Market Analysis for Life Science Start-ups Part One · Market Analysis for Life Science Start-ups ... Sales and marketing methods ... PTCL, Lung cancer

www.siegalconsulting.com

Market Analysis for Life Science Start-ups Part One: What you need to Get Funding and Plan for the Future

Carolyn Siegal

Principal, Siegal Consulting

[email protected]

626-798-8700

mailto:[email protected]


If you are….. • Filing an SBIR or similar grant applications

• Raising external capital

• Building a business plan for internal purposes and decision making

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MARKET ANALYSIS: A CLEAR UNDERSTANDING OF

PRODUCT, COMPETITION AND CUSTOMERS AND HOW

TO GET FROM ONE TO THE OTHER


Market Analysis Overview

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Defining the target market

Market environment

Selecting your market segment

Industry trends

Distribution channels

Customer profile

Competitive activity

Pipeline activity

Opportunities and barriers to entry

Sales and marketing methods

Building a winning target product

profile

Product positioning

Features and benefits

Cost and reimbursement

Buying process

Map the future

Tool kit:

Situational market analysis (landscape)

Secondary market research

SWOT analysis

Buying process

Gap analysis

Tool kit:

Target Product Profile

Primary market research

Income forecast

Competitive Matrix

SWOT analysis


Market Research for SBIR Proposals

• What customer needs will be addressed with your product or service?

• Describe who your target customer is. Providing generally-known examples may be helpful.

• How does the target customer currently meet that need that you are addressing? Is there is a significant problem that is not yet being addressed?

• What is the business model you plan to adopt to generate revenue from your innovation?

• Is the target market domestic, international or both?

• What channels would you employ to reach the targeted customer?

• What is the current size of the broad market you plan to enter and the “niche” market opportunity you are addressing?

• What are the growth trends for the market and the key trends in the industry that you are planning to target?

• What are the barriers to enter this market?

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Questions from the NSF Phase II Commercialization Plan


Defining Your Market Segment

5 Not too big…not too small, just right!


Finding the Right Level Right Level

• Market share is based on unmet needs, competitive attributes, displacement, buying patterns, customers • Ex. Neuromodulation for

Urinary Incontinence, 100 thousand patients per year, Product X can capture the 20% of the market that is dissatisfied with Product B

• Unit/demand driven • Ex. Relapsed, refractory multiple

myeloma, 20 thousand patients based on patients not responding to current 1st line standard of care

High Level

• Market $ # is not informative about competitive advantage

• Ex. Anti-cancer drug sales were $50 billion in 2009

• Ex. Clinical laboratory analysis for in vitro diagnostic (IVD) testing, $39 billion mark in 2008

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Need vs. Nice to Have

• Need

• Information that will inform business decisions

• That shows your market knowledge, expertise

• That dovetails and adds to the knowledge of an expert

• “No data before it’s time”

• Model is more sophisticated than the product knowledge/stage

• Anticipate the pivot

• Most early stage companies change direction on the way to market

• Priority vs. budget

• If you don’t have money DIY!!!

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DIY Market Research Resources

Secondary Data • Clinical literature

• Scientific literature

• Trade literature

• USPTO.gov

• SEC.gov

• http://report.nih.gov/

• clinicaltrials.gov

• Competitor websites

• Suppliers

• Medical Associations/Guidance documents

• Trade associations (BIO, AdvaMed, PMC)

• Patient advocacy groups (American Cancer Society)

• Government agencies (NIH, CDC, FDA, DARPA)

• Foundations (Gates, Michael J Fox)

• Public health (WHO, NICE)

• Google

• Wikipedia

Primary Data

• Customer and expert interviews

• LinkedIn groups

• Analyst reports

• Association lists and online survey resources

• Personal network

• Suppliers

• Conference competitive intelligence

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DIY Starter Kit

• Target Product Profile

• Market segment landscape analysis

• SWOT

• Competitive matrix

• Buying process

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Also a great foundation for business plan


Target Product Profile

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Single Agent Therapy

(3rd

Line Relapse/refractory ALL, Mantle

Cell, Waldenström’s

macroglobulinemia,?)

Combination Therapy

(2nd Line in MDS, Mantle, CLL,

DLBCL, ?? )

Product Description/

Mechanism of Action

A tissue targeted cytotoxic prodrug that is

bioreduced to PRODUCT (reduced

PRODUCT), a highly potent DNA

topoisomerase II inhibitor.

Same

Primary Indication(s)

Phase Ib

Patients with advanced Lymphoid

neoplasms. POC in patient with Multiple

Myeloma. Phase 1b would be a short run in

trial starting on dose level below the phase

1 solid tumor study. Phase II program will

be broad based in areas other than MM if

Product is successful. These include, ALL,

Mantle Cell, Waldenström’s, AML, CLL,

DLBCL, Follicular. Most of these would

require a randomized phase 3 studies and

require at lease 600+ patients. ALL,

Mantle, Waldenström’s would require

single arm studies but these are small

prevalence diseases

Phase Ib

MTD, DLT, PK/PD, Safety in combination

with Doxorubicin, Rituximab (?),

Fludarabine (?), lenalidomide (?) in selected

population of Lymphoid neoplam

Dependent on indication selected from

phase 1 and/or Product development

Phase 2

As above in a randomized phase II

Target Patient

Population/

Patients with DLBCL, Follicular, Mantle,

Waldenström’s who have failed front-line

and at least one second-line multi-agent

regimen with alkylating agents and/or

Rituximab. Patients with ALL who have

failed at least 1 regimen who are not

candidates for BMT.

In combination with chemotherapy or

biologic who have failed frontline therapy

or after first relapse.

Route of Administration Oral Oral

Dosage and

Administration

Administered daily (or BID) for 5 days with

nine day rest in a 28 day cycle or daily for 5

days for 2 weeks with 12 days rest. Dose to

be determined from Phase 1 trial in

lymphoid and/or solid malignancy trials.

Administered daily (or BID) for 5 days with

nine day rest in a 28 day cycle or daily for 5

days for 2 weeks with 12 days rest. Dose to

be determined from Phase 1b combination

trial in lymphoid neoplasm trial.

Duration of Treatment Up to 6-12 months Up to 6-12 months

Success Criteria

(efficacy) for modeling

purposes only

Very successful

Observed response rate (ORR) of > 50%

with RD of > 12 months

This is dependent on the indication selected.

ORR of > 80% with a PFS of > 12 months

Successful ( for modeling

purposes only)

ORR of 40-50% with RD of 10-12 months ORR of 50-60% with a PFS of 10-12

months

Minimally Successful ( for

modeling purposes only)

ORR of 30% with RD of 7-10 months

ORR 0 – 10% efficacy is not supportive of

further development as a single agent.

ORR of 45% with PFS of > 10 months

ORR of 0 – 10% efficacy is not supportive

of further development in combination.

Expected Primary

Endpoint (for modeling

purposes only)

Phase I

Preliminary Bioavailability

Phase II

ORR and Duration of Response

(need both endpoints for 3rd line approval)

Phase I

Phase II

ORR and Duration of Response

(need both endpoints for 1st or 2

nd line

approval)


Market Segment - Landscape Analysis • Merges situational analysis and mapping/landscapes

• Key market attributes

• Epidemiology/demographics

• Market behaviors

• Trends

• Key competitive attributes

• Competitive matrix

• SWOT of your Target Product Profile

• Map products against market attributes

• Drivers and hurdles

• Validate assumptions from secondary and primary market research 11


Market Landscape Analysis Tyrosine Kinase Inhibitors in Acute Myelogenous Leukemia

High Medium Low

Likely Impact on Standard of Care

Phase III

Phase II

Phase I

Sorafenib

Tipifarnib Lestaurtinib

Sunitinib

Tandutinib

Product

No combination data available for sunitinib, tipifarnib will not directly address flt3 mutant but overall benefit in

first line may impact treatment selection FLT3 selective

Other MTKI w/ flt3 Other targeting 10/11/2011 12


SWOT

• Strategic planning method

• Project/Product/Business Venture

• Factors that are helpful vs. harmful to achieving business objective

• Internal factors • Strengths

• Weaknesses

• External factors • Opportunities

• Threats

• A way to summarize hurdles and opportunities in one page • Do before market research to see what you are missing

• Do after market research to summarize what you learned 13


REMICADE – SWOT Analysis 1

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Sources:1Drugs@FDA – REMICADE Label, 2Jeff Stoll “Is the anti-TNF Market Facing a Seismic Shift?, Scientia Advisors, May 12, 2011, 3Jeffrey G Stoll and Uma Yasothan,

Rheumatoid Arthritis Market – Nature Review – Drug Disc, Vol 8, Sep, 2008, 694, 4“Anti-TNF Treatments and IBD” University of Manitob, 5“Positioning of REMICADE relative to

SIMPONI in leading indication – RA”, PharmaShare, 6“Surveyed Gastroenterologists Indicate That Humira Has Advantages Over Remicade In Maintaining Clinical Remission Of

Crohn’s Disease, Decision Resources, Jan 26, 2009, 7Armuzzi, A. & Pugliese, D., “Adalimumab for ulcerative colitis — is the glass half empty or half full?, Nat. Rev. Gastroenterol.

Hepatol. 8, 249–251 (2011), ” New Therapies in the Management of Rheumatoid Arthritis”, 8Maya H Buch et al., “New Therapies in the Management of Rheumatoid Arthritis”, Curr

Opin Rheumatol. 2011;23(3):245-251

• Largest number of approved indications, including a pediatric label in Crohn’s disease

• REMICADE was the only approved drug for Crohn’s disease and ulcerative colitis for almost 9 years and has an archive of experience across indications.

• Infusion once in 8 weeks limits office visits and improves patient convenience.

• As it is administered at a hospital/rheumatology clinic, patients usually do not have reimbursement concerns.

• Robust data supporting remission with full steroid withdrawal

at 1 year for CD; considered gold standard in IBD4

Opportunities

• Chimeric antibody vs. competitor mAbs that are

humanized • Black box warning of risk of infections especially

tuberculosis • Being an IV infusion drug, patients have to face

inconveniences of traveling to an infusion center. • Duration of infusion is long (2 hours) • Mostly reserved for second-line+ in rheumatoid arthritis,

psoriasis, and ankylosing spondylitis.

• REMICADE is the only IV anti-TNF and is thought to offer a

faster response than other anti-TNFs • Strong foothold in Crohn’s disease and ulcerative colitis indications

with robust patient registry data creates an advantage over other anti-TNFs.

• Cannibalization with SIMPONI IV formulation (Phase

III)8

• Results from HUMIRA 52-week trial in UC induction &

maintenance are pending. If data is positive, HUMIRA

could take away share from REMICADE in UC.7

• Abatacept (ORENCIA) has shorter infusion time (30

min vs. 1 hour for REMICADE) and similar efficacy to

anti-TNFs. • Due to different MoA, ORENCIA and ACTEMRA are better

positioned to target anti-TNF refractory patients. • Novel oral drugs in pipeline (JAK, MAPK, SYK

inhibitors)

Strengths

Threats

Weakness


Competitive Matrix

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Reaction

Yield Purity Capacity

Processing

Speed Scale-up

Additional

Requirements

Synthetron™ S3 98% Highest 1.5 Kg/hour Fastest Linear

No external cooling

required

Microreactor 90% Good 5 gram/hour Moderate Parallel

Requires cooling &

maintaining 0°C

Batch 76% Varies Multi-kg/hours Slow est Non-Linear

Cooling capabilities to -

40°C & below

Extrapolation of experimental results to commercial use characteristics

Chemical Synthesizer Competitor Comparison


Buying Process

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Tailor to your product/your industry 6 stages • Problem recognition • Information search • Evaluation of alternatives • Purchase decision • Purchase • Post-purchase evaluation

Validate your assumptions with secondary and primary market research


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Treatment Process: RA RA Patients

Under Treatment

NSAID Non-MTX DMARD

MTX

MTX

2nd TNF Blocker Other Biologic

TNF Blocker

Non-MTX DMARD

+

1% adult pop, 2.1 million

75%, 1.6 million

75%, 1.2 million

15%, 250 K

30-40% of TNF blocker treated will progress, annual ~40K

+ MTX or


Consulting Projects Type Area

Target Product Profile, identification of commercial challenges Device to provide visual cues to visually impaired

Commercialization Plan, support in- license agreement POC rapid diagnostic test, Q Fever

KOL IDS’s to support in-licensing-in-licensing HDAC-inhibitor, oncology, PTCL, Lung cancer

Top-line competitive clinical assessment to support venture investment Malignant melanoma

Partnering support; non-confidential docs and exhibit prep Antibody library generation

Strategic advisor; SBIR companies Antibody discovery and development, Power wheel chair, Lyme disease field inoculation, microneedle transdermal path, Alzheimer therapeutic, cardiac monitoring device, OCT imaging, early detection for Parkinson's, mobile wireless ECG, Oncology research tool based on modified B-lactam

Pipeline; resource allocation/prioritization Rheumatoid arthritis

Out-licensing strategic partner assessment, identification Oncology

Pricing analysis and recommendations for Phase 2 product to support out-licensing

Oncology

Deal comp analysis and recommendations Cardiac output monitor

Partner identification for out-licensing Cystic fibrosis

Acquisition opportunity vetting Oncology, supportive care

Commercial Assessment and Sales Forecast Pre-clinical TNF Blocker

Analysis of industry standards for SG&A and Product Marketing to support US sales forecast

Japanese Pharma

Corporate summary for investor review Lab equipment/process for rapid chemical reaction processing

Commercialization plan for SBIR grant application Liposomal formulation , chemotherapy, breast cancer

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Next Session

• Working group

• Hands-on web time/mentoring

• Pick a “Tool” to focus on.

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Documents

Market Analysis for Life Science Start-ups Part One · Market Analysis for Life Science Start-ups ... Sales and marketing methods ... PTCL, Lung cancer