Princeton, NJ – February 12, 2008 – Ophthotech Corp. (“Ophthotech”), a privately held biopharmaceutical company focused on developing ophthalmic therapies for back-of-the-eye diseases, today announced that the first patient has been enrolled in its Phase I clinical trial for the treatment of wet age-related macular degeneration (AMD). The Phase I trial will assess the safety and tolerability of E10030, an anti-PDGF aptamer, in combination with an anti-VEGF agent. This trial will enroll up to a maximum of 36 patients.
“The current treatment regimen for angiogenesis in AMD does not result in neovascular regression. The combination of anti-PDGF and anti-VEGF agents has been shown to cause neovascular regression, in both ocular and tumor angiogenesis preclinical models. We believe E10030 holds great promise for enhancing the visual outcome for patients with AMD,” said Samir Patel, M.D., President and CEO of Ophthotech Corp.
E10030 is the first of three compounds that Ophthotech Corp. is developing to treat AMD. Additional molecular entities include ARC1905, a complement (anti-C5) inhibitor, and volociximab, an anti-angiogenesis monoclonal antibody targeting α5β1 integrin.
About E10030 E10030, is an aptamer-based compound directed against PDGF-B. Pharmacology studies indicate that E10030 binds to PDGF-B with high specificity and affinity and inhibits the functions of PDGF-B both in vitro and in vivo. In preclinical studies, E10030 demonstrated the potential to regress neovascularization when used in combination with a VEGF-A inhibitor. In experiments involving models of ocular vascularization, concurrent inhibition of PDGF-B and VEGF-A signaling was superior to inhibition of the VEGF-A pathway alone.
About ARC1905 Anti-C5 aptamer ARC1905 inhibits C5, a central component of the complement cascade, which plays multiple roles in innate immunity and inflammatory diseases. Inhibition of this key step in the complement cascade at the level of C5 prevents the formation of key terminal fragments (C5a and C5b-9) regardless of which pathway (alternate, classical or lectin) induced their generation. The C5a fragment is an important inflammatory activator inducing vascular permeability, recruitment and activation of phagocytes. C5b-9 is involved in the formation of membrane attack complex (MAC: C5b-9) which initiates cells lysis. By inhibiting these C5-mediated inflammatory and MAC activities, therapeutic benefit may be achieved in both dry and wet AMD.
About Volociximab(M200) Volociximab is a monoclonal antibody targeting α5β1 integrin, a key protein involved in the formation of new blood vessels, a process known as angiogenesis. α5β1 integrin is a critical survival factor for proliferating endothelial cells involved in angiogenesis. Inhibition of α5β1 integrin has demonstrated potent anti-angiogenic effects in multiple pre-clinical models of angiogenesis.
About AMD AMD is the leading cause of blindness for people over the age of 50 in the United States and Europe. There are two forms of the disease, namely "dry" and "wet" AMD. The "wet" form is characterized by the growth of new blood vessels into the central region of the retina. These new vessels cause severe visual loss due to retinal damage caused by subsequent leakage and scar formation. Anti-VEGF therapies and photodynamic therapies have been approved for “wet” AMD. “Dry” AMD accounts for up to 90 percent of all cases of AMD. There is no approved therapy for "dry" AMD, which afflicts 8 million patients in the United States and an additional 8 million in Europe. Visual loss in "dry" AMD is typically not as severe as "wet" AMD, however, over time, dry AMD can progress to the wet form of the disease.
About Ophthotech Ophthotech Corp. is a privately held biopharmaceutical company focused on developing and commercializing therapies for back-of-the-eye diseases. Ophthotech plans to develop a pipeline of compounds with strong scientific foundations for the treatment of AMD and bring them to market in an accelerated manner. In August of 2007, Ophthotech announced a $36 million Series A venture financing and two separate in-licensing deals with Archemix Corp and (OSI) Eyetech, Inc. A third in-license from Biogen Idec and PDL BioPharma was announced in January of 2008. Ophthotech’s venture investors include SV Life Sciences, HBM BioVentures and Novo A/S. For more information, please visit www.ophthotech.com.
—Ophthotech to develop anti-angiogenesis antibody for Age-Related Macular Degeneration—
--Biogen Idec and PDL to receive future milestones and royalties--
Princeton, NJ, Cambridge, Mass., and Redwood City, Calif., January 8, 2008 – Ophthotech Corp. (“Ophthotech”), a privately held biopharmaceutical company focused on developing ophthalmic therapies for back-of-the-eye diseases, Biogen Idec Inc. (NASDAQ: BIIB) and PDL BioPharma, Inc. (NASDAQ: PDLI) today announced that they have entered into an exclusive worldwide licensing agreement for an anti-angiogenesis antibody to treat Age-Related Macular Degeneration (AMD). Under the terms of the agreement, Biogen Idec and PDL have granted Ophthotech worldwide development and commercial rights to all ophthalmic uses of volociximab (M200). Volociximab is an investigational monoclonal antibody targeting α5β1 integrin, a key protein involved in the formation of blood vessels, a process known as angiogenesis. Biogen Idec and PDL will each receive an equity position in Ophthotech as well as a combination of development milestone payments and royalties on future product sales. Other terms, including financial terms, related to the agreement have not been disclosed.
“α5β1 integrin is a critical survival factor for proliferating endothelial cells involved in angiogenesis,” said Samir Patel, MD, president and CEO of Ophthotech. "The preclinical studies to date provide very strong support for developing volociximab for ophthalmic indications. It represents a potential breakthrough for the treatment of AMD.”
"There remains a significant unmet need in treating AMD, and we're pleased that Ophthotech, founded by leaders in the development of new therapeutics for this disease, has chosen to explore the potential of volociximab in AMD," said Faheem Hasnain, executive vice president, oncology and rheumatology strategic business unit, Biogen Idec.
"We believe the anti-angiogenesis properties of volociximab, coupled with Ophthotech's expertise in developing ophthalmic therapies, provide an excellent opportunity to maximize volociximab's value," said L. Patrick Gage, PhD, PDL’s interim CEO. Separately, Biogen Idec and PDL are co-developing volociximab in solid tumor cancers and the companies retain rights in all other indications pursuant to their September 2005 collaboration agreement.
About Age-Related Macular Degeneration (AMD) AMD is the leading cause of blindness for people over the age of 55 in the United States and Europe. There are two forms of AMD, namely "dry" and "wet" AMD. The "wet" form is characterized by the growth of new blood vessels into the central region of the retina. These new vessels cause severe visual loss due to retinal damage caused by subsequent leakage and scar formation. Anti-VEGF therapies and photodynamic therapies have been approved for “wet” AMD. “Dry” AMD accounts for up to 90 percent of all cases of AMD. There is no approved therapy for "dry" AMD, which afflicts 8 million patients in the United States and an additional 8 million in Europe. Visual loss in "dry" AMD is typically not as severe as "wet" AMD, however, over time, dry AMD can progress to the wet form of the disease.
About Ophthotech Ophthotech Corp. is a biotechnology company focused on developing and commercializing therapies for back-of-the-eye diseases. Ophthotech plans to develop a pipeline of compounds with strong scientific foundations for the treatment of AMD and bring them to market in an accelerated manner. In August of 2007, Ophthotech announced a $36 million Series A venture financing and two separate in-licensing deals with Archemix Corp and (OSI) Eyetech, Inc. Ophthotech’s venture investors include SV Life Sciences, HBM BioVentures and Novo A/S. For more information, please visit www.ophthotech.com.
About Biogen Idec
Biogen Idec creates new standards of care in therapeutic areas with high unmet medical needs. Founded in 1978, Biogen Idec is a global leader in the discovery, development, manufacturing and commercialization of innovative therapies. Patients in more than 90 countries benefit from Biogen Idec's significant products that address diseases such as lymphoma, multiple sclerosis and rheumatoid arthritis. For product labeling, press releases and additional information about the company, please visit www.biogenidec.com.
About PDL BioPharma
PDL BioPharma, Inc. is a biopharmaceutical company focused on discovering, developing and commercializing innovative therapies for severe or life-threatening illnesses. For more information, please visit www.pdl.com.
NOTE: PDL BioPharma and the PDL BioPharma logo are considered trademarks of PDL BioPharma, Inc.
Forward-looking Statement This press contains forward-looking statements regarding the development of volociximab for ophthalmic indications. These statements are based on the companies' current beliefs and expectations. Drug development and commercialization involves a high degree of uncertainty and risk.
For more detailed information on the risks and uncertainties associated with the drug development and other activities of Biogen Idec and PDL BioPharma, see Item 1A "Risk Factors" in each company's most recent 10-Q or 10-K filing with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the companies assume no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.
The core management team that hit a home run with
the first vascular endothelial growth factor (VEGF) inhibitor
for the eye, which resulted in a $900 million merger and
acquisition deal, is taking another swing at bringing new
molecular entities to the marketplace with a newly formed
company.
Former Eyetech co-founders David R. Guyer and Samir
Patel have formed Ophthotech, which announced Series A
financing of $36 million and two in-licensing deals for compounds
for age-related macular degeneration (AMD).
SV Life Sciences led the round with HBM BioVentures
and Novo A/S participating.
Guyer, former chief executive officer for Eyetech, is
serving as Opthotech’s chairman of the board, and Patel is
president and CEO.
The entrepreneurs found success with the anti-VEGF
product Macugen (pegaptanib sodium), approved by the
FDA in 2004 for the treatment of neovascular (wet) AMD.
Wet AMD, the leading cause of blindness in the U.S.,
occurs when abnormal blood vessels start to grow under
the macula, the central portion of the retina that is responsible
for focusing central vision and aids with the ability to
read, recognize faces and colors and see objects in fine
detail.
Macugen binds to the VEGF protein that triggers the
abnormal blood vessel growth, ultimately slowing vision
loss.
Betting on Macugen’s potential for success, Melville,
N.Y.-based OSI Pharmaceuticals acquired Eyetech in
November 2005 for $935 million in cash and stock.
Many investors were baffled that OSI would take such a
risk on a one-drug firm like Eyetech, especially after results
of a Phase III study examining Genentech’s Lucentis
(ranibizumab), a humanized monoclonal antibody fragment,
showed that drug to be highly effective in treating
wet AMD, which set the stage for Lucentis to be an aggressive
rival to Macugen. (See BioWorld Today, Nov. 4, 2005.)
Indeed, after Lucentis entered the U.S. market in June
2006, sales of Macugen tumbled.
But Patel does not view Lucentis’ success as a failure for
Macugen.
The attention both drugs have received, he said, makes
it clear that there is still a “very large market space” available
for further opportunities in development and commercialization
of newer and better AMD therapies.
As part of its strategy to move from “infancy to adolescence”
in product development, Patel said, Ophthotech,
based in Princeton, N.J., entered into a licensing agreement
with Archemix, of Cambridge, Mass.
Archemix has granted Ophthotech worldwide rights to
all ophthalmic uses of the firm’s proprietary aptamers,
short oligonucleotides that form three-dimensional structures
that bind with high specificity and affinity to protein
and nonprotein targets, targeting the C5 component of the
complement cascade.
Ophthotech’s anti-C5 aptamer, Patel said, will be the
first breakthrough drug for dry age-related macular degeneration,
which occurs when the light-sensitive cells in the
macula slowly break down, gradually blurring central
vision in the affected eye.
Dry AMD is more common than wet AMD. There are
currently no treatments on the market for dry AMD.
“Dry AMD is an enormous market with major unmet
medical needs,” Patel said, noting that about 8 million
Americans and an additional 8 million Europeans have the
condition.
The other licensing agreement Ophthotech announced
involves a deal with OSI, the firm that acquired Eyetech.
OSI is transferring to Ophthotech all rights to Eyetech’s
anti-platelet derived growth factor (PDGF) aptamer program,
including rights to its preclinical compound E10030,
in exchange for an up-front cash payment, an equity interest
in Ophthotech and potential future milestones and royalties.
Financial details of the in-licensing agreements were not disclosed.
In preclinical studies, E10030 demonstrated the potential
to regress neovascularization when used in combination
with a VEGF inhibitor, Ophthotech said.
“Wet age-related macular degeneration is a huge
opportunity for combination therapy,” Patel said.
Anti-VEGF therapy, he said, “is still in its infancy.”
“Wet AMD is very similar to cancer therapeutics where
combination therapies have resulted in efficacy,” Patel said.
“Preclinical studies in both oncology and ophthalmology
have shown that when you have a therapeutic regimen that combines and targets anti-VEGF agent with anti-PDGF
agent, you get regression of the neovascularization.”
Patel said that Ophthotech has recruited a stellar team
of ex-Eyetech executives “who have tremendous expertise
and experience in developing these molecules in a very
accelerated fashion and bringing them to the marketplace
for patients with these very unfortunate diseases.”
In addition to Patel and Guyer, Ophthotech’s board
includes former Eyetech chairman of the board Henry
Simon of SV Life Sciences, Axel Bolte of HBM BioVentures,
and Thomas Dyrberg of Novo A/S.
Princeton, NJ – August 13, 2007 – Ophthotech Corporation (“Ophthotech”) today announced its Series A financing of $36 million and two in-licensing deals for compounds for macular degeneration. Ophthotech and Archemix Corp. ("Archemix") announced an exclusive, worldwide license agreement for the treatment of the wet and dry forms of Age-Related Macular Degeneration (AMD). Under the terms of the agreement, Archemix has granted Ophthotech worldwide rights to all ophthalmic uses of Archemix’s proprietary aptamers targeting the C5 component of the complement cascade. Specific terms related to the agreement have not been disclosed.
“Preclinical and human genetic linkage studies have demonstrated the significant role of complement mediated inflammation in both forms of AMD,” said Samir Patel, M.D., President and Chief Executive Officer of Ophthotech. “We are excited to have the opportunity to develop and commercialize anti-C5 aptamers, derived through Archemix’s proven leadership in therapeutic aptamers. We believe that the anti-C5 aptamer blockade represents a potential breakthrough therapy for dry and wet AMD.”
OSI Pharmaceuticals, Inc. (Nasdaq: OSIP) recently announced that its subsidiary, (OSI) Eyetech, Inc., has entered into an agreement with Ophthotech to divest its anti-platelet derived growth factor (PDGF) aptamer program. Under the terms of the agreement, OSI will transfer to Ophthotech all rights in the PDGF aptamer program, including rights to its pre-clinical compound E10030, in exchange for an up-front cash payment, an equity interest in Ophthotech and potential future milestones and royalties. Financial terms of the agreement have not been disclosed.
In pre-clinical studies, E10030 demonstrated the potential to regress neovascularization when used in combination with a vascular endothelial growth factor (VEGF) inhibitor. Anti-VEGF agents alone have shown the ability to slow or halt, but do not regress choroidal neovascularization. OSI elected to suspend further research on this compound in connection with its decision to divest its eye disease business. "We are pleased to transfer the development of this highly promising agent to an Ophthotech team that has the commitment to move this program forward aggressively," stated Colin Goddard, Ph.D., Chief Executive Officer of OSI Pharmaceuticals, Inc.
Dr. Patel continued, “We are excited to develop this Phase I ready anti-PDGF aptamer for AMD. Studies suggest that this compound, in combination with anti-VEGF agents, may cause regression of abnormal blood vessels in AMD. This is not observed with anti- VEGF therapy alone.”
With the $36 million of financing raised in the initial round of funding, Ophthotech expects to have sufficient resources to execute its strategy. Participants in the private
round include SV Life Sciences, HBM BioVentures and Novo A/S.
The lead investor, Lutz Giebel, Ph.D. of SV Life Sciences stated, “We are thrilled to invest in this all-star, ex-senior Eyetech management team that has a proven, stellar
track record in accelerated drug development and commercialization of therapeutics for the back-of-the-eye.”
The Ophthotech Board will consist of: Henry Simon (SV Life Sciences and former Chairman of the Board of Eyetech Pharmaceuticals, Inc. (“Eyetech”), Axel Bolte (HBM
BioVentures), Thomas Dyrberg, M.D., Ph.D. (Novo A/S), David R. Guyer, M.D. (former CEO and co-founder of Eyetech, who will also serve as Ophthotech’s Chairman of the Board), and Samir Patel, M.D. (President and CEO of Ophthotech and former co-founder of Eyetech).
The management team consists of former senior Eyetech personnel led by Chief Executive Officer, Samir Patel, M.D. Eyetech developed and commercialized Macugen,
the first anti-VEGF therapy for the eye.
Age-Related Macular Degeneration (“Wet” & “Dry”)
AMD is the leading cause of blindness for people over the age of 55 years, in the U.S. and Europe. There are two forms of AMD, namely "dry" and "wet" AMD. The "wet" form is characterized by the growth of new blood vessels into the central region of the retina. These new vessels cause severe visual loss due to retinal damage caused by subsequent leakage and scar formation. Anti-VEGF therapies and photodynamic therapies have been approved for “wet” AMD. “Dry” AMD accounts for up to 90 percent of all cases of AMD. There is no approved therapy for "dry" AMD, which afflicts 8 million patients in the U.S. and an additional 8 million in Europe. Visual loss in "dry" AMD is typically not as severe as "wet" AMD, however, over time, dry AMD can progress to the wet form of the disease.
About Ophthotech
Ophthotech Corporation, a recently-formed private company headquartered in Princeton, New Jersey with offices in New York, New York, focuses on developing and commercializing therapies for back-of-the-eye diseases. Ophthotech plans to develop compounds with strong scientific foundation for the treatment of AMD and bring them to market in an accelerated manner.
The core management team that hit a home run with
the first vascular endothelial growth factor (VEGF) inhibitor
for the eye, which resulted in a $900 million merger and
acquisition deal, is taking another swing at bringing new
molecular entities to the marketplace with a newly formed
company.
Former Eyetech co-founders David R. Guyer and Samir
Patel have formed Ophthotech, which announced Series A
financing of $36 million and two in-licensing deals for compounds
for age-related macular degeneration (AMD).
SV Life Sciences led the round with HBM BioVentures
and Novo A/S participating.
Guyer, former chief executive officer for Eyetech, is
serving as Opthotech’s chairman of the board, and Patel is
president and CEO.
The entrepreneurs found success with the anti-VEGF
product Macugen (pegaptanib sodium), approved by the
FDA in 2004 for the treatment of neovascular (wet) AMD.
Wet AMD, the leading cause of blindness in the U.S.,
occurs when abnormal blood vessels start to grow under
the macula, the central portion of the retina that is responsible
for focusing central vision and aids with the ability to
read, recognize faces and colors and see objects in fine
detail.
Macugen binds to the VEGF protein that triggers the
abnormal blood vessel growth, ultimately slowing vision
loss.
Betting on Macugen’s potential for success, Melville,
N.Y.-based OSI Pharmaceuticals acquired Eyetech in
November 2005 for $935 million in cash and stock.
Many investors were baffled that OSI would take such a
risk on a one-drug firm like Eyetech, especially after results
of a Phase III study examining Genentech’s Lucentis
(ranibizumab), a humanized monoclonal antibody fragment,
showed that drug to be highly effective in treating
wet AMD, which set the stage for Lucentis to be an aggressive
rival to Macugen. (See BioWorld Today, Nov. 4, 2005.)
Indeed, after Lucentis entered the U.S. market in June
2006, sales of Macugen tumbled.
But Patel does not view Lucentis’ success as a failure for
Macugen.
The attention both drugs have received, he said, makes
it clear that there is still a “very large market space” available
for further opportunities in development and commercialization
of newer and better AMD therapies.
As part of its strategy to move from “infancy to adolescence”
in product development, Patel said, Ophthotech,
based in Princeton, N.J., entered into a licensing agreement
with Archemix, of Cambridge, Mass.
Archemix has granted Ophthotech worldwide rights to
all ophthalmic uses of the firm’s proprietary aptamers,
short oligonucleotides that form three-dimensional structures
that bind with high specificity and affinity to protein
and nonprotein targets, targeting the C5 component of the
complement cascade.
Ophthotech’s anti-C5 aptamer, Patel said, will be the
first breakthrough drug for dry age-related macular degeneration,
which occurs when the light-sensitive cells in the
macula slowly break down, gradually blurring central
vision in the affected eye.
Dry AMD is more common than wet AMD. There are
currently no treatments on the market for dry AMD.
“Dry AMD is an enormous market with major unmet
medical needs,” Patel said, noting that about 8 million
Americans and an additional 8 million Europeans have the
condition.
The other licensing agreement Ophthotech announced
involves a deal with OSI, the firm that acquired Eyetech.
OSI is transferring to Ophthotech all rights to Eyetech’s
anti-platelet derived growth factor (PDGF) aptamer program,
including rights to its preclinical compound E10030,
in exchange for an up-front cash payment, an equity interest
in Ophthotech and potential future milestones and royalties.
Financial details of the in-licensing agreements were not disclosed.
In preclinical studies, E10030 demonstrated the potential
to regress neovascularization when used in combination
with a VEGF inhibitor, Ophthotech said.
“Wet age-related macular degeneration is a huge
opportunity for combination therapy,” Patel said.
Anti-VEGF therapy, he said, “is still in its infancy.”
“Wet AMD is very similar to cancer therapeutics where
combination therapies have resulted in efficacy,” Patel said.
“Preclinical studies in both oncology and ophthalmology
have shown that when you have a therapeutic regimen that combines and targets anti-VEGF agent with anti-PDGF
agent, you get regression of the neovascularization.”
Patel said that Ophthotech has recruited a stellar team
of ex-Eyetech executives “who have tremendous expertise
and experience in developing these molecules in a very
accelerated fashion and bringing them to the marketplace
for patients with these very unfortunate diseases.”
In addition to Patel and Guyer, Ophthotech’s board
includes former Eyetech chairman of the board Henry
Simon of SV Life Sciences, Axel Bolte of HBM BioVentures,
and Thomas Dyrberg of Novo A/S.
