American MedChem Nonprofit Corporation - Social Edge

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Dec 3, 2012 (4 years and 20 days ago)

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American MedChem Nonprofit Corporation

Salt Lake City, UT, USA

Robert Selliah, Ph.D., Founder/CEO

Email:
robert@americanmedchem.org


Introduction:

AMC’s drug discovery research

in

rare a
nd neglected diseases (RND) is structured

on
effective and dynamic

collaborations

with
biology experts in RND
(

initial
collaborators

, positioned to the left of AMC in the
value chain C
hart below,

in b
lue
)
. T
he
output

of these

collaborations

(i.e., measurable and tangible outcome)

will be high quality preclinical/clinical drug candidates
. These
will be provided

(partnered
with or out
-
licensed)

to other partners/collaborators (

downstream collaborators

, positioned t
o the right of AMC in the C
hart,

in red)

for clinical
development
, manufacturing and distribution/marketing. If clin
ical trials are successful (e.g.
, meet FDA approved end points) the drug candidates
discovered within these collaborations
w
ill become useful medicines to treat people suffering from RND
in the US and other countries.





Chart: Translational Research Workflow for Rare and Neglected Diseases





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Definitions:

Since
AMC business model
is
collaborative in nature, it is critical th
at
well
-
structured strategic partnerships
are put in place
to ensure overall success of the process of delivering much needed, high quality and effective medicines for patients who suf
fer from
RND.
For the purposes of this target market exercise, it is im
portant to clearly define the beneficiaries of AMC’s collaborative drug
discovery efforts.


P
atients suffering from RND

are the
Direct Beneficiaries

of
the collaborative drug discovery

efforts carried out

by AMC
-

i.e.,
patients
will benefit from
effective, quality medicines

and resulting better quality of life
.
Therefore,
in the target market analysis
only direct
be
neficiaries are included
.


There are two types of
Indire
ct Beneficiaries

within this collaborative drug discovery and development
mode
l
, and these are the
collaborators and partners of AMC. First type is the “Initial Collaborator” (the blue blocks to the left of AMC in the Chart)
, who are
experts in the biological research of RND. Tremendous progress is being made in basic and applied r
esearch in biology and genetics
of RND
, to identify biological pathways and processes which can be modulated to trea
t diseases. These findings must
ultimately

be

validated in the clinic through translational research which requires drug discovery as a key

step

(AMC’s core competency)
. Many of the
academic research investigators, research institutes and disease specific foundations dedicated to RND
biology
research do not have
access to high quality, systematic drug discovery research. Strategic drug disco
very collaborations with AMC will enable these biology
researchers to validate their discoveries in the clinical stage. Alternatively, lack of access to high quality systematic tr
anslational
research (i.e., no collaboration with AMC) may mean “non
-
consump
tion” of the results from biological research. AMC will exert a
“pull
effect”

by seeking out these collaborators as “early adopters” of the AMC discovery model for RND.


Second type

of indirect beneficiaries

of AMC

model are the “downstream collaborators”

(the red blocks to the right of AMC in the Chart)
who are experts in the clinical development, manufacturing and marketing/distribution of drugs for RND. These could be pharma
ceutical
companies, biotechnology companies, disease foundations, national labor
atories,

non
-
profits,

etc.
, who need high quality novel

clinical
(or preclinical) candidates for testing in patients

with RND
. Lack of systematic and high quality drug discovery efforts in RND currently
has lead to a nearly empty d
rug development pipeline

for these diseases
. AMC will impart a
“push effect”

by providing high quality
preclinical/clinical drug candidates
to the “downstream collaborators” for clinical testing and distribution.







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2.1 Estimate of the Total Available Market

for AMC Collaborations



Segments

Size

Needs

Rare Disease Global

Approx 350 million patients
**

New treatments; none exists for
nearly
6
000
diseases

**

Neglected diseases Global

(predominantly in less developed

Countries)

1

billion patients
##

Effective
, less toxic medicines;
better drugs to
overcome resistance to existing therapy

(e.g.,
malaria, dengue, Chagas, tuberculosis)
; better
medicines for kids
++




Rare Diseases USA

25
-
30 million
**

New and effective trea
tments; none exists for nearly
6
000 rare
diseases
**




Source:
**

R.A.R.E. Project (
http://rareproject.org/2012/02/01/the
-
rare
-
list
-
you
-
must
-
see
-
it
-
to
-
believe
-
it/
); Nature Reviews Drug
Discovery, Dec
2010, p921

##

World Health Organization; globalnetwork.org

++

“Make Medicines Child Size” initiative by WHO






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2.2 Total Addressable Market Positioning Statement

for AMC in Rare Diseases and Neglected Diseases


first three years


Alternative

Characteristics

Size

Positioning

No drugs available

Pediatric

cancers and
adult

rare
cancers


13,000 pediatric
patients

(various
types of cancers); 400,000 adult rare
cancers (various types)

Active

academic

research in biology in
USA and EU
;
early
adopters of AMC
collaborators of research model;
available research grants;
personalized medicine, along with
diagnostics (developed by others);
rapid

clinical development and

approval by FDA;
health insurance
pays for prescription drugs


No drugs available

Neuromuscular diseases

Including pediatric

Approx. 10,000



40,000

patients

(range includes various types of
diseses)

Highly active research in biology and
genetics of Duchene Muscular
Dystrophy, ALS, etc; early adopters of
AMC research model; research grants
accessible; health insurance pays for
prescription drugs

Current therapies available

but drug resistance is prevalent

Malaria (rare in the USA,

Most common infectious disease

Globally,)

300
-
500 million

patients

1.5
-
2 million deaths due to malaria in
Africa; 90% of these deaths are
children; NIH

has significant

effort in
malaria biology re
search; funding
through grants available; philanthropic
groups and large pharma
ceutical

are
major drug developers and buyers.








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2.3 Market Segmentation Table


Segment Definition

Size

Compelling Reason for Adoption

Reasons for Non
-
adoption of Product

or
Service

Pediatric cancers

13,500
kids/year
diagnosed;

35,000 in
treatment;

Cause of
largest
child
mortality
rates in
USA

There is a huge need for effective medicine to treat
childhood cancers; in 20 years FDA has approved only
one drug for pediatric cancer; more than half of drugs
currently used to treat kids with cancer
were discovered
more than 25 years ago
; pharmaceutical

companies
don’t do research in this area because of small market
size.

Genetic and biological understanding in pediatric
cancer is growing; compelling need for translational
research to advance drug candidates to clinical testing
stage; Creating Hope Act
2011; Better Pharmaceuticals
for Children Act 2007; and “Make Medicine Child Size”
are all incentives to find safe and effective medicines
specifically for children

Affordability of medicine; lack of access to diagnosis; non
-
consumption

Malaria

1
million
children
die in
African
due to
malaria;
300
-
500
million
globally;
rare
disease in
the USA

There is need for affordable, safe, effective and less
toxic drugs to treat malaria; especially with emerging
resistance strains require different drugs to tr
eat the
diseases.

Children afflicted with malaria suffer morbidity and
mortality. An African child on average has 1.6
-
5.4
episodes of malaria each year; every 30 seconds a
child dies of malaria (WHO)

Make Medicine Child Size is a WHO effort to promote
med
icines for children

Affordability, distribution of drugs to patients

Neuromuscular disorders
(e.g., Duchenne Muscular
Dystrophy)

1/3600
boys
born

Need for drugs to improve quality of life.

Advanced
biological and genetic studies
; high visibility of DMD
.

Non
-
consumption