"2010 was a very productive year for Regeneron as we reported positive Phase 3 results in four clinical trials: two with VEGF Trap-Eye in wet age-related macular degeneration, called wet AMD, one with VEGF Trap-Eye in central retinal vein occlusion, and one with ARCALYST® for the prevention of gout flares in patients initiating uric-acid lowering therapy," said
"In anticipation of potential product approvals," Dr. Schleifer added, "we are continuing to build our commercialization capabilities. We are also advancing our earlier-stage pipeline which currently includes eight fully-human monoclonal antibodies in clinical development for the treatment of various diseases and conditions including elevated LDL cholesterol, rheumatoid arthritis, atopic dermatitis, and cancer. We anticipate Phase 2 data from some of these programs in 2011."
"We entered 2011 in a strong financial position to support our development and commercialization activities," commented
Clinical Programs Update
VEGF Trap-Eye (aflibercept ophthalmic solution) — Ophthalmologic Diseases
VEGF Trap-Eye is a fusion protein locally administered in the eye that is designed to bind Vascular Endothelial Growth Factor-A (VEGF-A) and Placental Growth Factor (PLGF), proteins that are involved in the abnormal growth of new blood vessels. Regeneron maintains exclusive rights to VEGF Trap-Eye in
Phase 3 studies in wet age-related macular degeneration
In VIEW 1 and VIEW 2, VEGF Trap-Eye was administered either monthly or every two months, and compared to monthly doses of ranibizumab, which is the current standard of care in wet AMD. In these studies, all regimens of VEGF Trap-Eye including VEGF Trap-Eye dosed every two months successfully met the primary endpoint of non-inferiority compared to ranibizumab dosed every month. The primary endpoint was statistical non-inferiority in the proportion of patients who maintained (or improved) vision over 52 weeks compared to ranibizumab. At least 94% of patients in every VEGF Trap-Eye group, including those dosed every two months, as well as those receiving ranibizumab dosed monthly, maintained visual acuity over 52 weeks. Visual acuity was measured as a score based on the total number of letters read correctly on the Early Treatment Diabetic Retinopathy Study (ETDRS) eye chart, a standard chart used in research to measure visual acuity. Maintenance of vision was defined as losing fewer than three lines (equivalent to 15 letters) on the ETDRS eye chart.
In VIEW 1 and VIEW 2, a generally favorable safety profile was observed for both VEGF Trap-Eye and ranibizumab. The incidence of ocular treatment emergent adverse events was balanced across all four treatment groups in both studies, with the most frequent events associated with the injection procedure, the underlying disease, and/or the aging process. The most frequent ocular adverse events were conjunctival hemorrhage, macular degeneration, eye pain, retinal hemorrhage, and vitreous floaters. The most frequent serious non-ocular adverse events were typical of those reported in this elderly population who receive intravitreal treatment for wet AMD.
Phase 3 studies in central retinal vein occlusion
In the COPERNICUS study, VEGF Trap-Eye was generally well tolerated. The most common adverse events were those typically associated with intravitreal injections or the underlying disease. Serious ocular adverse events in the VEGF Trap-Eye group were uncommon (3.5%) and were more frequent in the control group (13.5%). The incidence of non-ocular serious adverse events was generally well-balanced between the treatment arms.
Initial results from GALILEO, the second of the two Phase 3 studies in CRVO, are expected in the first half of 2011.
Phase 2 study in diabetic macular edema
In DA VINCI, VEGF Trap-Eye was generally well tolerated. The most common adverse events reported were those typically associated with intravitreal injections or the underlying disease. The most frequent ocular adverse events reported among patients receiving VEGF Trap-Eye included conjunctival hemorrhage, eye pain, ocular redness (hyperemia), and increased intraocular pressure. There were no non-ocular serious adverse events judged by investigators to be drug-related during the first six months of the study and one during the second six months.
Based on these positive results,
Phase 3 study in choroidal neovascularisation
ARCALYST® (rilonacept) — Gout
ARCALYST® is a fusion protein that blocks the cytokine interleukin-1 (IL-1). ARCALYST® is currently available for prescription in
ARCALYST® is in a Phase 3 clinical development program for the prevention of gout flares in patients initiating uric acid-lowering therapy. In
Two other studies are ongoing in the Phase 3 program. The global PRE-SURGE 2 study, which has a similar trial design as PRE-SURGE 1, is evaluating the number of gout flares per patient over the first 16 weeks of initiation of allopurinol therapy. The global RE-SURGE study is evaluating the safety of ARCALYST® versus placebo over 16 weeks in patients who are at risk for gout flares because they are taking uric acid-lowering drug treatment. PRE-SURGE 2 and RE-SURGE are fully enrolled, and the Company expects to have initial data from both studies in the first quarter of 2011. Regeneron owns worldwide rights to ARCALYST®.
Aflibercept (VEGF Trap) — Oncology
Aflibercept, also known as VEGF Trap, is a fusion protein that is designed to bind VEGF-A and PLGF, proteins that are involved in the abnormal growth of new blood vessels in solid tumors.
Aflibercept is being developed worldwide by Regeneron and its collaborator, the sanofi-aventis Group, for the potential treatment of solid tumors. Three randomized, double-blind, Phase 3 trials, all of which are fully enrolled, are evaluating combinations of standard chemotherapy regimens with either aflibercept or placebo for the treatment of cancer. One trial (VELOUR) is evaluating aflibercept as a 2nd-line treatment for metastatic colorectal cancer in combination with FOLFIRI (folinic acid [leucovorin], 5-fluorouracil, and irinotecan). A second trial (VITAL) is evaluating aflibercept as a 2nd-line treatment for locally advanced or metastatic non-small cell lung cancer in combination with docetaxel. The third trial (
Final results from the VITAL and VELOUR studies are anticipated in the first half of 2011. Based on projected event rates, an interim analysis of the
In addition, a randomized Phase 2 study (AFFIRM) is evaluating aflibercept as a 1st-line treatment for metastatic colorectal cancer in combination with FOLFOX (folinic acid [leucovorin], 5-fluorouracil, and oxaliplatin). The AFFIRM study is fully enrolled, and initial data are anticipated in the second half of 2011.
Since 2007, Regeneron and sanofi-aventis have collaborated on the discovery, development, and commercialization of fully human monoclonal antibodies generated by Regeneron using its VelocImmune® technology. During the fourth quarter of 2009, Regeneron and sanofi-aventis expanded and extended their collaboration with the objective to advance an average of four to five antibodies into clinical development each year between 2010 and 2017. The following eight antibody candidates are currently in clinical development under the collaboration:
REGN727, an antibody to Proprotein Convertase Substilisin/Kexin type 9 (PCSK9), a novel target for LDL cholesterol ("bad cholesterol") reduction, has been evaluated in Phase 1 studies using both intravenous and subcutaneous routes of administration. REGN727 is being studied as a single agent and in combination with statin therapy. Phase 2 studies have been initiated in patients with hypercholesterolemia.
REGN88, an antibody to the interleukin-6 receptor (IL-6R), is in a Phase 2/3 study in rheumatoid arthritis and a Phase 2 study in ankylosing spondylitis, a form of arthritis that primarily affects the spine. Both studies are enrolling patients, and initial Phase 2 results are expected in 2011.
REGN421, an antibody to Delta-like ligand-4 (Dll4), a novel angiogenesis target, is in a Phase 1 study in patients with advanced malignancies.
REGN668, an antibody to the interleukin-4 receptor (IL-4R), a target for allergic and immune conditions, has completed Phase 1 testing in healthy volunteers. A Phase
REGN910, an antibody to angiopoietin-2 (ANG2), a novel angiogenesis target, is in a Phase 1 study in oncology.
REGN475, an antibody to nerve growth factor (NGF), has completed a Phase 2 trial in osteoarthritis of the knee. In
REGN728 and REGN846, whose targets remain undisclosed, have entered clinical development.
The Company's total revenues increased to
Net product sales of ARCALYST® in the fourth quarter of 2010 were
The Company's total operating expenses increased to
The Company had a net loss of
Regeneron is a fully integrated biopharmaceutical company that discovers, develops, and commercializes medicines for the treatment of serious medical conditions. In addition to ARCALYST® (rilonacept) Injection for Subcutaneous Use, its first commercialized product, Regeneron has therapeutic candidates in Phase 3 clinical trials for the potential treatment of gout, diseases of the eye (wet age-related macular degeneration and central retinal vein occlusion), and certain cancers. Additional therapeutic candidates developed from proprietary Regeneron technologies for creating fully human monoclonal antibodies are in earlier stage development programs in rheumatoid arthritis and other inflammatory conditions, pain, cholesterol reduction, allergic and immune conditions, and cancer. Additional information about Regeneron and recent news releases are available on Regeneron's web site at www.regeneron.com.
This news release discusses historical information and includes forward-looking statements about Regeneron and its products, development programs, finances, and business, all of which involve a number of risks and uncertainties and actual events or results may differ materially from these forward-looking statements. These risks and uncertainties include, among others, risks and timing associated with preclinical and clinical development of Regeneron's drug candidates, determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron's ability to continue to develop or commercialize its product and drug candidates, competing drugs that are superior to Regeneron's product and drug candidates, uncertainty of market acceptance of Regeneron's product and drug candidates, unanticipated expenses, the availability and cost of capital, the costs
of developing, producing, and selling products, the potential for any collaboration agreement, including Regeneron's agreements with the sanofi-aventis Group and
Michael Aberman, M.D.
REGENERON PHARMACEUTICALS, INC.
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