NEEDHAM, Mass.—A key issue in both clinical trials and regular disease management is patient compliance, particularly when it comes to treatments and therapeutics that are delivered via injection. Biopharmaceutical company Chiasma Inc. is one company working to address that stumbling block by offering more oral options, specifically with its Mycapssa (octreotide) capsules. Chiasma reported in August that data from its Phase 3 CHIASMA OPTIMAL (Ocetreotide capsules vs. Placebo Treatment In MultinationAL centers) clinical trial were published in the Journal of Clinical Endocrinology & Metabolism in a paper titled “Maintenance of acromegaly control in patients switching from injectable somatostatin receptor ligands to oral octreotide therapy.”
OPTIMAL was a randomized, double-blind, placebo-controlled trial assessing the safety and efficacy of Mycapssa capsules in acromegaly patients who had previously demonstrated biochemical control while receiving injectable somatostatin analogs (SSA), specifically octretide LAR or lanreotide. Fifty-six patients with biochemically controlled disease were enrolled and randomized to receive either Mycapssa or placebo, titrating up from 40 mg per day to a maximum of up to 80 mg per day. The trial met its primary and all secondary endpoints.
In patients who received Mycapssa, average IGF-1 levels were within the normal range (0.97 X upper limit of normal) at the end of treatment, compared to patients on placebo (1.69 X upper limit of normal).
Within the Mycapssa cohort, 77.7 percent of patients maintained growth hormone levels, compared to 30.4 percent in those on placebo. Seventy-five percent of Mycapssa patients successfully completed the trial without requiring reversion to their prior injectable treatment, and those that did revert re-established baseline response levels after just one dose. Mycapssa was well tolerated, with no new or unexpected safety signals observed. The paper notes that 18 patients discontinued treatment due to treatment failure in the placebo group, and five discontinued in the Mycapssa group. Treatment-emergent adverse events led to two discontinuations in the Mycapssa group and one in the placebo group.
Mycapssa received FDA approval as a long-term maintenance treatment for acromegaly patients in June, specifically for those patients who have responded to and can tolerate treatment with octreotide or lanreotide. The approval marks Mycapssa as the first and only oral somatostatin analog approved by the FDA.
“We are honored that the results from our Phase 3 CHIASMA OPTIMAL trial will be published in the Journal of Clinical Endocrinology & Metabolism,” said Dr. William Ludlam, senior vice president of clinical development and medical affairs at Chiasma. “This publication in a top-tier respected journal provides validation in the scientific community of our pivotal study results and the potential benefits Mycapssa could bring to patients with acromegaly [with] a novel, non-injectable oral option.”
Acromegaly is a condition in which a benign pituitary tumor produces too much growth hormone, resulting in side effects such as intense headaches, joint pain and impaired vision. If acromegaly progresses, it can result in health conditions such as type 2 diabetes, hypertension, respiratory disorders, and cardiac and cerebrovascular disease. While the preferred treatment is surgery to remove the tumor, not all patients are eligible for surgery and must rely on somatostatin receptor ligands (SRLs) instead.
Injectable SRLs require deep-tissue injection, and “can be associated with substantial treatment burden or deleterious long-term sequelae, including injection site pain, nodules, bruising, inflammation, and scarring,” according to the paper. In addition, while some patients can receive their injections at home, the authors report that only 17 percent of patients do so, and “With the majority of SRL injections occurring in the health care delivery setting, patients report a burden to their everyday life, including impacts from travel and administration time in addition to lost work.” According to Chiasma, roughly 8,000 patients with acromegaly are chronically treated with somatostatin analog injections in the U.S.
Patient compliance in the OPTIMAL trial was extremely high, “with a mean compliance of 98.2 percent in the OOC group and 96.9 percent in the placebo group,” the authors reported.
“Reflecting the positive experience of patients and their clinicians with OOC [oral octreotide capsules] treatments in this trial, 90% of patients receiving OOCs at the end of the DPC [double-blind placebo-controlled] period chose to remain on active treatment in an OLE [open-label extension] phase ... This pivotal placebo-controlled study indicates that OOCs may be an effective option in patients with acromegaly currently responding to injectable SRLs, while potentially avoiding side effects and compliance issues related to injectable regimens,” they concluded.