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Sarepta Therapeutics, Inc. Message Board

bionerd51 24 posts  |  Last Activity: 5 hours ago Member since: Jan 2, 2007
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  • Reply to

    If

    by thigrlsrk Mar 30, 2015 8:07 AM
    bionerd51 bionerd51 5 hours ago Flag

    The FDA has already given guidance on quantification methods. Where have you been. The IMF [IHC] and Western Blot. The only issue the FDA is weighing right now is what weight to give these findings. This was a major topic covered in workshop.

  • This quote says it all…: "Management Transition Aimed at Normalizing Relations with the FDA"

    Sentiment: Strong Buy

  • bionerd51 by bionerd51 Mar 31, 2015 9:18 PM Flag

    Board realized that CG was no longer an asset and was actually a hindrance in their efforts for eteplirsen approval. He's an example of a CEO pushing too hard at exactly the wrong times. FDA and his own researchers despised him.

  • Reply to

    A little more from Dirk.......

    by georgecantstansya Mar 27, 2015 8:28 AM
    bionerd51 bionerd51 Mar 27, 2015 10:48 AM Flag

    George, Dirk suffers from "target fixation". He focuses on a chemistry [in this case S-DNA's] so subjectively that he see's nothing else and flies right into it.

  • Reply to

    A little more from Dirk.......

    by georgecantstansya Mar 27, 2015 8:28 AM
    bionerd51 bionerd51 Mar 27, 2015 8:42 AM Flag

    George, Keep in mind that Dirk advocated for S-DNA's dating back to Genasense and discredited PMO's solely based on cell culture studies. I have been presenting clinical data to him for 17 years on the superior multicellular, intracellular delivery of PMO's. For 17 years I also have been warning S-DNA advocates of the renal and hepatic toxicity toxicity inherent in this oligo. He finally gets. I guess it's never too late.

    Sentiment: Strong Buy

  • Reply to

    not good, IMO

    by simp08801 Mar 23, 2015 8:40 AM
    bionerd51 bionerd51 Mar 23, 2015 8:12 PM Flag

    Ru, Stretching the truth again. Your following statement is absurd, "Yes, the immuno-florescence microscopy (IFM) methodology was second-rate and has to be repeated". This is a lie. Immunofluorescence microscopy is a very specific example of immunohistochemistry. It is not only used for quantification of dystrophin on fibers but qualification which is even more important then the limited data point that western blot provides. When ones objective is to count dystrophin that is actually incorporated in a functional position on a muscle fiber, IMF-microscopy is the preferred methodology. If SRPT was counting gross albumen levels in a sample then I would use WB. Identifying a phenotypical change in the underlining pathological protein [dystrophin] produced by a splice corrected oligo IMF/microscopy is superior. Your second rate comment is a false creation on your part because the IMF results generated by a lab came under scrutiny because of possible lab inconsistencies not a question of the underling IMF methodology or technology.

    Sentiment: Strong Buy

  • bionerd51 by bionerd51 Mar 20, 2015 9:45 AM Flag

    Since Biomarin's dystrophin's position is clear no one should project that Drisa produces additional, functional dystrophin ever again. That discussion is now over. Since there were no stat. sig. endpoints met in their trials why are they filing an NDA???The parallel universe that the FDA operates in is getting more bizarre.

  • bionerd51 bionerd51 Mar 19, 2015 3:40 PM Flag

    It's completely absurd that Biomarin/PTCT would argue against dystrophin as a biomarker for AA. Based on their position, what is the mechanism-of-action of Drisa and Translarna? This is slight of hand tomfoolery and if I was on the FDA panel I would ask this question. Well how does your drug[s] work? Cognizant that MOA's are not required for an NDA but a hypothesis for one will have to be answered. What do they posit, Our drug works like pixie dust, it's just magic????

    Sentiment: Strong Buy

  • Reply to

    comparing antisense

    by usagary1 Sep 21, 2011 7:19 AM
    bionerd51 bionerd51 Mar 19, 2015 11:50 AM Flag

    This research paper [Nature] was a piggy-back of a paper authored by MJ Wood [MDEX] from Aug. 2014 Molecular Therapeutics. Full paper is available. Here is the abstract. Antisense oligonucleotides (AOs) are currently the most promising therapeutic intervention for Duchenne muscular dystrophy (DMD). AOs modulate dystrophin pre-mRNA splicing, thereby specifically restoring the dystrophin reading frame and generating a truncated but semifunctional dystrophin protein. Challenges in the development of this approach are the relatively poor systemic AO delivery and inefficient dystrophin correction in affected non-skeletal muscle tissues, including the heart. We have previously reported impressive heart activity including high-splicing efficiency and dystrophin restoration following a single administration of an arginine-rich cell-penetrating peptide (CPPs) conjugated to a phosphorodiamidate morpholino oligonucleotide (PMO): Pip5e-PMO. However, the mechanisms underlying this activity are poorly understood. Here, we report studies involving single dose administration (12.5 mg/kg) of derivatives of Pip5e-PMO, consecutively assigned as Pip6-PMOs. These peptide-PMOs comprise alterations to the central hydrophobic core of the Pip5e peptide and illustrate that certain changes to the peptide sequence improves its activity; however, partial deletions within the hydrophobic core abolish its efficiency. Our data indicate that the hydrophobic core of the Pip sequences is critical for PMO delivery to the heart and that specific modifications to this region can enhance activity further. The results have implications for therapeutic PMO development for DMD.

  • bionerd51 by bionerd51 Feb 23, 2015 7:59 PM Flag

    New research demonstrates that PMO's can turn on calorie burning machinery in rodents. Publication: "New Scientist"

    Sentiment: Strong Buy

  • Reply to

    HIH Public Access...

    by bionerd51 Feb 7, 2015 1:29 PM
    bionerd51 bionerd51 Feb 9, 2015 11:24 AM Flag

    Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice

  • Reply to

    HIH Public Access...

    by bionerd51 Feb 7, 2015 1:29 PM
    bionerd51 bionerd51 Feb 8, 2015 9:35 AM Flag

    simp, What Biomarin believes they "see" in the PhaseII data is irrelevant. I have studied the data available and "see" nothing. The heart of Biomarin's case to the FDA will be "projection" and "extrapolation". They will extrapolate that a very few of the younger boys in their P-2 performed better then the older boys in their trial. They will ignore historical data. They will project to the FDA that if these older boys were treated earlier in progression they too would respond to Drisa. They will use the NIH PPMO paper as proof. Biomarin is looking for "extra-innings" from the FDA in hopes they [FDA] buy this projection and grant provisional AA until full PhaseIII dataset is in. If the FDA examines Biomarin's P-2 AA application without passion and decides case based on data this application has no chance of approval. I predict the FDA will shoot down their AA app. but will revisit PhaseIII data under a fast track protocol. Lose-win with the ultimate outcome being a full rejection of P-3 NDA application. When one analyzes their data without passion, and with objectivity, I do not understand how any biotech analyst can predict a positive outcome for Biomarin's Drisa.

    Sentiment: Strong Buy

  • bionerd51 by bionerd51 Feb 7, 2015 1:29 PM Flag

    Article which can be accessed via PubMed [full article] studies dystrophin the use of PPMO's and progression of DMD pathology. Abridged version….No correlation between dystrophin production and disease progression once a male is in advance stages of disease. Dystrophin does correlate in early to midstage of DMD. Something to think about. If SRPT did not enroll patients in their PhaseII trial that were expected to deteriorate rapidly [by certain criteria] the PhaseII data would have been much better. I believe this paper was used by RNA/Biomarin to design their next PhaseIII trial which will include boys

    Sentiment: Strong Buy

  • Reply to

    WHEN YOU HAVE A CANCER YOU CUT IT OUT

    by starfe11 Feb 6, 2015 12:22 PM
    bionerd51 bionerd51 Feb 6, 2015 12:49 PM Flag

    Starfe, You are now comparing CG to cancer. Really? A little extreme don't you think? If you have a problem with an action CG has taken, bring it up for peer review and let the most logical position prevail. Your constant, irrational rumblings are really tiresome.

  • bionerd51 bionerd51 Feb 5, 2015 11:28 AM Flag

    An increase of stability of the S-DNA's and their chemical permutations and PMO's, use the natural phosphate linkages found in DNA/RNA as the baseline. The thioate linkages used in the S-DNA constructs are more stable then phosphate linkages but the phosphorodiamidate linkages of the PMO are as stable as a biomolecule can be.

    Sentiment: Strong Buy

  • bionerd51 bionerd51 Feb 4, 2015 2:02 PM Flag

    S-DNA's and their spurious effects in part can be attributed to their affinity for certain cellular proteins like heparin growth factor, VEG factor and platelet derived growth factor. These toxic chemicals also increases the effect of ADP.

    Sentiment: Strong Buy

  • Reply to

    Anyone know if 4th biopsies have been done yet?

    by paris1785 Feb 3, 2015 12:03 PM
    bionerd51 bionerd51 Feb 4, 2015 1:43 PM Flag

    Yes. One can add a week or so for SRPT to evaluate this data set. Don't forget the biopsies will be received by the lab in a staggered timeframe because of the different days these biopsies are performed.

    Sentiment: Strong Buy

  • Reply to

    ISIS, TMKR, RNA, etc

    by tredleon Feb 4, 2015 11:25 AM
    bionerd51 bionerd51 Feb 4, 2015 11:44 AM Flag

    Tred, " ISIS is a $7B company with one approved drug that has a black box warning for liver issues" and has no sales or should I say Isis has not provided sales numbers since being approved as a second-line therapy for FHC.

    Sentiment: Strong Buy

  • Reply to

    Anyone know if 4th biopsies have been done yet?

    by paris1785 Feb 3, 2015 12:03 PM
    bionerd51 bionerd51 Feb 4, 2015 9:46 AM Flag

    Mush, Retail biopsies can take 3 months but SRPT has a contract with the labs processing these specimens and they will be conducted in a very timely manner. From date of biopsy to SRPT receiving data/slides it should take a ~ month.

    Sentiment: Strong Buy

  • bionerd51 bionerd51 Jan 31, 2015 5:09 PM Flag

    The source was a Prosensa presentation which has been mentioned on this board at least a dozen times.

    Sentiment: Strong Buy

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