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  • The Sarepta 12  [View article]
    When you have experts that are participating in the trial saying absolutely the drug works and there is clear-cut evidence of dystrophin production, what "totality of evidence" are you looking at to conclude that it probably should not be approved? The clinical evidence from the 6MWT is the weakest link - not because the performance by the treated boys is weak, but because it is such a flawed measurement - there are too many variables that can influence the performance of boys in that age group and it is impossible to establish a consistent baseline starting point for patients. Either way, the true clinical benefit will reveal itself in the confirmatory trial and beyond - exactly how the FDASIA regs intended.
    Dec 3, 2015. 12:39 AM | Likes Like |Link to Comment
  • Eteplirsen For Duchenne Muscular Dystrophy Should Receive FDA Accelerated Approval  [View article]
    A decent article, but your thinking that the "variability in patient population may limit the efficacy" to a subset of patients is off base. If you define efficacy or benefit strictly by the 6MWT (or the ability to remain ambulant for that matter), then you are assuming those already non-ambulant will never benefit, which is absurd. The two boys that lost ambulation at the start of the trial (prior to the time period where meaningful dystrophin production was achieved) are still being treated and presumably their pulmonary and upper body muscles are benefitting from the drug.

    It is clear that exon-skipping will never reverse the damage of DMD, so boys that have already lost most of the functional muscle mass in their legs will not benefit in their leg muscles - that does not mean Etep will not provide benefit to other muscle groups.
    Dec 1, 2015. 10:52 AM | 5 Likes Like |Link to Comment
  • Watch For A Potential 50% Drop In Omeros  [View article]
    You pass over the FDA approval Medicare reimbursement like it is meaningless. As one poster pointed out, there is pressure from the FDA to discontinue the use of compounded products and when it comes to the economics facing the doctors, why would they spend $40 out of pocket (they don't get reimbursed for a compounded drug) when they get fully reimbursed plus an administrative charge (I think it is 6% of cost) when using Omidria. So, the doctors make money by using Omidria vs a compounded drug and using a compounded drug opens them up to all kinds of legal issues if it proves to be inferior or harms the patient. Doesn't seem like much of a choice?
    Aug 31, 2015. 06:25 PM | 8 Likes Like |Link to Comment
  • Amicus Therapeutics Still A "Show Me" Story  [View article]
    It is not an argument - I'm simply stating that you provided no qualitative analysis about FOLD's ERT pipeline, but simply dismissed it based on its clinical status and historical odds given that status - are you saying otherwise?
    Jun 18, 2014. 10:12 AM | 1 Like Like |Link to Comment
  • Amicus Therapeutics Still A "Show Me" Story  [View article]
    Yes it does help. The fact that you rate the chances of the company's pipeline to be so low and assign baffling peak sale amounts (you state Sanofi's existing Pompe drug to sell $700M but assume the market potential for FOLD's Pompe ERT to be a peak of $250M), but then still come up with a value that is 30% higher of where it is today reinforces my belief that this stock is a bargain.

    When it comes to giving the Pompe program a 10% chance, because that is what the universe of drug development history would dictate, that is not much of an analysis. I thought the goal behind biotech investing was to find the diamonds in the rough - the drugs/treatments that have the best chances of beating the overall odds. No doubt, that is a dicey game before a company has "proof of concept" Phase II data, in most cases. But FOLD's ERT program isn't inventing a new mousetrap - they are improving on the existing mousetrap and have direct pre-clinical comparisons to show that it is magnitudes better. You don't even assess the qualitative value of the pre-clinical data for their combo Fabry or Pompe ERT's, so why bother to analyze the company at all?
    Jun 18, 2014. 07:04 AM | Likes Like |Link to Comment
  • Amicus Therapeutics Still A "Show Me" Story  [View article]
    A very comprehensive write-up, although your "chance" percentages seem low. With respect to Migalistat, is it a 35% chance that it meets the 012 non-inferiority goal or is there some marketing risk assigned to that %? Given the oral vs infusion advantage and the history of patients in the extension trials staying on Migalistat, I would think the marketing risk would be low. Also, aren't there patients that can't handle ERT due to immune response issues, so might the FDA leave the door open for approval for certain patients even if it Migalistat proves to be moderately inferior?

    With respect to the Pompe program, again, is the 10% all clinical risk or are you assuming there will be marketing/competitive risks? Given the pre-clinical response data and lack of immune issues, it is hard to imagine the risk of clinical success is only 10% - there is a lot of data comparing their formulas against the existing ERTs and beating them by a wide margin on response rates, so why so low?
    Jun 17, 2014. 10:42 PM | Likes Like |Link to Comment
  • Endocyte Fails Ovarian Cancer Trial, Now What?  [View article]
    I appreciate your hopeful comments about ECYT's prospects, but you need to do a little more homework. Vintafolide is not linked to either PLD or Dox - the folate receptor in Vinta is linked to a completely different chemo agent (the name is too long to print here, but the acronym is DAVLBH) - see any of their earlier press releases that disclose "About Vintafolide". Vinta is being used in combo with PLD in ovarian cancer and Dox in NSCLC, because those are standard therapies in those indications - the control arms in each case are PLD and Dox without Vinta.

    Either way, the long-term hope for ECYT is not in Vinta - while Merck may still pursue it in other indications, their Tubulysin-based agents are what will drive future value. Tubulysin is 100-times more potent than the chemo agent in Vinta and in pre-clinical studies generated complete responses where Vinta failed or became refractory. I'm actually surprised Merck invested so much in Vinta without getting an option on the Tubublysin agents.
    Jun 11, 2014. 01:23 PM | 1 Like Like |Link to Comment
  • Cyclacel Pharmaceuticals: The Devil's Advocate Be Damned  [View article]
    Wow - I have never seen an analyst dissect a presentation like that before - I admire your passion for the company and the nuances of the situation/presentation. Am I wrong in thinking that if the SEAMLESS trial is a success and they get approval in AML, that the planned Phase 2B trial in MDS would at least open the door for "off label" use of Sapa in MDS and at best provide a path for conditional approval in MDS? If so, it doesn't seem like a bad strategy, given their limited resources - it will certainly take less time to get there, relative to a 400+ patient pivotal trial. Is part of the issue not knowing what the proper control arm to use in R-MDS - could the standard of care shift in the time it would take them to do a pivotal trial? Thanks for the insight.
    Jun 4, 2014. 01:57 PM | 2 Likes Like |Link to Comment
  • Neuralstem And Brainstorm Cell Therapeutics: The Race To Treat ALS  [View article]
    The other issue the author does not touch upon is the how the FDA will approach Brainstorm's MSCs - how are they "differentiated" from the stem cells into the NurOwn cells and how pure is that differentiation. In watching Geron flounder with their stem cell efforts in spinal cord injury, one of the key issues they faced was the purity of the cells - does the differentiation process produce any cells other than those intended, the risk being the potential for tumors down the road, which if you are injecting them into the spinal cord, there is no room for error. Obviously, the FDA may put safety on the back burner for a deadly disease like ALS, but Cur may offer a safer alternative and since they are not autologous cells, they certainly have the cost advantage when it comes to producing the cells.
    Mar 11, 2014. 03:52 PM | 4 Likes Like |Link to Comment
  • Marketing Strategy Could Distinguish Neptune With Industry-Leading Status  [View article]
    You've got to be kidding? You make the case of Viagra becoming a market leader due to Pfizer's marketing efforts - Pfizer, a multi-billion $, international company that probably has spent hundreds of millions over the past decade marketing Viagra - and then somehow compare Neptune's opportunity in NKO, when Neptune needed to raise $25M just to stay alive - they used $8M in operating the company last quarter (exlcuding any capital improvements), so unless sales rebound dramatically, they raised enough cash to last about a year. Neptune may have a solid position in the krill oil market, but their ability to grow that market through their own marketing budget is limited, at best.
    Mar 4, 2014. 09:28 AM | Likes Like |Link to Comment
  • Wherein I Try To Understand Why Anyone Would Own Unilife Stock  [View article]
    The company is financially like a development stage biotech in the sense of where it is at with its product lifecycle - they have a whole stable of delivery technologies that have been in the R&D/testing stage for the past several years and will be entering the market over the next five years. The point is that historical financials are a meaningless indicator of the potential future revenue & profit picture and the fact that the company doesn't have a deferred tax asset or fails a bankruptcy scoring metric that is designed for operating companies is meaningless in determining the company's potential value.
    Feb 3, 2014. 10:56 PM | 1 Like Like |Link to Comment
  • Wherein I Try To Understand Why Anyone Would Own Unilife Stock  [View article]
    We get it - you don't believe the UNIS story, but UNIS is essentially a development stage biotech - no development stage biotech companies have deferred tax assets, all of them would fail your bankruptcy scoring, and none of them would look worth a dime based on some future extrapolation of historical financials. I'm sure most longs realize that UNIS is a high risk play - you have to believe the company will secure numerous long-term contracts that will generate revenues in the hundreds of millions over the next five years. Given the CEO's history for exaggeration and missing milestones, is it rational that the stock is trading at a market cap of $0.5B - absolutely not, but there are plenty of biotechs valued at $1B+ that are much further away from generating meaningful revenues than UNIS. The whole biotech space is in a bubble, so why not UNIS?
    Feb 3, 2014. 07:53 AM | 4 Likes Like |Link to Comment
  • Increasing Price Target On Neurocrine Bio After Phase 2 Success  [View article]
    Jason - Nice analysis - I appreciate the background on the TD market and current treatments, or lack thereof. One question I had was when you speak to the difference in baseline AIMS readings, I thought they said the AIMS scale they used in the second study was different - it didn't have as many gradations? Is that not true?
    Jan 8, 2014. 10:35 AM | Likes Like |Link to Comment
  • Sophiris Bio: Recent Price Action Offers An Attractive Entry Point  [View article]
    One issue that hasn't been addressed is with respect to the probability of Phase III success, the Phase IIb study did not show statistically significant differences in either IPSS or Qmax at 12 months, which is the timeline for measurement in the Phase III trial. Is everyone assuming that the 440 patients in the Phase III (vs the 92 patients in the Phase IIb trial) will push the reported difference in the Phase III trial to be statistically significant at 12 months? I am always leery of patient reported measurement scales like IPSS as primary endpoints - the placebo effect seems to trash these studies more often than not. It seems the IPSS has a number of more objective criteria (frequency, intermittency and having to go at night), so you would hope the patients' observations would have some objectiveness to them, but who knows?
    Jan 2, 2014. 07:51 PM | Likes Like |Link to Comment
  • KaloBios Pharmaceuticals: Undervalued Company With Huge Potential Catalyst  [View article]
    rootbeer - I agree. It is tempting to take a position now, as the phase I results were intriguing, showing efficacy after a single injection - The FEV benefit in the reversible population was >10% on average vs placebo, but that was based on only 12 patients. If you believe the 10% can be replicated, you should bet the house, as the Phase II trial only needs an ~7% differential to be statistically significant. Better to take a small position, if the results are positive, average up and if the results are positive, average down - if it trades near cash value (around $70M at year-end or $2 per share), then it is a bargain compared to the value of KB001 and the Sanofi partnership.
    Dec 29, 2013. 01:41 PM | 1 Like Like |Link to Comment