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Biogen (BIIB) Presents Positive Phase III Data on Spinraza

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Biogen Inc. (BIIB - Free Report) presented positive data from the phase III ENDEAR study evaluating Spinraza (nusinersen) for the treatment of spinal muscular atrophy (SMA).

Data from the study demonstrated a statistically significant reduction in the risk of death or permanent ventilation in infants with SMA when treated with Spinraza, compared to untreated infants.

Data were presented at the British Paediatric Neurology Association (BPNA) annual conference in Cambridge, UK, held on Jan 11–13.

Shares of Biogen have significantly outperformed the Zacks classified Medical-Biomedical/Genetics industry in last six months. The stock has gained 8.9% during the period, while the broader industry witnessed a drop of 4%.



In Dec 2016, the FDA approved Spinraza under Priority Review for the treatment of both pediatric and adult patients with SMA. This makes it the first and only treatment to be approved in the U.S. for SMA, allowing the product to tap a market with high potential.

The approval was based on positive results from multiple studies, including an interim analysis of the ENDEAR study in patients with infantile-onset SMA, which demonstrated sustained clinically meaningful improvement in motor function compared to the untreated study participants, as well as open-label data on pre-symptomatic and symptomatic patients with SMA, or who are likely to develop types 1, 2 and 3.

We note that Biogen licensed the global rights to develop, manufacture and commercialize Spinraza from Ionis Pharmaceuticals (IONS - Free Report) , a leader in antisense therapeutics.

We remind investors that Spinraza is currently under review in the EU. In Oct 2016, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) granted Accelerated Assessment to the product for this indication.

Biogen has also submitted regulatory filings for Spinraza in Japan, Canada and Australia. Regulatory filings in additional countries are expected in 2017.

SMA is estimated to affect about 30,000–35,000 patients in the U.S., the EU and Japan. In the U.S., one in every 50 individuals, or approximately six million people, carries the gene mutation that causes SMA.

Zacks Rank & Key Picks

Biogen currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the health care sector include Kite Pharma, Inc. and Enzo Biochem, Inc. (ENZ - Free Report) . Both the stocks carry a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Kite Pharma’s loss estimates narrowed from $5.60 to $5.48 for 2016 and from $6.86 to $6.85 for 2017 over the last 60 days. The company posted a positive surprise in two of the four trailing quarters with an average beat of 7.89%.

Enzo Biochem’s loss estimates narrowed from 17 cents to 16 cents for 2016 over the last 60 days. The company posted a positive earnings surprise in three of the four trailing quarters with an average beat of 22.50%.

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