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Yesterday, Biogen Inc. (BIIB - Free Report) and partner Ionis Pharmaceuticals, Inc. (IONS - Free Report) saw their shares soar on positive interim data from a late-stage study on Spinraza, their investigational treatment for spinal muscular atrophy (SMA). The companies said that Spinraza met the primary endpoint in the phase III CHERISH study for later-onset SMA. The results were statistically significant and clinically meaningful with a favorable safety profile and the study will be stopped early.

These are the second set of positive late-stage results to be presented by Biogen and Ionis on Spinraza and support the potential to use Spinraza across a wide range of SMA patients.

Spinraza is currently under FDA and EMA review with Biogen expecting a launch in the U.S. as early as the end of this year or the first quarter of 2017. While Biogen gained 6.7% on the interim results, Ionis’ shares were up 18.4%.

IONIS PHARMACT Price, Consensus and EPS Surprise

What is SMA?

SMA is a hereditary neuromuscular disease that progressively destroys lower motor neurons leading to severe and progressive muscular atrophy and weakness. The most severe type of SMA could result in paralysis and difficulty in performing basic functions like breathing and swallowing.

Market Potential

According to the SMA Foundation, SMA is believed to affect 10,000 - 25,000 children and adults in the U.S., making it one of the most common rare diseases. About 1 in 6,000 - 1 in 10,000 children are born with the disease while 1 in 40 - 1 in 50 people (approximately 6 million Americans) carry the gene mutation that causes SMA. SMA is divided into sub-categories Type 1 - 4, with Type 1 being the most severe.

At present, there is no cure for SMA and treatment involves managing the symptoms and preventing complications.

If all goes well, Spinraza could be the first therapy for SMA. Spinraza has blockbuster potential given the lack of FDA approved treatments in the market and enjoys oprhan drug status in both the U.S. and the EU.

BIOGEN INC Price, Consensus and EPS Surprise

Other SMA Treatments in Development

While it looks like Biogen and Ionis will most likely enjoy a head start in the SMA market, quite a few other companies are working on bringing SMA treatments to market.

AveXis, Inc. (AVXS - Free Report) , a clinical-stage gene therapy company, is looking to commence a pivotal study on AVXS-101 for SMA Type 1 in the first half of 2017. AveXis’ shares shot up 34.8% in November following a positive Type B meeting with the FDA related to AVXS-101. The company said that the pivotal study will reflect a single-arm design, using natural history of the disease as a comparator.

Moreover, the FDA has advised the company to seek an end-of-phase I meeting once an ongoing phase I study completes. The meeting could include a discussion as to whether the phase I data would be enough to support a marketing application. AveXis is a Zacks Rank #2 (Buy) stock. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

AVEXIS INC Price, Consensus and EPS Surprise

PTC Therapeutics, Inc. (PTCT - Free Report) has a joint development program for SMA with Roche Holding AG (RHHBY - Free Report) and the SMA Foundation -- RG7916, an oral small molecule survival motor neuron 2 (SMN2) splicing modifier, is in a phase II study in pediatric and adult Type 2/3 patients. The pivotal second part of the study is expected to start next year while a similarly designed two-part study in Type 1 patients is expected to commence in the coming months.

PTC THERAPEUTIC Price, Consensus and EPS Surprise

Roche is also working on developing olesoxime (RG6083), a small molecule neuroprotectant that preserves mitochondrial function, for SMA with a regulatory filing currently not expected before 2019.

ROCHE HLDG LTD Price, Consensus and EPS Surprise

South San Francisco, CA-based Cytokinetics, Inc. (CYTK - Free Report) is working with Astellas to develop CK-2127107, a fast skeletal troponin activator, for SMA. CK-2127107 is currently in a phase II study with data expected in the first half of 2017.

CYTOKINETCS INC Price, Consensus and EPS Surprise

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