Isis Pharmaceuticals, Inc. ISIS announced the commencement of an open-label extension study (SHINE) on ISIS-SMNRx in infants and children suffering from spinal muscular atrophy (SMA). With the initiation of this study, Isis earned a milestone payment of $11 million from its partner Biogen Inc. BIIB.
In the extension study, infants and children with SMA who have completed participation in the controlled portion of either of the phase III studies on ISIS-SMNRx – ENDEAR and CHERISH – are eligible to participate.
We note that Isis is developing ISIS-SMNRx under a collaboration agreement with Biogen. Currently, Isis is evaluating ISIS-SMNRx in two phase III studies – ENDEAR and CHERISH – for the treatment of infants and children suffering from SMA, respectively. Data from both studies is expected in late 2016 or early 2017. Isis is also evaluating ISIS-SMNRx in a phase II open-label study in infants and a phase II open-label extension study in children.
On the other hand, Biogen is conducting two additional phase II studies on ISIS-SMNRx – NURTURE and EMBRACE.
We are encouraged by Isis’ progress with ISIS-SMNRx. Per the company, approximately 30,000–35,000 patients in the U.S., Europe and Japan suffer from SMA (type I being the most severe form) and about 6 million people in the U.S. alone are carriers of a defective survival motor neuron 1 gene. ISIS-SMNRx enjoys Fast Track and orphan drug status in the U.S. for SMA.
We note that Isis has partnership agreements with several leading health care companies including Johnson & Johnson JNJ and GlaxoSmithKline plc GSK among others. The company’s agreements with these companies not only validate its antisense technology but also provide it with funds in the form of upfront, milestone and other payments.
Meanwhile, Isis has several candidates in mid-stage clinical development that could trigger future licensing opportunities. We expect investor focus to remain on pipeline updates from the company.
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