M
AY
2005
BioExeutive International
23
by Wayne Koberstein,
editor in chief
This scrappy little company has swiftly
boot-strapped its way up as it develops
a potential breakthrough in systems
biology--combining gene-based
science with chemistry for far-reaching
therapeutic applications.
I
t's small. It's growing fast. It's
raising and burning money
simultaneously. It's building
a technology platform rooted
firmly in the biological sciences.
It must be a biotech company, right?
Though the answer may seem obvi-
ous, it is unlikely to be unanimous.
From all corners of this industry, many
different answers come, as varied as
the many definitions of biotech. (See
"What Is a Biopharmaceutical? Part 2"
on page XX.) To some, only the pur-
est players in proteins may apply for
elite bio-status. To many others, the
inclusion rule must leave room for
biotech/pharmaceutical overlaps in
products and business models.
Like many companies that consider
themselves biotechs, PTC Therapeutics
(www.ptcbio.com) of South Plainfield,
NJ, sees no contradiction in applying
traditional pharmaceutical tools to
address processes at the core of cellular
biology. Nor does it mind bringing in
business and science expertise from
the traditional pharma side. Yet by
size, science, and culture, the com-
pany feels right at home in the biotech
community.
PTC's compounds selectively
modulate post-transcriptional control
mechanisms (PTCMs) to induce the
production of proteins in address-
ing genetic disorders, oncology, and
infectious diseases. PTC is also in
discussions to establish drug discov-
ery collaborations using its GEMS
(gene-expression modulation by small
molecules) technology. GEMS has
been successful identifying orally bio-
available small molecules, according
to the company. Thus, PTC's strategy
goes beyond using small molecules
to up- or down-regulate protein pro-
duction. For our study of this small,
self-coined "biopharmaceutical"
company--on a steep growth curve
to keep pace with its scientific prog-
ress--we interviewed the top members
of its management team: Founding
President and CEO Stuart Peltz, PhD;
CMO Langdon Miller, MD; and COO
William Ju, MD; as well as senior vice
presidents and vice presidents John
Babiak (drug discovery technologies),
Joseph Colacino (drug discovery),
Neil Almstead (chemistry), William
Baird (finance and strategic planning),
Cláudia Hirawat (corporate develop-
ment), and Mark Boulding (business
development and legal). Together,
they tell a remarkable story of entre-
preneurial creativity in building a
company from the ground up and in
short order--a story our readers may
well identify with and learn from.
O
FF
THE
G
ROUND
PTC Therapeutics scientists have led
the way in delineating the steps in
their entrepreneurial pathway with
a precision only biotechnology can
achieve. The company's lead product,
PTC124, now in phase 1 trials for
Duchenne muscular dystrophy (DMD)
and cystic fibrosis (CF), aims at one
particular type of genetic mutation:
nonsense mutations. The FDA has
granted orphan-drug status to PTC124
in the treatment of CF and DMD
caused by a nonsense mutation.
And the company announced on
March 31st that the agency had
designated its CF indication fast-
track status for review.
PTC124 restores protein produc-
tion inappropriately interrupted by
nonsense mutations. All genetic infor-
mation is contained in the DNA, in the
nucleus of the cell. For any gene to be
expressed, a copy of the relevant sec-
tion of the DNA is made, which is the
messenger RNA (mRNA). This process
of copying DNA into mRNA is called
transcription. The mRNA is sent to the
cytoplasm and is decoded by the ribo-
somes to make the specific proteins
it spells out in the mRNA--a process
known as translation. So a linear
process transcribes the genetic code
IN BRIEF
Company: PTC Therapeutics
Founded: 1998
Locations: South Plainfield, NJ
Employees (current): 97
Cash: $25 million
Burn: $2 million/month
Products in Development: PTC124
for Duchenne muscular dystrophy
and cystic fibrosis; PTC299 for cancer;
GEMS (gene-expression modulation
by small molecules) discovery
technology
