Regeneron Pharmaceuticals' EYLEA(R) (aflibercept) Injection Receives FDA Breakthrough Therapy Designation for Diabetic Retinopathy in Patients with Diabetic Macular Edema
Sep 16 14
Regeneron Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted EYLEA(R) (aflibercept) Injection Breakthrough Therapy designation for the treatment of diabetic retinopathy in patients with diabetic macular edema (DME). The designation is based on positive results in two Phase 3 trials (VIVID-DME and VISTA-DME), in which EYLEA demonstrated a statistically significant improvement in a pre-specified measure of diabetic retinopathy in patients with DME after two years of treatment. In the Phase 3 VIVID-DME trial in diabetic retinopathy patients with DME, 29% of evaluable patients in the 2Q4 group (monthly) and 33% of evaluable patients in the 2Q8 group (every two months, after 5 initial monthly injections) treated with EYLEA experienced at least a 2-step improvement on the diabetic retinopathy severity scale (DRSS), a grading system measuring the degree of retinopathy, compared to 8% of patients in the laser control group (p less than 0.001). In the Phase 3 VISTA-DME trial in diabetic retinopathy patients with DME, 40% of evaluable patients in both the 2Q4 and 2Q8 groups treated with EYLEA experienced at least a 2-step improvement on the DRSS compared to 17% of patients in the laser control group (p less than 0.0001). The most frequent ocular adverse events (AEs) observed in the VIVID-DME trial were conjunctival hemorrhage, cataract, and increased intraocular pressure. The most frequent ocular AEs observed in the VISTA-DME trial were conjunctival hemorrhage, eye pain, and vitreous floaters. The two-year results from these trials on the primary endpoint of best-corrected visual acuity (BCVA) and overall safety have been previously announced and are available here. The Breakthrough Therapy designation was created by the FDA to expedite the development and review of drugs for serious or life-threatening conditions. Drugs qualifying for this designation must show credible evidence of a substantial improvement on a clinically significant endpoint over available therapies, or over placebo if there is no available therapy. The designation includes all of the fast track program features, as well as more intensive FDA guidance and discussion. The Breakthrough Therapy designation is distinct from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met. EYLEA is approved in the United States, European Union (EU) and other countries for the treatment of wet age-related macular degeneration (AMD), macular edema following central retinal vein occlusion (CRVO), and DME. Regulatory submissions have been made for EYLEA in the U.S. and EU for macular edema following branch retinal vein occlusion (BRVO).
Regeneron Pharmaceuticals, Inc. Presents at EBD Group's Biopharm America 2014 Conference, Sep-22-2014
Sep 10 14
Regeneron Pharmaceuticals, Inc. Presents at EBD Group's Biopharm America 2014 Conference, Sep-22-2014 . Venue: Boston Marriott Copley Place, 110 Huntington Avenue, Boston, MA 02116, United States.
Regeneron Pharmaceuticals, Inc. and Sanofi Announce Presentation of Detailed Positive Results from Four Pivotal Alirocumab Trials at ESC Congress 2014
Aug 31 14
Regeneron Pharmaceuticals, Inc. and Sanofi announced detailed positive results from four Phase 3 ODYSSEY trials of alirocumab in people with hypercholesterolemia. Alirocumab is an investigational monoclonal antibody targeting PCSK9 (proprotein convertase subtilisin/kexin type 9). Results from the four ongoing trials, all of which met their primary efficacy endpoint, will be presented at a Hot Line session at the ESC Congress 2014 in Barcelona, Spain. ODYSSEY LONG TERM Trial: The ongoing 2,341-patient, double-blind ODYSSEY LONG TERM trial is designed to evaluate the long-term safety and efficacy of 150 milligrams (mg) alirocumab every two weeks versus placebo in patients with hypercholesterolemia who are at high or very-high cardiovascular (CV) risk, including patients with an inherited form of high cholesterol known as heterozygous familial hypercholesterolemia (HeFH). Both study groups are treated with statins at a maximally-tolerated dose and some patients also receive additional lipid-lowering therapies. A pre-specified interim analysis was performed when all patients reached one year and approximately 25% of patients reached 18 months of treatment. Key data to be presented on Aug. 31, 2014 include: On the primary efficacy endpoint of the trial, at 24 weeks, there was a 61% reduction from baseline in LDL-C levels in the alirocumab group as compared to a 1% increase in the placebo group (62% reduction in alirocumab group compared to placebo), p less than 0.0001. At 52 weeks, there was a 57% reduction from baseline in LDL-C levels in the alirocumab group as compared to a 4% increase in the placebo group (61% reduction in alirocumab group compared to placebo), p less than 0.0001. 81% of alirocumab patients achieved their pre-specified LDL-C goal (either 70 milligrams/deciliter [mg/dL] or 100 mg/dL depending on patients' baseline CV risk) compared to 9% for placebo (p less than 0.0001). The most common adverse events (greater than or equal to 5% of patients) were nasopharyngitis (13% alirocumab; 13% placebo), upper respiratory tract infection (7% alirocumab; 8% placebo), and injection site reactions (6% alirocumab; 4% placebo). In a post hoc safety analysis, there was a lower rate of adjudicated major CV events (cardiac death, myocardial infarction, stroke, and unstable angina requiring hospitalization) in the alirocumab group compared to placebo (1.4% compared to 3.0%, nominal p-value less than 0.01). These CV events comprise the composite primary endpoint of the ongoing 18,000-patient ODYSSEY OUTCOMES trial, which is prospectively evaluating the potential of alirocumab to demonstrate CV benefit. Three additional trials (ODYSSEY COMBO II, FH I and FH II) will also be presented on Aug. 31, 2014. In these three trials, alirocumab-treated patients receive an initial dose of alirocumab 75 mg every two weeks, increasing to 150 mg if needed to reach pre-specified LDL-C levels. The 75 mg and 150 mg alirocumab doses were delivered as a single, self-administered 1 milliliter (mL) injection. ODYSSEY COMBO II trial: ODYSSEY COMBO II is a double-blind, 720-patient trial designed to evaluate the safety and efficacy of alirocumab compared to ezetimibe in patients with hypercholesterolemia who are at high CV risk and had inadequate LDL-C reduction at baseline despite stable maximally-tolerated statin therapy. Key data to be presented on Aug. 31, 2014 include: On the primary endpoint of the trial, at 24 weeks, there was a 51% reduction from baseline in LDL-C levels in the alirocumab group compared to a 21% reduction in the ezetimibe group (30% reduction in alirocumab group compared to ezetimibe group), p less than 0.0001. At 52 weeks, there was a 50% reduction from baseline in LDL-C levels in the alirocumab group compared to an 18% reduction in the ezetimibe group (32% reduction in alirocumab group compared to ezetimibe group), p less than 0.0001. 77% of patients in the alirocumab group achieved an LDL-C level of 70 mg/dL at 24 weeks. Approximately 80% of patients in the alirocumab group remained on the initial 75 mg alirocumab dose. The most common adverse events (greater than or equal to 5% of patients) were upper respiratory tract infection (6.5% alirocumab; 6% ezetimibe), accidental overdose (6% alirocumab; 7% ezetimibe), dizziness (5% alirocumab; 5% ezetimibe), and myalgia (4% alirocumab; 5% ezetimibe). ODYSSEY FH I and FH II trials: The ODYSSEY FH I and FH II trials enrolled a total of 738 HeFH patients and compare alirocumab to placebo. All patients are on maximally-tolerated daily statin therapy and the majority of patients also receive ezetimibe. Despite receiving this high level of background therapy, patients in these studies had mean baseline LDL-C levels of 145 mg/dL (FH I) and 134 mg/dL (FH II).