Matt, good for you, like to see you demonstrating your trust here. I know we've been tested a lot by the volatility, and that will likely continue even as our share price leaves this trading range we've been in. We've had a lot of good news here since I've been around, the problem is (at least so far) that cumulatively, it doesn't seem to 'stick' to the successive valuation of the Company. That I believe is about to change forever.
Thanks stock, this program is in most capable hands. DMD by itself could end up driving the Company's net valuation to over $1.5B, if we can look into the future, assuming we progress on the successful pathway to stand at par with Sarepta today and their DMD drug candidate Eteplirsen, not yet FDA approved. I was not aware of our esteemed VP Ms. Kathryn Haviland. That's great to know she was directly from Sarepta, WOW, great to know, and what a resume'!
I was on the bid at 3.19 late in the day as well. Picked up a few more shares. I've been spending more time in the abstracts and presentations on the website lately. If you want to be more convinced of the depth and breadth of the Company's pipeline and where we're headed, everyone here should do the same.
This pivotal data release on WM is just the beginning of this juggernaut's evolution.
Continued from Idera's poster link: iderapharmaDOTcom/sites/default/files/uploads/presentations/idera_mda_2015_poster_final.pdf
TLR antagonism represents a non-steroidal anti-inflammatory
therapeutic approach applicable to all patients with DMD regardless
of dystrophin genotype
– TLRs are key drivers of muscle inflammation and are over-activated
in DMD patients at all stages of the disease
• Improved skeletal and cardiac muscle function was observed in
~1-year-old mdx mice with double MYD88 knockout, demonstrating
the role of the TLR pathway in the disease process
• An investigational TLR antagonist improved muscle inflammation,
cytokine gene expression, markers of muscle damage and immune
activation, and EDL muscle function following five weeks of treatment
in mdx mice
• Collectively, these data support advancing IMO-8400, an investigational
antagonist of TLRs 7, 8 and 9, into clinical development for the
treatment of DMD patients
People, watch this brief video on DMD: http://financeDOTyahooDOTcom/q?s=srpt&ql=1
Tuesday's binary event is being billed as the industry's major recent event, so why is this? Now consider this!
Today based on speculation about BMRN's treatment drug for DMD possibly not passing FDA scrutiny on Tuesday, the swing (in SRPT's stock price =$7.29) was nearly twice what IDRA's stock price is trading for today, and you know how broad our platform is and how many potential disease indications each of our scientific platforms can possibly treat given time and money to develop them. OUR phase two trial will likely start in Q1 for DMD, where TLR's play a significant role in the disease's pathogenesis.
THIS TREATMENT APPROACH is unique to IDRA. From the poster...
"A retrospective mRNA profiling analysis of muscle samples from fetopsies, infants (8-10 months), and symptomatic
patients (5-12 years) was conducted to assess stage-specific molecular pathways in DMD
• Results showed that TLR 7 was significantly over-expressed in symptomatic patients compared to age-matched controls (+6.6-fold, p
I don't like sarcasm but your point is well taken, there's a lot of good reason to believe this will be an important data release for the Company and all its shareholders. Doody, who I spoke to yesterday, seems to think so. We discussed how the market, viewing the muted share price, seems to think otherwise. I did ask him if he thought the market would treat it as a "sell the news" event. Nobody knows what the market will do, but he pointed out the future catalysts, also mentioned by Amano, and from our conversation and my due diligence, I think this will be a pivotal event for the Company and investors going forward. Good luck all longs, Jr.
Hope it's something like a vacation and not a crisis of some sort. He put out a lot of energy here to IDRA investors, same on TROV board, and well, you know that other company he made sure everyone knew about, last trade 2.88, low of day 2.55. If he used margin, that's the dreaded big one. Hope he's OK.
Since IDRA is entering this space in Q1 with their announced new trial for IMO-8400 in Duchenne Muscular Dystrophy, investors here may want to increase their knowledge of who the players are currently in this race for treatments of this Orphan Disease that affects only young boys. In that regard, here is a story about two of the leaders in the space.
November 20, 2015, 10:46 A.M. ET
BioMarin’s Pain is Sarepta’s Gain, Right? Maybe Not.
By Ben Levisohn
Shares of BioMarin Pharmaceutical (BMRN) have tumbled today after FDA reviewers fretted about the efficacy and safety of the company’s Duchenne muscular dystrophy treatment. Sarepta Therapeutics (SRPT), which makes a competing product, is soaring. Leerink’s Joseph Schwartz and team say BioMarin’s issues are “not necessarily positive for Sarepta.” They explain why:
We don’t think the FDA’s harsh analysis of drisapersen efficacy bodes well for Sarepta, which is likely to face similar if not worse concerns due to very low patient numbers and aggressive statistical analysis. The FDA may also reason that the very limited data for Sarepta doesn’t necessarily disprove a potential longer-term safety problem in large numbers of patients, despite mechanistic rationale for why this may be the case and obvious issues with drisapersen. However, like BioMarin, we believe Sarepta still has a good shot at approval, and BioMarin may be entitled to a significant royalty from Sarepta if they are able to reach the market.
Shares of Sarepta Therapeutics have soared 30% to $33.77 at 10:39 a.m. today, while BioMarin Pharmaceutical has dropped 4.9% to $98.11.
Sentiment: Strong Buy
And of course, there's the EU market and ROW (rest of world), so should they get approval from FDA, there is considerable further upside with this and their other targeted orphan disease medicines (DMD). Yesterday Tim Walbert, CEO of Horizon Pharma, was interviewed at a GS conference (archived presentation on their website), and they are moving their emphasis from primary care medicines to orphan diseases which they view as the strength of their future. That metamorphosis began with their purchase of Vidara and the drug Actimmune, approved in US for two indications, but they've started a phase 3 testing for Friedrich's Ataxia, which will potentially add 500M to $1B of revenues beginning second half of 2017. With IDRA's focus being split between oncology, autoimmune disorders, and rare diseases, their diversified approach will serve them extremely well. There will be many breakthrough therapies developed in all these areas, making them eventually the powerhouse we envision, going where ISIS cannot go and with fewer side effects.
With data release in April, assuming good date, share price spikes and they issue secondary into the strength of the higher share price, giving them strong balance sheet and stable company available for buyout or partnership.
CORRECTION: This article contained dated information. When I was talking to IR yesterday and mentioned my concern over the target market being so small, Bob directed me to the Biocentury slide presentation dated September 10th, slide#15, and from their research the disease incidence is much higher, indicating approximately 25,000 lives in the United States. Since this disease usually attacks the facial skin and/or the hands, the likelihood of people desperately seeking the best treatment options is quite high. Orphan diseases are very lucrative disease targets, and you won't have HMO's, PBM's or Congress interference with pricing. After what they demonstrated in their POC trial with psoraisis, clearing up these unsightly skin and damaging muscle diseases--there is a very high likelihood of similar successful trial outcomes.
When we have a gap like this, usually the MM comes back to fill the gap. I'd save some dry powder for another chance to get shares, if you are thinking of adding to your position. Breakaway gaps do happen, but more times than not they get filled.
Give it a rest Phil. Since when do message board opinions/projections have fulfilment
obligations? If you are entertained and can express yourself here, the venue is working. If you are looking for professional advice and accountability, you are looking in the wrong place. See all the red thumbs? You have misplaced expectations.
That was you buying today, good timing, the shares were weak in early going. Thanks for pushing us up! I'm light there, 2,500 shares. Here heavy, feeling it too. ASH and DLBCL can't get here fast enough for me. I have a big tax payment to make in January.
"lack of transparency, cutting conference calls short without being willing to answer questions" These things you mention here are beside two quarters in a row of disappointing results, were reasons I sold all my shares with a couple days of their ER. While I can forgive a miss, I cannot undue the complete loss of confidence suffered, as lack of transparency and shutting down the call after only two questions. Their secretive management style was something that I lost sleep over and never again! I saved myself a bundle by selling, and yet stayed six months too long by giving them the benefit of the doubt.
One seldom hears about these kind of real life stories, you would think there would be more stories like this? His plight is one that should be more often contemplated by the plethora of shorts out there. I'd rather read about it happening to hedge fund shorts operating in the dark pools, with massive capital funding and huge short positions, than to an isolated retail short. The small guys get hurt and more often it seems. Nice of anyone here to actually respond to his crowd funding plea.
Now to the point, shorts here are facing a real risk leading up to 8400 results at ASH. I am encouraged by the Company's plan to have a follow on conference call on Monday December 7th.
Wow, another investor who comes to the STML YMB! It's like nobody is home here. Thanks for your post, I agree with your assessments, we have a good institutional base. Some day the value here will be recognized. I hope we do have a good response after ASH, the stock has a bias toward selling off rather than gaining in share price, one would think it would be doing better with the poster presentations out, and ASH news anticipated.
...they could take this down again this close to ASH. By now I had expectations of being at a much higher level given anticipation of forthcoming data at ASH, but the market for biotech hasn't supported a run-up here.
How many here actually sold shares at the 3.60 level, expecting to replace with more shares, at lower price per share? I believe most here are not trading but merely adding. Every time this happens, it's deja vu. We are caught in what seems like a 'wash, rinse, repeat' cycle of being range bound, except that the recent high was a lower high historically in our range. After we hear WM results and listen to the conference call on December 7th, let's hope we can establish a new range, and new floor for the stock, for 2016.
Doody having stated that the Company is now thinking in terms of 5-year plans for each of their drug candidates, they need to set forth a timetable of forward catalysts so the stock won't go through another period like 2015 where there were long periods of no news, giving plenty of room for the bashers and short sellers to execute on their agenda.
We need what Amano refers to as "no room for scientific ambiguity" coming out of ASH. The conference call and December 7th morning PR will hopefully give us that. GLAL
Thanks, I was going to call Bob today myself, but you're absolutely right, there's no better place to get accurate up to the minute information.
Where do you see any pr about the Company planning a conference call? It's not on the website or Yahoo, and after lube yesterday spoke to IR, they told him there would be a PR Monday morning. I know you wouldn't post this unless you saw it somewhere. Thanks.