I hope you are correct. I was more than a little disappointed that the company just blew right past the deadline with no news release saying it was imminent, or they were all set just waiting on the FDA, or something. To just ignore investors as they missed the deadline is unthinkable.
I guess it is possible for them to have filed with the FDA and just not announced it, but that is pretty unlikely.
The company would be wise to release a press statement stating they were ready and done, whenever the FDA was prepared to receive the NDA filing.
Getting the government to do anything in December is a miracle. I don't expect it to be anything on the part of PTCT but rather, on the FDA. They said they would complete their filing by the end of the year, they never said the FDA would be at the office to accept it.
So I found one, PTCT's Ataluren, but it's efficacy is lower than Eteplirsen's. I own both stocks an believe that both drugs will get FDA approval. PTCT treats DMD patients wi a nonsense mutation, a condition for which there are no known treatments at all. They had to modify the Intended To Treat patient population to narrow it down to those who have not yet lost ambulatory ability, as efficacy dropped very low in those patients. Still it was hig in early treatment cases, so combined with a perfect safety record, it (Ataluren) should also get approved (in addition to Etwplirsen). PTCT is supposed to file their NDA before year end, so they are a half year behind SRPT in the approval cycle.
My guess is that they are waiting on the FDA so while they may be "ready to file" before the end of the year, I doubt anybody will be home at the FDA to receive it.
So I was parsing through the EMA website and did a search on Ataluren that came up with a ton of hits. Reading the 12-11-2015 (careful, their dates show day-month-year) and there is an update on the pediatric plan submitted by PTCT that was accepted (if I'm reading those minutes right). There are several sets of updates, including an update on Sareptas Eteplirsen. The updates are divided up into groups, some have a negative opinion, and we aren't in that category, so hopefully this is a favorable endorsement on the updated filing with the EMA.
I hoped to hear something early, like last week, but knew it was wishful thinking. Most of these. Omanis don't deal with the government much and don't understand that the government shuts down in December. Getting any Government agency to do anything after Thanksgiving is a miracle.
So my gut instinct tells me they have the NDA completed, ready to file, have addressed all the FDA concerns, has the mITT set right, and are all ready to file -- but nobody is home at the FDA to receive it.
In fact, if the FDA accepts the PCTC NDA before year end, I'll personally take a bunch of chocolate chip cookies down there to help them celebrate the Christmas season. I just don't see it happening (from the FDA's side).
I don't think it is any delay per se or anything wrong, just nobody home at the government. As usual.
With the FED move yesterday and several M&As in progress, short sellers are starting to cover en masse, gradually and in a disciplined manner to not cause a short squeeze. Watch for short positions across the biotech space to start covering for companies with high probability of FDA approval.
There will still be shorting of companies who have no products and long lead times on getting FDA fillings in place, but others will see a substantial decline in short interest.
Not just holding, actively buying more shares to take advantage of this dip. Yes, will hold through the Adcom and likely through most of 2016 as the EU will be approving soon after the Adcom.
Nobody does, they just said before year end. That may well be good for the company, but the government shuts down from Christmas to New Years so I'm looking for it before or on 12/18. Otherwise it is too close to the holidays and will slide into January.
Actually, it is more of a misunderstanding. BMRN failed because it didn't have any demonstrable efficacy in any group in the P3 study. Ataluren has outstanding efficacy for patients who were not in the advanced stages of the disease, and had already lost ambulatory ability. Perhaps PTCT should have gone with fewer patients like SRPT did. Etwplirsen showed amazing efficacy, but they get dinged for a small number of patients in the study.
PTCT did the opposite. They threw the net as wide as they could, enrolling any/all candidates, then looked at the data to see where it was most effective. While that was the lion's share of the patients in the study, it didn't show strong efficacy in a small subgroup of patients where the disease had advanced to an irreversible level.
Therefore, they have been able to identify a modified intent to treat (mITT) subgroup that they can show extremely high efficacy with. PTCT also is th ONLY treatment for DMD patients with th nonsense gene mutation. So that, combined with perfect safety - makes it absolutely compelling for FDA approval. Once people realize this, the stock will pop up repeatedly.
It will jump up considerably very soon when they file the NDA with the FDA before year end.
I own both SRPT and PTCT and think they both will get approved because both drugs are very safe. SRPT has higher efficacy across the full patient profile. PTCT treats a different group of patients, and they had to narrow the intent to treat (ITT) to a modified ITT (mITTT) to only include those patients who had not yet lost ambulatory ability. Meaning, once the disease progressed too far, Atalauren was no longer effective. However, it was extremely effective on patients who were still ambulatory. That, combined with the perfect safety profile (no adverse events at all) make it a compelling approval case for the FDA.
Also, PTCT doesn't have any competitors on the horizon. SRPT has BMRN and while Drisapersen is a joke in terms of efficacy, the company BMRN has had significant influence on the situation with Eteplirsen.
I think that PTCT has higher profit potential too, with targets as high as $168 by analysts upon approval. That said, PTCT had a tougher time on the P3 trials because they cast the net too wide on potential patients. They would have been far better served to more carefully cherry-pick the patients and shown perfect efficacy for the ITT group. So there are tradeoffs between them both. SRPT has a much smaller number of patients in the trial, PTCT had many more, but had to rule out a small portion of them because they were too far gone to help. That may be the same for SRPT in time, but is an unknown.
PTCT will file the NDA with the FDA by year end, so has an immediate catalyst. SRPT has their Adcom in January but could possibly get approval even before then.
So I have SRPT as a strong buy and PTCT as an absolutely SCREAMING BUY.
I own equal $ amount of both companies so hope they both do well. Same # of shares, bought in about the same price.
I can't confirm conclusively on the modified ITT, but know the updated NDA filing did narrow the mITT to specifically designate patients who had not yet lost ambulatory ability -- which is the area where P3 trials showed extremely high levels of efficacy.
Also, no adverse events reported still, so safety is running solid too.
I will try to get something more definitive on the NDA filing which is before year end, and I fully expect to be closer to mid-month December.