|
 Alnylam shares rose more than 17 percent, to $142.16. Ionis
Pharmaceuticals Inc also presented data for a rival drug that while
positive appeared to pale in comparison to the Alnylam results, and
its shares fell more than 9 percent to $52.39.
The Alnylam drug, patisiran, was being tested against hereditary
ATTR (hATTR) amyloidosis with polyneuropathy, which affects an
estimated 50,000 people worldwide. It is being developed in
partnership with Sanofi.
When Alnylam announced in September that the trial succeeded, its
shares surged 51 percent.
If approved, patisiran is forecast to reach annual sales of $1
billion by 2023, according to Thomson Reuters data.
More than 90 percent of those who received patisiran infusion every
three weeks in the 225-patient trial experienced a halt in disease
progression, and 56 percent showed an improvement in their condition
after 18 months, the companies said.
"Patients with hATTR amyloidosis ... have a profound need for
effective and safe treatment options," Dr. David Adams, the study's
lead investigator, said in a statement. "As a clinician, it has been
deeply rewarding to see the potential impact patisiran may have on
the lives of hATTR patients."
On a neurological disease impairment scale, patisiran patients'
scores on average declined by 6 points, indicating improvement,
while scores of those who received a placebo worsened by 28 points,
data presented at a medical meeting in Paris showed.
The mean 34-point difference was deemed highly statistically
significant.
In addition, 51 percent of patisiran patients reported improvement
on a quality of life scale, with a mean 6.7-point improvement at 18
months versus a 14.4-point worsening for placebo recipients, a
21.1-point difference.
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In the study of the Ionis drug, inotersen, the mean difference
versus placebo on neurological impairment was 19.7 points, and for
quality of life 11.7 points, both after 15 months.
Leerink Partners analyst Paul Matteis said patisiran safety data
also looked superior to the Ionis drug. "We think the Alnylam data
are confirming to the current consensus view that patisiran will be
the market leader."
Patisiran belongs to a new class of medicines that employ RNA
interference, or RNAi, which prevents a defective gene from making
disease-causing proteins.
If unchecked, the gene mutation causes amyloid proteins to
accumulate in peripheral tissues of the nerves, gut and heart,
causing damage to organs and tissue and a wide range of debilitating
symptoms. Patients have a life expectancy of 2.5 to 15 years from
symptom onset.
The companies plan to file for U.S. approval by year end and in
Europe shortly after. It would be Alnylam's first approved medicine
and validation for the long-held promise of RNAi technology.
The most common patisiran side effects were peripheral edema and
infusion-related reaction that led to one patient discontinuation.
Of serious adverse side effects reported, only a case of diarrhea
was deemed related to patisiran.
(Reporting by Bill Berkrot; Editing by Dan Grebler and Tom Brown)
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