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Additional write-up from FRAXA on last week's panel discussion: https://www.fraxa.org/making-drug-development-efficient-through-community-based-collaboration/
A few upcoming conference presentations folks might be interested in tracking. Of course the merits of these appearances may be hotly debated, but some may find them interesting to be aware of...
November 9-10 - Outsourcing in Clinical Trials New England
3:45 pm: Is the pharma industry still too conservative with wearable tech?
This session will give you 5 key takeaways for how and why you should incorporate wearables into your study.
• Discussing what wearable technologies are available and accepted by regulatory authorities
• Exploring home monitoring technologies to collect patient data in a timelier manner
• Overcoming challenges when shifting to wearables whilst ensuring patient safety
• Considering the infrastructure required for wearables when running a large multi-region trial
• How are wearables being more widely used to help with reimbursement?
Panelists - Christopher Missling and Daniel Klamer
11:00 am: Clinical Trials for Rare Diseases: Challenges and Opportunities
• Explore challenges in clinical trial execution in rare diseases
• Discuss strategies to overcome these challenges
• Share lessons learned from initiating and completing clinical trials in Rett Syndrome
Presenters - Christopher Missling and Daniel Klamer
https://www.arena-international.com/octnewengland
Nov 18: Microbiome Connect: Gut therapeutics USA
10.00 am: Unravelling The Gut-Brain Axis: The State Of The Gut-Brain Axis Field
• Bolster your understanding of a rapidly growing area of gut-organ axis research so you can make informed decisions pertaining to your investment and entry to the space.
• Create effective clinical trials pertaining to neurological conditions, which encompass multi-disciplinary approaches, by leveraging the lessons learned over many years of research by the expert panel.
Panelist - Christopher Missling
https://www.kisacoresearch.com/events/microbiome-connect-gut-therapeutics-usa
Dec 15: Neurodevelopmental Drug Development Summit
11:20 am: Utilizing Precision Genetic Medicine to Treat Rare Neurodevelopmental Diseases: Focusing on Rett Syndrome
Synopsis
• Requirement of clear understanding of target engagement
• Importance of predictive biomarker of response correlating with efficacy
• Relevance of appropriate efficacy endpoints
Presenter - Christopher Missling
12:50 pm: Live Q&A Session with Your Expert Speakers
Panelist - Christopher Missling
https://neurodevelopmental-drug-development.com/
I got you. Missling said on 1/30/17 at NobleCon13 - Noble Capital Markets' 13th Annual Institutional Investor Conference:
Simon Stott, who writes the Science of Parkinson's, is the deputy director of Research at the Cure Parkinson’s Trust. The organization posted this article a few days back: https://cureparkinsons.org.uk/2021/06/sigma/?fbclid=IwAR0WV0ifgbp906iFCGEwpjrdz0HGkFqH7JfgOB4-arUqLss2NGee6gN-SN4 (may have already made its way here, but just wanted to provide that additional context).
Simon's written about Anavex previously, so it's nice to see his thoughts here, and I appreciate his effort in explaining background concepts:
https://scienceofparkinsons.com/2021/06/29/anavex/?fbclid=IwAR3fx3LjVeI4koo9Rc1QT1l4dZRkC9oDUZLc0X9UI1d50xHwmInjwuEafFM
Here's another: https://markettactic.com/anavexs-parkinsons-disease-drug-reportedly-improves-cognitive-assessment-and-motor-responses/5517329/?fbclid=IwAR3_YgCDqd4id_bCkOyHk-J700cxUanIsakrpts4Ff2FEFOH1oCbw-TbgXo
Far less comprehensive than the excellent Science of Parkinson's post, but with some interesting assumptions.
Video posted today of Dr. Missling's presentation, Clinical Trials For Rare Diseases: Challenges And Opportunities, from the May 10th Precision in Clinical Trials Virtual Summit (East Coast) conference:
July 22, Dr. Walter E. Kaufmann will be speaking at the Collaborations That Transform - Fragile X Syndrome: In Pursuit of a Cure special global event to commemorate the annual Fragile X Awareness Day. He will be speaking under the topic New Directions in Fragile X Syndrome Therapeutics.
Complimentary webinar registration:
https://wxpress.wuxiapptec.com/events/fragile-x-syndrome-in-pursuit-of-a-cure/
Webinar Trailer:
June 9, Dr. Missling will be speaking at the ACCESS CHINA Biotech Forum 2021 Summer Showcase from 9:10-9:30am(CST). He will also have a Global Out-license Session, VOD presentation available there from June 10-26.
https://biotochina.org/agenda/
Dr. Walter E. Kaufmann will speak at AEDD TRIALS XVI on 6/18, delivering a 10 minutes presentation; Update on Adult and Pediatric Clinical Rett Syndrome ANAVEX2-73 Program. https://aedtrials.com/
Also, on 6/15, the International Rett Syndrome Foundation will be hosting a RettEd webinar titled Bringing Treatments & Cures Home. https://www.rettsyndrome.org/event/retted-bringing-treatments-cures-home/
In my opinion, the week of June 14th seems like it would be a good one to release the full US Rett data. We’ll see.
Dr. Missling is interviewed for this story, amongst the other featured biotech CEO's: https://www.investors.com/news/technology/biogen-stock-and-aducanumab-what-this-alzheimers-treatment-means-for-biotech-stocks
Coming on the heels of their recently announced collaboration with Sanofi in the auto-immune disease space, Ariana today announced a key new hire, referencing their work with Anavex within the PR. Nice to see Ariana's continued growth, as well as how they frame their current success.
My post that you replied to that started this chain, and your original comment, had nothing to do with the Needham conference. That post contained a link from the other conference Missling spoke at yesterday, a panel discussion at the World EPA Congress 2021. The youtube video was posted by RAM after the discussion was available for viewing by congress attendees on the congress website. Hope this clears up any confusion.
Link to the World EPA Congress panel discussion, posted to youtube by RAM:
Video of today's panel discussion from the WORLD EPA CONGRESS 2021:
Dr. Stephen Macfarlane was never an employee of Anavex. He is Head of Clinical Services of The Dementia Center at HammondCare and presumably, within that role, is still involved with Anavex clinical trials.
This "Australian Clinical & Scientific Lead" role at Anavex is a new, in-house, position.
Notable recent hire; Michael Kornhauser has joined the Anavex team as "Australian Clinical & Scientific Lead". From LinkedIn, it looks like as of March he left his position, “Associate Director, National Clinical Trials”, at HammondCare. This move comes after having worked with blarcamesine across trials at Alfred Health and HammondCare since 2014. He comments on this work in a post after the initial PDD results:
A drug which appears to slow Alzheimer's is being offered to more Victorians as part of a clinical trial. @EmilyRice28 #9News pic.twitter.com/IqKUXKzlhA
— 9News Melbourne (@9NewsMelb) January 28, 2020
A final AD recruitment push in Australia: https://m.facebook.com/story.php?story_fbid=2917159321897834&id=1641178672829245
Dr. Missling’s presentation, “Clinical Trials for Rare Diseases: Challenges and Opportunities”, at the Precision in Clinical Trials Virtual Summit (West Coast) on February 24th:
Excellent post - thank you.
This newly posted article is basically a repeat of year old news. Compare the funding numbers from this article from last year: https://news.sanfordhealth.org/research/kevin-francis-batten-disease/ The good news of that is that it means the preclinical research in mice has been going on for the past year.
An early 2017 quote from Missling that always stuck with me, as it struck me as both sincere and as a valuable indicator of how he intended to drive the company's clinical efforts. It's nice to see this approach starting to pay off for all those involved:
Videocast of this week's Rare Disease Day at NIH with Dr. Missling's discussion starting at 1:12:21: https://videocast.nih.gov/watch=41259
There was a previously available link put out by RAM of the discussion, but it cut off before the brief but informative Q&A portion.
That listing is from the 2020 meeting held on November 12th — Missling gave this talk on 1-41 last fall. The site is confusing, but they have not listed speakers/topics for 2021 yet and are still showing 2020 info. It's a little clearer from this link (see 2020 Speaker Faculty, then scroll down to Missling and click): https://npd-summit.com/about/speakers/
Effects of the Sigma-1 Receptor Agonist Blarcamesine in a Murine Model of Fragile X Syndrome: Neurobehavioral Phenotypes and Receptor Occupancy
https://www.researchgate.net/publication/349543848_Effects_of_the_Sigma-1_Receptor_Agonist_Blarcamesine_in_a_Murine_Model_of_Fragile_X_Syndrome_Neurobehavioral_Phenotypes_and_Receptor_Occupancy
Evaluation of ANAVEX2-73 (blarcamesine) in Participants with Parkinson’s Disease
Study Rationale:
We are testing ANAVEX2-73 (also known as blarcamesine), which previous research has shown helps improve behaviors as well as normalizes biochemical changes in a Parkinson’s disease animal model (6OHDA, which was supported by MJFF). In 132 patients with Parkinson’s disease dementia, the drug significantly improved cognitive function and memory as well as REM sleep. This includes complex cognitive tasks that impact quality of life such as making a choice between similar objects and remembering daily personal experiences, which could be impaired in Parkinson’s disease. In patients with Alzheimer’s disease a Phase 2a trial demonstrated a concentration dependent response in both cognition (MMSE) and function (ADCS-ADL) over 148 weeks (longer than 3 years).
The drug works by activating the Sigma-1 receptor protein in the brain. This protein helps brain cells stay healthy by reducing the effects of certain kinds of stress, preventing toxic proteins from building up in brain cells, and possibly protecting brain cells in other ways. This study is an important step in discovering if activating this protein will slow or reverse damage to brain cells and help them work normally again, thereby slowing or stopping Parkinson's progression.
Hypothesis:
We want to know if this new drug, ANAVEX2-73 (blarcamesine) can safely travel through the body to the final destination in parts of the brain most affected by Parkinson’s disease, which will help us determine if this new drug can help people with Parkinson’s disease.
Study Design:
In this study, researchers will give ANAVEX2-73 (blarcamesine) to up to 24 patients with Parkinson’s disease and healthy volunteers. The drug will be bound to a special tag or marker which will allow us to visualize the drug as it moves through the body to brain, using a medical imaging tool called Positron Emission Tomography (PET).
The researchers will also take blood samples from patients to learn about how the drug breaks down in the body to make sure it is safe and has minimal side effects.
Impact on Diagnosis/Treatment of Parkinson’s Disease:
This project will help us understand if this drug can safely make its way to the brain cells that are affected by Parkinson’s disease. This study may lead to a new treatment for Parkinson’s disease that will reduce or even reverse symptoms with improvements in the ability to walk, talk, eat, and smile, especially when combined with other medications.
Next Steps for Development:
If this study is successful, the drug will be tested in more patients and at different doses. This “efficacy testing” would determine the best dose of the drug to help people with Parkinson’s disease and whether it performs better than existing medications for Parkinson’s disease (or if a combination treatment is better).
https://www.michaeljfox.org/grant/evaluation-anavex2-73-blarcamesine-participants-parkinsons-disease
Nice to see the ERP Biomarker Qualification Consortium's initial observational trial was completed on 1/31 as per a clinicaltrials.gov update posted today. Will be interesting to hear of any learnings from this trial and next steps for the Consortium.
https://clinicaltrials.gov/ct2/show/NCT04025502?term=anavex&draw=2&rank=11
March 1 : Rare Disease Day at NIH
11:35 A.M. RARE STORY #2: INDUSTRY AND PATIENT ADVOCACY COLLABORATIONS — MAKING IT A WIN-WIN
Moderator:
• Shazia Ahmad, Senior Director, Patient and Physician Services, United BioSource LLC (UBC); Rare Disease Thought Leader, Patient and Stakeholder Engagement
Expert Panelists:
• Nadia Bodkin, Pharm.D., M.S., Rare Disease Patient Advocate, Rare Advocacy Movement (RAM)
• Christopher U. Missling, Ph.D., M.B.A., President and Chief Executive Officer (CEO), Anavex Life Sciences Corp.
https://ncats.nih.gov/news/events/rdd
https://events-support.com/Documents/RDD_NIH_2021_Agenda.pdf
Nice to note that it looks like Missling will be the only Industry representative speaking here, and this event will be free and open to the public for viewing.
2 more AD sites (both in Canada) flipped to recruiting today: https://clinicaltrials.gov/ct2/history/NCT03790709?A=16&B=17&C=merged#StudyPageTop
Dr. Missling is scheduled to speak at Rare Disease Day at NIH on March 1, 2021. He’ll be speaking along with Nadia Bodkin, founder of Rare Advocacy Movement, as well as Shazia Ahmad of United BioSource in a session titled Rare Story #2: Industry and Patient Advocacy Collaborations — Making It a Win-Win.
https://ncats.nih.gov/news/events/rdd
https://events-support.com/Documents/RDD_NIH_2021_Agenda.pdf
Speaking of IP protection around MS, it's tough to look at MS as being "disappeared" when a year ago Anavex issued this patent PR, specifically calling out MS from the numerous disorders referenced within the patent: https://www.anavex.com/anavex-life-sciences-issued-new-u-s-patent-for-anavex2-73-treatment-of-neurodevelopmental-disorders-including-rett-syndrome-and-multiple-sclerosis/
They also followed up a month or so later with: https://www.anavex.com/anavex-life-sciences-announces-publication-of-foundational-data-for-anavex2-73-blarcamesine-in-multiple-sclerosis-ms/
Yes, and likely part of the reason that AVATAR readout was pushed out from Q1 to 1H (along with Covid related delays to trial initiation in the UK — https://www.facebook.com/reverserett/posts/3421039837944917 )
EXCELLENCE was always a phase 2/3 trial — It was AVATAR that they recently announced the plan to change from a phase 2 into a pivotal phase 2/3.
Missling will be a Keynote Speaker at the Precision In Clinical Trials Virtual Summit (West Coast), Feb 24th-25th 2021. https://twitter.com/hashtag/VitualPCT?src=hash
More from Nadia Bodkin, one of the founders of RAM: https://www.rarerevolutionmagazine.com/blog/anavex-life-sciences-is-the-company-to-learn-from-according-to-the-rare-disease-advocacy-community
Dr. Randi J. Hagerman of the UC Davis MIND Institute, will be featuring Anavex 2-73 (among other potential therapies she is investigating) in her January 12th talk, The Fragile X Spectrum and new Targeted Treatments for FXS and ASD. Dr. Hagerman was the 2-73 Rett Syndrome trial Investigator at the UC Davis trial site: