Am I the only investor following this company? UniQure reminds me of BlueBird and I think that it has similar potential. I'm buying shares on weakness. Is anyone else out there following this story? Superb team and superb science.
I'm not saying it would go through, but a group could put an offer on the table tomorrow if they felt the urge. The good thing for us is that it props our valuation up while the company moves forward with the NDA filing. I don't think $50 could get a deal done, but it would be a good place to start discussions.
I think the story was a bit different then because of the timeline involved. We knew that it would be a late 2014 NDA filing so they really had no positive catalysts for investors to grab. It was easy to have weak hands, but now there are definite catalysts. We don't know what will happen between now and the late 2014 filing, but additional trial launches are possible (schizophrenia, sleep disorders in Parkinson's, etc). Any additional trial launches will force analysts and investors to reevaluate revenue projections. Also, it's not yet clear what impact Breakthrough Therapy Designation will have. My guess is that it drastically increases the odds of a priority review once the NDA is filed, but that remains to be seen. As another poster suggested, it could allow for a rolling submission but we're so close to the finish line now that my guess is that they'll submit all at once. They're clearly putting together a winning NDA filing as they're rushing nothing. That should be great for investors as a clear approval will open many avenues for Pimavanserin. It's a franchise drug in my opinion and finding the right indications will be the key. I think they will exploit other areas of Parkinson's as it has shown evidence of helping sleep issues and perhaps levodopa induced dyskinesia (movement disorder). Just sticking within Parkinson's could make Pimavanserin a tremendous success, but it clearly has potential well outside of Parkinson's.
Anyway, the bottom line in my mind is that investors are now recognizing the potential of Pimavanserin and even Cramer brought this up. The key factor holding this back is FDA approval and that timeline is right in front of us now. Acadia has stated that they will file the NDA late this year. That's not far off. The Breakthrough Therapy Designation strongly suggests that we will get a Priority Review designation. That's not far off either. Stay tuned as the timeline is not distant anymore.
Perhaps, but Acadia is far superior to Avanir in terms of having a proprietary new chemical entity that is likely going to be approved in PDP. I would say the difference between Acadia and Avanir will be the sustained surge in Acadia shares once pimavanserin gets to market for PDP. If clinical trials for additional indications start to click the price could run very high for a long period of time. Pimavanserin in a franchise drug....you can literally build an entire company around it and I'm starting to believe that's exactly what Acadia is going to do. People have joked about Acadia being another Celgene, but there is truth to it. It does have that kind of potential. No guarantees but Pima just has incredible potential throughout CNS. I personally don't believe the stock price will stay down for long. We're getting too close to big milestones.
All my opinion and please do your own DD.
It's not the same indication. Psychosis and agitation are distinct. Also, this was a Phase 2 and not a Phase 3. It's early days for the Avanir program which is also a mix of existing drugs. You can get dextromethorphan OTC already. I don't think the drop had anything to do with this. The drop is because Acadia was up a lot after breakthrough designation and then Cramer caused it to bump a bit more. However, timelines work in our favor now. The NDA filing and likely priority review designation are coming within the next several months. That will be a huge milestone.
Your description is exactly right. I'm fairly certain they'll go for a priority review and I believe that the FDA will grant it. I think this is why Acadia took so much time to get the NDA submitted. They need everything to be perfect so they can get the six month review. Things are changing here now. There are clear timelines so we know the worst case is an NDA filing in 3 and 1/2 months. Within 60 days of the filing we may get a priority review designation. If that happens there could be a summer approval as you mentioned. And we know Alzheimer's data will be coming late in 2015. There is a lot happening in 2015 so the stock may remain strong here and the timelines are shortened drastically from what they were when we were all talking about this a year ago. Exciting days for sure.
I want to point out that Acadia was granted Breakthrough Therapy Designation and people skilled in FDA processes know that this carries a lot of weight. That is the main reason it shot up so much. BTD increases the chances of a priority review drastically which means the review time will be cut way down. Also, it shows that the FDA understands the importance of this drug which increases the likelihood of approval. Finally, it helps Acadia justify a pricing premium for the drug because it's recognized as an extremely important and breakthrough drug where there is an unmet medical need. There are people who discount Acadia a great deal because formal FDA approval has not happened. However, BTD is assures most analysts, institutions and investors because it shows that the FDA is very serious. Cramer was an afterthought which is why the price barely moved. It's BTD that caused this and it's going to cause a lot more as the NDA submission dates draws near.
I agree. I think a schizophrenia may start soon as well. I love the schizophrenia market. I'm going to try to post some numbers later regarding the size of these markets, but they're obviously mega blockbuster for schizophrenia and ADP.
I'm starting to wonder how Pimavanserin would look in sleep disorders seen in the elderly in general. The current meds do not work well for the elderly at all. It's another blockbuster market.
Six months ago Acadia completed an offering at the $28.50 level and raised a bit over $180 million. What has changed since then? First, we're much closer to the NDA filing and no red flags have emerged. Second, the Alzheimer's study has continued to evolve. Third, we now have a breakthrough therapy designation which exponentially increases the odds that we will also get a priority review within 60 days of the NDA filing. That's a lot of added value and we still have the opportunity to buy at the price institutions paid six months ago.
The next big milestones are definitely the NDA filing, NDA acceptance and Priority Review Designation. I won't speculate beyond that, but it's my opinion that approval for PDP is highly likely and maybe much quicker than we originally speculated due to the breakthrough therapy designation. I don't know where the share price will be, but it should be much higher than today and the Alzheimer's data knocking on the door will bring excitement/speculation to a new high. The funny thing is that Pimavanserin doesn't really get the love it deserves as an off-label drug that psychiatrists and neurologists will try for many other conditions. It reminds me when drugs like gabapentin and provigil were approved for narrow indications, but had absolute booms in sales due to off-label indications. Maybe with time Acadia will expand the label to many indications, but it's just a fact that CNS rarely sees new medications. The indication that gets very little attention is schizophrenia, but if Pima can really be given alongside a very low dose of risperidone with equal efficacy to normal dose risperidone it's a huge breakthrough for patients. The side effects of atypicals can really be terrible. Very exciting asset for sure.
Finally, we never give love to the pipeline but Acadia will likely have another winner at some point.
All my opinion so please do your own DD. Looking good here fellow longs. Btw, I added.
My guess is that he will be on the show at some point. Uli is pretty low profile, but he does tell the pimavanserin story extremely well. Frankly, no one ever believed in Pimavanserin as much as Uli (at least outwardly). He has done one heck of a great job.
I'm very happy to see Cramer finally touting ACAD...it's about time. The pronunciation was classic...I think it just proves that the upside is simply tremendous. Most groups still don't even know the name of Pimavanserin and the stock is going to continue way up as that changes.
Interesting thoughts. I agree about CVR deals, but it's something that must be considered since the Neurogen/Ligand deal was built around the CVR's and Baker brought in Stephen Davis. I probably wouldn't be willing to hold most CVR deals until closure (and haven't held them in the past), but I would be willing to hold for ADP since there is so much potential value there.
There was the line of thinking that a company wouldn't buy a group post-NDA filing and before formal FDA approval, but Roche just killed this concept with the Intermune deal. Having said this, pirfenidone was already approved in the EU and Canada. Still, FDA approval hasn't yet occurred. I think the NDA filing is essential before a deal is done and I think a Priority Review designation will help a great deal. Any potential deal without CVR's would have to give close to full value to PDP and then a big bonus for ADP, schiz and all of the other potential indications. The struggle for Acadia is that the value will blast through the $50 mark really quickly if this works well in ADP. We already know there are signs of efficacy in schizophrenia and I have little doubt that it will capture significant market share there. Some analysts think schizophrenia is tapped out, but current meds are less than ideal and most doctors and patients would prefer a combo of Pimavanserin and risperidone (or another atypical) if it could offer equal/improved efficacy at a much lower dose (to avoid the nasty side effects of atypicals).
Could $50 get it done today? That's just shy of $5 billion. Intermune just got $8.3B and Acadia's Pimavanserin has way more potential than perfinidone. But that's just it...potential. We have the PDP market locked up most likely....if we lock up ADP the value will blast through $50 quickly. I could see $50 happening with a CVR for ADP. ADP is just too large to not get some reward for it.
Baker Bros was a big investor in a public company called Neurogen. It was a small company with Stephen Davis as CEO and a pipeline of several interesting mid and early stage candidates. This is the same Stephen Davis who is now our CFO. Neurogen was a troubled company so has very little in common with Acadia other than a major investor being Baker Brothers and Stephen Davis as the CEO. It's obvious that Baker Brothers likes the talent of Mr. Davis or they wouldn't have recruited him to Acadia. That brings me to Neurogen's M&A deal with Ligand that was executed while Stephen Davis was captain of the ship and while Baker Brothers was the largest (or one of the largest investors). The deal occurred in 2009.
I don't reference the deal for size because Neurogen never reached its true potential. I reference this deal because of its style and because of the players involved (Stephen Davis and Baker Bros). The deal called for a certain upfront and then a lot of contingency value rights once various milestones or events were triggered. Neurogen also retained the rights to sell some other assets before the deal closed. That part likely won't pertain to the Acadia situation if M&A does indeed take place here.
With Acadia, I think you're talking about a very nice upfront amount with a lot of back loaded items as well. For instance, approval for the PDP indication would trigger x payment, positive phase 2 results in ADP would trigger X payment, positive schizophrenia results would trigger X payment, etc. The problem with this style is that it doesn't really have a way of honoring longer term events such as the ADP indication getting formal approval or schizophrenia getting formal approval. Because of this, a partner is going to be required to either pay a great deal upfront to take out Acadia so that all shareholders are OK letting go today or there will have to be contingency rights so good ADP results reward shareholders. All my opinion.
I think that depends on the form the offer takes. In other words, if an actual term sheet is presented (representing a formal offer) it will go to the BOD and then I believe there is a legal requirement. However, I'm not sure an informal offer or "discussion purposes" offer is viewed the same way. I think there is some latitude here. Most of these talks sit in the "discussion" stage for a long time before anything formal is put on paper.
I don't think it's possible due to all the other variable. For instance, how many of those breakthrough therapies had a second Phase 3 waved off because the data was so compelling that the FDA said one Phase 3 is enough? I know this hasn't happened in CNS before so this case is almost without precedent.
In addition, I'm betting that a fair number of the breakthrough designations were for orphan indications.
You're not going to find many potential mega-blockbusters with a second Phase 3 waved off that were given this status. Acadia is a rare bird and she will fly high. IMHO!
Haha, well maybe I have too much time also! I actually sat here and calculated your shares and profit. You made that into a fun math problem so I had no choice! So you have 28,000 shares and you made $73,641.28 today. Well done!!
Thanks. Yes, I've been around and am always watching Acadia closely. We're starting to see things line up now. I'm looking forward to the actual NDA filing and then a Priority Review designation. It's not guaranteed but I believe that it's highly likely and the Breakthrough Therapy Designation today increases my confidence. A clock is now ticking and we haven't really had that with Acadia before. In the next four months the NDA will be filed and we may see additional clinical trial launches in areas such as schizophrenia and perhaps something in the sleep/wake area for Parkinson's patients since Pimavanserin clearly has a very positive impact here. Early 2015 will likely bring news regarding Priority Review which should set the stage for a lot of excitement going into the approval decision which I believe will be before the end of summer 2015. I don't have a price target, but it's certainly far north of where we are today. If we become a so-called bolt on the terms should be excellent with contingency rights should ADP, schizophrenia and other indications pan out. Exciting times for sure.
In other words, priority review is highly likely. I remember when risperidone was approved in 1994....20 years later a newer generation antipsychotic is finally ready to face the FDA. We all know how much revenue the atypicals generated as a class...it's around $14 billion for those who don't know. There is only one Nuplazid...no other class members to share this market with. Yes, we will only be approved for PDP at first, but Nuplazid will of course be looked at for ADP, schizophrenia and bipolar. Off-label and expanded label in CNS is almost a given because so few new drugs are approved. I have been waiting a long time for the next generation to appear. As an investor, Acadia is dreamy.