Seqens eBook - 19

Accelerating Drug Development
Through Repurposing,
Repositioning and Rescue
Nigel Walker
That's Nice

From Serendipity to Purpose
The pharmaceutical industry is facing intense pressure to reduce
the cost of prescription drugs and also more rapidly develop novel
medicines to treat unmet medical needs, including rare diseases. One
approach that can significantly reduce the time, cost and risk of drug
development is referred to as drug repurposing, or repositioning,
reprofiling, re-tasking, etc. and involves the application of existing
drugs (approved or unapproved candidates) for the treatment of
previously unconsidered indications.
Drug repurposing is not new. Historically, however, it was largely
the result of serendipity. Side effects and off-label uses of drugs
have suggested potential new indications, according to Gini
Deshpande, Founder & CEO of NuMedii, a drug discovery company
that applies its proprietary big data platform based on integrative
genomics, networkbased methods, large-scale machine learning,
and chemoinformatics to improve efficacy.1 Viagra (Pfizer's
sildenafil) is one such example; the drug initially failed as an
angina treatment in clinical studies, during which its effect on
erectile dysfunction was noted.
Today, many organizations are purposefully investing in drug
repurposing, repositioning and rescue (DRPx) programs. Drug
repurposing refers to the use of exiting approved drugs for new
indications, while repositioning involves the development of an
existing, previously evaluated but unapproved active pharmaceutical
ingredient for the treatment of a different disease.2
It is estimated that 30% of FDA approved new drug products3 and
repurposed drugs account for approximately 25% of pharmaceutical
industry revenues.4 The global market for drug repurposing is
estimated to reach $31.3 billion in 2020, growing at a compound
annual growth rate of 5.1% from $24.4 billion in 2015, according to
BCC Research.5
The growing interest is driven by the advantages of working with
existing compounds that have already undergone significant safety
testing. Repurposed drugs have shorter development times (5-8
years) compared to those associated with new chemical entities (1015 years).1 As a result, costs can be as much as 60% of those for NCEs.2
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In addition, the risk is lower: approval rates for repurposed drugs are
close to 30%.2
Repurposing/repositioning existing compounds is particularly
attractive for the development of treatments for rare diseases. There
are only approximately 400 approved drugs for the more than 7000
currently identified rare genetic conditions.6 Using existing APIs to
validate the biochemical pathways and facilitate the discovery of
new compounds can speed the development of drugs for patients
with no current options. There are approximately 2,800 drugs and
more than 4,000 compounds that have been discontinued at Phase
II development, according to Deshpande, providing a "rich pool" of
possible therapies.1

Many Successes
In recent years, many unsuccessful drug candidates and existing
drug products have been successfully investigated and approved
for other indications.
Thalidomide, which was originally developed as a racemic mixture
of enantiomers for the treatment of morning sickness but found
to be teratogenic due to the effect of the (S)-(-)- isomer, was later
successfully developed by Celgene as a single (R)-isomer product for
the treatment of leprosy and multiple myeloma.2
Celebrex, widely used for the treatment of osteoarthritis, works
by inhibiting COX-2 receptors. Recently it has been shown that
for patients that previously had colon cancer, taking Celebrex can
reduce the risk of additional polyp formation without negative
gastrointestinal effects associated with existing treatments.
Common diabetes drug metformin has been shown to reduce the risk
of breast cancer in diabetes patients and is being investigated as a
treatment for cancer in many different clinical trials.7
Acne medicine all-trans retinoic acid (ATRA), when combined with
traditional chemotherapy, results in complete remission of acute
promyelocytic leukemia in 90% of treated patients.7
The antibiotic minocycline, which is used to treat acne and sexuallytransmitted infections, also decreases the symptoms of patients
19

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Seqens eBook

Table of Contents for the Digital Edition of Seqens eBook

Contents
Seqens eBook - 1
Seqens eBook - Contents
Seqens eBook - 3
Seqens eBook - 4
Seqens eBook - 5
Seqens eBook - 6
Seqens eBook - 7
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Seqens eBook - 9
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