F4 Pharma GmbH i.G. is developing FX06 in diseases associated with systemic inflammation and capillary leak, thereby concentrating on orphan diseases with a high unmet medical need. The top priority development projects are Covid-19, Clarkson Disease, ECMO, and vasoplegic syndrome.
In many disease states, blood vessels become leaky.
A: normal endothelium
B: leaky endothelium
- Target Indication: Moderate to Severe Covid-19
In up to 15% of cases, predominantly in elderly patients with co-morbidities, infection with SARS-CoV-2 leads to a severe disease course with ground glass lung opacity due to alveolar infiltration and reduced blood oxygenation, which require intensive care, external oxygen supply and in many case mechanical ventilation and/or ECMO. Patients requiring mechanical ventilation have a case fatality rate of up to 80%. No approved treatment is available, which treats or prevents the ARDS like syndrome.
- Target Indication: Clarkson Syndrome (Systemic Capillary Leak Syndrome)
Clarkson disease or systemic capillary leak syndrome is an ultra-orphan disease affecting around 200 patients worldwide. It is characterized by episodic attacks of systemic capillary leak, massive hypotension and edema, cardiac and renal failure, stroke and compartment syndrome. Currently there is no treatment available for acute attacks and mortality is between 35 and 70 % within 10 years.
- Target Indication: Extracorporal Membrane Oxygenation ECMO
Extracorporal membrane oxygenation is used as supportive temporarily treatment in patients with cardiac and/or pulmonary failure. Vascular leak is a side effect of this life-saving therapy, treated mainly symptomatically with fluid resuscitation, corticosteroids and catecholamines. There are around 8.000 treatments/year in Europe and USA – nearly half of them children and neonates. Mortality can go up to 70 %.
- Target Indication: Vasoplegic Syndrome
The vasoplegic syndrome is a life-threatening condition which might develop after patients have been separated from cardiopulmonary bypass. It is associated with a high mortality. Vasoplegic syndrome is characterized by a catecholamine-resistant hypotension and treatment options are very limited. It is more common in older and multi-morbid patients and patients at risk can be identified during surgery according to their catecholamine need.