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Anavex Presents Preclinical Results of ANAVEX 2-73 in Rett Syndrome
- Data presentation at 2016 Epilepsy Pipeline Conference -
- Company exploring potential to advance ANAVEX 2-73 into human clinical studies
in rare disease indication -
New York, NY – February 25, 2016 – Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative diseases including Alzheimer’s Disease, other central nervous system (CNS) diseases, pain and various types of cancer, today announced positive data of ANAVEX 2-73 in an exploratory study in a Rett syndrome model at the 2016 Epilepsy Pipeline Conference being held February 25-26, 2016 in San Francisco, CA.
Chronic oral daily dosing of ANAVEX 2-73 starting at ~5.5 weeks of age was conducted in the MECP2 Rett syndrome disease mouse model and continued through a 12-week behavioral testing time point 60 minutes pre-treatment during the behavioral testing. Behavioral paradigms measure different aspects of muscular coordination, balance, motor learning and muscular strengths, some of the core deficits observed in Rett syndrome. The experiment was sponsored by the Rettsyndrome.org Scout Program and performed by PsychoGenics, Inc., NY.
“The data demonstrates dose related and significant improvements in an array of behavioral and gait paradigms in a mouse model with a MECP2-null mutation that causes neurological symptoms that mimic Rett syndrome. There is a tremendous need for therapeutic solutions for the individuals living with Rett syndrome and their families, and we are very encouraged by the data we have seen with ANAVEX 2-73,” said Steven Kaminsky, PhD, Chief Science Officer of Rettsyndrome.org.
Rett syndrome is rare non-inherited genetic postnatal progressive neurodevelopmental disorder that occurs almost exclusively in girls and leads to severe impairments, affecting nearly every aspect of the child’s life: their ability to speak, walk, eat, and even breathe easily. The hallmark of Rett syndrome is near constant repetitive hand movements while awake. It is characterized by normal early growth and development (6 to 18 months) followed by a slowing of development, loss of purposeful use of the hands, distinctive hand movements, slowed brain and head growth, problems with walking, seizures, and intellectual disability. There is currently no cure for Rett syndrome and treatment of the disorder is symptomatic. Management of the symptoms is done through a multidisciplinary approach utilizing medication for motor difficulties, breathing irregularities, and control of seizures through anticonvulsant drugs. Rett syndrome is caused by mutations in the MECP2 gene and strikes all racial and ethnic groups and occurs worldwide in approximately 1 in every 10,000-15,000 live female births.
Leader of the Rett syndrome Natural History Study, Alan Percy, MD, stated: “Given the strong clinical safety data profile of ANAVEX 2-73, it would be encouraging to explore the compound in patients with Rett syndrome, which is a very vulnerable patient population that in addition to its neurodevelopmental symptoms also experience a significant number of seizures.”
The presentation titled “Assessment of ANAVEX 2-73 in a MECP2 Rett Syndrome Mouse Model” will be presented by Christopher U. Missling, PhD at the 2016 Epilepsy Pipeline Conference in San Francisco, CA at 4:25 p.m. PST February 26, 2016 in the oral session and will be available on the publications page of the Anavex website.
“The positive preclinical signal in Rett syndrome seems to support the upstream mechanism of action of ANAVEX 2-73 with the potential to target not only neurodegenerative diseases such as Alzheimer’s, but also neurodevelopmental rare diseases like Rett syndrome,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex.
About Rettsyndrome.org
Rettsyndrome.org is accelerating research for treatments and a cure for Rett syndrome. As the world’s leading private funder of Rett research, they have funded more than $35 million in peer-reviewed research grants and programs to date. They are a 501(c) 3 organization, earning Charity Navigator’s most prestigious 4-star rating. Rettsyndorme.org empowers families to make a difference. Visit www.rettsyndrome.org to learn more, or call (800) 818-7388 (RETT).
No, another extension is unnecessary at this point and time, and, all factors have to be weighed. My bet is that they will not extend it.
Without knowing how many patients will be required for Phase 2/3, we really can't even guess at the cost.
I do think that AVXL will receive grant(s) and sign a lucrative partnership agreement provided they don't run into unexpected problems with the current trial.
Originally, AVXL's 2a trial was designed for a 5 week duration. The trial had a 26 week PART B added in order to give patients more incentive to join the trial. Then another 26 weeks was added to PART B due to patients and caregiver requests.
Donepezil alone loses all effectiveness by 52 weeks. The way it stands now, AVXL will be able to compare A2-73 and A2-73 Plus to donepezil at 52 weeks.
Extending the trial again would create unnecessary risk unless AVXL is 100% sure that nothing could go wrong. AVXL needs to secure Phase 2/3 funding and should not do anything that may jeopardize that funding.
Something going wrong could include events not related to A2-73. Such as, a high patient drop out rate during the last extension. Even though deemed unrelated to A2-73, this could still taint the trial. I'm sure there are numerous things that could go wrong.
Institution? I doubt that. The guy is back to playing golf.
Thanks for posting this-
Starting at 1:07 in the message Dr Missling said Last month was the most relevant information that we saw with Anavex 273. Very strong dose response curve in two independent cognitive markers.
Right, it would be a good thing if the patients continued to receive A 2-73.
I'm not a trial expert and don't know if the Company should extend the trial.
If the results from the patients that have finished 9 months are excellent and the Company feels that the results put them in an ideal position to bargain with potential partners, should the Company extend the trial?
Well said!!!
I wouldn't be surprised if all patients are requesting another extension. I just don't know if it's in the Company's best interest to extend the trial again.
Had Dr. M announced another extension, how many would have interpreted that as a negative? As in, AVXL is not seeing what they had hoped for in 12 months, so they extended the trial again.
The Company can't please everyone.
I trust the Company's many trial experts to do the right thing.
We were given preliminary data for the first 12 weeks of PART B in November. The data looks damn good to me -
In the interim analysis of the first 14 patients at week 12, the PART B portion of the study demonstrated a positive trend towards improvement over 12 weeks of ANAVEX 2-73 treatment on the secondary functional outcome measure, the Alzheimer’s Disease Co-operative Study – Activities of Daily Living Inventory (ADCS-ADL) by +3.21 points.
http://www.anavex.com/?news=anavex-announces-positive-primary-and-secondary-endpoints-were-achieved-in-a-phase-2a-clinical-trial-of-anavex-2-73-in-alzheimers-disease%e2%80%8e
Furthermore, this positive dose response data is huge -
NEW YORK, NY – January 11, 2016 – Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative diseases including Alzheimer’s disease, other central nervous system (CNS) diseases, pain and various types of cancer, today reported a positive dose-response relationship has been observed in a pre-planned interim analysis of data from the ongoing Phase 2a trial of ANAVEX 2-73 for treatment of mild to moderate Alzheimer’s disease.
http://www.anavex.com/?news=anavex-announces-positive-dose-response-data-for-anavex-2-73-in-alzheimers-disease-patients
It makes no sense to be negative regarding upcoming data at this time!
AVXL may assume a lot of unnecessary risk by extending the PART B again. The first extension made sense in that donepezil becomes pretty much worthless at 12 months. The 12 month comparison should look really good for A2-73.
Should A2-73's results become less remarkable after 12 months, it could hurt AVXL's partnering position and it's ability to gain grants.
If AVXL already had Phase 3 funding, the extension would make more sense.
However, the Company may not see additional risk.
IMHO
As I recall, Dr. Missling stated that they did not have the PK/PD data back yet. Previously, it was stated that the 12 week, PART B and 5 week, PART A, PK/PD data would be released together. I'm going to double check this.
Evidently, the PK/PD data is analyzed by a third party.
I think we will get some new data.
Dr. Missling released the dose response data in January and there wasn't even a conference. So, he's not necessarily saving all data for scientific conferences.
Can't we work out when the 12 wk data is due? Isn't next week the 12th week?
Let these words sink in -
From 10-K -
On December 22, 2015, the Company received a subpoena from the Securities and Exchange Commission (SEC) which indicates that the agency is conducting a formal investigation. The Company believes the subpoena and investigation relate to the recent unusual activity in the market for the Company’s shares. The Company is fully cooperating with the SEC in this investigation and is unable to predict when this matter will be resolved or what further action, if any, the SEC may take in connection with it.
Reply from IR -
hooperg83 Tuesday, 12/29/15 03:15:10 PM
Re: Bullish91 post# 46646
Post # of 46782
You are incorrect.
I just spoke with Christine Payne from Primoris. She indicates that AVXL received a subpoena from the SEC regarding abnormal trading of their security. She indicates that this subpoena does not involve the corporate officers of AVXL.
This is likely an investigation of what happened in November, specifically naked short selling.
According to this article, donepezil sold for $3,000 a year before patent expiration -
You can now buy a one year supply of Donepezil for $109.00.
This time last year you might have expected to pay $3,000.00 or more for a one year supply. Imagine.
http://www.alzheimersreadingroom.com/2011/09/donepezil-aricept-price-falls-by-90.html
That's $3,000 a year for a drug that's not very effective -
Alzheimer's disease[edit]
There is no evidence that donepezil or other similar agents alters the course or progression of Alzheimer's disease (AD). A 6 to 12-month controlled studies have shown modest benefits in cognition and/or behavior.[3] The UK National Institute for Clinical Excellence (NICE) recommends donepezil as an option in the management of mild to moderate Alzheimer's disease.[6] The person should, however, be reviewed frequently and if there is no significant benefit it should be stopped.[6] In 2006 the U.S. Food and Drug Administration also approved donepezil for treatment of mild, moderate and severe dementia in Alzheimer's disease.[7]
https://en.wikipedia.org/wiki/Donepezil
Anavex states that more than 25 million people are currently diagnosed with Alzheimer's -
About Alzheimer’s Disease
Today, Alzheimer’s disease remains the largest unmet medical need in neurology. More than 25 million people are currently diagnosed with Alzheimer’s, with the associated cost of care estimated to exceed $200 billion annually. By 2050, 100 million people are expected to be living with the disease. Alzheimer’s disease is a neurological disorder generally characterized by memory loss and cognitive decline. A neurodegenerative form of dementia, the disease begins with mild symptoms and becomes progressively worse.
http://www.anavex.com/?news=anavex-announces-preparation-of-regulatory-filings-based-on-guidance-from-the-fda
AVXL's plans for releasing data -
“We are encouraged by the preliminary safety and efficacy data. We look forward to continuing with PART B of the Phase 2a trial and expect to provide data updates at 12 week, 26 week, 38 week and 52 week time points,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “While we remain focused on Alzheimer’s, the remarkably fast onset of clinical effect of ANAVEX 2-73 increases our options to potentially pursue additional indications for diseases characterized by working memory impairment and may enable clinical trials to be completed within shorter time frames.”
http://www.anavex.com/?news=anavex-announces-positive-primary-and-secondary-endpoints-were-achieved-in-a-phase-2a-clinical-trial-of-anavex-2-73-in-alzheimers-disease%e2%80%8e
You're welcome. Have a great weekend!
Anavex 2-73 Phase 2a, PART A blockbuster data (bold by me)-
Positive Safety Data ...
... several Cogstate tests demonstrated highly statistically significant improvements
... highly statistically significant biomarker effects
... higher doses achieved a statistical significant improvement
Professor Paul Maruff, Chief Scientific Officer of Cogstate commented: “The Cogstate tests measure people’s ability to store and use information. The results of the Phase 2a study demonstrate that ANAVEX 2-73 improves psychomotor function, attention and working memory. For attention and working memory these improvements were statistically significant with a p-value of p<0.05 and p<0.001, respectively and their magnitude clinically important. To my knowledge, we have not yet seen a drug that has improved quantitatively working memory to such an extent as seen with ANAVEX 2-73”.
Anavex gave us an early glance at the 12 week PART B data that could be reported at anytime now -
In the interim analysis of the first 14 patients at week 12, the PART B portion of the study demonstrated a positive trend towards improvement over 12 weeks of ANAVEX 2-73 treatment on the secondary functional outcome measure, the Alzheimer’s Disease Co-operative Study – Activities of Daily Living Inventory (ADCS-ADL) by +3.21 points.
http://www.anavex.com/?news=anavex-announces-positive-primary-and-secondary-endpoints-were-achieved-in-a-phase-2a-clinical-trial-of-anavex-2-73-in-alzheimers-disease%e2%80%8e
http://www.anavex.com/?news=anavex-announces-positive-dose-response-data-for-anavex-2-73-in-alzheimers-disease-patients
The naysayers cling to the hope that the blockbuster trial data is due to placebo effect. Dr. Macfarlane, the trials lead investigator, throws cold water on that theory in this interview -
4. Of all the battery of tests which do you think most powerful for AD, and how did Anavex 2-73 perform in that test?
The One-back test (which measures working memory, a key domain of impairment in AD) within the Cogstate battery is the most difficult test, yet showed the greatest improvement. This outcome is unlikely to have been an artefact of any placebo effect...when placebo effects occur they are typically larger for the easier tests. In addition, whilst placebo effects are common with CNS sedative drugs, they are less likely to be present in tests of drugs used to enhance cognition...individuals cannot anticipate, or 'imagine' what a better score would consist of, and are unable to produce improved results through mere 'wishful thinking.'
http://seekingalpha.com/article/3672856-anavex-life-sciences-interview-with-dr-stephen-macfarlane
Miss Australia, as always, thanks for sharing your family members status.
You're absolutely correct here -
That article is the biggest load of BS. How can you have a placebo affect with a memory test? Either you can recall or you can not.
If you read some of McFarlanes interviews he points out that the placebo affect does not apply to these tests.
AVXL's beautiful share structure -
From the recent 10-K -
As of December 29, 2015, 34,601,173 shares of our common stock were issued and outstanding.
A summary of the Company’s share purchase warrants outstanding is presented below: Balance, September 30, 2015 4,272,890
At September 30, 2015, the following stock options were outstanding: Total 1,822,500
40,696,563 FULLY DILUTED!!!
I believe that -
The share price went up do to data that for the first time showed that a drug was working against Alz.
A social media short and distort campaign ambushed Anavex's share price.
The SA author made no attempt to shine light on how good the trial data is. He just alluded to hype.
There's more, but, screw it. The data will continue to be good and these guys will have egg on their face.
That's a stupid question!!!
Small biotechs are manipulated with no regard for the fundamentals. tootall's earlier posts are a prime example of this.
If you actually are in it for the jackpot, then ignore the short term price fluctuations. They're irrelevant.
Great post from another board -
hooperg83 • Jan 11, 2016 7:15 AM Flag
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Dose Response
.
A 5 week improvement of 2 points on the MMSE at approximately half maximum dosage is monumental. Dose dependence is a great indicator that the drug needs to saturate its ligands as expected, and thus very unlikely that this is placebo. Anavex is the real deal.
Melissa Davis, Feuerstein, and associated message board trolls, you lose.
Miss Australia, thanks for sharing. Sounds like your loved one and A2-73 are progressing nicely.
Time for some good news. Sorry can not hang about as the work for a living thing does get in the way.
Family member went in for two lots/two days of tests last week, medical (brain scans, bloods etc.) and memory/cognitive (with the researchers at The Alfred Group).
Don't have results yet for the medical tests (I assume they have to go via the boss, Steve McFarlane). However the doctors were very pleased. Yes, very pleased. Particularly with the memory tests.
Once again I can not state how great these folks are. Family member feels treated as part of "the team" and I don't know who is genuinely happier with the results to date, them or him!
The previous AD drug failures targeted the symptoms of AD rather than the causes.
Anavex approach -
“Our approach targets Alzheimer’s disease further upstream and apparently blocks these disease hallmarks, which may be a more promising approach. Combined with earlier findings that ANAVEX 2-73 restores mitochondrial functionality, this new report strengthens our compound’s potential in Alzheimer’s disease to also potentially act as a preventative drug.”
http://www.anavex.com/?post_type=news&p=1406
KMBJN,
There is a lot of info in this 11/7 data release presentation. It may be helpful -
http://www.anavex.com/wp-content/uploads/2015-11-07_Anavex_Presentation_CTAD_2015_Macfarlane.pdf
XL, here's some info from the recent 10-K-
As of December 29, 2015, there were approximately 71 holders of record of our common stock. As of such date, 34,601,173 shares of our common stock were issued and outstanding.
A summary of the Company’s share purchase warrants outstanding is presented below: Balance, September 30, 2015 4,272,890
At September 30, 2015, the following stock options were outstanding: Total 1,822,500
Common Stock -
Athanasios Skarpelos (Director) - 1,323,125 (2) 3.8%
(2) Includes options to purchase 16,667 shares of our common stock at $0.92 per share that have vested. Excludes options to purchase 33,333 shares of our common stock at $0.92 per share that do not vest within 60 days.
Christopher Missling (CEO/Director) - 1,701,167 (3) 4.7%
(3) Includes options to purchase 500,000 shares of our common stock at $1.60 that have vested, options to purchase 31,250 shares of our common stock at $1.32 that have vested, options to purchase 166,667 shares of our common stock at $0.92 per share that have vested, and 1,000,000 shares of restricted common stock that have vested pursuant to the achievement of certain objectives. Excludes options to purchase 93,750 shares of our common stock at $1.32 per share, options to purchase 333,333 shares of our common stock at $0.92 per share and options to purchase 187,500 shares of our common stock at $5.04 per share that do not vest within 60 days.
Directors & Executive Officers as a group (6 persons) - 3,107,672 8.6%
NEW YORK, Jan. 08, 2016 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp.(AVXL) (“Anavex” or the “Company”) , a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative diseases including Alzheimer’s disease, other central nervous system (CNS) diseases, pain and various types of cancer, today addresses the recent press releases by law firms.
The Company is aware of press releases issued by plaintiffs’ lawyers announcing the filing of one or more lawsuits against the Company. At this time, based on a review of federal court dockets, we are aware of only one case that has actually been filed. We have reviewed the complaint in that case and believe it has no merit.
The claims made by the ambulance chasers are false-
From Anavex's 10-K -
On December 22, 2015, the Company received a subpoena from the Securities and Exchange Commission (SEC) which indicates that the agency is conducting a formal investigation. The Company believes the subpoena and investigation relate to the recent unusual activity in the market for the Company’s shares. The Company is fully cooperating with the SEC in this investigation and is unable to predict when this matter will be resolved or what further action, if any, the SEC may take in connection with it.
Reply from Anavex's Investor Relations -
hooperg83 Tuesday, 12/29/15 03:15:10 PM
Re: Bullish91 post# 46646
Post # of 46782
You are incorrect.
I just spoke with Christine Payne from Primoris. She indicates that AVXL received a subpoena from the SEC regarding abnormal trading of their security. She indicates that this subpoena does not involve the corporate officers of AVXL.
This is likely an investigation of what happened in November, specifically naked short selling.
I don't think the shorts had much to do with the drop. The interest rate charged to the shorts, at Fidelity, did not change during this time period. There was no change in demand for shares to borrow from shorts.
AVXL closed at 7.02 on 12/28. News of the subpoena led to panic selling by longs on 12/29, 12/30, and early on 12/31.
Xena and 2014, as I stated (post #48179) IMO the shorts aren't done with AVXL and might believe there is one last chance to strike hard at it, which will be when the 12 week data is released. Their guns are loaded and will be out in force at that time.
I don't believe they have given up trying to suck the wind out of AVXL's sails and kill its momentum before NIH money comes to fruition or a new study based on FDA guidance is announced. If the shorts can create enough doubt within shareholders and outside support groups, then they might win.
Remember that a weak hit piece caused the SP to go from a high of $8.03 on 12/15 to $4.60 on the 31st, a loss of 42%+, so there are still weak hands in the s/holder group, regardless of what is professed here.
On 1/12/15, Anavex announced that the first patient had been dosed -
http://www.anavex.com/?news=anavex-doses-first-alzheimers-patient-in-phase-2a-trial-of-anavex-2-73-and-anavex-plus
57 weeks later, this patient should complete the trial.
I have read that patients in other trials would be allowed to continue dosing until the trial ended. I don't know if that is the case in this trial.
I think AVXL will continue to release data the way they did on 11/9 when they gave us all patients at 5 weeks and 14 patients after the additional 12 weeks. Next, I'm guessing we'll see all patients at 12 weeks of PART B and ~14 patients at 24 or 26 weeks.
bernardc gave us this excellent report that included this -
-12 weeks result should be out by mid-january if not before and they will be looking for a great venue to announce them
PART B runs for 52 weeks. The 5 week PART A is not included in PART B.
The ongoing, multicenter Phase 2a adaptive trial of ANAVEX 2-73 in both male and female mild-to-moderate Alzheimer’s patients enrolled 32 participants. It commenced in January 2015 and the participants, the majority of whom are also taking donepezil, have now completed PART A of the two-part trial. Lasting up to 36 days (5 weeks) for each patient, PART A was a simple randomized, open-label, two-period trial with an on-off-on not-yet-optimized dosing regimen to assess bioavailability, and cross-over between oral (30mg/50mg) and IV (3mg/5mg) administration. Event-related potentials (ERP) were used to assess cognitive effects and optimize dosing of ANAVEX 2-73. PART B is an open-label extension for a further 52 weeks. Initially planned for 26 weeks, PART B was extended to 52 weeks as a result of requests from patients and caregivers. PART B utilizes daily oral dosing in order to establish a longer drug effect.
Our focus should be on the following 2 questions -
Q - Is A2-73 efficacious?
Q - Is A2-73 safe?
A - Positive Safety Data, Statistically Significant Improvements on Exploratory Clinical Endpoints
NEW YORK, NY, November 9, 2015 – Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL). On Saturday, investigators presented positive safety and cognitive efficacy data for ANAVEX 2-73, the Company’s lead investigational oral treatment for Alzheimer’s disease targeting sigma-1 and muscarinic receptors, which are believed to reduce protein misfolding including reduction of beta amyloid, tau protein and inflammation at the international CTAD 2015 conference in Barcelona, Spain.
Initial analysis of Phase 2a data demonstrated that the study met the primary objective of safety as ANAVEX 2-73 was well tolerated and results were consistent with prior Phase 1 clinical trial data. The secondary objectives were also met, with ANAVEX 2-73 showing cognitive improvement across all doses in all exploratory cognitive measurements, including the Cogstate battery, Mini Mental State Examination (MMSE), event-related potentials (ERP) and P300 tests, which consistently demonstrated improvements from baseline in the completed PART A portion of the study in 32 mild-to-moderate Alzheimer’s patients. Even though PART A was designed as a 5 week bioavailability trial that included a built-in wash-out period of 12 days and without an optimized dosing regimen, several Cogstate tests demonstrated highly statistically significant improvements. This finding was supported by a trend towards improvement in median MMSE score, which increased by +1.5 over baseline at week 5.
Positive effects on cognition were further supported by highly statistically significant biomarker effects of treatment at week 5 on one event-related potential (ERP) measure with a p-value of p<0.0007 and improvement in the P300 signal. The ERP biomarker scores improved compared to the initial data presented at AAIC in Washington, DC in July 2015, by which time not all patients had yet completed PART A.
All patients who completed PART A volunteered to continue in the longitudinal PART B extension study.
In the interim analysis of the first 14 patients at week 12, the PART B portion of the study demonstrated a positive trend towards improvement over 12 weeks of ANAVEX 2-73 treatment on the secondary functional outcome measure, the Alzheimer’s Disease Co-operative Study – Activities of Daily Living Inventory (ADCS-ADL) by +3.21 points.
“While it is of prime importance to have confirmed that both the primary and secondary endpoints of the trial have been met, it is extremely encouraging to see the emergence of such strong cognitive signals after only 5 weeks of treatment. Such an outcome in a trial such as this is unprecedented in my experience, and the current results suggest that ANAVEX 2-73 could potentially make a significant difference in patients’ lives,” said Associate Professor Stephen Macfarlane, FRANZCP, Director of Aged Psychiatry at Alfred Health, who conducted the study. “We continue to receive extremely positive feedback about the effects of the drug from our study participants and their caregivers. The results justify a prospective comparison with current standard of care in a larger clinical trial.”
Professor Paul Maruff, Chief Scientific Officer of Cogstate commented: “The Cogstate tests measure people’s ability to store and use information. The results of the Phase 2a study demonstrate that ANAVEX 2-73 improves psychomotor function, attention and working memory. For attention and working memory these improvements were statistically significant with a p-value of p<0.05 and p<0.001, respectively and their magnitude clinically important. To my knowledge, we have not yet seen a drug that has improved quantitatively working memory to such an extent as seen with ANAVEX 2-73”.
Professor Harald Hampel, member of Anavex’s Scientific Advisory Board, and AXA Research Fund Chair at Sorbonne Universities’ Pierre and Marie Curie University (UPMC) in Paris, France, commented: “The collective data from this Phase 2a trial supports the concept of targeting the sigma-1 receptor with ANAVEX 2-73 with a degree of confidence that we did not foresee. Sigma-1 receptor presents an innovative interventional upstream approach to impact key cellular events believed to contribute to Alzheimer’s disease pathophysiology. The presented Phase 2a data underlines the importance of rigorously investigating a potential efficacy signal on co-primary outcomes such as cognition and function in larger and well-powered trials”.
“We are encouraged by the preliminary safety and efficacy data. We look forward to continuing with PART B of the Phase 2a trial and expect to provide data updates at 12 week, 26 week, 38 week and 52 week time points,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “While we remain focused on Alzheimer’s, the remarkably fast onset of clinical effect of ANAVEX 2-73 increases our options to potentially pursue additional indications for diseases characterized by working memory impairment and may enable clinical trials to be completed within shorter time frames.”
The presentation entitled “New Exploratory Alzheimer’s Drug ANAVEX 2-73: Assessment of Safety and Cognitive Performance in a Phase 2a Study in mild-to-moderate Alzheimer’s Patients” was presented by Professor Steve Macfarlane at CTAD in a late-breaking oral session and is available on the publications page of the Anavex website.
About the ANAVEX 2-73 Phase 2a Study
32 subjects who met NINCDS-ADRDA criteria for probable AD were recruited at five clinical sites in Melbourne, Australia. Subjects are between 55 and 85 years of age, and have a MMSE of 16 to 28. In PART A of the study, participants were administered ANAVEX 2-73 orally and IV in a randomized, open-label, 2-period, cross-over trial separated by a wash-out with adaptive study design lasting up to 36 days (5 weeks) for each participant. In PART B of the study, all participants are administered ANAVEX 2-73 daily orally.
At week 5 the MMSE score increased by +1.5 over baseline. The Cogstate Identification Task, Cogstate One Back Task and ERP Reaction Time all showed statistically significant improvements. In a preliminary interim readout of PART B data, ANAVEX 2-73 improved ADCS-ADL score, a functional measurement, over a period of 12 weeks by +3.21 over baseline, with 11 out of 14 (78.6%) patients improving. Both MMSE and ADCS-ADL are regulatory approved endpoints for pivotal Alzheimer’s disease trials.
The ongoing, multicenter Phase 2a adaptive trial of ANAVEX 2-73 in both male and female mild-to-moderate Alzheimer’s patients enrolled 32 participants. It commenced in January 2015 and the participants, the majority of whom are also taking donepezil, have now completed PART A of the two-part trial. Lasting up to 36 days (5 weeks) for each patient, PART A was a simple randomized, open-label, two-period trial with an on-off-on not-yet-optimized dosing regimen to assess bioavailability, and cross-over between oral (30mg/50mg) and IV (3mg/5mg) administration. Event-related potentials (ERP) were used to assess cognitive effects and optimize dosing of ANAVEX 2-73. PART B is an open-label extension for a further 52 weeks. Initially planned for 26 weeks, PART B was extended to 52 weeks as a result of requests from patients and caregivers. PART B utilizes daily oral dosing in order to establish a longer drug effect.
The primary endpoint of the Phase 2a trial is evaluation of safety and tolerability of ANAVEX 2-73, which had shown potential in preclinical studies to prevent, halt and/or reverse the course of the disease. Secondary endpoints were dose response, bioavailability, and exploratory cognitive effects using electroencephalographic (EEG) activity and event-related potentials (ERP), Cogstate battery, Mini Mental State Examination (MMSE), and evaluation of Alzheimer’s Disease Co-operative Study – Activities of Daily Living Inventory (ADSC-ADL) as well as the exploration of ANAVEX 2-73 as a potential add-on therapy to donepezil, the current standard of care. The combination of ANAVEX 2-73 and donepezil (Aricept®) is known as ANAVEX PLUS.
http://www.anavex.com/?news=anavex-announces-positive-primary-and-secondary-endpoints-were-achieved-in-a-phase-2a-clinical-trial-of-anavex-2-73-in-alzheimers-disease%e2%80%8e
http://www.anavex.com/wp-content/uploads/2015-11-07_Anavex_Presentation_CTAD_2015_Macfarlane.pdf
I am thinking that the SEC wanted access to all of the information held by AVXL's transfer agent.
The SEC may not have access to off shore trading accounts and this info may help them in their investigation.
Although, still early in the game, Anavex has already scored big with the first 2 trial results. No other AD drug has come close to matching their early results.
Good football games on TV today.