Areas of Focus
At Enzyvant, we apply ourselves to the most formidable challenges, focusing on problems that have proven difficult to solve. We aim to develop life-altering medicines for rare diseases where either limited options exist or current options fail to address the underlying disease. After delivering a first-of-its-kind advancement to improve the lives of pediatric patients with congenital athymia, we are advancing promising therapies for rare respiratory diseases including pulmonary arterial hypertension (PAH), bronchiolitis obliterans syndrome (BOS) and chemical lung injury.
Pediatric Congenital Athymia
Pediatric congenital athymia is an ultra-rare condition in which children are born without a thymus, which may cause profound immunodeficiency, vulnerability to potentially fatal infections and life-threatening immune dysregulation. Pediatric congenital athymia affects 17 to 24 infants born each year in the United States. In a natural history study, congenital athymia patients on supportive care alone did not survive beyond 2-3 years of age.
Pediatric congenital athymia is initially detected by T-cell deficiency observed in newborn screening for SCID (severe combined immune deficiency), which is now required in all 50 U.S. states. The “T” in T cell stands for thymus because it is where T cells are selected to fight infections or are destroyed if they have the potential to attack the body instead of invaders. SCID and congenital athymia are both primary immunodeficiency disorders, but they are distinct conditions.
Learn more about our scientific approach in pediatric congenital athymia here.
Learn more about our medicines in pediatric congenital athymia here.
Pulmonary Arterial Hypertension
Pulmonary arterial hypertension (PAH) is a rare disease characterized by a thickening and constriction of the smooth muscle layer of pulmonary arteries that is in part driven by excess serotonin signaling. This causes remodeling of pulmonary arteries, which leads to an increase in blood pressure and workload for the heart over time. Over 50,000 people have been diagnosed with PAH (WHO Group 1) in the United States, and this disease is almost twice as common in women than men.
PAH is a rapidly progressive disease carrying an approximate 50 percent mortality rate at five years, with those affected typically succumbing to right ventricular heart failure. The condition is currently treated with vasodilators, which offer some benefit but fail to address the underlying vessel remodeling believed to be responsible for the disease’s high morbidity and mortality rates. Reducing peripheral serotonin production is a novel approach for the treatment of PAH and has the potential to halt or reverse pulmonary remodeling. We are developing our peripherally acting tryptophan hydroxylase (TPH) inhibitor as an adjunctive treatment for PAH as our research suggests its mechanism of action is additive to the effects of standard of care.
Learn more about our scientific approach in PAH here
Learn more about our treatment in development for PAH here
Bronchiolitis Obliterans Syndrome
Bronchiolitis obliterans syndrome (BOS) is a life-threatening form of chronic lung allograft dysfunction (CLAD) that can occur following lung transplantation. It results from the immune system attacking the small airways in the lungs, leading to scarring and damage that impacts lung function and is characterized by increased interleukin-1 (IL-1) production. BOS can be found in more than 50 percent of lung transplant recipients who are alive at five years.
BOS is associated with poor long-term survival and is the leading cause of death in lung transplant recipients who survive beyond one year. It is irreversible with poor responses reported for the limited treatment options available. Current therapies may reduce the decline in pulmonary function but are not expected to halt or reverse the progression of BOS.
Learn more about our scientific approach in BOS here
Learn more about our treatment in development for BOS here
Chemical Lung Injury
Chemical lung injuries can result from the inhalation of noxious or irritant chemical gases (e.g., sulfur mustard, chlorine, ammonia or components of vaping products) as well as other toxicants such as smoke and particulates. These injuries may be a result of intentional or accidental exposure and may cause lifelong disability or possibly death.
Incidence figures are difficult to calculate owing to the diversity of compounds that may cause lung injury and the settings in which such injury may occur, such as accidental exposure in industrial or home settings or via chemical attack. It has been estimated by the National Occupational Exposure Survey, conducted by the CDC, that at least one million U.S. workers are at risk of exposure to respiratory irritants, with injuries occurring more frequently at home.
Learn more about our scientific approach in chemical lung injury here
Learn more about our treatment in development for chemical lung injury here