Age-related macular degeneration, or AMD for short, is a leading cause of acquired blindness for people over the age of 65 in the U.S. and Europe. AMD is of two types - wet and dry.
Patients with wet AMD develop abnormal growth of blood vessels under the the central part of the retina of the eye that exude or leak blood or fluid, making it harder to see. Though only about 10% of individuals who suffer from macular degeneration have wet AMD, it is the more severe type of AMD, accounting for 90% of all cases of severe vision loss from macular degeneration.
Wet AMD represents a blockbuster market and is estimated to be worth more than $3 billion. Monthly injections of Lucentis (ranibizumab), developed by Roche unit Genentech is the gold standard for treating wet AMD. The cost per treatment with Lucentis is high - about $2,000 ($24,000 per year). Avastin, a cancer drug, which costs $50 per treatment, is also commonly used off-label to treat wet AMD. Avastin, also developed by Genentech, is not approved by the FDA for treatment of wet AMD.
Each year, over 210,000 Americans are newly diagnosed with and treated for wet AMD. It is estimated that 1.8 million people in the U.S are affected by AMD, 1.2 million of whom have wet AMD. This number is expected to increase to nearly 3 million by 2020 as baby boomers age.
Let's take a look at what's making news in Regeneron Pharmaceuticals Inc. (REGN), one of the companies working to find less frequent and less expensive options for wet AMD.
Based in Tarrytown, New York, Regeneron currently has one marketed product and 11 product candidates in clinical development, including three that are in phase III studies.
The company's late-stage programs are VEGF Trap-Eye (aflibercept ophthalmic solution), which is being developed using intraocular delivery for the treatment of serious eye diseases; ZALTRAP (aflibercept), also known as VEGF Trap, which is being developed for cancer indications in collaboration with Sanofi-aventis and ARCALYST, which is being developed for the prevention of gout flares in patients initiating uric acid-lowering treatment.
Regeneron submitted a BLA (Biologics License Application) for VEGF Trap-Eye for the treatment of wet age-related macular degeneration in February 2011 and received a priority review designation. A Dermatologic and Ophthalmic Drugs Advisory Committee Meeting is scheduled to be held on June 17, to discuss the Biologics License Application for VEGF Trap-Eye for wet AMD. A final decision by the FDA on VEGF Trap-Eye for wet AMD is expected by August 20.
Regeneron and Bayer HealthCare are collaborating on the global development of VEGF Trap-Eye. The exclusive rights to VEGF Trap-Eye in the United States are maintained by Regeneron.
The VEGF Trap-Eye BLA was based on positive results from the VIEW program. The VIEW (VEGF Trap-Eye: Investigation of Efficacy and Safety in Wet AMD) program included two randomized, phase III clinical trials - VIEW 1 and VIEW 2, which evaluated VEGF Trap-Eye in the treatment wet AMD.
The VIEW 1 study, which randomized 1,217 patients, was conducted in the United States and Canada by Regeneron under a Special Protocol Assessment with the FDA. The VIEW 2 study, which randomized 1,240 patients, was conducted in Europe, Asia Pacific, Japan, and Latin America by Bayer HealthCare.
In each of the studies, VEGF Trap-Eye was dosed as an intravitreal injection on a schedule of 0.5 mg monthly, 2.0 mg monthly, or 2.0 mg every two months (following three monthly loading doses), compared with intravitreal Lucentis administered 0.5 mg every month during the first year of the studies.
The primary endpoint analysis was statistical non-inferiority in the proportion of patients who maintained (or improved) vision over 52 weeks compared to Lucentis.
According to the trial results, all doses of VEGF Trap-Eye, including VEGF Trap-Eye of strength 2 milligrams every two months (following three loading doses), successfully met the primary endpoint of non-inferiority, compared to Lucentis 0.5 mg dosed every month. A generally favorable safety profile was observed for both VEGF Trap-Eye and Lucentis.
The potential advantages of every two month dosing of VEGF Trap-Eye include less burden on patients as it requires fewer visits to doctor's office and fewer treatment injections.
In April of this year, Regeneron and Bayer initiated the first of two phase III clinical trials evaluating the efficacy and safety of VEGF Trap-Eye in the treatment of DME (Diabetic Macular Edema).
The first phase III trial in DME, named VIVID-DME, is being led by Bayer HealthCare and has started in Australia. The trial will also be conducted in Europe and Japan. A second study led by Regeneron, named VISTA-DME, is expected to begin later in 2011 in the United States, Canada, and other countries.
VEGF Trap-Eye is also in phase III development for the treatment of CRVO (Central Retinal Vein Occlusion), another cause of visual impairment. Regeneron has a license and collaboration agreement with Bayer HealthCare that was inked in October 2006 for the global development and commercialization outside the U.S. of VEGF Trap-Eye.
VEGF Trap-Eye for the treatment of CRVO is being studied in two phase III studies - COPERNICUS and GALILEO. The company reported positive results from the COPERNICUS study last December and reported positive results from the GALILEO study in April of this year.
Regeneron intends to submit a regulatory application for marketing approval for VEGF Trap-Eye in CRVO in the U.S. in the second half of 2011, and Bayer HealthCare is planning to submit regulatory applications in this indication in Europe in 2012.
And now, about the other drugs in the pipeline...
ZALTRAP is another drug candidate that is in phase III development stage in cancer indications. This investigational drug is developed by Regeneron in collaboration with Sanofi-Aventis.
In March 2011, Sanofi-Aventis and Regeneron announced results from a phase III trial dubbed VITAL evaluating ZALTRAP for the second-line treatment of non-small cell lung cancer, which did not meet the primary endpoint of improvement in overall survival.
The following month - in April, the companies reported positive results from a study dubbed VELOUR, which evaluated ZALTRAP in second-line metastatic colorectal cancer. In the VELOUR study, ZALTRAP was studied in combination with FOLFIRI chemotherapy regimen [folinic acid (leucovorin), 5-fluorouracil, and irinotecan] versus a regimen of FOLFIRI plus placebo. The company expects to submit a BLA for ZALTRAP to the FDA for the treatment of metastatic colorectal cancer in the second half of 2011.
In addition to the VELOUR trial, ZALTRAP is studied in a phase III trial dubbed VENICE in hormone-refractory metastatic prostate cancer patients, and in a phase II study named AFFIRM in first-line metastatic colorectal cancer.
An interim analysis of the VENICE trial is expected to be conducted by an Independent Data Monitoring Committee in mid-2011 and final results of the study are anticipated in 2012. The final results of the AFFIRM trial are expected during the second half of 2011.
Regeneron is also testing ARCALYST for a supplemental indication in preventing gout flares in patients initiating uric acid-lowering therapy. The company plans to submit a supplemental BLA to the FDA in mid-2011 for marketing approval in the U.S. of ARCALYST for the prevention of gout flares. ARCALYST is already approved for a rare orphan disease CAPS (Cryopyrin-Associated Periodic Syndromes). Last year, sales of ARCALYST brought in $25 million, up more than 35% over a year before.
The phase III clinical development program with ARCALYST for the prevention of gout flares consists of three studies - PRE-SURGE 1 (PRE vention S tudy against UR ate-lowering drug-induced G out E xacerbations), PRE-SURGE 2, and RE-SURGE (RE view of S afety U tilizing R ilonacept in G out E xacerbations).
The company has already reported positive results from the studies - PRE-SURGE 1, PRE-SURGE 2 as well as the RE-SURGE trial. A supplemental BLA to the FDA is expected to be submitted in mid-2011 for marketing approval in the U.S. of ARCALYST for the prevention of gout flares.
Beyond its phase III compounds, Regeneron has eight antibodies currently advancing through clinical development. According to the company, at least two to three of its investigational antibodies are likely to enter phase III by year-end 2012. In the next seven years, the company plans to introduce an additional 30 antibodies into clinical development.
Finally, a quick look at the company's balance sheet...
As of March 31, 2011, Regeneron had zero debt, an accumulated deficit of $1.1 billion and cash and securities of $607.6 million.
Shares of Regeneron have thus far hit a 52-week low of $20.45 and a 52-week high of $71.74. The stock closed Tuesday's trading at $60.02.
Given the fact that Regeneron is transforming into a commercial enterprise and advancing a fully-funded antibody pipeline, the upcoming events are worth the watch.
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