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5/15/17

New Market, New Players Biosimilars

A Pure Play Perspective

Brands (biologic) Generics

World Biosimilar Congress USA 2015

Brands (small molecule)

May 27, 2015

Medicinal drugs from natural sources (e.g., herbs, plants, roots, fungi)1

Pre-

Sarfaraz K. Niazi, Ph.D.,

2

Biosimilar Development has Accelerated

37 7

40 30 20 10 0

22 5

9 8 13-Jan

14

3

46

48

9

10

22

16

15

13-Aug

14-May

Approved

57

+4%

+24% +68%

Ph III

1906

1938

[KefauverHarris] Drug Amendments Pre-market proof of safety AND efficacy Required (IND)

First mass production of synthesized insulin using recombinant technology (1963-66 was small scale)3

Hatch-Waxman Act enacted Permits generic approval without new full NDA package

1962

1978

1984

US Food, Drug and Cosmetic ACT Pre-market safety evaluation for the first time (NDA)

1972

Biologics control transferred to the FDA from the National Institute of Health

1982

First BLA approved for Humulin (Eli Lilly & Co.’s human insulin)

First biosimilar approval in the US

2009

2015

BPCIA passed to allow for biosimilar versions of approved reference biologics

1. http://www.ncbi.nlm.nih.gov/pubmed/21698778, 2. http://pubs.acs.org/cen/coverstory/83/8325/8325emergence.html 3. http://www.karger.com/ProdukteDB/Katalogteile/isbn3_8055/_83/_53/Insulin_02.pdf

TheraProteins

+19%

50

1800’s

1869, first synthetic drug discovered1 Mid-late 1880’s, began wholesale production of drugs2

Founding Chairman TherapProteins® Chicago, Illinois, USA

60

Pure Food and Drugs Act passed, FDA modern regulatory functions begin

10

Focused only on biosimilars

Proprietary, patented, single-use technology platform

Fully integrated from cell line to fill-and-finish

cGMP compliant facility located in Chicago, Illinois

37 31

7

10

14-Oct

15-Apr

Ph I / Ph II

Double counting was avoided by using the most advanced status, i.e. a product with EU approval and US phase III counts as an approved product Source: 1st Annual Bernstein European Biosimilars Conference (FDA, EMA, Industry discussion, Bernstein analysis)

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5/15/17

Who’s Who? Brand

What is a Pure Play Biosimilar Company? Generic

Pure Play

Other

?

Brand

Other

NON-PHARMA INDUSTRIES

BRANDS

Generic

GENERICS

Pure Play

BIOSIMILARS

CRO/CMO/Emerging Market Domestic Players

A Pure Play Biosimilar Company focuses solely on biosimilar products

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6

New Market, New Paradigm Brand Launch

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Generic Launch

Challenges for Pure Play Companies Biosimilar Launch

Product

• Fully branded product driven • New name • New INN

• Unbranded company driven • No brand name • Same INN

• Hybrid • Brand name • Same INN (qualifier?)

• Not applicable

Education

• New product, MOA, treatment regiment, clinical safety and efficacy results

• Analytical characterization data • Safety data

Sales

• Large sales force • Pull-through on a prescriber level • MSL support

• Limited sales force • Focus on relationships with intermediaries

• Hybrid sales force? • MSL support for science story

• Not applicable

• Continuity of service

Services

• Patient services (e.g., PAP, copay, etc.) • Provider services

• Full clinical program • Prove safety and efficacy

• Not applicable

Clinical

• Analytical foundation • Safety studies • Prove safety, purity and potency

New Molecular Entity

Bioequivalent

Biosimilar

1 Competition: nonpure players

2 Getting to market

w Existing business relationships and practices

w Recognition in market

w Infrastructure and technology

w Immediate resources

w Financial resources

w Image of quality w Scientific finesse

Solutions: In the end it will be the COGS 8

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1

Traditional Infrastructure is a “Baggage”

1

Deep Tank System

2

3

Roller Bottle System

Single-use Bioreactor

u Single Container Expression, Harvest and Purification u Proprietary sparging and capture u ISO9 Friendly—no infrastructure u Modular, no scale-up u Transportable u Fast to Market u High Consistency—gentle movement u Lowest COGS—globally competitive

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TPI Single-use System

Bioreactor technology has experienced innovation over time, but what has to be given up to move to something new? Biologic Facility (2015)1 › ~323,000 ft2 › Six 15,000 L bioreactors › 400 employees › $900M investment › 4 Years › Ireland

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Biologic Facility (1994)2 › 300 employees › $150M investment › 3 Years for phase I Plant Expansion (2004)3 › 1,000+ employees › $600M investment

Biologics Facility (2012)4

Single-use Facility (2013)5

› $500M investment › 3 Years › Singapore

› $200M investment › 2 Years › Singapore

MayaBio® Bioprocess Platform (45 Patents)

› 5 Years

1. http://news.bms.com/press-release/financial-news/bristol-myers-squibb-construct-new-large-scale-biologics-manufacturing- 2. http://www.gene.com/media/press-releases/4420/1994-10-31/genentech-locatesmajor-new-facility-in- 3. http://articles.latimes.com/2004/apr/02/business/fi-genentech2 5. http://www.fiercepharmamanufacturing.com/story/amgen-opens-200m-continuous-purification-plant-singapore/2014-11-20 Roller Bottle Credit: REUTERS/V. RICHARD HARO/WIREPIX Source: http://www.reuters.com/article/2007/08/16/usamgen-idUSN8F26518720070816 (accessed June 2014)

No Compromise on Science—Smart Science Required

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Similarity Bar—Thinking Differently

Primary Structure

Product-related Impurities Intrinsic variants related to the protein

Processrelated Impurities Impurities that can be introduced from downstream process

The core DNA sequence and any post-translational modifications of the molecule

Higher Order Structure Secondary, tertiary and quaternary structure

Stability Particles and Aggregates

Product Properties

Biological Function How the molecule works including receptor binding

Finished drug properties including strength and formulation

w It takes a holistic approach to demonstrate a product is highly similar

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w It is in some sense more complex than producing a new molecular entity

w First test that measures 4D structure of protein solutions—replaces many tests, removes more residual uncertainty

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Education is one of the Biggest Hurdles to Adoption

2

Biosimilars Knowledge Remains Low 202

100

19%

16% 27%

38%

Extremely Familiar Very Familiar Moderately Familiar

39%

Not familiar

32% 10% 1% US

Slightly Familiar

17% 1%

“efforts to undermine trust in these products are worrisome and represent a disservice to patients who could benefit from these lower-cost treatments.”2

Key Takeaways u Pure play companies better poised to produce affordable biosimilars u Existing infrastructure is an archaic “baggage” u In the long run, it is the COGS that will determine market success u Cost of science will determine survival

Former FDA Commissioner Margaret Hamburg

Flexibility and Speed to Execution

EU5

As a pure play, there is only one story to tell 13

1. FirstWord: 2014 Physician Views Poll Results – A Tale of Two Continents; US and EU physicians paint contrasting picture of biosimilar educational space (80 US and 100 EU) and Xcenda pole of 122 US oncologists, 2014 2. http://www.worldipreview.com/article/biosimilar-biologics-is-the-us-being-left-behind

Reassess Every Aspect of the Process

Leverage Novel Technology Reduce Timelines One Focus, One Goal

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2

Thank You

Progress is Being Made, But More is Needed The FDA has Consistently Spoken about the New Paradigm of Biosimilars

› Fact based educational resources are emerging in the market › Industry groups are being formed with the goals of education and influencing policy in a positive way for biosimilars

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The Bar is Set Extremely High; Passing Puts you on the Playing Field

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New Brand Biologic

The Key will be the Value and the Value Add that each Competitor can Provide • Can you demonstrate biosimilarity? • Do you have a strong characterization package and can that story be told effectively? • Do you have the same product elements as the reference product? - Same presentation and delivery device? - Similar stability? - Similar packaging?

A New Type of Model Requires a New Set of Capabilities

w w w w

• Can you demonstrate confidence in quality?

New Biosimilar

w Being without “baggage” enables a certain level of flexibility and agility that is valuable in the biosimilar market

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w Reproduction of an reference biologic w Match the critical quality attributes of the reference product, including the productrelated impurities w Be within the band of acceptable variability of the reference product w Prove safety, purity, and potency, which comes first from matching structure

Analytics

Analytics

Drug Product

Upstream Downstream

Cell Line Analytics

Fill Finish Analytics

Within Reference Product Variability

• Can you provide consistency of patient experience?

w Replica of an branded small molecule product w No clinical studies required w 100% characterization of reference possible

New molecular entity Complex Prove efficacy and safety Set the criteria for acceptable variability batch to batch

• Can you provide a affordable alternative? • Can you be flexible and nimble in relationships, contracts etc.?

New Generic

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1

Requirements Rooted in Analytics and Science

1

Ingenuity Unleashed with a Clean Slate

New Biosimilar Clinical Studies

Clin Pharm

MayaBio® Bioreactors

MayaBio®

Clinical Studies

Clinical Pharmacology

w Proprietary and patented system (45 IP)

Nonclinical

w Mammalian or microbial cell expression systems

Nonclinical

w Single container expression, harvesting and purification

Analytical

w A flexible container with a proprietary sparging system w Suitable for ISO9 environment

Analytical Clinical Studies

w Completely closed system prevents cross-contamination Clin Pharm Nonclinical Analytical

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New Biologic

w Modular, transportable and instantly scaled w The base for biosimilarity is structural ‘sameness’ proven through analytical methods

w Consistent and gentle reliable expression and quality

w Clinical pharmacology studies are emphasized for function and safety w A pivotal phase III study to prove efficacy is not the model for biosimilars

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World biosimilars congress usa 2015  
World biosimilars congress usa 2015  
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