The search for an Alzheimer’s disease treatment has been
unsuccessful for the past 14 years.
The latest failure came in September, when Axovant said it failed
a key late-stage trial for its drug, intepirdine.
Alzheimer’s affects more than 5 million Americans, a
number that’s expected to balloon
13.8 million by
. There are only four drugs
that have been approved to treat the symptoms of the disease, and
the most recent drug approval happened in 2003.
2017 in particular has been a
tough year for Alzheimer’s failures. In February alone,
Lundbeck discontinued two of its trials, Merck
discontinued one of its studies, and Accera
failed a late-stage trial as well. Axovant’s trial
results are the last major results to come out in 2017, with the
next ones reading out in 2018 and a major wave of results
expected in 2019.
Research has determined that years — even decades — before a
person might start showing symptoms, amyloid beta deposits in the
brain that are characteristic of Alzheimer’s
start to accumulate. By the time we start seeing
symptoms of dementia, researchers think, it might already be too
late to do much.
So preventing any progression at that stage is something
researchers are pinning a lot of hope on. There’s one
research effort going
on in Colombia that’s testing out an amyloid-related
drug in an extended family with a rare genetic mutation that
leads to early-onset Alzheimer’s. That study is in people who are
still considered cognitively healthy, so if the drug is able to
prevent cognitive decline, it could be a breakthrough.
According to PhRMA, there are more than a dozen experimental
treatments that are still in
phase 3, the latest stage of clinical trials before companies
present their data to the FDA. Here are some of the ones to look
out for in the next year or two.
- VTV Therapeutics, a small company in
North Carolina, has an Alzheimer’s drug in late-stage
trials called azeliragon that inhibits the RAGE
receptor, ideally helping people with mild Alzheimer’s
delay cognitive decline. Its phase 3 trial is expected to wrap
up in early 2018. The company got encouraging phase 2 results
on the drug, which has been in the works for about 17 years.
Even so, there’s no guarantee it will succeed. “We are doing a sound and rigorous
experiment,” Dr. Larry Altstiel, VTV’s chief medical
officer told Business Insider.
- Biogen’s aducamumab, a BACE
inhibitor, is going after
hypothesis,” or the idea that targeting beta amyloid
deposits in the brain to clear them out is the way to go
about treating the disease. It’s expected to have results in
2019 or early 2020.
- Lanabecestat, AstraZeneca’s BACE
inhibitor, is going be reading out in 2019. Like
Biogen, it’s going after the amyloid hypothesis.
- Eli Lily’s solanezumab
failed a phase 3 trial in patients with mild dementia
in November 2016, but has plans to keep trying the drug in
pre-clinical stages of the disease to see if it works
preventatively. Solanezumab is going after the amyloid
hypothesis as well. While three trials have been disconnected,
the fourth in this preventive setting is expected to have
results in 2022.
- In February,
drugmaker Merck stopped its
late-stage trial of verubecestat in patients with mild to
moderate Alzheimer’s, after a committee found that there was
“virtually no chance of finding a positive clinical
effect.” The hope was to have the drug — a BACE inhibitor
— stop the disease from progressing. Merck said it’s still
working on another late-stage trial for the drug to treat
people with even earlier stages of the disease, and those
results are expected in 2019.
- Genentech has two Alzheimer’s drugs
in late-stage development, despite hitting
setbacks. In February, Genentech and its partner AC Immune
launched a phase 3 trial for crenezumab,
another drug that’s targeting amyloid deposits in the brain.
It’s expected to have data in 2020. The other is gantenerumab,
a drug also targeting amyloid that failed earlier trials. The
hope is that by
increasing the dose, it might work. Genentech started a new
phase 3 trial for gantenerumab in 2017.