Isis Pharmaceuticals Inc. recently announced that it has received a milestone payment of $3.5 million from its partner, Biogen Idec Inc. (BIIB - Free Report) . The milestone payment was triggered by dosing of the first patient in a phase II study evaluating ISIS-SMNin infants suffering from spinal muscular atrophy (SMA).
The open-label, multiple-dose, dose-escalation pilot phase II study will be conducted in eight infants (between three weeks and seven months) for the treatment of SMA. ISIS-SMN enjoys orphan drug as well as fast track status with the US Food and Drug Administration (FDA).
We remind investors that in Mar 2013, data from an open-label phase I study evaluating ISIS-SMN in children with SMA was presented. Results showed that ISIS-SMN was well tolerated at all the evaluated dose levels. Additionally, improvements in Hammersmith scores were also observed.
Isis Pharma is also evaluating ISIS-SMN in a multiple-dose phase Ib/2a study. The company expects to report data from this study by late 2013 or early 2014. Isis Pharma intends to initiate phase II/III studies on ISIS-SMN in infants in 2013 and children in the first half of 2014.
We note that as per the terms of the Isis Pharma-Biogen agreement, inked in Jan 2012, Isis Pharma is responsible for the global development of ISIS-SMN through the completion of phase II/III registrational studies, with input from Biogen. The latter will take over global development, regulatory and commercialization responsibilities if it decides to exercise its option. Biogen has the option to license ISIS-SMN until completion of the first successful phase II/III trial.
Isis Pharma currently carries a Zacks Rank #2 (Buy). Stocks such as Lannett Company, Inc. (LCI - Free Report) and Alkermes plc (ALKS - Free Report) look equally attractive with a Zacks Rank #2.