- Potential New Treatment Paradigm, PTC124 -
CAMBRIDGE, Mass. and SOUTH PLAINFIELD, N.J., July 17 /PRNewswire-
FirstCall/ -- Genzyme Corporation (Nasdaq: GENZ) and PTC Therapeutics, Inc.
(PTC) today announced an exclusive global collaboration to develop and
commercialize PTC124, PTC's novel oral therapy in late-stage development
for the treatment of genetic disorders due to nonsense mutations.
Under the terms of the agreement, PTC will commercialize PTC124 in the
United States and Canada, and Genzyme will commercialize the treatment in
all other countries. Genzyme will make an up-front payment of $100 million
to PTC, plus potential milestone and royalty payments. PTC will be
financially responsible for one ongoing and three additional clinical
trials of PTC124, which is potentially applicable to hundreds of genetic
diseases.
PTC124 is currently being evaluated in a phase 2b trial for Duchenne
muscular dystrophy (DMD), and a phase 2b trial in cystic fibrosis (CF) is
expected to begin by the end of this year. In its collaboration with PTC,
Genzyme will draw on its expertise in genetic disorders and its strong
regulatory, manufacturing and marketing infrastructure outside of the
United States. Genzyme has extensive experience with cystic fibrosis,
having conducted more than six clinical trials among CF patients. In the
field of DMD, Genzyme's experience with Myozyme(R) (alglucosidase alfa),
for the treatment of the genetic disorder Pompe disease, will be directly
applicable as patients with both diseases are treated by the same
specialist physicians.
"Over the past two decades, Genzyme has successfully developed four
therapies for patients with severe genetic diseases. PTC124 is a powerful
new approach that holds great potential to help CF and DMD patients, and
many others with a variety of devastating diseases," stated Henri A.
Termeer, Genzyme's chairman and chief executive officer. "This
collaboration is an excellent strategic fit for Genzyme and will be managed
within the company's stated financial guidance."
"One of PTC's earliest scientific insights was that targeting nonsense
mutations represented a novel approach to treating a large number of
genetic disorders. The translation of that insight through the discovery
and rapid development of PTC124 has been very gratifying," commented Stuart
W. Peltz, Ph.D., PTC's president and chief executive officer. "This
collaboration supports PTC's business strategy of establishing a fully
integrated biopharmaceutical company by retaining commercial rights in the
United States and Canada while engaging an experienced and capable partner
to swiftly address additional markets."
PTC initiated the clinical development of PTC124 in 2004. Based on
phase 2a clinical proof of concept in both DMD and CF, further development
in each of these indications is being pursued in international, multicenter
trials. A phase 2b trial of PTC124 in DMD is currently enrolling, and is
expected to include 165 patients. A phase 2b trial of PTC124 in CF is
planned to begin by the end of this year. With demonstration of clinical
benefit, these two trials are expected to serve as the basis for
registration of PTC124 in these indications. Further development of PTC124
will include clinical trials in multiple additional genetic disorders.
In the United States, there are an estimated 10,000 DMD patients,
approximately 13 percent of which have nonsense mutations. Of the more than
30,000 U.S. patients with CF, about 10 percent have nonsense mutations.
There is a significant unmet medical need for new treatments for these
diseases. In addition to DMD and CF, the companies plan to explore PTC124's
potential to make a difference for patients with other types of severe and
debilitating genetic diseases.
"We are impressed by the quality of the preliminary PTC124 data, which
suggest broad applicability to a large number of genetic disorders," said
Geoff McDonough, M.D., Genzyme's senior vice president and general manager
of LSD Therapeutics. "PTC124 is an excellent example of the promise that
personalized medicine holds to address significant unmet medical needs, and
we are excited about its potential to make a major positive difference in
the lives of patients and their families."
"We are delighted to enter into this collaboration with Genzyme, a
world- recognized pioneer and leader in the development of treatments for
genetic disorders," commented Claudia Hirawat, PTC's senior vice president
of corporate development. "Because of its novel mechanism of action, PTC124
has the potential to address the underlying cause of disease in a subset of
patients affected by more than 2,400 rare genetic disorders. PTC and
Genzyme are well suited as partners to realize the full potential inherent
in the broad applicability of PTC124."
Deal Terms
Under the terms of the agreement, Genzyme will make a $100 million up-
front payment to PTC Therapeutics. PTC will conduct and be financially
responsible for the phase 2b trial of PTC124 in DMD, the phase 2b trial in
CF, and two proof-of-concept studies in other indications to be determined.
Once these four studies are completed, the companies will share research
and development costs equally. Genzyme and PTC will each bear the sales,
marketing and other costs associated with commercialization of PTC124 in
their respective territories.
PTC is eligible to receive up to $337 million in total milestone
payments, as follows: up to $165 million in development and approval
milestones, the majority of which are to be paid upon approvals in Genzyme
territories; and up to $172 million in sales milestones, contingent upon
the achievement of specific sales levels. The sales milestone payments
begin when annual net revenues reach $300 million, and increase in
increments through revenues of $2.4 billion. PTC is also eligible to
receive tiered double-digit royalties from sales in Genzyme territories.
About PTC124
PTC124 is an orally delivered, investigational new small molecule drug
for the treatment of genetic disorders due to nonsense mutations. Nonsense
mutations are single-point alterations in the genetic code that prematurely
stop the translation process, preventing production of a full-length,
functional protein. In phase 2a clinical trials in
nonsense-mutation-mediated cystic fibrosis and in
nonsense-mutation-mediated Duchenne muscular dystrophy, PTC124 has
demonstrated the ability to produce functional protein across a variety of
nonsense mutation types.
Across all clinical studies to date, PTC124 has been generally well
tolerated and has achieved target plasma concentrations associated with
activity in preclinical models. PTC124 is currently in phase 2b development
with the goal of demonstrating that increasing functional protein levels in
patients with nonsense-mediated genetic disorders will provide clinical
benefits.
PTC124 has been granted orphan drug status for the treatment of DMD and
CF due to nonsense mutations by the FDA and the European Commission. The
FDA has also granted PTC124 Subpart E designation for expedited
development, evaluation and marketing. The development of PTC124 is
supported by grants from the Cystic Fibrosis Foundation, the Muscular
Dystrophy Association, Parent Project Muscular Dystrophy, FDA's Office of
Orphan Products Development and by General Clinical Research Center grants
from the National Center for Research Resources.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is characterized by rapid progression of
muscle degeneration, eventually leading to loss in ambulation, paralysis,
and death. DMD eventually affects all voluntary muscles, as well as the
heart and breathing muscles, and patients rarely survive beyond their early
30s. Each year, approximately 20,000 children worldwide are born with DMD
(one of every 3,500 male children), making it the most prevalent of
muscular dystrophies. There is a commercially available test to determine
whether a patient's DMD is caused by a nonsense mutation. More information
on DMD is available through the Muscular Dystrophy Association
(http://www.mdausa.org) and the Parent Project Muscular Dystrophy
(http://www.parentprojectmd.org).
About Cystic Fibrosis
Cystic fibrosis affects the mucus glands of the lungs, liver, pancreas,
and intestines, causing progressive disability due to multisystem failure.
It is among the most common life-threatening genetic disorders, affecting
nearly 70,000 people worldwide. There is a commercially available test to
determine whether a patient's CF is caused by a nonsense mutation. More
information regarding CF is available through the Cystic Fibrosis
Foundation (http://www.cff.org).
About Genzyme
One of the world's leading biotechnology companies, Genzyme is
dedicated to making a major positive impact on the lives of people with
serious diseases. Since 1981, the company has grown from a small start-up
to a diversified enterprise with more than 10,000 employees in locations
spanning the globe and 2007 revenues of $3.8 billion. In 2007, Genzyme was
chosen to receive the National Medal of Technology, the highest honor
awarded by the President of the United States for technological innovation.
With many established products and services helping patients in nearly
90 countries, Genzyme is a leader in the effort to develop and apply the
most advanced technologies in the life sciences. The company's products and
services are focused on rare inherited disorders, kidney disease,
orthopaedics, cancer, transplant, and diagnostic testing. Genzyme's
commitment to innovation continues today with a substantial development
program focused on these fields, as well as immune disease, infectious
disease, and other areas of unmet medical need.
Genzyme's press releases and other company information are available at
http://www.genzyme.com and by calling Genzyme's investor information line at
1-800- 905-4369 within the United States or 1-678-999-4572 outside the
United States.
About PTC Therapeutics Inc.
PTC is a biopharmaceutical company focused on the discovery,
development and commercialization of orally administered, proprietary,
small-molecule drugs that target post-transcriptional control processes.
Post- transcriptional control processes regulate the rate and timing of
protein production and are of central importance to proper cellular
function. PTC's internally-discovered pipeline addresses multiple
therapeutic areas, including genetic disorders, oncology and infectious
diseases. PTC has extensive knowledge of post-transcriptional control
processes and has developed proprietary technologies that it applies in its
drug discovery activities, including the Gene Expression Modulation by
Small-molecules (GEMS) technology, which has been the basis for
collaborations with leading biopharmaceutical companies such as Pfizer,
Celgene, CV Therapeutics and Schering-Plough. For more information, visit
the company's website http://www.ptcbio.com.
PTC Therapeutics(R) and PTC124(TM) are trademarks of PTC Therapeutics,
Inc. All rights reserved.
Genzyme Safe Harbor Statement
This press release contains forward-looking statements, including
without limitation, statements regarding the commercialization of PTC124;
the benefits and potential broad applicability of PTC124 to treat multiple
diseases; the development plan for PTC124 and expectations regarding phase
2b trials of PTC124 in Duchenne muscular dystrophy and cystic fibrosis; and
the management of the collaboration within Genzyme's stated financial
guidance. These statements are subject to risks and uncertainties that
could cause actual results to differ materially from those projected in
these forward-looking statements. These risks and uncertainties include,
among others, the ability of Genzyme and PTC to successfully complete the
necessary clinical research on PTC124, including the risk that PTC124 will
not meet its expected clinical endpoints; the parties' ability to obtain
and maintain the necessary regulatory approvals for PTC124 in the expected
indications and the timing of those approvals; the actual safety and
efficacy of PTC124; the parties' ability to manufacture and commercialize
PTC124; the possibility that other companies will seek to enter the same
market or markets; the availability and extent of reimbursement for PTC124;
and the risks and uncertainties described in reports filed by Genzyme with
the Securities and Exchange Commission under the Securities Exchange Act of
1934, as amended, including without limitation the information under the
heading "Risk Factors" in the Management's Discussion and Analysis of
Financial Condition and Results of Operations section of the Genzyme
Quarterly Report on Form 10-Q for the quarter ended March 31, 2008. Genzyme
cautions investors not to place substantial reliance on the forward-looking
statements contained in this press release. These statements speak only as
of the date of this press release, and Genzyme undertakes no obligation to
update or revise the statements.
Genzyme(R) is a registered trademark of Genzyme Corporation. All rights
reserved.
Genzyme Contacts
Patrick Flanigan (Investors) Erin Emlock (Media)
617-768-6563 617-768-6923
PTC Contacts
Jane Baj (Investors and Media) Andrea Johnston (Investors and Media)
PTC Therapeutics, Inc. Pure Communications
(908) 912-9167 (910) 616-5858
jbaj@ptcbio.com andrea@purecommunicationsinc.com
Diane Goetz (Patients, Patients' Families,
Investigators and Patient Organizations)
PTC Therapeutics, Inc.
(908) 912-9256
patientinfo@ptcbio.com
SOURCE Genzyme Corporation and PTC Therapeutics Inc.
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CONTACT:
Investors, Patrick Flanigan, +1-617-768-6563,
or Media, Erin Emlock, +1-617-768-6923, both of Genzyme
Corporation; or Jane Baj (Investors and Media), PTC Therapeutics,
Inc., +1-908-912-9167, jbaj@ptcbio.com; or Andrea Johnston
(Investors and Media), Pure Communications, +1-910-616-5858,
andrea@purecommunicationsinc.com, for PTC Therapeutics, Inc.; or
Diane Goetz (Patients, Patients' Families, Investigators and
Patient Organizations), PTC Therapeutics, Inc., +1-908-912-9256,
patientinfo@ptcbio.com
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