1st Cushing’s patient dosed in ST-002 Phase 2 clinical trial
Study in Europe tests treatment's safety, tolerability, efficacy
The first participant has been dosed in a Phase 2 clinical trial testing ST-002 in people with Cushing’s syndrome and high blood sugar levels.
“ST-002 has the potential to change the treatment landscape for patients who currently have limited therapeutic options,” Manohar Katakam, PhD, CEO of treatment developer Sterotherapeutics, said in a company press release.
The study (2023-508322-10-00) is being conducted at multiple sites in Europe. Sterotherapeutics said several sites are active, and more are expected to open in the coming months.
Cushing’s syndrome refers broadly to disorders that are characterized by excess levels of cortisol, a hormone involved in many bodily functions, including metabolism, blood pressure, and inflammation.
One of the most common forms of Cushing’s syndrome is Cushing’s disease, in which a tumor growing in the brain’s pituitary gland secretes a signaling molecule that triggers cortisol production, ultimately leading to excess cortisol levels. Prolonged high cortisol levels can cause a variety of Cushing’s symptoms, including weight gain, skin problems, and mental health issues.
Blood sugar and Cushing’s
Cushing’s also can lead to diabetes (marked by high blood sugar levels) and metabolic dysfunction-associated steatotic liver disease (MASLD), a form of fatty liver disease marked by excess liver fat that typically occurs in combination with metabolic risk factors such as obesity or diabetes. Over time, MASLD can lead to liver inflammation, scarring, and in severe cases liver failure and liver cancer.
ST-002, also known as fluasterone, works by blocking the activity of cortisol and related hormones. It is also thought to have anti-inflammatory and anti-diabetic effects, according to the company.
The therapy received U.S. orphan drug designation to treat high blood sugar in people with Cushing’s syndrome. This status, meant to incentivize the development of treatments for rare diseases, provides tax credits, exemption from certain fees, and seven years of market exclusivity should the treatment ultimately be approved.
The Phase 2 trial, launched earlier this year, is evaluating the safety, tolerability, and efficacy of ST-002 against a placebo in adults aged 18-75 with Cushing’s syndrome and poorly controlled diabetes or high blood sugar levels.
Participants will be randomly assigned to receive buccal tablets (medication placed against the inside of the cheek to dissolve and enter the bloodstream) of either ST-002 or a placebo for 12 weeks (about three months), after which participants will receive the alternate treatment for 12 weeks.
Changes in blood sugar levels and other health markers will be assessed in both parts of the study.
“We remain focused on generating high-quality data that will inform the future development of ST-002 and provide hope for patients living with this challenging disorder and its associated complications, including diabetes and MASLD, the latter being assessed by magnetic resonance imaging and targeted measurements in our clinical study,” said Constantine Stratakis, MD, PhD, executive medical director of Sterotherapeutics.
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