Sparrow Pharmaceuticals Announces First Patient Rolled-Over to Open-Label Extension of RESCUE, a Phase 2 Clinical Trial of SPI-62 for ACTH-Dependent Cushing’s Syndrome

RESCUE trial continues to enroll eligible patients; data expected in 2024

PORTLAND, Ore.--(BUSINESS WIRE)--Sparrow Pharmaceuticals, an emerging, clinical-stage biopharmaceutical company developing novel, targeted therapies to address unmet needs in both rheumatology and endocrinology, today announced the first patient has elected to enroll in the open-label extension (OLE) phase of the RESCUE trial, a phase 2 clinical trial of SPI-62, a potent and selective HSD-1 inhibitor, for the treatment of ACTH-dependent Cushing’s syndrome. The long-term OLE will be open to patients who complete the initial RESCUE cross-over treatment protocol and then elect to continue to participate in the research.

“Cushing’s syndrome can be life-threatening and the preferred first-line surgical treatment often fails. While medical therapies are increasingly used, there are many drawbacks and innovative medical approaches have lagged,” said Frank Czerwiec, MD, PhD, Chief Medical Officer of Sparrow. “We’re excited to graduate patients from the initial RESCUE study to a new phase of investigation of SPI-62, one which will allow us to deepen our understanding of the potential for long-term safety and efficacy of this completely novel mode of treatment for Cushing’s.”

RESCUE is evaluating the pharmacologic effect, efficacy, and safety of SPI-62 in patients with adrenocorticotropic hormone (ACTH)-dependent Cushing’s syndrome. Evidence suggests that by inhibiting HSD-1, SPI-62 can lower intracellular levels of cortisol in key organs, thereby potentially mitigating the adverse effects of hypercortisolism. SPI-62 could represent the first new mechanism of action to treat Cushing’s syndrome in decades. The OLE will seek to gather new long-term safety and tolerability data, as well as allow continued monitoring of SPI-62’s effects on signs and symptoms of Cushing’s syndrome.

Sparrow is actively enrolling the RESCUE trial at approximately 16 sites in the U.S., Bulgaria, and Romania. Criteria for participation include male and female subjects, ages 18 years or older, with active and consistent cortisol excess and documented diagnosis of ACTH-dependent Cushing’s syndrome.

To learn more about the RESCUE trial (NCT05307328), please visit www.sparrowtrials.com.

About Cushing’s Syndrome and SPI-62

Patients develop Cushing’s syndrome due to an excess of glucocorticoids. In Cushing’s disease, this is due to a pituitary tumor that leads to high levels of adrenocorticotropic hormone, which stimulates excess cortisol secretion. ACTH, or its releasing hormone CRH, may also be produced by non-pituitary tumors. By targeting the intracellular activation of cortisol in key organs, SPI-62 can potentially mitigate multiple signs and symptoms of cortisol excess. HSD-1 inhibition would be the first new mechanism of action in decades to treat Cushing’s. In phase 1 studies, SPI-62 demonstrated the ability to reduce intracellular cortisol in the liver and to inhibit HSD-1 in both the brain and adipose tissue, which are three key tissues in which corticosteroid toxicity leads to morbidity.

About Sparrow Pharmaceuticals

Sparrow Pharmaceuticals was founded to spare patients the ravages of steroids. Leveraging underappreciated scientific insights into glucocorticoid biology, the company is working to provide better treatment options for serious disorders of hypercortisolism, and to revolutionize the treatment of autoimmune and inflammatory conditions. Its lead product, SPI-62, is an oral, small molecule, novel therapeutic treatment designed to target the source of active intracellular glucocorticoids in key tissues.