cello health bioconsulting · • scientific literature and conference proceedings • industry...

43
CELLO HEALTH BIOCONSULTING Capabilities Presentation 2020

Upload: others

Post on 12-Aug-2020

1 views

Category:

Documents


0 download

TRANSCRIPT

Page 1: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

CELLO HEALTH BIOCONSULTINGCapabilities Presentation

2020

Page 2: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

2Capabilities deck© Cello Health BioConsulting 2020

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

Page 3: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

3Capabilities deck© Cello Health BioConsulting 2020

Cello Health BioConsulting is a Part of Cello HealthA worldwide team of more than 400 based in the US and UK

A UNIQUE FUSION OF EXPERTISE TO SOLVE PROBLEMS, REDUCE RISK AND UNLOCK THE FULL POTENTIAL OF ORGANIZATIONS, ASSETS AND BRANDS

London

Farnham

Chicago

San Francisco

Philadelphia

New York & New Jersey

Boston

Offices in London, Farnham, New York, New Jersey, San Francisco, Chicago, Philadelphia and Boston

INSIGHT & ANALYTICS

STRATEGY & COMMERCIAL

SCIENCE & EVIDENCEMarket Researchers, Master Practitioners,Digital Specialists, Innovation leads, Field and Compliance

70+ Consultants with a blend of industry experts and consulting specialists

200

Communication Specialists, Scientists & Creatives including Client Service, Project Managers and Medical Writers

60+

140

PhDs and MDs providing a depth of understanding into cutting edge science

Page 4: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

4Capabilities deck© Cello Health BioConsulting 2020

Cello Health BioConsulting Areas of Expertise and CapabilitiesCore Services

Opportunity Assessments

Portfolio and Platform Strategy

Opportunity Search & Evaluation

Page 5: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

5Capabilities deck© Cello Health BioConsulting 2020

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

Page 6: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

6Capabilities deck© Cello Health BioConsulting 2020

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

Page 7: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

7Capabilities deck© Cello Health BioConsulting 2020

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

Page 8: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

8Capabilities deck© Cello Health BioConsulting 2020

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

Page 9: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

9Capabilities deck© Cello Health BioConsulting 2020

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.

Page 10: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

10

Opportunity Assessments

Page 11: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

11Capabilities deck© Cello Health BioConsulting 2020

Opportunity Assessments

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

Page 12: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

12Capabilities deck© Cello Health BioConsulting 2020

Opportunity Assessments

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

Page 13: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

13Capabilities deck© Cello Health BioConsulting 2020

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

1

3

2

4

Page 14: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

14Capabilities deck© Cello Health BioConsulting 2020

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

Page 15: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

15Capabilities deck© Cello Health BioConsulting 2020

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

Page 16: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

16Capabilities deck© Cello Health BioConsulting 2020

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

Page 17: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

17Capabilities deck© Cello Health BioConsulting 2020

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

Page 18: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

18

Opportunity Search & Evaluation

Page 19: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

19Capabilities deck© Cello Health BioConsulting 2020

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.

Page 20: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

20Capabilities deck© Cello Health BioConsulting 2020

1 CRITERIA

2SCREEN

3 EVALUATE

4RATE & PRIORITIZE

5 OVERVIEWS/BUSINESS CASES FOR PRIORITIZED OPPORTUNITIES

Search & Evaluation of 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

Page 21: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

21Capabilities deck© Cello Health BioConsulting 2020

Search & Evaluation of Opportunities

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

Page 22: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

22

Portfolio and Platform Strategy

Page 23: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

23Capabilities deck© Cello Health BioConsulting 2020

Portfolio and Platform Strategy

• 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

0

2

4

6

8

10

0 2 4 6 8 10

Potential to Overcome Risk & Challenges

Op

po

rtu

nit

yA

ttra

ctiv

en

ess

Indication C

Indication A

Indication E

Indication D

Indication F

Indication G

Indication B

Page 24: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

24

Therapeutic Area KnowledgeExpertise & ExperienceSelected Case Studies

Page 25: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

25

OncologyExpertise & ExperienceSelected Case Studies

Page 26: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

26Capabilities deck© Cello Health BioConsulting 2020

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

Page 27: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

27Capabilities deck© Cello Health BioConsulting 2020

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

Page 28: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

28Capabilities deck© Cello Health BioConsulting 2020

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

Page 29: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

29

Advanced Therapeutics – Cell and Gene TherapyExpertise & ExperienceSelected Case Studies

Page 30: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

30Capabilities deck© Cello Health BioConsulting 2020

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

Page 31: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

31Capabilities deck© Cello Health BioConsulting 2020

Advanced Therapeutics

Advanced TherapeuticsRecent Case Study: Prioritization of Putative Targets & Indications for Differentiated but Early-Stage ACT Platform

The Client

• well-financed public company with differentiated PC, 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

• elucidate optimal positioning (targets, indications to be pursued) based on identification and consideration of key differentiating features of the platform

• focused consideration on the context of extant unmet needs and the rapidly evolving competitive landscape

Our Approach

• identified starting list of potential targets, along with scoring criteria, inputs, and supporting assumptions that reflect scientific, clinical & commercial considerations

• user-friendly tool to generate rank-ordered lists of opportunities corresponding to differential weighting scenarios

Valued Outcome

• provided client with well-supported tool to identify the most attractive targets and indications to be pursued with its ACT platform

• tool able to generate rank-ordered lists associated with alternative corporate priorities (e.g. speed to market, attractiveness to potential partners, or competitive gaps)

Page 32: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

32

Central Nervous SystemExpertise & ExperienceSelected Case Studies

Page 33: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

33Capabilities deck© Cello Health BioConsulting 2020

Central Nervous SystemExperience & Expertise

Practice Leaders:Ginger Johnson, PhD, CEOJoel Sandler, PhD, Associate Principal

Unique Experience and Insights

• experience performing in-depth evaluations of across the spectrum of CNS indications including those in neurology, psychiatry and pain

• particularly deep knowledge of novel therapies for neurodegenerative disease ranging from large indications (e.g., Alzheimer’s disease) to orphans (e.g., ALS, Huntington’s)

Central Nervous System

Sample Project Work

• assess the potential of early-stage novel mechanism agent to address the needs of specific patient subsets, clinical/regulatory risk, commercial positioning and value compared to future standard of care

• define clinical development / opportunity sequencing strategies to maximize the value of a CNS asset and/or platform

• value and prioritized a defined group of potential in-licensing candidates focused on the treatment target CNS indications

Page 34: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

34Capabilities deck© Cello Health BioConsulting 2020

Central Nervous System

Central Nervous SystemRecent Case Study: Valuation of Company Portfolio & Platform

The Client

• US-based early-stage biotech founded on an enzyme-based discovery platform that promises to address a wide range of CNS indications; lead program partnered with big pharma

• Blue chip venture backed company positioning for next round of financing

Business Challenge

• valuation of company including all programs in development and platform

• independent, informed valuation of company to support financing discussions

Our Approach

• provide comprehensive, risk-adjusted assessments, revenue forecasts and NPV estimate for entire development portfolio (~15 programs) and platform

• define target product profiles for early-stage assets to guide development prioritization plans and prioritization

Valued Outcome

• clear valuation and prioritization of potential indications, mapping points of value inflection; identified opportunities most suitable for partnering versus client development

• supported successful financing; client returned for additional work

Page 35: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

35Capabilities deck© Cello Health BioConsulting 2020

Central Nervous System

Central Nervous SystemRecent Case Study: Portfolio Assessment in Support of IPO

The Client

• EU-based biotech; early-stage assets in development for several orphan neurological disorders

• Venture-backed company preparing for initial public offering

Business Challenge

• required valuation of portfolio by informed, third-party firm with a detailed understanding of clinical value and pricing/market access issues in EU

• articulation and support of valuation in formats appropriate for a range of audiences, including management team, company board and public investors

Our Approach

• leverage internal knowledge, augmented by in-depth discussions with relevant KOLs, front-line clinicians and payers

• work with bankers to provide description and valuation of portfolio to be included in company prospectus

Valued Outcome

• oversubscribed IPO

• prioritized and sequenced indications for further development and deployment of company resources

Page 36: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

36

Autoimmune and Inflammatory DisordersExpertise & ExperienceSelected Case Studies

Page 37: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

37Capabilities deck© Cello Health BioConsulting 2020

Autoimmune and Inflammatory DisordersExperience & Expertise

Practice Leaders:David J. Lomb, PhD, PrincipalDanielle Marra, MBA, Associate Principal

Unique Experience and Insights

• extensive experience performing opportunity assessments of specific products, search projects to identify licensing and acquisition opportunities, and indication prioritization projects for products with broad mechanisms that are applicable across multiple AIID indications

• evaluations have covered most common (e.g., RA, SLE, psoriasis, etc.) and many rare AIID indications (e.g., CIDP, Guillain-Barré, pemphigus vulgaris, etc.)

Autoimmune andInflammatory Disorders

Sample Project Work

• search project to identify, evaluate and rank high-value in-licensing and acquisition candidates to support strategic initiative of bolstering AIID pipeline

• target and indication prioritization project to help a pre-commercial biotechnology company choose the best combination of target and indication for an immune checkpoint agonist AIID program

• indication prioritization project to identify additional development paths for a B cell targeting agent with a focus on rare AIID indications with a high unmet need

Page 38: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

38Capabilities deck© Cello Health BioConsulting 2020

Autoimmune and Inflammatory Disorders

Autoimmune and Inflammatory DisordersRecent Case Study: Target and Indication Prioritization

The Client

• clinical stage biotechnology company with a drug discovery platform for creating modified proteins

• core therapeutic focus on immuno-oncology (IO) with rich pipeline

Business Challenge

• investigate potential to reverse the principles of IO to target AIID and generate additional value

• prioritize selected IO targets for evaluation as potential AIID targets

• identify AIID indications where manipulating these targets might have a therapeutic benefit

Our Approach

• exhaustive literature review of each target to understand level of support of the role in AIID

• collegial interviews with key thought-leaders on each target and potential to impact AIID with IO approaches

• indication assessments to evaluate market size, unmet needs, development challenges, & competitive intensity

Valued Outcome

• prioritization of the selected targets

• recommendations for preclinical development for the 2 lead targets and identification of most attractive indications to pursue

• rich understanding of potential challenges of clinical development in this nascent area

Page 39: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

39Capabilities deck© Cello Health BioConsulting 2020

Autoimmune and Inflammatory Disorders

Autoimmune and Inflammatory DisordersRecent Case Study: Search & Evaluation

The Client

• large Japanese Pharmaceutical company heavily dependent on three marketed products for immunology, genitourinary, and oncology indications

• additional therapeutic areas of interest include cardiology, diabetes, gastrointestinal disease, infectious disease and neurology

Business Challenge

• company’s immunology franchise largely dependent on a single product which recently lost patent protection and internal pipeline lacks compelling replacements

• identify, evaluate and rank high-value development stage in-licensing and acquisition candidates to support strategic initiative of bolstering AIID pipeline

Our Approach

• conducted initial “triage” screen of ~2,300 AIID assets in Phase I to Phase III development to generate starting list of all assets of potential interest

• conducted secondary (~800 assets) and tertiary (~400 assets) screens to identify top 10 biologic and small molecule AIID programs

Valued Outcome

• curated catalog of the entire universe of assets currently in development for, or that have potential for, any AIID indication

• actionable list of nine potential partnering candidates with the greatest clinical and commercial potential based on extensive secondary research-based analysis

Page 40: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

40

Cardiovascular & MetabolicExpertise & ExperienceSelected Case Studies

Page 41: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

41Capabilities deck© Cello Health BioConsulting 2020

Cardiovascular & Metabolic

Cardiovascular & MetabolicRecent Case Study: Strategic Guidance on Development of Novel Cholesterol Lowering Drug

The Client

• small US-based pharma company with a history of successful commercial programs in the CVD space

• aiming to refocus from acute care products to broader opportunities and novel therapeutic modalities within larger CVD patient populations

Business Challenge

• strategy for entering the increasingly competitive hypercholesterolemia landscape crowded with large pharma players who have historical presence in the space and far more advanced clinical programs

• estimating market share and WW revenue projections assuming launch into various patient segments

Our Approach

• stakeholder engagement and evaluation of competitive landscape to understand how novel modality can differentiate from leading agents

• 10-year patient-based WW forecast with various extent of market penetration and rates of clinical uptake among different patient groups

Valued Outcome

• defined shortcomings of current agents and drafted roadmap towards an achievable best-in-class TPP

• advised client to initially pursue genetically defined patient populations in order to drive uptake and strengthen early value story

Page 42: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

42Capabilities deck© Cello Health BioConsulting 2020

Cardiovascular & Metabolic

Cardiovascular & MetabolicRecent Case Study: Commercial Assessment to Support Partnering Discussions

The Client

• single-asset, pre-commercial US biotechnology company

• Phase 2b product with a novel delivery formulation in development for a defined subgroup of the type 2 diabetes mellitus (T2DM) population

Business Challenge

• clear articulation of the product’s value proposition, positioning, and market opportunity, particularly to payers

• company seeking to attract buyers to execute on remaining clinical trials and commercialize the product

Our Approach

• leverage extensive experience in the T2DM space to conduct primary research with physicians and payers

• translate findings from primary research into defendable assumptions driving a 10-year, patient-based revenue model

Valued Outcome

• refined value proposition and clear product messaging for future payer engagements

• thorough and well-supported revenue forecast to enable/support discussions with potential investors and collaboration partners

Page 43: CELLO HEALTH BIOCONSULTING · • scientific literature and conference proceedings • industry publications • information gathered from publicly available resources One-to-one

43Capabilities deck© Cello Health BioConsulting 2020

Cello Health BioConsulting Principals

Senior Management

Joel S. Sandler, PhD 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 Principal [email protected]

Janet F. [email protected]

David J. Lomb, [email protected]

James T. Lee, PhD [email protected]

Principals

Senior Consultants

Brent W. Osborne, PhD Senior [email protected]

Beth Fordham-Meier, BS, CLPVice President, Business [email protected]