H4 Orphan Pharma is developing an innovative therapeutic approach to treat fibrosis diseases. Two drug candidates, issue from a class of molecules heretofore unexploited in fibrosis management, are currently in preclinical development:
- 554-001 for idiopathic pulmonary fibrosis
- 554-002 for dry eye in Sjögren syndrome
H4 Orphan Pharma is dedicated to the research and development of drugs against fibrosis with a new therapeutic approach.
The term "fibrosis" describes the fibrous transformation of tissue due to the excessive development of collagen, which, in time, blocks the circulation of water within cells. This phenomenon progresses from cell to cell and within them sets off a process called apoptosis, or programmed cell death. As the cells die, the affected organ (lung, liver, kidney, secretory glands of the eye, etc.) degenerates and can no longer fulfill its function.
Pulmonary fibrosis occurs in several diseases:
- Idiopathic pulmonary fibrosis
- Sjögren syndrome
- Systemic sclerosis
- Severe COPD
- Severe asthma
The excess of collagen causes accelerated apoptosis. The death of the cells ultimately alters the function of the affected organ, which may be the lung, liver, kidney, heart or secretory glands of the eye, among many others.
554-001for idiopathic pulmonary fibrosis
Idiopathic pulmonary fibrosis (IPF) affects 200,000 people in Europe and in the United States1. The disease is very aggressive, causing the death of 50% of those diagnosed with it in two to five years2. Currently, there is no curative treatment available for IPF.
554-002 for dry eye in Sjögren syndrome
- Dry eye syndrome affects more than 300 million people worldwide3. It occurs when lacrimal fluid is insufficient in quality or quantity, and increases the risk of eye infections or inflammation. Corneal ulcers may occur in the most serious cases.
- Sjögren syndrome is a rare disease affecting 2.5 million people worldwide4. Its symptoms are due to lymphocytic infiltration of certain glands and the resulting reduction of their secretions. The most-frequently affected glands are the salivary glands in the mouth and the lacrimal glands in the eyes, resulting in the hallmark dry mouth and dry eye symptoms of Sjögren syndrome. Other organs, such as the lungs, kidneys, joints or small blood vessels may also show infiltration and inflammation due to the disease.
H4 Orphan Pharma deploys an innovative therapeutic approach in its committed to the research and development of novel treatments for fibrosis.
1 - Epidemiology of idiopathic pulmonary fibrosis: a population-based study in primary care. Harari S, Davì M, Biffi, A, Caminati A, Ghirardini A, Lovato V, Cricelli C, Lapi F. Internal and emergency Medicine. Springr. 2019 Sep 2019
2 - Raghu G., Collard HR, et al. An Official ATS/ERS/JRS/ALAT Statement: Idiopathic Pulmonary Fibrosis: Evidence-based Guidelines for Diagnosis and Management, AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE VOL 183 2011
3 - Costabel U. Eur Respir Rev 2012;21:140
4 - Société nationale française de médecine interne [French Society for Internal Medicine]: http://www.snfmi.org/content/gougerot-sjogren-syndrome-de