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Actually, the FDA is facing a backlog with site inspections and other bottlenecks that is causing delays with drug approval. To date, there are 48 drugs that are facing unanticipated delays and while the good news is that the FDA is starting to get back to a regular cadence, it's going to take some time before everything is back to normal. Pandemics can sometimes do that.
I don't think the FDA wants bad press, but you seem to think that the FDA is going to prioritize Rett over any of the number of other rare pediatric diseases that are deserving of the agency's attention (and have equally powerful advocacy groups). There is nothing in my post that I believe creates fear, uncertainty, or doubt. I am hopeful that Rett will be approved. I just believe it will take longer than some here are speculating.
Maybe I'm wrong. Wouldn't be the first time and it won't be the last. I'd love to see Rett approved tomorrow. But I've also been here long enough to know that everything always takes longer than expected.
The fun part is that we'll know soon enough whether I am correct or not. My belief is that we'll see a decision with Rett syndrome sometime between May 2022 and September 2022. Others think it'll happen sometime between September 2021 and December 31st, 2021. Time will tell.
Stay safe, Fireman. I enjoy your posts and perspectives, but in this instance I think you're wrong to state that it's absurd for me to believe it will take another 9-12 months for the FDA to approve Rett for pediatric use so that AVXL can get the priority review voucher.
It might get priority for Rett. It might not. I don't know, I'm just making my best guess.
There are a lot of other drugs that have these various designations. It's nice to have, but breakthrough designation is the crown jewel and we don't have that yet.
We'll see what happens. Obviously if Rett is approved ASAP that would be fantastic. I just think it'll take longer than many here believe it will. Every few years everyone gets all worked up about timelines that are way off. The FDA is backlogged as it is, and a good portion of its resources right now are dedicated to the pandemic. It'll get to Rett when it gets to it. Hopefully sooner. But I think approval is probably closer to 9-15 months away instead of the 1-6 months a lot of people here seem to think will happen.
Agreed. Usually the system works well. The FAA is another example. But every now and then the system doesn't work because the FDA isn't a machine. It's run by humans, who all have opinions and motivations and every now and then, buckle to pressure from bigger fish.
That's how Aducanumab got approved in the first place, right? It certainly wasn't approved on objectivity. It was the one that slipped through the cracks. I'd be willing to bet the farm that if Anavex had been the company to develop Aducanumab, there's zero chance the drug gets approved on that data.
Like it or not, Biogen has power. That's all I'm saying. It seems likely it'll do everything it can to flex that power to keep its new drug on the market for as long as possible. And I'd be willing to bet my other farm that if Dr. Missling created a SWOT board, Biogen would be listed under "Threats." And maybe "Opportunity" because if Aducanumab were even halfway decent, the FDA would be under no pressure to approve another Alzheimer's drug immediately.
Everyone keeps talking about a partnership with a big pharmaceutical.
I'd love to see a partnership with Cassava if their drug works as advertised and can be prescribed alongside A273.
I think the potential for massive profits is significantly higher that way, and Dr. Missling will retain a lot more influence and power over future decision making.
No, Anavex gets the FDA rare disease priority review voucher if/when Rett is approved. It's Anavex's reward for spending the time to research a rare disease and it's supposed to incentivize companies to pursue diseases that may not be profitable to treat.
As time goes on and as these vouchers become worth less and less now that there's directive to speed up the approval process for all drugs, I can see a day in the distant future when the FDA needs to add more value to these vouchers. Perhaps a guaranteed re-submission if the drug isn't approved. Or maybe add priority review for BTD. Otherwise the incentive to research rare and tropical diseases will continue to be diminished and companies like Anavex will determine that it's better to hold them than sell them.
Yup. I had SRPT, too. Duchenne's seems very similar to Rett.
I wish I shared your idealism for the morality and ethical values government is supposed to uphold.
I suppose in a virtuous world such as that, Biogen wouldn't have ever had its drug approved.
I agree that there will not be any more back room deals, no more secret handshake agreements. Biogen will defend its newly held position the old fashioned way -- in public, with lobbyists, many of whom have so much power they literally write the bills that become laws.
I've worked in and with government agencies too. I agree with much of what you're saying. But I disagree wholeheartedly that money doesn't influence political decisions.
Do you know where the FDA gets a large percentage of its budget? It's from the fees they charge for companies to submit their drug applications. Biogen pays the FDA a lot of money every year as do other whale pharmaceuticals. The FDA has to restore its reputation but to be honest I don't think it's in quite the hurry you believe it is. It's reputation is already plenty tarnished with 1/3 of the country hating the FDA because they believed it rushed through a COVID vaccine that is secretly the mark of the beast...
Like I said, I can name a dozen companies working on horrific rare diseases that are in P2/P3 trials. All of these companies want the same thing as Anavex: approval as quickly as possible. And all have passionate advocates with connections to the FDA, just like Rett Foundation.
I'm not saying that Rett isn't deserving of Accelerated Approval. It is. But it's a numbers game and there is a lot of equally deserving competition. Some here are going to be biased toward Rett because of their financial stake in Anavex.
I would be thrilled if your prediction comes true.
I hope you're right. Money is a powerful tool and Biogen has plenty of it. It also spent a lot of money developing this drug and it's going to do all it can to protect its interests.
I once went up against Wilbur Ross in a multi-billion dollar MLP oil restructuring deal. I represented the equity interests, he represented the creditors. Who do you think won that battle? As it turned out, years later the facts would support the financial models my team had put together were correct, but it didn't matter other than as a moral victory. Wilbur isn't a stupid man. He knew damn well if facts and the public's interest were the sole determiners in how the bankruptcy court would make its ruling, he'd have lost. But money is power and he had plenty of both. Just like Biogen...
No, I am dismissing the influence that Rett org has with the FDA when it comes to advancing Rett treatments ahead of any other ones, each of which have their own foundations dedicated to helping their patients and ability to advocate to the FDA decision-makers.
Putting aside your financial interest in Anavex, what makes Rett syndrome more important than, say, the diseases that other rare disease biotechs are focusing on? Allievex, NeuroVive, there are dozens of companies just like Anavex.
The Rett Foundation can advocate for accelerated approval all it wants. I don't think it'll receive it because Rett is competing with a bunch of other really horrific diseases and there is limited manpower at the FDA to accelerate the timelines for all of them.
The case for accelerated Rett approval is simple: What else is there to help those who are suffering?
The safety issue is moot because there are years of trial data, and if early results are promising what is there to lose with approval? A P4 may be required but that too seems unlikely.
That said, I do not expect the FDA to grant Rett accelerated approval. I don't think there is a large enough population in the world who have Rett to sway the FDA to grant accelerated approval, especially considering all the other drugs for lots of other diseases without a treatment that may be more prevalent.
The drug itself may be far superior. But I'm not naive enough to believe that the FDA doesn't occasionally buckle to political and outside pressures, otherwise Biogen's drug wouldn't have even made it to the AdComm stage of the review process let alone being granted approval.
The FDA will eventually have to approve Aducanumab's replacement but there's no guarantee that Anavex will be first in line. Breakthrough designation would be nice but its far from guaranteed now that there is a recently approved drug on the market -- one that Biogen is willing to spend hundreds of millions to keep on the market for as long as possible.
Assuming Anavex gets the priority review voucher, it then has a 90% chance of having a decision within 6 months (the voucher doesn't promise immediate review, only that the FDA will try its hardest to do so). To me, that is far more valuable than $100 million or whatever they are going for these days. If AVXL does get breakthrough designation, it can always use the voucher to advance A371.
If you were Dr. Missling and just received a rare pediatric disease priority voucher, how would you use it?
Agreed, but BTD is going to be significantly more difficult now that Biogen's drug was approved recently. We all know it's not the long-term answer but Biogen put a lot of money and time developing it, and is going to use all its lobbying influence to ensure that it remains the sole Alzheimer's alternative to Aricept for as long as possible.
BTD is more likely for Rett Syndrome, in my opinion. And while that will help Anavex generate revenue quickly, the goal for Anavex should be to do everything in its power to mitigate the interference it's going to face from Biogen and the others who do not want to see A273 in front of the FDA's advisory committee. The voucher is just another tool to help advance A273 as quickly as possible, whether in conjunction with BTD or not.
If AVXL had 30mm shares outstanding, I'd say sell the voucher and offer a one-time special dividend to shareholders. But with 80mm shares out there, at $100mm it's probably better to hold it and use it than to sell it, especially given the 5 year runway we currently have.
Wolf,
I don't think any of the competitors are currently pursuing rare pediatric diseases. I do not believe this was originally Dr. Missling's intent, either, as I doubt any of us invested in Anavex because of its involvement with Rett syndrome. However, Dr. Missling saw the writing on the wall with BIIB, SAVA, ANVS, etc and realized that competition was going to be robust in the Alzheimer's space.
Pivoting to Rett syndrome was a short-term setback -- it required time and money being taken away from the Alzheimer's trials -- but in the long-term the priority review voucher was a worthwhile goal. Not because of its monetary value but rather because it offers the one thing money cannot buy -- the ability to be first in line in the increasingly likely scenario that multiple companies submit to the FDA around the same time.
If AVXL is proven to be effective, and if it gets to market first because of the priority voucher, it'll make it increasingly difficult for the competition to retain trial participants. If you were taking SAVA's experimental drug and a proven drug became available, why would you continue in that trial when dealing with a degenerative disease?
$100mm to a small startup is a lot of money. But for Anavex, that's maybe 5 million shares in an offering. Better to use the voucher than to sell it for what amounts to peanuts when looking at the bigger picture strategy.
Well, back in January of this year Rhythm Pharma sold theirs for $100mm.
And back in 2018, Spark Therapeutics sold theirs to Jazz Pharma for $110mm.
Also in 2018, Ultragenyx sold theirs for $80mm.
I could keep on going but I think you get the idea. The value of these vouchers today isn't worth nearly what they went for half-a-decade ago, namely because the FDA has tried to streamline the approval process per President Trump's request.
The rare priority review voucher is worth more for Anavex to use to expedite its Alzheimer's pipeline, particularly if there is competition from other drug companies like SAVA...
https://pharmaphorum.com/news/spark-sells-priority-review-voucher-to-jazz/
https://www.biopharmadive.com/news/ultragenyx-prv-sale-value-regulation-fast-pass/529221/
Rare disease vouchers are worth maybe $80-$100 million today. Your article is 6 years old.
Better use would be to use the voucher to expedite Alzheimer’s review. Even if Anavex sold the voucher for $350 million, that’s only like $4.50/share value because there are so many outstanding shares now.
Exactly. I knew this stock was doomed in the short term when all the AVXL millionaires club talk started.
Exactly. The stock gods have a fun way of punishing those who brag about their gains. Nothing ever goes straight up.
Pardon my ignorance, but how does AVXL's method of action differ from SAVA and ANVS?
Is there any concern that ANVS's failure could be a harbinger for AVXL or do the MOAs differ so much that it's apples to oranges?
Thanks.
I'm sorry to hear
You're nearly at your end
But please take solace in knowing those certificates
Will benefit your family and iHub friends
Fair enough. I don't know that your timeline is accurate but let's suppose that it is.
I guess for traders, waiting another 4 years would really be disappointing, particularly in terms of lost opportunity cost. For long-term investors who subscribe to Buffett's advice that the ideal holding period is forever, 2022 vs 2025 revenue is relatively insignificant.
I don't know what is going to happen to AVXL one way or the other. I'm still in the minority here that I do not think there is even an 80% chance of approval. I'd put the current odds at 50%. But in 2015, I had the odds pegged around 10%. As we saw with a competitor today, unexpected things happen during these trials and sometimes drugs that look promising turn out to be duds later on. Especially with Alzheimer's candidates.
That said, AVXL is up nicely this year. And from its relatively recent lows of ~$2, it's a 10x return. Suckers wager or not, the profits generated (on paper anyway) are real. Even if AVXL is riding the coattails of other drug companies, their success is our success at least in the short-term and vice-versa.
You've been saying for a long time to sell AVXL. When it was at $3, $4, $5, $6, etc...
One of these days you may be right. The historical odds are in your favor. But for the time being, you should at least admit that in the short-term your sentiment has been incorrect. AVXL has presented a good opportunity to make money. There's virtue in humility, Squid.
Good luck with your investments.
The plan is unfolding nicely.
Step 1: Seek Rett Approval
Step 2: Get Rare Disease Priority Review voucher from FDA
Step 3: Use Priority Review voucher to expedite the A273 Alzheimer's or PDD approval process
Step 4: Profit
Step 5: Use those profits to advance A371 and other pipeline candidates
In my opinion, it all hinges on A273 being approved for Rett syndrome. Let the other companies cannibalize themselves over being first to market with Alzheimer's -- they may even submit their NDA before Anavex, but Anavex will be able to leapfrog them once it's in the FDA's hands thanks to its initial focus on Rett and securing the priority review voucher.
I like your optimism and hope you’re right but that would mean Rett is a $7.5 billion disease and I think it’s too rare for a treatment to be worth that much.
Perhaps it gets over $100 on Rett approval if investors extrapolate the data and bet on Alzheimer’s approval is likely but I am not sure Rett alone justifies a $7.5 billion market cap.
That's a great question. I think it's okay that the trials aren't in the United States. Approval elsewhere will move the needle here, and it's not like Australia is considered to be second or third-world when it comes to modern medicine.
I'd expect to see something similar happen as to what the FDA did a few years back with Project Orbis. When one domino falls, they all fall.
https://www.fda.gov/news-events/press-announcements/fda-takes-first-action-under-new-international-collaboration-australia-and-canada-designed-provide
Exactly. I am a bit more loose with my margins. As long as it holds $18, it's a step in the right direction. I think $20 is the magic number the market doesn't want to see if break under, if only because it's a nice milestone dollar amount. If/when it does drop under $20, I can see stop losses triggered and another 10% drop as a result. At that point I think if it can find its footing, it'll have a quick run back to recent highs with another one or two positive PR announcements.
I remember the Mudcats. I think they were playing AA ball. Glenn Davis was a solid player. He grew up not too far down the road in North Florida. I'm glad to hear he's doing well over there, his brief stint in GA must have made a wonderful impression if he's still got connections to the city.
You make a great point about AVXL being seen as more of a Rett stock than an Alzheimer's stock, which. is interesting because I don't think anyone on this board invested initially because of its interest in Rett syndrome. To me, at least, this has always been Alzheimer's-related and any other indications are a pleasant bonus.
I've been critical of Dr. Missling in the past, but I do applaud his decision to focus on an immediate, unmet need to create revenue -- Rett is significantly more likely to be approved quickly than Alzheimer's and I think the plan all along has been to use Rett to get the priority review voucher, which will then be used to speed up the FDA decision for Alzheimer's assuming the P3 readout hits all the endpoints. That is probably worth more than the $100 million or so he'd fetch selling it on the open market. With Biogen's drug having its own set of safety and efficacy issues, the opportunity to strike has never been better for Anavex.
I used to be primarily concerned that A273 won't have good P3 results. It's still possible, but my concern now is whether a competitor is able to sway the FDA into accelerated approval. If the Biogen approval showed us anything, being first is better than being better. Dr. Missling hopefully knows this and Rett approval will indirectly provide a catapult for Alzheimer's that puts us over the finishing line first.
We'll see...
Columbus is nice. I remember they had a pretty decent minor league baseball team, the RedStixx, that sent a number of players to the majors.
My account is back up and running now. Hopefully everyone else's is, too.
AVXL looking solid today. Lots of bumps to be expected but if you look at SAVA and ANVS, both of which are trading in triple digits, AVXL seems significantly discounted -- even if our O/S is greater than both of those companies.
I'm still trying to wrap my head around why AVXL is getting the short end of the stick. Dr. Missling has been on plenty of investor roadshow conferences so its not like funds are unaware of Anavex or what its accomplished over the years. And I don't think the negative perceptions from 2015/16 by the likes of Feuerstein, Sykes, Shkreli, et al. are still lingering years later.
Anyone have any thoughts on what the disconnect is between AVXL and its pre-revenue peers?
Hall County. And I'm having issues, too.
I’m hopeful that we get approval but I think discussing pricing before we get approval is counting chickens before they hatch.
Pro tip: complaining on a message board isn’t going to affect the price. Buy golf or sell, choice is yours, but there’s no reason to raise your blood pressure over something beyond your control.
Nothing has changed from when the stock was $30. Also, nothing has changed since it was $2. It’s still an experimental drug that may or may not get approval. We will know within 12-24 months.
It’ll be a while before it gets back there. We need $20 to hold, otherwise it looks like support will be in the $14-$17 range.
You grab the tar and I'll grab the feathers.
In all seriousness I don't pay any mind to any of those posts. I expected a pullback, but today's particularly brutal.
Let's see how July plays out...
Probably not a good idea to taunt the stock gods with those new AVXL millionaire lists. Anavex is now trading below its close on Friday.
I think it'll go back up, but if it's true that funds want in now (and I have no idea if that's the case), they're going to want these shares at cheap as they can get them.
I suspect many newly minted millionaires will want to preserve that status and will sell when they think there is a chance their account falls back to six digits. This may be what we're seeing playing out today.
Yes, thanks for the correction!
Some consolidation would actually be preferable for the long-term. It's fun watching these gains but the chart looks really oversold in the short-term.
Exactly. If Rett is approved and Anavex begins generating revenue from it, the days of needing to issue shares to fund future operations may be over. That's the big event I think that will get a lot of conservative institutions who may not be willing to invest today interested in a future investment down the line.
I could live with that.