Heterocyclic Diamidines

Simple SummaryContent extracted from patent full text and abstract with AI.

This patent describes new pharmaceutical compositions containing heterocyclic diamidines, specifically targeting the transcription factor PU.1, as effective treatments for diseases characterized by fibrosis (excessive tissue scarring) and certain cancers. These compounds, such as DB1976 and DB1977, reduce collagen production and the activation of fibroblasts, which are central to both tissue fibrosis and tumor progression. The invention covers compositions for treating fibrotic diseases (like systemic sclerosis, pulmonary fibrosis, hepatic cirrhosis) and a range of tumors (including breast, pancreas, colon, and lung cancers), and can be used alone or in combination with existing cancer therapies.

Use CasesContent extracted from patent full text and abstract with AI.

• Treatment of systemic sclerosis (scleroderma) and other fibrotic skin diseases.
• Therapy for pulmonary fibrosis (lung scarring).
• Management of hepatic cirrhosis and other liver fibrosis conditions.
• Therapy for renal (kidney) fibrosis.
• Treatment of chronic graft-versus-host disease and Crohn's disease.
• Reduction of arthrofibrosis, myelofibrosis, and Dupuytren's disease.
• Cancer therapy, particularly for breast, endometrial, ovarian, lung, colon, and pancreatic tumors.
• Supportive therapy in combination with standard anti-cancer drugs or radiotherapy.
• Prevention or reduction of excessive scarring (e.g., keloids, hypertrophic scars).

BenefitsContent extracted from patent full text and abstract with AI.

• Targets a central molecular driver (PU.1) in fibrosis and tumor-associated fibroblasts, offering a new therapy where current options are limited.
• Demonstrated efficacy in multiple animal models and human fibroblast cultures with a strong reduction in pathological collagen deposition.
• Potential for disease modification in chronic, progressive fibrotic diseases, not just symptom management.
• Can be used for both non-malignant fibrotic diseases and malignant tumors, broadening therapeutic applications.
• May reduce tumor invasion and progression by modulating tumor-associated fibroblasts.
• Can be combined with existing anti-cancer drugs for potentially synergistic or additive effects.
• Several administration options (oral, injectable, topical, inhalation), enhancing patient flexibility and compliance.
• No major toxicity or apoptosis observed in preclinical studies, suggesting a favorable safety profile.

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Inventors & Applicants

Patent Abstract

The present invention relates to pharmaceutical compositions comprising heterocyclic diamidines and their use in the prophylaxis and/or treatment of diseases associated with fibrosis, in particular for the treatment of diseases such as systemic sclerosis (limited cutaneous scleroderma and diffuse cutaneous scleroderma), pulmonary fibrosis, hepatic cirrhosis, renal fibrosis, chronic graft-versus-host disease, Crohn's disease, arthrofibrosis, myelofibrosis, Dupuytren's disease and nephrogenic systemic fibrosis and tumours selected from mamma carcinoma, endometrial adenocarcinoma, ovarian serous tumor, lung adenocarcinoma, lung squamous cell tumor, colorectal tumor and pancreatic tumor.