Highlights from Isis Pharma ISIS investor event (excerpts c/o SeekingAlpha) with updated commentary on various drug development programs in the pipeline. For archival purposes (June 2013)
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Earnings call transcript excerpt c/o SeekingAlpha.
We're making progress with our mid-stage pipeline as well and also the important work of keeping the sustainability of the pipeline going. We expect data readouts for daclizumab in 2014, TYSABRI and SPMS in 2015 and potentially SMNRx in 2016. 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. Not much to report from Isis Pharma (NASDAQ: ISIS) partners from 3q-2012 among major pharma/biotech companies:
Sanofi SNY (transcript via SeekingAlpha) "You've seen the advisory committee, which voted in favor of KYNAMRO for Homozygous Familial Hypercholesterolemia just last week, PDUFA date in January and a European opinion in the fourth quarter of this year. " Biogen BIIB - no comments about Isis partnerships A quiet quarter on the partner front for ISIS news. See below for the details:
Sanofi $SNY - only mentioned KYNAMRO (mipomersen) in passing...still awaiting US and EU regulatory decisions and FDA advisory committee vote in October 2012. Biogen $BIIB (transcript via Morningstar) "Through a second collaboration with Isis Pharmaceuticals, we added an interesting preclinical program intended to develop and commercialize the treatment for myotonic dystrophy. This program fits squarely within our strategy to focus on highly differentiated therapies for serious, unmet needs in neurology." "As George mentioned, we recently added a promising preclinical program targeting myotonic dystrophy or DM1 through a second collaboration with Isis. DM1 is a debilitating neuromuscular disease that affects about 150,000 people in the U.S., Europe and Japan. It's caused by a genetic defect in the dystrophia myotonica-protein kinase or DMPK gene and there are currently no disease modifying therapies to treat DM1." "As discussed during our Analyst Day one of our focus areas within neurology is genetically well-defined diseases. DM1 fits squarely within that particular approach. The disease is caused by the accumulation of toxic RNAs in the nucleus of affected individuals, because of CTG repeats and the Isis molecule is designed to specifically correct this accumulation. Isis is working to discover the lead drug candidate for the program, which we hope to take in the Phase I next year." |
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