Jones Lang LaSalle
PTC, CV In Potential $345M Cardiovascular Agreement
By Karen Pihl-Carey
Senior Staff Writer
Less than three months after signing its first major preclinical deal focused on infectious disease, PTC Therapeutics Inc. topped it with a new cardiovascular collaboration worth up to $345 million with CV Therapeutics Inc.
South Plainfield, N.J.-based PTC will apply its Gene Expression Modulation by Small-Molecules (GEMS) technology to develop orally bioavailable small molecules. Together, the companies will select five targets, including those known to raise HDL and be involved in dyslipidemia and diabetes.
"The technology can be applied virtually to any therapeutic area," said Claudia Hirawat, the company's senior vice president of corporate development. "This is our first effort into addressing a therapeutic area where we don't have internal expertise."
While the main focus of the collaboration will be on cardiovascular indications, PTC does have the freedom to "dabble into other areas," she said.
CV Therapeutics, of Palo Alto, Calif., will pay PTC $10 million up front, consisting of $2 million in cash and two loans of $8 million total. One loan is forgivable as long as the research remains in effect, and the other loan is convertible into PTC equity.
If CV Therapeutics chooses to take certain compounds into development, it has an exclusive option to gain worldwide, exclusive rights for each or several targets.
If exercised, PTC would receive royalties on worldwide net sales. Assuming all five targets are selected by CV Therapeutics, PTC could earn up to $335 million in milestone payments based on development, regulatory and commercial achievements. It also retains an option to co-fund research and development in return for increased royalties or co-promotion rights.
The licensing deal with CV Therapeutics is part of PTC's strategy to leverage its GEMS technology outside of its core research areas, which include genetic disorders, oncology and infectious diseases.
Late last year, the company licensed certain compounds from its anti-angiogenesis program to Bausch & Lomb, of Rochester, N.Y., for use in ophthalmology indications. That agreement is in line with the same strategy, although it is not a discovery collaboration like the one with CV Therapeutics. "We had already identified those compounds as part of our oncology screen," Hirawat said.
In March, PTC signed a deal potentially worth $200 million with Kenilworth, N.J.-based Schering-Plough Corp. focused on small-molecule candidates that inhibit the hepatitis C virus internal ribosome entry site (IRES)-mediated production of viral proteins. Schering is responsible for clinical development and worldwide commercialization. (See BioWorld Today, March 21, 2006.)
Internally, PTC is advancing PTC124 in three Phase II trials for genetic disorders, including Duchenne's muscular dystrophy and cystic fibrosis in cases in which a nonsense mutation is the cause of the diseases. The drug has orphan status for both indications.
Interim results of the cystic fibrosis trial, announced in April, suggested that PTC124 might have pharmacological activity that addresses the underlying cause of CF in patients with a nonsense mutation. Both the CF and DMD trials are expected to be completed in the second half of this year. The DMD nonsense mutation, which occurs in about 15 percent of the DMD cases, is found in the dystrophin gene. About 10 percent of the cystic fibrosis cases are caused by a nonsense mutation of the CF transmembrane regulator gene.
PTC also has its anti-angiogenic compound PTC299 - an oral inhibitor of vascular endothelial growth factor - in Phase I testing for cancer. Derived from the GEMS screening technology, it modulates gene expression by targeting the post-transcriptional control processes that act through the untranslated regions of messenger RNA molecules.
"In a matter of four years, we went from a concept to being in Phase I," Hirawat said, adding that a major advantage of the GEMS technology is its ability to address targets like IRES that researchers are "not able to tackle with traditional drug discovery."
"With GEMS," she told BioWorld Today, "you can actually either up-regulate or down-regulate protein expression using small-molecule drugs. As far as we know, there's no other way to do that."
John Bluth, CV Therapeutics' senior director of corporate communications, said his company's interest in the technology is its ability to "accelerate the arrival of treatments to the market."
Among CV Therapeutics' approved products are Ranexa (ranolazine) for chronic angina and Aceon (perindopril erbumine) for coronary artery disease or hypertension. It also is developing regadenoson in Phase III trials as a pharmacologic stress agent in myocardial perfusion imaging studies, and it is conducting a study under a special protocol assessment agreement with the FDA to gain approval of Ranexa as first-line chronic angina therapy.
The company went public in 1996. Its stock (NASDAQ:CVTX) fell 77 cents Monday to close at $14.16.
Founded in 1998, PTC raised $26.6 million in a private placement in November, bringing the total venture capital and grant money brought in since inception to $139 million. The money was expected to last the firm through early 2007.
In April, PTC filed for an initial public offering to raise $86.3 million.
Published June 13, 2006
PTC, CV In Potential $345M Cardiovascular Agreement
By Karen Pihl-Carey
Senior Staff Writer
Less than three months after signing its first major preclinical deal focused on infectious disease, PTC Therapeutics Inc. topped it with a new cardiovascular collaboration worth up to $345 million with CV Therapeutics Inc.
South Plainfield, N.J.-based PTC will apply its Gene Expression Modulation by Small-Molecules (GEMS) technology to develop orally bioavailable small molecules. Together, the companies will select five targets, including those known to raise HDL and be involved in dyslipidemia and diabetes.
"The technology can be applied virtually to any therapeutic area," said Claudia Hirawat, the company's senior vice president of corporate development. "This is our first effort into addressing a therapeutic area where we don't have internal expertise."
While the main focus of the collaboration will be on cardiovascular indications, PTC does have the freedom to "dabble into other areas," she said.
CV Therapeutics, of Palo Alto, Calif., will pay PTC $10 million up front, consisting of $2 million in cash and two loans of $8 million total. One loan is forgivable as long as the research remains in effect, and the other loan is convertible into PTC equity.
If CV Therapeutics chooses to take certain compounds into development, it has an exclusive option to gain worldwide, exclusive rights for each or several targets.
If exercised, PTC would receive royalties on worldwide net sales. Assuming all five targets are selected by CV Therapeutics, PTC could earn up to $335 million in milestone payments based on development, regulatory and commercial achievements. It also retains an option to co-fund research and development in return for increased royalties or co-promotion rights.
The licensing deal with CV Therapeutics is part of PTC's strategy to leverage its GEMS technology outside of its core research areas, which include genetic disorders, oncology and infectious diseases.
Late last year, the company licensed certain compounds from its anti-angiogenesis program to Bausch & Lomb, of Rochester, N.Y., for use in ophthalmology indications. That agreement is in line with the same strategy, although it is not a discovery collaboration like the one with CV Therapeutics. "We had already identified those compounds as part of our oncology screen," Hirawat said.
In March, PTC signed a deal potentially worth $200 million with Kenilworth, N.J.-based Schering-Plough Corp. focused on small-molecule candidates that inhibit the hepatitis C virus internal ribosome entry site (IRES)-mediated production of viral proteins. Schering is responsible for clinical development and worldwide commercialization. (See BioWorld Today, March 21, 2006.)
Internally, PTC is advancing PTC124 in three Phase II trials for genetic disorders, including Duchenne's muscular dystrophy and cystic fibrosis in cases in which a nonsense mutation is the cause of the diseases. The drug has orphan status for both indications.
Interim results of the cystic fibrosis trial, announced in April, suggested that PTC124 might have pharmacological activity that addresses the underlying cause of CF in patients with a nonsense mutation. Both the CF and DMD trials are expected to be completed in the second half of this year. The DMD nonsense mutation, which occurs in about 15 percent of the DMD cases, is found in the dystrophin gene. About 10 percent of the cystic fibrosis cases are caused by a nonsense mutation of the CF transmembrane regulator gene.
PTC also has its anti-angiogenic compound PTC299 - an oral inhibitor of vascular endothelial growth factor - in Phase I testing for cancer. Derived from the GEMS screening technology, it modulates gene expression by targeting the post-transcriptional control processes that act through the untranslated regions of messenger RNA molecules.
"In a matter of four years, we went from a concept to being in Phase I," Hirawat said, adding that a major advantage of the GEMS technology is its ability to address targets like IRES that researchers are "not able to tackle with traditional drug discovery."
"With GEMS," she told BioWorld Today, "you can actually either up-regulate or down-regulate protein expression using small-molecule drugs. As far as we know, there's no other way to do that."
John Bluth, CV Therapeutics' senior director of corporate communications, said his company's interest in the technology is its ability to "accelerate the arrival of treatments to the market."
Among CV Therapeutics' approved products are Ranexa (ranolazine) for chronic angina and Aceon (perindopril erbumine) for coronary artery disease or hypertension. It also is developing regadenoson in Phase III trials as a pharmacologic stress agent in myocardial perfusion imaging studies, and it is conducting a study under a special protocol assessment agreement with the FDA to gain approval of Ranexa as first-line chronic angina therapy.
The company went public in 1996. Its stock (NASDAQ:CVTX) fell 77 cents Monday to close at $14.16.
Founded in 1998, PTC raised $26.6 million in a private placement in November, bringing the total venture capital and grant money brought in since inception to $139 million. The money was expected to last the firm through early 2007.
In April, PTC filed for an initial public offering to raise $86.3 million.
Published June 13, 2006
posted by Jones Lang LaSalle NJ News Blog at 12:10 PM
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