Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
I think coverage is a possibility given the additional questions by the moderator/analyst.
To answer my own question. AI buddy says.
EMEA (European Medicines Agency) MAA (Marketing Authorization Application) filings can fail to get approved for various reasons:
1. **Insufficient Evidence**: If the submitted data doesn't adequately demonstrate the safety, efficacy, and quality of the drug, the application may be rejected.
2. **Safety Concerns**: If there are significant safety issues identified during the review process, such as unexpected side effects or risks outweighing benefits, the application may be denied.
3. **Efficacy Concerns**: If the drug's effectiveness is not proven to a satisfactory degree, the application may be rejected.
4. **Quality Issues**: If there are deficiencies in manufacturing processes or product quality, it could lead to rejection.
5. **Regulatory Compliance**: Failure to meet regulatory requirements, including documentation and labeling standards, can result in rejection.
6. **Incomplete Data**: Missing or incomplete data required for evaluation can lead to delays or rejection.
7. **Ethical or Legal Concerns**: Issues related to ethics, patient consent, or legal compliance can also result in rejection.
8. **Insufficient Benefit-Risk Profile**: If the benefits of the drug do not outweigh its risks, the application may be rejected.
Each case is unique, and the reasons for rejection can vary depending on the specific circumstances surrounding the drug and its application.
These percentages are not common knowledge to the average investor and institutional money knows they have time to get in. Getting the actual MAA filed will make that 85-95% more of a reality. That's when the smart money comes in.
FDA NDA percentages courtesy of our AI friend Chat GPT
The percentage of FDA NDA (New Drug Application) filings resulting in an approved drug varies from year to year, but historically it has been around 70-75%. However, this figure can fluctuate due to various factors such as the complexity of the drug, the quality of the data submitted, and evolving regulatory standards.
All this talk about percentages has me feeling pretty optimistic about our chances.
It’s pretty obvious that the high percentage of positive opinions due to the thorough examination of the data, trials, processes, etc. by CHMP allows them to weed out the “losers”. These crucial prior steps save them and the drug companies’ time.
Getting through this part seems to be the toughest hurdle that we have evidently passed.
I’m wondering about the reasons for the 5-15 percent failures. Any ideas/guesses as to why this relatively small group didn’t make it?
A closer look at data revealed issues, couldn’t handle the magnitude of the submission, misstep discovered in trial design? Any others?
We have a shot here and it’s a good one. The data is the data is the data and ours seems pretty good. Successful trial with never before seen biomarkers.
The slowing of brain shrinkage is huge and that alone should give the regulators an “oh dam!” moment. Couple that with our safety and the Alz landscape, we are looking pretty good.
I don’t think Missling will F this up. This is in his control and the EMA will not penalize for minor mistakes in “paperwork”. Not sure what type of communication eligible MAA filers are entitled to but I’m sure it’s not nothing.
Will it be timely? I have my doubts on that.
I hope Missling wasn’t penny wise and pound foolish when hiring a medical writing team to complete the MAA.
Some things you need to pay for to be done right and fast.
Any thoughts as to whether or not our PDD data can be used as supplemental support in our MAA?
It’s odd that we didn’t pursue a P3 for PDD sooner as we had pretty robust data from our P2. And like others have pointed out, we never received guidance on that end on how to proceed. I mean it wasn’t made public. However I’m sure guidance was given.
So that program gets halted and we gleaned as much info as possible from it including the genetic mapping. All good while we wait for our Alz data. I guess now we plan a “real” PDD P3 with all that we learned.
Hope to see some “real precision medicine” plans in our design as we have spent so much time analyzing. Or do we just get a standard trial?
No point in getting all this data if it’s not put to good use. Yeah, we can tell you that possibly you may get a better effect based on your profile but does that really make a difference? Everyone is going to take the drug anyway because there’s nothing else out there.
It seems like 2-73 works pretty broadly so parsing out the differences in sub groups may not make much of a difference anyway in our success/failure/approval decision.
So we learn and learn and waste more time to end up with the same result and same customer base.
Maybe I just can’t see the forest.
Rett topline was released on Jan 2nd. Assuming they requested an FDA meeting around that date and I can’t see any reason to wait for guidance, then the earliest date for a meeting would be mid March.
Hope to hear some guidance soon.
We know the MAA application needs to include details about manufacturing, does anyone know if it needs to include a distribution plan as well?
It seems that we’ve had the manufacturing nailed down for years as Missling has stated in the past at one point that we had enough 2-73 to supply all of NYC.
I’m just trying to gauge if it would be in our best interest to partner before the MAA application is filed.
I of course think it is for other reasons in addition to this one.
Good post. Let’s see what new data comes out of this conference.
Missling may have been holding back to release some at this conference and then the rest in the peer review.
Then announce our new European BP partner for the extra political influence.
Hope he’s playing the game. Up til now he hasn’t played nice. Now is probably the time to change that.
Mayo partially answered this in his article in regard to Alz.
Anavex is likely pursuing an industry-defining genomic study for their completed Alzheimer's 2b/3 trial. Spirit of the Coast Analytics expects this robust analysis to correlate genomics, disease pathology, dosing, and efficacy to fully elucidate 'responders' (classified as patients with absolute improvement/reversal) and varying level of moderate-to-non responder. We believe Anavex is well positioned to accomplish this task with the assistance of Dr. Marwan Sabbagh, Chairman of the Scientific Advisory Board, whom is a frontrunner in disease classification and biomarker analysis.
Can anyone here shed some light on the value of our “whole genome and exome analysis” in our Rett and Alz trial and what impact it could have
-with regulators when deciding our fate? I’m guessing that that data will be revealing and could benefit/sway.
-in designing new trials?
We spent a lot of time and money doing this. The payoff is unclear.
Sadly your characterization of Missling has been correct so far. All will be forgiven if he can right the ship.
Hopefully he can go from zero to hero.
Need that partner and their resources! They need to get the filings correct with speed and an experienced team.
OLE data is not required for an FDA decision or guidance. Why open yourself up for a possible unknown instead of going in with the strong hand you’ve got.
Unless they have spoken to the FDA already and it was suggested that they wait for the OLE data. If that’s the case, Missling should have communicated that we are doing what they advised us to do. He made it seem like we had very little to no dialogue so far.
The EMA seems to be fine without it or with only a partial peak at the OLE. A lot of time has been wasted already by not approaching them earlier. Missling stated that KOLs said the OLE could be confirmatory. Hearing that from the decision makers would have been better.
What if the OLE turns out less than stellar? Then Misslings decision to wait, if that’s what he’s doing, can possibly be detrimental to our filing instead of supporting it.
Please God. Give us some EMA and FDA clarity before the summer!
All the data crunching is done. There’s nothing left to do but have meetings and file.
Any further analysis of the Alz data will bear little fruit and is probably a waste of time and money.
I’m still hoping for that partnership announcement. Otherwise it seems 2-73 may be stalled for a while. A lot of time and money dedicated to filing, analysis of a Rett data, and 3-71 trial.
They can’t be spread much thinner. We have all the data that is needed to design and move forward with multiple 2-73 trials. With confidence. We’ve learned from our mistakes hopefully and will likely emerge stronger but desperately need that muscle.
Years go by quick, patent expirations go by quick and competitors move quickly. Now is the right time for expertise, money and infrastructure. For Alz and all other trials.
Missling is no dummy. He has his pulse on the cns world and is aware of who and what’s moving forward. Try to salvage Rett without letting the voucher cloud his judgement and move on full speed.
Missling is not as dumb as we think he is or some of us think he is. Although at times we think he acts unilaterally, he doesn’t. He has a great team in place and they do provide oversight. Especially after Missling gave up the chairmanship.
There is a plan. And some have speculated the no dilution of late and the move to 3-71 could allow a partner to takeover the bigger 2-73 trials in Pk, etc. That could make sense.
AA for Alz with the fda has a good shot, imo considering the failure of the Man’s to gain traction. We offer a common sense alternative which cannot be ignored. The EMA filing could put some pressure on the FDA but not much. However, the EU has nothing and needs something considering the trajectory of Alz patients and the burden on their health system.
With our safety profile, there’s little to lose.
Rett is not dead but waiting for the voucher is clearly not a good decision right now. With the rett “failure” or better stated, unclear success, there’s no point in not pressing forward with Alz. Something they should have done before. Dollar foolish.
The peer review will clear up a lot of things and give a better picture of where we stand. Why Missling hasn’t released more data, super responder, ADL numbers, etc is suspect but I imagine there is good reason for is and it wasn’t made by himself.
Kun is here and he clearly knows what the fda expects. Missling said they are proceeding with dialogue which to me means it has been in the works. After the biomarker data and the clear labeled success with our two endpoints, it is pass time to approach them. I’m guessing after that, Kun said we’re ready.
Missling gamble on small trials and big data was a chance he took and it may pay off. With the genome data that no one else has ever done, it can give the FDA some confidence in moving forward with an AA.
The Partex partnership will prove useful in adding an additional layer to our precision medicine approach. The groundwork being laid will hopefully encourage the EMA to move.
Regardless of all this, Anavex needs a partner, sooner rather than later. Hopefully George’s predictions come true. M and A is heating up and we are a prime candidate. Maybe Missling’s wheelhouse will be fruitful.
Although the “slam dunk” Rett didn’t work out like we thought, it is far from dead. The fragile x and other rare disease trials will and should move forward with lessons learned. I just hope a BP with experience and deep pockets can help us run multiple trials simultaneously.
The future is still bright and we have many options. We just need some muscle behind us. It’s coming.
Yes. That what I meant to say.
FDA has nothing to lose by giving us AA.
Cost vs benefit is pretty good.
Anyone care to give a percentage?
I’ll go with 85%
I believe we need a partner to overcome the BP stranglehold
I pray Misslings commercial preparation talk is about negotiations with a partner. What is there to prepare that a partner, which we know he has to get, isn’t going to double check, redo or be miffed that it was done incorrectly?
Let the experienced professionals do it right.
Let’s get some adults up in here.
The problem is no one that we know knows the truth. No one here can really assess how well we did in our Alz trial.
The peer review will give everyone a clearer picture. Sadly we just wait as usual.
Sad that Misslings word is not good enough.
The conversation with the fda must have been planned a while ago and I’m assuming there’s an on going dialogue. They probably initiated after the biomarker data and the “met endpoints” pr reveal.
Missling never wants to say what has been done. Maybe he feels like it’s a jinx if it’s mentioned. We never knew they were talking to the ema until the news came out. I hope this is the same case.
The peer review coupled with positive FDA feedback will propel us.
Hope continues to be the word of the day, month, year.
There will be fomo if the FDA tells us to file.
They will most likely wait for peer review before having a meeting.
He said it's perfect timing, which it is. Then again we've had many instances of perfect timing in the past.
He keeps talking about commercial readiness. I’m assuming that’s for Alz and may need to be included with MAA package. Anyone?
Also, wondering if he’s waiting to speak to fda because he’s worried their input/decision on how to proceed will influence EMA decision.
With all that said about Dr. Jin and our data, and how we are superior to the MABs in virtually every aspect, wouldn’t make sense to approach the FDA?
It makes so much sense that he isn’t doing it and not telling us if he is doing it or when or if they even met.
If the potential voucher is his excuse at this point then it makes it even worse.
I still believe in the science and the company even though they haven’t told or shown us the full truth. Maybe that’s my problem.
The street definitely doesn’t believe it either. It’s hard to change someone’s personality or way of communicating. If Missling was more shareholder friendly, would our SP be different? Probably. Would it change our prospects of approval? Probably not.
We are all here to make some money. We are mad at Missling because we aren’t making any.
I’ve been here as long as anyone and it’s sad to see the morale at what seems like an all time low.
If Missling
-gives zero clarity on Rett with FDA
-shifts focus to 3-71
-Doesn’t mention any direction with Alz in regard to FDA
-give more of the same non transparent spiel. We’re working on it, we’ll give it when it’s ready, we’re doing more analysis, etc. They may be true statements but it doesn’t mean they suck any less.
I will sadly reduce my position here even more than I did after the Rett results.
Yep, and all will say it’s the darkest before the dawn because it’s all anyone can say at this point.
I’ll prob keep a smaller position and just set it and forget it.
Hard not to jump on the bash Missling bandwagon.
We need a partner and waiting until after we get Alz approval like he originally planned seems like a dumb idea.
Missling makes it damn hard to have faith in the company.
Sad
He should have released the “super responder” data if there indeed were any which I believe there were.
No reason to hold that.
If you have a successful Alz trial, you make your rounds at investor conferences. You pay the money for the right people to file with regulators world wide asap.
Is he just too cheap?
Is he not confident in the results?
Why drip out this data and cause self inflicted wounds unnecessarily?
No one believes him. He’s surely not acting like we’ve had a raging success.
We are ripe for a deal.
I wonder if Missling is still holding out for the voucher hoping Rett eventually gets approved.
Is this why we haven’t had any Alz movement with the FDA?
Where is the FDA guidance on Alz?
No mention at all. Messed up
Are they going to surprise us or were they surprised at the FDA’s decision?
Let’s see if we hear them mentioned at all during the next CC.
Why hasn’t Missling released the FDA’s guidance to our Alz program? Surely they have met several times, especially after the brain shrinkage and amyloid data was released.
They started with EMA because they take the longest. That’s dumb. Why not start with the shortest? EMA seems to have a higher bar since they denied the others, so another reason not to start there.
If they are waiting for OLE to complete first, then say it. And say it clearly.
It’s been a long time. Too long not to have any guidance.
It’s a shame that we need a peer review in order for people to believe us.
It seems that some think that the peer review is going to be some aha moment where everything changes.
-Institutional investors will pile in.
-Regulators will have renewed confidence in blessing our approval.
-Partners will sign deals.
-Sigma one research will accelerate more than it is already
-All our new trials will be able to move forward
Or
It will be just a ho hum that leads to more questions.
Let’s see. It should make for some good conversation here at least.
Need to hear some FDA guidance. Please!
It seems like there’s a lot riding on this peer review. Until then, the trial results and Missling’s words are fake news.
Validation of our trial and results will help in partnership negotiations and regulatory filings. SP will surely get a boost from it as new investors can be assured the results are what we say they are.
Be nice to see the “super responder” results and what actually led to the Odds Ratio. It will answer a lot of questions.
The paper may be the final piece before an agreement is made with a partner.
Why release this pre-market on a Tuesday??
Messed up!
So for the entirety of 2023, the 40 plus employees of Anavex focused on data analysis, regulatory and partnership meetings, preparation for filings, and preparation for new trials hopefully.
They had one corporate presentation and very few investor presentations.
No visible movement on any other pipeline initiatives.
Does anyone know how long it took Acadia to file an NDA after their trial ended?
Wonder if Missling could surprise us with an already filed an NDA for Rett.
All this time, in addition to EMA talks, FDA talks had to have been going on as well. They would not wait this long to get guidance from them especially after our biomarker data was revealed.
Also after all this time the previous mentioned Rett partnership discussions have matured. He even mentioned term sheets last time which was premature.
All this 2023 leg work should allow for a strong start in 2024.
Happy New Year to all!!
Rett tomorrow? They had control over the timing of today’s PR.
Quite possible that they wanted it out before the Excellence release.
Don’t want to eat leftover frozen spam from Thanksgiving on Christmas.
Last week for shiny bicycles before Christmas!