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Isis Pharmaceuticals, Inc. (ISIS - Analyst Report) 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.

ISIS-TTRRx, an antisense drug, is being developed by Isis Pharma, in collaboration with GlaxoSmithKline (GSK - Analyst Report). 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 deal with Biogec Idec (BIIB - Analyst Report) 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. (ALXN - Analyst Report) carrying a Zacks Rank #1 (Strong Buy).

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