Isis Pharmaceuticals, Inc. (ISIS) announced that it has earned a milestone payment of $7 million from its partner Biogen (BIIB). The milestone payment was related to the advancement of the ongoing open-label extension study on ISIS-SMNRx in children suffering from spinal muscular atrophy (SMA).
The open-label extension study is evaluating a 12 mg dose of ISIS-SMNRx in children with SMA every six months over the course of the study period.
We remind investors that in Nov 2014, Isis Pharma had started a pivotal phase III study (CHERISH) on ISIS-SMNRx for the treatment of children with SMA. Biogen had then announced its intention of conducting two additional phase II studies on ISIS-SMNRx – NURTURE and EMBRACE.
Currently, ISIS-SMNRx is also being studied for the treatment of infants suffering from SMA in the phase III ENDEAR trial. Both ENDEAR and CHERISH are being conducted under the FDA’s special protocol assessment program.
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.
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 now expects to end 2014 with cash of more than $725 million, exceeding its initial cash guidance by over $150 million.
We note that the company recently inked an agreement with Johnson & Johnson (JNJ) for the discovery and development of antisense drugs for treating autoimmune disorders of the gastrointestinal tract.
Isis Pharma currently carries a Zacks Rank #3 (Hold). A better-ranked stock in the health care sector is Biogen carrying a Zacks Rank #2 (Buy).
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