Isis Pharmaceuticals, Inc.
) announced that the Committee for Orphan Medicinal Products
(COMP) has given a positive opinion regarding orphan drug status
for ISIS-TTRRx in the EU for the treatment of ATTR-Amyloidosis.
The European commission (EC) will review the recommendation
before granting the status to the candidate.
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ISIS-TTRRx, an antisense drug, is being developed by Isis Pharma,
in collaboration with
). Isis Pharma said that ISIS-TTRRx is currently in a phase
II/III study for the treatment of patients suffering from
familial amyloid polyneuropathy.
Meanwhile, Isis Pharma is also making progress with its spinal
muscular atrophy (SMA) candidate, ISIS-SMNRx.
The company said that a 12 mg dose of ISIS-SMNRx was administered
to the first child in an ongoing phase II study being conducted
in children with SMA. Isis Pharma also said that dosing in a
phase II study being conducted in infants with SMA is being
extended so that infants who have completed the first three doses
(3 mg, 6 mg and 9 mg) can move on to the 12 mg dose. The company
said that the first infant has been administered the fourth dose.
Based on these milestones, Isis Pharma is eligible to receive
$9.3 million from partner Biogen.
Top-line results from multiple-dose studies in infants with type
I SMA and in children with type II and type III SMA are expected
in the first quarter of 2014.
We remind investors that in Jan 2012, Isis Pharma had stuck a
) for the development of ISIS-SMNRx. Altogether, Isis Pharma and
Biogen have four collaboration agreements - one for ISIS-SMNRx,
the second for the development of antisense drugs to treat
myotonic dystrophy type 1, a third for the discovery and
development of antisense drugs against three novel targets for
neurological disorders and the final agreement for the use of
Isis Pharma's antisense technology for the development of
treatments for neurological diseases
Isis Pharma carries a Zacks Rank #3 (Hold). A better-ranked stock
in the biotech sector is
Alexion Pharmaceuticals, Inc.
) carrying a Zacks Rank #1 (Strong Buy).