Is this from Insmed's Letter to the EMA?
Also, very puzzling where all of our recent *Industry Veteran* BOD members and Lewis were during this process. Lack of competence on Management's part or fooled by the EMA dangling a carrot? So much for skipping QT CC's and having the CFO jawboning the Conferences while Lewis was away doing....what?!
Excellent post. I would add, are we now planning to skip EMA all together and just go back to an FDA filing (assuming IF the data is positive!)?!?
Excellent post...this board seemingly is either Uber Pumpers or Crazy Shorts. Relief to actually hear some pragmatism. Maybe, Mr. Lewis figured we had plenty of money and applying with the EMA would buy some time after the Gupta Trial debacle, knowing that it would be years (at that time) for the CONVERT results and the EMA tends to be MUCH more lenient approving drugs than the FDA. Who knows? Yet another failure, either way.
As we now head to all-time lows, it all comes down to absolutely needing success of a PHIII trial. Fugly. Can only imagine what our monthly burn rate is now with all of the sleek new offices and lots of new employees with little to do for a while but it would certainly be nice to know how many patients will be in the Compassionate Use Programs...need some positive light...especially with our leader and company, inexplicably being silent for so long. I guess he is above having impromptu C/C's to answer the unknowns and calm the markets.
One bright spot, as we are getting creamed and heading South in a BIO bear market, I am actually glad he raised money when he did so at least we're not getting diluted at these pathetic levels.
Very good post for any that heavily follow the sector. The IPLEX data, especially from the Italy Extension is MUCH more compelling than what SRPT has been presenting...and I believe SRPT is using an extremely small sample size too boot. Having said that, Mr. Lewis and his Team need to validate all of the money and immense time spent and deliver something from the Transave IP! Management has basically been saying the past year that full-enrollment in the CONVERT Trial is their top objective but perhaps, they actually feel an EMA Approval for Arykace is a slam-dunk and are sand-bagging just in case...especially after the very expensive Gupta situation during (and after) the FDA PIIB trial.
As far IPLEX, you HAVE to assume Shire would love the rest of the IPLEX IP and would also love an EMA approved indication for NTM and Arykace. For suffering longs, these current PPS levels really need to improve because if we go it alone, you KNOW they're gonna have to raise again and if the BUYOUT option is in play then you would prefer to see a much higher PPS to justify the metrics and multiples of a potential offer, IMO.
Nice compilation work, as always, JTFM.
Besides the recent Conference Presentations, I think some of the current strength with the stock might be coming from the M & A in the sector (and related positioning) which could be a terrific set-up if your correct about guidance, etc. Great Balance Sheet, significant Growth and still slightly under the radar.
IF (still a big if or at least a regular *if*..) EMA gives Marketing authorization approval, it's sort of a no-brainer that Arikayce will be prescribed off-label, especially with all of this type of data...right?
Good to see this pr. These posters will provide some very interesting reading when they're public for sure.
nice reading this below again in the most recent 10k too...
*the protocol for the convert study incorporates feedback from the fda and the ema via its scientific advice working party process, as well as local health authorities, including japan’s pharmaceuticals and medical devices agency, and was approved in the us by a central institutional review board (irb). We initiated the global trial in early 2015 and expect to complete patient enrollment in the second half of 2016. If the convert study meets the primary endpoint of culture conversion at month 6, we believe we would be eligible to submit an nda pursuant to 21 cfr 314 subpart h (accelerated approval of new drugs for serious or life-threatening illnesses), which permits fda to approve a drug based on a ‘‘surrogate endpoint’’ provided the sponsor commits to study the drug further to verify and describe the drug’s clinical benefit. We believe that efficacy data from the convert study after month 6 will suffice to meet this commitment. We are currently conducting convert at over 115 sites in the us, europe, australia, new zealand, asia and canada. The convert study is designed to enroll enough subjects to ensure at least 261 subjects are evaluable for the primary endpoint at month 6.*
It is a shame, indeed.
Civil, rational debate and the exchange and evolution of ideas is actually part of the enjoyment of investing. Yahoo hasn't done a good job here, beyond some particular glitchy software.
Insurance reimbursement, aside, one would have to assume that off-label Rx will occur as well, right? (I am jumping WAY ahead, obviously.)
Ummmm, not a *do over* but the Primary endpoint of the Phase IIB failed and the Scientist that was running the trial was fired and then Management did indeed botched the timeline to investors and had to correct which wasn't good at all ($$$), if you remember!
Full Enrollement now scheduled for the end of this year...maybe we get good news that it is enrolled sooner, though.
You do realize that Mr. Dreschler has lead these Presentations solo before, right?! You, also realize that you're being as dishonest and fraudulent as the *shorts* and their prognostications by making up this *idea* and publishing it, right?!
How about we get just get some dang positive news from Management that has spent a 100 mil in the past year about Trial Enrollment, Partnership Money (anyone else remember Mr. Lewis numerously teasing the Japanese Partner talks 2 YEARS ago?!) and LEGIT EMA news that is honestly revealed...PUBLICLY?
It's been a VERY long time and enormous dilution for Common Shareholders since Geoff Allan's success, that's for sure. I could care less about anonymous Message Boards p-contest that hurt so many insecure feelings but I do hope for ONCE this Management Team/ BOD can actually close the deal and create meaningful value for common shareholders and not the traders and shorts that have continually feasted on their mistakes...for years!