What a dimwit - the guy says cash is $5.58 per share with very little debt - the $8.65M of debt is over $2 per share. On top of that, the $5.58 of cash was the June 30 balance of $23.0M - they were burning almost $8.0M per quarter, so that $23.0M is likely down to $15M or so, so after paying off the debt they likely have around $8.0M, or roughly $2 per share, which is what it was trading at. If anyone believes Martin is going to pour more money into this company to support their early stage pipeline, they are dreaming (certainly not at $10+per share) and he likely already cashed out his windfall and is moving on to the next group of suckers.
I disagree. Unless she believes her testimony will aid the chances of Etep getting approved, there really is no rationale for advocating for a Drisa approval. She has been adamant and vocal about her conviction in knowing that Etep is working for her boys - her testimony in favor of Drisa will at the very least diminish her message on Etep and as other posters have pointed out, a Drisa approval could very well cause complications in the market (patent issues, insurance issues, etc.) that may complicate her access to the drug she really believes in.
Perhaps her motive is to remove the potential excuse the AdCom will have in assessing Drisa by assuring them there is a more viable drug in Etep and they don't have to settle for worse drug, simply because there is an unmet need. Either way, her testimony has as many pitfalls as benefits for the prospects of Etep.
I can understand Jenn's motives, but the other question is who decided she would be invited to speak to the AdCom? She has no direct experience with Drisa, which is what the AdCom is set up to analyze. I don't believe BMRN has any control/input into who is allowed to speak at the meeting and I doubt they would allow her to - she can only cloud the picture for Drisa. If they wanted an objective advocate for the parent community to address the unmet medical need, etc., I'm sure they could have found someone who has no ties to either drug. Am I wrong, or is it strange she would be invited to speak?
The probability ratings are "intriguing", but again, does this clown provide any rationale for why SRPT would get rejected? Is there any analyst that has dug into and addressed the case for conditional approval, dystrophin production, etc. or is it simply they don't believe the FDA will approve a drug based on a trial with N=12? SRPT has provided the FDA with every piece of evidence they have requested and by any objective measure all that evidence has been overwhelmingly positive. So what exactly is the case for rejection?
Only a "brilliant move" because of retail suckers. KBIO tried to shop their assets and got no takers - their technology doesn't work - what do the buyers of the stock think Martin is going to do with the company? More likely, he is going to cash out on this wave of stupidity this morning and the shares will retreat back to $1?
The potential breadth of their hypoxia platform is downright outrageous - enhances the efficacy of conventional chemo, EGFR and now immunotherapy - and they have no competition in the space. The also stated they have hit the required "events" in both the pancreatic and STS trials - they (inc Merck) are in the data scrubbing stage (i.e. verifying the accuracy of the data from the various sites) and expect to announce top-line results for both trials around year-end. If both trials succeed, pancreatic and STS are just the tip of the iceberg. If they fail, there may still be plenty of value in the TH-4000 drug. GLTA.
That's BS - you can't get forced to cover from AH activity. The normal rule is 3-5 days to cover and that is based strictly on end-of-day pricing in the regular trading session.
Omidria is the only FDA-approved drug - there is absolutely no relation to what Valeant was doing, which was circumventing doctors' prescriptions and/or insurance company rules in order to get their drug sold over a cheaper generic or other FDA-approved alternative. Beyond that, this is a one-time charge (vs a potentially recurring prescription) at cost that is reasonable given the benefit it provides the doctors. Any comparison to Valeant's issues is laughable.
The chances are between zero and nil - why do you indulge in such fantasies? Etep has an established PDUFA date in February and the chances that the FDA renders and opinion before that date are zilch.
bionerd - what is your read on the implications of the safety issues? Are you aware of any drug for a chronic illness being approved with a safety profile as bad as Drisa? I'm vaguely familiar with the AdCom process in that they take separate votes on efficacy and safety (DVAX got burned on the safety call a couple of years ago), but is the vote whether it is safe enough for approval or some other rendering on safety?
usagary - a confirmatory trial is only required if conditional approval is given - if the Drisa is given full approval, there is no requirement for a confirmatory trial. BMRN has put the FDA in a box, because if they give them conditional approval and require them to do a confirmatory study, their ability to enroll that study (at least in the US) would be severely compromised by the approval of Etep. Frankly, given the "messy" clinical benefit data and horrible safety profile, the FDA should kick Drisa to the curb, but it doesn't seem like that is in the cards?
The patient advocates will include a mom who's son was on Drisa but couldn't handle the injections and is now on Etep, but she will reportedly still advocate for Drisa's approval. Jenn McNary will also speak as an advocate for Drisa #$%$) under the banner that DMD parents need choices. When you have the CEO of SRPT speaking to the positive impact of a Drisa approval and parents that know the value of Etep jumping on that bandwagon, you have to believe the political stars are aligning for BMRN. Looks like the best we can hope for is the FDA puts a black box on the Drisa label and/or delays the approval for a REMS or further safety data from their confirmatory trial, which they have yet to launch. AF's article doesn't even speak to dystrophin production evidence and the lack thereof from BMRN - in the long run, how could they ever get the smaller Exons approved without dystrophin production evidence. Etep will own the DMD space.
How often does the CHMP meet - they said in the latest earnings call they did not expect a CHMP review until "late this year or early 2016", so is this really a delay?
I found it strange that in the statement recapping the safety profile they noted there have been no pulmonary embolisms - is that an AE that has occurred with Drisa or some other RNAi drugs?
Have you read any analysis of the BMRN's prospects for approval that speaks to the quality of any of their data? Everything I have seen is just platitudes about BMRN's expertise in the rare disease space, the political pressure on the FDA to approve something and the perceived advantage of BMRN having their drug reviewed two months earlier. Show me one analyst that points to the core efficacy data that will sway the AdCom panel, let alone the FDA. The analysts that asked the tough questions on BMRN's last earnings call were dismissed - in BMRN's view dystrophin production evidence is not important, the failure of the Phase III trial can be rationalized with excuses about the lack of expertise of the clinicians that administered the trial, and safety issues are manageable and "seldom" lead to discontinuations. They are left with a small subset of boys under 7 that supposedly benefited in the 6MWT for a short period of time, when the historical evidence of the same group shows that boys are just as likely to improve as decline at that age. Show me any analyst opinion that is backed up by a robust review of the data that BMRN has publicly disclosed?
The key to valuing this going forward is to guess what the final share count will be by the time the bleeding stops - hopefully it will be less than 500 million shares? Even at that number, the market cap is only $10M - you would think the technology is worth at least $50M+, depending on how much needs to be invested to get it past the FDA?
They're in the dilution trap - until they have the capital to get them to self-supporting/breakeven operations, buyers will sit on the sidelines - in the absence of buyers, sellers have to take what the bottom-feeders will offer.
Pricing is never an issue addressed by an AdCom - their only directive is to determine whether the clinical evidence at hand is sufficient to support that the drug is effective and safe - separate votes are taken on each. No doubt, their vote can be influenced by the severity of the disease and the availability of other treatments - the whole risk-reward context that the parent groups have pushed to make part of the equation. Pricing isn't even an issue the FDA can consider in its deliberations on whether to approve a drug, so the idea that a pricing "pledge" may come into play is ridiculous.
speed - when it comes to dystrophin and the 6MWT, there will never be a tight correlation of one measure to the other. This issue should have been put to rest by now - CG tried to nip it in the bud, but some analysts keep resurrecting it and EK is too nice to put them in their place. You are taking what is generally agreed to be an imprecise measure (% of dystrophin positive fibers) from a different location on the body and trying to correlate that to a performance measure of muscles in a completely different location and that is not even the biggest problem - the performance of the patients leg muscles is primarily influenced by the degree to which the muscle has already been destroyed by the disease - something that is impossible to measure accurately. There is so much "noise" in each measurement that any effort to correlate the two is foolhardy. The ultimate clinical proof of Etep will be confirmed when the younger boys make it past their teens still on their feet and the older boys continue to breathe and use their arms into their twenties - that evidence is a good decade away.