Maybe someone else has noted this, but the press release specifically states the FDA informed the company on 19 April. However, the news was released after the market closed on 20 April.
That means anyone who bought shares today did so in what amounts to a false market.
I think it's technically legal to delay announcing key business news by 3 days but I'm struggling to think of another example where a company with a critical pending NDA or 510k who delayed informing a rejection by one business day.
It also defies belief that - despite sitting on it for a whole day - they were apparently unable to give a brief summary of the FDA's key points in its rejection.
My guess is the FDA response is absolutely brutal and the company will probably have to do substantial additional clinical work before submitting it through the alternate PMA pathway.
I think a new trial (taking that to be a full Phase III trial) is a severe issue here:
1) They could attempt to use the just started EU Phase III trial (it was a bit of a tell that half the sites for this EU trial are in the US, despite this trial being apparently only for the EU authorities). More likely, the FDA will seek amendments on end-points and may want a bigger trial - adding time and cost.
2) Even if somehow the FDA wants no amendments, ClinicalTrials shows the EU Phase III isn't due to report until late 2018. That means, at the earliest, the US launch is now deferred to H1 2020. The company has nowhere near enough cash to last that long and a substantive interim equity raise will be essential.
3) The company has said they would only proceed with the NET indication if this approval came through. According to the recent 10-K, The NET market is 50% bigger than the acromegaly market. This larger indication is now dead.
4) I think the Servier monthly injection drug goes off patent in 2017. Therefore, the acromegaly market should be much more genericised by 2020 (making a new premium-priced launch much more difficult)
Given the now locked-in cash burn until 2020, I don't think you can rely at all on the book value acting as a floor for the share price next week (the market may move down initially to book value on the Monday opening, if so I'd grab it and sell). Bear in mind there are also nearly 2M warrants at $0.09 so the 'real' share count is about 10% bigger than officially shown.
Having said all that, you could well be right and I'm just being too pessimistic - perhaps Monday's PR will be relatively mild (say the FDA just wants a small additional PK-type trial).
As you've no doubt guessed, i was short here. However, I've been long into NDA's multiple time and been bitterly disappointed several times. My deep learning from those CRL's: have a beer or a glass of wine this evening. No one was hurt here & you'll win this back sooner or later.
The fact they are deferring the CRL explanation until Monday morning (which I've never seen before!) tells me it's most likely a severe rejection (e.g. the first Phase III trial has been completely rejected and/or the FDA have identified safety concerns).
I think was has happened here is as soon as they were informed by the FDA of the outcome (i.e. just after 5PM EST) they halted the stock. This was to prevent any leaks of the outcome being used to illegally profit from the news.
If that assumption is correct, the company has been spending the last hour thinking about the press release they must issue.
I have to say if the news was good, the press release should have come by now as they surely have a pre-prepared version waiting to go that would require very little amendment..
However, if the news was bad, having a pre-prepared version isn't much help. In particular, they will need to give a broad outline of why the FDA rejected the NDA - e.g. a drug manufacturing problem would be a 'minor' fail and likely fixable within a few months at low cost but the FDA being unhappy with the first Phase III trial woudl be a lethal blow.
The longer this goes on, then frankly the more likely an FDA rejection has happened here.
Anyways, just my 2 cents ...
I strongly suspect this is a run-up to Takeda's 27/1/13 NDA on NESINA. NESINA is already on sale in Japan and has a sales run-rate of $475m there. Furiex is entitled to a $25m milestone on US approval & royalties of 7%-12%.
This is also a resubmission as the FDA turned down the original NDA - as they wanted to see more safety data (given general nervousness over side-effect profiles of diabetes medicines). I'd give this 2nd NDA a very good chance of approval - as the FDA have narrowed their concerns to getting more safety data which Takeda included in their resubmission.
If this is approved, then I'd expect a pop to the $25-$30 price range. I'd sell out quick & come back in late 2013 for the MuDelta Phase III readout - probably due in Q1 14.
I hope this is of help.
I think the recent vicious sell-off has to do with institutional owners. According to Reuters, institutions own a relatively high 62% of shares - even worse, the institutions own 97% of the free float.
Here's the problem as I see it: a lot of those institutions have to disclose their positions at the end of each quarter to their investors. Based on the current $2.34 price, that equates to a fall on their Anthera position of 62% during Q1 12. That's not a pretty statistic. However, the fund manager can make his/her presentational problem go away by selling before the quarter end. My guess is several fund managers have been doing precisely that - particularly once they realised any run-up to the Lupus PIIb catalyst in June wasn't going to start in March.
If this theory is right, then there'll be more carnage next week. I wouldn't be shocked if it fell to a $1.50-$1.75 range by Friday week.
That would make it a brilliant entry point for the Lupus catalyst. It also suggests that no matter how painful it gets next week then existing shareholders should stick with their position - as the lack of persistent institutional selling in April should create a strong rebound.
Well, that's my 2 cents.
Some points :
* The investigator sponsored PII trial had no placebo comparator - i.e. it was just a baseline comparison. Therefore, the recorded discomfort response could just be a 'placebo effect'.
* I appreciate a different dye was used - but I'm conceding that it has a fighting chance in the signs end-point. All my concerns are focussed on the symptoms end-point.
* A point I didn't make in my original E-Mail is a dry eye indication trial success is almost an oxymoron. Restasis has barely any efficacy. I had intended to short ISTA but the result came earlier than I expected. I think Regenerx management have had dollar signs in their eyes (dollar eye disease?) chasing after dry eye while an orphan indication like eye ulcers would be much better - I do believe failure here will say more about management than thymosin-beta 4.
I've researched this stock but decided to pass playing on it. I thoroughly agree success could quickly lead to a $50m-$100m enterprise value but here's the problem ...
This trial has 2 co-primary end-points : signs & symptoms of dry eye versus placebo. ISTA Pharmaceutical failed a Phase III dry eye trial on 28 July 2011. It too had 2 co-primary end-points : signs (measured by dye staining - like Regenerx) & symptoms (measured by patient questionnaire - like Regenerx) of dry eye. ISTA showed <5% for the primary end-points versus baseline but only signs had p<5% when compared to placebo (a saline eye-drop solution). The failure on symptoms versus placebo cost ISTA $73m in market capitalisation on result's day.
The ISTA failure to beat placebo on symptoms I think had 2 causes :
* Patient symptoms of dry eye are all pain-related and placebos are notorious for affecting pain related end-points
* Several commentators on the result felt the saline solution did actually relieve discomfort in the eye and so has a real (but non-disease modifying) therapeutic impact.
I would note that RGN-259 did show decent sign (i.e. eye staining) efficacy in animal pre-clinical tests but animals aren't great at filling out discomfort questionnaires so there's no evidence for efficacy on that score.
Putting all this together, I think they have a fighting chance of passing on the signs end-point but will fail on the symptoms end-point (versus placebo). Given they are co-endpoints, that's a trial failure. Bear in mind ISTA had an SPA for their Phase III so the FDA want both signs & symptoms efficacy.
It is possible getting a result on the sign end-point and/or spinning a yarn about getting p<5% versus baseline rather than placebo could lead to a temporary spike on results day - maybe a 2-3 hour blip.
In my view, the only real thing going for this trial is management have been very specific on the catalyst timeline. This may lead to a share price run-up & I have retained the stock on my watchlist for this reason - and this reason alone.
Sorry to be harsh here, but they just failed a Phase III trial on a $500m+ indication. Who cares about a beat or not on earnings?
I reckon this will be down 30% tomorrow.
They do have another Phase III read-out on a reduced BROMDAY dosage in Q4 2011, so a swinging stockmarket reaction over the next few days could actually represent a decent entry point.
I think it's falling because it will be deleted from the Russell 3000 index at end June 2011. The Russell indices are 'reconstituted' once a year at the end of every June. Russell published the proposed 2011 additions and deletions to their funds on 10 June - NuPathe was on the deletions list. In addition, traders can second guess the likely additions and deletions a couple of weeks in advance as Russell publish the technical rules behind their reconstitution technique.
Deletion from the Russell index is a real technical hit - index trackers have to unload it & many institutional fund mandates require stocks to be on Russell indices. I suspect the price will drift down 'til end June - probably with a nasty fall around the reconstitution date.
In my view, this would be a perfect buying opportunity for the upcoming NDA catalyst.
Actually, I was more saying that the FDA could well have faxed the news to Xenoport by now but the licensing agreement with GSK gives GSK control over how the public announcement (approved, CRL or delay) is made.
I came across an example of this with the Canadian biotech Bioniche who had ceded complete media disclosure to their partner, Endo Pharmaceuticals, of the outcome of a Phase III trial in bladder cancer.
... or am I the only one to consider that a legitimate reason for delaying the PDUFA announcement is the partnership deal with GSK?
For instance :
* Might a common statement have to be agreed which inevitably takes time?
* Might GSK have sole control over announcements (particularly after their deal was restructured last year)?
* Maybe with GSK being a British company (with a 5 hour time difference) can't be bothered releasing press statements after midnight local time?
Have a look at SKYEY.PK, ARDM.OB and CRMD. All have major catalysts in the next 3-6 months.
PCRX is also a larger cap stock that's been overlooked - IPO in Feb. 2011 means not enough investors know about it yet.
I'm I the only person who reads the newswires?
It passed!! Key facts
* P-value < 0.01%
* All 3 radiologists got an improvement
* Huge improvement versus standard of care (e.g. zero SAE versus 55 SAE)
I was doing some DD on this stock on the basis today's fall may be overdone.
However, I noticed on Clinicaltrials.gov that their Azixa Phase II trial on Glioblastoma Multiforme (brain cancer) was marked down as suspended last Thursday, 19 August. The trial's Identifier is NCT00892931 if you wish to verify this.
Oddly, neither Epicept or their partner, Myrexis, have made any comment on this development.
To be fair, until the background to the suspension is explained, it's not fully clear what the implications are. However, it has to be marked down as a negative development as of right now. It never rains but it pours ...
Overall, this seems like a fantastic development to me.
* Their last 10-Q had already disclosed another diabetes trial would be needed to demonstrate safety so this isn't news. Stopping it after the efficacy readout will also heavily reduce cash burn as they seek to partner.
* They were always going to have to partner after the Phase III efficacy results given the salesforce & marketing budget needed for a diabetes drug. Again, move on – there’s nothing to see here.
* A new drug to treat high triglycerides is a big deal. Take Amarin Corp whose only pipeline compound is in Phase III to treat high triglycerides. It currently has an enterprise value of $211m. Given this diabetes trial could now also be regarded as a Phase IIIa trial on triglycerides, an enterprise value of perhaps 50% of Amarin's could be ascribed to Spherix. That's a mere 5 times Spherix current capitalisation.
* I haven't done DD on Spherix's patents but I'm guessing they don't mention D-Tagatose as effective against triglycerides. If so, they could file a method-of-use patent with coverage beyond 2030 (and short patent coverage is Spherix's main current problem).
* The press release hints the triglyceride efficacy is dose-related - i.e. 7.5g dose led to a 21% lower count than 2.5g. The Phase III trial is using a higher dose again at 15g.
* The Phase II trial is also reasonably powered with 172 patients spread across 3 dosing arms. That’s an average of 57 patients per dosing arm being followed for 6 months.
* A reduction of 21% looks clinically meaningful. For instance, Amarin's latest presentation on their website shows 6 Japanese studies of their compound gave an average 21.4% triglyceride reduction.
* The press release says they are “… eagerly awaiting the efficacy results in a few weeks” which is a lot tighter than other mentions of “later this summer”. I’d speculate this means data in early August – when also taking account of enrollment completing on 12 January 2010 & the 6 month treatment phase.
The quick answer – May/June 2010.
The more detailed answer is I’m aware of 3 ways to estimate the results date here :
* On 31 March, 2009 Bioniche issued a press release saying they had completed recruitment in the trial. The primary end-point is measured 12 months after treatment. Therefore, the final recruited patient will have reached their primary end-point measurement at end March 2010 (assuming they didn’t drop out earlier). At that stage, with all primary end-points measurements made, the analysis phase for the trial’s primary end-point begins. This varies from trial to trial but should be in the 4-10 week range. This gives end April 2010 to June 2010 as the answer.
* The FDA requires details of all human drug trials to be logged on clinicaltrials.gov. Linked to this, the FDA requires the ‘Estimated Primary Completion Date’ for the trial to be shown. This currently shows April 2010. The analysis phase of the trial would take place after this (discussed above) and so gives May/June 2010 as the answer. Finally, the April 2010 entry was updated on 13 May 2009 which is after the final patient had been recruited and so should be accurate – some trials leave out-of-date estimates on their clinicaltrials.gov entries for long periods of time.
* Bioniche’s 5 November 2009 presentation to shareholders states “results to be available mid-2010”. I would interpret this to mean June/July 2010.
These 3 different methods produce a range from end April 2010 to July 2010. However, the strongest area of overlap occurs in May/June 2010 and that, as a result, is my estimate.
I hope this is of help to you.