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PTC Therapeutics - ptcmay 2005 (Page 12)

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PTC Therapeutics - ptcmay 2005
BioExeutive International
chemists and the biologists were
mixed together in the lab space, and
even today they're all sitting in the
same offices and talking to each other
everyday. That's different than a larger
organization with its vast separation
between disciplines."
"And it's not just chemistry and
biology," adds Peltz. "Business develop-
ment and legal has to come in, clinical
in a very regulated environment versus
discovery in a sort of wild west envi-
ronment. To put those all together and
have it stick without exploding means
everybody's got to move over a little so
that everybody lives happily together."
To be fair to large companies,
however, some traditions in phar-
maceutical R&D lend themselves to
integrating disciplines--though per-
haps less extensively than PTC's ideal,
in Langdon's view. "People in chem-
istry, biology, the discovery, and the
preclinical groups have had the experi-
ence of working in other companies
where bringing the key concepts to a
compound, the product, was a multi-
disciplinary and integrated process.
They have been primed to know that
they can't work in isolation. But many
people who have worked on multidis-
ciplinary teams before probably went
through the period where they were in
silence, but then realized that to suc-
ceed they would have to take a more
involved approach."
Hirawat points to a cultural advan-
tage in the company's shoulder-rubbing
structure: "It is unique that the disci-
plines were folded together here right
from the beginning--from biology and
chemistry sharing offices and labs to
the very early focus on being project-
based as opposed to discipline-based.
As the leading example, Stuart never
thought that what he was doing was
necessarily more important than what
anyone else was doing. That kind of
sentiment carried through the com-
pany. No one thinks that pharmacology
is more important than chemistry--it's
just part of an overall project."
Putting traditional pharmaceutical
people into the more entrepreneurial
environment at PTC, in effect, liberated
them to practice the collaboration their
former companies preached widely but
practiced awkwardly. That, according
to Ju, made his operations-building job
easier--a true scaling-down to a more
effective level, he says.
"First comes assembling the proj-
ect team itself, and then creating a
governance structure. So you have the
line managers who supply the func-
tional expertise, the project teams that
actually do the work, a governance
structure, all in a matrix organization.
With those elements in place here, it
was really more a question of scale. We
realized that with this slimmed down
organization, we can actually get what
we got done in big pharma without so
many people."
Ju also says that a critical qualita-
tive factor--that the company's mix
of disciplines would change as it
grew--guided the creation of PTC's
scaled-down matrix. "We had to be
sensitive about the company evolv-
ing from a discovery organization to
one that would be integrated, with
research and development and all the
other aspects of a complete company.
Because of the quality and the collab-
orative nature of the people recruited
here, we were able to make that transi-
tion very quickly."
Leave it to Peltz, the lead scientist
(and CEO), to underline business
education as a chief integrator of the
young company. "Training people
in the business was critical," he says.
"You may be doing great science, but
doing great science alone won't get
you a sustainable platform. What you
need are business and products. That
requires getting people to realize that
you can do great science and still make
Even the earliest stages of business
development demanded universal
involvement of the company team,
Peltz recalls. "At all levels, everyone
understood the requirements of
growth, not just in purely scientific
terms, but also in business terms."
Most readers by now will have made
up their minds on the question, "Is
it Bio or Pharma?" in the case of
PTC Therapeutics and others like it.
If it's bio to you, identification with
the protagonist should come easy. If
it's pharma, take a deep breath and
perhaps draw in some useful lessons.
Foremost among them will be the
way the company mixes science and
business--or rather, spins them in
a creative feedback loop so that one
always drives the other on.
It may be that the best examples
of that are the very ones that set PTC
apart from "pure" biopharma, not
those that bring it closer. Baird calls
attention to the company's progress in
product development as a less famil-
iar but more fruitful area of study for
many biopharma companies. Part of
that was the early acquisition of an
in-house legal counsel to establish a
strong IP and contracting portfolio.
"We did three to five agreements a
week back then--CDAs, MTAs, service
agreements, consulting agreements,
manufacturing agreements, clinical
trials agreements, and so on,"
Baird says.
Even so, whatever the business case,
it may turn out that science ultimately
leads this company and all others in this
sphere. Would it have made scientific
sense for PTC to take a protein-based
rather than small-molecule product
approach to the pathway it pioneered?
Despite the perceived business advan-
tage of raising money as a pure player
in the biotech community, what pos-
sible benefit would that have brought?
If it seems wrong to let companies like
PTC share the special bio mantle, it
should seem even more unfair to brand
them as "drug companies" in the tradi-
tion of Big Pharma.
For statistical reasons, a strict
process-based definition of
biopharmaceuticals may be positive
or even necessary. But from a business
point of view, it might pay to loosen
up a little. Sometimes the mainstream
finds it has the most to learn from its
supposed fringes.

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