I'm sure if there is a market for it, they will develop it. They have spoken to how flexible the technology is - it is just a question of prioritizing opportunities/investments.
Just to show how broken the market is, they last raised cash two years ago (Feb 2014), selling 6.325M shares at $18.95 per share for a total raise of $120M - the current enterprise value is around $120M. You can argue that the market was inflated when they sold the shares, but given the pipeline progress since then, it is hard to imagine the value cratering to where it is now.
By all accounts, the earnings call was solid - plenty of reasons to believe this company will be worth a lot more in 18 months (w bacteria panel on the market and hemostasis on the horizon) and they have the cash to get there. I could understand the lack of speculative value if the technology was unproven, but that is certainly not the case here. There may be some execution risk on the marketing/adoption side, but there really is no competition that can touch this technology and the odds are that they will dominate the market within 5 years.
They didn't miss - they only pre-announced revenue (which was the same) and the earnings of -0.17 include warrant revaluation of -.06 , so the adjusted earnings was -0.11 vs the estimate of -0.15. Analyst estimates never include warrant revaluation.
Your "simple" analysis assumes that the FDA's preliminary assessment of the data vs natural history is the final word. The company has already pointed out flaws in the FDA's assessment of the data and the AdCom could come to a completely different conclusion - unless you believe the FDA will ignore the AdCom, it is not that clear. The AdCom is likely to conclude that there is some dystrophin being produced, but nobody has the answer on what level is required to produce a clinical benefit and the data on the 12 boys may not be conclusive of a clinical benefit, but is sufficient for conditional/accelerated approval.
Unfortunately, yes - even if you assume that the guidance is conservative (it better be), they won't be close to breakeven until late 2017 or 2018. As long as the placements and recurring revenue continue to grow as they have (10%+ quarter over quarter), then the share price shouldn't get hit too bad in the near term. The first half of 2015 was weak, so the comparisons the next couple of quarters should be easy to beat on the 35% guidance - they would only need to do $16M of revenue in Q1 this year to exceed 35% growth over Q1-2015 ($11.7M).
The technology and the growth story is still attractive, but I still am underwhelmed with the execution by this CEO. They have talked about penetrating international markets for 2+ years and I believe in today's call he indicated that international revenue should grow at a similar rate as the US market - the international revenue is currently negligible, so 35% on top of that is meaningless. Two years ago, I thought they would be profitable by now and was hoping they wouldn't sell out too soon - now I am hoping they sell the company to a larger firm that realizes the potential of the technology - waiting for this CEO to tap that potential is frustrating.
I am hoping their guidance is ultra-conservative - they have already indicated that they feel comfortable with the Q1 analyst estimate of $17.6M, which is 50% above the q1-2015 revenue ($11.7M). With the added sales reps that are supposed to start producing in the 2nd half and traction in the DermaCell line, it is hard to imagine them only doing 30% in the back half of the year?
PTCT and Etep address completely different patient groups/mutations - approving Etep will do nothing for the patients that are targeted to benefit from PTCT.
The float is 32M shares and 26M shares were traded yesterday - I'm guessing that the shorts closing out their positions is what kept the above 15 at the open yesterday. I wouldn't count on any meaningful squeeze from here.
Your post makes no sense. They are expecting a CHMP decision next month and they don't have a meeting with the FDA until next quarter. The EC is not waiting on the FDA opinion - that won't come until 2017.
#$%$ are you talking about - what does Listeria have to do with Migalistat? As the CEO spoke about on the call, the life-threatening issues of Fabry are renal and heart issues, which was the focus of the data package that they presented to the FDA last year. Then, out of left field, the FDA decided they wanted to see data on the impact of Migalistat on GI issues - Fabry apparently causes serious diarrhea, which is not life-threatening, but a quality of life issue.
IMO, this is just another case of the FDA protecting an established market for big pharma - in this case Fabrazyme from Genzyme. It was clear from the Migalistat clinical data (the largest phase III studies ever done in Fabry) that Migalistat was at least as effective as ERT in dealing with renal complications and better than ERT for heart complications. When it comes to quality of life issues, Migalistat is an oral medication, while Fabrazyme is an IV infusion every two weeks. From a safety perspective, many patients can't tolerate Fabrazyme due to adverse immune reactions. The delay by the FDA is a travesty.
Regardless of how you perceive JC, the idea that the EC is waiting on the FDA is senseless. Is JC too enthusiastic about the prospects for Migalistat - possibly, but he is in a position of knowing that the drug works, has substantive clinical data supporting efficacy and safety, and unless he is lying, initially received positive feedback from the FDA on whether their data package was supportive of approval. No doubt, he has toned down the enthusiasm - other than saying that he was proud and happy with how the team dealt with the oral presentation at CHMP, he offered no positive spin or speculation on the prospects for approval. What comments from today's call require a "heavy dose of salt"?
I am calm - I'm simply calling you out on what I consider to be an incorrect assessment of the situation. There are a number of examples of the EC allowing drugs on the market before the FDA and there is no reason to believe that the EC is waiting on the FDA in this case. Your characterization of the FDA is a bit simplistic - like most govt agencies, they aren't attracting the best and brightest and political/monetary influence can play a significant role in their decisions. Your implication is that there is something controversial or missing from the data that FOLD has provided the agencies and these delays are warranted because of these shortcomings. I choose to believe that the data is substantive enough and they are just getting the normal, capricious bureaucratic treatment that most companies do, possibly influenced by pressures from Genzyme.
What are you talking about - the drug does not cause GI issues - this is not a safety issue with the drug - the FDA wants data on what level it improves the GI issues associated with Fabry disease. The company is putting together the data that they have, but because it was not the focus of either of the trials, my guess is that the data will be incomplete. Then the FDA will be in the position of saying "We told you to design the two Phase III trials to measure the impact on renal and heart performance, but now that you have that data, we decided we want a new trial to measure the impact on GI symptoms". If that happens, you know the game is fixed for Genzyme,
I don't think you have this right. They certainly don't have Phase III trials started for Exons 45 & 53, but the 10-K shows drugs for both Exons already in the clinic - 53 is in Europe and 45 was "started in 2015" in the US.
avii - ESSENCE is the Phase III trial for 45 & 53. I don't think the Etep approval in any way hinges on 45 & 53 being in Phase III trials.
This is a Phase 1/2 trial with a 12-week study period that is placebo controlled (8 on drug, 4 on placebo) - the primary endpoint is safety. I believe after the 12 weeks all 12 patients are eligible to stay on drug in an open-lable extension. The final completion date being two years out may involve final safety readings during the extension phase?
My Fidelity account showed a news ticker that Wedbush had reiterated their outperform rating on 2/29/16, but there was no narrative on price target or what prompted the reiteration. I did find a narrative on their reiteration on 11/20/14 - that had a price target of $52 and was prompted by their enthusiasm over OMS-721. They believe OMS-721 will provide strong competition against Soliris, which is on pace to generate revenue of $2B annually.