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Isis Pharmaceuticals $ISIS and Sanofi $SNY announced the FDA approval of KYNAMRO (mipomersen) for the treatment of homozygous familial hypercholesterolemaia on January 29, 2013 and held a conference call the next day. This approval followed the December 2012 approval of JUXTAPID (lomitapide) from Aegerion Pharma for the same patient population and the October 2012 FDA Advisory Committees that voted in favor of both drugs. My notes from the webcast can be found below. Keep in mind this is reporting what management said on the call, which always includes a fair amount of "ISIS speak" statements that often later turn out to be hogwash, as I have noted when possible.
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(Continue reading for full post) Isis Pharma stock (click here for index of all ISIS content on BiotechDueDiligence) has been on fire ever since the positive buzz and commentary about early signs of clinical benefit surrounding their early stage antisense program for the devastating childhood disease of spinal muscular atrophy (SMA). The drug, targeting the splicing of the SMN gene, is partnered with Biogen $BIIB and is in phase 1/2 development.
Early-stage clinical trial data for ISIS-SMNrx will be presented at the American Academy of Neurology (#AAN13) annual meeting on March 20, 2013. Continue reading below for the fulltext abstract of the data to be presented. Investors, patients, and the medical community will all be hoping for greater details at the actual platform presentation. Oh, and don't forget that ISIS and Sanofi/Genzyme $SNY have an upcoming FDA PDUFA date for KYNAMRO (mipomersen) for homozygous familial hypercholesterolemia. Competitor Aegerion Pharma AEGR recently won FDA approval for JUXTAPID (lomitapide), but ISIS was rejected by the EU and faced greater criticism from a November FDA panel, leading to greater concern about mipomersen's prospects for approval. The race to developed novel cholesterol-lowering monoclonal antbodies targeting PCSK9 is heating up as Regeneron REGN and Sanofi SNY are the first to embark on a massive phase 3 program. While this battle is the realm of the big pharmas, small-cap biotech investors should also track the progress of these hypercholesterolemia drugs which are competitors in the familial HoFH/severe HeFH arena to KYNAMRO/mipomersen (developed by Isis Pharma ISIS with SNY - note Isis abandoned their PCSK9 antisense program), lomitapide (Aegerion AEGR), and ALN-PCS (RNAi against PCSK9 from Alnylam ALNY, which is seeking a parter for phase 2 development). Continue reading below the jump for the latest updates from 3q-2012 earnings conference calls about these candidates.
Quick notes from the Isis Pharma $ISIS conference call after tepid endorsement of KYNAMRO (mipomersen) by the FDA metabolic and endocrine products advisory committee. You can find more of my commentary on this event (as well as competitor Aegerion Pharma $AEGR and their drug lomitapide) on the Chimera Research Group blog.
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