Sanofi and Regeneron Announce Publication of Positive Phase 2 Results for Lipid-Lowering PCSK9 Antibody in The Lancet
Global Phase 3 program of SAR236553/REGN727 to be initiated in June across multiple patient populations
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Claims outcomes studies and carcinogenic requirements will delay. He says the delay will be upfront.
Well guess we are talking about grains of salt here- if one assume SNY know what they are doing, and they are going into P3 for a broader indication with PSK9, maybe they have a differing view than that of the Isis CEO. And unless he has actual head-to- head data it must be he is making he claims based on pre-clinical work, as SNY didn't mention any of his speculations in their clinical findings.
Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), and Isis Pharmaceuticals Inc. (ISIS), announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for KYNAMRO™ (mipomersen sodium) for the treatment of patients with homozygous familial hypercholesterolemia (HoFH). The NDA filing with the FDA triggers a $25 million milestone payment to Isis from Genzyme.
"The NDA filing with the FDA represents a significant achievement in the development of KYNAMRO™ and our efforts to get this important new drug to the market for patients who are at high-risk of a cardiovascular event,” said David Meeker, M.D., President and CEO, Genzyme. “We look forward to continuing the review process with the U.S. and EU regulatory authorities to bring KYNAMRO™ to patients in need.”
Genzyme submitted an application for U.S. marketing approval of KYNAMRO™ for the treatment of patients with HoFH in March 2012. The application will be subject to a standard review and will have a Prescription Drug User Fee Act (PDUFA) date of January 29, 2013. In July 2011, Genzyme submitted an application for EU marketing approval of KYNAMROTM for the treatment of patients with HoFH and severe heterozygous FH (Severe HeFH).
“We believe that KYNAMRO™ has the potential to bring real benefit to patients in the United States with HoFH who are unable to adequately control their LDL-C with currently available treatments,” said B. Lynne Parshall, Chief Operating Officer and CFO of Isis. “The successes of our joint development efforts for KYNAMRO™ are evident in the significant progress made in bringing this important new drug to the regulatory agencies for review. We are pleased to have earned the first regulatory milestone payment for KYNAMRO™ from this collaboration.”
You guys seem to be missing the history on this one. Psk9 works by expediting excretion. MIPS works by limiting production. So a combo seems likely. For what it's worth, dr crooke said FDA is concerned about safety (kidney damage as I recall). something about psk9 being involved in other pathways. Isis dropped its psk9 inhibitor in Jan (in stealth Isis fashion). I suspect it had to do with less than ideal potency. But crooke said the FDA would require a high burden of proof (ie, very long term follow up). Check the conference call transcripts from Jan.
More than an FYI- and says something that this has escaped comment by analysts and the BB. From the release, this seems basically quite effective, many patients below target at the top doses, no evidence of poor tolerance and importantly no LFT abnormalities, and the clincher is the P3 plan for HeFH.
Doesn't this suggest that Sanofi think this is a better and more broadly acceptable drug than Mipo? Hence the P3 development for this in HeFH. I would bet that Mipo will be a short-term niche product for HoFH, that SNY will not invest further in Mipo, and that the resultant royalty stream will be less than projected by Isis for this product.
You stated, "importantly no LFT abnormalities...."
---Actually the release said, "There were no elevations in liver function tests (LFT) >3 times the upper limit of normal (ULN).
Also, I wonder whether it might be possible to combine the effects of REGN's 727 PCSK9 blocker and Mipo in some way.