Isis Pharmaceuticals, Inc. (ISIS) has commenced a pivotal phase III study on ISIS-SMNRx for the treatment of children suffering from spinal muscular atrophy (SMA). With the initiation of this study, Isis Pharma earned a milestone payment of $27 million from its partner Biogen (BIIB).

The randomized, double-blind, sham-procedure controlled 15-month second phase III study (:CHERISH) will be conducted on approximately 120 non-ambulatory SMA children aged between 2 to 12 years. The study will evaluate the efficacy and safety of a 12 mg dose of ISIS-SMNRx.

Isis Pharma’s association with Biogen goes back to 2012 when the companies had entered into a collaboration agreement for the development and commercialization of ISIS-SMNRx for the treatment of SMA.

Meanwhile, Biogen intends to conduct two phase II studies on ISIS-SMNRx – NURTURE and EMBRACE. While NURTURE will evaluate ISIS-SMNRx in up to 25 pre-symptomatic newborns who are genetically susceptible to the disease, EMBRACE will evaluate the safety and exploratory efficacy of ISIS-SMNRx in approximately 20 patients with infantile or childhood-onset SMA. The studies are expected to begin in the first half of 2015.

We are encouraged by Isis Pharma’s progress with ISIS-SMNRx. As per the company, approximately 30,000–35,000 patients in the U.S., Europe and Japan suffer from SMA and about 6 million people in the U.S. alone are carriers of a defective SMN1 gene.

Currently, ISIS-SMNRx is also being developed for the treatment of infants suffering from SMA in the phase III ENDEAR study. Both ENDEAR and CHERISH are being conducted under the FDA’s special protocol assessment program.

We expect investor focus to remain on pipeline updates from the company.

Isis Pharma currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include AMAG Pharmaceuticals, Inc. (AMAG), ANI Pharmaceuticals, Inc. (ANIP) and Biogen. All three carry a Zacks Rank #1 (Strong Buy).

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