ENDO 2022 saw the presentation of positive clinical data from the open-label extensions (OLEs) of two of the Phase 3 trials of oral octreotide for patients with acromegaly. Amryt is marketing the drug as Mycapssa.
Data presented in a late-breaking poster presentation of the 2nd year of the OLE of OPTIMAL (NCT03252353), a randomized, double-blind placebo-controlled (DPC) trial, further support the long-term safety and efficacy of Mycapssa in acromegaly patients who were previously biochemically controlled on monthly injectable somatostatin receptor ligands (iSRLs).
Susan L. Samson, MD, PhD, FRCPC, FACE, chair of Endocrinology at the Mayo Clinic in Jacksonville, Fla., and lead investigator of the OPTIMAL study, tells Endocrine News that this oral formulation has provided another option for patients who already responded to injectable therapies, but as a clinician her focus was on whether her patients would have sustained success on the oral medication. “That’s what was really key about the data presented this year at ENDO 2022 was looking at an open label extension, which wasn’t just a short period of time,” she says. “This was 96 weeks. We had patients exposed for drug for I think it was a median of two years, some patients a handful up to three.”
Data highlights from the late-breaking poster presentation titled, “Second Year Outcomes of the Open-Label Extension of OPTIMAL, a Phase 3 Study of Oral Octreotide Capsules in Acromegaly,” include:
• Maintenance of biochemical response (defined as insulin-like growth factor I [IGF-I] levels of less than the upper limit of normal [ULN]) to Mycapssa was durable up to 96 weeks. 100% of subjects (n= 17) who were responders at week 48 and 93% of subjects overall (n=29) demonstrated a biochemical response at week 96.
• Median exposure to Mycapssa was 2.1 years, with exposure >3 years for 5 patients.
• Mycapssa’s safety profile was consistent with previous studies throughout the OLE; no serious adverse events were reported.
Samson says that next she would like to see how oral octreotide works in combination therapy (one of the arms of the MPOWERED trial is looking at cabergoline with oral octreotide capsules). “Also, I think some of us might like to see not just how the biochemistry is controlled, but what happens with the actual tumor, and we don’t have that data right now,” she says.
For now, Samson says that these findings mean she can be assured that patients who fit the criteria for oral treatment of their acromegaly will maintain control over the disease for the long term. “It was really exciting to see that those patients coming into that second year of those that were responders 100% maintained their response,” she says. “For all of us in endocrinology that treat patients with acromegaly, this is a really exciting option for our patients beyond what we’ve had in the past. It adds to our armamentarium, it’s safe, it has durable effect in our patients to respond, and I think that’s a really positive thing.”