Stocks of small pharmas developing new drugs for Alzheimer’s disease sank on Thursday

Key Points​

  • Clinical-trial data that Annovis Bio characterized as successful was actually more than a little disappointing.
  • Anavex Life Sciences and Cassava Sciences haven't presented any new clinical-trial data for their Alzheimer's disease programs, but they're falling, too.

What happened​

Shares of Annovis Bio(NYSEMKT:ANVS) are getting hammered in response to disappointing data for the company's Alzheimer's disease candidate. The biotech stock was down 59.4% at 2:45 p.m. EDT.
Annovis Bio's losses have spread to other clinical-stage biotech companies with Alzheimer's disease programs of their own. Shares of Anavex Life Sciences(NASDAQ:AVXL) and Cassava Sciences (NASDAQ:SAVA) were down 14.3% and 20.3%, respectively.
 

So what​

Leading up to yesterday's presentation at the Alzheimer's Association International Conference (AAIC), Annovis Bio tried to convince investors its new drug candidate for Alzheimer's disease and Parkinson's disease could be more effective at improving cognition than Aduhelmfrom Biogen. Annovis Bio stock imploded today because data from 14 Alzheimer's disease patients wasn't nearly sufficient to make such a claim.

Caregiver with an Alzheimer's disease patient.

IMAGE SOURCE: GETTY IMAGES.
After 25 days of treatment, cognition scores, as measured by the ADAS-Cog11 test, improved by 30%, compared to these patients' baseline scores. As is often the case in neuroscience studies, the placebo group also improved. This resulted in a difference between patients who received Phosiphen and the placebo group that wasn't statistically significant.

Cognitive abilities, as measured by the mini-mental state examination (MMSE) after 25 days, were more discouraging. Parkinson's disease patients treated with Posiphen showed no improvement, while the placebo group improved slightly. Both groups of Alzheimer's disease patients showed some improvement on the MMSE, but the placebo group improved slightly more than those given Posiphen.


Alzheimer's disease and Parkinson's disease progress too slowly to expect measurable cognitive improvements in just 25 days, but that didn't stop Annovis from trying. Today's flop was a harsh reminder that Anavex Life Sciences has been testing its lead candidate, blarcamesine, in different forms of dementia for over a decade, and the company still hasn't shown evidence that it significantly reduces cognitive decline for Alzheimer's disease patients, compared to a placebo.

In June, Cassava Sciences told investors its lead candidate simufilam improved ADAS-Cog11 scores by just 18%, compared to baseline after nine months of treatment. Since this was an open-label study, we can't say it wouldn't have been significantly better than a placebo group.
 

Now what​

Annovis Bio's results today probably wouldn't have caught any investors off guard if they had done some homework. The company in-licensed Posiphen all the way back in 2008, so it's already had plenty of time to shine in an early-stage dementia study.

As you can imagine, Annovis Bio isn't throwing in the towel on Posiphen just yet. The company has finished treating 40 Parkinson's disease patients and should be ready to share the results in about two months. Given Posiphen's lack of success so far, its outlook is downright lousy.


Simufilam from Cassava Sciences showed impressive biomarker data in June. Unfortunately, actual patient outcomes, as measured by cognitive ability tests, are what matters most.

Blarcamesine from Anavex Life Sciences produced arguably successful clinical-trial data in an open-label phase 2 Alzheimer's disease trial five years ago. Today, the company reminded investors that it's still planning a placebo-controlled phase 3 trial to confirm the results.

When it comes to biotechnology investing, it's important to understand that companies rarely just come out and say something isn't going well. The appearance of foot dragging doesn't necessarily mean a drug is doomed to fail. With failure rates in this industry so high, though, biotechs that appear to be dragging their feet aren't worth the risk.
 
ginfreely said:
In June, Cassava Sciences told investors its lead candidate simufilam improved ADAS-Cog11 scores by just 18%, compared to baseline after nine months of treatment. Since this was an open-label study, we can't say it wouldn't have been significantly better than a placebo group.
Click to expand...
An open-label trial, or open trial, is a type of clinical trial in which information is not withheld from trial participants. In particular, both the researchers and participants know which treatment is being administered.

Open-label trial - Wikipedia​

 
What is blinded clinical trial?


A blinded (or masked) clinical trial is a field study of a drug in which the recipient does not know if he is receiving the actual drug versus a placebo.

Blinded Clinical Trial - SEER Training​

 
What is a single blinded trial?


Listen to pronunciation. (SING-gul-blind STUH-dee) A type of clinical trial in which only the researcher doing the study knows which treatment or intervention the participant is receiving until the trial is over. A single-blind study makes results of the study less likely to be biased.

Definition of single-blind study - NCI Dictionary of Cancer Terms ...​

 
Why do double blind trials?


A double-blind study is one in which neither the participants nor the experimenters know who is receiving a particular treatment. This procedure is utilized to prevent bias in research results. Double-blind studies are particularly useful for preventing bias due to demand characteristics or the placebo effect.

What Is a Double-Blind Study? - Verywell Mind​

 
ginfreely said:
An open-label trial, or open trial, is a type of clinical trial in which information is not withheld from trial participants. In particular, both the researchers and participants know which treatment is being administered.

Open-label trial - Wikipedia​

Click to expand...
When is an open label trial used?


Open-label trials can be used to compare treatments or gather additional information about the long-term effects in the intended patient population.24 Jun 2019

Understanding Clinical Trial Terminology: What is an Open ...​

https://www.concertpharma.com › understanding-clinic...
 
Naturally, in open-label trials in anticoagulation there is a risk of a reporting bias of adverse events. Patients may research the new drug and its side-effects in publications and may be influenced in their reporting behaviour of potential side-effects.

External and internal validity of open label or ... - Wiley Online Library​

 
Are open-label studies blinded?


Many trial designs do not permit blinding, and are therefore designed as open-label, with patients, clinicians, and other study investigators aware of treatment allocation. Research has suggested that these trials should use blinded outcome assessment to avoid bias in estimated treatment effects [6–10].21 Nov 2014

Reducing bias in open-label trials where blinded outcome ...​

https://trialsjournal.biomedcentral.com › articles
 
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