I thought the reason that the Aldox was supposed to be superior was that they could dose it at higher levels and for longer - with dox, there is a cumulative cap, so even if the drug is working, once you hit the cap, the patient must go off drug. If Aldox doesn't have that cumulative dose cap, maybe there is some hope, but nothing that is going to warrant big ticket pricing.
There is nothing "boilerplate" about this delay. Less than three months ago in their Q1 conference call the CEO said the bacteria panel would be on the market by early 2017. When an analyst on the call challenged him to identify any "hurdles" they needed to overcome in order to meet that timeline, the CEO said the risk to the timeline from the work they needed to do was low, but he could not place the risk on a potential delay in the FDA review. Three months later, they are saying they won't even FILE with the FDA until mid-2017 - how is that a "boilerplate" change in guidance.
Well, the answer to the Q2 revenue question has been answered and it is not good - 6 contracts closed. I think the stock could shrug off the Q2 miss, as well as the problem/disruption with the cartridges, but the delay of the bacterial panel will scare many investors - they tried to spin it as a minor delay, but they were previously guiding to an early 2017 filing and now they are talking about "mid-2017" - 6 months is an eternity for a company spending cash the way they are.
Where did you get that the FDA asked for another trial? The last word was that they suddenly were hung up on GI issues and the Crowley said they were going to try to extract some GI data from the current/ongoing trials. A "reasonable" path would be to at least provide conditional approval, subject to a new trial - no approval until a new trial is completed would be a kick in the balls.
Thanks for sharing the info - unfortunately, technical superiority hasn't been the problem for this company - they have had it for years - it is execution in the market. With Arun gone, maybe there is hope, but they simply moved the current marketing head up to CEO, so it is doubtful that he will shake things up dramatically?
The sad part of your analysis is that the $30M only "keeps us going for about 2 months" - with the cash on hand at the end of Q1 ($165M) and the $16M sold in April, that gets us out to probably mid 2017 - with revenue from Migalistat and reduced R&D when the P3 trial for Scioderm is completed, hopefully into Q3 of next year before cash becomes critical. Obviously, if Scioderm is approved by then, we won't have much of an issue, but if Scioderm is delayed and/or the FDA forces them to do another trial on Migalistat, it could get ugly.
If their next generation Pompe and Fabry ERTs show clear benefits over existing ERTs, I agree that we will eventually get bought out, My hope is that the FDA will provide a reasonable clinical path for Migalistat and the Scioderm P3 leaves no doubt about efficacy - that should get us into double digits and after that, anything else is gravy.
Your guess is as good as any and well thought out - unfortunately, as you describe, there are a number of factors and players involved with closing a contract, so to a certain extent it is #$%$ shoot. Hopefully, they will be reporting the results of the bacteria panel testing in Q3 - maybe that will provide some support. Does anyone know if the performance of blood culture in bacterial sepsis is as bad as it is with candida? If blood culture is in the 60% range and T2 is in the 95%+ range for sensitivity, then blockbuster status should be forthcoming in 2017+?
You're welcome - I actually hope Q2 will show a nice recovery, as continued softness may limit the company's ability to raise cash. They are in somewhat of a race to get the bacteria panel approved and on the market before their cash runs out. They are on pace to spend around $50M in 2016 and have about $60M on hand at the end of March. The approval of the bacteria panel should support a higher share price - the question is whether it will allow them to borrow money to fund the necessary expansion of the marketing effort or whether they will have to do an offering. If sales in the back half are stagnant and the share price dwindles, it could be painful for the company to have to raise money in early 2017?
Hard to imagine they could be worse, but I doubt they recovered substantially. The company spoke to the fact that it appeared that some prospective buyers were going to wait until the bacteria panel was approved before taking the leap - the bacteria panel is not expected to be approved until early 2017. This would seem to make sense in that they also described how transitioning the T2 system into the hospital's protocols for testing takes some time and so some hospitals would rather not go through a second transition next year. The one potential driver for the remainder of the year are the studies releases at ASM in June - hospitals that have already adopted are sharing glowing reports about how fast & accurate the test is (compared to blood culture), which is leading to savings on the use of expensive anti-fungals. These studies may prompt some hospitals to not wait until next year, but they did not come out in time to impact Q2 sales.
Can anyone provide even a remote explanation as to how Brexit will impact a US drug company like DEPO? Probably not, but the momentum players will jump in and take any excuse provided - stupidity of the herd will dominate today!
not sure what you are looking at - the Form 4 filings represent option grants - there is a Form 3 from Redmile that shows them increasing their position?
He is missing the boat here - OMS721 won't start a "price war" - it will dominate the market. With a better safety profile - Soliris has a REMS (worse than "black box" safety warning) and requires a vaccine against meningitis two weeks prior to treatment - OMS721 will crush Solirist. OMS721 is already being used on a compassionate use basis in patients that haven't responded to Soliris. What doctor can ignore the safety risks with Soliris and choose it over OMS721 - they would be sued out of practice if a patient got sick or died because of Soliris and a safer alternative was available.
These gene therapy companies are a tough nut to crack from a valuation standpoint. There are two critical factors that are impossible at this stage to reasonably estimate - first, how long will the safety portion of a pivotal trial be and second, what kind of pricing will they be able to get for the treatment. The fear with these gene therapies is that they will create some sort of genetic mutation that will result in cancer or other "off target" side effects, so the FDA may require 2+ years of safety reviews before approving the treatment. The pricing is even more of a wild card - the value to the patient's quality of life and the economic cost is clearly there (e.g. saving a hemophiliac from routine infusions could save $300K+ per year), but will that warrant a price of $1M, $5M, $10M for these single-treatment cures - who knows? I have small investments in both QURE and SGMO, because the technology is so promising - curing people with a single treatment - but the time to approval and ultimate pricing issues (as more and more pressure is coming down on the industry) may not drive much value to shareholders. Gene therapy is really something the govt should be investing in and keep private industry out of it - it will save the healthcare system hundreds of billions if these treatments are successful, but all that value may just end up in the pockets of another healthcare company, rather than saving the taxpayers and those paying private insurance premiums any money.
The point is they have less than 6 months of cash left and will have to raise money soon - any pop from a Phase 2a trial cannot withstand the drop from a dilutive offering.
Yes, that was an interesting exchange about Firefly - you could sense that Arun was implying that NVDQ probably gave up too much to get SPY into ISRG's robots (their quarterly "partnered" revenues have been stuck around $3M for some time, despite increased procedures using Firefly?) and if ISRG wants to get the next generation of SPY technology and software, they are going to have to pay more than they are currently?
investor73498 - did you listen to the GS presentation - everything I said about Gala was confirmed - JC said it would take 6-12 months to "launch" (i.e. begin marketing) in most of the EU countries, as they have to negotiate pricing and other issues before getting individual approval - that is why the PR for the EU approval said it gives them "... a platform to begin accessing..." the various markets - they said two of the larger markets outside the EU (Japan and Brazil) will likely take even longer. He said it will take them until this time next year before they have a feel for the market potential in the EU. That is why I said you have to hope for one of three things over the next 6-9 months to drive the stock - a favorable ruling by the FDA on the US approval path for Gala, solid top-line data from the EB trial and/or solid early clinical data from the Pompe trial.
Of the three, I think the Pompe results are the least risky - lab data on the performance of ERT's translate pretty cleanly to the clinic. While the EB results will most likely be favorable, I think the "market" may not respond significantly, because they will not be final data - they are only a little over 50% enrolled and JC spoke to the fact that they are still increasing sites over the next 6 months (from 16 to 25+) in order to complete enrollment. They are starting to prepare a rolling NDA for EB this year, but EB approval is probably late 2017, at the earliest. The FDA guidance on Gala approval is the the driver that will be revealed first and the one with the most risk - any additional trial requirements will likely push the share price back to 52-week lows, before the prospects of recovery from EB and Pompe results.
Wow - your last statement confirms my point - if you believe Etep is as toxic as Drisa, your are off-the-charts stupid.
careful - BMRN BOUGHT the drug after the 100 patient trial "showed it did not work" because they looked at the pre-clinical data and early clinical data and concluded that the drug did work, but the P3 trial was flawed - they gave up the drug after the FDA and the EMA told them their drug was too toxic to ever be approved. Etep may have a "similar" mechanism as Drisa, but because of Etep's pristine safety profile, they are able to dose Etep at 5X+ higher levels than Drisa. The fact that you would bring BMRN in as a "logical" comparison shows how clueless you are.