Sparrow Pharmaceuticals Introduces Open-label Extension in RESCUE Phase 2 Trial of SPI-62 for ACTH-dependent Cushing’s Syndrome, Enrolling First Patient
Thursday, August 17, 2023
Sparrow Pharmaceuticals has made a noteworthy announcement regarding its progress in tackling Cushing's syndrome, a challenging medical condition. The company has entered the open-label extension (OLE) phase of the RESCUE trial, a phase 2 clinical trial focused on evaluating SPI-62—a potent and selective HSD-1 inhibitor—as a potential treatment for ACTH-dependent Cushing's syndrome. This marks an important step as the first patient has chosen to enroll in this extension phase.
Cushing's syndrome can be life-threatening and is often resistant to conventional treatments, especially when surgery proves ineffective. While medical therapies are being explored, they come with limitations. Sparrow's SPI-62 presents a novel approach by inhibiting HSD-1, which could help reduce cortisol levels in crucial organs and alleviate the negative effects of excessive cortisol—an approach not seen in Cushing's treatment for a long time.
The RESCUE trial focuses on evaluating the effects, efficacy, and safety of SPI-62 in patients diagnosed with ACTH-dependent Cushing's syndrome. By inhibiting HSD-1, SPI-62 is expected to decrease cortisol levels and mitigate the adverse effects associated with high cortisol levels in the body. The move to the OLE phase is a significant step forward, enabling researchers to gather valuable data on the long-term safety and effectiveness of SPI-62.
Sparrow is actively enrolling participants for the RESCUE trial at multiple sites in the US, Bulgaria, and Romania. Eligible participants include individuals aged 18 and above with consistent cortisol excess and a confirmed diagnosis of ACTH-dependent Cushing's syndrome.
Cushing's syndrome results from an excess of glucocorticoids, often caused by a pituitary tumor that elevates adrenocorticotropic hormone levels or by non-pituitary tumors. SPI-62 targets intracellular cortisol activation in key organs, offering a potential solution to multiple signs and symptoms of excessive cortisol. Notably, SPI-62 introduces a new mechanism of action for treating Cushing's, which is a significant advancement after decades of limited options. Phase 1 studies have already shown SPI-62's effectiveness in reducing intracellular cortisol and inhibiting HSD-1 in the liver, brain, and adipose tissue—critical areas linked to corticosteroid-related complications.
