By Marta Falconi
Results from a late-stage trial showed that Novartis AG's (NVS)
drug Ilaris helped relieve symptoms in patients suffering from the
most severe form of childhood arthritis, the Swiss drug maker said
Wednesday, confirming plans to submit the drug to regulators for
this indication later this year.
The Phase III study showed that 62% of participating children
with a rare and serious auto-inflammatory disease called systemic
juvenile idiopathic arthritis, known as SJIA, became symptom-free
when treated with the drug, also known as ACZ885 or canakinumab,
against 32% of those taking a placebo after an initial treatment
with the drug. The study also showed that one-third of the patients
were able to stop taking steroids within five months and that
ACZ885 can extend the time to the next flare-up of the disease.
SJIA affects fewer than one child in every 100,000. These young
patients can face joint destruction and growth retardation, with
serious developmental and psychological consequences.
The Basel-based company, which will present the study's results
on Thursday at the annual congress of the European League Against
Rheumatism in Berlin, said ACZ885 regulatory submissions for use in
SJIA are "on track for 2012."
Ilaris is already approved in more than 60 countries for the
treatment of adults and children as young as four years suffering
from cryopyrin-associated periodic syndrome, a rare lifelong
auto-inflammatory disease with debilitating symptoms and few
treatment options.
A successful expansion of the drug's use would prove in line
with the company's drug development strategy as it aims to develop
drugs for rare diseases that can later be used to treat illnesses
that affect more people, thus helping sales and profit.
Novartis also said a separate Phase II trial showed that 90% of
patients with TRAPS, another rare auto-inflammatory disease,
experienced clinical remission after one week of treatment with
ACZ885. There is currently no approved treatment for TRAPS.
"We are committed to investigating new treatments that can
address the existing unmet medical need in immune-mediated
diseases, no matter how rare some of these conditions may be," said
John Hohneker, head of development for integrated hospital care for
Novartis's pharma division.
ACZ885 is a monoclonal antibody which works by blocking an
inflammatory protein called interleukin-1 beta, an important part
of the body's immune system defenses. Excessive production plays a
major role in certain inflammatory diseases, including SJIA and
TRAPS.
The positive results in both indications may add $200 million to
current sales forecasts of the drug of $80 million, which are
solely based on the approved indication of CAPS, said Zurich-based
analyst Karl-Heinz Koch with Helvea. "However, for Ilaris to become
a meaningful drug for Novartis, Phase III studies in
atherosclerosis would have to be positive, with results not
expected before 2014," said Koch, who has a Buy rating on
Novartis.
At 0934 GMT, Novartis shares were trading 0.5% higher at
CHF49.78.
Write to Marta Falconi at marta.falconi@dowjones.com