28 BioExecutive International
M
AY
2005
PTC124 boosted dystrophin produc-
tion. Having passed human safety
tests, the compound is heading for
phase 2 orphan-drug trials this
year in both DMD and CF. Both
development programs for PTC124
have won the support of their respec-
tive patient groups, such as the
Muscular Dystrophy Association, the
Parent Project Muscular Dystrophy
(PPMD) and the Cystic Fibrosis
Foundation (CFF). CFF's therapeutics
research affiliate granted PTC a
$1.7 million grant for developing the
CF indication.
Such support has become a critical
component of the development pro-
grams in other ways as well. PTC says
it has always prioritized efforts to form
close relations with patients, families,
and physicians--relations that already
inform the design of clinical programs.
And the same connections may pay off
later when those constituents, along
with payers, make choices about a
premium priced therapy in the context
of its therapeutic value.
Besides the compelling need for
better treatment, DMD and CF present
the other key criteria for development
targets: relatively small patient popula-
tions and specialist audiences. Thus
research, development, marketing,
and production can all proceed on a
scale small enough for the fledgling
company. PTC will likely form its own
sales force for PTC124's initial indica-
tions and has already manufactured
enough of the compound for full
clinical development. It has similar
plans for its other pipeline products,
including the most advanced candi-
dates: anti-angiogenesis compound
PTC299 and hepatitis C inhibitors.
Many of the compounds target differ-
ent PTCMs spanning a variety of areas
and indications.
Building on that scale, together
with potential for much wider applica-
tion of the core technology over time,
has proved an attractive business plan
for investors, as Peltz explains:
"We were lucky enough to get
investors who understood that our
project would take a little bit lon-
ger term than licensing in a phase 3
compound and trying to turn it into
a company. With the first investment
in 2000, we had an idea of something
and a little bit of data, but nothing that
would go to market rapidly. So the
dream that we were talking to people
about was to take advantage of this
new area in biology. Don't necessarily
bet on any individual program; bet on
our execution of our efforts to target
new biology. And that's what we did."
Peltz adds that the company has
stuck close by that plan, with evident
results thus far:
"PTC124 is a first-in-class drug
for diseases of high medical need
but small enough markets so we can
be in a position to commercialize
it ourselves. Following PTC124, we
have oncology, infectious diseases,
inflammation, and other programs
percolating up. We are also working
on our first business development
deal this year. So the combination
of product, technology, and pipeline
makes us a top-tier organization with
new products for years to come."
P
ARTS
A
SSEMBLY
Strong science, business plans, and
investment are all very nice, of course.
But it is thereafter that the fun really
begins. For all new companies,
building and operating an effective
organization is where the rubber
meets the road--and, unfortunately,
for many of them it is also where the
rubber leaves the road. Peltz and his
team have so far shown every sign of
meeting that challenge.
They have put together an appar-
ently well-oiled machine from many
disparate parts--including themselves.
As the top managers join Peltz in his
office for this interview, they seem like
jovial astronauts, each from a differ-
ent planet, all tossed into a common
adventure. (See Biographies.)
Actually, two of them come from
the same planet--the late, great
Pharmacia--CMO Langdon Miller
and COO William Ju. Finance and
strategic planning VP William
Baird hails from LEK Consulting;
Corporate development VP Claudia
Hirawat, from recruiter Ledbetter-
Stevens; and business development
and legal senior VP Mark Boulding,
from MedicaLogic/Medscape. Other
executives who were not present at the
interview include John Babiak, senior
VP of drug discovery technologies,
from Pharmacopeia and Wyeth-Ayerst;
Neil Almstead, PhD, VP of chemistry,
from Proctor & Gamble; and Joseph
Colacino, PhD, VP of drug discovery,
from Eli Lilly.
PTC's six-member scientific
advisory board brings additional dis-
ciplines to the team. Cofounder Allan
Jacobson is chairman of the molecular
you have to look pretty hard, and you
have to know your product pretty well.
And most of the time, the products
have some warts associated with them.
Otherwise, why would people be selling
them? And then from an investor point
of view, what's the pipeline that will
continue growth? What we're trying to
build is a product focused company,
and we have products in the pipeline.
BIOGRAPHY: Affectionately known
as "Stu" among his close colleagues,
Dr. Peltz cofounded PTC Therapeutics
in 1998. Since then, he has served as
the company's "champion," leading the
science, raising the funds, and managing
the fast-growing enterprise. Peltz began
his career as a cancer researcher at
the University of Wisconsin's McArdle
Laboratory for Cancer Research, where
he earned his PhD. Before PTC, he had
already won widespread recognition for
his groundbreaking investigations into
post-transcriptional control processes,
which became the core science of PTC
Therapeutics. He helped develop the first
biochemical assays for exploring how
those processes control mRNA turnover
and cellular protein production. His work
subsequently led to the elucidation of all
components in the post-transcriptional
control pathway. His publications are
many and well-known, and he serves on
the review committees of the National
Institutes of Health and the American
Cancer Society.
DIALOGUE
CONTINUED
