- US NDA and EU MAA have been accepted for review
- Expect FDA advisory committee meeting late 3q or early 4q 2012
- Expect US approval late 2012 and EU approval first half 2013
- Project profitability in 2014
- Working on patient registry for homozygous familial hypercholesterolemia (HoFH), won't say how many patients are on it until closer to launch
- Expect mipomersen and lomitapide to be revieweed at same panel. Topics of discussion could include trial design, liver safety, REMS
- AEGR has submitted a proposed REMS to FDA in NDA filing focused around strict on-label use and monitoring of liver enzymes.
- Don't see a place for PCSK9 drugs in HoFH
- Now can't forecast a date for access program/reimbursement in France (had been projecting by year-end 2011)
- Need to reformulate before starting pediatric trials -won't start until 2013, and probably after initial regulatory decisions. Need to get same bioavailability, stability, palatability for whatever presentation given to kids - so even if plan to open capsules and mix with food, would have to do the work. Liquid formulation has not been tried but should be technically feasible.
- Market size estimate ~3000 patients in US holds up across multiple methods of calculation so far.
- In these patients, neither mipomersen nor lomitapide is going to get all patient to goal LDL levels
- Intellectual property: Excited about patent issued in 2011 (administration patent through 2025-2026 in US/EU). Composition of matter (COM) patent expires in 2015, Hatch-Waxman extension through mid-2020. 10 years EU market exclusivity. Patent term extension for COM in US "yet to be determined"
Aegerion Pharma $AEGR held its first quarter 2012 earnings call on May 3rd. Here are a few quick notes regarding their drug candidate lomitapide and its potential competitor KYNAMRO (mipomersen) from Isis Pharma $ISIS.
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Next week on 3/29 Isis Pharma will once again present further long-term safety data for its lipid lowering drug candidate KYNAMRO (mipomersen) at the XVI INTERNATIONAL SYMPOSIUM ON ATHEROSCLEROSIS. See below for the full abstract. For an extensive discussion on mipomersen safety and efficacy, see my prior report. Isis Pharma is presenting two abstracts at the American College of Cardiology Scientific Sessions this weekend, related to two drugs in their cardiovascular franchise. See below for complete details. While Isis Pharma recently abandoned their PCKS9 antisense program, blaming regulatory concerns, other companies push forward with therapeutics against this important target, including Alnylam with a RNAi approach. A more advanced program, from Sanofi $SNY and Regeneron $REGN, will release data this weekend at the American College of Cardiology Scientific Sessions. See below for the complete abstract. Note that this antibody was also featured in a recent New England Journal of Medicine article. Presentation Number: 911-5 Abstract Title: The Effects of Co-administering a Monoclonal Antibody to Proprotein Convertase Subtilisin/Kexin Type 9 Serine Protease, REGN727/SAR236553, with 10 and 80 mg Atorvastatin Compared to 80 mg Atorvastatin Alone in Patients with Primary Hypercholesterolemia (NCT: 01288469) Presentation Time: Sunday, Mar 25, 2012, 8:25 AM - 8:38 AM Topic: 9. Prevention: Clinical Author Block: Eli M. Roth, James McKenney, Corinne Hanotin, Gaelle Asset, Evan Stein, Sanofi-aventis US, Inc, Bridgewater, NJ, USA, Regeneron Pharmaceuticals, Inc, Tarrytown, NY, USA Keywords: Hypercholesterolemia, Low‐density lipoprotein cholesterol Background: Low-density lipoprotein cholesterol (LDL-C) reduction significantly lowers cardiovascular risk in at-risk patients. Atorvastatin 80 mg/day (A80) is a proven and highly efficacious LDL-C-lowering treatment. However, many patients do not achieve goals with maximal dose of statin. Proprotein convertase subtilisin/kexin 9 (PCSK9) binds to LDL receptors (LDLRs), increasing LDLR degradation and reducing the rate of LDL-C removal from the circulation. REGN727/SAR236553, a fully human monoclonal antibody, binds to PCSK9, enhances LDLR expression, and further reduces LDL-C levels in statin-treated patients. The primary objective of this study was to assess the LDL-C efficacy of high dose of A (A80) alone compared to both A 10 mg/day (A10) and A80 combined with subcutaneously (sc) administered REGN727/SAR236553. Methods: This was a multicenter, double-blind, placebo-controlled study conducted in patients with hypercholesterolemia and LDL-C ≥100 mg/dL on A10 for ≥6 weeks prior to randomization to 3 parallel treatment groups: A80 + placebo (n=29), A80 + REGN727/SAR236553 sc every 2 weeks (q2w) (n=30), or A10 + REGN727/SAR236553 sc q2w (n=29). All patients were treated for 8 weeks and then followed for an additional 8 weeks. Results: After 8 weeks of treatment, A10 + REGN727/SAR236553 patients had an additional [mean (SD)] LDL-C reduction of 66.7% (12.5), and those on A80 + REGN727/SAR236553 achieved a 72.3% (14.4) reduction, compared to a 17.7% (27.2) reduction in patients on A80 + placebo (p<0.0001). Significant reductions were also observed in apolipoprotein B, non-high density lipoprotein cholesterol and lipoprotein(a) with A80 + REGN727/SAR236553 versus A80 alone. Statistical comparisons with A10 were not performed. There was one serious adverse event of dehydration in the A80 + REGN727/SAR236553 group, which was deemed not treatment related. No other significant safety concern was observed. Conclusions: Co-administration of REGN727/SAR236553 with A10 or A80 resulted in a significant and substantial decrease in LDL-C and was well tolerated. |
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