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AVXL is the only stock in my portfolio that is green.
Does this mean the shorts are getting a little nervous?
Probably the best NDA roadmap I have ever seen. (Take special note of pages 16 and 26/27)
https://www.fdli.org/wp-content/uploads/2021/04/Mulkey-Elizabeth.pdf
Makes me wonder if this had anything to do with it? (Read both headlines below)
FDA Selects Ariana's KEM Platform for Biomarker Signature Validation
FDA has chosen to implement Ariana Pharma's KEM® (Knowledge Extraction Management) decision-support platform to facilitate data analysis for the validation of biomarker signatures. The technology will be used by FDA's reviewers to analyze pharmacogenomic and other data submitted through the agency's Voluntary Exploratory Data Submission (VXDS) program.
Ariana claims the collaboration will help FDA more systematically identify potential genomic fingerprints and develop recommendations relating to the analysis of genomic data prior to the submission of biomarker signatures. "We are looking forward to this collaboration to help the agency systematically analyze all equivalent signatures combining both genomic and phenotypic data, thus increasing chances of selecting the best biomarker signature," comments Federico Goodsaid, Ph.D., FDA's associate director for operations in genomics at the Center for Drug Evaluation and Research's Office of Clinical Pharmacology.
Ariana provides data-mining solutions and decision-support services for clinical and safety studies, drug discovery, diagnostics, and biomarker development. The firm's KEM platform is a rules-based method developed to mine data and systematically extract and manage all consistent hypotheses. Ariana claims the technology can be applied to biomarker discovery to identify the best subset of markers in order to maximize patient coverage, through the ability to evaluate heterogeneous information, including genomic, proteomic, polypharmacology, and clinical data. The firm also suggests that unlike existing numerical methods, KEM can generate and prioritize hypotheses, carry out more exhaustive analysis, and handle missing data. [From September, 2010]
https://www.genengnews.com/topics/omics/fda-selects-arianas-kem-platform-for-biomarker-signature-validation/
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Anavex Life Sciences and Ariana Pharma Collaborate to Accelerate Timelines and Improve Efficiency
October 5, 2016
[You see, one of the first things Dr. Missling did was to follow the FDA's lead in AI technology...so now both the FDA and Anavex are using Ariana's KEM technology to analyze data. (Not bad for a German, aye?)]
https://www.anavex.com/post/anavex-life-sciences-and-ariana-pharma-collaborate-to-accelerate-timelines-and-improve-efficiency
Great Story.
A House of Sticks Won’t Save You from the Big Bad Wolf
by Jay Martin
Picture this: a gym class, eighth grade. A line of students standing against the wall, waiting their turn to be measured. I was there, too, and when my turn came, the tape measure stopped just shy of 5 feet. That day, I earned a nickname that would stick with me for years: “Four foot eleven and a half.”
I was a late bloomer, a fact I came to terms with early on. I knew I would eventually realize my dreams, but it would take longer than I, or anyone else, wanted. This realization has been a constant companion, a prophecy that's unfolding just as I had expected.
Now, let's shift gears for a moment. Imagine a cold water fish.
Researchers at the University of Las Palmas de Gran Canaria, Spain, have been studying the effects of temperature on growth rates across a variety of species. Their findings are fascinating: when an organism is raised in colder than natural temperatures, its growth rate lags behind the average. Warm temperatures, on the other hand, have the opposite effect.
Consider the humble goldfish. If you raise one in warmer than natural water, it will grow at an astonishing pace. But its sibling, raised in colder water, will grow more slowly. Yet, when both reach maturity and are placed in water matching their natural habitat, they revert to the mean, and become indistinguishable.
There's one key difference, though: the fast-growing fish, raised in warm water, has a life expectancy reduced by up to 16%. The slow-growing fish, raised in cold water, lives up to 30% longer.
Why does this happen?
Warm temperatures speed up metabolism, leading to higher calorie consumption. It's a simple equation: more input equals more output. But the issue of life expectancy is more complex. As an organism grows, it uses its energy and resources for various activities - cellular replication, repair, and maintenance. If the rate of replication is so high that it demands resources that would have been applied to repair and maintenance, the organism grows with an abundance of tissue damage.
A slower-growing organism, however, has a more balanced distribution of resources. This ensures a strong and healthy foundation, reducing mutant cells, inflammation, and tissue damage. While these conditions may not cause trouble at a young age, as an organism ages and becomes more prone to disease, their vulnerabilities are exposed.
This principle isn't limited to goldfish. It applies to most mammals, reptiles, fish - and yes, humans.
This lesson, I believe, is universal.
Fast-growing trees produce soft, airy wood. Slow-growing trees yield hard, dense timber. As German forester Peter Wohlleben puts it in The Hidden Life of Trees, "A tree that grows quickly, rots quickly, and therefore never has a chance to grow old."
I see this principle at work among my friends, many of whom are entrepreneurs.
One friend, a software company owner, is always on the cutting edge of every emerging industry. He pivots with remarkable agility, captures market share, and is often recognized as a pioneer. Yet, his business hasn’t grown in the ten years that I have known him. He has had some phenomenal years, followed by some remarkably insolvent ones. Ten years later, there has been no actual growth, just a relentless pursuit of shiny objects.
Another friend built a seemingly mundane, blue-collar service business. For twenty years, he stayed focused on exceptional customer service and slow organic growth into new geographies. He eventually sold to a strategic competitor for a life-changing sum.
He was the cold water fish.
I appreciate concepts like this because I believe there are certain natural laws that govern us.
Investors seeking the quick win or the easy trade are the ones who get wiped out.
Entrepreneurs who fixate on shortcuts always face their day of reckoning.
Even my kids understand that a quick house made of sticks won’t save you from the big bad wolf.
So take your time. Be patient, and trust your process. Be the cold water fish.
Great post, tred.
Quote: "Dr. Kaufmann has played different roles, including site investigator, Principal Investigator, and DSMB member/chair, in almost 20 drug trials for neurodevelopmental disorders. In this context, he has been involved in virtually all neurobiologically-based drug trials for Rett syndrome."
Now THAT is impressive.
Thanks for sharing.
You have to have a market that will pay for your drug before you can make any money...and there's not too many people that can afford $3.2 Million for treatment!
$3.2 Million Gene therapy per patient. Initial market reaction is based on price shock and questions about reimbursement.
Some of the issues highlighted in article below.
https://www.investors.com/news/technology/srpt-stock-halted-sarepta-wins-fda-approval-for-duchenne-gene-therapy/?src=A00220
Looks like AVXL is taking a breather before it moves up another 50 cents.
I follow several other biostocks and they're not doing so well.
ANVS...........up a penny
BIVI..............down 25 cents after getting a nice pop a few days ago
BIIB..............down 75 cents but that's nothing for a $270+ stock
NWBO..........down a penny which is significant for that stock
SRPT............down over $1 and keeps falling. Heck, it's lower than before the FDA approved their drug a few weeks ago! The trend is definitely down for this stock. Watching its downward spiral is like watching death with a thousand cuts. You have to wonder what will it take to turn this around if it can't appreciate after FDA approval? (ouch!)
Quote: One can make a reasonably strong argument that having 2-73 approved for Rett improves the chances of approval for AD.
Yes. The FDA has two specific pathways for NDA approval.
1) You must submit two successful Phase 3 trials - but there are a few exceptions; or
2) One successful Phase 3 trial and the submission of that same drug (A2-73) for an already approved disease...which, in this case, would be Rett.
And frankly, I believe that was the original plan that Dr. Missling intended. So, it wouldn't surprise me if Dr. Missling follows through with that strategy. Especially if it increases his chances for approval and expedites his submission timeline.
This is where Dr. Jin's expertise and experience is most needed.
Will be over $9/share tomorrow if we get another day like this.
Would like to see more volume...but hey, I'll take it!
As you already stated, he could've used some boiler plate verbiage that is typical for incoming executives...but he didn't.
Read the words carefully:"I believe I can make a substantial contribution at this very important time in the Company’s transition towards commercialization.”
Doc,
What's your opinion on the recent insider purchase by Dr. Missling of 232,000 shares...with a current value of $1.9MM?
He already had 1 MM shares in his personal account. In all practicality, he should've sold those shares and reinvested into other "less-risky" investments. But he didn't! Instead he held onto those shares and added them to his stash.
IMO, a nice vote of confidence for the future of Anavex.
2H 2023
Again, that was "old Anavex" talking. Dr. Jin wasn't hired until Jan/Feb of this year.
Things change.
If you don't adapt to the new changes, you'll be stuck in the past...and miss the opportunities that lie ahead.
IMO, he's basing his (Feb-ish) prediction on the "old Anavex" that was under-staffed and overworked.
With all of the new hires, Dr. Missling now has Anavex running like a fine running machine.
We still have a few key positions that need to be filled...but we're very focused on what's on our plate now...so we should easily attain the goals that are set for 2H 2023.
Quote: "I get what your saying. I've been surprised at the dates I see on stories...2018...2015 .... So I get it. But I have a feeling we are hitting a new phase just now."
Yes. You hit the nail on the head!
We are definitely hitting a new phase. We have new people on board (like Dr. Jin - the top statistician from the FDA) that will expedite our data and present it with clarity.
And, with a staff of approx 40 employees it's obvious that we are now preparing for commercialization.
Been here since 2010 (with powerwalker and a few others) and I can assure you that we are definitely in a "new phase". The second half of 2023 will be the year that Anavex presents the TLR for the EXCELLENCE trial...and, even more exciting, the full data for the 2b/3 AD study.
Your timing is impeccable...especially with AVXL shares being pushed down below $10
Once 2-73 is approved, it will push the value of our pipeline to 10x...imo, of course.
AVXL is a Piece of SSSSSSS-SILVER!
LOL
Wow...AVXL is flexing its muscles today and will probably close in the green.
Even the XBI is clearly in the red.
LOL...it reminds of a quote by a famous golfer who had just made a very long 20-foot put.
Someone in the crowd yelled: That was pure luck!
The golfer looked into the crowd and said: The more I practice, the luckier I get.
Quote: "I think neuroinflammation is a great target for AD and drugs that regulate microglia/macroglia or promote M1 to M2 phenotype switching are reasonable to trial for this indication."
Wow...it's nice to see that you have finally accepted this MOA to address AD. (Very encouraging!)
Thanks for your perspective.
That's good DD. Thanks WM
Here's some additional history about Anavex and Dr. Missling's contributions to the company.
When Dr. Missling came aboard in 2013 the SP was about $1/share and the company was on the brink of bankruptcy. Below is the auditor's comments:
"The accompanying consolidated financial statements have been prepared assuming that the Company will continue as a going concern. As described in Note 1 to the consolidated financial statements, the Company had an accumulated deficit of $41,204,972 and negative working capital of $1,559,211 at September 30, 2013 and incurred a net loss of $3,700,046 for the year then ended. These conditions raise substantial doubt about the Company’s ability to continue as a going concern. Management’s plans in regard to these matters are also described in Note 1. The consolidated financial statements do not include any adjustments that might result from the outcome of this uncertainty. Our opinion is not modified with respect to this matter."
/s/ BDO USA, LLP
New York, NY
December 30, 2013
https://www.sec.gov/Archives/edgar/data/1314052/000106299313006497/form10k.htm#page_33
Once they hired Dr. Missling in 2013, he successfully executed a plan to turn the company around.
Since then, the SP has increased 800%...now at $8/share...they have $150 MM in the bank and no longer in debt...the employment growth has exploded in preparation for the commercialization of their new drug, Blarcamesine. (See below)
2013...Dr. Missling is hired (Thank God!)
2014 = 4 employees
2015 = 7 employees
2016 = 10 employees
2017 = 10 employees
2018 = 13 employees
2019 = 16 employees
2020 = 20 employees
2021 = 25 employees
2022 = 38 employees
https://www.macrotrends.net/stocks/charts/AVXL/anavex-life-sciences/number-of-employees
I thought my post was pretty straightforward.
Confirmed... Missling exercised 500k shrs and sold 268k.
"Anavex Life Sciences Corp.'s President and CEO just picked up 232,000 shares"
I responded with the following...
Since Dr. Missling could've easily sold those 232,000 shares on the open market, I now consider them as a purchase...and thus, he's now invested $1.9 MM in AVXL shares.
232,000 shares x $8.15 (which is the closing price) = $1.9 MM
IMO, this represents a purchase from an insider. (Thank you Dr. Missling!)
That's very good news!
Since Dr. Missling could've easily sold those 232,000 shares on the open market, I now consider them as a purchase...and thus, he's now invested $1.9 MM in AVXL shares.
Welcome to the club.
Correct me if I'm wrong, but aren't most families with Rett children automatically covered by some kind of federal insurance program? (I can't imagine any family having to pay all of the special medical care that is required to raise a Rett child)
And if they are covered by a federal insurance program, wouldn't they automatically qualify to receive Blarcasimine once it is approved by the FDA?
Anavex has only one shot to get FDA approval for their P2b/3 Alzheimer's NDA...so they need to make sure they include all of the P1s, P2s and P3 trials.
Everyone knows that a successful P3 trial is worth its weight in gold because it's a double-blind, placebo-controlled study...and the FDA values P3 results over all of the other P1s and P2s trials.
And remember, our P2b/3 trial (with only 509 patients) is probably the smallest n that has ever applied for an NDA for AD.
Waiting a few more months to add the Rett P3 trial to our NDA portfolio is just common sense. TGD knows what he's doing.
Cheers.
abe
What are you smoking?
In light of today's PR, would you like to change your statement?
Quote: "Rett has a 20-25% chance of showing data later this year good enough for an NDA submission in early 2024 (potential decision Q3)."
Interesting. Thanks for that insight Raja.
Yes. "Sufficient" is the key word there. No one knows for sure how the FDA is going to rule once they get all of the data for the NDA...so Anavex has only one shot to prove their case!
Everyone knows that a successful P3 trial is worth its weight in gold because it's a double-blind, placebo-controlled study...and the FDA will value those results over all of the other trials.
If it weren't for the Covid delays, I believe we would've had the Rett trials finished and approved by the FDA by now...which was Dr. Missling's original plan.
It would be a lot easier to get the FDA to approve our AD application if we could include all of the data from a drug that is already approved for Rett. Unfortunately we can't do that because, like I said, we had Covid delays.
But we can do the next best thing...which is to submit the P3 data from the Excellence trial...so I think it's smart to wait a few more months until it's finished.
LOL...even with all of the insider selling, NVCR is still valued at $4.5 Billion. Please explain why you think they're going to be obsolete?
Novocure Announces LUNAR Phase 3 Clinical Trial Demonstrates Statistically Significant and Clinically Meaningful Extension in Overall Survival for Patients with Metastatic Non-Small Cell Lung Cancer After Platinum-Based Therapies
"The LUNAR trial is the first study in more than seven years to show a significant improvement in overall survival in metastatic non-small cell lung cancer post-platinum chemotherapy. I am heartened by this progress and the potential of this innovative therapy to help many metastatic lung cancer patients in need of new treatment choices following platinum therapy, without added systemic toxicity."
"The significant improvement in overall survival as demonstrated in the LUNAR study is groundbreaking. Lung cancer is the leading cause of cancer-related death in China, and non-small cell lung cancer is the most common form of this disease. The prognosis is often very poor for patients who progress after treatment with a platinum-containing regimen in the front-line setting," said professor Li Zhang, M.D., Sun Yat-sen University Cancer Center. "I am very excited that this novel, non-invasive device can bring significant benefit to patients living with metastatic NSCLC in China."
And while you're trying to explain why Novocure is going to be "obsolete", please explain what happened to SRPT's share price?
They actually received FDA approval for their drug...and yet, SRPT's share price has dropped from $160 to just $105 in the last 30 days!
Quote: "What else explains this (lack of) SP action?"
Try comparing AVXL with another biotech company like NVCR.
NVCR reported a successful TLR on June 6th...and yet, their SP lost over 50% in 3 weeks!
Did their CEO "fumble" the TLR? NO
Is their CEO dragging his feet with other data? NO
Does the market not trust their CEO because he has no credibility? NO
So, why did their SP drop from $120 to just $42 after they reported a successful TLR?
If you can't answer that question, then you certainly can't accuse Dr. Missling of causing our SP to drop when the exact same thing is happening to other biotech companies.
When applying for an NDA, it's my understanding that you must submit all of the data that you've accumulated for a specific drug...which would include all P1s, P2s, and P3s from any and all trials.
The more successful trials that you submit with your NDA, the more evidence you have to strengthen your case for FDA approval...especially the P3s because they are double-blinded and placebo-controlled.
Therefore, doesn't it make sense for Anavex to include the P3 data from the Rett EXCELLENCE trial when they submit the NDA for the P2b/3 AD trial?
If you agree with this logic, then that would explain why Anavex hasn't submitted their NDA yet. They're simply waiting for the completion of the Rett Excellence trial.
Click on the previous post and you'll get the numbers.
One other point...
It is not uncommon for the market to give pharmaceutical companies a multiple of approximately 5x their gross revenue.
If Anavex treats just half the Rett population in the US, we would generate approx $4 Billion x 5x multiple = $20 Billion per year market cap.
Divide $20 Billion by 100 million shares and you get $200/share.
At the 22:40 Minute mark:"We would look to market the Rett Syndrome indication at approximately $500,000 annually, which is about the going rate for Trofinetide."
There are approximately 16,000 people with Rett syndrome in the United States today. If we treat only half of them, Anavex would generate $4 Billion/year in revenue.
8,000 patients x $500,000 each per year = $4.0 Billion per year.
And that's only treating half of the Rett population in the U.S. (I did not include the Rett population in Asia or Europe...which could easily add another ~$4Billion)
https://healthresearchfunding.org/18-amazing-rett-syndrome-statistics/
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Acadia is our only competitor to treat this disease. Their product to treat Rett is called: DAYBUE (chemical name: Trofinetide)
Acadia's PRICING
Acadia announced on their webcast that the list price of DAYBUE is $21.10 per mL. That comes to $9,495 per 450 mL bottle. Given the dosage included in the prescribing information, cost per patient, depending on weight, will be between $385,075 and $924,180 per year.
Acadia has not yet announced if there will be discounts or rebates on pricing.
Key Summary Facts of Acadia's LavenderTM and Lilac-1 Trials for Rett Syndrome
61% of patients taking DAYBUE did not improve
Only 13% of patients were rated as “much improved”
No data are provided regarding which specific symptoms improve
85% of patients treated with DAYBUE had diarrhea and 29% had vomiting
In the study where everyone received DAYBUE, 46% of patients withdrew before completing the study
TROFINETIDE DEVELOPMENT TIMELINE
Trofinetide is the chemical name for DAYBUE. Based upon efficacy in a rat model for traumatic brain injury (TBI), a clinical trial with trofinetide for treatment of TBI was initiated in 2008. In that study, trofinetide was administered intravenously. It did not demonstrate efficacy for treating TBI.
Trofinetide was then re-formulated as an oral solution and tested in individuals with concussion, Rett syndrome and Fragile X syndrome. DAYBUE’s official prescribing information states that its “mechanism of action” is unknown. This means that how DAYBUE produces an effect in Rett syndrome is not known. Although there is a publication reporting the efficacy of trofinetide in a Fragile X mouse model, there are currently no publications evaluating the efficacy of trofinetide in any animal model of Rett.
DAYBUE has been previously tested in a number of clinical trials in individuals with Rett syndrome. A clinical trial in adults (initiated in 2012) and a clinical trial in pediatrics (initiated in 2016) assessed its safety and tolerability.
The FDA approval of DAYBUE is based on the LavenderTM clinical trial, which began in 2019. In the prescribing information the trial is referred to as Study 1.
As shown in the table above, the LavenderTM trial had two primary clinical outcome assessments: RSBQ and CGI-I. A clinical outcome assessment aims to reflect how a patient feels, functions, or survives. Both the RSBQ and CGI-I are scales that require caregivers and clinicians to interpret how their child and patient are doing and are therefore subjective by nature.
WHAT IS THE RSBQ?
The Rett Syndrome Behavioral Questionnaire (RSBQ) is a 45-item survey that assesses behavioral and emotional characteristics of Rett.
Here are some examples of symptom statements included in the RSBQ.
Her breathing is sometimes deep and fast (hyperventilation).
Spells of screaming for no apparent reason during the day.
There are certain days/periods where she performs much worse than usual.
Has frequent naps during the day.
Expressionless face.
Bright wide-open eyes.
There are times when she is irritable for no apparent reason.
Rocks self when hands are prevented from moving.
https://reverserett.org/news/articles/daybue-trofinetide-key-facts-for-parents/
That's simply not true. Try comparing apples with apples.
NVCR is down 65% from their 1-year high of $120. Shares are now at $42...after they reported a successful TLR on June 6th.
AVXL is down 46% from their 1-year high of $15.24. Shares are now at $8.20...after we reported a successful TLR on December 1st.
And note that NVCR had an MC of $13 Billion before they reported their TLR. It is now down to just $4.5 Billion. What happened?
Did their CEO fumble the TLR? NO
Is their CEO dragging his feet with other data? NO
Does the market not trust their CEO because he has no credibility? NO
So, why did their SP drop from $120 to just $42 after they reported a successful TLR?
The same question can be asked of SRPT. They received FDA approval for their drug last Thursday...and yet, their SP has dropped from $160 to just $109. A drop of over 30% in less than a month! What happened to them?
The bottom line is that you can't point the finger at Dr. Missling when the exact same thing is happening to other biotech companies.
On June 6th, Novocure (NVCR) announced the following:
Novocure Announces Phase 3 Clinical Trial Demonstrates Statistically Significant and Clinically Meaningful Extension in Overall Survival for Patients with Metastatic Non-Small Cell Lung Cancer After Platinum-Based Therapies
Novocure (NVCR) is a multi-billion dollar company. But take a look at what happen to their SP after they announced the full Top Line Results!
I'm wondering if TGD is waiting for the results from the P2b/3 OLE so that he can include them with the AA submission?
Quote: "...we are 2 years behind ACAD in Rett,"
I'm not happy about being 2 years behind ACAD, but I am happy that ACAD was approved first because they established a cost approximately $500,000/year for us to compete with. (See my post below)
8:20 minute mark: Dr. Missling alludes to the fact that EXCELLENCE data will/should be extremely good considering the age of the patients. Later he mentions data is expected 2H 2023.
EXACTLY!!!
This reinforces my post a few weeks ago. Wake up people!
This is a $2B/year company with just 50% of Rett patients in the US!!! (Frankly, I think we'll capture 75 - 90% because the Rett families are a close-knit society...but I'm trying to be conservative...and I haven't even factored in Europe, Japan, or Australia!
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abew4me
Tuesday, April 04, 2023
Post#409874
You can use the data from the two Rett (adult) trials to determine the outcome of our pediatric (children) EXCELLENCE trial.
If A2-73 was effective with adults, the odds are extremely high that it will be more effective with children.
Once you accept that, you can realistically calculate the revenue that Anavex will make by comparing A2-73 (Blarcamesine) with Acadia's product, Daybue. (See below)
There are approximately 16,000 people with Rett syndrome in the United States today. If we treat only half of them, that would be 8,000 patients.
8,000 patients x $250,000 each per year = $2.0 Billion per year.
And that's only treating half of the population in the U.S.
https://healthresearchfunding.org/18-amazing-rett-syndrome-statistics/
****************************************************************************************************************
Acadia's product to treat Rett is called: DAYBUE
PRICING (From our only competitor)
Acadia announced on their webcast that the list price of DAYBUE is $21.10 per mL. That comes to $9,495 per 450 mL bottle. Given the dosage included in the prescribing information, cost per patient, depending on weight, will be between $385,075 and $924,180 per year. Acadia has not yet announced if there will be discounts or rebates on pricing.
KEY SUMMARY FACTS OF LAVENDERTM AND LILAC-1 TRIALS
61% of patients taking DAYBUE did not improve
13% of patients were rated as “much improved”
No data are provided regarding which specific symptoms improve
85% of patients treated with DAYBUE had diarrhea and 29% had vomiting
In the study where everyone received DAYBUE, 46% of patients withdrew before completing the study
TROFINETIDE DEVELOPMENT TIMELINE
Trofinetide is the chemical name for DAYBUE. Based upon efficacy in a rat model for traumatic brain injury (TBI), a clinical trial with trofinetide for treatment of TBI was initiated in 2008. In that study, trofinetide was administered intravenously. It did not demonstrate efficacy for treating TBI.
Trofinetide was then re-formulated as an oral solution and tested in individuals with concussion, Rett syndrome and Fragile X syndrome. DAYBUE’s official prescribing information states that its “mechanism of action” is unknown. This means that how DAYBUE produces an effect in Rett syndrome is not known. Although there is a publication reporting the efficacy of trofinetide in a Fragile X mouse model, there are currently no publications evaluating the efficacy of trofinetide in any animal model of Rett.
DAYBUE has been previously tested in a number of clinical trials in individuals with Rett syndrome. A clinical trial in adults (initiated in 2012) and a clinical trial in pediatrics (initiated in 2016) assessed its safety and tolerability.
The FDA approval of DAYBUE is based on the LavenderTM clinical trial, which began in 2019. In the prescribing information the trial is referred to as Study 1.
As shown in the table above, the LavenderTM trial had two primary clinical outcome assessments: RSBQ and CGI-I. A clinical outcome assessment aims to reflect how a patient feels, functions, or survives. Both the RSBQ and CGI-I are scales that require caregivers and clinicians to interpret how their child and patient are doing and are therefore subjective by nature.
WHAT IS THE RSBQ?
The Rett Syndrome Behavioral Questionnaire (RSBQ) is a 45-item survey that assesses behavioral and emotional characteristics of Rett.
Here are some examples of symptom statements included in the RSBQ.
Her breathing is sometimes deep and fast (hyperventilation).
Spells of screaming for no apparent reason during the day.
There are certain days/periods where she performs much worse than usual.
Has frequent naps during the day.
Expressionless face.
Bright wide-open eyes.
There are times when she is irritable for no apparent reason.
Rocks self when hands are prevented from moving.
https://reverserett.org/news/articles/daybue-trofinetide-key-facts-for-parents/