The randomized, double-blind, sham-procedure controlled 13-month phase III study is being conducted on approximately 110 infants suffering from SMA. The study will evaluate the efficacy and safety of a 12 mg dose of ISIS-SMNRx with survival or time to permanent ventilation being the primary endpoint.
Currently, ISIS-SMNRx is also being studied for the treatment of children suffering from SMA in the phase III CHERISH trial. Both ENDEAR and CHERISH are being conducted under the FDA’s special protocol assessment program. The phase III program on ISIS-SMNRx is expected to be completed in 2016.
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. Apart from ISIS-SMNRx, other candidates under their collaboration include ISIS-DMPKRx (myotonic dystrophy type 1) and two neurodegenerative disease candidates – ISIS-BIIB3Rx and ISIS-BIIB4Rx.
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 survival motor neuron 1 gene.
Meanwhile, Isis Pharma continues to progress with its pipeline and earn milestone payments for the same. Owing to its partnership with several leading health care companies including GlaxoSmithKline plc GSK among others, the company recorded revenues of $214.2 million in 2014, out of which nearly two-thirds of revenues came from milestone payments.
For 2015, the company expects revenues of more than $220 million including amortization of upfront fees and milestone payments.
Isis Pharma currently carries a Zacks Rank #2 (Buy). A better-ranked stock in the health care sector is Theravance Inc. THRX carrying a Zacks Rank #1 (Strong Buy).
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