Ten years have passed since the publication of the ‘rule
of five’ physical property guidelines for drug permea-
bility
1,2
. The rule of five, which was derived from a
database of clinical candidates reaching Phase II trials or
further, states that poor absorption or permeability are
more likely when cLogP (the calculated 1-octanol–water
partition coefficient) is >5; molecular mass is >500 daltons
(Da); the number of hydrogen-bond donors (OH plus
NH count) is >5; and the number of hydrogen-bond
acceptors (O plus N atoms) is >10 (REFS 1,2). Although the
rule-of-five properties are interrelated (cLogP actually
being a composite property dependent on molecular
size, polarity and hydrogen bonding
3
), its conceptual
simplicity and ease of calculation has made it the leading
measure of drug-likeness, with the original article having
more than 1,500 literature citations.
The physicochemical profiles of oral drugs
4–6
are con-
sistent with the rule of five, and recent studies on drug
absorption have also highlighted the importance of polar
surface area (PSA), which is closely correlated to O plus
N atom count, and LogD (1-octanol–water coefficient at
various pH values)
7,8
. Overall oral bioavailability depends
not just on absorption but also on dissolution, gut transit
time and first-pass metabolism, so it is perhaps surpris-
ing that rat bioavailability can be categorized by simple
physical properties such as PSA and rotatable bonds
9,10
,
and number of rule-of-five violations, PSA and ioniza-
tion state
11
. However, these models do not readily explain
human bioavailability data
12
. One further aspect that has
received less attention is that bulk molecular properties
are relevant to drug safety as well as pharmacokinetics and
metabolism: marketed oral drugs, which are rule-of-five
compliant
4–6
, have by definition successfully passed
rigorous toxicological and clinical safety hurdles.
Analyses of drug-likeness inevitably depend on the
medicinal chemistry innovation and prevailing strate-
gies of the past. However, it is clear that compounds
produced by more recent medicinal chemistry efforts
do not occupy the same chemical space as historical
drugs. From three studies of oral drugs
4–6
, the mean
cLogP values were 2.3, 2.5 and 2.5 and mean molecular
masses were 333, 337 and 344 Da. By contrast, 1,117
GlaxoSmithKline compounds
9
and 553 Abbott com-
pounds
11
that had advanced to pharmacokinetic studies
had, respectively, mean cLogP values of 4.3 and 3.9, and
molecular mass values of 480 and 434 Da. Additionally,
a group of 1,680 optimized compounds from the recent
medicinal chemistry literature had a mean cLogP of 4.0
and molecular mass of 435 Da
13
; and in another literature
study, more than 50% of compounds with high potency
had cLogP values >4.25 and molecular mass values
>425 Da
14
. Drugs and research compounds also differ
in physicochemical properties between target protein
classes
13,15,16
and therapy areas
17
. Molecular mass and
other properties of oral drugs
6,17
, as well as of literature
compounds
16
, are also increasing with time.
These changes are potentially a concern because
physical property inflation may prove detrimental
to the health of drug development pipelines. Several
studies
4,14,16,18
of compounds in development concur
that mean molecular mass declines as compounds
progress through Phase I, II and III; moreover, the more
AstraZeneca R&D
Charnwood, Bakewell Road,
Loughborough LE15 5RH, UK.
Correspondence to P.D.L.
e-mail:
paul.leeson@astrazeneca.com
doi:10.1038/nrd2445
LogP
Log of the octanol–water
partition coefficient, which
is a measure of a drug’s
lipophilicity. Defined as the
ratio of un-ionized drug
distributed between the
octanol and water phases at
equilibrium. Higher values
imply greater lipophilicity.
Molecular mass
The molecular mass of a
substance, frequently called
molecular weight, is the mass
of one molecule of that
substance, and its units are the
unified atomic mass unit (u)
or Dalton (Da), which equals
1/12 the mass of one atom
of carbon-12.
Polar surface area
(PSA). This is defined as the
surface sum over all polar
atoms, (usually oxygen and
nitrogen), also including
attached hydrogens.
The influence of drug-like concepts on
decision-making in medicinal chemistry
Paul D. Leeson and Brian Springthorpe
Abstract | The application of guidelines linked to the concept of drug-likeness, such as the
‘rule of five’, has gained wide acceptance as an approach to reduce attrition in drug
discovery and development. However, despite this acceptance, analysis of recent trends
reveals that the physical properties of molecules that are currently being synthesized in
leading drug discovery companies differ significantly from those of recently discovered oral
drugs and compounds in clinical development. The consequences of the marked increase
in lipophilicity — the most important drug-like physical property — include a greater
likelihood of lack of selectivity and attrition in drug development. Tackling the threat of
compound-related toxicological attrition needs to move to the mainstream of medicinal
chemistry decision-making.
NATURE REVIEWS | DRUG DISCOVERY VOLUME 6 | NOVEMBER 2007 | 881
ANALYSIS
© 2007 Nature Publishing Group