Isis Pharmaceuticals announced 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.
"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.
"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.
There there's a person to give me the results of the press conference of the Isis 02/24/2014 ...?
ReplyDeleteContacting Isis Pharm would probably be the best option.
ReplyDeletehttp://www.isispharm.com/About-Isis/Contact-Us.htm