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Kind of felt the opposite. The analyst seemed to be focused on asking the right questions and Missling was focused on clearly explaining our science and path forward.
Missling has been talking more and more about commercialization, pricing, partnerships, etc. He has also been using more buzz words that exude confidence. IMO
Words like plattform, Rett franchise, correct target, loaded with biomarkers.
He shows he understands our drugs in and out like he always has, and can clearly compare and contrast our position in the market compared to other companies and their respective trials.
He continues to confidently reiterate timelines and his "let the data tell us what to do" approach.
The data told him to go for more indications despite not having revenue. Pretty bold!
Don't really get the SP movement as of late but I'm thinking that will all chance soooon. There goes that darn word again.
Also said we are beyond dose finding work and that too much can actually have an opposite effect. The doses they are using show dose dependent improvements.
Great conference. The analyst seemed well prepared.
I liked how Missling came out emphasizing that we are a plattform
Also liked how he said the difference in effect between the wild type and variant let them know that they have the right target. Even though there is only 1 amino acid difference between the 2, it still showed a difference from the full functioning wild type.
Great to hear that he felt our trial could be registrational because we are loaded with biomarkers and Aduhelm set a precedent.
Hopefully we get some coverage from them.
If the Rett data is ready, I would think a release tomorrow before the conference would make a perfect set up.
I keep revisiting that wording change of the fragile x trial from potentially pivotal to pivotal. I know another poster mentioned it as well.
He def didn’t do it based on an assumption. And the FDA would not give this a nod without seeing the 2nd Rett trial results.
So... any other reasons. It was clearly deliberate.
He’s going to be and so far he’s sold very few shares considering how many he has.
I just thought about how he always likes to mention that the effect could be larger with this Rett trial since we are dose dependent.
He said it this time with even more German enthusiasm. He Knows!
Especially now, since he most likely seen the results already. He chooses his words carefully and was like. Uh hum. Don’t forget we can see a larger effect!
Confirmed dose dependent improvements is what we need.
Yes, I’m making an inference that it is the OLE data they are talking about.
Unidentified Analyst
All right. Thank you. And my last question regards to the Parkinson's disease dementia program, can you provide any updates on that program, have you met with the agency to discuss it and or do you plan to anytime in the near future?
Christopher Missling
Yeah, we plan, Right. Thank you very much. Yes. We plan to do that. We actually are in the process of now discussing the data with the foundations and we are expecting to get a valuable feedback for input on the design of pivotal studies for Parkinson and also pivotal study for Parkinson dementia. And with that, in our package, if you like, we then feel more robustly educated and fully informed to go to do the discussion with the agency about a proper pivotal study in the respective communications.
There’s definitely more to the PDD story. Wonder why they are discussing the data with the foundations first.
If the results were so so, no need to bring the data to them. IMO. Just submit it to the FDA and collaborate with them to design a phase 3 or 2B.
However, if the OLE results were “robust”, then maybe discuss with the foundations to get backing/support on something better. They may have sway with the FDA, if not, they definitely have away with the public which may be just as important.
Unprecedented results should receive special treatment, especially if there is follow through or greater improvement in our OLE.
Why give the data to the foundations first? Why not give it to both at the same time since there will inevitably be back and forth in the trial design regardless?
Just thought that was a strange step. Let’s give our data to someone else first before we give it to who really matters and waste some time doing it.
Anyone think of a good reason to do that first?
It's insane to go after all these indications. Especially for a tiny company like ours. I can see how people might view this as snake oil considering we don’t have an approved drug yet. Some may also think Missling is just initiating trials in anything that gives a signal hoping something sticks and hoping it reflects in our SP. We are starting so many without even finishing one.
Rett is the gateway. If it fails, then everything else will be viewed as less likely to be successful. And of course the opposite is also true. Getting solid dose dependent Rett data will help move us over to the launch pad. It may be the spark that gets us our turn to have the fomo momo moment that others have enjoyed already.
The breadth of our amazing drug platform will no longer be viewed as a pipe dream for many. Each trial initiation PR will have a more meaningful affect.
If Rett, the low hanging fruit, fails to prove our drug has merit, then we will go down just like all the others regardless of how many indications we have in our pipeline chart.
Exciting times ahead!
When does data analysis become a waste of time?
Especially when after all this analysis results in choosing the same common gold standard primary and secondary endpoints anyway.
It seems time would be better spent actually starting the trials.
Thanks.
With the unprecedented PDD results, why haven’t they applied for BTD?
If all they want or are aiming for is a well designed pivotal trial, you’d think BTD would have been applied for already.
Could they be aiming for more? Possible AA in light of all the other failures as of late.
Seems like overkill to spend all this time analyzing in order to get a robust package to the FDA just to seek guidance on a trial.
He said he was discussing the PDD results with the foundation. I’m assuming he means MJF.
If so, it shows a very close working relationship. Just hope it doesn’t take another year to analyze the OLE data in order to provide a compete package to the FDA.
With 2 FDA guys working with us, I’m assuming Missling has inquired about this.
It’s a pretty straightforward question. Do we need 2 phase 3 trials for approval?
Seems like something that even the most inexperienced CEO would ask.
Be pretty bad to get a delay tomorrow. Even though a delay is not necessarily bad news, it will cause the SP to drift even lower in the absence of expected near term catalysts.
Adding insult to injury. I guess dates matter. It seems more than words for now.
Why couldn’t he file any other day?
This week, Monday, Tuesday... pick a day. Any day.
This was an unnecessary amateur move that caused needless speculation and FUD.
He should know better.
Maybe. What kind of sick and twisted man wants to give people bad news before Thanksgiving?
He doesn’t strike me as that kind of guy.
Maybe he wants us to get an extra fat turkey and be thankful for Anavex.
Wonder why initiation of p2/3 PDD trial isn’t on there anymore.
“Let's not under-estimate the rest-of-world market and that some ROW approvals may (likely) come ahead of the FDA. “
Yes, I hope so. Missling has been bypassing the FDA for the most part. I don’t see why he wouldn’t apply elsewhere. Unless he was advised against it. Unless it would leave a bad taste in the mouth of the fda.
Missling knows the fda is the gold standard and I’m sure is aiming for their nod. If the relationship is productive, he may not go for an overseas approval, etc. Even though we all want him to. It actually may force their hand. I’m sure it has been discussed in their boardrooms.
If the TGA hints at some accelerated path especially for PDD, I’m sure he would jump at the opportunity. This news drought is indeed frustrating, however, I believe news is in the horizon.
I’m expecting big things this year. I’m also expecting him to check off many of the boxes he has slated as catalysts for this year.
I use to doubt him and his timelines. However, after those 3 blockbuster PRs over the Summer, I feel like something changed in him. I think he has his pulse on shareholder enthusiasm or criticism. Any good CEO should to some degree.
I also believe a reason for the change is that more eyes are in us. It’s a different type of investor. We can see him honing in on his speaking and message delivery skills. We also see the much needed change in our website. Obviously appearance matters.
An appearance of a CEO that doesn’t know or understand his own timelines is definitely not a good look. What good is your word then? In deals, negotiations, shareholder messaging, presentations, etc.
If he doesn’t meet a decent amount of these self imposed timelines, he would be like a kid that’s flapping his gums all the time and receiving a “yeah ok buddy” from those listening.
I pray he’s not flapping.
So maybe we didn’t apply for BTD since we should have heard something already.
Maybe Missling said, we don’t need no stinkin BTD cause we are going for approval after topline Rett.
Maybe he said the same for PDD. That we will wait for our OLE to finish and apply for AA instead.
A lot of catalysts waiting in the wings for 2021. How many will we actually get?
Thanks for posting this. So great that Missling is tapping into the expertise of FRAXA. They seem eager to get started.
Developing the trial around the patients’ needs is so important especially during these Covid times. It makes entering a trial so much easier. I like they way they said parents are eager to get into trials and they have hundreds waiting.
Tailored trials to these communities. Nice job Missling. We can help so many! A budding rare disease company. Nice!
Got the game plan and expertise from the Rett trial. Now just rinse and repeat with whatever 3rd party rare disease organization that wants to give it a go.
Who’s next. Step right up. With your funding, expertise, patient pool, eagerness, etc. We have a very safe drug using your own body’s defense mechanism. Step right up. 2-73 here!! Step right up!!
Yes, that is curious. I haven’t listened to any others to confirm, but this format seemed to allow Missling to dive deeper into our MOA.
It’s almost as if Cantor said, “We want people to not only know about the company but to understand the science behind it”.
I think it was beneficial to anyone who listened. Especially when your using a diff approach. New takes time to absorb. New needs explaining.
It’s hard for investors to plunk money down on new. I mean “smart” money. Missling is doing a good job on getting the message out there.
Why wouldn’t he apply for adult Rett approval right after Avatar?
Why would the Rett launch be so expensive?
Hey frrol
The horizon for filing an S3 is relatively short
Can you explain what this means? Does this mean they only have a certain amount of time to sell these shares?
I didn’t see a timeline requirement when doing my DD on this.
Thanks
New coverage needs to be conservative to give room to move. No one wants to see crazy unrealistic SAVA type numbers. That would be foolish and can tarnish the analysts rep.
The BTIG coverage hopefully will entice other firms to initiate. I’m not sure how big or reputable BTIG is. Anyone?
What is Missling seeing? What does he know? Big statements and results being implied.
Pre-revenue biotech with no guarantee of approval, yet moving ahead with more and more phase 3 trials.
No way is this all based on pre-clinical data. Does any company decide to run a bunch of expensive P3 trials on one drug based on pre-clinical?
People that don’t know Missling and have not been following our science must think he’s nuts. This guy wants to run what? With no revenue?
This is a guy who’s looking at all the data and science in front of him and saying “Oh Damn! Let’s hit the gas!”
https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/breakthrough-therapy
Seems silly not to have as it would be a big mistake.
https://www.raps.org/news-and-articles/news-articles/2019/5/almost-half-of-all-new-drug-approvals-in-2018-reli
Wishful thinking!!
If they applied for BTD, they should have heard already.
Let’s go!
Yep. And it’s all being driven by data, data and more data.
And that data is telling him to go, go, go!!
Let’s go!!!
“which could lead to some unblinding of the study or inadvertedly to learning about the effect of the drug and making a calculation back on the envelope as more drug is given, how much the effect would be based on the existing data of the 5 milligram arm [ph]. So that's the reason why we keep this right now blinded,”
Yep. Because parents would be fighting for higher doses because they will see that higher doses equals x amount of improvement. Why else would they do back of the envelope calculation?
Dose dependent improvements! Pretty much implied that it is being seen in the trial.
"All of that makes sense, after examining and understanding how blarcamesine works, by uniquely activating the sigma-1 receptor protein. Only good things happen when that happens. "
Nice!!
Agreed. Thanks
The optimist in me says we will get top line readout from Avatar and BTD for Rett very "soon".
I also believe we will get BTD for PDD. There's no reason for Missling to not apply for it.
After we get BTD for PDD, he will prepare the larger more comprehensive data package that he mentioned. Who knows, maybe apply for AA. I guess that depends on how compelling the data is.
No reason to not apply, especially with 2 ex-FDA employees on our team.
With the top-line read-out of Avatar, Missling will mention how he will be submitting an NDA for Rett while also preparing a more detailed presentation of the data, getting a 2fer like he did with PDD.
We will be seen and valued as a soon to be revenue generating company with the pediatric indication and voucher being pretty much a slam dunk. Confirming dose dependent improvements will surely add confidence to all our other trials.
Praying and hoping to have this happen in late September to early October. All other catalysts and trial initiations would be that much more meaningful and will add to our SP appreciation. These would not have as meaningful of an affect on our SP if the above doesn't happen first
I'm calling $50, possibly more by Christmas. If we don't get Avatar or BTD, I will whine like a child who didn't get his new bike under the tree. We will likely be in the mid 20s then.
So undervalued!
What CNS company has a safer drug with more indications?
What company is ahead of us in the Alz game with our late stage potentially pivotal trial fully enrolled? (Crtx maybe - not much competition IMO)
What company has shown better PDD results than we just did with our OLE finishing in October?
Rett patients have never seen improvement. Hope is here!
Autism is here. So many late stage trials ready to go.
So much third party validation and support.
So undervalued. Share price appreciation inevitable. Once you start doing DD on our company, you want to buy as much as you can because sales like this don’t last very long.
Could they have peeked already?
How closely are the working with the TGA?
Why did that guy jump ship from Hammond?
Why sprinkle in some PDD patients in Australia?
Would TGA approve a dosing scheme that was not patented?
How much OLE, SAS data do they have?
Random thoughts. What was needed for the TGA to grow balls?
FDA not the only game. Missling is not one to pigeon hole us.
Yep. Unfortunate news that needs to be investigated regardless.
Black eye for the company any way you look at it. Fake or not.
Good time for Missling to drop some credible news!
That would be great. He could get a 2fer like he did with the PDD results. Present top line now and then a more detailed analysis when it’s ready.
The Rett results along with a positive statement in regards to approval would def be SP moving.
With a BTD or two to boot.
Good post. They are taking the time to do it right. Does it really matter if it takes another 6 months for big things to develop? Nope! This is the set up for years and years of growth, possibly decades.
They have so much on their plate right now, I can just imagine what it's like to be in one of their many high level meetings going on.
Up on the big board
-Partnerships - list of all potential partners, pros and cons of what each and what they offer
-List of manufacturers and distributers and analysis of each
-Timelines for each trial about to be initiated and details about sites, dosing, progress, etc, etc.
-FDA, TGA and AEMPS, etc. what we have applied for, timelines, expectations, etc.
-Marketing strategies by region, penetration, pricing, etc..
-Expenses, what to do with all that money we accumulated and when we need more
-How to spread our message more efficiently - peer reviews, conferences, speaking engagements, etc.
Just some of the few things off the top of my head on what they are thinking about and working on daily.
A company planning for big growth despite what we see in the daily price moves.
Once we get approval for Rett, every other trial we initiate will become more meaningful. Chances for success will become more realistic. Each will become a catalyst for an SP bump. Instead of just being a company with no approved drugs, running a bunch of trials, we will be a revenue generating company with a proven effective drug expanding on our success.
Good times ahead.
True. And raising all that cash so quickly was also uncharacteristic and unexpected.
Wonder what it’s for. I’m sure he has a few ideas already in motion.
Hopefully another unexpected surprise!