payers willing to pay for

♦ Current unmet needs

♦ Plan type by segment♦ Costs and unmet needs by

segment♦ Reimbursement by segment

♦ Coverage determinations based on pharmacy or medical benefit

♦ Expected copays

♦ Endpoints to show value♦ Patient criteria to show the

greatest value♦ Level of improvement to justify

price

♦ Restrictions of currently available drugs

♦ Anticipated future restrictions♦ Pricing thresholds for

stricter/looser restrictions

♦ Current cost of care in the target patient segments

♦ Target price point♦ Floor and ceiling price points

Cello Health BioConsulting’s payer research evaluates key questions in the following six areas:

Value Inflection

Points


Forecasting and Valuation

• Financial forecast modeling is an essential component of Cello Health BioConsulting’s valuation capabilities.

• We build revenue forecasts (patient- or prescription-based) in a user-friendly Excel format to allow our clients to see in real-time how changes and sensitivities around distinct variables impact the forecast.

• In addition, we provide benchmarking valuation of individual assets, platforms and company portfolios as well as risk-adjusted NPVs in Excel with sensitivity analysis surrounding the key inputs.

Pricing• Appropriate/acceptable price based on current & future

market

• Impact of market access landscape on pricing potential

• Potential level of rebating/discounts

Penetration• Anticipated use based on product’s value proposition

• Potential adoption & uptake

• Appropriate analogs

Competition• Current and future competitive landscape

• Impact of new entrants/generics/ biosimilars

• Impact of competition on peak revenue potential

Patient Population & Treatment• Epidemiology

• Key patient segments based on treatment practices

• Addressable patient segments

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Opportunity Search & Evaluation


Search & Evaluation of Opportunities

• Cello Health BioConsulting assists clients by helping to define and execute on strategic goals to either establish a new presence or grow an existing presence in specific therapeutic and specialty areas.

• Cello Health BioConsulting has more than 25 years of experience that result in an incomparable ability to evaluate and prioritize markets and potential opportunities.


1 CRITERIA

2SCREEN

3 EVALUATE

4RATE & PRIORITIZE

5 OVERVIEWS/BUSINESS CASES FOR PRIORITIZED OPPORTUNITIES


• Cello Health BioConsulting has established a comprehensive & transparent process for identification and qualification of actionable partnering and in-licensing opportunities.

• We evaluate potential opportunities with our broad understanding of the current state of scientific, development and commercial environments within specific diseases.

• Cello Health BioConsulting provides valuable insights and assists in prioritizing of the most compelling assets available to meet the client’s objectives.

Develop screening criteria and generate initial list of potential opportunity candidates (assets or companies)

Screen for assets and companies against inclusion criteria

Compile data, review and evaluate opportunities

Rating & tiering with rationale



We take a comprehensive, multi-pronged approach to evaluating potential licensing/acquisition candidates.

• Scientific Evaluation: understand the base mechanistic approach, and potential application to disease areas, as well as the current level of validation

• Commercial Evaluation: provide an assessment of the market need and growth potential for the intended indication; compound’s ability to address unmet need, impact on the treatment paradigm, and competitive environment challenges

• Clinical/Regulatory Evaluation: identify the clinical development risk for the product; likelihood of success in late-stage clinical trials and/or with FDA approval

Scientific

Clinical & Regulatory

CommercialOpportunity

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• Cello Health BioConsulting works with many emerging biotech companies to prioritize pipeline portfolios and define pathways to value inflection

• Typically, these programs are at pre-clinical to early clinical development (Phase I-Phase 2a) so opportunity remains to shape the development program to maximize potential for clinical and commercial success

• Output often includes a definition of therapeutic strategy and best transaction model going forward

Portfolio Identification & Prioritization

Path to value inflection is completely clear and facile

Path to value inflection is moderately clear and facile

Path to value inflection is uncertain and/or difficult

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Potential to Overcome Risk & Challenges

Op

po

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yA

ttra

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Indication C

Indication A

Indication E

Indication D

Indication F

Indication G

Indication B

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Therapeutic Area KnowledgeExpertise & ExperienceSelected Case Studies

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Rare Disorders and Advanced TherapeuticsExpertise & ExperienceSelected Case Studies


Cello Health BioConsulting Rare Disease Experience

THE TYPE OF WORK WE DELIVER

• Asset identification and evaluation • Clinical Study support • Forecasting and valuation • Landscape analysis and gap analysis • Digital landscape assessment• Launch strategy development • Opportunity assessment• Organizational capability assessment • Partnering and BD support

>800 >80

1025 RARE DISEASE PROJECTS IN THE LAST 4 YEARS

RARE DISEASES EXPLORED

>160IN DEPTH RARE DISEASE COVERAGE SINCE 2011

RARE DISEASES COVERED IN LAST 12 MONTHS

>40MARKETS COVERED

• Patient journey and patient flow analysis • Physician, payer, patient, caregiver insight mapping• Portfolio and Platform strategy • Publication planning• Social media monitoring• Scenario development • Scientific platform development • Strategic and competitive planning • Thought leader identification and engagement


Rare Oncology16%

Rare Non-Onc16%

Large Oncology23%

Large Non-Onc45%

Oncology vs. Non-Onc Project Count by Indication Size

(2013 to 2017e)

Rare Disorders Represent Nearly 1/3 of CHBC Projects:Non-Malignant Genetic Diseases and Rare Cancers are Common Subjects

Rare Disease

32%

Large Population68%

5 year Project Count by Indication Size(2013 to 2017e)

CHBC Internal Project Data


CHBC’s Rare Disease Experience Crosses All Therapeutic Areas, Involving Patient Segments with a Wide Degree of Disease Burden

Dermatology2%

Pulmonology3% Gastrointestinal

2%

Musculoskeletal3%

Ophthalmology3%

Hematology4%

Cardiometabolic6%

Immunology7%

Neurology8%

Metabolism 9%

Oncology53%

Number of Rare Disease Projects by Therapeutic Area⧫ Each afflict a relatively small population, but combined

make up one of the highest value categories

⧫ Some are idiopathic or acquired disorders, but many are inherited, monogenetic diseases

⧫ Have a wide degree of severity, age of onset and clinical presentation

⧫ Represent attractive opportunities for clinical development as a result of their:

• well understood etiologies & pathologies

• straightforward paths of intervention (e.g. enzyme replacement, substrate reduction, mRNA knockdown)

• accelerated development timelines

• premium pricing models

CHBC Internal Project Data

• Half of CHBC’s projects focus on orphan oncology while the other half on non-malignant disorders scattered across all therapy areas:


CHBC Assessments of Therapies for Rare Disorders Cross All Platforms

⧫ Assessments in rare disorders typically cross all technologies platforms from traditional small molecule and biologics to complex therapeutics including nucleic acid based oligonucleotide and gene/vector based therapies, viral therapies, cell therapies and drug device combinations

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Cello Health’s Rare Disorders and Genetic Disease Database


Cello Health Genetic Disease Database

⧫ Cello Health has created a database of genetic diseases sourced from OrphaNet, National Institutes of Health Genetics Health Reference (NIH GHR), National Organization for Rare Disorders (NORD), Online Mendelian Inheritance in Man (OMIM)

⧫ CHBC is able to efficiently filter the database based on multiple categories characterizing genetic diseases. This process allows CHBC to prioritize and identify genetic diseases to enhance the positioning of potential new gene therapies in the competitive landscape.

Orphanet OMIM

NORD NIH GHR

Prioritized Genetic Diseases

Prevalence

Mutation Type

Disease Severity

Age of Onset

Organs Affected

Competitive Pipeline

CHBC Genetic/Rare

Disease Database


Information Provided By CHBC Rare Disease Database

⧫ CHBC utilizes the secondary the sources on the right to characterize each disease:

• therapeutic area

• description/disease manifestation

• synonyms

• segmentation

• genetic transmission

• gene mutated/cause

• gene size

• protein mutated or impacted

• gene product locations (tissue, intra/extracellular)

• gene function in normal vs. mutated form

• type of mutation (loss/gain of function, missense, etc.)

• diversity of mutations (# of different mutations with key mutations identified, % patients with mutations)

• epidemiology: prevalence, incidence

• age of onset

• SoC, severity / unmet need

• Competitive pipeline and financing

Selected Data Sources• NCBI OMIM ClinVar

• HGMD

• GeneTests / Gene Reviews

• National Organization for Rare Disorders [NORD]

• Orphanet

• National Institutes of Health Genetics Health Reference [NIH GHR]

• Clarivate Analytics Cortellis

• ADIS R&D Insight

• EvaluatePharma

• Medical info sites (Medscape, UpToDate)

• Company websites and press releases

• Analyst reports

• Clinical trial databases

• Cello Health’s Knowledgebase


Example Screenshot: Cello Health Genetic Disease Database

⧫ Below is a representative screenshot of the CHBC genetic disease databased filtered on: monogenic, loss of function mutation type, and liver affecting.

⧫ Final output can be further tailored to include additional criteria of interest not already captured.

Source: CHBC genetic disease database

Monogenic/Polygenic Mutation Type Organs Affected


Example: Categorization of Genetic Diseases by Therapeutic Area and ADVTX Platform Approach


Example Evaluation Prioritization Criteria and ProcessSummary of Relative Rankings For Opportunities Identified as Attractive

Criteria High Medium Low

Epi/Market Size

Larger epidemiology and/or Market size: (>2K candidates for gene therapy) AND (Current or e2022 WW market >$500M

Larger US epi or Market size:(>2K candidates for gene therapy) OR(Current or e2022 WW market >$500M

Low US epi and Market size:(<2K candidates for gene therapy) AND (Current & e2022 WW market <$500M

Unmet Need

High unmet need:No effective and safe therapies for management of indication

Moderate unmet need:Current SoC is somewhat effective

Low unmet need:Current SoC is very effective and safe for management of indication

CompetitionLimited number of gene/cell therapies in clinical or preclinical development:(<5 programs)

Moderate number of gene/cell therapies in clinical or preclinical development:(5-15 programs)

High number of gene/cell therapies in clinical or preclinical development:(>12 programs)

Biologic Validation

HSC Transplant is SOC for some or all patients affected by indication

HSC Transplant rarely used in clinic, but there are BMT case reports and/or clinical development of ex vivo therapies requiring conditioning regimen

No use of HSC Transplant or interest in developing ex vivo products requiring conditioning regimen

ORF Size (bp)

Small ORF size of affected gene:(<1,000 bp)

Moderate ORF size of affected gene:(1,000-6,000 bp)

Large ORF size of affected gene OR multiple genes affected:(>6,000 bp)

Time to POC Endpoint(Months)

Short post-treatment follow up time, per patient, for readout of efficacy endpoint:(<3 months)

Moderate post-treatment follow up time, per patient, for readout of efficacy endpoint:(3-6 months)

Long post-treatment follow up time, per patient, for readout of efficacy endpoint:(>6 months)

Time to Approval(Yr.)

Shorter required pivotal trial:(0-3 years)

Moderate required pivotal trial:(3-5 years)

Longer required pivotal trial:(>5 years)

Approval Trial Size(Pat. #)

Smaller required # of patients:(0-25 patients)

Moderate required # of patients:(25-75 patients)

Larger required # of patients:(>75 patients)

Starting Rare Disease List~1,500 Groups of Rare disorders

Review indications, Apply selection criteria, Rank order

Select Indications for Expert Opinion

Case Assessments• Technological Fit• Clinical Need• Commercial Opp.• Partnerability


Monogenetic Disorders Have Diverse Etiology Depending on the Location of Expression and Function of a Mutated Gene Product

Monogenetic Neuromuscular Disorders are caused by inherited and sporadic mutations in genes involved in motor unit structural and electrical function, and also metabolic and biochemical physiology providing energy and support for neuromuscular health and functioning.


Example Output: Graphic of Clinical Validation vs. Unmet Need


Example Output: Rare Disorder Profile


Advanced Therapeutics

Advanced TherapeuticsRecent Case Study: Analysis of Gene Therapy Landscape

The Client

• US-based biotech with single asset gene therapy in late-stage development for treatment of debilitating monogenetic disorder

• planning follow-on development with same agent in analogous disease subtypes

Business Challenge

• seeking to broaden its gene therapy portfolio by bringing additional assets into its pipeline

• considering acquisition of new gene therapy technologies within the context of existing domain expertise

Our Approach

• exhaustive analysis of gene therapy landscape including overview of gene transfer systems and various enabling technologies

• interaction with key personnel at vector cores, research institutes and biotechs in order to assess licensing or M&A opportunity

Valued Outcome

• comprehensive searchable database of gene therapy programs in academia and industry

• facilitate discussion on gene transfer systems, platforms and most tractable indications with client and relevant stakeholders



Advanced TherapeuticsRecent Case Study: Strategic Development of Gene-Editing Platform Technology in Rare Disease Space

The Client

• US-based biotech with clinical stage best-in-class gene editing platform in development for various oncology settings

• looking to leverage the broad applicability of proprietary gene editing technologies to a broad range of human diseases beyond oncology

Business Challenge

• develop rational strategic plan for pursuing development of platform technology in areas outside of core oncology expertise

• prioritize therapeutic targets with genetic driver among the multitude of opportunities for gene editing within the rare disease space

Our Approach

• develop methodology for filtering rare genetic disease landscape based on selection criteria amenable to client platform technology

• map clinical stage gene therapy programs for genetic diseases with assessment of clinical value proposition

Valued Outcome

• pursuit of ex vivo cell-based development found to represent most attractive near-term opportunity in light of current regulatory and potential in-vivo delivery issues

• detailed profiles of 20 prioritized opportunities for client’s platform gene editing technologies within the rare disease space



Advanced TherapeuticsRecent Case Study: M&A guidance to support pipeline expansion

The Client

• clinical stage US biopharma with large market cap looking to implement portfolio expansion strategy

• client has single clinical stage asset & several discovery programs, aims to build pipeline that delivers new products to new rare diseases in rapid succession

Business Challenge

• identify M&A opportunities within rare disease space that align with client’s core capabilities, expertise and strategic focus

• filter list of selected opportunities that may be poised for acquisition – focus in ophthalmology disease

Our Approach

• profile leading and novel approaches for GT assets being developed for rare diseases with ophthalmologic phenotype

• solicit expert opinion on the potential of specific gene therapy technologies to address the unmet needs of patients with ophthalmologic disease

Valued Outcome

• Framework for vetting novel gene therapy opportunities within the rare sensory disorder space

• list of prioritized assets with recommended development plans including clinical endpoints and proposed strategic fit within client’s expanding portfolio

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Oncology and Adoptive Cell Therapy PracticeExpertise & ExperienceSelected Case Studies


OncologyExperience & Expertise

Unique Experience and Insights

• leading area of research at CHBC

• we have completed hundreds of commercial assessments and forecasts of novel MOAs, as well as opportunity search projects, across all MOAs and tumors

• our immuno-oncology experience goes back over a decade, long before IO became in vogue

Sample Project Work

• recent evaluations of: checkpoint inhibitors and costimulatory agonists, CARTs, cancer vaccines and oncolytic viruses, as well as agents targeting key Hanahan and Weinberg cancer hallmarks such as apoptosis, angiogenesis, cell cycle, epigenetics, and classic oncogenes

• looked extensively at next-generation platform biologics including adoptive cell therapies, bispecifics, ADC, and non-antibody scaffolds, as well as oligonucleotide therapies like RNA or DNA-based approaches

Oncology

Practice Leaders:Jeff Bockman, PhD, Executive Vice PresidentMike Rice, MBA, PrincipalJames Lee, PhD, Associate PrincipalJoel Sandler, PhD, Associate Principal


Oncology

OncologyRecent Case Study: Portfolio Strategy & Opportunity Assessment to Support BD Decisions and Clinical Development

The Client

• venture-backed pre-commercial biotech

• novel MOA with two potentially distinct paths to market:

• direct anticancer strategy

• one based on the agent’s immunomodulatory anticancer effects

Business Challenge

• PC data suggest the small molecule can provide both inhibition of a common cell signaling pathway terminal kinase and may have a role in regulating T cells and their recruitment for effector function

• identification of optimal indications based on PoC, timing to value inflection, and partnerability

Our Approach

• systematic approach to prioritizing indications based on clinical and commercial risks and attractiveness

• opportunity assessment on top indications to determine best positioning (including biomarker-driven strategy) to inform target product profile development

• 10-year market forecast for direct comparison between top indications.

Valued Outcome

• provided client ability to strategize portfolio by triaging indications and identified new indications that were not previously championed internally

• provided optimal registrational settings and clinical development strategies to inform partnering discussions which led to formation of a clinical collaboration with a large biopharmaceutical entity


Oncology

OncologyRecent Case Study: US Opportunity Assessment to Inform Client to US Commercialization Strategy for Target Asset

The Client

• Chinese biotech with an internal drug discovery platform

• looking to expand their portfolio within China and US

• interested in investment & licensing opportunities in China (and potentially globally) to further establish their footprint as a global player in oncology

Business Challenge

• client looked to invest or in-license a novel MOA asset that can target certain virally-induced cancers and was not familiar with what indication opportunities existed in the US and additional western areas for novel MOA

• sought help with performing due diligence on the asset in the context of the US opportunity

Our Approach

• worked in tandem with client company and the potential partner company for the novel MOA asset

• performed independent assessment of indication opportunities via secondary research and primary research which helped to inform the risks and opportunities for asset in the US

Valued Outcome

• vetted MOA and clearly identified market size for various indications and potential clinical development strategies in the US that could synergize with their China development

• outlined context of development in China vs. US, providing strong guidance for deal sheet & BD strategy


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


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


Adoptive Cell TherapyStrategic Insight

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


Adoptive Cell TherapyExperience & Expertise

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


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 remained a trusted partner for ongoing projects


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


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


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


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 helping to build internal consensus and inform positioning, partnering, and messaging strategy

• Client provided with strategic recommendations for how to position itself for value inflection and future success


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

⧫ 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.

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

Detailed, robustly-supported & QC’ed input

Intuitive, tailored interface

Actionable output (e.g. target prioritization)


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

⧫ 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.

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


Cello Health BioConsulting Principals

Senior Management

Joel S. Sandler, PhD Associate Principal [email protected]

Ed Saltzman Executive [email protected]

Ginger S. Johnson, PhD Chief Executive [email protected]

Jeffrey M. Bockman, PhDEVP, Oncology Practice [email protected]

Michael C. Rice, MS, MBA [email protected]

Danielle M. Marra, MS, MBA Associate Principal [email protected]

Janet F. [email protected]

David J. Lomb, PhDAssociate [email protected]

James T. Lee, PhD Associate [email protected]

Principals & Associate Principals

Senior Consultants

Brent W. Osborne, PhD Senior [email protected]

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