Last week’s negative FDA advisory committee vote may be the final nail in the coffin in the up-and-down registration path of Biogen (NASDAQ:BIIB) and collaboration partner Eisai’s aducanumab (formerly BIIB037) for the treatment of patients with mild Alzheimer’s disease (AD).
This is the latest setback for therapies targeting the underlying pathology of AD, a notoriously treatment-resistant neurodegenerative disorder. The advisory committee voted 8-1 (2 uncertain) that the Phase 3 EMERGE study, in which the highest dose achieved the primary endpoint, was insufficient to demonstrate efficacy.
As you can see from the chart above, the news cost Biogen $4 billion in market cap. Even big pharma’s top cheerleader, Adam Fraudstein couldn’t spin the news (which tells you how bad it is for Biogen in that Fraudstein has never, ever before said anything bad about BP).
So… going into today, I thought there was little that the $BIIB FDA panel could say or do to stop FDA from approving aducanumab.
After listening to that panel for 7 hrs, I have flipped. I was wrong. I can’t see how FDA approves the drug now.
— Adam Feuerstein 💉 (@adamfeuerstein) November 6, 2020
We all knew last year that aducanumab didn’t work, but Biogen wouldn’t give up. Biogen was working behind-the-scenes with scientists, regulators, and statisticians to give aducanumab another go. It should come as no surprise to anyone following the sector.
Where Biogen Got It Wrong
Aducanumab was derived from human B cells collected from healthy elderly people with no symptoms of cognitive impairment and from cognitively impaired seniors with unusually slow clinical decline. Screening the libraries of human memory B cells for reactivity against beta-amyloid led to aducanumab, a monoclonal antibody that binds to beta-amyloid aggregates.
Ridding the brain of these aggregates, commonly called amyloid plaques, has been an AD drug target for many years. All efforts to date, including those by members of Big Biopharma including Eli Lilly, and Johnson & Johnson, have come up empty, fueling the debate on whether amyloid plaques are a product of AD rather than the cause.
The thing is, we at Insider Financial knew this was going to happen. We have been saying time and time again that Biogen, Eli Lilly, and Johnson & Johnson have been barking up the wrong tree the whole time. Here is what we said last month in our write-up on Anavex Life Sciences (NASDAQ:AVXL), which you can read here.
We believe ANAVEX 2-73 is the most promising drug being developed right now for Alzheimer’s. ANAVEX 2-73 is based on oxidation and nitration causing Alzheimer’s. Other drug companies have instead focused on the cause being amyloid. They have misdiagnosed the cause while we believe Anavex has the correct approach.
Anti-amyloid and anti-tau drugs have proven to only slightly slow down the progression of Alzheimer’s disease. The prevention or removal of either has shown little effect on the progression of the disease.
Inflammation, tau, and amyloid all add to oxidative stress but are themselves the products of this stress. The key then is not only to inhibit oxidative stress (which only moderately slows down the progression of the disease over time), but to remove compounds that are causing oxidation and nitration, and reverse part of the damage that they are doing to the brain.
Oxidation and nitration also likely play a role in Parkinson’s Disease Dementia and Rett syndrome. This is why we believe Anavex 2-73 is the most promising drug being developed today.
Anavex Life Sciences Update
On November 6th, Anavex Life Sciences reported additional details on and presented the results from the proof of concept Phase 2 controlled trial evaluating the safety, tolerability, and efficacy of ANAVEX®2-73 (blarcamesine) in patients with Parkinson’s disease dementia (PDD) at the 13th international conference on Clinical Trials on Alzheimer’s Disease (CTAD).
The study found that ANAVEX®2-73 (blarcamesine) 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 (blarcamesine), confirming that ANAVEX®2-73 (blarcamesine) acts through SIGMAR1 activation. These results support continued development in PDD / PD as well as the currently ongoing Phase 2 and Phase 2/3 clinical studies with ANAVEX®2-73 (blarcamesine) in Rett syndrome and Alzheimer’s disease.
Highlights of the study results:
- 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.
- 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).
- ANAVEX®2-73 (blarcamesine) does not impair sleep and has a positive effect on REM sleep behavior disorder.
- ANAVEX®2-73 (blarcamesine) was generally safe, well-tolerated, and improved safety profile compared to dementia drugs associated with typical adverse effects.
The results were excellent, especially considering there are no other drugs on the market or horizon that can do what A2-73 does for PDD suffers. I think the big surprise will be how well the drug performed for the secondary PD results, hopefully, this will all be revealed in the upcoming Parkinson Conference on November 16-17, 2020, a short wait. Here are the highlights of the presentation:
- ANAVEX®2-73 (blarcamesine) is delivered via oral capsules, which does not require a hospital visit or specialized equipment.
- Significant improvements in episodic memory with increased dose. Notice the improvement compared to placebo.
- Data indicates that ANAVEX®2-73 (blarcamesine) does not impair sleep and has a positive effect on REM sleep behavior disorder. REM sleep is when the body repairs and regrows tissues, builds bone and muscle, and strengthens the immune system.
- Data will be submitted to the FDA to seek regulatory guidance.
Overall, the data was positive, but we are still playing the waiting game. AVXL stock sold off on the news for two reasons:
- Misinformed investors sold because of the bad news from Biogen. Again, if anyone sold because of this, they are wrong. It’s apples and oranges.
- Investors keep expecting more with each data release. The key to remember is that ANAVEX®2-73 (blarcamesine) keeps showing improvement when compared to placebo. That is what’s important.
Why Biogen Should Buy Anavex Life Sciences
Biogen needs a win. Alzheimer’s is the Holy Grail. It’s worth a fortune as there is no effective treatment on the market for Alzheimer’s. We believe ANAVEX®2-73 (blarcamesine) has the most potential and why it would make sense for Biogen. Biogen has a market cap of $50 billion and over $3 billion in cash. Anavex, on the other hand, has a market cap of just $496 million.
We have said repeatedly that most biotechs and researchers have missed the root cause of Alzheimer’s and as a result, have approached treatment in the wrong manner. Anavex is the only biotech with a pathway for a cure. With or without Biogen, 2021 and beyond is looking big for Anavex Life Sciences. The big payday many are banking on might just be around the corner.
As always, good luck to all (except the shorts)!
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Disclosure: We have no position in NASDAQ:AVXL and NASDAQ:BIIB, nor any of the securities mentioned. We wrote this article ourselves and it expresses our own opinions. We are not receiving compensation for it. We have no business relationship with any company whose stock is mentioned in this article. Insider Financial is not an investment advisor and does not provide investment advice. Always do your own research and make your own investment decisions. This article is not a solicitation or recommendation to buy, sell, or hold securities. This article is meant for informational and educational purposes only and does not provide investment advice.