Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
You're equating "the Street" with those entities that keep manipulating the price down.
That is NOT a good comparison.
All you're doing is creating another false narrative.
I had time to reflect on your post and wondered if you were aware that Deep Track Capital invested $50 Million in Anavex back in June, 2021? (They executed this deal at $21/share with far less data than today)
Anavex Life Sciences Announces $50 Million Registered Direct Offering
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) disorders, today announced that it has entered into a definitive purchase agreement with Deep Track Capital for the issuance and sale of an aggregate of 2,380,953 shares of its common stock at a purchase price of $21.00 per share of common stock a registered direct offering. The registered direct offering is expected to close on or about June 24, 2021, subject to the satisfaction of customary closing conditions.
H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.
The gross proceeds from the offering are expected to be approximately $50 million before deducting placement agent fees and other offering expenses. Anavex currently intends to use the net proceeds from the offering for advancing its pipeline and for working capital and general corporate purposes.
The shares of common stock described above are being offered pursuant to Anavex’s shelf registration statement on Form S-3 (File No. 333-232550) filed with the Securities and Exchange Commission (the “SEC”) on July 3, 2019 and declared effective on July 15, 2019. Such shares of common stock may be offered only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and the accompanying prospectus relating to the shares of common stock being offered in the registered direct offering will be filed with the SEC. Electronic copies of the final prospectus supplement and the accompanying prospectus may be obtained, when available, on the SEC’s website at http://www.sec.gov or by contacting H.C. Wainwright & Co., LLC, 430 Park Avenue, 3rd Floor, New York, NY 10022, by e-mail: placements@hcwco.com or by telephone: (212) 856-5711.
This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of any of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of such jurisdiction.
About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of 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, pain and various types of cancer. Anavex’s lead drug candidate, ANAVEX®2-73 (blarcamesine), successfully completed a Phase 2a clinical trial for Alzheimer’s disease and recently a Phase 2 proof-of-concept study in Parkinson’s disease dementia and a Phase 2 study in adult patients with Rett syndrome. ANAVEX®2-73 is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer’s disease. ANAVEX®2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson’s Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX®2-73 for the treatment of Parkinson’s disease. ANAVEX®3-71, which targets sigma-1 and muscarinic receptors, is a promising clinical stage drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer’s disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid and tau pathologies. In preclinical trials, ANAVEX®3-71 has shown beneficial effects on mitochondrial dysfunction and neuroinflammation. Further information is available at www.anavex.com. You can also connect with the company on Twitter, Facebook, Instagram and LinkedIn.
Forward-Looking Statements
Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks set forth in the Company’s most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Anavex Life Sciences Corp. undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.
https://www.anavex.com/post/anavex-life-sciences-announces-50-million-registered-direct-offering-1
Obviously you are fairly certain that the P2b/3 AD trial did not meet both endpoints...based on the data that's been presented to the public (so far).
However, can you play devil's advocate and assume that Dr. Missling is correct that both endpoints have been met...and give us the best possible scenario that would validate Dr. Missling's claim?
Thanks.
And don't forget this PR from 2022 that talks about the genomic analysis.
Anavex Announces First Entire Clinical Alzheimer’s Gene Pathway Data of ANAVEX®2-73 at AAIC 2022
Expression levels of pathological dysregulated neurodegenerative genes of both Alzheimer’s and Parkinson’s disease were significantly restored by the therapeutic effect of ANAVEX®2-73 (p<0.005)
These findings will facilitate contextualization of upcoming readout of ANAVEX®2-73 Phase 2b/3 Alzheimer’s disease clinical trial
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 announced the first entire clinical gene pathway data from the ANAVEX®2-73-PDD-001 Parkinson’s Disease Dementia (PDD) study at the Alzheimer’s Association International Conference (AAIC), taking place in San Diego, CA and virtually on July 31 – August 4, 2022.
The poster presentation titled, “Study of the mechanism of action of Blarcamesine (ANAVEX®2-73): Whole blood transcriptomics analysis (RNAseq) identifies treatment impact on compensatory pathways by restoring key neurodegenerative pathways functionality, including Alzheimer’s and Parkinson’s disease pathways” is being presented by the principal author, Dr. Mohammad Afshar, MD, PhD, Ariana Pharma, Paris, France and Cambridge, US.
Randomized, placebo-controlled clinical trial in 132 patients with Parkinson’s disease dementia (PDD) included prespecified biomarkers of response as well as Whole Exome Sequencing DNA data and full RNA exome expression data collection.
ANAVEX®2-73 transcriptomics analysis (RNAseq) identified a gene network that is differentially expressed in Parkinson’s disease dementia (PDD) patients treated with ANAVEX®2-73 compared to placebo after 14 weeks of treatment. The expression of 14,150 genes were analyzed from both placebo and ANAVEX®2-73 treated patients. Biological relevance of this gene network was assessed through pathway analysis and confirmed the impact of ANAVEX®2-73 treatment on pathways involved in neurodegenerative diseases, especially Alzheimer’s disease and Parkinson’s disease.
While genes are known to be down-regulated in Alzheimer’s disease [1] and Parkinson’s disease [2], representing pathology for these diseases, ANAVEX®2-73 singularly impacted expression levels of these genes in multiple pathways by countering the pathological down-regulation of genes in both Alzheimer’s (p<0.005) and Parkinson’s disease (p<0.005) and other degenerative diseases (p<0.005).
The scope of these detected gene expressions identified through ANAVEX®2-73 effect may represent additional potential biomarkers of disease pathology and response.
Previously, this study demonstrated dose-dependent, statistically significant improvement of dementia assessment, Quality of Episodic Memory with ANAVEX®2-73 (p=0.003) as well as significant improvement of Parkinson’s assessment, MDS-UPDRS Total score (p=0.034), in patients treated with ANAVEX®2-73 high oral dose once daily during the 14-week trial. SIGMAR1 mRNA expression significantly increased in ANAVEX®2-73-treated patients vs placebo (p=0.035) over the course of treatment and was significantly associated with improvements of MDS-UPDRS scores and cognitive efficacy endpoints CDR system. [3]
Dr. Jaime Kulisevsky, MD, PhD, Principal Investigator of the trial, commented, "To my knowledge, this represents the first extensive transcriptomics analysis (RNAseq) of a therapeutic agent in patients with Parkinson’s disease dementia (PDD). It is very intriguing to confirm this robust correlation of the clinical improvements of motor impairment (MDS-UPDRS) and cognition (CDR system) with compensation of expression levels of dysregulated neurodegenerative genes, especially Alzheimer’s disease and Parkinson’s disease, through the therapeutic effect of ANAVEX®2-73. PDD is a debilitating disorder with significant co-morbidities and there has not been a mechanistically novel medication approved for PDD in over 20 years. Hence, new therapies are urgently needed to alleviate this suffering and disability.”
Christopher U Missling, PhD, President & Chief Executive Officer of Anavex, stated, "It is exciting to witness ANAVEX®2-73’s (blarcamesine) demonstration of its platform Precision Medicine potential for both Alzheimer’s disease and Parkinson’s disease, and likely other neurodegenerative diseases. We believe these results will facilitate contextualization of upcoming readout of ANAVEX®2-73 Phase 2b/3 Alzheimer’s disease clinical trial and further supports pivotal studies in Parkinson’s disease and Parkinson’s disease dementia. We would like to thank all the patients and participating families as well the investigators and clinical site coordinators for their dedication to this study."
The presentation of the Abstract #59024 is available on the Anavex website (www.anavex.com).
About ANAVEX®2-73-PDD-001 Clinical Study
The ANAVEX®2-73-PDD-001 study was an international, double-blind, multicenter, placebo-controlled proof of concept Phase 2 clinical study that randomized 132 patients with Parkinson’s disease dementia (PDD) equally (ratio of 1:1:1) to target doses of 30 mg, 50 mg ANAVEX®2-73 or placebo, respectively. As previously reported, in addition to prespecified ANAVEX®2-73-related biomarker of response, SIGMAR1, safety and cognitive efficacy, MDS-UPDRS, actigraphy and sleep function was assessed during the study duration of 14 weeks.
Study inclusion required diagnosis of idiopathic Parkinson's disease (PD) consistent with the UK Parkinson's Disease Society Brain Bank diagnostic criteria and diagnosis of probable PD dementia (PDD) according to the Movement Disorder Society Task Force clinical diagnostic criteria as well as Montreal Cognitive Assessment (MoCA) score of 13 to 23. DNA and RNA from blood samples were analyzed using NGS.
Study participants were allowed to be on stable regimen of anti-Parkinson's disease medications (including levodopa, dopamine agonists, MAO-B inhibitors, or entacapone). Treatment with cholinesterase inhibitors, rivastigmine, donepezil and galantamine (Exelon®, Aricept®, or Reminyl®) were also permitted.
The study found that ANAVEX®2-73 was well tolerated in oral doses up to 50 mg once daily. The results showed clinically meaningful, dose-dependent, and statistically significant improvements in the Cognitive Drug Research (CDR) computerized assessment system analysis. The study validated the precision medicine approach of targeting SIGMAR1 as a genetic biomarker of response to ANAVEX®2-73, confirming that ANAVEX®2-73 acts through SIGMAR1 activation.
Broad and statistically significant improvements in CDR system Cognitive Domain of Attention assessed by Choice Reaction Time (p = 0.039) and Digital Vigilance (p = 0.008) and CDR system Episodic Memory (p = 0.047), representing complex cognitive tasks with impact on quality of life such as making a choice between similar objects and remembering daily personal experiences, which are mostly impaired in both PD and AD. [4] [4]
Statistically significant dose-dependent (p = 0.003) improvement of Episodic Memory, which has been shown to be highly correlated (70%) with the Alzheimer’s Disease Assessment Scale–Cognitive score (ADAS-Cog; r = 0.7). [5]
ANAVEX®2-73 does not impair sleep and has a positive effect on REM sleep behavior disorder.
ANAVEX®2-73 was generally safe, well tolerated, and improved safety profile compared to dementia drugs associated with typical adverse effects.
After completing the ANAVEX®2-73-PDD-001 trial, participants were able to enroll in a voluntary 48-week open-label extension study, ANAVEX®2-73-PDD-EP-001, which continued to assess safety, long term efficacy and changes in gut microbiota. [6]
About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company 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. Anavex’s lead drug candidate, ANAVEX®2-73 (blarcamesine), has successfully completed a Phase 2a clinical trial for Alzheimer’s disease, a Phase 2 proof-of-concept study in Parkinson’s disease dementia and both a Phase 2 and a Phase 3 study in adult patients with Rett syndrome. ANAVEX®2-73 is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer’s disease. ANAVEX®2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective, and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson’s Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX®2-73 for the treatment of Parkinson’s disease. ANAVEX®3-71, which targets sigma-1 and muscarinic M1 receptors, is a promising clinical stage drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer’s disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid, and tau pathologies. In preclinical trials, ANAVEX®3-71 has shown beneficial effects on mitochondrial dysfunction and neuroinflammation. Further information is available at www.anavex.com. You can also connect with the company on Twitter, Facebook, Instagram and LinkedIn.
Forward-Looking Statements
Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks set forth in the Company’s most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Anavex Life Sciences Corp. undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.
https://www.anavex.com/post/anavex-announces-first-entire-clinical-alzheimer-s-gene-pathway-data-of-anavex-2-73-at-aaic-2022
Makes sense. Thanks
Very rare to see AVXL in the green while the XBI is in the red.
Maybe the shorts are tired of selling shares to the Institutions...as they gobble up everything below $8.
In any event, AVXL is close to hitting $8 now.
Yes. I agree it was at a higher level. But he specifically stated that "the indices were in an uptrend" the week after the OLE was announced...which was false.
And thus, I pointing out that the XBI finished that week lower than it started.
No need to kick this dead horse again.
Yup. That's exactly why I posted the PR from 2018.
This should give us a better feel for what is in the SAP too, no?
****************************************************************************************************************
abew4me post# 412170
Monday, April 24, 2023
Artificial Intelligence enables first biomarker driven precision medicine therapy against Alzheimer
September, 2018
Ariana Pharma helps ANAVEX® demonstrate efficacy of its Alzheimer’s therapy in phase 2a clinical trial.
Ariana’s KEM® Artificial Intelligence technology has resulted in the identification of the first actionable genetic variant biomarkers, selecting a specified Alzheimer’s disease population, who demonstrated a confirmed response with ANAVEX®2-73.
These results provide support for a faster precision medicine clinical development of ANAVEX®2-73 focusing on the right patients who could benefit from the drug.
“This is the first full genomic analysis of ANAVEX®2-73 in Alzheimer’s disease which resulted in the identification of actionable genetic variants, bringing us one step closer to realizing the full potential of a precision medicine and precision pharmacology approach to treating this devastating disease,” Professor Harald Hampel, M.D., Ph.D., MA, MSc, AXA Research Fund & Sorbonne University Excellence Chair, Department of Neurology, Sorbonne University, Paris.
“The innovative study findings based on ANAVEX®2-73 moves precision medicine and pharmacology a step closer in Alzheimer’s therapy trials,” Christopher U. Missling, Ph.D., President and Chief Executive Officer of Anavex.
“… in CNS-based studies, whether AD or MS, the data are complex across multiple endpoints. As such, having KEM® system with Ariana in place to help analyze complex data is, in our view, a strategic positive for Anavex.” Jason Kolbert, former Executive Managing Director and Head of Healthcare Research at Maxim Group, Managing Director of Research at HC Wainwright & Co.
Identifying the right patient population, the use of Ariana’s proprietary KEM® AI technology has a proven track of increasing the chances of success, accelerating clinical timelines and creating immediate value for the Company.
KEM® is a comprehensive and FDA-tested clinical data analysis system that enables full exploitation of complex datasets. It has uniquely demonstrated its ability to extract biomarkers from small sets of patients.
Beyond CNS, Ariana’s expertise spans multiple medical indications and therapeutic areas, including cancer, metabolic and immunological diseases.
Further reading
Anavex presents results at CTAD, Barcelona (October 2018)
PRESENTATION – Longitudinal 148-Week Extension Study for ANAVEX®2-73 Phase 2a Alzheimer’s Disease Demonstrates Maintained Activities of Daily Living Score (ADCS-ADL) and Reduced Cognitive Decline (MMSE) for Patient Cohort on Higher Drug Concentration and Confirms Role of Patient Selection Biomarkers [Link: Document]
Biomarker data presented at AAIC, Chicago (July 2018)
Anavex Life Sciences Presents New Data Identifying Treatment Response Biomarkers in Alzheimer’s Disease Patients Treated with Investigational ANAVEX®2-73 at 2018 Alzheimer’s Association International Conference (AAIC) Read more
ABSTRACT – Full Genomic Analysis of ANAVEX®2-73 Phase 2a Alzheimer’s Disease Study Identifies Biomarkers Enabling Targeted Therapy and a Precision Medicine Approach [Link: Document]
ABSTRACT – Systematic Processing of Full Genomic Analysis of ANAVEX®2-73 Phase 2a Alzheimer’s Disease Study Identifies Biomarkers Enabling a Precision Medicine Approach [Link: Document]
POSTER – Full Genomic Analysis of ANAVEX®2-73 Phase 2a Alzheimer’s Disease Study Identifies Biomarkers Enabling Targeted Therapy and a Precision Medicine Approach [Link: Document]
PRESENTATION – Systematic Processing of Full Genomic Analysis of ANAVEX®2-73 Phase 2a Alzheimer’s Disease Study Identifies Biomarkers Enabling a Precision Medicine Approach [Link: Document]
NASDAQ – Anavex Life Sciences Presents New Data Identifying Treatment Response Biomarkers in Alzheimer’s Disease Patients Treated with Investigational ANAVEX®2-73 at 2018 Alzheimer’s Association International Conference (AAIC) Read more
Mohammad Afshar presents results at CTAD, Boston (November 2017)
Anavex Life Sciences Reports PK and PD Data from Phase 2a Trial of ANAVEX®2-73 in Mild-to-Moderate Alzheimer’s Disease Patients Read more
ABSTRACT – Clinical pharmacokinetics and pharmacodynamics characterization of ANAVEX™2-73 for designing a phase 2/3 study in mild-to-moderate Alzheimer’s disease [Link: Document]
PRESENTATION – Clinical Pharmacokine/cs and Pharmacodynamics Characteriza/on of ANAVEX®2-73 for Designing a Phase 2/3 Study in Mild-to-Moderate Alzheimer’s Disease – CTAD November 2017 [Link: Document]
Initiating collaboration (October 2016)
Anavex Life Sciences and Ariana Pharma Collaborate to Accelerate Timelines and Improve Efficiency of Alzheimer’s and Parkinson’s Clinical Development Programs Read more
NASDAQ – Anavex Life Sciences (AVXL) was up 9,3% after teaming up with Ariana Pharma [Link: Document]
https://www.arianapharma.com/artificial-intelligence-enables-first-biomarker-driven-precision-medicine-therapy-against-alzheimer/
LOL...I'm the one that has proven my points with hard data by giving the daily closing prices for the XBI.
I understand that you're upset with AVXL's low price...but all of the biotech companies are scraping the bottom right now.
There's no need to create a false narrative that AVXL is in a downtrend specifically because of the OLE results. That is simply not true.
Wrong again. The indices were not in an uptrend as you claim. The week after the OLE announcement, the XBI looked like this...
Monday..........77.06
Tuesday.........75.57 (Which is a huge drop...and AVXL's share price followed the same path)
Wednesday....75.38 (Closed even lower...and so did AVXL)
Thursday........76.87 (Closed lower than Monday)
Friday............(Good Friday)
So, the XBI closed DOWN for the week...which contradicts your analysis that the indices were in an uptrend.
This is how false narratives are created where there isn't any.
Especially if the FDA is still using Ariana's KEM technology to evaluate and analyze the data. (See below)
FDA taps Ariana KEM platform for biomarker signature analysis
September 9th, 2010
The US Food and Drug Administration is collaborating with Ariana Pharma, a provider of decision support software for pharmaceutical discovery, development and safety, in the agency’s efforts to improve the analysis of genomic data in support of personalised medicine.
Under the collaboration, Ariana is providing its KEM biomarker technology to help FDA reviewers analyse pharmacogenomic data and patient characteristics for biomarker signatures filed through the agency’s Voluntary Exploratory Data Submission (VXDS) programme.
As things stand, the FDA notes, most pharmacogenomic data are “of an exploratory or research nature”. Accordingly, the agency’s regulations do not require the inclusion of these data in investigational new drug application (INDs), nor do they ask for complete reports on pharmacogenomic data in new drug applications (NDAs) or biologic license applications (BLAs).
Nonetheless, the FDA adds, voluntary submissions “can benefit both the industry and the FDA in a general way by providing a means for sponsors to ensure that regulatory scientists are familiar with and prepared to appropriately evaluate future genomic submissions”.
Launched in 2003 and based in central Paris, France, Ariana Pharma is a spin-off from the Institut Pasteur. Its KEM (Knowledge Management and Extraction) platform is a proprietary decision-support technology for the rapid analysis of parametric/multi-objective data, with applications ranging from drug discovery to optimisation of design and outcomes in clinical trials and early signal detection for drug safety.
According to Ariana, the KEM platform can cut the risk rate in selecting panels for biomarker development from 40% to 10%, potentially saving “billions of dollars”. The technology will help the FDA to identify systematically potential genomic ‘fingerprints’ and to develop recommendations for the analysis of genomic data prior to the submission of biomarker signatures through the VXDS programme, the company said.
https://www.pharmatimes.com/news/fda_taps_ariana_kem_platform_for_biomarker_signature_analysis_982469
No. You're wrong. AVXL's price ended the week at its highest point after he announced the PDD OLE results...which were announced on Thursday.
Monday...........$8.51
Tuesday..........$8.32
Wednesday.....$8.56
*Thursday.........$8.32
Friday..............$8.57
Nothing "cute" about it except the obvious manipulation downward on Thursday after the announcement.
If it was such a failure, the price would've continued downward on Friday. Instead, it closed at its highest point for the entire week.
Can you provide a link to that quote?
Thanks
Artificial Intelligence enables first biomarker driven precision medicine therapy against Alzheimer
September, 2018
Ariana Pharma helps ANAVEX® demonstrate efficacy of its Alzheimer’s therapy in phase 2a clinical trial.
Ariana’s KEM® Artificial Intelligence technology has resulted in the identification of the first actionable genetic variant biomarkers, selecting a specified Alzheimer’s disease population, who demonstrated a confirmed response with ANAVEX®2-73.
These results provide support for a faster precision medicine clinical development of ANAVEX®2-73 focusing on the right patients who could benefit from the drug.
“This is the first full genomic analysis of ANAVEX®2-73 in Alzheimer’s disease which resulted in the identification of actionable genetic variants, bringing us one step closer to realizing the full potential of a precision medicine and precision pharmacology approach to treating this devastating disease,” Professor Harald Hampel, M.D., Ph.D., MA, MSc, AXA Research Fund & Sorbonne University Excellence Chair, Department of Neurology, Sorbonne University, Paris.
“The innovative study findings based on ANAVEX®2-73 moves precision medicine and pharmacology a step closer in Alzheimer’s therapy trials,” Christopher U. Missling, Ph.D., President and Chief Executive Officer of Anavex.
“… in CNS-based studies, whether AD or MS, the data are complex across multiple endpoints. As such, having KEM® system with Ariana in place to help analyze complex data is, in our view, a strategic positive for Anavex.” Jason Kolbert, former Executive Managing Director and Head of Healthcare Research at Maxim Group, Managing Director of Research at HC Wainwright & Co.
Identifying the right patient population, the use of Ariana’s proprietary KEM® AI technology has a proven track of increasing the chances of success, accelerating clinical timelines and creating immediate value for the Company.
KEM® is a comprehensive and FDA-tested clinical data analysis system that enables full exploitation of complex datasets. It has uniquely demonstrated its ability to extract biomarkers from small sets of patients.
Beyond CNS, Ariana’s expertise spans multiple medical indications and therapeutic areas, including cancer, metabolic and immunological diseases.
Further reading
Anavex presents results at CTAD, Barcelona (October 2018)
PRESENTATION – Longitudinal 148-Week Extension Study for ANAVEX®2-73 Phase 2a Alzheimer’s Disease Demonstrates Maintained Activities of Daily Living Score (ADCS-ADL) and Reduced Cognitive Decline (MMSE) for Patient Cohort on Higher Drug Concentration and Confirms Role of Patient Selection Biomarkers [Link: Document]
Biomarker data presented at AAIC, Chicago (July 2018)
Anavex Life Sciences Presents New Data Identifying Treatment Response Biomarkers in Alzheimer’s Disease Patients Treated with Investigational ANAVEX®2-73 at 2018 Alzheimer’s Association International Conference (AAIC) Read more
ABSTRACT – Full Genomic Analysis of ANAVEX®2-73 Phase 2a Alzheimer’s Disease Study Identifies Biomarkers Enabling Targeted Therapy and a Precision Medicine Approach [Link: Document]
ABSTRACT – Systematic Processing of Full Genomic Analysis of ANAVEX®2-73 Phase 2a Alzheimer’s Disease Study Identifies Biomarkers Enabling a Precision Medicine Approach [Link: Document]
POSTER – Full Genomic Analysis of ANAVEX®2-73 Phase 2a Alzheimer’s Disease Study Identifies Biomarkers Enabling Targeted Therapy and a Precision Medicine Approach [Link: Document]
PRESENTATION – Systematic Processing of Full Genomic Analysis of ANAVEX®2-73 Phase 2a Alzheimer’s Disease Study Identifies Biomarkers Enabling a Precision Medicine Approach [Link: Document]
NASDAQ – Anavex Life Sciences Presents New Data Identifying Treatment Response Biomarkers in Alzheimer’s Disease Patients Treated with Investigational ANAVEX®2-73 at 2018 Alzheimer’s Association International Conference (AAIC) Read more
Mohammad Afshar presents results at CTAD, Boston (November 2017)
Anavex Life Sciences Reports PK and PD Data from Phase 2a Trial of ANAVEX®2-73 in Mild-to-Moderate Alzheimer’s Disease Patients Read more
ABSTRACT – Clinical pharmacokinetics and pharmacodynamics characterization of ANAVEX™2-73 for designing a phase 2/3 study in mild-to-moderate Alzheimer’s disease [Link: Document]
PRESENTATION – Clinical Pharmacokine/cs and Pharmacodynamics Characteriza/on of ANAVEX®2-73 for Designing a Phase 2/3 Study in Mild-to-Moderate Alzheimer’s Disease – CTAD November 2017 [Link: Document]
Initiating collaboration (October 2016)
Anavex Life Sciences and Ariana Pharma Collaborate to Accelerate Timelines and Improve Efficiency of Alzheimer’s and Parkinson’s Clinical Development Programs Read more
NASDAQ – Anavex Life Sciences (AVXL) was up 9,3% after teaming up with Ariana Pharma [Link: Document]
https://www.arianapharma.com/artificial-intelligence-enables-first-biomarker-driven-precision-medicine-therapy-against-alzheimer/
Back in 2010, the FDA selected Ariana's KEM software to analyze biomarkers. Does anyone know if they are still using it?
FDA taps Ariana KEM platform for biomarker signature analysis
September 9th, 2010
The US Food and Drug Administration is collaborating with Ariana Pharma, a provider of decision support software for pharmaceutical discovery, development and safety, in the agency’s efforts to improve the analysis of genomic data in support of personalised medicine.
Under the collaboration, Ariana is providing its KEM biomarker technology to help FDA reviewers analyse pharmacogenomic data and patient characteristics for biomarker signatures filed through the agency’s Voluntary Exploratory Data Submission (VXDS) programme.
As things stand, the FDA notes, most pharmacogenomic data are “of an exploratory or research nature”. Accordingly, the agency’s regulations do not require the inclusion of these data in investigational new drug application (INDs), nor do they ask for complete reports on pharmacogenomic data in new drug applications (NDAs) or biologic license applications (BLAs).
Nonetheless, the FDA adds, voluntary submissions “can benefit both the industry and the FDA in a general way by providing a means for sponsors to ensure that regulatory scientists are familiar with and prepared to appropriately evaluate future genomic submissions”.
Launched in 2003 and based in central Paris, France, Ariana Pharma is a spin-off from the Institut Pasteur. Its KEM (Knowledge Management and Extraction) platform is a proprietary decision-support technology for the rapid analysis of parametric/multi-objective data, with applications ranging from drug discovery to optimisation of design and outcomes in clinical trials and early signal detection for drug safety.
According to Ariana, the KEM platform can cut the risk rate in selecting panels for biomarker development from 40% to 10%, potentially saving “billions of dollars”. The technology will help the FDA to identify systematically potential genomic ‘fingerprints’ and to develop recommendations for the analysis of genomic data prior to the submission of biomarker signatures through the VXDS programme, the company said.
https://www.pharmatimes.com/news/fda_taps_ariana_kem_platform_for_biomarker_signature_analysis_982469
Joseph, you make some good points but it comes down to whether you believe the company has the supportive data or not. I think we'll have an updated TLR by the time the ASM starts on May 23rd.
And, of course, this begs the question...if you didn't believe they have the supportive data, why are you invested "deep" in AVXL shares?
Just curious...
Yup. It's one thing to build a company from scratch, but Dr. Missling took Anavex from near bankruptcy to where it is now...which is pre-commercialization.
The next time someone complains about the number of options Dr. Missling has, I'll point to this quote from BDI below.
"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
You're kidding, right?
How can a biotech company generate revenue when it takes approximately 10+ years to get a drug approved?
Dr. Missling is approaching his 10 year anniversary here and A2-73 should be approved within the next 6 - 12 months...which is the norm for any new drug.
If you can't understand that, then you shouldn't be invested in biotech. It's that simple.
Now you know why Dr. Missling has my respect. He took Anavex from the brink of bankruptcy and has built it into a company on the brink of commercialization!
And don't forget that he simultaneously built the employee staff from 1 to 39 employees.
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 + Dr. Jun Kin = 39 employees
https://www.macrotrends.net/stocks/charts/AVXL/anavex-life-sciences/number-of-employees
Yes. Anavex was in deep financial trouble when Dr. Missling took over. Below is a statement from their 10k back in 2013.
"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
Compare that to today's financial condition that the company has no debt and over $143 million in the bank...is indeed a financial accomplishment!
Unless they are focusing on a specific type of epilepsy. Then it could be categorized as a rare disease. (See below)
*******************************************************************************************************************
Experts now divide epilepsy into four basic types based on the seizures you're having:
Generalized epilepsy
Focal epilepsy
Generalized and focal epilepsy
Unknown if generalized or focal epilepsy
My wife suffers from Temporal Lobe Epilepsy (TLE) which falls into the Focal category...because it only affects one side of the brain.
https://www.webmd.com/epilepsy/guide/types-epilepsy
Can they present the updated slides without jeopardizing the peer-reviewed submission? Or do they have to wait until after the new information is published?
If this post is correct, then it explains why Anavex took down all of the the slide decks for the P2b/3 trial because they're still accumulating "new, updated information". (See the post below)
hnbadger
Friday, February 17, 2023
Post# of 404558
This is from AVXL IR:
The company is working on another
video and an upcoming press release
that will address many of the
questions that you asked. Moreover,
some of the answers will elaborate
on specifics, all of which we believe
will provide a thorough
understanding of our initiatives. At
this time some of your questions can
not be answered today, because new,
updated data will be available to us
in the near future, all of which we
would need to disseminate such
information in accordance with
Regulation FD - via press releases
and/or 8k filing, not through an
individual email or one-on-one
conversation.
Thanks 100fold from ST
Exactly. No one jumps aboard a sinking ship!
As a reminder, both Dr. Kun Jin and Dr. Jiong Ma accepted their positions after Anavex announced the P2b/3 results at CTAD (December 1st)...and was reiterated on the Quarterly CC (February 7th)...
and continues to be posted on the company's official website. (See below)
***************************************************************************************************************
ANAVEX®2-73 (BLARCAMESINE) PHASE 2B/3 STUDY MET PRIMARY AND KEY SECONDARY ENDPOINTS, SHOWING STATISTICALLY SIGNIFICANT REDUCTION OF CLINICAL DECLINE IN GLOBAL CLINICAL STUDY OF PATIENTS WITH EARLY ALZHEIMER’S DISEASE
- Robust, Statistically Significant and Clinically Meaningful Absolute Improvement in Cognitive Function as Measured by ADAS-Cog and ADCS-ADL
- Key Secondary Endpoint CDR-SB Also Met, Demonstrating Statistically Significant Results
- Plan to Meet with Regulatory Authorities to Determine Next Steps
NEW YORK –December 1, 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) disorders, today announced positive topline results from its Phase 2b/3 ANAVEX®2-73-AD-004 clinical trial of oral ANAVEX®2-73 (blarcamesine) for the treatment of mild cognitive impairment (MCI) due to Alzheimer’s disease (AD) and mild AD (collectively known as early AD).
ANAVEX®2-73 met the primary endpoints ADAS-Cog[1] and ADCS-ADL[2] and key secondary endpoint CDR-SB[3] with statistically significant results. Next step, in light of this data, is meeting with regulatory authorities to discuss this data in the context of ongoing development with an aim to bring this therapy to patients in Europe, Asia-Pacific, and the U.S.
ANAVEX®2-73 (blarcamesine) is an orally available, small-molecule activator of the sigma-1 receptor (SIGMAR1), which, data suggest, is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.[4]
ANAVEX®2-73-AD-004 was a randomized, double-blind, multicenter, placebo-controlled 509 patient Phase 2b/3 study (randomized 1:1:1 to mid or high dose of ANAVEX®2-73 or placebo), for the treatment of early Alzheimer’s disease over 48 weeks. Top line data will be presented later today in the late breaking oral communication presentation at the Clinical Trials on Alzheimer’s Disease (CTAD) Congress 2022, December 1, 2022, at 4:30pm PT in San Francisco, CA. Further analysis of the data remains ongoing, and the Company plans to submit the data for publication in a peer-reviewed medical journal. The open-label extension study ATTENTION-AD will continue to follow participants over a 96 week period.
ANAVEX®2-73 treatment met the primary endpoints and reduced clinical decline on the global cognitive and functional scales over 48 weeks in the analysis of the Intent-to-treat (ITT) population.
ANAVEX®2-73 demonstrated visible improvement in patients with Alzheimer’s disease. Patients treated with ANAVEX®2-73 were 84% more likely, to have improved cognition by ADAS-Cog score change of -0.50 points or better from baseline to end of treatment than patients on placebo, Odds Ratio = 1.84 (p = 0.015). On average, patients, who improved cognitively with ANAVEX®2-73 treatment, improved by ADAS-Cog cognition score of -4.03 points. ANAVEX®2-73 treatment was 167% more likely to improve function compared with placebo, at a clinically meaningful improvement of ADCS-ADL score change of +3.5 points or better, Odds Ratio = 2.67 (p=0.0255). This reflects a robust improved and clinically meaningful outcome in cognition and function from baseline.
Additionally, treatment with ANAVEX®2-73 statistically significantly reduced cognitive decline, measured with ADAS-Cog, compared to placebo at end of treatment by 45%, representing a treatment difference in mean score change of -1.85 points (p=0.033).
ANAVEX®2-73 treatment also met the secondary endpoint of reduction in clinical decline of cognition and function assessed by the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB) compared to placebo, by a treatment difference in mean score change of -0.42 points (p=0.040), representing 27% reduction in the ITT population.
ANAVEX®2-73 (blarcamesine) was generally safe and well tolerated. The incidence of treatment emergent adverse events (TEAEs) was similar in the active and placebo arms with dizziness being the most common TEAE. TEAEs ≥7.5% threshold were predominantly mild or moderate. No clinically significant changes in vital signs, laboratory values and ECG parameters in active and placebo arms were observed. Safety findings in the study were consistent with the known safety profile of ANAVEX®2-73.
In addition to safety and efficacy demonstrated on the primary and key secondary endpoints, a pre-specified analysis of patients without SIGMAR1 gene mutation provides further confidence of the robustness of the SIGMAR1 activation in the treatment of neurodegenerative diseases. Approximately 80% of the total worldwide population lack a SIGMAR1 gene mutation.[5] ANAVEX®2-73 was more efficacious in this pre-specified population. This effect is consistent with prior clinical trials of ANAVEX®2-73.[6]
“People living with Alzheimer’s disease desperately need new therapies and I am truly impressed with the outcome of this study, which demonstrated reversal of cognitive decline,” said A/Professor Stephen Macfarlane, FRANZCP, Head of Clinical Services at the Dementia Centre, HammondCare and Principal Investigator. “These results complement and are consistent with findings from the previously completed Alzheimer’s disease Phase 2a ANAVEX®2-73 trial, which also demonstrated therapeutic effect on cognition and function. ANAVEX®2-73 might be a very much needed solution for many patients with Alzheimer’s disease.”
“We are very pleased to see such positive clinical data in patients with Alzheimer’s disease, which is otherwise a progressive disease, thereby emphasizing the potentially significant implications these findings have for patients, caregivers, and healthcare systems worldwide,” said Edward R Hammond, MD, PhD, MPH, Chief Medical Officer of Anavex. “We intend to discuss these findings with regulatory authorities in the context of the ongoing clinical development of ANAVEX®2-73 in this indication, with the goal of providing a much-needed treatment to the millions of patients living with Alzheimer’s disease.”
“The successful results of the ANAVEX®2-73-AD-004 clinical trial would not be possible without the truly motivated and dedicated study participants, their families and caregivers and the clinical investigators around the world. We thank all the people involved in the study for their invaluable contributions,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “These clinical study findings confirm the robustness of the ANAVEX precision medicine platform, and we look forward to advancing ANAVEX®2-73 as a potential new treatment option for Alzheimer’s disease while we continue to focus on the effect of ANAVEX®2-73 leveraging this approach to drug development to provide intelligent solutions beyond many traditional neurology trials in disease areas with high unmet needs.”
Economic Burden of Alzheimer's Disease[7]
Alzheimer's disease is the most common cause of dementia and the fifth leading cause of death in adults older than 65 years. The estimated total healthcare costs for the treatment of Alzheimer's disease in 2020 were estimated at $305 billion, with the cost expected to increase to more than $1 trillion as the population ages. Most of the direct costs of care for Alzheimer's disease are attributed to skilled nursing care, home healthcare, and hospice care. Indirect costs of care, including quality of life and informal caregiving, are likely underestimated, and are associated with significant negative societal and personal burdens.
About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company 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. Anavex's lead drug candidate, ANAVEX®2-73 (blarcamesine), has successfully completed a Phase 2a clinical trial for Alzheimer's disease, a Phase 2 proof-of-concept study in Parkinson's disease dementia, and both a Phase 2 and a Phase 3 study in adult patients with Rett syndrome. ANAVEX®2-73 is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer's disease. ANAVEX®2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective, and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson's Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX®2-73 for the treatment of Parkinson's disease. ANAVEX®3-71, which targets sigma-1 and M1 muscarinic receptors, is a promising clinical stage drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer's disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid, and tau pathologies. In preclinical trials, ANAVEX®3-71 has shown beneficial effects on mitochondrial dysfunction and neuroinflammation. Further information is available at www.anavex.com. You can also connect with the company on Twitter, Facebook, Instagram, and LinkedIn.
Forward-Looking Statements
For Further Information:
Anavex Life Sciences Corp.
Research & Business Development
Toll-free: 1-844-689-3939
Email: info@anavex.com
Investors:
Andrew J. Barwicki
Investor Relations
Tel: 516-662-9461
Email: andrew@barwicki.com
[1] AD Assessment Scale-Cognitive subscale. ADAS-Cog is the most common cognitive assessment instrument used in AD clinical trials all over the world.
[2] AD Cooperative Study-Activities of Daily Living Scale. ADCS-ADL is the most common functional assessment instrument used in AD clinical trials all over the world.
[3] Clinical Dementia Rating-Sum of Boxes.
[4]Advances in Experimental Medicine and Biology Volume 964 (2017) Sigma Receptors: Their Role in Disease and as Therapeutic Targets.
[5] https://clinvarminer.genetics.utah.edu/variants-by-submitter/1012/gene/SIGMAR1/benign
[6] Hampel et al. A precision medicine framework using artificial intelligence for the identification and confirmation of genomic biomarkers of response to an Alzheimer’s disease therapy: Analysis of the blarcamesine (ANAVEX2-73) Phase 2a clinical study. Alzheimer’s Dement. 2020;00:1–14
[7] W Wong Economic Burden of Alzheimer Disease and Managed Care Considerations Am J Manag Care. 2020;26:S177-S183.
https://www.anavex.com/post/anavex-2-73-blarcamesine-phase-2b-3-study-met-primary-and-key-secondary-endpoints
Quote: "Thank you Old Mystic. Makes you wonder how much data comes out of that trial that supports our science? Just like the MIT paper that came out claiming it can reverse Alzheimer’s by blocking the Ckk5(?) gene in a preclinical study. I love how Dr. Missling recerred to this as a “flashy PR”
3:45 min: Dr. Missling notes that S1R-related research and understanding is at all-time-highs.
You're combining two different scenarios.
The rolling submission was for the Rett trials in which the adult Rett was filed first and the pediatric Excellence trial would be submitted later.
When he referenced that TGD wanted to wait until he had "all the data", it was in reference to all of the data for the Alzheimer's trial i.e. genome, etc...
If this post is correct, then it explains why Anavex hasn't filed for the NDA because they're still accumulating information...and it also explains why they took down all of the the slide decks for the P2b/3 trial...again, they're still accumulating new information. (See the post below)
hnbadger
Friday, February 17, 2023
Post# of 404558
This is from AVXL IR:
The company is working on another
video and an upcoming press release
that will address many of the
questions that you asked. Moreover,
some of the answers will elaborate
on specifics, all of which we believe
will provide a thorough
understanding of our initiatives. At
this time some of your questions can
not be answered today, because new,
updated data will be available to us
in the near future, all of which we
would need to disseminate such
information in accordance with
Regulation FD - via press releases
and/or 8k filing, not through an
individual email or one-on-one
conversation.
Thanks 100fold from ST
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 closer to 75% 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!
****************************************************************************************************************
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/
Hmm...since Dr. Jun Kin and Dr. Jiong Ma have accepted their senior positions as Vice President and Chairman of the Board (respectively), doesn't that automatically make them part of the WGT group?
(I mean, c'mon man they've obviously seen the data, right?)
FYI...found a picture of Dr. Jun Kin. Thought I'd add it to the post since someone posted a pic of Dr. Ma the other day. Wonder if those charts in the background show some additional data for the Phase 3 AD trial? (LOL)
http://images.angelnexus.com/wd/20080416-wd_pic1.jpg
Yes. I fully agree.
People forget that Dr. Missling is one of the largest shareholders (via options) in the company. He has the most to lose if things aren't running right...so I'm sure he's given his approval for this change.
Quote: "She was brought in by Missling a few years ago"
Do you have ANY information to prove that your statement is true and correct?
When she joined the BOD for Anavex a few years ago, Doc328 made an astute observation that Blackrock had just accumulated a large percentage of AVXL shares...and it was customary for Blackrock to request a BOD outsider.
Interesting that she is also a Kaufman fellow...which is a two year program. Also note that the founder (Ewing Marion Kaufman) made his fortune as a pharmaceutical entrepreneur.
(Click the link below for more details)
https://en.wikipedia.org/wiki/Kauffman_Fellows_Program
Thanks for the detailed answer. Much appreciated!
"Probably" isn't a very convincing answer.
If so, does that mean we have to conduct all new trials to prove it's safety and efficacious?
Yes. After hiring Dr. Kun Jin, I think Anavex has the expertise to speed things up...so I don't think the SH meeting on May 23rd is out of the question.
Dr. Jin will draw on his extensive experience, including recently as the Statistical Team Leader at the U.S. Food and Drug Administration (FDA). Dr. Jin provided statistical review coverage and expertise for neurological drug products for the Center for Drug Evaluation and Research (CDER), and performed timely and quality reviews of marketing applications, including New Drug Applications (NDA), Biologic License Applications (BLA), and Investigational New Drug (IND) applications. Under the leadership of Dr. Jin, the neuropharmacological statistical team has completed several hundred statistical reviews of NDAs, BLAs, and efficacy supplements.
https://www.anavex.com/post/anavex-life-sciences-appoints-former-fda-lead-neurology-statistician-as-vp-head-of-biostatistics
If the LPLV is April 30th, I think they have a good chance of getting the TLR finished by the SH meeting on May 23rd.
*********************************************************************
ANAVEX2-73-RS-003 is a Phase 2/3, Double-blind, Randomized, Placebo-controlled Safety and Efficacy Study in Pediatric Patients With RTT
Actual Study Start Date : July 1, 2020
Estimated Primary Completion Date : April 30, 2023 Last Participant Last Visit
Estimated Study Completion Date : June 1, 2023
https://clinicaltrials.gov/ct2/show/NCT04304482
I would also add the TLR of the P3 Rett EXCELLENCE trial to the list of possibilities prior to the SH Meeting.
LOL...sure, if that will help you sleep better at night.
And don't disregard the TLR for the P3 results from the Rett EXCELLENCE trial. We should have the LPLV by mid-May or sooner...so those results are in play.
Read my post at the beginning of this thread.
https://investorshub.advfn.com/boards/read_msg.aspx?message_id=171663988