This is going to be a 500m plus market cap. Just be patient and buy. I see similar signals in mstx as cpxx. I was a long time buyer and bought in the low ones for cpxx and sold around 15. The shorts on cpxx were so stupid it was amusing as some shorts are on this board.
Culley's job was to ensure that there was enough money to enroll and get a readout for phase 3. He's done more than that which is hire former FDA consultants to work on the NDA. If the readout is as good as I think it will be, the financing side won't be a concern.
How do any biotech companies finance for commercialization? Your statement makes you look stupid. If they have a successful phase 3 readout they will not have any trouble financing. There will be many institutional holders that will want in. These statements are common for all biotech companies.
No, I'll keep it simple for you. This is a 67 M market cap company that should be around 100-150 M market cap pre-phase 3 readout. If it's a positive readout you are looking at 300-500 M market cap. I think a 500 M+ may now be reasonable because BLUE's therapy has been disappointing and years away from approval. If you short a 67 M market cap that is undervalued you may gain if it drops to a 30 M market cap (still has a phase 2 and other indications it can pursue). This trade favours going long, not to mention an interim look at data now aligns to their projected/modeled statistical model. This is similar to CPXX's positive induction response rate in advance of readout.
Stop posting, you don't know what you're talking about.
I expect share prices to run to 0.5-0.6 prior to data release and then with the positive topline release jump to 500 M+ market cap. Just patiently buy and enjoy.
Most CEOs will only say "weeks" after 30 day observation for QAQC. You can then provide an additional 2-3 weeks for the statistician. This trial has a large number of patients and sites but the primary and secondary endpoints are simple to evaluate. My investment thesis is that we will see something in the next two weeks. Enjoy.
Data lock pr are not common. Judging by my estimates they are already past data lock and the statistician is now reviewing.
The mechanism for P-188 has been demonstrated at a biological level independently by many experts and will translate to a benefit. Do your own literature search and you will see that the pervious phase 3 before should have been more of an exploratory study...Tough luck that the amount of time between two type of treatments could cause a slightly less p value. Also, the age group matters in this disease and lack of patients would contribute to this. I would hazard a guess that had the previous phase 3 was able to enroll the full number of patients they would have been P
CHMA mislead investors. The didn't disclose that when they had their meetings with Roche the FDA asked for a placebo trial. A lawsuit is warranted in that situation. Also acromegaly already had a treatment available. Sickle cell does not have an alternative treatment for VOCs and the endpoint has been up for discussion for a long time. The age of the patient population and the rate at which patients are accepted into the trial gives me confidence that the phase 3 result will be a success. Not to mention an extension is ongoing. If you treat MSTX previous phase 3 trial as being more of a phase 2 where a subpopulation was examined you can reasonably conclude that this Phase 3 for the specific age group will be a success. They also relaxed their endpoint so their statistical model is very lenient. This is EXACTLY similar to CPXX. Enjoy.
Also, because I work in this field - two big signals 1) extension for Epic probably a request from site investigators 2) Hiring of consultants to start the NDA prior to phase 3 results. It is very labor intensive and costly so companies will not do this without good guidance.
Would it be fair to say at a premium for the following reasons? 1) No alternatives for the indication even when compared to gene therapy 2) Orphan exclusivity 3) Repeat treatments are expected several times a year 4) Strong IP 5) Strong support in the scientific community judging by independent publishing. I plan to hold between $3-5 depending on what management plans to do in the EU. This drug has had the misfortune of being used with an impurity so abandoned because of safety concerns, being run on a trial that was under powered and subjective endpoint, and diluted for an additional phase 2. Management got a bargain on this drug. Investing in this company is about timing...and in 2 weeks the price will continue rising.
I've been taking large positions in this company the past couple of weeks. Based on the available data and independent ex-vivo studies via microarrays I'm 100% confident this will be positive. Management did a good job of relaxing their endpoint and increasing the number of patients in trial. I clocked the release of data to be around 70 work days from Feb 22, with a generous amount of time for the statistician to review. Relax and enjoy! Next two weeks should see some positive moves.
Can anyone explain to me why the FDA would grant orphan drug approval when this give 7 year exclusivity to Sarepta? Wouldn't this block other companies with potentially better treatments from entering? I think based on this the FDA will hand out a CRL.
What does going to the sideline mean? They should just admit it and downgrade to an actual price...but I suspect the company is working on a raise with Oppenheimer as we speak.
No it's folly to think the FDA goes against staff and the panel. Those are rare instances...so you are betting on a an less than 5% chance of approval (instances/ all adcoms). It's not looking good.