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2023 Top shelf Due Diligence for those seeking information.
Thanks to MayoMobile for the best comprehensive data and indepth analysis. See link at bottom.
(( The following part is my opinion only and not an advice to buy or sell AVXL.
I have been investing since Moses was a little boy it seems, and occasionally I have witnessed a new company that comes along destined for greatness. We all know the familiar names The-Wheel ...fast forward > Microsoft, Amazon, Amgen, etc. that gave stunning ROI. If you believe that AVXL will receive a drug approvals for treating Alzheimer, then IMO the returns on the investment will be stunning based on 7 Million with AD in Europe, 6.7 Million in the US, 401,300 in Australia, and 55 Million WW.
No matter how conservative you figure the revenue, if the drug receives approval then the revenue will be record breaking in the CNS space; and quite likely for any drug WW considering all the indications treated, like Parkinson and Rett; and the ubiquitous rare diseases with no current treatments. That is my personal take)).
https://www.sotcanalytics.com/update-compendium-2023
As of 2022 10K accumulated deficit $245,563,781
Today's analysis from Mayo Mobile: regarding the EMA/AVXL news:
Bottom-line Up Front (BLUF): On 20 Nov 2023, Anavex re-ignited investor interest by disclosing that the company has been in discussions with the European Medicines Agency (EMA), and that the company has made initial filing for Centralized Procedure.
Assessment: It has been Spirit of the Coast's position for nearly a year that Anavex's first - or at least most emphasized - regulatory effort would be with the EMA. In fact, on the SOTC homepage we write: we assess it is likely Anavex will pursue European approval first and foremost, as they designed the 2b/3 trial to accommodate EMA Alzheimer's guidance, and the European population has the absolute most favorable genomic qualities for effective therapy.
According to a large genome database, the European population has approximately 7.5% greater S1R WT prevalence than the American population, 2.5% greater than the African population, and 18% greater than the Asian population. This statistic is bolstered by the fact that Americans with European ancestry have the highest S1R WT prevalence of American population groups.
Beyond having the most favorable genomic qualities for efficacious therapy, the Alzheimer's 2b/3 trial design and outcomes are both in alignment with EMA guidance and approval values. Earlier in the year, we had this to say:
Anavex approached their trial design deliberately with EMA (European) approval as a primary consideration. Unlike the Aducanumab and Lecanemab trials (ENGAGE, EMERGE & CLARITY), the Anavex Alzheimer's 2b/3 utilized guidance directly from the EMA's 2018 Alzheimer's instruction. This is important because so far, MaB treatments like Aducanumab and Lecanemab have failed to garner approval in Europe and Australia. Reason being, the EMA noted that although Aducanumab reduces amyloid beta in the brain, the link between this effect and clinical improvement had not been established. In addition, the studies did not show that the medicine was sufficiently safe. Therefore, the EMA's opinion was that the benefits did not outweigh the risks.
Anavex has excellent opportunity for approval in Europe due to their deliberate accommodating design. These are some of the items implemented towards this end.
Favors co-primary endpoints with a cognitive outcome (ADAS-COG) and a functional outcome (ADCS-ADL).
Secondary endpoint with global measure (CDR-SB).
Company clearly identified the 'responder' criteria.
Beyond those design considerations, differences between FDA & EMA Alzheimer's guidance is favorable towards Blarcamesine as shown below:
EMA favors disease modification whereas FDA favors a 'persistent effect on disease course.
This is interesting because when Lecanemab patients stopped dosing, the little improvement they had began to once again degenerate.
Blarcamesine is easily identifiable as the only true disease modifying compound to complete pivotal trials. [Edit: with Donanemab data now available, I do believe it is sound to consider the drug disease modifying]
EMA favors trial designs showing dose dependency (30mg + 50mg).
EMA approves less drugs than the FDA, but the EMA places higher value on high therapeutic value.
From a semi-recent analysis:
Expedited drug approvals requiring high therapeutic value: 45% (FDA), 67% (EMA)
Standard drug approvals requiring high therapeutic value: 13% (FDA), 27% (EMA)
"In mild to moderate AD to accept an effect on cognition it should be clinically meaningful. The clinical relevance should be confirmed by an effect on function or clinical global assessment in a co-primary endpoint approach."
More information can be found here, but as a brief summation, Anavex has postured themselves in clinical design to approach the EMA for approval. Additionally, their 2b/3 outcomes meet approval preferences by the EMA, being an efficacious, inexpensive, and safe drug. In our opinion, Lecanemab still fails to meet many of the criteria set out by the European regulatory agency, and Lecanemab is unlikely to garner approval there; we give an ~20% chance.
More MayoMobile analysis information in this link:
https://www.sotcanalytics.com/update-compendium-2023#h.xkrui77mb8s8
On the Fri after Thanksgiving the stock market closes early at 1:00 et.
An estimated 6.7 million Americans age 65 and older are living with Alzheimer's in 2023. However the European Alzheimer population in Europe is a little larger at 7 million.
Todays bullish EMA news targets an European Alzheimer population of approximately 7 Million.
"It is estimated that 7 million people in Europe alone already live with Alzheimer’s disease. With a rapidly ageing population, it is a growing public health concern worldwide as this number is projected to rise to a staggering 14 million by 2030."
https://www.braincouncil.eu/projects/rethinking-alzheimers-disease/
Thanks for the chart NWDR, I recall seeing you post that in the past.
One item I question as being even below conservative estimates is Alzheimer, Epilepsy, and Parkinson being treated at 20% of the disease population. Seems in my estimation you could double that to 40% and I still would see that figure as being too low. I cannot imagine people with AD and PD not take a drug that would improve their situation.
I was the care giver of my grandmother who succumbed to Alzheimer, and know first hand that any help would be a blessing.
Then there is Australia's TGA, where most of the trials were held; I'm speculating that Australia could be the next hammer to drop; and before the FDA.
I'm seeing this vol already over a million shares on good news, yet shares are available in the $6 range. IMO it has to be shorts selling to keep the lid on the SP preventing a break out, there are plenty of sellers.
Who would be selling shares in the $6 - $7 range after the bullish PR: "Anavex Initiates Regulatory Submission of Oral Blarcamesine for Alzheimer’s Disease to European Medicines Agency (EMA)" it has to be the shorts selling shares down here.
Multiple PR train will break the shorts, and it is coming.
I was surprised to see this important PR being released on a holiday week when some fund managers take a break. This tells me that there is much more good news to come and so little time to space them out. We shall see and we won't have to wait long.
MarketBeat Analysts' Consensus Price Target $43.25
589.79% Upside
High Forecast $80.00
Average Forecast $43.25
Yahoo Finance
Average Price Target: 47.75
Wall Street Journal
Stock Price Target AVXL
High $58.00
Median $47.00
Low $39.00
Average $47.75
Based on all data announced to date. These targets were before todays good news.
Thanks to Brian5220 on ST.
FWIW Higher than avg vol in AH trades
AH Close: Consolidated Last Trade $6.22 +0.47 (+8.17%)
After-Hours Volume 100,288
After-Hours High $6.22 (05:00:34 PM)
After-Hours Low $6.22 (05:00:34 PM)
https://www.nasdaq.com/market-activity/stocks/avxl/after-hours
'Anavex Alzheimer's And Parkinson's Drug Underappreciated By Investors' ... thanks for the summary bas; I thought this point was laid out well. I'll go with Dr Jin looking at the data over any contradictory poster which doesn't have Dr Jin's skills, and not having the data that he has at his disposal and purview.
"The September press release declaring again the AD trial a "success" was published six months after the hiring of Dr. Jin, so if there is any question about the veracity of the "successful" Phase 2b/3 trial, it is on Dr. Jin's name. And it is up to you, the individual investor, to decide whether you trust Dr. Jin or if you trust certain writers who claimed that Anavex could not even subtract numbers accurately.
My hunch is that Dr. Jin will not risk his reputation by writing a press release that does not meet strict statistical standards. So when Anavex says that a p<.025 on both the ADAS-Cog and the CDR-SB combined with statistically significant findings in two important biomarkers spells a successful trial, I expect it to be true."
With the SP up almost 8% on no news, I am posting this for new eyes that are looking for info:
Near term, see pg 8 in the link below. Missling has reiterated these actions before this year ends:
Alzheimer’s disease: Full data ANAVEX®2-73-AD-004:
Potentially pivotal Phase 2b/3 clinical trial
Parkinson’s disease dementia: Data of 48-week OLE
Phase 2 study
Parkinson’s disease: Initiation of ANAVEX®2-73
imaging-focused trial and pivotal 6 months trial
Top-line data Rett syndrome: EXCELLENCE
potentially pivotal Phase 2/3 pediatric clinical trial:
2H2023
Fragile X: Initiation of potentially pivotal ANAVEX®2-73
Phase 2/3 clinical trial
Schizophrenia: Initiation of ANAVEX®3-71 Phase 2
clinical trial
New Rare disease: Initiation of potentially pivotal
ANAVEX®2-73 Phase 2/3 clinical trial
Publications: Several clinical publications involving
ANAVEX®2-73, ANAVEX®3-71 and Rett syndrome
Burden of Illness study
Source, page 8:
https://www.anavex.com/_files/ugd/79bcf7_97da6311c3b643bea8e9a6659bb51422.pdf
.29 Gapper.
Speculation on when I think the PRs will start rolling out;
Last year: "NEW YORK – November 28, 2022
Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, today reported financial results for its fiscal year ended September 30, 2022."
News is highly likely coming this week if the news will be out before this year's financial results are reported for its fiscal year ended September 30, 2023. The following week will be TG week and nothing important is announced on Holiday weeks.
Time is getting really tight to roll out the significant number of announcements he indicated would be out before the year ends. I am speculating he is planning on running a train of PRs to force short covering, and I logically speculate with reasons that the PR train starts next week.
I don't think Missling will want to report in the company web cast empty handed.
Missling reiterates every time he speaks that the Rett TLD and Alzheimer full data will be PRed before this year ends; now only 2 months left. That will happen if for no other reason than to not get in trouble with the SEC for false statements, and to keep shareholders from filing law suits. There are other important PRs that he said will be out this year as well. I think he is going to run a PR train once the Rett PR is out (I'm expecting it to be in front of the AD PR) with a string of PRs till the end of this year.
I also think he will PR during the first hour of trading with multiple 'Halts'. Shorts will be slaughtered on the halts as Institutes, Funds, retail, and turncoat shorts rush to buy any shares available.
MayoMobile Many thanks for the effort you put into this report on A3-71. I'm surprised and impressed by the positive results after the rats completed the wash out period "Anavex 3-71 was able to prevent cognitive decline in McGill-APP mutation rats. Efficacious and significant results were garnered in working memory, spatial navigation, and reference memory tests. In addition, the drug had profound affect on the decline in social interaction. Social engagement has a protective effect against age-related cognitive decline, and social withdrawal is one of the earliest signs in Alzheimer's progression. Importantly, these combined cognitive tests show that Anavex 3-71 has marked improvement on multiple brain regions and region interaction. Finally, it is my opinion that some of the results seen lend to the possibility that completely healthy people could see improvement over baseline for both spatial learning and working memory, as well as possible minor social/behavioral benefits."
Mayo's Full and detailed report:
https://www.sotcanalytics.com/update-compendium-2023#h.ldx1og7f1mpm
And another one bites the dust: (competition SAVA)
https://www.science.org/do/10.1126/science.adl3444/full/cuny_wang_final_report-1697142265780.pdf
"Almost Half of All New Drug Approvals in 2018 Relied on One Clinical Trial"
"Most recently, IQVIA released a report finding that 25 of 59 (42%) novel drugs approved in 2018 were approved on the basis of only one trial. And one out of eight approvals relied only on Phase 1 or 2 trials, with no Phase 3 trials. But as in previous years, a large portion of the drugs relying on only one trial were new orphan and cancer drugs."
The source:
https://www.raps.org/News-and-Articles/News-Articles/2019/5/Almost-Half-of-All-New-Drug-Approvals-in-2018-Reli
Best post of the day, and most informative IMO.
Braking News "House passes 45-day government funding bill, putting Congress on a path to avoid a shutdown"
Good news for the markets.
Looks like the shorts are having a difficult covering with the shorted share count up.
They have painted themselves in the proverbial corner, with no way out.
I'm looking for a hard short squeeze on a surprise excellent PR. I hope it happens with a halt right after the market opens.
A PR showing Rett Excellent with outstanding data would do it.
A PR on the Alzheimer trial showing patients with significant improvement and over the base line;
along with a significant number of patients that did not decline. That would be a mega-squeeze..
New short number is out, and it is an increase and no surprise:
Settlement Date Short Interest Percent Change Average Daily Share Volume Days to Cover
09/15/2023 17,087,678 1.24 1,098,054 15.56
08/31/2023 16,878,865 0.94 621,130 27.17
08/15/2023 16,721,112 1.12 1,075,407 15.55
07/31/2023 16,536,540 (1.15) 671,751 24.62
07/14/2023 16,728,610 (0.07) 623,329 26.84
06/30/2023 16,739,768 (5.64) 1,148,109 14.58
06/15/2023 17,739,562 7.45 1,165,885 15.22
05/31/2023 16,508,942 3.82 763,358 21.63
05/15/2023 15,901,191 10.58 937,267 16.97
04/28/2023 14,379,183 (7.80) 798,137 18.02
04/14/2023 15,594,970 (1.84) 981,502 15.89
03/31/2023 15,887,617 1.34 1,275,882 12.45
03/15/2023 15,677,855 (3.93) 1,171,753 13.38
02/28/2023 16,320,041 (4.70) 850,227 19.19
02/15/2023 17,125,449 2.43 1,160,761 14.75
01/31/2023 16,719,384 (8.98) 1,128,968 14.81
01/13/2023 18,368,256 0.63 1,504,154 12.21
12/30/2022 18,252,546 15.66 1,422,909 12.83
12/15/2022 15,780,885 32.45 7,073,260 2.23
11/30/2022 11,914,762 17.19 1,787,726 6.66
11/15/2022 10,167,456 0.61 986,389 10.31
10/31/2022 10,105,713 0.62 985,598 10.25
10/14/2022 10,043,451 (2.12) 718,819 13.97
09/30/2022 10,260,594 5.37 936,366 10.96
https://nasdaqtrader.com/Trader.aspx?id=ShortInterest
Alzheimer Agent Blarcamesine Shows Significant Reduction of Amyloid-ß Biomarkers in Phase 2b/3 Trial
Sep 15, 2023
Isabella Ciccone, MPH (Thanks to My_Best_Guess and Guzzi on ST)
Newly announced findings from a follow-up analysis to the phase 2b/3 study (NCT03790709) assessing blarcamesine (Anavex Life Sciences), an investigational therapy, demonstrated a significant reduction in pathological amyloid-ß levels in plasma, as well as a significant slowing in the rate of pathological brain atrophy on MRI scans in treated patients with early Alzheimer disease (AD). According to Anavex, blarcamesine is one of the first therapies to demonstrate efficacy on biomarkers of neurodegeneration and may slow cognitive decline among patients with AD.1
In the study, blarcamesine-treated patients showed significant increases in validated biomarkers of amyloid-ß pathology, plasma Aß42/40 ratio (P = .048), further demonstrating the agent’s strong antiamyloid effect. Additionally, MRI findings showed significant reduction in brain volume loss, including whole brain (P = .0005), when blarcamesine was comparedwith placebo.
“There is hope that new therapies for Alzheimer that target the disease beyond amyloid that may slow progression of the disease for many people with the earliest forms of the disease,” Marwan Noel Sabbagh, MD, professor of neurology and chairman of the Scientific Advisory Board said in a statement.1 “The advantage of blarcamesine (ANAVEX2-73) is that it is a small oral molecule that exerts clinical benefits on cognition and neurodegeneration and could be appealing because of its route of administration and excellent safety profile.”
The trial was a multicenter, randomized, double-blind, placebo-controlled, phase 2b/3 study that enrolled 508 participants with early symptomatic AD. The participants, recruited from 52 medical research centers and hospitals in 5 countries, were randomized to receive blarcamesine (n = 338) or placebo (n = 170) oral capsules once daily for 48 weeks. The Alzheimer’s Disease Assessment Scale-Cognitive (ADAS-Cog) and Alzheimer’s Disease Cooperative Study-Activities of Daily Living (ADCS-ADL) subscales were used as primary end points to assess the cognitive and functional efficacy of blarcamesine. Using a mixed model for repeated measures, all prespecified clinical end points were analyzed.
The differences in the least-squares mean (LS) change from baseline to 48 weeks between the blarcamesine and placebo groups were -1.783 (95% CI, -3.314 to -0.251) for ADAS-Cog (P = .0226). In addition, at the same time point, the LS difference between the 2 groups were -0.456 (95% CI, -0.831 to -0.080) for Clinical Dementia Rating Scale Sum of Boxes (P = .0175).
“These data are very exciting, particularly in a study that can demonstrate objective slowing of markers of neurodegeneration,” Michael Weiner, MD, professor of radiology and biomedical imaging, medicine, psychiatry, and neurology at the University of California, San Francisco (UCSF) and principal investigator of the Alzheimer's Disease Neuroimaging Initiative (ADNI) said in a statement.1
One of the most common treatment-emergent adverse events in the respective safety population was dizziness, which was transient and mostly mild to moderate in severity. This adverse event was reported in 120 participants (35.8%) during titration and in 76 participants (25.2%) during maintenance with blarcamesine. In comparison, this adverse effect occurred in 10 participants (6.0%) during titration and 9 (5.6%) during maintenance with placebo.
“Alzheimer disease is such a devastating disease that affects tens of millions worldwide, and Anavex’s clinical development is a testament to our determination to follow the science,” Christopher U Missling, PhD, president and chief executive officer of Anavex said in a statement.1 “We like to thank all the people involved in the study for their invaluable contributions and we look forward to advancing blarcamesine as a potential new convenient orally available treatment option for Alzheimer disease.”
https://www.neurologylive.com/view/ad-agent-blarcamesine-significant-reduction-amyloid-beta-biomarkers-phase-2b3-trial?fbclid=IwAR2EgknljYnKnrpPYx7PuHRv8lElnBPmW0Ng0gnjsMDGRgNO6S1449oXbMc
Unusually high vol 251,091 in after hours.
https://www.nasdaq.com/market-activity/stocks/avxl/after-hours
Mirae Asset Global Investments Co. Ltd. lifted its holdings in shares of Anavex Life Sciences Corp. (NASDAQ:AVXL - Free Report) by 51.5% in the 1st quarter, according to the company in its most recent disclosure with the Securities and Exchange Commission. The firm owned 51,724 shares of the biotechnology company's stock after buying an additional 17,579 shares during the period. Mirae Asset Global Investments Co. Ltd. owned approximately 0.06% of Anavex Life Sciences worth $443,000 at the end of the most recent reporting period.
Other hedge funds and other institutional investors have also made changes to their positions in the company. Arizona State Retirement System increased its holdings in shares of Anavex Life Sciences by 4.5% in the fourth quarter. Arizona State Retirement System now owns 21,993 shares of the biotechnology company's stock valued at $204,000 after buying an additional 957 shares in the last quarter. Legal & General Group Plc raised its stake in Anavex Life Sciences by 3.2% during the 4th quarter. Legal & General Group Plc now owns 36,150 shares of the biotechnology company's stock valued at $335,000 after purchasing an additional 1,111 shares during the period. ProShare Advisors LLC raised its stake in Anavex Life Sciences by 8.2% during the 4th quarter. ProShare Advisors LLC now owns 15,345 shares of the biotechnology company's stock valued at $142,000 after purchasing an additional 1,165 shares during the period. Raymond James Financial Services Advisors Inc. raised its stake in Anavex Life Sciences by 6.4% during the 1st quarter. Raymond James Financial Services Advisors Inc. now owns 25,770 shares of the biotechnology company's stock valued at $317,000 after purchasing an additional 1,561 shares during the period. Finally, Principal Financial Group Inc. raised its stake in Anavex Life Sciences by 10.5% during the 4th quarter. Principal Financial Group Inc. now owns 20,704 shares of the biotechnology company's stock valued at $192,000 after purchasing an additional 1,962 shares during the period. 33.42% of the stock is currently owned by institutional investors and hedge funds.
Thanks to imatrader65 on ST for the link,
https://www.marketbeat.com/instant-alerts/nasdaq-avxl-sec-filing-2023-08-21/
"The first Alzheimer’s drugs meant to slow the progression of the fatal disease may only be available to a tiny fraction of patients, a study published Wednesday in the journal Neurology suggests."
"The drugs, Eisai’s Leqembi and Biogen’s Aduhelm, are approved for older adults with mild cognitive impairment or early-stage Alzheimer’s disease but, according to the study, less than 1 in 10 patients at this point in the disease may be prescribed them."
https://www.nbcnews.com/news/amp/rcna100011
I predict that the less than 1 in 10 will become 0 in 10 once 2-73 Blarcamesine is approved and in the market for one year. Biogen will pull the drug because of adverse side effects, IV injections at the doctors office, low efficacy, and brain scans monitoring for bleeding and brain swelling; because of all those reasons it will be driving what few patients they do treat over to 2-73 Blarcamesine.
Biogen will eventually pull their AD drugs when the revenue is so low that it cannot justify the cost to run the program.
10,000+ Distinct types of Rare and Genetic Diseases.
400 Million People Suffer From a Rare Disease Globally.
1 of 2 Patients Diagnosed with a Rare Disease is a Child.
3 of 10 Children with a Rare Disease Won’t Live to See Their 5th Birthday.
Anavex specializes in Rare Diseases.
(Thx sokol for the good news)
https://globalgenes.org/?gclid=CjwKCAjw5_GmBhBIEiwA5QSMxECJqixb15LMHqL9rMs42Aivft45UErC_A6a1CPgg3Ipyy4tGgMhxhoCTAEQAvD_BwE
I like this figure boi , and if it is somewhat close we have a Lunar Mission in the works:
"Here, again using public information from Anavex (plus arithmetic) -- and a couple of assumptions from Peter Karol's blog and a 12x post analyzing the December results -- I can both confirm my original analysis and determine the "Super Responder" n as 70."
+One Million Vol.
Closing Volume - 3,071,688
Lets see if this run continues on high vol; way overdue IMO.
Best post of the week so far:
https://investorshub.advfn.com/boards/read_msg.aspx?message_id=172500290
"dedicated to the development of novel therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders, including Alzheimer's disease, Parkinson's disease, Rett syndrome, and other central nervous system (CNS) diseases, pain, and various types of cancer."
I find it odd that recently "various types of cancer' is in the PRs, when there has not been any known activity; yet diseases like Fragile X and Frontotemporal Dementia (FTD) that are in the works are not mentioned.
Makes me wonder if there is a little fire behind that smoke? Maybe mice being given various types of cancer for testing?
https://www.anavex.com/post/anavex-life-sciences-to-present-at-the-btig-virtual-biotechnology-conference-2023?mc_cid=8c880e0384&mc_eid=74368b19ab
Moon Man, that guy flip flops and jams the ST board with BS like this, and is not worth ever quoting.
Settlement Date Short Interest Percent Change Average Daily Share Volume Days to Cover
06/30/2023 16,739,768 (5.64) 1,148,109 14.58
06/15/2023 17,739,562 7.45 1,165,885 15.22
05/31/2023 16,508,942 3.82 763,358 21.63
05/15/2023 15,901,191 10.58 937,267 16.97
04/28/2023 14,379,183 (7.80) 798,137 18.02
04/14/2023 15,594,970 (1.84) 981,502 15.89
03/31/2023 15,887,617 1.34 1,275,882 12.45
03/15/2023 15,677,855 (3.93) 1,171,753 13.38
http://nasdaqtrader.com/Trader.aspx?id=ShortInterest
With a strong PR, shorts in a squeeze heading for the exit door will require waiting to get through the key hole.
Powerwalker, this was from the Pub Med abstract with a pay wall that Mayo bought.
The data is from the 2-73-RS-001 trial, which was low dose, and that bodes extremely well for the two Rett trials that followed.
https://pubmed.ncbi.nlm.nih.gov/37429704/
Best part:
From Mayo's post on ST in the reply section: ...effects were measured using Cohens D. Effect sizes being the ‘impact’ of the therapy. A drug could have statistically significant results with a low effect size. What you want is a high effect size (impact) with statistical significance.
0.2 would equate to a low effect size
0.5 would equate to a medium effect size
0.8 would equate to a large effect size
Anything over about 1.1 would be extremely large.
The Rett patients with S1R WT genes (our preferred patient) had effect sizes in excess of 1.8
You read that correctly.
(( What is even better is that this was the first Rett trial, the adult 'low dose'. Extrapolate that to the higher dose 2nd Adult trial; and especially to the pediatric 'Excellent Rett' trial that should blow away the adult trials.))