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Quote: "This is very important and relevant guidance to address multiplicity. Until the last 5-6 years, almost all AD trials were in mild AD and with clinical rather than PET entry criteria. As the anti-amyloid drugs showed some activity in the mildest patients but no activity in the moderate patients, trials evolved to test either MCI (with proof of amyloid) only or Mild Ad / MCI (with proof of amyloid). ADAS-Cog is more sensitive to follow progression of mild AD than it is for MCI. Therefore, some studies the past 5 years have used CDR-SB (lecanemab among others as example) or iADRS (donanemab as only example). iADRS is basically the subset of ADAS-Cog found to be most sensitive for MCI and mild AD combined with the ADCS-iADL scale (a modified instrumental ADL for MCI and milder AD). Therefore the 2017 example for AD in the draft guidance became a poor example and not included in the final guidance to industry."
Good for you, Doc!
Is it fair to say that Anavex's (Dr. Jin's) newly released TLR for the 2b/3 results has a stronger chance of FDA approval than you initially thought?
Yes. I remember that. Well done, boi.
And correct me if I'm wrong, but statistically speaking, isn't one co-primary endpoint at .025 and the secondary also at .025 is just as strong as both co-primaries at .05 and the secondary at .05?
I saw an order to buy 60,000 shares at $6.97. (That's almost half a million dollars)
Nice to see the buyers are stepping in with big orders.
No. Not at all...because they continue to affirm that they've achieved ADCS-ADL on their website as per their "met all endpoints" position.
And frankly, I don't believe Dr. Kun Jin would allow today's Press Release without acknowledging that one of the endpoints didn't make it...if that was the case.
Secondly, we know that they've submitted the data for publication in a peer-reviewed journal...so it makes sense that an embargo would require them to withhold specific data from the public.
NO. They will wait until A2-73 is approved for Rett first, then repurpose the drug for AD. This will also allow for the OLE to be completed and expedite the entire NDA process for AD approval.
Anavex's website still shows that they met all end points. (see below)
ANAVEX®2-73 (blarcamesine) Meets Co-Primary and Key Secondary Endpoints for Patients with Early Alzheimer’s Disease
• ANAVEX®2-73 (blarcamesine) treatment slowed decline of cognition and function in patients with early Alzheimer’s disease over 48 weeks
• ANAVEX®2-73 (blarcamesine) treatment reduced cognitive decline by 45%, measured by ADAS-Cog, as compared to placebo, representing a treatment difference in mean score change of -1.85 points (p=0.033) over 48 weeks
• Patients treated with ANAVEX®2-73 had 1.84 times higher odds, or likelihood, to improve cognitively compared to placebo, with an ADAS-Cog score threshold of -0.5 points or better [Odds Ratio = 1.84 (p = 0.015)] and had 2.67 times higher odds, or likelihood, to improve function with an
ADCS-ADL score threshold of +3.5 points or better compared to placebo [Odds Ratio = 2.67 (p = 0.0255)]
• Compared to placebo, ANAVEX®2-73 (blarcamesine) reduced clinical decline of cognition and function by 27% with mean score difference of -0.42 points (p=0.040) as measured by CDR-SB
• ANAVEX®2-73 (blarcamesine) was generally safe and well tolerated Next Steps
• Publish full analyses, including MRI data, prespecified biomarkers of response as well as Whole Exome Sequencing DNA data and full mRNA exome expression data collection data on biomarkers of neurodegeneration in a peer-reviewed medical journal
• Continue open-label extension study ATTENTION-AD to follow participants over 96 weeks
• Meet with regulatory authorities to discuss data with aim to bring this therapy to patients in Europe, Asia-Pacific, and the U.S., including potential Accelerated Approval Pathway based on newly available preliminary efficacy results of surrogate biomarkers
Odds Ratio of ADAS-Cog meaningful improvement in cognition at threshold of -0.5 points or less (90% CI)
Improvement
-1 0 1 2 3
ODDS RATIO
Odds Ratio of ADCS-ADL meaningful improvement in function at threshold of +3.5 points or higher (90% CI)
P = 0.015
1.839 (1.17, 2.94)
-0.5 0
1 2
3 4 5 6
ODDS RATIO
Improvement
P = 0.0255
2.67 (1.17, 6.13)
17
https://www.anavex.com/_files/ugd/79bcf7_97da6311c3b643bea8e9a6659bb51422.pdf
And do you think that information was left out of the press release...
1) purposely?
2) negligently?
Choose wisely grasshopper!
Indeed...and to paraphrase TGD at the Annual Shareholder's Meeting: You will enjoy reading the results when they are released in the 2nd half of this year.
And thus, I fully expect the peer-reviewed results to validate what Dr. Kun Jin has concluded in today's press release.
2+2=4
Joseph.
I don't have an answer for your specific question...but I take comfort in knowing that the FDA's former lead statistician was probably the one that finalized all of the numbers that were published in today's press release...Dr. Kun Jin
Anavex Life Sciences Appoints Former FDA Lead Neurology Statistician as VP Head of Biostatistics
https://www.anavex.com/post/anavex-life-sciences-appoints-former-fda-lead-neurology-statistician-as-vp-head-of-biostatistics
Good post.
The other option is to wait until Rett is approved and repurpose the drug for AD.
Submitting the P2b/3 trial results with a FDA-approved drug should be sufficient to substitute the need for a second P3/4 trial.
Remember, the FDA can approve an NDA (New Drug Application) with one successful P3 trial and the repurposing of an FDA-approved drug.
So, if the FDA approves A2-73 for Rett, that would qualify for the first half of our Alzheimer's application. The other half would be our P2b/3 AD trial.
IMO, this is the main reason why Anavex wants the FDA to approve Rett first. Once it is approved, they can expedite the entire process for our Alzheimer's application by simply repurposing A2-73. (Qualifying for the $100M voucher is a bonus)
I'm not worried at all because our FDA guys know what they're doing...especially Dr. Jin.
A simple phone call to IR should easily clear this up. This should be public information so why wait until Sept/Oct?
Good post.
Raja..."I would have preferred a PR, sometime in June-July which stated ' Seeking FDA Approval'. (Not very difficult to do, since the company stated the they met both the primary and secondary end points)."
If they're going to repurpose A2-73 for AD, they'll have to wait for Rett approval. Once that happens, I believe they will indeed make the statement: "seeking FDA approval".
Raja...quote:
A) 9 months and counting since CTAD (especially after assuring that there would be more data to share, sooner than later. Each of us can interpret this delay to suit our ego/convenience by making assumptions accordingly).
B) If one considers the time since the AD trial was done and dusted, it has been more than a year.
So, what has changed since the CTAD data release 9 months ago?
Anavex Life Sciences Corp. Appoints Dr. Kun Jin as Head of Biostatistics
March 09, 2023
Anavex Life Sciences Corp. announced the appointment of Kun Jin, Ph.D., as Head of Biostatistics. 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. Prior to joining Anavex, Dr. Jin had a distinguished career of more than 27 years at the FDA.
During his tenure at the agency, Dr. Jin has contributed extensively to statistical review issues and trial designs surrounding the regulatory approval of drugs for the treatment of neurological diseases including Alzheimers disease, Parkinsons disease, migraine, epilepsy, and multiple sclerosis, as well as rare diseases, such as ALS and DMD. Dr. Jin was the lead author in top theoretical statistics, biostatistics, and molecular genetics journals. He was also a winner in a worldwide innovation competition on clinical cardiology data processing (Predicting Acute Hypotensive Episodes).
Dr. Jin has extensive research experience in Alzheimers clinical trials. He has been an invited speaker and has authored publications on topics in Alzheimers disease endpoints and trial designs. He conducted FDA/CDER The Oak Ridge Institute for Science and Education (ORISE) Summer Fellowship projects, built the Integrative Alzheimers Trial Database, FDA/CDER Regulatory Science and Review (RSR) Project and the results have been communicated at Accelerate Cures/Treatments for All Dementias (ACT-AD), Drug Information Association (DIA), and Joint Statistical Meetings (JSM).
Before joining the FDA, Dr. Jin was Assistant Professor at the Division of Biostatistics, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania. Dr. Jin received his Ph.D. in Statistics from the University of California at Berkeley, California.
Such a ridiculous accusation. You just earned an emphatic IGNORE.
Bye-bye
Great story. Thanks for sharing Hoskuld.
It's important to see how NWBO's stock price was manipulated down (~35%) to frustrate the weak-handed investors who sold their shares for a big loss...while the strong-handed investors kept their shares and positioned themselves to reap the rewards of a possible approval.
I've seen this happen numerous times with other companies just before good news is announced. You can practically set your watch to it...IMO, of course.
"The Company anticipates submitting the MAA in approximately the next 30-45 days. The Company plans to request that the MHRA review the MAA under the 150-business day process that the MHRA [equivalent to the U.S. FDA] has established to accelerate the availability of new medicines for patients in the U.K."
https://www.prnewswire.com/news-releases/northwest-biotherapeutics-announces-completion-of-prerequisites-and-plans-for-submission-of-marketing-authorization-application-301912602.html
Yeah, I get it.
But if they have 6 P3s planned for trials (and no P2s), they must be pretty comfortable with the data they've acquired for dosages.
That's up to the Anavex team to decide whether or not they want to conduct a separate P2 trial for dosage.
IMO, they have enough data with all of the P1s, P2s, and P3 trials (which includes pediatric and adults) to determine dosages for most every age group.
The question is whether those dosages can be applied to other diseases? IMO, yes.
Well, let me try to explain repurposing a drug in simpler terms...
Let's assume that the FDA approves A2-73 for Rett. That means that the FDA has approved a specific dosage for that specific disease.
Now, Anavex has to prove to the FDA that A2-73 is also effective for Fragile X - which is a new disease that is listed in our pipeline. And the only way to prove that it is effective for Fragile X is by conducting a separate P3 trial that is double-blind, placebo-controlled, multi-centered with enough n to satisfy the FDA. (We can skip the P1 and P2 trials because the FDA already knows the drug is safe via the Rett approval)
So, for every new disease that is listed in our pipeline, we will need to conduct a P3 trial to prove its efficacy.
Fortunately, we already conducted a P2b/3 trial for Alzheimer's Disease so Anavex can use that study for the P3 requirement. (When you repurpose an FDA-approved drug and submit a P3 trial, the FDA will consider it for an NDA)
BTW, we only conducted a P2 trial for PDD, so we will need to conduct a P3 trial for that disease.
I hope that makes sense to you.
Yup. The only way that you can skip the required P1 and P2 trials and go directly to a P3 trial is if you are going to repurpose a drug for another disease.
IMO, this is confirmation that Anavex will in fact be repurposing A2-73 after it is approved for Rett...which is exactly what I had outlined in my previous post a few weeks ago. (See below)
*****************************************************************************************************
abew4me
Post# 426544
Wednesday, August 09, 2023
Yes. I think they'll present the OLE as an alternative to a full-blown confirmatory trial because their NDA (New Drug Application) will be focused on repurposing A2-73.
This has been Anavex's plan from the beginning (IMO)...but the Covid virus delayed the approval of Rett. Now everything is back on track!
Drug repurposing, or repositioning, is a strategy aimed at identifying new uses for already approved drugs, which fall outside their original medical indication. This strategy has led not only to the successful preclinical and clinical testing in multiple neurological disorders, but also to the reevaluation of disused drugs, epitomized by the remarkable case of thalidomide.
In recent years drug repurposing has covered a wide range of neurological disorders, from neurodegenerative and neuropsychiatric to drug abuse-related disorders.
Alzheimer’s disease and Parkinson's disease, for example, are the two most common neurodegenerative diseases, for which only symptomatic therapies are available, while neuroprotective drugs are still an urgent unmet need. They are therefore in the top list of neurological disorders for the investigation of repurposed drugs, targeting both the disease neuropathology and symptoms.
**************************************************************************************************
The FDA can approve an NDA (New Drug Application) with one successful P3 trial and the repurposing of an FDA-approved drug.
So, if the FDA approves A2-73 for Rett, that would qualify for the first half of our Alzheimer's application. The other half would be our P2b/3 AD trial.
IMO, this is the main reason why Anavex wants the FDA to approve Rett first. Once it is approved, they can expedite the entire process for our Alzheimer's application by simply repurposing A2-73. (Qualifying for the $100M voucher is a bonus)
Our FDA guys know what they're doing...especially Dr. Jin.
TGD and his team know what they're doing. Just sit back and enjoy the ride.
"The S1R plays an important role in neuronal health and it is an established therapeutic target for neurodegenerative and neuropsychiatric disorders."
Very Nice!
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10455549/
Nice find, George.
The S1R plays an important role in neuronal health and it is an established therapeutic target for neurodegenerative and neuropsychiatric disorders. Despite its importance in physiology and disease, the biological function of S1R is poorly understood. To gain insight into the biological and signaling functions of S1R, we took advantage of recently reported crystal structures of human and Xenopus S1Rs and performed structural modeling of S1R interactions with ligands and cholesterol in the presence of the membrane. By combining bioinformatics analysis of S1R sequence and structural modelling approaches, we proposed a model that suggests that S1R may exist in two distinct conformations—“dynamic monomer” (DM) and “anchored monomer” (AM). We further propose that equilibrium between AM and DM conformations of S1R is essential for its biological function in cells, with AM conformation facilitating the oligomerization of S1R and DM conformation facilitating deoligomerization. Consistent with experimental evidence, our hypothesis predicts that increased levels of membrane cholesterol and S1R antagonists should promote the oligomeric state of S1R, but S1R agonists and pathogenic mutations should promote its deoligomerization. Obtained results provide mechanistic insights into signaling functions of S1R in cells, and the proposed model may help to explain neuroprotective effects of S1R modulators.
Co-Existence of Two Conformations of Sigma 1 Receptor BIND Domain
The structure of Sigma 1 receptor (S1R) was recently solved using X-ray diffraction (XRD) crystallography in lipid cubic phase for human S1R (hS1R) [24,25] and with the sitting drop method for Xenopus S1R (xS1R) [26] as a single transmembrane domain protein.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10455549/
Fully agree. As further proof that Anavex is preparing for commercialization, Just look at how they've restructured their chain of command by hiring Dr. Kun Jin as Vice President Head of Biostatistics....and promoting Dr. Jiong Ma to Chairman of the Board. (See below)
Anavex Life Sciences Appoints Former FDA Lead Neurology Statistician as VP Head of Biostatistics
https://www.anavex.com/post/anavex-life-sciences-appoints-former-fda-lead-neurology-statistician-as-vp-head-of-biostatistics
Anavex Life Sciences Announces Appointment of Ms Jiong Ma, PhD to Board of Directors
https://chavantcapital.com/team/dr-jiong-ma/
https://finance.yahoo.com/news/anavex-life-sciences-announces-appointment-110000415.html?fr=sycsrp_catchall
Since it's a slow weekend, I thought I'd re-post Gator's comments from the last CC. It's a great read.
Gator328
Wednesday, August 09, 2023
Some thoughts on today's conference call.
1. This is by far the most confident Dr. Missling has ever been in a public meeting. I can't understand a single thing MayoMobile writes about the science, but I can spot a con artist a mile away. In my former career, I dealt with scumbags and fraudsters all the time. They're easy to spot if you know how because they all hide behind a veneer of confidence. Deep down they know there's a chance they'll be exposed and that creates a different sort of confidence than what I heard from Dr. Missling today. He spoke with conviction and authority.
2. I don't think Dr. Missling ever forgot about or forgave the likes of Feuerstein, Fonteneau, Michaud, etc. They made his job far more stressful and difficult than it should have been, and I have a feeling his "take the market by surprise" could result in some max pain if he ends up on the winning side of the bet. The guy's got an MBA from Northwestern. He knows all the ways to maximize shareholder value at the expense of shorts. I'd much rather have someone like that than an MD who has zero years of professional financial experience or education.
3. We have 4 years of runway. That's insanely good cash management. While we don't know the terms of these partnerships, we do know that there isn't a significant cash outflow so he's striking financially prudent deals. My guess is there's no shortage of companies wanting to partner with Anavex, which leads me to the next comment...
4. Dr. Missling verbatim told us that he has received offers. These are easily verifiable claims if an investigation were to ever be launched. I have no reason not to believe him. There's been more M&A spending in 2023 than in previous years and there's still lots of money on the sidelines. This is the best possible time to have a marketable drug candidate.
5. Dr. Missling's suggestion that the extended trial could substitute for a P4 confirmatory was interesting, but it's not something that affects the calculus of my investment thesis. If it happens, great! But I've always believed we'll need another Alzheimer's trial and in the meantime, Rett will provide enough short-term revenue to prevent further dilution. The confirmatory trial is more or less pointless because everyone will know within a few months if Blarcamesine works once it becomes available, and there will be a direct comparison against those taking the -Mab treatments. Let the better drug win. If it takes another two years to get to market for Alzheimer's, that's just two more years of building demand for the product launch.
Sometimes in a poker game there's a point where it's no longer necessary to be secretive. I was pleased to see Dr. Missling finally reveal his hand when he spoke of offers received, releasing data within the next 4 months, and that he's firmly aware of current market forces and expects to counter them at the time of his choosing.
Also, thanks to all who sent private messages after I announced I'd be leaving this board. This site will not allow me to reply without paying a monthly fee. It's been a nice break from thinking about Anavex in general because at this point I feel like my money is parked and there's nothing anyone can say or do to influence the outcome. If I win I win big. If I lose I lose relatively small. That's how I look at it.
When this all finally plays out, there will be longs who made money and longs who lost money. Same with the shorts. But the musical chairs game is about to come to a screeching halt and I'm pretty confident I'll be able to find a place to sit when it does.
I don't understand your post in response to Sab's post.
Where is the accusation of corruption?
Exactly. And if the data wasn't so great I'm sure he would've sold the other 232,000 shares that he received when he exercised his options.
It was a very positive move for him (as an insider) to purchase those shares and increase his position to 1.23 Million shares.
Quote: "He doesn't seem to hold current or potential future shareholders in high regards."
You do realize that Dr. Missling is one of the biggest shareholders of the company, right?
FDA Approved 65 Percent of New Drugs in 2022 Based on a Single Study
by Megan Redshaw, J.D.
Aug 10 2023
The 21st Century Cures Act (Cures Act), signed into law in December 2016, was created to help accelerate medical product development and “bring new innovations and advances” to patients quicker and more efficiently. Yet some researchers suggest the law is being used to bypass the once rigorous and evidenced-based standards for new drug approvals, allowing novel drugs to flood the market without adequate data and public transparency.
According to a research letter published on August 8 in the Journal of the American Medical Association Network Open (JAMA), 24 of the 37 drugs approved in 2022 by the U.S. Food and Drug Administration (FDA) were based on a single study, with only four drugs having more than three studies to support their approval.
“I’m not surprised,” David Gortler, a pharmacologist, pharmacist, and FDA reform advocate at the Ethics and Public Policy Center, told The Epoch Times in an email. As a former senior advisor to the FDA commissioner, Mr. Gortler said he saw the agency grant expedited approval to a medication called aducanumab—used to treat Alzheimer’s disease “based on zero positive studies.”
“They did the same with other monoclonal antibodies for Alzheimer’s disease,” Mr. Gortler said.
According to the research letter, most of the 413 studies evaluating the 37 drugs approved in 2022 were sponsored by the industry—meaning they were manufactured, funded, and analyzed by the company producing the product, seeking FDA approval, and standing to benefit financially from the drug.
Of the studies available for analysis, only 25 percent of study results have been made publicly available, with the results of six percent of those studies published after the FDA had already approved the drug for use.
Furthermore, researchers found that only 55 percent of studies evaluating drugs in 2022 consisted of randomized clinical trials—the “gold standard” of evidence-based medicine—despite the FDA justifying most approvals based on randomized clinical trial data.
For comparison, only 20 percent of medical products in 2016 were approved based on a single study, and 55 percent were approved based on three or more studies, whereas 65 percent of drugs in 2022 were approved based on a single study, with only 11 percent having three or more studies.
“We believe consumers deserve access to the full range of evidence for the drugs they are considering, not just from the selected studies released to the public,” the authors wrote.
The researchers say their results “highlight a trend toward less rigorous standards for novel drug approvals that has evolved over the past few decades” and are consistent with other reports showing a widespread decrease in the number of trials used for drug approvals.
“The authors point to the deterioration of the quality and rigor of the regulatory review and approval of new drugs over time,” Sasha Latypova told The Epoch Times in an email. Ms. Latypova is a retired pharmaceutical industry executive with 25 years of experience in pharmaceutical research and development and co-founder of several organizations that work with pharmaceutical companies to design, execute, collect data, and submit clinical trial data to the FDA.
Ms. Latypova says this trend began with a “fast track” designation implemented in 1988 that increased the number of special regulatory programs available by the FDA and decreased the evidentiary requirements for approval. In the 2000s, Ms. Latypova said many blockbuster drugs became generic medicines, which started a “patent cliff” where industry investments began to focus on narrower niches in an effort to get patent exclusivity—which is more profitable for a pharmaceutical company.
“For example, approvals receiving an ‘orphan’ designation or what is considered rare disease increased to over 50% percent,” Ms. Latypova said. “These products are sometimes approved on as little as a single observational study with fewer than 20 subjects, however, once approved, the drug’s price increased one million to three million dollars per treatment and was fully covered by the taxpayer and private insurance—driving the costs of premiums.”
Thus, the “regulatory requirements are minimal, but the profits are outsized,” she added.
FDA Cures Act Made It Easier for Pharma and Regulatory Agencies to Cut Corners
The FDA, on its website, states the intent of the Cures Act (pdf) passed by Congress in December 2016 was to “incorporate the perspectives of patients into the development of drugs, biological products, and devices in FDA’s decision-making process” and enhance its ability to “modernize clinical trial designs,” including the use of “real world evidence” to speed up the development and review of novel medical products, including emergency and preparedness response countermeasures used to justify rapid authorization of COVID-19 vaccines.
Allowing the FDA to consider real-world evidence instead of randomized trials previously required under its strict methodological standards used to evaluate the safety and efficacy of a drug relaxed requirements for pharmaceutical companies and opened the door for bias.
The Cures Act gave new authority to the FDA to “recruit and retain scientific, technical, and professional experts and it establishes new expedited product development programs” and directed the agency to create one or more intercenter institutes to assist with coordination of activities between the drug, biologics, and device centers to improve the regulation of combination products.
The 312-page Act provided $500 million U.S. tax dollars to help the FDA implement the law over nine years and provided $6.3 billion in funding, mainly to the National Institutes of Health (NIH), a major funder of American universities and research institutions.
According to the National Center for Health Research (NCHR), the Cures Act dramatically benefits pharmaceutical and medical device companies, lowers the standards for drugs and devices, and makes it difficult for patients and physicians to decide whether to try a new treatment without knowing if it is safe or effective. This may explain why the Act was originated and promoted by major pharmaceutical companies, universities, and other organizations that hired more than 1,455 lobbyists to advance the bill.
The NCHR says the bill has had the following effects:
- Allowed anecdotal, unreliable, and easily manipulated health data to be used to approve new drugs.
- Allowed pharmaceutical and device companies to bypass public reporting requirements related to funding and gifts provided to physicians.
- Weakened patient safety by lowering the evidentiary standards required to prove a new drug or medical device is safe and effective.
- Allowed companies to disseminate potentially inaccurate scientific information not evaluated as part of the FDA approval process, opening the door for widespread use of drugs and treatments not FDA-approved.
- Reduced the FDA’s authority to regulate electronic health records systems and other medical software, which, if defective, can lead to deaths and permanent harm.
- Encourages smaller and shorter-term studies that are less likely to measure product safety and effectiveness for excluded parts of the population who may rely on them.
FDA Is Not Enforcing Reporting Requirements for Clinical Trials
Problems with clinical trial reporting go back to a law passed in 2007 requiring companies, universities, and other institutions to publish most clinical trial data in a federal database so that doctors and patients can determine whether a new product is safe or effective, according to a Science analysis. After trial sponsors failed to follow the law, the NIH and FDA attempted in 2017 to enact a final rule explaining the requirements and penalties for failing to disclose clinical trial results. Yet many sponsors ignored the requirements, and federal officials have done “little or nothing” to enforce the law.
The analysis of more than 4,700 clinical trials that should have been published on the NIH database ClinicalTrials.gov under the 2017 rule showed improved compliance rates of most large pharmaceutical companies and some universities. Yet the performance of many other sponsors, including the NIH, was “lackluster.”
ClinicalTrials.gov is an online registry of clinical trials run by the National Library of Medicine at NIH, where researchers, doctors, and patients are supposed to be able to see data on trial outcomes from peer-reviewed publications and can compare results across trials. Yet according to the analysis, thousands of trials are never published, especially when treatments are shown to be ineffective.
The Science analysis showed roughly 67 percent of studies from 30 of the 184 sponsor organizations with at least five trials failed to report any results on ClinicalTrials.gov, reinforcing the 2022 data published in the JAMA research letter.
Perhaps even more concerning is that these organizations consisting of pharmaceutical companies, universities, and medical centers failed to meet a single deadline. Those considered “habitual violators” didn’t report results in 67 percent of their trials and were an average of 268 days late disclosing data past their original deadlines.
These institutions included Harvard University, the University of Minnesota, and Baylor College of Medicine—leading recipients of NIH grants in 2019. Researchers found that The University of Texas MD Anderson Cancer Center and Mayo Clinic both failed to report results on time, or at all, in nearly two-thirds of their clinical trials. Yale University did not report results in 84 percent of its trials.
The NIH is tasked with reporting results when they sponsor studies done by agency staff or certain grantees, and the top four NIH institute sponsors reported results late or not at all in more than six of every ten trials assessed by Science. In addition, the Science analysis found the sponsors violated the reporting law more than 55 percent of the time and identified hundreds of cases where sponsors were credited for reporting results where the results themselves were not publically posted.
Despite the 2017 rule promising “aggressive enforcement and stiff penalties,” the NIH and FDA have not penalized sponsors who have not followed the requirements. The FDA in 2019 said it would not enforce penalties of up to $12,103 a day for failing to report a trial’s results until the agency issues further guidance on how it will exercise its power.
The FDA and NIH did not respond to requests for comment at the time of press.
Megan Redshaw
J.D.
Megan Redshaw is an attorney and investigative journalist with a background in political science. She is also a traditional naturopath with additional certifications in nutrition and exercise science.
https://www.theepochtimes.com/health/fda-approved-65-percent-of-new-drugs-in-2022-based-on-a-single-study-5453802?utm_source=brightnoe&src_src=brightnoe&utm_campaign=bright-2023-08-23&src_cmp=bright-2023-08-23&utm_medium=email&est=ZEY2sBilrl3%2F33K45ssrKxiNDzZ%2Ba0%2BiyRlKL1WP8uXw7pL2Eb3EY%2FnFzg%3D%3D
plex...we have a lot of investors here with different personalities.
You don't know how a person is going to react if something doesn't go their way. They may voice their frustration on the Annalise FB page and blame them for their loss.
Yes, their FB page is available to anyone who takes the time to search for it...and we should keep it that way.
Just my opinion.
Sorry. You're correct. Their full name is not on the post. Disregard my request
Joseph...out of respect for Annelise and her family's privacy, I think you should remove the link.
Thanks
Any idea how long ago they posted that message on FB?
Thanks
When Anavex submits the NDA for Rett, the FDA requires them to submit all of the data related to A2-73...so they will most likely submit the OLE data using the ~350 patients...rather than wait for the completion at the end of July, 2024.
Side note: If Anavex has had this information for some time, I would think the TGD is privy to the results...and thus, it could be another reason why Dr. Missling chose to buy the 320,000 shares of AVXL when he exercised his options.
(I'm pretty sure this doesn't violate FCC insider rules because TGD was forced to exercise his options before they expired...but maybe SAB or boi can enlighten us?)
Yup. Thanks
Mr. Palmer...I think he said that he was going to aggressively approach the FDA...which is much different than claiming that he was going to pursue the FDA for Accelerated Approval.
And remember, Dr. Jun Kin came aboard in early January and has undoubtedly made several recommendations to TGD that will increase our chances of getting FDA approval.
Perhaps Dr. Jun Kin told Dr. Missling to wait for the OLE before approaching the FDA. Or...Perhaps Dr. Jun Kin told Dr. Missling to wait for FDA approval for Rett...and then repurpose the drug for Alzheimer's Disease. (Which was the original game plan anyway, IMO)
One other thing to remember, TGD told everyone at the ASM that we will enjoy seeing the P2b/3 data once it is presented in the 2H of 2023. (paraphrasing)
The next 3 - 4 months should be very exciting for those of us that have the patience to wait.
Scratch that request.
Notice he said that AVXL is his top holding within his portfolio.
Some encouraging news after the Maui fire