Reinventing the CMO industry - Amazon Web Services · 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 ... 30% of the 45 ANDs were developed for the treatment of cancer ... Reinventing

Reinventing the CMO industry

CPhI 2017 | Joseph Bihm | Account Management Small Molecules

A mature CMO market commoditizing?Contract manufacturing for small molecules

CPhI 2017 | Joseph Bihm | Account Management Small Molecules 2

CMO market for small molecules exists for more than forty years and matured over time

Fragmented market with many players, perceived oversupply of capacity

Over time the small molecule API market has shown elements of commoditization

Some pharma and biotech customers started to select CMO partners primarily based on price vs quality

New challenges drive need for CMO partner


More complex, highly potent molecules

Poor solubility, decreased bioavailability

Accelerated timelines

Uncertain demand

Innovation needs

Increase in new drug filings


VC fundings fuel new opportunities

• 2015 record breaking year for VC Deals

• Continued reinvestment in VC funds

• More companies with more funding has impacted the CMO market

Key factsUS Annual VC Deals: 2007-2016

Source: National Venture Capital Association, PWC, MoneyTree report

$6.0

$4.8 $4.9 $4.9

$4.3$4.8 $4.9

$7.1

$9.6

$7.8

$0.0

$1.0

$2.0

$3.0

$4.0

$5.0

$6.0

$7.0

$8.0

$9.0

$10.0

2007 2008 2009 2010 2011 2012 2013 2014 2015 2016

Year

Aggregate Deal Value ($bn)

Increase in new drug filings


Fast track programs create opportunities

• Increased New Drug Filings

• The number of NDAs/BLAs filed per year has increased slightly over the past decade.

• Between 2005 and 2013, an average of 34 filings were made per year, compared with 41 filings in 2014 and 40 filings in 2015

Key facts Number of NME/BLA filings

Source: FDA CDER report

32

2022

18

2426

21

30

39

27

41 40

0

5

10

15

20

25

30

35

40

45

2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015

# NME Applications Filed

Ø 34

Increase in approval rates


Fast track programs create opportunities

• Fast-track regulatory approval programs have been introduced to meet new needs by shortening the time to market and compressing timelines for developers

• More drugs are being approved at a faster rate

• Increasing number of drug applications approved in the first review cycle1

Key facts Number of NME/BLA approvals by FDA

Source: FDA CDER report

36

2022

18

2426

21

30

39

27

41

45

0

5

10

15

20

25

30

35

40

45

50

2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015

# NME/BLA Approved

Ø 25

1 2008 - 2012, the FDA approved 54.8% of drug applications in the first review cycle vs 78% and 87% in 2014 and 2015



Increase in approvals – contributing factors

First in Class and Orphan Approvals

Shift in the types of new drugs that are submitted to the FDA for approval:

Acceleration in first in class approvals, compared to an average of 15 agents approved p.a by FDA between 1987-2011

Number of FDA orphan approvals has been steadily increasing since 2000

Unique and innovative qualities of the drugs submitted to the FDA in 2014 and 2015 may have contributed to the increase in CDER approvals.

Source: FDA CDER

1716

17

21

2014 2015

First in class Orphan

Number of FDA approvals 2014 -2015 for First in class andorphan drugs




FDA Expedited Programs

The way the FDA works with industry on new drug development programs evolved

FDA now offers 4 paths for expedited development and/or review:

• Fast track, breakthrough therapy, priority review, and accelerated approval.

• In 2015, 14 of the 45 approved new drugs had fast track designation1

The use of fast track, accelerated approval, and priority review programs has been steadily increasing since 1987.

The breakthrough therapy designation was created in 2012

• Use of the designation is increasing: there were 21 approvals in 2015, 14 approvals in 2014, and 3 approvals in 2013.

Expedited programs increase the speed at which new drugs are developed and reviewed, which could contribute to the number of CDER approvals in recent years.

1 10 breakthrough therapies, 24 were given priority review, and 6 received accelerated approval. Similar to 2014.




Therapeutic Area

1987 - 2014, oncology related therapeutics generated more fast track, accelerated, and priority approvals than any other therapeutic area.

• 2015 oncology was the single largest therapeutic area for new drug approval

In 2014, 20% of the 41 ANDs and in 2015, 30% of the 45 ANDs were developed for the treatment of cancer

The constant need for cancer therapeutics, coupled with their proven track record for obtaining accelerated approval (based on surrogate endpoints), may have contributed to their approval rate in recent years.



New market – new needs

Speed Flexibility Innovation

• Need for secure supplycovering a variety of different market demand scenarios

• Need for shortered timelinesin process development

• New ways of collaborationand risk sharing

• Technologies supportingflexibility needs and costefficiency for smaller volumemedicines

Case StudySmall molecule program with breakthrough therapy designation status


Case Study


Specific customer needs

Speed Flexibility Innovation

• Need for secure supplycovering a variety of different market demand scenarios

• Small volumes expected

• Accelerated developmenttimeline, shortened cycle but robust and scalable processrequired

• Supply chain control required

• Technology innovation toshorten lead times of productto market

• Innovative new molecule with a new mode of action, running against several competing products to market approval –highly uncertain final demand due to competitive landscape

• Good clinical trial read-outs led to breakthrough therapy designation from FDA

Case Study


Solution to address customer problem

Schematic view of multi-purpose plant vs monoplant

Lonza was able to offer a so-called “monoplant”, i.e. a dedicated manufacturing assets

• Lonza to design, engineer, and build dedicated manufacturing assets within existing facility

• Monoplant includes extensive online analytical monitoring designed to facilitate real-time release testing

• During construction of new facility, Lonza grants dedicated access to its existing production trains –bridging campaigns

Monoplant commitment triggered on FDA approval

Q1

Q2

Q3

Q4

Cus1

Cus1

Cus1

Cus2

Cus3

Cus1

Cus1

Cus1

Cus1

Multi-purpose plant Monoplant

Case Study


Key benefits for the customer

VALUE

Lead time from order to

delivery reduced from more

than 24-36 month down

to 6 weeks

Intermediate “Buffer Stock”

management: Rapid response

to demand fluctuation

Guaranteed capacity access

for critical manufacturing

steps every year

COGS reduction by 50%

compared to initial campaigns



New needs – innovative solutions

36%

Agile

development for

your accelerated

approval

timelines

Breakthrough Therapy Designations for commercialized medicines and Lonza

share, 2013-161

1 For medicines that have received commercial approval by FDA between 2013-16 and had a breakthrough therapy designationPlease note that drugs receiving more than one BTD are counted as 1 in this graphicSource: FDA CDER, Lonza



VISIT US

To learn more about our drug substance process dev. and mfg capabilities/services

• Please stop by Lonza Booth # 11.1C30 in Hall 11.1

To learn more about some of our specific drug product differentiated tools and services such as Tech selection methodologies, Xcelodose-based powder in capsule / microdosing studies

• Please stop by at the Capsugel Booth # 4.1B10 Hall 4.1

Thank you!


Documents

Reinventing the CMO industry - Amazon Web Services · 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 ... 30% of the 45 ANDs were developed for the treatment of cancer ... Reinventing