Pyrazole Compounds and Uses Thereof Small Molecules Aimed at the JAK Signalling Pathway

This patent covers a family of pyrazole compounds that interfere with protein kinases, with claimed uses spanning cardiovascular disease, cancers, inflammatory conditions and eye diseases such as glaucoma. It comes from Alcon Inc., the Swiss-headquartered eye care company.

The Problem

Protein kinases are enzymes that attach phosphate groups to other proteins, and by doing so they act as switches in almost every biological process that matters: immunity, cell adhesion and migration, the growth of new blood vessels, programmed cell death, bone development and remodelling, the cell cycle, metabolism and gene transcription. Because they sit at so many control points, kinases that misbehave show up in a correspondingly wide range of diseases.

One group in particular, the Janus kinases or JAKs, comprises four members: JAK1, JAK2, JAK3 and TYK2. They work with STAT proteins in the JAK-STAT pathway, which is how cytokines, interferons and growth factors outside a cell reach in and change which genes that cell expresses. Mutations in the pathway’s components, or simply too much or too little of the cytokines that use it, are implicated in cancers and immune-related conditions. The difficulty for drug developers is selectivity. Kinases share similar structures, so a molecule that hits the intended one often hits several others, and in the eye there is a further constraint: a compound has to reach ocular tissue at a useful concentration without causing local irritation or systemic effects.

What This Invention Does

The application discloses pyrazole compounds according to several defined chemical formulas, along with specific named compounds, their pharmaceutically acceptable salts, and compositions containing them. These compounds affect the function of phosphotransferases such as protein kinases, and are presented both as therapeutic agents in their own right and as agents to be used alongside other treatments.

The claimed therapeutic scope follows the biology of the pathway rather than a single indication. It covers cardiovascular diseases, JAK-associated diseases including those characterised by abnormal growth such as cancers, inflammatory diseases, and eye diseases such as glaucoma. Given the applicant’s business, the ophthalmic uses are the ones to watch: glaucoma is driven by pressure inside the eye damaging the optic nerve, and inflammatory signalling contributes to a range of surface and posterior eye conditions where a kinase-directed small molecule could be delivered locally as a drop or an injection.

Key Features

• Defined pyrazole formulas. Compounds of Formula 1, Formula 2 and Formula 3 are claimed, along with specifically enumerated compounds.
• Pharmaceutically acceptable salts. Salt forms of the compounds are covered as well as the free molecules.
• Kinase and phosphotransferase activity. The compounds act on phosphotransferases such as protein kinases, including the JAK family.
• Compositions. Pharmaceutical compositions comprising the pyrazole compounds are claimed.
• Broad indication set. Uses span cardiovascular disease, JAK-associated diseases and cancers, inflammatory disease, and eye diseases including glaucoma.
• Standalone or adjunct use. The compounds are described both as therapeutic agents and for use with other therapeutic agents.

Who Is Behind It

The applicant is Alcon Inc., headquartered in Geneva, Switzerland, one of the largest eye care companies in the world across surgical equipment, intraocular lenses, contact lenses and ophthalmic pharmaceuticals. The named inventors are David Ellis, Heeren Gordhan, Cynthia Lichorowic, Jill Sturdivant and Mitchell DeLong. The application is a divisional of Australian application 2022361055, with priority from October 2021.

Why It Matters

Glaucoma affects roughly 300,000 Australians and is a leading cause of preventable blindness, with prevalence rising as the population ages. Current treatment relies heavily on drops that lower eye pressure, and adherence is a persistent problem, so new mechanisms of action attract sustained interest. More broadly, JAK inhibitors have already become important medicines in rheumatology and dermatology, which is why chemistry claiming this pathway is filed widely. As with any compound-class application, the patent establishes a commercial position rather than demonstrating clinical benefit: safety and efficacy for any specific use would need to be established through trials and assessed by the Therapeutic Goods Administration before a product could be supplied in Australia.

Related Concepts

• Protein kinase – the enzyme family these compounds act on.
• Janus kinase – the specific kinase group central to the claimed indications.
• JAK-STAT signalling pathway – the route by which external signals change gene expression.
• Glaucoma – the eye disease named among the target conditions.
• Pyrazole – the chemical scaffold the compounds are built on.
• Therapeutic Goods Administration – the Australian regulator any resulting medicine would face.

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AU 2026202032 was published in the Australian Official Journal of Patents on 2 April 2026 and is open for public inspection. Patent applications represent inventions that are sought to be protected and do not necessarily reflect commercially available products.