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Ah yes. MAA not NDA. I'm working through a case of the Flu and nowhere close to 100%. Thank you for the correction.
Missling has never been very communicative about meetings with the FDA. We mostly find out about them well after the fact and then only when the decision reached at the meeting becomes a point of serious contention in the public discussion.
We don't know exactly what data was presented to the EMA. Apparently enough data was presented such that the EMA thought it was worth filing an NDA and looking at the full NDA package.
The meetings and initial date presentation is the start of the approval process. The submission of the full NDA package can be 7 months in the future.
If that question is asked the company may not want to answer directly as part of its market surprise strategy.
Exhibit B.
I provided a piece of information. If that makes everything OK for you, great.
I found it an interesting overview of the current state of the developmental biotech space and thought others might find it interesting also.
Overall view of the small biotech industry slump this year.
https://techxplore.com/news/2023-11-biotech-slump-labs-threatening-drugs.html
NOVEMBER 24, 2023
Editors' notes
A biotech slump is emptying labs and threatening research on new drugs
by Gerry Smith, Bloomberg News
Credit: Pixabay/CC0 Public Domain
Sangamo Therapeutics Inc.'s cost cuts didn't hurt just the roughly 162 U.S. workers who lost their jobs this month. It's also a blow to Jerry Walter, who's lost five family members and suffered kidney, lung, hearing and heart damage from a rare disease.
Citing the "challenging economic environment" that's sending shock waves through the biotech industry, Sangamo paused research on an experimental gene therapy—possibly a cure—for Fabry disease. Walter, a 69-year-old retired U.S. Army colonel who's already had a heart transplant due to the inherited enzyme deficiency, said it's disappointing to see work stop on yet another potential new medicine.
"The promise of a better treatment keeps getting pushed down the road," he said.
Sangamo will finish a current Fabry trial as planned and is seeking a partner for help with moving its studies to the final stage, a spokesperson said. Yet it's been another bleak year for biotech companies as a closely watched index of stocks, often called the XBI, just passed 1,000 days from its early 2021 peak. The number of companies exploring strategic options—typically a sign of desperation—just hit a record high, according to analysts from Stifel.
"Nobody knows where the bottom of this is," said Sam Fazeli, an analyst at Bloomberg Intelligence.
Money for research is getting harder to find: New flows into biotech venture funds are estimated to fall to $23 billion this year from about $31 billion in 2021, the Stifel analysts said. Rising interest rates, threats to prices of new drugs and a post-pandemic letdown have forced biotechs to pull back on research and development.
Last year, global pharma R&D spending fell 2.2% to $244 billion, the first drop in a decade, according to Evaluate Pharma, and slowed growth is expected in the coming years. It was just the fourth such decline in almost four decades, according to the PhRMA industry group.
The sector has seen "serious slimming down of pipelines at the small end," Evaluate said in its August report.
Crucial work
Big drugmakers have increasingly moved into areas that used to be biotech's domain—like cancer and rare diseases—seeking profits from drugs that aren't easily replaced by competitors. French drugmaker Sanofi is boosting R&D spending, while Eli Lilly & Co., maker of blockbuster weight-loss drugs, increased this year's R&D budget by $600 million.
But the industry giants can't replace the crucial work done by smaller biotech companies, said Derek Lowe, a longtime drug researcher who writes the industry blog "In the Pipeline." In many cases, biotech companies handle risky early-stage research that big drugmakers may invest in if it shows promise.
Biotech companies often take on challenges that are a bit "out there," Lowe said. Big manufacturers "let the little companies take a crack at it and see how it goes."
Gone is the pandemic enthusiasm that drove COVID-19 shot-maker Moderna Inc.'s 12-month trailing price as high as 188 times diluted earnings per share from continuing operations in June 2021. Now the company is expected to report losses on an adjusted basis over the next year, and more than half the analysts covering it don't recommend buying shares.
An industry slowdown was inevitable, Fazeli said. Firms are shifting their strategies in response. Unable to find investors who can provide more funds, small biotechs are skipping the long shots to focus on sure bets that are more likely to generate income soon or attract a deal with a bigger drug company, said John LaMattina, a former head of R&D at Pfizer Inc.
That means less creativity in the sector that gave rise to COVID-19 shots, life-saving gene therapies and other treatments for once-fatal diseases. Those include Merck & Co.'s Keytruda, which first took shape about two decades ago at biotech company Organon & Co. Today the drug has treated more than 1 million patients with more than 20 different cancers, generating some $21 billion in sales last year.
Medicine paucity
"The concern is that in three or four years from now there will be a paucity of innovative medicines," said Jeff Jonas, former chief executive officer of the biotech company Sage Therapeutics Inc. "There is potential for the early ecosystem to suffer."
The impact is hitting companies like Charles River Laboratories International Inc. that does work including early safety research on rats that precedes human testing. The firm has been involved in studying more than 80% of all U.S.-approved drugs in the past three years.
Biotech companies that once investigated four or five products at a time now just have enough funding for two, said Jim Foster, Charles River's chief executive officer. The result: organic revenue is expected to grow about 6% this year, roughly half its 2022 level.
"People are reprioritizing, being more judicious," Foster said in an interview. "We are a canary in the coal mine."
Lab space vacancies in the Boston biotech industry hub have hit a 10-year high, according to the real estate firm Colliers. For the first time, there's about 5 million square feet of space on the market, up from 300,000 two years ago.
IRA fears
Biotech's woes could be a problem for big drugmakers, said Lowe, the pharma blogger: a smaller crop of candidates for big drug companies to buy later on. Those drugmakers need new products to make up for older ones that will be facing competition when their exclusivity runs out.
Aging drugs are also eligible for Medicare price negotiations under the Inflation Reduction Act, President Joe Biden's signature legislation. Drugmakers have said that the law effectively shortens their products' most profitable period, and that some early development programs will have to be sacrificed.
All that is bad news for Walter, the former colonel who founded the National Fabry Disease Foundation. Twice a month, the Hillsborough, North Carolina, resident sits for three hours getting enzyme replacement through an IV.
Before running short of money, Sangamo was pursuing a one-time treatment aimed at fixing the gene flaw responsible for the disease, meaning Fabry patients wouldn't need those twice-monthly infusions. Those who got it in a trial "continue to report improvements in their quality of life," the company said.
"We believe this is an important potential medicine and are doing everything in our power to still get this to patients in need," the Sangamo spokesperson said in an email.
The treatment could have been "revolutionary," had work on it continued, Walter said, adding he hopes that Sangamo can resolve its funding issues.
Would it make sense for the CC to be it's usual not much and then after the call to make any announcements?
The year end 10K is really about the past. Subsequent events are those between the end of the fiscal year and the publication of the 10K.
Get the CC over which clears the decks for new information.
As of 2022 10K accumulated deficit was $245,563,781.
With Missling, there is no predicting what he will do with the voucher.
It hits the books as an asset with about $100,000,000 value. He may want to use that asset value to help secure additional financing. He has said that as soon as possible he wants to use conventional bank financing going forward. Having that on the books should help secure a much better loan rate as it would be tangible collateral. At this point the company doesn't have much in assets other than Intellectual Property. A bank valuing 2-73 as a drug will have to be done once it is approved and generating an actual revenue stream. As we saw with the mabs recently approved, getting the drug on the market doesn't always translate into a big revenue stream and the banks know that.
You make a good argument except for ignoring what Missling has repeatedly said about Rett first in the US.
I'm Sooo proud of you.
What does that 12X1:GR mean?
Thanks for the reply. I wish you well in you endeavors.
Where did the 12/1 requirement come from? Missling said EOY.
Raja what is your thinking on that?
I'll be surprised if there is any significant short covering in that report.
Probably not a PR tomorrow. That is a holiday in the US. Not to mention the very negative spin you put on that "theory".
2-73 for AD will be considerably cheaper than the liquid form for Rett 2-73. My guess is the price for Rett treatment will drop considerably when AD is approved and there is a mass market for 2-73.
2-73 for insomnia maybe an occasional pill rather than an every day thing. We will have to wait and see what the price of 2-73 is for AD treatment.
Of course those tests are necessary. Adding any indication to a drugs labeling requires a standard demonstration of efficacy.
Sleep is a complex phenomena. Data about REM sleep doesn't say anything about the other three stages of sleep. Disrupt any one of those and it is not a good thing.
At present there is some consideration that disrupted deep sleep contributes to AD and other CNS issues because it is deep sleep where the brain flushes out the metabolic wastes generated during the day. Incomplete flushing is thought to result in increased inflammation.
I think that happened yesterday.
A patent is no substitute for trial results.
Each indication will require a trial.
Right now, an approval for any indication is the priority and the necessary first step to additional indications.
Once 2-73 is approved for anything I can see Anavex starting trials for a number of other indications.
Once a drug has applied for a trial it is always a drug and can not become a supplement. It can on the basis of a safety profile become an over the counter drug.
Voltaren is a recent example of that.
Wasn't that also mentioned in the AD trial that sleep improved.
No problem with whatever level of certainty or lack thereof anyone has.
Rather, I find it curious that predicting the reactions of others as if it was somehow important when the issue is a matter opinion is worthy of a dismissive comment.
And you care about that why?
If we really get nothing this year the WGT lot will immediately come up epically wonderful and cunning reasons with a lot Great Post accolades.
Such an amazing leap to a conclusion. I am in awe.
And what? All you know is that he hasn't publicized any meetings with the FDA.
Thanks for the heads up on that.
Many a slip twixt the cup and the lip.
I think that Anavex has clearer case to be made for making the drug available to treat a disease with massive impact and few real choices. Even so, it is wise to be aware of the road ahead.
Given the timeline for EMA approval it looks like the Rett application will be in the hands of the FDA and most likely approved before the EMA can approve 2-73 for AD.
Based on the information provided by boi568, I would expect the FDA application for AD to be in process and possibly approved before the EMA application.
Seems to me that an application in Australia for AD will be next up. Looks like the AU procedures are faster.
a pre-submission planning phase where applicants lodge details of a proposed application at least 2¼ months prior to lodgement of the dossier allowing the TGA to identify milestone dates and plan resource requirements (this is not required for submissions lodged in eCTD format if the sponsor selects the PPF-only option)
2.4.5 Priority review designated applications
TGA now has a formal Priority review pathway for faster assessment of vital and life-saving prescription medicines for which a complete data dossier is available. The target timeframe of 150 working days is up to three months shorter than the standard prescription medicines registration process. A valid Priority review designation must be held in order to access the Priority review pathway.[/quo
Based on the eligibility requirements it appears that Anavex is eligible for priority review but Anavex must apply for that designation.
The EU process makes the FDA process look swift.
How many times have you ignored what Missling has said about Rett first in the US?
Wishful thinking?
If you believe there are users using multiple aliases send a message to admin with names. Admin is pretty good at catching multiple aliases when alerted.
I
Peer review wont help the stock, PW.
The problem with that theory is Anavex can not control when the peer review paper will be published.
Generally the authors will be notified in advance of when the paper is going to be published. So perhaps Anavex could work that angle.
Releasing TLD can be done in advance of the PR paper. The PR would likely not be as in depth as it would be after the paper is published, but then afterwards the info would already be out there.
Actually that's not true.
Someone has to do it.
Thank you for stating the obvious.
Thank you for stating the obvious.
My take on the odds of Rett approval are 95%. YMMV
What seems to be forgotten in the bitching is that the voucher is 100% profit.
That is revenue. That is not profit is it?
Whoopee!. Once in how long?
I find zero value in the predictions.
Keep pounding that narrative. It will be interesting to see what the narrative will be when the Rett NDA is filed and then approved.