Oct 26 Previously untreated patients experienced
significant and lasting reductions in signs and symptoms of
psoriatic arthritis when given Celgene Corp's
experimental oral drug, apremilast, according to data from a
late stage clinical trial.
Patients taking either the 20 milligrams or 30 mg dose of
apremilast twice a day had a statistically significant
improvement in symptoms, such as painful, swollen joints, after
16 weeks of treatment compared with those who received a
placebo, researchers said.
Signs and symptoms of the painful disease continued to
improve on apremilast through a year of treatment, according to
data to be presented next week at the American College of
Rheumatology meeting in San Diego.
If approved, apremilast will compete with injected biotech
medicines that have proved to be highly effective, but have more
serious potential side effects, such as opportunistic infections
and tuberculosis because the biologics suppress the immune
"Psoriatic arthritis can be one of the most crippling types
of arthritis that we see," said Dr. Alvin Wells, the lead
investigator of the 527-patient study who will present the data
at the ACR meeting on Tuesday.
"To be able to have a patient that can take a pill twice a
day, I think this is going to be a game changer, not only for me
as a physician, but for my patients. They're going to have a new
treatment option," added Wells, director of the Rheumatology and
Immunotherapy Center in Franklin, Wisconsin.
The U.S. Food and Drug Administration is expected to make an
decision on the drug in March.
Celgene has said sales of apremilast could reach $1.5
billion by 2017. Wall Street analysts' projections have been
more modest, with RBC Capital Markets forecasting sales of $1
billion in 2017, while Cowen and Co sees annual sales reaching
$475 million in 2018.
The primary goal of the study dubbed Palace 4 was a 20
percent reduction in signs and symptoms of the disease, known as
ACR20, after 16 weeks versus placebo.
At that point, 29.2 percent of those who got the 20 mg dose
and 32.3 percent to took 30 mg achieved ACR 20, compared with
16.9 percent on placebo.
After 52 weeks on the drug, 53.4 percent on the lower dose
and 58.7 percent on the higher dose achieved an ACR20 response.
"These patients are on drug a long time so I want to see
that it's going to last," said Wells, calling the results "a
pretty dramatic response."
Placebo patients who did not respond early in the trial were
switched to apremilast. By week 24 there were no longer any
patients receiving placebo, Wells explained.
Researchers also looked at how many patients experienced
improvements of 50 percent and 70 percent.
At 52 weeks, for those on the higher dose of apremilast,
31.9 percent had achieved ACR50 and 18.1 percent ACR70,
About 125 million people worldwide suffer from the scaly
skin condition psoriasis and about 30 percent of them can
develop psoriatic arthritis, a chronic, progressive disease in
which joints become swollen and inflamed.
Apremilast is the first drug in a new class of medicine
called PDE4 inhibitors.
"It inhibits an enzyme involved in making all these angry
proteins that get into the skin, causing psoriasis, and they
also get into the joints, causing arthritis," Wells said.
The most common side effects with apremilast were diarrhea,
nausea and headache. No more serious adverse side effects were
reported, and fewer than 2 percent of patients in the study
discontinued treatment due to side effects.