I'm guessing this may have been posted before, but I missed it, so for those of you that haven't seen it - go to YouTube and watch the video of Christine discussing her son Jett - it's titled "Is Eteplirsen Effective in Non-Ambulatory Duchenne Patients". She describes the quality of life improvements that Jett has experienced after being on Etep for 9+ weeks. I defy any AdCom member to listen to this testimony , which I am sure she will provide at the meeting, and determine that Etep is not worthy of AA.
The convenience advantage of 721 vs Soliris is secondary to the safety advantage - Soliris weakens the immune system, subjecting patients to life-threatening infections - that is why patients must be pre-treated with a vaccine to prevent meningitis. You've heard of drugs with "black box warnings" for safety issues - Soliris has what is called a REMS warning - it means Risk Evaluation and Mitigation Strategy - which is beyond black box on the safety spectrum. Assuming 721 is as effective as Soliris, the idea that doctors (or patients) would continue to stay with Soliris is laughable - the doctors would be subjecting themselves to malpractice suits if they kept a patient on Soliris and they developed a serious infection. Within a year, OMS721 would have 90% of the market.
Unfortunately, it will be a long trip - the pivotal EG-1962 trial will be completed in 2018, unless the interim results are strong enough to stop the trial. The biotech market used to value strong Phase II data, but not much lately - will that change over the next 18 months or are we bound to flounder below $10 until 2018?
Exactly - they will be cash flow positive the 2nd half of the year and the possibly getting accelerated approval for 721 in the first half of 2017 (per the call today, the FDA put the prospect of accelerated approval on the table) - combine that with three more potential blockbusters, and this has a good chance of hitting triple digits by the end of 2017..
More importantly, they will be seeking accelerated approval, so it could be approved and marketed before the Phase III trial is completed - early next year, perhaps?
You missed - that was way too aggressive. They did $6.7M in Q4 and would need $20M+ per quarter to be cash flow positive - not sure why you think 40% per month is a reasonable growth rate?
The delay probably has to do with the way the FDA is dealing with Etep - dismissing their 6MWT data and their dystrophin production levels. If the Exon 53 trial is designed in a similar manner and is expected to generate comparable results, why go ahead with it?
A "TRUCKLOAD" of studies behind it - the prescription insert only references a few studies with a total of 178 patients. Soliris also requires pre-vaccination against meningitis infections. Based on the clinical experience of OMS 721, doctors are looking to use it in compassionate use because Soliris is not effective enough. Not sure why you are so hyped up about Soliris - by all accounts it is a moderately effective drug with severe side effects.
A treatment that is just as efficacious, safer and more convenient would not compete? The long-term issue is that Soliris comes off patent in 2017, so will payers push patients to a generic Soliris, even if OMS 721 is safer and more convenient?
Correction/Update - Soliris generated $2.6B in revenue in 2015. From what I have read, OMS 721 will is more effective, is safer (Soliris has a REMS - worse than a black box warning) and more convenient dosing (subcutaneous injection vs IV infusion for Soliris.
As promising as OMS 721 seems to be, your point about no news on the EU front for Omidria is disturbing. It was approved for sale in the EU on 8/3/15, so we are a full 7 months since approval and no marketing arrangements have been established? Maybe the patent protection in EU won't prevent a generic competitor from entering or compounding is a more accepted practice, although if that was the case you think they wouldn't have spent the resources to gain approval. Either way, it is a stain on the potential value of Omidria.
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.
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?
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.
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.
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,