Anavex Life Sciences Corp (AVXL)

[abew4me]

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/
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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/