Insilico announces ISM6200 AI-designed drug candidate for ovarian cancer and cortisol disorders

A new artificial intelligence-powered drug candidate targeting multiple serious conditions has been announced by Insilico Medicine.

The company reported the nomination of ISM6200, a potentially best-in-class preclinical candidate designed to target NR3C1, a key receptor involved in cortisol regulation. The drug is being developed for ovarian cancer, hypercortisolism including Cushing’s syndrome and other disorders linked to excess cortisol such as obesity.

The discovery process was driven by Insilico’s generative chemistry engine Chemistry42, part of its broader Pharma.AI platform, which supports molecule design and optimisation.

Addressing urgent unmet needs

“In platinum-resistant ovarian cancer, patients face an overall survival of only about a year, so the field is calling for new therapies,” said Dr Feng Ren, Co-CEO and Chief Scientific Officer of Insilico Medicine. “ISM6200 was specifically engineered with outstanding properties, including lower DDI risks, which allow broader combination options. More importantly, the molecule can deliver strong efficacy at a much lower projected human dose, offering a safer and more effective treatment path for patients in need.”

In platinum-resistant ovarian cancer, patients face an overall survival of only about a year, so the field is calling for new therapies

Preclinical studies indicate that ISM6200 demonstrates low drug-drug interaction risk and strong efficacy across several animal models. In one study using a cell line-derived xenograft model, the drug showed a significant and dose-dependent increase in anti-tumour activity when combined with paclitaxel.

Researchers also reported favourable absorption, distribution, metabolism and excretion characteristics, alongside a pharmacokinetic profile that supports strong systemic exposure and a low projected effective dose in humans.

AI unlocks complex drug targets

The NR3C1 target was initially identified through dual target research related to ageing, with links to multiple age-associated diseases. Insilico then developed a portfolio of small molecules aimed at using the target’s therapeutic potential while avoiding known risks.

“The nomination of ISM6200 highlights the transformative power of generative AI in addressing complex medicinal chemistry challenges while reducing traditional risks such as metabolic instability and off-target toxicity,” said Alex Zhavoronkov, Founder and Co-CEO of Insilico Medicine. “I am especially encouraged by multi-purpose candidates like this one, as it is indicated in systems across the body, even exceeding our expectation of targeting both ageing and certain diseases. By using AI, we can identify high-quality molecules with broad potential, in record time, and we are advancing more pipelines with AI-driven efficiency.”

Promising results across multiple conditions

Beyond cancer, ISM6200 has shown encouraging results in models of metabolic disease. In a diet-induced obesity mouse model, the drug achieved significant weight loss, primarily by reducing fat while preserving muscle mass. After 28 days of treatment, animals receiving ISM6200 alone recorded an average weight reduction of 8.8 percent, while those treated in combination with semaglutide saw weight loss reach 23.8 percent.

Beyond cancer, ISM6200 has shown encouraging results in models of metabolic disease

Further studies demonstrated benefits in other cortisol-related conditions. In a rat model of Cushing’s syndrome, ISM6200 reduced insulin resistance by 68 percent and lowered blood pressure towards normal levels after six days of treatment. In a glaucoma model, the drug also reduced intraocular pressure following short-term administration.

Expanding a growing pipeline

The nomination of ISM6200 represents the 29th preclinical or developmental candidate identified by Insilico Medicine. Since 2021, 12 of the company’s candidates have received investigational new drug clearance, with three progressing to Phase II trials, including one completed Phase IIa study.

The development also reinforces the company’s cardiometabolic portfolio, first introduced at BIO-Europe 2025, where the NR3C1 programme was highlighted for its potential to address cortisol-driven metabolic dysfunction and enhance weight loss therapies.

As AI continues to change drug discovery, ISM6200 gives researchers a look into how advanced computational tools may accelerate the development of treatments for complex and multifaceted diseases