01 / Vitamin A / retinol
Retinol enters tissue biology
Dietary vitamin A is transported and processed across tissue-specific pathways.
Pioneering first-in-class antagonists for retinoid and rexinoid nuclear receptor pathways
Kayothera specializes in inhibiting two historically difficult nuclear receptor pathways: retinoid and rexinoid signaling. Its oral small-molecule approach is designed to block formation of nuclear receptor ligands across cardiometabolic disease, obesity, cancer biology, and other unmet medical needs.
01First-in-class approach
02Oral small molecules
03Multi-program pipeline
04Peer-reviewed foundation
Mechanism
A new way to block disease-driving nuclear receptor signaling
Kayothera focuses on inhibiting the formation of retinoid and rexinoid ligands upstream, before nuclear receptor activation.
Active viewVitamin A / retinol
02 / Upstream ligand formation
ALDH enzymes generate ligands
ALDH1a enzymes participate in forming retinoid and rexinoid nuclear receptor ligands.
03 / Nuclear receptor signaling
Ligands activate RAR / RXR
Retinoic acid and rexinoid ligands activate nuclear receptor signaling and transcription.
04 / Multiple disease areas
Pathway activity shapes disease biology
Scientific evidence connects retinoid signaling with metabolism, immunity, cancer biology, and reproduction.
05 / Inhibition strategy
Kayothera targets the source
Kayothera is developing oral small molecules designed to inhibit ligand formation upstream.
01 / 05
Scientific inflection point
From pathway activation to pathway inhibition
For decades, retinoid biology has been studied largely through activation. Kayothera is focused on the other side of the pathway: inhibiting retinoid and rexinoid ligand formation upstream.
Historically difficult pathway
Two of the last remaining nuclear receptor pathways to be drugged with inhibitors.
Upstream ligand strategy
Target ligand formation before RAR or RXR nuclear receptor activation.
One biological foundation
A platform rationale spanning metabolism, cancer biology, and reproductive health.
Disease opportunities
Multiple opportunities from one biological foundation
01KAYO-1732
Diabetes / cardiometabolic disease
ALDH1a3 and beta-cell biology
Explore science →02KAYO-2000
Obesity
Rexinoid signaling, adipogenesis and energy expenditure
Explore science →03Platform science
Cancer biology
Retinoid signaling and immune / metabolic tumor biology
Explore science →04KAYO-1609
Reproductive health
ALDH1a2, retinoic acid synthesis and Sertoli cells
Explore science →Pipeline
Pipeline command center
Three programs connected by one upstream biological foundation.
Program selector03 active programs
Development pathwayPreclinical focus
Current development focus
KAYO-1732
Designed to inhibit retinoid synthesis in tissues relevant to type 2 diabetes and cardiometabolic disease, with a development focus on beta-cell function and metabolic health.
- Disease area
- Type 2 diabetes / cardiometabolic disease
- Supporting science
- ALDH1a3 biology, pancreatic beta-cell function, adipose tissue and vasculature.
- Next milestone
- Preclinical development priorities
Development pathwayPreclinical focus
Current development focus
KAYO-2000
Designed to target rexinoid pathway biology tied to thyroid function, mitochondrial function, adipogenesis, and energy expenditure.
- Disease area
- Obesity
- Supporting science
- Rexinoid signaling, thyroid and mitochondrial function, and energy expenditure.
- Next milestone
- Lead program development priorities
Development pathwayPreclinical focus
Current development focus
KAYO-1609
Designed to inhibit ALDH1a2-mediated retinoic acid synthesis in reproductive biology, with relevance to spermatogenesis and male contraceptive development.
- Disease area
- Male contraceptive / reproductive health
- Supporting science
- ALDH1a2, retinoic acid synthesis, Sertoli cells, and spermatogenesis.
- Next milestone
- Future development priority
| Program | Disease area | Pathway relevance |
|---|---|---|
| KAYO-1732 | Type 2 diabetes / cardiometabolic disease | Retinoid synthesis / ALDH1a3-related biology |
| KAYO-2000 | Obesity | Rexinoid pathway / ALDH1a1-related biology |
| KAYO-1609 | Male contraceptive / reproductive health | ALDH1a2 / retinoic acid synthesis / Sertoli cells |
Progress + roadmap
Progress mapped to value creation
Completed milestones build the scientific and organizational foundation for disciplined future development.
Completed progressForward roadmap
Advisory
Clinical Advisory Board expansion
Key opinion leaders in beta-cell health and type 2 diabetes joined the Clinical Advisory Board.
Science
Anti-tumor immunity research
New research and news advanced understanding of retinoic acid signaling in cancer immunity.
Publication
Foundational iScience research
Research on retinoid nuclear receptor pathway antagonists through targeting ALDH1A3 was published.
Pipeline
KAYO-1732 nominated
KAYO-1732 was selected as a development candidate for type 2 diabetes and cardiometabolic indications.
Forward
roadmap
roadmap
Development priority
Lead program selection
Advance program-specific small molecules and development priorities.
Development priority
Preclinical development
Evaluate selected candidates in development-enabling studies.
Development priority
IND-enabling preparation
Prepare development packages subject to study outcomes and strategy.
Forward-looking development priorities are subject to study outcomes, regulatory feedback, funding, and company strategy.
View forward roadmap →Investor perspective
The investment thesis
First-in-class biology
Targeting retinoid and rexinoid ligand formation upstream.
Oral small-molecule platform
A modality designed for development across major disease areas.
Multiple disease opportunities
Programs in cardiometabolic disease, obesity, and reproductive health.
Clear milestone path
A roadmap grounded in scientific evidence and program development.
JEMJournal of Experimental Medicine
Peer reviewed2024 / Review
Featured peer-reviewed foundation
The pathogenic role of retinoid nuclear receptor signaling in cancer and metabolic syndromes
A major review connecting retinoid nuclear receptor signaling to cancer biology, metabolic dysfunction, diabetes, obesity, and emerging therapeutic inhibition strategies.
Read the publicationCompany proof
A growing foundation of science, leadership, and recognition
Publications
Peer-reviewed work in JEM, iScience, Nature Communications, and other journals.
Advisory expertise
Scientific and clinical advisors spanning beta-cell health, diabetes, and translational development.
Recognition
American Diabetes Association Innovation Challenge recognition.
Funding ecosystem
Backed by investors and grant funding supporting company development.
Latest
Company news
Advisory
Kayothera Announces Expansion of Clinical Advisory Board
Key opinion leaders in beta-cell health and type 2 diabetes added to the Clinical Advisory Board.
Read more →Science
A nuclear target to revive anti-tumor immunity
Research advances understanding of how all-trans retinoic acid compromises anti-cancer immunity.
Read more →Publication
Development of retinoid nuclear receptor pathway antagonists through targeting ALDH1A3
Foundational iScience research on drugging the retinoid nuclear receptor pathway.
Read more →Connect with Kayothera