AI-empowered Pipeline-in-a-drug Option: Insilico Medicine Nominates ISM9077, Potential First-in-class Target Y Inhibitor, as Preclinical Candidate (PCC) for Ocular Diseases, Inflammatory Disorders and Aging

• As a dual-purpose, potential first-in-class, "pipeline-in-a-drug" candidate, ISM9077 provides an innovative solution for ocular diseases, inflammatory disorders, and the fundamental aging process.
• ISM9077 was designed, evaluated and optimized utilizing Chemistry42's integrated generative AI models, marking Insilico's 32nd PCC nominated since 2021.
• In preclinical studies, ISM9077 outperformed current therapies in dry AMD, uveitis and dry eye models, while demonstrating a favorable safety profile represented by wide safety margin.
• Aside from the promising efficacy results, the candidate, with a novel target and a novel molecule structure driven by AI, also exhibits good permeability, excellent retinal tissue exposure, supporting novel delivery method potential (both oral and eye drop)

CAMBRIDGE, Mass. , Aug. 5, 2026 /PRNewswire/ -- Insilico Medicine ("Insilico"; HKEX: 3696), a clinical-stage generative artificial intelligence (AI)-driven drug discovery company, today announced the nomination of ISM9077, the AI-empowered, potential first-in-class Target Y inhibitor with pipeline-in-a-drug potential, as a Preclinical Candidate (PCC) for the treatment of ocular diseases including dry Age-Related Macular Degeneration (dry AMD), uveitis, and dry eye disease. As the 32nd PCC nominated since 2021, ISM9077 boasts potent inhibition across species, excellent exposure and permeability traits, and a favorable safety profile in preclinical studies.

Also, the novel mechanism modulates pathological inflammation, supporting broad indication expansion into wide-ranging therapeutic areas, represented by aging, the complex biological progress associated with chronic inflammation, and other inflammation-related disorders including neurodegenerative diseases, Inflammatory Bowel Disease (IBD), cardiovascular conditions, Metabolic Dysfunction-Associated Steatohepatitis (MASH), and obesity.

"ISM9077 marks our 32nd preclinical candidate nominated since 2021, so this is an example of how generative AI delivers at scale," said Alex Zhavoronkov, PhD, Founder, CEO and CBO of Insilico Medicine. "This candidate features a moderately novel target, a novel molecule designed from scratch, and a novel formulation possibility of eye drop. Beyond offering a non-invasive, highly effective option for millions of patients suffering from blinding ocular diseases, ISM9077 further enhances Insilico's dual-purpose strategy to treat specific diseases while targeting aging and extending healthspan, as Target Y is strongly implicated in aging, longevity, and multiple age-related conditions."

According to recent research, the global market of ocular diseases exceeded USD 40 billion in 2025, and is projected to grow to USD 71.58 billion by 2034. Among the multiple conditions related to inflammation, uveitis alone accounts for more than 10% of severe visual handicaps in the United States. Given that options of effective treatments are still limited, the field has been working on innovative solutions, and venturing into novel drug delivery methods for better bioavailability.

To fulfil the unmet needs, Insilico R&D set out to discover an innovative inhibitor, guided by the structure-based drug design (SBDD) approach with Chemistry42, Insilico's generative chemistry platform. With binding pocket understanding and structural foundation established through in-house co-crystal structures, the team utilized the integrated generative models within Chemistry42 for de novo design, optimizing pharmacophore fit, drug-likeness, three-dimensional shape, and structural novelty.

An AI-driven target-specific model was subsequently developed to predict molecular activity, enabling rapid screening and prioritization of generated compounds. Rounds of evaluation and optimization yielded ISM9077, a novel, AI-empowered structure with low patent risk, targeting a unique mechanism of action supported by crystallographic validation.

"Ocular diseases treatment calls for novel options, as current solutions haven't reached a balance between toxicity management, efficacy results, and dosing adherence," said Feng Ren, PhD, Co-CEO and CSO of Insilico Medicine. "ISM9077 overcomes these limitations through AI-driven structural design, achieving superior target exposure and potential for eye drop formulation. Dry AMD is a leading cause of irreversible central vision loss in older adults, and the positive efficacy results in therapeutic studies, as well as Cynomolgus monkey models, first validated the therapeutic value of Target Y for dry AMD, in a way much closer to human mechanism. We look forward to future clinical translation based on such AI-driven outcomes."

In preclinical studies, ISM9077 demonstrated robust in vivo efficacy across ocular disease models, after both oral administration and topical eye drop delivery. Moreover, the compound exhibits favorable oral bioavailability, low-to-moderate in vivo clearance, a retinal exposure 2 to 5.5 times of plasma levels, good permeability supporting future development as eye drop, and a favorable safety profile marked with a wide safety margin.

In dry AMD models, ISM9077 significantly improved retinal structural integrity and visual function. On key evaluation endpoints, ISM9077 demonstrated efficacy approximately three times of currently available therapy, while simultaneously demonstrating superior histopathological improvement.

Across independent uveitis models, ISM9077 reduced ocular inflammation, suppressed the release of inflammatory cytokines, and restored retinal function. In dry eye models, ISM9077 improved tear production, and suppressed corneal inflammation with a fast onset, outperforming Cyclosporine A (CsA), the current standard-of-care. These findings validate Target Y's potential as a key therapeutic target addressing a broad range of inflammatory and degenerative ocular diseases.

As an AI-native biotechnology company, Insilico is redefining the efficiency of preclinical drug development through its advanced AI and automation platform, setting a new standard for the industry. While traditional early-stage drug discovery typically takes 2.5 to 4 years, Insilico has consistently reached preclinical candidate (PCC) nomination in an average of just 12 to 18 months, with only 60 to 200 molecules synthesized and tested per program. Since 2021, the company has nominated 32 PCCs, 13 of which have received IND approval or clearance.

While expanding the practical applications of its technology in drug discovery and life science research, Insilico is also continuously enhancing the performance of its AI platform. Drawing on extensive experience and datasets from its training platform, the company has distilled thousands of benchmarks and integrated them into MMAI Gym. Serving as both a "trainer and benchmark" for scientific AI, MMAI Gym enables organizations to train models for domain-specific reasoning while rigorously evaluating their performance on real-world tasks, advancing the path toward pharma superintelligence. To date, Human Longevity and Liquid AI have collaborated with Insilico, joining as partners of MMAI Gym.

About Insilico Medicine

Insilico Medicine is a pioneering global biotechnology company dedicated to integrating artificial intelligence and automation technologies to accelerate drug discovery, drive innovation in the life sciences, and extend health longevity to people on the planet. The company was listed on the Main Board of the Hong Kong Stock Exchange on December 30, 2025, HKEX:3696.

By integrating AI and automation technologies and deep in-house drug discovery capabilities, Insilico is delivering innovative drug solutions for unmet needs including fibrosis, oncology, immunology, pain, and obesity and metabolic disorders. Additionally, Insilico extends the reach of Pharma.AI across diverse industries, such as advanced materials, agriculture, nutritional products and veterinary medicine. For more information, please visit www.insilico.com.

SOURCE Insilico Medicine