PTC Therapeutics and Genzyme Announce Restructuring of Collaboration
- PTC Regains Worldwide Rights to Ataluren in Duchenne and Becker Muscular Dystrophies -
SOUTH PLAINFIELD, NJ AND CAMBRIDGE, MA – SEPTEMBER 2, 2011 – PTC Therapeutics, Inc. and Genzyme, a Sanofi company, announced today the restructuring of their collaboration. Under the original agreement, PTC held commercial rights for the U.S. and Canada and Genzyme held commercial rights in all other countries. Under the restructured agreement, PTC regains worldwide rights to ataluren and Genzyme retains an option to commercialize ataluren in indications other than nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) outside the U.S. and Canada. PTC will continue to conduct worldwide development in all indications with a near-term focus on late-stage programs in nmDBMD and nonsense mutation cystic fibrosis.
"We have been very pleased with our long-standing relationship with PTC," said David Meeker, M.D., Chief Operating Officer, Genzyme. "The decision to restructure this agreement is a result of a recent portfolio assessment, and will enable us to intensify our focus on other areas of development within Genzyme where we believe we have the best ability to develop therapies to benefit the rare disease community. However, our option to reengage the collaboration reflects our belief in the potential of this approach for the treatment of nonsense mutation genetic disorders. While we will no longer be involved in advancing ataluren in nmDBMD, we remain committed to the neuromuscular community."
"PTC and Genzyme share a strong commitment for bringing treatments to patients with rare and life-threatening genetic disorders," said Stuart Peltz, Ph.D., President and Chief Executive Officer, PTC Therapeutics, Inc. "We have benefited greatly from our partnership with Genzyme and we look forward to rapidly advancing the development of ataluren."
ABOUT ATALUREN
An investigational new drug discovered by PTC Therapeutics,
ataluren is a protein restoration therapy designed to enable the
formation of a functioning protein in patients with genetic
disorders caused by a nonsense mutation. A nonsense mutation is an
alteration in the genetic code that prematurely halts the synthesis
of an essential protein. The resulting disorder is determined by
which protein cannot be expressed in its entirety and is no longer
functional, such as dystrophin in nmDBMD. The development of
ataluren has been supported by grants from Cystic Fibrosis
Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic
Fibrosis Foundation); Muscular Dystrophy Association; FDA's Office
of Orphan Products Development; National Center for Research
Resources; National Heart, Lung, and Blood Institute; and Parent
Project Muscular Dystrophy.
ABOUT PTC THERAPEUTICS, INC.
PTC is a biopharmaceutical company focused on the discovery,
development and commercialization of orally administered
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 rare genetic
disorders, oncology and infectious diseases. PTC has developed
proprietary technologies that it applies in its drug discovery
activities and has served as the basis for collaborations with
leading biopharmaceutical companies. For more information, visit
the company's web site at www.ptcbio.com.
ABOUT GENZYME, A SANOFI COMPANY
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 its founding in 1981, the company has
introduced breakthrough treatments that have provided new hope for
patients. The company's areas of focus are rare genetic diseases,
multiple sclerosis, cardiovascular disease, and endocrinology.
Genzyme is a Sanofi company. Genzyme's press releases and other
company information are available at www.genzyme.com.
FOR MORE INFORMATION:
Investor and Media
Jane Baj Sheryl Seapy
PTC Therapeutics, Inc. Pure Communications
(908) 912-9167 (949) 608-0841
jbaj@ptcbio.com sheryl@purecommunicationsinc.com
Media contact: Investor contact:
Lori Gorski Kristen Galfetti
Genzyme Sanofi US
(617) 768-9344 (617) 768-6563
Lori.gorski@genzyme.com Kristen.Galfetti@Sanofi.com
Posted: September 2011
