Under the terms of the agreement, PTC will commercialize PTC124 in
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
"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
"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
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
"We are impressed by the quality of the preliminary PTC124 data, which
suggest broad applicability to a large number of genetic disorders," said
"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
Deal Terms
Under the terms of the agreement, Genzyme will make a
PTC is eligible to receive up to
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 (www.mdausa.org) and the Parent Project Muscular Dystrophy (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 (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
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
www.genzyme.com and by calling Genzyme's investor information line at 1-800-
905-4369 within
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 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
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.