That won't happen. INCY is right to fear CTIC as their monopoly may be challenged. We will know much more on Saturday morning after CTIC's oral session. Baxalta (CTIC's partner) has the size and bandwidth to give JAKAFI from Incyte a real challenge. Baxter's own words were that they wanted this to become the first line therapy for all myelofibrosis patients regardless of low platelets of anemia. In other words, the standard of care.
The market is big enough for both JAKAFI and Pacritinib. It's just that JAKAFI had a monopoly and those days may be coming to an end. Baxalta has the power to truly fund and market pacritinib so Incyte has a real challenge. CTIC should be trading much higher in my opinion. The covers are about to be pulled back on pacritinib and soon we will see the full data. I'm expecting great things, but of course won't know for sure until we see it.
Good luck to all and please do your own DD.
Thank you. I don't know about the USA portion because they didn't break down the numbers. However, Incyte's monopoly could be challenged. CTI is not being appreciated. That could change soon.
Thanks. I'm really looking forward to the ASCO data. June should be a very interesting month.
If you listen to Baxalta's extensive 4+ hour presentation you will clearly see that pacritinib is a core piece of their strategy going forward. They are not looking to fit pacritinib behind Jakafi. They clearly state (paraphrasing) that pacritinib could be a new first line therapy for all myelofibrosis patients (standard of care) regardless of blood platelet counts or anemia. All I can say is watch out Incyte...this drug is for real and Baxalta is for real. The stock price isn't reflecting any of its potential, but that should change drastically in time.
Finally, the discuss that it may work in a variety of cancers and they will be targeting many different liquid tumors.
They cited a potential peak market for pacritinib and put it at $6+ billion.
Again, I am paraphrasing but this is very close to what was said. Please do your own due diligence and good luck!
Absolutely agree. They have covered every aspect of preparation from basic science to the use of an (approved) baculovirus expression system for manufacturing. In terms of being prepared for market, they are the top gene therapy company. I hope to see them trade at BLUE levels within the next year or so.
Interestingly, if you listened to their investors days conference, they also have a lot going on in CNS. The potential applications for QURE's platform are vast. This is definitely only just the very beginning.
Definitely a good point. That we have orphan drug status in DMD for cardiomyopathy is a big deal.
Okay, thanks. I do remember the insomnia candidate. That's interesting since Acadia's Pimavanserin is said to possibly have sleep promoting properties. This could be an interesting program. Like Acadia's Pimavanserin, I'm sure they'll target Parkinson's disease psychosis and perhaps Alzheimer's.
Very interesting. I actually haven't followed Arena's inverse 2A agonist, but it's interesting that it failed. I wonder how it compares to Pimavanserin. I'll have to keep a closer eye on this one.
I just think you have to look at it as a buying opportunity. The missed earnings are meaningless and the delay in the ITP results doesn't matter. The mentioned pursuing other collaborations to monetize assets in their pipeline. That statement caught my eye for sure...I'm a buyer this morning.
Personally, I would love to see a partner for the ITP program. There is no reason to spin the future of the entire company around it. Rigel is stronger than that. I would partner and then expand the pipeline.
Thanks for sharing. I don't think there is any question about the potential value of BLUE. However, I don't think CLDN in any way diminishes the upside for QURE. I didn't see any safety issues in the CLDN trial and there wasn't an indication that AAV didn't function correctly. I would say the issue was with CLDN's gene target or poor execution at some level. Both lenti and AAV are likely to play a large role in the future of gene therapy IMO.
Very hard to know. QURE pulled back a lot already from recent highs and part of the reason may have been the potential competition from CLDN. The gene therapies are different, but it's obviously much better for QURE if CLDN isn't a competitor. Also, BMS bought just under 5% of the company at $33.84 so that provides some support.
At the same time, gene therapy companies in general could take a hit just because they've gone up so much. However, UniQure seems very fairly valued to me as they were undervalued for a long time.
Ultimately, this isn't bad news for UniQure. It might be bad news for the gene therapy sector but that's not clear. In the long term, it obviously benefits QURE to not have CLDN as a competitor and market leader.
Like you said, it's really hard to predict whether this goes up or down. It just as easily could rally since QURE is now the definite dominant player in the cardio gene therapy space.
I was in INCY and YMI before CTIC all for their JAKs. I made a lot of money on both...bet you weren't in either. Pacritinib seems to be the real deal....I'm long and strong...and you're ignored.
Centeritide definitely works through the natriuretic system. The news to watch in this space is Novartis LCZ696 which also impacts the natriuretic system. LCZ696 is up for FDA approval this August and is expected to be a mega blockbuster. This should fuel interest in other groups in the space. I'm an investor in CAPR for this reason and also in PTN as they have a problem at a similar stage of development and that program is PT3994 which is a synthetic natriuretic peptide antagonist.
Both companies are interesting above and beyond these programs, but I do believe the approval of LCZ696 will fuel interest in cardio companies and especially companies with natriuretic programs.
First Phase 3 is done. Results are being presented at ASCO. Second Phase 3 has a very good chance of generating excellent results considering you don't see signals like these in myelofibrosis without true efficacy. The safety also looks very good. This isn't a typical 50/50 situation by any stretch in my opinion.
You definitely have nothing to worry about in my profit category. I can guarantee you that we're not in the same ballpark.
Apologies to quality and long term posters. I don't typically respond to the loudmouth ignorant ones but couldnt help it. As I said, I may be back. Looking forward to seeing how the execute over the next few months.
I'm concerned about three major things probably in this order:
1. FDA Panel. I had hoped that the priority review designation would negate the need for a panel. However, Uli indicated on a call that they would indeed have to face a panel. I don't have any particular reason to fear a panel, but panels in general are a bit unpredictable. Pima should get approval, but it's hard to know if there will be any concerns regarding things like cardio or additional requirements to get to market. I simply cannot predict what a panel might want to see and that alone makes me a bit uncomfortable.
2. Alzheimer's results. I truly have no idea how these will turn out. The disease pathology is so different even if the symptoms are similar. It's another unknown for me and I don't typically invest around unknowns unless i can handicap enough other variables in my favor.
3. Cash. As others pointed out, past fund raises haven't hurt the stock so this one isn't a major concern.
I'm also just turned off by the filing delays. I accept the reason, but it does cause me to worry more about the FDA panel. They will need to be prepared completely. I assume that they will be ready. However, the core variables I was counted on changed so that the biggest risk was no longer Alzheimer's data. Now it's a panel, but I do believe they should get through it.
A key question of course is the extent to which serotonin 2a antagonism causes cardiac concern and of this is the reason atypicals carry the black box. It's not the dopamine component of atyoicals so I'm wondering how the panel will perceive a highly selective 2a antagonist.
I'm definitely not arguing with you on this point. I think Pima could have tremendous upside. I just want to see how Acadia executes over the next several months. I can always move back in, but not today.
This was my final 2000 shares and I was waiting for them to mature to long term capital gains which happened today. I sold off the bulk of my holdings for about $35 which was my prespecified exit point. I realize that many see this as a low target, but it's a target I'm comfortable with. I might buy again depending on developments.
Thank you very much. I'm a long time Acadia follower so I will definitely post now and then. I might buy again...we'll see.