It took four years for Chiasma to bring its acromegaly pill Mycapssa back from the dead, but in late June, the Needham, Massachusetts-based biopharma did just that, scoring a nod for what's now the first FDA-approved oral treatment for the rare organ and limb growth disorder.
With a full rollout planned for the year's final quarter, Chiasma is touting its rare disease pill as an easier alternative to the injectable standard-of-care, and now it has results from an extension study to pad its case.
After a 36-week, phase 3 trial that won Mycappsa (octreotide) its acromegaly green light, data from the first 48 weeks of its extension study are in: The drug kept subjects' limb and organ growth in check without any major safety deviations from the original late-stage test.
All patients who responded to Mycapssa in the phase 3 completed the 48-week regimen, with 93% of those subjects maintaining a similar response to the drug throughout that time. The average rate of growth hormones remained stable in all Mycapssa patients, and no new side effect patterns cropped up with longer-term use, the company said.
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The longer-term results could help Chiasma pitch the drug as a new standard treatment for acromegaly patients who depend on monthly injections of somatostatin analogs—such as Novartis' Sandostatin LAR Depot—to keep their disease under control.
The data "reaffirm our belief in the potential for [Mycapssa] to be the new standard of pharmacological care for patients with acromegaly who face significant challenges with their octreotide and lanreotide injectables,” CEO Raj Kannan said. The pill is given once or twice daily, depending on dosage.
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Chiasma has been laying the groundwork for a fourth-quarter launch, planning to target the small pool of endocrinologists currently treating the disease in the U.S. Roughly 90% of acromegaly patients are handled by just 1,000 physician practices, Chiasma said.
To reach those doctors, the company will pivot 45 of its existing sales staff to the Mycapssa team. It plans to offer support services—including remote doctor visits and mobile blood testing—to help patients obtain Mycapssa prescriptions. And Chiasma will offer services aimed at insurance providers, specialty pharmacies and patients to help them make the Mycapssa switch, the company said.