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Isis Pharmaceuticals to Investigate a Higher Dose of ISIS-SMNRx in Children with Spinal Muscular Atrophy.

November 22, 2013.

Isis Pharmaceuticals, Inc. announced today that it plans to add a 12 mg cohort to the ongoing Phase 1b/2a study evaluating ISIS-SMNRx in children with spinal muscular atrophy (SMA). In addition, a 12 mg dose will be included in an open-label extension study for those children who have completed dosing in previous studies. The addition of the 12 mg cohort will allow for the investigation of this dose in support of the Phase 2/3 registration-directed program planned to begin next year.

Click here for the full press release: http://www.4-traders.com/ISIS-PHARMACEUTICALS-INC-9734/news/ISIS-Pharmaceuticals-Inc--Isis-Pharmaceuticals-to-Investigate-a-Higher-Dose-of-ISIS-SMN-Rx-in-Chi-17487793/

"We are pleased to be able to increase the dose of ISIS-SMNRx based on the safety and tolerability data we have seen to date in both infants and children with SMA. We have already begun the infant 12 mg dose cohort and look forward to sharing data from this study early next year. Considering the encouraging safety profile we have observed in children with SMA in the lower dose cohorts, the evaluation of the 12 mg dose will provide additional information as we move into our Phase 3 program. We are also pleased that after reviewing the data, the FDA agrees that we can proceed in our investigation of a higher dose," said B. Lynne Parshall, chief operating officer at Isis. "This addition of patients to the ongoing Phase 1b/2a study does not change our plan to report data on the first three cohorts by early next year."

The Phase 1b/2a study of ISIS-SMNRx is an open-label, multiple-dose, dose-escalation study designed to assess the safety, tolerability and pharmacokinetic profile of the drug in children with SMA between the ages of 2-15 who are medically stable. In the ongoing Phase 1b/2a study in children with SMA, all patients have completed dosing in the initial three dose cohorts (3 mg, 6 mg and 9 mg) and ISIS-SMNRx has been well tolerated over multiple doses. Patients who have participated in the Phase 1b/2a study are eligible to enter an open-label extension study, which is designed to provide a single additional dose of 12 mg to the more than 50 children with SMA who are eligible to roll over into this study. The investigation of the 12 mg dose in both studies is anticipated to begin in December or January.

ABOUT ISIS-SMNRx
ISIS-SMNRX is designed to alter the splicing of a closely related gene (SMN2) to increase production of fully functional SMN protein. The United States Food and Drug Administration granted orphan drug status and fast track designation to ISIS-SMN(Rx) for the treatment of patients with SMA. Isis is currently in collaboration with Biogen Idec to develop and potentially commercialize the investigational compound, ISIS-SMN(Rx), to treat all types of SMA. Under the terms of the January 2012 agreement, Isis is responsible for global development and Biogen Idec has the option to license the compound until completion of the first successful Phase 2/3 study.

Isis acknowledges support from the following organizations for ISIS-SMNRx: Muscular Dystrophy Association, SMA Foundation, Families of SMA and intellectual property licensed from Cold Spring Harbor Laboratory and the University of Massachusetts Medical School.

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