Positive long-term safety and efficacy data for Mycapssa.
Positive long-term safety and efficacy data for Mycapssa from the 2nd year of OPTIMAL open label extension study in acromegaly patients
Amryt presents long-term safety and efficacy data from the 2nd year open-label extension (OLE) of its global Phase III OPTIMAL clinical trial that compared Mycapssa (octreotide capsules) to placebo for maintenance of biochemical response in patients with acromegaly.
The OPTIMAL trial supported the approval of Mycapssa in the United States for long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with injectable octreotide or lanreotide.
OPTIMAL Phase III Trial– Open-Label Long-Term Safety & Efficacy Data: 40 patients that completed the 9 months double-blind placebo controlled (DPC) core treatment phase elected to continue treatment with Mycapssa in the OPTIMAL open label extension study (20 patients that were originally randomized to Mycapssa and 20 that were randomized to placebo). Results from the first year were published previously and demonstrated that all patients who responded to Mycapssa (IGF-1 within normal limits) during the DPC period and enrolled in the OLE (n=14) completed the 48-week period and 93% (13/14) maintained their IGF-1 response within the normal limit at the end of this period. 32 patients continued treatment into the 2nd year of the OLE (18 of those originally randomized to Mycapssa during the DPC and 14 of those randomized to placebo).
Key 2nd year study outcomes included: i. 31 out of 32 patients (97%) of those enrolled to the 2nd year of the OLE completed 96 weeks in the OLE period. ii. 100% of evaluable patients, who entered the 2nd year OLE phase of the study as responders (IGF-1 within normal limits; N=17), maintained their long-term biochemical response at the end of the study. The average IGF-1 levels of enrolled patients were stably maintained within the normal limits at the end of the OLE period (mean IGF-1 levels at baseline OLE and at the end of the OLE were 0.92 and 0.84 respectively). iii. 93% of all patients who entered the 2nd year OLE phase (N=32) were responders at the end of the 96 weeks OLE. iv. The average GH levels of enrolled patients improved at the end of the OLE period (mean GH levels at baseline OLE and at the end of the OLE were 0.79 and 0.45 respectively). v. Acromegaly patients were exposed to Mycapssa during the OPTIMAL study (including its OLE phase), for a median treatment duration of 2.1 years and a maximal exposure of 3.2 years. vi. Patients in the OLE demonstrated a median compliance rate of 98% over this period of time.
The long-term safety profile of Mycapssa during the OLE, was consistent with the safety profile observed during previous studies with Mycapssa with no new safety signals with long-term exposure.