Octreolin demonstrated a PK profile similar to that of subcutaneously injected octreotide acetate. In addition, no serious adverse safety events were reported for Octreolin.
Chiasma said that it was advancing towards its goal of beginning a pivotal trial by the end of the year for Octreolin in acromegaly, a hormonal disorder that results from an excess of growth hormone (GH).
The Phase I trial was conducted in 12 healthy volunteers with a cross-over design in which subjects were each administered three single oral doses of Octreolin and a single subcutaneous injection of octreotide acetate as the reference drug (Sandostatin).
The primary objectives of the trial were to evaluate the safety, tolerability and PK of the three oral doses of Octreolin in comparison to the subcutaneous injection of octreotide acetate.
In the trial, Octreolin was well tolerated by all subjects, measurable drug levels were noted in all subjects at all 3 doses of Octreolin.
The trial results also noted a similar elimination half life (T(1/2)) of octreotide regardless of route of administration or dose.
Therapeutically relevant blood levels of Octreotide, known to induce maximal inhibition of GH in acromegaly patients, were achieved and were maintained for a longer time with Octreolin than after Sandostatin subcutaneous injection. The effective blood levels of octreotide (as measured by maximal GH inhibition) are well established clinically based on data from decades of octreotide injections.
The study outcomes support advancement of clinical development of Octreolin and allow dose selection for the pivotal study in which the clinical efficacy of Octreolin will be tested in acromegaly patients.
Chiasma has submitted an orphan drug application to the FDA for acromegaly and is expected to submit an application for orphan medicinal product designation to the European Agency for the Evaluation of Medicinal Products (EMEA) shortly. The company intends to file an NDA using the 505(b)(2) regulatory pathway in the US and its EMEA equivalent, the Hybrid Application, in Europe.
Shlomo Melmed, senior vice president, academic affairs & dean of the medical faculty at Cedars-Sinai Medical Center, said: “Chiasma has demonstrated achievement of an important first clinical step in the process of developing a safe and effective oral therapy for acromegaly patients who would prefer the convenience of oral therapy and the avoidance of the side effects associated with injectable forms of somatostatin analogs.”